NMD Pharma Begins Phase 2 Trial of NMD670 for Charcot-Marie-Tooth Disease Types 1 and 2

NMD Pharma A/S, a biotech company focused on developing new treatments for neuromuscular diseases, has initiated a Phase 2 study of its drug NMD670 for patients with Charcot-Marie-Tooth Disease Types 1 and 2 in the United States and Europe. The first patient was dosed by Dr. Yessar Hussain at the Austin Neuromuscular Center in Austin, Texas, following the FDA's clearance of the investigational new drug application in June 2024.

NMD670 is a pioneering small molecule inhibitor targeting the skeletal muscle-specific chloride ion channel 1 (CIC-1). This drug has been shown to enhance muscle responsiveness to weak signals, thereby improving neuromuscular transmission and restoring muscle function. The current Phase 2a clinical trial, named SYNAPSE-CMT (NCT06482437), is a randomized, double-blind, placebo-controlled study designed to assess the effectiveness, safety, and tolerability of NMD670. The trial involves a 21-day treatment period with an oral dose administered twice daily to 80 adult patients who have genetically confirmed CMT1 or CMT2 subtypes.

The trial will measure changes in several physical performance tests, including the 6-minute walk test (6MWT), the time required to complete a 10-meter walk/run test (10MW/R), and the time needed for the timed-up-and-go test (TUG), among other safety and efficacy endpoints. These evaluations will be conducted across multiple clinical sites in the US and Europe.

Dr. Yessar Hussain from the Austin Neuromuscular Center expressed optimism about the study: “Patients with CMT are very excited about entering into this study. This is the only clinical study with a clinically feasible treatment to address the muscle weakness and fatigue with a pharmaceutical therapy at the moment. The fact that this study enrolls patients with all types of CMT 1 and 2 is also encouraging and motivating to the community. I am happy to be involved in this exciting clinical study.”

Thomas Holm Pedersen, Chief Executive Officer of NMD Pharma, highlighted the urgent need for new treatments for CMT, a debilitating disease with no approved medicines or cure. He stated, “As a highly debilitating disease with no currently approved medicines or a cure, there is an urgent need for new therapeutic approaches to help improve the quality of life of patients with CMT and their families. NMD670 has demonstrated promising clinical results in a proof-of-mechanism study in patients with NMJ dysfunction in the rare neuromuscular disease myasthenia gravis, and we are confident that our novel therapeutic approach has the potential to also provide benefit to those affected by CMT. Our focus is to develop treatments that address the symptoms of CMT across multiple subtypes and provide needed muscle strength, power, and endurance that can help patients gain and maintain the ability to independently perform daily activities. I look forward to keeping patient communities updated on our progress.”

In June 2023, NMD Pharma shared findings from an international observational study, ESTABLISH1, at the Peripheral Nerve Society Annual Meeting. The study focused on neuromuscular transmission deficits and muscle function in CMT types 1 and 2, highlighting neuromuscular junction (NMJ) transmission deficits as an underappreciated characteristic of the disease.

With the start of this Phase 2 CMT clinical trial, NMD Pharma is currently running three global clinical trials investigating NMD670 in various rare neuromuscular conditions. These include a Phase 2 study for adults with spinal muscular atrophy (SMA) type 3 and a Phase 2b study for patients with generalized myasthenia gravis (gMG).

Patients with CMT types 1 and 2 in the US and Europe are encouraged to participate in this study. New clinical trial sites are being announced, and more information on these sites can be found on ClinicalTrials.gov.