NMD Pharma Receives FDA IND Clearance to initiate a Phase 2b Clinical Trial of NMD670 in Generalized Myasthenia Gravis Patients in the US

AARHUS, Denmark I March 22, 2024 I NMD Pharma A/S, a clinical-stage biotech company dedicated to developing novel and improved treatments for patients living with neuromuscular diseases, today announces that it has received clearance from the US Food and Drug Administration (FDA) for its Investigational New Drug (IND) application to initiate a Phase 2b clinical trial of NMD670 in patients with generalized myasthenia gravis (gMG) who are anti-acetylcholine receptor (AChR) or anti-muscle-specific tyrosine kinase (MuSK) antibody positive.

The Phase 2b clinical trial is a dose range-finding, double-blind, placebo-controlled study of NMD670, a twice daily oral muscle-targeted therapy, in gMG patients experiencing persistent symptoms or fluctuating symptoms despite treatment on current standard of care. The trial will take place in both US and European clinical sites. The study is expected to initiate enrollment of patients in the coming months.

NMD670 is a first-in-class, muscle-targeted 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 of NMD670 in patients.

Thomas Holm Pedersen, Chief Executive Officer of NMD Pharma, said: “This is an important milestone for NMD Pharma, enabling us to progress our lead development candidate NMD670 into a Phase 2b trial in the US in generalized myasthenia gravis patients with AChR and MuSK positive antibodies. It follows positive Phase 1/2a data which provided proof-of-mechanism and where clinically and statistically significant effects were seen in patients. We hear from myasthenia gravis patients, patient organizations and physician experts that a great number of myasthenia gravis patients continue to experience on-going severe symptoms of muscle weakness and fatigue despite treatment with new or conventional treatments. We are excited to bring forward a unique and validated muscle-targeted approach with promise to provide improvements in muscle function leading to clinically meaningful improvements in activities of daily living and quality of life.”

In November 2023, NMD Pharma announced  that it had raised €75 million (~$80 million) in a Series B financing to complete three Phase 2 studies with NMD670, the Company’s lead ClC-1 inhibitor in AChR and MuSK antibody positive myasthenia gravis, spinal muscular atrophy, and Charcot-Marie-Tooth disease.

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 neuromuscular transmission and restores skeletal muscle function, and this novel treatment approach has demonstrated compelling preclinical and clinical data in animal models of MG, 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 MG.

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 and it can be life-threatening when affecting the muscles responsible for breathing. There are approximately 100,000 people in the European Union, 65,000 people in the United States and 20,000 people in Japan living with the disease.