NMD Pharma Receives Approval to Start a combined Phase I/IIa Clinical Trial of NMD670 for the Treatment of Symptoms of Myasthenia Gravis

Aarhus, Denmark, 14 July 2020 – NMD Pharma A/S, a biotech company leading in the development of novel therapeutics for neuromuscular disorders, today announces that it has received approval from the Dutch ethics committee and regulatory authorities to advance its development candidate, NMD670, into clinical trials as a novel treatment for the symptoms of myasthenia gravis (MG).

NMD670 is a first-in-class small molecule inhibitor of the muscle specific chloride ion channel, the ClC-1 ion channel. NMD Pharma has demonstrated that ClC-1 inhibition can strengthen neuromuscular transmission and ultimately skeletal muscle function and this novel treatment approach has demonstrated compelling preclinical safety and efficacy data for MG.

The combined Phase I/IIa clinical trial is a randomized, double-blind, placebo controlled, single and multiple dose escalation study designed to assess the safety, tolerability, pharmacokinetics and pharmacodynamics of NMD670 in male and female healthy subjects and patients with MG.  The study will take place at the Centre for Human Drug Research (CHDR) in Leiden in the Netherlands and the first dosing is expected to take place over the next few months.

Thomas Holm Pedersen, Chief Executive Office of NMD Pharma, said: “We are very pleased to receive this approval to start our first clinical trial in humans with NMD670. MG is a rare autoimmune disease and there is a need for new, differentiated, treatment options for patients who are not adequately managed by standard of care therapies. Our pre-clinical work has already shown the potential benefit that CIC-1 inhibition can have, and so we are excited to start this trial over the next couple of months. I would like to take this opportunity to thank the team at NMD Pharma for all their hard work and dedication which has allowed the Company to reach this important milestone.”

Further information on the study can be found on the Netherlands Trial Register:

https://www.trialregister.nl/trial/8692

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 seed financing from Novo Seeds, Lundbeckfonden Emerge and Capnova in 2016, and in 2018 raised a €38 million Series A financing, led by new investor INKEF Capital, together with new investor Roche Venture Fund and existing investors Novo Seeds and Lundbeckfonden Emerge.

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 muscle specific chloride ion channel, the ClC-1 ion channel. NMD Pharma has demonstrated that ClC-1 inhibition can strengthen neuromuscular transmission and ultimately skeletal muscle function and this novel treatment approach has demonstrated compelling preclinical safety and efficacy data for 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 most commonly affects the muscles that control the eyes and eyelids, facial expressions, chewing, swallowing and speaking but it can affect most parts of the body. More than 85% of people with MG progress to generalized MG (gMG) within 18 months and in more life-threatening cases, MG can affect the muscles responsible for breathing. Patients with confirmed acetylcholine receptor (AChR) antibodies account for 80-90% of the total gMG population. 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.