NMD Pharma has reported phase 1/2a data on its myasthenia gravis candidate, furthering its efforts to treat an aspect of the disease that it thinks therapies from AstraZeneca, argenx, Johnson & Johnson and UCB are unable to address.
Myasthenia gravis, a rare long-term condition that causes muscle weakness, is a competitive space. As it stands, AstraZeneca and argenx have treatments on the market, and UCB has two prospects that are near to approval. J&J, which entered the space through its $6.5 billion Momenta Pharmaceuticals buyout, is following closely behind with its phase 3 candidate.
Viewing that scrum of advanced, deep-pocketed companies, NMD has identified a potential gap that has enabled it to sign up Novo Holdings and Roche Venture Fund as investors. The phase 1/2a data reported Tuesday represent an early test of whether the Danish biotech is onto something.
In the phase 1 portion of the trial, investigators enrolled 67 healthy volunteers to receive single and multiple ascending doses of NMD670, a ClC-1 chloride ion channel inhibitor. The phase 2a tested the effects of giving a single dose of NMD670, at one of two dose levels, to 12 myasthenia gravis patients in a placebo-controlled, three-way crossover study.
According to NMD, “administration of single doses of NMD670 was associated with clinically significant improvements in the Quantitative Myasthenia Gravis (QMG) score with up to 50% of the patients meeting pre-specified responder criterion.” In argenx’s phase 3 efgartigimod trial, 63% of patients with acetylcholine receptor-antibody positive disease met the QMG responder definition.
Because NMD670 is designed to restore muscle function rather than focus on autoimmune aspects of the disease like other drugs, NMD CEO Thomas Holm Pedersen has speculated in the recent past that it could be useful as monotherapy in some patients while being “used in combination with other therapies including the newer treatment options” in other people.
In a statement to disclose the phase 1/2a data, the CEO said the results “provide the first clinical proof of mechanism for our novel ClC-1 inhibitor treatment approach,” adding that they “further establish the relevance of pursuing the development of ClC-1 inhibitors across a range of diseases associated with neuromuscular dysfunction.”