APN-501 series (Cleveland Clinic)

NMD Pharma Initiates Phase 2b Trial of NMD670 in Generalized Myasthenia Gravis Patients AARHUS, Denmark I June 11, 2024 I 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. Further information on the study can be found here: Study Details | Safety and Efficacy of 3 Dose Levels of NMD670 in Adult Patients With Myasthenia Gravis | ClinicalTrials.gov Generalized myasthenia gravis patients who are AChR or MuSK antibody positive in the US and Europe are encouraged to participate in the study. Further information and a list of currently active investigational sites can be found via email at contact@nmdpharma.com . About NMD Pharma NMD Pharma A/S is a clinical-stage biotech company developing a first-in-class platform of small molecule therapies selectively targeting the skeletal muscle chloride ion channel (ClC-1) for the treatment of severe neuromuscular disorders. The Company was founded on more than 15 years of muscle physiology research with a focus on regulation of skeletal muscle excitability under physical activity. NMD Pharma has built a world-leading muscle electrophysiology platform leveraging the in-depth know-how of muscle physiology and muscular disorders, small molecule modulators, enabling technologies and tools as well as in vivo pharmacology models for discovering and developing proprietary modulators of neuromuscular function. The Company has built significant clinical and development expertise as its programmes have progressed through the clinic. NMD Pharma has raised ~€155 million from investors including Novo Holdings, Lundbeckfonden BioCapital, INKEF Capital, Roche Venture Fund, and Jeito Capital. Find out more about us online at http://www.nmdpharma.com . About NMD670 NMD670 is NMD Pharma’s lead development program. It 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. About Myasthenia Gravis (MG) MG is a rare and chronic autoimmune disease where IgG antibodies disrupt communication between nerves and muscles causing debilitating and potentially life-threatening muscle weakness. It commonly affects the muscles that control the eyes and eyelids, facial expressions, chewing, swallowing, and speaking but in most patients, it eventually impacts most skeletal muscles. More than 85% of people with MG progress to generalized MG within 18 months, with symptoms that can be life-threatening when affecting the muscles responsible for breathing. While new and conventional existing therapies for MG fight the immune system, many patients are still left to deal with severe persistent and unpredictable symptoms. There are approximately 100,000 people in the European Union, and more than 65,000 people in the United States and 20,000 people in Japan with living with generalized myasthenia gravis. SOURCE: NMD Pharma