Efzofitimod - (EFZO-FIT)

Sept. 24, 2026: aTyr Pharma (NASDAQ: ATYR) said it reached alignment with the FDA on the protocol for a planned Phase 3 trial of efzofitimod in chronic, symptomatic pulmonary sarcoidosis with restrictive lung disease. The company expects to begin study-related activities in Q4 2026 and focus on regulatory submissions in the U.S. and Europe.

The global, randomized, double-blind, placebo-controlled 54-week study will enroll about 372 patients with moderate to severe pulmonary sarcoidosis receiving stable background therapy. Patients will be randomized to efzofitimod 5.0 mg/kg or placebo, given IV every 3 weeks for 17 doses. The primary endpoint is change from baseline in forced vital capacity (FVC) at week 48; the key secondary endpoint is change from baseline in King’s Sarcoidosis Questionnaire-Lung score at week 48.

aTyr said the design is supported by a subgroup analysis from the Phase 3 EFZO-FIT study showing clinically meaningful FVC benefit and improvements in multiple patient-reported outcomes, including KSQ-Lung, versus placebo in patients with restrictive lung disease; these data were presented at the WASOG 2026 Congress.

Efzofitimod is also being studied in the Phase 2 EFZO-CONNECT trial in systemic sclerosis-related ILD. The company said further Phase 3 development will require additional capital.