A Phase 2 Randomized, Double-blind, Placebo-controlled Study to Evaluate the Effect of EDG-5506 on Safety, Biomarkers, Pharmacokinetics, and Functional Measures in Adults and Adolescents with Becker Muscular Dystrophy

Becker Muscular Dystrophy (BMD)

1. Cohorts 1 and 2: Incidence, frequency, and severity of adverse events (AEs) and serious adverse events (SAEs) during treatment with sevasemten or placebo., 2. Cohorts 1 and 2: Change from Baseline in serum creatine kinase (CK) averaged across months 6, 9 and 12., 3. Cohorts 4 and 5: Incidence, frequency, and severity of adverse events (AEs) and serious adverse events (SAEs) during treatment with sevasemten or placebo, 4. Cohort 6: Month 18 change from Baseline in total North Star Ambulatory Assessment (NSAA) score, 5. Cohort 6: Incidence, frequency, and severity of adverse events (AEs) and serious adverse events (SAEs) during treatment with sevasemten or placebo

1. Cohorts 1 and 2: Month 12 change from Baseline in total North Star Ambulatory Assessment (NSAA) score., 2. Cohorts 1 and 2: Cumulative loss of function on the NSAA over 12 months, 3. Cohorts 1 and 2: Month 12 Change from Baseline in: − Total North Star Assessment for Limb Girdle Muscular Dystrophies (NSAD) score − 10-meter walk/run − 100-meter timed test, 4. Cohorts 1 and 2: • Change from Baseline in serum myoglobin averaged across Months 6, 9, and 12 • Change from Baseline in TNNI2 averaged across Months 6 and 12, 5. Cohorts 1 and 2: Pharmacokinetics (PK) of sevasemten as measured by steady state (Css) plasma concentration of sevasemten., 6. Cohorts 1 and 2: Incidence of treatment-emergent abnormal laboratory test results (clinical chemistry, hematology, coagulation). Change from Baseline in: − Safety laboratory parameters − Vital signs − Electrocardiogram (ECG) parameters − Cardiac function as assessed by an echocardiogram − Pulmonary function as assessed by forced expiratory volume in 1 second (FEV1) , forced vital capacity (FVC), 7. Cohorts 4 and 5: Pharmacokinetics (PK) of sevasemten as measured by steady state (Css) plasma concentration of sevasemten., 8. Cohorts 4 and 5: Incidence of treatment-emergent abnormal laboratory test results (clinical chemistry, hematology, coagulation). Change from Baseline in: − Safety laboratory parameters − Vital signs − Electrocardiogram (ECG) parameters − Cardiac function as assessed by an echocardiogram − Pulmonary function as assessed by forced expiratory volume in 1 second (FEV1) , forced vital capacity (FVC) − Growth (as assessed by height centile on WHO growth charts), 9. Cohort 6: Month 18 change from Baseline in: - 100-meter timed test - 10-meter walk/run - Stride velocity (95th percentile) -Total North Star Assessment for Limb-Girdle Type Muscular Dystrophies (NSAD) score, 10. Cohort 6: Month 18 change from Baseline in fat fraction of upper leg muscles (as assessed by MRI), 11. Cohort 6: Pharmacokinetics (PK) of sevasemten as measured by steady state (Css) plasma concentration of sevasemten, 12. Cohort 6: Incidence of treatment-emergent abnormal laboratory test results (clinical chemistry, hematology, coagulation). Change from Baseline in: − Safety laboratory parameters − Vital signs − Electrocardiogram (ECG) parameters − Cardiac function as assessed by an echocardiogram − Pulmonary function as assessed by forced expiratory volume in 1 second (FEV1) , forced vital capacity (FVC), 13. Cohort 6: Month 18 change from Baseline in: - Serum Creatine kinase (CK) - Fast skeletal muscle troponin I (TNNI2). - Serum myoglobin, 14. Cohort 6: Cumulative loss of function on the North Star Ambulatory Assessment (NSAA) over 18 months

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