Half-Year 2022 Financial and Clinical Trials Update slide image

Half-Year 2022 Financial and Clinical Trials Update

Duchenne muscular dystrophy franchise update Pivotal Ph III development program expected to read out in 2023 Delandistrogene moxeparvovec NH1 R1 R2 R3 R5 R6 R7 R8 R9 R10 R11 R12 R13 R14 R15 R16 R17 R18 R19 H3 R20 R21 R22 R23 R24 H4 CR CT Ph lb ENDEAVOR (Study 103) Functional results: NSAA LSM change from baseline SAREPTA THERAPEUTICS PhI (Study 101) Roche ICNMD 2022 17TH INTERNATIONAL CONGRESS ON NEUROMUSCULAR DISEASES 5-9 July 2022 Brussels, Belgium NSAA total score over 4 years in treated patients vs. EC (unadjusted mean) 34 32 145 bp 795 bp 97 bp MHCK7 PROMOTER INTRON ITR 3,591 bp 53 bp 145 bp MICRO-DYSTROPHIN PA ITR ABD 581 582 583 2 CR AAVrh74 NSAA total score starting from mean score at baseline (mean ± SD) 2222220 0 4 8 12 24 36 52 Week LSM change from baseline in NSAA total score (mean ± SE) 5 4 3 2 10 WK 52 EC 1 year comparison Delandistrogene moxeparvovec Propensity-score-weighted EC Changes from baseline in NSAA were measured at Week 52 and compared to a propensity-score-weighted EC Commercially representative delandistrogene moxeparvovec led to improvements in motor function NNNNN WWW NSAA total score starting from mean score at baseline, +SD 16 14 Baseline Year 1 Year 2 Year 3 Year 4 Number of patients Delandistrogene moxeparvovec EC 4 21 4 4 4 21 19 20 21 A treated patients vs. EC: 9.9* Delandistrogene moxeparvovec (N=4) Propensity score-weighted EC (n=21) Targeted delivery of micro-dystrophin transgene to key muscle tissue can enable meaningful and durable functional response AAVrh74 vector: low likelihood of pre-existing immunity and high tropism for skeletal & cardiac muscles Expression potentiated by the MHCK7 promoter in cardiac & skeletal muscles • • In ENDEAVOR participants gained a mean 4.0 points in NSAA over 1 year vs baseline. The treatment difference vs an external control was 3.2 points which is clinically meaningful and highly statistically significant (p <0.0001) Consistent transduction, expression and safety demonstrated 4-year follow up for Study 101 (n=4): Patients maintained NSAA gain over 4 years at an age at which a decline would be expected (8-10 yrs) Ph III (EMBARK) on track to be fully enrolled by H2 2022; Ph III (ENVOL; study 302) in 0-3 year olds and Ph III (ENVISION, study 303) in older ambulatory / non ambulatory patients to be initiated in H2 2022 NSAA=North Star Ambulatory Assessment; LSM-least square mean(s); Ph 1/2a Trial of Delandistrogene Moxeparvovec in Patients with DMD: 4-year Update. Mendell J.R. et al., Journal of Neuromuscular Diseases, 2022: 9; s1, p. S97, PS03.01; One-year ENDEAVOR Data (Ambulatory, ≥4 to <8-year-olds): Phase 1b Trial of Delandistrogene Moxeparvovec in DMD. Lehman K. et al, Journal of Neuromuscular Diseases, 2022: 9; s1, p. S201, eP02.05.01. 30 50
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