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Recently, NS Pharma announced the failure of its Phase III clinical trial for viltolarsen (brand name: Viltepso), a candidate drug for Duchenne muscular dystrophy.Duchenne Muscular Dystrophy(DMD) is a fatal rare X-linked degenerative neuromuscular disease. Worldwide, approximately 1 in every 3,500 to 5,000 male newborns is affected by DMD. It is caused by genetic mutations in the body that encode dystrophin, often leading to respiratory and/or cardiac failure, with patients' life expectancy rarely exceeding 40 years.Viltepso is an antisense oligonucleotide, by skipping exon 53. It was approved in Japan in March 2020 for the treatment of DMD patients confirmed to have exon 53 skipping mutations. In August 2020, Viltepso received accelerated approval from the U.S. FDA, becoming the second antisense oligonucleotide (ASO) therapy for the treatment of DMD amenable to exon 53 skipping. The first one was Golodirsen (brand name: Vyondys 53), a drug from Sarepta Therapeutics approved by the FDA in December 2019.®)。These two drugs are both ASO therapies, a type of small nucleic acid therapy. They share the same mechanism of action, promoting the production of functional dystrophin by masking (skipping) exon 53 in the dystrophin gene.Viltepso is administered via intravenous infusion once a week at a dose of 80mg/kg. This study recruited a total of 77 DMD patients (ambulatory), primarily evaluating whether Viltepso improves standing time in DMD patients.After 48 weeks of treatment, patients receiving Viltepso did not show a statistically significant difference compared to the placebo.。In terms of safety, the adverse reactions of Viltepso were mild or moderate, and no patients withdrew from the study due to side effects.Despite failing to pass the Phase III study,Tsugio Tanaka, President of NS PharmaStated: "The company still believes that Viltepso can bring benefits to patients with Duchenne muscular dystrophy. NS Pharma will collaborate with the FDA and other regulatory agencies to determine the next steps based on the analysis results."Coincidentally, in early May, Pfizer suspended its DMD gene therapy after the death of one pediatric patient.Fordadistrogene MovaparvovecPhase 3 clinical trial.In recent years, Duchenne muscular dystrophy has been a highly productive direction for the development of small nucleic acid drugs. Currently, four ASO therapies have been approved. In addition to Viltepso, the other three are small nucleic acid therapies for DMD developed by Sarepta Therapeutics, namely Eteplirsen, which was approved for marketing in 2016, Golodirsen, which was approved for marketing in 2019, and Casimersen, which was approved for marketing in 2021. All of them received accelerated approval from the FDA. In 2023,Eteplirsen sales reached $540 million, a year-on-year increase of 6%; Golodirsen sales amounted to $130 million, a year-on-year increase of 11%; Casimersen sales hit $274 million, a year-on-year increase of 28%.。
About NS Pharma
NS Pharma is a wholly-owned subsidiary of Nippon Shinyaku, focusing on the research and development of drugs for treating DMD. Currently, it has 8 pipelines, 7 of which are for treating DMD. The one with the fastest R&D progress is viltolarsen, which has been approved for marketing in Japan and conditionally approved in the United States for DMD patients who can skip exon 53.
R&D Pipeline
References:
https://www.biospace.com/article/ns-pharma-s-dmd-drug-viltespo-fails-confirmatory-phase-iii-trial/
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