Efzofitimod

Results reported for Phase 3 EFZO-FIT™ study of efzofitimod in pulmonary sarcoidosis. Company plans to meet with the FDA to determine path forward for efzofitimod in pulmonary sarcoidosis in the first quarter of 2026. Company expects to complete enrollment in Phase 2 EFZO-CONNECT™ study of efzofitimod in systemic sclerosis-related interstitial lung disease (SSc-ILD) in the first half of 2026. Ended the third quarter 2025 with $92.9 million in cash, cash equivalents, restricted cash and investments. SAN DIEGO, Nov. 06, 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 third quarter 2025 results and provided a corporate update. “In September, we announced results from our Phase 3 EFZO-FIT™ study of efzofitimod in pulmonary sarcoidosis, a major form of interstitial lung disease (ILD). While we did not meet the primary endpoint, efzofitimod is the first investigational therapy to exhibit improvements in quality of life across multiple disease-related health outcomes while also reducing steroid burden in patients with pulmonary sarcoidosis,” said Sanjay S. Shukla, M.D., M.S., President and Chief Executive Officer of aTyr. “Treatment options for pulmonary sarcoidosis are limited and can include a significant reliance on the use of oral corticosteroids, which often have significant side effects. There remains an urgent need for a new safe and effective treatment for patients with chronic, symptomatic disease.” “Based on the findings from EFZO-FIT™, we believe there is drug activity for efzofitimod as evidenced by improvements across multiple clinically relevant efficacy measures. Based on this, coupled with the ongoing high unmet medical need in pulmonary sarcoidosis, we plan to meet with the U.S. Food and Drug Administration (FDA) in the first quarter of 2026 to review the results of the study and determine the path forward for efzofitimod in pulmonary sarcoidosis. We plan to provide an update regarding the next steps for the program following that meeting.” Third Quarter 2025 and Subsequent Period Highlights Reported results from the global Phase 3 EFZO-FIT™ study to evaluate the efficacy and safety of 3.0 mg/kg and 5.0 mg/kg of efzofitimod or placebo in 268 patients with pulmonary sarcoidosis. The study did not meet its primary endpoint of change from baseline in mean daily oral corticosteroid dose at week 48. Clinical benefit for 5.0 mg/kg efzofitimod was observed across multiple pre-specified study efficacy parameters at week 48 compared to placebo, including the King’s Sarcoidosis Questionnaire (KSQ)-Lung score, KSQ-General Health score, Fatigue Assessment Scale, and complete steroid withdrawal and improvement in KSQ-Lung score. Additionally, treatment with efzofitimod maintained lung function as a measure of forced vital capacity and was well-tolerated with a safety profile consistent with prior trials conducted to date. Based on these findings, the Company plans to meet with the FDA in the first quarter of 2026 to review the results and determine the path forward for efzofitimod in pulmonary sarcoidosis. Presented the results from the Phase 3 EFZO-FIT™ study in a late-breaking oral abstract at the European Respiratory Society Congress 2025, which took place September 27 – October 1, 2025, in Amsterdam, Netherlands. Daniel Culver, D.O., Chair of the Department of Pulmonary Medicine at the Cleveland Clinic and principal investigator of the study, delivered the presentation, titled “EFZO-FIT: The Largest Ever Interventional Trial in Pulmonary Sarcoidosis.” Enrollment is progressing 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). 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, and the Company expects to complete enrollment in the study in the first half of 2026. Presented at the Federation of European Biochemical Societies (FEBS) Special Meeting, “Expanding frontiers in aminoacyl-tRNA synthetase research,” which took place September 28 – October 3, 2025, in Dubrovnik, Croatia. Leslie Nangle, Ph.D., Vice President of Research at aTyr, delivered a presentation titled, “Advancing a therapeutic platform based on tRNA synthetases for treatment of fibrotic lung diseases.” Third Quarter 2025 Financial Highlights and Cash Position Cash & Investment Position: Cash, cash equivalents, restricted cash and available-for-sale investments as of September 30, 2025, were $92.9 million. R&D Expenses: Research and development expenses were $22.1 million for the third quarter 2025, which consisted primarily of clinical trial costs for the Phase 3 EFZO-FIT™ and Phase 2 EFZO-CONNECT™ studies, manufacturing costs for a potential Biologics License Application (BLA) filing and commercial supply for the efzofitimod program, and research and development costs for the efzofitimod and discovery programs. G&A Expenses: General and administrative expenses were $4.8 million for the third quarter 2025. 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. In addition to the global Phase 3 EFZO-FIT™ study of efzofitimod in patients with pulmonary sarcoidosis, a major form of ILD, efzofitimod is also being investigated in the Phase 2 EFZO-CONNECT™ study in patients with systemic sclerosis (SSc, or scleroderma)-related 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. About aTyr aTyr is a clinical stage biotechnology company leveraging evolutionary intelligence to translate tRNA synthetase biology into new therapies for fibrosis and inflammation. tRNA synthetases are ancient, essential proteins that have evolved novel domains that regulate diverse pathways extracellularly in humans. aTyr’s discovery platform is focused on unlocking hidden therapeutic intervention points by uncovering signaling pathways driven by its proprietary library of domains derived from all 20 tRNA synthetases. aTyr’s lead therapeutic candidate is efzofitimod, a novel biologic immunomodulator in clinical development for the treatment of interstitial lung disease, a group of immune-mediated disorders that can cause inflammation and progressive fibrosis, or scarring, of the lungs. For more information, please visit www.atyrpharma.com. Forward-Looking Statements This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements are usually identified by the use of words such as “aims,” “anticipates,” “believes,” “can,” “designed,” “expects,” “hopes,” “intends,” “look toward,” “may,” “plans,” “potential,” “project,” “will,” and variations of such words or similar expressions. We intend these forward-looking statements to be covered by such safe harbor provisions for forward-looking statements and are making this statement for purposes of complying with those safe harbor provisions. These forward-looking statements include, among others, statements regarding the potential therapeutic benefits and applications of efzofitimod; the potential for efzofitimod to improve patient quality of life across multiple disease related health outcomes in pulmonary sarcoidosis; the expected size of, and number of patients to be enrolled in the Phase 2 EFZO-CONNECT™ study; our belief that there is drug activity for efzofitimod; and timelines and plans with respect to certain development activities and development goals, including our plans to meet with the FDA in the first quarter of 2026 to determine the path forward for efzofitimod in pulmonary sarcoidosis as well as our expectations with respect to the outcome of that meeting and next steps for the development of efzofitimod in pulmonary sarcoidosis, and our expectation that our Phase 2 EFZO-CONNECT™ study of efzofitimod in patients with SSc-ILD will complete enrollment in the first half of 2026. These forward-looking statements also reflect our current views about our plans, intentions, expectations, strategies and prospects, which are based on the information currently available to us and on assumptions we have made. Although we believe that our plans, intentions, expectations, strategies and prospects, as reflected in or suggested by these forward-looking statements, are reasonable, we can give no assurance that the plans, intentions, expectations, strategies or prospects will be attained or achieved. All forward-looking statements are based on estimates and assumptions by our management that, although we believe to be reasonable, are inherently uncertain. Furthermore, actual results may differ materially from those described in these forward-looking statements and will be affected by a variety of risks and factors that are beyond our control including, without limitation, risks related to our reliance on third-party partners and the potential that such partners may not perform as anticipated, the fact that NRP2 and tRNA synthetase biology is not fully understood, uncertainty regarding the ultimate long-term impact of evolving macroeconomic and geopolitical conditions, the risks inherent in using the results from the EFZO-FIT™ study to pursue FDA approval for efzofitimod in pulmonary sarcoidosis, the risk of delays in our clinical trials, risks associated with the discovery, development and regulation of our product candidates, including the uncertainty of related costs and regulatory filings and the risk that results from clinical trials or other studies may not support further development, the risk that we may cease or delay preclinical or clinical development activities for any of our existing or future product candidates for a variety of reasons, the fact that our collaboration agreements are subject to early termination, and the risk that we may not be able to raise the additional funding required for our business and product development plans, as well as those risks set forth in our most recent Annual Report on Form 10-K, Quarterly Reports on Form 10-Q and in our other SEC filings. Except as required by law, we assume no obligation to update publicly any forward-looking statements, whether as a result of new information, future events or otherwise. Contact: Ashlee Dunston Sr. Director, Investor Relations and Public Affairs adunston@atyrpharma.com Source: aTyr Pharma, Inc.

Clinical benefit for efzofitimod across multiple disease-related health outcomes reported. Improvement in Fatigue Assessment Scale Total Score at week 48 observed in the 5.0 mg/kg efzofitimod treatment group vs placebo (p=0.0226). Improvement in King’s Sarcoidosis Questionnaire-General Health score at week 48 observed in the 5.0 mg/kg efzofitimod treatment group vs placebo (p=0.0197). SAN DIEGO, Sept. 30, 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 presented additional findings from the Phase 3 EFZO-FIT™ study of efzofitimod in 268 patients with pulmonary sarcoidosis, a major form of interstitial lung disease, in a late-breaking oral abstract presentation by Daniel Culver, D.O., Chair of the Department of Pulmonary Medicine at the Cleveland Clinic and principal investigator of the study, at the European Respiratory Society (ERS) Congress 2025 in Amsterdam, Netherlands. “Building on the positive data we presented in the topline results for the King’s Sarcoidosis Questionnaire (KSQ)-Lung score, the clinical improvements in the Fatigue Assessment Scale (FAS) and KSQ-General Health scores presented at ERS are compelling because these directly address critical symptoms and quality of life measures that profoundly impact pulmonary sarcoidosis patients,” said Sanjay S. Shukla, M.D., M.S., President and Chief Executive Officer of aTyr Pharma. “Crucially, the improvements observed for the KSQ-Lung, FAS and KSQ-General Health scores were achieved rapidly and sustained robustly throughout the study. Despite missing the primary endpoint of the study, efzofitimod has clearly demonstrated the potential to durably improve multiple disease-related health outcomes, including cough, shortness of breath, fatigue and general health. These consistent clinical benefits reinforce our belief in efzofitimod’s potential to meaningfully improve quality of life and reduce reliance on chronic steroids for pulmonary sarcoidosis patients.” The ERS presentation included analyses of additional pre-specified outcomes that demonstrated clinical improvements in mean change from baseline in the FAS Total Score (p=0.0226) and KSQ-General Health score (p=0.0197) in patients treated with 5.0 mg/kg efzofitimod vs placebo. Treatment with efzofitimod was also associated with a trend toward a greater proportion of patients achieving steroid-free status for at least six months. As previously reported in the topline results, the study did not meet its primary endpoint of change from baseline in mean daily oral corticosteroid dose at week 48. However, clinical benefit for 5.0 mg/kg efzofitimod was observed across multiple study efficacy parameters at week 48 compared to placebo, including complete steroid withdrawal and KSQ-Lung score improvement (p=0.0196), improvement in KSQ-Lung score change from baseline (p=0.0479), preservation of forced vital capacity and a well-tolerated safety profile. Note, the study’s statistical analysis plan was designed on a hierarchical assessment basis. Since the primary endpoint was not met, all subsequent statistical testing is reported as nominal findings. Details of the ERS presentation are as follows: Title: EFZO-FIT: The Largest Ever Interventional Trial in Pulmonary Sarcoidosis Authors: Daniel Culver, Francesco Bonella, Lisa Carey, Pavithra Ramesh, Abhijeeth Chandrasekaran, Nelson Kinnersley, Vis Niranjan, Robert Baughman Presenter: Daniel Culver, D.O., Chair of the Department of Pulmonary Medicine, Cleveland Clinic Presentation Number: RCT5337 Session: 436 Clinical Trials Session, ALERT 3: Interstitial Lung Disease, Pulmonary Hypertension and Intensive Care Unit The corresponding abstract is available on the ERS conference website for registered attendees. The ERS presentation will be available on the aTyr website once presented. About Pulmonary Sarcoidosis Pulmonary sarcoidosis is an inflammatory disease characterized by the formulation of granulomas, clumps of inflammatory cells, in one or more organs of the body. Approximately 200,000 Americans live with pulmonary sarcoidosis and the prognosis ranges from benign and self-limiting to chronic, debilitating disease, permanent loss of lung function and death. Current treatment options include corticosteroids and other immunosuppressive therapies, which have limited efficacy and are associated with serious side-effects that many patients cannot tolerate long-term. About Efzofitimod Efzofitimod is a first-in-class 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. In addition to the global Phase 3 EFZO-FIT™ study of efzofitimod in patients with pulmonary sarcoidosis, a major form of ILD, efzofitimod is also being investigated in the Phase 2 EFZO-CONNECT™ study in patients with systemic sclerosis (SSc, or scleroderma)-related 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. About aTyr aTyr is a clinical stage biotechnology company leveraging evolutionary intelligence to translate tRNA synthetase biology into new therapies for fibrosis and inflammation. tRNA synthetases are ancient, essential proteins that have evolved novel domains that regulate diverse pathways extracellularly in humans. aTyr’s discovery platform is focused on unlocking hidden therapeutic intervention points by uncovering signaling pathways driven by its proprietary library of domains derived from all 20 tRNA synthetases. aTyr’s lead therapeutic candidate is efzofitimod, a first-in-class biologic immunomodulator in clinical development for the treatment of interstitial lung disease, a group of immune-mediated disorders that can cause inflammation and progressive fibrosis, or scarring, of the lungs. For more information, please visit www.atyrpharma.com. Forward-Looking Statements This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements are usually identified by the use of words such as "anticipate," “believes,” “can,” “could,” “designed,” “expects,” “intends,” “may,” “plans,” “potential,” “upcoming,” “will,” and variations of such words or similar expressions. We intend these forward-looking statements to be covered by such safe harbor provisions for forward-looking statements and are making this statement for purposes of complying with those safe harbor provisions. These forward-looking statements include, among others, statements regarding the potential therapeutic benefits and applications of efzofitimod; the potential for efzofitimod to improve multiple disease related health outcomes and reduce reliance on chronic steroids for treatment; timelines and plans with respect to certain development activities (such as the timing of data from clinical trials); and certain development goals. These forward-looking statements also reflect our current views about our plans, intentions, expectations, strategies and prospects, which are based on the information currently available to us and on assumptions we have made. Although we believe that our plans, intentions, expectations, strategies and prospects, as reflected in or suggested by these forward-looking statements, are reasonable, we can give no assurance that the plans, intentions, expectations, strategies or prospects will be attained or achieved. All forward-looking statements are based on estimates and assumptions by our management that, although we believe to be reasonable, are inherently uncertain. Furthermore, actual results may differ materially from those described in these forward-looking statements and will be affected by a variety of risks and factors that are beyond our control including, without limitation, uncertainty regarding geopolitical and macroeconomic events, risks associated with the discovery, development and regulation of efzofitimod, the risk that we or our partners may cease or delay preclinical or clinical development activities for efzofitimod for a variety of reasons (including difficulties or delays in patient enrollment in planned clinical trials), the possibility that existing collaborations could be terminated early, and the risk that we may not be able to raise the additional funding required for our business and product development plans, as well as those risks set forth in our most recent Annual Report on Form 10-K, Quarterly Reports on Form 10-Q and in our other SEC filings. Except as required by law, we assume no obligation to update publicly any forward-looking statements, whether as a result of new information, future events or otherwise. Source: aTyr Pharma, Inc.