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Promising Clinical Trial Results for Brogidirsen in DMD

On October 14, 2025, NS Pharma, Inc., a leader in the biopharmaceutical industry focused on rare diseases and a subsidiary of Nippon Shinyaku Co., Ltd., unveiled significant clinical trial data for brogidirsen (NS-089/NCNP-02) during the 30th annual International Congress of the World Muscle Society. The data presented highlighted the efficacy and safety of brogidirsen over a 3.5-year period, offering hope to many families affected by Duchenne muscular dystrophy (DMD).

Understanding Duchenne Muscular Dystrophy

Duchenne muscular dystrophy is a severe form of muscular dystrophy primarily impacting young males, leading to progressive muscle weakness and loss. Early symptoms include delayed motor milestones, such as the inability to sit or stand independently, which often results in the need for a wheelchair by adolescence. As the disease progresses, vital muscular functions, including cardiac and respiratory muscle strength, diminish, increasing the risk of life-threatening complications.

The Role of Brogidirsen

Brogidirsen is an antisense oligonucleotide designed specifically for DMD patients with mutations amenable to exon 44 skipping. Co-discovered by Nippon Shinyaku and the National Center of Neurology and Psychiatry (NCNP), this investigational therapy aims to facilitate the production of functional dystrophin protein, which is crucial for muscle health.

Dr. Yukiteru Sugiyama, President of NS Pharma, expressed optimism regarding the latest findings, stating, "These long-term motor function data suggest the potential for brogidirsen to slow disease progression in DMD patients amenable to exon 44 skipping and supports the robust increases in dystrophin production seen earlier in the trial."

Key Findings from the Clinical Trial

The clinical data stem from an investigator-initiated trial involving participants receiving weekly intravenous dosing of brogidirsen. The study involved small participant groups, thus emphasizing the importance of these early findings.

Significant Functional Benefits

The results showed a high efficiency for exon 44 skipping as well as elevated dystrophin expression levels observed in muscle biopsies at both the 25th and 99th weeks of treatment.

Maintenance of Motor Function

Participants who remained ambulant throughout the course of the long-term brogidirsen administration were able to preserve their motor function, as measured by assessments like the North Star Ambulatory Assessment.

Acceptable Safety Profile

Importantly, participants did not report any serious or severe adverse effects, nor instances of anaphylaxis associated with the treatment after 3.5 years. There were also no treatment discontinuations, indicating a favorable safety profile for brogidirsen over the trial period.

These promising results solidify the potential for brogidirsen not only to enhance motor function but also to alter the trajectory of DMD. Given the success seen so far, ongoing studies will continue to investigate its efficacy and safety over a longer duration.

Future Directions

Currently, NS Pharma is conducting a global Phase II study involving brogidirsen, as part of its commitment to exploring innovative therapeutics for DMD. The perseverance demonstrated by the DMD community and research organizations remains vital in the pursuit of effective treatments.

For those keen to learn more about DMD or the clinical trials related to brogidirsen, an information hub is maintained at wespeakduchenne.com and clinical trial details can be found at ClinicalTrials.gov.

In a landscape where hope often seems sparse, the findings presented at the World Muscle Society Congress represent a beacon for families grappling with Duchenne muscular dystrophy. The commitment of NS Pharma and its collaborators underscores the importance of continued research to evolve our understanding and treatment of this challenging condition.