September 25, 2025

The need-to-know this morning

Following a meeting with FDA officials, Capricor Therapeutics said improvement in upper limb muscle function will remain the primary efficacy goal of a Phase 3 clinical trial for its cell therapy in Duchenne muscular dystrophy. The company had asked the agency for permission to use ejection fraction, a measure of the heart's squeezing ability, as the study's primary goal. Top-line results from the study will be reported in the fourth quarter.
Bristol Myers Squibb added the psoriasis medicine Sotyktu to its direct-to-consumer sales program.

cancer

Harvard oncologist Anthony Letai eyed as NCI director

Anthony Letai, a Dana-Farber cancer researcher and Harvard oncologist, has emerged as a top contender to lead the National Cancer Institute, multiple sources tell STAT's Matthew Herper and Angus Chen. The role — the only NIH directorship appointed by the president — has been vacant since January, leaving cancer researchers uneasy amid ongoing turmoil at the agency.

Letai, praised by colleagues as both kind and scientifically rigorous, is known for championing functional precision oncology, an approach that tests living tumor samples against hundreds of drugs, as well as for discoveries that paved the way for AbbVie's blockbuster leukemia drug Venclexta. His potential appointment has sparked optimism across the cancer community, though questions remain about how much he could reshape NCI policy.

vaccines

Military pursues mRNA research as HHS pulls back

Although health secretary Robert F. Kennedy Jr. abruptly axed nearly $500 million in BARDA-backed mRNA vaccine research contracts, spooking labs and biotechs, the Pentagon is quietly keeping parts of the effort alive, Nature reports. The Department of Defense's Joint Program Executive Office has told Seattle-based biotech HDT Bio to restart its Phase 1 mRNA vaccine trial for Crimean-Congo haemorrhagic fever, a deadly tick-borne disease that kills up to 40% of people infected.

That said, other BARDA-JPEO collaborations continue to face "restructuring." AstraZeneca has also begun human studies of two avian-flu mRNA shots, though the question of who's paying now is murky. Experts warn the policy whiplash risks gutting U.S. biodefense.

"A lot of us are at least relieved the Department of Defense is not abandoning mRNA research," Johns Hopkins infectious disease specialist Amesh Adalja said. "The whole biodefence structure is completely derailed ... I've never seen it be disconnected like this."

RARE disease

More on Uniqure's gene therapy win in Huntington's

Yesterday's results from Uniqure's study of its experimental Huntington's disease gene therapy were a boost for the gene therapy field after months of depressing headlines. Shares of Uniqure, a company whose viability was in doubt one year ago, tripled, as STAT's Adam Feuerstein notes.

The breakthrough was enabled by a study that provided external control arm data, though regulators will still need to scrutinize the trial design and the FDA review could be contentious. For now though, the Huntington's community has something to celebrate.

RARE DIsease

Acadia's Prader-Willi drug fails in late study

Acadia Pharmaceuticals' experimental drug ACP-101 flopped in a pivotal study of Prader-Willi syndrome, STAT's Adam Feuerstein writes. It failed to ease hyperphagia — the relentless food cravings that define the rare genetic disease — or any other key symptoms after 12 weeks of treatment.

The nasal spray, a reformulated version of oxytocin acquired from Levo Therapeutics, had previously shown hints of benefit at lower doses, but results were inconsistent and not statistically significant. The setback sent Acadia shares down 9%, while rival Soleno, maker of the only approved therapy for the condition, rose 13%.

diabetes

Sparrow raises $95 million to target cortisol-driven diabetes

Sparrow Pharmaceuticals closed a $95 million Series B to advance clofutriben, a once-daily HSD-1 inhibitor aimed at Type 2 diabetes patients with elevated cortisol, into a Phase 2b trial. Results expected in 2027.

Clofutriben modulates intracellular cortisol to improve glycemic control — which could help manage weight, lipids, blood pressure, bone metabolism, sleep, and quality of life. This positions the experimental drug as a potential add-on to current diabetes regimens for people who are treatment-resistant.

Sparrow is also testing the drug in Cushing syndrome and exploring its use with prednisone in polymyalgia rheumatica, a common inflammatory condition. The company was founded back in 2013 by ex-Abbott/AbbVie scientist David Katz, FierceBiotech points out, and raised a $50 million Series A in 2021.

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