NS Pharma, Inc., a subsidiary of Nippon Shinyaku Co., Ltd., has released preliminary findings from its global Phase 3 clinical trial of NS-065/NCNP-01, commonly known as viltolarsen.

Viltolarsen, marketed as VILTEPSO®, gained approval from the United States Food and Drug Administration (FDA) in 2020 for treating Duchenne muscular dystrophy (Duchenne) in patients with a confirmed mutation of the dystrophin gene amenable to exon 53 skipping. The FDA granted accelerated approval based on increased dystrophin production observed in skeletal muscle following treatment. However, continued approval in the US may require confirmation of clinical benefit in a subsequent trial.

The RACER53 Study involved 77 ambulatory boys with Duchenne in a randomized, double-blind, placebo-controlled format, assessing the efficacy and safety of an 80 mg/kg once weekly dose of viltolarsen over 48 weeks. The primary endpoint, Time to Stand from Supine evaluated as velocity (rise/sec), showed a trend of increased velocity in the viltolarsen group compared to baseline. However, there was no statistically significant difference between the viltolarsen and placebo groups.

Initial safety data indicated that adverse events associated with viltolarsen were generally mild or moderate, with no treatment discontinuations due to adverse events during the study.

"We are currently conducting detailed analyses to better understand the results, considering factors such as age, treatment duration, and concomitant medications including glucocorticoid therapy," stated NS Pharma President Tsugio Tanaka, MSc. "Based on previous clinical findings, we remain optimistic about viltolarsen's potential as a beneficial treatment for eligible Duchenne patients."

Previous studies, including a Phase 2 long-term extension study, have shown significant improvements in Time to Stand in Duchenne patients treated with viltolarsen compared to historical controls. Adverse events in these studies were predominantly mild or moderate, with no treatment discontinuations due to adverse events.

NS Pharma intends to conduct further analyses, including post-hoc assessments, and collaborate closely with regulatory authorities to determine the next steps in the best interest of patients. Additional updates on analyses and discussions with regulatory bodies will be provided in due course.

VILTEPSO® (viltolarsen) Injection received Priority Review, Rare Pediatric Disease, Orphan Drug, and Fast Track Designations prior to its US approval in August 2020. It was also approved in Japan in March 2020 for Duchenne patients amenable to exon 53 skipping therapy.

VILTEPSO is indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients with a confirmed mutation of the DMD gene amenable to exon 53 skipping, granted accelerated approval based on increased dystrophin production. Continued approval may require verification of clinical benefit in further trials.

Important Safety Information highlights the need for kidney function monitoring due to potential kidney toxicity observed in animal studies, though not in clinical trials. Common adverse reactions include upper respiratory tract infection, injection site reaction, cough, and pyrexia.

Duchenne muscular dystrophy (Duchenne) is a progressive condition predominantly affecting males, causing weakness and loss of skeletal, cardiac, and respiratory muscles. Early signs include delayed motor milestones, leading to mobility issues and, eventually, cardiac and respiratory complications. More information about Duchenne can be found at wespeakduchenne.com.

 

Source: prnewswire.com

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