On March 15, 2024 aTyr Pharma, Inc. (Nasdaq: LIFE) ("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, reported fourth quarter and full year 2023 results and provided a corporate update (Press release, aTyr Pharma, MAR 15, 2024, View Source [SID1234641208]).
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"Throughout 2023 we made meaningful progress with our clinical development program for our lead therapeutic candidate, efzofitimod, in interstitial lung disease (ILD)," said Sanjay S. Shukla, M.D., M.S., President and Chief Executive Officer of aTyr. "Our primary focus for 2024 is completing enrollment in our global pivotal Phase 3 EFZO-FIT study in patients with pulmonary sarcoidosis, a major form of ILD, which is anticipated in the second quarter."
"We ended 2023 with more than $100 million in cash, restricted cash, cash equivalents and investments. Based on our current cash position and operational plans, we believe our financial resources are sufficient to fund the Company’s operations through the filing of a Biologics License Application (BLA) for efzofitimod in pulmonary sarcoidosis."
Fourth Quarter 2023 and Subsequent Period Highlights
Continued enrollment in the global pivotal Phase 3 EFZO-FIT study to evaluate the efficacy and safety of efzofitimod in patients with pulmonary sarcoidosis. This is a randomized, double-blind, placebo-controlled, 52-week study consisting of three parallel cohorts randomized equally to either 3.0 mg/kg or 5.0 mg/kg of efzofitimod or placebo dosed intravenously monthly for a total of 12 doses. The study intends to enroll up to 264 patients with pulmonary sarcoidosis. The study is currently enrolling at more than 90 centers in 9 countries. A positive data and safety monitoring board review assessed that the study could continue unmodified. Based on current enrollment projections, the Company anticipates completing enrollment in the study in the second quarter of 2024.
Announced an Individual Patient Expanded Access Program (EAP) for efzofitimod for patients with pulmonary sarcoidosis. The EAP has been initiated based on blinded EFZO-FIT study investigator and patient participant feedback. The program is designed to allow access for patients who complete the Phase 3 EFZO-FIT study and wish to receive treatment with efzofitimod outside of the clinical trial.
Continued enrollment in the Phase 2 EFZO-CONNECT study to evaluate the efficacy, safety and tolerability of efzofitimod in patients with 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 dosed intravenously monthly for a total of 6 doses. The study intends to enroll up to 25 patients with SSc-ILD and is open for enrollment at multiple centers in the U.S.
Poster for efzofitimod accepted for presentation at the upcoming American Thoracic Society (ATS) 2024 International Conference. The conference is scheduled to take place May 17 – 22, 2024, in San Diego, CA.
Poster 8837 – Efzofitimod is an Immunomodulator of Myeloid Cell Function and Novel Therapeutic Candidate for Interstitial Lung Diseases on Sunday, May 19, 2024, at 2:15 p.m. PDT.
Presented two posters highlighting the importance of neuropilin-2 (NRP2) in immune regulation at the Keystone Symposia on Myeloid Cell Diversity. The findings further demonstrate that efzofitimod modulates myeloid cells via the NRP2 receptor to promote a unique anti-inflammatory mechanism and validates the role of NRP2 in the immune system by the activity of an NRP2 blocking antibody in preclinical models.
Announced Wayne A. I. Frederick, M.D., President Emeritus of Howard University, as an advisor to the Company. Dr. Frederick is a distinguished physician executive with extensive knowledge on disparities in healthcare and will advise the Company on its efzofitimod program in ILD.
Poster for ATYR0750 accepted for presentation at the upcoming Gordon Research Conference Fibroblast Growth Factors in Development and Disease. The conference is scheduled to take place March 24 – 29, 2024, in Galveston, TX.
Poster – Alanyl-tRNA Synthetase Fragment Binds to FGFR4 and Induces Morphological Changes and Downstream Signaling in Liver Cells with Functional Similarities to FGF2.
Year Ended 2023 Financial Highlights and Cash Position
Cash & Investment Position: Cash, cash equivalents, restricted cash and investments as of December 31, 2023, were $101.7 million. Based on the Company’s current operational plans and existing cash, the Company maintains its prior guidance and believes its cash runway will be sufficient to fund the Company’s operations through the filing of a BLA for efzofitimod in pulmonary sarcoidosis.
R&D Expenses: Research and development expenses were $42.3 million for the year ended 2023, which consisted primarily of clinical trial costs for the Phase 3 EFZO-FIT and Phase 2 EFZO-CONNECT studies, manufacturing costs for the efzofitimod program and research and development costs for the efzofitimod and discovery programs.
G&A Expenses: General and administrative expenses were $13.0 million for the year ended 2023.
Conference Call and Webcast Details
aTyr will host a conference call and webcast today at 5:00 p.m. EDT / 2:00 p.m. PDT to discuss its financial results and provide a corporate update. Interested parties may access the call by registering here in order to obtain a dial in, personalized passcode and webcast information. Links to a live audio webcast and replay may be accessed on the aTyr website Events page at: View Source An audio replay will be available for at least 90 days following the event.
About Efzofitimod
Efzofitimod is a first-in-class 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. aTyr is currently investigating efzofitimod in the global Phase 3 EFZO-FIT study in patients with pulmonary sarcoidosis, a major form of ILD, and 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.