Achievement of Improved Post-Intervention Status in Patients with Generalized Myasthenia Gravis Treated with Ravulizumab during the CHAMPION MG Study (S5.008)

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Objective: To determine whether ravulizumab treatment enables patients with anti-acetylcholine receptor antibody-positive generalized myasthenia gravis (AChR Ab+ gMG) to achieve the Myasthenia Gravis Foundation of America Post-Intervention Status (MGFA-PIS) of “improved”, with or without achieving minimal manifestation status (MMS). Background: The 26-week, phase 3, randomized, double-blind, placebo-controlled CHAMPION MG study demonstrated the efficacy and safety of ravulizumab in patients with AChR Ab+ gMG. Patients who completed the randomized controlled period (RCP) could receive ravulizumab in an open-label extension (OLE); blinding to original treatment was maintained. Design/Methods: In a prespecified exploratory analysis, modified MGFA-PIS (improved and achieved MMS, improved without MMS, unchanged, worsened) was assessed at Week 26 (patients who completed the RCP with Week 26 MGFA-PIS data) and Week 60 (patients who received ≥1 ravulizumab dose during the OLE with Week 60 MGFA-PIS data) relative to RCP baseline. A proportional-odds cumulative logit model (cumulated across categories starting from best outcome) was used to analyze outcomes at Week 26, with treatment as a fixed categorical effect and adjusting for geographic region (estimated odds ratio [OR] >1 indicates beneficial treatment effect). Results: At Week 26, improved status with and without MMS, respectively, was achieved by 25.6% (20/78) and 21.8% (17/78) of ravulizumab-treated patients, and by 9.9% (8/81) and 22.2% (18/81) of patients who received placebo (adjusted OR: 2.236, 95% confidence interval: 1.210, 4.130; p=0.01). At Week 60, improved status, with/without MMS, was achieved by 33.9% (19/56)/32.1% (18/56) of patients who continued ravulizumab treatment and 31.6% (18/57)/42.1% (24/57) of patients who switched from placebo to ravulizumab. Conclusions: Ravulizumab-treated patients were more likely than those receiving placebo to achieve MGFA-PIS of improved, with or without MMS. The increase in the proportion of patients achieving MGFA-PIS of improved, with or without MMS, with continued treatment, suggests that longer-term treatment may be needed for some patients to achieve this status. Disclosure: Dr. Muppidi has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Alexion. Dr. Muppidi has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for argenx. Dr. Muppidi has received personal compensation in the range of $500-$4,999 for serving as a Consultant for UCB/Ra Pharma. Dr. Muppidi has received publishing royalties from a publication relating to health care. Dr. Narayanaswami has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for UCB. Dr. Narayanaswami has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Novartis. Dr. Narayanaswami 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. Narayanaswami has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Janssen. Dr. Narayanaswami has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Argenx USA. Dr. Narayanaswami 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. Narayanaswami has received personal compensation in the range of $5,000-$9,999 for serving as an Editor, Associate Editor, or Editorial Advisory Board Member for Muscle and Nerve, Wiley. Dr. Narayanaswami has stock in Moderna. Dr. Narayanaswami has stock in Dr. Reddys laboratories. Dr. Narayanaswami has stock in Pfizer. Dr. Narayanaswami has stock in Viatris. Dr. Narayanaswami has stock in Doximity. The institution of Dr. Narayanaswami has received research support from Alexion. Dr. Narayanaswami has a non-compensated relationship as a Member, Medical and Scientific Advisory Board with Myasthenia Gravis Foundation of America that is relevant to AAN interests or activities. Dr. Narayanaswami has a non-compensated relationship as a Member, Board of Directors with AANEM that is relevant to AAN interests or activities. 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. 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 res

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