Nippon Shinyaku announced efficacy and safety data from a 3.5-year application based on an open-label extension study that also included the initiation of a clinical trial for brogidirsen (NS-089/NCNP-02).
These data were presented as a poster by the National Center of Neurology and Psychiatry (NCNP, Kodaira City, Tokyo; President, Kazuyuki Nakagome) at the 30th annual International Congress of the World Muscle Society held in Austria from October 7 to 11, 2025.
What is Brogidirsen?
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．
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 in 6 patients who received weekly intravenous administration of brogidirsen.
Read More: Upcoming Exon 44 Skipping Therapies
Safety Results
The results showed trends of high exon skipping efficiency and dystrophin expression level in biopsied muscles at week 25/26 and week 99/100. Furthermore, participants who remain ambulant after long-term brogidirsen administration maintained their motor function ability in assessment including the North Star Ambulatory Assessment. Safety evaluation results showed no serious or severe adverse events related to brogidirsen, or adverse events related to infusion-related reactions such as anaphylaxis, or discontinuation observed due to adverse events.
The results suggest the potential of brogidirsen to slow the disease progression in patients with DMD who are amenable to exon 44 skipping.
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 its subsidiary NS Pharma, Inc.
Learn More: Mutations and Deletions Amenable to Exon 44 Skipping Therapies
