NS Pharma has developed first-of-its-kind NS089/NCNP-02, an investigational candidate for individuals diagnosed with Duchenne muscular dystrophy (DMD) who could potentially benefit from exon 44 skipping therapy.
NS089/NCNP-02 is an antisense nucleotide, discovered through collaborative research between Nippon Shinyaku, and the National Centre of Neurology and Psychiatry.
The therapy aims to address the specific genetic mutation associated with Duchenne muscular dystrophy by skipping exon 44 during the production of dystrophin, a protein critical for muscle function.
NS089/NCNP-02 has the potential to restore the reading frame of the dystrophin gene, thereby potentially improving the production and function of dystrophin in patients with the relevant genetic mutation.
The clinical development of NS089/NCNP-02 involves planned Phase 2 studies, one to be conducted by NS Pharma in the United States and another to be conducted by Nippon Shinyaku in Japan. These studies are designed to assess the safety and efficacy of the investigational therapy in patients with Duchenne muscular dystrophy.