Late Breaking Abstract - EFZO-FIT, the largest ever interventional trial in pulmonary sarcoidosis.

Intro/Background: Sarcoidosis is a granulomatous disorder primarily affecting the lungs. Oral corticosteroids (OCS) are first line treatment for pulmonary sarcoidosis. Guidelines recommend OCS reduction as a critical goal in pulmonary sarcoidosis (PS). Efzofitimod, a novel biologic immunomodulator, previously showed a positive exposure response relationship across multiple efficacy endpoints including the ability to taper steroids. EFZO-FIT is a Phase 3 global, multicenter, randomized, double-blind, placebo-controlled study evaluating the efficacy and safety of efzofitimod in PS. Enrolment completed July 2024. Aims/Objectives: The primary endpoint in this trial is the change from baseline (CFB) in mean daily OCS dose at Week 48. Besides safety, key secondary endpoints are the CFB in KSQ-Lung score at Week 48; steroid withdrawal rate and CFB in absolute value of FVC % predicted (pp) at Week 48. Methods: The study included adults (≥ 18 years) with chronic (≥ 6 months), symptomatic (MRC dyspnea score ≥1, KSQ-Lung Score ≤70) PS on OCS for 3 months (stable for 4 weeks) with a FVC ≥50%pp. The starting OCS dose was ≥ 7.5 and ≤ 25 mg/day (Japan ≥ 5 mg/day and ≤ 25 mg/day). Up to one concomitant immunosuppressant was allowed. Eligible patients were randomized 1:1:1 to efzofitimod 3.0 or 5.0 mg/kg, or placebo, intravenously Q4W for 12 doses. All subjects had a protocol directed steroid taper from Day 15 through Day 85. Results: The study enrolled 268 patients. Four patients were randomized but not dosed. The study population was consistent with the eligibility criteria. Primary efficacy and safety results will be presented at the conference. Conclusion: We have completed the largest interventional study in PS.