Pharnext Announces On-Schedule Completion of Patient Enrollment in its Pivotal Phase III Trial of PXT3003, the PREMIER Trial, for the Treatment of Charcot-Marie-Tooth Disease Type 1A (CMT1A)

Pharnext, an advanced late-clinical stage biopharmaceutical company and one of HNF Therapeutic Research In Accelerated Discovery (TRIAD) partners, has just announced its PREMIER trial for CMT1A has reached and has exceeded its enrollment goal of 350 randomized patients at 52 centers across the US, Canada, Europe and Israel for their novel therapeutic – PXT3003 – in Phase III clinical trial to treat CMT1A, the most common type of CMT. The top-line data results are expected to be announced in Q4 2023.

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.