BioSpace

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as Zepbound bests CagriSema in a head-to-head trial sponsored by Novo; The FDA kicked off Rare Disease Week, providing draft guidance on its new plausible mechanism pathway, while a bipartisan senate hearing on Thursday will focus on the authorization process for rare conditions; Another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Meanwhile, the FDA kicked off Rare Disease Week with draft guidance on the new Plausible Mechanism Pathway for personalized therapies that was first teased in November.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.  

Hosts
Jef Akst, Managing Editor, BioSpace
Heather McKenzie, Senior Editor, BioSpace
Annalee 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.

Host
Annalee Armstrong, Senior Editor, BioSpace

Guests
Rena Denoncourt, Chief Operating Officer, Corsera Health
Meredith Kaya, Chief Financial Officer, Corsera Health

Disclaimer: 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.

Host
Jennifer Smith-Parker, Director of Insights, BioSpace

Guests
Jane Hughes, President of R&D and Co-founder, Verdiva Bio
Jon Rees, CEO and Co-founder, MitoRx Therapeutics

Disclaimer: 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.