aTyr Pharma Completes Global Enrollment For Pivotal Phase 3 Study

aTyr Pharma, Inc. (Nasdaq: ATYR), a biotechnology firm specializing in pioneering therapies from its unique tRNA synthetase platform, has announced the successful completion of patient recruitment for its Phase 3 EFZO-FIT™ trial. This global study focuses on evaluating efzofitimod, its leading candidate, in individuals with pulmonary sarcoidosis—a challenging form of interstitial lung disease with few treatment options. The trial surpassed its enrollment goal, involving 268 participants across 85 centers in 9 countries. Preliminary results are anticipated in the third quarter of 2025.

Sanjay S. Shukla, M.D., M.S., President and Chief Executive Officer of aTyr, commented “Completing enrollment in this landmark study is an important milestone that brings us one step closer to delivering a potentially groundbreaking treatment to address the significant unmet need for pulmonary sarcoidosis patients. We are grateful to all of the patients and their caregivers, our principal investigators and their teams, our many advocacy partners and our partner Kyorin Pharmaceutical Co., Ltd., who helped make this accomplishment possible. The historic number of patients enrolled in this study signifies the strong patient demand for a new treatment option such as efzofitimod.”

Efzofitimod is a novel therapy based on tRNA synthetase that targets activated myeloid cells via neuropilin-2, aiming to reduce inflammation without suppressing the immune system, and could potentially halt the advancement of fibrosis. It has been granted orphan drug status for sarcoidosis in the U.S., E.U., and Japan, and has also received Fast Track designation in the U.S. specifically for pulmonary sarcoidosis.

“This is a monumental achievement for the sarcoidosis community. It is by far the largest interventional study ever to be conducted in sarcoidosis. We expect the results of this trial to yield valuable insights that will inform sarcoidosis research and treatment in the years to come. We are optimistic based on the positive Phase 1b/2a results that efzofitimod could be a potentially transformative therapy for sarcoidosis patients, which is greatly needed. We look forward to the readout from this study in 2025,” said Daniel A. Culver, D.O., Chair of the Division of Pulmonary Medicine at The Cleveland Clinic and Lead Primary Investigator of the study.