NS Pharma to Provide Update on its Investigational Therapy Viltolarsen in Webinar

Joana Carvalho, PhD avatar

by Joana Carvalho, PhD |

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NS Pharma will share future plans for viltolarsen, its investigational exon-skipping therapy for Duchenne muscular dystrophy (DMD) in a webinar hosted by Parent Project Muscular Dystrophy (PPMD) on Wednesday.

The company will discuss viltolarsen’s mechanism of action and its current regulatory status during the one-hour webinar, which will be held Nov. 20 at 1 p.m. EST. It will also provide updates on ongoing clinical trials evaluating the treatment’s safety and efficacy. Please click here to register.

The webinar will be moderated by Abby Bronson, PPMD’s senior vice president of research strategy. Presenters will be Angela Melia, associate director of research and development at NS Pharma and Paula R. Clemens, MD, an investigator in the clinical trials.

DMD is caused by mutations in the DMD gene. This leads to no or very little production of a protein called dystrophin, which results in subsequent cell death and muscle weakness. In some cases, these mutations result in the loss of entire exons — the sequence of a gene that provides instructions to make proteins — leading to a dysfunctional form of dystrophin.

Exon-skipping therapies work by excluding lost or damaged exons, using specific molecules called antisense oligonucleotides. This approach skips over the faulty exon to produce a shorter but functional protein.

Viltolarsen (NS-065/NCNP-01) excludes exon 53, meaning that only patients carrying specific defects in this exon will be amenable to treatment with this investigational therapy.

NS Pharma and Nippon Shinyaku, NS Pharma’s parent company, are conducting a double-blind Phase 3 trial called RACER53 (NCT04060199) to confirm the efficacy and safety of viltolarsen seen in previous studies.

RACER53 is recruiting patients in the U.S. and Japan. A total of 74 boys, 4 to 7 years old, with DMD are expected to join, all able to walk without assistance. They will be randomly assigned to receive either weekly intravenous infusions of viltolarsen (80 mg/kg) or a placebo, for up to 48 weeks.

The study will evaluate the effects of treatment on the boys’ ability to stand, run, walk, and climb steps, as well as their ability to perform movements such as extending and flexing their elbows and knees.

Investigators will also collect blood samples to study how viltolarsen is absorbed, distributed, metabolized, and eliminated by the body.

RACER53 is expected to conclude by the end of 2024. More information about clinical sites, study design, and inclusion criteria will be discussed by NS Pharma during the webinar.

In a previous Phase 2 trial (NCT02740972) carried out in the U.S. and Canada, viltolarsen restored the production of dystrophin in the muscles of boys with DMD carrying a mutated exon 53 after 20 to 24 weeks of treatment.

The treatment was well-tolerated, with boys experiencing mild or moderate side effects. None of the boys participating in the study were forced to stop therapy due to side effects.

Meanwhile, Nippon Shinyaku has completed a Phase 1/2 trial assessing viltolarsen in Japan.

The U.S. Food and Drug Administration has granted fast track, orphan drug, and rare pediatric disease designations to viltolarsen and is currently reviewing a new drug application submitted by Nippon Shinyaku requesting its approval in the U.S.

In Japan, viltolarsen has also received SAKIGAKE (intended for early introduction of innovative medicines or medical devices) and orphan drug designations. The Japanese Ministry of Health, Labour and Welfare is currently reviewing an application requesting the treatment’s approval.