June 25, 2025

The need-to-know this morning

Viking Therapeutics announced the start of Phase 3 studies involving its injectable, GLP-1/GIP-targeted obesity drug, called VK2735.
Gilead Sciences and Kymera Therapeutics announced a partnership and licensing agreement to develop a new class of cancer drugs that eliminates a protein called CDK2 from cells — a contributor to tumor growth.
Biogen is advancing a next-generation treatment for spinal muscular atrophy into a Phase 3 clinical trial.

politics

As FDA center director exits, a new hire is announced

A day after the top drug regulator at the Food and Drug Administration announced she was leaving her post, a new deputy director of the office she led, the Center for Drug Evaluation and Research, was named yesterday.

Mike Davis, a psychiatrist and pharmacologist, was most recently chief medical officer at the Usona Institute, a nonprofit organization developing psychedelic drugs for the treatment of depression and PTSD, STAT's Lizzy Lawrence reports. His appointment comes as the Trump administration, in various ways, is showing a surge of interest in the potential approval of psychedelics as mental health treatments.

At a staff meeting where she introducing Davis, the outgoing acting director of CDER, Jacqueline Corrigan-Curay, acknowledged much is still in flux at the FDA. "We are leaner and therefore we have to find ways to be efficient and do things in new ways," she said at a town hall meeting.

gene therapy

FDA probes deaths of boys taking Sarepta Duchenne drug

From my colleague Adam Feuerstein: The FDA said yesterday that it is investigating the deaths from liver failure of two non-ambulatory boys with Duchenne muscular dystrophy who were treated with Sarepta Therapeutics' gene therapy, Elevidys.

"FDA is investigating the risk of acute liver failure with serious outcomes, including those such as hospitalization and death, following Elevidys, and is evaluating the need for further regulatory action," the agency said in a safety bulletin posted to its website.

The death of the second Elevidys patient earlier this month led Sarepta to voluntarily halt the use of Elevidys in non-ambulatory Duchenne patients. The company believes a stronger immunosuppression regimen administered prior to Elevidys might mitigate the gene therapy's toxic effects on the liver, although any changes to treatment will need to be cleared by the FDA.

The initiation of an FDA investigation into patient deaths isn't unusual. Regulators may decide to add more stringent safety warnings to Elevidys' prescriber label, or take more aggressive actions, possibly withdrawing Elevidys' accelerated approval for non-ambulatory Duchenne patients.

regulation

Makary reportedly asked staff to reject KalVista's drug application

FDA Commissioner Marty Makary asked agency staff to reject a drug application from KalVista Pharmaceutics, Endpoints reported yesterday, citing unnamed sources.

The staff did not ultimately carry out the request, as scientists pushed back and raised legal concerns, Endpoints reported. Still, it was an extremely rare case of a political appointee intervening in what is traditionally a purely scientific review. Read more of the story by Endpoints.

In another case of reported political interference under the Trump administration, Politico previously reported that the FDA's principal deputy commissioner, Sara Brenner, paused the approval process of Novavax's Covid-19 vaccine to ask for more data.

biotech

Regeneron seeks to revamp ties with patient charity

In a bid to rework the controversial relationship between drugmakers and patient charities, Regeneron Pharmaceuticals has announced a new program in which it will match up to $200 million in donations this year to a leading foundation.

At issue is funding for Good Days, also known as the Chronic Disease Fund, which provides financial assistance to people who have difficulty affording 20% insurance copays for medicines — including Regeneron's eye therapy called Eylea — or need help covering travel costs to appointments.

Such charitable donations can prove challenging for companies. A drugmaker is not permitted to dictate or suggest how its funds may be used by a charity. As a result, some of its money can end up being distributed to help patients afford medicines sold by rival drugmakers.

Regeneron CEO Len Schleifer said: "There's no connection, as there shouldn't be, in what we give and … how the foundations dole out the resources."