Sarepta Announces Duchenne Gene Therapy and Exon-Skipping Presentations at World Muscle Society Congress

Key Takeaways

• Sarepta Therapeutics will present six posters on its Duchenne muscular dystrophy (DMD) portfolio at the 31st Annual Congress of the World Muscle Society (WMS), September 29 to October 3 in Hiroshima, Japan.
• Five of the posters cover ELEVIDYS (delandistrogene moxeparvovec), the company's gene therapy, including a late-breaking poster on efficacy and safety in ambulatory patients who were 8 to 12 years old at treatment.
• The sixth covers the Phase 3 ESSENCE study of golodirsen and casimersen versus placebo. All six posters are scheduled for Wednesday, September 30.

Sarepta Therapeutics, a Cambridge, Massachusetts-based company focused on precision genetic medicines for rare diseases, announced it will present new data from its DMD portfolio at this year's WMS Congress. Sarepta says the poster abstracts and presentations will be posted in the Events & Presentations section of its investor relations website after they are presented.

ELEVIDYS is a single-dose, AAV-based gene transfer therapy given by intravenous infusion. It is designed to address the genetic cause of DMD, mutations in the DMD gene that result in a lack of dystrophin protein, by delivering a transgene that produces a micro-dystrophin protein in skeletal muscle. It is indicated for ambulatory patients 4 years of age and older with a confirmed DMD mutation. The prescribing information carries a boxed warning for acute serious liver injury and acute liver failure.

The late-breaking poster (1.103LBP), led by C. McDonald, reports functional and safety outcomes in older ambulatory patients from Sarepta's clinical studies who were 8 to 12 years old when treated. Four more ELEVIDYS posters are scheduled for the same day:

• Functional and safety outcomes up to three years after infusion in the EMBARK study (J. Mendell)
• Micro-dystrophin expression and safety in 2- to 3-year-olds in the ENDEAVOR and ENVOL studies (C. McDonald)
• A pooled safety analysis across Phase 1 to Phase 3 trials (C. Proud)
• A preclinical study of early intervention in DMDMDX mice (N. Pukos)

The sixth poster covers the Phase 3 ESSENCE study of golodirsen and casimersen compared with placebo, including a post hoc analysis (F. Muntoni).

The late-breaking poster is scheduled for 2:30 to 3:30 PM JST on Wednesday, September 30 (1:30 to 2:30 AM Eastern). The other five will be presented from 5:15 to 6:15 PM JST the same day (4:15 to 5:15 AM Eastern).