2024 American Academy of Neurology Abstract Website

Miriam Freimer¹, Raghav Govindarajan², Bhupendra Khatri³, Todd Levine⁴, Babak Boroojerdi⁵, Eumorphia Maria Delicha⁶, Brittany Harvey⁷, Andreea Lavrov⁵, James F. Howard⁸
¹Department of Neurology, The Ohio State University Wexner Medical Center, Columbus, OH, USA, ²HSHS St. Elizabeth’s Hospital, O’Fallon, IL, USA, ³The Regional MS Center and Center for Neurological Disorders, Milwaukee, WI, USA, ⁴Department of Neurology, HonorHealth, Phoenix, AZ, USA, ⁵UCB Pharma, Monheim, Germany, ⁶UCB Pharma, Braine-l’Alleud, Belgium, ⁷UCB Pharma, Tampa, FL, USA, ⁸Department of Neurology, The University of North Carolina at Chapel Hill, Chapel Hill, NC, USA

Objective:

To evaluate safety, efficacy, patient satisfaction and preference after switching from intravenous (IV) complement component 5 (C5) inhibitors to subcutaneous (SC) zilucoplan in adults with acetylcholine receptor autoantibody-positive (AChR Ab+) generalized myasthenia gravis (gMG).

Background:

Existing antibody C5 inhibitors for gMG are administered intravenously by healthcare professionals, whereas the peptide C5 inhibitor zilucoplan is intended for self-administration by daily SC injection, which may be preferred by some patients.

Design/Methods:

MG0017 (NCT05514873) is an ongoing Phase 3b, multicenter, open-label, single-arm study with a 12-week main treatment period, and an optional extension period, of daily SC zilucoplan 0.3 mg/kg, with a target enrollment of 20 patients. Adults with AChR Ab+ gMG were eligible if they had clinically stable disease on an IV C5 inhibitor and were willing to switch to zilucoplan. Primary endpoint was incidence of treatment-emergent adverse events (TEAEs). Secondary endpoints included change from baseline (CFB) in Myasthenia Gravis Activities of Daily Living (MG-ADL) and Quantitative Myasthenia Gravis (QMG) scores at Week 12. Patient-reported outcomes included treatment satisfaction, using the Treatment Satisfaction Questionnaire for Medication (TSQM-9) global satisfaction subscore (scored from 0–100), and preference.

Results:

At data cut-off (August 11, 2023), eight participants had received zilucoplan and five had completed the 12-week main treatment period. Four (50.0%) participants reported TEAEs; one TEAE was considered treatment related (injection-site pruritus). No serious TEAEs or study discontinuations were reported. At Week 12, mean (standard deviation) CFB in MG-ADL and QMG scores were −1.80 (3.49) and −3.00 (1.63) respectively, and 26.43 (19.50) for TSQM-9 global satisfaction subscore. All five participants preferred zilucoplan to their previous IV C5 inhibitor.

Conclusions:

In this interim analysis, switching from IV C5 inhibitors to SC zilucoplan did not raise any safety concerns. After 12 weeks, data showed trends towards higher treatment satisfaction, symptom improvement, and preference for zilucoplan over IV C5 inhibitors.