The Safety and Efficacy of Chronic Weekly Rozanolixizumab Treatment in Patients with Generalized Myasthenia Gravis (MG0004)
Vera Bril1, Artur Drużdż2, Julian Grosskreutz3, Ali A. Habib4, Henry J. Kaminski5, Renato Mantegazza6, Sabrina Sacconi7, Kimiaki Utsugisawa8, Tuan Vu9, Marion Boehnlein10, Franz Woltering10, Bernhard Greve10, Maryam Gayfieva11, John Vissing12
1University Health Network, Toronto, ON, Canada, 2Department of Neurology, Municipal Hospital, Poznań, Poland, 3Precision Neurology, Department of Neurology, University of Lübeck, Lübeck, Germany, 4MDA ALS and Neuromuscular Center, University of California, Irvine, Irvine, CA, USA, 5Department of Neurology & Rehabilitation Medicine, George Washington University, Washington, DC, USA, 6Department of Neuroimmunology and Neuromuscular Diseases, Fondazione IRCCS, Istituto Nazionale Neurologico Carlo Besta, Milan, Italy, 7Université Côte d’Azur, Peripheral Nervous System & Muscle Department, Pasteur 2 Hospital, Centre Hospitalier Universitaire de Nice, Nice, France, 8Department of Neurology, Hanamaki General Hospital, Hanamaki, Japan, 9Department of Neurology, University of South Florida Morsani College of Medicine, Tampa, FL, USA, 10UCB Pharma, Monheim, Germany, 11UCB Pharma, Slough, UK, 12Department of Neurology, Rigshospitalet, University of Copenhagen, Copenhagen, Denmark
Objective:
To evaluate the safety, tolerability and efficacy of chronic, weekly rozanolixizumab in patients with generalized myasthenia gravis (MG).
Background:
In the Phase 3 MycarinG study (MG0003/NCT03971422), six once-weekly subcutaneous infusions of rozanolixizumab 7mg/kg or 10mg/kg significantly improved MG-specific outcomes versus placebo. After MycarinG, patients could enroll in open-label extension MG0004 (NCT04124965).
Design/Methods:
In MG0004, patients were randomized (1:1) to once-weekly subcutaneous rozanolixizumab 7mg/kg or 10mg/kg for 52 weeks, followed by an 8-week observation period. Patients could switch dose at the investigators’ discretion. After ≥6 visits, patients could roll over into MG0007 (NCT04650854), which replaced MG0004. Analyses are presented by first dose received unless specified otherwise. Efficacy data are presented to Week 33.
Results:
In MG0004, 70 patients received rozanolixizumab 7mg/kg (n=35) or 10mg/kg (n=35). Mean duration of rozanolixizumab was 22.9 (7mg/kg) and 23.7 (10mg/kg) weeks. Patient numbers after Week 33 (17/70 [24.3%]) were low, primarily due to rollover into MG0007. Eight (11.3%) patients completed 52 weeks in MG0004. Treatment-emergent adverse events by most recent dose were reported in 76.0% (38/50; 7mg/kg) and 78.6% (33/42; 10mg/kg) of patients; most mild or moderate. Headache occurred in 30.0% (15/50; 7mg/kg) and 28.6% (12/42; 10mg/kg) of patients and infections in 26.0% (13/50; 7mg/kg) and 21.4% (9/42; 10mg/kg) of patients. Clinically relevant improvements in MG-Activities of Daily Living score were observed; mean reduction from baseline between Weeks 7–33 ranged from −2.7 to −3.1 (7mg/kg) and −3.4 to −4.1 points (10mg/kg). Mean Quantitative MG score improvements ranged from −2.6 to −5.4 (7mg/kg) and −4.2 to −6.2 points (10mg/kg). Mean maximum reduction from baseline in total serum immunoglobulin G was 74.7% (7mg/kg) and 78.4% (10mg/kg). No clinically relevant reductions in albumin were observed.
Conclusions:
Chronic, weekly rozanolixizumab was well tolerated, with a safety profile similar to repeated cycles of rozanolixizumab treatment. Clinically relevant mean improvements were maintained across MG-specific outcomes.