July 21, 2025

The need-to-know this morning

iTeos Therapeutics, the defunct cancer drug developer, is being acquired and shut down by Concentra Biosciences, the private company run by investor Kevin Tang.
The Food and Drug Administration named George Tidmarsh, a longtime biotech executive who has a background in oncology and pediatrics, its top drug regulator. Tidmarsh will be the director of the FDA's Center for Drug Evaluation and Research, or CDER. Read more about Tidmarsh from my colleague Andrew Joseph here.

duchenne muscular dystrophy

Sarepta defies FDA amid gene therapy crisis

The Food and Drug Administration and Sarepta Therapeutics are at loggerheads.

Late Friday, after the FDA asked Sarepta to voluntarily stop all shipments of Elevidys, its gene therapy for Duchenne muscular dystrophy, based on safety concerns, the company refused. It said it had seen "no new or changed safety signals," despite the deaths of two patients.

Exactly what happens next is unclear, but the FDA is unlikely to give up its effort; Sarepta, meanwhile, must contend with growing investor unrest. The company's shares plunged 36% to $14.07, their lowest level in nine years, on Friday.

Elevidys represented immense hope for the Duchenne patient community, even as its maker never quite delivered clear and decisive efficacy results in clinical trials. Many patients still believe the therapy offers their best chance against a universally fatal disease, STAT's Jason Mast and Adam Feuerstein write.

Read more about Sarepta's controversial decision to continue shipping its therapy. And then read an opinion piece from two Duchenne advocates: "A search for absolute safety guarantees will come up short," they write. "That luxury is not afforded to our club — and never will be."

DRUG DEVELOPMENT

Alkermes drug shows promise in narcolepsy trial

Alkermes said this morning that its experimental narcolepsy drug, alixorexton, hit key goals in a mid-stage trial, with all doses significantly improving wakefulness and reducing daytime sleepiness over six weeks — a promising result in the race for a multibillion-dollar market, STAT's Elaine Chen writes.

Only the middle dose tested reduced the frequency of sudden muscle control loss, however, which CEO Richard Pops attributes to limitations in the trial methodology. Patients also showed improvements in fatigue and cognition, which the company says reflect real-world benefits beyond just staying awake.

The full data will be presented at the World Sleep Congress in September.

VENTURE CAPITAL

Omega Funds raises $647 million amid industry slump

Omega Funds, the Boston-based biotech venture firm led by the outspoken Otello Stampacchia, has closed a $647 million fund — its eighth and a rare bright spot in a bleak VC landscape. Just four biotech firms raised new capital in the first quarter of 2025, but Omega's steady M&A pipeline — 14 exits since 2022 — has helped buck the trend.

While 30% of Omega's cash historically went into creating new companies from scratch, STAT's Allison DeAngelis writes, the firm plans to pare back on that front — focusing instead on existing clinical-stage companies with promising drug candidates that may have made operational mistakes.

policy

Biotech leaders push back against 'sloppy' mifepristone report

More than 50 biotech leaders are urging the FDA to reject what they describe as a politically motivated, error-ridden report from the Ethics and Public Policy Center, a conservative think tank, on the safety of mifepristone. The drug, used to induce abortion, is backed by decades of data and is one of the drugs most studied by the agency.

The report, already weaponized by lawmakers and anti-abortion groups, inflates risk through flawed methodology and dubious sourcing, threatening to undermine the scientific basis of drug regulation, opines Grace E. Colón, president and CEO of InCarda Therapeutics and a former venture partner who serves on several biotech boards.

"We can and should debate how to make our health systems stronger, more responsive, and more affordable," she writes in a new First Opinion essay. "But that debate must be grounded in facts, not fear, and in data, not ideology."