A Phase 2/3 Placebo-Controlled, Parallel Group, Randomized Withdrawal Study to Evaluate the Efficacy and Safety of Nipocalimab for Adults with Chronic Inflammatory Demyelinating Polyneuropathy: the ARISE study (P14-8.006)

Abstract

<h3>Objective:</h3> Chronic inflammatory demyelinating polyneuropathy (CIDP) is a rare, chronic autoimmune disease of the peripheral nervous system characterized by progressive weakness and impaired sensation. The ARISE study (NCT05327114) is a Phase 2/3 multicenter, double-blind, placebo-controlled, randomized withdrawal study evaluating the efficacy and safety of nipocalimab in adults with CIDP. <h3>Background:</h3> Nipocalimab is a fully human aglycosylated immunoglobulin (Ig)G1 monoclonal antibody (mAb) designed to selectively bind, saturate, and block the IgG binding site on the endogenous neonatal Fc receptor. Nipocalimab is administered via intravenous infusion. <h3>Design/Methods:</h3> The study consists of the following periods: (1) Screening (≤4 weeks)/Run-in (≤12 weeks), which includes identification of patients with active CIDP; (2) Stage A (Open-label, 12 weeks), in which participants receive nipocalimab loading dose, then once every 2 weeks (q2w); (3) Stage B (Double-blind, placebo-controlled, ≤52 weeks), in which participants are randomized 1:1 to nipocalimab q2w versus placebo; (4) Open-label extension (variable duration), in which participants receive nipocalimab q2w. Key inclusion criteria include adults ≥18 years with CIDP, progressing/relapsing forms, confirmed by independent adjudication committee; CIDP disease activity score ≥3; and adjusted INCAT disability score 2–9. Patients with pure sensory CIDP or CISP, or other diagnoses that could better explain their clinical presentation are excluded. The primary endpoint is time to first occurrence of a relapse event in Stage B. Secondary efficacy endpoints include time to initial response to nipocalimab and percentage of nipocalimab responders in Stage A, and change from baseline in functional measures (e.g. grip strength, MRC sum score, etc.) in Stage B. Other secondary endpoints include safety/tolerability, pharmacokinetics, immunogenicity, and pharmacodynamics of nipocalimab. <h3>Results:</h3> The study is currently enrolling patients, targeting approximately 300 patients, with primary study completion date anticipated in 2026. <h3>Conclusions:</h3> This ongoing phase 2/3 study will assess the efficacy, safety, PK, and PD of nipocalimab in adults with CIDP. <b>Disclosure:</b> Dr. Ford has received personal compensation for serving as an employee of Janssen . Dr. Ford has stock in Janssen. Dr. Wong has received personal compensation for serving as an employee of Janssen Research &amp; Development. Dr. Wong has received personal compensation for serving as an employee of Biogen. Dr. Wong has stock in Biogen. Dr. Youssef has received personal compensation for serving as an employee of Janssen. Dr. Youssef has stock in Janssen. Mr. Murray has received personal compensation for serving as an employee of Janssen. Dr. Lim has received personal compensation for serving as an employee of Johnson &amp; Johnson. Dr. Lim has stock in Johnson &amp; Johnson. Dr. Nobile-Orazio has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for ArgenX, Belgium. Dr. Nobile-Orazio has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for LFB, France. Dr. Nobile-Orazio has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Roche, Switzerland. Dr. Nobile-Orazio 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. Nobile-Orazio has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Janssen, USA. The institution of Dr. Nobile-Orazio has received research support from Takeda/Shire. Ingemar Merkies has nothing to disclose. Dr. Cornblath has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Octapharma. Dr. Cornblath has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Annexon. Dr. Cornblath has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Pfizer. Dr. Cornblath has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Grifols. Dr. Cornblath has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Cigna. Dr. Cornblath 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. Cornblath has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Anavex. Dr. Cornblath 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. Cornblath has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for MPTP. Dr. Cornblath has received intellectual property interests from a discovery or technology relating to health care. Dr. Sun has received personal compensation for serving as an employee of Janssen.