NMD Pharma late breaking oral presentation at the MDA Conference with top-line safety and efficacy data from SYNAPSE-CMT, an exploratory Phase 2a clinical study of ignaseclant in Charcot-Marie-Tooth disease Types 1 & 2

• NMD Pharma will also present an update on the ongoing SYNAPSE-MG Phase 2b clinical trial of ignaseclant in generalized myasthenia gravis
  
  

Aarhus, Denmark, 10 March 2026 – NMD Pharma A/S, a clinical-stage biotechnology company dedicated to developing novel therapies to restore skeletal muscle health, announces today a poster and late-breaking oral presentation describing safety and efficacy data 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 at the 2026 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference being held in Orlando, Florida from March 8-11, 2026. The oral late-breaking presentation will be given by Dr. David Arnold, 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, and SYNAPSE-CMT investigator.

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 adult patients with any genetically confirmed CMT1 or CMT2 subtype across clinical sites in the US and Europe.

Important SYNAPSE-CMT Phase 2a clinical trial study findings
The presentation by Dr. Arnold will show the benefit observed in functional improvements with ignaseclant on handgrip strength, 9-hole peg test, and patient-reported CMT-Health Index (CMT-HI) scale. Dr. Arnold will further show the observations on lower limb function including dynamometry, 10MWR, and 6 minute walk test.

Improvement with ignaseclant versus placebo on the multi-item CMT-Functional Outcome Measure Scale (CMT-FOM) clinical assessment scale in patients with CMT will be presented.

Across these endpoints improvements were seen with ignaseclant treatment after 7 days, increased further after 21 days, and improvements were sustained at day 28. The sustained effect align with recent pre-clinical data demonstrating ClC-1 inhibition facilitating reinnervation and/or attenuating denervation in animal models of CMT2 and MuSK+ antibody myasthenia gravis.

Ignaseclant was safe and well tolerated over 21 days. The incidence of any treatment-emergent adverse events was higher with ignaseclant than on placebo (71.4% vs. 60.5%), but all adverse events with ignaseclant were mild to moderate (i.e. Grade ≤3), and none led to study drug discontinuation. No serious adverse events were observed in the ignaseclant group.

“What is particularly encouraging about this data is the consistency of the effects with ignaseclant treatment observed across multiple measures of muscle strength and function in such a short study,” said W. David Arnold, M.D., Executive Director of the NextGen Precision Health Initiative and Professor of Physical Medicine and Rehabilitation at the University of Missouri School of Medicine. “Taken together, the totality of the data provides a strong rationale to further investigate ignaseclant as a potential new treatment approach for people living with Charcot-Marie-Tooth disease.”

Timing of the oral presentation are as follows:

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

See the full poster presentation online here.

SYNAPSE-MG Phase 2b clinical trial update
In addition, NMD Pharma will present an update on the ongoing SYNAPSE-MG study evaluating ignaseclant in generalized myasthenia gravis (gMG) patients who are AChR or MuSK antibody positive in an oral presentation, details below:

Date: Wednesday, March 11, 2026
Start Time: 9:00 AM ET
Location: Hilton Orlando, Orlando, FL
Room Name: Florida 4
Presentation Name: Ignaseclant (NMD670) for Generalized Myasthenia Gravis - the SYNAPSE-MG study status update
Presenter: Thomas Holm Pedersen, PhD

See the full poster presentation online here.

Patients living with gMG who are AChR or MuSK antibody positive in the US and Europe are encouraged to participate in the ongoing study. Further information and a list of currently active investigational sites can be obtained via email at contact@nmdpharma.com.

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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.