First Patient Dosed in Phase 2 Clinical Study of NS-065/NCNP-01 for the Treatment of Duchenne Muscular Dystrophy Amenable to Exon 53 Skipping
Nippon Shinyaku Co., Ltd. (Nippon Shinyaku; Headquarters, Kyoto City; President, Shigenobu Maekawa) announced today that the first patient dosed in the phase 2 clinical trial of NS-065/NCNP-01 in patients with Duchenne muscular dystrophy (DMD) amenable to exon 53 skipping. NS-065/NCNP-01 is the first antisense oligonucleotide discovered in Japan, which was granted Fast Track and Orphan Drug Designation from the FDA.
DMD is an inherited muscle disorder with the highest incidence that is limited to male children. It causes a severe loss of muscle strength and function due to a deficiency of normal dystrophin, a protein involved in constructing the framework of muscle cells. This deficiency arises from a 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/NCNP-01 is a morpholino antisense oligonucleotide, which is expected to improve muscle function by skipping a part of genetic information of the dystrophin gene. A phase 2 clinical study in the US and a phase 1/2 study in Japan began recruitment in December 2016 and July 2016, respectively. NS-065/NCNP-01 was registered as "SAKIGAKE designation(Japanese version of Breakthrough Therapy Designation) " of the Ministry of Health, Labour and Welfare in Japan in October 2015. The clinical study in the US is being conducted by NS Pharma, Inc. (Headquarters, New Jersey, US; President, Masato Matsuoka) which is a US subsidiary of Nippon Shinyaku.
The phase 2 clinical study in the US will enroll approximately 16 patients aged 4 to 9 years. Two dose level cohorts (40mg/kg or 80mg/kg) will be studied. Once confirmed eligible, each enrolled participant will start in the study in a 4-week double-blind period followed by a 20-week open label period. NS-065/NCNP-01 is administered by IV infusion.
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.