Preliminary results from the PREMIER trial do not confirm the efficacy of PXT 3003 in CMT 1A

PXT 3003 is a drug candidate developed by Pharnext. It has already been the subject of two clinical trials, launched in 2010 and 2015, and of an international phase 3 trial between 2021 and 2023, notably in France. Its preliminary results were announced in a press release in December 2023.

• The ONLS score was reported to have improved in both patients treated with PXT 3003 and those treated with placebo.
• The treatment is well tolerated.

For the time being, these are only preliminary results, and Pharnext says it is continuing to analyse them in greater depth.

 

 Pharnext reports topline results from the pivotal Phase III clinical trial (PREMIER trial) of PXT3003 in Charcot-Marie-Tooth disease type 1A – 11 December 2023 Pharnext press release.

 

Pharnext intends to prepare registration and marketing authorization dossiers for PXT3003, its drug candidate in Charcot-Marie-Tooth disease type 1A – 19 December 2023 Pharnext press release.