NMD Pharma, a clinical-stage biotech company dedicated to developing novel and improved treatments for patients living with neuromuscular diseases, has announced that it has dosed the first Charcot-Marie-Tooth disease patient in its Phase 2a clinical trial of NMD670 following FDA IND clearance in June 2024. The first patient was dosed under the direction of Yessar Hussain, MD at the Austin Neuromuscular Center in Austin, TX in the US.
NMD670 is a first-in-class small molecule inhibitor of the skeletal muscle specific chloride ion channel 1 (CIC-1). NMD Pharma has demonstrated that CIC-1 inhibition amplifies the muscle’s responsiveness to weak signals, improving neuromuscular transmission and restores skeletal muscle function. To date CMTRF has not funded this project but is pleased by its success.
“The initiation of this Phase 2 clinical trial for NMD670 marks an exciting milestone for the CMT community, igniting hope for patients and families who have long awaited meaningful progress toward effective treatments,” says Laura MacNeill, CMTRF’s CEO. “We commend NMD Pharma for their dedication to addressing the unmet need in CMT care and are inspired by the potential of NMD670 to improve muscle function and enhance daily living for those affected by CMT.”
The Phase 2a clinical trial (NCT06482437), named SYNAPSE-CMT, is a randomized, double-blind, placebo-controlled study, evaluating the efficacy, safety, and tolerability of a twice daily oral dose of NMD670 over 21 days in 80 adult patients with any genetically confirmed CMT1 or CMT2 subtype. The study will evaluate changes in the 6-minute walk test (6MWT), time to complete the 10-meter walk/run test (10MW/R) and time to complete timed-up-and-go test (TUG), among other safety and efficacy endpoints over a 21-day study period, taking place across clinical sites in both the US and Europe.
With the initiation of this Phase 2 CMT clinical trial, NMD Pharma now has three ongoing global clinical trials investigating NMD670 across rare neuromuscular diseases characterized by a high degree of patient impact and need, including a Phase 2 study in adults living with spinal muscular atrophy (SMA) type 3 and a Phase 2b study in gMG patients.
Further information on the study can be found here: Study Details | Safety and Efficacy of NMD670 in Adult Patients With Type 1 and Type 2 Charcot-Marie-Tooth Disease | ClinicalTrials.gov
