June 11, 2025

The need-to-know this morning

Argenx and Cabaletta Bio, separately, reported new data on potential treatments for myositis, an autoimmune disease that causes muscle weakness. The data are being presented at a European rheumatology conference.
RayzeBio, the radiopharmaceutical unit of Bristol Myers Squibb, licensed an experimental drug and diagnostic agent for prostate cancer from Swiss-based Philochem AG. Under terms of the agreement, Philochem received a $350 million upfront payment and is eligible for another $1 billion in contingency payments.

politics

Drugmakers try to strategize with little info on MFN

In the coming days, the Trump administration is expected to release more details on its "most-favored nation" plan aimed at lowering drug prices, and pharma companies have been trying to game out possible scenarios and strategize how they may respond, my colleague Daniel Payne reports.

A Washington representative for one pharmaceutical company, granted anonymity to discuss internal thinking, said the firm expects more details this week, likely on Wednesday. The difficulty is that so far, the current information being shared "is a bit of a black hole," the person said.

The company has been mapping out the impacts of different potential actions the White House could take, depending, for example, on the way price targets are set (whether it's a price reduction by a certain dollar amount, a percentage reduction, or something else entirely) and the breadth of drugs targeted (whether it's all drugs, certain classes of drugs, or only drugs that are new to the market.)

genetic testing

Anne Wojcicki grilled on 23andMe data privacy

Andrew Harnik/Getty Images

23andMe cofounder Anne Wojcicki and interim CEO Joe Selsavage faced questioning from lawmakers yesterday about how they intend to protect users' data as the genetic testing firm undergoes bankruptcy proceedings.

They pointed to a history of encryption, de-identification of stored genetic data, and opt-in consent for use of customer data in research. But House Oversight Committee members remained unconvinced that these self-imposed standards were enough to ensure that genetic data would be protected by a new owner.

In May, Regeneron had entered into an agreement to buy 23andMe, but a court reopened the auction to final bids last week. Wojcicki has now put in her own bid to buy the company under a new nonprofit entity.

Concerns over where the company's genetic data will end up are precisely why she put in a bid, she told lawmakers.

pharma

Novo Nordisk strikes small molecule, AI deals

We've got several Novo Nordisk deals this morning.

Deep Apple Therapeutics said it's reached an agreement with the Danish company to develop and commercialize oral small molecule treatments for obesity. Deep Apple did not disclose the specific drug target, but said it's a G protein-coupled receptor that is not GLP-1.

Deep Apple will be eligible to receive up to $812 million in upfront and milestone payments, as well as potential royalties from sales.

This is Novo's latest deal involving small molecule candidates, after it reached an agreement with biotech company Septerna last month. Small molecules are expected to be easier to manufacture and distribute than the current injectable obesity medications. As more competitors, particularly Eli Lilly, advance small molecules, Novo is starting to warm up to the modality after it had previously shied away from it, as STAT recently reported.

Also, Nvidia announced a new collaboration with Novo to create AI models for drug development.

Nvidia did not disclose the financial terms, but said that Novo researchers will focus on several programs, including using single-cell models to predict cellular responses to drug candidates, as well as designing models to build molecules with drug-like properties.

infectious disease

Gilead pauses trials of HIV combination therapy

Gilead said yesterday that the FDA has ordered a pause on trials of its investigational HIV combination treatment, after the emergence of safety signals involving a decrease in measures of immune function.

Specifically, the studies center around the drugs GS-1720 and GS-4182, and researchers have seen decreases in CD4+ T-cell and absolute lymphocyte counts in a subset of patients.

Shares of Gilead dropped over 2% yesterday.

The pause doesn't affect other HIV medications that Gilead is testing. Notably, it has no effect on lenacapavir, which is under review with the FDA to be cleared as a twice-yearly preventive drug. A decision is due by June 19.

Correction: Yesterday's newsletter incorrectly spelled Insmed's treatment for pulmonary arterial hypertension, treprostinil.

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