Sarepta Therapeutics, Inc.
NEWS
Faced with the encroaching threats of patent expirations and generics, biopharma companies in 2024 invested 33% more in licensing deals, on average, than in 2023 with an eye toward enriching their pipelines with novel and potentially more effective therapies.
While the last decade has brought considerable progress for patients with DMD, substantial unmet need remains. Several companies including Wave, Dyne and Avidity are looking to answer the call with investigational therapies targeting greater efficacy and broader reach.
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.
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