NS Pharma Study Shows Exon 44 Skipping Drug Efficacy

National Center of Neurology and Psychiatry and Nippon Shinyaku announce positive results for a Phase I/II study evaluating the efficacy and safety of their exon 44 skipping drug (NS-089/NCNP-02) 

Results have just been released for the first exon-skipping clinical study in Duchenne patients amenable to exon 44 skipping and demonstrate highly efficient production of dystrophin in a dose-dependent manner. 

NS-089/NCNP-02 (weekly intravenous administration for 24 weeks) increased dystrophin expression to an average of 10.27% of normal at 40 mg/kg and 15.79% of normal at 80 mg/kg, with no discontinuation due to adverse events during the trial. 

Based on these results, Nippon Shinyaku is now preparing for the next phase of the clinical study. More information is available in the press release below.