Abstract Details

Title Ravulizumab for the Treatment of Generalized Myasthenia Gravis: Timing of Response

Background

The phase 3 CHAMPION MG study (NCT03920293) demonstrated that ravulizumab is an effective and well-tolerated treatment for anti-acetylcholine receptor antibody-positive (AChR Ab+) gMG. Endpoints for primary data analysis were assessed at Week 26 of the randomized controlled period (RCP); responses at earlier timepoints have not been examined.

Objective To assess the timing of first response to ravulizumab in patients with generalized myasthenia gravis (gMG) in the CHAMPION MG study.

Design/Methods Data were included from patients with a Myasthenia Gravis-Activities of Daily Living (MG-ADL) total score of ≥6 at ravulizumab initiation who received ravulizumab in the RCP or in the open-label extension. Cumulative MG-ADL response rates at various timepoints after ravulizumab initiation were evaluated. Time to first response (improvement in MG-ADL total score ≥3 points) after ravulizumab initiation was also assessed, using the Kaplan–Meier product-limit method.

Results The analysis included 139 patients who were treated with ravulizumab for up to 60 weeks. Cumulative proportions of patients achieving MG-ADL response by Weeks 2, 4, 12, 26, and 60 were 45.3%, 55.4% 66.2%, 75.5%, and 82.0%, respectively. Median (interquartile range) time to first MG-ADL response was 29.0 (170.0) days.

Conclusions

Cumulative response rates indicate that first response was achieved by almost half of patients after one ravulizumab infusion (by Week 2) and by two-thirds of patients by Week 12; however, some patients (15.8%) achieved a first response after longer treatment. Median time to first response to ravulizumab treatment in patients with AChR Ab+ gMG was approximately 4 weeks.

Authors/Disclosures
Ali A. Habib, MD (University of California, Irvine) PRESENTER	Dr. Habib has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Alexion. Dr. Habib has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for argenx. Dr. Habib has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Immunovant. Dr. Habib has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for UCB. Dr. Habib has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Pfizer. Dr. Habib has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Genentech. Dr. Habib has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for NIH/NINDS. Dr. Habib has received personal compensation in the range of $5,000-$9,999 for serving on a Speakers Bureau for Alexion. Dr. Habib has received personal compensation in the range of $5,000-$9,999 for serving on a Speakers Bureau for argenx. The institution of Dr. Habib has received research support from Alexion. The institution of Dr. Habib has received research support from RA pharmaceuticals. The institution of Dr. Habib has received research support from Immunovant. The institution of Dr. Habib has received research support from UCB. The institution of Dr. Habib has received research support from argenx. The institution of Dr. Habib has received research support from CabalettaBio. The institution of Dr. Habib has received research support from Genentech. The institution of Dr. Habib has received research support from Regeneron.
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 BVBA. 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 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 $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Immunivant. 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 Viela Bio (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 $10,000-$49,999 for serving on a Speakers Bureau for Academic 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. The institution of Dr. Howard has received research support from Alexion Pharmaceuticals. The institution of Dr. Howard has received research support from argenx BVBA. The institution of Dr. Howard has received research support from Ra Pharma (now 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 Millenium Pharmaceuticals. 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.
Tuan Hoang Vu, MD (University of South Florida)	Dr. Vu has received personal compensation in the range of $500-$4,999 for serving as a Consultant for BPL. Dr. Vu has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Takeda. Dr. Vu has received personal compensation in the range of $10,000-$49,999 for serving on a Scientific Advisory or Data Safety Monitoring board for ARGENX. Dr. Vu 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. Dr. Vu has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for UCB. Dr. Vu has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for Alexion. Dr. Vu has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for CSL Behring. Dr. Vu has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for AbbVie. Dr. Vu has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for Argenx. Dr. Vu has received personal compensation in the range of $5,000-$9,999 for serving on a Speakers Bureau for MedScape. The institution of Dr. Vu has received research support from CSL Behring. The institution of Dr. Vu has received research support from Alexion. The institution of Dr. Vu has received research support from RA Pharma/UCB. The institution of Dr. Vu has received research support from Mitsubishi Tanaka. The institution of Dr. Vu has received research support from Mass Gen Hospital/Healy Platform Study. The institution of Dr. Vu has received research support from Amylyx Pharma. The institution of Dr. Vu has received research support from ARGENX. The institution of Dr. Vu has received research support from Alector. The institution of Dr. Vu has received research support from Annexon. The institution of Dr. Vu has received research support from Apellis. The institution of Dr. Vu has received research support from Cytokinetics. The institution of Dr. Vu has received research support from Sanofi. The institution of Dr. Vu has received research support from Horizon. The institution of Dr. Vu has received research support from Woolsey Pharma. The institution of Dr. Vu has received research support from CSL Behring. The institution of Dr. Vu has received research support from Cartesian Therapeutics. The institution of Dr. Vu has received research support from Janssen/Momenta.
Shahram Attarian, MD, PhD (CHU La Timone Aix Marseille University)	Prof. Attarian has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for SANOFI, Pfizer LFB, Alnylam Biogen . Prof. Attarian has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for SANOFI, Biogen. The institution of Prof. Attarian has received research support from National Institut of Health .
Michael G. Benatar, MBChB, DPhil, FAAN (University of Miami)	Dr. Benatar has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Biogen. The institution of Dr. Benatar has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Arrowhead. Dr. Benatar has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Alexion. Dr. Benatar has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Annexon. Dr. Benatar has received personal compensation in the range of $500-$4,999 for serving as a Consultant for UCB. Dr. Benatar has received personal compensation in the range of $500-$4,999 for serving as a Consultant for UniQure. Dr. Benatar has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Immunovant. Dr. Benatar has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Alector. Dr. Benatar 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. Benatar 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. Benatar has received intellectual property interests from a discovery or technology relating to health care. Dr. Benatar has received intellectual property interests from a discovery or technology relating to health care.
Kathleen Beasley, PharmD (Alexion Pharmaceuticals)	Dr. Beasley has received personal compensation for serving as an employee of Alexion, AstraZeneca Rare Disease. Dr. Beasley has received stock or an ownership interest from Alexion, AstraZeneca Rare Disease.
Masahisa Katsuno, MD,PhD (Nagoya University)	Prof. Katsuno has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Esai. Prof. Katsuno has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Ono Pharmaceutical. Prof. Katsuno has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Takeda Pharmaceutical. Prof. Katsuno has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Biogen Japan. The institution of Prof. Katsuno has received research support from Alexion. The institution of Prof. Katsuno has received research support from Alnylam. The institution of Prof. Katsuno has received research support from Zenyaku Kogyo. The institution of Prof. Katsuno has received research support from Mitsubishi-Tanabe Pharmaceutical. The institution of Prof. Katsuno has received research support from JB. The institution of Prof. Katsuno has received research support from Esai. The institution of Prof. Katsuno has received research support from Takeda Pharmaceutical. The institution of Prof. Katsuno has received research support from CSL Behling. The institution of Prof. Katsuno has received research support from Otsuka Pharmaceutical. The institution of Prof. Katsuno has received research support from Daiichi Sankyo. The institution of Prof. Katsuno has received research support from Sumitomo Dainippon Pharma.
Andreas Meisel, MD (Charité Universitätsmedizin Berlin)	Prof. Meisel has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Alexion. Prof. Meisel has received personal compensation in the range of $500-$4,999 for serving as a Consultant for argnx. Prof. Meisel has received personal compensation in the range of $500-$4,999 for serving as a Consultant for UCB. Prof. Meisel has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Janssen. Prof. Meisel has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for argnx. Prof. Meisel has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Alexion. Prof. Meisel has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for argnx. Prof. Meisel has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Grifols. Prof. Meisel has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Hormosan. The institution of Prof. Meisel has received research support from Octapharma. The institution of Prof. Meisel has received research support from Alexion. Prof. Meisel has received personal compensation in the range of $500-$4,999 for serving as a Auditor with Deutsche Myasthenie Gesellschaft / Bundesinstitut für Qualität und Patientensicherheit.
Serena Liao, PhD (Alexion)	Dr. Liao has received personal compensation for serving as an employee of Alexion.