Switching to Subcutaneous Zilucoplan from Intravenous Complement Component 5 Inhibitors in Myasthenia Gravis: Patient Preference and Satisfaction from a Phase 3b Study
Miriam Freimer1, Urvi Desai2, Raghav Govindarajan3, Min K. Kang4, Shaida Khan5, Bhupendra Khatri6, Todd Levine7, Samir Macwan8, Perry B. Shieh9, Michael D. Weiss10, Jos Bloemers11, Babak Boroojerdi12, Eumorphia Maria Delicha13, Andreea Lavrov12, Puneet Singh14, James F. Howard Jr.15
1The Ohio State University Wexner Medical Center, Columbus, OH, USA, 2Atrium Health, Wake Forest University, Charlotte, NC, USA, 3HSHS St. Elizabeth’s Hospital, O’Fallon, IL, USA, 4University of California, San Francisco, San Francisco, CA, USA, 5UT Southwestern Medical Center, Dallas, TX, USA, 6The Regional MS Center and Center for Neurological Disorders, Milwaukee, WI, USA, 7HonorHealth Neurology, Bob Bové Neuroscience Institute, Scottsdale, AZ, USA, 8Eisenhower Health, Rancho Mirage, CA, USA, 9University of California, Los Angeles, Los Angeles, CA, USA, 10University of Washington Medical Center, Seattle, WA, USA, 11UCB, Brussels, Belgium, 12UCB, Monheim, Germany, 13UCB, Braine-l’Alleud, Belgium, 14UCB, Slough, UK, 15The University of North Carolina at Chapel Hill, Chapel Hill, NC, USA
Objective:
To evaluate safety, efficacy, patient preference and satisfaction with zilucoplan in adults with acetylcholine receptor autoantibody–positive generalized myasthenia gravis (MG) switching from their current intravenous complement component 5 (C5) inhibitors to subcutaneous zilucoplan.
Background:
Zilucoplan, a macrocyclic peptide C5 inhibitor, is self-administered as a daily subcutaneous injection, offering an alternative to intravenous infusion of antibody-based complement C5 inhibitors.
Design/Methods:
MG0017 (NCT05514873) is a Phase 3b, open-label, single-arm study with a 12-week main treatment period of daily subcutaneous zilucoplan. Patients were required to have stable disease on an intravenous C5 inhibitor and be willing to switch to zilucoplan. Incidence of treatment-emergent adverse events (TEAEs; primary endpoint), change from baseline in Myasthenia Gravis Activities of Daily Living score at Week 12 (secondary endpoint) and preference for intravenous or subcutaneous C5 inhibitors at Week 12 (exploratory endpoint) were assessed. Treatment satisfaction was measured using the 9-item Treatment Satisfaction Questionnaire for Medication (TSQM-9; scored 0–100; exploratory endpoint).
Results:
Twenty-six patients enrolled and received zilucoplan; 16 switched from eculizumab and 10 from ravulizumab. Of these, 23 patients completed the main treatment period and three discontinued (two due to TEAEs). TEAEs occurred in 19/26 (73.1%) patients and were mostly mild in severity. At Week 12, MG symptoms were improved or unchanged in approximately 75% of patients. Subcutaneous treatment was preferred by 20/26 (76.9%) patients; 4/26 (15.4%) preferred intravenous treatment and 2/26 (7.7%) had no preference. At Week 12, mean (standard deviation) changes from baseline in the TSQM-9 global satisfaction, effectiveness and convenience subscores were +19.410 (27.429), +13.889 (21.534) and +21.739 (19.955), respectively.
Conclusions:
Zilucoplan demonstrated a favorable safety profile. More patients preferred subcutaneous to intravenous treatment, and overall treatment satisfaction increased after switching from intravenous C5 inhibitors to subcutaneous zilucoplan.
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