NS Pharma, a subsidiary of Japanese pharmaceutical company Nippon Shinyaku, has announced preliminary findings from the global Phase III RACER53 study of viltolarsen (NS-065 / NCNP-01) for Duchenne muscular dystrophy (DMD) treatment.

The randomised, placebo-controlled study enrolled 77 male patients with DMD who were able to walk.

It aimed to assess the safety and efficacy of an 80mg/kg weekly dose of viltolarsen compared to a placebo over 48 weeks.

The primary endpoint, ‘Time to Stand from Supine’, measured as velocity (rise/sec), indicated a trend of increased velocity in the viltolarsen group from baseline after 48 weeks.

Similar trends were also observed in the placebo group, resulting in no statistically significant difference.

Preliminary safety data from the trial showed that all adverse events were mild or moderate without any discontinuations observed due to treatment-emergent adverse events.

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A previous open-label Phase II long-term extension study reported significant improvements in ‘Time to Stand’ at week 205 in subjects receiving viltolarsen versus the control group.

A rise in dystrophin production in skeletal muscle following viltolarsen treatment was reported, leading to approval from the US Food and Drug Administration in 2020 under the accelerated approval pathway.

Marketed as Viltepso, the product is indicated to treat DMD in patients with confirmed dystrophin gene mutation amenable to exon 53 skipping.

NS Pharma is conducting further detailed data analyses and plans to engage with regulatory authorities to determine future actions.

The company will provide updates on additional analyses and regulatory discussions at a later point.

NS Pharma president Tsugio Tanaka said: “We are currently conducting further detailed data analyses and identifying factors that may have influenced the results.

“Considering the results of prior clinical studies, we have confidence that viltolarsen can be a beneficial treatment for amenable patients with Duchenne.”