aTyr Pharma Await FDA Feedback for Pulmonary Sarcoidosis Study
SAN DIEGO, Sept. 01, 2026 /CourierPR/ -- aTyr Pharma, a clinical-stage biotechnology company developing first-in-class medicines from its proprietary tRNA synthetase platform, has provided an update on its ongoing efforts to advance efzofitimod, a novel biologic immunomodulator, for the treatment of pulmonary sarcoidosis. The company anticipates receiving feedback from the U.S. Food and Drug Administration (FDA) by mid-September 2026 regarding the protocol submitted in June 2026 for a planned Phase 3 study in patients with chronic, symptomatic pulmonary sarcoidosis characterized by restrictive lung disease.
The planned Phase 3 trial is a global, randomized, double-blind, placebo-controlled study designed to evaluate efzofitimod’s efficacy and safety in treating patients with moderate to severe pulmonary sarcoidosis. The 54-week study will consist of two parallel cohorts, each randomized equally to receive 5.0 mg/kg efzofitimod or placebo, administered intravenously once every three weeks for a total of 17 doses. The study aims to enroll up to approximately 372 patients who are currently receiving a stable dose of ≤5.0 mg daily oral corticosteroids and/or a background immunosuppressant, with all background treatment remaining stable throughout the study.
The primary endpoint of the study will be the change from baseline in forced vital capacity at week 48, while the key secondary endpoint will be the change from baseline in the King’s Sarcoidosis Questionnaire-Lung score at the same time point. This trial is part of a broader strategy to address the limited therapeutic options available for patients with interstitial lung disease (ILD), a group of immune-mediated disorders that can cause inflammation and progressive fibrosis of the lungs.
Efzofitimod, derived from aTyr’s tRNA synthetase platform, selectively modulates activated myeloid cells through neuropilin-2, aiming to resolve inflammation without immune suppression and potentially prevent fibrosis. The biologic is currently in Phase 2 clinical development for the treatment of ILD, including SSc-related ILD, and is now being investigated for its potential in pulmonary sarcoidosis.
According to aTyr, the urgency of this regulatory response stems from the critical need for safer and more effective, disease-modifying treatments that can improve outcomes for patients suffering from ILD. While the company remains optimistic about the potential of efzofitimod, the timeline for regulatory approval and the results of the Phase 3 study remain uncertain, as the process is subject to various risks and uncertainties, including interactions with the FDA and external factors beyond the company's control.