AI & Machine Learning in Biotechnology
STAT+: Sarepta Therapeutics shares rise on early promise for rare disease drugs
Sarepta Therapeutics’ next crop of medicines for muscular dystrophy appear to be safe and effective in early clinical trials, the company said, a positive though incremental update for a drugmaker rebuilding its business after a tumultuous 2025.
Shares of Sarepta rose more than 20% in early trading Wednesday after the company presented preliminary data on a pair of treatments for rare, muscle-wasting diseases. Sarepta, which laid off more than one-third of its workforce last year after multiple patient deaths derailed its top-selling gene therapy for muscular dystrophy, has staked its future on a pipeline of unproven treatments for related conditions.
The early data on two of those drugs, SRP-1001 and SRP-1003, will be followed by more substantive updates later this year, Sarepta said, and pivotal studies will come in 2027.
Sarepta Therapeutics’ next crop of medicines for muscular dystrophy appear to be safe and effective in early clinical trials, the company said, a positive though incremental update for a drugmaker rebuilding its business after a tumultuous 2025.
Shares of Sarepta rose more than 20% in early trading Wednesday after the company presented preliminary data on a pair of treatments for rare, muscle-wasting diseases. Sarepta, which laid off more than one-third of its workforce last year after multiple patient deaths derailed its top-selling gene therapy for muscular dystrophy, has staked its future on a pipeline of unproven treatments for related conditions.
The early data on two of those drugs, SRP-1001 and SRP-1003, will be followed by more substantive updates later this year, Sarepta said, and pivotal studies will come in 2027.