- Positive topline results show the Phase 3 RAISE (NCT04115293) zilucoplan trial met primary and all key secondary endpoints in adults with generalized myasthenia gravis.
- The results show a favorable safety profile and good tolerability.
- UCB plans to proceed with zilucoplan regulatory submissions later this year.
- Results follow recent positive topline data from the Phase 3 MycarinG study investigating rozanolixizumab, a monoclonal antibody also being developed by UCB in the same indication.
- These results are the latest in a series of positive phase 3 data announcements by the company across its product pipeline.
UCB, a global biopharmaceutical company, announced positive topline results from the RAISE (NCT04115293) trial evaluating its investigational treatment zilucoplan, a self-administered, subcutaneous (SC) peptide inhibitor of complement component 5 (C5 inhibitor), 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 (MG-ADL) 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 (QMG) score, Myasthenia Gravis Composite (MGC) 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 (TEAEs) 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 (US), European Union (EU) and Japan, beginning later this year.
These findings from RAISE build on the positive results from the Phase 3 MycarinG study evaluating UCB’s investigational treatment rozanolixizumab, an SC-infused monoclonal antibody targeting the neonatal Fc receptor (FcRn) which also met its primary and secondary endpoints with statistical significance in gMG.2
UCB is currently the only company investigating two potential treatments with different mechanisms of action in gMG. Detailed results from both Phase 3 trials will be presented at forthcoming medical meetings in 2022.
“While there has been recent progress in the treatment of MG, there is still a significant unmet need for new, effective treatment options that address the unpredictable, fluctuating symptoms of MG – some of which require urgent treatment or hospitalization – to improve patient outcomes and quality of life,” said Raquel Pardo, Spanish Myasthenia Association (AMES), Spain. “New research into additional treatment options will be welcomed by the global MG community.”
Iris Loew-Friedrich, Executive Vice-President and Chief Medical Officer added: “This is the latest in a series of positive Phase 3 data announcements across UCB’s product pipeline validating our patient value strategy and laying foundations for future sustainable growth. Today’s results represent another significant milestone in UCB’s efforts to bring transformational outcomes to those living with myasthenia gravis. Positive results for zilucoplan and rozanolixizumab – each with a different mechanism of action – bring us one step closer to achieving our ambition of delivering choice and flexibility for a broad range of patients and physicians at each step of their treatment journey, addressing significant unmet needs and offering unique patient value. We thank the MG community for their continued insights, partnership and participation in this study.”