Sarepta Therapeutics, Inc. (SRPT) reported the first clinical results from two of its investigational siRNA programs, showing high muscle drug concentrations and favorable tolerability in early-stage studies for facioscapulohumeral muscular dystrophy type 1 and myotonic dystrophy type 1. The company said the findings reinforce confidence in its avß6 integrin-targeted delivery platform.
Facioscapulohumeral muscular dystrophy type 1 (FSHD1) and myotonic dystrophy type 1 (DM1) are rare, progressive neuromuscular diseases caused by toxic protein or mRNA overexpression. Sarepta noted that RNA-targeted therapies have historically struggled with rapid degradation before reaching muscle cells, a challenge the company aims to overcome with its targeted siRNA chemistry.
Early Phase 1/2 data from SRP-1001, an investigational siRNA therapy designed to reduce DUX4 protein in patients with FSHD1 and SRP-1003 (DM1), an investigational siRNA therapy for DM1 that targets and knocks down DMPK mRNA showed dose-dependent increases in muscle exposure, early biomarker effects, and no dose-limiting toxicities. Sarepta also reported proof-of-concept evidence that a single dose of each therapy supported knockdown of the intended target protein or mRNA. Most adverse events were mild to moderate and not dose dependent.
"These early clinical results showed high level of siRNA delivery to muscle, with no saturation of muscle uptake or dose-limiting safety signals to date," said Louise Rodino-Klapac, Ph.D., President, Research & Development and Technical Operations. She added that the data strengthen confidence in the platform's potential to deliver meaningful clinical benefit.
SRPT has traded between $10.42 and $74.40 over the past year. The stock is currently trading at $22.58, up 28.25%.
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