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  • LP-184 Rationale and design of two phase 3, Randomized, double-blind, placebo-controlled studies of the efficacy and safety of litifilimab in adults with active systemic lupus erythematosus: TOPAZ-1 and TOPAZ-2

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12. SLE treatment
LP-184 Rationale and design of two phase 3, Randomized, double-blind, placebo-controlled studies of the efficacy and safety of litifilimab in adults with active systemic lupus erythematosus: TOPAZ-1 and TOPAZ-2
  1. Ronald FVan vollenhoven1,
  2. Richard A Furie2,
  3. Eric F Morand3,
  4. Kenneth Kalunian4,
  5. Maria Dall’era5,
  6. Stacey Goode-sellers6,
  7. Puja Joshi7,
  8. George Kong8,
  9. Ting Wang8,
  10. Xing Wei8,
  11. Youmna Lahoud9 and
  12. Catherine Barbey10
  1. 1Department of Rheumatology and Clinical Immunology, Amsterdam University Medical Centers, Amsterdam, Netherlands
  2. 2Division of Rheumatology, Northwell Health, Great Neck, NY, USA
  3. 3School of Clinical Sciences, Monash University, Victoria, Australia
  4. 4Division of Rheumatology, Allergy and Immunology, University of California San Diego, CA, USA
  5. 5Division of Rheumatology, University of California San Francisco, CA, USA
  6. 6Research and Development, Biogen, Cambridge, MA, USA
  7. 7MS Immunology Clinical Development, Biogen, Cambridge, MA, USA
  8. 8Biostatistics, Biogen, Cambridge, MA, USA
  9. 9MS-Immunology Development Unit, Biogen, Cambridge, MA, USA
  10. 10MS-Immunology Development Unit, Biogen, Baar, Switzerland

Abstract

Background Litifilimab is a humanized IgG1 monoclonal antibody targeting BDCA2, a receptor predominantly expressed on plasmacytoid dendritic cells (pDCs). In Part A of the Phase 2 LILAC study (NCT02847598), administration of litifilimab at a dose of 450 mg was superior to placebo in reducing the total active joint count at Week 24 and resulted in a greater frequency of SLE Responder Index 4 (SRI-4) responses versus placebo.¹ Two multicenter, Phase 3, randomized, double-blind, placebo-controlled studies (TOPAZ-1, NCT04895241; TOPAZ-2, NCT04961567), described here, are ongoing to further evaluate litifilimab efficacy and safety in patients with SLE.

Methods Eligible participants are randomly assigned to receive either subcutaneous litifilimab (at a high or low dose) or placebo at Weeks 0 and 2, then Q4W until Week 48 (figure 1). Appropriate representation of individuals from underrepresented populations is a focus of the TOPAZ program. The SRI-4 response at Week 52 was chosen as the primary endpoint based on its performance in Part A of the LILAC study. Multiplicity-adjusted secondary endpoints of TOPAZ-1/-2 include Joint-50 response (a 50% reduction from baseline in the active joint count, where an active joint is defined as both swollen and tender, based on a 28-joint assessment) and oral corticosteroid (OCS) tapering. Additional secondary endpoints include the Cutaneous Lupus Erythematosus Disease Area and Severity Index-Activity score, Lupus Low Disease Activity State, and safety. Key features of this study design are the inclusion of several organ-specific evaluations, stringent rules for concomitant medication, and protocol-specified OCS tapering.

Results Both studies are currently recruiting, aiming to enroll 540 participants each; estimated primary completion dates are April 2025 (TOPAZ-1) and June 2025 (TOPAZ-2).

Conclusions Data from the TOPAZ studies will help characterize the efficacy and safety of litifilimab in SLE, a disease in need of greater treatment options.

Abstract LP-184 Figure 1
Abstract LP-184 Figure 1

TOPAZ-1 and TOPAZ-2 study design

Reference

  1. Furie RA, et al. N Engl J Med 2022;387:894–904

  • SLE
  • Phase 3
  • Litifilimab
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This is an open access article distributed in accordance with the Creative Commons Attribution Non Commercial (CC BY-NC 4.0) license, which permits others to distribute, remix, adapt, build upon this work non-commercially, and license their derivative works on different terms, provided the original work is properly cited, appropriate credit is given, any changes made indicated, and the use is non-commercial. See: http://creativecommons.org/licenses/by-nc/4.0/.

http://dx.doi.org/10.1136/lupus-2023-KCR.259

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