NMD Pharma A/S, a spin-out from Aarhus University in Denmark, has reported positive top-line data from a Phase 1/2a trial of a small molecule drug for myasthenia gravis, a chronic autoimmune disease. The drug, NMD670, recently received an orphan designation from the US Food and Drug Administration in light of the seriousness of the disease. Myasthenia gravis causes potentially life-threatening muscle weakness due to impaired neuromuscular transmission. NMD670 inhibits the skeletal muscle-specific chloride ion channel CIC-1.
