AB1185 Sustained maintenance of adapted ACR pediatric response with canakinumab in patients with active systemic juvenile idiopathic arthritis

Background Systemic juvenile idiopathic arthritis (SJIA) is a rare autoinflammatory disease. Phase 3 data showed canakinumab (CAN), a fully human selective anti-IL-1β monoclonal antibody, provided robust initial adapted ACRpedi response in SJIA patients (pts) following a single dose. Objectives Demonstrate sustained efficacy of CAN in the maintenance of adapted ACRpedi response. Methods This was a two-part Phase 3 study in SJIA pts (2-20 yrs; CAN naive or rolled-over from earlier studies). Part 1 (P1), an open label, active treatment maximum 33 wk long period (including a max 20 wk corticosteroid [CS] dose reduction subpart) was followed by Part 2 (P2), a randomized, DB, placebo-controlled, event-driven period. In P1, pts received CAN sc (4mg/kg to 300mg max) every 4 wks. Primary endpoint for P1 was the proportion of pts on CS at study entry who were able to taper it. We present data for the secondary endpoints: maintenance of adapted ACRpedi30/50/70/90/100 criteria, number of active joints and physician’s global assessment of disease activity (PGDA; 0-100 mm VAS) during P1. ACR response was assessed at Days 15, 29 and every 4 weeks thereafter. PGDA and number of active joints were assessed at these same visits and at Baseline (BL) and Day 3. Results 177 pts (128 on CS; 49 steroid-free) entered P1, of which 100 pts (CAN, n=50; placebo, n=50) entered P2; most of the 77 pts who did not enter P2 were eligible (>ACR30 at Day 15) to enter an open-label extension study directly when they discontinued. Of 92 pts attempting CS taper according to pre-specified criteria, 57 (62%) were successful, representing 44.5% (57/128; p Conclusions CAN allowed for successful steroid tapering with sustained maintenance of high adapted ACRpedi response while demonstrating an acceptable safety and tolerability profile in pts with active SJIA. Disclosure of Interest P. Quartier Grant/Research support from: Novartis, J. Anton Grant/Research support from: Novartis, Pfizer, Consultant for: Novartis, Roche, Paid Instructor for: Novartis, Roche, Abbot, Speakers Bureau: Novartis, Pfizer, Abbot, SOBI, J. Barash Grant/Research support from: Novartis, R. Berner Grant/Research support from: Novartis, K. Abrams Shareholder of: Novartis, Employee of: Novartis, K. Lheritier Shareholder of: Novartis, Employee of: Novartis, D. Kim Shareholder of: Novartis, Employee of: Novartis, N. Wulffraat: None Declared