NMD Pharma A/S has announced the initiation of a Phase 2a clinical trial of NMD670 in patients with Charcot-Marie-Tooth (CMT) disease types 1 and 2. The first patient was dosed at the Austin Neuromuscular Center in Austin, TX, under the direction of Yessar Hussain, MD.
The Phase 2a clinical trial, named SYNAPSE-CMT (NCT06482437), is designed as a randomized, double-blind, placebo-controlled study. It aims to evaluate the efficacy, safety, and tolerability of NMD670, administered orally twice daily over 21 days, in 80 adult patients with genetically confirmed CMT1 or CMT2 subtypes. The study is being conducted across clinical sites in both the US and Europe.
NMD670: A Novel Therapeutic Approach
NMD670 is a first-in-class small molecule inhibitor targeting the skeletal muscle-specific chloride ion channel 1 (ClC-1). Preclinical studies have demonstrated that inhibiting ClC-1 enhances the muscle's responsiveness to weak signals, thereby improving neuromuscular transmission and restoring skeletal muscle function.
Study Endpoints and Design
The SYNAPSE-CMT trial will primarily assess changes in the 6-minute walk test (6MWT), time to complete the 10-meter walk/run test (10MW/R), and time to complete the timed-up-and-go test (TUG). Secondary endpoints include additional measures of safety and efficacy over the 21-day study period.
Expert Commentary
Yessar Hussain, MD, from the Austin Neurological Center, noted, "Patients with CMT are very excited about entering into this study... The fact that this study enrols patients with all types of CMT 1 and 2 is also encouraging and motivating to the community."
Thomas Holm Pedersen, Chief Executive Officer of NMD Pharma, 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."
Charcot-Marie-Tooth Disease: An Unmet Need
CMT comprises a group of hereditary sensory and motor neuropathies causing damage to peripheral nerves. This damage leads to muscle weakness, fatigue, and atrophy, significantly impairing mobility, independence, and overall quality of life. CMT affects approximately 136,000 individuals in the United States and 3.2 million worldwide. Symptoms typically manifest during adolescence or early adulthood.
NMD Pharma's Broader Clinical Program
With the initiation of the Phase 2 CMT clinical trial, NMD Pharma now has three ongoing global clinical trials investigating NMD670 across rare neuromuscular diseases. These include a Phase 2 study in adults with spinal muscular atrophy (SMA) type 3 and a Phase 2b study in generalized myasthenia gravis (gMG) patients.
