Topline results expected in the third quarter of 2025. SAN DIEGO, July 22, 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 that the last patient has completed their last visit in the Company's Phase 3 EFZO-FIT™ study of its lead therapeutic candidate, efzofitimod, in patients with pulmonary sarcoidosis ...

Efzofitimod for Interstitial Lung Disease (ILD) - Efzofitimod is a first-in-class biologic immunomodulator in Phase 3 development for ILD, addressing a significant unmet medical need[10] - aTyr is advancing Efzofitimod as the standard-of-care for ILD, which encompasses over 200 types of rare lung diseases[11, 13] - The company's current focus is estimated at a $2-5 billion global market opportunity[13] - Efzofitimod is positioned to be the first approved product for sarcoidosis in over 60 years[31] Clinical Development and Trial Design - Phase 3 catalyst for multi-billion dollar indication in Q3 2025[6] - The Phase 3 EFZO-FIT study in pulmonary sarcoidosis is fully enrolled with 268 patients, and topline data is expected in Q3 2025[50] - Interim data from the Phase 2 EFZO-CONNECT study in SSc-ILD is expected in Q2 2025[70] - The addressable population for Efzofitimod in sarcoidosis is estimated to be 50-75% of all sarcoidosis patients[28] Financial Position and Pipeline - The company had approximately $68.9 million in cash, restricted cash, cash equivalents, and investments as of Q3 2024, with an additional $19.4 million raised subsequently[70] - The company's cash runway extends through the filing of a Biologics License Agreement (BLA) for efzofitimod in pulmonary sarcoidosis[70]

Group 1 - aTyr Pharma, Inc. is expected to be added to the Russell 2000 Index and the Russell 3000 Index effective after the U.S. market close on June 27, 2025, as part of the 2025 Russell U.S. Indexes annual reconstitution [1] - The Russell 3000 Index tracks the performance of the largest 3,000 publicly traded U.S. companies, while the Russell 2000 Index focuses on small-cap companies [2] - Membership in the Russell Indexes lasts for one year and results in automatic inclusion in appropriate growth and value style indexes, which are utilized by investment managers and institutional managers [2] Group 2 - aTyr is a clinical stage biotechnology company that focuses on developing first-in-class medicines from its proprietary tRNA synthetase platform [4] - The company's lead therapeutic candidate is efzofitimod, which is in clinical development for treating interstitial lung disease, a group of immune-mediated disorders [4]

Summary of aTyr Pharma (ATYR) 2025 Conference Call Company Overview - **Company**: aTyr Pharma (ATYR) - **Event**: 2025 Jefferies Global Healthcare Conference - **Focus**: Lead program for pulmonary sarcoidosis and updates on other indications Key Points on Pulmonary Sarcoidosis Program - **Phase Three Study**: Baseline demographics presented at ATS, showing expected characteristics in race, gender, and age of sarcoidosis patients [3][4] - **Steroid Dosage**: Average steroid dose at entry for the phase three trial was 10.5 mg, slightly lower than the 12-13 mg in phase two, aligning with treatment guidelines [4][6] - **Patient Management**: Patients on lower steroid doses are more sensitive to reductions, with a focus on managing flare-ups in the placebo group [12][13] - **FDA Feedback**: FDA recommended simplifying the statistical analysis plan to focus on the last four weeks of treatment, which increases the power to detect smaller differences [17][18] - **Power Assumptions**: The trial is powered to show a statistically significant absolute reduction of 3 mg in steroid use compared to placebo [22][23] - **Market Opportunity**: The target population for the therapy is larger than initially estimated, with 150,000 to 160,000 steroid-dependent patients in the U.S. [48][49] Other Indications and Updates - **Scleroderma-Related ILD Study**: Interim data showed promising results in skin improvement, particularly in diffuse scleroderma patients [60][62] - **Potential for Broader Applications**: Interest in expanding the use of epsofitimod to other forms of interstitial lung disease (ILD) and connective tissue disease ILD, with a potential market value exceeding $5 billion [70][71] Financial Position - **Cash Position**: The company has sufficient cash to support operations through the upcoming readouts and for at least another year beyond that [72] Conclusion - aTyr Pharma is positioned to potentially change treatment paradigms in pulmonary sarcoidosis and other ILDs, with significant market opportunities and a strong financial position to support ongoing and future trials.

Core Insights - The interim analysis of the Phase 2 EFZO-CONNECT™ study indicates that three out of four patients treated with efzofitimod showed clinically significant improvement in the modified Rodnan Skin Score (mRSS) at 12 weeks [1][3][7] - Efzofitimod is generally safe and well tolerated across all doses, with no serious adverse events reported [1][7] Company Overview - aTyr Pharma, Inc. is a clinical stage biotechnology company focused on developing first-in-class medicines from its proprietary tRNA synthetase platform [2][9] - The lead therapeutic candidate, efzofitimod, is being investigated for the treatment of interstitial lung disease (ILD) related to systemic sclerosis (SSc) [8][9] Study Details - The EFZO-CONNECT™ study is a Phase 2 randomized, double-blind, placebo-controlled trial designed to evaluate the efficacy, safety, and tolerability of efzofitimod in patients with limited or diffuse SSc-ILD [4] - The study involves 28 weeks of treatment with three parallel cohorts randomized to receive either 270 mg or 450 mg of efzofitimod or placebo, with a total of 25 patients expected to be enrolled [4] Key Findings - The interim analysis revealed stable or improved mRSS for all patients, with a notable improvement of 4 points or greater for three out of four efzofitimod-treated patients with diffuse SSc-ILD [7] - Preliminary signals of improvement were observed in inflammatory biomarkers such as interferon gamma (IFN-γ) and monocyte chemoattractant protein-1 (MCP-1) [7] Disease Context - Systemic sclerosis (SSc) is a chronic autoimmune disease that can lead to interstitial lung disease (SSc-ILD), affecting approximately 100,000 people in the U.S., with up to 80% potentially developing ILD [6] - Current treatment options for SSc-ILD are limited and primarily focus on slowing lung function decline, often with significant toxicity [6]

Summary of Atyre Pharma Conference Call Company Overview - Atyre Pharma is positioned as a leading company in the interstitial lung disease sector, focusing on the development of therapies for conditions such as pulmonary sarcoidosis with its lead program, esofitimod [4][5] Key Points and Arguments Upcoming Phase Three Catalyst - Atyre Pharma is anticipating a major phase three trial readout for esofitimod in pulmonary sarcoidosis, which could be the first new therapy for this condition in 70 years [4][5] - The company has transitioned esofitimod through various stages of development, culminating in a highly derisked asset ready for pivotal trials [5] Data-Driven Approach - Atyre emphasizes a data-driven methodology, showcasing significant preclinical and phase two data that supports the efficacy of esofitimod in modulating immune responses without the heavy immunosuppressive effects typical of existing treatments [6][7][9] - The company has published extensively, including in high-impact journals, to validate its findings and maintain transparency with investors [12][14][15] FDA Interaction and Endpoint Adjustments - Recent discussions with the FDA have led to a refined approach to analyzing steroid reduction as a primary endpoint, which is now expected to yield more favorable statistical outcomes [18][19][22] - The FDA's guidance has allowed Atyre to adjust its statistical model positively, potentially lowering the threshold for demonstrating statistical significance [19][22] Market Potential and Demographics - Atyre's updated epidemiological work indicates that the steroid-dependent population for sarcoidosis is larger than previously estimated, now believed to be around 75% of the 200,000 patients in the U.S. [44][45] - The potential market opportunity for esofitimod is now projected to be between $5 billion to $6 billion, significantly higher than earlier estimates [46] Clinical Trial Insights - The trial involves a diverse patient population across 90 centers in nine countries, with baseline demographics aligning closely with previous studies [30][31][34] - The company has taken steps to ensure data integrity and quality, which is crucial for the pivotal submission [39] Future Indications and Pipeline - Atyre is exploring additional indications for esofitimod, including scleroderma ILD, although the readouts from this trial are not expected to directly influence the sarcoidosis data [50][53] - The company has introduced a new IND program targeting myofibroblast apoptosis, which could have significant implications for treating idiopathic pulmonary fibrosis (IPF) [58][60] Other Important Content - The company has received positive feedback from the medical community regarding its innovative approach to treating sarcoidosis, with many expressing gratitude for the efforts to reduce steroid dependency [10][26] - Atyre's commitment to rigorous clinical trial design and data publication is highlighted as a key differentiator in the biotech space [12][15] This summary encapsulates the critical insights and developments discussed during the conference call, reflecting Atyre Pharma's strategic direction and market positioning.

Core Insights - aTyr Pharma, Inc. is a clinical stage biotechnology company focused on developing first-in-class medicines from its proprietary tRNA synthetase platform [1][2] - The company will present at several investor conferences in May and June 2025, including the RBC Capital Markets Global Healthcare Conference and the Jefferies Global Healthcare Conference [1] Group 1: Upcoming Conferences - aTyr Pharma's President and CEO, Sanjay S. Shukla, will present at the RBC Capital Markets Global Healthcare Conference on May 21, 2025, at 11:00am EDT in New York, NY [1] - The company will also participate in the Piper Sandler 3 Annual Virtual Lung Symposium on May 27, 2025, at 2:00pm EDT [1] - Additionally, aTyr will present at the Jefferies Global Healthcare Conference on June 5, 2025, at 2:00pm EDT in New York, NY [1] Group 2: Company Overview - aTyr leverages evolutionary intelligence to develop therapies targeting fibrosis and inflammation through its tRNA synthetase biology [2] - The company's lead therapeutic candidate, efzofitimod, is a first-in-class biologic immunomodulator in clinical development for interstitial lung disease [2]

Blinded baseline demographics and disease characteristics for ongoing Phase 3 EFZO-FIT™ study of efzofitimod in pulmonary sarcoidosis largely balanced and representative of targeted trial population. Real-world evidence shows target market for efzofitimod in pulmonary sarcoidosis is higher than previously estimated with increased morbidity. Treatment practices in the U.S. show approximately 75% of diagnosed pulmonary sarcoidosis patients require treatment with steroids. SAN DIEGO, May 19, 2025 (GLOBE NEWSWI ...

Preclinical data demonstrate ATYR0101's unique anti-fibrotic mechanism through interaction with LTBP-1. About ATYR0101 ATYR0101 to be showcased in oral presentation at the American Thoracic Society (ATS) 2025 Respiratory Innovation Summit. SAN DIEGO, May 14, 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 announ ...

UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 FORM 10-Q (Mark One) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended March 31, 2025 or ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from to Commission File Number: 001-37378 ATYR PHARMA, INC. (Exact name of registrant as specified in its charter) Delaware 20-3435077 (State or other jurisdiction ...