NMD Pharma: HNF TRIAD Partner Community Update on the SYNAPSE-CMT (NMD670) Clinical Trial

NMD Pharma: HNF TRIAD Partner Community Update on the SYNAPSE-CMT (NMD670) Clinical Trial

NMD Pharma, a proud HNF TRiAD Partner, has announced the successful completion of patient enrollment in their SYNAPSE-CMT clinical trial, marking a major milestone in the development of new treatments for Charcot-Marie-Tooth (CMT) disease. The Phase 2 study is designed to evaluate the safety and efficacy of NMD670, a first-in-class therapeutic candidate targeting muscle weakness associated with neuromuscular disorders.

About NMD670

NMD670 is a novel small molecule that enhances muscle contraction by inhibiting a calcium-activated chloride channel (ClC-1) found exclusively in skeletal muscle. By blocking this channel, the compound promotes stronger and more consistent muscle contractions. This mechanism is expected to improve muscle strength, endurance, and overall function—including mobility, balance, and performance in daily activities—by amplifying weak signals from nerves to muscles. For individuals living with CMT, this may help sustain steady muscle performance and mitigate fatigue.

The SYNAPSE-CMT Clinical Trial

The SYNAPSE-CMT trial successfully enrolled 81 participants across global sites, including individuals with CMT Type 1 and Type 2, surpassing initial recruitment projections. Participants received either NMD670 or a placebo twice daily for 21 days. Following completion, NMD Pharma will analyze the results to assess safety and functional improvements between the treatment and placebo groups.

Beyond CMT, NMD670 is being explored for additional neuromuscular conditions that affect the nerve-to-muscle connection, such as myasthenia gravis and spinal muscular atrophy (SMA).

Encouraging Early Findings

In an earlier study involving 12 patients with myasthenia gravis, NMD670 demonstrated a strong safety and tolerability profile. While the compound does not address the genetic root cause of CMT, it is designed to improve muscle performance, reduce fatigue, and enhance balance and motor coordination, ultimately supporting better quality of life for patients.

A Step Toward Comprehensive Care

Ongoing research at academic institutions and biotech companies aims to develop gene-targeted therapies that address the underlying genetic causes of CMT. In the future, patients may benefit from combination therapies—using treatments like NMD670 to strengthen the nerve-muscle connection alongside genetic therapies that correct the source of the disease.