Muscular Dystrophy, Duchenne · registered primary completion 2026-10-31 · NCT03992430
105,627,021 shares outstanding against 133,471,021 if every outstanding option, warrant and convertible were exercised. In that case 20.9% of a gain in enterprise value would accrue to those holders rather than to current shareholders.
Registered design for NCT03992430: randomized, quadruple-masked, 160 participants (actual). Primary outcome: Part 1: Incidence of Adverse Events (AEs). 2 further primary outcome(s) registered.
Designations disclosed: RMAT, Breakthrough Therapy, Fast Track, Orphan Drug, Priority Review Voucher. These accelerate review and, for Orphan, confer exclusivity if approved; none is evidence of efficacy.
11 distinct molecules in 10 active company-sponsored trials; delandistrogene moxeparvovec accounts for 3 of them at Phase 1/Phase 3.