Sarepta shares PhII data for next-gen exon skipping drug, but regulatory pathway is unclear

Sarep­ta Ther­a­peu­tics an­nounced Mon­day that its ex­per­i­men­tal Duchenne mus­cu­lar dy­s­tro­phy drug led to some ex­pres­sion of dy­s­trophin — the mus­cle pro­tein that is mu­tat­ed in peo­ple with Duchenne — in a Phase II study.

In 20 pa­tients who re­ceived a high dose of SRP-5051 for 28 weeks, Sarep­ta re­port­ed a mean 5.17% in dy­s­trophin ex­pres­sion, and re­sults were rough­ly sim­i­lar be­tween am­bu­la­to­ry and non-am­bu­la­to­ry pa­tients. At the low dose, mean dy­s­trophin ex­pres­sion was 2.81% in 20 pa­tients. Those ex­pres­sion lev­els equat­ed to a change of 4.53% and 2.00%, re­spec­tive­ly, in ex­pres­sion lev­els from base­line.

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