September 8, 2025

The need-to-know this morning

A positive readout of a Rapport Therapeutics seizure medicine in a mid-stage clinical trial sent company shares soaring, as the biotech announced it would move the experimental drug into Phase 3 trials. Read more from STAT reporter Andrew Joseph.
Dianthus Therapeutics said its experimental treatment for myasthenia gravis achieved the primary goals of a Phase 2 study.
Pfizer and BioNTech reported top-line Phase 3 study results showing their Covid-19 vaccine Comirnaty elicited an immune response against the most prevalent circulating strain of the SARS-Cov-2 virus.

startups

Arena BioWorks refocuses with a new CEO

Arena BioWorks, the $500 million research institute launched last year by billionaire backers, has tapped ARIAD Pharmaceuticals co-founder Harvey Berger as CEO, reuniting him with longtime collaborator Stuart Schreiber, who shifts to chief scientific officer. The move follows a 30% staff cut that largely eliminated the company's gene and cell therapy group, STAT's Allison DeAngelis writes, with Berger arguing the institute had been overextended.

"We had done some really beautiful work in the gene-editing area in the first year of Arena's history," he told STAT. "But let's face it, to be the best at small molecules, proteins, antibodies, and at the same time, gene and cell therapy and gene editing, I just thought it was too much to take on."

Despite the cutbacks and refocus, Berger said the institute remains ambitious and expects to unveil its first projects and spinouts next year.

xenotransplantation

Pig kidneys to enter FDA-backed human trial next year

The Food and Drug Administration has cleared eGenesis to launch the first U.S. clinical trial of gene-edited pig kidneys for patients with end-stage kidney disease, marking a milestone in xenotransplantation. The Cambridge, Mass., biotech, co-founded by George Church and Luhan Yang, will begin by transplanting organs into three patients at Massachusetts General before expanding to 30.

The announcement follows months of expanded-access surgeries, STAT's Eric Boodman and Megan Molteni write. Among the recipients is a patient named Bill Stewart, who is back at work and off dialysis thanks to a kidney from a CRISPR-modified Yucatan mini-pig named Lavender.

Rival Revivicor, a United Therapeutics subsidiary, is advancing its own FDA-cleared trial. Together, the companies' competing approaches reflect growing momentum in the race to use genetically engineered pig organs to ease America's chronic organ shortage.

cancer

Summit lung cancer drug may have a geography problem

Ivonescimab, a lung cancer drug made by Summit Therapeutics, showed stronger results in Chinese patients than in those from North America and Europe, raising questions about its regulatory path in the West. In the Phase 3 HARMONi study, tumor progression risk fell 45% in Chinese participants but just 33% in Western patients, missing statistical significance in the latter cohort.

Overall, STAT's Adam Feuerstein writes, the drug delayed progression by 43% versus placebo, but survival gains were modest and not statistically significant. Experts remain divided: Some see the geographic gap as clinically minor, while others point to the lack of clear survival benefit as a bigger hurdle for FDA approval.

narcolepsy

Alkermes, Takeda advance rival narcolepsy therapies

Alkermes and Takeda reported promising results this morning for experimental narcolepsy drugs.

In a Phase 2 study, a once-daily pill from Alkermes helped increase the amount of time patients stayed awake on a test by 26 minutes more than the changes experienced by the placebo group. Patients on the highest does of Takeda's twice-daily pill experienced increases of 20 minutes and 17 minutes more than placebo groups in a pair of Phase 3 trials.

Both orexin receptor agonists, STAT's Elaine Chen writes, appear more effective at treating narcolepsy than current therapies on the market. Drugmakers have ambitions to test the treatments in much more common conditions that lead people to feel sleepy, like Alzheimer's and depression.

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