NS-050/NCNP-03 in Boys With DMD (Meteor50)

A Phase 1/2, First in Human, Multiple-dose, 2-part Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of NS-050/NCNP-03 in Boys With Duchenne Muscular Dystrophy (DMD)

This is a Phase 1/2 study of Multiple-Ascending Dose (MAD) levels for 12 weeks of treatment followed by 24 weeks of open-label treatment with a selected dose of NS-050/NCNP-03 administered once weekly to ambulant boys with DMD, who have a DMD exon deletion amenable to exon 50 skipping.

Inclusion Criteria:

• Male ≥ 4 years and <16 years of age;
• Confirmed DMD exon deletion in the dystrophin gene that is amenable to skipping of exon 50 to restore the dystrophin mRNA reading frame;
• Able to walk independently without assistive devices;
• Able to complete the TTSTAND without assistance in <20 seconds;
• Stable dose of glucocorticoid for at least 3 months and the dose is expected to remain on a stable dose for the duration of the study.

Other inclusion criteria may apply.

Exclusion Criteria:

• Evidence of symptomatic cardiomyopathy;
• Current or previous treatment with anabolic steroids (e.g., oxendolone, oxandrolone) or products containing resveratrol or adenosine triphosphate within 3 months prior to first dose of study drug;
• Currently taking another investigational drug or has taken another investigational drug within 3 months prior to the first dose of study drug;
• Surgery within the 3 months prior to the first dose of study drug or planned during the study duration;
• Having taken any gene therapy.

Other exclusion criteria may apply.