NMD Pharma announces topline results from its Phase 2a study of ignaseclant in Charcot-Marie-Tooth disease Types 1 and 2
- Exploratory Phase 2a study showed consistent and clinically meaningful functional improvements across muscle strength, motor performance, and patient-reported outcomes in patients with Charcot-Marie-Tooth disease (CMT)Benefits emerged early, were durable beyond treatment discontinuation, and were supported by patient-reported outcomes, with ignaseclant being safe and well tolerated and no serious adverse events reported
- NMD Pharma is accelerating ignaseclant’s clinical development in CMT alongside multiple near-term clinical catalysts across its neuromuscular pipeline, with Phase 2 studies in spinal muscular atrophy (SMA) and generalised myasthenia gravis (gMG) expected to deliver additional data in 2026
Aarhus, Denmark, 3 February 2026 – NMD Pharma A/S, a clinical-stage biotechnology company dedicated to developing novel therapies to restore skeletal muscle health, today announced topline results from its Phase 2a SYNAPSE-CMT study evaluating ignaseclant (formerly NMD670), an investigational first-in-class small molecule inhibitor of the skeletal muscle-specific chloride ion channel 1 CIC-1, in patients living with Charcot-Marie-Tooth disease (CMT) types 1 or 2. There are currently no FDA-approved therapies for the treatment of Charcot-Marie-Tooth disease.
SYNAPSE-CMT (NCT06482437) was a randomized, double-blind, placebo-controlled Phase 2a study designed to explore the clinical activity, safety, and tolerability of twice-daily oral ignaseclant administered over 21 days, with follow-up assessments at day 28. The trial enrolled 81 adult patients with any genetically confirmed CMT1 or CMT2 subtype across clinical sites in the US and Europe.
Although the study did not demonstrate a treatment difference on the 6-minute walk test (6MWT) at 21 days, the pre-specified primary endpoint for this exploratory study, there was improvement across multiple pre-specified secondary endpoints: Patients receiving ignaseclant demonstrated consistent improvements in muscle strength, functional performance, and patient-reported outcomes. These secondary endpoints are recognized as highly relevant by the CMT clinical and patient community.
Key findings from both CMT1 and CMT2 included:
- Improvements on the validated Charcot-Marie-Tooth Functional Outcome Measure (CMT-FOM), a composite scale assessing strength and function across multiple domains, with separation from placebo observed during treatment and maintained through day 28
- Robust improvements in handgrip strength, a clinically meaningful domain for patients with CMT
- Improvements in fine hand function, supporting functional benefit beyond gross motor performance
- Patient-reported improvements in physical function and disease impact, as measured by the CMT-Health Index (CMT-HI), align with objective functional findings