On May 15, 2026 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, reported first quarter 2026 results and provided a corporate update.
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"2026 is off to a productive start, as we now have a clear path forward for efzofitimod in pulmonary sarcoidosis, a major form of interstitial lung disease (ILD), following our recent Type C meeting with the U.S. Food and Drug Administration (FDA)," said Sanjay S. Shukla, M.D., M.S., President and Chief Executive Officer of aTyr. "Based on feedback from the FDA, we plan to file an investigational new drug (IND) application next month for a new Phase 3 study in patients with chronic, symptomatic pulmonary sarcoidosis with restrictive lung disease utilizing forced vital capacity (FVC) as the primary endpoint of the study and the King’s Sarcoidosis Questionnaire (KSQ)-Lung score as a key secondary endpoint. We look forward to the continued advancement of efzofitimod in this form of ILD where there remains a high unmet medical need."
First Quarter 2026 and Subsequent Period Highlights
Announced plans to continue the development of efzofitimod in pulmonary sarcoidosis following a Type C meeting with the FDA to review the results of the Phase 3 EFZO-FIT study and determine the path forward for efzofitimod in pulmonary sarcoidosis. The Company plans to file an IND in June 2026 for a new Phase 3 study in patients with chronic, symptomatic pulmonary sarcoidosis with restrictive lung disease utilizing FVC as the primary endpoint of the study and the KSQ-Lung score as the key secondary endpoint. The Phase 3 trial is expected to be a global, randomized, double-blind, placebo-controlled study to evaluate the efficacy and safety of efzofitimod in patients with moderate to severe pulmonary sarcoidosis. The 54-week study will consist of two parallel cohorts randomized equally to either 5.0 mg/kg efzofitimod or placebo dosed intravenously once every 3 weeks for a total of 17 doses. The study is intended to enroll up to approximately 372 patients with symptomatic pulmonary sarcoidosis with restrictive lung disease who are receiving a stable dose of ≤ 5.0 mg daily oral corticosteroid and/or a background immunosuppressant. All background treatment will remain stable throughout the duration of the study. The primary endpoint of the study will be change from baseline in FVC at week 48 and the key secondary endpoint will be change from baseline in the KSQ-Lung score at week 48.
On track to complete enrollment in the Phase 2 EFZO-CONNECT study to evaluate the efficacy, safety and tolerability of efzofitimod in patients with limited or diffuse systemic sclerosis (SSc, or scleroderma)-related ILD (SSc-ILD) in the first half of 2026. 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. Promising interim data from the study were reported in the second quarter of 2025.
Poster related to the Company’s investigational new drug candidate, ATYR0101, accepted for presentation at the Extracellular Matrix Pharmacology Congress, which is scheduled to take place June 14 – 17, 2026 in Copenhagen, Denmark. The poster, which is titled, "Natural Asp-tRNA Synthetase Fragment Interacts with LTBP-1 on the ECM Promoting Myofibroblast Apoptosis and Reducing Fibrosis," presents research indicating that AYTR0101 selectively induces myofibroblast apoptosis via modulation of focal adhesion kinase (FAK) signaling through a novel binding interaction with latent-transforming growth factor beta binding protein 1 (LTBP-1) and results in a significant reduction of fibrosis in lung and kidney models. The poster will be available on the Company’s website once presented.
First Quarter 2026 Financial Highlights and Cash Position
Cash & Investment Position: Cash, cash equivalents, restricted cash and available-for-sale investments as of March 31, 2026, were $68.3 million.
R&D Expenses: Research and development expenses were $7.3 million for the first quarter 2026, which consisted primarily of costs for the Phase 3 EFZO-FIT and Phase 2 EFZO-CONNECT studies and research and development costs for the Company’s preclinical product candidates.
G&A Expenses: General and administrative expenses were $4.1 million for the first quarter 2026.
About Efzofitimod
Efzofitimod is a novel biologic immunomodulator in clinical development for the treatment of interstitial lung disease (ILD), a group of immune-mediated disorders that can cause inflammation and fibrosis, or scarring, of the lungs. Efzofitimod is a tRNA synthetase derived therapy that selectively modulates activated myeloid cells through neuropilin-2 to resolve inflammation without immune suppression and potentially prevent the progression of fibrosis. Efzofitimod is currently being investigated in the Phase 2 EFZO-CONNECT study in patients with systemic sclerosis (SSc, or scleroderma)-related ILD, and aTyr intends to submit an investigational new drug (IND) application in June 2026 for a global Phase 3 study of efzofitimod in patients with pulmonary sarcoidosis, a major form of ILD. These forms of ILD have limited therapeutic options and there is a need for safer and more effective, disease-modifying treatments that improve outcomes.
(Press release, aTyr Pharma, MAY 15, 2026, View Source [SID1234665786])