BioSpace

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 The FDA issued on December 2 a draft guidance outlining specific product types for which the agency believes six-month non-human primate toxicity testing can be eliminated or reduced.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.

Host

Jennifer Smith-Parker, Director of Insights, BioSpace

Guests

Patrick Smith, Senior Vice President, Translational Science, Certara

Stacey Adam, PhD, Vice President of Science Partnerships, Foundation for the National Institutes of Health

Disclaimer: 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.

Host

Chantal Dresner, Vice President of Marketing, BioSpace

Guest

Leslie Loveless, Co-CEO and Managing Partner, Slone Partners

Disclaimer: 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.

Host

⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠Lori Ellis⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠, Head of Insights, BioSpace

Guests

Oliver Eden, Senior Business Unit Director, Jabil

Travis Webb, Chief Scientific Officer, PII

Disclaimer: 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.

Hosts
Jennifer Smith-Parker, Director of Insights, BioSpace
Lori Ellis, Head of Insights, BioSpace

Guests

Dr. Jihye Jang-Lee, Director of Technical Services, Element Materials Technology

Dr. Khanh Courtney, Biologics Technical Strategy Manager, Element Materials Technology

Disclaimer: 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.

Hosts
Jennifer Smith-Parker, Director of Insights, BioSpace
Lori Ellis, Head of Insights, BioSpace

Guests

Dr. Jihye Jang-Lee, Director of Technical Services, Element Materials Technology

Dr. Khanh Courtney, Biologics Technical Strategy Manager, Element Materials Technology

Disclaimer: The views expressed in this discussion by guests are their own and do not represent those of their organizations.