Sarepta Therapeutics, Inc.
NEWS
The FDA published briefing documents ahead of advisory committee meetings for therapies from Capricor Therapeutics and Replimune indicate an uphill battle for the biotechs; GSK and AstraZeneca outline ambitious plans in second quarter presentations; Sarepta taps Michael Severino as its new CEO; and Amgen launches its official defense of Tavneos.
After leaving Tessera Therapeutics earlier this month, Michael Severino will take over Sarepta Therapeutics as the biotech awaits two key regulatory decisions in Duchenne muscular dystrophy.
Sarepta Therapeutics is seeking to convert the accelerated approval of its therapeutic exon-skippers for Duchenne muscular dystrophy to full despite the drugs’ failure to improve motor function in a confirmatory trial.
Analysts homed in on Duchenne muscular dystrophy and myotonic dystrophy type 1 assets during first quarter earnings as major players like REGENXBIO and Novartis as well as Dyne, Wave, Solid and Sarepta near the regulatory finish line.
The recent approval of Regeneron’s Otarmeni underscores the maturation of gene therapies across a range of diseases. Here, BioSpace reviews genetic medicines in development for the central nervous system, retinal, cardiac and neuromuscular diseases.
Sarepta Therapeutics has put in place several initiatives to help its gene therapy Elevidys return to growth, but recovery will take a long time, according to company executives.
As Q1 earnings arrive, three biotechs have big quarters ahead, with two—Amylyx and Neumora Therapeutics—betting at least partly on novel assets for obesity.
At Sarepta Therapeutics, we’ve seen it all. Here are the questions I believe we should be asking to move forward in Duchenne muscular dystrophy.
Biomarker data for Sarepta Therapeutics’ RNA programs, licensed from Arrowhead Pharmaceuticals, are “competitive” and “strong,” according to analysts at Jefferies, which projected over $1 billion in peak sales.
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