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FDA Grants Breakthrough Therapy Designation to NS-089/NCNP-02 for Duchenne Muscular Dystrophy Treatment

Saturday, July 29, 2023

NS Pharma, Inc. announced today that the U.S. Food & Drug Administration (FDA) has granted Breakthrough Therapy Designation to NS089/NCNP-02, an investigational candidate developed for patients with Duchenne muscular dystrophy amenable to exon 44 skipping therapy.

The Breakthrough Therapy Designation for NS-089/NCNP-02 is based on promising results from a first-in-human clinical trial conducted in Japan. The FDA grants Breakthrough Therapy Designation to speed up the development and review of medications intended to treat serious or life-threatening diseases. To receive this designation, there must be early clinical evidence suggesting that the drug could bring significant improvements over currently available treatments on clinically meaningful endpoints.

Notably, in June 2023, NS-089/NCNP-02 was also granted Rare Pediatric Disease Designation by the FDA.

NS-089/NCNP-02 is an antisense nucleotide resulting from collaborative research between NS Pharma's parent company, Nippon Shinyaku, and the National Center of Neurology and Psychiatry (located in Kodaira City, with Kazuyuki Nakagome as its President).

The clinical development of NS-089/NCNP-02 includes plans for a Phase 2 study in the United States, which will be conducted by NS Pharma, and another Phase 2 study in Japan, to be conducted by Nippon Shinyaku. Further details about these trials will be provided once they are ready to begin enrolling participants.

Duchenne Muscular Dystrophy is a progressive muscle disorder primarily affecting males. It leads to gradual weakness and loss of skeletal, cardiac, and respiratory muscles. Early signs may include delays in sitting, standing, or walking. As the disease progresses, mobility is further affected, often necessitating wheelchair use during adolescence. Serious and life-threatening complications arise due to cardiac and respiratory muscle problems in the teenage years.

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