FDA Guidance Document Supportive of Sarepta (SRPT) - William Blair

William Blair analyst Tim Lugo said the FDA's guidance document released today entitled “Human Gene Therapy for Rare Diseases" is likely supportive of Sarepta Therapeutics (NASDAQ: SRPT) ongoing microdystrophin gene therapy trial for Duchenne muscular dystrophy (DMD) as pivotal for a BLA filing under accelerated approval conditions

Lugo highlights the guidance document: "efforts should be made early in the GT product development program to identify and validate biomarkers … Some biomarkers or endpoints are very closely linked to the underlying pathophysiology of the disease … Changes in such biomarkers could be used during drug development for dose-selection, or even as an early demonstration of drug activity."

He comments, "Based on precedence set by DMD-specific guidance highlighting exon-skipped dystrophin expression as an appropriate surrogate endpoint for accelerated approval basis (Exondys 51 and golodirsen under rolling NDA review), we believe microdystrophin expression post-gene therapy could also satisfy this requirement with preclinical and clinical data at Sarepta’s R&D day supporting this notion (see our note: Microdystrophin Gene Therapy the Star at R&D Day, Limb Girdle Gene Therapy Next Up as Dosing to Begin Soon ). Recall, data from 3 patients (age 4-7 years) dosed with 2x1014vg/kg rAAVrh74.MHCK7.micro-dystrophin showed a mean 76.2% micro-dystrophin positive fibers assessed by gastrocnemius biopsy at three months post-infusion (exhibit 1). Micro-dystrophin protein expression was also quantified by Western blot displaying a mean of 38.2% or 53.7% compared to normal (full-length dystrophin standard curve) using Sarepta’s or Nationwide Children’s hospital method respectively (exhibit 2)."

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Shares of Sarepta Therapeutics closed at $138.04 yesterday.

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