A PHASE 2, MULTICENTER, RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED, PARALLEL-GROUP CLINICAL TRIAL OF NIPOCALIMAB IN PARTICIPANTS WITH ACTIVE IDIOPATHIC INFLAMMATORY MYOPATHIES (SPIREA): STUDY DESIGN

Background: Idiopathic inflammatory myopathies (IIM) are an uncommon class of systemic autoimmune diseases characterized by progressive weakness of muscles and involvement of internal organs, frequently resulting in physical impairment and a reduced quality of life. Nipocalimab targets the underlying disease pathology by selectively blocking the neonatal Fc receptor to reduce pathogenic autoantibodies. In a phase 2 study (NCT03772587), nipocalimab reduced pathogenic immunoglobulin G autoantibody levels and demonstrated significant clinical benefits and acceptable safety with a favorable benefit-risk profile in patients with generalized myasthenia gravis. Objectives: SPIREA (NCT05379634) is designed to evaluate the efficacy and safety of nipocalimab in patients with IIM. Methods: SPIREA, a phase 2, double-blind, placebo-controlled, randomized clinical trial, is enrolling approximately 200 adults with active IIM. The study consists of screening (≤6 weeks), double-blind treatment (52 weeks), long-term extension (48 weeks), and safety follow-up (8 weeks) periods (Figure 1). Intravenous nipocalimab or placebo is administered to randomized participants every two weeks until Week 50. Tapering of background oral glucocorticoid doses will occur from Weeks 24–44. Results: The primary endpoint is the percentage of participants who achieve at least minimal improvement (≥20) in American College of Rheumatology/European League Against Rheumatism (ACR/EULAR) Total Improvement Score (TIS) at Week 52 and on ≤5 mg/day of oral glucocorticoids from Weeks 44–52. Secondary endpoints include the percentage of participants who achieve ≥20-point improvement in TIS at Week 24 and Week 52. Conclusion: The ongoing SPIREA study investigating the safety and efficacy of nipocalimab in adults with IIM will aid in validating the ACR/EULAR-TIS endpoints in IIM and nipocalimab’s role as a steroid sparing agent in IIM. Figure 1. Study Design REFERENCES: NIL. Acknowledgements: NIL. Disclosure of Interests: Catherine E Najem is an employee of Janssen Pharmaceutical Companies of Johnson & Johnson, Jagriti Craig is an employee of Janssen Pharmaceutical Companies of Johnson & Johnson, Lisa Christopher Stine have served as consultants/on advisory boards of Janssen, Federico Zazzetti is an employee of Janssen Medical Affairs Global Services, LLC, Cathye Shu is an employee of Janssen Pharmaceutical Companies of Johnson & Johnson, Wim Noel is an employee of Janssen Pharmaceutica, Christopher Blango Sr. is an employee of Janssen Pharmaceutical Companies of Johnson & Johnson, Chetan S Karyekar is an employee of Janssen Scientific Affairs, Rohit Aggarwal have served as consultants/on advisory boards of Janssen. DOI: 10.1136/annrheumdis-2024-eular.3020 Keywords: Targeted synthetic drugs, Randomized controlled trial, Autoantibodies, Glucocorticoids, Disease-modifying Drugs (DMARDs) Citation: , volume 83, supplement 1, year 2024, page 1365Session: Inflammatory myopathies (Publication Only)

Targeted synthetic drugs, Randomized controlled trial, Autoantibodies, Glucocorticoids, Disease-modifying Drugs (DMARDs)