July 17, 2025

The need-to-know this morning

Bristol Myers Squibb and Pfizer announced a plan that will allow uninsured or under-insured U.S. patients to buy the blood-thinning medicine Eliquis directly from the companies at a 40% discount from its list price. The move follows similar direct-to-consumer services offered by Eli Lilly and Novo Nordisk, respectively, for their obesity drugs. It also comes amid efforts by the Trump administration to force drugmakers to lower drug prices.
Novartis reported second-quarter earnings and announced the retirement of CFO Harry Kirsch after 22 years at the company. His replacement is current Novartis executive Mukul Mehta.

rare disease

Sarepta slashes jobs amid gene therapy fallout

Sarepta Therapeutics is laying off roughly 500 employees — over a third of its workforce — and halting several gene therapy programs in a sweeping restructuring move aimed at saving $400 million annually. The cuts follow two teenagers' deaths tied to the company's Duchenne muscular dystrophy gene therapy, Elevidys, prompting safety restrictions on its use.

Once projected to draw in $3 billion annually, Elevidys is now limited to ambulatory children and under intense scrutiny. Investors, however, welcomed the dramatic cost-cutting, sending Sarepta shares up 36% after hours yesterday, STAT’s Jason Mast and Adam Feuerstein write.

Meanwhile, Generate:Biomedicines is trimming 10% of its 330-person workforce amid broader biotech turbulence. The company told STAT’s Brittany Trang, however, that it intends to “end the year at close to that same number.”

research

Scientists develop capsule to dose mRNA orally

In a potential breakthrough for mRNA therapeutics, researchers have created an oral capsule — dubbed RNACap — capable of delivering liquid mRNA directly to the gut, bypassing the need for injections. Published yesterday in Science Translational Medicine, their study shows that the capsule’s protective coating survives stomach acid and releases its payload in the intestines using natural digestive contractions.

“For years, people thought the stomach would destroy mRNA before it could even reach the intestines, but RNACap solves that,” the study’s lead author told STAT’s Marissa Russo.

In animal models, the therapy reduced inflammation by prompting cells to produce IL-10, an anti-inflammatory molecule. This could pave the way for oral mRNA therapies, though scalability and stability remain challenges.

stem cells

RFK Jr.'s FDA may embrace state-led deregulation of biologics

Under health secretary Robert F. Kennedy Jr., the FDA appears poised to loosen its grip on stem cell oversight. Interestingly, this is because it’s aligned with red-state laws that permit the use of non-FDA-approved biologics. This could potentially empower dubious stem cell clinics, STAT’s Paul Knoepfler writes, which might endanger patients — and sideline science in favor of political ideology and profit.

Florida and Alabama are the latest states to say that FDA approval isn’t necessary for stem cells to be used clinically. Coupled with Kennedy’s ties with stem cell donors, the agency risks becoming a passive bystander as unproven therapies flood the market under the guise of access and innovation.

ivf

Eight babies born using three-parent IVF technique

Eight children have been born healthy through mitochondrial replacement therapy in the U.K., the first large-scale clinical use of the controversial “three-parent baby” technique designed to prevent transmission of devastating mitochondrial diseases. In two papers published in the New England Journal of Medicine, researchers from Newcastle University reported that the children — now up to 2 years old — are meeting developmental milestones, though some showed low levels of inherited mutated mitochondrial DNA, a known limitation of the procedure.

“As parents, all we ever wanted was to give our child a healthy start in life,” said the mother of one of the babies born with the help of the technology, in a statement issued by Newcastle University. “Mitochondrial donation IVF made that possible. After years of uncertainty this treatment gave us hope — and then it gave us a baby.”

While long-term and even multigenerational follow-up is still needed, the results strengthen the case for expanding access to the technology in carefully selected patients.

clinical trials

Rethinking ethics and equity in research pay

For decades, institutional review boards have erred on the side of conservatism when it comes to paying medical research trial participants — capping compensation out of fear of influencing them unethically. But this caution, well-intentioned though it may have been, has backfired, opines clinical trial advocate Jake Eberts of the nonprofit 1Day Sooner and University of North Carolina bioethicist Jill Fisher.

Instead, trials systematically underpay participants, which slows recruitment and disproportionately harms low-income individuals who depend on such studies to make ends meet. In a recent open letter, dozens of experts called this model misguided, pointing out there’s little evidence that higher pay clouds risk perception. If the research is ethical and the risks are reasonable, they argue, participants should be trusted — and fairly compensated.

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