June 17, 2025

The need-to-know this morning

Eli Lilly is buying Verve Therapeutics, developer of gene-editing treatments for cardiovascular disease, for $1 billion plus a CVR potentially worth another $300 million.
Dyne Therapeutics received FDA clearance to seek accelerated approval for its drug to treat myotonic dystrophy type 1. However, the company pushed back the timing of the submission to "late 2026" from its previous guidance of "first half 2026."

gene therapy

Duchenne families are fearful and divided after patient's death

Following Sarepta Therapeutics' disclosure that a second boy who took its gene therapy for Duchenne muscular dystrophy has died, the patient community is angry, fearful, and divided over whether to maintain hope in the therapy.

The news also renewed questions about the Food and Drug Administration's standards for approving drugs for fatal rare diseases and whether it had allowed the therapy, called Elevidys, on the market with inadequate testing.

My colleagues have been talking to families of other children with the debilitating muscle-wasting disease.

One person's son experienced signs of liver failure after receiving the therapy and has been hospitalized multiple times. “This kid died from the same thing we’re trying to fight off right now,” the father said. “It really sent it home for us where we’re at right now.”

Read more from my colleagues Adam Feuerstein and Jason Mast.

biotech

China has become an unlikely hotbed for obesity trials

China has one of the lowest rates of obesity in the developed world. And yet, it's emerged as one of the top locations for testing new weight loss drugs.

According to a report from clinical research organization Novotech, China is the second-most-popular country for obesity trials in recent years, behind the U.S. and ahead of Australia.

The reality is that China is one of the most important geographic regions for the weight loss drug market, as rates of people with obesity are rising. The clinical trial activity also reflects Chinese companies' drive to strike lucrative deals licensing drugs out to Western companies.

Incentives from the Chinese government have supercharged the native biotech industry and made it easier to run clinical trials.

Read more from STAT's Allison DeAngelis.

This story accompanies a new update to STAT’s Obesity Drug Tracker. Our database tracks more than 150 drugs in development, including several from Chinese companies Sciwind Biosciences and Zhejiang Doer Biologics, among others.

science

Private equity finances a Harvard lab after Trump's funding cuts

Kent Dayton/Harvard T.H. Chan School of Public Health

As Harvard grapples with steep cuts to federal funding undertaken by the Trump administration, a private equity firm has stepped in to finance a biological research lab at the university and launch a biotech alongside it.

İş Private Equity, a Turkish firm, has committed $39 million to a lab run by Harvard professor Gökhan Hotamışlıgil (pictured above). The firm also plans to invest an undisclosed amount of money in any drug candidates that come out of the lab and are moved into a new biotech called Enlila.

Given the uncertain state of federal funding, some university officials believe this unusual arrangement could be at least one model to fund other academic research in the future.

obesity

Lilly adds highest Zepbound doses to DTC offering

Eli Lilly said yesterday that the highest doses of its weight loss treatment Zepbound will be offered in vials through its online pharmacy service, as obesity drugmakers increasingly compete in the direct-to-consumer space.

Lilly began directly selling Zepbound in vials to self-pay patients last year, advertising it as a more affordable way for patients to get the treatment, which carries a list price of $1,086 per month. The company was offering all but the two highest doses in vials at prices ranging from $349 to $699.

With the new announcement, Lilly will also now sell its highest 12.5 mg and 15 mg doses, but at prices approaching the list price: $849 and $1,049, respectively.

Experts previously cast doubt on Lilly's statements that the vial offerings would expand access, noting that the new prices are still unreachable for many patients.

Competitor Novo Nordisk this year also entered the DTC space, selling its obesity drug Wegovy at $499 a month across all doses for self-pay patients.

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