2025 American Academy of Neurology Abstract Website

Zabeen K. Mahuwala¹, Ali A. Habib², Robert M. Pascuzzi³, John Vissing⁴, Tuan Vu⁵, Fiona Grimson⁶, Thaïs Tarancón⁷, Jos Bloemers⁸, Vera Bril⁹
¹Department of Neuromuscular Medicine, Epilepsy and Clinical Neurophysiology, University of Kentucky, Lexington, KY, USA, ²MDA ALS & Neuromuscular Center, Department of Neurology, University of California, Irvine, Orange, CA, USA, ³Neurology Department, Indiana University School of Medicine, Indiana University Health, Indianapolis, IN, USA, ⁴Department of Neurology, Rigshospitalet, University of Copenhagen, Copenhagen, Denmark, ⁵Department of Neurology, University of South Florida Morsani College of Medicine, Tampa, FL, USA, ⁶UCB, Slough, UK, ⁷UCB, Madrid, Spain, ⁸UCB, Brussels, Belgium, ⁹University Health Network, Toronto, ON, Canada

Objective:

To investigate the effect of rozanolixizumab on ocular symptoms in patients with generalized myasthenia gravis (gMG) in the Phase 3 MycarinG study (NCT03971422).

Background:

Patients with gMG may experience disabling ocular symptoms such as diplopia/double vision and ptosis/eyelid drooping due to ocular muscle weakness. In MycarinG, rozanolixizumab demonstrated clinically meaningful improvements across myasthenia gravis (MG)-specific outcomes and was generally well tolerated in patients with gMG.

Design/Methods:

Adults with MGFA Disease Class II–IVa, acetylcholine receptor or muscle-specific tyrosine kinase autoantibody–positive gMG were randomly assigned 1:1:1 to once-weekly subcutaneous rozanolixizumab 7mg/kg, 10mg/kg or placebo for 6 weeks (to Day 43). This descriptive post hoc analysis evaluated mean change from baseline (CFB) in ocular item scores within the MG Activities of Daily Living (MG-ADL), Quantitative MG (QMG) and MG Symptoms Patient-Reported Outcome (MG Symptoms PRO) Ocular Muscle Weakness scales for patients with baseline score ≥1 in that item.

Results:

Overall, 200 patients received rozanolixizumab 7mg/kg (n=66), 10mg/kg (n=67) or placebo (n=67). Mean baseline scores for ocular items were 1.6–1.9 for MG-ADL, 1.8–2.1 for QMG and 1.5–1.8 for MG Symptoms PRO. For rozanolixizumab 7mg/kg, 10mg/kg and placebo, mean CFB at Day 43 in MG-ADL ocular item scores was −0.6, −0.6 and −0.2, respectively, for double vision, and −0.5, −0.7 and 0.0, respectively, for ptosis. Mean CFB in clinician-assessed QMG scores was −0.6, −0.8 and 0.1, respectively, for double vision, and −0.5, −1.0 and −0.5, respectively, for ptosis. Mean CFB in MG Symptoms PRO scores was −0.5, −0.6 and −0.2, respectively, for double vision and −0.5, −0.7 and −0.1, respectively, for eyelid drooping. Most treatment-emergent adverse events with rozanolixizumab were mild/moderate.

Conclusions:

Overall, greater improvements in ocular item scores across MG-specific outcomes were observed with rozanolixizumab compared with placebo in the MycarinG study, suggesting a benefit for gMG patients with ocular signs and symptoms.