NMD Pharma initiates Phase II trial of NMD670 in Spinal Muscular Atrophy
Aarhus, Denmark, 26 September 2023 – NMD Pharma A/S, a clinical-stage biotech company developing first-in-class, small molecule ClC-1 inhibitors directly targeting muscle function within neuromuscular disorders, today announces that the first patient has been dosed in a Phase II clinical trial with the ClC-1 inhibitor NMD670 in patients impacted by spinal muscular atrophy (SMA). The patient was dosed under the direction of Prof. dr. Kristl Claeys, MD, PhD at the Kliniekhoofd Neurologie, Neuromusculaire Ziekten, Laboratory for Muscle Diseases and Neuropathies at UZ Leuven University Hospital in Belgium.
The Phase II clinical trial (NCT05794139) is a randomized, double-blind, placebo-controlled, 2-way crossover study to evaluate the efficacy, safety, and tolerability of twice-a-day oral dosing of NMD670 for 21 days in ambulatory adult patients with SMA Type 3. The study is an international multicenter study including sites in North America and Europe.
“SMA is a rare neuromuscular disorder characterised by severe muscle weakness and fatigue that greatly affects the quality of life of patients and their families.” commented Jorge A. Quiroz, EVP and Chief Medical Officer NMD Pharma. “NMD670 has already been shown to be safe and efficacious in a proof of mechanism study in patients with myasthenia gravis (MG) and we are confident that it could also be beneficial for the treatment of patients affected by SMA.”
Thomas Holm Pedersen, Chief Executive Officer of NMD Pharma, added: “Despite great advances in the therapeutic armamentarium of therapies to treat SMA, patients can still experience muscle weakness and impaired mobility. This represents a significant unmet need for new interventions that can help patients independently perform daily activities and better manage the symptoms of SMA. We are very pleased to announce the dosing of the first patient in this Phase II study. This represents a significant milestone for NMD Pharma as we further expand our pipeline into new indications, and I look forward to keeping the patient communities and market updated as we progress.”
“We are excited for the start of this Phase II clinical trial that will test a new approach for SMA. Building on the impact from our current genetically targeted disease-modifying therapies, NMD670 may help to restore muscle strength and function by targeting the neuromuscular junction which is known to be affected in SMA," said Kenneth Hobby, President, of Cure SMA.
NMD670 is a first-in-class small molecule inhibitor of the muscle-specific chloride ion channel, the ClC-1 ion channel. NMD Pharma has pre-clinically demonstrated that ClC-1 inhibition can enhance neuromuscular transmission and ultimately skeletal muscle function in multiple animal models of neuromuscular diseases. NMD670 has already demonstrated positive clinical study results in a Phase I/II study in patients with MG. Based on these preclinical and clinical data, it is expected that this novel approach could be beneficial in the treatment of patients impacted by SMA.