Safety and Efficacy of KER-065 in Participants With Duchenne Muscular Dystrophy

Prior treatment

• Exposure to any approved or investigational dystrophin restoration gene therapy product.
• Exposure to any approved or investigational dystrophin restoration product other than gene therapy (Except for exon-skipping therapy for Cohort A2).
• Exposure to any approved or investigational histone deacetylase inhibitor, antimyostatin therapy, therapy targeting transforming growth factor-beta ligands, or cell-based therapy.
• Treatment with immunosuppressant therapy (other than CS)

Other illness

• Clinical symptoms or signs of cardiomyopathy or heart failure.
• History of fracture of the upper limb
  • Nonambulatory Participants Only (Cohort N1):

Other

• Use of any other pharmacological treatment, except for CS
• Elbow-flexion contractures \> 30° in both upper extremities.
• Forced vital capacity (FVC) of \< 50% or requirement for daytime or nocturnal ventilation, except for nocturnal non-invasive ventilation AND inability to perform consistent FVC measurements within ± 15% during paired testing.

• Diagnosis of DMD, defined as the presence of phenotypic features at screening consistent with DMD AND documented mutation in the dystrophin gene consistent with the diagnosis of DMD using a clinically validated genetic test.
• Receiving a stable regimen of systemic CS (including, but not limited to, prednisone, prednisolone, deflazacort, or vamorolone) for at least 90 days before screening.
• Body weight of ≥ 25.0 kg.
  • Ambulatory Participants Only (Cohort A1 and A2):
• Ambulatory, defined as able to walk independently without assistive devices.
• Able to TTR in \< 10 seconds.
• Has a NSAA score ≥ 15 points.
• Cohort 2 only: Documentation of a stable dose of an approved exon-skipping therapy.
  • Nonambulatory Participants Only (Cohort N1):
• Nonambulatory, characterized as being unable to ambulate for a minimum of 3 months before first dose with onset of nonambulatory status AND a NSAA walk score of 0 and inability to perform the 10MWR.
• PUL v2.0 entry item score of 3 to 5, inclusive.