February 18, 2025

The need-to-know this morning:

Solid Biosciences reported initial results from a study of its experimental gene therapy for Duchenne muscular dystrophy, showing increases in a lab-made version of the dystrophin protein and improvements in muscle and heart-related biomarkers.
Ultragenyx said the FDA granted priority review for its gene therapy to treat Sanfilippo syndrome Type A, with an approval decision expected by Aug. 18.

BIOTECH

Biogen partners with Stoke Therapeutics to sell severe epilepsy drug

From my colleague Adam Feuerstein: Biogen and Stoke Therapeutics have signed a partnership agreement to co-develop and sell Stoke's experimental treatment for Dravet syndrome, a severe form of epilepsy, outside the U.S., the companies said this morning.

For Biogen, acquiring international commercial rights to the Stoke drug bolsters its rare disease and neurology pipeline, and provides another product that could help it return to growth in the latter part of the decade. Called zorevunersen, the drug has shown potential to become the first treatment to target the underlying, genetic cause of Dravet, which induces frequent seizures and impairs cognitive function in children and teens.

Stoke will benefit from an experienced partner already capable of marketing neurology and rare disease drugs internationally. Most importantly, the deal provides Stoke with $165 million in upfront cash to fully fund a Phase 3 study of zorevunersen that will start in the second quarter.

politics

Firings at U.S. health agencies appear far-reaching

The Trump administration initiated mass terminations across federal health agencies — including HHS, FDA, CDC, NIH, and CMS — starting Friday and continuing through the weekend. While the administration has stated publicly that it believes staffing reductions will make the agencies and the programs they run more efficient, those who were fired, along with officials who remain in their roles, argued the cuts would be deeply felt, not only by the federal workforce but by ordinary Americans who rely on them.

The FDA's Center for Devices and Radiological Health, which reviews medical devices, artificial intelligence, and diagnostic tests, appears to be one of the hardest units hit, with outside sources saying that 200 people, or 10% of all its staff, were cut.

It remains unclear to what extend FDA employees whose work is funded by industry user fees were affected by the cuts. Some workers cut were covered at least in part by the Medical Device User Fee Act.

POLITICS

Firings will have outsize impact on AI oversight at FDA

The Trump administration's firings at the FDA have hit the artificial and digital health divisions particularly hard, raising concerns over the agency's ability to regulate the rapidly growing use of AI in medical devices, STAT's Lizzy Lawrence reports. There's concern that the cuts could slow the approval process, reduce oversight, and shift the burden of ensuring patient safety to hospitals.

"I fear if there's going to be even less rigor because we can't keep up with the bandwidth and we can't do important research, that burden is going to go to the hospitals," one FDA employee, who requested anonymity to protect against retaliation, told STAT. "It's going to go to the patients."

The FDA's Center for Devices and Radiological Health has had a strained relationship with one of Elon Musk's companies, Neuralink, which is testing a brain implant. Among the employees fired over the weekend were several reviewing Neuralink's technology, Reuters reports.

ethics

The medical ethics of abruptly canceling USAID trials

The abrupt termination of USAID-funded clinical trials, which impact critical HIV and TB research, is profoundly unethical, opine Ruth Faden and Nancy Kass of the Berman Institute of Bioethics. The move, they argue, poses immediate risk to trial participants, since it withdraws potentially lifesaving treatments and can hasten the creation of drug-resistant strains.

Ethical medical research, shaped by lessons learned from historical atrocities, prioritizes participant welfare — which means closing studies gradually and transparently. Immediate action is necessary, the author write, to continue funding the USAID research sites and to protect medical professionals while they wind down the affected trials.

RESEARCH

Former NIH director calls agency cuts 'devastating'

Former NIH director Monica Bertagnolli told STAT that she finds the large-scale firings at federal health and science agencies "devastating." She worries how the cuts will impact early-career scientists and the future of biomedical research — fearing that the lack of support will deter a new generation from entering scientific research.

"I got very little sleep [Friday] night. It was just such an emotional thing for me," she told STAT's Anil Oza at the annual meeting of the American Association for the Advancement of Science in Boston. "They are numbers to most people, but they are people to me, who have now lost their jobs and whose talents won't be used the way they have been."

Despite such concerns, Bertagnolli emphasized the importance of working with the new administration to advance public health, maintaining that science should transcend politics.

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