[Pubmed] Switching to Subcutaneous Zilucoplan from Intravenous Complement Component 5 Inhibitors in Myasthenia Gravis: P

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Neurology. 2025 Apr 8;104(7_Supplement_1):1862. doi: 10.1212/WNL.0000000000208791. Epub 2025 Apr 7.

ABSTRACT

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. Disclosure: 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. 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 UCB. 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. The institution of Dr. Freimer has received research support from Dept of defense. The institution of an immediate family member of Dr. Freimer has received research support from Abcurro. Dr. Freimer has received personal compensation in the range of $10,000-$49,999 for serving as a presentations/teaching with UCB. Dr. Desai has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Alexion. Dr. Desai has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Biogen. Dr. Desai has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Takeda. Dr. Desai 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. Desai has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Fulcrum. Dr. Desai 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. Desai has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for UCB. Dr. Desai has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Alexion. Dr. Desai has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau 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 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 $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for UCB. 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. Dr. Govindarajan has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for Argenx. Dr. Govindarajan has received personal compensation in the range of $5,000-$9,999 for serving on a Speakers Bureau for Biohaven. Dr. Govindarajan has received personal compensation in the range of $5,000-$9,999 for serving on a Speakers Bureau for UCB. Dr. Govindarajan has received personal compensation in the range of $5,000-$9,999 for serving on a Speakers Bureau for Takeda. Dr. Govindarajan has received personal compensation in the range of $5,000-$9,999 for serving on a Speakers Bureau for Abbvie. 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. Dr. Kang has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Samsung Bioepis. Dr. Kang has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for UCB. Dr. Kang 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. Kang has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for J&J. The institution of Dr. Kang has received research support from UCSF resource allocation program . Dr. Khan has received personal compensation in the range of $500-$4,999 for serving as a Consultant for UCB pharma. Dr. Khan has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for UCB pharma. 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. 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. Dr. Macwan has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Alexion,Argenx,Catalyst,Kabafusion, UCB. Dr. Macwan has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for Abbvie,Alexion,Argenx,Biohaven,Catalyst,Grifols,Supernus. The institution of Dr. Macwan has received research support from Alexion, Disimmune Disease Foundation . Dr. Shieh has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Sarepta. Dr. Shieh has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Genentech. Dr. Shieh has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Biogen. Dr. Shieh has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Alexion. Dr. Shieh has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Argenx. Dr. Shieh has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for UCB. Dr. Shieh has received personal compensation in the range of $5,000-$9,999 for serving on a Speakers Bureau for CSL Behring. Dr. Shieh has received personal compensation in the range of $5,000-$9,999 for serving on a Speakers Bureau for Grifols. Dr. Shieh has received personal compensation in the range of $5,000-$9,999 for serving on a Speakers Bureau for Alexion. Dr. Shieh has received personal compensation in the range of $50,000-$99,999 for serving on a Speakers Bureau for Biogen. Dr. Shieh has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for Genentech. Dr. Shieh has received personal compensation in the range of $5,000-$9,999 for serving on a Speakers Bureau for Argenx. Dr. Shieh has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for Catalyst. Dr. Weiss has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for UCB-RA. Dr. Weiss has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Argenx. Dr. Weiss has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Biogen. Dr. Weiss has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Soleo. Dr. Weiss has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Immunovant. Dr. Weiss has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Cytokinetics. Dr. Weiss has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Sanofi. Dr. Bloemers has received personal compensation for serving as an employee of UCB. Dr. Bloemers has stock in UCB. Dr. Boroojerdi has received personal compensation for serving as an employee of UCB Biosciences. Dr. Boroojerdi has stock in UCb Biosciences. Eumorphia DELICHA has nothing to disclose. Andreea Lavrov has received personal compensation for serving as an employee of UCB Biosciences GmbH. An immediate family member of Andreea Lavrov has received personal compensation for serving as an employee of UCB Biosciences GmbH. Andreea Lavrov has stock in UCB Biosciences GmbH. An immediate family member of Andreea Lavrov has stock in UCB Biosciences GmbH. Dr. Singh has nothing to disclose. 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 $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for AMGEN (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 $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Biohaven Ltd. 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 CorEvitas. 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 Merck EMD Serono. 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 received personal compensation in the range of $5,000-$9,999 for serving on a Speakers Bureau for MJH LifeSci. 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. Dr. Howard has stock in Viatris. 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. The institution of Dr. Howard has received research support from NMD Pharma. The institution of Dr. Howard has received research support from Ad Scientiam. 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. Disclaimer: Abstracts were not reviewed by Neurology® and do not reflect the views of Neurology® editors or staff.

PMID:40194119 | DOI:10.1212/WNL.0000000000208791


Source: https://pubmed.ncbi.nlm.nih.gov/4019411 ... t9+e462414