The National Center of Neurology and Psychiatry is announced the publication of a research paper in Cell Reports Medicine detailing the results of an investigator-initiated trial for NS-089/NCNP-02, known as “brogidirsen.” This innovative treatment for Duchenne muscular dystrophy (DMD), jointly developed with Nippon Shinyaku Co., Ltd., represents a significant advancement in exon 44 skipping therapy.
Key Findings:
- Brogidirsen shows dose-dependent dystrophin restoration in DMD patients’ trials.
- High-dose brogidirsen reached 24.47% of normal dystrophin level in the DMD cohort.
- The trial demonstrated therapeutic benefits with stable or improved motor function and a favourable safety profile with no severe adverse events.
- Serum biomarkers for DMD were identified, including PADI2, TTN, MYOM2, and MYLPF.
- Brogidirsen showed high efficiency in DMD urine-derived cells, supporting human trials.
What is Brogidirsen?
Brogidirsen (NS-089/NCNP-02) is a nucleic acid drug co-discovered by Nippon Shinyaku and NCNP, and is expected to be a therapeutic drug for DMD patients with dystrophin gene mutations amenable to exon 44 skipping.
