Aarhus, Denmark, 17 June 2026 – NMD Pharma A/S, a clinical-stage biotechnology company dedicated to developing novel therapies to restore skeletal muscle health, today announces 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, The Netherlands from June 13-16, 2026. The late-breaking presentation was given by David Herrmann, MD, Chief of the Neuromuscular Division and Vice Chair for Strategic Initiatives in the Department of Neurology at the University of Rochester Medical Center on Sunday, June 14, 2026.
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 ambulatory adult patients with any genetically confirmed CMT1 or CMT2 subtype across clinical sites in the US and Europe.
The presentation by Dr. Herrmann 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. A similar pattern of improvements in hand grip strength at Day 7 led to significant gains in hand grip strength at Day 21 (p=0.02) and Day 28 (p<0.01). Additional improvement trends were seen in the 9-hole peg test with positive trends for improvement in lower limb outcomes such as the 10 meter walk/run test.
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.
Dr. Herrmann, MD, Chief of the Neuromuscular Division and Vice Chair for Strategic Initiatives in the Department of Neurology at the University of Rochester Medical Center, said: “We are encouraged by the improvements in muscle strength and patient-reported outcomes, as well as the apparent persistence of benefits after treatment discontinuation, which may point to a meaningful effect on neuromuscular function in patients. Taken together, these findings support ignaseclant’s potential as a novel therapeutic approach for CMT and warrant further investigation in larger and longer-term studies.”
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NMD Pharma A/S
Dan Brennan, SVP Corporate and Commercial Strategy
E-mail: contact@nmdpharma.com
ICR Healthcare
Mary-Jane Elliott / Ashley Tapp / Lindsey Neville
E-mail: NMDPharma@icrhealthcare.com
Tel: +44 (0)20 3709 5700
About NMD Pharma
NMD Pharma A/S is a privately held, clinical-stage biotechnology company dedicated to enabling better lives through novel therapies designed to restore skeletal muscle health. The company is advancing a first-in-class platform of small-molecule therapies that selectively target skeletal muscle to address rare neuromuscular diseases as well as broader age-related conditions with significant unmet medical need.
Building on more than 20 years of pioneering research in muscle physiology, NMD Pharma has established a world-leading muscle electrophysiology and translational research platform integrating deep biological insight, proprietary enabling technologies, small-molecule drug discovery capabilities, and robust in vivo pharmacology models. The platform is designed to translate fundamental muscle biology into clinically meaningful and sustained improvements in strength, function, and quality of life for patients.
NMD Pharma’s lead development candidate, ignaseclant (formerly NMD670), is a first-in-class skeletal muscle-targeted therapy currently in clinical development for Charcot-Marie-Tooth disease (CMT), generalized myasthenia gravis (gMG), and spinal muscular atrophy (SMA). The company is also advancing next-generation compounds and exploring additional biologic pathways that may support durable neuromuscular function across a range of disorders that result in a lack of normal muscle function.
Headquartered in Aarhus, Denmark, with operations in the United States, NMD Pharma has raised approximately $180 million from leading life science investors, including Novo Holdings, Lundbeckfonden BioCapital, INKEF Capital, Roche Venture Fund, and Jeito Capital.
For more information, please visit www.nmdpharma.com.
About ignaseclant (NMD670)
Ignaseclant (formerly known as NMD670) is NMD Pharma’s lead development compound and an investigational first-in-class small molecule inhibitor of the skeletal muscle-specific chloride ion channel 1 (CIC-1). By inhibiting ClC-1 ignaseclant enhances skeletal muscle excitability and the muscle’s responsiveness to weak signals, improving neuromuscular transmission, restoring muscle activation and skeletal muscle function.
Preclinical have demonstrated and clinical studies indicate that ClC-1 inhibition can improve muscle strength and functional performance across multiple disease settings. Emerging preclinical findings also suggest that ClC-1 modulation may influence additional biologic pathways relevant to sustained muscle and nerve function, areas that remain under active investigation.
Ignaseclant has previously demonstrated clinically meaningful improvements in a Phase 1b/2a study in generalized myasthenia gravis (gMG) and has shown preclinical evidence of activity in spinal muscular atrophy (SMA), Charcot-Marie-Tooth disease (CMT), and age-related muscle disorders such as sarcopenia. Ignaseclant has received orphan drug designations from the U.S. Food and Drug Administration for the treatment of gMG and CMT.
About the SYNAPSE-CMT Phase 2a Trial
SYNAPSE-CMT was a randomized, double-blind, placebo-controlled Phase 2a clinical trial evaluating the investigational drug ignaseclant in 81 adult patients with genetically confirmed Charcot-Marie-Tooth disease (CMT) types 1 or 2.
The study incorporates a range of validated clinical and functional assessments, including measures of muscle strength, motor performance, walking ability, endurance, balance, and patient reported outcomes, to evaluate the impact of skeletal muscle activation via inhibition of the ClC-1 ion channel in patients with CMT. The trial was designed to explore clinical activity, characterize safety and tolerability, inform dose and endpoint selection, and support advancement into subsequent stages of clinical development.
About Charcot-Marie-Tooth Disease (CMT)
CMT is a rare, inheritable neuromuscular disease affecting approximately one in 2,500 people worldwide, including an estimated 135,000 individuals in the United States, making it one of the most prevalent rare orphan neuromuscular diseases. CMT causes progressive dysfunction of peripheral nerves, leading to sensory loss, debilitating muscle weakness, impaired balance, declining motor control and substantial limitations in daily function that typically worsen over time. There are no FDA-approved treatments for CMT, and patient care is supportive, relying on physical therapy, orthotic devices, and mobility aids. The significant and lifelong burden of CMT underscores the urgent need for new therapeutic approaches, including strategies designed to directly improve muscle strength and function independent of underlying nerve degeneration, such as the mechanism being investigated with ignaseclant.
