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Abstract Details

Safety, Efficacy, and Patient Preference for Subcutaneous Zilucoplan in Myasthenia Gravis After Switching from Intravenous Complement Component 5 Inhibitors: An Interim Analysis of a Phase 3b Study
Neuromuscular and Clinical Neurophysiology (EMG)
P10 - Poster Session 10 (11:45 AM-12:45 PM)
11-006

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.

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).

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.

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.

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. 
Authors/Disclosures
Miriam L. Freimer, MD, FAAN (The Ohio State University)
PRESENTER
Dr. Freimer has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for argenx. Dr. Freimer has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for alexion. Dr. Freimer has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for J and J. The institution of Dr. Freimer has received research support from Alnylum. The institution of Dr. Freimer has received research support from UCB. The institution of Dr. Freimer has received research support from NIH. The institution of Dr. Freimer has received research support from Janssen. Dr. Freimer has received research support from Avidity. Dr. Freimer has received research support from Fulcrum. Dr. Freimer has received personal compensation in the range of $10,000-$49,999 for serving as a presenations/teaching with UCB.
Raghav Govindarajan, MD, FAAN (HSHS St. Elizabeth Medical Group) Dr. Govindarajan has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for MT pharma. Dr. Govindarajan has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Alexion. Dr. Govindarajan has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Argenx. Dr. Govindarajan has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Catalyst. Dr. Govindarajan has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Roche . Dr. Govindarajan has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Sarepta. Dr. Govindarajan has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Amicus. Dr. Govindarajan has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for Alexion. Dr. Govindarajan has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for MT pharma . Dr. Govindarajan has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Catalyst. The institution of Dr. Govindarajan has received research support from Band of Hope . The institution of Dr. Govindarajan has received research support from Alexion. Dr. Govindarajan has received publishing royalties from a publication relating to health care.
Bhupendra O. Khatri, MD, FAAN (Center for Neurologic Disorders/St. Francis OP Center) Dr. Khatri has received personal compensation in the range of $50,000-$99,999 for serving as a Consultant for Alexion. Dr. Khatri has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Biogen. Dr. Khatri has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for EMD Serono. Dr. Khatri has received personal compensation in the range of $100,000-$499,999 for serving as a Consultant for ER Squibb (Bristol Myer Squibb). Dr. Khatri has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Horizone. Dr. Khatri has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Janssen. Dr. Khatri has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for TG Therapeutics. Dr. Khatri has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Genzyne. Dr. Khatri has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Genentech. Dr. Khatri has received personal compensation in the range of $10,000-$49,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Genzyme. Dr. Khatri has received personal compensation in the range of $50,000-$99,999 for serving on a Speakers Bureau for Alexion. Dr. Khatri has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for Biogen. Dr. Khatri has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for EMD Seronoe. Dr. Khatri has received personal compensation in the range of $100,000-$499,999 for serving on a Speakers Bureau for E.R. Squibb. Dr. Khatri has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for Horizon. Dr. Khatri has received personal compensation in the range of $5,000-$9,999 for serving on a Speakers Bureau for Janssen. Dr. Khatri has received personal compensation in the range of $5,000-$9,999 for serving on a Speakers Bureau for TG Therapeutic. Dr. Khatri has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for Genzyme. Dr. Khatri has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for Genentech. Dr. Khatri has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Argenx US. The institution of Dr. Khatri has received research support from Biogen.
Todd D. Levine, MD (Honor Health) Dr. Levine has received personal compensation for serving as an employee of CND life sciences . Dr. Levine has received personal compensation in the range of $100,000-$499,999 for serving as a Consultant for Nufactor. Dr. Levine has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for Grifols. Dr. Levine has received personal compensation in the range of $50,000-$99,999 for serving as an Expert Witness for PNA. Dr. Levine has stock in CND Life Sciences. Dr. Levine has stock in Corinthian reference lab.
Babak Boroojerdi, MD (UCB Biosciences GmbH) Dr. Boroojerdi has received personal compensation for serving as an employee of UCB Biosciences. Dr. Boroojerdi has stock in UCb Biosciences.
Eumorphia DELICHA (UCB) No disclosure on file
Brittany Harvey (UCB) No disclosure on file
Andreea Lavrov No disclosure on file
James F. Howard, Jr., MD, FAAN (The University of North Carolina, Dept of Neurology, CB 7025) Dr. Howard has received personal compensation in the range of $10,000-$49,999 for serving on a Scientific Advisory or Data Safety Monitoring board for arenx . Dr. Howard has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Regeneron Pharmaceuticals. Dr. Howard has received personal compensation in the range of $10,000-$49,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Alexion Pharmaceuticals. Dr. Howard has received personal compensation in the range of $10,000-$49,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Ra Pharma (now UCB Biosciences). Dr. Howard has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Horizon Therapeutics. Dr. Howard has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Sanofi US. Dr. Howard has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Novartis. Dr. Howard has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for Academic CME. Dr. Howard has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for PeerView CME. Dr. Howard has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for Platform Q CME. Dr. Howard has received personal compensation in the range of $5,000-$9,999 for serving on a Speakers Bureau for Zai Laboratories. Dr. Howard has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for Medscape CME. Dr. Howard has stock in General Electric dividends. Dr. Howard has stock in Johnson & Johnson dividends. Dr. Howard has stock in Pfizer dividends. An immediate family member of Dr. Howard has stock in GlaxoSmithKline dividends. An immediate family member of Dr. Howard has stock in GE Healthcare dividends. The institution of Dr. Howard has received research support from Alexion Pharmaceuticals. The institution of Dr. Howard has received research support from argenx . The institution of Dr. Howard has received research support from UCB Biosciences. The institution of Dr. Howard has received research support from NIH. The institution of Dr. Howard has received research support from Centers for Disease Control/Research Triangle Institute. The institution of Dr. Howard has received research support from Duke University (DCRI). The institution of Dr. Howard has received research support from Cartestian Therapeutics. Dr. Howard has a non-compensated relationship as a Scientific Advisiory Board member, Committee member with Myasthenia Gravis Foundation of America that is relevant to AAN interests or activities. Dr. Howard has a non-compensated relationship as a Committee member with American Assoc Neuromuscular and Electrodiagnostic Medicine that is relevant to AAN interests or activities.