Safety and efficacy of chronic weekly rozanolixizumab treatment in patients with generalized myasthenia gravis (MG0004)

Topic:

Clinical Trials

Poster Number: P352

Author(s):

Vera Bril, MD, University Health Network, Toronto, ON, Canada, Artur Drużdż, MD, Department of Neurology, Municipal Hospital Poznan, Poland, Julian Grosskreutz, MD, Precision Neurology of Neuromuscular Diseases, Dep. of Neurology, Univ. of Lubeck, Lubeck, Germany, Ali Habib, MD, ALS & Neuromuscular Center, University of California, Irvine, Orange, CA, USA, Renato Mantegazza, MD, Emeritus & Past Dir., Neuroimmunol. & Neuromusc. Dis., Fondazione IRCCS Carlo Besta, Milan, Italy, Sabrina Sacconi, MD, Peripheral Nervous System and Muscle Department, University Côte d’Azur, Kimiaki Utsugisawa, MD, PhD, Hanamaki General Hospital, Tuan Vu, MD, Department of Neurology, University of South Florida, Tampa, FL, USA, Marion Boehnlein, PhD, UCB, Manheim, Germany, Franz Woltering, MSc, UCB, Manheim, Germany, Bernhard Greve, MD, UCB, Manheim, Germany, Maryam Gayfieva, MD, MPH, UCB, Slough, UK, John Vissing, MD, University of Copenhagen

Introduction

In the Phase 3 MycarinG study (MG0003/NCT03971422), six, once-weekly infusions of rozanolixizumab 7mg/kg or 10mg/kg significantly improved myasthenia gravis (MG)-specific outcomes versus placebo. After MycarinG, patients could enroll in open-label extension MG0004 (NCT04124965) which evaluated safety, tolerability and efficacy of chronic weekly rozanolixizumab in patients with generalized MG.

Methods

In MG0004, patients were randomized (1:1) to once-weekly subcutaneous infusions of rozanolixizumab 7mg/kg or 10mg/kg for ≤52 infusions, followed by an 8-week observation period. Patients could switch dose at investigators’ discretion. After ≥6 visits, patients could enroll 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; 10mg/kg, n=35). Mean duration of rozanolixizumab treatment was 22.9 (7mg/kg) and 23.7 (10mg/kg) weeks, respectively. Rollover into MG0007 resulted in low patient numbers after Week 33 (n=17; 24.3%). Eight (11.3%) patients completed 52 weeks. 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 were 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 and 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 had a safety profile similar to repeated cycles of rozanolixizumab treatment. Clinically relevant mean improvements were maintained across MG-specific outcomes.

These data were previously presented at ICNMD, October 25–29, 2024.