aTyr Pharma presents efzofitimod data

aTyr Pharma announced the results of a post-hoc analysis of data from its Phase 1b/2a study of efzofitimod in patients with pulmonary sarcoidosis. The analysis was presented in a poster at the European Respiratory Society International Congress 2023, which is taking place September 9 – 13, 2023, in Milan, Italy. The poster presents findings from a pooled, post-hoc analysis of data from a Phase 1b/2a randomized, double-blind, placebo-controlled, multiple ascending dose 24-week study of efzofitimod in patients with pulmonary sarcoidosis receiving oral corticosteroid dose greater than or equal to 10.0 mg/day. Patients were randomized 1:2 and underwent a forced steroid taper in the first 8 weeks of the study. Dose dependent improvements in steroid burden, FVC and patient reported outcomes were noted, though the study was not powered for efficacy. In this pooled analysis, the 3.0 mg/kg and 5.0 mg/kg efzofitimod arms were considered therapeutic, and pooled. The placebo and 1.0 mg/kg efzofitimod arm, which was considered subtherapeutic, were pooled. Time to relapse for steroid use, rate of change for FVC and proportion of patients with changes that are multiples of the minimally clinically important difference in PRO were compared. Additionally, a responder endpoint was proposed and an analysis was performed. Key findings include: 7.7% of patients in the therapeutic group relapsed for steroid use compared to 54.4% of patients in the placebo/subtherapeutic group; The rate of change for FVC was significantly improved for the therapeutic group compared to the placebo/subtherapeutic group; 52.9% of patients in the therapeutic group showed an increase greater than or equal to12 for KSQ-L compared with 15.0% in the placebo/subtherapeutic group; and 64.7% of patients in the therapeutic group achieved response compared to 20.0% in the placebo/subtherapeutic group. aTyr is currently conducting EFZO-FIT, a global Phase 3 randomized, double-blind, placebo-controlled 52-week study to evaluate the efficacy and safety of 3.0 mg/kg and 5.0 mg/kg of efzofitimod in 264 patients with pulmonary sarcoidosis. The trial design incorporates a forced steroid taper. The primary endpoint of the study is steroid reduction. Secondary endpoints include measures of lung function and sarcoidosis symptoms.