Sarepta Reports One Heart Inflammation Case In Duchenne Gene Therapy Trial

  • Sarepta Therapeutics Inc SRPT shared new functional data across multiple studies from the SRP-9001 (delandistrogene moxeparvovec) program for Duchenne muscular dystrophy. 
  • SRP-9001 is an investigational gene therapy under development in partnership with Roche Holdings AG RHHBY
  • Results from Cohort 1 demonstrated a 3.8-point improvement and 3.2-point improvement on the Duchenne functional scale 52 weeks after treatment compared to a propensity-score weighted external control.
  • Related: Sarepta Therapeutics' Shares Plummet Following FDA's Clinical Hold On MOMENTUM Study.
  • Additionally, across multiple new analyses, SRP-9001 treated patients showed statistically significant and clinically meaningful benefits versus propensity-matched external controls.
  • After four years, SRP-9001-treated participants had a positive mean 7.0-point difference on the Duchenne functional scale compared to baseline. 
  • The safety and tolerability profile of SRP-9001 is similar to past reports. The most common treatment-related adverse event was vomiting. Increases in liver enzymes were transient and responsive to steroids. 
  • In Study 9001-103, there was one new serious adverse event of myocarditis in Cohort 2, resolved after one month.
  • Price Action: SRPT shares are down 1.98% at $74.24 during the market session on the last check Wednesday.

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