Today, French pharmaceutical company Pharnext SA announced positive topline results from its pivotal Phase 3 clinical trial (PLEO-CMT) evaluating two doses of PXT3003 compared to placebo during 15 months for the treatment of Charcot-Marie-Tooth disease type 1A (CMT1A).

PLEO-CMT was a pivotal, 15-month, double-blind Phase 3 study that assessed the efficacy and safety of PXT3003 compared to placebo for the treatment of patients with mild to moderate CMT1A. The study evaluated two doses of PXT3003 in a randomized group of 323 patients age 16 to 65 years old. The primary endpoint was the Overall Neuropathy Limitation Scale (ONLS) measuring patient disability. Pharnext reports that a reduction of 0.3 point on this scale was determined to be meaningful according to previously described methodology.

Compared with placebo, a mean reduction of 0.4-point ONLS (95% CI [0.1,0.6], p=0.008) was observed in the trial group receiving the highest dose of PXT3003.

Additionally, PXT3003 was safe, well tolerated and showed a similar safety profile as seen in the Phase 2 study. In this study, PXT3003 provided first evidence of a meaningful improvement of CMT1A effects. Based on these results, Pharnext intends to file for market approval in the U.S. and Europe. For more information about this and ongoing PXT3003 trials, read Pharnext’s full press release here.