Sarepta Therapeutics (SRPT) said Wednesday that early clinical results showed that its investigational siRNA treatments SRP-1001 and SRP-1003 achieved high muscle concentrations in patients with neuromuscular diseases.

SRP-1001 and SRP-1003 demonstrated dose-dependent muscle exposure, early biomarker effects, and favorable tolerability in patients with facioscapulohumeral muscular dystrophy type 1 and myotonic dystrophy type 1, respectively, the company said.

In both phase 1/2 ascending dose studies, the majority of adverse events were mild to moderate and were not dose dependent, Sarepta said.