Sarepta CEO Doug Ingram is stepping down

Doug Ingram is stepping down as Sarepta Therapeutics' CEO after a nearly decade-long run defined by aggressive regulatory wins in Duchenne muscular dystrophy and, more recently, a stock collapse and deep scrutiny into the safety of the company's treatments.

Ingram is retiring for family reasons, he said — his wife and son were recently diagnosed with myotonic dystrophy, a form of muscular dystrophy that Sarepta actually started working on in 2024 through a new partnership. He described the discovery as "a fairly shocking and ironic twist of fate."

Sarepta is in search of a successor, STAT's Jason Mast reports. Ingram will retire by the end of the year, if a new CEO is not found before then.

REGULATION

FDA rejection is a reality check on agency rhetoric

An experimental cell therapy for an ultra-rare, often fatal post-transplant blood cancer appeared to be on track for FDA approval last year. Reviewers were said to have been supportive of the treatment, developed by Atara Biotherapeutics and Pierre Fabre Pharmaceuticals, once the companies resolved some manufacturing issues. But in January the agency reversed course and rejected it, citing insufficient clinical evidence, STAT's Adam Feuerstein writes.

The drug, Ebvallo, is already approved in Europe based on the same single-arm trial data. It showed tumor responses in about half of treated patients with no major safety concerns, in a disease where survival is often measured in months and alternatives are scarce.

This represents a "complete reversal that I can't help but think was due to the FDA's new leadership," one former FDA employee told STAT. The decision is particularly notable given recent public pledges by Commissioner Marty Makary to exercise regulatory flexibility for rare disease treatments.

rare disease

Regulatory shifts disappoint rare disease community

Two years after Megan Selser learned her infant son Ben had Hunter syndrome — a devastating, often fatal rare disease — she believed gene therapies designed to alter the disease's course might give him a chance he otherwise wouldn't have.

Instead, STAT's Jason Mast writes, the FDA has rejected one experimental treatment for the condition, along with several other rare disease gene therapies over the past year. The decisions are raising concerns that regulators under new leadership are pulling back from the flexibility once championed for ultra-rare conditions.

The applications were imperfect, often relying on biomarkers and single-arm trials in diseases where randomized studies are ethically and logistically fraught. But families and investors alike are now grappling with a deeper uncertainty: whether prior FDA guidance still holds, and whether the evidentiary bar has quietly shifted.

adam's biotech scorecard

On the upcoming readout of Xenon's epilepsy drug

Xenon Pharmaceuticals is up next in the parade of stock-moving readouts, STAT's Adam Feuerstein writes in his Biotech Scorecard newsletter (sign up, STAT+ subscribers!) Phase 3 data for its epilepsy drug, azetukalner, are due in March.

The company is running two identical trials, called X-TOLE2, in roughly 360 patients each. It has a straightforward goal in focal onset seizures: To achieve a statistically significant reduction in seizures over 12 weeks. Ideally, the company is looking for something close to the Phase 2b result, where the 25 mg dose cut seizures by 53% versus 18% for placebo and had a 55% response rate.

"Our mantra for X-TOLE2 has been 'just replicate X-TOLE,'" Xenon CEO Ian Mortimer told Adam. "In epilepsy, you do get good consistency and reproducibility across studies, so that should give us confidence going into the readout."

A positive outcome would allow the company to file for U.S. approval by year's end. With about 1.8 million Americans living with focal seizures, half of them inadequately controlled, azetukalner has blockbuster potential in epilepsy alone

opinion

Why supplement trials rarely deliver clarity

Americans love supplements — roughly three-quarters take them — but most nutraceuticals remain thinly tested. Elise Felicione, an independent clinical research scientist, writes for STAT that she recently attempted to design a rigorous randomized trial for a botanical extract — and came upon five hurdles that got in the way of an effective study.

The sector, she says, sits in an awkward middle ground: companies want pharmaceutical-grade credibility without pharmaceutical-grade budgets. Patents are an issue: Natural ingredients are hard to exclusively own, so investing hundreds of thousands in trials benefits competitors. She also noted that tight budgets shrink sample sizes and make it difficult for follow-up studies, which creates weaker results and dilutes scientific clarity.

"If we want to stop arguing about whether name-your-supplement works, or if it is safe, and start knowing, we need a research ecosystem capable of answering the question realistically, rigorously, and sustainably," Felicione writes.

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