NMD Pharma Initiates Phase 2 Study of NMD670 in Patients with Charcot-MarieTooth Disease Type 1 and 2
NMD Pharma A/S, a clinical-stage biotech company dedicated to developing novel and improved treatments for patients living with neuromuscular diseases, announces that it has dosed the first Charcot-Marie-Tooth (CMT) disease patient in its Phase 2a clinical trial of NMD670 following FDA IND clearance in June 2024. The first patient was dosed under the direction of Yessar Hussain, MD at the Austin Neuromuscular Center in Austin, TX in the US.
NMD670 is a first-in-class small molecule inhibitor of the skeletal muscle specific chloride ion channel 1 (CIC1). NMD Pharma has demonstrated that CIC-1 inhibition amplifies the muscle’s responsiveness to weak signals, improving neuromuscular transmission and restores skeletal muscle function.
The Phase 2a clinical trial (NCT06482437), named SYNAPSE-CMT, is a randomized, double-blind, placebo controlled study, evaluating the efficacy, safety, and tolerability of a twice daily oral dose of NMD670 over 21 days in 80 adult patients with any genetically confirmed CMT1 or CMT2 subtype. The study will evaluate changes in the 6-minute walk test (6MWT), time to complete the 10-meter walk/run test (10MW/R) and time to complete timed-up-and-go test (TUG), among other safety and efficacy endpoints over a 21-day study period, taking place across clinical sites in both the US and Europe.
Yessar Hussain, MD, Austin Neurological Center, stated: “Patients with CMT are very excited about entering into this study. This is the only clinical study with a clinically feasible treatment to address the muscle weakness and fatigue with a pharmaceutical therapy at the moment. The fact that this study enrolls patients with all types of CMT 1 and 2 is also encouraging and motivating to the community. I am happy to be involved in this exciting clinical study.”
Thomas Holm Pedersen, Chief Executive Officer of NMD Pharma, added: “As a highly debilitating disease with no currently approved medicines or a cure, there is an urgent need for new therapeutic approaches to help improve the quality of life of patients with CMT and their families. NMD670 has demonstrated promising clinical results in a proof-of-mechanism study in patients with NMJ dysfunction in the rare neuromuscular disease myasthenia gravis, and we are confident that our novel therapeutic approach has the potential to also provide benefit to those affected by CMT. Our focus is to develop treatments that address the symptoms of CMT across multiple subtypes and provide needed muscle strength, power and endurance that can help patients gain and maintain ability to independently perform daily activities. I look forward to keeping patient communities updated on our progress.”
In June 2023, NMD Pharma announced the results of ESTABLISH1, an international observational study of neuromuscular transmission deficits and muscle function in CMT types 1 and 2, at the Peripheral Nerve Society Annual Meeting, highlighting neuromuscular junction (NMJ) transmission deficits as an unappreciated disease characteristic in CMT.
With the initiation of this Phase 2 CMT clinical trial, NMD Pharma now has three ongoing global clinical trials investigating NMD670 across rare neuromuscular diseases characterized by a high degree of patient impact and need, including a Phase 2 study in adults living with spinal muscular atrophy (SMA) type 3 and a Phase 2b study in gMG patients.
Further information on the study can be found here: Study Details | Safety and Efficacy of NMD670 in Adult Patients With Type 1 and Type 2 Charcot-Marie-Tooth Disease | ClinicalTrials.gov
Patients with CMT disease type 1 and type 2 in the US and Europe are encouraged to participate in the study. New clinical trial sites are being announced, starting this month and continuing until March 2025. For further information and a list of currently active clinical trial sites, please look to the ClinicalTrials.gov link about or email us at [email protected].
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