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.
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