A Study of CAP-1002 in Ambulatory and Non-Ambulatory Patients With Duchenne Muscular Dystrophy

- Approximately 84 eligible study participants will be randomized to either CAP-1002 or
placebo in a 1:1 ratio.

- The trial will include visits at Screening, Baseline/Day 1, Week 4, and Months 3, 6, 9,
and 12 with IV infusions of CAP-1002 or placebo on Day 1 and Months 3, 6, and 9.

- Safety evaluations will include adverse events, concomitant medications, physical exam,
vital signs, 12-lead ECG, and clinical laboratory testing.

- Efficacy will be evaluated in the Performance of the Upper Limb, pulmonary function
testing, North Star Ambulatory Assessment (ambulatory subjects only), strength testing,
cardiac MRI, and quality of life.

- If trial data suggests an appropriate risk/benefit profile of CAP-1002, Capricor, upon
the recommendation of the Data Safety Monitoring Board (DSMB), will introduce an
open-label extension study to offer CAP-1002 to study participants who were randomized
to placebo and completed all trial visits during the 12-month period.

Inclusion Criteria:

1. Genetically confirmed DMD

2. Reduced upper arm strength as measured by the Performance of Upper Limb

3. Reduced ability to walk/run (if ambulatory)

4. Treatment with systemic glucocorticoids for at least 12 months and at a stable dose at
least 6 months prior to study participation, except for weight-based or
toxicity-related adjustments

5. Current and up-to-date immunizations

Exclusion Criteria:

1. Left ventricular ejection fraction < 35%

2. BMI > 45

3. Ambulant if ≥ 18 years of age

4. Exon 44 skip-amenable mutation(s) in the DMD gene

5. Deletion mutation(s) encompassing exons 3-7 of the DMD gene

6. Percent-predicted forced vital capacity (FVC) < 35%

7. Chronic respiratory disease not related to DMD (for example, asthma, bronchitis, and
tuberculosis)

8. History of diabetes requiring treatment with metformin or insulin within 3 months
prior to randomization

9. Treatment with an FDA-approved exon skipping therapy for the treatment of DMD if on a
stable dose for less than 24 months prior to randomization

10. Treatment with human growth hormone (HGH) within 3 months prior to randomization,
unless on a stable dose for at least 24 months prior to randomization

11. Treatment with idebenone within 3 months prior to randomization

12. Treatment with a cell therapy product within 12 months prior to randomization

13. Treatment with an investigational product within 6 months prior to randomization