Sarepta's Gene Therapy for Duchenne Muscular Dystrophy Fails Phase 3 Trial, Shares Plummet as Analysts Downgrade Price Targets

Despite Trial Failure, Analysts Still See Significant Sales Potential for Elevidys and Sarepta Plans to Seek Expanded Approval in Other Age Groups

Sarepta's EMBARK Phase 3 trial on Elevidys treatment for Duchenne muscular dystrophy in ambulatory boys aged 4-7 years failed to meet primary endpoint. However, patients showed improvements, and key secondary endpoints exhibited positive results, leading to continued support for existing use in 4-5 year olds.
The failure of the trial led to a sharp fall in Sarepta's share price and triggered several analysts to lower their price targets. Despite this, continued support for the efficacy of Elevidys means it can remain on the market.
The trial data has cast uncertainty over the therapy's future growth due to potential reluctance from health insurers to cover it. Regardless, analysts predict the treatment is not likely to be pulled from the market.
Despite the trial failure, Elevidys still represents a significant financial opportunity for Sarepta, potentially bringing in nearly $2 billion in top-line durable sales. The exon skipping platform retains considerable potential, particularly in treating limb-girdle muscular dystrophy.
Sarepta is planning to seek approval for Elevidys use in other age groups beyond the current 4-5 year-olds, maintaining that the data collected holds enough evidence to capture most of the U.S. DMD market.