Aarhus, Denmark, 12 February 2026 - NMD Pharma A/S, a clinical-stage biotechnology company dedicated to developing novel therapies to restore skeletal muscle health, today announced that top-line data results from its Phase 2a SYNAPSE-CMT study evaluating ignaseclant (formerly known as NMD670) in patients living with Charcot-Marie-Tooth disease (CMT) types 1 or 2 has been accepted for a late-breaking oral presentation at the 2026 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference being held in Orlando, Florida from March 8-11, 2026.
Presentation details are below:
Date: Wednesday, March 11, 2026
Start Time: 2:15 PM ET
Location: Hilton Orlando, Orlando, FL
Room Name: Florida 4
Presentation Name: Efficacy and safety of ignaseclant in adults with Charcot-Marie-Tooth Disease: Top Line Results of a Phase 2a study
Presenter: W. David Arnold, M.D., Executive Director of the NextGen Precision Health Initiative,
Professor of Physical Medicine and Rehabilitation, Co-Director of Tom and Anne Smith MD/PhD Program, University of Missouri School of Medicine
The Muscular Dystrophy Association (MDA) has announced the agenda for its 2026 MDA Clinical & Scientific Conference and the focus for this year's Conference, the nonprofit says, is on "groundbreaking research and clinical achievements." Over 2,000 participants from over 40 countries are expected to attend.
Ignaseclant is an investigational first-in-class small molecule inhibitor of the skeletal muscle-specific chloride ion channel 1 (CIC-1). There are currently no FDA-approved therapies for the treatment of CMT.
On 3 February 2026, NMD Pharma announced topline results from its Phase 2a study of ignaseclant in Charcot-Marie-Tooth disease Types 1 and 2.
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Contact
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 15 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 and clinical studies have demonstrated 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.
