BioSpace

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news, BioSpace recaps the now nine-month-old Commissioner’s National Priority Voucher pilot program and unpacks new draft guidance on animal testing alternatives.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. 

Host

Jennifer Smith-Parker, Director of Insights, BioSpace

Guests

Viswa Colluru, CEO & Founder, Enveda

Akshay Rai, Principal, Healthcare & Biotech Investments, Premji Invest

Disclaimer: 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.

Host

⁠Jennifer C. Smith-Parker⁠, Director of Insights, BioSpace

Guests

Oxana Iliach, Senior Director of Regulatory Strategy, Certara

Vera Pomerantseva, Director of Product Management for RBQM, eClinical Solutions

Disclaimer: 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.

Host

Annalee Armstrong, Senior Editor, BioSpace

Guests

Emile Nuwaysir, CEO, Stylus Medicine

Jason Fontenot, Chief Scientific Officer, Stylus Medicine

Disclaimer: 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.

Host

⁠Jennifer Smith-Parker⁠, Director of Insights, BioSpace

Guests

Ram May-Ron, Managing Partner, FreeMind Group

Ravi Kiron, Managing Director, Biopharma Strategy Advisors

Disclaimer: 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.

Host

Jennifer Smith-Parker, Director of Insights, BioSpace

Guests

⁠Indu Navar⁠, CEO and Founder, EverythingALS

Dr. Olga Uspenskaya, Chief Medical Officer, VectorY Therapeutics

Disclaimer: 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.  

Hosts

Jef Akst, Managing Editor, BioSpace

Heather McKenzie, Senior Editor, BioSpace

Annalee Armstrong, Senior Editor, BioSpace