Pharma Update

Elevidys development program Largest development plans in the broadest patient population Ph III EMBARK study design1 Additional Ph II/III studies Randomisation (N=120) Definitive diagnosis of DMD Confirmed DMD mutation within 1:1 exons 18-44 or 46-79 Roche Elevidys Part 1:52 weeks* Week 129 Week 52 Part 2:52 weeks' Up to 5 years ENVOL Week 645 Week 104 delandistrogene moxeparvovec Muscle Muscle NSAA placebo NSAA biopsy biopsy OLE Study Muscle placebo NSAA biopsy delandistrogene moxeparvovec Muscle NSAA biopsy Part 1 patients will be randomized to treated or placebo (1:1) and stratified according to age and NSAA Primary endpoint Change in NSAA total score from baseline to Week 52 in Part 1 Study 302 21 patients Ages 0-3 Open label •Safety (primary) and Expression (secondary) Excludes mutations 1-17 EU study population ENVISION Study 303 Older ambulatory (n=28) / non-ambulatory patients (n=120) Total 148 patients Double-blind, placebo-controlled No upper age restrictions for non-ambulatory patients • Ambulatory: 8-18 ●Primary endpoint: PUL Excludes mutations 1-17 • Pivotal Ph III (EMBARK) trail: Two part design with an OLE study of up to 5 years; primary endpoint of change in NSAA total score from baseline to Week 52 in Part 1 ⚫ Ph III (ENVOL; Study 302): Open label trial in 0-3 year olds with safety as primary endpoint and expression as secondary ⚫ Ph III (ENVISION; Study 303): Randomized trial in older ambulatory/non-ambulatory patients without an upper age restriction • SV95C-SYDE, first ever digital endpoint approved as primary endpoint by EMA 1.Muntoni et al MDA 2022; *Double-blind, placebo-controlled. *Patients, caregivers, Investigators, and site staff remain blinded. Only a subset of patients will receive a muscle biopsy for expression assessments; DMD=Duchenne muscular dystrophy; NSAA=North Star Ambulatory Assessment; PUL-performance of upper limb; OLE=Open label extension; Delandistrogene moxeparvovec in collaboration with Sarepta Therapeutics; SV95C-Stride Velocity 95th Centile; SV95C-SYDE in collaboration with Sysnav 102

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