NMD Pharma, a clinical-stage biotech company leading the development of novel first-in-class therapies for severe neuromuscular disorders, recently announced that the first SMA patient has been dosed in their Phase II clinical trial of the CIC-1 inhibitor NMD670.
To read the full press release, visit this link.
MD670 is a first-in-class small molecule inhibitor of the muscle-specific chloride ion channel, the ClC-1 ion channel. NMD Pharma has pre-clinically demonstrated that ClC-1 inhibition can enhance neuromuscular transmission and ultimately skeletal muscle function in multiple animal models of neuromuscular diseases.
NMD670 has already demonstrated positive clinical study results in a Phase I/II study in patients with MG. Based on these preclinical and clinical data, it is expected that this novel approach could be beneficial in the treatment of patients impacted by SMA.
