NMD670 is an experimental oral treatment for myasthenia gravis (MG) being developed by NMD Pharma.
How does NMD670 work?
MG is a rare disease in which a person’s immune system produces antibodies that mistakenly recognize acetylcholine receptors on muscle cells. These receptors convey signals from nerve cells to muscle cells, telling them to contract. The antibodies can alter, block, or destroy the acetylcholine receptors, leading to disease symptoms.
While muscles receive signals to contract through the activation of the acetylcholine receptors, they also receive signals to relax from another channel called ClC-1. NMD670 is a small molecule therapy that inhibits, or blocks, the activity of ClC-1. This could help restore the balance between the excitatory signals telling a muscle to contract, and the inhibitory signals telling it to relax. Early research into the function of ClC-1 supports this idea.
NMD670 in clinical trials
A Phase 1/2a clinical trial (CHDR1948) is recruiting an estimated 79 healthy volunteers and generalized MG patients with muscle weakness and anti-acetylcholine receptor antibodies. The three-part trial, taking place in the Netherlands, will investigate the safety, tolerability, pharmacokinetics (movement in the body), and pharmacodynamics (effects on the body) of NMD670.
In part A, healthy men ages 18 to 45, and healthy women ages 18 to 65 will receive a single oral dose of NMD670 or a placebo. In part B, healthy men ages 18 and 65 will be given multiple doses of NMD670 or a placebo. Adults with MG will receive a single dose of NMD670 or a placebo in part C. Dose levels given successive participants will increase as the study continues, allowing investigators to find the maximum tolerated dose and establish the treatment’s safety.
The trial dosed a first participant in October 2020, and is continuing to enroll at the Centre for Human Drug Research (CHDR) in Leiden, Netherlands. It is expected to conclude in December 2021.
Last updated: Dec. 22, 2020
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