Clinical Study Alert: Phase 2 Study of Brogidirsen in Boys with DMD

Researchers at NS Pharma are seeking boys with Duchenne muscular dystrophy (DMD) amenable to exon 44 skipping therapy to participate in a phase 2 clinical trial to evaluate the safety, efficacy, and dynamics in the body of the investigational exon 44 skipping therapy brogidirsen (NS-089/NCNP-02-201). DMD is caused by mutations that lead to the loss of the dystrophin protein, which is important for the structure and function of muscle cells. Brogidirsen is designed to target and promote skipping over a section of genetic code (exon 44) in order to avoid the gene mutation and produce more of the dystrophin protein. In a previous Phase 1/2 study (NCT04129294), skeletal muscle dystrophin levels increased significantly after 24 weeks of brogidirsen treatment (mean change from baseline: 13.1%). The current study is examining whether this treatment is safe and well-tolerated in people with DMD amenable to exon 44 skipping, and whether treatment may increase dystrophin levels and help stabilize or improve motor function.

This is a two-part, open-label study that will enroll 20 ambulant boys with DMD amenable to exon 44 skipping. This means that all participants will receive brogidirsen treatment. The study is composed of two parts; the first part will include 6 boys who will be treated with increasing doses of the drug for 12-weeks and the second part will include 14 additional boys who will receive the optimal dose (as determined by the first part) for 24 weeks. Participants will have weekly doctor visits to monitor safety and efficacy during the drug administration.

The drug will be administered intravenously (in the vein). The effects of brogidirsen will be evaluated using a number of tests and procedures at each visit including but not limited to blood draws, urine collections, and physical exams. Motor function tests will be performed every 12 weeks, and muscle biopsies will be performed at the start and end of part 2 for those participants joining for part 2.

To be eligible, individuals must meet the following inclusion criteria:

• Male, 4 to 14 years old
• DMD amenable to exon 44 skipping
• Able to walk independently without assistive devices
• Able to complete TTSTAND without assistance in <7 seconds
• On a stable dose of glucocorticoid for at least 3 months prior to entering the study

Individuals may not be eligible to participate if they are affected by another condition or receiving another treatment that might interfere with the ability to undergo safe testing.

Please visit this link for more detailed listing of inclusion and exclusion criteria.

Travel support will be available for eligible participants.

To learn more about the study or inquire about participation, contact the study coordinator at email: trialinfo@nspharma.com.