Sarepta Therapeutics announced on Mar. 26 that early biomarker data from two of its RNA interference therapies, licensed from Arrowhead Pharmaceuticals, show promising results as the company moves forward following challenges with its previous gene therapy programs.
The announcement comes after Sarepta shifted focus away from gene therapies due to safety concerns and a significant drop in company valuation. The move is seen as a critical step for the biotech firm, which had faced multiple patient deaths linked to its Elevidys gene therapy for Duchenne muscular dystrophy and subsequent financial losses.
According to Sarepta, a single dose of SRP-1001—an investigational treatment for facioscapulohumeral muscular dystrophy (FSHD)—led to a reduction of 90% to 93% in the expression of genes abnormally activated in FSHD. The company also reported rapid decreases in serum creatine kinase levels, an indicator of muscle damage. Jefferies analysts described these findings as “competitive,” highlighting the longer dosing interval compared to other candidates for FSHD.
The second asset highlighted by Sarepta is SRP-1003, being studied for myotonic dystrophy type 1 (DM1). Sarepta said that at its lowest dose, SRP-1003 "significantly outperforms" similar therapies from Dyne Therapeutics and Avidity Biosciences based on available data. However, the company cautioned that no direct head-to-head clinical studies have been conducted so far and stated that “cross-trial comparisons may not be reliable.”
Jefferies analysts called the DM1 data “strong,” stating: "SRP-1003 could show superior efficacy/safety and provide less-frequent dosing." H.C. Wainwright analysts commented: "In our view, the early data are compelling and appear to validate the core thesis underpinning the deal." Both firms estimate around $1 billion in peak sales potential for each program.
The positive readouts offer renewed optimism not only for Sarepta but also reflect well on Arrowhead Pharmaceuticals’ technology platform as both companies look ahead to further development.
