NMD Pharma Starts Phase 2b Trial of NMD670 in Myasthenia Gravis

NMD Pharma, a biotechnology company focused on advancing treatments for neuromuscular disorders, announced the commencement of a Phase 2b clinical trial for its drug candidate NMD670 in patients with generalized myasthenia gravis (gMG). The trial, following FDA's IND clearance in March 2024, marks a significant step in the development of this novel therapy.

Clinical Trial Overview

The first patient in the United States has been dosed under the supervision of Dr. Marc Feinberg at SFM Clinical Research in Boca Raton, Florida. The Phase 2b trial is designed as a dose range-finding, double-blind, placebo-controlled study. It will evaluate the effectiveness of NMD670, an oral small molecule that inhibits the ClC-1 chloride ion channel specifically in skeletal muscles. The study will span 21 days and include gMG patients who test positive for either anti-acetylcholine receptor (AChR) or anti-muscle-specific tyrosine kinase (MuSK) antibodies. Researchers will assess various endpoints, including changes in the Quantitative Myasthenia Gravis Total Score and Myasthenia Gravis Activities of Living.

Expert Opinions

Jorge A. Quiroz, Executive Vice President and Chief Medical Officer of NMD Pharma, highlighted the criticality of addressing the persistent symptoms experienced by gMG patients despite available therapies. Quiroz noted that NMD670, being a first-in-class treatment, aims to enhance muscle power and endurance, thus managing both persistent and fluctuating symptoms of gMG.

Samantha Masterson, President and CEO of the Myasthenia Gravis Foundation of America (MGFA), emphasized the ongoing unmet needs of gMG patients. She expressed optimism that NMD670 could offer additional strength and endurance, improving the quality of life for patients. The MGFA is closely monitoring the trial’s progress, hoping that the promising early results translate into substantial clinical benefits.

Background on NMD670

NMD670 is a pioneering oral small molecule designed to inhibit the ClC-1 chloride ion channel in skeletal muscles. Earlier phases of clinical trials have already shown positive results, establishing the clinical proof-of-mechanism for ClC-1 inhibitors. Published data in "Science Translational Medicine" affirmed the safety and tolerability of a single dose of NMD670 in patients with gMG.

Participation and Further Information

The study encourages participation from gMG patients who are AChR or MuSK antibody positive in both the US and Europe. Details about active investigational sites can be found on ClinicalTrials.gov.

About NMD Pharma

NMD Pharma A/S, established on 15 years of muscle physiology research, is a clinical-stage biotech company focused on developing treatments for severe neuromuscular disorders. The company leverages a unique muscle electrophysiology platform to discover and develop small molecule modulators targeting the skeletal muscle chloride ion channel (ClC-1). NMD Pharma has garnered considerable investment backing, securing approximately €155 million from notable investors such as Novo Holdings, Lundbeckfonden BioCapital, and others.

Understanding Myasthenia Gravis

Myasthenia gravis (MG) is a rare autoimmune disorder characterized by muscle weakness due to disrupted nerve-muscle communication caused by IgG antibodies. It often affects eye and facial muscles initially but can progress to involve most skeletal muscles. Life-threatening complications may arise, particularly when respiratory muscles are affected. Despite existing treatments targeting the immune system, many patients continue to suffer from severe and unpredictable symptoms. The incidence of generalized myasthenia gravis affects about 100,000 individuals in the European Union, 65,000 in the United States, and 20,000 in Japan.

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