NMD Pharma starts phase 2b trial of NMD670 in generalized myasthenia gravis patients

NMD Pharma A/S, a clinical-stage biotech company dedicated to developing novel and improved treatments for patients living with neuromuscular diseases, announces today that it has dosed the first generalized myasthenia gravis (gMG) patient in a Phase 2b clinical trial of NMD670, after receiving FDA IND clearance in March 2024 to conduct the study. The first US patient was dosed under the direction of Dr. Marc Feinberg at SFM Clinical Research, LLC in Boca Raton, Florida, USA.

The Phase 2b clinical trial is a dose range-finding, double-blinded, placebo-controlled study of NMD670, a twice daily, small molecule oral inhibitor of the skeletal muscle specific ClC-1 chloride ion channel, in patients with gMG, over 21 days, who are anti-acetylcholine receptor (AChR), or anti-muscle-specific tyrosine kinase (MuSK) antibody positive. The study will evaluate changes in the Quantitative Myasthenia Gravis Total Score and the Myasthenia Gravis Activities of Living, among other endpoints and take place across both US and European clinical sites.

Jorge A. Quiroz, EVP, Chief Medical Officer of NMD Pharma, commented: “Generalized myasthenia gravis is a rare disorder characterized by neuromuscular transmission dysfunction that leads to severe and fluctuating muscle weakness and fatigue. While new and approved therapies address the autoimmune response, many patients with myasthenia gravis have persistent symptomatology. NMD670 is a first-in-class development candidate which is expected to improve muscle power and endurance to help manage persistent and fluctuating symptoms.”

Samantha Masterson, President and Chief Executive Officer of the Myasthenia Gravis Foundation of America (MGFA), added: “Many gMG patients continue to have critical unmet needs and we hope that new therapies, like this muscle-directed therapy NMD670, can be developed to help provide additional strength and endurance so patients can live a less-restricted life with their disease. We will eagerly and closely follow the trial’s progress and outcomes, and hope the promising results shown to date translate into further clinically meaningful benefits for patients.”

NMD670 is a first-in-class, orally administered small molecule inhibitor of the skeletal muscle specific ClC-1 chloride ion channel. NMD Pharma has recently published positive Phase 1/2a data in the journal Science Translational Medicine, establishing the first clinical proof-of-mechanism of CIC-1 inhibitors in patients suffering from gMG while also confirming safety and tolerability after a single dose of NMD670 in patients.