Isis has completed a Phase 1 study evaluating the safety of ISIS-DMPKRx in healthy volunteers. In this study, ISIS-DMPKRx was well tolerated at all dose levels tested with no safety or tolerability concerns. The compound was delivered by a subcutaneous injection. All study participants completed the study.

A Phase I/IIa study to evaluate the safety and dose-range finding of ISIS-DMPKRx in patients with DM1 is being planned for the end of the year 2014. Details on this study, including eligibility criteria, will be posted on this resource page and on www.clinicaltrials.gov as soon as they will become available, which is anticipated to be late this year.