Sarepta: Phase 3 clinical development considerations for SRP-5051 in patients with Duchenne muscular dystrophy
Sarepta Non-confidential report
Sarepta’s application for advice sought to gain input on the development of a new candidate therapeutic, SRP-5051, a peptide-conjugated phosphorodiamidate morpholino oligomer (PPMO), for exon 51 skipping in eligible patients with Duchenne muscular dystrophy. SRP-5051 has demonstrated increased delivery in cell culture and animals, and improved exon skipping and dystrophin production in cardiac and skeletal muscle. Preclinical studies in animal models show target engagement, efficacy, and tolerability.
TACT welcomed the opportunity to review this application and stressed the importance to also refer back to advice given in 2018. Suggestions were given by the panel for additional pre-clinical studies, learnings from Phase 1 and 2 and for the design of Phase 3.
Name of applicant: Jon Tinsley Reviewed: October, 2019 in Toronto, ON, Canada
Sarepta’s application for advice sought to gain input on the development of a new candidate therapeutic, SRP-5051, a peptide-conjugated phosphorodiamidate morpholino oligomer (PPMO), for exon 51 skipping in eligible patients with Duchenne muscular dystrophy. SRP-5051 has demonstrated increased delivery in cell culture and animals, and improved exon skipping and dystrophin production in cardiac and skeletal muscle. Preclinical studies in animal models show target engagement, efficacy, and tolerability.
TACT welcomed the opportunity to review this application and stressed the importance to also refer back to advice given in 2018. Suggestions were given by the panel for additional pre-clinical studies, learnings from Phase 1 and 2 and for the design of Phase 3.
