NS-065 / NCNP-01 is an exon-skipping nucleic acid drug created through joint research between National Center for Neurological and Medical Research (NCNP) and Nippon Shinyaku. Itt is targeted as a remedy for patients with DMD who have gene mutations responding to exons 53 skipping of the dystrophin gene. In this study, NS – 065 / NCNP – 01 was administered to humans for the first time and 10 patients with DMD were treated at a low dose (1.25 mg NS-065 / NCNP-01 was administered once a week for 12 weeks, 3 cases were assigned to the middle dose (5 mg group) and 4 cases to the high dose (same, 20 mg) group. The test was carried out as an open-label, non-contrasted study to be administered intravenously. The recruitment of patients for this study was done by utilizing Neuromuscular Disease Patient Registration System (Remudy). The main endpoint was set as safety, the secondary endpoint was set as efficacy such as pharmacokinetics and dystrophin expression. The main inclusion criteria were to have mutations that would apply exon 53 skip to the dystrophin gene, to be unable to walk as a rule under the age of 5 years of age. Regarding dystrophin expression, muscle biopsy was performed before and after the administration period and evaluated. As a result, no serious adverse events occurred with regard to safety, and there were no discontinuation cases. Based on the above results, Nippon Shinyaku started Phase I / II clinical trials in Japan in January 2016 and Phase II clinical trials in the United States since March of that same year, DMD is a severe disease in which muscle weakness of the whole body progresses and organ dysfunction also merges, and NS-065 / NCNP-01 has an innovative mechanism of action based on exon skipping action etc. This product has been designated as a pioneer review designation system in Japan.

Nikkei Biotech news release, April 19, 2014