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NMD Pharma Receives FDA IND Clearance to initiate a Phase 2b Clinical Trial of NMD670 in Generalized Myasthenia Gravis Patients in the USNMD Pharma Receives FDA IND Clearance to initiate a Phase 2b Clinical Trial of NMD670 in Generalized Myasthenia Gravis Patients in the US Aarhus, Denmark, 22 March 2024 – 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, atient 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. -END- Contacts NMD Pharma A/S ICR Consilium About NMD Pharma About Myasthenia Gravis (MG) |