Editorial note: This post refers to Pharnext and its experimental CMT1A therapy PXT3003. Pharnext is no longer operating, and PXT3003 is not an approved or available treatment. We have kept this post as part of our historical record.

The Hereditary Neuropathy Foundation (HNF) is a strong advocate for accurate and early diagnosis of Charcot-Marie-Tooth (CMT) disease, recognizing its critical importance for patient care, research, and treatment. For those living with CMT and its progressive muscle weakness in the legs, feet, arms, and hands, there are currently no approved curative or symptomatic medications.

That may be changing.

In December 2016, Pharnext, a French biopharmaceutical company, completed patient enrollment for the international Phase 3 clinical trial of PXT3003 (Pharnext’s lead PLEODRUG) for treatment of CMT1A, the most common form of CMT. The trial’s primary endpoint is the change in the Overall Neuropathy Limitation Score (ONLS), used to determine improvement in patient disability after 12 and 15 months of treatment. Trial results were expected in the second half of 2018.

HNF has partnered with Pharnext to support this clinical trial’s development. We want to thank everyone involved, including the researchers and developers at Pharnext, the clinicians at HNF-designated Centers of Excellence, and the patient-participants who play a critical role.

HNF contributed to this research by providing U.S. clinical site identification and patient recruitment support for this pivotal Phase 3 trial. HNF resources, including the Global Registry for Inherited Neuropathies (GRIN), the Inspire Patient Community, and CMT-Connect local patient empowerment programs, have been instrumental. We are enthusiastic that this joint effort with Pharnext could lead to the first-ever therapy for CMT, and we look forward to continuing our collaboration to address the rarer forms of CMT.

Building a full pipeline of potential therapeutics to serve the diverse needs of the entire CMT community will require many clinical trials. That is why HNF created the Clinical Trial Support Fund, specifically to raise funds for the clinical trials our patients need. Your tax-deductible donation helps ensure HNF can continue to support clinical trials for years to come.