Phase 1/2 Clinical Study of NS-065, a Treatment for Duchenne Muscular Dystrophy, Initiated
Nippon Shinyaku Co., Ltd. (Nippon Shinyaku; Headquarters, Kyoto City; President, Shigenobu Maekawa) has submitted to the Pharmaceuticals and Medical Devices Agency (PMDA) today a notification of a clinical trial plan for a phase 1/2 clinical study of NS-065, the first antisense oligonucleotide discovered in Japan for the treatment of Duchenne Muscular Dystrophy (DMD).
DMD is an inherited muscle disorder with the highest incidence that is limited to male children. It causes a severe loss of muscle power due to a deficiency of normal dystrophin, a protein involved in constructing the framework of muscle cells. This deficiency arises from mutation of the dystrophin gene. Because there is no effective treatment for DMD other than steroids, the development of an effective new treatment is desired.
NS-065 was discovered during a collaboration between Nippon Shinyaku and National Center of Neurology and Psychiatry (NCNP, Kodaira City; President, Teruhiko Higuchi), and it is expected to improve muscle function by skipping a part of genetic information of the dystrophin gene (Exon 53 skipping*).
Nippon Shinyaku has decided to proceed to a Phase 1/2 clinical study on the basis of the favorable results of an investigator-initiated early exploratory clinical trial (from June 2013 to March 2015) conducted by NCNP.
In addition, NS-065 was registered as "pioneer review designation system" of the Ministry of Health, Labour and Welfare on October 27, 2015.
Nippon Shinyaku has been working actively having a sense of mission to develop agents for the treatment of intractable and rare diseases, with a view to launching products for patients as soon as possible.
*: The subjects of the clinical study of NS-065 are patients who can be treated by Exon 53 skipping (patients with deletion mutation of Exon 42-52, 43-52, 45-52, 47-52, 48-52, 49-52, 50-52, or 52 of the dystrophin gene).