NMD Pharma recently announced that it has received clearance from the US Food and Drug Administration (FDA) for its Investigational New Drug (IND) application to initiate a Phase 2 clinical trial of NMD670 in patients living with CMT types 1 and 2.

NMD670 is a first-in-class, muscle-targeted small molecule inhibitor of the skeletal muscle specific ClC-1 chloride ion channel. The Phase 2 clinical trial is a randomized, double-blinded, placebo-controlled study of twice daily orally administered NMD670 in a study of consisting of approximately 84 adult patients with type 1 and type 2 CMT. The trial will take place at clinical sites in both the US and Europe, and it is expected to start enrolling patients soon.

In June 2023, NMD Pharma presented the results from an observational study of neuromuscular function in patients with CMT types 1 and 2which revealed, for the first time, that neuromuscular junction (NMJ) transmission deficits are evident in patients with CMT types 1 and 2 and associated with disease severity, assessed through a range of clinical measurements of muscle strength and motor function. These results demonstrated that the disease symptoms that NMD670 has the potential to treat are present in CMT type 1 and 2 and also provided important information to aid in the design of their upcoming CMT clinical trial with NMD670.

“Although we did not fund this NMD Pharma project, this is an exciting moment for the CMT community,” says Cleary Simpson, CMTRF CEO. “We are pleased to see a Phase 2 trial for CMT with NMD670, a treatment with the potential for improving muscle function in people living with multiple types of CMT.”