UCB announced positive topline results from the RAISE trial evaluating its investigational treatment zilucoplan, a self-administered, subcutaneous peptide inhibitor of complement component 5, versus placebo in adults with generalized myasthenia gravis (gMG).
"gMG patients can experience varying and debilitating symptoms that impact their everyday lives in unique ways," said James F. Howard, MD, Distinguished Professor of Neuromuscular Disease, Chief, Neuromuscular Disorders Section, University of North Carolina School of Medicine and lead investigator in the RAISE trial. "These exciting results give us additional reason to believe that zilucoplan can offer an important step forward in addressing the unmet needs of people living with gMG. As we strive to improve the management of this complex and unpredictable disease, any new medicines will be welcomed by physicians to help us realize our goal of offering effective and flexible treatment approaches in gMG which are tailored to the needs of individual patients."
The primary endpoint of the trial was met; a clinically meaningful and statistically significant improvement from baseline in Myasthenia Gravis-Activities of Daily Living Profile total score at Week 12 was observed for the zilucoplan treatment group vs placebo.
All key secondary endpoints were also met, including statistically significant improvements from baseline in Quantitative Myasthenia Gravis score, Myasthenia Gravis Composite score and MG-QoL15r score at Week 12 for the zilucoplan treatment group vs placebo.
The results show zilucoplan was well-tolerated and no major unexpected safety findings were identified compared to earlier zilucoplan studies. The incidence of serious treatment emergent adverse events in the zilucoplan and placebo treatment arms was similar.
The safety and efficacy of zilucoplan have not been established, and it is not approved for use in any indication by any regulatory authority worldwide. Based on these results, UCB plans to progress with regulatory filings for zilucoplan in gMG in the United States, European Union and Japan, beginning later this year.
