baclofen/naltrexone/sorbitol (PXT3003) / GeneNet, Pharnext - LARVOL DELTA

A phase III clinical trial of PXT3003 in Chinese patients with Charcot-Marie-Tooth disease type 1A. (PubMed, iScience) - "Significant improvements were observed in the ONLS leg subscore and ankle-dorsiflexion strength. These findings support PXT3003 as a promising therapeutic option for alleviating limb symptoms in patients with CMT1A."

Journal • P3 data • Genetic Disorders • Pain

The current status of Charcot-Marie-Tooth disease type 1 A treatment. (PubMed, Acta Neurol Belg) - "Notably, PXT3003 has shown phase III clinical efficacy in relieving symptoms and reducing neuropathy, and is expected to be the earliest CMT1A-targeted drug on the market...In addition, the rapid development of induced pluripotent stem cell (iPSC) technology has paved the way for stem cell therapies, which may be a promising therapeutic approach. This article reviews the existing literature on therapeutic strategies for CMT1A and aims to provide a valuable reference for the clinical treatment of CMT1A."

Journal • Review • Gene Therapies • Genetic Disorders • Muscular Atrophy • Pain

A Study to Assess the Efficacy and Safety of PXT3003 in Charcot-Marie-Tooth Type 1A (clinicaltrials.gov) - P3 | N=176 | Completed | Sponsor: Tasly GeneNet Pharmaceuticals Co., Ltd | Recruiting ➔ Completed

Trial completion • Genetic Disorders

PREMIER: Phase III Trial Assessing the Efficacy and Safety of PXT3003 in CMT1A Patients (clinicaltrials.gov) - P3 | N=350 | Active, not recruiting | Sponsor: Pharnext S.C.A. | Trial primary completion date: Oct 2023 ➔ Apr 2024

Trial primary completion date • Genetic Disorders

The Current State of Charcot-Marie-Tooth Disease Treatment. (PubMed, Genes (Basel)) - "Compounds such as PXT3003, which are being clinically and preclinically investigated, and a broad array of therapeutic agents and their corresponding mechanisms are discussed. Furthermore, the progress in established gene therapy techniques, including gene replacement via viral vectors, exon skipping using antisense oligonucleotides, splicing modification, and gene knockdown, are appraised. Each of these gene therapies has the potential for substantial advancements in future research."

Journal • Review • Gene Therapies • Genetic Disorders • KIF1B

Gene therapy and other novel treatment approaches for Charcot-Marie-Tooth disease. (PubMed, Neuromuscul Disord) - "PXT3003 is the compound in the most advanced phase for CMT1A, as a second phase-III trial is ongoing...Other potential therapeutic strategies include targeting macrophages, lipid metabolism, and Nav1.8 sodium channel in demyelinating CMT and the P2×7 receptor, which regulates calcium influx into Schwann cells, in CMT1A. Further approaches are aimed at correcting metabolic abnormalities, including the accumulation of sorbitol caused by biallelic mutations in the sorbitol dehydrogenase (SORD) gene and of neurotoxic glycosphingolipids in HSN1."

Gene therapy • Journal • Review • Gene Therapies • Genetic Disorders • Metabolic Disorders • NTF3

Gene therapy and other novel treatment approaches for CMT (WMS 2022) - "PXT3003 is the compound in the most advanced phase for CMT1A, as a second phase-III trial is ongoing...Other potential therapeutic strategies include targeting macrophages, lipid metabolism, and Nav1.8 sodium channel in demyelinating CMT and the P2X7 receptor, which regulates calcium influx into Schwann cells, in CMT1A. Further approaches are aimed at correcting metabolic abnormalities, including the accumulation of sorbitol caused by biallelic mutations in the sorbitol dehydrogenase (SORD) gene and of neurotoxic glycosphingolipids in HSN1."

Gene Therapies • Metabolic Disorders • NTF3

PREMIER: Phase III Trial Assessing the Efficacy and Safety of PXT3003 in CMT1A Patients (clinicaltrials.gov) - P3 | N=350 | Active, not recruiting | Sponsor: Pharnext SA | Recruiting ➔ Active, not recruiting

Enrollment closed • Genetic Disorders