Data presented at ACR Convergence 2025 highlight Sobi’s commitment to advancing care across rare and underserved inflammatory conditions

Group 1: NASP and Uncontrolled Gout - NASP (Nanoencapsulated Sirolimus plus Pegadricase) is an investigational therapy aimed at reducing serum uric acid (sUA) levels in patients with uncontrolled gout, which affects over 200,000 individuals in the U.S. [42][43] - In the DISSOLVE Trials, NASP demonstrated significant efficacy, with median sUA levels reducing by 97% and 98% in high-dose (HD) and low-dose (LD) groups, respectively, while the placebo group saw a 1.5% increase [8][12]. - The treatment resulted in a reduction in the number of gout flares, with patients on HD NASP experiencing 2.3 times fewer flares compared to placebo [12]. - Complete resolution of tophi was observed in 31% of patients receiving HD NASP and 48% receiving LD NASP, compared to only 5% in the placebo group [9]. Group 2: Gamifant and Macrophage Activation Syndrome - Gamifant (emapalumab) is the first approved treatment for adults and children with macrophage activation syndrome (MAS) in Still's disease, receiving FDA approval in June 2025 [11][46]. - In clinical trials, Gamifant showed efficacy across diverse MAS presentations, with significant reductions in key pharmacodynamic markers such as CXCL9 and ferritin [13]. - The most common adverse reactions associated with Gamifant included viral infections (44%), rash (21%), and anemia (18%) [30]. Group 3: Vonjo and VEXAS Syndrome - Vonjo (pacritinib) is a kinase inhibitor indicated for treating adults with intermediate or high-risk myelofibrosis, with a focus on spleen volume reduction [48]. - The PAXIS trial is the first randomized, double-blind, placebo-controlled study for VEXAS syndrome, aiming to establish a standardized definition of "flare" for clinical research [19].