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. 2023 Jan;67(1):12-16.
doi: 10.1002/mus.27742. Epub 2022 Nov 12.

The best and worst of times in therapy development for myasthenia gravis

Michael Benatar  1 , Gary Cutter  2 , Henry J Kaminski  3
Affiliations

The best and worst of times in therapy development for myasthenia gravis

Michael Benatar et al. Muscle Nerve. 2023 Jan.

Abstract

Within the last 5 years, the US Food and Drug Administration (FDA) has approved complement and neonatal Fc receptor (FcRN) inhibitors for treatment of generalized myasthenia gravis, and several other therapies are in late-stage clinical trials or under regulatory review. However, questions about which patients are most likely to benefit from which therapies, and the relative effectiveness of these very expensive drugs, has resulted in uncertainty around the place that they should occupy in the existing therapeutic armamentarium. MGNet (a Rare Diseases Clinical Research Consortium funded by the National Institute of Neurological Diseases and Stroke) held two meetings during the 14th International Conference of the Myasthenia Gravis Foundation of America to discuss the most critical needs for clinical trial readiness and biomarker development in the context of therapy development for myasthenia gravis. Herein we provide a summary of these discussions, but not a consensus opinion, and offer a series of recommendations to guide focused research in the most critical areas. We welcome ongoing discussion through comments on this work.

Keywords: Myasthenia Gravis---Activities of Daily Living; clinical trials; myasthenia gravis; outcome measures; quantitative myasthenia gravis score.

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Conflict of interest statement

Disclosures of Conflict of Interest

Michael Benatar has consulted for Alexion, Immunovant, Takeda, UCB, Ad Scientam, and Sanofi. He receives research funding from Alexion and Immunovant. He has served as the site-principal investigator for MG trials sponsored by Alexion, UCB and the National Institutes of Health.

Gary Cutter serves on the Data and Safety Monitoring Boards of AI Therapeutics, AMO Pharma, Astra-Zeneca, Avexis Pharmaceuticals, Biolinerx, Brainstorm Cell Therapeutics, Bristol Meyers Squibb/Celgene, CSL Behring, Galmed Pharmaceuticals, Green Valley Pharma, Horizon Pharmaceuticals, Immunic, Mapi Pharmaceuticals LTD, Merck, Mitsubishi Tanabe Pharma Holdings, Opko Biologics,Prothena Biosciences, Novartis, Regeneron, Sanofi-Aventis, Reata Pharmaceuticals, NHLBI (Protocol Review Committee), University of Texas Southwestern, University of Pennsylvania, Visioneering Technologies, Inc. and Consulting or Advisory Boards for Alexion, Antisense Therapeutics, Biogen, Clinical Trial Solutions LLC, Entelexo Biotherapeutics, Inc., Genzyme, Genentech, GW Pharmaceuticals, Immunic, Klein-Buendel Incorporated, Merck/Serono, Novartis, Osmotica Pharmaceuticals, Perception Neurosciences, Protalix Biotherapeutics, Recursion/Cerexis Pharmaceuticals, Regeneron, Roche, SAB Biotherapeutics. Dr. Cutter is employed by the University of Alabama at Birmingham and President of Pythagoras, Inc. a private consulting company located in Birmingham AL.

Henry J. Kaminski is a consultant for Roche, Cabeletta Bio, and UCB Pharmaceuticals; and is CEO and CMO of ARC Biotechnology, LLC based on US Patent 8,961,98. He is principal investigator of the Rare Disease Network for Myasthenia Gravis (MGNet) National Institute of Neurological Disorders & Stroke, U54 NS115054, Targeted Therapy for Myasthenia Gravis. R41 NS110331 to ARC Biotechnology, and co-investigator for R43NS124329 MV2C2 antibody as a new therapeutic for myasthenia gravis to Mimivax, LLC.

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