NMD Pharma A/S announces that it has dosed the first generalized myasthenia gravis patient in a Phase 2b clinical trial of NMD670, after receiving FDA IND clearance in March 2024 to conduct the study.
NMD670 is a first-in-class small molecule inhibitor of the skeletal muscle specific chloride ion channel (CIC-1). NMD Pharma has demonstrated that CIC-1 inhibition enhances weakened neuromuscular transmission and restores skeletal muscle function, and this novel treatment approach has demonstrated compelling preclinical and clinical data in animal models of myasthenia gravis, spinal muscular atrophy and a range of other neuromuscular disorders. NMD670 has also been granted orphan-drug designation by the U.S. FDA for treatment of gMG.