Alnylam clears an FDA hurdle, but not without skepticism

A panel of FDA advisers voted in favor of Alnylam Pharmaceuticals' treatment for a debilitating heart disease yesterday, but only after a day-long debate challenging whether the drug's modest observed effects were actually meaningful for patients.

The group voted 9-3 that the benefits of Alnylam's patisiran outweigh its risks for patients with ATTR-CM, a progressive and increasingly common disease that leads to fatal heart failure if left untreated. The FDA is expected to make a final decision on the application by Oct. 3.

While the final tally went in Alnylam's favor, panelists spent the bulk of the meeting picking apart its case and concluding that while the supporting study met its primary goal, the actual benefit to patients was small enough to sow doubt. "It's like a light wind blowing in favor of patisiran over placebo," said David Cella, a neurologist at Northwestern University's Feinberg School of Medicine.

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Venture

There's always money in AI

Biotech's prolonged downturn has made it difficult for late-stage startups to find the kind of venture capital to which they were once accustomed. But Generate Biomedicines, which just raised a $273 million Series C, has something most don't: a foundational interest in AI.

As STAT's Allison DeAngelis reports, Generate recruited an investor syndicate that includes the venture arm of Nvidia, the booming chipmaker riding high on the AI hype cycle, and Amgen.

Generate launched out of Flagship Pioneering in 2018 with the promise of using machine learning algorithms to identify antibodies, peptides, cell therapies, and other medicines. The company started its first clinical trial in July, studying a monoclonal antibody for Covid-19, and it's working on starting another study for an asthma treatment.

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Washington

The White House quietly blazed a trial in drug pricing

Amid the legalese in a contract between Regeneron Pharmaceuticals and the federal government was a clause with vast potential implications, one guaranteeing the U.S. won't pay a penny more than other high-income nations.

As STAT's Rachel Cohrs reports, the deal covers a future Regeneron treatment for Covid-19, and the contract didn't specify which countries will serve as benchmarks or how exactly the data will be tabulated. But the mere fact of its existence sets in motion a proof-of-concept study for using an international index to negotiate drug prices, an idea academics and policy experts have floated for years.

"To my knowledge, this is the first time that the Biden administration has included a reasonable pricing provision in a new investment in pharma, and that makes it one of the most significant executive actions Biden has taken on drug pricing," said Christopher Morten, an associate clinical professor of law at Columbia University.

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Digital health

Farewell to the prescription video game

Back in 2020, Akili Interactive made history by winning FDA clearance for a doctor-prescribed video game designed to treat the symptoms of ADHD. Yesterday the company said it was rethinking its entire model, laying off staff and pivoting to consumers.

As STAT's Mario Aguilar reports, Akili is cutting 40% of its workforce as it seeks over-the-counter labeling for EndeavorRx, its therapeutic video game. The company, founded by PureTech Health, expects to submit the required data to the FDA by 2024.

Akili's move away from a prescription business model that relies on insurer reimbursement underscores the challenges prescription digital therapeutics companies have faced. Despite FDA clearances and published evidence that suggests the products can deliver positive outcomes, insurers have been slow to embrace the novel treatments.

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