Core Insights - aTyr Pharma, Inc. is advancing its clinical development of 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][3][4] Company Updates - The company completed enrollment in the Phase 3 EFZO-FIT™ study, which is the largest interventional study for pulmonary sarcoidosis, involving 268 patients across 85 centers in nine countries [4] - The study is designed as a randomized, double-blind, placebo-controlled trial with a primary endpoint focused on steroid reduction and secondary endpoints assessing sarcoidosis symptoms and lung function [4] - The fourth positive review from the Data Safety Monitoring Board (DSMB) confirmed the favorable safety profile of efzofitimod, allowing the study to continue as planned [1][4] Financial Highlights - For the year ended 2024, aTyr reported research and development expenses of $54.4 million, primarily due to clinical trial costs for the EFZO-FIT™ and EFZO-CONNECT™ studies [6][14] - The company had cash and cash equivalents of $75.1 million as of December 31, 2024, and raised approximately $18.8 million in gross proceeds from an at-the-market offering [6][15] - General and administrative expenses for the year were $13.8 million, reflecting an increase from the previous year [14] Research and Development - The Phase 2 EFZO-CONNECT™ study is ongoing, evaluating efzofitimod in patients with systemic sclerosis-related interstitial lung disease, with interim data expected in Q2 2025 [4] - A manuscript published in Science Translational Medicine validated efzofitimod's mechanism of action, highlighting its unique anti-inflammatory effects [1][5][8] Upcoming Events - aTyr will host a conference call and webcast to discuss financial results and corporate updates on March 13, 2025 [1][7] - Posters related to efzofitimod will be presented at the American Thoracic Society (ATS) 2025 International Conference scheduled for May 16-21, 2025 [10]

Core Insights - The publication validates the unique anti-inflammatory mechanism of action of efzofitimod through the neuropilin-2 (NRP2) receptor, supporting its clinical program for interstitial lung disease (ILD) [1][3][4] 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][7] - The lead therapeutic candidate, efzofitimod, is in clinical development for treating ILD, specifically targeting inflammation and fibrosis in the lungs [5][7] Mechanism of Action - Efzofitimod is derived from a splice variant of histidyl-tRNA synthetase (HARS) and selectively binds to NRP2, which is highly expressed in myeloid cells at inflammation sites [3][4] - This binding inhibits pro-inflammatory receptors and cytokines, downregulating inflammatory pathways in macrophages, potentially disrupting chronic inflammation and fibrosis cycles [3][4] Clinical Development - Efzofitimod is currently being investigated in the Phase 3 EFZO-FIT™ study for pulmonary sarcoidosis and the Phase 2 EFZO-CONNECT™ study for systemic sclerosis-related ILD [4][5] - The drug has received orphan drug designation in the U.S., E.U., and Japan for sarcoidosis, as well as Fast Track designation in the U.S. for both pulmonary sarcoidosis and systemic sclerosis-related ILD [4][5] Scientific Validation - The peer-reviewed publication in Science Translational Medicine provides extensive preclinical data supporting efzofitimod's immunomodulatory activity and its potential application in chronic inflammatory conditions [3][4]

Core Insights - The independent data and safety monitoring board (DSMB) has recommended the continuation of the Phase 3 EFZO-FIT™ study of efzofitimod without any modifications, indicating no safety concerns [1][2] - The study aims to position efzofitimod as a potential chronic maintenance therapy for pulmonary sarcoidosis, with topline results expected in Q3 2025 [2] Company Overview - aTyr Pharma, Inc. is a clinical stage biotechnology company focused on developing first-in-class medicines from its proprietary tRNA synthetase platform [1][4] - The lead therapeutic candidate, efzofitimod, is being investigated for its efficacy and safety in treating pulmonary sarcoidosis and other interstitial lung diseases (ILD) [3][4] Study Details - The EFZO-FIT™ study is a global Phase 3 randomized, double-blind, placebo-controlled trial involving 268 subjects, assessing the efficacy of efzofitimod administered intravenously at doses of 3.0 mg/kg or 5.0 mg/kg [2][3] - The primary endpoint is steroid reduction, while secondary endpoints include lung function and sarcoidosis symptoms [2]

Core Viewpoint - aTyr Pharma, Inc. is set to report its fourth quarter and full year 2024 financial results on March 13, 2025, and will provide a corporate update during a conference call and webcast [1] Group 1: Financial Reporting - The financial results will be announced after market close on March 13, 2025 [1] - A conference call and webcast will be held at 5:00 p.m. EDT / 2:00 p.m. PDT to review the results [2] Group 2: Company Overview - aTyr Pharma is a clinical stage biotechnology company focused on developing first-in-class medicines from its proprietary tRNA synthetase platform [3] - The company aims to translate tRNA synthetase biology into new therapies for fibrosis and inflammation [3] - The lead therapeutic candidate, efzofitimod, is in clinical development for treating interstitial lung disease, which involves immune-mediated disorders causing lung inflammation and progressive fibrosis [3]

Group 1 - 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 the Leerink Partners Global Healthcare Conference on March 10, 2025, at 1:40pm EDT in Miami, FL [1] - aTyr's lead therapeutic candidate, efzofitimod, is an immunomodulator in clinical development for treating interstitial lung disease, which involves inflammation and progressive fibrosis of the lungs [2] Group 2 - The company leverages evolutionary intelligence to translate tRNA synthetase biology into new therapies for fibrosis and inflammation [2] - aTyr's discovery platform aims to uncover therapeutic intervention points by exploring signaling pathways from its proprietary library of domains derived from all 20 tRNA synthetases [2] - Management will be available for one-on-one meetings with registered investors during the conference [1]

Clinical Development and Presentations - The company will present three posters for its lead therapeutic candidate, efzofitimod, at the American Thoracic Society (ATS) 2025 International Conference [1] - The posters include details on the EFZO-FIT trial design, real-world treatment patterns, and the incidence, prevalence, and mortality of pulmonary sarcoidosis in the US [2] - The EFZO-FIT trial is the largest placebo-controlled trial in pulmonary sarcoidosis, with topline data expected in Q3 2025 [1][2] About Efzofitimod - Efzofitimod is a first-in-class biologic immunomodulator in clinical development for the treatment of interstitial lung disease (ILD) [3] - It selectively modulates activated myeloid cells through neuropilin-2 to resolve inflammation without immune suppression and potentially prevent fibrosis progression [3] - The company is investigating efzofitimod in the global Phase 3 EFZO-FIT study for pulmonary sarcoidosis and the Phase 2 EFZO-CONNECT study for systemic sclerosis-related ILD [3] About aTyr - aTyr is a clinical-stage biotechnology company focused on translating tRNA synthetase biology into new therapies for fibrosis and inflammation [4] - The company's discovery platform leverages a proprietary library of domains derived from all 20 tRNA synthetases to uncover novel signaling pathways [4] - Efzofitimod is the lead therapeutic candidate, targeting immune-mediated disorders that cause inflammation and progressive fibrosis in the lungs [4]

Core Points - aTyr Pharma, Inc. is a clinical stage biotechnology company focused on developing first-in-class medicines from its proprietary tRNA synthetase platform [1][3] - The company will present at the 43rd Annual J.P. Morgan Healthcare Conference scheduled for January 13-16, 2025, in San Francisco, CA [1][2] - The lead therapeutic candidate of aTyr is efzofitimod, an immunomodulator in clinical development for treating interstitial lung disease [3] Presentation Details - The presentation will take place on January 16, 2025, at 8:15 am PST [2] - Company management will be available for one-on-one meetings with registered investors during the conference [2] - A webcast of the presentation will be available on the company's investor section of the website, with a replay accessible for at least 30 days post-event [2] Company Overview - aTyr leverages evolutionary intelligence to develop therapies targeting fibrosis and inflammation through its tRNA synthetase biology [3] - The company’s discovery platform aims to identify therapeutic intervention points by exploring signaling pathways from its proprietary library of tRNA synthetase domains [3] - Interstitial lung disease, the focus of aTyr's lead candidate, is characterized by immune-mediated disorders leading to lung inflammation and progressive fibrosis [3]

Core Insights - aTyr Pharma, Inc. has appointed Eric Benevich to its Board of Directors, effective December 10, 2024, to support its commercialization efforts [1][2] - Mr. Benevich brings over 30 years of commercial experience in the pharmaceutical industry, having held significant roles at various biopharmaceutical companies [2] - aTyr is focused on developing first-in-class medicines from its proprietary tRNA synthetase platform, with its lead candidate being efzofitimod for treating interstitial lung disease [3] Company Overview - aTyr Pharma is a clinical stage biotechnology company that utilizes evolutionary intelligence to develop therapies targeting fibrosis and inflammation [3] - The company’s discovery platform aims to identify therapeutic intervention points by exploring signaling pathways from its library of tRNA synthetase-derived domains [3] - The lead therapeutic candidate, efzofitimod, is an immunomodulator currently in clinical development for immune-mediated disorders affecting the lungs [3]

SAN DIEGO, Nov. 15, 2024 (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 Company will present two posters related to its tRNA synthetase candidate ATYR0101 at the Keystone Symposia on Fibrosis: Inflammation, Drivers, and Therapeutic Resolution, which is scheduled to take place December 8 – 11, 2024, ...

Efzofitimod Clinical Development and Trials - The company's lead therapeutic candidate, efzofitimod, has shown positive results in a Phase 1b/2a clinical trial for pulmonary sarcoidosis, with consistent dose response and improvements in key efficacy endpoints compared to placebo[75][76] - The global pivotal Phase 3 EFZO-FIT study for efzofitimod in pulmonary sarcoidosis completed enrollment of 268 patients, exceeding the target of 264, with topline data expected in Q3 2025[77] - The company initiated an Individual Patient Expanded Access Program for efzofitimod, allowing patients who complete the Phase 3 EFZO-FIT study to receive treatment outside the clinical trial[78] - A Phase 2 proof-of-concept study (EFZO-CONNECT) for efzofitimod in SSc-ILD was amended to include a 24-week open-label extension, with interim data expected in Q2 2025[79] Collaboration and Licensing Agreements - The company has a collaboration with Kyorin Pharmaceutical for the development and commercialization of efzofitimod in Japan, generating $20 million in upfront and milestone payments, with potential for an additional $155 million and tiered royalties[80] - Kyorin Agreement milestone payment of $10.0 million triggered by the first patient dosing in Japan for the EFZO-FIT study, recorded as revenue in 2022 and received in February 2023[91] - Total upfront and milestone payments from Kyorin Agreement to date: $20.0 million, with potential for an additional $155.0 million upon achieving development, regulatory, and sales milestones[91] Pipeline and Therapeutic Candidates - The company has identified target receptors for domains of two additional tRNA synthetases, forming the basis of new pipeline candidates[83] - ATYR0101, a fusion protein derived from aspartyl-tRNA synthetase, shows potential for treating multiple fibrotic diseases by inducing apoptosis of myofibroblasts in a TGFβ-dependent manner[84] - ATYR0750, a fusion protein derived from alanyl-tRNA synthetase, is a novel ligand to FGFR4, with potential therapeutic benefits in diseases related to inflammation and fibrosis[85] Financial Performance and Cash Position - As of September 30, 2024, the company had $68.9 million in cash, cash equivalents, restricted cash, and available-for-sale investments, sufficient to meet material cash requirements for at least one year[87] - The company raised $48.1 million in a February 2023 public offering and $21.4 million through an at-the-market offering program in the first nine months of 2024[89][90] - Net cash used in operating activities for the nine months ended September 30, 2024: $56.0 million, primarily due to increased costs for EFZO-FIT and EFZO-CONNECT studies and BLA preparation[92] - Net cash provided by investing activities for the nine months ended September 30, 2024: $23.3 million, driven by timing differences in investment activities[93] - Net cash provided by financing activities for the nine months ended September 30, 2024: $21.0 million, primarily from the issuance of common stock[94] Expenses and Cost Analysis - Research and development expenses expected to increase, primarily driven by clinical development and manufacturing of efzofitimod[106] - Research and development expenses increased to $14.8 million in Q3 2024, up from $10.3 million in Q3 2023, primarily due to a $3.5 million increase in efzofitimod expenses[110] - Efzofitimod expenses rose to $11.9 million in Q3 2024, compared to $8.5 million in Q3 2023, driven by the advancement of the EFZO-FIT study and increased manufacturing efforts[110] - General and administrative expenses increased to $3.3 million in Q3 2024, up from $2.6 million in Q3 2023, mainly due to higher personnel-related costs[111] - Research and development expenses for the nine months ended September 30, 2024, were $42.1 million, up from $29.5 million in 2023, with a $12.5 million increase in efzofitimod expenses[114] - General and administrative expenses for the nine months ended September 30, 2024, were $10.2 million, compared to $9.8 million in 2023, due to higher personnel-related costs and professional fees[115] - Efzofitimod expenses for the nine months ended September 30, 2024, were $34.2 million, up from $21.7 million in 2023, driven by the EFZO-FIT study and manufacturing efforts[119] - Total research and development expenses for the nine months ended September 30, 2024, were $42.1 million, up from $29.5 million in 2023, with a $12.6 million increase[119] Revenue and Other Income - License and collaboration agreement revenues were $0.2 million for the nine months ended September 30, 2024, down from $0.4 million in the same period in 2023[113] - Other income, net decreased to $0.8 million in Q3 2024, down from $1.3 million in Q3 2023, primarily due to a lower cash balance[112] - Other income, net for the nine months ended September 30, 2024, was $3.0 million, slightly down from $3.3 million in 2023, due to a lower cash balance[116] Corporate Operations and Leasing - Corporate headquarters lease base rent: $5.75 per square foot per month for the first 12 months, with annual upward adjustments of approximately 3.0%[100] - Financing lease liabilities as of September 30, 2024: $1.6 million, with $2.2 million in cash collateral included in restricted cash[101] Accounting and Financial Policies - Critical accounting policies and estimates remain consistent with those disclosed in the 2023 Annual Report[109] - General and administrative expenses include salaries, stock-based compensation, and costs for legal, accounting, and patent maintenance[107]