aTyr Expects FDA Response on Phase 3 Sarcoidosis Study

A mid-September 2026 FDA response to aTyr Pharma's Phase 3 protocol submission will determine whether the company can advance efzofitimod into a pivotal trial for chronic, symptomatic pulmonary sarcoidosis, a condition with few approved disease-modifying options. For regulatory affairs leads tracking orphan-indication review timelines, the roughly three-month turnaround from aTyr's June 2026 protocol submission offers a current reference point for FDA responsiveness on IND-stage rare disease programs.

The planned study is a global, randomized, double-blind, placebo-controlled design enrolling up to approximately 372 patients with moderate to severe pulmonary sarcoidosis with restrictive lung disease. Patients must be on a stable dose of ≤5.0 mg daily oral corticosteroid and/or a background immunosuppressant, with all background therapy held constant across the 54-week duration. Two parallel cohorts will receive either 5.0 mg/kg efzofitimod or placebo intravenously once every three weeks for 17 doses total.

The primary endpoint is change from baseline in forced vital capacity at week 48, with the King's Sarcoidosis Questionnaire-Lung score at week 48 designated as the key secondary endpoint. The endpoint selection reflects a regulatory preference for objective pulmonary function measures in ILD programs, alongside patient-reported outcome instruments that capture symptom burden, a pairing QA and clinical operations teams will need to build into data collection and site monitoring plans from the outset.

Efzofitimod is a tRNA synthetase-derived biologic that modulates activated myeloid cells via neuropilin-2, targeting inflammation resolution without broad immune suppression. The mechanism distinguishes it from corticosteroid-dependent regimens that dominate current sarcoidosis management. The asset is also under investigation in the Phase 2 EFZO-CONNECT study in systemic sclerosis-related ILD, meaning manufacturing, supply chain, and comparability considerations will span two concurrent clinical programs.

Receipt of the FDA response by mid-September 2026 will set the immediate timeline for protocol finalisation, site activation planning, and any required amendments before global enrolment opens.

Source: aTyr Pharma, Inc. via GlobeNewswire, 1 September 2026.