The
Myasthenia Gravis Foundation of America (MGFA) recently conducted a survey which found that most
myasthenia gravis patients are not satisfied with their current treatment regime. These patients wanted a therapeutic option that they could administer in their own home by themselves.
Ra Pharmaceuticals has just brought this patient population closer to to this goal. Results from a recent Phase 2 trial showed statistically significant efficacy for the drug zilucoplan in MG treatment, and the best part is- it could be administered at home.
About MG
MG is an autoimmune condition characterized by weak muscles and fatigue. It affects the limbs, respiratory muscles, and even movement of the eye. 60,000 people in the United States have the generalized form of the disease. There is also a severe form of the condition.
Typically, patients become more weak as they use their muscles, with periods of recovery when they rest. It’s considered a chronic disease.
The Trial
Zilucoplan is a synthetic, macrocyclic peptide which works to bind C5, prohibiting it from binding to C5a or C5b. It also interferes with the interaction of C5b and C6. For MG, it is designed to be administered subcutaneously once every day. The patient would be able to administer zilucoplan by themselves easily.
To investigate the tolerability, efficacy and safety of Zilucoplan, a Phase 2 trail was conducted over 12 weeks. The results were statistically significant for both refractory and non-refractory MG patients.
“Zilucoplan has the potential to become the first convenient, self-administered, complement inhibitor expanding access for patients living with this chronic, debilitating, neuromuscular disease.”
The trial included 44 patients from both the United States and Canada. Participants were randomized and received either a .3 mg/kg dose of zilucoplan, a .1 mg/kg dose of zilucoplan or a placebo.
For all patients, there were few adverse events during the trial. Most of the events that did take place were mild and researchers did not consider them to be occur as a result of zilucoplan. Overall the drug showed safety and tolerability. These results were consistent with earlier Phase 1 and Phase 2 trials.
Looking Forward
Ra Pharma is planning a Phase 3 trial which should begin in the first half of 2019. This trial will specifically focus on a .3 mg/kg dose of the drug.
The goal is to ultimately improve the quality of life for MG patients, and this study is another step in the right direction. Hopefully, the coming Phase 3 trial will show the same success as this recent study and zilucoplan will gain FDA approval for the treatment of MG soon.
This type of self-administered treatment could truly be life-changing for patients.
You can read more about this study and Ra Pharmaceuticals’ research for myasthenia gravis here.
