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Pharma Update

Elevidys, first FDA approved DMD gene therapy by partner Sarepta Ph III (EMBARK) results expected in Q4 Pooled analysis of studies 101/102/103* MDA 2023 Change from baseline in NSAA total score over 1 year** Development program Roche SAREPTA THERAPEUTICS LSM change in NSAA total score from baseline to Year 1 (±SE) 3.0 2.5 2.3 2.0 1.5 1.0 0.5 LSM A=2.4; P<0.0001 0.0 -0.1 -0.5 -1.0 Delandistrogene moxeparvovec (N=52) EC (n=105") Study DMD subgroup PhI Ph II Ph III Comment 101 Ambulatory, 4-7 yrs. 102 103 (ENDEAVOR) 301 (EMBARK) 302 (ENVOL) 303 (ENVISION) Ambulatory, 4-7 yrs. Ambulatory, 3-18 yrs Non-ambulatory, all ages Ambulatory, 4-7 yrs. Ambulatory, 0-3 yrs. Ambulatory, 8-18 yrs Non-ambulatory, all ages US approval (Sarepta) US approval (Sarepta) US approval (Sarepta)* EU filing and US label extension Expansion to younger DMD pts Expansion to older ambulatory and non-ambulatory DMD pts • Positive functional and clinically meaningful results up to 4 years after treatment with consistent safety US accelerated approval achieved by Sarepta in Q2 · First patient treated in the US in August · First ex-US approval in the UAE achieved • Ph III (ENVISION) in older ambulatory and all ages non-ambulatory patients started in Q2 2023 . Ph II (ENVOL) in 0-3 year old ambulatory patients to initiate in H2 2023 • Ph III (EMBARK) results in Q4 2023; data to be filed in the EU / International and to facilitate non-age-restricted expansion of the US label Elevidys (delandistrogene moxeparvovec) accelerated US approval by partner Sarepta Therapeutics; Zaidman, et al. MDA 2023; *For study 103 (ENDEAVOR) only cohort 1 was used; **Functional data from patients who received the 1.33x1014 vg/kg dose of delandistrogene moxeparvovec and the propensity-score-weighted EC cohort were compared; DMD-Duchenne muscular dystrophy; NSAA=North Star Ambulatory Assessment; LSM-least-squares mean; EC-external control; SE-standard error; UAE-United Arab Emirates 101
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