June 9, 2025

The need-to-know this morning

Merck said its experimental, oral PCSK9 inhibitor lowered levels of bad cholesterol in people with genetically high cholesterol, achieving the goals of two Phase 3 studies. No data were reported in its press release. Merck is conducting a large, cardiovascular outomes study.
Keros Therapeutics said it will return $375 million in "excess capital" to shareholders. The decision was made at the end of a strategic review of its operations, and notably, after Adar1 Capital Management, a biotech hedge fund and Keros' largest shareholder, accused management of wasting money.
Avidity Biosciences said it received clearance from the FDA to seek accelerated approval for an RNA-based drug to treat facioscapulohumeral muscular dystrophy (FSHD). Separately, the company reported new study results showing its drug, called del-brax, improved function in patients with FSHD.
Axsome Therapeutics said the FDA issued a "refuse to file" letter, turning back its application seeking the approval of a treatment for fibromyalgia.
Struggling "zombie" biotech Elevation Oncology is being acquired by Concentra Biosciences and will be shut down.

obesity

Metsera's amylin drug shows early potential

Metsera, the obesity-focused biotech, said this morning that its drug candidate targeting the amylin hormone showed marked efficacy in an early trial and the potential to have longer-lasting effects than current weight loss treatments on the market that are injected weekly.

In one part of a Phase 1 trial where patients received a single dose, those on the highest dose of 2.4 mg experienced an average of 5.3% placebo-adjusted weight loss at one week. In the other part of the trial where patients received weekly doses for five weeks, those on the highest dose of 1.2 mg experienced 8.4% placebo-adjusted weight loss at the end of the five weeks.

The most popular drugs currently on the market, Novo Nordisk's Wegovy and Eli Lilly's Zepbound, showed 15% and 21% weight loss in late-stage trials lasting over a year, though it's difficult to directly compare early and late-stage results.

Read more from STAT's Elaine Chen.

POLICY

A clearer look at Trump's budget proposal at NIH

President Trump's 2026 budget proposes a 43% cut to the NIH's central function — awarding grants to universities, academic medical centers, and other institutions — from 2025 levels, according to new documents released by the agency.

The $11.6 billion reduction in extramural research funding would decimate support for universities, junior scientists, and startups, while the agency also is moving to cap overhead payments at 15%.

"This budget proposal is misguided and dangerous," said Belinda Avalos, president of the American Society of Hematology, in a statement. Read more from STAT's Jonathan Wosen and Daniel Payne.

Meanwhile, other budget documents released by the administration provide the first clear indication of the priorities for the Administration for a Healthy America, a proposed new agency focused on preventing chronic disease. Read more from STAT's Elizabeth Cooney.

gene therapy

FDA aims to 'be flexible' on gene therapy regulation

In an unusual roundtable on Friday, the administration's top health officials — including health secretary Robert F. Kennedy Jr., NIH Director Jay Bhattacharya, and Mehmet Oz, administrator of the Centers for Medicare and Medicaid Services — signaled openness to gene therapies for rare diseases, STAT's Jason Mast writes. Their remarks are likely to calm fears that the FDA would erect new roadblocks for these costly medicines.

Though once a fierce critic of accelerated approvals, Vinay Prasad, now head of the FDA's Center for Biologics Evaluation and Research, struck a conciliatory tone — emphasizing flexibility and longterm safety tracking.

The hearing was "a terrific sign for us," according to Alliance for Regenerative Medicine lobbyist Timothy Hunt. "We always welcome converts." Whether the shift in rhetoric translates into new policy, however, remains to be seen.

obesity

EMA flags rare blindness risk tied to semaglutide

The European Medicines Agency has warned about a very rare but serious potential side effect of Novo Nordisk's blockbuster drugs Wegovy and Ozempic: non-arteritic ischemic neuropathy, a condition that can cause vision loss.

Affecting perhaps 1 in 10,000 longterm users of semaglutide, this disease is the second-leading cause of blindness from optic nerve damage after glaucoma.

While the EMA is now requiring the risk be listed on drug labels, analysts say the finding is unlikely to dent prescribing. Novo, facing growing competition from Eli Lilly, maintains that the benefits of semaglutide outweighs the risks.

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