Phase 2 study of Viltolarsen (Formerly NS-065/NCNP-01) in people with Duchenne amenable to Exon 53 Skipping

Safety, Dose Finding, and Efficacy Studies of Viltolarsen (NS-065/NCNP-01) in Boys with Duchenne

Questions and Answers

What stage is this research?

Study NS-065/NCNP-01-201 is a Phase 2 study which has completed enrollment (16 patients) and dosing at clinical sites in North America (United States and Canada).

All participants elected to enroll into Study NS-065/NCNP-01-202 which is an extension study of NS-065/NCNP-01-201. All participants continue to be dosed.

Submission of a rolling New Drug Application (NDA) for viltolarsen (NS-065/NCNP-01) was initiated for the treatment of Duchenne Muscular Dystrophy (DMD) in patients amenable to exon 53 skipping.

What is viltolarsen (NS-065/NCNP-01)?

Viltolarsen (NS-065/NCNP-01) is an investigational drug designed to skip exon 53 in the dystrophin gene.

What is the goal or purpose of this study?

Study NS-065/NCNP-01-201 was conducted to evaluate the safety and tolerability of a low dose and high dose of viltolarsen (NS-065/NCNP-01) injection delivered as a weekly intravenous infusion in patients with Duchenne who are amenable to exon 53 skipping. Additional objectives of the study were to assess the medication’s effect on induction of dystrophin, muscle function and strength, pharmacokinetics and pharmacodynamics.

Study NS-065/NCNP-01-202 is being conducted to continue evaluating the safety and tolerability of low and high doses of viltolarsen (NS-065/NCNP-01) Injection up to an additional 144 week period or until the product is commercially available (total 168 weeks of treatment in Study NS-065/NCNP-01-201 and 202). Additional objectives of the study are to assess the medication’s effect on muscle function and strength.

Who is funding this study?

The study is sponsored by NS Pharma, Inc.

Who was eligible to participate in this study?

To be in Study 201 boys must have had a confirmation of Duchenne by genetic test with a mutation amenable to skipping exon 53 and be between the ages of 4 and less than 10 years old. They must have been able to walk independently and to complete standard timed tests. Boys must have been on a stable dose of steroid medication for at least 3 months.

Did all boys enrolled in this trial receive treatment with the investigational therapy?

Study NS-065/NCNP-01-201 was a 2-period, randomized, dose finding study for 24 weeks.

Period 1 was a 4-week, blinded, placebo-controlled phase and Period 2 was a 20-week, open-label phase where all participants received the study medication.

Study NS-065/NCNP-01-202 is an open-label, extension study of NS-065/NCNP-01-201 for an additional 144 weeks.

All participants who completed Study 201 have enrolled into Study 202 and all participants continue to receive the investigational therapy, viltolarsen (NS-065/NCNP-01).

How long will this study last?

Study NS-065/NCNP-01-201 lasted 24 weeks.

Study NS-065-NCNP-01-202 will last for up to an additional 144 weeks (total 168-week treatment) or until the product is commercially available.

Why did Study NS-065/NCNP-01-201 include a placebo group of participants?

In Study 201 the initial 4-week placebo arm served as a safety control arm. The safety information collected during the 4-week placebo period will be compared to those participants who received viltolarsen (NS-065/NCNP-01).

Where do the studies take place?

This study was and continues to be conducted at selected participating centers of the Cooperative International Neuromuscular Research Group (CINRG) in the North America. For more information on site locations, please visit Identifiers NCT02740972 and NCT03167255.

Where can I learn more about this study?

You can learn more about this study at (NCT# NCT02740972 and NCT03167255).

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