Safety and tolerability of zilucoplan in RAISE-XT: A multicenter, open-label extension study in patients with myasthenia gravis

Topic:

Clinical Trials

Poster Number: 145

Author(s):

Angela Genge, MD, Montreal Neurological Institute, Yessar Hussain, Department of Neurology, Dell Medical School, The University of Texas at Austin, Austin, TX, USA, Henry J. Kaminski, Department of Neurology & Rehabilitation Medicine, George Washington University, Washington DC, USA, Maria Isabel Leite, Nuffield Department of Clinical Neurosciences, University of Oxford, Oxford, UK, Renato Mantegazza, MD, Fondazione IRCCS Istituto Neurologico Carlo Besta, Kimiaki Utsugisawa, MD, PhD, Hanamaki General Hospital, Tuan Vu, MD, University of South Florida, Melissa Brock, UCB Pharma, Raleigh, NC, USA, Babak Boroojerdi, UCB Pharma, Monheim, Germany, Mark Vanderkelen, UCB Pharma, Braine-l’Alleud, Belgium, Guillemette de la Borderie, UCB Pharma, Brussels, Belgium, Petra W. Duda, MD, PhD, UCB Pharma, Cambridge, MA, USA, James Howard, MD, FAAN, The University of North Carolina at Chapel Hill

Background
Collating long-term clinical data will contribute to an increased understanding of the safety profile of zilucoplan in generalized myasthenia gravis (gMG).
Objective
To evaluate the safety and efficacy of zilucoplan in an interim analysis of RAISE-XT (NCT04225871).
Methods
RAISE-XT, a Phase 3, multicenter, open-label extension study, recruited patients with gMG who participated in randomized Phase 2 (NCT03315130) and Phase 3 (NCT04115293) zilucoplan studies. All patients self-administered daily subcutaneous injections of 0.3 mg/kg zilucoplan. Primary outcome was incidence of treatment-emergent adverse events (TEAEs). Key secondary outcomes included Myasthenia Gravis Activities of Daily Living (MG-ADL) score.
Results
199 patients enrolled in RAISE-XT; 104 continued zilucoplan from their qualifying study (zilucoplan group) and 95 switched to zilucoplan from placebo (placebo-switch group). Median exposure at data cut-off was 253.0 (range 29–1434) days. 169 (84.9%) patients experienced a TEAE; 46 (23.1%) patients experienced a serious TEAE. Most common TEAEs were headache and worsening of MG, both in 33 (16.6%) patients. At extension study Week 12, after 24 weeks of zilucoplan, the zilucoplan group achieved a least squares mean change in MG-ADL score from double-blind study baseline of −6.30 (95% CI: −7.44, −5.15). MG-ADL reduction from baseline for the placebo-switch group, after 12 weeks of zilucoplan, was −6.32 (95% CI: −8.00, −4.65).
Conclusion
In this interim analysis of RAISE-XT, zilucoplan demonstrated a favorable long-term safety profile. Efficacy in patients who had previously received zilucoplan continued to improve and was demonstrated for those who switched from placebo. The study is ongoing. Funding: UCB Pharma. These data were previously presented at the MGFA Scientific Session at AANEM, September 21–24, 2022.