Abstract Details

Title Long-Term Efficacy of Efgartigimod PH20 SC in Patients with Chronic Inflammatory Demyelinating Polyneuropathy: Interim Results from the ADHERE+ Study

Topic Neuromuscular and Clinical Neurophysiology (EMG)

Presentation(s) S16 - Updates on Nerve and Muscle Disorders (1:12 PM-1:24 PM)

Poster/Presentation
Number 002

Background

Efgartigimod, a human immunoglobulin (Ig)G1 antibody Fc fragment, blocks the neonatal Fc receptor, decreasing IgG recycling and reducing IgGs that may play a role in CIDP pathophysiology. The multi-stage, double-blinded, placebo-controlled ADHERE (NCT04281472) and open-label extension ADHERE+ (NCT04280718) trials assessed the efficacy and safety of efgartigimod PH20 SC in participants with CIDP.

Objective

Assess the efficacy of subcutaneous (SC) efgartigimod PH20 (co-formulated with recombinant human hyaluronidase PH20) in chronic inflammatory demyelinating polyneuropathy (CIDP) in ADHERE+ (Interim analysis, data cutoff: February 16, 2024).

Design/Methods

Enrolled participants had CIDP (off treatment or on standard treatments withdrawn during ≤12-week run-in).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). Participants with clinical deterioration in stage-B or who completed ADHERE could enter ADHERE+ (weekly efgartigimod PH20 SC 1000 mg). Changes from run-in baseline (CFB) (study enrollment) in adjusted Inflammatory Neuropathy Cause and Treatment (aINCAT), Inflammatory Rasch-built Overall Disability Scale (I-RODS) (centile metric), and (dominant hand) grip strength scores to ADHERE+ Week 36 in all enrolled participants are reported.

Results

322 participants entered stage-A; 221 were randomized and treated in stage-B (111 efgartigimod, 110 placebo). 99% of eligible participants entered ADHERE+. Mean (SE) aINCAT, I-RODS, and grip strength scores were 4.0 (0.10), 47.9 (1.05), and 46.6 (1.75), respectively, at ADHERE run-in baseline (N=191). Mean CFB (SE) in aINCAT, I-RODS, and grip strength scores were −1.2 (0.15) and 8.8 (1.46) points and 17.5 (2.02) kPa, respectively, at ADHERE+ Week 36 (N=150), representing clinically meaningful improvements. Additionally, half of the participants who experienced clinical deterioration (aINCAT) during ADHERE stage-B restabilized on efgartigimod PH20 SC as early as ADHERE+ Week 4.

Conclusions

Interim results from ADHERE+ indicate long-term effectiveness of efgartigimod PH20 SC in clinical outcomes in participants with CIDP.

Authors/Disclosures
Jeffrey A. Allen, MD (University of Minnesota) PRESENTER	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 $500-$4,999 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 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 Octapharma. 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 $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Argenx. 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 Alnyla,m. Dr. Allen has received personal compensation in the range of $5,000-$9,999 for serving on a Speakers Bureau for Alexion.
Jie Lin	No disclosure on file
Mark Stettner, MD (University of Duesseldorf)	No disclosure on file
Jeff Guptill, MD, FAAN (argenx US)	Dr. Guptill has received personal compensation for serving as an employee of argenx. Dr. Guptill has 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 $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 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. 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 $500-$4,999 for serving as a Consultant for Johnson & Johnson. Dr. Querol has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Sanofi. 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.
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 $5,000-$9,999 for serving as a Consultant for Alnylam. Dr. Karam has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Ionis. Dr. Karam has received personal compensation in the range of $5,000-$9,999 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 $500-$4,999 for serving as a Consultant for Alexion. Dr. Karam has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Neuroderm.
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 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 Roche. 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 Anexxon. 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. Dr. Van Doorn has received publishing royalties from a publication relating to health care.
Richard A. Lewis, MD, FAAN (Cedars-Sinai Medical Center)	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 Seismic. 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 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.

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