NMD Pharma presents preclinical data highlighting increased innervation of skeletal muscle with ClC-1 ion channel inhibition treatment in neuromuscular disease model

• Company presented clinical and preclinical data at the MDA Conference last week in Dallas, Texas
• Oral presentation included data from MuSK-MG rat model depicting increases in muscle function and improvements in the structural integrity of the neuromuscular junction and innervation of skeletal muscle
• Other poster presentations included data from Phase 2a clinical study in myasthenia gravis and status updates for the three ongoing Phase 2 trials with its novel, oral, skeletal-muscle-targeted drug therapy
  
  

Aarhus, Denmark, 25 March 2025 – NMD Pharma A/S, a clinical-stage biotech company dedicated to developing novel and improved treatments for patients living with neuromuscular diseases, today announces that it has presented data on its novel, orally bioavailable skeletal muscle specific chloride ion channel (ClC-1) inhibitors in one invited oral presentation and four poster presentations at the Muscular Dystrophy Association (MDA) Clinical and Scientific Conference, Dallas, Texas from March 16-19 2025.

Martin Brandhøj Skov, Senior Innovation Manager at NMD Pharma, delivered the oral presentation on the positive effects of NMD Pharma’s orally bioavailable small molecule ClC-1 inhibitor in a muscle-specific kinase (MuSK) myasthenia gravis (MG) rat model on improved disease symptoms at the conference. The study showed that NMD Pharma’s small molecule ClC-1 inhibitor had a positive effect on electromyography (EMG), leading to higher body weight, increased survival, and increased muscle and respiratory function. In addition to the alleviation of disease symptoms and improved function, the structural integrity of the neuromuscular junction was improved, with animals treated with a ClC-1 inhibitor demonstrating an increase in the number of fully innervated muscle fibers from 21% (vehicle) to 46% (treated with ClC-1 inhibitor).

This unanticipated finding highlights a potential disease-modifying mechanism of ClC-1 inhibition in neuromuscular disorders, potentially involving facilitation of reinnervation or attenuation of denervation processes. NMD Pharma intends to further characterize these effects in additional preclinical models and integrate these mechanistic insights into the design of future clinical trials evaluating ClC-1 inhibitors across a spectrum of neuromuscular diseases with limited therapeutic options.

Thomas Holm Pedersen, Chief Executive Officer of NMD Pharma commented, “These presentations highlight NMD Pharma’s scientific leadership in neuromuscular research, an important area implicated in many diseases in which patients have limited treatment options. We look forward to continuing to translate these new scientific insights into therapeutics to improve the quality of life of many patients.”

Further details can be found below, and the posters can be accessed by registered conference participants on the MDA Clinical and Scientific Conference website.

Oral presentation

Session: Molecular Insights into NMDs
Title: Skeletal Muscle Targeted ClC-1 Ion Channel Inhibition Improves Skeletal Muscle Function and Respiratory Function in a Rat Model of MuSK-MG
Presenter/Authors: J. J. Morgen M. Skals, M. B. Skov, N. Huus, N. M. Kelly, M. Broch-Lips, T. K. Petersen

Poster presentations

Session: MDA Poster sessions
Title: Skeletal Muscle Targeted ClC-1 Ion Channel Inhibition Improves Skeletal Muscle Function and Respiratory Function in a Rat Model of MuSK-MG   
Authors: J. J. Morgen, M. Skals, M. B. Skov, N. Huus, N. M. Kelly, M. Broch-Lips, T. K. Petersen

Session: MDA Poster sessions
Title:    Investigation of the Effect of Combinatorial Treatment with a ClC-1 Inhibitor and a Rat Specific FcRn Blocker in a Preclinical Model of Myasthenia Gravis.
Authors: J. J. Morgen, M. Skals, M. B. Skov, N. Huus, N. M. Kelly, M. Broch-Lips, T. K. Petersen

Session: MDA Poster sessions
Title: Status Update of Phase 2 Trials in SMA, CMT and MG with NMD670 - a Novel, Oral Skeletal-Muscle-Targeted Therapy
Authors: V. Kiyasova, T. S. Grønnebæk, C. Cornwall, T. Breuer, J. Gupte, C. Sampson, T. Gidaro, T. H. Pedersen

Session: MDA Poster sessions
Title: Post-hoc Responder Analyses from the Proof-of-mechanism Study of NMD670
in Patients with Myasthenia Gravis
Authors: V. Kiyasova, T. S. Grønnebæk, T. Breuer, J. Gupte, C. Sampson, T. Gidaro, T. H. Pedersen

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Contacts

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 clinical-stage biotech company developing a first-in-class platform of small molecule therapies selectively targeting the skeletal muscle chloride ion channel (ClC-1) for the treatment of rare neuromuscular disorders and age-related neuromuscular diseases with high levels of patient unmet. The Company was founded on more than 15 years of muscle physiology research with a focus on regulation of skeletal muscle excitability under physical activity. NMD Pharma has built a world-leading muscle electrophysiology platform leveraging the in-depth know-how of muscle physiology and muscular disorders, small molecule modulators, enabling technologies and tools as well as in vivo pharmacology models for discovering and developing proprietary modulators of neuromuscular function. The Company has built significant clinical and development expertise as its programmes have progressed through the clinic. NMD Pharma has raised ~€155 million from investors including Novo Holdings, Lundbeckfonden BioCapital, INKEF Capital, Roche Venture Fund, and Jeito Capital. Find out more about us online at http://www.nmdpharma.com.

About NMD670
NMD670 is NMD Pharma’s lead development program. It is a first-in-class small molecule inhibitor of the skeletal muscle specific chloride ion channel 1 (CIC-1). NMD Pharma has demonstrated that CIC-1 inhibition amplifies the muscle’s responsiveness to weak signals, improving neuromuscular transmission and restores skeletal muscle function. This novel treatment approach has resulted in clinical evidence of CIC-1 inhibition in myasthenia gravis (MG) and preclinical evidence in spinal muscular atrophy, Charcot-Marie Tooth (CMT) disease and sarcopenia. NMD670 has also been granted orphan-drug designation by the U.S. FDA for treatment of generalised MG and CMT.