The results of an investigator-initiated clinical trial of NS-089/NCNP-02 is published in Cell Reports Medicine

PARAMUS, NJ: January 13, 2025 – NS Pharma, Inc. (NS Pharma), a subsidiary of Nippon Shinyaku Co., Ltd. (Nippon Shinyaku), announced that the National Center of Neurology and Psychiatry (NCNP) has published in the journal Cell Reports Medicine the results of an investigator-initiated clinical trial (first in human trial) of NS-089/NCNP-02 (brogidirsen), which is being developed by Nippon Shinyaku for the treatment of Duchenne muscular dystrophy (Duchenne). A global Phase II study of NS-089/NCNP-02 is being conducted by Nippon Shinyaku and NS Pharma. Please check the press release from NCNP for a summary of the paper. Additionally, the paper is available under open access here.

NS-089/NCNP-02 is an antisense oligonucleotide co-discovered by Nippon Shinyaku and NCNP and is expected to be a therapeutic drug for Duchenne patients who have dystrophin gene mutations amenable to exon 44 skipping.

NS Pharma has been actively working to develop agents for the treatment of intractable and rare diseases, with a goal of launching treatments for patients with Duchenne as soon as possible.

About Duchenne Muscular Dystrophy (Duchenne)

Duchenne is a progressive form of muscular dystrophy that occurs primarily in males. It causes progressive weakness and loss of skeletal, cardiac, and respiratory muscles. Early signs of Duchenne may include delayed ability to sit, stand or walk. There is a progressive loss of mobility, and by adolescence, patients with Duchenne may require the use of a wheelchair. Cardiac and respiratory muscle problems begin in the teenage years and lead to serious, life-threatening complications. For more information about Duchenne, please visit wespeakduchenne.com.

About NS Pharma, Inc.

NS Pharma, Inc., is a wholly owned subsidiary of Nippon Shinyaku Co., Ltd. NS Pharma is a registered trademark of the Nippon Shinyaku group of companies. For more information, please visit nspharma.com.

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