FDA names Szarama as acting CBER director

The FDA has tapped Katherine Szarama as acting head of the Center for Biologics Evaluation and Research, following Vinay Prasad’s abrupt and contentious exit. Szarama, who joined the agency late last year as Prasad’s deputy, brings a mix of academic training and policy experience, with past roles at CMS, Arnold Ventures, and ARPA-H.

Her appointment appears to be interim while the agency continues its search for a permanent leader, with Commissioner Marty Makary signaling a decision could come within weeks and industry chatter still pointing to outside contenders like ophthalmologist and biopharma entrepreneur Houman Hemmati.

obituary

Genomics pioneer J. Craig Venter reshaped biotech's trajectory

J. Craig Venter, the brash and polarizing scientist who helped turn genetics into a data-driven industry, has died from cancer at 79.

“Along the way, he did things that can only be described as really cool,” STAT’s Matthew Herper writes. “He raced against a government-funded project to sequence the first human genome, grabbing headlines around the world; sailed the oceans collecting the genetic information of life in the sea, and removed a bacterium’s genome and rebooted the organism with an identical set of genes he and his team had synthesized. He drove fast cars, drank red wine, and pissed people off.”

Venter pushed ideas like synthetic biology and large-scale sequencing well before the field could deliver on them commercially, and many of his early business models fizzled. His real impact wasn’t just sequencing genomes, but helping create the systems and ambition that made modern genomics possible — even if the world he envisioned is only now starting to take shape.

rare disease

A new bespoke drug startup from Mila's mom

Julia Vitarello, whose daughter Mila received a custom therapy tailored to her mutation, is launching a new company to try to scale individualized medicines. Her prior effort, EveryONE Medicines, shut down amid investor concerns over regulatory uncertainty, STAT’s Andrew Joseph writes. That company had tested a model in the U.K. that aimed to treat multiple patients with slightly customized versions of a drug under a single protocol. But Vitarello said recent FDA guidance — while a step forward — still leans too heavily on disease-by-disease review, limiting scalability and commercial appeal.

“I still believe just as much as I always have in making this work,” she told STAT at a conference in Rome organized by the Alliance for Regenerative Medicine.

Vitarello said the current system, which has produced about 80 bespoke therapies largely through one-off efforts, isn’t sustainable. Making these treatments viable will require clearer regulatory pathways, she said, as well as reimbursement — and investors willing to back a different model.

alzheimer's disease

Axsome wins FDA nod for Alzheimer's agitation

From STAT's Elaine Chen: Axsome Therapeutics said yesterday that the FDA approved its drug Auvelity for agitation in Alzheimer’s disease, opening up a potentially safer and more appealing treatment option for patients.

The only other drug approved for the condition, Lundbeck and Ostuka’s Rexulti, carries a black box warning for increased risk of death in patients with dementia-related psychosis.

The treatment, which was initially approved to treat major depression, is a combination of dextromethorphan, commonly found in cough suppressants, and a generic form of the depression treatment Wellbutrin. Auvelity succeeded in three late-stage trials for Alzheimer’s agitation, but did not show a statistically significant benefit in one study.

The patient population for the drug is broad. Agitation — which includes pacing, restlessness, and verbal and physical aggression — is reported in up to three quarters of patients with Alzheimer’s. Since Auvelity is already approved for depression, it may be especially appealing for doctors to use since many Alzheimer’s patients also experience depression.

podcast

Hair-raising trial results, and Servier's M&A wishlist

Why are investors excited about hair loss drugs? Will artificial intelligence make clinical trials run more smoothly? And how does a nonprofit pharma company compete in the M&A arena?

We get into all that and more on this week's episode of "The Readout LOUD," STAT's weekly biotech podcast.

Veradermics CEO Reid Waldman joined us to discuss his company’s data, and why hair loss is such a trendy topic in biotech. Then, Servier Pharmaceuticals CEO David Lee joined us to discuss the company's acquisition of Day One Biopharmaceuticals. The hosts also discussed the latest news in biotech.

Listen here.