Home / Ionis Pharmaceuticals Reports on Phase I & I/II Ionis-DMPKrx Studies

Ionis Pharmaceuticals Reports on Phase I & I/II Ionis-DMPKrx Studies

Dr. Laurence Mignon, Director of Clinical Development at Ionis Pharmaceuticals in Carlsbad, CA, and Dr. Charles Thornton, neurologist and a principal investigator at the University of Rochester, reported on the status of the Phase I and Phase I/II clinical trials for IONIS-DMPKRx, a potential DM therapy, at the annual meeting of the American Academy of Neurology in Vancouver, BC last month. Dr. Mignon shared the results of the presentations with MDF so we could report out to the MDF community.

Dr. Mignon presented a poster focused on the IONIS-DMPKRx Phase I study that was conducted in 2014 as a first-phase Phase I safety and toxicity pharmacokinetic study in healthy volunteers. Dr. Thornton presented a poster focused on the first four cohorts or groups of participants in the Phase I/II study in affected patients. Information from these cohorts, which have completed the study, indicate that IONIS-DMPKRx was well tolerated by participating patients, and the strength outcome measures used in the trial correlate very well between the baseline and screening visits, and between participating sites. Hence the training sessions conducted with physical therapists before the trial began were effective, and the tests being used are reliable.

Ionis Pharmaceuticals expects to present the results of the full Phase I/II trial in early 2017 after the trial concludes.  

Questions about the Ionis Pharmaceuticals clinical trial? Contact Ionis at patients@ionisph.com.





© Myotonic Dystrophy Foundation. All rights reserved.