NMD Pharma A/S, a Denmark-based clinical-stage biotechnology company, announced on Wednesday that further safety and efficacy data from its Phase 2a SYNAPSE-CMT exploratory study evaluating ignaseclant in patients living with Charcot-Marie-Tooth disease (CMT) types 1 or 2 has been presented in a late-breaking oral presentation at the 2026 Peripheral Neuroscience Association (PNS) Annual Meeting held in Maastricht in the Netherlands.

SYNAPSE-CMT was a randomised, 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 ambulatory adult patients with any genetically confirmed CMT1 or CMT2 subtype across clinical sites in the United States and Europe.

The presentation showed that ignaseclant demonstrated consistent signals of benefit across multiple secondary functional endpoints. Signals for improvement versus placebo were observed in the CMT Functional Outcome Measure (CMT-FOM) composite scale at Day 7, continued improvements to Day 21 with improvements sustained and significant at Day 28.

According to NMD, Ignaseclant was well tolerated, with all adverse events reported as mild or moderate and no serious adverse events or discontinuations. Overall, the findings indicate that 21 days of ClC-1 inhibition with ignaseclant improved muscle strength and motor function in adults with CMT, with effects persisting seven days after treatment cessation, suggesting potential functional or structural recovery at the neuromuscular junction.