Abstract Details

Title Evaluating the Comparative Effectiveness of Emerging Immunomodulatory Therapies for Patients with Generalized Myasthenia Gravis

Topic Neuromuscular and Clinical Neurophysiology (EMG)

Presentation(s) P1 - Poster Session 1 (11:45 AM-12:45 PM)

Poster/Presentation
Number 11-033

Objective To assess the relative effectiveness of immunomodulatory treatments for generalized myasthenia gravis (gMG).

Background Several novel therapies have recently been approved in the United States for gMG, while inebilizumab and nipocalimab are either currently under FDA review or expected to undergo evaluation. Comparative evaluations of these treatments are necessary for guiding informed clinical decision-making.

Design/Methods A Bayesian network meta-analysis NMA was conducted to compare the efficacy of neonatal Fc receptor inhibitors (efgartigimod intravenous [IV], rozanolixizumab) and complement inhibitors (ravulizumab, zilucoplan) versus placebo. Efficacy outcomes included changes from baseline, and ≥3- and ≥5-point reductions in MG-ADL and QMG scores. Number needed to treat (NNT) versus placebo were derived from NMA outputs. The analysis will be updated to include inebilizumab and nipocalimab as new data becomes available.

Results The NMA results suggest efgartigimod IV was associated with the greatest improvement in changes from baseline in MG-ADL (not significantly better than other active treatments) and QMG (significantly better than ravulizumab and zilucoplan). Efgartigimod IV also had the lowest NNT vs. placebo for achieving ≥5-point reduction in MG-ADL and ≥3-point reductions in QMG (significantly lower vs. ravulizumab and zilucoplan), and ≥5-point reduction in QMG (significantly lower than zilucoplan). Rozanolixizumab had the lowest NNT for ≥3-point reduction in MG-ADL (not statistically lower than other active treatments). The poster will present updated results incorporating nipocalimab and inebilizumab.

Conclusions Fc receptor inhibitors, particularly efgartigimod IV, show a more favorable efficacy profile compared to ravulizumab and zilucoplan. Further results, including data on nipocalimab and inebilizumab, will be presented in updates to the analysis.

Authors/Disclosures
Cynthia Qi (argenx) PRESENTER	Cynthia Qi has received personal compensation for serving as an employee of argenx.
A. Gordon Smith, MD, FAAN (VCU Health System Department of Neurology)	Dr. Smith has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Alexion. Dr. Smith has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Argenx. Dr. Smith has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Merz. Dr. Smith has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Sangamo. Dr. Smith has received personal compensation in the range of $500-$4,999 for serving as a Consultant for US Department of Justice. Dr. Smith has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Janssen. Dr. Smith has received personal compensation in the range of $0-$499 for serving on a Scientific Advisory or Data Safety Monitoring board for Eidos. Dr. Smith has received personal compensation in the range of $0-$499 for serving on a Scientific Advisory or Data Safety Monitoring board for Lexicon. Dr. Smith has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Alexion. The institution of Dr. Smith has received research support from NIH (NINDS, NIDDK). Dr. Smith has received publishing royalties from a publication relating to health care. Dr. Smith has received personal compensation in the range of $0-$499 for serving as a Study Section with NIH.
Gil I. Wolfe, MD, FAAN (Univ. At Buffalo, SUNY)	Dr. Wolfe has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Grifols. Dr. Wolfe has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Alexion. Dr. Wolfe has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for ArgenX. Dr. Wolfe has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for UCB/Ra. Dr. Wolfe has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Janssen. Dr. Wolfe has received personal compensation in the range of $0-$499 for serving on a Scientific Advisory or Data Safety Monitoring board for Cartesian. Dr. Wolfe has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for Grifols. The institution of Dr. Wolfe has received research support from UCB/Ra. The institution of Dr. Wolfe has received research support from Immunovant. The institution of Dr. Wolfe has received research support from Roche. Dr. Wolfe has received personal compensation in the range of $0-$499 for serving as a Advisor with FDA Advisory Panel for Cellular, Tissue and Genetic Therapies.
Ali A. Habib, MD (University of California, Irvine)	Dr. Habib has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Alexion. Dr. Habib has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for argenx. Dr. Habib has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Immunovant. Dr. Habib has received personal compensation in the range of $10,000-$49,999 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/Roche. Dr. Habib has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for NMD Pharma. Dr. Habib has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Horizon/ Amgen. Dr. Habib has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Grifols. Dr. Habib has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Kyverna. Dr. Habib has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Jansen/Johnson & Johnson. Dr. Habib has received personal compensation in the range of $500-$4,999 for serving as a Consultant for EMDSerono/Merck. 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 $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Immunis Biomedical. 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 Hoffman-La Roche. Dr. Habib 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. Habib has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for Alexion. Dr. Habib has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for argenx. Dr. Habib has received personal compensation in the range of $5,000-$9,999 for serving on a Speakers Bureau for UCB. Dr. Habib has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Jansen/Joohnson & Johnson. The institution of Dr. Habib has received research support from Alexion. The institution of Dr. Habib has received research support from Horizon/Amgen. 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 Regeneron. The institution of Dr. Habib has received research support from Arcellx. The institution of Dr. Habib has received research support from Novartis. The institution of Dr. Habib has received research support from EMDSerono/Merck. The institution of Dr. Habib has received research support from Cour Pharmaceuticals.
Hongbo Yang, PhD	Dr. Yang has nothing to disclose.
Mandy Du, PhD	Dr. Du has received personal compensation for serving as an employee of Analysis Group. Dr. Du has received personal compensation in the range of $100,000-$499,999 for serving as a Consultant for Analysis Group.
Xin Chen	Ms. Chen has received personal compensation for serving as an employee of Analysis Group. Ms. Chen has received personal compensation in the range of $50,000-$99,999 for serving as a Consultant for Analysis Group.
Deborah Gelinas, MD (ARGENX)	Dr. Gelinas has received personal compensation for serving as an employee of argenx. Dr. Gelinas has stock in argenx.
Edward Brauer	Edward Brauer has received personal compensation for serving as an employee of argenx. Edward Brauer has stock in argenx.
Glenn A. Phillips, PhD	Dr. Phillips has received personal compensation for serving as an employee of argenx. Dr. Phillips has stock in argenx.

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