FDA approves Takeda drug for narcolepsy

The Food and Drug Administration has approved Takeda’s Orzeyful for narcolepsy type 1, marking the debut of a promising new drug class of therapies aimed at treating the disorder’s underlying biology rather than simply managing symptoms. In Phase 3 studies the drug, an orexin receptor agonist helped patients stay awake longer during the day while reducing excessive sleepiness and episodes of muscle weakness, STAT’s Elaine Chen writes.

Drugmakers see broader potential for orexin agonists in related sleep disorders, as well as neurologic and psychiatric conditions such as Alzheimer’s disease and depression — also diseases where fatigue and impaired alertness are common.

BIOTECH

Greg Verdine's long bet starts paying off

Biotech entrepreneur Greg Verdine is finally seeing several long-running bets come together, STAT’s Allison DeAngelis writes.

His former startup Warp Drive Bio laid the groundwork for Revolution Medicines’ groundbreaking pancreatic cancer drug daraxonrasib. Meanwhile, peptide drugmaker Parabilis Medicines completed one of biotech’s biggest IPOs in recent years. This morning, Verdine’s startup LifeMine announced it has raised $263 million to advance a next-generation calcineurin inhibitor into kidney transplant studies.

The new funding will also revive LifeMine’s fungal drug discovery platform, with plans to use AI to sift through hundreds of potential targets.

“I’m on a roll this year,” he said. “I planted a lot of seeds way back when, and they’ve really started to bear fruit.”

Vaccines

FDA approves Moderna’s mRNA flu vaccine

The FDA late yesterday approved Moderna’s influenza vaccine for older adults, making it the first licensed flu shot made using messenger RNA technology.

As STAT's Helen Branswell notes, the approval was virtually unthinkable just six months ago, after Vinay Prasad, then head of CBER, overturned the recommendation of career staff and issued a rare “refusal-to-file” notice to Moderna, saying the agency wouldn’t review the vaccine based on the available data package.

A week later — after Moderna disclosed the FDA’s decision as well as its own displeasure over the agency’s position — Prasad’s decision was reversed, and he left the agency a few months later.

exclusive

Child dies in China-based gene therapy trial for Duchenne

A young boy in China died during an experimental gene therapy trial for Duchenne muscular dystrophy, STAT’s Jason Mast and Brian Yang write. The company, HuidaGene, is developing CRISPR-based therapies and took center stage last year to present early data on the therapy at a meeting of the American Society for Gene and Cell Therapy in New Orleans. That was followed by more than a year of silence, during which time the CEO and CTO left the company.

After a months-long STAT investigation, HuidaGene said yesterday that the child died from acute respiratory distress syndrome following a severe immune response to the viral delivery system, prompting the company to halt the study.

The “thing that bothers me is the lack of transparency,” said Kiran Musunuru, a gene editing researcher at the University of Pennsylvania, who argued HuidaGene should have released the results in a preprint months ago. “There could be many of these events and we have no idea.”

microbiome

Fecal transplants show promise in peanut allergies

A small Phase 1 study suggests fecal microbiome transplants could one day become a new way to treat peanut allergies. Six of 15 adults with peanut allergies were able to tolerate substantially larger amounts of peanut protein for up to four months after swallowing capsules containing donor stool, with researchers linking the response to increases in immune-regulating T cells, bile acid metabolism, and beneficial gut bacteria.

“We, as the food allergy community, have been waiting for these results,” said Stephen Tilles, an allergist-immunologist and clinical professor at the University of Washington, who was not a part of this study. Tilles described the study findings as “very exciting,” even if it may not be “ready for prime time” yet.

Researchers are now launching a larger Phase 2 trial using more refined, easier-to-administer capsules designed for repeated dosing.

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@megkesh