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]

Financial Performance - Total revenues for Q3 2024 were $0, compared to $353,000 in Q3 2023, indicating a decrease in license and collaboration agreement revenues[12] - Consolidated net loss for Q3 2024 was $17.3 million, compared to a net loss of $11.3 million in Q3 2023[12] - General and administrative expenses for Q3 2024 were $3.3 million, compared to $2.6 million in Q3 2023[12] - Research and development expenses for Q3 2024 were $14.8 million, up from $10.3 million in Q3 2023, reflecting increased clinical trial costs[7] Cash and Investments - Cash and investments as of September 30, 2024, were $68.9 million, down from $101.7 million at the end of 2023[13] - Total assets as of September 30, 2024, were $91.6 million, down from $120.7 million at the end of 2023[13] - The company raised approximately $19.4 million in gross proceeds from an at-the-market offering after Q3 2024[7] Clinical Trials - Enrollment completed in the Phase 3 EFZO-FIT™ study with 268 patients, exceeding the target, and topline data expected in Q3 2025[5] - The Phase 2 EFZO-CONNECT™ study is ongoing, aiming to enroll up to 25 patients with interim data expected in Q2 2025[6] - The efzofitimod program showed statistically significant improvement in corticosteroid relapse rates, as published in the European Respiratory Journal[2]

Financial Position - The company has an accumulated deficit of $499.8 million as of June 30, 2024, and expects to continue incurring net losses for the foreseeable future[78]. - As of June 30, 2024, the company had cash, cash equivalents, restricted cash, and available-for-sale investments totaling $81.4 million, sufficient to meet material cash requirements for at least one year[78]. - Net cash used in operating activities for the six months ended June 30, 2024, was $42.8 million, compared to $12.6 million for the same period in 2023, primarily due to increased clinical study costs[83]. - Net cash provided by investing activities for the six months ended June 30, 2024, was $5.4 million, a significant improvement from $(42.3) million in 2023[84]. - Net cash provided by financing activities for the six months ended June 30, 2024, was $21.1 million, down from $57.5 million in 2023, mainly due to reduced proceeds from stock offerings[85]. - As of June 30, 2024, the company has $1.7 million in financing lease liabilities and $2.2 million in cash collateral for the financing lease[91]. Fundraising Activities - The company completed a public offering in February 2023, raising approximately $48.1 million from the sale of 23,125,000 shares at $2.25 per share[80]. - Under the Jefferies ATM Offering Program, the company sold 10,530,795 shares at an average price of $1.82 per share, generating net proceeds of approximately $18.4 million in 2023[81]. - Kyorin triggered a $10.0 million milestone payment for dosing the first patient in Japan in the EFZO-FIT study, recorded as revenue in 2022[82]. - Total milestone payments from the Kyorin Agreement to date amount to $20.0 million, with potential additional payments of up to $155.0 million upon achieving specific milestones[82]. Research and Development - The company initiated a global pivotal Phase 3 clinical trial (EFZO-FIT study) for efzofitimod, enrolling 268 patients, with topline data expected in Q3 2025[69]. - The EFZO-CONNECT study for efzofitimod in patients with systemic sclerosis-associated ILD is designed to enroll up to 25 patients, with interim data expected in Q2 2025[71]. - The company is developing additional tRNA synthetase candidates, including ATYR0101 and ATYR0750, targeting fibrosis and inflammation[75][76]. - The company expects research and development expenses to continue increasing, primarily for the clinical development of efzofitimod and other potential therapeutics[96]. - Research and development expenses increased to $14.0 million for the three months ended June 30, 2024, up from $9.8 million in 2023, representing a $4.1 million increase[100]. - Efzofitimod expenses accounted for $11.3 million in the three months ended June 30, 2024, compared to $6.8 million in 2023, reflecting a $4.5 million increase due to advancements in the EFZO-FIT study[100]. - Total research and development expenses for the six months ended June 30, 2024, were $27.3 million, an increase of $8.1 million from $19.2 million in 2023[103]. - Efzofitimod expenses for the six months ended June 30, 2024, were $22.3 million, up from $13.2 million in 2023, marking a $9.1 million increase[103]. Revenue and Expenses - The company has not generated any revenues from product sales to date and anticipates increased expenses related to ongoing clinical development and potential commercialization efforts[86]. - License and collaboration agreement revenues were $0.2 million for the six months ended June 30, 2024, with no revenues reported in the same period of the prior year[102]. - General and administrative expenses decreased to $3.3 million for the three months ended June 30, 2024, down from $3.7 million in 2023, a reduction of $0.4 million[101]. - General and administrative expenses for the six months ended June 30, 2024, were $6.8 million, a decrease of $0.3 million from $7.1 million in 2023[104]. - Other income, net for the six months ended June 30, 2024, was $2.2 million, compared to $2.1 million in 2023, showing a slight increase[105]. - Non-cash expenses related to research and development increased to $0.6 million for the three months ended June 30, 2024, from $0.5 million in 2023[101]. - Non-cash expenses for general and administrative purposes increased to $1.2 million for the six months ended June 30, 2024, from $1.0 million in 2023[104]. Market and Operational Considerations - The company continues to monitor macroeconomic uncertainties that may impact its operations and financial performance[77]. - The company has no sales or marketing capabilities and will need to expand its organization to support commercialization activities[86]. - The company has a lease agreement for approximately 24,866 rentable square feet with a base rent starting at $5.75 per square foot, subject to annual adjustments[90].

Exhibit 99.1 IMMEDIATE RELEASE Contact: Ashlee Dunston Director, Investor Relations and Public Affairs adunston@atyrpharma.com aTyr Pharma Announces Second Quarter 2024 Results and Provides Corporate Update Phase 3 EFZO-FIT™ study of efzofitimod in pulmonary sarcoidosis enrollment completed with 268 patients; topline data from this 52-week study expected in the third quarter of 2025. Ended the second quarter 2024 with $81.4 million in cash, cash equivalents, restricted cash and investments. SAN DIEGO – Augu ...

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, 2024 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 ...

Drug Development and Clinical Trials - Efzofitimod is a first-in-class biologic immunomodulator in clinical development for interstitial lung disease (ILD) with orphan drug designations from the FDA for sarcoidosis and systemic sclerosis (SSc) [12] - The EFZO-FIT study, a pivotal Phase 3 trial for efzofitimod in pulmonary sarcoidosis, is currently enrolling up to 264 subjects and aims to complete enrollment by Q2 2024 [14][15] - The Phase 1b/2a clinical trial demonstrated consistent dose response for efzofitimod on key efficacy endpoints, including improvements in lung function and symptom measures compared to placebo [13] - The primary endpoint of the EFZO-FIT study is steroid reduction, with secondary endpoints including forced vital capacity (FVC) and quality of life assessments [14] - The EFZO-CONNECT study for efzofitimod in patients with SSc-ILD was initiated in Q3 2023, with the first patient dosed in October 2023 [17][22] - The ongoing Phase 3 clinical trial, EFZO-FIT, aims to enroll up to 264 patients with symptomatic pulmonary sarcoidosis, evaluating the efficacy and safety of IV efzofitimod at doses of 3.0 mg/kg and 5.0 mg/kg [43] - The Phase 1 clinical trial of efzofitimod in healthy volunteers indicated that the drug was well-tolerated at all dose levels tested, with no significant adverse events [57] - The Phase 1b/2a clinical trial for efzofitimod in pulmonary sarcoidosis demonstrated a 58% overall steroid reduction from baseline and a 22% relative reduction compared to placebo in the 5.0 mg/kg treatment group [51] - The EFZO-CONNECT study, targeting SSc-ILD, is a 28-week trial with a planned enrollment of 25 patients, with the first patient dosed in October 2023 [17] - The Phase 2 clinical trial for efzofitimod in COVID-19 patients showed that a single IV dose was generally safe and well-tolerated, with a signal of activity in the 3.0 mg/kg cohort [56] Financial and Market Considerations - The Kyorin Agreement has generated $20.0 million in upfront and milestone payments, with potential for an additional $155.0 million upon achieving certain milestones [18] - The global market opportunity for pulmonary sarcoidosis and SSc-ILD is estimated to be between $2-3 billion [72] - The company has incurred significant losses since inception and anticipates continued losses for the foreseeable future [10] - The market for ILD treatments remains underserved, presenting a high unmet medical need for effective therapies [12] - The company faces significant uncertainty regarding coverage and reimbursement from third-party payors, which may limit sales of approved products [125][132] - Third-party payors are increasingly scrutinizing pricing and cost-effectiveness, potentially impacting reimbursement rates for approved products [126][129] - The U.S. government and state legislatures are implementing cost containment programs that may reduce the profitability of drug products, including price controls and mandatory discounts [127] Regulatory and Compliance - The FDA requires extensive preclinical and clinical testing before any new biologic can be marketed, which involves significant time and financial resources [96] - A Biologics License Application (BLA) must include all relevant data from preclinical studies and clinical trials, and FDA approval is required before marketing [104] - The FDA may issue a Complete Response Letter (CRL) if the BLA is not ready for approval, outlining deficiencies that need to be addressed [108] - The FDA grants orphan drug designation for drugs treating rare diseases affecting fewer than 200,000 individuals in the U.S., providing financial incentives and exclusivity for seven years if approved first [117][118] - The company is subject to various federal and state healthcare laws, including the Anti-Kickback Statute and the False Claims Act, which could impact operations [136] - The company has implemented safeguards to discourage improper payments under the U.S. Foreign Corrupt Practices Act (FCPA) [135] Research and Development Strategy - The company is focused on advancing efzofitimod toward regulatory approval and exploring its applications in other ILDs [21][22] - The company has advanced two additional tRNA synthetase programs, ATYR0101 and ATYR0750, into preclinical development [23] - The company plans to further elucidate the therapeutic potential of efzofitimod through mechanistic investigations, including in vitro and in vivo preclinical studies [23] - The company plans to invest significant financial and management resources to develop the appropriate commercial infrastructure for its product candidates [75] - The company aims to expand its intellectual property estate by filing patent applications for new methods of treatment and therapeutics [83] Employee and Operational Insights - As of December 31, 2023, the company had 59 employees, with 56 being full-time, including 36 in research and development roles [137] - The company emphasizes the importance of attracting and retaining talented employees to execute its strategy effectively [138] - The company operates in a single accounting segment, indicating a focused business model [141] Drug Mechanism and Efficacy - Efzofitimod targets the NRP2 receptor, which is upregulated during differentiation and activation of myeloid cells, potentially providing a novel therapeutic approach for immune-mediated and fibrotic diseases [32] - Efzofitimod has shown significant reduction in lung inflammation and fibrosis, improving respiratory function parameters in multiple animal models of lung fibrosis [29] - Efzofitimod has been shown to reduce lung and skin fibrosis in an animal model of systemic sclerosis (SSc-ILD), indicating its potential as a therapeutic candidate for this condition [42] - The company has demonstrated that efzofitimod downregulates key inflammatory cytokines such as IL-6, TNF-α, and IFN-γ in both animal models and human clinical trials [29] - Efzofitimod is a novel immunomodulatory Fc fusion protein that selectively modulates NRP2 to downregulate immune responses in inflammatory diseases [24]

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 September 30, 2023 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 | --- | --- | --- | |-----------------------------------------------------------------------------------------|------ ...