Isis Pharmaceuticals, Inc. (NASDAQ: ISIS) has initiated a Phase I/II trial for ISIS-DMPKrx in patients affected by myotonic dystrophy type 1 (DM1).
This study is designed to evaluate the safety and tolerability of multiple doses of an investigational compound, ISIS-DMPKrx, in adult patients with DM1. ISIS-DMPKrx is designed to reduce the production of toxic dystrophia myotonic-protein kinase (DMPK) RNA in cells, including muscle cells, for the treatment of DM1.
[Update: Results from the Phase I/II Trial study were announced on January 5, 2017: Ionis Pharmaceuticals Reports DMPKRx Phase 1/2 Trial Results. Ionis Pharmaceuticals and Biogen also released a letter to the DM community in January 2017.]