2025 American Academy of Neurology Abstract Website

Vera Bril¹, Carlo Antozzi², Tomasz Berkowicz³, Artur Drużdż⁴, Rachana K. Gandhi Mehta⁵, Zabeen K. Mahuwala⁶, Jana Zschüntzsch⁷, Marion Boehnlein⁸, Andreea Lavrov⁸, Mark Morris⁹, Puneet Singh⁹, M. Isabel Leite¹⁰
¹University Health Network, Toronto, ON, Canada, ²Neuroimmunology and Muscle Pathology Unit, Multiple Sclerosis Center, Fondazione Istituto di Ricovero e Cura a Carattere Scientifico, Istituto Nazionale Neurologico Carlo Besta, Milan, Italy, ³Miejskie Centrum Medyczne JONSCHER im. dr Karola Jonschera w Łodzi, Łódź, Poland, ⁴Department of Neurology, Municipal Hospital, Poznań, Poland, ⁵Department of Neurology, Wake Forest University School of Medicine, Winston-Salem, NC, USA, ⁶Department of Neuromuscular Medicine, Epilepsy and Clinical Neurophysiology, University of Kentucky, Lexington, KY, USA, ⁷Department of Neurology, University Medical Center Göttingen, Göttingen, Germany, ⁸UCB, Monheim, Germany, ⁹UCB, Slough, UK, ¹⁰Nuffield Department of Clinical Neurosciences, University of Oxford, Oxford, UK

Objective:

To assess successful self-administration, efficacy and safety of rozanolixizumab using manual push (MP) and syringe driver (SD) methods in patients with generalized myasthenia gravis (gMG).

Background:

Rozanolixizumab, indicated for the treatment of adults with gMG, is currently administered by healthcare professionals (HCPs) using programmable SDs. Options for patients to self-administer rozanolixizumab are of clinical interest; MP and SD are potential self-administration methods.

Design/Methods:

In the Phase 3, open-label, randomized, crossover MG0020 study (NCT05681715), adults with gMG received once-weekly rozanolixizumab (weight-tiered dosing or 7 mg/kg) for 18 weeks, comprising a 6-week training period and two 6-week self-administration periods where patients were randomized 1:1 to Sequence 1 (SD then MP) or Sequence 2 (MP then SD) to self-administer at clinic and at home. Primary endpoint: successful self-administration of rozanolixizumab (choosing correct infusion site, administering subcutaneously and delivering intended dose) evaluated by an HCP at Weeks 12 and 18. Secondary endpoints included occurrence of treatment-emergent adverse events (TEAEs). Additional endpoints included change from baseline in total IgG and MG Activities of Daily Living (MG-ADL) score.

Results:

62 patients received treatment; 55 were randomized to Sequence 1 (n=28) or Sequence 2 (n=27). The rozanolixizumab self-administration success rate was 100%; all patients successfully self-administered with MP or SD at clinic and at home. Decreases from baseline in median total IgG and mean MG-ADL score were observed at Week 7 and maintained during self-administration. TEAEs occurred in 75.8% (47/62) of patients; the most common TEAE was headache (21.0% [n=13]). Incidence of TEAEs was comparable for both self-administration methods (31.5% [n=17; SD] and 34.0% [n=18; MP]). Most TEAEs (97.6% [161/165 events]) were mild/moderate.

Conclusions:

In MG0020, all patients successfully self-administered rozanolixizumab with both MP and SD methods. Efficacy and safety were consistent with the known profile of rozanolixizumab. These findings support MP or SD self-administration of rozanolixizumab in patients with gMG.