NS Pharma Presents 5-Year Data for Duchenne Therapy

PARAMUS, N.J., Oct. 1, 2026 /CourierPR/ -- NS Pharma, Inc., a biopharmaceutical company based in Paramus, New Jersey, announced the presentation of five-year clinical trial data for its investigational therapy, brogidirsen (NS-089/NCNP-02), at the 31st annual International Congress of the World Muscle Society, held in Hiroshima, Japan, from September 29 to October 3, 2026.

Brogidirsen is an antisense oligonucleotide co-discovered by Nippon Shinyaku Co., Ltd., and the National Center of Neurology and Psychiatry (NCNP) in Japan. It is being developed as a potential treatment for Duchenne muscular dystrophy (DMD) patients with dystrophin gene mutations that are amenable to exon 44 skipping. The trial evaluated the efficacy and safety of brogidirsen in six participants who received weekly intravenous (IV) dosing.

The presented data showed that brogidirsen maintained motor function in participants who remained ambulant during the course of the study. Notably, upper limb function was maintained in all participants, including those who became non-ambulant. The safety profile of the therapy was also favorable, with no serious or severe adverse events reported after five years of administration. There were no discontinuations due to adverse events.

Comparing the observed functional outcomes to the natural history of DMD, the data suggested that brogidirsen could slow disease progression in patients who are amenable to exon 44 skipping. The extension trial is ongoing to further investigate the long-term efficacy and safety of the therapy.

Duchenne muscular dystrophy (DMD) is a form of muscular dystrophy that primarily affects males, causing progressive weakness and loss of skeletal, cardiac, and respiratory muscles. Early signs of DMD may include delayed ability to sit, stand, or walk. By adolescence, patients with DMD often require the use of a wheelchair. Cardiac and respiratory muscle problems typically begin in the teenage years and can lead to serious, life-threatening complications.

The National Center of Neurology and Psychiatry (NCNP) conducted the initial investigator-initiated clinical trial, while Nippon Shinyaku and NS Pharma extended the study. The trial involved six participants who received weekly IV dosing. The data presented at the conference highlighted the maintenance of motor function and the acceptable safety profile of brogidirsen over a five-year period. These findings are significant as they provide further evidence of the potential of brogidirsen to slow disease progression in DMD patients.