Ra Pharmaceuticals, which is now enrolling patients with generalized myasthenia gravis into a Phase 2 clinical trial, joined millions of health care advocates worldwide for the 11th annual Rare Disease Day, held to raise awareness and public understanding of rare diseases.
The 2018 Rare Disease Day had as a slogan “Show Your Rare, Show You Care,” and focused specifically on research being developed for rare diseases.
RA Pharma’s RA101495 is a potential therapy for generalized myasthenia gravis and for paroxysmal nocturnal hemoglobinuria, a serious and chronic blood disorder.
Patients are often treated with the FDA-approved Soliris (eculizumab), by Alexion, which inhibits the terminal part of the complement cascade, a part of the immune system that, when uncontrolled, can cause rare immune disorders.
However, treatment with Soliris may increase the risk for hemolysis — ruptured red blood cells — particularly at the end of the two-week administration cycle.
The Phase 2 clinical trial (NCT03315130), currently recruiting at 30 sites across the U.S. and Canada, aims to enroll 36 patients with generalized myasthenia gravis. Patients will be randomized to a placebo (control) or one of two treatment arms.
Researchers will test two doses of RA101495 — 0.1 mg/kg and 0.3 mg/kg — administered as a daily subcutaneous injection, and matching placebo for 12 weeks (3 months).
Its primary objective (endpoint) is to determine the changes in the Quantitative Myasthenia Gravis (QMG) score from baseline up to 12 weeks of treatment. As an additional (secondary) endpoint, researchers will assess how the therapy improved patients’ daily activity, according to the changes in the Myasthenia Gravis-Activities of Daily Living (MG-ADL) scale.
Enrollment information is available here; the trial is expected to conclude in early 2019.
“After more than a decade of raising awareness through the observance of Rare Disease Day and achieving great progress in rare disease research, there is still significant work to be done to develop treatments and Ra Pharma is committed to the fight against rare diseases,” Doug Treco, president and chief executive officer of Ra Pharma, said in a press release.
“Since Rare Disease Day 2017, Ra Pharma has implemented and completed a global Phase 2 program for our lead compound RA101495 SC … We remain committed to advancing the development of RA101495 SC across a broad range of indications to address the significant unmet need for these patients,” he concluded.