June 16, 2025

The need-to-know this morning

Supernus Pharmaceuticals said it is acquiring Sage Therapeutics, the distressed maker of a drug for postpartum depression, for $561 million. Last January, Sage rebuffed a $469 million takeover offer from Biogen, which co-markets the postpartum depression pill, called Zurzuvae.

gene therapy

Second death tied to Sarepta's Duchenne treatment

Sarepta Therapeutics has suspended shipments of its Duchenne muscular dystrophy gene therapy, Elevidys, to non-ambulatory patients after a second death from acute liver failure. This is the same side effect that was seen in the first fatal case disclosed this past March. Both patients were boys who had lost the ability to walk, STAT’s Andrew Joseph writes — a stage of disease progression common in adolescence.

The company is pausing dosing in its ENVISION trial and is working on a more aggressive immunosuppressive regimen. It said it plans to talk to the Food and Drug Administration about this approach. The death intensifies scrutiny over the agency’s controversial decision to approve Elevidys broadly, despite scant data in older patients — a move made by Peter Marks, the former chief of the FDA's Center for Biologics Evaluation and Research, who was ousted in March.

Obesity

Lilly’s amylin candidate shows early promise

From STAT's Elaine Chen: Eli Lilly’s early obesity candidate targeting the amylin hormone showed marked weight loss in a Phase 1 trial.

The treatment, called eloralintide, led to an average 11.3% weight loss over 12 weeks in one of the dose groups, while those on placebo experienced 0.2% weight loss, according to an abstract posted by the American Diabetes Association ahead of its annual meeting.

Notably, the treatment had a half-life of 13.9 to 15.8 days, longer than what’s seen with current drugs on the market. Side effects also appeared less common than what’s been seen in other trials, with 8% of treated patients experiencing nausea and 4% experiencing vomiting. Larger studies will be needed to better assess the tolerability of the treatment, though. Lilly is currently running a Phase 2 trial.

A growing number of drugmakers are targeting amylin, a hormone that’s co-secreted with insulin. Novo Nordisk has been furthest along with a candidate called CagriSema, which activates receptors of both the amylin and GLP-1 hormones, but its recent weight loss results failed to live up to investors’ expectations. Investors have been excited, though, by results of candidates targeting only amylin, such as those being developed by Zealand Pharma and Metsera.

There’s going to be more data coming out of the ADA conference happening this weekend — stay tuned for our coverage.

POLICY

RFK Jr.'s vaccine panel appointees raise ethics questions

The vaccine advisory panel appointed by HHS chief Robert F. Kennedy Jr. last week was formed after the previous 17 members were dismissed over what he described as conflicts of interest with the pharmaceutical industry. However, the new slate of advisers is drawing scrutiny for their own conflicts of interest — as well as a lack of infectious disease expertise.

Several appointees, including Robert Malone and Martin Kulldorff, have served as paid expert witnesses in lawsuits against vaccines or publicly questioned vaccine safety, raising concerns among public health experts and ethicists about how conflicts are being defined.

“I don't know what conflict of interest vetting will look like with this administration,” Robert Besser, CEO of the Robert Wood Johnson Foundation and former acting CDC director, told STAT. He said asking questions about vaccine safety isn’t a conflict, but “when you have people who have not just asked those questions, but spread misinformation and disinformation, that should be a disqualifier.”

GENETICS

Wojcicki's nonprofit wins 23andMe bidding war

23andMe co-founder Anne Wojcicki will acquire the bulk of the consumer genetic data company’s assets for $305 million via her nonprofit TTAM Research Institute. She outbid Regeneron in a final round of bidding designed to maximize value for shareholders. The move transitions the struggling company — which was once valued at more than $3.5 billion — into a nonprofit.

Concern has been swirling about the fate of 23andMe’s vast trove of genetic data, STAT’s Matthew Herper writes — but TTAM has pledged to uphold existing privacy policies, refrain from selling data, and form new identity protections.

“We believe it is critical that individuals are empowered to have chocie and transparency with respect to their genetic data and have the opportunity to continue to learn about their ancestry and health risks as they wish,” Wojcicki said in a statement.

POLICY

Academics propose fix to research overhead crisis

Facing a backlash over the Trump administration’s 15% cap on research overhead — a move that could cost some universities more than $100 million annually — a coalition of academic groups has floated two alternative models aimed at preserving scientific funding while reducing bureaucratic pressures.

The Joint Associations Group, or JAG, proposes either tiered payments based on research type and institution — or a granular accounting system that turns indirect costs into line items in each grant proposal. Both models would replace the current complex system of negotiated rates with the hopes of boosting transparency, flexibility, and fairness, STAT’s Jonathan Wosen writes.

In a recent Senate hearing, it appears that lawmakers and NIH Director Jay Bhattacharya were intrigued by this approach.

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