NMD Pharma A/S has reported positive top-line results from its phase I/IIa clinical trial of NMD670, recently granted orphan drug designation by the FDA for treatment of myasthenia gravis (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. Electrophysiological endpoints demonstrated target engagement and restoration of neuromuscular transmission associated with increases in muscle strength and function.
The trial took place at the Centre for Human Drug Research (CHDR) in Leiden in the Netherlands.
Geert Jan Groeneveld, chief scientific officer and chief medical officer at the Centre for Human Drug Research (CHDR) and the principal investigator of the trial, said: “I would like to thank the study participants, their families and caregivers for participating in the NMD670 clinical study. These positive clinical data provide valuable early insights into the potential of ClC-1 inhibition to enhance neuromuscular transmission for myasthenia gravis and other neuromuscular diseases.”
Thomas Holm Pedersen, chief executive officer of NMD Pharma, said: “These trial results represent an important milestone for NMD Pharma as they provide the first clinical proof of mechanism for our novel ClC-1 inhibitor treatment approach. With these data we complete an important journey from conceptualizing a new treatment concept to obtaining clinical proof of mechanism, and further establish the relevance of pursuing the development of ClC-1 inhibitors across a range of diseases associated with neuromuscular dysfunction. I would like to thank the NMD Pharma team for their many contributions to making this trial a success and, most importantly, the patients who participated in the study.”
NMD Pharma will publish the full trial data in a peer reviewed journal and present the findings at a leading industry conference over the coming months.