If approved, this will be the first therapeutic to treat any type of CMT.
The PREMIER trial is an international, randomized, doubleblind, two-arm placebo-controlled, pivotal Phase III study, where the primary objective is to evaluate the efficacy and safety of PXT3003 versus placebo in mild-to-moderate CMT1A patients, over a 15-month period. The dose of PXT3003 tested in the PREMIER trial corresponds to the high dose (‘HD’) tested in the prior Phase III clinical study, the PLEO-CMT trial, and the ongoing open-label extension Phase III study, the PLEOCMT-FU trial. As agreed with regulatory agencies, the primary efficacy endpoint will be the Overall Neuropathy Limitations Scale (‘ONLS’) which measures functional motor disability.
Allison Moore, founder & CEO of HNF congratulates Pharnext on reaching this major milestone and continues to applaud Pharnext for their innovation and dedication to supporting patient focused research with their CMT&Me app and the importance of documenting the impact CMT has on quality of life.