As search for new FDA chief begins, Diamantas takes hold for now

The Trump administration is scrambling to find a permanent replacement for former FDA Commissioner Marty Makary, and it’s on the lookout for someone who can somehow satisfy two increasingly incompatible goals: restoring trust among a demoralized FDA workforce, and still carrying out the White House’s highly political “Make America Healthy Again” directive.

Internally, staff reactions to Makary’s departure have varied — from relief to deep skepticism about what comes next, STAT’s Daniel Payne and Lizzy Lawrence write. There’s ongoing concern about how political interference will impact scientific decision making around issues like abortion drugs, peptides, and flavored vapes.

Separately, my colleague Adam Feuerstein takes a closer look at the acting commissioner, Kyle Diamantas. Based on what Adam is hearing, he says "no drama" Diamantas should serve biotech well.

exclusive

CREATE reloads CAR-T push with $122 million

CREATE Medicines, the rebranded cell therapy company formerly known as Myeloid Therapeutics, has raised $122 million to push its in vivo CAR-T programs through early clinical testing, STAT’s Allison DeAngelis writes. Cofounded by oncologist and author Siddhartha Mukherjee, CREATE originally focused on using myeloid cells to better target solid tumors, but has since expanded into autoimmune conditions amid growing enthusiasm for CAR-T beyond cancer.

The company’s therapies are designed to generate CAR-T cells directly inside patients rather than through laborious ex vivo manufacturing. CEO Daniel Getts argues they could ultimately outperform rivals like treatments from Capstan Therapeutics, drawing a comparison to how Keytruda emerged as the dominant PD-1 drug despite not being first to market.

“I think we know from what we've observed and what we've done, that we do have superior products, and I think… we'll win the foot race, as well,” he said.

gene therapy

Regenxbio stakes claim in Duchenne gene therapy

Regenxbio says its experimental Duchenne muscular dystrophy gene therapy produced high levels of microdystrophin — the miniature muscle protein used as a proxy for efficacy. This, STAT’s Adam Feuerstein and Jason Mast write, could potentially position the company as a formidable challenger to Sarepta Therapeutics and its embattled therapy Elevidys.

In a 31-patient study, most boys achieved protein expression levels far above the threshold thought to matter clinically. There were also early signs of improved muscle function, and a relatively manageable safety profile. But the results arrive at a turbulent moment for the FDA’s gene therapy division: The leadership turnover there is prompting Regenxbio to delay filing for approval until next year.

“We’re not in a rush to go to the FDA at this point. We want to give time for the leadership changes to occur. But that’s okay, because we can still meet our timeline of securing an approval in 2027,” Simpson said.

Alzheimer's

Biogen’s tau-targeting Alzheimer’s drug posts mixed results in mid-stage study

Biogen reported mixed results this morning from a mid-stage clinical trial investigating a treatment for Alzheimer’s disease that targets the protein tau that, like the better-known amyloid, is toxic to neurons and is believed to play a role in the cognitive decline of patients with the disease.

In the Phase 2 study, the Biogen drug, called diranersen, or BIIB080, reduced levels of tau in the spinal fluid and brains of patients with early-stage Alzheimer’s. Those tau reductions also correlated to a slowing of cognitive decline, the company said.

Biogen investigated three escalating dosing regimens in its study, with the lowest dose showing the best results. For that reason, the study failed to achieve its primary efficacy goal, which was a dose response.

Read my colleague Adam Feuerstein's story for more perspective on the tau-targeting approach to treating Alzheimer's. If you're hoping for actual diranersen numbers, you'll need to wait. Biogen will present data from the study in July at an Alzheimer's research meeting.

cancer

BeOne wins FDA approval in lymphoma race

BeOne Medicines has officially entered the BCL-2 arms race with an FDA approval for Beqalzi in relapsed or refractory mantle cell lymphoma. This is the first BCL-2 inhibitor specifically cleared for this indication in the U.S. —challenging AbbVie and Roche’s blockbuster drug Venclexta, FiercePharma notes.

Beqalzi is being pitched by BeOne as both more potent and easier to manage, arguing its shorter half-life could dramatically reduce the cumbersome tumor lysis syndrome monitoring that has long complicated Venclexta use. While the initial approval is in a relatively niche blood cancer setting, the real commercial fight is shaping up in chronic lymphocytic leukemia. Already, BeOne is touting early measurable residual disease data and advancing multiple Phase 3 studies with ambitions to be a front-line therapy.