Ra Pharmaceuticals Delivers on Midstage Muscle Study

Health and Healthcare

Published: December 10, 2018 1:40 pm

Last Updated: January 11, 2020 1:55 pm

Ra Pharmaceuticals Inc. (NASDAQ: RARX) shares jumped on Monday after the company announced positive midstage results for the treatment of generalized myasthenia gravis (gMG).

Ultimately, the Phase 2 clinical trial evaluating zilucoplan achieved clinically meaningful and statistically significant reductions in both the primary and key secondary endpoints for both zilucoplan dose groups tested versus placebo at 12 weeks.

Zilucoplan achieved a mean reduction from baseline of 6.0 points in the Quantitative Myasthenia Gravis (QMG) score and a mean reduction from baseline of 3.4 points in the MG Activities of Daily Living (MG-ADL) score, with no patients treated with the 0.3 mg/kg dose of zilucoplan requiring rescue therapy.

For some quick background: gMG is a chronic, autoimmune, neuromuscular disease characterized by weakness and fatigue of skeletal muscles. Patients with gMG experience muscle weakness that becomes increasingly severe with repeated use and recovers with rest.

James F. Howard, M.D., Distinguished Professor of Neuromuscular Disease and Chief of the Neuromuscular Disorders Section, Department of Neurology, University of North Carolina School of Medicine, commented:

The rapid, profound, and sustained reductions in QMG and MG-ADL observed in this Phase 2 study confirm that complement inhibition was effective across a wide spectrum of MG patients in this study, whether refractory or non-refractory. Zilucoplan has the potential to become the first convenient, self-administered, complement inhibitor expanding access for patients living with this chronic, debilitating, neuromuscular disease.

Shares of Ra Pharmaceuticals were last seen up about 13% at $18.58, in a 52-week range of $4.78 to $19.75. The consensus analyst price target is $24.80.