Aarhus, Denmark — February 12, 2026 — Leads & Copy — NMD Pharma A/S announced that top-line data from its Phase 2a SYNAPSE-CMT study, which evaluated ignaseclant (formerly known as NMD670) in patients 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. The conference will take place in Orlando, Florida, from March 8-11, 2026.
The presentation is scheduled for Wednesday, March 11, 2026, at 2:15 PM ET in Florida 4 at the Hilton Orlando. W. David Arnold, M.D., Executive Director of the NextGen Precision Health Initiative and Professor at the University of Missouri School of Medicine, will present the findings, titled “Efficacy and safety of ignaseclant in adults with Charcot-Marie-Tooth Disease: Top Line Results of a Phase 2a study.”
The Muscular Dystrophy Association (MDA) anticipates over 2,000 participants from more than 40 countries will attend its 2026 Clinical & Scientific Conference, which will focus on “groundbreaking research and clinical achievements.”
Ignaseclant, a first-in-class small molecule inhibitor of the skeletal muscle-specific chloride ion channel 1 (ClC-1), is currently under investigation. There are no FDA-approved therapies available for CMT treatment.
NMD Pharma announced topline results from its Phase 2a study of ignaseclant in Charcot-Marie-Tooth disease Types 1 and 2 on February 3, 2026.
NMD Pharma A/S is a clinical-stage biotechnology company focused on developing therapies to restore skeletal muscle health. The company is advancing a platform of small-molecule therapies that selectively target skeletal muscle to address rare neuromuscular diseases and age-related conditions.
NMD Pharma’s lead candidate, ignaseclant (formerly NMD670), is in clinical development for Charcot-Marie-Tooth disease (CMT), generalized myasthenia gravis (gMG), and spinal muscular atrophy (SMA). The company is also exploring next-generation compounds and additional biologic pathways to support neuromuscular function across various 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 investors, including Novo Holdings, Lundbeckfonden BioCapital, INKEF Capital, Roche Venture Fund, and Jeito Capital.
Ignaseclant (formerly known as NMD670) enhances skeletal muscle excitability and the muscle’s responsiveness to weak signals by inhibiting ClC-1, improving neuromuscular transmission, restoring muscle activation, and skeletal muscle function.
Preclinical and clinical studies have indicated 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.
Ignaseclant has demonstrated 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.
The SYNAPSE-CMT Phase 2a trial was a randomized, double-blind, placebo-controlled study evaluating ignaseclant in 81 adult patients with genetically confirmed Charcot-Marie-Tooth disease (CMT) types 1 or 2.
The study used clinical and functional assessments 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.
Charcot-Marie-Tooth Disease (CMT) is a rare, inheritable neuromuscular disease affecting approximately one in 2,500 people worldwide. CMT causes progressive dysfunction of peripheral nerves, leading to sensory loss, debilitating muscle weakness, impaired balance, declining motor control, and limitations in daily function that worsen over time. There are no FDA-approved treatments for CMT, and patient care is supportive. The burden of CMT underscores the need for new therapeutic approaches.
Source: NMD Pharma A/S
