According to a story from Markets Insider, the biotechnology company NMD Pharma A/S has recently announced that the Dutch ethics committee has approved the company’s plan for a combined phase 1/2a clinical trial that will test its experimental therapy NMD670 as a treatment for myasthenia gravis (MG). NMD focuses on the development of treatments for severe neuromuscular disorders.
About Myasthenia Gravis
Myasthenia gravis is a long term autoimmune disease that is most characterized by weakness of the skeletal muscles which can affect a patient’s ability to move and breathe. As an autoimmune disease, myasthenia gravis is ultimately the result of abnormal immune system behavior in which the system attacks normal body tissue. In this disease, the immune system produces antibodies that inhibit regular neuromuscular function. Symptoms of the illness include muscle weakness that worsens after activity and gets better after resting. This weakness may affect the face, eyes, neck, breathing, and limbs. It may cause speech and swallowing difficulties, eyelid drooping, shortness of breath, and blurred vision. In severe cases, patients may require breathing assistance with a ventilator. Treatment options for myasthenia gravis include immunosuppressive medications, thymus gland removal, IVIG, and plasmapharesis. Most patients can effectively manage the condition with treatment. To learn more about myasthenia gravis, click here.
NMD670 is classified as an inhibitor of the ClC-1 ion channel and is the first medication of this kind. This mechanism of action can improve skeletal muscle function and improve neuromuscular signal transmission for patients living with the disease according to preclinical research findings.
About the Trial
The clinical trial will include a single and multiple dose escalation design that is intended to evaluate pharmacodynamics, pharmacokinetics, tolerability, and safety of NMD670. As a combined phase 1/2a trial, it will include both healthy participants and myasthenia gravis patients. The trial operators expect to begin dosing in the next few months.
While there are therapies available for myasthenia gravis, there is a great disparity in patient response with a significant number of patients having trouble maintaining control of symptoms. Therefore, the availability of a new treatment in the not-too-distant future would be a significant development.