Evaluated in a robust clinical trial program in Duchenne1
ELEVIDYS was evaluated in multiple clinical trials that included a broad patient population of more than 200 ambulatory (n=206) and non-ambulatory (n=8) individuals. Clinical trials are ongoing to verify long-term data as well as outcomes in non-ambulatory individuals.22-24
KEY DEMOGRAPHICS1
Characteristic |
---|
Race group, White (%) |
Mean age, years (range) |
Mean weight, kg (range) |
STUDY 1 (Part 1)
ELEVIDYS n=20 | Placebo n=21 |
---|---|
65% | 81% |
6.3 (4.5 to 7.9) | 6.2 (4.3 to 7.9) |
23.3 (18.0 to 34.5) | 21.6 (15.0 to 30.0) |
STUDY 2*
ELEVIDYS N=48 |
---|
77% |
7.7 (3.2 to 20.2) |
30.1 (12.5 to 80.1) |
STUDY 3
ELEVIDYS n=63 | Placebo n=62 |
---|---|
78% | 74% |
6.0 (4.1 to 7.9) | 6.1 (4.0 to 7.9) |
21.3 (13.5 to 38.5) | 22.4 (14.4 to 41.6) |
Characteristic |
---|
Race group, White (%) |
Mean age, years (range) |
Mean weight, kg (range) |
STUDY 1 (Part 1)
ELEVIDYS n=20 | Placebo n=21 |
---|---|
65% | 81% |
6.3 (4.5 to 7.9) | 6.2 (4.3 to 7.9) |
23.3 (18.0 to 34.5) | 21.6 (15.0 to 30.0) |
Characteristic |
---|
Race group, White (%) |
Mean age, years (range) |
Mean weight, kg (range) |
STUDY 2*
ELEVIDYS N=48 |
---|
77% |
7.7 (3.2 to 20.2) |
30.1 (12.5 to 80.1) |
Characteristic |
---|
Race group, White (%) |
Mean age, years (range) |
Mean weight, kg (range) |
STUDY 3
ELEVIDYS n=63 | Placebo n=62 |
---|---|
78% | 74% |
6.0 (4.1 to 7.9) | 6.1 (4.0 to 7.9) |
21.3 (13.5 to 38.5) | 22.4 (14.4 to 41.6) |
* Cohorts 1, 2, 4, and 5a included 40 ambulatory patients; Cohorts 3 and 5b included 8 non-ambulatory patients.
Patients with moderate to severe left ventricular ejection fracture (LVEF) impairment have not been studied in clinical trials with ELEVIDYS.
Completed, multicenter trial in 41 ambulatory males aged 4 to 7 years1:
- Part 1: 48-week, double-blind, placebo-controlled randomized period; randomization stratified by age (4 to 5 years vs 6 to 7 years)
- Part 2: 48-week, double-blind, placebo-controlled crossover† period after Part 1
PRIMARY OBJECTIVES
- ELEVIDYS micro-dystrophin expression in skeletal muscle, as measured by western blot
- Motor function, as measured by NSAA total score
KEY BASELINE CHARACTERISTICS (PART 1)
Characteristic | All N=41 | ELEVIDYS n=20 | Placebo n=21 |
---|---|---|---|
Mean age, years (range) | 6.3 (4.3 to 7.9) | 6.3 (4.5 to 7.9) | 6.2 (4.3 to 7.9) |
Mean weight, kg (range) | 22.4 (15.0 to 34.5) | 23.3 (18.0 to 34.5) | 21.6 (15.0 to 30.0) |
Mean NSAA total score (range) | 21.2 (13 to 29) | 19.8 (13 to 26) | 22.6 (15 to 29) |
Mean time to rise from floor, seconds (range) | 4.3 (2.7 to 10.4) | 5.1 (3.2 to 10.4) | 3.6 (2.7 to 4.8) |
† In the crossover period, patients randomized to placebo in Part 1 received ELEVIDYS in Part 2, and patients randomized to ELEVIDYS in Part 1 received placebo in Part 2.
NSAA=North Star Ambulatory Assessment; TTR=time to rise.
Ongoing, open-label, multicenter trial in 40 ambulatory males (cohorts 1, 2, 4, and 5a) and 8 non-ambulatory males (cohorts 3 and 5b)1
PRIMARY OBJECTIVE
- ELEVIDYS micro-dystrophin in skeletal muscles, as measured by western blot
KEY BASELINE CHARACTERISTICS
Characteristic | Cohort 1 n=20 | Cohort 2 n=7 | Cohort 3 n=6 | Cohort 4 n=7 | Cohort 5a n=6 | Cohort 5b n=2 |
---|---|---|---|---|---|---|
Mean age, years (range) | 5.8 (4.4 to 7.9) | 10.1 (8.0 to 12.1) | 15.3 (9.9 to 20.2) | 3.5 (3.2 to 3.9) | 6.7 (4.7 to 8.6) | 13.4 (12.3 to 14.6) |
Mean weight, kg (range) | 21.2 (15.2 to 33.1) | 37.1 (28.0 to 50.5) | 59.9 (36.1 to 80.1) | 15.2 (12.5 to 16.5) | 32.1 (19.1 to 47.4) | 51.2 (43.4 to 59.0) |
Mean NSAA total score (range) | 22.1 (18 to 26) | 20.7 (17 to 26) | NA‡ | 12.9 (11 to 17) | 22.5 (18 to 30) | NA‡ |
Mean TTR from floor, seconds (range) | 4.2 (2.4 to 8.2) | 5.9 (3.8 to 9.7) | NA‡ | 5.2 (3.8 to 6.7) | 4.6 (2.5 to 7.7) | NA‡ |
Characteristic | Cohort 1 n=20 | Cohort 2 n=7 | Cohort 3 n=6 |
---|---|---|---|
Mean age, years (range) | 5.8 (4.4 to 7.9) | 10.1 (8.0 to 12.1) | 15.3 (9.9 to 20.2) |
Mean weight, kg (range) | 21.2 (15.2 to 33.1) | 37.1 (28.0 to 50.5) | 59.9 (36.1 to 80.1) |
Mean NSAA total score (range) | 22.1 (18 to 26) | 20.7 (17 to 26) | NA‡ |
Mean TTR from floor, seconds (range) | 4.2 (2.4 to 8.2) | 5.9 (3.8 to 9.7) | NA‡ |
Characteristic | Cohort 4 n=7 | Cohort 5a n=6 | Cohort 5b n=2 |
---|---|---|---|
Mean age, years (range) | 3.5 (3.2 to 3.9) | 6.7 (4.7 to 8.6) | 13.4 (12.3 to 14.6) |
Mean weight, kg (range) | 15.2 (12.5 to 16.5) | 32.1 (19.1 to 47.4) | 51.2 (43.4 to 59.0) |
Mean NSAA total score (range) | 12.9 (11 to 17) | 22.5 (18 to 30) | NA‡ |
Mean TTR from floor, seconds (range) | 5.2 (3.8 to 6.7) | 4.6 (2.5 to 7.7) | NA‡ |
‡ Not evaluated in non-ambulatory patients.
NSAA=North Star Ambulatory Assessment; TTR=time to rise.
Ongoing, randomized, multicenter, double-blind, placebo-controlled trial in 125 ambulatory males aged 4 to 7 years1
PRIMARY OBJECTIVE
- Motor function, as measured by NSAA total score
KEY SECONDARY OBJECTIVES
- ELEVIDYS micro-dystrophin expression in skeletal muscle, as measured by western blot
- Motor function, as measured by:
- TTR from floor
- Time of 10MWR
ADDITIONAL OBJECTIVES
- Motor function, as measured by:
- Time of 100MWR
- Time to ascend 4 steps
KEY BASELINE CHARACTERISTICS1,2,25
Characteristic | ELEVIDYS n=63 | Placebo n=62 |
---|---|---|
Mean age, years (range) 4-5 years, n (%) 6-7 years, n (%) | 6.0 (4.1 to 7.9) | 6.1 (4.0 to 7.9) |
Mean weight, kg (range) | 21.3 (13.5 to 38.5) | 22.4 (14.4 to 41.6) |
Mean time since corticosteroid treatment started, years (range) | 1.07 (0.23 to 6.17) | 0.97 (0.24 to 4.01) |
PRIMARY OBJECTIVES | ||
Mean NSAA total score, points (range) | 23.10 (14 to 32) | 22.82 (15.5 to 30) |
KEY SECONDARY OBJECTIVES | ||
Mean TTR from floor, seconds (range) | 3.52 (1.9 to 5.8) | 3.60 (2.3 to 5) |
Mean 10MWR, seconds (range) | 4.82 (3.2 to 6.9) | 4.92 (3.7 to 7) |
ADDITIONAL OBJECTIVES | ||
Mean 100MWR, seconds (range) | 60.67 (38.0 to 129.2) | 63.01 (38.7 to 118.1) |
Mean time to ascend 4 steps, seconds (range) | 3.17 (1.6 to 7.1) | 3.37 (1.5 to 7.1) |
MWR=meter walk/run; NSAA=North Star Ambulatory Assessment; TTR=time to rise.