THU0328 SAFETY AND EFFICACY OF SUBCUTANEOUS TOCILIZUMAB IN SYSTEMIC SCLEROSIS: RESULTS FROM THE OPEN-LABEL PERIOD OF THE PHASE 3 FOCUSSCED TRIAL

Abstract

Background:The anti–interleukin-6 (IL-6) receptor-α antibody tocilizumab (TCZ) demonstrated skin score improvement and forced vital capacity (FVC) preservation in patients with systemic sclerosis (SSc) in a phase 2 randomized controlled trial.1,2Data from the 48-week, double-blind (DB), placebo (PBO)-controlled period of the focuSSced phase 3 trial were previously presented,3and open-label (OL) data up to week 96 are presented herein.Objectives:To assess the long-term safety and efficacy of TCZ in SSc patients.Methods:Adult patients with active SSc (≤60-month duration, modified Rodnan skin score [mRSS] 10-35, and elevated acute-phase reactants) treated with PBO or TCZ in the DB period received OL TCZ 162 mg SC weekly from weeks 48 to 96 in the OL period (PBO→OL TCZ and TCZ→OL TCZ, respectively). Exploratory analysis of data up to week 96 included no formal statistical analyses. Changes in mRSS and percent predicted FVC (ppFVC) were assessed.Results:Overall, 92/105 TCZ (88%) and 89/107 PBO (83%) patients entered the OL TCZ treatment period at week 48, and 85/105 TCZ→OL TCZ (81%) and 82/107 PBO→OL TCZ (77%) patients completed treatment up to week 96. Continued decline in mRSS was observed in the OL period for PBO→OL TCZ and TCZ→OL TCZ patients (Table). Change in ppFVC for patients who switched from PBO to TCZ (PBO→OL TCZ) was comparable between weeks 48 and 96 (OL period) to the change in patients who received TCZ from BL to week 48 in the DB period (Table). Rates (95% CI) of serious adverse events from weeks 48 to 96 were 15.8 (8.6, 26.5) per 100 PY for TCZ→OL TCZ patients, 14.8 (7.9, 25.3) per 100 PY for PBO→OL TCZ patients, and 15.4 (11.0, 20.9) for all TCZ exposure over 96 weeks (n = 193). Rates (95% CI) of serious infections were 2.3 (0.3, 8.1) per 100 PY for TCZ→OL TCZ patients, 3.4 (0.7, 10.0) per 100 PY for PBO→OL TCZ patients, and 3.0 (1.3, 5.9) for all TCZ exposure over 96 weeks. One death occurred during the OL period in each arm.Conclusion:Although OL data have to be interpreted with caution, results from OL TCZ treatment show numeric improvements in mRSS and FVC preservation similar to those of the DB period, with a beneficial effect on trajectory of FVC decline in patients who switched from PBO to TCZ. Long-term safety results were consistent with the known safety profile of TCZ, and no new or unexpected events were observed.