aTyr Pharma Announces First Quarter 2025 Results and Provides Corporate Update

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aTyr Pharma, Inc.
aTyr Pharma, Inc.

Topline data from Phase 3 EFZO-FIT™ study of efzofitimod in pulmonary sarcoidosis on track for the third quarter of 2025.

Blinded baseline demographics and disease characteristics from Phase 3 EFZO-FIT™ study and current U.S. epidemiology and treatment practices for pulmonary sarcoidosis to be presented at ATS 2025.

SAN DIEGO, May 07, 2025 (GLOBE NEWSWIRE) -- aTyr Pharma, Inc. (Nasdaq: ATYR) (“aTyr” or the “Company”), a clinical stage biotechnology company engaged in the discovery and development of first-in-class medicines from its proprietary tRNA synthetase platform, today announced first quarter 2025 results and provided a corporate update.

“Throughout the first quarter of 2025 we have maintained strong and steady progress with our clinical program for efzofitimod in interstitial lung disease (ILD),” said Sanjay S. Shukla, M.D., M.S., President and Chief Executive Officer of aTyr. “The program’s cornerstone is our Phase 3 EFZO-FIT™ study in pulmonary sarcoidosis, the most prevalent form of ILD for which no new treatments have been approved in more than 70 years. We look forward to reporting topline data from this study next quarter with the hopes of advancing standard of care beyond oral corticosteroids for sarcoidosis patients.”

First Quarter 2025 and Subsequent Period Highlights

  • On track to announce topline data in the third quarter of 2025 from the global pivotal Phase 3 EFZO-FIT™ study to evaluate the efficacy and safety of efzofitimod in patients with pulmonary sarcoidosis. This is a randomized, double-blind, placebo-controlled, 52-week study consisting of three parallel cohorts randomized equally to either 3.0 mg/kg or 5.0 mg/kg of efzofitimod or placebo administered intravenously monthly for a total of 12 doses. The study enrolled 268 patients with pulmonary sarcoidosis at 85 centers in nine countries. The trial design incorporates a forced steroid taper. The primary endpoint of the study is steroid reduction measured as the absolute change from baseline to week 48. Secondary endpoints include measures of sarcoidosis symptoms and lung function. Patients who complete the study and wish to receive treatment with efzofitimod outside of the clinical trial are eligible to participate in an Individual Patient Expanded Access Program.

  • Enrollment ongoing in the Phase 2 EFZO-CONNECT™ study to evaluate the efficacy, safety and tolerability of efzofitimod in patients with limited and diffuse systemic sclerosis (SSc, or scleroderma)-related ILD (SSc-ILD). This proof-of-concept study is a randomized, double-blind, placebo-controlled, 28-week study consisting of three parallel cohorts randomized 2:2:1 to either 270 mg or 450 mg of efzofitimod or placebo administered intravenously monthly for a total of six doses. The study intends to enroll up to 25 patients at multiple centers in the United States. Patients who complete the study are eligible to participate in a 24-week open-label extension. Interim data from the study are expected in the second quarter of 2025 and will focus on skin assessments measured at baseline and week 12 in approximately eight patients, including patients in both the efzofitimod and placebo arms. The data will include skin histopathology, including immune biomarkers, and the modified Rodnan skin score.

  • Appointed Dalia R. Rayes as Head of Commercial, Global Efzofitimod Franchise. Ms. Rayes has over 25 years of experience building and leading commercial organizations at biotechnology and pharmaceutical companies, including leading rare disease product launches. She will serve as a member of the Company’s executive leadership team, overseeing global commercial strategy and operations for the efzofitimod program in ILD, including its lead indication in pulmonary sarcoidosis.

  • Posters for efzofitimod accepted for presentation at the upcoming American Thoracic Society (ATS) 2025 International Conference. The conference is scheduled to take place May 16 – 21, 2025, in San Francisco, CA.

    • Poster 9320 – Real-World Treatment Patterns Among Pulmonary Sarcoidosis Patients with Parenchymal Involvement in the US on Sunday May 18, 2025, at 11:30 a.m. PDT.

    • Poster 6808 – EFZO-FIT, Largest Placebo-Controlled Trial in Pulmonary Sarcoidosis – Trial Design and Patient Characteristics on Monday, May 19, 2025, at 11:30 a.m. PDT.

    • Poster 9092 – Incidence, Prevalence, and Mortality of Pulmonary Sarcoidosis with Parenchymal Involvement in the US on Tuesday May 20, 2025, at 11:30 a.m. PDT.

  • Presented preclinical data for its neuropilin-2-targeting antibody ATYR2810 at the American Association for Cancer Research (AACR) Annual Meeting 2025. The findings from the research, which was conducted in collaboration with Michael Lim, M.D., Chair of the Department of Neurosurgery at Stanford Medicine, demonstrate ATYR2810’s ability to enhance anti-tumor activity and increase survival in a model of glioblastoma multiforme, a primary form of brain cancer.