ATYR

Company awaiting comments from FDA by the end of August 2026 on protocol submitted for planned Phase 3 study of efzofitimod in patients with pulmonary sarcoidosis. Workforce reduction of approximately 60% and program prioritization conserves capital and aligns resources on efzofitimod program in ILD. Enrollment completed in Phase 2 EFZO-CONNECT™ study of efzofitimod in SSc-ILD; topline results expected in the first quarter of 2027. Ended the second quarter 2026 with $58.9 million in cash, cash equivalents, restricted cash and investments; cash runway into late 2028 based on current operations. SAN DIEGO, Aug. 07, 2026 (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 second quarter 2026 results and a corporate restructuring to prioritize its efzofitimod program in interstitial lung disease (ILD), including pulmonary sarcoidosis and systemic sclerosis (SSc)-related ILD (SSc-ILD). The program prioritization aims to focus the Company’s resources on advancing its lead asset, efzofitimod, and targeted pipeline development to conserve capital in anticipation of receiving comments from the U.S. Food and Drug Administration (FDA) on the protocol for its planned Phase 3 study of efzofitimod in pulmonary sarcoidosis, a major form of ILD. The Company submitted the protocol in June 2026 and is expecting feedback from the FDA by the end of August 2026. “We are proactively taking decisive, necessary action to focus our resources on our lead therapeutic candidate, efzofitimod, as we await feedback from the FDA on the protocol we submitted for our planned Phase 3 study in pulmonary sarcoidosis patients with restrictive lung disease. This approach positions aTyr to advance this planned Phase 3 study efficiently and continue completing the Phase 2 EFZO-CONNECT™ study in SSc-ILD,” said Sanjay S. Shukla, M.D., M.S., President and Chief Executive Officer of aTyr. “We remain confident in the potential of efzofitimod to become a meaningful therapy for patients with these forms of ILD, and these changes are essential to our ability to achieve that goal. We are deeply grateful to our dedicated team members for their outstanding contributions, commitment, and perseverance, including those who have helped advance tRNA synthetase biology over the years.” Program Prioritization and Corporate Restructuring Workforce reduction of approximately 60% will align organizational resources to support the efzofitimod program in ILD in anticipation of comments from the FDA on a protocol submitted for a planned Phase 3 study in pulmonary sarcoidosis and to complete the Phase 2 EFZO-CONNECT™ study in SSc-ILD. Jill Broadfoot, aTyr’s Chief Financial Officer (CFO), will step down as of September 30, 2026, and transition to serve as a consultant to the Company. Brandon Yaras, aTyr’s Vice President of Finance, will be appointed as CFO as of October 1, 2026. Nancy Denyes, aTyr’s General Counsel, will step down as of September 30, 2026, and transition to serve as a consultant to the Company. The Company expects that this restructuring and additional cost saving measures will reduce annualized operating expenses by approximately $13 million, beginning in the fourth quarter of 2026. Second Quarter 2026 and Subsequent Period Highlights Protocol submitted to FDA in June 2026 for planned Phase 3 study in patients with chronic, symptomatic pulmonary sarcoidosis with restrictive lung disease. The Company is expecting feedback from the FDA by the end of August 2026. 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 forced vital capacity (FVC) at week 48 and the key secondary endpoint will be change from baseline in the King’s Sarcoidosis Questionnaire-Lung score at week 48. Enrollment completed in the Phase 2 EFZO-CONNECT™ study to evaluate the efficacy, safety and tolerability of efzofitimod in patients with limited or diffuse SSc-ILD. Topline results are expected in the first quarter of 2027. 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 enrolled 23 patients at multiple centers in the United States. Promising interim data from the study were reported in the second quarter of 2025. Post hoc analysis of Phase 3 EFZO-FIT™ study in subgroup of patients with restrictive lung disease presented in a poster at the World Association of Sarcoidosis and Other Granulomatous Disorders (WASOG) 2026 Congress in Porto, Portugal. The poster, which is titled, “Evaluating Efzofitimod in a Subset of Sarcoidosis with the Restrictive Phenotype,” demonstrated clinically meaningful benefit for FVC and improvement in multiple patient-reported outcomes for patients treated with 5.0 mg/kg efzofitimod compared to placebo. The poster is available on the Company’s website. Second Quarter 2026 Financial Highlights and Cash Position Cash & Investment Position: Cash, cash equivalents, restricted cash and available-for-sale investments as of June 30, 2026, were $58.9 million. Based on its current cash and new operating expense forecast and plans, the Company anticipates that this cash position will be sufficient to fund the Company’s current operations into late 2028. Future development of efzofitimod in the planned Phase 3 study in pulmonary sarcoidosis will require the Company to obtain additional capital through equity or debt offerings, grant funding, collaborations, strategic partnerships and/or licensing arrangements. R&D Expenses: Research and development expenses were $6.7 million for the second quarter 2026, which consisted primarily of costs for the Phase 2 EFZO-CONNECT™ study 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 second 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 recently submitted a protocol to the FDA 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. 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,” “suggest,” “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 timelines and benefits with respect to our workforce reduction and program prioritization, including the resulting conservation of capital, reduction in operating expenses, ability to timely and efficiently advance our planned Phase 3 study of efzofitimod in pulmonary sarcoidosis and ability to complete the Phase 2 EFZO-CONNECT study in SSc-ILD; our new operating expense forecast and plans and the resulting cash runway sufficiency into late 2028 based on current operations; expected cash needs to develop efzofitimod in our planned Phase 3 study in pulmonary sarcoidosis and the means of raising such capital, including through equity or debt offerings, grant funding, collaborations, strategic partnerships and/or licensing arrangements; the potential therapeutic benefits and applications of efzofitimod; timelines and plans with respect to certain development activities and development goals, including the potential receipt of comments from the FDA by late August 2026 on a protocol submitted to the FDA in June 2026 for a Phase 3 study of efzofitimod in pulmonary sarcoidosis; the proposed design of our planned Phase 3 study of efzofitimod in pulmonary sarcoidosis, including the dosing regimen, enrollment expectations, targeted endpoints, and strategy to focus on a more limited patient population; our interpretation of the results of the Phase 3 EFZO-FIT™ study and the meaning of those interpretations for our planned Phase 3 study; and our expectation that we will report topline results from the Phase 2 EFZO-CONNECT™ study in the first quarter of 2027. 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 related to interactions with the FDA in general, 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 associated with targeting a more limited patient population in our planned Phase 3 study of efzofitimod in pulmonary sarcoidosis, the risk of delays in our clinical trials, risks associated with the discovery, development and regulation of our existing or future 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 (including difficulties or delays in patient enrollment in planned clinical trials), the fact that our collaboration agreements are subject to early termination, the risk that our assumptions and forecasts with respect to our workforce reduction and program prioritization may be materially inaccurate, the risk that our existing cash resources may be insufficient to fund our current operations for as long as anticipated due to changes in our operating plans, unanticipated expenses or other factors, 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.

Company to continue development of efzofitimod in pulmonary sarcoidosis following Type C meeting with the FDA. Company plans to submit an IND in June 2026 for a Phase 3 study of efzofitimod in patients with chronic, symptomatic pulmonary sarcoidosis with restrictive lung disease utilizing FVC as primary endpoint and KSQ-Lung as key secondary endpoint. On track to complete enrollment in Phase 2 EFZO-CONNECT™ study of efzofitimod in SSc-ILD in the first half of 2026. Ended the first quarter 2026 with $68.3 million in cash, cash equivalents, restricted cash and investments. SAN DIEGO, May 15, 2026 (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 2026 results and provided a corporate update. “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. 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,” “suggest,” “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 and ATYR0101; timelines and plans with respect to certain development activities and development goals, including the submission (and planned timing of submission) of an IND for a Phase 3 study of efzofitimod in pulmonary sarcoidosis in June 2026, the proposed design of our planned Phase 3 study of efzofitimod in pulmonary sarcoidosis, including the dosing regimen, enrollment expectations, targeted endpoints, and strategy to focus on a more limited patient population; our interpretation of the results of the Phase 3 EFZO-FIT™ study and the meaning of those interpretations for our planned Phase 3 study; the expected size and number of patients to be enrolled in the Phase 2 EFZO-CONNECT™ study; and our expectation that the EFZO-CONNECT™ study 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, uncertainty related to interactions with the FDA in general, 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 associated with targeting a more limited patient population in our planned Phase 3 study of efzofitimod in pulmonary sarcoidosis, the risk of delays in our clinical trials, risks associated with the discovery, development and regulation of our existing or future 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 (including difficulties or delays in patient enrollment in planned clinical trials), 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.

Efzofitimod’s global Phase 3 EFZO‑FIT narrowly missed its primary steroid‑reduction endpoint because placebo steroid withdrawal was higher than expected, but the 5 mg/kg dose showed statistically significant quality‑of‑life improvements (KSQ lung), a positive steroid‑free+KSQ composite with dose response, preserved FVC versus placebo, and a favorable safety profile. aTyr has an FDA Type C meeting in mid‑April to review EFZO‑FIT; management says a filing would be “very challenging” after the primary miss and is preparing for the possibility the FDA will request another study, with any future trial likely to emphasize quality‑of‑life and pulmonary function endpoints by patient phenotype. The company’s platform leverages tRNA synthetase fragments to modulate innate immunity in inflammatory ILDs, is advancing ATYR0101 as a potential anti‑fibrotic and completing the Phase 2 EFZO‑CONNECT scleroderma‑ILD study, and reported roughly $80 million in cash at year‑end. Interested in aTyr Pharma, Inc.? Here are five stocks we like better. aTyr Pharma (NASDAQ:ATYR) CEO Sanjay Shukla outlined the company’s strategy to develop new therapies for inflammation and fibrosis based on tRNA synthetase biology during a presentation at the Leerink Partners Global Healthcare Conference. Shukla focused primarily on efzofitimod, the company’s lead program, including results from a global Phase 3 study in pulmonary sarcoidosis and the upcoming U.S. FDA Type C meeting planned for mid-April. Shukla said aTyr is translating biology related to tRNA synthetases—enzymes historically known for their role in attaching amino acids to tRNA—into a “new class of medicines.” He said the company’s research has identified that these enzymes can break into fragments that interact with the immune system, creating an opportunity to develop therapies that modulate inflammation and fibrosis. → Microsoft Positioned to Win AI Race With Dual-Model Strategy He positioned the approach as differentiated and focused on leveraging what he described as “evolutionary intelligence” embedded in human biology. aTyr’s near-term development focus is on interstitial lung diseases (ILDs) that have a significant inflammatory component, including sarcoidosis and scleroderma-related ILD, with a longer-term ambition to potentially expand into more fibrotic settings. Efzofitimod, a tRNA synthetase fragment-based therapy, is being advanced for ILD, with an emphasis on inflammatory phenotypes. Shukla described ILDs as chronic inflammatory diseases that can progress to fibrosis, worsen lung function, and significantly impair quality of life. He also noted that some fibrotic lung diseases have survival rates worse than many common cancers and said existing therapies are often “toxic, not disease modifying or both.” → FuelCell Energy Is Burning Cash Faster Than It’s Building Momentum According to Shukla, efzofitimod is designed to modulate innate immunity at the site of inflammation, primarily by interacting with myeloid cells such as macrophages. He said the drug works upstream through the Neuropilin-2 receptor on macrophages, nudging these cells toward a “less inflammatory phenotype” and downregulating pro-inflammatory and pro-fibrotic signals. He emphasized that the company views the mechanism as restorative and not associated with overt immunosuppression. Shukla also referenced a publication in Science Translational Medicine that he described as a landmark paper on the mechanism of action. → Why This Defense ETF Could Keep Rallying as the Iran Conflict Escalates Shukla reviewed topline data presented in September in Amsterdam from EFZO-FIT, a global Phase 3 trial in pulmonary sarcoidosis conducted at about 90 centers across nine countries. The study compared efzofitimod at 5 mg/kg and 3 mg/kg versus placebo, enrolling 268 patients with a target of about 88 per arm. Patients entered the trial on a stable prednisone regimen (7.5 mg to 25 mg), and the protocol included a rigorous, every-two-week steroid taper based on patient global assessment. He said discontinuations were low across arms, supporting statistical power. He also highlighted that baseline characteristics were largely balanced, and he pointed to the inclusion of Black patients as evidence that sarcoidosis trials can enroll diverse populations. Participants had longstanding disease (roughly 7–9 years duration on average), and about one-third had extrapulmonary involvement. Average steroid dose at baseline was around 10 mg, he said. The primary endpoint was steroid reduction. Shukla said efzofitimod showed nearly 80% reductions in steroid use, but the trial did not meet the primary endpoint because placebo patients reduced steroids more than expected, resulting in overlapping curves. He said the company “just missed the p-value by a couple patients,” attributing the outcome to a roughly 40% placebo steroid withdrawal rate that exceeded assumptions used in trial planning. Despite the primary endpoint miss, Shukla argued the study generated learnings that could affect clinical practice, including how aggressively patients may be tapered off steroids. He relayed an anecdote from a pulmonologist who said she began taking about 40% of her patients off steroids after returning from the European Respiratory Society meeting. Shukla emphasized what he characterized as clear drug activity on quality of life measures. He highlighted results on the King’s Sarcoidosis Questionnaire (KSQ) lung domain, describing a statistically significant improvement for the 5 mg/kg group that emerged around week eight and was maintained across time points. He said other pre-specified measures, including Fatigue Assessment Score (FAS) and KSQ general health, showed consistent and durable benefits, particularly at the higher dose. He also pointed to pre-specified composite analyses combining steroid withdrawal with symptom improvement. Specifically, he highlighted a composite of being steroid-free and achieving more than an eight-point improvement in KSQ lung, which he said showed a statistically significant result and dose response. Shukla said patients on 5 mg/kg were “more than 2.5x more likely” to be steroid-free and significantly improved versus placebo. On lung function, Shukla discussed forced vital capacity (FVC), noting that in an aggressive steroid taper setting the expectation would typically be for FVC to decline as prednisone is withdrawn. He said large declines were not observed and characterized the data as suggesting nominal declines with efzofitimod, while placebo showed about a 2%–3% decline. He also said sarcoidosis includes heterogeneous phenotypes and suggested that a more restrictive population might show greater benefit—an area he said is part of discussions with regulators. Regarding safety, Shukla described efzofitimod as well tolerated, with low serious adverse events and few adverse events leading to discontinuation over one year, and no apparent dose-related safety signal. He also discussed anti-drug antibodies, stating the company did not observe concerning “treatment-boosted” effects. Shukla said aTyr has an FDA Type C meeting scheduled for mid-April to review EFZO-FIT results and determine the path forward in sarcoidosis. In Q&A, he said the company believes the program is viable, citing pre-specified analyses showing activity, including steroid weaning alongside improved patient-reported outcomes and preserved lung function. However, he also said filing based on the existing dataset would be “very challenging” because the primary endpoint was not met. Shukla said the company is preparing for the possibility that FDA may request another study and indicated that any future late-stage trial could emphasize quality of life and potentially pulmonary function, with careful selection of endpoints by patient phenotype (e.g., FEV1, FVC, DLCO). He said the company has built a large natural history database in sarcoidosis and has not presented post-hoc analyses publicly. On communications, Shukla said the company expects to wait for written FDA correspondence before updating investors, citing the current environment at the agency. He suggested feedback typically arrives about four weeks after the meeting, while noting conditions may not be typical. Beyond efzofitimod, Shukla highlighted ATYR0101, another tRNA synthetase fragment that he said modulates myofibroblasts and could have utility in fibrosis. He said he anticipates that program could enter the clinic next year and described it as potentially “a true anti-fibrotic.” Shukla also provided an update on the ongoing Phase 2 EFZO-CONNECT study in scleroderma-related ILD. He said the company expects to complete enrollment in the first half of the year and expects data roughly six to seven months later. Later in the presentation, he described the scleroderma-ILD trial as a six-month study evaluating fixed doses intended to approximate the weight-based Phase 3 sarcoidosis dosing. He said an interim finding based on early unblinding suggested three of four patients with diffuse skin disease had responded at three months, and he noted that he believes no drug has previously improved skin symptoms in scleroderma. He said enrollment was expected to finish in the first half of 2026, with results anticipated toward the end of the year. Shukla said the company reported just over $80 million in cash as of year-end and described aTyr as being in a strong position to complete ongoing work and prepare for the upcoming FDA interaction. aTyr Pharma, Inc is a clinical-stage biotechnology company focused on the discovery and development of novel protein-based therapies that modulate the extracellular matrix and immune pathways. Headquartered in San Diego, California, the company applies its proprietary Extracellular Modulation® platform to identify and optimize biologic candidates for the treatment of rare and serious diseases, including pulmonary sarcoidosis, muscular dystrophy, and solid tumors. The company's research efforts are centered on harnessing extracellular proteins to regulate tissue remodeling, cell adhesion, and immune signaling. The article "aTyr Pharma Spotlights Efzofitimod Phase 3 Learnings, Eyes Mid-April FDA Type C Meeting" was originally published by MarketBeat.

Company scheduled to meet with the FDA in mid-April 2026 to review the results of the Phase 3 EFZO-FIT™ study and determine the path forward for efzofitimod in pulmonary sarcoidosis. Phase 2 EFZO-CONNECT™ study of efzofitimod in systemic sclerosis-related interstitial lung disease (SSc-ILD) on track to complete enrollment in the first half of 2026. Ended 2025 with $80.9 million in cash, cash equivalents, restricted cash and investments. SAN DIEGO, March 05, 2026 (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 fourth quarter and full year 2025 results and provided a corporate update. “In 2025 we announced results from our Phase 3 EFZO-FIT™ study of efzofitimod in pulmonary sarcoidosis, a major form of interstitial lung disease (ILD) where a significant proportion of patients develop chronic or progressive disease with debilitating symptoms despite current treatment options. This marked an important milestone, not only for the broader sarcoidosis community, but also for aTyr, as it was the Company’s largest and first Phase 3 study of a tRNA synthetase-derived therapy generated from our platform,” said Sanjay S. Shukla, M.D., M.S., President and Chief Executive Officer of aTyr. “We are ready and look forward to engaging with the U.S. Food and Drug Administration (FDA) in mid-April 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 the receipt of the official FDA meeting minutes.” Fourth Quarter 2025 and Subsequent Period Highlights Announced the scheduling of a Type C meeting with the FDA in mid-April 2026 to review the results of the Phase 3 EFZO-FIT™ study and determine the path forward for efzofitimod in pulmonary sarcoidosis. The Company expects to provide an update regarding the outcome of the meeting following the receipt of the official meeting minutes. EFZO-FIT™ was a Phase 3 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 symptomatic 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 (p=0.0479), Fatigue Assessment Scale score (p=0.0226), KSQ-General Health score (p=0.0197), and complete steroid withdrawal with KSQ-Lung score improvement (p=0.0196). Additionally, treatment with efzofitimod maintained lung function as measured by forced vital capacity and was well-tolerated with a safety profile consistent with prior trials conducted to date. 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. Presented a poster related to its investigational new drug candidate, ATYR0101, at the Keystone Symposia on Fibrosis: Cross Organ Pathology and Pathways to Clinical Development. The poster demonstrated that subcutaneous delivery of ATYR0101 yielded a comparable pharmacokinetic and immunogenicity profile, which is favorable to other delivery methods, while reducing lung inflammation. The findings presented in the poster suggest the ability of ATYR0101 to potentially resolve the cycle of chronic inflammation and fibrosis utilizing a novel mechanism and further support a compelling therapeutic profile for patients suffering from fibrosis. The poster is available on the Company’s website. Published an article demonstrating the generation of a functional neuropilin-2 (NRP2)/plexinA1 (PLXNA1) bispecific antibody in the Journal of Biological Chemistry. The publication, entitled, “A bispecific antibody designed to act as a NRP2/PLXNA1 agonist mimics anticancer activity of SEMA3F,” demonstrates that the bispecific antibody selectively mimics the beneficial aspects of semaphorin 3F (SEMA3F)/NRP2 signaling while avoiding potentially cross-toxic reactivity, serving as a basis for a novel anticancer therapy. The publication is available on the Company’s website and at: https://www.jbc.org/article/S0021-9258%2825%2902908-4/fulltext. Year Ended 2025 Financial Highlights and Cash Position Cash & Investment Position: Cash, cash equivalents, restricted cash and available-for-sale investments as of December 31, 2025, were $80.9 million. R&D Expenses: Research and development expenses were $60.2 million for the year ended 2025, 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 $17.6 million for the year ended 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,” “suggest,” “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 and ATYR0101; 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; timelines and plans with respect to certain development activities and development goals, including the occurrence and timing of our 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 as well as our expectations with respect to the outcome of that meeting, the timing of our update for 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, uncertainty related to interactions with the FDA in general, 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 existing or future 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 (including difficulties or delays in patient enrollment in planned clinical trials), 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.

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 [email protected]