NMD670 Shows Signs of Efficacy in Small Phase 1/2a Trial

NMD Pharma's investigational therapy improved muscle function

Marisa Wexler, MS avatar

by Marisa Wexler, MS |

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NMD670 safely improved muscle function and strength in a small clinical trial of people with myasthenia gravis (MG), the therapy’s developer, NMD Pharma, announced.

“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,” Thomas Holm Pedersen, CEO of NMD Pharma, said in a press release.

“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,” Pedersen said.

In MG, an autoimmune attack interferes with the communication between nerve and muscle cells, blocking biochemical signals sent from nerves instructing muscle cells to contract and ultimately leading to muscle weakness.

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NMD670 is designed to improve muscle contraction — and, by extension, reduce muscle weakness — by blocking the activity of a protein called ClC-1 that normally prompts muscles to relax. The experimental oral therapy  recently was granted orphan drug designation by the U.S. Food and Drug Administration (FDA).

NMD conducted a Phase 1/2a clinical trial (CHDR1948), the main goal of which was to assess the safety and tolerability profile of NMD670.

In the Phase 1 portion of the study, one or multiple doses of NMD670 were administered to 67 healthy volunteers in the Netherlands. Then, in the Phase 2 portion, 12 people with generalized MG were assigned randomly to receive one of two doses of NMD670, or a placebo. The study used a one-dose, three-way crossover design, meaning that all participants took one dose of all three possible treatments (both dose levels of NMD670, and a placebo) over the course of the study.

According to NMD, top-line data from the study showed that NMD670 was generally well-tolerated in both healthy volunteers and MG patients. The company also said that assessments of muscles’ electrical activity demonstrated that inhibiting ClC-1 appeared to improve muscle function and strength in MG patients, as intended.

“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,” Pedersen said.

Among MG patients, there were signs of clinical efficacy, with up to half of the patients meeting pre-specified responder criteria on the Quantitative Myasthenia Gravis score, a measure of MG severity. NMD did not provide further details on safety or efficacy outcomes. The company said full results will be published in a peer-reviewed journal and presented at scientific conferences in the coming months.

NMD Pharma is part of the portfolio of life sciences investor Novo Holdings. In a separate press release, Novo partner and NMD board director Morten Graugaard Døssing commented:

“We have been working collaboratively alongside CEO Thomas Holm Pedersen and the NMD Pharma team even before the company was founded and are incredibly proud of the progress the team has made to date,” he said. “I look forward to continuing supporting the company alongside our fellow investors and progress its portfolio of CIC-1 inhibitors for the treatment of patients suffering from rare neuromuscular diseases.”