Efficacy

AGAMREE Improved Muscle Strength and Motor Function

AGAMREE significantly improved motor function as assessed by the Time to Stand Test (TTSTAND velocity)1,2*

Line graph showing that in clinical studies AGAMREE significantly improved motor function as assessed by the Time to Stand Test (TTSTAND velocity): Primary endpoint: AGAMREE 6 milligrams per kilogram of weight per day improved TTSTAND velocity at Week 24 vs placebo (least-square mean difference vs placebo, 0.06 rises/s [P=0.002]). Prespecified secondary endpoint: AGAMREE 2 milligrams per kilogram of weight per day improved TTSTAND velocity at Week 24 vs placebo (least-square mean difference vs placebo, 0.045 rises/s [P=0.017]) Improvements with AGAMREE 6 milligrams per kilogram of weight per day and 2 milligrams per kilogram of weight per day were clinically meaningful (MCID, >0.02 rises/s).
Line graph showing that in clinical studies AGAMREE significantly improved motor function as assessed by the Time to Stand Test (TTSTAND velocity): Primary endpoint: AGAMREE 6 milligrams per kilogram of weight per day improved TTSTAND velocity at Week 24 vs placebo (least-square mean difference vs placebo, 0.06 rises/s [P=0.002]). Prespecified secondary endpoint: AGAMREE 2 milligrams per kilogram of weight per day improved TTSTAND velocity at Week 24 vs placebo (least-square mean difference vs placebo, 0.045 rises/s [P=0.017]) Improvements with AGAMREE 6 milligrams per kilogram of weight per day and 2 milligrams per kilogram of weight per day were clinically meaningful (MCID, >0.02 rises/s).
  • Primary endpoint: AGAMREE 6 mg/kg/d improved TTSTAND velocity at Week 24 vs placebo (least-square mean difference vs placebo, 0.06 rises/s [P=0.002])1
  • Prespecified secondary endpoint: AGAMREE 2 mg/kg/d improved TTSTAND velocity at Week 24 vs placebo (least-square mean difference vs placebo, 0.045 rises/s [P=0.017])1
  • Improvements with AGAMREE 6 mg/kg/d and 2 mg/kg/d were clinically meaningful (MCID, >0.02 rises/s)2,3

Study Design: Study 1 was a multicenter, randomized, double-blind, placebo- and active-controlled clinical trial in 121 boys aged 4 to <7 years of age with a confirmed DMD diagnosis who were ambulatory and who had not been previously treated with corticosteroids. Patients were randomized to treatment with AGAMREE 6 mg/kg/d (n=30), AGAMREE 2 mg/kg/d (n=30), prednisone 0.75 mg/kg/d (n=31), or placebo (n=30) for 24 weeks.1 At baseline, patients had a mean age of 5.4 years, mean height of 109 cm, and mean weight of 20 kg.2

TTSTAND velocity measures the time required to stand to an erect position from a supine position (floor).1

Baseline mean TTSTAND velocity, rises/s (SD): AGAMREE 6 mg/kg/d: 0.19 (0.06); AGAMREE 2 mg/kg/d, 0.18 (0.05); placebo, 0.20 (0.06).2

DMD, Duchenne muscular dystrophy; MCID, minimal clinically important difference; SD, standard deviation; SEM, standard error of the mean.

Prespecified Secondary Endpoints

AGAMREE significantly improved motor function as assessed by the 6-Minute Walk Test (6MWT)1,2‡

  • AGAMREE 6 mg/kg/d and 2 mg/kg/d improved 6MWT vs placebo (least-square mean change from baseline vs placebo, 42 m [P=0.002] and 40 m [P=0.004], respectively)1
    • Improvements with AGAMREE were clinically meaningful (MCID >30 m)2

See study design for Study 1, above.

‡6MWT measures the distance a patient can walk on a flat hard surface in a period of 6 minutes.1

Baseline mean 6WMT, m (SD): AGAMREE 6 mg/kg/d: 313 (56); AGAMREE 2 mg/kg/d, 316 (58); placebo, 355 (78).2

DMD, Duchenne muscular dystrophy; MCID, minimal clinically important difference; SD, standard deviation; SEM, standard error of the mean.

AGAMREE significantly improved motor function as assessed by Time to Run/Walk 10 Meters (TTRW velocity)1,2¶

  • AGAMREE 6 mg/kg/d improved TTRW velocity at Week 24 vs placebo(least-square mean change from baseline vs placebo, 0.24 m/s [P=0.002])1
    • Improvement with AGAMREE was clinically meaningful (MCID, >0.21 m/s)3

See study design for Study 1, above.

TTRW velocity measures the time it takes a patient to run or walk 10 meters.1

Baseline mean TTRW velocity, m/s (SD): AGAMREE 6 mg/kg/d: 1.6 (0.4); AGAMREE 2 mg/kg/d, 1.6 (0.3); placebo, 1.7 (0.3).2

DMD, Duchenne muscular dystrophy; MCID, minimal clinically important difference; SD, standard deviation; SEM, standard error of the mean.

Safety

AGAMREE Was Well Tolerated in Clinical Studies

  • The most common adverse reactions (>10% and greater than placebo) in boys with DMD treated with AGAMREE in Study 1 were cushingoid features, psychiatric disorders, vomiting, weight increased, and vitamin D deficiency1
Table showing adverse reactions to AGAMREE in a clinical study: these reactions occurred in greater than or equal to 5 percent of patients treated with AGAMREE and more frequently than in patients who received placebo during 24 weeks. Cushingoid features were reported in 0% of patients given a placebo (n=29), 7% of patients given 2 milligrams of AGAMREE per kilogram of weight per day (n=30), and 29% of patients given 6 milligrams of AGAMREE per kilogram of weight per day (n=28); psychiatric disorders were reported in 14% of patients given a placebo (n=29), 7% of patients given 2 milligrams of AGAMREE per kilogram of weight per day (n=30), and 21% of patients given 6 milligrams of AGAMREE per kilogram of weight per day (n=28); vomiting was reported in 7% of patients given a placebo (n=29), 17% of patients given 2 milligrams of AGAMREE per kilogram of weight per day (n=30), and 14% of patients given 6 milligrams of AGAMREE per kilogram of weight per day (n=28); a weight increase was reported in 3% of patients given a placebo (n=29), 0% of patients given 2 milligrams of AGAMREE per kilogram of weight per day (n=30), and 11% of patients given 6 milligrams of AGAMREE per kilogram of weight per day (n=28); a vitamin D deficiency was reported in 0% of patients given a placebo (n=29), 7% of patients given 2 milligrams of AGAMREE per kilogram of weight per day (n=30), and 11% of patients given 6 milligrams of AGAMREE per kilogram of weight per day (n=28); a cough was reported in 3% of patients given a placebo (n=29), 10% of patients given 2 milligrams of AGAMREE per kilogram of weight per day (n=30), and 7% of patients given 6 milligrams of AGAMREE per kilogram of weight per day (n=28); headache was reported in 3% of patients given a placebo (n=29), 7% of patients given 2 milligrams of AGAMREE per kilogram of weight per day (n=30), and 7% of patients given 6 milligrams of AGAMREE per kilogram of weight per day (n=28); diarrhea was reported in 3% of patients given a placebo (n=29), 3% of patients given 2 milligrams of AGAMREE per kilogram of weight per day (n=30), and 7% of patients given 6 milligrams of AGAMREE per kilogram of weight per day (n=28); increased appetite was reported in 3% of patients given a placebo (n=29), 3% of patients given 2 milligrams of AGAMREE per kilogram of weight per day (n=30), and 7% of patients given 6 milligrams of AGAMREE per kilogram of weight per day (n=28); and rhinitis reported in 3% of patients given a placebo (n=29), 3% of patients given 2 milligrams of AGAMREE per kilogram of weight per day (n=30), and 7% of patients given 6 milligrams of AGAMREE per kilogram of weight per day (n=28).
Table showing adverse reactions to AGAMREE in a clinical study: these reactions occurred in greater than or equal to 5 percent of patients treated with AGAMREE and more frequently than in patients who received placebo during 24 weeks. Cushingoid features were reported in 0% of patients given a placebo (n=29), 7% of patients given 2 milligrams of AGAMREE per kilogram of weight per day (n=30), and 29% of patients given 6 milligrams of AGAMREE per kilogram of weight per day (n=28); psychiatric disorders were reported in 14% of patients given a placebo (n=29), 7% of patients given 2 milligrams of AGAMREE per kilogram of weight per day (n=30), and 21% of patients given 6 milligrams of AGAMREE per kilogram of weight per day (n=28); vomiting was reported in 7% of patients given a placebo (n=29), 17% of patients given 2 milligrams of AGAMREE per kilogram of weight per day (n=30), and 14% of patients given 6 milligrams of AGAMREE per kilogram of weight per day (n=28); a weight increase was reported in 3% of patients given a placebo (n=29), 0% of patients given 2 milligrams of AGAMREE per kilogram of weight per day (n=30), and 11% of patients given 6 milligrams of AGAMREE per kilogram of weight per day (n=28); a vitamin D deficiency was reported in 0% of patients given a placebo (n=29), 7% of patients given 2 milligrams of AGAMREE per kilogram of weight per day (n=30), and 11% of patients given 6 milligrams of AGAMREE per kilogram of weight per day (n=28); a cough was reported in 3% of patients given a placebo (n=29), 10% of patients given 2 milligrams of AGAMREE per kilogram of weight per day (n=30), and 7% of patients given 6 milligrams of AGAMREE per kilogram of weight per day (n=28); headache was reported in 3% of patients given a placebo (n=29), 7% of patients given 2 milligrams of AGAMREE per kilogram of weight per day (n=30), and 7% of patients given 6 milligrams of AGAMREE per kilogram of weight per day (n=28); diarrhea was reported in 3% of patients given a placebo (n=29), 3% of patients given 2 milligrams of AGAMREE per kilogram of weight per day (n=30), and 7% of patients given 6 milligrams of AGAMREE per kilogram of weight per day (n=28); increased appetite was reported in 3% of patients given a placebo (n=29), 3% of patients given 2 milligrams of AGAMREE per kilogram of weight per day (n=30), and 7% of patients given 6 milligrams of AGAMREE per kilogram of weight per day (n=28); and rhinitis reported in 3% of patients given a placebo (n=29), 3% of patients given 2 milligrams of AGAMREE per kilogram of weight per day (n=30), and 7% of patients given 6 milligrams of AGAMREE per kilogram of weight per day (n=28).

Includes the following adverse reactions that occurred more frequently in the AGAMREE group than in placebo: abnormal behavior, aggression, agitation, anxiety, irritability, mood altered, sleep disorder, and stereotypy.

  • In a separate open-label safety study of pediatric patients with DMD aged 2 to <4 years (n=16) and pediatric patients aged 7 to <18 years (n=16), adverse reactions were similar to Study 11

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