Neuromuscular diseases (NMDs) are a variable group of disorders with different etiologies, but most NMDs lead to muscle weakness, fatigue and major disability. In a group of NMDs, such as myasthenia gravis (MG), Charcot-Marie-Tooth disease (CMT), and spinal muscular atrophy (SMA), failure of muscle activation due to compromised neuromuscular transmission contributes to muscle weakness and fatigue. Drugs that improve neuromuscular junction (NMJ) transmission have been suggested as candidates for treatment of muscle symptoms in these disorders.
NMD670 is a first-in-class inhibitor of skeletal muscle-specific chloride channel ClC-1, developed to improve muscle weakness and fatigue in certain NMDs. Preclinical data in animal models of MG, SMA and CMT and phase 1 study results in healthy volunteers and MG patients demonstrated that NMD670 improves muscle strength and indicated a favorable safety profile of the drug thus supporting further clinical development for these indications.
Three phase 2 studies with NMD670 are currently ongoing. SYNAPSE-SMA enrolls patients with SMA type 3 with NMJ deficits. Participants receive NMD670 or placebo twice a day for 21 days. SYNAPSE-MG is a dose finding study in which Acetylcholine Receptor positive (AchR+) and Muscle Specific Kinase positive (MuSK+) patients with MG are enrolled. The aim is to evaluate the efficacy and safety of 3 dose levels of NMD670, administered twice a day for 21 days versus placebo.
SYNAPSE-CMT is aiming to assess the efficacy, safety, and tolerability of NMD670 in ambulatory adults with CMT type 1 and 2. Participants receive NMD670 or placebo twice a day for 21 days.
Detailed study designs and updates on recruitment will be provided by NMD Pharma at 2025 MDA Clinical & Scientific Conference in March 2025.