NMD Pharma Advances Ignaseclant to Late-Stage CMT Trials Despite Phase IIa Miss

NMD Pharma will accelerate late-stage development of its lead rare disease candidate ignaseclant in Charcot-Marie-Tooth (CMT) disease, despite missing the primary endpoint in a Phase IIa study. In the 28-day SYNAPSE-CMT trial (NCT06482437), ignaseclant did not significantly improve six-minute walk test scores versus placebo.

However, the oral small molecule met several key secondary endpoints. Patients showed improvements in CMT Functional Outcome Measures (CMT-FOM), clinically meaningful gains in handgrip strength and fine motor function, and better patient-reported outcomes on the CMT-Health Index (CMT-HI). These benefits persisted after treatment discontinuation. Ignaseclant was well tolerated, with only mild-to-moderate adverse events.

Given the lack of approved therapies for CMT, researchers highlighted the functional relevance of the secondary data. Other firms active in CMT include Applied Therapeutics with govorestat and Novartis, which is developing CKD-510 and gene therapy EDK060.

10-02-2026