Abstract Details

Title The Phase 3 PREVAIL Study Assessing the Efficacy and Safety of Subcutaneous Gefurulimab in Adults with Generalized Myasthenia Gravis: Trial in Progress

Topic Neuromuscular and Clinical Neurophysiology (EMG)

Presentation(s) P7 - Poster Session 7 (5:00 PM-6:00 PM)

Poster/Presentation
Number 11-021

Objective Provide an overview of PREVAIL, which is evaluating the efficacy and safety of gefurulimab in adults with anti-acetylcholine receptor antibody-positive (AChR-Ab+) generalized myasthenia gravis (gMG).

Background Complement component 5 (C5) inhibitors are effective treatments for AChR-Ab+ gMG. Gefurulimab is a new investigational complement inhibitor that binds C5, blocks its enzymatic cleavage, and thus, inhibits the terminal complement pathway. Gefurulimab is suitable for subcutaneous (SC) self-administration. Its extended half-life allows for once-weekly injection. Gefurulimab is currently under investigation for treatment of AChR-Ab+ gMG in a phase 3, multicenter, randomized, double-blind, placebo-controlled study (PREVAIL; NCT05556096).

Design/Methods PREVAIL will enroll up to 254 adults with AChR-Ab+ gMG, including those with mild disease. Patients may continue taking previously prescribed therapies, including immunoglobulins. Patients are randomized 1:1 to weekly SC self-injection of gefurulimab or placebo. The primary endpoint is change from baseline in Myasthenia Gravis Activities of Daily Living (MG-ADL) total score at week 26. Secondary endpoints include change from baseline in Quantitative Myasthenia Gravis (QMG) total score and Myasthenia Gravis Composite (MGC) total score. Safety, pharmacokinetics, pharmacodynamics, immunogenicity, and quality of life will also be assessed. PREVAIL includes an open-label extension.

Results PREVAIL is recruiting patients at ~ 160 sites in North America, South America, Europe, Asia, and the Pacific region.

Conclusions This study examines the potential of the C5 inhibitor gefurulimab as a new treatment for patients with AChR-Ab+ gMG that can conveniently be self-administered at home through once-weekly SC injection. Results from PREVAIL will inform the clinical development program of gefurulimab.

Authors/Disclosures
Joachim Scholz, MD (Alexion AstraZeneca Rare Disease) PRESENTER	Dr. Scholz has received personal compensation for serving as an employee of Alexion AstraZeneca Rare Disease. Dr. Scholz has stock in Alexion AstraZeneca Rare Disease. Dr. Scholz has stock in Biogen. Dr. Scholz has received intellectual property interests from a discovery or technology relating to health care.
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 argenx . 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 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 $10,000-$49,999 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 $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Cartesian 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 NMD Pharma. 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 H. Lundbeck A/S. 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 Seismic Therapeutics. 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 UCB Pharma. 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 Vor Biopharma. 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 $10,000-$49,999 for serving on a Speakers Bureau for MJH LifeSci. Dr. Howard has or had stock in Johnson & Johnson dividends.Dr. Howard has or had stock in Pfizer dividends. An immediate family member of Dr. Howard has or had stock in GlaxoSmithKline dividends.Dr. Howard has or had stock in Bristol Myer Squbb dividends.Dr. Howard has or had stock in AbbieVie Inc. 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 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. The institution of Dr. Howard has received research support from Merck EMD Serono. 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.
Kelly G. Gwathmey, MD (VCU Neuroscience, Orthopedic, and Wellness)	Dr. Gwathmey has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Alexion Pharmaceuticals. Dr. Gwathmey has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Argenx. Dr. Gwathmey has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Argenx. Dr. Gwathmey has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Strongbridge. Dr. Gwathmey has received personal compensation in the range of $500-$4,999 for serving as a Consultant for UCB. Dr. Gwathmey has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Amgen. Dr. Gwathmey has received personal compensation in the range of $500-$4,999 for serving as a Consultant for UCB. Dr. Gwathmey has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Novartis. Dr. Gwathmey 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. Gwathmey has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Cabaletta. Dr. Gwathmey has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Alexion Pharmaceuticals.
Chongbo Zhao (Chongbo Zhao, Neurologist, Department of Neurology, Huashan Hospital, Fudan Uni)	Chongbo Zhao has nothing to disclose.
Sanjay N. Rakhade, MBBS, PhD (Alexion Pharmaceuticals)	Dr. Rakhade has received personal compensation for serving as an employee of Alexion Pharmaceuticals . Dr. Rakhade has stock in Alexion Pharmaceuticals. Dr. Rakhade has stock in Astra Zeneca. Dr. Rakhade has received intellectual property interests from a discovery or technology relating to health care.
Alexandra Pelaez, MD (Alexion)	Dr. Pelaez has received intellectual property interests from a discovery or technology relating to health care.
Annie Racine, PhD (Alexion)	Annie Racine has received personal compensation for serving as an employee of Biogen. Annie Racine has received stock or an ownership interest from Biogen.
Alanna McEneny (Alexion Pharmaceuticals)	Alanna McEneny has received personal compensation for serving as an employee of Alexion, AstraZeneca Rare Disease. Alanna McEneny has stock in AstraZeneca.
Shulian Shang, PhD	Dr. Shang has received personal compensation for serving as an employee of Alexion, AstraZeneca Rare Disease. Dr. Shang has stock in Alexion, AstraZeneca Rare Disease.
Tuan H. Vu, MD (University of South Florida)	Dr. Vu has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Dianthus. Dr. Vu has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Amgen. Dr. Vu has received personal compensation in the range of $500-$4,999 for serving as a Consultant for NMD Pharma. 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 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 $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for UCB. Dr. Vu has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Amgen (Horizon). 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 Johnson & Johnson. Dr. Vu has received personal compensation in the range of $50,000-$99,999 for serving on a Speakers Bureau for Alexion. Dr. Vu has received personal compensation in the range of $5,000-$9,999 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 Argenx. 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 Mass Gen Hospital/Healy Platform Study. The institution of Dr. Vu has received research support from ARGENX. The institution of Dr. Vu has received research support from Horizon/Amgen. 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/Johnson & Johnson. The institution of Dr. Vu has received research support from Immunovant. The institution of Dr. Vu has received research support from Regeneron. The institution of Dr. Vu has received research support from Dianthus. The institution of Dr. Vu has received research support from COUR.

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