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Page last updated: 30th April 2024


At the 4th International Scientific Congress on SMA, Martin Skov (NMD Pharma, Aarhus, Denmark), innovation manager at NMD Pharma, presented data from a trial of a new, investigational drug called NMD670 with potential for SMA.

NMD670 is an orally-bioavailable, small molecule that specifically targets a protein called ClC-1 that is found almost exclusively on the surface of muscles cells. The function of ClC-1 is to dampen the excitability of muscles and prevent them from becoming overstimulated.

By inhibiting ClC-1 function, NMD670 can increase and improve the passage of signals from motor neurons to muscles, which can increase how easily muscles are able to contract. NMD670 therefore has potential for the treatment of SMA, where the passage of electrical signals from motor neurons to muscles can be severely affected.

Dr. Skov presented work using a rat model of a neuromuscular disease called myasthenia gravis. The data indicate that single and prolonged administration of NMD670 can improve muscle function and mobility of the model. In addition, NMD670 was also tested in 12 people with myasthenia gravis and shown to be safe and cause clinically meaningful improvements in the disease.

Given that NMD670 improves muscle function, it was hypothesised that it could be valuable for the treatment of SMA. Hence, experiments were performed showing that NMD670 was capable of improving the electrical signalling between motor nerves and muscles in a mouse model of SMA, leading to a marked increase in muscle strength.

Given these results, the SYNPASE-SMA trial of NMD670 was initiated in late 2023 in North America and Europe. Designed to assess the efficacy, safety, and tolerability of NMD670 in 50 ambulatory participants with SMA Type 3, SYNPASE-SMA is a phase 2, randomized, double-blind, placebo-controlled study.

SYNPASE-SMA is estimated to be completed in late 2024, and the findings will be published and publicised in due course.

To read more about NMD670, see: