Report from the 2018 New Direction in Biology and Disease of Skeletal Muscle Conference

The third day of the conference focused on stem cells, new small molecule screens, poster sessions and clinical trial updates.

• Hirofumi Komaki (National Center of Neurology and Psychiatry, Japan) presented results of a Japanese Phase I/II dose-finding clinical study with NS-065, an exon-53 skipping drug (NS Pharma) for the treatment of Duchenne. The drug was well tolerated, and exon-53 skipping was detected in all patients who participated in the study.
• Paula Clemens (University of Pittsburgh) provided an early update on the North American Phase 2 study with NS-065. The drug was again well tolerated with no serious adverse advents noted. Restoration of dystrophin expression and was seen in patient’s muscle samples following 20-24 weeks of weekly infusions. Analysis of the clinical endpoints are expected to be reported later this year.
• Francesco Muntoni (University College London, UK) presented an update for Golodirsen, an exon-53 skipping drug (Sarepta Therapeutics) in 6 to 15-year-old ambulant Duchenne patients. Treatment was associated with an increase in dystrophin expression in all patients who participated in the study at the 48-week timepoint, and the new dystrophin demonstrated the correct sarcolemmal membrane localization. The trial continues, and an update on the clinical data will be performed after 144-weeks of dosing.
• Jill Rafael-Fortney (Ohio State University) reported on their recent non-clinical mouse study with the non-steroidal mineralocorticoid agonist Finerenone (BAY-94-8862, finerenone is currently in clinical development for the treatment of diabetes and other conditions). The mdx mouse study showed that treatment with finerenone was associated with improvements in grip strength, reduction in cardiac strain compared to untreated mice. This work may provide the foundation for a future clinical study in Duchenne patients.