Key clinical point: PXT3003 showed a clinically meaningful improvement in CMT1A neuropathy symptoms.
Major finding: Patients taking PXT3003 had a 0.37-point decrease in Overall Neurology Limitations Scale score, compared with placebo (P = .0008).
Study details: The findings are based on the PLEO-CMT study, an international, multicenter, randomized, double-blind, placebo-controlled, phase 3 trial of 235 patients (per-protocol) who received drug PXT3003 or placebo twice daily for 15 months.
Disclosures: Pharnext funded the research. Dr. Thomas is a researcher with Pharnext and Acceleron and has received speaking or advisory board fees from Novartis, Acceleron, Sanofi, and Genentech. The other seven authors are employees of Pharnext.
Thomas F et al.