bobby, Author at NSPharma

PARAMUS, NJ: March 9, 2026 – NS Pharma, Inc. (NS Pharma, New Jersey, USA; President, Yukiteru Sugiyama ), a biopharmaceutical leader in rare diseases and subsidiary of Nippon Shinyaku Co., Ltd. (Nippon Shinyaku), announced today that the National Center of Neurology and Psychiatry (NCNP, Kodaira City; President, Kazuyuki Nakagome) presented 4.5-year safety and efficacy data based on the open-label extension study, including an investigator-initiated clinical trial of brogidirsen (NS-089/NCNP-02) for the treatment of Duchenne muscular dystrophy (DMD) at the 2026 MDA Clinical & Scientific Conference from March 8-11, 2026.

Brogidirsen is an antisense oligonucleotide co-discovered by Nippon Shinyaku and NCNP as an investigational therapy for DMD patients with dystrophin gene mutations that are amenable to exon 44 skipping.

“We are thrilled to see the data continue to demonstrate long-term efficacy in DMD patients amenable to exon 44 skipping and while maintaining patient safety,” said NS Pharma President, Yukiteru Sugiyama, Ph.D. “We are committed to expanding available treatment options to the DMD community and we look forward to realizing that ambition.”

The presented data are based on the first-in-human Phase 1/2 open-label investigator-initiated clinical trial conducted by NCNP and the subsequent Phase 2 open-label extension trial conducted by Nippon Shinyaku. These studies evaluate the safety and efficacy of brogidirsen in six participants who received weekly IV dosing of brogidirsen.

Findings include:

Maintenance of motor function – From the initiation of weekly administration, participants maintained motor function in assessments such as North Star Ambulatory Assessment (NSAA). All participants, including those who transitioned to full-time wheelchair use due to disease progression, maintained their total scores in Performance of Upper Limb (PUL 2.0).
Acceptable safety profile – After 4.5-year of receiving brogidirsen, no serious or severe adverse events were reported related to long-term brogidirsen administration; anaphylaxis was not observed and no patients discontinued administration.

These findings support the potential of brogidirsen to modify the progression of DMD. This extension trial is ongoing to investigate the safety and efficacy of longer-term administration.

A second, ongoing global Phase II study of brogidirsen is being conducted by Nippon Shinyaku and NS Pharma to further evaluate the safety and efficacy of brogidirsen. Learn more at ClinicalTrials.gov.

About Duchenne Muscular Dystrophy (DMD)
Duchenne is a form of muscular dystrophy that occurs primarily in males. It causes progressive weakness and loss of skeletal, cardiac, and respiratory muscles. Early signs of DMD 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, 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 Co., Ltd. For more information, please visit nspharma.com.

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PARAMUS, NJ: October 14, 2025 – NS Pharma, Inc. (NS Pharma), a biopharmaceutical leader in rare disease and subsidiary of Nippon Shinyaku Co., Ltd. (Nippon Shinyaku), announced today that the National Center of Neurology and Psychiatry (NCNP, Kodaira City; President, Kazuyuki Nakagome) presented 3.5-year efficacy and safety data from the open-label extension of an investigator-initiated clinical trial of brogidirsen (NS-089/NCNP-02) for the treatment of Duchenne muscular dystrophy (DMD)at the 30th annual International Congress of the World Muscle Society from October 7 to 11, 2025.

“These long-term motor function data suggest the potential for brogidirsen to slow disease progression in DMD patients amenable to exon 44 skipping and support the robust increases in dystrophin production seen earlier in the trial,” said NS Pharma President, Yukiteru Sugiyama, Ph.D. “We are proud to offer hope to families and continue to demonstrate our commitment to the DMD community

The presented data are based on the investigator-initiated clinical trial conducted by NCNP and its extension study conducted by Nippon Shinyaku. These studies evaluated the efficacy and safety of brogidirsen in 6 participants who received weekly IV dosing of brogidirsen.

Findings include:
• Consistent functional benefits – High exon 44 skipping efficiency and dystrophin expression levels were observed in biopsied muscles at week 25/26 and week 99/100.
• Maintenance of motor function – Participants who remain ambulant after long-term brogidirsen administration maintained their motor function in evaluations including North Star Ambulatory Assessment.
• Acceptable safety profile – After 3.5-year of receiving brogidirsen, no serious or severe adverse events, or anaphylaxis related to long-term brogidirsen administration, were reported, and there were no discontinuations.

These findings support the potential of brogidirsen to modify the progression of DMD. This long-term extension trial is ongoing to investigate the efficacy and safety of longer-term administration.

Further, a global Phase II study of brogidirsen is being conducted by Nippon Shinyaku and NS Pharma, Inc. (Headquarters: New Jersey, USA; President: Yukiteru Sugiyama). Learn more at ClinicalTrials.gov.

US Media Contact:
[email protected]

PARAMUS, NJ: September 19, 2025 – NS Pharma, Inc. announced today that the U.S. Food & Drug Administration (FDA) has granted Orphan Drug Designation to NS-051/NCNP-04 which is being developed for the treatment of Duchenne muscular dystrophy (Duchenne) in patients amenable to exon 51 skipping. The FDA issues Orphan Drug Designations to support the development and evaluation of new treatments to prevent, diagnose, or treat a rare disease or condition.

NS-051/NCNP-04 previously received Rare Pediatric Disease Designation from the FDA in January 2025. For more information, see the press release here.

Duchenne is a progressive muscle wasting disease caused by a deficiency of the dystrophin protein. It leads to weakness of skeletal, cardiac, and respiratory muscles. There are many types of genetic mutations that can cause Duchenne, and NS-051/NCNP-04 is being developed to treat patients with confirmed gene mutations amenable to exon 51 skipping therapy.

NS-051/NCNP-04 is an antisense oligonucleotide co-discovered by the National Center of Neurology and Psychiatry (NCNP) and Nippon Shinyaku. NS-051/NCNP-04 promotes the skipping of exon 51 within the dystrophin gene, causing the production of a shortened dystrophin protein containing essential functional portions. This is expected to have the effect of stabilizing or improving muscle function.

About Duchenne Muscular Dystrophy (Duchenne)
Duchenne is a 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.

US Media Contact:
[email protected]