Sarepta’s disappointing data put the FDA in a difficult spot

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Good morning, all. Damian here with disappointing news on a closely watched gene therapy, an interesting deal in genome editing, and the long shadow of biotech’s slump.

The need-to-know this morning
• Shares of Sarepta Therapeutics are down 40% in pre-market trading, following disappointing results from a confirmatory study of its gene therapy for Duchenne muscular dystrophy announced late yesterday.
• Amgen reported third-quarter adjusted earnings of $4.96 per share, better than the analyst consensus. Revenue rose 4% year over year to $6.9 billion, in-line with consensus. The company slightly raised its earnings and revenue guidance for the remainder of the year.
• Pfizer posted a third-quarter adjusted loss of 17 cents per share, impacted by $5.6 billion non-cash inventory write-offs and other charges. Analysts, on average, were expecting a net loss of 8 cents per share. Revenue fell 42% year over year to $13.2 billion on the previously announced decline in Covid product sales. Excluding Covid sales, “operational revenue” grew 10%.
• A group of FDA advisers will spend today debating the merits of the first CRISPR medicine up for U.S. approval, a treatment for sickle cell disease from Vertex Pharmaceuticals and CRISPR Therapeutics. Follow STAT’s live coverage.

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