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 ...

Exhibit 99.1 IMMEDIATE RELEASE Contact: Ashlee Dunston Sr. Director, Investor Relations and Public Affairs adunston@atyrpharma.com aTyr Pharma Announces First Quarter 2025 Results and Provides Corporate Update Topline data from Phase 3 EFZO-FIT™ study of efzofitimod in pulmonary sarcoidosis on track for the third quarter of 2025. Blinded baseline demographics and disease characteristics from Phase 3 EFZO-FIT™ study and current U.S. epidemiology and treatment practices for pulmonary sarcoidosis to be present ...

Core Insights - aTyr Pharma is progressing with its clinical program for efzofitimod, a first-in-class biologic immunomodulator targeting pulmonary sarcoidosis, with topline data from the Phase 3 EFZO-FIT™ study expected in Q3 2025 [1][4][3] Company Updates - The first quarter of 2025 saw aTyr Pharma report a net loss of $14.88 million, compared to a loss of $15.49 million in the same period of 2024, with total revenues remaining at $0 [13] - Research and development expenses for Q1 2025 were $11.8 million, primarily for clinical trial costs related to the EFZO-FIT™ and EFZO-CONNECT™ studies [10][13] - The company appointed Dalia R. Rayes as Head of Commercial for the Global Efzofitimod Franchise, bringing over 25 years of experience in biotechnology and pharmaceutical commercial organizations [4] Clinical Trials - The Phase 3 EFZO-FIT™ study is a randomized, double-blind, placebo-controlled trial involving 268 patients across 85 centers in nine countries, focusing on steroid reduction as the primary endpoint [4][7] - The ongoing Phase 2 EFZO-CONNECT™ study aims to evaluate efzofitimod in patients with systemic sclerosis-related interstitial lung disease, with interim data expected in Q2 2025 [4][7] Financial Position - As of March 31, 2025, aTyr Pharma had cash and cash equivalents totaling $78.8 million, which is projected to fund operations for one year following the Phase 3 EFZO-FIT™ readout [10][15] - General and administrative expenses for Q1 2025 were reported at $4.0 million, slightly higher than the previous year's $3.5 million [10][13]

Core Insights - aTyr Pharma, Inc. is presenting preclinical data for ATYR2810, a monoclonal antibody targeting neuropilin-2 (NRP2), at the AACR Annual Meeting 2025, indicating progress in its clinical development [1][2] Group 1: Company Overview - aTyr Pharma is a clinical stage biotechnology company focused on developing first-in-class medicines from its proprietary tRNA synthetase platform [1][4] - The company's lead therapeutic candidate is efzofitimod, which is in clinical development for treating interstitial lung disease [4] Group 2: Research Findings - ATYR2810 has shown potential in combating drug resistance mechanisms in aggressive cancers like glioblastoma multiforme (GBM) [2][3] - Preclinical research indicates that ATYR2810 enhances anti-tumor immunity and significantly increases overall survival when used as a single agent [3] - The combination of ATYR2810 with anti-PD-1 therapies further improves survival rates and reduces tumor size in GBM models [3]