December 3, 2025

The need-to-know this morning

Pharvaris said its oral, on-demand treatment for the swelling "attacks" commonly experienced by patients with a genetic condition called hereditary angioedema achieved the goals of a Phase 3 clinical trial.
Bristol Myers Squibb said it will enroll more patients in a key Phase 3 trial studying its drug Cobenfy in psychosis associated with Alzheimer's disease, after the company found "irregularities due to clinical trial execution" at a small number of study sites.

exclusive

Sam Altman-backed longevity company seeks a $5 billion valuation

Retro Bio, a longevity startup backed by OpenAI CEO Sam Altman, is close to raising what could be one of the drug industry's largest investment rounds. It estimates that it will be worth $5 billion, STAT has learned.

My colleague Allison DeAngelis obtained two slide decks used in Retro's fundraising, one of which includes a slide that projects its market value could surpass that of Eli Lilly and Novo Nordisk, nearing the value of tech companies like Alphabet and Microsoft.

"Longevity will be the greatest pharma market of all time," one slide reads.

biotech

Capricor posts positive data on recently rejected Duchenne drug

Capricor said this morning that in a Phase 3 trial, its cell therapy significantly improved muscle and heart function among patients with Duchenne muscular dystrophy.

The FDA had rejected the treatment in July, telling the company that its application, based on mixed results from a prior study, lacked "substantial evidence of effectiveness." The decision was reportedly made by Vinay Prasad, the FDA's top regulator of cell and gene therapies, overruling staffers who supported the therapy's approval.

Capricor CEO Linda Marban said she thinks the results from the new study, which is placebo-controlled, are sufficient to persuade the FDA to reverse its prior decision.

Read more from STAT's Adam Feuerstein and Jason Mast.

politics

FDA drug chief Rick Pazdur set to leave agency

Less than a month after Rick Pazdur agreed to take on the role of top drug regulator at he FDA, he's decided to leave.

Pazdur filed papers to retire from the agency at the end of the month. Sources told STAT that he's been concerned about several issues: the legality of a program that offers expedited review to drugs deemed to align with national priorities, agency leaders inserting themselves into drug review decisions, and efforts by Commissioner Marty Makary to hand-pick hires for the drug center.

His planned departure adds to the ongoing leadership turmoil at the FDA. Pazdur, who previously directed the agency's cancer center, is the fourth person to lead the drug center this year.

artificial intelligence

Startup aims to combine AI with new small molecule technology

From my colleague Brittany Trang: Excelsior Sciences, an AI-focused drug developer, launched today with $95 million in funding, including a $70 million series A co-led by Deerfield Management, Khosla Ventures, and Sofinnova Partners, as well as a $25 million grant from New York's Empire State Development.

The company is using what it calls "smart blocks" to modularize small molecule synthesis, similar to the way that peptide synthesis makes any oligopeptide synthesizable on demand. Excelsior plans to have its own pipeline, using artificial intelligence to come up with drug candidates based on its "blocks" that it can quickly synthesize. It also plans to use its technology to manufacture at scale for not only the drug industry but also the materials science, agriculture, flavor, and fragrance sectors as well.

At this moment in time, "we feel that [the way to] fully leverage AI is to create chemistry that machines can do, meaning we can actually, for the first time, automate chemistry in a meaningful way," said Excelsior CEO Michael Foley, who co-founded the company with COO Jana Jensen and chemistry professors Marty Burke and Bartosz Grzybowski.

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