Positive results from myasthenia gravis trial

Published Date
Jenny Sharpe

A phase 2 clinical trial has shown that zilucoplan can reduce the severity of generalised myasthenia gravis (MG).

Zilucoplan blocks part of our immune response called the ‘complement system’. This contributes to the destruction of the neuromuscular junction in MG. Zilucoplan can be administered at home, like an insulin injection.

The trial, which was carried out at sites in the US and Canada by Ra Pharmaceuticals, included 44 people with generalised myasthenia gravis. Participants were split into three groups; one high-dose group, one low-dose and one placebo. After 12 weeks of daily injections, the higher dose group had a significantly improved disease severity score compared to the placebo group.

Nancy Law, Chief Executive Officer of the Myasthenia Gravis Foundation of America (MGFA) said:

This represents a potential breakthrough for all patients who are struggling every day with their MG, and seeking more effective and convenient treatment options. This unmet need was highlighted in a recent survey of patients from the MGFA database, where we learned that a majority of patients with MG are not satisfied with their current treatments and are interested in effective, at home, self-injectable treatment options.

Based on these results, Ra Pharmaceuticals will test the higher dose of zilucoplan in a larger phase 3 trial. The company plans to confirm the design of this trial with the FDA and other regulatory agencies by the first quarter of 2019.

For further information, read Ra Pharmaceuticals’ press release.

If you have any questions about this news story or any other myasthenia gravis research, please contact the MDUK Research Line on 020 7803 4813 or email research@musculardystrophyuk.org.

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