In this episode of Denatured, you'll hear from Mark Lowdell, CSO & co-founder at INmuneBio Inc. and Vishwas Seshadri, CEO & director at Abeona Therapeutics. We examine how recessive dystrophic epidermolysis bullosa serves as a model for cell and gene therapy, and what this ultra-rare disease teaches us about scaling advanced treatments for the future. Host Jennifer C. Smith-Parker, Director of Insights, BioSpace Guests Mark Lowdell, CSO & Co-Founder, INmuneBio Inc Vishwas Seshadri, CEO & Director, Abeona Therapeutics Disclaimer: The views expressed in this discussion by guests are their own and do not represent those of their organizations.

Moderna’s mRNA-based flu vaccine made headlines in February when the FDA declined to even review the application. Days later, the agency reversed course, setting a decision date for August and later scheduling an advisory committee meeting for June 18. In documents released ahead of that meeting, the FDA and Moderna seem to have reached alignment, though the agency did flag certain data gaps for advisors to review.Biotech IPOs are off the charts—literally. This past month has seen not one but two record-setting public debuts. First there was Kailera Therapeutics, which hit the market with $625 million in April, outpacing Moderna’s 2018 IPO of $600 million. And now we have Parabilis Medicines, which last week dethroned Kailera as the largest biotech IPO of all time, with $670 million.Meanwhile, the number of employees laid off spiked by almost 50% year-over-year in May, though that jump is mostly due to significant cuts at Takeda and BioNTech.Genentech also made a big change last week.Eli Lilly continued its dealmaking spree with AlzeCure, striking a licensing agreement worth up to $1 billion centering on a small-molecule asset for Alzheimer’s disease. But the bigger Lilly news in the past week was clinical results that show promise for the pharma’s $2.3 billion acquisition of Ajax Therapeutics in April.Earlier this month, the FDA held a public session to glean feedback regarding the Commissioner’s National Priority Voucher program, in which several groups called for a temporary pause to the pilot, citing concerns about transparency and political involvement. While the future of that program hangs in the balance, two voucher holders got updates this week. First, Sanofi’s diabetes drug Tzield was greenlit for older kids and teenagers, though it’s unclear whether a voucher was associated with the approval. Meanwhile, Disc Medicine’s rejected rare disease drug bitopertin appears to be back on track, with the biotech announcing last week that the FDA will allow its current Phase 3 trial to support another regulatory filing.

In this episode of Denatured, as part of our series on the European life sciences investment ecosystem, you'll hear from Hannah Franklin, associate at Biovance Capital and Pablo Gabriel Cironi Lopez, director of life science investment at Caixa Capital Risc. We examine the rise of Southern Europe’s biotech ecosystem—from Portugal’s efforts to turn strong science into startups to Spain’s growing depth of capital and talent—and what it will take for the region to compete globally.Host⁠Jennifer C. Smith-Parker, Director of Insights, BioSpaceGuestsHannah Franklin, Associate, Biovance CapitalPablo Gabriel Cironi Lopez, Director, Life Science Investment, Caixa Capital RiscDisclaimer: The views expressed in this discussion by guests are their own and do not represent those of their organizations.

Everything came up roses for Eli Lilly at the American Diabetes Association, as the pharma boasted positive results for its new pill Foundayo and next-gen asset retatrutide—not just for weight loss but also for other indications.Obesity rival Novo Nordisk, meanwhile, held a dinner, during which executives may have convinced analysts that the company is turning a corner after a rough couple of years.Also check out ADA updates from Roche and partner Zealand Pharma, Boehringer Ingleheim, Pfizer (touting results from its Metsera buy), AstraZeneca, Kailera Therapeutics and more.Outside of ADA, the biggest news of the past week came on Tuesday with GSK striking the biggest traditional pharma/biotech M&A of the year so far with its $10.6 billion acquisition of oncology focused Nuvalent Bio. And on Monday, J&J notched a smaller deal, buying out Firefly Bio for $1B. These deals add to continued uptick in M&A seen in biopharma this year, with much of that momentum being driven by Eli Lilly and its GLP-1 cash.At an FDA listening meeting last week on the Commissioner’s National Priority Voucher program, the agency heard multiple calls to pause it. Confusion also still persists around all of the new rare disease pathways, including the new plausible mechanism framework.

In this episode of Denatured, you'll be hearing from Daniel Gil, CEO of Pelage Pharmaceuticals and Francisco Ramírez-Valle, senior vice president of immunology discovery at Eli Lilly. We dive into the long-overlooked hair loss space, exploring why true innovation has lagged, how a regenerative approach aims to reactivate dormant follicles and what early proof-of-concept means for patients.HostJennifer C. Smith-Parker, Director of Insights, BioSpaceGuestsDaniel Gil, CEO, Pelage PharmaceuticalsFrancisco Ramírez-Valle, Senior Vice President, Immunology Discovery, Eli LillyDisclaimer: The views expressed in this discussion by guests are their own and do not represent those of their organizations.

At the American Society of Clinical Oncology (ASCO) annual meeting in Chicago this past weekend, packed plenary sessions from Revolution Medicines and Summit Therapeutics’ Chinese partner Akeso stole the show. For RevMed, analysts anticipate a potential approval in pancreatic cancer as early as this year, while Summit still has a tough road ahead showing that the survival benefit seen in Akeso’s clinical trial in China will hold up in a global population.Immuneering, BMS/BioNTech, Merck, Pfizer and many more also scored oncology wins at the annual meeting, as did Moderna, with “encouraging” 5-year survival for its mRNA-based personalized melanoma vaccine. Outside of ASCO, the past week saw Pfizer strike an unusual pact with China’s Innovent Biologics as it seeks to bolster its oncology pipeline. Reminiscent of another recent deal from BMS and Hengrui Pharma, it could signal a more collaborative approach to working with Chinese companies. We’ll also cover the latest deals from Eli Lilly, which continues to rack up partners with its GLP-1 windfall. Finally, learn about how BrainStorm is planning another FDA bid for its experimental ALS therapy NurOwn with former regulator Peter Pitts now on the board, and check out a preview of the 2026 American Diabetes Association, or ADA, which kicks off this weekend in New Orleans.

In this episode of Denatured, as part of our series on the European life sciences investment ecosystem, you'll be hearing from Ksenija Pavletic, partner and chief commercial officer at Jeito Capital and Thierry Laugel, managing partner at Kurma Partners. We dive into France’s biotech ecosystem and what still needs to happen for more early innovation to translate into investable, scalable biotech.Host⁠Jennifer C. Smith-Parker, Director of Insights, BioSpaceGuestsKsenija Pavletic, Partner and Chief Commercial Officer, Jeito CapitalThierry Laugel, Managing Partner, Kurma PartnersDisclaimer: The views expressed in this discussion by guests are their own and do not represent those of their organizations.

Eli Lilly has been as acquisitive as ever, striking five new deals in the past week alone. On Tuesday, the company dove into the deep end of vaccine development with three separate buys in the space. Outside of vaccines, Lilly scooped up preclinical biotech Engage Bio for $202 million and teamed up with AI company Collaborative Drug Discovery for its life sciences data management solutions.These deals and Lilly’s skyrocketing revenue pushed the Indianapolis-based company to the top of IDEA Pharma’s list of best inventors and innovators.Lilly also continues to make headlines for its impressive data readouts, especially in the weight-loss space that it now reigns supreme. Last week, Lilly announced that next-gen asset retatrutide led to weight loss of 70 pounds, or 28.3% at 80 weeks, setting a new benchmark for the space.Moderna also made news this week with a surprising uptick in its share price that analysts are attributing to the so-called “fear trade” that is rising amid reports of a cluster of cases of hantavirus. Moderna also announced that its mRNA flu shot—initially turned away from the FDA but later accepted for review—will be discussed at an advisory committee on June 18.The annual conference of the American Society of Clinical Oncology starts on Friday in Chicago, where all eyes will be on Revolution Medicines’ investigational pancreatic cancer pill and Akeso’s Phase 3 trial HARMONi-6 for its Summit Therapeutics-partnered PD-1/VEGF bispecific ivonescimab.Finally, sign up for BioPharma Executive to receive this week’s special edition breaking down executive compensation packages across the biggest pharmas.

In this episode of Denatured, as part of our series of the European life science investment ecosystem, you'll be hearing from Regina Hodits, managing director at Angelini Ventures and Sofia Ioannidou, VC partner at Andera Life Sciences. They examine Germany’s biotech and life sciences landscape, focusing on the scientific, infrastructure and policy shifts required to enable European companies to scale globally while remaining anchored in Europe.Host⁠Jennifer C. Smith-Parker, Director of Insights, BioSpaceGuestsRegina Hodits, Managing Director, Angelini VenturesSofia Ioannidou, VC Partner, Andera Life SciencesDisclaimer: The views expressed in this discussion by guests are their own and do not represent those of their organizations.

FDA Commissioner Marty Makary officially resigned last week following reports of his ouster. Then, the acting directors for the agency’s two main review units also left their posts, as did the FDA chief of staff and chief AI officer. Domino effect aside, the reaction from the industry has been mostly positive, given Makary’s tumultuous reign. But he might be hard to replace. If it were up to the biotech industry, former longtime oncology regulator and short-lived CDER director Richard Pazdur would take the role. For now, FDA Deputy Commissioner for Food Kyle Diamantas is in charge.Eli Lilly’s David Ricks was the highest paid pharma CEO last year, but J&J’s Joaquín Duato made the most relative to rank-and-file employees, with a median pay ratio of 358 to one. He was on the top of BioSpace’s list last year, too, with a ratio of 293 to 1. Last month, Revolution Medicines’ RAS inhibitor doubled survival in a Phase 3 pancreatic cancer trial. This week, Truist Securities went so far as to nominate RevMed as “the next oncology titan,” a title currently held by Merck and its blockbuster cancer drug Keytruda. Safety continues to challenge the gene therapy space, especially in Duchenne muscular dystrophy. Late last week, REGENXBIO announced mixed results from a Phase 3 program—the gene therapy did lead to functional improvements, but two serious adverse events caused the stock to drop 37%.Finally, Amgen’s rare disease drug Tavneos continues to face scrutiny. Last month, the FDA alleged that doctored data were filed to support Tavneos’ initial approval. Now, it’s been linked to 20 deaths in Japan.

In this episode of Denatured, you'll be hearing from Miguel Forte, president of the International Society for Cell and Gene Therapy (ISCT), and John Ellis, co-founder & CEO of Trenchant Bios, speaking live from the ISCT annual meeting. We dive into mesenchymal stem cells and induced pluripotent stem cells, exploring the science behind them, the manufacturing challenges, and the potential for scalable, engineered next-generation therapies.HostJennifer C. Smith-Parker, Director of Insights, BioSpaceGuestsJon Ellis, Co-founder & CEO, Trenchant BioMiguel Forte, President, International Society for Cell & Gene TherapyDisclaimer: The views expressed in this discussion by guests are their own and do not represent those of their organizations.

Moments after recording this episode of The Weekly, FDA Commissioner Marty Makary announced his resignation. Reports first circulated last Friday that President Donald Trump had signed off on his ouster. The president initially denied this account, originally from The Wall Street Journal, on Saturday morning, but appears to have now confirmed the plan. This confusion underscores a key theme in Robert F. Kennedy Jr.’s health department: lack of transparency and clear communication. Makary’s exit also highlights the high level of senior leadership turnover across the Department of Health and Human Services. With Makary is on his way out, the FDA will reportedly be helmed by an acting commissioner, Kyle Diamantas. This would also be fitting for an agency whose two key review divisions—the Center for Biologics Evaluation and Research (CBER) and Center for Drug Evaluation and Research (CDER)—are currently led by temporary directors. Meanwhile, one of Makary’s key initiatives, the Commissioner’s National Priority Review (CNPV) program, hit a snag last week as Sanofi has reportedly requested that the FDA remove its diabetes prevention drug Tzield from the scheme. This comes after acting CDER director Tracy Beth Høeg apparently intervened in the drug’s review. And in the business realm, BioSpace kicks off our annual compensation report. Who made the most money in 2025? Was it Eli Lilly’s David Ricks or Johnson & Johnson’s Joaquin Duato? Read BioPharm Executive to find out. Not subscribed? Sign up here for all of BioSpace’s insightful newsletters.

In this episode of Denatured, you'll be hearing from Georg Vo Beiske, CEO of Tribune Therapeutics and Jonas Hallén, co-founder & Chief Medical Officer of Calluna Pharma. We dive into IPF and fibrosis challenges, unpacking treatment hurdles, emerging targets, unmet needs and expansion paths beyond the lung.HostJennifer C. Smith-Parker, Director of Insights, BioSpaceGuestsGeorg Vo Beiske, CEO, Tribune TherapeuticsJonas Hallén, Co-Founder & CMO, Calluna PharmaDisclaimer: The views expressed in this discussion by guests are their own and do not represent those of their organizations.

First quarter earnings continue to roll in, with Pfizer, Eli Lilly, Amgen and more reporting in the past week. While Pfizer beat consensus estimates, it wasn’t enough for analysts, who had greater expectations for the New York pharma. Analysts were more than satisfied with Lilly’s 56% revenue increase, though the company itself appeared to want more—a feat executives believe it could have achieved had it not lowered prices on many of its medicines. Meanwhile, a host of small– to-medium biotechs are beginning to report, with analysts focused squarely on key near-term catalysts. On the regulatory beat, the FDA tapped Katherine Szarama as a temporary replacement for outgoing Center for Biologics Evaluation and Research (CBER) Director Vinay Prasad, whose controversial reign over the biologics division ended April 30. And the FDA held its first advisory committee meeting in nine months, for two AstraZeneca cancer drug applications, which former regulator Harpreet Singh said was missing the “Pazdur moment,” after stalwart oncology leader Richard Pazdur left the agency last November.Over in R&D, an optimistic story is unfolding in a treatment space that has endured much heartache over the past few years: ALS. In the past week, QurAlis and Corcept Therapeutics both reported positive mid-stage data for their respective candidates, with QurAlis’ QRL-201 eliciting an up to 50% in decrease progression, and Corcept reporting a two-year survival advantage for patients talking its dazucorilant.Finally, in BioPharm Executive this week, senior BioSpace editor Annalee Armstrong sits down with biotech founders to discuss the challenges of being a founder today.

In this episode of Denatured, you'll be hearing from Yaniv Sneor, founder of the Mid Atlantic Bio Angels and Alex Pederson, an investor at Mid Atlantic Bio Angels and partner at Alloy Bio Consulting. We discuss why a life sciences-only angel group matters, how they evaluate opportunities, and the importance of strong teams, capital efficiency and a realistic path to exit.HostJennifer C. Smith-Parker, Director of Insights, BioSpaceGuestsYaniv Sneor, Founder, Mid Atlantic Bio AngelsAlex Pederson, Investor, Mid Atlantic Bio Angels & Partner, Alloy Bio ConsultingDisclaimer: The views expressed in this discussion by guests are their own and do not represent those of their organizations.

First quarter earnings are coming in at a rapid pace, with Sanofi and Novartis defending patents for Dupixent and Lutathera, respectively, and Sanofi welcoming Belén Garijo as CEO. Still to come this week are Eli Lilly, AstraZeneca, Regeneron and many more.Lilly will undoubtedly discuss its recent streak of dealmaking, including a $2.25 billion pact with AI biotech Profluent, plus buyouts of Ajax Therapeutics for up to $2.3 billion and Kelonia Therapeutics for up to $7 billion.Meanwhile, Regeneron earned FDA approval for the highly anticipated gene therapy that will now be known as Otarmeni. The same day the approval came down, Regeneron also struck a deal with the White House.Over at the FDA, the agency has requested—again—that Amgen remove the autoimmune therapy Tavneos from the market. Separately, the FDA has issued three Commissioner’s National Priority Vouchers to unnamed psychedelic drug developers. Finally, who will replace Vinay Prasad, the head of the agency’s Center for Biologics Evaluation and Research (CBER), who departs at the end of April after one year as the biologics chief?

In this episode of Denatured, you'll be hearing from Dr. Sarah Howell, CEO at Arecor Therapeutics and Dr. Wendy S. Lane, clinical endocrinologist and diabetologist. We examine how increasingly connected and tailored diabetes technologies are reframing the field’s central opportunity around minimizing the day-to-day demands of managing the condition.

After entering the CAR T arena in February, Eli Lilly jumped onto the in vivo bandwagon, penning a deal worth up to $7 billion for Kelonia Therapeutics and its gene therapy delivery tech iGPS. Meanwhile, Belgium-based UCB scooped up Neurona Therapeutics for $650 million upfront and up to $500 million in milestone payments.On the policy front, President Donald Trump signed a new executive order that analysts believe could help psychedelic therapies become the “key next wave” of mental health therapies. The EO instructs the FDA to grant Commissioner’s National Priority Vouchers (CNPVs) to “appropriate psychedelic drugs” that have secured breakthrough designation, a move that could cut review timelines from 10-12 months to 1-2 months for these therapies.Elsewhere, the FDA’s rebuff of Replimune’s advanced melanoma drug RP1 continues to make waves, with CEO Sushil Patel slamming the agency for failing to exercise flexibility, while experts have lamented the regulator’s inconsistency.On the business side, Kailera managed to break biopharma’s all-time IPO record, raising $625 million last week. The new all-time high prompted BioSpace to take a look back at other historic IPOs in the industry, with mRNA heavyweight Moderna’s 2018 debut falling into second place.BioSpace also spoke with Denali Therapeutics CEO Ryan Watts about the company’s long-awaited FDA approval for Hunter syndrome drug Avlayah. “It was the greatest professional moment of my life,” Watts said during an event put on by Utah’s biotech community hub, BioHive.

In this episode of Denatured, you'll be hearing from Edoardo Negroni, co-founder & managing partner at AurorA-TT and Naveed Siddiqi, senior partner, Venture Investments at Novo Holdings. We debate whether Europe’s world-class science can be matched by a truly integrated venture ecosystem—and what it would take, in practice, to get there.HostJennifer C. Smith-Parker, Director of Insights, BioSpaceGuestsEdoardo Negroni, Co-Founder & Managing Partner, AurorA-TTNaveed Siddiqi, Senior Partner, Venture Investments, Novo HoldingsDisclaimer: The views expressed in this discussion by guests are their own and do not represent those of their organizations.

Johnson & Johnson kicked off Q1 earnings season with a “modest” beat, raking in $24.1 billion in sales as it targets $100 billion in revenue in 2026. J&J, as always, is the first to report results from a first quarter that has seen a wave of deals across biopharma; this M&A rush is expected to headline investor calls as more companies, including Novo Nordisk and Eli Lilly, report their Q1 results in the coming days. Meanwhile, IPOs are also on an upswing, with Kailera Therapeutics eyeing a raise that could reach as high as $533 million. On the regulatory front, Replimune failed for a second time to secure approval for its advanced melanoma therapy RP1, as the FDA held its ground, requesting a Phase 3 trial that CEO Sushil Patel indicated in a statement last week “will not be viable.” This comes as analysts say the FDA’s new policy of making complete response letters public is increasing accountability. Along with transparency, another key theme for the FDA this year has been its promise to be flexible, particularly when it comes to therapies for rare diseases. While BioSpace has closely covered the myriad cases where the agency has appeared to reverse its guidance to sponsors, two companies—Rezolute & CERo Therapeutics—lauded recent interactions with the FDA, calling reviewers “collaborative” and “curious.” Finally, heading into the American Association for Cancer Research’s annual meeting this weekend, several companies—including Revolution Medicines, Allogene and IDEAYA Biosciences and Servier—got a jump start, reporting positive data in a number of indications. Revolution’s report was especially seminal. The company’s therapy, daraxonrasib, doubled survival in pancreatic cancer—a disease that has just a 13% survival rate five years after diagnosis.

Listen to BioSpace’s Vice President of Marketing Chantal Dresner and Careers Editor Angela Gabriel discuss 2026 job market performance so far, sharing the latest BioSpace data.They touch on positive signs they saw between January and March, including a reduced number of layoffs, and what this year might have in store for those seeking employment in 2026.

In this episode of Denatured, you'll listen to Jason Jones, head of global business development at Cellular Origins and Alexander Seyf, founder & CEO of  Autolomous. They discuss how cell and gene therapy manufacturing is facing growing pressure to scale more quickly and efficiently, driving developers and manufacturers toward automation, digitization, robotics and broader collaboration across the ecosystem.HostJennifer C. Smith-Parker, Director of Insights, BioSpaceGuestsJason Jones, Head of Global Business Development, Cellular OriginsAlexander Seyf, Founder & CEO, AutolomousDisclaimer: The views expressed in this discussion by guests are their own and do not represent those of their organizations.

Eli Lilly won FDA approval last week for orforglipron—now Foundayo— officially launching what promises to be a heated battle between Lilly and chief rival Novo Nordisk.  Elsewhere, the M&A space keeps chugging along, with Gilead Sciences gobbling up partner Tubulis for up to $5 billion and Neurocrine Biosciences nabbing Soleno Therapeutics for $2.9 billion. The industry will be on high alert for more deals, as analysts say Amgen, AbbVie and Bristol Myers Squibb all have more money to spend. At the White House, President Donald Trump levied his long-promised tariffs on the pharma industry, but myriad carveouts mean many companies will be safe from the 100% tax, at least for now. And Trump’s Most Favored National pricing scheme is endangering access to new drugs in Europe as companies forgo launches  in countries that could pull down U.S. prices. Meanwhile, the Trump administration came out with its proposed 2027 budget on Friday, with several requests for the FDA and Department of Health and Human Services overall. In line with the administration’s efforts to accelerate development of therapies for rare diseases, the FDA proposes is seeking to permanently authorize the rare pediatric disease priority review voucher program. Other proposals include a new clinical trial notification pathway and expanded authority to regulate post approval manufacturing changes. These requests come during a time when the agency is, as always, walking the precarious tightrope of rigor vs. unmet need—with rare disease leaders calling for clarity around topics like externally controlled trials.

In this episode of Denatured, you'll listen to Dr. Marc Hedrick, President and CEO of Plus Therapeutics and Dr. Phil Kantoff, CEO & Co-founder of Convergent Therapeutics. They discuss breakthroughs like alpha-emitting isotopes, supply chain solutions and why radiotherapeutics is biotech's next big frontier.Host Jennifer C. Smith-Parker, Director of Insights, BioSpace Guests Marc Hedrick, President & CEO, Plus Therapeutics Philip Kantoff, Co-founder & CEO, Convergent TherapeuticsDisclaimer: The views expressed in this discussion by guests are their own and do not represent those of their organizations.

Biogen, Eli Lilly and Merck spent more than $20 billion in the past week to swallow biotechs with approved products or promising drug candidates—representing three of this year’s four biggest takeovers. First, Merck picked up Terns Pharmaceuticals and its mid-stage leukemia drug for $6.7 billion. Then, on Tuesday, Lilly and Biogen struck, acquiring Centessa Pharmaceuticals and Apellis Pharmaceuticals respectively.Those big-ticket deals aren’t the only recent transactions, however. Others include Novartis’ up to $2 billion pick up of Excellergy and Gilead’s $2.1 billion purchase of Ouro Medicines. Meanwhile, Kevin Tang—the newly minted CEO of Aurinia Pharmaceuticals—again has his sights set on Kezar Life Sciences, which he previously targeted in 2024. This time, biopharma’s “clean-up” man is offering $50 million for the chronic disease–focused biotech.Last week also saw nods for Denali Therapeutics’ Avlayah, the first treatment for Hunter syndrome to target the disease’s neurological complications, and Rocket Pharmaceuticals’ Kresladi for leukocyte adhesion deficiency-I. And it’s been a big week for Biogen, which besides moving on Apellis, won approval of a high-dose formulation of spinal muscular atrophy drug Spinraza and scored a Phase 2 win for lupus candidate litifilimab in cutaneous lupus erythematosus.On the weight loss front, Wave Life Sciences’ stock was cut in half after its obesity candidate WVE-007 failed to impress investors in a Phase 1 trial.

In this episode, you'll be listening to Sergey Jakimov, managing partner at LongeVC and Artem Trotsyuk, operating partner, US, LongeVC. We discuss how developers in the longevity space should stop chasing aging as an abstract target and concentrate on specific mechanisms that can clearly tackle age-related conditions. HostJennifer C. Smith-Parker, Director of Insights, BioSpaceGuestsSergey Jakimov, Managing Partner, LongeVCArtem Trotsyuk, Operating Partner-US, LongeVC

Immunology & inflammation stole the show this week, as Gilead dropped up to $2.1 billion for Ouro Medicines and its T cell engager OM336—an investment it hopes to split with longtime partner Galapagos. Meanwhile, Sanofi also added a T cell engager in a licensing deal with Kali Therapeutics worth $180 million upfront.One I&I partnership that bore fruit last week was that of Johnson & Johnson and Protagonist Therapeutics which got their IL-23 receptor blocker Icotyde across the FDA finish line for plaque psoriasis. Icotyde is the first commercial product for Protagonist.Even the drama this week came from the I&I space, as Kevin Tang took the reins as CEO of Aurinia Pharmaceuticals, which you may remember was the company at the heart of former Center for Drug Evaluation and Research Director George Tidmarsh’s exit from the FDA.Outside of I&I, the CDC’s Advisory Committee on Immunization Practices made headlines once again as Vice Chair Robert Malone posted to social media that the group was being disbanded. The Department of Health and Human Services quickly corrected the record, but Malone posted a few hours later that “dissolving and reforming remains one of options being considered.”In FDA policy news, BioSpace recaps the now nine-month-old Commissioner’s National Priority Voucher pilot program and unpacks new draft guidance on animal testing alternatives. Plus, check out an opinion article on the need for precision ALS drugs and features on gene therapy for hearing loss and Big Pharma’s overall R&D spending in 2025.

In this episode, you'll be listening to  Akshay Rai, principal, Healthcare & Biotech Investments at Premji Invest and Viswa Colluru, CEO and founder, Enveda. They discuss how AI platforms must now prove themselves through data, focused pipelines and clinical readouts and that promises of faster, cheaper drug discovery are not enough to entice strong investor engagement.  HostJennifer Smith-Parker, Director of Insights, BioSpaceGuestsViswa Colluru, CEO & Founder, EnvedaAkshay Rai, Principal, Healthcare & Biotech Investments, Premji InvestDisclaimer: The views expressed in this discussion by guests are their own and do not represent those of their organizations.

Biopharma’s weight loss warriors have led the news this week, with Structure Therapeutics and Rhythm Pharmaceuticals announcing new data. Structure delivered a “competitive profile” for its investigational GLP-1 pill compared to Eli Lilly’s orforglipron and Novo Nordisk’s oral Wegovy, BMO Capital Markets wrote to investors, after the candidate elicited 16.3% weight loss after 44 weeks in a Phase 2 trial. Rhythm was not as successful, announcing that Imcivree failed in a Phase 3 basket trial of various genetically driven obesities. Meanwhile, Lilly and Novo made headlines outside of the clinical realm, as Lilly warned the public of the potential safety risks of taking compounded versions of tirzepatide—marketed as Zepbound for obesity and Mounjaro for type 2 diabetes. And Novo was hit with an FDA warning letter for failing to investigate reported adverse events—including three deaths—potentially linked to its own diabetes drug Ozempic. The letter only adds to what has been a challenging recent run for Novo, one that led to a 34% drop in total assets under management for its controlling shareholder Novo Holdings. On the regulatory front, Robert F. Kennedy Jr.’s vaccine agenda is stalled after Massachusetts District Court Judge Brian Murphy found that his efforts to overhaul policies in this area were likely unlawful. Murphy specifically cited Kennedy’s move last June to empty the CDC’s Advisory Committee on Immunization Practices (ACIP). This comes on the heels of a White House crackdown in which officials would like the health secretary to tone down his vaccine skepticism, according to a report by The Wall Street Journal. At the FDA, tensions continue to escalate, with Sen. Ron Johnson (R-WI) launching an investigation into recent rejections of rare disease drugs. And transparency issues have come to light once again regarding the circumstances around biologics chief Vinay Prasad’s imminent departure and recent request to remain anonymous during a media briefing about uniQure’s gene therapy for Huntington’s disease.

In this episode of Denatured, you'll listen to Oxana Iliach, senior director of regulatory strategy at Certara and Vera Pomerantseva, director of product management for risk-based quality management at eClinical Solutions. We speak about how the FDA's latest decision to have one, rather than two pivotal studies, for new drug applications raises the bar for data collection and risk-based management.Host⁠Jennifer C. Smith-Parker⁠, Director of Insights, BioSpaceGuestsOxana Iliach, Senior Director of Regulatory Strategy, CertaraVera Pomerantseva, Director of Product Management for RBQM, eClinical SolutionsDisclaimer: The views expressed in this discussion by guests are their own and do not represent those of their organizations.

After Friday’s news that Center for Biologics Evaluation & Research Chief Vinay Prasad will leave the FDA—again—at the end of April, stocks for several rare disease drug developers popped. UniQure, in particular, was up 51% in premarket trading on Monday. Prasad in a meeting last Thursday with select journalists called the biotech’s Huntington’s treatment AMT-130 a “failed” therapy, according to STAT News. Shares of Replimune and REGENXBIO—which have suffered rejections during the past year—also rose.One person who is not impressed with the plethora of rare disease drug rejections of late—H.C. Wainwright said in a note Tuesday that there have been at least five cell and gene therapies they believe could have been approved under prior FDA officials—is Wisconsin Senator Ron Johnson. Tuesday, Bloomberg News reported that Johnson has launched an investigation into these recent denials.Johnson called the FDA’s request that uniQure conduct a sham surgery-controlled trial of AMT-130 “bureaucratic idiocy,” according to the publication. Meanwhile, uniQure and the FDA appear to be on different pages regarding the design of this prospective trial, with uniQure Chief Medical Officer Walid Abi-Saab referring to a 10-12 hour surgery during which [burr] holes would be drilled in patients’ skulls and Prasad claiming on a media call last week that it would require only “one to three nicks in the scalp.”In other news, no episode of The Weekly would be complete without our weekly weight loss segment. Roche and Zealand Pharma’s amylin analog fell short of Eli Lilly’s rival candidate eloralintide; AbbVie reported what analysts called “competitive” results, with its amylin analog eliciting nearly 10% weight loss at 13 weeks in a Phase 1 trial; and Regeneron touted a much-needed Phase 3 win for Hansoh-partnered dual GLP-1/GIPR agonist olatorepatide in China. Beyond data, Novo Nordisk and Hims & Hers are together again, with Novo striking a deal to sell its injectable and oral GLP-1 medicines through the telehealth provider.Elsewhere on the business side of biopharma, experts are reporting a cut-throat atmosphere behind doors on the M&A front as the supply of companies available to buy dwindles.

Stylus Medicine CEO Emile Nuwaysir and Chief Scientific Officer Jason Fontenot speak with BioSpace about the biotech's work on next-generation genetic medicines—and why they think it’s the perfect time to be in the cell therapy game. Stylus Medicine is featured in BioSpace's  list, the top startups to watch in the U.S.HostAnnalee Armstrong, Senior Editor, BioSpaceGuestsEmile Nuwaysir, CEO, Stylus MedicineJason Fontenot, Chief Scientific Officer, Stylus MedicineDisclaimer: The views expressed in this discussion by guests are their own and do not represent those of their organizations.

In this Denatured episode, Jennifer C. Smith-Parker speaks to Ram May-Ron, managing partner at FreeMind Group, and Ravi Kiron, managing director at Biopharma Strategy Advisors. We'll discuss how best to tailor an investment approach of both nondilutive funding and family offices to overcome the drug development valley of death. Host⁠Jennifer Smith-Parker⁠, Director of Insights, BioSpaceGuestsRam May-Ron, Managing Partner, FreeMind GroupRavi Kiron, Managing Director, Biopharma Strategy AdvisorsDisclaimer: The views expressed in this discussion by guests are their own and do not represent those of their organizations.

The FDA is dominating the headlines once again thisweek.  Days after FDA Commissioner Marty Makary appeared to question uniQure’s gene therapy candidate for Huntington’s disease, the company revealed that the agency will require it to conduct a randomized, double-blind, sham surgery–controlled Phase 3 study. The FDA also published anothercomplete response letter (CRL), this one for REGENXBIO’s gene therapy for Hunter syndrome. The rejection, sustained by the biotech early last month, was driven by issues with the study’s population, controls and use of surrogate markers to measure efficacy, according to the document.  Meanwhile, regulatory experts have expressed concernsthat the FDA’s circle of trust is shrinking, making many decisions feel like “fiat”—both in terms of individual drug applications and policy. The FDA has reportedly initiated a probe into complaints that a toxic workplace is fostered by CBER director Vinay Prasad, who is at the heart of many of these decisions. Finally, the biopharma industry continues to react to the agency’s pivot from a requirement of two pivotal trials to one for approval, asking why now, what are the risks and what exactly the FDA expects from this one trial.   Still on the gene therapy front, Sarepta Therapeutics CEO Doug Ingram stepped down last week to spend more time with family as the company’s muscular dystrophy mission hits home. Also during the company’s fourth quarter earnings call, Sarepta projected that sales of its embattled Duchenne muscular dystrophy gene therapy Elevidys will be flat or down as far as 15% in 2026.  On the obesity front, Eli Lilly topped Novo Nordisk again in a weight loss trial, this time in a Lilly-sponsored study of patients with type 2 diabetes. But don’t count Novo out yet. The company is actively seeking out new obesity assets, according to business development executive Tamara Darsow. Just last week, Novo linked with Boston’sVivtex to advance novel weight loss pills.Finally, check out BioPham Executive this week for a rundown of 2025’s top-selling assets—spoiler: Merck’s Keytruda held onto its crown as number one—and a story on former2seventy exec Chip Baird’s new role as CEO of recently launched Poplar Therapeutics, which secured a $45 million series A extension this week.

In this episode, you'll be listening to Indu Navar, CEO and founder of EverythingALS and Dr. Olga Uspenskaya, chief medical officer at VectorY Therapeutics. We'll be speaking about patient-pharma collaborations accelerating trials and hope, advances in ALS biology understanding and biomarker-driven endpoints.HostJennifer Smith-Parker, Director of Insights, BioSpaceGuests⁠Indu Navar⁠, CEO and Founder, EverythingALSDr. Olga Uspenskaya, Chief Medical Officer, VectorY TherapeuticsDisclaimer: The views expressed in this discussion by guests are their own and do not represent those of their organizations.

Eli Lilly notches another win over Novo Nordisk, as Zepbound bests CagriSema in a head-to-head trial sponsored by Novo; The FDA kicked off Rare Disease Week, providing draft guidance on its new plausible mechanism pathway, while a bipartisan senate hearing on Thursday will focus on the authorization process for rare conditions; Another leadership change shakes up CDC; and Gilead acquires CAR T partner Arcellx for nearly $8 billion.  Everything is coming up Lilly. The Indianapolis-based pharma bested its chief rival, Novo Nordisk in a head-to-head test. In a Phase 3 trial initiated by Novo itself, Lilly’s Zepbound generated 25.5% weight loss while the Danish pharma’s CagriSema elicited 23%. The results sent Novo’s shares plummeting by an unprecedented 20% to a pre-Wegovy valuation while Lilly’s market cap continues to climb.   Novo attempted a comeback on Tuesday, announcing that its triple-G agonist UBT251 scored almost 20% weight loss after 24 weeks in a Phase 2 trial in China. By comparison, Lilly’s own triple-G competitor retatrutide led to 17.5% weight loss over the same timeframe, according to BMO Capital Markets analysts. Novo also sweetened the pot, announcing that it would slash the prices for all three of its GLP-1 medicines starting in 2027.    Meanwhile, the FDA kicked off Rare Disease Week with draft guidance on the new Plausible Mechanism Pathway for personalized therapies that was first teased in November. Jumping off last summer’s Baby KJ success story, the new pathway is aimed at advancing treatments for ultra-rare diseases. And a bipartisan senate hearing on Thursday will focus on the authorization process for rare disease therapies.  While the rare disease space has enjoyed recent regulatory progress, funding these vital therapies remains a challenge. Companies like the Orphan Therapeutics Accelerator (OTXL), a non-profit biotech, are trying to change this with creative approaches including tax exempt status and unique partnerships with CDMOs and CMOs. Finally, in a move that also has implication for the rare disease space, the FDA’s official pivot from a two clinical trial requirement to just one for new drug applications is lighting up biopharma social media.   And over at the CDC, there is more upheaval on the leadership front as National Institutes of Health Director Jay Bhattacharya replaces acting director Jim O’Neill as head of the agency, and principal deputy director Ralph Abraham steps down, citing “unforeseen family obligations.”    On the business front, Gilead inked the biggest M&A deal of the year so far, acquiring CAR T partner Arcellx for nearly $8B. And Merck’s Keytruda should have a few extra years of dominance thanks to a web of patents, with billions on the line. Check it out in BioPharm Executive, in your inboxes Wednesday.  HostsJef Akst, Managing Editor, BioSpaceHeather McKenzie, Senior Editor, BioSpaceAnnalee Armstrong, Senior Editor, BioSpace

Corsera Health’s Chief Operating Officer Rena Denoncourt and CFO Meredith Kaya speak with BioSpace about the biotech’s mission and vision for the next generation of cardiovascular care in this special bonus episode. Corsera Health is featured in BioSpace's NextGen: Class of 2026 list, the top startups to watch in the U.S.HostAnnalee Armstrong, Senior Editor, BioSpaceGuestsRena Denoncourt, Chief Operating Officer, Corsera HealthMeredith Kaya, Chief Financial Officer, Corsera HealthDisclaimer: The views expressed in this discussion by guests are their own and do not represent those of their organizations.

In this episode, you'll be listening to Jane Hughes, President of R&D and Co Founder of Verdiva Bio, and Jon Rees, CEO and cofounder of MitoRx Therapeutics. We'll discuss next-generation obesity solutions tackling GLP-1's muscle loss and adherence challenges, through innovative muscle preservation, oral administration and combination therapy.HostJennifer Smith-Parker, Director of Insights, BioSpaceGuestsJane Hughes, President of R&D and Co-founder, Verdiva BioJon Rees, CEO and Co-founder, MitoRx TherapeuticsDisclaimer: The views expressed in this discussion by guests are their own and do not represent those of their organizations.

Last week, the FDA declined to review Moderna’s mRNA-based flu vaccine, with Center for Biologics Evaluation and Research Director Vinay Prasad himself signing the refuse-to-file (RTF) letter. Prasad said Moderna’s comparator group “does notreflect the best-available standard of care,” while Moderna pushed back, saying CBER had previously found a standard-dose flu shot to be an “acceptable” comparator. Despite the shocking turn of events, HHS backed Prasad, saying Moderna didn’t “follow very clear FDA guidance from 2024.”Regardless of the reasons, the development imperilsModerna’s breakeven plans, affecting not just the company’s investigational flu vaccine but also a flu-COVID combo shot and other parts of the company’s pipeline. And for the broader industry, it highlights the regulatory uncertainty that persists into 2026, even as other countries move full speed ahead with novel modalities.That regulatory uncertainty extended this week to DiscMedicine, whose rare disease drug bitopertin was rejectedby the FDA. The company tried to convince investors that its planned Phase 3 trial would pave the way toward a refiling, but analysts raised concerns that the study was developed with oldFDA leadership. Disc’s situation also raises questions about the FDA’s Commissioner’s National Priority Voucher, which bitopertin had received.Meanwhile, Compass Pathways dropped long-awaited data from two Phase 3 trials of its psilocybin-based COMP360, showing strong durability data that “clearly met the Street’s bar for success,” according to Stifel analysts. The company plans to complete a rolling new drug application by the end of the year.Finally, the CDC has been left leaderless, again, with the departure of Jim O’Neill, who had been servingas acting director after the ouster of Susan Monarez last summer. And Sanofi’s Paul Hudson was removed as CEO last week. He’ll be replaced by Merck KGaA’s Belén Garijo, who becomes just the second woman in charge of a Big pharmacompany.

Last year, the FDA announced it would be phasingout animal testing requirements for some therapies. The NIHfollowed suit. According to Thomas Hartung, professor and chair at Johns Hopkins Bloomberg School of Public Health, these policy shifts are an “overdue adaptation to scientific progress.” In this special edition of The Weekly, Hartung discusses howartificial intelligence (AI) and various non-animal models such as human organoids will transform drug development and delivery, especially now that the regulatory side is catching up. The FDA Modernization Act, now making its way through Congress, codifies the FDA’s stated goal of starting to move away from animal testing. And in making that announcement last year, the FDA put out a roadmap for how to do this. In May, Hartung will attend the National Biotechnology Conference in San Diego as a keynote speaker. BioSpace is a media partner of the meeting, and Managing Editor Jef Akst will also attend to moderate the executive track. The 2026 National Biotechnology Conference runs May 11–14.You can find the agenda here.

In this episode of Denatured, Jennifer C. Smith-Parker speaks with Dr. Rob Monroe, Vice President and Chief Scientific Officer of Oncology at Danaher Corporation and Chief Medical Officer at Leica Biosystems, and Jennifer Fakish, Vice President and Franchise Head of Oncology at Danaher Corporation. We'll be discussing how antibody drug conjugates (ADCs) are transforming cancer care and with AI-powered pathology, doctors can now measure her HER2 more precisely to match patients with the best treatments. HostJennifer Smith-Parker, Director of Insights, BioSpaceGuestsDr. Rob Monroe, Vice President and Chief Scientific Officer, Oncology, Danaher Corporation; Chief Medical Officer, Leica BiosystemsJennifer Faikish, Vice President and Franchise Head, Oncology, Danaher CorporationDisclaimer: The views expressed in this discussion by guests are their own and do not represent those of their organizations.

Last Thursday, Hims & Hers launched a compounded versionof Novo’s oral Wegovy, approved just before Christmas and launched in early January. Novo was, not surprisingly, none too pleased. FDA Commissioner Marty Makary denounced“illegal copycat drugs” in an X post the same day without naming Hims, which then said it would notsell the oral weight loss drug. Novo sued the consumer healthcare company for patent infringement, seeking potentially hundreds of millions in damages. Thenit emerged that FDA hit Hims with a warning letter last year about infestations of vermin at a facility it owns, including rodents, birds and insects, as well as a live spider in a production area.The timing of all the Hims drama is interesting, because BioSpace was already covering Novo, Lilly and their blockbuster GLP weight loss drugs as both companies—and others—reported 2025 fourth quarter and full year earnings. EliLilly and Novo both reported last Wednesday, and both spoke of Novo’s oral Wegovy launch positively. But the calls had two very different tones, as Lilly beat Q4 2025 analyst consensus by more than a billion dollars while Novo projected sales to decline by 5% in 2026.In other recent earnings calls, AbbVie touted strong Skyriziand Rinvoq sales, and the company continues to follow Johnson & Johnson’s lead into the psychedelics space. And Biogen discussed the highly anticipated readout of its tau-targetingtherapy for Alzheimer’s.Finally, in gene therapy, Sarepta struggles to recover from last year’s patient deaths as other DMD contenders near the market; uniQure pauses two arms of a clinical trial for Fabry disease; and Ultragenyx resubmits its in vivo gene therapy UX111 for the treatment of Sanfilippo syndrome type A.

In this episode of Denatured, Jennifer C. Smith-Parker speaks to Erik Digman Wiklud, CEO of Circio and Jacob Becraft, CO-founder and CEO of Strand Therapeutics. Since the mRNA vaccine breakthroughs of the COVID-19 era, attention has turned to what's next for programmable medicines. While first- generation mRNA prove the power of transient genetic instruction, its instability, immune reactivity, and short-lived expression have limited its use mainly to vaccines. Emerging platforms like circular and logic circuit RNA are expanding the field's therapeutic horizons.HostJennifer Smith-Parker, Director of Insights, BioSpaceGuestsErik Digman Wiklund⁠, CEO, CircioJacob Becraft, Co-founder and CEO, Strand TherapeuticsDisclaimer: The views expressed in this discussion by guests are their own and do not represent those of their organizations.

Pfizer reported the first data from its new obesity pipeline, picked up in the nearly $10 billion acquisition of Metsera last fall. While BMO Capital Markets said in a Tuesday note that the data “look competitive,” analysts clamored for more details on Pfizer’s earnings call the same morning—and were left wanting more. Meawhile, Merck batted away accusations of “modest growth” from analysts on its own earnings call, as CEO Robert Davis touted “probably the broadest and widest pipelinewe’ve had in years.” These calls followed Roche last week and Johnson & Johnson before that, but earnings season is just getting started. On the docket today is Eli Lilly, which has been acquisitive of late, plus Novo Nordisk, Novartis, AbbVie and more.  On the regulatory front, the FDA is expected to decideon eight products this month, including REGENXBIO’s Hunter syndrome gene therapy RGX-121. The biotech ran into a regulatory snag last week, however, as the FDA placed a clinical hold on two of its programs, including RGX-121. The agency also launched its much-anticipated PreCheck pilot program, which aims to make it easier for companies to build manufacturing plants in the U.S. And President Donald Trump’s TrumpRx platform is delayed, potentially amid anti-kickback concerns.  In ClinicaSpace this week, we took a deep dive intothe resurgent psychedelics space, which could see two companies—Compass Pathways and Definium—submit FDAapplications this year. H.C. Wainwright analyst Patrick Trucchio told BioSpace 2026 is set to be “the biggest year from a clinical data standpoint,” since the firm began covering Compass in 2018. And check your inboxes Wednesday for BioPharmExecutive, where we take a look back at the banner IPO year that was 2021: Where are these companies now?

In this episode of Denatured, Jennifer C. Smith-Parker speaks with RTW’s Rod Wong and Stephanie Sirota how shifting JPM deal timing masks record M&A potential; why oncology, obesity, psychedelics, and neuroscience are attracting fresh capital; and how “alpha stacking” shapes their investment edge in an age of chronic uncertainty. They cover topics discussed in RTW's new book, "Innovation is the Best Medicine."Hosts⁠⁠⁠⁠Jennifer Smith-Parker⁠⁠, Director of Insights, BioSpaceGuestsRoderick Wong, Managing Partner, Chief Investment Officer, RTW InvestmentsStephanie Sirota, Partner, Chief Business Officer, RTW InvestmentsDisclaimer: The views expressed in this discussion by guests are their own and do not represent those of their organizations.

Roche made the biggest splash this week so far, announcing on Tuesday that GLP-1/GIP injectable CT-388 led to 22.5%weight loss in a Phase II trial. These numbers appear to put CT-388, which Roche acquired in its $2.7 billion Carmot buy, in line with Eli Lilly’s Zepbound, according to William Blair analysts. Roche plans to start a Phase III study of CT-388 in the first half of this year and is also pairing the drug with a therapy from Zealand Pharma, with the aim of offering a weight lossoption with fewer gastrointestinal side effects. Meanwhile, Baseline Therapeutics debuted to challenge Lilly with a Phase III–ready GLP-1 for alcohol use disorder.  In the vaccines sector, Moderna took perhaps the biggest action to date amid Health Secretary Robert F. Kennedy’s anti-vaccine policies and rhetoric, last week announcing that the company will no longer run late-stage vaccine trials for infectious diseases. “You cannot make a return on investment if you don’t have access to the U.S. market,” CEO Stéphane Bancel saidthe World Economic Forum at Davos, Switzerland. Pfizer CEO Albert Bourla, also speaking at Davos, called RFK Jr.’s rhetoric and policies on vaccines “anti-science.”  Finally, Sarepta released new data on Monday for Elevidys, the company’s embattled gene therapy for neuromuscular disease Duchenne muscular dystrophy. Plus, check out up-and-coming treatments for Alzheimer’s and Parkinson’s.

In this episode of Denatured, BioSpace editorial team members, Senior Editor, Annalee Armstrong, and News Editor, Dan Samorodnitsky, discuss their post-JPM takeaways and 2026 forecasts after speaking to a range of pharma and biotech executives and investors last week.Hosts⁠Jennifer Smith-Parker⁠, Director of Insights, BioSpace⁠Guests⁠Annalee Armstrong, Senior Editor, BioSpace⁠Dan Samorodnitsky, News Editor, BioSpaceDisclaimer: The views expressed in this discussion by guests are their own and do not represent those of their organizations.

The 2026 J.P. Morgan Healthcare Conference concludedlast week without a big M&A deal. But the meeting was anything but boring, with President Donald Trump’sMost Favored Nation drug pricing scheme and the perpetual battle for supremacyin the obesity space between Eli Lilly and Novo Nordisk prime topics of conversation. Meanwhile, other companies like Biohaven refuse tocede the entire weight loss market, stating their intention to carve out apiece of the massive pie.  On the competitive front, Novo took a step forwardMonday with the news that its Wegovy pill reached nearly 3,100 patients since its launch on Jan. 5. Meanwhile, an FDAdecision for Lilly’s own oral offering, orforglipron, has been delayed from March28 to April 10. Orforglipron is being reviewed under the FDA’s new Commissioner’sNational Priority Voucher (CNPV) program, which aims to shorten the reviewperiod from 10–12 months to 1–2 months. Other voucher recipients—includingSanofi and Disc Medicine—have also had their decisions delayed.  While the FDA wasn’t a primary focus at JPM, severalbiotechs sought to make clear during their investor presentations that they hadsecured alignment with the regulator on trial design or pathways to approval. This comesafter a year that has seen many of their peers experience regulatory whiplashas previous FDA guidance was seemingly reversed.  And on the business front, IPOs are back! This week, SpyGlass Pharma and AgomAb Therapeutics joined Veradermicsand Eikon Therapeutics on the rapidly accelerating IPO train. Aktis Oncology,which announced its bid in late 2025, debuted on the Nasdaq earlier this month. And in BioPharm Executive this week, Annalee Armstrongand Dan Samorodnitsky catch up with execs from Novo Nordisk, Korro Bio, Biohaven and Galapagos.

In this episode, BioSpace’s Vice President of Marketing Chantal Dresner and Careers Editor Angela Gabriel discuss Q4 2025 job market performance, sharing the latest BioSpace data. They touch on positive signs they saw last quarter and what this year might have in store for those seeking employment. Want to receive our latest quarterly job market reports as soon as they’re published? ⁠⁠⁠Subscribe⁠⁠⁠ to Career Insider for our job market updates, job trends, career advice and more.

AbbVie bet nearly $5 billion for the ex-China rights to RemeGen’s PD-1/VEGF bispecific and Novartis struck a $1.5 billion Alzheimer’s-focused deal with China-based SciNeuro Pharmaceuticals as the 2026 J.P. Morgan Healthcare Conference got underway on Monday. These and other recent tie-ups speak to the therapeutic trends predicted to be hot this year by experts who spoke with BioSpace, who particularly noted that cancer and neuroscience would remain top of the list.  Meanwhile, attendees are still waiting for that “one big M&A deal.” Could it come from Merck? CEO Rob Davis expressed high confidence during the company’s JPM presentation on Monday, revealing that Merck is open to deals in the range of “multi tens of billions of dollars.” On the weight loss front, Eli Lilly and Novo Nordisk both updated attendees on the state of play, with Lilly expressingconfidence in its supply chain to support the launch of obesity pill orforglipron and Novo continuing to lament the business it is still losing to compounders.  Just in time for JPM, the IPO window cracked open, with Aktis Oncology debuting on the public markets last week and Veradermics and Eikon Therapeutics announcing their bids to follow suit. A similar thaw occurred last year before the window slammed shut again as myriad policy changes frightened investors. How will this year play out?  2025 had its challenges, especially on the policy front. In his annual letter, Flagship CEO Noubar Afeyan cited “growing contempt” in the U.S. for the scientific method and arapidly emerging Chinese biopharma ecosystem among the greatest challenges facing the industry. And while the funding may now be flowing, it still favors later-stage companies. A .J.P. Morgan report released prior to the conference showed VC funding was down across the board last year, but especially for biotechs recruiting early rounds.  Finally, while JPM is the primary focus, the FDA hasn’t taken the week off. Just prior to recording, the regulator requestedthat Lilly and Novo remove the suicide warning from the labels of certain GLP-1 medications after a comprehensive review found no increased risk of suicidal ideation and behavior with these drugs. On the policy front, the FDA continued its quest to accelerate the development of cell and gene therapies with new manufacturing exemptions.  The regulator also took action on two applications this week, approving a rare disease drug developed by Sentynl Therapeutics and Fortress Biotech and denying a cell therapy submitted by Atara Biotherapeutics. To learn about key upcoming FDA action, check out 6 FDA Decisions To Watch in Q1 2026 in ClinicaSpace this week.

In this episode of Denatured, Jennifer C. Smith-Parker speaks to Maha Katabi, general partner at Sofinnova Investments and Andrew Lam, managing director, head of Biotech Private Equity at Ally Bridge Group, about how M &A dynamics, dealmaking and global partnerships are reshaping portfolio valuations and paths to growth in 2026. HostJennifer Smith-Parker, Director of Insights, BioSpaceGuestsMaha Katabi, General Partner, Sofinnova InvestmentsAndrew Lam, Managing Director, Head of Biotech Private Equity, Ally Bridge Group (ABG)Disclaimer: The views expressed in this discussion by guests are their own and do not represent those of their organizations.

Obesity titans Novo Nordisk and Eli Lilly characteristically kicked off 2026 in the headlines. After capping 2025 with the first-ever FDA approval of an oral GLP-1 for obesity, Novo launched its Wegovy pill on Monday, starting at $149 per month out of pocket for the lower doses. Meanwhile, all eyes are on Lilly, which expects a greenlight for orforglipron as early as March. The oral weight loss play is set to be a key aspect of a pivotal year for Lilly, which hopes to more than double its annual revenue in just a few years.   Also pulling a rabbit out of a hat just before Christmas were nine drugmakers—including Amgen, Bristol Myers Squibb, Boehringer Ingelheim—which signed Most Favored Nation drug pricing agreements with the Trump administration, likely securing their businesses against the threat of tariffs. However, drug pricing will continue to be top of mind in 2026, experts told BioSpace, as only about half of Big Pharmas have signed a deal, and small and mid-sized companies haven’t yet begun.   Another area biopharma leaders are watching with bated breath is the FDA. The overwhelming consensus from experts who spoke with BioSpace is that last year was a disaster—particularly from a workforce standpoint. Still, the agency approved 56 novel drugs, down just a tick from 2024’s 59. Advisory committees fell off considerably, in both number and concordance, however, according to a report by Jefferies, as these meetings came under scrutiny by FDA leadership.  In 2026, will the unprecedented leadership turnover—especially at CDER—settle down? Will the new rare disease and cell and gene therapy policies bear first fruits? Also on the regulatory front, the CDC has already been busy this year, axing several childhood vaccines from its recommended schedule with potential implications for biopharma companies including Pfizer and Merck.   And if there’s one area biopharma watchers anticipate even more than regulatory action, it’s M&A action. Last month, analysts shared their top targets with BioSpace, listing names like Summit Therapeutics, Apogee Therapeutics and Axsome Therapeutics. Here at BioSpace, we’ll stick with Viking.   Finally, don’t forget to check out our NextGen Class of 2026—featuring 15 biotech startups whose mission and platforms cut through a challenging environment. And make sure to join us next week when senior editor Annalee Armstrong and news editor Dan Samorodnitsky will provide dispatches from the J.P. Morgan Healthcare Conference.

In this episode of Denatured, Jennifer Smith-Parker speaks to Kenneth Galbraith, CEO at Zymeworks and Josh Smiley, president and COO at Zai Lab, about how renewed confidence is driving biotech entering 2026. As we look towards conversations that will shape this year's J.P. Morgan Healthcare Conference, the biotech industry is fueled by improving market sentiments, visible at recent meetings like Jeffries in London, and reinforced by a wave of partnerships, strategic acquisitions and Chinese collaboration that is recalibrating investor expectations.HostJennifer Smith-Parker, Director of Insights, BioSpaceGuestsKenneth Galbraith, CEO, ZymeworksJosh Smiley, President and Chief Operating Officer, Zai LabDisclaimer: The views expressed in this discussion by guests are their own and do not represent those of their organizations.

Eli Lilly is wrapping up 2025 with record-breaking weight loss in a late-stage trial for its triple hormone receptor agonist retatrutide. Results from the Phase III TRIUMPH-4 trial exceeded analyst expectations, leading BMO Capital markets to cleverly dub it “a true TRIUMPH.” Also in the weight loss arena, Zealand Pharma inked a deal with China’s OTR Therapeutics worth up to $2.5 billion to collaborate on next-gen drugs for obesity and other metabolic diseases, and Rhythm Pharmaceuticals awaits a Dec. 20 FDA verdict for Imcivree in hypothalamic obesity.   Turning to the FDA, reports broke late last week that the agency was considering slapping a black box label—its strictest warning—on COVID-19 vaccines. Commissioner Marty Makary denied those reports on Monday, stating on Bloomberg TV that the FDA has “no plans” to make such a move. This follows an internal memo from Vinay Prasad leaked over Thanksgiving in which the CBER director claimed that “at least” 10 children have died “because of” COVID-19 vaccines. An internal safety review published last week refuted this conclusion, instead concluding that between zero and seven deaths could be linked to the shots.   Pfizer CEO Albert Bourla, for one, is tired of the recent rhetoric from HHS on vaccines and hopes they are “an anomaly” that will be corrected soon. With strong words about the administration’s sentiment on vaccines, Bourla prominsed Pfizer’s continued investment in vaccines despite declining revenue. Pfizer this week lowered its 2026 guidance to $62.5 billion in revenue, missing analyst consensus.  The FDA has also granted several approvals in the past week, to Amgen, Milestone Pharmaceuticals and AstraZeneca and Daiichi Sankyo. USAntibiotics also snagged a greenlight, for Augmentin XR, the first approval to be given under the agency’s new Commissioner’s National Priority Voucher (CNPV) program. Also this week, Johnson & Johnson scored a CNPV ticket—without even having to apply—for its investigational combo of Tecvayli plus Darzalex for relapsed or refractory multiple myeloma after the FDA was impressed by Phase III data.   In ClinicaSpace this week, we highlighted 5 of 2025’s Defining Clinical Wins and The 5 Most Painful Clinical Trial Failures of 2025. This past week provided a few more on each front. In the winner’s circle, Immunome’s desmoid tumor drug and and Kyverna’s CAR T for stiff person syndrome both aced pivotal trials, while Sanofi’s MS drug tolebrutinib and Gilead and Arcus’ TIGIT therapy domvanalimab each failed Phase III tests.   And in BioPharm Executive, we highlight 6 Biotechs That Could Be Big Pharma’s Next M&A Target, and more M&A predictions for 2026.

In this episode presented by AnaptysBio, Jennifer Smith-Parker speaks to Dr. Joe Murray, professor of medicine, Division of Gastroenterology and Hepatology, Department of Internal Medicine, and Department of Immunology, The Mayo Clinic; Marilyn Gellar, CEO, The Celiac Disease Foundation; and Dr. Paul Lizzul, chief medical officer, AnaptysBio, about the unmet need for effective treatments for celiac disease, the limitations of the gluten-free diet and the development of ANB033, a first-in-class CD122 antagonist designed to modulate IL-2/IL-15 signaling.HostJennifer Smith-Parker, Director of Insights, BioSpaceGuestsDr. Joe Murray, Professor of Medicine, Division of Gastroenterology and Hepatology, Department of Internal Medicine and Department of Immunology, The Mayo ClinicMarilyn Geller, CEO, Celiac Disease FoundationDr. Paul Lizzul, Chief Medical Officer, AnaptysBio Disclaimer: The views expressed in this discussion by guests are their own and do not represent those of their organizations.

Pfizer followed up its winning $10 billion bid forobesity startup Metsera by adding another molecule to its obesity cabinet, thisone from Chinese firm YaoPharma in a deal worth up to $1.9 billion licensing deal announced Tuesday. Meanwhile, Wave Life Sciences and Structure Therapeutics may have changed the weight loss landscape with early and mid-stagedata from their candidates that analysts called “very disruptive” and “highlycompetitive,” respectively.  At the FDA, the confusion and instability continuewith the retirement of agency veteran Richard Pazdur just three weeks into hisrole as CDER director. Pazdur’s exit leaves the FDA with just three veteran leaders from one year ago. The CDC similarly made headlines last week as therevamped vaccines advisory committee voted to recommend postponing the hepatitis B vaccination from birth to two months of age for some infants. Meanwhile, the Clinical Trials on Alzheimer’s Disease (CTAD)conference generated excitement in the space as Roche, Eisai and others presentednew data from their respective candidates. The star of the show was Roche’snext-gen antibody trontinemab, which lowered amyloid levels below the threshold of positivity in 92%of treated patients in a mid-stage study. Meanwhile, Eisai isdetermined to buck the losing the trend in the anti-tau space. The company’s etalanetugreduced all measurable forms of MTBR-tau243—a specific biomarker of tau tanglepathology in Alzheimer’s—in a Phase Ib/II trial, according to an oralpresentation at CTAD.  Finally, check outthis week’s deep dives in BioPharm Executive on what pharmas are learning fromthe Chinese biotech sector and how Japan’s pharma industry is setting itssights on the global market.

Momentum is building behind New Approach Methodologies (NAMs) that offer stronger human relevance than traditional animal testing. The FDA issued on December 2 a draft guidance outlining specific product types for which the agency believes six-month non-human primate toxicity testing can be eliminated or reduced.  The guidance followed a proposed agency template for NAMs in April. There is also an initiative called the Validation and Qualification Network, with dozens of partners from regulators, like the FDA and European Commission, to Big Pharmas and CROs, such as Sanofi, Novo Nordisk, GSK and Charles River Laboratories, that had a July meeting. In addition, Reuters reported in September that AI-driven drug discovery picks up as FDA pushes to reduce animal testing.In this episode of Denatured, Jennifer C. Smith-Parker speaks to Stacey Adam, PhD, Vice President of Science Partnerships at the Foundation for the National Institutes of Health and Patrick Smith, Senior Vice President, Translational Science at Certara, to discuss the latest regulatory news and the future for NAMs development.HostJennifer Smith-Parker, Director of Insights, BioSpaceGuestsPatrick Smith, Senior Vice President, Translational Science, CertaraStacey Adam, PhD, Vice President of Science Partnerships, Foundation for the National Institutes of HealthDisclaimer: The views expressed in this discussion by guests are their own and do not represent those of their organizations.

Vinay Prasad’s memo, leaked over the weekend, has sent vaccine makers’ stocks slipping and experts clamoring for more evidence to support the CBER director’s claim that COVID vaccines have led to the deaths of at least 10 children. It’s the latest insult to the vaccine sector from the healthcare administration this year, with other challenges coming on the CDC side, where the recently revamped advisory committee is heading into its next meeting with a brand new chair.  Elsewhere at the FDA, newly promoted Center for Drug Evaluation and Research Director Richard Pazdur has filed the paperwork to retire from the agency just weeks after reportedly reluctantly accepting the position.   Meanwhile, the Alzheimer’s space is buzzing as the 2025 Clinical Trials on Alzheimer’s Disease (CTAD) conference continues in San Diego. Last week saw a mid-stage flop for Johnson & Johnson’s anti-tau candidate and the “definitive” failure of Novo’s GLP-1 semaglutide—which analysts say may actually help uptake of anti-amyloid therapies from Biogen and Eli Lilly. Meanwhile, Roche announced positive results for its latest antibody, putting the pharma back in the game it had once stepped back from in the tumultuous days of Aduhelm.   In the weight loss space, Novo Nordisk revealed ‘competitive’ mid-stage data for its next-gen amycretin, which showed no weight-loss plateau over 36 weeks in patients with type 2 diabetes. Meanwhile, the pricing war for approved and future GLP-1s from Novo and obesity rival Eli Lilly rages on, with Lilly this week announcing another price drop for Zepbound through its LillyDirect self-pay platform.  In BioPharm Executive this week, we review the top venture capital rounds for female-founded biotechs and examine the 2026 biotech market outlook.

Acquisitions have ticked up this month as all eyes were on Pfizer and Novo’s fight over Metsera. The past week saw that deal finalized with Pfizer as the winner plus a handful of other M&A stories, including Merck’s $9.2 billion buyout of Cidara Therapeutics and Johnson & Johnson’s $3 billion bet on Halda Therapeutics. Additionally, a separate bidding war has broken out between Lundbeck and Alkermes for sleep biotech Avadel Pharmaceuticals. Outside of M&A, the FDA dominated headlines again this week. The agency effectively closed the loop on one of the messiest stories of the year—Sarepta’s gene therapy Elevidys—when it formally added a black box warning for serious liver toxicities and risk of death and removed the nonambulatory indication from the label. The agency will also require that the company complete a postmarketing observational study of around 200 patients. This happened just as Richard Pazdur accepted the role of director of the Center for Drug Evaluation and Research (CDER) that was recently vacated by George Tidmarsh amidst conflict with Center for Biologics Evaluation and Research (CBER) Director Vinay Prasad. By all accounts, Pazdur, a 26-year FDA veteran, is a highly respected and thoughtful regulator, and sources told BioSpace that if Prasad tries to supersede Pazdur’s authority, “there will be hell to pay and resignations to be handed in.” In other FDA drama, FDA Commissioner Marty Makary is apparently butting heads with Health Secretary Robert F. Kennedy Jr. Meanwhile, the agency revealed details of its plausible mechanisms pathway teased by Makary earlier in his tenure. Finally, in Biopharm Executive this week, check out our deep dives into GLP-1 pricing. Coming off Trump’s deal with both Eli Lilly and Novo Nordisk to offer their GLP-1s at a lower cost through the president’s new direct-to-consumer platform, Novo CEO Maziar Mike Doustdar announced a sale on its company’s products. Bargain bin pricing like this so early in a drug’s market life is unprecedented. It’s changing the investment calculations for the next-generation of obesity treatments.

A lean funding environment has changed how biotechs approach workforce organization and executive hiring. Searches are becoming more targeted and intentional, and accountability–both for employers and candidates–is high. In this episode of Denatured, Leslie Loveless of Slone Partners describes how biotechs should approach building their executive teams, working with boards and investors to make decisions that will enable growth. She also discusses how the search and hiring processes has changed for both employers and candidates.This episode is presented in partnership with Slone Partners. HostChantal Dresner, Vice President of Marketing, BioSpaceGuestLeslie Loveless, Co-CEO and Managing Partner, Slone PartnersDisclaimer: The views expressed in this discussion by guests are their own and do not represent those of their organizations.

One of biopharma’s most memorable bidding wars finally came to an end on Friday—with Metsera right back in the arms of its original suitor, but with Pfizer paying around $10 billion for the rights to the obesity biotech, a nearly $3 billion increase over its original bid. But while Novo Nordisk may have bowed out of that race, the company still made headlines this past week, with CEO Maziar Mike Doustdar joining Eli Lilly head David Ricks at the White House on Thursday to announce a deal that will see their GLP-1 drugs offered at about $350 per month.   This marks a significant discount to the current list prices of $1086 and $1350 for Lilly’s obesity drug Zepbound and Novo’s comparator Wegovy, respectively. No matter how low they go, however, the GLP-1 leaders can still be undercut by compounders, Steven Grossman, policy and regulatory consultant and author of the FDA Matters blog, told BioSpace this week.   Speaking of Lilly, the Indianapolis-based pharma had a busy week, reporting 20% weight loss in a mid-stage study of its amylin agonist eloralintide that William Blair analysts said “validates [the] amylin agonist class.” Lilly also netted two new partners, inking a $1.2 billion RNAi pact with SangeneBio to target metabolic diseases and licensing a genetic eye disease therapy from MeiraGTx Holdings for up to $475 million.   On the regulatory front, the FDA awarded the second round of priority review vouchers under its new Commissioner’s National Priority Vouchers program. Unlike the first cohort of vouchers, which was announced in October, this group mostly consisted of products already on the market—with the exception of Lilly’s orforglipron.   Finally, BioSpace dives into one the hottest trends in the immunology and inflammation (I&I) space—pipeline-in-a-product. Possibly motivated by blockbuster drugs like AbbVie’s Skyrizi and Rinvoq and Regeneron and Sanofi’s Dupixent, companies are optimizing shots on multiple goals in this lucrative space.

This conversation features insights from Oliver Eden, senior business unit director at Jabil, and Travis Webb, chief scientific officer at PII. Our guests continue their discussion on how autoinjectors and smart technologies can be integrated into clinical trials in a way that isn’t problematic or burdensome for patients, particularly for patients that may not be tech savvy. They discuss that by focusing on patient experience, clinical trials can increase engagement and compliance.This episode is presented in partnership with ⁠PII⁠.Host⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠Lori Ellis⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠, Head of Insights, BioSpaceGuestsOliver Eden, Senior Business Unit Director, JabilTravis Webb, Chief Scientific Officer, PIIDisclaimer: The views expressed in this discussion by guests are their own and do not represent those of their organizations.

Many of the top stories of 2025 are currently being written. We’re on the edge of our keyboards, watching and waiting as Pfizer and Novo Nordisk duke it out over the right to acquire glittery obesity startup Metsera. In the latest development, Pfizer raised its original bid of around $7.27 billion to about $8.1 billion on Monday—only to be usurped again by the indefatigable Novo, which upped its own bid to a cool $10 billion.  Meanwhile, the unprecedented drama in the uppermost ranks of the FDA—another top story of 2025—continues as CDER Director George Tidmarsh exits the agency. Tidmarsh reportedly resigned Sunday after being placed on administrative leave amid an investigation into his “personal conduct” at the agency. On Monday, however, Tidmarsh told Endpoints News that he was “second-guessing” his decision.  Speaking of the FDA, the regulator appears to have done its own 180—on uniQure’s investigational gene therapy for Huntington’s disease, three-year data from which sent the biotech’s stock into the stratosphere just five weeks ago. Despite previous agreements on protocols and statistical analyses, the agency “no longer agrees” that Phase I/II data for AMT-130 are adequate to provide primary evidence for the application, uniQure said, throwing the timeline for the BLA into question.  Another gene therapy player, Sarepta Therapeutics, took a hit this week, as two of its Duchenne muscular dystrophy drugs, Vyondys 53 and Amondys 45, failed a confirmatory trial. Sarepta still plans to file for full approval of the two exon-skipping therapies, however, based on what it called “encouraging trends” in efficacy. Finally, on the genetic medicine front, CBER director Vinay Prasad teased an upcoming paper that will detail the regulator’s thinking and a new approach to gene editing approvals.  On top of all that, Q3 earnings continue to roll in, with Pfizer, Eli Lilly, Vertex, Bristol Myers Squibb, AbbVie, and more reporting results.  One more thing: Have you ever wanted to know more about the inner workings of the Biogen-Eisai Alzheimer’s partnership? Check out this profile on BioSpace 40 under 40 honoree Neena Bitritto-Garg, Eisai alum and current CEO of Ensho Therapeutics.

In this episode of Denatured, guests Dr. Jihye Jang-Lee and Dr. Khanh Courtney discuss how China, historically focused on manufacturing, is increasingly becoming an innovation leader, particularly in pharmaceuticals. Ultimately, balanced strategies involve domestic capacity investments coupled with global collaboration.This episode is presented in partnership with Element Materials Technology.HostsJennifer Smith-Parker, Director of Insights, BioSpaceLori Ellis, Head of Insights, BioSpaceGuestsDr. Jihye Jang-Lee, Director of Technical Services, Element Materials TechnologyDr. Khanh Courtney, Biologics Technical Strategy Manager, Element Materials TechnologyDisclaimer: The views expressed in this discussion by guests are their own and do not represent those of their organizations.

Novartis started the week early with a Sunday afternoon announcement of the acquisition of neuromuscular drug developer Avidity Biosciences for $12B. That’s the second biggest buy of the year after Johnson & Johnson’s January acquisition of Intra-Cellular. The Avidity buy could read through positively to Dyne Therapeutics, as both are aiming to treat neuromuscular ailments with RNA-targeting therapies. Dyne shares have nearly doubled over the past month, jumping approximately 40% after Novartis’ news dropped.  The Avidity deal is the latest in an uptick on the pharma M&A front. Also this week, Eli Lilly doubled down on gene therapy with a pick up of Adverum Biotechnologies and its lead program for wet age-related macular degeneration. And Roche, which last month acquired 89bio in a $3.5 billion deal centered on a MASH candidate, said in its third-quarter earnings call on Thursday that more deals could be in the future. Finally, beyond the big guys, Leerink Partners predicts which small- to mid-cap firms might also be on the hunt for new pipeline goodies.  Following the dealmaking news, Novartis held its earning call on Tuesday. CEO Vas Narasimhan downplayed the deals Pfizer, AstraZeneca and Amgen have made with the White House, saying they don’t address the root of the drug pricing problem President Donald Trump hopes to solve.   On other earnings calls, BioMarin announced plans to divest the hemophilia gene therapy Roctavian. Regeneron faced further questions about Eylea and issues with the Catalent plant that’s been tripping up its regulatory applications. But the company didn’t address last week’s news that it was dropping a CAR T asset picked up from 2seventy bio. These are but two of the latest examples of underperforming assets in the cell and gene therapy space.  BridgeBio had positive news for patients with limb-girdle muscular dystrophy this week after acing a Phase III trial for an investigational substrate supplementation therapy. Analysts predict the asset could be before the FDA later this year or early next.   Finally, with the U.S. government shutdown going on a month, BioSpace takes a look at how the FDA is operating.

In this episode of Denatured, guests Dr. Jihye Jang-Lee and Dr. Khanh Courtney how in light of economic uncertainty, small biotechs can turn to international harmonization standards as a stable template. U.S. tariffs are redefining competitiveness in manufacturing nations like India, China, and other parts of Asia, with China emerging as a global innovator.This episode is presented in partnership with Element Materials Technology.HostsJennifer Smith-Parker, Director of Insights, BioSpaceLori Ellis, Head of Insights, BioSpaceGuestsDr. Jihye Jang-Lee, Director of Technical Services, Element Materials TechnologyDr. Khanh Courtney, Biologics Technical Strategy Manager, Element Materials TechnologyDisclaimer: The views expressed in this discussion by guests are their own and do not represent those of their organizations.

Novo Nordisk dominated the news cycle this week, with more leadership changes as the Novo Foundation replaced the company’s board, which will now be headed by former CEO Lars Rebien Sørensen. Meanwhile, President Donald Trump promised last week that Novo’s Ozempic will cost about $150 when he and Centers for Medicare & Medicaid Services Administrator Mehmet Oz are done negotiating, though Oz clarified that said negotiations have not yet begun. Over in Berlin, the 2025 European Society for Medical Oncology featured presentations from Akeso and Summit Therapeutics on PD-1/VEGF inhibitor ivonescimab in first linenon-small cell lung cancer (NSCLC) and Exelixis’ oralkinase inhibitor zanzalintini in colorectal cancer. In addition to reporting that ivonescimab “significantly improved” progression-free survival in first-line NSCLC, Summit said on a Q3 call Monday that it would submit a regulatory application with the FDA for the drug in second-line EGFR-mutatedNSCLC. In other cancer news, shares of Replimune soared after the FDA accepted its resubmitted biologics license applicationfor RP1 in advanced melanoma, nearly three months after its July rejection. Also on the regulatory front, the FDA named the first nine recipients of its Commissioner’s National Priority Voucherprogram. Winners of the expedited review vouchers include Regeneron, Disc Medicine and Sanofi. The FDA agency also awarded its second-ever platform designation to Krystal Biotech—after granting the first such designation to Sarepta Therapeutics earlier this year for its AAV vector andthen rescinding it after the platform was linked to multiple deaths. Finally, Sandra Retzky, formerly director of the FDA’s Office of Orphan Products Development, joins the lengthy leadership exodus at the agency this year. In BioPharm Executive, BioSpace look at how Johnson & Johnson weathered the erosion of its cornerstone drug Stelara. And is hair loss the new weight loss? Two biopharma companies—Veradermics and Pelage Pharmaceuticals—reeled in large financing rounds for their respective hair loss/regrowth programs. They’re part of an uptick in mega rounds of late, butexperts say it’s not a full biotech comeback just yet.

In this episode of Denatured, guests Dr. Jihye Jang-Lee and Dr. Khanh Courtney will explore how healthcare and pharmaceutical players — from hospitals and universities to manufacturers and investors — are rethinking their strategies amid global supply chain disruption.The conversation highlights both immediate operational responses and long-term structural shifts shaping a more resilient, agile, and locally grounded ecosystem, especially in light of U.S. tariffs.This episode is presented in partnership with Element Materials Technology.HostsJennifer Smith-Parker, Director of Insights, BioSpaceLori Ellis, Head of Insights, BioSpaceGuestsDr. Jihye Jang-Lee, Director of Technical Services, Element Materials TechnologyDr. Khanh Courtney, Biologics Technical Strategy Manager, Element Materials TechnologyDisclaimer: The views expressed in this discussion by guests are their own and do not represent those of their organizations.

This episode continues the discussion with Oliver Eden, senior business unit director at Jabil, and Travis Webb, chief scientific officer at PII, as they dive into the nuance, challenges and opportunities of autoinjectors and combination drug delivery systems.In this conversation they focus on supporting decentralized clinical studies and how connected technology can improve patient compliance, trust and cleaner data processing.This episode is presented in partnership with ⁠PII⁠.Host⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠Lori Ellis⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠, Head of Insights, BioSpaceGuestsOliver Eden, Senior Business Unit Director, JabilTravis Webb, Chief Scientific Officer, PIIDisclaimer: The views expressed in this discussion by guests are their own and do not represent those of their organizations.

Johnson & Johnson kicked off Q3 earnings season on Tuesday with the announcement that it is splitting its orthopedics and medtech operations and that it has yet to reach a drug pricing deal with the White House, though CEO Joaquin Duato did say those discussions are ongoing.  In advance of its Nov. 5 earnings call, Novo Nordisk, under the direction of new CEO Maziar Mike Doustdar, has been busy making moves, doubling down on MASH last week with its $5.2 billion buy of Akero. Then this week, Novo became the latest company to cut cell therapy, following Takeda’s recent exit from the space. Meanwhile, as the government shutdown continues, so too does the chaos at the CDC, where more than 1,000 employees received termination notices last Friday—only for hundreds to be told never mind. At the FDA, however, there is some consistency despite the overhaul, with the agency on track for an average number of approvals this year. And a recent report on breakthrough designations shows that the regulatory award often leads to an FDA greenlight. On the legislative front, the BIOSECURE Act is back, as a slimmed down version passed the Senate last week as part of the defense spending bill. The latest version of BIOSECURE, which is meant to distance American biopharma from Chinese collaborators, doesn’t name specific companies as previous iterations did and must still gain the Senate’s support.

In this discussion, BioSpace’s vice president of marketing ⁠⁠Chantal Dresner⁠⁠ and careers editor ⁠⁠Angela Gabriel⁠⁠ take a look at job market performance in the second quarter of 2025.They discuss biotech and pharma job posting trends, wider U.S. employment data, the new $100,000 H-1B visa fees and more.Want to receive our latest quarterly job market reports as soon as they’re published? ⁠⁠Subscribe⁠⁠ to Career Insider for our job market updates, job trends, career advice and more.

The U.S. government is now in its second week of a shutdown—with the FDA having paused acceptance of all new drug applications for the duration. But it was business as usual at the CDC, which adopted the recent recommendations of its newly revamped advisory committee on chickenpox and COVID-19 vaccines. And another senior leader, National Institute of Allergy and Infectious Diseases Director Jeanne Marrazzo, was fired last week, after filing a whistleblower report.  Across the country, cell and gene therapy leaders arrived in Phoenix for the annual Meeting on the Mesa, as the space remains in a state of flux—with regulatory and M&A momentum being stalled by commercial and market challenges. Takeda, for one, is looking to offload its cell therapy platform after years of hefty investment.  President Donald Trump’s long-awaited tariffs did not hit on Oct. 1 as promised. But in the face of the looming levies, Pfizer signed a drug pricing deal with the White House that provides a three-year exemption. Amgen appears to be climbing onboard as well, announcing that its lipid-lowering drug Repatha will be available at a steep discount. These moves are all well and good for Big Pharma players, but a recent report from CRB reveals most smaller biopharma companies are not planning any investments to offset tariffs.  In our weekly weight loss segment, Skye Bioscience’s cannabinoid receptor 1-targeting candidate nimacimab failed to outpace placebo in reducing body weight but elicited “intriguing synergy” in combination with Novo Nordisk’s Wegovy, according to William Blair analysts. And regulatory documents shed further light on Pfizer’s $4.9 billion takeover of Metsera, in which the New York pharma beat out two higher bidders for the promising obesity startup.  Finally, make sure to check out The 5 Most Powerful Women in Biopharma and BioSpace’s inaugural 40 Under 40, highlighting 40 young leaders who have made an impact on the biopharma industry.

Clinical trials can present many challenges for patients–particularly injections.Oliver Eden, senior business unit director at Jabil, and Travis Webb, chief scientific officer at PII, discuss nuance, challenges and opportunities of autoinjectors and combination drug delivery systems.Additionally, they advocate that drug delivery systems enter the conversation earlier in clinical development stages given interdependencies. This episode is presented in partnership with PII.Host⁠⁠⁠⁠⁠⁠⁠⁠⁠Lori Ellis⁠⁠⁠⁠⁠⁠⁠⁠⁠, Head of Insights, BioSpaceGuestsOliver Eden, Senior Business Unit Director, JabilTravis Webb, Chief Scientific Officer, PIIDisclaimer: The views expressed in this discussion by guests are their own and do not represent those of their organizations.

Genmab closed out a busy third quarter for M&A in biopharma, picking up cancer biotech Merus for $8 billion. This deal—the year’s fifth largest—came just a week after Pfizer acquired rising obesity star Metsera for $4.9 billion. Just seven days later, Metsera made the New York–based pharma look like a genius with mid-stage data for one of the deal’s centerpiece therapies, MET-097i, showing 14% placebo-adjusted weight loss over 28 weeks.After months of tarrying and threats, President Donald Trump announced last week that 100% tariffs would take effect Oct. 1—with broad exceptions for companies that have taken steps to build out their domestic manufacturing footprints. One company that has answered that call—as well as a letter sent by the president to 17 of the largest pharma companies requesting action on his most-favored-nation drug pricing policy—is Pfizer. In a joint oval office announcement with Pfizer CEO Albert Bourla and HHS officials, Trump said Pfizer would offer all new medicines at MFN prices.Meanwhile, over at the FDA, CDER Director George Tidmarsh, who has flown relatively under the radar since taking the post in July, took to LinkedIn to address the subject of relying on surrogate endpoints in drug approvals. In a since deleted post, Tidmarsh called out Aurinia Pharmaceuticals’ lupus drug as an “egregious” example of this phenomenon. CDER’s sister agency, CBER, also made a splash last week, publishing three draft recommendations intended to accelerate the development of cell and gene therapies.Speaking of CGT, maybe the biggest clinical development news of the year emerged from this space last week when uniQure announced that its gene therapy for Huntington’s disease, AMT-130, slowed disease progression by 75% after three years. With these data in hand, uniQure plans to file for FDA approval of the treatment in the first quarter of 2026. If successful, AMT-130 would be the first genetic therapy for the intractable neurodegenerative disease.Finally, biopharma’s glass ceiling just got a little more tightly sealed. Emma Walmsley, the industry’s first female CEO, is stepping down after nine years at GSK, handing the reins to current chief commercial officer, Luke Miels. When Walmsley officially departs on Dec. 31, she will leave Vertex CEO Reshma Kewalramani and incoming Takeda CEO Julie Kim to represent the sisterhood at the highest ranks of the biopharma industry.

The FDA is working to reapprove GSK’s long-dormant drug Wellcovorin (leucovorin) for cerebral folate deficiency, which the agency linked to “developmental delays with autistic features.” This immediately followed a much-anticipated press conference in which President Donald Trump, flanked by Health Secretary Robert F. Kennedy Jr and other healthcare administrators, linked the use of Tylenol during pregnancy to rising rates of autism.  Meanwhile, Pfizer woke us all up Monday with the news that it had acquired breakout obesity rockstar Metsera for $4.9B. The deal should pump new life into Pfizer’s portfolio, which over the last two years has suffered three discontinued assets. Bite-sized deals—or those at or below the $5 billion mark—have defined biopharma recently, with Roche picking up metabolic dysfunction-associated steatohepatitis biotech 89bio for a potential $3.5 billion last week and Novartis putting another $5.7 billion on the line with partner Monte Rosa Therapeutics in a second molecular glue agreement.  Another therapeutic space primed for M&A action is psychedelics. After AbbVie bought Gilgamesh Pharmaceuticals’ lead depression asset for $1.2B last month, BioSpace spoke sought opinions from experts on who might be next to take the plunge. A few potential names included Eli Lilly, Bristol Myers Squibb and Merck.  On the policy front, the CDC’s revamped vaccine advisory committee convened for their first meeting to discuss COVID-19, MMRV and hepatitis B vaccine schedules. Industry watchers who spoke to BioSpace commented on the “lack of knowledge” and dearth of previous experience on the committee. And while the advisors ultimately voted to change the schedule for the MMRV vaccine, it appears unlikely to significantly affect manufacturers’ bottom lines. Finally, in rare disease, Stealth BioTherapeutics secured its long-sought approval for elamipretide—now Forzinity—in Barth syndrome—a disorder that would fall under the purview of the FDA’s new Rare Disease Evidence Principles framework for ultra rare diseases affecting less than 1,000 people in the U.S. And we said “Bye Bye Bluebird,” as the famed gene therapy biotech—which was recently bought out by two private equity firms—returned to its original moniker, Genetix Biotherapeutics.  Lastly, make sure to sign up for Biopharm Executive here for access to a special deep dive into China biopharma.

In this episode, Lori Ellis and Colin Zick spend a little time further discussing some of the points brought up in the Bioprocessing Summit last month. AI is a tool, a powerful one but a tool. Understanding this, they explore the connections between hammers, AI, The Planet of the Apes and monoliths.In the end, it is all about compliance.Host⁠⁠⁠⁠⁠⁠⁠⁠⁠Lori Ellis⁠⁠⁠⁠⁠⁠⁠⁠⁠, Head of Insights, BioSpaceGuestColin Zick, Partner, Foley Hoag LLP Disclaimer: The views expressed in this discussion by guests are their own and do not represent those of their organizations.

A global shift may be underway in biopharma as the White House prepares legislation that would would place restrictions on drugs brought to the U.S. from China, and pharma companies exit the U.K. in droves. President Donald Trump is reportedly writing an executive order that would clamp down on the pharmaceutical industry’s ability to buy new molecules from biotechs based in China, while Sanofi, Merck and more have canceled or suspended investments in the U.K. following a sizeable increase in a mandatory levy in the region. In other business news, Novo Nordisk’s newly appointed CEO Maziar Mike Doustdar hit the ground running, cutting around 9,000 employees and informing those who remained that they would need to return to the office. Novo’s headcount had climbed 81% in five years as its revenue soared—and then fell. The obesity juggernaut has been a key presence at the European Association for the Study of Diabetes’ annual meeting this week, announcing that it would seek FDA approval for a high-dose formulation of Wegovy, and presenting new data for long-acting amylin analog cagrilintide. Meanwhile, Reuters reported that Eli Lilly’s orforglipron could potentially qualify for the FDA’s recently launched Commissioner’s Priority Voucher, which could see the oral obesity candidate approved this year.  The gene therapy space was hit with more bad news as Capsida Biotherapeutics reported the death of a patient being treated with its investigational gene therapy for epileptic disorders. This follows an unfortunate trend in 2025 that has also seen deaths attributed to Sarepta’s approved Duchenne muscular dystrophy gene therapy Elevidys and a monoclonal antibody used for lymphodepletion in a study of Allogene’s CAR T cell therapy cema-cell. According to a new analyst survey, however, doctors are still prescribing Elevidys to ambulatory patients.  Meanwhile, on the regulatory front, FDA Center for Drug Evaluation and Research Director George Tidmarsh reportedly told two separate groups last week that he “would like to get away” from advisory committee meetings, but quickly appeared to walk the comments back in a statement to Endpoints News.  Finally, in BioPharm Executive, BioSpace takes a deep dive into the FDA’s new crackdown on pharmaceutical drug ads, and spotlights Akeso CEO Michelle Xia who built the biotech from a $3 million angel fundraising round to its current $15 billion valuation.

The Make America Healthy Again Commission released itssecond report Tuesday, recommending, among other efforts, an investigation into a possible link between vaccines and the uptick in chronic disease. At a livestreamed MAHA commission meeting, Health Secretary Robert F. Kennedy painted a dire picture of the country’s health, saying the U.S. now has “the highest chronic disease burden of any country in the world.” Looking back to last week, all eyes were on HealthSecretary Robert F. Kennedy Jr.’s appearance before the Senate Finance Committee. The combative showdown amounted to little more than political theater, according to industry watchers, with Kennedy accusing former CDC Director Susan Monarez of lying in an op-ed published in the Wall Street Journal about his alleged request that she approve vaccine advisors’ recommendations in advance of their meeting later this month.Over at the FDA, BioSpace combed through the latest cache of publicized complete response letters (CRLs), including one for Lykos’ MDMA-based therapeutic for post-traumatic stress disorder. Going forward, the agency has promised to release CRLs in real time. The greater transparency could help companies spinning on a carousel of confusion caused by all the recent regulatory change. In other FDA news, we take a deep dive into new expert panels, which some commentators view as one-sided, and into the new rare disease approval framework, which one critic called “all wrapper and no gift.” And in the weight loss space, the FDA debuted a consumer “green list” for GLP-1 ingredients. Meanwhile, at the World Conference on Lung Cancer inBarcelona, several data readouts caught our attention. In particular, Summit Therapeutics released disappointing data for its PD-L1/VEGFa bispecific antibody ivonescimab in Western populations—a finding some analysts said could have readthroughs to Bristol Myers Squibb/BioNTech’s first ever global data readout for its L1/VEGFa bispecific.Finally, in Biopharm Executive this week, check out features on contingent value rights, which have been getting tacked on to biopharma deals more and more, Amgen’s pipeline beyond the obesity drug MariTide and Novartis’ recent deal spree, which included the $1.4B acquisition of Tourmaline on Tuesday.

In this episode, Mike Garrett, CEO of Taconic Biosciences, discusses how preclinical research companies are helping drug developers navigate the current challenging funding environment. He explains that investors now demand more robust translational data showing real potential to impact human health, rather than just basic in vitro results, forcing researchers to generate better evidence packages earlier in development.This episode is presented in partnership with ⁠⁠Taconic Biosciences.Host⁠⁠⁠⁠⁠⁠⁠⁠⁠Lori Ellis⁠⁠⁠⁠⁠⁠⁠⁠⁠, Head of Insights, BioSpaceGuestMike Garret, CEO, Taconic BiosciencesDisclaimer: The views expressed in this discussion by guests are their own and do not represent those of their organizations.

We returned from the Labor Day holiday to a spate of intriguing deals, including two that could surpass $2 billion: Vertex’s new pact with Enlaza for autoimmune disease—which the Casgevy maker hopes could ease conditioning for the sickle cell/beta thalassemia gene therapy—and Novartis’ agreement with Arrowhead for neurodegenerative diseases such as Parkinson’s.  But as we look ahead, Thursday’s Senate Finance Committee will be the focus this week, as Health and Human Services Secretary Robert F. Kennedy Jr. will answer questions after the ousting of CDC Director Susan Monarez. Her departure is reportedly linked to changes to the regulation of COVID-19 vaccines, for which the FDA last week issued restricted approvals to Moderna, Pfizer/BioNTech and Novavax and rescinded the emergency use authorizations. The next ACIP meeting—where COVID-19 vaccines will be on the agenda—is set for Sept. 18 and 19.   In the weight loss arena, Novo Nordisk presented results from a real-world study this weekend at the European Society of Cardiology Congress in Madrid showing that Wegovy cut the risk of heart attack, stroke or death by 57% compared to Eli Lilly’s tirzepatide in people with obesity and cardiovascular disease. The company also continues to throw money into the space, last week inking a $550 million deal with Replicate for RNA-based treatments for obesity and diabetes. Meanwhile, Lilly is dropping studies of one oral obesity candidate as another nears a regulatory filing. Finally, the FDA greenlit the first GLP-1 generic for obesity.   We also discuss reactions to the FDA’s new guidance on radiopharma drug development, four recent approvals for rare diseases, and everything you ever wanted to know about SPACs.

Eli Lilly posted data Tuesday from a second Phase III trial of its oral weight loss therapy orforglipron, providing the company with all it needs to head to the FDA with a new drug application. For more in-depth discussion on the oral weight-loss space, check out a special episode of The Weekly. And stay tuned to BioSpace for more unique coverage of this market as we learn which investigational assets will make it across the regulatory finish line and which will join the weight-loss wasteland. On the business side of biopharma, AbbVie turned back to neuropsychiatric therapies this week, acquiring partner Gilgamesh Pharmaceuticals’ lead depression candidate for $1.2 billion. The deal is a display of resilience for AbbVie in the neuropsychiatric space as well, after the stunning failure last November of schizophrenia asset emraclidine—picked up in its nearly $9 billion acquisition of Cerevel Therapeutics. And is the cooler late summer weather thawing the IPO market? Neuropsych-focused LB Pharma revealed in an SEC filing on Friday that it plans to take the plunge, ending a six-month stalemate in biotech IPOs.  Finally, the Department of Health and Human Services (HHS) has been busy this past week. On Monday, the CDC named Retsef Levi—a known vaccine critic—to head the agency’s COVID-19 immunization working group, just as reports surfaced that the Trump administration could be “within months” of banning the COVID-19 vaccine in the U.S. Meanwhile, the FDA issued new draft guidance for cancer drug developers that prioritizes the use of survival data, and the agency began publishing drug-related adverse event reports daily.   HHS itself has also been the subject of recent headlines, announcing that it will no longer recognize employee unions, and last week, hundreds of HHS staffers penned an open letter calling on Secretary Kennedy to tone down what they called “dangerous and deceitful statements” that have fostered distrust against federal health workers, exposing them to physical harm. This followed the Aug. 8 shooting at CDC headquarters in Atlanta.   Finally, multinational pharmaceutical companies spent more than $48 billion on partnerships with China in the first half of 2025 alone, according to a new report from IQVIA—more than in all of 2024.

Oral therapies are projected to account for 25% of the anti-obesity medication market by 2030—but first returns have largely disappointed. This month, shares of both Eli Lilly and Viking Therapeutics took a hit as investors reacted negatively to highly anticipated Phase III and Phase II results for their respective candidates.While the 9.1% placebo-adjusted weight loss generated by Lilly’s orforglipron over 72 weeks was an efficacy miss by most analyst accounts, tolerability tripped up oral VK2735’s otherwise best-case efficacy scenario—10.9% weight loss after just 13 weeks. These murky results have left observers wondering, just how game-changing these pills will be and which ones will be most effective?In such a hot space, Lilly’s and Viking’s results—which follow Novo Nordisk’s new drug application for an oral form of Wegovy in May—are only the tip of the iceberg. Stay tuned to BioSpace for further in-depth coverage of the space as we learn which investigational assets will make it across the regulatory finish line and which will join the weight-loss wasteland.

While AI represents a significant advancement in efficiency for early-stage drug discovery, it won't dramatically change the 90% clinical trial failure rate. Most failures stem from fundamental gaps in biological understanding rather then the processes where AI is able to have the most impact.The discussion highlights AI's strengths and ability to reduce preclinical costs. However, they caution that AI faces significant limitations in predicting complex biological properties like toxicity due to insufficient data, and regulatory acceptance of AI-only safety assessments remains unlikely, meaning traditional clinical trials will continue to be necessary.This episode is presented in partnership with ⁠Cresset⁠.Host⁠⁠⁠⁠⁠⁠⁠⁠Lori Ellis⁠⁠⁠⁠⁠⁠⁠⁠, Head of Insights, BioSpaceGuests⁠Mutlu Dogruel⁠, VP of AI, Cresset⁠Mark Mackey⁠, CSO, CressetDisclaimer: The views expressed in this discussion by guests are their own and do not represent those of their organizations.

Three months after taking heat over an error-riddled first report, Health Secretary Robert F. Kennedy’s Make America Healthy Again Commission was due to submit a new strategy report to President Donald Trump last week. While the report is delayed—for disputed reasons— Politico obtained a draft copy, which homes in on creating a new vaccine framework and streamlining access to investigational drugs, specifically for children. The new strategy also touches on pharma lobbying, with plans to address alleged conflicts of interest within HHS—which, incidentally, a new JAMA study found have dropped dramatically over the past 25 years. In other HHS news, Kennedy is reviving the Task Force on Safer Childhood Vaccines—which has been dormant for 30 years, and the secretary voiced his support of mRNA vaccines for cancer.   Meanwhile, weight loss continues to coral the headlines. Viking Therapeutics reported greater than 12% weight loss after 13 weeks for its obesity pill VK2735, but tolerability tanked the biotech’s stock. As Viking, Eli Lilly and Novo Nordisk begin to report data from these oral options, manufacturing comes into focus, and Novo’s pending approval of an oral form of Wegovy specifically puts the spotlight on the variability of high-dose peptides.  Speaking of Novo, the Danish pharma appears to be sucking in some oxygen after a difficult run. Last week, the FDA approved Wegovy as just the second drug for metabolic dysfunction-associated steatohepatitis (MASH), and on Monday, Novo teamed up with GoodRx to offer Wegovy and Ozempic and $499 per month if patients skip insurance. Not to be outdone, fellow weight loss warrior Lilly announced a $1.3 billion partnership with AI/ML startup Superluminal Medicines to advance new small molecules for cardiometabolic diseases and obesity. Lilly has also been active on the policy front, saying that it will soon announce higher prices for its drugs in markets like Europe in an effort to “align prices across developed countries.”  Always a contentious topic, pricing plays a key role in why the pharmaceutical industry fails to command the same respect as steelworkers or other all-American pursuits. Also in BioPharm Executive this week, BioSpace takes a deep dive into how the Trump administration is using Most Favored Nation pricing to target pharma companies and pursue a broader trade war.

In this Denatured discussion, the conversation revolves around unpacking the patent policy changes that could make or break biotech companies. Guests Aaron Cummings and Anne Li of Brownstein Hyatt Farber Schreck highlight critical issues posed by the Patent Office including recent changes to the Inter Partes Review (IPR) such as discretionary denial and a proposed patent property tax.Cummings and Li also discuss how biopharma can work with the administration as changes are being proposed and evaluated and stress the importance of individual advocacy and engagement.Host⁠⁠⁠⁠⁠⁠⁠⁠Lori Ellis⁠⁠⁠⁠⁠⁠⁠⁠, Head of Insights, BioSpaceGuestsAaron CummingsAnne Elise Herold Li, Shareholder, Brownstein Hyatt Farber SchreckDisclaimer: The views expressed in this discussion by guests are their own and do not represent those of their organizations.

Vinay Prasad is back at the FDA as chief of the FDA’s Center for Biologics Evaluation and Research. Prasad’s return—which hit the news wires Saturday morning—came just 10 days after his unexpected exit on July 29, following blowback over the saga involving Sarepta Therapeutics’ Duchenne muscular dystrophy gene therapy Elevidys, and a campaign by conversative personalities to oust the outspoken physician.Elsewhere in the government, Gray Delany, former head of HHS’ and President Donald Trump’s Make America Healthy Again agenda, has been fired—days after Health and Human Services Secretary Robert F. Kennedy Jr. axed 22 mRNA vaccine contracts under the Biomedical Advanced Research and Development Authority. Delany’s ouster came after he allegedly butted heads with other agency officials over how announcements were made, according to reporting from Endpoints News. The mRNA contract cuts, meanwhile, have sparked criticism from the scientific community and concern that the growing anti-mRNA sentiment could also have a negative effect on research for cancer therapeutics.In somewhat related news, the Annals of Internal Medicine is refusing to retract a large-scale study it ran in July that pointed to the lack of an association between childhood aluminum exposure through vaccination and chronic conditions, despite Kennedy’s criticism. In an op-ed published earlier this month, the Health Secretary took issue with the study’s design.In obesity news, biopharma darling Eli Lilly suffered a rare chink in its normally impenetrable GLP-1 armor. The first Phase III trial for oral weight loss therapy orforglipron read out last week, and the results were underwhelming, at least to Wall Street. Truist Securities wrote that the data support approval but “leaves room for competition.” This is good news for a whole host of companies, including Roche, Viking, Terns and Rhythm Pharmaceuticals, who all have weight loss pills at various stages of development.And in gene therapy, the hits just keep coming. The FDA has limited the use of bluebird bio’s gene therapy Skysona to patients with cerebral adrenoleukodystrophy for whom no other therapies or stem cell donors are available, due to a heightened risk of blood cancers. This follows safety issues in gene therapy trials experienced by Allogene, and, of course, Sarepta Therapeutics. This week in ClinicaSpace, Dan Samorodnitsky explored the future of AAV technology. And in BioPharm Executive, Annalee Armstrong sat down with Chris Anzalone, CEO of Arrowhead Pharmaceuticals—Sarepta’s key strategic partner—to learn how his company has been weathering the storm.

In this thought-provoking episode, Cresset Group’s CSO, Mark Mackey, and VP of AI, Mutlu Dogruel, dissect the emerging geopolitical battle for AI supremacy, revealing how China's DeepSeek model fundamentally disrupted the AI landscape by achieving GPT-4 level performance for just $6 million versus OpenAI's reported $100+ million investment. The discussion exposes a critical divide in global AI governance: Europe's "regulatory fortress" approach with the AI Act creates strict but vague rules that leave companies navigating a complex maze, while the US employs a "Wild West" philosophy of sector-specific oversight and red-teaming strategies. In the race for global AI dominance, China is leaping ahead of the US and the EU.This episode is presented in partnership with Cresset.Host⁠⁠⁠⁠⁠⁠⁠Lori Ellis⁠⁠⁠⁠⁠⁠⁠, Head of Insights, BioSpaceGuestsMutlu Dogruel, VP of AI, CressetMark Mackey, CSO, CressetDisclaimer: The views expressed in this discussion by guests are their own and do not represent those of their organizations.

Center for Drug Evaluation and Research Head George Tidmarsh will oversee the Center for Biologics Evaluation and Research on an acting basis after Vinay Prasad’s abrupt departure hours after the recording of last week’s episode of The Weekly. While the situation is being billed as temporary, rumors are swirling that structural changes may be afoot at the FDA with Commissioner Marty Makary looking for better drug, biologic alignment.Meanwhile, the agency has come under criticism for another recent decision—one apparently driven by CDER’s Oncology Center of Excellence director Richard Pazdur: the rejection of Replimmune’s advanced melanoma drug, RP1. According to reporting by multiple outlets, Pazdur opposed the consensus opinion of CBER staff to approve the drug. The research team behind Replimmune’s Phase III study penned an open letter to the FDA on Friday responding to the issues outlined in the agency’s complete response letter. Meanwhile, the FDA’s Sarepta saga continues, highlighting “unprecedented” FDA leaks and a veritable communications disaster.On the business side of biopharma, Q2 earnings continue to unfurl, with Pfizer, Vertex, BioNTech, Merck and Moderna all reporting this past week. Merck’s $3 billion savings push has claimed 6,000 jobs, contributing to a brutal July that saw the entire biopharma industry axe 7,900 employees, a 487% year-over-year increase, based on BioSpace tallies. Meanwhile, Pfizer CEO Albert Bourla confirmed he has been in direct contact with President Donald Trump to negotiate a path forward on Most Favored Nation drug pricing after the president sent letters to 17 Big Pharma companies—and posted on his Truth Social platform—asking them to comply with the policy within 60 days or face potential unspecified consequences.In other policy news, the Centers for Medicare and Medicaid Services is reportedly considering coverage of GLP-1 drugs for weight management and obesity—reviving a Biden era proposal the Trump admin scrapped earlier this year.Finally, in BioPharm Executive this week, we have a special report on the situation in China as international drugmakers swoop into the region to find new drug candidates, while other companies build their therapeutic farm systems from incubators and venture arms. And check out BioSpace’s brand new Manufacturing Brief, where we bring you the latest news and analysis in the area of biopharma manufacturing, starting with a feature on how to make cell and gene therapies commercially viable.

While AI excels at repetitive tasks like triaging medical information calls, document retrieval, and adverse event detection, the industry must address valid concerns from physicians (41% are excited but concerned, according to AMA studies) and patients who distrust AI systems. In this episode, IQVIA’s Louise Molloy advocates for complete transparency, including clear disclaimers when AI generates or supports responses, arguing that the industry serves as "custodians, not owners" of patient data with ethical responsibilities.This episode is presented in partnership with ⁠IQVIA⁠.Host⁠⁠⁠⁠⁠⁠⁠Lori Ellis⁠⁠⁠⁠⁠⁠⁠, Head of Insights, BioSpaceGuestLouise Molloy, Associate Director Medical Information & Pharmacovigilance, IQVIADisclaimer: The views expressed in this discussion by guests are their own and do not represent those of their organizations.

The Sarepta saga continued into another week as the FDA recommended that the voluntary hold on the company’s Duchenne muscular dystrophy gene therapy be lifted for ambulatory patients, after determining that the death of an 8-year-old Brazilian Duchenne patient who had received Elevidys’ was not caused by the drug.  Sarepta’s stock has swung wildly and its transparency questioned after it elected not to reveal the death of a third patient—a participant in a trial of a gene therapy for limb girdle muscular dystrophy—during a business update last week.    Speaking of entities—or individuals—who have trouble staying out of the news, Health Secretary Robert F. Kennedy Jr. plans to dissolve the U.S. Preventive Services Task Force because it is “too woke.” Also on Monday, Kennedy addressed what he called the “broken” vaccine injury compensation program. Without offering details, he vowed to “fix” the U.S.’s VICP and return it to its “original Congressional intent.”   On the business side of the biopharma house, Q2 earnings are in full swing, with AstraZeneca announcing estimate-beating numbers and CEO Pascal Soriot saying the world “needs to share” in global pharma R&D, while Merck cut $3 billion to support an aggressive launch schedule. Meanwhile, a week ahead of its own earnings report, Novo Nordisk named a new CEO and lowered its 2025 sales guidance for the second time this year.   In clinical development, the Alzheimer’s Association Annual Conference is underway in Toronto, with Roche’s trontinemab the standout so far. In a Phase Ib/IIa trial, the next-gen anti-amyloid antibody rapidly cleared amyloid from the brains of patients with Alzheimer’s disease after just seven months—besting the 18-month timeframe for Biogen and Eisai’s Leqembi and Eli Lilly’s Kisunla. While Leqembi and Kisunla have shown some progress is slowing down the progression of Alzheimer’s, their effect size is modest and they don’t work for all patients—leaving plenty of room for symptomatic treatments, such as those being developed by Bristol Myers Squibb and Acadia Pharmaceuticals. The space is gearing up for several readouts, for both symptomatic and disease-modifying therapies alike.   And in BioPharm Executive this week, we dig into the top VC rounds so far this year and highlight a few scrappy biotechs walking the solo road.

Just a few weeks ago, it seemed like Sarepta had weathered a spate of bad news, after two patients died from liver injuries from its Duchenne muscular dystrophy gene therapy Elevidys. Then came news of a third patient death. Last Wednesday, the company announced a major restructuring and 500-person layoff.  Then, in just a few days time, Sarepta Therapeutics went from enjoying a notable stock bump in response to that corporate update to its lowest price in nearly 10 years as it halts shipments of Elevidys. In addition to requesting the shipment hold, the FDA revoked the company’s technology platform designation and paused all clinical trials for Sarepta’s limb-girdle muscular dystrophy (LGMD) gene therapy. The turmoil was set in motion by media reports that a patient who received the LGMD treatment had died—a fact the company chose not to disclose during an investor call. In other news, the FDA’s Center for Drug Evaluation and Research gets a new director in biotech veteran George Tidmarsh, also an adjunct professor of pediatrics and neonatology at Stanford University’s School of Medicine. Tidmarsh enters the agency at a time of mass layoffs as well as voluntary departures. Meanwhile, Replimmune and Roche suffer FDA rejections as therapies from Otsuka/Lundbeck and GSK fail to earn adcomm support, as the bar for acceptable controls and demonstrations of efficacy continue to change under FDA commissioner Marty Makary and CBER director Vinay Prasad.  Finally, Big Pharmas continue to pump billions into U.S. manufacturing, with Biogen and AstraZeneca joining the list of companies to have made such pledges, pledging $2 billion and $50 billion, respectively. These latest announcements come as President Donald Trump reiterates that pharma-specific tariffs of up to 200% could come as soon as Aug. 1.

This episode continues the discussion regarding the rapid evolution of mRNA technologies since COVID-19. Guests discuss the improvements that have occurred within just a few years, which are making these therapies more reliable, cost-effective and viable for personalized cancer, rare disease chronic disease treatment.Today’s episode is sponsored by Eclipsebio. From AI-ready datasets to sequencing validation, they drive RNA success.Explore their solutions at ⁠https://eclipsebio.com/⁠.Host⁠⁠⁠⁠⁠⁠Lori Ellis⁠⁠⁠⁠⁠⁠, Head of Insights, BioSpaceGuests⁠Andy Geall⁠, Co-founder and Chief Development Officer, Replicate Bioscience; Chair of the Board, Alliance for mRNA Medicines⁠Pad Chivukula⁠, Co-founder, CSO & COO, Arcturus TherapeuticsDisclaimer: The views expressed in this discussion by guests are their own and do not represent those of their organizations.

This episode focuses on how AI is transforming pharmacovigilance (PV) on a global scale, particularly focusing on the evolving role of local qualified persons for pharmacovigilance in the EU.This episode is presented in partnership with IQVIA.Host⁠⁠⁠⁠⁠⁠Lori Ellis⁠⁠⁠⁠⁠⁠, Head of Insights, BioSpaceGuestAna Pedro Jesuíno, Global Head Local QPPV Network, IQVIADisclaimer: The views expressed in this discussion by guests are their own and do not represent those of their organizations.

Up to 3,500 FDA staffers received their final walking papers Monday after the U.S. Supreme Court found last week that the government is “likely to succeed” in arguing that its overhaul of HHS is “lawful.” Meanwhile, FDA Commissioner Marty Makary floated policy changes for the agency, including a proposal to lower prescription drug user fees for the next iteration of the program, and one to offer speedier reviews to companies willing to lower the cost of their drugs.  Last week, the regulator opened its cache of complete response letters (CRLs), offering transparency into the rationale behind more than 200 recent rejections for ultimately approved therapies, including those for Eli Lilly’s Alzheimer’s drug Kisunla and Sarepta’s Duchenne muscular dystrophy (DMD) treatment Vyondys 53. The FDA did not, however, release the CRLs for two new rejections: those of therapies from Ultragenyx and Capricor Therapeutics in Sanfilippo syndrome type A and cardiomyopathy associated with DMD, respectively. It was an especially rough week for Ultragenyx, which also, along with partner Mereo BioPharma, released seemingly negative Phase II/III data for their osteogenesis imperfecta therapy.  On a more positive note, two bustling therapeutic spaces continue to see positive data. In obesity, Hengrui Pharma’s Kailera Therapeutics–partnered dual GLP-1/GIP receptor agonist elicited 17.7% average weight loss in a pivotal Chinese trial. And the psychedelic therapeutics space is again generating excitement with two recent positive readouts in treatment-resistant depression. BioSpace took a deep dive into the market reaction to these readouts for Compass Pathways and Beckley Psytech and atai Life Sciences, and what exactly investors are looking for in a successful psychedelic therapy.  Finally, we examine the progress of AI biotech unicorns and kick off our series on women in biopharma with profiles on Mayo Venture Partner Audrey Greenberg and the all-female CEO/R&D tandem at Acadia Pharmaceuticals.

In this discussion, BioSpace’s vice president of marketing ⁠⁠Chantal Dresner⁠⁠ and careers editor ⁠⁠Angela Gabriel⁠⁠ take a look at job market performance in the second quarter of 2025. They discuss job posting trends, application rates and the most significant layoffs of 2025 so far, plus wider trends impacting biopharma including Massachusetts’ $30M tax incentives and factors affecting California’s success.  Want to receive our latest quarterly job market reports as soon as they’re published? ⁠⁠Subscribe⁠⁠ to Career Insider for our job market updates, job trends, career advice and more.

This discussion focuses on how mRNA and self-replicating RNA (srRNA) technologies are expanding far beyond COVID vaccines into revolutionary therapeutic applications for cancer and rare diseases. It is clear that mRNA therapeutics offer three major application areas: infectious disease vaccines, therapeutic vaccines for oncology, and protein replacement for monogenic rare diseases.Today’s episode is sponsored by Eclipsebio. From AI-ready datasets to sequencing validation, they drive RNA success. Explore their solutions at https://eclipsebio.com/.Host⁠⁠⁠⁠⁠Lori Ellis⁠⁠⁠⁠⁠, Head of Insights, BioSpaceGuestsAndy Geall, Co-founder and Chief Development Officer, Replicate Bioscience; Chair of the Board, Alliance for mRNA MedicinesPad Chivukula, Co-founder, CSO & COO, Arcturus TherapeuticsDisclaimer: The views expressed in this discussion by guests are their own and do not represent those of their organizations.

While most of the U.S. was celebrating the 4th of July holiday, President Donald Trump was busy signing the One Big, Beautiful Bill into law. This wide-ranging tax law has a few implications for the biopharma industry, including expanded IRA exemptions for orphan drugs.  Looking ahead to the second half of 2025, BioSpace reviews some of the upcoming catalysts highlighted by Jefferies’ “Halftime Show” report,including a highly anticipated Phase III readout for Eli Lilly’s oral obesity candidate orforglipron and an eye on rare disease decisions under the “new” FDA.  Speaking of FDA decisions, this week kicked off with a surprise approval—that of KalVista’s Pharmaceuticals’ Ekterly for hereditary angioedema. The road to approval for Ekterly was not a smooth one, after the FDA delayed its target action date and Endpoints News reported that FDA Commissioner Marty Makary tried to have the application rejected.  More regulatory controversy is afoot as Health Secretary Robert F. Kennedy Jr. is facing yet another lawsuit. A group of medical organizations have sued Kennedy and other health leaders in an attempt to reverse Kennedy’s recent decision to remove COVID-19 shots from the routine immunization guidelines for healthy children and healthy pregnant women. In other vaccine news, Kennedy endorsed the expanded use of RSV vaccines for people 50 through 59 years old who are at risk of severe disease—following the recommendation of the CDC vaccine advisory committee he turfed last month.  This seeming reversal of sentiment largely mirrors the Secretary’s massive HHS overhaul, which has already seen several of these layoffs reversed. In ClinicaSpace this week, we take a deep dive into the numbers. Also in ClinicaSpace, we feature four therapies hanging tough in a troubled TIGIT space that has seen several companies burn billions of dollars on failed assets. And BrainStorm Cell Therapeutics is back in the news after signaling support for a Citizens’ Petition submitted to the FDA requesting the approval of its cell therapy NurOwn, whose Biologics License Application was withdrawn in 2023.  Finally, in BioPharm Executive, we take a deep dive into the burgeoning longevity space and unpack the short-lived marriage between Novo Nordisk and Hims & Hers Health.

The U.S. Supreme Court closed out its session before breaking for summer on Friday, ruling that decisions around coverage of HIV PrEP drugs should be left up to the U.S. Preventive Services Task Force. The ruling thwarted a lawsuit brought by a group of insurance providers who challenged the Affordable Care Act’s requirement that they cover preventive medicines, such as HIV drugs, recommended by the task force. The high court also determined that members of this task force can be removed at will by HHS Secretary Robert F. Kennedy Jr.Speaking of HHS committees from which members have been removed at will by Kennedy, the CDC’s revamped Advisory Committee on Immunization Practices convened last week for its first meeting since the roster overhaul, where a vote on Merck’s recently approved RSV-targeting monoclonal antibody Enflonsia and a discussion around the inclusion of preservative thimerosal in influenza vaccines was on the docket, but an expected vote on Moderna’s mRNA-based RSV shot mRESVIA was not.Also dominating the headlines on Friday was the FDA’s decision to remove the Risk Evaluation and Mitigation Strategies (REMS) program from the six approved CAR T therapies it was applied to. Analysts and patient groups lauded the move, which is expected to potentially double access to these life-saving therapies, saying it reflects “thoughtful consideration of real-world evidence” and “regulatory trust.”Willliam Blair noted that it could be a “positive signal” to companies developing CAR T therapies for autoimmune diseases, which now includes AbbVie. In an all-cash buyout on Monday, the Illinois-based pharma dropped $1.2 billion for Capstan Therapeutics and its in vivo edited CAR T therapy for B cell–mediated autoimmune diseases. M&A has been on an uptick of late, and private equity companies—such as those that snapped up bluebird bio—are also getting in on the game.Finally, after Lykos Therapeutics’ high profile failure last summer, the psychedelics space is heating up once again. While a Phase III readout of Compass Pathways’ psilocybin drug last week in treatment-resistant depression received a muted reaction from investors, the response to a Phase II readout for Beckley Psytech and atai Life Sciences’ intranasal psychedelic was more positive.

In this episode of Denatured, Archana Hegde from IQVIA discusses the practical challenges faced by pharmacovigilance (PV) professionals as they navigate the FDA's first draft guidance for AI in drug development, published in January 2025. Hegde explains that the vagueness of the current framework is like “a recipe with no pictures and mystery ingredients." During the discussion, she highlights areas of existing confusion.This episode is presented in partnership with IQVIA.Host⁠⁠⁠⁠⁠⁠Lori Ellis⁠⁠⁠⁠⁠⁠, Head of Insights, BioSpaceGuestArchana Hegde, Senior Director, PV Systems & Innovations, IQVIADisclaimer: The views expressed in this discussion by guests are their own and do not represent those of their organizations.

Robert F. Kennedy Jr. testified in front of the House Committee on Energy and Commerce Tuesday, where Democrats confronted the health secretary on hot button issues ranging from his recent overhaul over the CDC’s Advisory Committee on Immunization Practices (ACIP) committee, Kennedy’s recently published—and error-ridden—MAHA report, and his threat to ban government scientists from publishing in certain medical journals.  Meanwhile, at the FDA, the mass exodus of senior leadership continues. On Monday, Jacqueline Corrigan-Curay, acting head of the Center for Drug Evaluation and Research (CDER), announced her retirement as of July. This follows the ouster of Nicole Verdun—the wildly popular director of the FDA’s Office of Therapeutic Products—and her deputy, Rachael Anatol. Their involuntary departure sent shock waves through the biopharma industry, as Verdun had been considered a stabilizing force at the rapidly reshaping agency.  Speaking of the revamped ACIP, the new panel will meet for the first time Wednesday and Thursday to discuss COVID-19 vaccine safety, maternal and pediatric RSV vaccines and more, as experts question the experience and anti-vaccine views of some of Kennedy’s recently appointed members and others express concern about the potential politicization of the committee.   On the clinical front, Eli Lilly, Novo Nordisk and others presented new data from their next-generation obesity programs at the American Diabetes Association’s 85th Scientific Congress. After failing to impress investors—and meet its own high expectations—with CagriSema, Novo sought to reassure investors by touting a safety profile “in line with the GLP1-RA class,” and Eli Lilly reported that bimagrumab, when used alongside Novo’s Wegovy, led to additional weight loss while also preserving muscle mass.  Finally, we recap BIO2025, where Jef Akst, Lori Ellis and Heather McKenzie moderated panels on cell and gene therapy, cybersecurity and AI, and accelerating market entry for rare disease treatments. Relevant to the latter discussion, congressional Republicans dropped the Orphan Cures Act from their version of President Donald Trump’s “One Big Beautiful Bill Act,” and congresspeople, including Rep. Gus Bilirakis (R-Fla.) at Tuesday’s hearing asked Kennedy to commit to supporting the priority review program for rare pediatric diseases, which expired at the end of last year.

Lori Ellis, head of insights at BioSpace, discusses some of the recent events and topics that are buzzing around BIO and DIA in 2025 with Rich Daly, CEO of Catalyst Pharmaceuticals, Peter Ronco, CEO of Emmes Corporation, and Phil Vanek, founder of Redline Bio Advisors. They address funding, the partnering market, AI, and also the recent FDA and CGT roundtable discussion.HostLori Ellis, Head of Insights, BioSpaceGuests⁠⁠Peter Ronco⁠⁠, CEO, Emmes⁠Phil Vanek⁠⁠, Founder, Redline Bio Advisors; Chief Commercial Officer, ISCT; Entrepreneur in Residence, Georgetown University School of MedicineRichard Daly, President & Chief Executive Officer, Catalyst PharmaceuticalsDisclaimer: The views expressed in this discussion by guests are their own and do not represent those of their organizations.

This week, BioSpace is at 50% power as Heather McKenzie and Jef Akst are off attending this year’s BIO Conference in Boston. The half-team discusses this week’s biggest news: the death of another patient who took Sarepta’s Duchenne muscular dystrophy gene therapy Elevidys. The patient was a non-ambulatory teenager who experienced acute liver failure after receiving the gene therapy, which is the same cause of death for an Elevidys patient reported in March. Sarepta announced that it was halting treatments to non-ambulatory patients and on a media call discussed new steps in its therapeutic protocol for preventing further liver injuries.  Elsewhere, mergers and acquisitions are surging across biopharma. Eli Lilly picked up the gene editing company Verve Therapeutics for $1.3 billion, which helped bolster the gene editing space —particularly after Sarepta’s report of the death. Supernus bought Sage Therapeutics for $795 million, five months after Sage rejected a smaller offer from Biogen.  BioNTech also got in on the dealmaking, buying its German rival—scientifically and in the courtroom—CureVac for about $1.25 billion. The deal seemed focused mostly on CureVac’s early-stage cancer immunotherapy pipeline, but analysts were otherwise left scratching their heads on what BioNTech was getting for its money.  Last week on The Weekly the team discussed the sudden dismissal of the CDC’s entire ACIP committee, and this week we have a new slate of members. The eight people replacing the 17 members that were removed last week include allies of HHS Secretary Robert F. Kennedy Jr., many of whom are vaccine skeptics who seem to share his skeptical view of vaccination in general.

This week the BioSpace team was sent into an after-hours scramble by the news that Health and Human Services Secretary Robert F. Kennedy Jr. had fired the remaining members of the CDC’s Advisory Committee on Immunization Practices. The secretary argued in an op-ed that accompanied the announcement that the “clean sweep” was necessary to “reestablish public confidence in vaccine science.” The move came amid heightening rhetoric from Kennedy about the safety of vaccines and sparked concerns that the Secretary will replace the committee with people who sympathize with his anti-vaccine positions. On the drug development front, Metsera once again made waves with new data for an amylin drug candidate showing weight loss of 8.4% at just 36 days. The drug could be a major competitor for Novo Nordisk’s Cagrisema, which has failed to meet the sky-high expectations of investors despite being the leading amylin candidate in the space. And cell and gene therapy was back in the spotlight last week when the FDA held a round table discussion with industry leaders aimed at eliminating barriers to approval for these complex medicines. This support from the FDA came as Sarepta became the first company to receive a platform technology designation intended to streamline future gene therapy reviews and create predictability for new drug applications created using the same technologies.Over in Biopharm Executive, BioSpace looks at right of first negotiation deals in light of Sanofi’s deal to buy Vigil Neuroscience. How often do these agreements turn into M&A? After some analysis, Jefferies found that a ROFN is not a golden ticket to a buyout.

In a dynamic regulatory environment, IQVIA’s Michelle Gyzen suggests that AI may be the best and only way to keep with changes that are happening daily–and sometimes hourly.In this discussion Lori and Michelle touch on the governance frameworks for cybersecurity, risk, and how AI transformation and integration is evolving to help regulatory professionals navigate the speed and complexity of global requirements.Host⁠⁠⁠⁠⁠Lori Ellis⁠⁠⁠⁠⁠, Head of Insights, BioSpaceGuestMichelle Gyzen, Sr. Director, Strategic Regulatory Solutions; Head of Regulatory Services Innovation & Technology, IQVIA

The words of the week so far in biopharma are “deals” and “cancer”—or, more specifically, money being invested in cancer and other key therapeutic areas. With the American Society of Clinical Oncology’s annual conference underway in Chicago, Bristol Myers Squibb got in the PD-1/PD-L1xVEGF game, paying potentially more than $11 billion to co-develop BioNTech’s solid tumor bispecific BNT327. Elsewhere, Sanofi nabbed the year’s second-biggest buyout, picking up Blueprint for $9.5 billion, expanding its rare disease portfolio. And Regeneron plunked down up to $2 billion to license a dual GLP-1/GIP receptor agonist from Chinese biopharma Hansoh Pharmaceuticals Group.  Back in Chicago, presentations by AstraZeneca, Gilead and Amgen drew rave reviews from investor analysts, while Pfizer and Arvinas elaborated on mixed data from a PROTAC that showed positive results in only a subsection of breast cancer patients, failing to impress Wall Street. Meanwhile, Bicara’s solid survival stats in head and neck cancer weren’t enough to clear the high bar set by rival Merus. At the meeting, BioSpace’s own Dan Samorodnitsky sat down with Jazz Pharmaceuticals’ CMO Rob Iannone to discuss the company’s recently acquired pediatric glioma drug, and talked AI strategy with AstraZeneca’s head of U.S. oncology for lung cancer Arun Krishna. Dan recaps his ASCO experience here.  Speaking of buzzy therapeutic spaces, there was more action on the vaccines front last week as Health and Human Services Secretary RFK Jr. announced that healthy children and healthy pregnant women would no longer be advised to get vaccinated against COVID-19. However, as of publication, the CDC still recommends a COVID vaccine for healthy children but instead of a universal recommendation advises that the decision should be made between parents and healthcare providers. Against this backdrop, the FDA signed off on Moderna’s next-gen COVID-19 vaccine, mNEXSPIKE, for a limited population in line with its new guidelines. This was a much-needed win for Moderna, which last week had a $760 million-plus government contract for its mRNA-based bird flu vaccine terminated.  Also on the policy front, the Trump administration released its Make America Healthy Again report last week to much scrutiny after reports found studies and references that did not exist.

The name of the biopharma game this season is vaccines—and RFK Jr. wasted no time returning from Memorial Day Weekend before making news on this front, removing the COVID-19 vaccine from the list of recommended immunizations for healthy kids and pregnant women on Tuesday. This follows a rash of recent moves, including a new risk-based strategy for the approval of new COVID vaccines focused on adults over 65 and high-risk individuals six months to 64 years of age and a request that Moderna and partners Pfizer and BioNTech update the myocarditis risk on their vaccines’ labels.This increased vaccine scrutiny by the FDA and Department of Health and Human Services is having a significant impact on biopharma companies, several of whom have received stop-work orders on next-gen COVID vaccines. Meanwhile, Moderna last week pulled the biological license application for its combination COVID-19/flu vaccine, anticipating a request for additional data on flu shot efficacy from the FDA, as mRNA technology continues to be scrutinized after playing the hero during the pandemic. Also last week, the White House released its Make America Healthy Again (MAHA) report, which took aim at vaccines, along with pharma lobbying and GLP-1s.Also on the policy front, a California judge issued an order Thursday that indefinitely stops HHS’ goal of reducing its divisions from 28 to 15 and firing upwards of 10,000 employees, among other reorganization and mass layoff plans. And speaking of government plays that could receive judicial pushback, we received a couple of new updates on the Most Favored Nation (MFN) front: first, President Donald Trump appointed his CMS chief Mehmet Oz as the leader of drug pricing negotiations, calling him “one tough hombre,” and second, HHS provided new guidance for streamlining the process for states to import drugs from Canada.In the obesity realm, Eli Lilly is calling out the Centers for Medicare and Medicaid Services. In an open letter last week, Lilly voiced its displeasure with the agency for a final ruling that left its Zepbound and Novo Nordisk’s Wegovy out of Medicare Advantage and Part D coverage in 2026.And in the R&D realm, we returned from the long weekend to sad news from Rocket Pharmaceuticals, as the company reported that its pivotal Danon disease trial is on hold after the death of a young patient. The death—extremely sad on a human level—is also a setback for the gene therapy space, which had been buoyed earlier this month by the success of a personalized CRISPR treatment received by baby KJ.Finally, BioSpace looks ahead to ASCO#25 where Dan Samorodnitsky will be on the ground in Chicago.

Lori Ellis, head of insights and Ian Fisher, head of development of analytics at IQVIA, discusses the critical importance of Target Product Profiles (TPPs) for life sciences companies, especially during uncertain times with funding challenges. Fisher emphasizes that TPPs serve as strategic guiding light which help companies articulate their development goals and demonstrate value to potential investors and partners.This episode is presented in partnership with IQVIA.Host⁠⁠⁠⁠⁠Lori Ellis⁠⁠⁠⁠⁠, Head of Insights, BioSpaceGuestIan Fisher, Head of Development Analytics, IQVIA

Pfizer stole the headlines this week with a pact worth up to $6 billion with Chinese biotech 3SBio for a PD-1/VEGF candidate just three months after inking a clinical trials collab for a similar drug with Summit Therapeutics. It’s the largest Chinese licensing deal in recent memory, as pharmas continue to turn to the country to fill their pipelines.  Also on Tuesday, the Department of Health and Human Services offered a smidge more detail on President Donald Trump’s Most Favored Nation executive order. A press release explained that drug prices will be tied to the lowest price in certain countries with a GDP at least 60% that of the U.S. and that the effort will focus on branded drugs.   Over at the FDA, the strategy around COVID-19 vaccines is evolving. According to an editorial published in the New England Journal of Medicine Tuesday by FDA Commissioner Marty Makary and CBER director Vinay Prasad, future COVID-19 approvals will focus on adults over 65 and high-risk individuals six months to 64 years old—a strategy they say will better align the U.S. with other high-income nations. This is also in sync with the Novavax approval, which came in Friday after a few delays.   Meanwhile, the FDA’s Oncologic Drugs Advisory Committee (ODAC) is currently in the midst of a two-day meeting, but acquiring the necessary expertise was “absolute chaos,” according to an agency insider who spoke with BioSpace. This is partly due to the decimation of an FDA office that includes staff responsible for screening scientific and therapeutic area experts for conflicts of interest.  Going back to last week—which seems like a year ago at this point—we were somewhat shocked to learn that the CEO of one of the world’s most valuable pharma companies is on his way out. Friday morning, Novo Nordisk announced that CEO Lars Fruergaard Jørgensen will be leaving the company after eight years at the helm. After peaking last June at about $155 apiece, the obesity leader’s shares are currently worth just $68. Novo said the decision for Jorgensen to leave was mutual, but Jorgensen was not made available to speak on a call following the announcement.  Finally, a couple of milestones are worth noting: First, the Alzheimer’s space got a big win on Friday when the FDA approved the first blood-based test for the disease—news that could be a boon to Eli Lilly’s Kisunla and Biogen & Eisai’s Leqembi. And second, a nine-month-old boy named KJ with a disease called CPS1 deficiency that affects just 1 in 1.3 million U.S. babies was treated with a single-use CRISPR treatment created just for him. It’s an incredible story that highlights just how far gene editing has come, but it also highlights a rare disease crisis, with these sorts of ultra- and nanorare diseases lacking the necessary financial incentive to motivate biopharma’s focus.

President Donald Trump unveiled a sweeping drug pricing policy this week, seeking to lower drug prices in the U.S. by up to 80% through a reprisal of the Most Favored Nation rule he attempted to introduce in his first term. The rule would essentially link U.S. prices to those paid in other nations where medications are cheaper. Biopharma reaction was one of tentative relief, with BMO Capital Markets analysts suggesting the executive order had “more bark than bite.” Meanwhile, the Centers for Medicare and Medicaid Services announced that among the next 15 drugs to undergo IRA-prescripted price negotiations could be drugs payable through Medicare Part B, and not just Part D, where the first two rounds have applied.  Into all of this action steps Vinay Prasad, the outspoken oncologist and hematologist who was named last week as the next director of the FDA’s Center for Biologics Evaluation and Research. While the S&P Biotech ETF fell by more than 5% upon the news, overall reaction was fairly measured, with cell and gene therapy executive Audrey Greenberg summing up Prasad’s selection as “anything but a status quo appointment.”  Over in the weight loss and obesity space, Eli Lilly can’t seem to lose. This weekend, Lilly announced full data from a head-to-head trial showing a “superior benefit-to-risk ratio” for its Zepbound over Novo Nordisk’s Wegovy. And last week, the Indiana-based pharma won a court battle against compounders when a judge sided with the FDA, stating that tirzepatide—the active ingredient in both Zepbound and diabetes sister drug Mounjaro—was no longer in shortage. Add on a presidential shoutout during Trump’s Monday press conference for its U.S. manufacturing investments, and it really was Lilly’s week.  Flying less high are some 2,000 Bayer employees who lost their jobs in the first quarter of 2025 as part of the company’s new operating model, which is intended to make Bayer “much more agile.” On a less direct flight is Galapagos, which reversed course on plans to spin out a portion of the company and find a new CEO. Instead, CEO Paul Stoffels will make a quicker exit and the Belgian biotech could sell off its cell therapy assets as it looks to build up a new pipeline in house, having abandoned the spinout idea altogether. Stay tuned.  Finally, in ClinicaSpace this week, we took a deep dive into the HIV treatment space, where companies like Gilead and Immunocore are targeting a cure, while the Trump administration slashes funding for HIV research.

Paul Offit, MD, Director of the Vaccine Education Center at the Children's Hospital of Philadelphia and the Maurice R. Hilleman Professor of Vaccinology, Professor of Pediatrics at the Perelman School of Medicine at the University of Pennsylvania, identifies himself as a "vaccine skeptic"–someone who demands data and evidence–which he believes is the appropriate stance for medical professionals and regulators.After the Cutter incident in 1955, which resulted in 250 cases of polio caused by batches of polio vaccine containing live polio virus given to the public, the FDA took up this mantle in evaluating vaccines. He warns that public discourse has moved dangerously past healthy skepticism into harmful cynicism, particularly through conspiracy theorists who dismiss scientific evidence by claiming researchers are "in the pocket of Big Pharma." This cynicism threatens public health as vaccine-preventable diseases like measles and pertussis are re-emerging due to declining vaccination rates, with polio potentially following if immunization continues to decrease.Host⁠⁠⁠⁠⁠Lori Ellis⁠⁠⁠⁠⁠, Head of Insights, BioSpaceGuestsPaul Offit, MD, Director of the Vaccine Education Center, Children's Hospital of Philadelphia; Maurice R. Hilleman Professor of Vaccinology and a Professor of Pediatrics, Perelman School of Medicine, University of PennsylvaniaDisclaimer: The views expressed in this discussion by guests are their own and do not represent those of their organizations.

In his effort to onshore manufacturing, President Donald Trump issued an executive order on Monday afternoon ordering the FDA to ease permitting processes for new and expanded U.S. facilities. The announcement comes as more and more Big Pharma companies commit billions to expanding their U.S. footprints. Bristol Myers Squibb CEO Christopher Boerner announced this week that the company will pump $40 billion into its stateside operations over the next five years, even as the pharma executes a massive cost-cutting effort that involves shaving $3.5 billion from expenses by 2027 and cutting thousands of jobs, including another 516 in New Jersey, according to a May WARN notice.  In other policy news, the Department of Health and Human Services on Wednesday said it will require all new vaccines to be tested in placebo-controlled trials to earn FDA approval but some vaccine experts have raised concerns about this approach. Meanwhile, turmoil still envelopes the FDA, with staff cuts and rehires continuing at a dizzying pace. On Monday, several states sued HHS, saying that the cuts offload critical functions and costs onto the states and impede public health efforts.  As Q1 earnings season for Big Pharma begins to wind down, there are still headlines coming from the biotech sector. Vertex revealed last week that it is abandoning all of its adeno-associated virus vector work, while BioNTech on Monday announced that tariffs could get in the way of its ambitious plans for a closely watched PD-L1-VEGF therapy. Moderna, meanwhile, continues its fall from COVID grace, missing Q1 revenue expectations and announcing plans to reduce operating expenses by around $1.5 billion by 2027.   In the weight loss space, Novo Nordisk announced on Friday that the FDA has accepted the application for a pill version of Wegovy, with a decision expected this fall. Novo has also struck partnerships with CVS and Hims & Hers pharmacies to market injectable Wegovy, drawing the attention of Eli Lilly CEO David Ricks.   Also this week, check out BioSpace’s deep dives into advances in base editing—a technology that’s been touted as a “safer” CRISPR—and Summit Therapeutics’ push to bring closely watched PD-1/VEGF immunotherapy ivonescimab to the U.S. market after its recent approval in China.

Policy issues—particularly tariffs—loom large as Q1 2025 earnings season rolls on, with Pfizer , Novartis, AstraZeneca and many more all reporting this week. On Pfizer’s call, CEO Albert Bourla called the Trump administration’s national security concerns “legitimate,” but objected to the proposed tariffs in general. Meanwhile, Novartis CEO Vas Narasimhan brushed off the tariff risk but expressed concern over President Donald Trump’s desire to bring back the ‘Most Favored Nations’ rule.  Meanwhile, cancer conference season is in full swing, with the American Association for Cancer Research’s annual event continuing in Chicago. Merck, GSK, Boehringer Ingelheim, Roche and others have presented highly anticipated data from some of their cornerstone cancer drugs and other candidates.  Turning to the latest news out of the FDA, newly minted Commissioner Marty Makary gave his first two big press interviews last week, making statements that both jibed with and contradicted recent reporting. And in the midst of the ongoing Novavax vaccine saga, many have raised concerns that the FDA could become politicized under the watch of HHS Secretary RFK Jr.  Finally, check out this week’s BioPharm Executive stories, including a deep dive into the trend toward mining innovative therapies from China and BioSpace’s annual report on pharma CEO-to-employee pay ratios.

This discussion features BioSpace's head of insights Lori Ellis, Kearney partner Martin Hadosi, and Melissa Laitner, director of strategic initiatives at the National Academy of Medicine emphasizing the need for collaboration to improve women's health.This discussion features Lori Ellis, Martin Hadosi, and Melissa Laitner, who emphasize the need for collaborative effort across multiple sectors including industry, healthcare organizations, researchers, and patient advocacy groups. Regarding investment challenges, they acknowledge the current difficult economic environment affects all biomedical research, not just women's health specifically.This episode is presented in partnership with ⁠DIA⁠, in support of their ⁠2025 Global Annual Meeting⁠ taking place June 15-19 in Washington DC.Host⁠⁠⁠⁠⁠Lori Ellis⁠⁠⁠⁠⁠, Head of Insights, BioSpaceGuests⁠Martin Hodosi⁠, Partner, Kearney⁠Melissa Laitner⁠, Director of Strategic Initiatives, National Academy of MedicineDisclaimer: The views expressed in this discussion by guests are their own and do not represent those of their organizations.

While much of the country was off celebrating Easter and Passover this weekend, the FDA—and its new leader Marty Makary—were busy making news. Makary, who was confirmed as FDA commissioner last month, gave his first major interview, where he unveiled a new regulatory pathway for rare disease therapies and addressed the “epidemic of distrust” he feels is imperiling the agency. Makary also shared his thoughts on the conflicts of interest he sees between the FDA and pharma industry, highlighting a new directive to ban industry representatives from serving on the agency’s advisory committees—a move experts believe will have minimal impact in reducing COIs.Makary also touched on vaccines and what he believes to be the cause of autism. On this, he was more restrained than his boss, HHS Secretary Robert F. Kennedy Jr., who continues to insist on re-investigating what he believes is a link between autism and vaccines. The interview comes as more former FDA officials sound the alarm about the changes happening within the agency.Elsewhere on the policy front, President Donald Trump continues to threaten tariffs on pharma products. In response, two more companies, Roche and Regeneron, are committing billions of dollars to enhance their U.S. footprints. They join Novartis, J&J and Eli Lilly in this regard, with the latter additionally promising to manufacture its investigational oral obesity drug orforglipron in the U.S.That announcement followed the news that orforglipron generated “injectable-like efficacy” in a Phase III diabetes trial. Lilly intends to submit for approval of the pill for weight management by the end of this year and diabetes in 2026. Not to be outdone, chief rival Novo Nordisk revealed Saturday that it has already filed for FDA approval of an oral formulation of its own blockbuster, semaglutide, for overweight and obesity.Finally, make sure to check out this week’s edition of BioPharm Executive where we take a deep dive into another sizzling space—transthyretin amyloid cardiomyopathy (ATTR-CM)—and find out which pharma CEO has the biggest golden parachute.

It’s all about tariffs again this week. No sooner had Donald Trump announced that “major” tariffs on the pharmaceutical industry would be coming “very shortly”—sending stocks spiraling—the president announced a 90-day pause on most taxes for imports from countries not named China. But this respite was not to last. On Sunday, Secretary of Commerce Howard Lutnick told ABC News that tariffs would be coming for pharma in the next month or two, according to Leerink.  A Federal Register Notice revealing an investigation into pharmaceutical product imports and the associated potential national security threats additionally signaled that tariffs might be imminent. Accordingly, tariffs were a major theme as Johnson & Johnson kicked off Q1 earnings season on Tuesday, where CEO Joaquin Duato urged President Donald Trump to consider tax policy changes instead. Last week, Novartis joined J&J and Eli Lilly in preparing for said tariffs with a $23 billion pledge to expand its own U.S. R&D and manufacturing. And amid all of these trade tensions, the National Security Commission on Emerging Biotechnology released a report warning that China is “dangerously close” to overtaking U.S. biotech.  Meanwhile, after unprecedented cuts to its workforce, the FDA is looking toward a future of drug review delays and increased executive oversight, according to experts. Industry watchers have also expressed concern that the cuts could trigger a little-known mechanism built into the Prescription Drug User Fee Act that could potentially cost the FDA nearly half of its funding and set the agency—and U.S. patients—back 35 years.  Also fighting for headlines on Monday morning was Pfizer’s announcement that it is ending development of its lead obesity candidate danuglipron after detecting a potential case of drug-induced liver injury. Pfizer’s loss could be Viking Therapeutics’ gain, however, as analysts immediately floated the idea of a marriage between the two companies. Viking has long been a top takeover target, and the biotech’s shares rose 13% upon Pfizer’s announcement.  Finally, it’s that time of year again: In BioPharm Executive this week, BioSpace looked at the top paid CEOs in biopharma. Who is this year’s highest paid CEO? Click here to find out.

In this bonus episode, BioSpace’s vice president of marketing ⁠Chantal Dresner⁠ and careers editor ⁠Angela Gabriel⁠ take a look at job market performance in the first quarter of 2025. They discuss job posting trends, application rates and layoffs. Also discussed are recent decisions of the Trump administration and how they are impacting the workforces of the private sector, HHS and academia.Want to receive our latest quarterly job market reports? ⁠Subscribe⁠ to Career Insider for our job market updates, job trends, career advice and more.

This discussion features BioSpace's head of insights Lori Ellis, Kearney partner Martin Hadosi, and Melissa Laitner, director of strategic initiatives at the National Academy of Medicine examining the underrepresentation of women in clinical trials. They stress that rather than waiting for regulatory guidance, the industry should proactively improve trial accessibility and inclusivity, as there's mutual benefit in faster enrollment and more diverse participation.This episode is presented in partnership with ⁠DIA⁠, in support of their ⁠2025 Global Annual Meeting⁠ taking place June 15-19 in Washington DC.Host⁠⁠⁠⁠⁠Lori Ellis⁠⁠⁠⁠⁠, Head of Insights, BioSpaceGuests⁠Martin Hodosi⁠, Partner, Kearney⁠Melissa Laitner⁠, Director of Strategic Initiatives, National Academy of MedicineDisclaimer: The views expressed in this discussion by guests are their own and do not represent those of their organizations.

Pushback against the Trump administration’s massive government cuts exploded this weekend across America—and biopharma is no exception. Taking a deep dive into the leadership cuts, we found that more than half of the senior FDA leaders from six months ago are no longer there, and that’s just the leadership. Across the Department of Health and Human Services, some 20,000 people will be leaving—of their own accord or otherwise—amounting to a 25% reduction in headcount that would leave HHS with around 62,000 employees. With many of these people likely destined for biopharma, does this pose an ethical problem?   Meanwhile, the escalating tariff war is beginning to ring alarm bells for the economy, with Goldman Sachs now predicting a 45% chance of a recession happening in the next 12 months. While pharma has historically been considered resistant to the challenges of a recession—people always need medicines—this time around could be different, in part because of the consumer-led obesity market. Thanks to the Trump administration’s decision to nix a Biden era proposal to cover GLP-1s under Medicare Part D—at least for now—these drugs will continue to cost consumers a pretty penny.   Even without a recession being declared, Eli Lilly’s and Novo Nordisk’s stocks are falling significantly with the rest of the market. Lilly has lost more than $95 billion in market value in one month. Novo’s value has declined $72 billion.   The drugmakers are also still battling makers of knockoff versions of their drugs. Most recently, Lilly filed a lawsuit against a med spa in Indianapolis for allegedly taking vials of tirzepatide and splitting them up into single doses that are sold to patients without the packaging inserts.  Of course, Lilly and Novo aren’t the only pharma drug rivalry out there. There’s also Keytruda vs. Opdivo, Leqembi vs. Kisunla and more.   Finally, we take a look at seven late-stage MASH candidates that could hit the market in the next few years, following the FDA’s 2024 approval of Madrigal’s Rezdiffra, the first treatment for the metabolic disorder.

Biopharma’s reaction to the forced resignation of venerable CBER director Peter Marks has been swift and furious, with former FDA Commissioner Robert Califf saying on LinkedIn that “the FDA as we’ve known it is finished.” Analysts, meanwhile, called Marks’ exit “arguably biotech investors’ greatest fear,” as company shares across the industry tumbled.   Marks’ announcement added insult to injury for the agency, which was already reeling from the announced cuts of 10,000 employees across the Department of Health and Human Services, including 3,500 FDA staffers. Those layoffs began to roll out on Tuesday as some employees showed up to work only to discover they no longer had a job. Amid all this chaos, Cantor Fitzgerald analysts called for Kennedy himself to get the axe, saying in part that he was “undermining the trusted leadership of health care in this country.”    Despite the turmoil, drug development continues in the obesity space, with Novo Nordisk presenting mixed data from its semaglutide franchise at the American College of Cardiology’s annual conference last weekend. While an oral version of the blockbuster drug showed cardiovascular benefits for some patients, it failed to change the trajectory of other major adverse cardiovascular events. Meanwhile, Novo continues its battle against compounding pharmacies manufacturing copycat versions of semaglutide—as multiple players scramble for a piece of this massive pie.    On the Alzheimer’s front, Eli Lilly’s Kisunla failed last week to win the recommendation of the EU’s Committee for Medicinal Products for Human Use. This decision is consistent with CHMP’s recent stance on anti-amyloid antibodies, as Biogen and Eisai have also struggled to get Leqembi approved in Europe.    On a positive note, pharma R&D returns grew again in 2024, but Deloitte warned that this progress is “fragile.” The firm urged companies to be bold and embrace cutting edge technology like gene therapy and AI. These returns can’t help everyone, however, as the past week has seen an uptick in layoffs across biopharma, including at Carisma Therapeutics, Organon and Tenaya Therapeutics.   Finally, as April kicks off cancer conference season, BioSpace took a deep dive into the recent action in the always-hot in radiopharmaceuticals space.

Martin Hodosi, partner at Kearney and Melissa Laitner, director of strategic initiatives, National Academy of Medicine, join Lori Ellis, head of insights, to discuss the evolution of women's inclusion in clinical trials. They note that while overall representation has improved, significant challenges remain. They highlight how industry mindset has evolved from being protectionist to inclusion.This episode is presented in partnership with DIA, in support of their 2025 Global Annual Meeting taking place June 15-19 in Washington DC.Host⁠⁠⁠⁠Lori Ellis⁠⁠⁠⁠, Head of Insights, BioSpaceGuestsMartin Hodosi, Partner, KearneyMelissa Laitner, Director of Strategic Initiatives, National Academy of MedicineDisclaimer: The views expressed in this discussion by guests are their own and do not represent those of their organizations.

President Donald Trump doubled down on tariff threats targeting pharma, saying additional levies on pharmaceuticals will come “at some point,” per CNBC. Meanwhile, Johnson & Johnson became the latest big pharma to respond to Trump’s warning of potential tariffs if companies don’t reshore their manufacturing, announcing a massive $55 billion U.S. manufacturing and R&D investment. Not all companies are on board, however: AstraZeneca is looking eastward, pumping $2.5 billion into a new research facility in Beijing.    Also on the policy front, Trump nominated acting CDC director Susan Monarez for the top job after pulling his first nominee, Dave Weldon, days before his senate hearing was expected to begin. If confirmed, Monarez would be the first CDC director since 1953 to not have a medical degree; she holds a Ph.D. in microbiology and immunology from the University of Wisconsin.   In weight loss news, Novo Nordisk is paying China-based United Laboratories $200 million upfront to license a triple agonist of the GLP-1, GIP and glucagon receptors that could one day compete with Eli Lilly’s retatrutide. And BioSpace examines the next great challenge for GLP-1s: oral formulation manufacturing.    Two more therapeutic spaces in focus last week are Duchenne muscular dystrophy and spinal muscular atrophy, where companies including Dyne Therapeutics, REGENXBIO and Novartis presented new data on their respective candidates. And the Duchenne community continued to react to news of the death of a patient taking Sarepta’s approved gene therapy Elevidys.  In cardiovascular news, Alnylam won a much-anticipated approval for Amvuttra as the first RNAi silencer for transthyretin amyloid cardiomyopathy, setting up a three-way race with Pfizer’s tafamidis—marketed as Vyndaqel and Vyndama—and BridgeBio’s Attruby. Next up is Milestone Therapeutics’ CARDAMYST in paroxysmal supraventricular tachycardia, which has a PDUFA date of March 27.   Finally, the saga of Cassava Sciences’ Alzheimer’s hopeful simufilam is over, as the company announced it has ended development of the controversial candidate.

A patient with Duchenne muscular dystrophy taking Sarepta’s gene therapy Elevidys has died of acute liver failure, possibly related to a recent viral infection. Sarepta, which said it will update Elevidys’ label to reflect the new safety signal, saw its shares drop 22% on the news but analysts still seem positive on the drug, as treatment options for Duchenne remain limited.Meanwhile, both AstraZeneca and Taiho Pharmaceuticals announced acquisitions worth up to $1 billion or more in two sizzling therapeutic spaces, cell therapy and antibody-drug conjugates, respectively.Despite canceling a vaccine advisory committee late last month, the FDA on Thursday selected flu strains to be targeted in the upcoming 2025-2026 flu season. And at another federal agency, the Centers for Disease Control and Prevention, employees will have to wait a bit longer to see who will take the helm under Donald Trump, as the president’s nominee, Dave Weldon, was pulled hours before he was set to appear before a Senate committee on Thursday. Like HHS Secretary Robert F. Kennedy Jr., Weldon has expressed anti-vaccine views in the past, particularly his continued suggestion of the link between vaccines and autism. Guggenheim Partners called the move to revoke Weldon’s nomination “a positive sign for reigning in vaccine criticism.”In the weight loss arena, BioSpace takes deep dives into the tendency for biopharma to develop fast-followers, or me-too drugs—following a pattern seen with PD-1 checkpoint inhibitors after the approvals of Merck’s Keytruda and Bristol Myers Squibb’s Opdivo. One key difference between these two markets, however, is that when it comes to GLP-1s for weight loss, patients are not staying on these medicines. Drug developers are trying several approaches to improve treatment persistence, including titration, combinations and even secondary drugs that address side effects. They’re also making other moves to differentiate themselves, including focusing on overall health outcomes—in areas like cardiovascular, sleep apnea and kidney disease.Following on BioSpace’s coverage of the major patent cliffs that many Big Pharma companies are facing in coming years, we also take a look back at some of the companies that have already weathered such loss of exclusivity. It’s rarely a straightforward story of sales crashing off patent, as companies take various tacks to extend their blockbuster sales.Finally, the cardiovascular space is expecting some movement this week. First, Alnylam is anticipating a decision on its RNAi silencer Amvuttra in ATTR-CM. An approval—which is widely expected—would make three companies on the market in this rapidly expanding space after Pfizer’s tafamidis was approved in May of 2019, and BridgeBio’s Attruby got the greenlight in November last year. And second, Milestone Pharmaceutical has a PDUFA coming up for etripamil in paroxysmal supraventricular tachycardia.

On the sidelines of this year’s J.P. Morgan Healthcare Conference in San Francisco, BioSpace Senior Editor Annalee Armstrong sat down with Mark McKenna, CEO of Mirador Therapeutics, a member of BioSpace’s NextGen Class of 2025. Their discussion here focused on the company’s two-pronged approach to developing therapies for inflammatory and fibrotic diseases, as well as the importance of operating under stealth at this time for the biotech.This is the second episode in a special series of The Weekly focused on how NextGen companies are navigating the current business environment.

Miruna Sasu, CEO of COTA, has been labeled as a disrupter in the industry. In this episode, she discusses the challenges of stringent inclusion and exclusion criteria of clinical trials. She also dives into her main takeaways from this year's JP Morgan Healthcare Conference and Scope Summit around exits and investment in women's health.Host⁠⁠⁠Lori Ellis⁠⁠⁠, Head of Insights, BioSpaceGuests⁠Miruna Sasu⁠, President and CEO, COTADisclaimer: The views expressed in this discussion by guests are their own and do not represent those of their organizations.

As we mark five years since the World Health Organization officially declared the COVID-19 pandemic, the BioSpace editorial team reflects on the health crisis of a generation and how it changed us—and the biopharma industry. While companies like Pfizer, BioNTech and Moderna raked in billions from their vaccines, J&J and Novavax struggled to capture a significant piece of the market.   Five years later, much has changed. A substantial number of us now work from the comfort of our homes—though that may be changing for some in the life sciences—and biopharma has a new obsession: obesity. In this space, Novo Nordisk has had a rough week, reporting lower-than-expected results from a second straight trial of its next-gen weight loss drug CagriSema. And in an effort to protect revenues from its blockbuster weight-loss drug Wegovy, Novo jumped into the legal battle between the FDA and compounding pharmacies over the regulator’s decision to declare the shortage of Wegovy over. Viking Therapeutics had a better week, securing “multiple metric tons” of its investigational obesity medication VK2735 in a deal with CordenPharma. Meanwhile, companies continue to tackle adverse events associated with GLP-1s.  Another space that has seen its fair share of failures in the past couple of years is depression. The latest flop comes from J&J, which announced it would discontinue its a kappa opioid receptor blocker aticaprant after a disappointing Phase III readout. J&J joins Biogen and Sage Therapeutics, Relmada Therapeutics and Alto Neuroscience, all of whom have suffered regulatory misses or trial flops. Finally, two of Donald Trump’s healthcare nominees, Marty Makary and Jay Bhattacharya, sailed through their confirmation hearings in the Senate last week. Both are up for confirmation on Thursday.

Donald Trump’s tariffs have headlined myriad news stories this week—including at BioSpace, where we reported Pfizer CEO Albert Bourla’s claim that his company is prepared to reshore manufacturing if the president makes good on threats made last month. Eli Lilly also appears to be preparing, commiting $27 billion to boost its U.S. manufacturing capacity.   Meanwhile, another regulatory meeting has been canceled under new HHS Secretary Robert F. Kennedy Jr. Reuters revealed last week that an upcoming meeting of the FDA’s external advisers for vaccine policy on March 13 has been canceled—just a week after the CDC Vaccine Advisory Board’s first meeting of 2025 was postponed. Also on the policy front, BioSpace took a deep dive into priority review vouchers (PRVs) after Congress failed to reauthorize the rare pediatric disease PRV program at the end of 2024. Our reporting shows this will be painful for many biopharma companies who rely on funds from the sale of PRVs.   Speaking of money, AbbVie and Eli Lilly struck a pair of mid-size deals in hot spaces. AbbVie made a late obesity play this week, inking a licensing deal worth up to $2.2 billion with service provider Gubra to bring a long-acting amylin drug to the market, while Lilly hopped onto the hot molecular glue train, paying more than $1.2 billion in a licensing deal with Magnet Biomedicine.  Finally, we examined the somewhat lethargic immuno-oncology space, which has companies, including BMS, Roche, Summit Therapeutics and BeiGene, targeting TIGIT, VEGF, RAS and more in their quest to bring the next Keytruda—which led the way in 2024 as the world’s best-selling drug—to the market.

On the sidelines of this year’s J.P. Morgan Healthcare Conference in San Francisco, BioSpace Senior Editor Annalee Armstrong sat down with Kevin Marks, CEO of Delphia Therapeutics, a member of BioSpace’s ⁠NextGen Class of 2025⁠. Their discussion here focused on the company’s novel science of activation lethality, which involves forcing cancer cells to overactivate and thereby overload cell stress pathways, as well as Delphia's relatively smooth path to a $67 million series A raise thanks to contributions from Google Ventures (GV), where Marks was previously employed.This is the second episode in a special series of The Weekly focused on how NextGen companies are navigating the current business environment.Host⁠Annalee Armstrong⁠, Senior Editor, BioSpaceGuest⁠Kevin Marks⁠, President and CEO, Delphia TherapeuticsFor a complete list of NextGen 2025 companies, ⁠click here⁠.

Miruna Sasu, CEO of COTA, has been labeled as a disrupter in the industry. In this discussion, she highlights the challenges of investing in the life sciences industry. She also suggests how the investor mindset needs to change as well as offer solutions that benefit both investors and developers seeking investment.Host⁠⁠Lori Ellis⁠⁠, Head of Insights, BioSpaceGuestsMiruna Sasu, President and CEO, COTADisclaimer: The views expressed in this discussion by guests are their own and do not represent those of their organizations.

There was a high point this week for employees riding the rollercoaster that is currently the FDA, as 300 recently fired employees are reportedly being asked to return. This could serve to boost spirits at the agency, which is reeling from unpredictable and seemingly ‘indiscriminate’ layoffs—and low morale as a result.   Trump made additional waves over the weekend, threatening to enact tariffs on the largest pharmaceutical companies—unless they relocate their manufacturing operations to the U.S. And the early impacts of the new administration continue to be felt, as the CDC’s vaccine advisory board postponed its first meeting of 2025—just a week after RFK Jr.’s confirmation as HHS secretary.   In weight loss news, the Outsourcing Facilities Association (OFA), which represents compounders, is suing the FDA after the agency declared an end to the shortage of semaglutide, marketed by Novo Nordisk as Ozempic and Wegovy. This followed a similar OFA lawsuit last fall, filed after the FDA removed Eli Lilly’s tirzepatide— Zepbound and Mounjaro—from its shortage list. In a possible attempt to protect Zepbound from compounder competition, Lilly announced Tuesday that it will expand single dose vials options of the blockbuster weight loss drug at a reduced price. This all comes as a new study compares physician wishlists with investment in the obesity space—and reveals key indicators for drug developers.  And it’s been a good news/bad news kind of week in gene therapy, as Pfizer discontinued hemophilia B drug Beqvez worldwide, Regeneron reported more positive data from its gene therapy for genetic deafness and, facing a cash crunch, bluebird bio elected to go private in an acquisition by global investment firms Carlyle and SK Capital Partners.   Finally, let us leave you with a ghost story—the story of so-called “ghost assets” that have found a new home, and a new purpose.

At the 2025 National Biotechnology Conference, gene therapies, bispecific antibodies and other novel modalities—relative newcomers to medicine—will be much discussed. In this curtain raiser, BioSpace speaks with conference chair Prathap Nagaraja Shastri of J&J about these highly anticipated topics. HostJef Akst, Managing Editor, BioSpaceGuestPrathap Nagaraja Shastri, Scientific Director and Group Leader, Clinical Pharmacology and Pharmacometrics, Johnson and JohnsonBioSpace is a media partner of the National Biotechnology Conference.

President Trump has been on a staff cutting rampage since taking office, and this weekend, the hammer came down on an undisclosed number of FDA employees. The firings—which reportedly affected staffers within the FDA’s centers for food, medical devices and tobacco products—came days after Robert F. Kennedy Jr. was confirmed as HHS secretary.One space that could have an ally in RFK Jr. is psychedelic therapies, which the new HHS boss has accused the FDA of “suppressing,” along with stem cell therapies. On the flip side, Kennedy is a well-known vaccine critic, though his confirmation didn’t affect the approvals of a pair of vaccines—one for GSK’s meningococcal shot and another for Bavarian Nordic’s chikungunya jab.One notable vaccine maker, however, is struggling. Moderna reported Q4 and 2024 earnings last week, revealing full-year revenue of just over $3 billion—less than half of the nearly $7 billion the company brought in the year before. Adding insult to financial injury, Moderna is also laying off around 50 employees on its digital team.Moderna isn’t the only company facing challenges. Several biopharmas, including Merck, Regeneron and Bristol Myers Squibb are facing patent headwinds that, according to William Blair, are the strongest they’ve been in nearly 15 years. It’s also a difficult climate in which to stay alive as a biotech, to which the 13 bankruptcies in 2024 can attest.Finally, BioSpace launched a new NextGen podcast series this week, in which senior editor Annalee Armstrong speaks with execs from four of our NextGen Class of 2025 companies—starting with COUR Pharmaceuticals.

On the sidelines of this year’s J.P. Morgan Healthcare Conference in San Francisco, BioSpace Senior Editor Annalee Armstrong sat down with Dannielle Appelhans, CEO of COUR, a member of BioSpace’s NextGen Class of 2025. Their discussion here, focused on the company’s long history leading up to its $105 million in series A funding at the start of 2024 and the possibility of an initial public offering in the future, among other things, represents the first of a special series of The Weekly focused on how NextGen companies are navigating the current business environment. HostAnnalee Armstrong, Senior Editor, BioSpaceGuestDannielle Appelhans, CEO, COURFor a complete list of NextGen 2025 companies, click here. The NextGen Class of 2025 is brought to you by Pliancy.

BioSpace’s Head of Insights Lori Ellis and Tom Whitehead, co-founder of the Emily Whitehead Foundation discuss the challenges navigated by cancer patients, weaving in their personal experiences with treatment and how they would encourage those undergoing treatment to speak up and advocate for themselves.This episode is presented by the ⁠Genscript Biotech Global Forum 2025.⁠Host⁠⁠Lori Ellis⁠⁠, Head of Insights, BioSpaceGuests⁠⁠Tom Whitehead,⁠⁠ Author, Keynote speaker; Co-Founder, Emily Whitehead FoundationDisclaimer: The views expressed in this discussion by guests are their own and do not represent those of their organizations.

We saw more action on the M&A front this week as Novartisbought back its blood thinner abelacimab and the rest of Anthos Therapeutics for up to $3.1 billion and still ahead, a potential buyout of SpringWorks Therapeutics by Merck KGaA, which confirmed it’s in “advanced discussions” with the biotech. Meanwhile, Eli Lilly signed aglobal licensing deal for a MASH asset with South Korea’s OliX Pharmaceuticals.As Q4 and full-year 2024 earnings continue to roll in, BMS, Eli Lilly,Novo Nordisk, AstraZeneca, Eisai, Vertex and Gilead all reported their results in the past week. Notably, BMS continues to reorganize, now targeting an additional $2 billion in savings through 2027, and AstraZeneca axed two rare disease drugs from its $39 billion acquisition of Alexion. When it comes to revenue growth, Novo’s Wegovy and Lilly’s Zepbound continue to climb at a striking pace—a phenomenon that has at times driven the weight loss drugs into shortage and spawning a controversialshadow market of off-brand versions.Speaking of controversy, Regeneron is suing its Dupixent partner Sanofi, claiming the French pharma has failed to provide it with adequate information regarding sales of the blockbuster anti-inflammatory drug—which missed analyst expectations in Q4.On the clinical side, the pulmonary fibrosis space continues to see positive results, with Boehringer Ingelheim notching its second Phase III win in six months for nerandomilast. It wasn’t all good news, however, as Pliant Therapeutics suspended dosing and enrollment in a Phase IIb/III study of its idiopathic pulmonary fibrosis candidate, causing its stock to crash.Finally, BioSpace released a special edition of ClinicaSpace this week focused on the resurgent cardiovascular space—just in time for Valentine’s Day! Sign uphere to receive your copy.

U.S. political maneuverings are keeping biopharma on its toes. Tuesday, Robert F. Kennedy, Jr. passed a major hurdle as the Senate Finance Committee voted to advance his nomination for HHS secretary to a floor vote of the entire Senate. If confirmed, RFK Jr. could have an impact on myriad regulatory issues, including PDUFA negotiations. In other political news, Donald Trump announced his promised tariffs on Canada, Mexico and China—the last of which could have particular implications for biopharma companies looking to China for promising new drug candidates. Both were subjects of conversation as leading pharma companies—including Pfizer, Merck and Regeneron—reported their Q4 and full-year 2024 earnings. Notably, Pfizer is eyeing deals between $10 billion and $15 billion, while Merck took a 12% stock hit on Gardasil challenges in China and Regeneron issued its first-ever dividends. On the regulatory front, the FDA greenlit Vertex’s Jounavx last week as the first new drug for acute pain in more than two decades. Journavx leads a non-opioid pain space that is seeing significant momentum this year, with Tris Pharma, Algiax Pharamceuticals and SiteOne Therapeutics all announcing positive data in the past month. Meanwhile, in obesity news, the U.K.’s pharmacies regulator rolled out stricter guidelines for online pharmacies selling medicines including Novo Nordisk’s Wegovy and Eli Lilly’s Mounjaro, and an obesity-focused deal inked with Novo last year wasn’t enough to keep Omega Therapeutics afloat as the Massachusetts-based biotech revealed it is heading toward bankruptcy. Finally, a new CEO will be taking the reins at Takeda in 2026—but the selection of Julie Kim is an exception in an industry still struggling for gender parity at the top leadership ranks.

BioSpace's Head of Insights Lori Ellis and Tom Whitehead sit down and discuss cell and gene therapies from the perspectives of patients. What do caregivers and patients consider to be risks when lives are on the line? This episode is presented by the Genscript Biotech Global Forum 2025. Host ⁠Lori Ellis⁠, Head of Insights, BioSpace Guests ⁠Tom Whitehead,⁠ Author, Keynote speaker; Co-Founder, Emily Whitehead Foundation Disclaimer: The views expressed in this discussion by guests are their own and do not represent those of their organizations.

In the week since Donald Trump took office, he’s caused quite the stir with healthcare-centered moves that include ordering the Department of Health and Human Services to stop communications, hiring and travel and announcing that he would withdraw the U.S. from the World Health Organization. Wednesday, the U.S. Senate Finance Committee convenes to vote on the controversial nomination of Robert F. Kennedy, Jr. for health secretary—a vote that Jefferies analysts said they expect to be “close.” Biogen continues to grab headlines this month, as the latest chapter in the Sage saga saw the smaller biotech rejecting its neuro partner’s unsolicited buyout offer. Meanwhile, Biogen laid off an undisclosed number of employees from its research unit, just as a higher dose of its Ionis-partnered spinal muscular atrophy therapy Spinraza was accepted for review by both the FDA and EMA. Elsewhere, the weight loss space continues to click on all cylinders, with Versant Ventures debuting its newest obesity biotech Helicore Biopharma on Tuesday with $65 million in series A funds, and two obesity-focused companies, Aardvark Therapeutics and Metsera, seeking entry to the public markets. These up-and-comers will have to compete against the likes of Eli Lilly and Novo Nordisk, the latter of which reported data last week showing that its next-gen obesity drug amycretin could elicit up to 22% weight-loss. And Veru announced that its enobosarm could significantly improve the quality of weight loss in seniors also taking Novo’s Wegovy. Another busy therapeutic space is Duchenne muscular dystrophy, where analysts predict a lot of action in the next couple of years, with a number of data readouts and regulatory submissions. And finally, Annalee Armstrong caught up at JPM with Novavax CEO John Jacobs, who said the vaccine maker is at a pivot point.

Donald Trump was sworn in as the 47th president of the United States on Monday, bringing with him a host of healthcare nominees and potential changes to the FDA, M&A and drug pricing. The new administration was a key focus point at the 2025 J.P. Morgan Healthcare Conference last week, where Annalee Armstrong spoke to executives from several companies about what they’re expecting from a second Trump term. Also at JPM, Mirador Therapeutics CEO Mark McKenna boldly predicted that 2025 would see the return of the megamerger. Speaking of M&A, Annalee spoke with leaders from Biogen, who declined to address the company’s unsolicited takeover bid for Sage Therapeutics. For its part, Sage sued Biogen last week, “seeking preliminary injunctive relief to enforce a standstill agreement.” Meanwhile, Gilead CEO Daniel O’Day addressed the split of Galapagos—a company Gilead has poured more than $5 billion into since 2019. Check out more personal stories from JPM here. In the obesity space, Novo Nordisk reported data from a Phase III trial showing that a high dose of Wegovy elicited more weight loss than the approved regimen—but still fell short of results posted by Eli Lilly’s Zepbound. Relatedly, Wegovy and sister drug Ozempic are both on the list of the next fifteen drugs whose prices could be negotiated under the Inflation Reduction Act. Finally, the FDA on Friday made its first high-profile decision of the year, greenlighting AstraZeneca and Daiichi Sankyo’s Dato-DXd—now Datroway—to treat certain types of advanced breast cancer. The approval is the first for the highly touted antibody-drug conjugate, which is also under FDA review for EGFR-mutated non-small cell lung cacner. Analysts expect 2025 to be a pivotal year for the ADC.

In this bonus episode, BioSpace's vice president of marketing Chantal Dresner and careers editor Angela Gabriel take a look at Q4 job market performance and what we expect to see ahead. We dive into year-on-year data and recent job trends. Want to receive our latest quarterly job market reports? Subscribe to Career Insider for our job market updates, job trends, career advice and more.

In this episode of Denatured, Dr. Peter Marks M.D., PhD. gives his thoughts on the future of cell and gene therapies. He speaks candidly about opportunities and realities, manufacturing and its role in efficacy and the CGT runway.  Dr. Marks was keynote speaker at the GenScript Biotech Forum in San Francisco this week. Host ⁠Lori Ellis⁠, Head of Insights, BioSpace Guest ⁠Peter Marks M.D., PhD.⁠, Director, Center for Biologics Evaluation and Research (CBER)

The J.P. Morgan Healthcare Conference kicked off Monday with a flood of high-value deals, reinvigorating sentiment across the biopharma industry. Johnson & Johnson made the biggest splash, acquiring neurology leader Intra-Cellular Therapies for $14.6 billion, while GSK picked up precision therapy specialist IDRx for $1B upfront and Eli Lilly laid down up to $2.5 billion for Scorpion’s PI3Kα inhibitor program. Meanwhile, the immunology and inflammation space continues to fire on all cylinders as Gilead invests up to $1.7 billion for LEO Pharma’s preclinical oral small molecule STAT6 program. And those are only the deals accepted by both parties. Prior to the conference, Biogen offered to acquire its struggling neuro partner Sage Therapeutics for around $469M. The proposal follows a catastrophic run for Sage, which has seen its shares fall more than 90% in the past two years. 2024 sales and earnings forecasts have also generated attention this week, with Sarepta reporting that Duchenne muscular dystrophy gene therapy Elevidys beat analysts’ expectations in the fourth quarter, and Eli Lilly projecting a full-year revenue miss driven largely by lower-than-expected sales of GLP-1 blockbusters Zepbound and Mounjaro. As expected, obesity has been a hot topic at JPM, with Pfizer CEO Albert Bourla announcing that his company is going “all in” in the space. This follows new FDA guidance revealed last week recommending a minimum weight loss threshold for drug developers. Among the many companies taking notice is newcomer Verdiva Bio, which launched last week with more than $410 million in opening funds. Also debuting last week was Kardigan, which raised $300 million to tackle heart disease. Kadigan joins a resurgent cardiovascular space, where several companies—including those developing gene therapies—are targeting myriad diseases. Finally, BioSpace senior editor Annalee Armstrong caught up with Daphne Zohar, CEO of BioSpace NextGen 2025 company Seaport Therapeutics Daphne Zohar, who offered her thoughts on the current state of the neuropsychiatric space.

TEASER: This episode is little sneak peak into upcoming podcasts as well as topics that will be discussed at the 2025 GenScript Biotech Global Forum on January 15 in San Francisco. Listen in as Peter Marks and Tom Whitehead share their thoughts on the CGT space for 2025. Register for the event here. Host Lori Ellis, Head of Insights, BioSpace Guests Peter Marks M.D., PhD., Director, Center for Biologics Evaluation and Research (CBER) Tom Whitehead, Author, Keynote speaker; Co-Founder, Emily Whitehead Foundation

Happy New Year! BioSpace released our NextGen: Class of 2025 this week, highlighting 25 biopharma startups to watch this year. The companies on this list are not afraid of a challenge, wading into some of biopharma’s most competitive therapeutic spaces. As analysts predict an uptick in M&A in ’25, could some of them be potential targets? One NextGen 2025 company that could already be fielding calls is Metsera, which reported promising Phase II data for its investigational subcutaneous GLP-1 therapy on Tuesday. The GLP-1 space continues to garner significant attention, with both Eli Lilly and Novo Nordisk seeking to protect their drugs against competition from compounders. This strategy makes a lot of sense as the sizzling class continues to expand into heart disease, sleep apnea and metabolic dysfunction-associated steatohepatitis, among other indications. On the regulatory side, the FDA approved 55 novel medicines in 2024—including a few inaugural company approvals—and is looking ahead to several key PDUFA dates in Q1, 2025. Meanwhile, the agency released three new guidance documents seeking to provide clarity around confirmatory trials supporting accelerated approvals, the use of AI in drug development and the use of tissue biopsies in clinical trials. Finally, BioSpace senior editor Annalee Armstrong heads to the J.P. Morgan Healthcare Conference next week as biopharma seeks an M&A spark to improve the currently a gloomy sentiment. Stay with us for all the biggest news coming out of JPM.

This conversation focused on a few remaining topics discussed at length at Jefferies late last year, with the same topics anticipated to make an appearance at JPM. In this episode, our guests the industry’s need to catch up with women’s health issues and the innovative lead the APAC region has taken in clinical trials. Host ⁠⁠Lori Ellis⁠⁠, Head of Insights, BioSpace Guests ⁠⁠Miguel Forte⁠⁠, President, International Society for Cell & Gene Therapy; Board Member, ARM; CEO and Board Member, Kiji Therapeutics ⁠⁠Ali Pashazadeh,⁠⁠ Founder, Treehill Partners

2025 is set to change the industry. The new administration is poised to challenge many existing processes. The question is whether they will be based on data-driven analysis or ideologies and beliefs. Our guests address this concern along with the existing challenges CEOs face, particularly manufacturing processes. Additionally, they discuss weight loss drugs, focusing on GLP-1s, with medical precision. Host ⁠Lori Ellis⁠, Head of Insights, BioSpace Guests ⁠Miguel Forte⁠, President, International Society for Cell & Gene Therapy; Board Member, ARM; CEO and Board Member, Kiji Therapeutics ⁠Ali Pashazadeh,⁠ Founder, Treehill Partners

Note: BioSpace is taking a break for the holidays. The next episode of The Weekly will air Jan. 8, 2025. Happy New Year! Novo dominated headlines over the last week, with a kidney disease label expansion for Novo Nordisk’s Ozempic in Europe but a newly flagged safety risk for the drug, with a Danish regulator requesting that the EMA review reports of a rare eye disease in patients who took the GLP-1. Meanwhile, over the weekend Novo Nordisk’s parent company, Novo Holdings, and CDMO Catalent announced that they had received all regulatory clearances to close the proposed $16.5 billion acquisition. The deal, which will see Novo Holdings sell three sites to Novo Nordisk, is the biggest manufacturing investment this year but certainly not the only, with supply chain becoming a focus for biotech and Big Pharmas alike. Meanwhile, 2024 proved to be a snacky year for M&A activity and a tough environment for IPOs, with this year’s newly public biotechs mostly seeing their stock prices plummet after entering the public markets. Moderna, while not new to the markets, has similarly seen its stock tank. President-elect Donald Trump’s nomination of well-known anti-vaccine advocate Robert F. Kennedy Jr. to head the Department of Health and Human Services certainly didn’t help. On the clinical research side of the industry, 2024 saw its fair share of flops and victories. One of the most striking in each category was the failure of AbbVie’s emraclidine. The Big Pharma acquired the schizophrenia hopeful with its nearly $9B Cerevel buy late last year. The flops came not long after the approval of BMS’ Cobenfy, which came out of the even-bigger purchase of Karuna and was the first new schizophrenia medication in more than three decades.

This week, GSK and Gilead and Arcellx presented key data at the American Society of Hematology (ASH) annual meeting as they vie for a competitive advantage in multiple myeloma. Meanwhile, Vertex unveiled positive long-term data for its CRISPR Therapeutics–partnered gene therapy Casgevy in sickle cell disease—results BMO Capital Markets analysts said should help Casgevy keep its edge over bluebird bio’s Lyfgenia. On that note, Casgevy and Lyfgenia have a new outcomes-based payment model after the Centers for Medicare and Medicaid Service (CMS) said both companies have agreed to participate in a voluntary program to improve access to the gene therapies. Speaking of access, Eli Lilly and Novo Nordisk both announced significant manufacturing investments aimed at shoring up production of their diabetes and weight loss blockbusters tirzepatide and semaglutide. And in related news, the European Commission gave its blessing to Novo Holdings’ controversial acquisition of contract manufacturing firm Catalent. Elsewhere, AbbVie got a much-needed win for Cerevel-acquired Parkinson’s disease therapy tavapadon—a month after the deal’s cornerstone asset emraclidine failed in schizophrenia—while uniQure announced it has aligned with the FDA on “key elements” of the accelerated approval pathway for its investigational gene therapy for Huntington’s, AMT-130.

“Cautious optimism” is circling once again as the industry approaches 2025. However, many companies are treading water, barely holding on as the market continues at a slow pace. Ali Pashazadeh and Miguel Forte have an open discussion about the state of the market. Host Lori Ellis, Head of Insights, BioSpace Guests Miguel Forte, President, International Society for Cell & Gene Therapy; Board Member, ARM; CEO and Board Member, Kiji Therapeutics Ali Pashazadeh, Founder, Treehill Partners

It’s officially holiday shopping season, and biopharma is getting into the spirit. Novartis expanded its presence in Huntington’s, paying up to $2.9 billion to advance PTC Therapeutics’ Phase II candidate, while as Gilead committed up to $415 million to Tubulis to develop novel ADCs for solid tumors and Roche’s Genentech inked an autoimmune development deal with COUR Pharmaceuticals potentially worth more than $900 million. Finally, Takeda put up to $1.3 billion on the line for Keros’ blood cancer anemia drug, a potential competitor to Bristol Myers Squibb’s Reblozyl. On the regulatory front, Applied Therapeutics’ stock crashed more than 80% after the FDA last week denied approval of govorestat in the rare metabolic disorder classic galactosemia. Also last week, the FDA announced it is looking into the safety of bluebird bio’s gene therapy Skysona after new reports of secondary blood cancers in treated patients. On a more positive note this week, Intra-Cellular Therapies submitted an application for Caplyta in major depressive disorder, potentially opening up an additional $1 billion in revenue. Finally, R&D investment in glioblastoma is ticking up as Merck, Kazia Therapeutics and Black Diamond Therapeutics look to advance treatments for the rare but devastating brain tumor.

Biopharma had its collective deal-making hat on heading into the Thanksgiving holiday, with Roche buying Poseida for up to $1.5 billion, and Sarepta and Arrowhead tying up in a collaboration potentially worth up to $10 billion. These announcements followed last week’s news from Novartis, which snatched up Kate Therapeutics for a little over $1 billion while promising more acquisitions below $5 billion. On the regulatory front, the FDA gave BridgeBio—and transthyretin amyloid cardiomyopathy (ATTR-CM) patients—something to be thankful for late last week with the approval of Attruby for the rare, cardiovascular disease. The nod sets up a potential three-way race with Pfizer’s tafamidis and Alnylam’s Amvuttra, the latter of which was accepted for FDA review in ATTR-CM on Monday.   On the opposite end of the clinical development spectrum, Cassava Sciences’ controversial Alzheimer’s drug failed to reduce cognitive or functional decline in a Phase III trial. And investors were unimpressed by the 20% weight loss generated by Amgen’s MariTide in a much-anticipated Phase II trial, as the company’s stock tumbled 11% Tuesday. Drawing much attention from the biopharma industry, President-elect Donald Trump continued with his nominees for top healthcare posts, announcing COVID-19 critic Marty Makary to lead the FDA and well-known vaccine skeptic Dave Weldon for CDC director. Meanwhile, the FDA is grappling with the loss of Chevron Deference pertaining to a legal challenge over Eli Lilly’s GLP-1 shortages. Finally, the next generation of antibody-drug conjugates remains hot, with Danish biotech Adcendo reeling in $135M in a Series B financing round.

Women represent 51% of the population; not investing is a poor investment strategy. Incorporating women into leadership positions, into board rooms, is the only way to tap this market. Host ⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠Lori Ellis⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠, Head of Insights, BioSpace Guests ⁠Konstantina Katcheves⁠, Senior Vice President, Innovative Medicines Global Business Development and Acquisitions, Teva Pharmaceuticals ⁠Sanskriti Thakur⁠, Chairwoman, TOWER Capital Group

President-elect Donald Trump announced last week that he would nominate prominent anti-vaxxer Robert F. Kennedy Jr. to lead the HHS. While this news caused a dip in biotech stocks, one analyst said the selloff was an overreaction. Meanwhile, fall conference season continues with the American College of Rheumatology Convergence (ACR) and American Heart Association’s 2024 Scientific Sessions (AHA). Lupus was the indication du jour at ACR, where BMS, Kyverna, Fate Therapeutics and Cabaletta Bio presented data from early-stage trials of their CAR T candidates, and Biogen and UCB detailed data behind their unexpected late-stage victory for dapirolizumab pegol. This was a much-needed win for Biogen, which has seen its shares decline 36% this year.  Neurogene’s stock, meanwhile, tumbled 36% on Monday alone, following the release of details about an adverse event in a trial for its Rett syndrome gene therapy. This followed a 35% fall last week when the adverse event was first announced. And the company decided to drop its gene therapy in Batten disease after the FDA declined to grant Regenerative Medicine Advance Therapy designation to support the program. We also took a look at bluebird bio’s cash problem, with the company slated to exhaust its runway to reach a breakeven point before the end of next year. On a more positive note, RegenxBio announced this week it has aligned with the FDA on path to possible accelerated approval for its investigational gene therapy for Duchenne muscular dystrophy, following behind Sarepta’s Elevydis, which faced controversy after it received accelerated approval in June 2023, only to miss the primary functional endpoint in its confirmatory trial four months later. And speaking of accelerated approval, we released a special edition of ClinicaSpace Monday focused on this very topic. Sign up to receive it here.

Possibly the biggest news this week comes from the schizophrenia space, where AbbVie’s emraclidine failed two mid-stage trials. Acquired by AbbVie in its $8.7 billion Cerevel Therapeutics buy, this result is in stark contrast to that of Bristol Myers Squibb’s $14 billion acquisition of Karuna Therapeutics, which yielded Cobenfy—the first novel schizophrenia drug approved in 35 years. Also having a tough month is AstraZeneca, which despite reporting strong Q3 sales Tuesday has been dealing with drama at its China headquarters as a top executive there is being investigated for alleged medical insurance fraud. Meanwhile, Bayer had less good fortune in Q3 as CEO Bill Anderson acknowledged that the company’s earnings were “not pretty.” And while much of the attention during earnings season focuses on Big Pharma, several biotech companies, including Nkarta, Intellia and Cassava Sciences, also announced their earnings last week. As we head into the holiday season, some employees at Sana Biotechnology and genetic testing firm 23andMe aren’t feeling very fesitve as those companies announced layoffs. Finally, BioSpace takes a deep dive into one of the most intractable neurodegenerative diseases, Huntington’s, where the first disease-modifying drug just might be on the horizon.

No discussion regarding the future is complete unless AI is incorporated. In this episode, Konstantina Katcheves of Teva Pharmaceuticals and Sanskriti Thakur of TOWER Capital Group provide their insights on the impact of not only the benefits of the technology but the regulatory challenges and uncertainty surrounding AI. Host ⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠Lori Ellis⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠, Head of Insights, BioSpace Guests Konstantina Katcheves, Senior Vice President, Innovative Medicines Global Business Development and Acquisitions, Teva Pharmaceuticals Sanskriti Thakur, Chairwoman, TOWER Capital

Last week, Eli Lilly suffered a rare third-quarter earnings miss as diabetes and obesity drugs Mounjaro and Zepbound fell short of Wall Street expectations, in part due to wholesalers’ stocking decisions. On the flip side of the GLP-1 race, Novo Nordisk’s Wegovy aced part 1 of a pivotal Phase III trial in metabolic dysfunction–associated steatohepatitis (MASH), with results comparable to Madrigal’s Rezdiffra, according to analysts.  In ClinicaSpace this week, BioSpace explores the shift in the CAR T cell therapy space from cancer to autoimmune disease as early data spark excitement and companies recruit autoimmune experts to fill in knowledge gaps. In the neurodegenerative space, we take a deep dive into the Alzheimer’s and Parkinson’s pipelines after recent news of a few terminated programs and returned assets. Finally, we look at the lawsuits filed by Henrietta Lacks’ estate against life sciences companies and the history of the cells that bear her name.

Earnings heat up as Pfizer got a much-needed Q3 beat amidst criticism from activist investor Starboard Value. Novartis and Sanofi are among others that have outpaced Wall Street expectations this quarter, as Eli Lilly, Merck, AbbVie, Amgen, Biogen, GSK, Bristol Myers Squibb and Takeda are all reporting today and tomorrow. The past week has also seen a pack of deals, with AbbVie’s $1.4 billion buy of Aliada Therapeutics, Roche’s potential $1 billion deal with Dyno Therapeutics and Novartis’ up to $2.1 billion commitment to Monte Rosa’s molecular glue degraders. With less than a week until Election Day, we unpack what biopharma might expect under a Trump or Harris administration. We also take a look back at 10 years of BioSpace’s NextGen list of top up-and-coming startups. A lot have been bought out by bigger companies—some for big money—while some, such as CRISPR Therapeutics, continue to operate independently. Finally, we took a close look at questions stemming from Sarepta’s new data for Duchenne muscular dystrophy gene therapy Elevidys, and separately but not unrelatedly, at the FDA’s accelerated approval pathway.

In this episode, we’re talking money. The guests in this discussion recognize that the economic climate has been different (and more chaotic) in last three to four months, but expect a steady but slow improvement as we move into the new year. This episode reviews 2024’s investment landscape and the policies influencing investment going into 2025. Host ⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠Lori Ellis⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠, Head of Insights, BioSpace Guests Konstantina Katcheves, Senior Vice President, Innovative Medicines Global Business Development and Acquisitions, Teva Pharmaceuticals Sanskriti Thakur, Chairwoman, TOWER Capital

Last week, Gilead withdrew Trodelvy in bladder cancer after the antibody-drug conjugate failed to meet the primary endpoint in a confirmatory study. This follows Pfizer’s recent withdrawal of another therapy that had earned FDA accelerated approval, Oxbryta for sickle cell disease. With few other options available to patients, BioSpace took a look at 5 sickle-cell candidates currently in clinical trials.   Following a disappointing Alzheimer’s readout, the company’s third neuro stumble in six months, Sage Therapeutics will lay off over 165 employees—about 33% of its workforce. The company is reporting Q3 earnings on Oct. 29.  On a more positive note, Vertex reported full Phase III data this week for its non-opioid pain treatment, su-zetri-gine. If approved, suzetrigine, which has a PDUFA date of Jan. 30, 2025, would be the first new class of acute pain medicine in more than two decades. And on Monday, a company that never leaves the news, Novo Nordisk, announced positive results from a cardiovascular study with its oral version of semaglutide, Rybelsus. In other Novo news, scrutiny around Novo Holdings’ acquisition of Catalent is heating up with a coalition of unions, consumer groups and public interest organizations last week expressing their concerns about the buyout. This prompted BioSpace to unpack the unique structure of the collection of organizations that is Novo. Finally, Sanofi is having a busy month, securing the sale of its healthcare unit Opella and separately paying approximately $326 million to obtain a 16% stake in European radiopharma biotech Orano Med. Radiopharma is skyrocketing in popularity, and some companies are even trying to marry it with another hot therapeutic spaces: antibody-drug conjugates. Could radiolabeled ADCs overcome some of the side effects of radiation treatments, speed up treatment times and enable lower doses than traditional therapies?

It’s that time again: earnings season. Q3 calls started out with a bit of a snore from J&J, which did beat analyst expectations but announced no big shakeups or surprises. As expected, the company saw shrinking revenue for its blockbuster Stelara, which just lost to Lilly’s Omvoh in a head-to-head clinical trial in Crohn’s disease. Last week, a concerning study was published regarding seven children who developed blood cancers after being treated with bluebird bio’s gene therapy Skysona. This publication comes at a time when the company is struggling to regain its share price, which has dropped below the Nasdaq minimum bid price requirement. The bad news about Skysona also fits with another trend BioSpace covered this week: accelerated approvals gone wrong. In other news, Sanofi is following some of its pharma peers and looking to sell its consumer healthcare unit. This follows similar moves from J&J, which spun off Kenvue last year, as well as GSK, Pfizer and others. And Senator Elizabeth Warren (Mass.-D) is upping the pressure on Novo in relation to its acquisition of Catalent, penning a letter to the Federal Trade Commission voicing concerns about the potential effects of such a deal, should it go through. Finally, bispecific antibodies make a comeback in oncology.

Designating something "safe" in this industry means acceptable risk. AI can help us improve acceptable risks if it is used appropriately. However, industry experts must be thoughtful and responsible in their use of AI within clinical trials. In the third episode of our mini series on artificial intelligence, our guests explore the challenges and benefits of integrating technology into the lives of patients. Host ⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠Lori Ellis⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠, Head of Insights, BioSpace Guests ⁠⁠Mike King⁠⁠⁠, Senior Director of Product and Strategy, IQVIA ⁠⁠⁠⁠Moritz von Stosch⁠⁠⁠⁠ Chief Innovation Officer, DataHow ⁠⁠⁠Nindhana Paranthaman⁠⁠⁠, Executive Medical Director, Clinical Development, Summit Therapeutics ⁠⁠⁠⁠Paul Agapow⁠⁠⁠⁠, Head of Data Science, BioNTech

The cell and gene therapy sector may be on the road to recovery after being met with investment headwinds following the highs seen during the pandemic, according to data presented Monday at the 2024 Cell & Gene Meeting on the Mesa hosted by the Alliance for Regenerative Medicine. BioSpace News Editor Greg Slabodkin reports from Phoenix. Last week, news broke that WuXi AppTec and WuXi Biologics, two companies named in the BIOSECURE Act, are looking to unload facilities in the U.S. and abroad as uncertainty looms over their U.S. business prospects. As Eli Lilly resolves shortages of its GLP-1 blockbusters, the company remains confident in its massive lead, along with competitor Novo Nordisk, over other companies with weight loss drug candidates—GLP-1s or other modalities such as next-gen CB1 inhibitors—looking to compete in the lucrative space. Not only are both companies making deals to expand their pipelines beyond GLP-1s, Lilly and Novo are actively pursuing broader markets for their current diabetes and weight loss blockbusters. Meanwhile, Big Pharma’s layoffs continue with announcements last week from Bayer, J&J and Pfizer.

Bristol Myers Squibb notched one of this year’s biggest approvals as the FDA greenlit Cobenfy, formerly KarXT, as the first novel treatment for schizophrenia in 35 years. Cobenfy’s origins go nearly as far back, beginning as an Alzheimer’s hopeful developed by current obesity rivals Eli Lilly and Novo Nordisk. On a lower note, Pfizer withdrew sickle cell medicine Oxbryta from the market, sending patients and advocates scrambling for more information. In other news, Roche hosted a Pharma Day event where it touted the $850 million acquisition of a portfolio of CDK inhibitors from Regor Pharmaceuticals and projected $3.6 billion in annual sales from three Carmot Therapeutics-acquired obesity and diabetes drug candidates. For those companies not bringing in billions of dollars, royalty financing provides an attractive option. And news editor Greg Slabodkin gives a preview of the upcoming Meeting on the Mesa. Plus, BioSpace looks at how the new abortion and IVF laws could impact women’s health research. With nearly a dozen abortion-related measures on state ballots this year, this will be an area to watch during next month’s elections.

Globalization, return on investment, diverse data sets undiscovered: this episode continues the exploration of AI. As we see the industry level out AI adoption, the future is still exciting and yet uncertain. With quantum around the corner, there are still challenges with AI at every turn. Host ⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠Lori Ellis⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠, Head of Insights, BioSpace Guests ⁠⁠Mike King⁠⁠⁠, Senior Director of Product and Strategy, IQVIA ⁠⁠⁠⁠Moritz von Stosch⁠⁠⁠⁠ Chief Innovation Officer, DataHow ⁠⁠⁠Nindhana Paranthaman⁠⁠⁠, Executive Medical Director, Clinical Development, Summit Therapeutics ⁠⁠⁠⁠Paul Agapow⁠⁠⁠⁠, Head of Data Science, BioNTech

Novo Nordisk CEO Lars Fruergaard Jørgensen testified Tuesday in front of Sen. Bernie Sanders’ Senate health committee regarding the prices of Ozempic and Wegovy—with some interesting takeaways. Meanwhile, PhRMA scored a rare win as the Fifth Circuit Court of Appeals ruled in the trade group’s favor, sending its complaint against the Inflation Reduction Act (IRA) back to a lower Texas court. Looking ahead, 15 more drugs are expected to be negotiated under the IRA next year, including Novo’s Ozempic.   Last week, the Federal Reserve cut the federal interest rate by half a percentage point—a move that was expected and met with a collective shrug from the biotech market, with experts skeptical of its impact. Elsewhere, the schizophrenia space is gearing up for an FDA decision on BMS’ KarXT. If approved, KarXT would be the first novel drug for the neuropsychiatric disease in decades. And on the obesity front, Novo’s oral cannabinoid receptor 1 drug elicited impressive weight loss in a Phase IIa trial, with some adverse events, and young startup Metsera touted robust Phase I data for its injectable GLP-1 candidate.

After their groundbreaking approval last year, infusions of Vertex and CRISPR Therapeutics’ and bluebird bio’s sickle cell gene therapies have begun, bringing hope to patients and the companies closer to realizing revenue. Meanwhile, bispecifics and anti-TIGIT therapies were all the rage at ESMO 2024 as BioNTech, GSK and iTeos, BMS and more reported positive results across multiple cancers. Last week, Moderna announced it would slash its R&D budget by $4 billion as it targets 10 new approvals through 2027. Possibly boding well for this target, the biotech features prominently on our list of 5 late-stage mRNA vaccines to watch. Meanwhile, the oral obesity drug race continues to heat up, with Terns Pharmaceuticals, Roche and Novo Nordisk all reporting new data from their respective trials. And in the equally hot radiopharmaceuticals space, Sanofi inked a $110 million licensing deal with RadioMedix to develop a neuroendocrine tumor candidate. Finally, BioSpace takes a deep dive into the HEALEY ALS Platform Trial, which has so far seen quick failures and small victories as leaders and early participants remain hopeful.

As 2024 closes, it is only right to discuss where we are when it comes to AI and where we will be in the future. As technology continues to evolve and blend into science in 2025 and beyond, a practical approach to what it can and cannot do must be explored. Additionally, how overpromising while underdelivering has affected investors' and the industry’s confidence in AI. Host ⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠Lori Ellis⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠, Head of Insights, BioSpace Guests ⁠⁠Mike King⁠⁠⁠, Senior Director of Product and Strategy, IQVIA ⁠⁠⁠⁠Moritz von Stosch⁠⁠⁠⁠ Chief Innovation Officer, DataHow ⁠⁠⁠Nindhana Paranthaman⁠⁠⁠, Executive Medical Director, Clinical Development, Summit Therapeutics ⁠⁠⁠⁠Paul Agapow⁠⁠⁠⁠, Head of Data Science, BioNTech

Summit Therapeutics made headlines this weekend at the 2024 World Conference on Lung Cancer, revealing that its bispecific antibody outperformed Keytruda in first-line non-small cell lung cancer. Meanwhile, bispecifics are one of several therapeutic classes on the agenda at the 2024 European Society for Medical Oncology (ESMO) Congress in Barcelona later this week. Last week, BioMarin held a public address intended to calm anxious investors after rounds of layoffs and pipeline changes—but many were left wanting. Also undergoing major upheaval is Lykos Therapeutics, which laid off 75% of its workforce after failing to secure approval for its MDMA-based post-traumatic stress disorder therapy and announced last week that CEO Amy Emerson is stepping down from her role. In Washington, D.C., Congress is back in session this week and wasted no time in reviewing the BIOSECURE Act, which passed a House vote on Monday.  And on the weight loss front, Terns Pharmaceuticals is moving ahead to Phase II after its investigational GLP-1 pill elicited positive results in Phase I, and Amgen is pushing MariTide into a broad late-stage development program that will test the obesity treatment in other weight-related conditions, such as heart, kidney and liver diseases.

Eli Lilly shook up the weight loss market again last week, announcing plans to sell single-dose vials of its blockbuster GLP-1 drug Zepbound directly to consumers. Meanwhile, Novo Nordisk said Monday that it expects the shortage for the lower doses of its own GLP-1 therapy Ozempic to persist into the fourth quarter of 2024. This week, we take a closer look at eyes, where gene therapy is breaking through against wet AMD, a common cause of blindness—potentially significantly minimizing the number of treatments required by patients—and cell therapy is making strides against another common foe: dry eye disease. Lastly, an unfortunate trend—layoffs—continues to play out, with BioMarin, Genentech and Astellas Gene Therapies all parting with staff members.

As the legal landscape continues to evolve, the uncertainty surrounding diversity, equity and inclusion (DEI) initiatives increases though the life sciences industry continues to move forward with DEI initiatives to include all patients in clinical trials. In this episode, our guests discuss framing strategies designed to protect DEI initiatives from legal challenges. Additionally, the guests acknowledge the importance of clinical trial sites in gaining patient trust. We also address the increased burden technology is putting on the sites as well as patients, suggesting potential ways to reduce these burdens. Host ⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠Lori Ellis⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠, Head of Insights, BioSpace Guests ⁠⁠Otis Johnson, PhD, MPA⁠⁠, Principal Consultant and Co-founder, Trial Equity ⁠⁠Pamela Tenaerts, MD, MBA⁠⁠, Chief Science Officer, Medable ⁠⁠Chris Hart,⁠⁠ Partner, Co-Chair, Privacy and Data Security Group, Foley Hoag LLP ⁠⁠Patrick Floody⁠⁠, Executive Director, Global Clinical Trial Services, Regeneron ⁠⁠Ken Getz⁠⁠, Executive Director, Tufts Center for the Study of Drug Development; Professor, Tufts University School of Medicine Disclaimer:The views expressed in this discussion by Patrick Floody are his own and do not represent those of Regeneron.

Gene editing startup Tome Biosciences is laying off nearly its entire workforce, as biopharma companies continue to face challenges in the current market. Meanwhile, Bayer, which cut 1,500 jobs in May, is parting with another 150 in Basel, Switzerland. On the other side of the spectrum, two new companies debuted last week. Novartis and Versant Ventures launched Borealis Biosciences to develop RNA medicines for kidney diseases, and BridgeBio spawned GondolaBio to focus on therapies for genetic and rare diseases. In Q2, biopharma VC funding reached its highest quarterly level since the same quarter in 2022, according to PitchBook. Separately, U.S.-China biopharma relations are making headlines again, with a House committe writing to the FDA comissioner about U.S. companies working with the Chinese military on potentially unethical clinical trials. And in case you missed it, a special edition of ClinicaSpace this week focuses on the obesity and diabetes space—a combined market that is expected to exceed $200 billion within the next decade.

With the Democratic National Convention underway, the Biden Administration’s announcing the first 10 drug prices negotiated under the under the Inflation Reduction Act seems likely to be a political move. But it’s still unclear how much the government is really saving—and what sorts of discounts patients will see. Separately, the biopharma industry saw a flood of layoffs. Lykos Therapeutics slashed around 75% of its staff following the FDA’s rejection of its MDMA-assisted therapy for PTSD. Meanwhile, diagnostic company GRAIL parted with about a quarter of its workforce, and German biotech Evotec could cut 400 roles globally. And as vaccine makers fall off the COVID-19 cliff, some are turning to combination COVID-flu vaccines, with Pfizer and BioNTech last week announcing mixed data following Moderna’s positive results earlier this summer.

In the second part of the discussion, our guests address clinical trial design which if done without careful consideration of the patient population can exclude patients from clinical trials instead of being inclusive. Using specific examples, our guests highlight the progress the industry has made to design inclusive clinical trials but stress the continuing need for improvement. In parallel, our guests discuss the continual judicial actives at the federal and state level surrounding women's health issues. As states begin to challenge other states for access to women's data, the future participation of women patients in clinical trials is becoming uncertain. Host ⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠Lori Ellis⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠, Head of Insights, BioSpace Guests ⁠Otis Johnson, PhD, MPA⁠, Principal Consultant and Co-founder, Trial Equity ⁠Pamela Tenaerts, MD, MBA⁠, Chief Science Officer, Medable ⁠Chris Hart,⁠ Partner, Co-Chair, Privacy and Data Security Group, Foley Hoag LLP ⁠Patrick Floody⁠, Executive Director, Global Clinical Trial Services, Regeneron ⁠Ken Getz⁠, Executive Director, Tufts Center for the Study of Drug Development; Professor, Tufts University School of Medicine Disclaimer:The views expressed in this discussion by Patrick Floody are his own and do not represent those of Regeneron.

In one of the year’s most highly anticipated decisions, the FDA rejected Lykos Therapeutics’ MDMA-assisted therapy for post-traumatic stress disorder (PTSD). Reaction from Lykos was swift, with the company stating its intention to “ask for reconsideration of the decision.”   Meanwhile, against the backdrop of the CDC’s recent RSV guidelines, Pfizer scored a big Phase III win for its shot in immunocompromised adults. Separately, Merck halted a Phase III trial of its Keytruda, anti-TIGIT, chemo combo in small cell lung cancer but made a splash with the potential $1.3 billion acquisition of Curon’s B cell depletion therapy. And AI-focused biotechs Recursion and Exscientia are merging to create a new company that will take Recursion’s name. Plus, as we reflect on Q2 earnings, it’s becoming clear that Eli Lilly is catching up to Novo Nordisk in the weight loss sphere, while others faced challenges in the vaccine space and the continued COVID cliff. Finally, BioSpace highlights five obesity data readouts to watch in the second half of 2024.

This week, Kamala Harris selected Minnesota Governor Tim Walz as her running mate. In July, Harris’ campaign netted a whopping $310 million—so where do pharmaceutical-connected contributions stand?   Meanwhile, Q2 earnings continue to roll in, and the picture isn’t looking so bright for some companies particularly vaccine makers like BioNTech, Moderna and Pfizer, who have been hit hard by the COVID-19 cliff and new CDC guidelines around RSV vaccines.   The FDA has also been busy of late, approving the first engineered cell therapy for solid tumors in Adaptimmune’s Tecelra. The regulator has another potential first on the docket this week in the form of Lykos Therapeutics’ MDMA-assisted treatment for PTSD—but Lykos will need to overcome a decidedly negative advisory committee vote and concerns surrounding its Phase III trial design. If approved, it would be the first classic psychedelic therapy authorized in the U.S.

The 2023 New Benchmarks on Demographic Disparities in Pivotal Trials study indicates that as Black representation increases, clinical trial enrollment time decreases. This is contradictory to what has been reported in the past. However, the highest disparity in clinical trial enrollment remains to be Black or African descent patients, with only a third being enrolled in clinical trials. Understanding not just the benefits on patient lives, but also the cost-effectiveness of adequate representation, pharma companies are continuing to lean into DEI practices for their clinical trials. In parallel, the legal environment for DEI initiatives has become unstable. With organizations actively pursuing legal cases against DEI initiatives, pharmaceutical and biotech companies must keep a watchful eye on judicial and political activities surrounding DEI and how these will shape the future of the clinical trial space in the United States. Host ⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠Lori Ellis⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠, Head of Insights, BioSpace Guests Otis Johnson, PhD, MPA, Principal Consultant and Co-founder, Trial Equity Pamela Tenaerts, MD, MBA, Chief Science Officer, Medable Chris Hart, Partner, Co-Chair, Privacy and Data Security Group, Foley Hoag LLP Patrick Floody, Executive Director, Global Clinical Trial Services, Regeneron Ken Getz, Executive Director, Tufts Center for the Study of Drug Development; Professor, Tufts University School of Medicine Disclaimer:The views expressed in this discussion by Patrick Floody are his own and do not represent those of Regeneron.

Second-quarter earnings season continues with Big Pharma beating Wall Street expectations, the author of an encrypted email sent to BioSpace has a proposal for Moderna and Merck, Roche and Viking seek quicker entry to the obesity market, and AAIC is in full swing. As July comes to a close, biopharma second-quarter earnings continue to roll in with Pfizer, Merck, AbbVie, AstraZeneca and many more reporting. So far, everything is coming up roses with most major companies beating Wall Street expectations. But every rose has its thorn and for biopharma executives this has long been drug price negotiations under the Inflation Reduction Act. Recently, however, drugmakers have expressed confidence that the IRA will not greatly impact their bottom lines. Speaking of bottom lines, in an anonymous email sent to the CFOs of Merck and Moderna and shared with BioSpace, a shareholder made the case that Merck should buy Moderna out of its 50/50 partnership involving Keytruda and their shared investigational personalized cancer vaccine. Meanwhile, M&A activity picked up in the second quarter of 2024 with nearly $18 billion changing hands, according to J.P. Morgan. Q3 has been no stranger to deals so far, either, with Boehringer Ingelheim acquiring Nerio Therapeutics for up to $1.3 billion to boost its immuno-oncology pipeline, and GSK and Flagship Pioneering striking a potential $7B deal to develop a portfolio of novel vaccines and medicines starting with immunology and respiratory treatments. Pfizer raised its full-year outlook after reporting positive Q2 results and announced layoffs this week at two North Carolina sites, with a total of 210 workers losing their jobs. Cuts at the Sanford, N.C. gene therapy manufacturing site come on the heels of Pfizer’s Duchenne muscular dystrophy gene therapy failed a Phase III trial. On the obesity front, both Viking Therapeutics and Roche indicated that they will seek entry to the market more quickly than originally anticipated, and the FDA issued a warning on Monday about possible safety risks associated with compounded versions of Novo Nordisk’s semaglutide. And the Alzheimer’s Association International Conference (AAIC) this week revealed some interesting nuggets, including long-term data regarding Eisai and Biogen’s Leqembi, and a small Phase IIb study showing that Novo’s GLP-1 liraglutide slowed cognitive decline in Alzheimer’s patients by up to 18%.

While the biopharma industry has seen glimmers of economic optimism, there were still more than 14,000 employees laid off in the first half of 2024. And BioSpace’s readers are among them. In this week’s Job Market Trends, H2 Update, 43% of respondents indicated they are currently unemployed, the highest-ever rate since BioSpace started conducting surveys. One large company that intends to eliminate around 680 jobs in its project development sector over the next few years is Novartis. Despite this, the Swiss pharma announced positive Q2 results last week, exceeding revenue expectations thanks to the continued revenue generated by its blockbuster drugs. Johnson & Johnson, which released its second quarter results on Wednesday, also beat analyst estimates.  Interestingly, Novartis CEO Vas Narasimhan said in a statement that coming into the market late with another GLP-1 drug would not be a “prudent approach” for the company. Meanwhile, several companies, including start-ups, are firmly committed to the weight loss space. Recently launched companies like Metsera, ProFound Therapeutics and Metaphore are developing GLP-1s or leveraging other mechanisms to target obesity, while market leaders Novo Nordisk and Eli Lilly are expanding their GLP-1 pipelines beyond weight loss. Novo and Lilly are also now marketing their GLP-1 medicines, semaglutide and tirzepatide, for weight loss in China, where GLP-1 biosimilars will soon hit the market. And finally, the Cassava Sciences saga continued, with CEO Remi Barbier and SVP of Neuroscience Lindsay Burns both resigning their posts. Cassava has faced allegations of data manipulation relating to its Alzheimer’s drug simufilam. The company has denied any wrongdoing.

This is the third episode of Denatured's discussion on diversity, equity and inclusion (DE&I). AI represents unlimited opportunities to increase efficiency, but as our guests note, it can also do incredible harm to patients and society. It largely comes down to training of the professionals using AI, as well as understanding how the AI model was grounded or trained. The data quality must be accurate and clean, but as they noted, DE&I is an issue the industry has been struggling with for decades. The solution lies in humans identifying problems, such as the lack of patient diversity in data, as well as studies in certain disease states, such as HPV-derived cancers. Going further, it is up to the industry to collaborate with each stake holder within the life sciences and healthcare spaces to use AI in ways that are patient focused. Host ⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠Lori Ellis⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠, Head of Insights, BioSpace Guests Dr. ⁠⁠⁠Ali Pashazadeh⁠⁠⁠, CEO,Treehill Partners Dr. ⁠Charlotte Jones-Burton⁠, Board Member, bluebird bio; Founder & President, Women of Color in Pharma⁠⁠⁠⁠⁠ ⁠Chia Chia Sun⁠⁠⁠⁠, Chief Commercial Officer, ⁠⁠⁠Fab Biopharma; CEO, Damiva ⁠Phyllis Greenberger⁠, Senior Vice President, Policy & Regulatory, Healthy Women Dr. ⁠Todd Rudo⁠, Chief Medical Officer, Clario

Pfizer stole the show late last week with the announcement that it will move forward with its oral GLP-1 analog danuglipron. While Pfizer may hope to advance in the already crowded race to bring an oral GLP-1 to market, some analysts reacted to the news with caution, mainly due to lack of data. Meanwhile, in the injectable GLP-1 space, it’s primarily a two-horse race between Novo and Lilly. While Novo had a two-year head start, Lilly has been picking up ground since the November 2023 approval of Zepbound (tirzepatide). A recent observational study saw tirzepatide outperform Novo’s semaglutide in helping patients to lose weight, and some analysts predict Lilly will indeed gain majority market share within a few years in part due to this potential efficacy advantage, though manufacturing is a complicating factor that currently favors Novo. Separately, the drug pricing debates heat up after the Federal Trade Commission released a scathing report last week about the business practices of pharmacy benefit managers (PBMs), noting how they charge payers higher prices than what they pay to pharmacies, profiting at the expense of patients and pharmacies. But biopharma firms must share the blame, and the Senate last week unanimously passed a bipartisan bill that would limit their use of patent thickets to maintain exclusivity, thereby making it easier for generic competitors to enter the market. Finally, several neurology-focused companies are hoping to bring new drugs to the market. BioSpace highlights five key neuro readouts to watch in the second half of 2024 in the Alzheimer’s, schizophrenia, depression and rare disease spaces.

After securing approval last week for Alzheimer’s drug Kisunla, Eli Lilly was back in the news Monday with the $3.2 billion acquisition of immunology biotech Morphic Holding. This represents another deal in the lucrative immunology and inflammation (I&I) space, which saw $12.3 billion in M&A activity last year, with analysts predicting this momentum would carry over into 2024.  Investment continues to pour into another hot therapeutic space: antibody-drug conjugates (ADCs). British biopharma company Myricx Bio netted a £90 million ($115.5 million) Series A round to expand its ADC platform and advance its pipeline into the clinic. Several other pharmaceutical companies are looking to capitalize on the potential of the ADC market—which is estimated to reach nearly $30 billion by 2028—including Johnson & Johnson, Genmab and Ipsen, who have all struck M&A deals in the space this year. Still others, including Sutro Biopharma and Mersana Therapeutics, are hoping to supercharge their potential via the immune system with immunostimulatory ADCs. And GLP-1s continue to make headlines, with one study linking semaglutide to an optic nerve condition that can cause sudden vision loss, and another showing the drug class could significantly lower the risk of 10 obesity-related cancers in diabetes patients. Novo Nordisk’s Wegovy has successfully expanded into China, where its patent will expire in less than two years, paving the way for biosimilar competition, and Lilly’s Mounjaro elicited greater weight loss than Novo’s Ozempic in a new observational study.

The biggest news of the week was the FDA approval of Eli Lilly’s Kisunla (donanemab) on Tuesday. While not unexpected, it was one of the year’s most highly anticipated decisions. And last week saw another big approval from the FDA—that of Verona’s novel COPD drug. But the regulator has also dropped three Complete Response Letters on drugmakers in the last seven days. Two of these were directly related to issues with third-party manufacturers. Though it’s unclear if those contract development and manufacturing organizations (CDMOs) were overseas, the FDA has flagged several concerns regarding manufacturers in India and China that have contributed to extreme drug shortages in the U.S. This will be particularly important in the face of a potential decoupling from Chinese CDMOs should the BIOSECURE Act become law, as India has been one country eyeing the opportunity. Meanwhile, Korean company Samsung Bio struck a $1 billion manufacturing contract with an undisclosed U.S. biopharma company. Among the many products the manufacturer makes are the piping hot antibody-drug conjugates, or ADCs. The global market has already exceeded $10 billion and is estimated to grow to nearly $30 billion by 2028. As evidence of the excitement surrounding ADCs are five major deals struck by biopharma companies this year. It’s not all good news for the ADC space, though, as one of those three FDA rejections was handed to Merck and partner Daiichi Sankyo’s investigational ADC for the treatment of certain non-small cell lung cancers. Meanwhile, BMS backed out of a collaboration with Eisai to develop an ADC being investigated in ovarian, peritoneal and fallopian tube cancers, as well as non-small cell lung cancer. We will continue to watch the industry’s strategies unfold as biopharma firms compete for a piece of that exploding ADC market.

This is the second episode of Denatured's discussion on diversity, equity and inclusion (DE&I). Data is the essential piece for both science and technology. The lack of DE&I data is becoming even more imperative as we move towards personalized medicines. Without understanding why there are differences in outcomes, the industry will continue to see worsening outcomes. However, DE&I is still not at the forefront of the mind during clinical trial designs, which influences not only the training but the application of AI models. Additionally, what this means not only for life sciences but also for healthcare providers as more of the healthcare industry adopts AI to assist with patient care. Host ⁠⁠⁠⁠⁠⁠⁠⁠⁠Lori Ellis⁠⁠⁠⁠⁠⁠⁠⁠⁠, Head of Insights, BioSpace Guests Dr. ⁠⁠Ali Pashazadeh⁠⁠, CEO,Treehill Partners Dr. Charlotte Jones-Burton, Board Member, bluebird bio; Founder & President, Women of Color in Pharma⁠⁠⁠ Chia Chia Sun⁠⁠⁠, Chief Commercial Officer, ⁠⁠⁠Fab Biopharma; CEO, Damiva Phyllis Greenberger, Senior Vice President, Policy & Regulatory, Healthy Women Dr. Todd Rudo, Chief Medical Officer, Clario

The biggest news of the last week was easily the expanded approval of Sarepta’s Duchenne muscular dystrophy gene therapy, Elevidys. CBER Director Peter Marks again overruled FDA staff members and review teams to grant the therapy full approval and a broad label expansion despite its missing the primary endpoint in a Phase III confirmatory study. This approval has us thinking about other big FDA decisions to watch this year, first and foremost, Eli Lilly’s anti-amyloid antibody donanemab. If given the green light, donanemab will be a direct competitor to Biogen and Eisai’s Leqembi, also a disease-modifying anti-amyloid antibody. Beyond that, Verona Pharma is expecting a decision Wednesday on ensifentrine, which could be the first novel mechanism for chronic obstructive pulmonary disease in over a decade, and in August, the FDA is set to decide on Lykos’ MDMA-assisted PTSD therapy, which an advisory committee voted against earlier this month. Then this week, Alnylam scored big with a Phase III win in transthyretin amyloidosis with cardiomyopathy (ATTR-CM). In what Alnylam CMO Pushkal Garg called “overwhelmingly positive data,” Amuvttra significantly lowered the risk of death and recurrent cardiovascular events in patients with ATTR-CM. Meanwhile, biopharma conference season continues with the American Diabetes Association’s annual conference held this past weekend. Eli Lilly’s blockbuster drug Zepbound “significantly improved” disease severity in patients with obesity and sleep apnea, and Altimmune racked up a Phase II victory for pemvidutide, which was effective at helping patients lose weight while retaining lean muscle.

BioSpace's ⁠Lori Ellis⁠ and ⁠Chantal Dresner⁠ are bringing live updates from ⁠#DIA2024⁠ in San Diego this week where we've been attending sessions on trial design, digital twins, cell and gene therapy regulation, clinical research workforce trends, professional development and many more. We discuss some of our key takeaways and event highlights, including Lori's exclusive interview with CBER director Dr. Peter Marks. Hosts ⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠Lori Ellis⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠, Head of Insights, ⁠⁠⁠BioSpace⁠⁠⁠   Chantal Dresner, VP of Marketing, BioSpace⁠⁠⁠

Diversity, equity and inclusion (DEI) continues to be a challenge for drug and medical device developers. While there has been some improvement in clinical trials, our guests note that unless there is a commercial or regulatory need, DEI is still an afterthought for many developers when designing clinical trials. Host ⁠⁠⁠⁠⁠⁠⁠⁠⁠Lori Ellis⁠⁠⁠⁠⁠⁠⁠⁠⁠, Head of Insights, BioSpace Guests ⁠⁠Ali Pashazadeh⁠⁠, CEO,Treehill Partners Charlotte Jones-Burton, Board Member, bluebird bio; Founder & President, Women of Color in Pharma⁠⁠⁠ Chia Chia Sun⁠⁠⁠, Chief Commercial Officer, ⁠⁠⁠Fab Biopharma; CEO, Damiva Phyllis Greenberger, Senior Vice President, Policy & Regulatory, Healthy Women Todd Rudo, Chief Medical Officer, Clario

We’re off to a rocky start this week on the clinical side, with some safety issues. BioNTech and MediLink’s ADC was placed on partial hold after multiple patient deaths, and some safety concerns were also revealed for Gilead’s blood cancer therapy magrolimab.  But on a more upbeat note, DIA in San Diego is filled with hope of collaboration and a focus on the patient experience. Head of Insights Lori Ellis joins BioSpace’s editorial team to discuss what’s she’s observed so far at the meeting and what she’s looking forward to in the coming days.  In global geopolitical news, the BIOSECURE Act was unexpectedly left out of the DoD’s spending bill. But that doesn’t mean the legislation is going anywhere, and the U.S. House of Representatives committee’s recent request that the FBI put together a briefing on GenScript Biotech highlights the ripple effect the act is having on the Chinese market.  Other news includes the busy regulatory activity from the FDA, approving the first new treatment in nearly a decade for a rare liver disease. This week, the agency will rule on full approval and a potential label expansion for Sarepta’s Elevidys.   And GLP-1s, as always, are making headlines. A new report from Global Data forecast the obesity market will reach $111 billion by 2033. This includes the blockbuster GLP-1s as well as alternatives being pursued by multiple companies. Just last week, Sytis Bio launched with a lead program consisting of an oral drug meant to mimic the effects of oral bypass surgery.   Finally, there’s some good news on the financing front, as J.P. Morgan raised over $500 million in its first biotech VC fund. It’s the latest sign of the uptick in VC funds devoted to biopharma and tech industries.

BioSpace's Lori Ellis and Chantal Dresner are bringing live updates from #DIA2024 in San Diego this week where the first day kicked off with an inspiring story from Tom and Emily Whitehead of the Emily Whitehead Foundation, setting the tone for patient-centric discussions. The following panel featuring Emer Cooke, Chair, ICMRA an Executive Director, European Medicines Agency; Peter Marks, Director, CBER, FDA; Carsten Linnerman, CEO, Neogene Therapeutics, AstraZeneca Group; Dean Kamen, Founder, DEKA; and Stacy Hurt, Chief Patient Officer, Parexel emphasized the imperative of collaboration to improve outcomes, between regulatory bodies as well as with patients. Tune in this week to hear more from the event!

The big news of this week so far was Monday’s FDA advisory committee for Eli Lilly’s Alzheimer’s drug donanemab, where the vote was unanimous in support of the anti-amyloid antibody. If approved, donanemab would be a direct competitor of Biogen and Eisai’s Leqembi, but analysts believe there’s plenty of room in the market for both, and even predict that donanemab’s potential approval could be beneficial for Leqembi in the long run by increasing investments in advocacy and infrastructure. The mood was decidedly different from last week’s FDA adcomm, which voted overwhelmingly against approving Lykos Therapeutics’ MDMA-assisted PTSD therapy. Interestingly, that vote came down less an hour before BioSpace Senior Editor Heather McKenzie hosted a panel at BIO where Dan Karlin, chief medical officer at MindMed, said he’d hoped the conversation would have been different. In other conference news, Eli Lilly and partners Boehringer Ingelheim and Zealand Pharma presented mid-stage results for their GLP-1 products in the MASH space at the Congress of the European Association for the Study of the Liver. These companies are eyeing a piece of the MASH market that Madrigal Pharmaceuticals first tapped into earlier this year when its therapy, Rezdiffra, was approved in March. Finally, CBRE published a report on challenges in finding R&D and manufacturing talent, and thousands of layoffs continue to hit the biopharma industry. According to our Layoff Tracker, there have been 14,000 positions cut this year, with BMS and Bayer being the unfortunate leaders, each with more than 1,500 layoffs underway. Still, analysts are optimistic that the second half of 2024 could be better, as an uptick in the financial markets might provide companies with money to spend on growing their workforces.

How do we ensure strategic vision and thoughtful implementation when pursuing new opportunities in science and technology? Our guests discuss challenges around reimbursement, intellectual property, change management and the critical nature of early engagement.  Additionally, we are joined by Tamei Elliott of DIA who shares insights on key discussions and themes of the upcoming meeting in San Diego. This is the third and final episode in our preparation for the roundtable discussion: ⁠⁠Pioneering New Frontiers: Advanced Drug Delivery Technologies and Cell/Gene Therapies in Combination Products⁠⁠ taking place at the ⁠⁠DIA 2024 Global Annual Meeting⁠⁠. James Wabby, Global Head, Regulatory Affairs, Emerging Technologies and Combination Products, AbbVie is chairing the discussion between Rob Schulz, President and Chief operating officer of Suttons Creek, William Daunch, Chief Technology Officer, Focal Medical, Inc, Andrea Gray, Biomedical Engineer Advisor, CBER, FDA, and Michael Lehmicke, Senior Vice President, Science and Industry Affairs, Alliance for Regenerative Medicine. Lori Ellis will be covering DIA Global Annual meeting 2024, June 16-20 in San Diego. Host  ⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠Lori Ellis⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠, Head of Insights, ⁠⁠⁠BioSpace⁠⁠⁠   Guests ⁠⁠James Wabby⁠⁠, Global Head, Regulatory Affairs, Emerging Technologies and Combination Products, ⁠⁠AbbVie, United States⁠⁠ ⁠⁠Rob Schulz⁠⁠, President and COO, ⁠⁠Suttons Creek, Inc., United States Tamei Elliott, Associate Director, Scientific Programs (Americas), DIA

Conference season kicked into high gear this week with the American Society of Clinical Oncology (ASCO) annual meeting in Chicago and the BIO International Convention in San Diego. Check out BioSpace’s ASCO24 Tracker with all the biggest data and other news from the cancer meeting, which wrapped up on Tuesday. Among the trial results highlighted at ASCO24 were BMS’ Phase III study results in liver cancer. There was also a lot of news on non-small cell lung cancer (NSCLC): Pfizer presented results from a Phase III trial evaluating its ALK inhibitor Lorbrena, BMS provided three updates on its immunotherapy Opdivo and Opdivo-based combinations, while Gilead unveiled additional details on a failed Phase III trial for its antibody-drug conjugate (ADC) Trodelvy. Currently, there is one ADC approved to treat NSCLC, AstraZeneca and Daiichi Sankyo’s Enhertu, for patients with HER2-mutated metastatic disease. While the companies’ investigational ADC Dato-DXd is making some headway against NSCLC, experts recently told BioSpace that more compelling surface targets for lung cancer are needed in order for ADCs to really make a difference. When it comes to M&A, Big Pharma continues to show strong interest in oncology, particularly ADCs and radiopharmaceuticals. However, last week’s biggest deal was Merck’s $3 billion acquisition of EyeBio and its first-in-class trispecific antibody Restoret, marking the company’s return to the ophthalmology space after nearly a decade. For now, it appears Big Pharma favors smaller M&A over megabillion-dollar deals. Recent buying activity also indicates a potential resurgence in the appetites of larger companies for psychiatric drug development.

Regulators suggest developers engage in discussions early, however those conversations cannot take place unless developers are sure of the asset's category. Existing confusion surrounding advanced therapy products may become more challenging as both science and technology evolve. With both developers and regulators, two risk adverse entities, addressing this evolution and continually updated regulations, advanced therapy product development continues to be a collaborative process. This is the second episode in our preparation for the roundtable discussion: ⁠Pioneering New Frontiers: Advanced Drug Delivery Technologies and Cell/Gene Therapies in Combination Products⁠ taking place at the ⁠DIA 2024 Global Annual Meeting⁠. James Wabby, Global Head, Regulatory Affairs, Emerging Technologies and Combination Products, AbbVie is chairing the discussion between Rob Schulz, President and Chief operating officer of Suttons Creek, William Daunch, Chief Technology Officer, Focal Medical, Inc, Andrea Gray, Biomedical Engineer Advisor, CBER, FDA, and Michael Lehmicke, Senior Vice President, Science and Industry Affairs, Alliance for Regenerative Medicine. Lori Ellis will be covering DIA Global Annual meeting 2024, June 16-20 in San Diego. Host  ⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠Lori Ellis⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠, Head of Insights, ⁠⁠BioSpace⁠⁠   Guests ⁠James Wabby⁠, Global Head, Regulatory Affairs, Emerging Technologies and Combination Products, ⁠AbbVie, United States⁠ ⁠Rob Schulz⁠, President and COO, ⁠Suttons Creek, Inc., United States

At its Investor Day 2024 event last week, AstraZeneca announced plans to reach $80 billion in total revenue by 2030—up from $45.8 billion in 2023—and to launch 20 new medicines by the end of the decade. Does AstraZeneca have the assets and developmental pipeline to hit this lofty target? Meanwhile, Bayer, BMS and Pfizer are looking to conserve cash through cost-savings plans that include layoffs. It’s a trend we’ll be keeping a close eye on as companies face potential implications from upcoming patent expirations, the Inflation Reduction Act’s Drug Price Negotiation Program and investor expectations. On the R&D front, Takeda took a deep dive into the hot molecular glue degrader space, signing a potential $1.2 billion exclusive licensing deal with China-based Degron Therapeutics. The new partners will be going after various oncology, neuroscience and inflammatory disease targets. Takeda follows Novo Nordisk, Roche’s Genentech and Merck, who have all recently struck R&D deals focused on molecular glues degraders. And the conference season again kicks into high gear this weekend with the American Society of Clinical Oncology (ASCO)’s annual meeting starting Friday in Chicago and the 2024 BIO International Convention beginning Monday in San Diego. BioSpace’s Heather McKenzie will be moderating a BIO panel focused on closing the gaps in neuropsychiatric drug development, so if you’re heading to San Diego, please pop in and say hello!

With every new advancement in either science or technology there is increased excitement as we see the potential. Cell and gene therapies (CGT) as combination products are emerging as the stage in the evolution of technology and science merging together. While the potential benefits for patients are great, there are risks and challenges that must be carefully considered. This discussion is part of one of a precursor of the roundtable discussion: Pioneering New Frontiers: Advanced Drug Delivery Technologies and Cell/Gene Therapies in Combination Products taking place at the DIA 2024 Global Annual Meeting. James Wabby, Global Head, Regulatory Affairs, Emerging Technologies and Combination Products, AbbVie is chairing the discussion between Rob Schulz, President and Chief operating officer of Suttons Creek, William Daunch, Chief Technology Officer, Focal Medical, Inc, Andrea Gray, Biomedical Engineer Advisor, CBER, FDA, and Michael Lehmicke, Senior Vice President, Science and Industry Affairs, Alliance for Regenerative Medicine. Lori Ellis will be covering DIA Global Annual meeting 2024, June 16-20 in San Diego. Host  ⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠Lori Ellis⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠, Head of Insights, ⁠BioSpace⁠   Guests James Wabby, Global Head, Regulatory Affairs, Emerging Technologies and Combination Products, AbbVie, United States Rob Schulz, President and COO, Suttons Creek, Inc., United States

Bayer’s recent layoffs of 1,500 mark a step toward CEO Bill Anderson’s stated goal of removing managerial layers at the company—“fewer bosses, fewer rules,” he told  The Wall Street Journal. The move follows BMS’ announcement that it will let go more than 2,000 people. In other news, J&J expanded its dermatology portfolio, paying $850 million in cash plus potential milestone payments for California-based Proteologix and its Phase I–ready atopic dermatitis candidate. J&J is competing with Eli Lilly, which continues to actively pursue this space as well, with positive Phase IIIb results for its candidate in March. Roche’s Genentech is looking to jump into the hot GLP-1 space with positive results from a Phase Ib trial for its investigational weight-loss treatment CT-388. The once-weekly subcutaneous injection was part of Roche’s acquisition of Carmot Therapeutics in December for $2.7 billion—a hefty sum as Roche looks to compete with frontrunners Novo Nordisk and Eli Lilly. Separately, Lilly announced a late-stage win for its weekly insulin product, which outperformed Novo’s Tresiba in adults with type 2 diabetes. We also touch on Novo’s latest positive study on Wegovy and a couple of big approvals last week, including BMS’ third indication for its CAR T therapy Breyanzi and Amgen’s Imdelltra, the first bispecific T-cell engager therapy for advanced small cell lung cancer. Finally, we discuss this week’s special edition of ClinicaSpace, focused on psychedelics for mental health disorders. There’s a pretty extensive pipeline of therapies of psilocybin, DMT and more, for depression, substance abuse disorders and other neuropsychiatric diseases. The first psychedelic therapy for a neuropsychiatric disease—Lykos Therapeutics MDMA for PTSD—is up for FDA approval this summer. The FDA is convening an adcomm on June 4 to discuss the therapy.

Bayer made news this week announcing 1,500 layoffs as part of an effort to save €500 million ($540 million) in 2024 and €2 billion ($2.16 billion) in 2026, reminiscent of BMS’ announcement of a major restructuring that will involve 2,200 layoffs and aims to save $1.5 billion by the end of 2025. Bayer’s news just about rounds out the Q1 2024 earnings season highlighted by strong sales of GLP-1 weight loss drugs. However, it was Amgen that emerged as a big winner, with revenue increasing over 22% from Q1 2023. In other news this week, Takeda took another deep dive into the Alzheimer’s space with a deal worth up to $2.2 billion with AC Immune for global rights to an immunotherapy that targets toxic forms of amyloid beta. And the BIOSECURE Act got an update, now naming WuXi Biologics among the Chinese firms that U.S. companies are expected to cut ties with, and setting a deadline of 2032. Finally, we discuss the major takeaways from last week’s 2024 American Society of Gene & Cell Therapy (ASGCT) annual meeting. While the cell and gene therapy space faces many challenges, promising updates included Regeneron’s news that its gene therapy improved hearing in two young deaf children. And throughout the week, Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research (CBER), spoke with positivity on how the community is now working to address the challenges it now faces, touching on topics such as accelerated approval, global regulatory convergence and the Bespoke Gene Therapy Consortium, aimed at accelerating the delivery of AAV-based gene therapies for rare diseases.

12 billion dollars: one of the boldest women’s health research moves an administration has made for women’s health research. On a global scale, this has caused countries and sovereign states to start investing in women’s health. But in the United States, the funding is not guaranteed. It depends on congressional approval, which is complicated during an election year. As the United States continues to grapple with politics and science meeting, the global healthcare sector is already beginning to reframe the language and invest in women’s health research. Host  ⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠Lori Ellis⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠, Head of Insights, ⁠⁠BioSpace⁠⁠   Guests ⁠⁠Sans Thakur⁠⁠, Founder and Chairwoman of ⁠⁠Tower Capital⁠⁠ ⁠⁠Chia Chia Sun⁠⁠, Chief Commercial Officer at ⁠⁠Fab Biopharma⁠⁠ ⁠⁠Elisa Cascade⁠⁠, Chief Product Officer of at ⁠⁠Advarra⁠⁠ Shawana Moore, Medical Advisor on the ⁠⁠Advisory Council for Healthy Women⁠⁠ ⁠⁠Katie Schubert⁠⁠,  President and Chief Executive Officer of the ⁠⁠Society for Women’s Health

In this edition of The Weekly, Managing Editor Jef Akst and News Editor Greg Slabodkin share their insights from the American Society of Gene & Cell Therapy’s 27th annual conference, including advances around adeno-associated viruses and the ongoing discussion about the FDA’s accelerated approval program and how it relates to rare diseases. Also, on Tuesday, Eli Lilly announced that an advisory committee meeting will be held for its Alzheimer’s drug donanemab on June 10. Lilly is aiming for full approval of the anti-amyloid antibody after accelerated approval was denied in January 2023. And the race between Vertex and bluebird bio’s gene therapies Casgevy and Lyfgenia is heating up.

In this special edition of The Weekly, we discuss the much-anticipated annual meeting of the American Society of Gene & Cell Therapy in Baltimore. It starts Tuesday and will hit on wide-ranging issues facing the cell and gene therapy (CGT) space, from new therapeutic advances to safety concerns and regulatory considerations to manufacturing and commercialization. Big hitters in the CGT space will be there. Sarepta, whose gene therapy for Duchenne muscular dystrophy Elevidys received accelerated approval in June 2023, will be presenting, as will CRISPR Therapeutics, which in the last few months brought to market Casgevy, its Vertex-partnered CRISPR-based therapy, for sickle-cell disease and beta thalassemia. There will also be plenty of big names, including Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research. Marks will be speaking in two sessions at ASGCT 2024, one on global regulatory convergence and the other on the regulation of CGTs, from IND to BLA. Stay tuned for this week’s regular episode on Wednesday when we discuss what we’re seeing and hearing in Baltimore.

J&J and BMS’ challenges to Medicare drug price negotiations shut down in federal court less than a week after BMS announced it was laying off more than 2,000 employees.    Earlier this week, a federal judge in New Jersey added fuel to the ongoing legal battle between Big Pharma and the government over the Inflation Reduction Act. The judge on Monday dismissed claims from Johnson & Johnson and Bristol Myers Squibb that Medicare’s drug price negotiations program is unconstitutional.     The big news last week also involved BMS, which announced it will let go 2,200 people by the end of 2024 in what the company is calling a “strategic productivity initiative.” Layoffs are already underway in New Jersey, California and elsewhere, and may be related to the pharma giant’s recent M&A spending spree.   Elsewhere around the industry, Q1 earnings season rolls on, with Sanofi and AstraZeneca also announcing cuts to their pipeline, while Gilead, Merck, and Lilly all touted numbers that exceeded expectations.  Finally, on the regulatory front, X4 Pharmaceuticals got a win this week with the approval of its first commercial drug—and the first therapy for an ultra-rare disease called WHIM.

The potential impacts on clinical trial outcomes warrant a discussion surrounding the current definition of women’s health. In the second episode on our series on women's health, we discuss what may happen to future generations if women, the custodians of generational health, are not comfortable sharing complete medical histories. Additionally, we discuss how broadening the definition will open funding to address women’s health. Host  ⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠Lori Ellis⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠, Head of Insights, ⁠BioSpace⁠   Guests ⁠Sans Thakur⁠, Founder and Chairwoman of ⁠Tower Capital⁠ ⁠Chia Chia Sun⁠, Chief Commercial Officer at ⁠Fab Biopharma⁠ ⁠Elisa Cascade⁠, Chief Product Officer of at ⁠Advarra⁠ Shawana Moore, Medical Advisor on the ⁠Advisory Council for Healthy Women⁠ ⁠Katie Schubert⁠,  President and Chief Executive Officer of the ⁠Society for Women’s Health

Sanofi will cut an undisclosed number of jobs as it revamps its U.S. vaccines commercial operations, as well as 99 employees from its Belgian sites—moves that are part of a full pipeline reprioritization. Meanwhile, the company got a win this week, with its investigational BTK inhibitor rilzabrutinib generating positive results in a Phase III study for adult patients with persistent or chronic immune thrombocytopenia. Sanofi will present its Q1 earnings tomorrow. Meanwhile, the FDA announced an update to the boxed warnings it now requires for CAR T cell therapies regarding the heightened risk of secondary T cell malignancies. Additional requirements involve various aspects of the label, including the warnings and precautions, post-marketing experience, patient counseling information and medication guide sections. And finally, GLP-1s are once again making headlines, as Eli Lilly’s Zepbound produced positive Phase III results that could earn the weight-loss drug a label expansion into sleep apnea and a new company, New York–based Metsera emerged from stealth with $290 million in financing. The startup’s pipeline includes multiple GLP-1 agonists in early-stage development.

The past two weeks have seen Amylyx lose its only marketed product but receive numerous accolades for its decision to voluntarily withdraw Relyvrio after a failed Phase III trial in amyotrophic lateral sclerosis. Analysts and insiders spoke with BioSpace this week about the company's future and a possible regulatory precedent for other drugs approved based on a single trial. Elsewhere, antibody-drug conjugates continue to steal the show in oncology. At the American Association for Cancer Research’s annual meeting, Merck and Kelun Biotech presented Phase I/II data showing that their TROP2-directed ADC elicited promising disease control and a potential survival benefit in gastric cancer patients, and Bristol Myers Squibb–backed TORL netted $158 million in an oversubscribed Series B to advance its pipeline of novel ADC candidates. Finally, drug shortages in the U.S. have reached an all-time high, according to the latest data from the American Society of Health-System Pharmacists, with both basic and life-saving products on the list.

Last month, President Biden signed an Executive Order on Advancing Women’s Health Research and Innovation, signalling the significance of women's health to the government and consequently investors. While women’s clinical trial participation has increased, clinical trials still largely are not designed for women. In this episode, our guests discuss the many areas where trials can be improved, such as human and historical diagnosis and screening biases, accessibility, data capturing, and more. They further discuss the importance of women’s health for the future. Host  ⁠⁠⁠⁠⁠⁠⁠⁠⁠Lori Ellis⁠⁠⁠⁠⁠⁠⁠⁠⁠, Head of Insights, BioSpace   Guests Sans Thakur, Founder and Chairwoman of Tower Capital Chia Chia Sun, Chief Commercial Officer at Fab Biopharma Elisa Cascade, Chief Product Officer of at Advarra Shawana Moore, Medical Advisor on the Advisory Council for Healthy Women Katie Schubert,  President and Chief Executive Officer of the Society for Women’s Health

Conference season is in full swing, with the American Association for Cancer Research and American College of Cardiology concurrently holding their annual meetings in San Diego and Atlanta, respectively. From opposite coasts, biopharma companies are presenting their latest data in the cancer and cardiology spaces, both hits and misses alike. Oncology continues to be a hot area for investment, with several highly anticipated readouts coming down the pike. In addition to all the data coming out of AACR, conference organizers this year put together the meeting’s first-ever industry event, bringing together investigators, investors and Big Pharma. Over at ACC, one exciting race to watch will be between Ionis and Arrowhead, both of whom presented positive data on their ApoC3-targeting drugs for diseases associated with elevated triglyceride levels. Meanwhile, as the BIOSECURE Act sits before Congress, biopharma companies detail the potential fallout to the growing geopolitical tensions between the U.S. and China.

WuXi AppTec was once again in the news this week, with speculation that the China-based biotech allegedly handed a U.S. client’s intellectual property over to the Chinese government without consent. Meanwhile, Lonza’s $1.2 billion buy of a Roche biologics plant in California—one of the world’s largest biologics manufacturing facilities by volume—bodes well for the CDMO market, and BMS pulls ahead of Amgen in the race to bring a fully approved KRAS inhibitor to market for patients with certain types of non-small cell lung cancer, acing a confirmatory Phase III trial for Krazati.

The pharmaceutical industry is facing critical attention, particularly around drug pricing and development costs. Drug development cost is about 10% of the total healthcare spend in the United State. Broader issues such as local monopolies, utilization, unit, and costs and local monopolies, politics and a fragmented payer system contribute to the increasingly high costs to patient. Challenging as these issues are, they must be addressed to lower patient costs. In this episode, our guests discuss more efficient drug development practices but also some of the broader issues within the United States healthcare system and the IRA’s impacts on not only US patients but the world.  Host  ⁠⁠⁠⁠⁠⁠⁠⁠⁠Lori Ellis⁠⁠⁠⁠⁠⁠⁠⁠⁠, Head of Insights, BioSpace     Guests ⁠⁠Ali Pashazadeh⁠⁠, CEO,Treehill Partners ⁠⁠Dan Smithey⁠⁠, President, CEO and Co-founder, Serán BioScience ⁠⁠Matthew Price⁠⁠, COO and Co-founder, Promontory Therapeutics ⁠⁠Peter Rubin⁠⁠, Executive Director, No Patient Left Behind ⁠⁠Rob Williamson⁠⁠, President and COO, Triumvira Immunologics

It was another busy week! Bayer reduced its executive suite from 14 to 8 as part of a major restructuring. Meanwhile, the government announced it will pay for Wegovy for patients with heart disease after the Novo Nordisk therapy was approved earlier this month for the cardiovascular indication. Plus, Mirador’s emergence from stealth and the FDA’s approval of the third therapy for Duchenne muscular dystrophy in less than a year.

This week, we're talking money! Following the release of BioSpace's 2024 Salary Report, we discuss salary trends and how they are impacting the biopharma workforce. Considering growth rates, we also discuss asking for raises vs looking for a new job and what salary trends may spell out for the rest of the year. Guest Chantal Dresner, VP of Marketing, BioSpace Angela Gabriel, Content Manager - Careers, BioSpace Host  ⁠⁠⁠⁠⁠⁠⁠Lori Ellis⁠⁠⁠⁠⁠⁠⁠, Head of Insights, BioSpace

Phew, what a week! One of the most anticipated FDA decisions of the year dropped last week when the regulator approved Madrigal’s Rezdiffra as the first therapy for MASH (formerly known as NASH).   Then on Friday, BioSpace kept a close eye on an advisory committee discussing J&J’s Carvykti and BMS’s Abecma, two CAR-T therapies seeking label expansions as earlier lines of treatment in multiple myeloma despite the risk of early death. Also discussed: BIO’s decision from last week to cut ties with the China-based biotech WuXi AppTec.

Continuing on from our previous episode "The gap is where the pain is", this episode focuses on a healthy discussion regarding the IRA, particularly the unintended consequences to small molecule development within the industry and for patients. Topics explored in this discussion are potential reasons behind the small molecule negotiation timeline decision, the antibody drug conjugates (ADC) boom, the investment environment, and positive steps the government is making through legislation to equalize the negotiation timeline. Host  ⁠⁠⁠⁠⁠⁠⁠⁠Lori Ellis⁠⁠⁠⁠⁠⁠⁠⁠, Head of Insights, BioSpace   Guests ⁠Ali Pashazadeh⁠, CEO,Treehill Partners ⁠Dan Smithey⁠, President, CEO and Co-founder, Serán BioScience ⁠Matthew Price⁠, COO and Co-founder, Promontory Therapeutics ⁠Peter Rubin⁠, Executive Director, No Patient Left Behind ⁠Rob Williamson⁠, President and COO, Triumvira Immunologics

The weight loss market is the gift that keeps on giving—at least for Novo Nordisk—as Wegovy continues to demonstrate its prowess with label expansion. Also discussed: biosimilar approvals for Fresenius Kabi, and Sandoz. More news at the forefront of Alzheimer's treatment: Eli Lilly's donanemab decision date got pushed and now the FDA wants to discuss the drug’s safety and efficacy at an upcoming adcomm. And finally, politics: President Biden's State of the Union address ruffles feathers as he strikes out at Big Pharma and drug prices, and how the industry is reacting to the election.

These days you can hardly move without figuratively bumping into antibody-drug conjugates (ADCs). This week we discuss Pfizer's strategic priorities for oncology - focused on ADCs and less on small molecules - and how the market is being influenced by the IRA.

When it comes to drug pricing, there have been (and are) bad actors in the industry, but the conversation usually ends there. Five KOLs from different parts of the industry come together to discuss the complexities surrounding drug spend and pricing, innovation, funding, and the overall healthcare system. This episode explores the drug pricing gap between consumer experience and industry norms. Join Lori and our KOLs as we explore the drivers of healthcare costs, the CBO, rebates, and overall spend in this new series. Host  ⁠⁠⁠⁠⁠⁠⁠Lori Ellis⁠⁠⁠⁠⁠⁠⁠, Head of Insights, BioSpace   Guests Ali Pashazadeh, CEO,Treehill Partners Dan Smithey, President, CEO and Co-founder, Serán BioScience Matthew Price, COO and Co-founder, Promontory Therapeutics Peter Rubin, Executive Director, No Patient Left Behind Rob Williamson, President and COO, Triumvira Immunologics

This week Lori, Greg and Tyler discuss AbbVie: how longtime CEO Richard Gonzalez navigated Humira's LOE, his victorious retirement announcement, and the future of biosimilars. Also discussed: Earlybird Health's $186m VC fund; and definitely not the IRA.

This week Lori, Greg and Tyler discuss the Accelerated Approval of Amtagvi, the first one-time cell therapy for solid tumors and the first TIL therapy; the FTC and HHS probe into generic drug shortages and some recent ADC-focused raises from ProfoundBio and Firefly Bio.

This week, Greg, Heather and Tyler discuss reaction to Novo Nordisk's purchase of Catalent and speculate on what that means for existing manufacturing contracts, customers and consequences with regulators; as well as discussion around the ongoing issues with drug shortages and manufacturing challenges and whether it the move will help accelerate manufacturing for Wegovy and Ozempic. Plus, what's next for Alzheimer's treatment following Biogen's withdrawal of Aduhelm? As mentioned in this episode: you can subscribe to ClinicaSpace for our latest special edition on Leqembi.

This week Lori, Greg and Tyler discuss drug pricing reforms. CMS sent offers to manufacturers of the 10 drugs that have been selected for Medicare price negotiations. What's the best way forward that benefits patients while still supporting the innovation that makes these drugs possible? How will the election impact negotiations? Also discussed earnings including Novo Nordisk, who beat analyst expectations largely due to sales of Ozempic and Wegovy, AbbVie, Merck, and GSK.

Almost 30,000 patients have now been treated with CAR-T cell therapies since it was the first approval in 2017. In November 2023, the FDA launched a probe into malignancies caused by CAR T therapies, and just last week the agency called for a class-wide box warning on all commercial CAR-T therapies with ongoing investigations into cases of secondary malignancies. How do we approach this balancing act of treatment and side effects? Also this week: IPOs march on with ArriVent, CG Oncology, Alto Neuroscience, Fractyl Health; and what's happening re: Congressional Budget Office, Centers for Medicare & Medicaid Services and drug pricing. Join BioSpace's Lori Ellis, Greg Slabodkin and Tyler Patchen as they discuss.

Following a challenging year of layoffs and limited funding, Chantal Dresner, VP of Marketing at BioSpace, discusses the findings of our most recent Employment Outlook Report including unemployment, anticipated job search activity, hiring trends and current workforce sentiment.

The first surge of IPO activity this year sees Alto Neuroscience, ArriVent Biopharma, CG Oncology, Kyverna Therapeutics and Metagenomi making announcements. Lori, Greg and Tyler also discuss gene therapy pricing including Vertex/CRISPR's Casgevy.

The team comes together to discuss the dominant themes of what was an incredibly busy week of events in San Francisco. There was an air of hope and optimism concerning biopharma funding - but will it last? Lori Ellis, Greg Slabodkin and Tyler Patchen discuss.

We're still in San Francisco covering the JP Morgan Healthcare Conference and Biotech Showcase, bringing you key themes from day three: disruptive AI ⁠is still a hot topic including generative AI; M&A and VC funding; and general whiffs of cautious optimism. Join Lori, Greg and Tyler as they discuss what's been top of mind today. You can ⁠⁠⁠follow our coverage⁠⁠⁠ as our team adds updates throughout the day.

Once again we're recording from San Francisco covering the JP Morgan Healthcare Conference and Biotech Showcase, bringing you key themes from day two: AI, cell and gene therapy innovation and pricing, and the risks of CAR T cell therapy. Join Lori, Greg and Tyler as they discuss what's been top of mind. You can ⁠⁠follow our coverage⁠⁠ as our team adds updates throughout the day. Join us tomorrow for Day 3 highlights!

Hello from San Francisco! BioSpace is reporting from #JPM2024 bringing you key takeaways and highlights from day one. Join Lori, Greg and Tyler as they discuss what's moving and shaking. You can ⁠follow our coverage⁠ as our team adds updates throughout the day. Get Greg's take on ⁠what this year might hold⁠ in store and how the Biden administration's policies may ⁠play a role in discussions⁠. Join us tomorrow for Day 2 highlights!

The last year has demonstrated that a sound financing strategy and compelling company narrative are essential to securing funding – it’s become more important than ever that investors are excited before taking the plunge. Given the current economic environment that is causing challenges in biopharma, how should companies be approaching funding in 2024? Listen to this discussion on different kinds of funding options available, how companies can optimize their chances to secure funding and how they should be assessing VCs in addition to alternative partners – particularly if your company will be seeking funding at JPM next week. Our host and guests also discuss current challenges, how to weather adversity, and what investment trends are anticipated for next year. You can also watch the roundtable on BioSpace. Host  ⁠⁠⁠⁠⁠⁠Lori Ellis⁠⁠⁠⁠⁠⁠, Head of Insights, BioSpace   Guests Jeff Burton, CFO, Halia Therapeutics Karen Harris, CFO and Head of Mission-Related Investing, The Alzheimer's Drug Foundation Rob Williamson, President and COO, Triumvira Immunologics

Welcome to 2024! This week we're discussing BioSpace's tenth annual list of the hottest new life sciences companies in the U.S. NextGen Class of 2024⁠ includes organizations that launched between September 2022 and September 2023 with a Series A funding.  BioSpace's Greg Slabodkin, Heather McKenzie and Tyler Patchen discuss.

How can startup leaders support long-term sustainable growth and investment? BioSpace’s Lori Ellis speaks with venture capital guests Ansbert Gadicke, Martin Gershon and Mike Goguen for their advice and recommendations on how biopharma startups should approach funding. Host  ⁠⁠⁠⁠⁠Lori Ellis⁠⁠⁠⁠⁠, Head of Insights, BioSpace   Guests ⁠Ansbert Gadicke⁠, Managing Director, MPM Capital ⁠Martin Gershon⁠, Managing Partner & CIO, Endeavor Venture Fund ⁠Mike Goguen⁠, Founder and Managing Partner, Two Bear Capital

Biden administration flexes regulatory muscles, putting pressure on the biopharma industry over ‘price gouging’ and invoking march-in rights; meanwhile Sanofi throws in towel over threat of injunction  Renewed interest in psychedelics after MindMed’s Phase IIb trial of its LSD-based candidate meets primary endpoint in patients with generalized anxiety disorder.   Bluebird bio changes its tune, signing an ‘outcomes- based’ agreement with commercial payer, and Pfizer struggles continue. BioSpace's Lori Ellis, Greg Slabdodkin and Tyler Patchen discuss. And that's a wrap on 2023! We'll see you in the New Year.

What needs to happen for funding in biopharma to bounce back? BioSpace’s Lori Ellis discusses the macroeconomic environment and biopharma funding outlook with venture capital guests Ansbert Gadicke, Martin Gershon and Mike Goguen.  Host  ⁠⁠⁠⁠Lori Ellis⁠⁠⁠⁠, Head of Insights, BioSpace   Host  ⁠⁠⁠⁠⁠⁠Lori Ellis⁠⁠⁠⁠⁠⁠, Head of Insights, BioSpace   Guests ⁠⁠Ansbert Gadicke⁠⁠, Managing Director, MPM Capital ⁠⁠Martin Gershon⁠⁠, Managing Partner & CIO, Endeavor Venture Fund ⁠⁠Mike Goguen⁠⁠, Founder and Managing Partner, Two Bear Capital

That was Week with a capital W. Two major FDA approvals for sickle cell came through on Friday - Casgevy, the first-ever CRISPR-based gene editing therapy from Vertex and CRISPR Therapeutics, and bluebird bio’s Lyfgenia, which comes with a hefty price tag and a black box warning.  Axcella announced its closure; what does the future of long-covid treatments look like, especially as attention shifts to different markets like ADCs and neurological treatments?  Also discussed - Vanda drops $100m on rights to MS drug Ponvory, AI regulatory developments in Europe.  BioSpace's Lori Ellis, Greg Slabodkin and Tyler Patchen discuss.

This is the second part of our discussion on the evolution of artificial intelligence and its impact on life sciences with guests from Microsoft and IQVIA. We dive into regulation, real-time management, and AI's various applications and how it can streamline different processes. We also discuss the cost and affordability of AI; the tremendous investment required and when holding off on new tech is valuable. Hear how AI is highlighting society's flaws and may become the catalyst for societal change. Host  ⁠⁠⁠⁠Lori Ellis⁠⁠⁠⁠, Head of Insights, BioSpace   Guests  ⁠Matt O’Donnell⁠, Industry Executive Health & Life Sciences, Microsoft  ⁠⁠⁠Mike King⁠⁠⁠⁠, Senior Director of Product and Strategy, IQVIA

Over the last year, the reaction to generative AI has changed - and so have behaviors. People are integrating AI to become more productive and it is happening now in healthcare and life sciences. Hear from senior leaders at Microsoft and IQVIA to get their take on how generative AI is impacting productivity, employee engagement and how to mitigate risks.  Host  ⁠⁠⁠Lori Ellis⁠⁠⁠, Head of Insights, BioSpace   Guests  Matt O’Donnell, Industry Executive Health & Life Sciences, Microsoft  ⁠⁠Mike King⁠⁠⁠, Senior Director of Product and Strategy, IQVIA

The weight loss market sees more ups and downs - Altimmune joins the fray and sees a stock jump while Pfizer experiences setbacks; and European Medicines Agency seeks additional information as part of its ongoing review of the potential risk of suicide and self-harm thoughts associated with the class. Meanwhile, AbbVie buys ImmunoGen for a cool $10B and scores a win with Teliso-V as ADC momentum continues to build. Plus, the FDA investigates malignacies linked to CAR-T. BioSpace's Lori Ellis, Greg Slabodkin and Tyler Patchen discuss.

Patient behavior has always had an influence on clinical trials - so how do we contend with missing and/or irregular data now that it is feeding AI models? And how do providers continue to work with patients to optimize and grow data pools? This is the third and final part of a discussion focused upon data bias, accuracy, access and the future of AI in drug development. Host  ⁠⁠Lori Ellis⁠⁠, Head of Insights, BioSpace   Guests  ⁠⁠Paul Agapow⁠⁠, Director of Innovation, Data Science and Strategy, GlaxoSmithKline⁠⁠⁠ ⁠Mike King⁠⁠, Senior Director of Product and Strategy, IQVIA  ⁠⁠Nindhana Paranthaman⁠⁠, Executive Medical Director, Exelixis  ⁠⁠Moritz von Stosch⁠⁠, Chief Information Officer, DataHow

The breadth of AI in healthcare applications is broad. How much human oversight is necessary or preferred when leveraging AI in the interest of patient safety? Listen to this in-depth discussion on how AI can help identify end-to-end data weaknesses, as well as broader implications regarding the inevitability of human interaction. This is part two of a discussion focused upon data bias, accuracy, access and the future of AI in drug development. Host  ⁠Lori Ellis⁠, Head of Insights, BioSpace   Guests  ⁠Paul Agapow⁠, Director of Innovation, Data Science and Strategy, GlaxoSmithKline⁠ Mike King⁠, Senior Director of Product and Strategy, IQVIA  ⁠Nindhana Paranthaman⁠, Executive Medical Director, Exelixis  ⁠Moritz von Stosch⁠, Chief Information Officer, DataHow

This week we talk struggles with GLP-1 drug shortages and what that might mean for Novo/Lilly competitors; Regeneron and Sanofi positive results for ⁠⁠Dupixent⁠⁠ in COPD. Plus, Merck buys Caraway, Beigene's deal with Ensem, ups and downs for Flagship. Join BioSpace's Lori Ellis, Greg Slabodkin and Tyler Patchen as they discuss the most important biopharma news this week.

Last Thursday, the U.K.’s Medicines and Healthcare products Regulatory Agency (MHRA) approved CRISPR/Cas9 gene-edited therapy exagamglogene autotemcel (exa-cel). Will the FDA follow suit? What can patients expect the price tag to be? Plus, a good handful of approvals for AstraZeneca, Pfizer and Astellas' Xtandi, and Keytruda. Join BioSpace's Lori Ellis, Greg Slabodkin and Heather McKenzie as they discuss the news this week.

This is part one of a discussion focused upon data bias, accuracy, access and the future of AI in drug development. Topics explored are ROI, human bias, data challenges, data management plans, and human expertise. Host Lori Ellis, Head of Insights, BioSpace  Guests Paul Agapow, Director of Innovation, Data Science and Strategy, GlaxoSmithKlineMike King, Senior Director of Product and Strategy, IQVIA Nindhana Paranthaman, Executive Medical Director, Exelixis Moritz von Stosch, Chief Information Officer, DataHow

Last week, the FDA approved Eli Lilly's obesity drug Zepbound, creating an anticipated intense competition between it and Novo Nordisk's Wegovy. BioSpace's Greg Slabodkin, Tyler Patchen and Lori Ellis discuss the weight loss race and the future of this drug class. Plus, a quick recap on Valneva, the FDA's first Chikungunya vaccine approval.

This week (our inaugural episode!) BioSpace's Greg Slabodkin, Tyler Patchen and Lori Ellis discuss the good, the bad and the ugly of biopharma's reported Q3 earnings. They also tackle what's going on at Pfizer, ADCs, deals and more. Want to dive deeper on BioSpace? Read more on Pfizer's recent cuts⁠ as part of their cost-savings program and more insight on Q3 earnings for Gilead, ⁠⁠Novo Nordisk,⁠⁠ ⁠⁠Moderna⁠⁠, ⁠⁠Vertex⁠⁠, ⁠⁠BMS⁠⁠, ⁠⁠Lilly,⁠⁠ ⁠⁠GSK⁠⁠. Meanwhile, industry-wide ⁠layoffs⁠ march on and in case you missed it - bankruptcies reached a record high.