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
101View entire presentation