NDM Pharma provides POC for NMD670

Researchers at NDM Pharma A/S and Aarhus University have demonstrated that their oral CIC-1 blocker NMD670 restores crosstalk between neurons and muscles in Myasthenia gravis.


In Science Translational Medicine the team headed by Thomas Holm Pedersen from NDM Pharma A/S report that they have conducted animal studies and a clinical trial to prove that blocking the Chloride ion Channel CIC-1 could address the need for treatments that can reverse muscle decline in people with myasthenia gravis.

Myasthenia gravis weakens muscles by interrupting the electrical signals that permit neurons to communicate with muscle fibers, leading to severe muscle weakness and fatigue. Patients usually take combinations of drugs that boost muscle signal transmission and target the immune system, but there is no cure that can completely restore muscle function in day-to-day life.

Martin Skov and colleagues now provided data from a new treatment option dubbed NMD670 that supported muscle function in rat models of myasthenia gravis. The compound works by inhibiting CIC-1. This sensitizes the muscle to commands from motor neurons and partially restores neuromuscular communication, leading to improved grip strength and movement in the rodents.

Moving to humans, the researchers then administered the drug to 12 patients with mild myasthenia gravis in a randomised clinical trial, where the compound showed a good safety profile. It also led to improvements in a clinical score of myasthenia gravis, including by augmenting grip strength. Researchers have noted similar deficits in signal transmission in other neuromuscular diseases, indicating that CIC-1 inhibitors could also prove valuable beyond myasthenia gravis.


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