NMD Pharma Receives IND Clearance to Start a Phase II Clinical Trial of NMD670 for the Treatment of Symptoms of Spinal Muscular Atrophy Type 3

NMD Pharma Receives IND Clearance to Start a Phase II Clinical Trial of NMD670 for the Treatment of Symptoms of Spinal Muscular Atrophy Type 3

Aarhus, Denmark, 14 December 2022 – NMD Pharma A/S, a clinical stage biotech company developing first-in-class, small molecule ClC-1 inhibitors for neuromuscular disorders, today announces that it has received clearance for its Investigational New Drug (IND) application from the US Food and Drug Administration (FDA) to advance NMD670 into a Phase II clinical study in patients of spinal muscular atrophy (SMA) Type 3.

The Phase II clinical trial is a randomized, double-blind, placebo-controlled, 2-way crossover study to evaluate the efficacy, safety, and tolerability of NMD670 in ambulatory adult patients with SMA Type 3. The study is an international multicenter study and will include sites in North America and Europe with first dosing of patients expected to take place in Q1 2023.

NMD670 is 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. In October, NMD announced positive topline data from NMD670 single dose in a Phase I/IIa study in patients with myasthenia gravis (MG) confirming safety, tolerability and initial efficacy data in subjects suffering from neuromuscular disorders. Based on these preclinical and clinical data, it is expected that this novel approach could be beneficial in the treatment of patients suffering from SMA.

Thomas Holm Pedersen, Chief Executive Officer of NMD Pharma, said: “Receiving IND clearance to start our first clinical trial in SMA patients with NMD670 reflects the progress of NMD Pharma’s clinical development into new indications and geographies and comes only two months after we reported successful Phase I/IIa data on NMD670 in myasthenia gravis. SMA is a rare disease and despite recent treatment advances, there is still a substantial unmet medical need to alleviate weakness and fatigue in these patients.”



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 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/II clinical trial 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/.

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), spinal muscular atrophy (SMA) and a range of other neuromuscular disorders as well as clinical efficacy in a Phase IIa clinical proof of mechanism study in MG. NMD670 has recently been granted orphan-drug designation (ODD) by the U.S. Food and Drug Administration (FDA) for treatment of MG.

About Spinal Muscular Atrophy
Spinal muscular atrophy (SMA) is a group of genetic diseases characterised by progressive destruction of nerve cells that control essential skeletal muscle activity leading to weakness and fatigue. SMA is the most common genetic cause of mortality in infants with an incidence of 1:5000 to 1:10.000 new-born babies. The most common form of SMA is caused by defects in both copies of the survival motor neuron 1 gene (SMN1) on chromosome 5q. Chromosome 5q-related SMA, is commonly classified into types 1 to 4 (sometimes 0 to 4) dependent on age of onset. Type 1 (0) is the most severe form of the disease with symptoms appearing at birth or in infancy (before birth, type 0).

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