Research & Development
Ra Pharmaceuticals Announces Positive gMG Phase 2 and Open-Label, Long-Term Extension Data at the 2019 AAN Annual Meeting
10 May 2019 - - US-based biopharmaceutical company Ra Pharmaceuticals, Inc. (NASDAQ: RARX) has presented data from the company's Phase 2 clinical trial evaluating zilucoplan for the treatment of generalized myasthenia gravis, including data from the open-label, long-term extension study, at the 2019 American Academy of Neurology annual meeting, in Philadelphia, PA, from May 4-10, 2019, the company said.

According to the base study results, the 0.3 mg/kg dose of zilucoplan consistently achieved rapid, sustained, and near-complete complement inhibition. The 0.1 mg/kg dose of zilucoplan achieved rapid, sustained, but sub-maximal complement inhibition.

Based on the pharmacokinetic, pharmacodynamic, and efficacy results, the 0.3 mg/kg dose of zilucoplan was selected for evaluation in the upcoming pivotal Phase 3 trial.

As previously reported, the pre-specified primary efficacy endpoint of change from baseline to week 12 in Quantitative Myasthenia Gravis score was met with zilucoplan dosed at 0.3 mg/kg SC daily, resulting in a clinically meaningful and statistically significant improvement over placebo (QMG reduction from baseline at week 12 = -6.0; placebo-corrected change in QMG at week 12 = -2.8; p=0.05).

The key secondary efficacy endpoint of change from baseline to week 12 in the MG Activities of Daily Living (MG-ADL) score was met with zilucoplan dosed at 0.3 mg/kg SC daily, resulting in a clinically meaningful and statistically significant improvement over placebo (MG-ADL reduction from baseline at week 12 = -3.4; placebo-corrected change in MG-ADL at week 12 = -2.3; p=0.04).

The 0.3 mg/kg dose of zilucoplan led to rapid, statistically significant, and clinically meaningful reductions in additional pre-specified secondary endpoints, the MG Composite (MG-COMP) and the 15-item MG Quality of Life revised scale (MG-QoL15r), versus placebo at week 12 (MG-COMP reduction from baseline at week 12 = -7.4; placebo-corrected change at week 12 = -4.1; p=0.04); MG-QoL15r reduction from baseline at week 12 = -5.9; placebo-corrected change at week 12 = -3.7; p=0.06).

Rescue therapy with intravenous immunoglobulin or plasma exchange was required by 3/15 patients in the placebo arm, 1/15 patients in the 0.1 mg/kg zilucoplan arm, and in zero patients in the 0.3 mg/kg zilucoplan arm.

Treatment with zilucoplan had a favorable safety and tolerability profile in the study, consistent with previously-completed Phase 1 and Phase 2 studies. The majority of adverse events reported were mild and were not considered by the investigators to be related to study drug. There were no serious AEs observed related to treatment with zilucoplan.

Based on feedback provided by the US Food and Drug Administration, Ra Pharma plans to initiate a single, 12-week, pivotal, Phase 3, randomised, double-blind, placebo-controlled trial evaluating the efficacy of a once-daily, SC self-administered dose of 0.3 mg/kg of zilucoplan versus placebo.

The trial is expected to enroll approximately 130 patients with gMG who are acetylcholine receptor -antibody-positive, regardless of their prior therapies.

The primary endpoint will be the change in the MG-ADL score from baseline to week 12. Following completion of the Phase 3 clinical trial, patients will have the option to enroll into an open-label, long-term extension study.

Ra Pharmaceuticals is a clinical-stage biopharmaceutical company focused on leading the field of complement biology to bring innovative and accessible therapies to patients with rare diseases. The company discovers and develops peptides and small molecules to target key components of the complement cascade.
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