Sarepta Therapeutics, Inc.
NEWS
Biopharma executives were busy Monday, striking high-value deals and providing updates on cancer, obesity and vaccine pipelines.
Sarepta Therapeutics’ Duchenne muscular dystrophy therapy Elevidys handily beat analysts’ expectations in the fourth quarter of 2024, reflecting the biotech’s “world-class” execution, according to BMO Capital Markets analysts.
Other notable greenlights this year include Bristol Myers Squibb’s Cobenfy, the first novel therapeutic for schizophrenia in 35 years, and Madrigal Pharmaceuticals’ Rezdiffra, the first-ever treatment for MASH.
Not exactly known for its dealmaking, Sarepta Therapeutics has thrown down a massive wad of cash to work with Arrowhead Pharmaceuticals on RNAi-based medicines.
With Elevidys expansion in hand, Sarepta commits up to $10 billion to develop short interfering RNA–based drugs to build out its pipeline.
The investigational therapy, vesleteplirsen, had been positioned as an updated version of Sarepta’s original exon 51-skipping Duchenne muscular dystrophy drug Exondys 51.
With Sarepta’s gene therapy Elevidys now available to a majority of Duchenne muscular dystrophy patients, experts express cautious optimism while emphasizing the need for further data.
Since its inception in 1992, the FDA’s accelerated approval pathway has helped shepherd nearly 300 new drugs to the market. However, recent years have seen a number of high-profile market withdrawals and failed confirmatory trials.
The recent invalidation of an AAV gene therapy patent overlooks the complexity of innovation in biotechnology and could put a broad swath of intellectual property at risk.
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