Sarepta Stock Collapses After Duchenne Gene Therapy Disappointment

Sarepta Therapeutics (SRPT) saw its stock plunge 35% on Tuesday after revealing that a late-stage study testing two gene-targeted therapies for Duchenne muscular dystrophy (DMD) did not meet its main efficacy goal.

Trial Miss Sparks Investor Concern

The failed study represents a major setback for Sarepta’s pipeline in treating DMD — a rare genetic disorder that causes progressive muscle weakness and degeneration in young boys. Investors had been hopeful the therapies would demonstrate strong functional improvements.

While the company did not release detailed secondary endpoint data, the miss on the primary goal immediately shook confidence in Sarepta’s future revenue prospects tied to its gene therapy programs.

Market Reaction

Shares tumbled over 35% in early trading, marking one of the steepest single-day declines in Sarepta’s history. Analysts warned that the company could face regulatory and commercial hurdles if additional data fails to show meaningful clinical benefit.

What’s Next

Sarepta said it plans to analyze the full dataset and discuss next steps with regulators. The biotech firm may seek alternative endpoints or follow-up studies to assess longer-term benefits.