NMD Pharma Reports Positive Top-Line Data from a Phase I/IIa Clinical Trial of NMD670 in Patients with Myasthenia Gravis

NMD Pharma Reports Positive Top-Line Data from a Phase I/IIa Clinical Trial of NMD670 in Patients with Myasthenia Gravis

  • Data provides first clinical proof of the mechanism of action of NMD Pharma’s ClC-1 chloride ion channel inhibitor in patients suffering from myasthenia gravis
  • NMD670 was safe and well tolerated, with clinically significant improvements in the Quantitative Myasthenia Gravis Score in patients and changes in electrophysiological endpoints that demonstrated target engagement and restoration of neuromuscular transmission
  • The clinical validation of ClC-1 inhibition to restore neuromuscular function leading to clinical meaningful effects even with a single dose in patients with myasthenia gravis provides a unique translational platform for NMD Pharma and informs new clinical studies in additional indications characterized by neuromuscular dysfunction

Aarhus, Denmark, 11 October 2022 – NMD Pharma A/S, a clinical stage biotech company developing first-in-class, small molecule ClC-1 inhibitors for neuromuscular disorders, today reported positive top-line results from a Phase I/IIa clinical trial of NMD670, recently granted orphan drug designation by the FDA for treatment of myasthenia gravis (MG).

The Phase I part included single ascending dose and multiple ascending dose in 67 healthy volunteers. The Phase IIa part was a single dose, proof of mechanism, randomized, placebo-controlled, double-blind, three-way cross-over study, investigating the safety and pharmacodynamic effects of NMD670 at two dose levels in 12 patients with MG.

NMD670 was safe and well tolerated in healthy volunteers and patients. Administration of single doses of NMD670 was associated with clinically significant improvements in the Quantitative Myasthenia Gravis Score with up to 50% of the patients meeting pre-specified responder criterion. Electrophysiological endpoints demonstrated target engagement and restoration of neuromuscular transmission that in patients were associated with increases in muscle strength and function.

The trial took place at the Centre for Human Drug Research (CHDR) in Leiden in the Netherlands. Further information on the study can be found on the International Clinical Trials Registry: https://trialsearch.who.int/Trial2.aspx?TrialID=NL8692.

Dr. Geert Jan Groeneveld, Chief Scientific Officer and Chief Medical Officer at the Centre for Human Drug Research (CHDR) and the Principal Investigator of the trial said: “I would like to thank the study participants, their families and caregivers for participating in the NMD670 clinical study. These positive clinical data provide valuable early insights into the potential of ClC-1 inhibition to enhance neuromuscular transmission for myasthenia gravis and other neuromuscular diseases.”

Thomas Holm Pedersen, Chief Executive Officer of NMD Pharma, said: “These trial results represent an important milestone for NMD Pharma as they provide the first clinical proof of mechanism for our novel ClC-1 inhibitor treatment approach. With these data we complete an important journey from conceptualizing a new treatment concept to obtaining clinical proof of mechanism, and further establish the relevance of pursuing the development of ClC-1 inhibitors across a range of diseases associated with neuromuscular dysfunction. I would like to thank the NMD Pharma team for their many contributions to making this trial a success and, most importantly, the patients who participated in the study.”

NMD Pharma will publish the full trial data in a peer reviewed journal and present the findings at a leading industry conference over the coming months.



NMD Pharma A/S
Thomas Holm Pedersen, CEO
E-mail: contact@nmdpharma.com

Consilium Strategic Communications
Mary-Jane Elliott / Ashley Tapp / Lindsey Neville
E-mail: NMDPharma@consilium-comms.com
Tel: +44 (0)20 3709 5700

About NMD Pharma
NMD Pharma A/S, is a private biotech company leading in the development of novel first-in-class therapies for severe neuromuscular disorders. The Company was incorporated as a spin-off from Aarhus University, Denmark in 2015 and 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. NMD Pharma received initial seed financing in 2016 and have since raised ~€80 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 (ClC-1). NMD Pharma has demonstrated that ClC-1 inhibition enhances neuromuscular transmission and restores skeletal muscle function, and this novel treatment approach has demonstrated compelling preclinical efficacy data in animal models of myasthenia gravis (MG) and a range of other neuromuscular disorders. NMD670 has recently been granted orphan-drug designation (ODD) by the U.S. Food and Drug Administration (FDA) for treatment of MG.

About Myasthenia Gravis (MG)
MG is a rare and chronic autoimmune disease where IgG antibodies disrupt communication between nerves and muscles causing debilitating and potentially life-threatening muscle weakness. It commonly affects the muscles that control the eyes and eyelids, facial expressions, chewing, swallowing, and speaking but in most patients it eventually impacts most skeletal muscles. More than 85% of people with MG progress to generalized MG (gMG) within 18 months and it can be life-threatening when affecting the muscles responsible for breathing. There are approximately 100,000 people in the European Union, 65,000 people in the United States and 20,000 people in Japan living with the disease.

About the Centre for Human Drug Research
The Centre for Human Drug Research (CHDR) is an independent institute that specialises in cutting-edge early-stage clinical drug research. Combining innovative methods and technologies, state-of-the-art facilities, and talented, motivated researchers helps CHDR maximise their clients’ success. CHDR’s overall mission is to improve the drug development process by collecting as much information as possible regarding a candidate drug in the early phases of development. This information helps clients make informed decisions regarding the course of clinical development for their product. CHDR places the highest priority on their subjects’ comfort and safety, and they play an active role in helping to educate the medical and clinical research communities. Find out more about the CHDR online at https://chdr.nl/.

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