Pharnext announced Thursday that it has completed its pivotal Phase III clinical trial in Charcot-Marie-Tooth disease type A (CMT1A) on schedule.

In a press release, the biopharmaceutical company states that the last randomized patient in the trial, which involves its lead drug candidate PXT3003, has now completed his end-of-trial visit.

The trial, initiated in March 2021, aims to evaluate the molecule's efficacy and safety over a 15-month period in patients with mild-to-moderate CMT1A.

A total of 387 patients were included in the trial, exceeding the 350 patients initially defined in the protocol. Of these, 315 received high-dose PXT3003.

Preliminary results of this study are still expected in Q4 2023.

Charcot-Marie-Tooth disease type A, characterized by progressive, hereditary and severe neuropathies, is an orphan disease with a prevalence of one in 5,000 people affected.

Copyright (c) 2023 CercleFinance.com. All rights reserved.