, /PRNewswire/ -- NS Pharma, Inc. (NS Pharma), a subsidiary of Nippon Shinyaku Co., Ltd.
, announced today that it has received preliminary analysis results from the global Phase 3 clinical trial (RACER53 study, NCT04060199) of NS-065/NCNP-01 (generic name: viltolarsen). Viltolarsen was approved by (US) Food and Drug Administration (FDA) in 2020 under the brand name VILTEPSO – for the treatment of Duchenne muscular dystrophy (Duchenne) in patients who have a confirmed mutation of the dystrophin gene that is amenable to exon 53 skipping – under the FDA accelerated approval pathway based on an increase in dystrophin production in skeletal muscle observed in treated patients. In the US, continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial.
The RACER53 Study is a randomized, double-blind, placebo-controlled, comparative study of 77 ambulatory boys with Duchenne. The study evaluated the efficacy and safety of an 80 mg/kg once weekly dosing of the treatment – versus placebo – for 48 weeks and was intended to serve as a confirmatory study. The primary endpoint of the study was Time to Stand from Supine evaluated as velocity (rise/sec).
The viltolarsen group showed a trend of increased velocity from baseline after treatment for 48 weeks. However, the placebo group also showed a trend of increased velocity, and there was no statistically significant difference between the viltolarsen group and the.