aTyr Pharma’s Efzofitimod Fails Primary Endpoint in Pulmonary Sarcoidosis Phase III Trial

aTyr Pharma’s Phase III EFZO-FIT trial (NCT05415137) evaluating efzofitimod in pulmonary sarcoidosis did not meet its primary endpoint. The therapy showed no significant change in mean daily oral corticosteroid (OCS) dose at week 48, with reductions of 2.79 mg for 5 mg/kg efzofitimod versus 3.52 mg for placebo. Complete steroid withdrawal occurred in 52.6% of efzofitimod-treated patients compared to 40.2% on placebo. Improvements were also observed in the King’s Sarcoidosis Questionnaire (KSQ)-Lung score, with 10.36 points for efzofitimod versus 6.19 for placebo, and 29.5% of patients achieved both KSQ-Lung improvement and steroid withdrawal versus 14.4% for placebo.

Despite the trial outcome, aTyr plans further discussions with the FDA, citing clinically meaningful trends across multiple endpoints. The 268-patient study underscores the continued unmet need in pulmonary sarcoidosis, where no new therapies have been FDA-approved since 1952.

22-09-2025