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NS Pharma: VILTEPSO® (viltolarsen) Injection Long-Term Efficacy and Safety Data

July 6, 2021

N.S. Pharma, Inc. has announced new long-term efficacy and safety data from the open-label extension trial of a Phase 2 study of VILTEPSO® (viltolarsen) injection.

VILTEPSO is an exon-skipping therapy for individuals with Duchenne muscular dystrophy with deletion mutations amenable to exon 53 skipping.

The efficacy and safety results were based on interim analyses at week 109 from the open-label extension trial of a VILTEPSO Phase 2 study. The primary endpoint of Time to Stand showed a statistically significant benefit for multiple time points over two years of treatment with VILTEPSO compared to Duchenne matched historical control group.

“These analyses showed that, after more than two years of treatment with VILTEPSO, patients maintained their motor function based on clinically relevant measurements while the DMD historical controls showed a functional decline,”

Leslie Magnus, MD, Vice President, Medical Affairs

dditional secondary endpoints of motor function, including Time to Run/Walk and 6-Minute Walk Test, demonstrated consistent and statistically significant benefits compared to DMD historical controls. There were no treatment-related serious adverse events, and no patients discontinued treatment.

About VILTEPSO® (viltolarsen) injection

Prior to its approval in the U.S. in August 2020, VILTEPSO was granted Priority Review as well as Rare Pediatric Disease, Orphan Drug and Fast Track Designations. VILTEPSO is not approved in Canada; however, if your child is between the ages of 4-7 and walking, you could qualify for their clinical trial at one of three clinic sites:

  1. Toronto
  2. Calgary
  3. Quebec City

Do you have questions about the clinical trial? Contact [email protected] for more information.

For any further support, please email Defeat Duchenne Canada at [email protected].