NMD1343 is a novel small molecule inhibitor of the muscle-specific chloride ion channel, the ClC-1 ion channel, and NMD Pharma’s second clinical development candidate

AARHUS, Denmark I May 23, 2023 I NMD Pharma A/S, a clinical-stage biotech company developing first-in-class, small molecule ClC-1 inhibitors for neuromuscular disorders, today announces that the first healthy volunteers have been dosed at the study site in London, UK.

The Phase I clinical trial is a randomized, double-blind, placebo-controlled, single and multiple ascending dose study to evaluate the safety, tolerability, and pharmacokinetics of oral NMD1343 in healthy male and female volunteers. All participants will be screened up to four weeks prior to dosing and each cohort of eight healthy volunteers will be randomized to receive, initially, either a single dose of NMD1343 or a matching placebo. The study was reviewed and approved by the Medicines & Healthcare products Regulatory Agency (MHRA).

Jorge A. Quiroz, SVP and Chief Medical Officer NMD Pharma commented: “It is an exciting time at NMD Pharma, now launching our second ClC-1 inhibitor NMD1343 that, with a differentiated profile compared to other development candidates in our pipeline, could be beneficial for patients with a range of rare neuromuscular diseases.”

Thomas Holm Pedersen, Chief Executive Officer of NMD Pharma, said: “We are very pleased to announce that the first healthy subject has been dosed in this Phase I study with NMD1343. NMD Pharma is focused on the development of novel first-in-class therapies for severe neuromuscular disorders, and the initiation of this study expands our clinical pipeline and our vision of delivering life-transforming therapies for those patients and their families.”

In addition to NMD1343, NMD Pharma is also developing NMD670, a first-in-class small molecule inhibitor of the muscle specific chloride ion channel, the ClC-1 ion channel. NMD Pharma has pre-clinically demonstrated that ClC-1 inhibition can enhance neuromuscular transmission and, ultimately, skeletal muscle function and NMD670 has already demonstrated positive results in a Phase I and proof of mechanism study in patients with myasthenia gravis confirming safety, tolerability and initial efficacy data in subjects suffering from neuromuscular disorders.

About NMD Pharma
NMD Pharma A/S, is a clinical-stage biotech company leading in the development of novel first-in-class therapies for severe neuromuscular disorders. The Company was incorporated as a spin-out 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 its in-depth know-how of muscle physiology and muscular disorders and is developing a pipeline of ClC-1 inhibitors for the treatment of patients with neuromuscular disorders including myasthenia gravis, spinal muscular atrophy and Charcot-Marie Tooth. Positive top line data reported from a Phase I and proof of mechanism study of lead program NMD670 in myasthenia gravis has provided clinical validation of ClC-1 inhibition to restore neuromuscular function. NMD Pharma has 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/.