aTyr Pharma Plans June IND Submission for 372-Patient Phase 3 Efzofitimod Trial
aTyr Pharma will submit an IND in June 2026 for a Phase 3 trial of efzofitimod in 372 pulmonary sarcoidosis patients with restrictive lung disease. The 54-week study will use forced vital capacity as primary endpoint and KSQ-Lung score as secondary and dose 5 mg/kg every three weeks.
1. FDA Type C Meeting and Path Forward
aTyr Pharma engaged with FDA in a Type C meeting to review Phase 3 EFZO-FIT results and define next steps for efzofitimod in pulmonary sarcoidosis. The FDA agreed that forced vital capacity (FVC) is a direct measure of function and endorsed FVC as primary endpoint, while recommending additional validation of the King’s Sarcoidosis Questionnaire-Lung (KSQ-Lung) as a key secondary measure.
2. Phase 3 Trial Design and Endpoints
The planned global, randomized, double-blind, placebo-controlled Phase 3 study will enroll approximately 372 adult patients with chronic, symptomatic pulmonary sarcoidosis and restrictive lung disease. Patients will receive 5.0 mg/kg efzofitimod or placebo intravenously every three weeks for 17 doses over a 54-week period, with change in FVC at week 48 as the primary endpoint and KSQ-Lung score change at week 48 as key secondary endpoint.
3. IND Submission and Timeline
aTyr Pharma intends to submit an investigational new drug (IND) application for the trial in June 2026 and will host a conference call to discuss regulatory and clinical updates. Preparations include final protocol design, dosing strategy adjustments, and safety surveillance plans to support increased drug exposure without added safety concerns.