February 12, 2025

The need-to-know this morning

The Food and Drug Administration approved a new therapy from Springworks Therapeutics to treat children and adults with neurofibromatosis type 1, a rare, but generally non-fatal tumor that forms along nerve sheaths. The drug will be sold under the brand name Gomekli.
Gilead Sciences earnings are here, and Biogen earnings are here. Biogen said it will also receive $250 million in financing under a new royalty agreement with Royalty Pharma, tied to Biogen's experimental lupus drug, litifilimab.

politics

Drugmakers stay mum on NIH funding cuts

As universities and medical centers scramble to respond to the Trump administration's move to cut NIH funding for research overhead, the biopharma industry has largely stayed silent, my colleague Adam Feuerstein reports.

Though the industry is a major beneficiary of academic research, drugmakers contacted by STAT — including Vertex, Gilead, Eli Lilly, and Regeneron — declined to comment or did not respond to requests for comment. The industry lobbying group PhRMA also declined to comment.

This stance reflects, with few exceptions, an industry-wide decision to favor appeasement and behind-the-scenes engagement with the administration, rather than arguing in public in a way that risks triggering the president.

politics

Is Trump's new order a reprieve for biotech?

From my colleague Matt Herper: Since last week, there have been rumors that President Trump would sign an executive order making dramatic cuts in the Department of Health and Human Services, including at the FDA.

That made investors in pharmaceutical and medical device companies nervous, because it seemed possible that across-the-board cuts at the FDA would hit even those employees who are involved in approving medicines and devices. Those employees are paid for by user fees paid by manufacturers; user fees amount to about $2.7 billion, or about 45%, of the FDA budget.

So perhaps a little bit of relief for now: An executive order signed last night gives the so-called Department of Government Efficiency (DOGE) tons of authority, but there were no specific targets for cuts at the FDA. The agency could be protected by phrasing in the order referring to functions "mandated by statute" and to functions "related to public safety."

Still, as one observer noted, it's impossible to predict what is next with this administration, and the order still gives a great deal of power to DOGE and said that agencies should hire no more than one replacement for every four employees who depart — which could be tough for the FDA, which has long said it has trouble retaining its best employees.

Ahead of Trump signing this order, there was a growing effort by biotech executives to publicly oppose the rumored plans to cut FDA staff. Dozens of people working at small biotechs and in venture capital signed onto an open letter saying they're "concerned that public health will be put at risk if arbitrary and excessive staffing cuts are made to the FDA."

exclusive

Tome founders launch an RNA contract research firm

Omar Abudayyeh, Jonathan Gootenberg, and Patrick Hsu have come together to launch a new company called Terrain Biosciences that will offer RNA contract research and manufacturing services.

The startup has raised $9 million from Magnetic Ventures, Bruker Corporation, and industry veterans Hans Bishop, John Evans, and John Maraganore. The plan is to raise a Series A round later this year.

Terrain aims to use artificial intelligence tools to design RNA for clients, and proprietary manufacturing techniques to produce it.

Abudayyeh and Gootenberg's most recently founded Tome Biosciences, which launched in December 2023 but laid off nearly all of its employees last year after sources told STAT it struggled to raise more money from investors.

Read more from STAT's Allison DeAngelis.

opinion

HHS' settlement with Gilead was disappointing

Earlier this year, the U.S. government settled with Gilead on a lawsuit alleging that the drugmaker infringed on patents owned by the Department of Health and Human Service on HIV PrEP. The U.S. alleged that Gilead ignored the government's scientific contributions that later formed the basis of the company's drugs, called Truvada and Descovy.

In a new opinion piece, 13 academics say that the settlement is disappointing and threatens the future of pharmaceutical public-private partnerships.

The authors point out that public-private partnerships drive some of the biggest pharmaceutical breakthroughs, including cancer treatments and Covid vaccines. Sustaining these partnerships requires mutual respect for contributions, including a willingness by private players to acknowledge and pay for the public technologies they use, the authors argue.