FDA Approves Phase 2b Trial of NMD670 for US Myasthenia Gravis Patients

NMD Pharma, a biotech firm specializing in neuromuscular disorder treatments, has been granted FDA approval to commence a Phase 2b clinical trial for NMD670, a potential treatment for generalized myasthenia gravis (gMG). The trial is designed to evaluate the efficacy and dosage of NMD670, an oral muscle-targeted therapy, in patients with persistent or fluctuating symptoms despite standard care.

The study will be conducted across the United States and Europe and is set to begin enrolling participants shortly. NMD670 is a novel small molecule that inhibits the ClC-1 chloride ion channel in skeletal muscles. It has shown promise in a Phase 1/2a trial, where it demonstrated a proof-of-mechanism and was well-tolerated by patients.

The CEO of NMD Pharma, Thomas Holm Pedersen, highlighted the significance of this milestone, which follows the positive results from earlier trials. He emphasized the need for new treatments, as many gMG patients continue to suffer from severe symptoms despite current therapies. The company is eager to offer a new muscle-targeted approach that could lead to significant improvements in muscle function and quality of life.

In late 2023, NMD Pharma secured approximately $80 million in Series B financing, which will support the completion of three Phase 2 studies for NMD670, targeting gMG, spinal muscular atrophy, and Charcot-Marie-Tooth disease.

NMD Pharma is a clinical-stage company founded on extensive research into muscle physiology, with a focus on the regulation of skeletal muscle excitability. The company has developed a platform of therapies that selectively target the ClC-1 chloride ion channel, aiming to treat severe neuromuscular disorders. They have demonstrated expertise in muscle electrophysiology and have successfully developed proprietary modulators of neuromuscular function.

Myasthenia gravis is a rare autoimmune disease characterized by muscle weakness caused by antibodies disrupting nerve-muscle communication. It can be life-threatening and affects approximately 185,000 people across the European Union, the United States, and Japan. NMD670 has received orphan-drug designation from the U.S. FDA for the treatment of MG, indicating its potential as a significant advancement in patient care.