Aarhus, Denmark, 19 December 2024 – NMD Pharma A/S, a clinical-stage biotech company dedicated to developing novel and improved treatments for patients living with neuromuscular diseases, today announced that findings from its Phase 1 trial evaluating the safety, pharmacokinetic (PK) and pharmacodynamic (PD) profile of NMD670 in healthy volunteers were published in the peer-reviewed journal Clinical Pharmacology & Therapeutics. NMD670 is a novel, oral, small molecule inhibitor of the skeletal muscle-specific chloride ion channel ClC-1. By inhibiting the ClC-1 ion channel, NMD670 has been shown to enhance muscle excitability and improve muscle function by alleviating the neuromuscular transmission dysfunction that is present in various neuromuscular diseases.
The aim of the Phase 1 study was to evaluate the safety, PK and PD of single ascending doses (SAD) and multiple-ascending doses (MAD) of NMD670 in healthy male and female volunteers. A total of 87 healthy volunteers were enrolled and randomized to receive single-ascending doses (50-1,600 mg) or multiple-ascending doses (200-600 mg once daily or 400 mg twice daily). The study was performed at the Centre for Human Drug Research in Leiden, the Netherlands.
NMD670 was generally safe and well-tolerated in healthy subjects, with no serious adverse events (SAE). Myotonia occurred at the highest dose levels in the SAD study but resolved fully and spontaneously within hours and was not considered a safety hazard to the volunteers. Importantly, the results of this study indicate pharmacological target engagement at well-tolerated dose levels in healthy subjects because myotonia was an expected exaggerated on-target pharmacological effect.
Thomas Holm Pedersen, Chief Executive Officer of NMD Pharma, commented: “This Phase 1 safety data for our lead asset, NMD670, provides a solid base for translation to patients with neuromuscular diseases. NMD670 is currently being evaluated in three separate Phase 2 clinical trials for neuromuscular diseases with high unmet clinical needs, including a Phase 2 study in adults living with spinal muscular atrophy type 3 and a Phase 2b study in generalized myasthenia gravis patients. Thank you to all the study participants and clinical staff for their continued effort as we look forward to progressing NMD670 to the market.”
The full paper can be accessed here:
Safety, Pharmacokinetics, and Pharmacodynamics of a First‐in‐Class ClC‐1 Inhibitor to Enhance Muscle Excitability: Phase I Randomized Controlled Trial - Ruijs - Clinical Pharmacology & Therapeutics - Wiley Online Library
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Contacts
NMD Pharma A/S
Thomas Holm Pedersen, CEO
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 and preclinical evidence in spinal muscular atrophy, Charcot-Marie Tooth disease and sarcopenia. NMD670 has also been granted orphan-drug designation by the U.S. FDA for treatment of gMG.
