NMD Pharma Doses the First Myasthenia Gravis Patient in a Combined Phase I/IIa Clinical Trial of NMD670

NMD Pharma Doses the First Myasthenia Gravis Patient in aCombined Phase I/IIaClinical Trialof NMD670

Aarhus, Denmark, 30 June 2021 – NMD Pharma A/S, a biotech company leading in the development of novel therapeutics for neuromuscular disorders, today announces that it has dosed the first myasthenia gravis (MG) patient in a combined Phase I/IIa clinical trial of NMD670, NMD Pharma’s lead program.

Thomas Holm Pedersen, Chief Executive Officer of NMD Pharma, said: “We are pleased to announce the dosing of the first myasthenia gravis patient today withour lead development candidate, NMD670. This represents a significant milestonein this study andfor NMD Pharma.”

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 added:“Myasthenia gravis is a debilitating autoimmune disease which can lead to life-threatening symptoms. There is a real unmet need for a therapy to help relieve these symptoms and so it is particularly exciting that this trial has now moved into patients. The pre-clinical data generated by NMD Pharma demonstrates the potential of CIC-1 inhibition in strengthening neuromuscular transmission in myasthenia gravis and I am hopeful that we can replicate those promising pre-clinical results in patients.”

NMD670 is in development to treat the symptoms of MG and is also being evaluated for the treatment of other neuromuscular diseases. It is a first-in-class small molecule inhibitor of the muscle specific chloride ion channel, the ClC-1 ion channel. NMD Pharma has already demonstrated that ClC-1 inhibition can strengthen neuromuscular transmission and ultimately skeletal muscle function and this novel treatment approach has demonstrated compelling preclinical 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. Healthy male and female subjects have received single doses of NMD670 and safety, tolerability and pharmacokinetic parameters have been assessed. The Phase I program now continues with the administration of NMD670 dosed daily for 10 days, and with single doses given to patients with MG. The primary outcome of the study includes a number of safety and tolerability endpoints and the secondary outcomes are a number of pharmacokinetic and pharmacodynamic endpoints.

Further information on the study can be found on the Netherlands Trial Register: https://www.trialregister.nl/trial/8692

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Contacts

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 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 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.

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/.