Moderna goes to Washington

Tomorrow, Stéphane Bancel, the well-compensated CEO of Moderna, will stand before Sen. Bernie Sanders, a well-documented critic of Moderna, to explain why exactly the company will soon quadruple the list price of its Covid-19 vaccine. It should be interesting.

Ahead of Bancel's appearance before the Senate's health committee, we put together a cheat sheet with a few burning questions facing Moderna's CEO. The impetus for the hearing — and the source of Sanders' ire — is that Moderna received about $1.7 billion in federal funding to develop and manufacture its vaccine, and that investment, some argue, obligates the company to limit any price increases to its product.

The underlying question boils down to: How big a reward do drug companies deserve when they do something society wants? Moderna has already banked about $40 billion from its Covid-19 vaccine, and Bancel made nearly $400 million last year, most of which he has promised to donate. After making all that money from a federally subsidized vaccine, why does Moderna feel the need to raise prices?

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Neuroscience

Another ALS drug heads to the FDA

A Biogen treatment for ALS is up for approval despite failing to show a definitive benefit in a clinical trial, bringing the latest test of the FDA's flexibility when it comes to new medicines for dire conditions.

Tomorrow, a panel of FDA advisers will convene to pore over data on tofersen, an injectable treatment meant to treat an inherited form of ALS caused by a mutation in a gene called SOD1. About 100 people are diagnosed with SOD1 ALS each year, accounting for roughly 2% of the overall cases of the disease.

In a Phase 3 study disclosed in 2021, tofersen led to a modest slowing of neurologic and functional decline in ALS patients compared to a placebo, not enough to reach statistical significance or achieve the study's main efficacy goal. Biogen submitted the treatment for approval based on a further analysis showing tofersen's benefits on surrogate markers of ALS, including an oft-debated biological signal called neurofilament.

The panel's vote, followed by the FDA's final decision next month, will illuminate just how flexible the agency's neurology division is willing to be. Last month's departure of division chief Billy Dunn, widely perceived as industry-friendly, has spurred debate over whether the FDA is taking a turn for the conservative.

Regulatory

Top FDA official: Time to speed up gene therapy approvals

The FDA needs to start using its accelerated approval pathway, generally reserved for new cancer medicines, to advance gene therapies for rare diseases, a top agency official said.

As STAT's Jason Mast reports, Peter Marks, head of the FDA's Center for Biologics Evaluation and Research, said the agency should consider approving gene therapies based on early-stage evidence rather than waiting for long-term data demonstrating their benefits.

"We always are going to keep safety front and foremost," Marks told attendees at the annual meeting of the Muscular Dystrophy Association. "But I think the issue here is we can't be so careful about our approvals under accelerated approval that we prevent potentially life-saving therapies from getting to market in a timely manner."

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Markets

When good data go over poorly

Yesterday's news that Karuna Therapeutics' treatment for schizophrenia succeeded in a second Phase 3 trial seemed to all but guarantee the company's treatment will win FDA approval, which would make it the first novel treatment for the disease in decades. And then Karuna's stock price fell as much as 9%.

The likeliest explanation, Mizuho analyst Graig Suvannavejh wrote in a note to investors, is that Karuna's drug, KarXT, missed a secondary endpoint of improving certain symptoms of schizophrenia and showed some signs of elevating blood pressure. Whether those concerns were worth the $500 million Karuna lost in market value remains unclear.

Meanwhile, Cerevel Therapeutics, one of biotech's few SPAC success stories, rose as much as 8% on the same data. Cerevel's lead drug is a schizophrenia medicine similar to KarXT, and while it remains years behind Karuna, any hint of a hiccup is arguably good for its competitive positioning. That's despite the fact that Cerevel recently delayed the readouts of three clinical studies, meaning the company will have no data to offer in all of 2023.

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