Abstract Details

Title Treatment Impact of Efgartigimod PH20 SC on I-RODS Daily Activity Assessment in Patients with Chronic Inflammatory Demyelinating Polyneuropathy: Post Hoc Analysis of the Registrational ADHERE Study

Topic Neuromuscular and Clinical Neurophysiology (EMG)

Presentation(s) P10 - Poster Session 10 (5:00 PM-6:00 PM)

Poster/Presentation
Number 11-025

Objective

Assess the effect on functionality of subcutaneous (SC) efgartigimod PH20 (co-formulated with recombinant human hyaluronidase PH20) in chronic inflammatory demyelinating polyneuropathy (CIDP).

Background

CIDP is a rare, progressive, immune-mediated polyneuropathy characterized by proximal and distal weakness, and sensory disturbance that can lead to irreversible disability. Efgartigimod, a human IgG1 antibody Fc fragment, blocks the neonatal Fc receptor, decreases immunoglobulin G (IgG) recycling, and reduces IgGs.

Design/Methods

ADHERE (NCT04281472), a multistage, multicenter, randomized, double-blinded, placebo-controlled trial, enrolled participants with CIDP (off treatment or on standard treatments withdrawn during ≤12-week run-in). Participants with active disease received open-label, weekly efgartigimod PH20 SC 1000 mg (stage-A). Responders were randomized (1:1) to weekly efgartigimod PH20 SC 1000 mg or placebo for ≤48 weeks (stage-B). We report a post hoc analysis of changes from run-in baseline (study enrollment) to stage-B last assessment in centile metric total (all participants) and individual items of the Inflammatory Rasch-built Overall Disability Scale (I-RODS) (participants with improvements of 1–2 points on individual I-RODS items of clinical interest from run-in baseline).

Results

322 participants entered stage-A; 221 were randomized and treated in stage-B (111 efgartigimod, 110 placebo). 191/221 participants had run-in baseline and post–stage-B timepoints. Mean (SE) I-RODS change at stage-B last assessment vs run-in baseline was 5.7 (1.88) and −4.9 (1.82) in participants randomized to efgartigimod PH20 SC and placebo, respectively. 37/97 (38.1%) and 24/92 (26.1%) participants randomized to efgartigimod PH20 SC and placebo, respectively, experienced ≥4-point improvements in I-RODS score. Efgartigimod-treated participants improved ≥1 point in I-RODS items of Dress Upper Body (24/57, 42.1%), Wash Upper Body (19/54, 35.2%), Walk Outdoors (27/83, 32.5%), Walk One Flight of Stairs (25/83, 30.1%), Walk Avoiding Obstacles (19/72, 26.4%), and Wash Lower Body (17/69, 24.6%).

Conclusions

Participants who received efgartigimod PH20 SC in stage-B experienced improvements in I-RODS score from study enrollment to stage-B last assessment.

Authors/Disclosures
Richard A. Lewis, MD, FAAN (Cedars-Sinai Medical Center) PRESENTER	Dr. Lewis has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Argenx. Dr. Lewis has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for CSL Behring. Dr. Lewis has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Sanofi. Dr. Lewis has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Grifols. Dr. Lewis has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Takeda. Dr. Lewis has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Nuvig. Dr. Lewis has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Dianthus. Dr. Lewis has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Janssen. Dr. Lewis has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Medscape. Dr. Lewis has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Annexon. Dr. Lewis has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Immunovant. Dr. Lewis has received personal compensation in the range of $500-$4,999 for serving as a Consultant for BioCryst. Dr. Lewis has received personal compensation in the range of $500-$4,999 for serving as a Consultant for NervoSave. Dr. Lewis has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Annexon. Dr. Lewis has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Alexion. Dr. Lewis has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Amgen. Dr. Lewis has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Immunovant. Dr. Lewis has received personal compensation in the range of $500-$4,999 for serving as a Consultant for TGTX. Dr. Lewis has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Boehringer-Ingleheim. Dr. Lewis has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Intellia. Dr. Lewis has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Cartesian. Dr. Lewis has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for CME Outfitters. Dr. Lewis has received personal compensation in the range of $500-$4,999 for serving as a Consultant for PeerVision. Dr. Lewis has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Up to Date. Dr. Lewis 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. Lewis has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Boehringer Ingelheim. Dr. Lewis has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Alnylam. Dr. Lewis has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Novartis. Dr. Lewis has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Intellia. Dr. Lewis has received personal compensation in the range of $5,000-$9,999 for serving as an Expert Witness for Shernoff et al. Dr. Lewis has received publishing royalties from a publication relating to health care.
Jeffrey A. Allen, MD (University of Minnesota)	Dr. Allen has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Argenx. Dr. Allen has received personal compensation in the range of $500-$4,999 for serving as a Consultant for csl behring. Dr. Allen has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Takeda. Dr. Allen has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Grifols. Dr. Allen has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Alnylam. Dr. Allen has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Sanofi. Dr. Allen has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Astra Zeneca. Dr. Allen has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Johnson and Johnson. Dr. Allen has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Alexion. Dr. Allen has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Annexon. Dr. Allen has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Immunovant. Dr. Allen has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Amgen. Dr. Allen has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Dianthus. Dr. Allen has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for CSL Behring. Dr. Allen has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Annexon. Dr. Allen has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Takeda. Dr. Allen 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. Allen has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Dianthus. Dr. Allen has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for CSL behring. Dr. Allen has received personal compensation in the range of $5,000-$9,999 for serving on a Speakers Bureau for Takeda. Dr. Allen has received personal compensation in the range of $5,000-$9,999 for serving on a Speakers Bureau for Alnylam. Dr. Allen has received personal compensation in the range of $5,000-$9,999 for serving on a Speakers Bureau for Alexion. Dr. Allen has received personal compensation in the range of $5,000-$9,999 for serving on a Speakers Bureau for Johnson and Johnson.
Jie Lin	No disclosure on file
Mark Stettner, MD (University of Duesseldorf)	No disclosure on file
Ingemar S. Merkies, MD, PhD	The institution of Prof. merkies has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Sanofi/Argenx/.
Jeff Guptill, MD, FAAN (argenx US)	Dr. Guptill has received personal compensation for serving as an employee of argenx. Dr. Guptill has or had stock in argenx.
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.
Geoffrey Istas	No disclosure on file
Arne De Roeck, PhD	Mr. De Roeck has received personal compensation for serving as an employee of argenx. Mr. De Roeck has stock in argenx.
Satoshi Kuwabara	Satoshi Kuwabara has nothing to disclose.
Giuseppe Lauria	Giuseppe Lauria has nothing to disclose.
Luis Querol, MD, PhD (Hospital de la Santa Creu i Sant Pau)	Dr. Querol has received personal compensation in the range of $500-$4,999 for serving as a Consultant for CSL Behring. The institution of Dr. Querol has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for UCB. Dr. Querol has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for ArgenX. Dr. Querol has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Takeda. Dr. Querol has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Dianthus. Dr. Querol has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Alnylam. Dr. Querol has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Annexon. Dr. Querol has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Avilar. Dr. Querol has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Biocryst. Dr. Querol has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Immunovant. Dr. Querol has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Lundbeck. Dr. Querol has received personal compensation in the range of $500-$4,999 for serving as a Consultant for KPL. Dr. Querol has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Lycia. The institution of Dr. Querol has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Sanofi. Dr. Querol has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Sanofi. Dr. Querol has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Novartis. Dr. Querol has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Roche. The institution of Dr. Querol 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. Querol has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for ArgenX. Dr. Querol has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Roche. Dr. Querol has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Merck. Dr. Querol has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Grifols. The institution of Dr. Querol has received research support from GBS-CIDP Foundation International. The institution of Dr. Querol has received research support from Grifols. The institution of Dr. Querol has received research support from ISCIII. The institution of Dr. Querol has received research support from CIBERER. The institution of Dr. Querol has received research support from UCB. The institution of Dr. Querol has received research support from ArgenX.
Niraja S. Suresh, MD (Lakeland Regional Medical Center)	Dr. Suresh has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Anylam . Dr. Suresh has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Alexeion. Dr. Suresh has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Argenex. Dr. Suresh has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Takeda . Dr. Suresh has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Pfizer.
Chafic Y. Karam, MD (University of Pennsylvania)	Dr. Karam has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Alnylam. Dr. Karam has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Sanofi. Dr. Karam has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Argenx. Dr. Karam has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Annexon. Dr. Karam has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Nuvig. Dr. Karam has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Applied therapeutics. Dr. Karam has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Amgen. Dr. Karam has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Astra Zeneca. Dr. Karam has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Intellia. Dr. Karam has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Takeda. Dr. Karam has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Vertex.
Thomas Skripuletz	Thomas Skripuletz has received personal compensation in the range of $50,000-$99,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Alexion, Alnylam Pharmaceuticals, Biogen, Centogene, CSL Behring, Grifols, Hexal AG, Janssen-Cilag, Merck Serono, Novartis, Roche, Sanofi, Swedish Orphan Biovitrum, Viatris. Thomas Skripuletz has received personal compensation in the range of $50,000-$99,999 for serving on a Speakers Bureau for from Alexion, Alnylam Pharmaceuticals, argenx, Bayer Vital, Biogen, Bristol Myers Squibb, Celgene, Centogene, CSL Behring, Euroimmun, Grifols, Hexal AG, Horizon, Janssen-Cilag, Merck Serono, Novartis, Pfizer, Roche, Sanofi, Siemens, Swedish Orphan Biovitrum, Teva, Viatris.
Simon Rinaldi, PhD, MBChB	Dr. Rinaldi has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Aregnx. Dr. Rinaldi has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Hansa. Dr. Rinaldi has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Annexon. Dr. Rinaldi has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Takeda. Dr. Rinaldi has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for CSL Behring. The institution of Dr. Rinaldi has received research support from MRC. The institution of Dr. Rinaldi has received research support from BMA. The institution of Dr. Rinaldi has received research support from GBS/CIDP Foundation International.
Andoni ECHANIZ-LAGUNA, MD, PhD	Prof. ECHANIZ-LAGUNA has received personal compensation in the range of $500-$4,999 for serving as a Consultant for LFB. Prof. ECHANIZ-LAGUNA has received personal compensation in the range of $500-$4,999 for serving as a Consultant for ARGENX. Prof. ECHANIZ-LAGUNA has received personal compensation in the range of $500-$4,999 for serving as a Consultant for SANOFI.
Benjamin Van Hoorick	Dr. Van Hoorick has received personal compensation for serving as an employee of argenx. Dr. Van Hoorick has stock in argenx.
Ryo Yamasaki	Ryo Yamasaki has nothing to disclose.
Pieter Van Doorn, MD (Erasmus University Medical Center)	The institution of Dr. Van Doorn has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Argenx. The institution of Dr. Van Doorn has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Hansa. The institution of Dr. Van Doorn has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Sanofi. The institution of Dr. Van Doorn has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Octapharma. The institution of Dr. Van Doorn has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Annexon. The institution of Dr. Van Doorn has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Grifols. The institution of Dr. Van Doorn has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Octapharma. The institution of Dr. Van Doorn has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Argenx. Dr. Van Doorn has received publishing royalties from a publication relating to health care.

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