AARHUS, Denmark, June 17, 2026
NMD Pharma A/S announced new positive clinical findings from its Phase 2a SYNAPSE-CMT study evaluating ignaseclant in adults with Charcot-Marie-Tooth (CMT) disease. The late-breaking results were presented during an oral session at the 2026 Peripheral Nerve Society (PNS) Annual Meeting held in Maastricht, Netherlands. The randomized, double-blind, placebo-controlled trial demonstrated consistent improvements in muscle strength, motor function, and patient-reported outcomes, while maintaining a favorable safety profile. The study evaluated 81 ambulatory adults with genetically confirmed CMT type 1 or type 2 who received twice-daily oral ignaseclant for 21 days, followed by a one-week post-treatment assessment. The findings provide further clinical evidence supporting ignaseclant, a first-in-class skeletal muscle-targeted ClC-1 inhibitor, as a potential treatment for patients living with this rare inherited neuromuscular disorder.
Clinical Trial Demonstrates Significant Functional Improvements
The SYNAPSE-CMT (NCT06482437) study was designed to evaluate the clinical activity, safety, and tolerability of ignaseclant across multiple functional endpoints. Patients receiving treatment demonstrated early improvements in the Charcot-Marie-Tooth Functional Outcome Measure (CMT-FOM) beginning on Day 7, with benefits continuing throughout the treatment period and remaining statistically significant by Day 28, one week after dosing ended. Investigators also reported significant improvements in hand grip strength, reaching statistical significance at Day 21 (p=0.02) and strengthening further at Day 28 (p<0.01). Additional positive trends were observed in the 9-Hole Peg Test and 10-Meter Walk/Run Test, suggesting improvements in both upper- and lower-limb motor performance. According to the investigators, the persistence of clinical benefits beyond the treatment period may indicate ongoing functional recovery at the neuromuscular junction, supporting the potential disease-modifying effects of the therapy.
Strong Safety Profile Supports Continued Clinical Development
Safety findings from the Phase 2a study were equally encouraging. Ignaseclant was well tolerated, with all reported adverse events classified as mild or moderate. Importantly, investigators reported no serious adverse events, no treatment discontinuations, and no unexpected safety concerns during the study. The favorable safety profile, combined with measurable improvements in muscle strength and functional performance, strengthens the clinical rationale for advancing ignaseclant into larger and longer-duration studies. Dr. David Herrmann, Chief of the Neuromuscular Division at the University of Rochester Medical Center, stated that the observed improvements in muscle strength and patient-reported outcomes, together with the sustained effects after treatment discontinuation, support the potential of ignaseclant as a novel therapeutic approach for Charcot-Marie-Tooth disease and justify further clinical investigation.
Advertisement
Ignaseclant Advances as a Promising Therapy for Rare Neuromuscular Diseases
The encouraging Phase 2a data further reinforce NMD Pharma’s strategy of developing innovative therapies that directly improve skeletal muscle function rather than targeting the underlying genetic mutation alone. Ignaseclant, formerly known as NMD670, selectively inhibits the ClC-1 chloride channel in skeletal muscle, enhancing muscle excitability and improving neuromuscular performance. Beyond Charcot-Marie-Tooth disease, the investigational therapy is also being evaluated in generalized myasthenia gravis (gMG) and spinal muscular atrophy (SMA), expanding its potential across multiple rare neuromuscular conditions with significant unmet medical needs. The positive results presented at the 2026 PNS Annual Meeting provide important clinical validation of the company’s muscle-targeted therapeutic platform and establish a strong foundation for future late-stage clinical development. With sustained efficacy signals, a favorable safety profile, and evidence of continued functional improvement following treatment, ignaseclant represents a promising candidate that could potentially improve muscle strength, mobility, and quality of life for patients living with inherited neuromuscular disorders.
Source: NMD Pharma press release
