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NMD Pharma Reports Positive Top-Line Data from a Phase I/IIa Clinical Trial of NMD670 in Patients with Myasthenia Gravis
- Data provides first clinical proof of the mechanism of action of NMD Pharma’s ClC-1 chloride ion channel inhibitor in patients suffering from myasthenia gravis
- NMD670 was safe and well tolerated, with clinically significant improvements in the Quantitative Myasthenia Gravis Score in patients and changes in electrophysiological endpoints that demonstrated target engagement and restoration of neuromuscular transmission
- The clinical validation of ClC-1 inhibition to restore neuromuscular function leading to clinical meaningful effects even with a single dose in patients with myasthenia gravis provides a unique translational platform for NMD Pharma and informs new clinical studies in additional indications characterized by neuromuscular dysfunction
Aarhus, Denmark, 11 October 2022 – NMD Pharma A/S, a clinical stage biotech company developing first-in-class, small molecule ClC-1 inhibitors for neuromuscular disorders, today reported positive top-line results from a Phase I/IIa clinical trial of NMD670, recently granted orphan drug designation by the FDA for treatment of myasthenia gravis (MG).
The Phase I part included single ascending dose and multiple ascending dose in 67 healthy volunteers. The Phase IIa part was a single dose, proof of mechanism, randomized, placebo-controlled, double-blind, three-way cross-over study, investigating the safety and pharmacodynamic effects of NMD670 at two dose levels in 12 patients with MG.
NMD670 was safe and well tolerated in healthy volunteers and patients. Administration of single doses of NMD670 was associated with clinically significant improvements in the Quantitative Myasthenia Gravis Score with up to 50% of the patients meeting pre-specified responder criterion. Electrophysiological endpoints demonstrated target engagement and restoration of neuromuscular transmission that in patients were…
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