Abstract Details

Title Efficacy and Safety of Efgartigimod PH20 in Chronic Inflammatory Demyelinating Polyneuropathy: Results of ADHERE/ADHERE+

Topic Autoimmune Neurology

Presentation(s) C5 - Peripheral Nervous System Autoimmunity (2:21 PM-2:28 PM)
P2 - Poster Session 2 (2:45 PM-3:45 PM)

Poster/Presentation
Number 029

Background Efgartigimod, a human immunoglobulin G (IgG)1 antibody Fc fragment, blocks the neonatal Fc receptor, decreasing IgG recycling and reducing pathogenic IgG autoantibody levels. The multi-stage, double-blind, placebo-controlled, randomized-withdrawal ADHERE study, and ongoing, open-label extension ADHERE+ study assessed the efficacy and safety of efgartigimod PH20 subcutaneous (SC; coformulated with recombinant human hyaluronidase PH20) in chronic inflammatory demyelinating polyneuropathy (CIDP).

Objective To report efficacy and safety results from ADHERE/ADHERE+ trials.

Design/Methods Enrolled participants with CIDP (off treatment or on standard treatments withdrawn during run-in period) had active disease and received weekly efgartigimod PH20 SC 1000 mg (stage A). Responders were randomized (1:1) to weekly efgartigimod PH20 SC 1000 mg or placebo (stage B). Participants with clinical deterioration in stage B or those who completed ADHERE could enter ADHERE+ (weekly efgartigimod PH20 SC 1000 mg). The primary endpoints were evidence of clinical improvement (ECI; stage A), the risk of relapse (time to first adjusted Inflammatory Neuropathy Cause and Treatment deterioration between participants in the efgartigimod PH20 SC and placebo groups; stage B), and safety (ADHERE+; data cut-off: 15 June 2023).

Results

In stage A, 214/322 (66.5%) participants demonstrated ECI. In stage B, efgartigimod significantly reduced the risk of relapse (HR: 0.394 [95% CI, 0.253–0.614]) vs placebo (P=0.00004). The reduced risk of relapse was also shown in participants regardless of prior CIDP therapy. A total of 99% of eligible participants entered ADHERE+. The safety profile of efgartigimod was consistent over 137.42 total patient-years of follow-up for ADHERE+. Most treatment-emergent adverse events were mild/moderate, and their incidence/severity did not increase in ADHERE+.

Conclusions

ADHERE demonstrated the effectiveness of efgartigimod PH20 SC in reducing the risk of relapse. Efgartigimod PH20 SC was well-tolerated; its safety profile was similar between ADHERE and ADHERE+ and was consistent with the previously demonstrated safety profile of efgartigimod.

Authors/Disclosures
Niraja S. Suresh, MD (Lakeland Regional Medical Center) PRESENTER	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.
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 $0-$499 for serving on a Scientific Advisory or Data Safety Monitoring board for Immunics. The institution of Dr. Van Doorn has received personal compensation in the range of $0-$499 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 $0-$499 for serving on a Scientific Advisory or Data Safety Monitoring board for Roche. Dr. Van Doorn has received publishing royalties from a publication relating to health care.
Jeffrey A. Allen, MD (UMN)	Dr. Allen has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Argnyx. 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 $500-$4,999 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 $500-$4,999 for serving as a Consultant 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 grifols. 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 Alexion. Dr. Allen has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for CSL behring.
Ivana Basta	No disclosure on file
Tina Dysgaard	Tina Dysgaard has nothing to disclose.
Christian Eggers, MD (Krankenhaus der Barmherzigen Brueder Linz)	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 $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.
Channa A. Hewamadduma, MBBS, PhD, FRCP, FRC (Royal Hallamshire Hospital)	Dr. Hewamadduma has received personal compensation in the range of $500-$4,999 for serving as a Consultant for UCB. Dr. Hewamadduma has received personal compensation in the range of $500-$4,999 for serving as a Consultant for ARENX. Dr. Hewamadduma has received personal compensation in the range of $500-$4,999 for serving as a Consultant for BIOGEN. Dr. Hewamadduma has received personal compensation in the range of $500-$4,999 for serving as a Consultant for ROCHE. The institution of Dr. Hewamadduma has received research support from Neurocare . The institution of Dr. Hewamadduma has received research support from Sheffield Charitable Trust. Dr. Hewamadduma has received personal compensation in the range of $5,000-$9,999 for serving as a Adult Expert with National Health Service England (NHSE).
Erik Hofman	Erik Hofman has received personal compensation for serving as an employee of argenx BV. Erik Hofman has stock in argenx BV.
Yessar M. Hussain, MD (Austin Neuromuscular Center)	The institution of Dr. Hussain has received research support from Alnylam Pharma.
Satoshi Kuwabara	No disclosure on file
Gwendal Lemasson, MD (Hopital De Haut Leveque)	Dr. Lemasson has received personal compensation in the range of $0-$499 for serving on a Scientific Advisory or Data Safety Monitoring board for ARGENX. Dr. Lemasson has received personal compensation in the range of $0-$499 for serving on a Scientific Advisory or Data Safety Monitoring board for SANOFI.
Frank Leypoldt, MD (University Hospital Schleswig-Holstein, Campus Kiel Department of Neurology)	Frank Leypoldt, MD has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Roche. Frank Leypoldt, MD has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Argen. The institution of Frank Leypoldt, MD has received research support from German Ministry of Research BMBF.
Jie Lin	No disclosure on file
Marta Lipowska (Medical University of Warsaw)	No disclosure on file
Murray Lowe	No disclosure on file
Giuseppe Lauria	No disclosure on file
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.
Mihaela A. Simu, MD, PhD (University of Medicine and Pharmacy Timisoara)	Dr. Simu has received personal compensation in the range of $0-$499 for serving on a Scientific Advisory or Data Safety Monitoring board for Novartis PHarma . Dr. Simu has received personal compensation in the range of $0-$499 for serving on a Scientific Advisory or Data Safety Monitoring board for Sanofi Romania. Dr. Simu 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. Simu has received personal compensation in the range of $0-$499 for serving on a Speakers Bureau for ABBVIE. Dr. Simu has received personal compensation in the range of $0-$499 for serving on a Speakers Bureau for ABBVIE. Dr. Simu has received personal compensation in the range of $0-$499 for serving on a Speakers Bureau for ABBVIE. Dr. Simu has received personal compensation in the range of $0-$499 for serving on a Speakers Bureau for Merck. Dr. Simu has received personal compensation in the range of $0-$499 for serving on a Speakers Bureau for Merck. Dr. Simu has received personal compensation in the range of $0-$499 for serving on a Speakers Bureau for Merck. Dr. Simu has received personal compensation in the range of $0-$499 for serving on a Speakers Bureau for TEVA. Dr. Simu has received personal compensation in the range of $0-$499 for serving on a Speakers Bureau for Sanofi Romania. Dr. Simu has received personal compensation in the range of $0-$499 for serving on a Speakers Bureau for Sanofi Romania. Dr. Simu has received personal compensation in the range of $0-$499 for serving on a Speakers Bureau for Ipsen . Dr. Simu has received personal compensation in the range of $0-$499 for serving on a Speakers Bureau for Bayer. Dr. Simu has received personal compensation in the range of $0-$499 for serving on a Speakers Bureau for Roche. Dr. Simu has received personal compensation in the range of $0-$499 for serving on a Speakers Bureau for Merck. Dr. Simu has received personal compensation in the range of $0-$499 for serving on a Speakers Bureau for ABBVIE. Dr. Simu has received personal compensation in the range of $0-$499 for serving on a Speakers Bureau for UCB. Dr. Simu has received personal compensation in the range of $0-$499 for serving on a Speakers Bureau for Novartis Pharma. Dr. Simu has received personal compensation in the range of $0-$499 for serving on a Speakers Bureau for Ipsen. Dr. Simu has received personal compensation in the range of $0-$499 for serving on a Speakers Bureau for Johnson &Johnson. Dr. Simu has received personal compensation in the range of $0-$499 for serving on a Speakers Bureau for Roche . Dr. Simu has received personal compensation in the range of $0-$499 for serving on a Speakers Bureau for Sanofi. Dr. Simu has received personal compensation in the range of $0-$499 for serving on a Speakers Bureau for Sanofy Romania. Dr. Simu has received personal compensation in the range of $0-$499 for serving on a Speakers Bureau for ABBVIE. Dr. Simu has received personal compensation in the range of $0-$499 for serving on a Speakers Bureau for Roche. Dr. Simu has received personal compensation in the range of $0-$499 for serving on a Speakers Bureau for Sanofi Romania. Dr. Simu has received personal compensation in the range of $0-$499 for serving on a Speakers Bureau for Merck. Dr. Simu has received personal compensation in the range of $0-$499 for serving on a Speakers Bureau for Sanofi. Dr. Simu has received personal compensation in the range of $0-$499 for serving on a Speakers Bureau for Roche. Dr. Simu has received personal compensation in the range of $0-$499 for serving on a Speakers Bureau for Novartis. Dr. Simu has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Abbvie. Dr. Simu has received personal compensation in the range of $0-$499 for serving on a Speakers Bureau for Merck. Dr. Simu has received personal compensation in the range of $0-$499 for serving on a Speakers Bureau for Abbvie . Dr. Simu has received personal compensation in the range of $0-$499 for serving on a Speakers Bureau for Sanofi. Dr. Simu has received personal compensation in the range of $0-$499 for serving on a Speakers Bureau for Novartis. Dr. Simu has received personal compensation in the range of $0-$499 for serving on a Speakers Bureau for Abbvie. Dr. Simu has received personal compensation in the range of $0-$499 for serving on a Speakers Bureau for Sanofi. Dr. Simu has received personal compensation in the range of $0-$499 for serving on a Speakers Bureau for Johnson&Johnson. Dr. Simu has received personal compensation in the range of $0-$499 for serving on a Speakers Bureau for Sanofi. Dr. Simu has received personal compensation in the range of $0-$499 for serving on a Speakers Bureau for Roche. Dr. Simu has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Novartis. Dr. Simu has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Johnson & Johnson . Dr. Simu has received personal compensation in the range of $0-$499 for serving on a Speakers Bureau for Novartis. Dr. Simu has received personal compensation in the range of $0-$499 for serving on a Speakers Bureau for Abbvie. Dr. Simu has received personal compensation in the range of $0-$499 for serving on a Speakers Bureau for Sanofi. Dr. Simu has received personal compensation in the range of $0-$499 for serving on a Speakers Bureau for Sanofi. Dr. Simu has received personal compensation in the range of $0-$499 for serving on a Speakers Bureau for UCB. Dr. Simu has received personal compensation in the range of $0-$499 for serving on a Speakers Bureau for Merck. Dr. Simu has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Sanofi. Dr. Simu has received personal compensation in the range of $0-$499 for serving on a Speakers Bureau for Ewopharma A G. Dr. Simu has received personal compensation in the range of $0-$499 for serving on a Speakers Bureau for Roche. Dr. Simu has received personal compensation in the range of $0-$499 for serving on a Speakers Bureau for Ipsen. Dr. Simu has received personal compensation in the range of $0-$499 for serving on a Speakers Bureau for Novartis. Dr. Simu has received personal compensation in the range of $0-$499 for serving on a Speakers Bureau for Ipsen.
Ting Chang	No disclosure on file
Anissa Tse	No disclosure on file
Peter Ulrichts	Peter Ulrichts has received personal compensation for serving as an employee of argenx. Peter Ulrichts has stock in argenx.
Benjamin Van Hoorick	No disclosure on file
Ryo Yamasaki	No disclosure on file
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 $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 $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.