aTyr Pharma Reports Q1 2024 Results and Corporate Update

aTyr Pharma, Inc. (Nasdaq: LIFE), a clinical stage biotechnology company based in San Diego, recently released its first quarter financial results for 2024 and provided updates on its corporate activities. The company, known for leveraging its proprietary tRNA synthetase platform to develop novel therapeutics, is currently focusing on its lead candidate, efzofitimod, for treating interstitial lung disease (ILD).

During the first quarter of 2024, aTyr continued to advance its clinical studies for efzofitimod. The company's President and CEO, Sanjay S. Shukla, M.D., M.S., expressed optimism about the progress of the Phase 3 EFZO-FIT™ study, which targets pulmonary sarcoidosis, a severe form of ILD with significant unmet medical needs. Enrollment for this pivotal global trial is expected to be completed by the second quarter of 2024.

The EFZO-FIT™ study is a 52-week, randomized, double-blind, placebo-controlled trial involving 264 patients with pulmonary sarcoidosis. Participants are divided equally into three cohorts to receive either 3.0 mg/kg or 5.0 mg/kg of efzofitimod or a placebo, administered intravenously monthly for a total of 12 doses. The study is being conducted across more than 90 centers in nine different countries. Patients who complete the trial and wish to continue treatment with efzofitimod may participate in an Individual Patient Expanded Access Program (EAP).

In addition to the EFZO-FIT™ study, aTyr is also enrolling patients in the Phase 2 EFZO-CONNECT™ study, which aims to assess the efficacy, safety, and tolerability of efzofitimod in patients with systemic sclerosis-associated ILD (SSc-ILD). This proof-of-concept study involves 25 patients in a randomized, double-blind, placebo-controlled format. The participants are divided into three cohorts to receive either 270 mg or 450 mg of efzofitimod or a placebo, administered intravenously monthly over six doses. The trial is being conducted at several centers in the United States.

aTyr's research on other therapeutic candidates also continued to gain attention. The company presented a poster on ATYR0750 at the Gordon Research Conference, highlighting its selective binding to FGFR4 and the resultant morphological changes and signaling in liver cells. Additionally, a poster on efzofitimod will be presented at the upcoming American Thoracic Society (ATS) 2024 International Conference in San Diego.

Financially, aTyr concluded the first quarter of 2024 with $87.7 million in cash, cash equivalents, restricted cash, and investments. The company believes that this funding will support its operational needs through the filing of a Biologics License Application (BLA) for efzofitimod in pulmonary sarcoidosis. Research and development expenses for the quarter amounted to $13.4 million, primarily driven by clinical trial costs, manufacturing expenses, and other related activities. General and administrative expenses were $3.5 million, while revenue from collaboration and licensing agreements with Kyorin was $0.2 million.

Efzofitimod is a first-in-class biologic immunomodulator designed to treat ILD by selectively modulating activated myeloid cells through neuropilin-2, thereby reducing inflammation and potentially preventing fibrosis without suppressing the immune system. Efzofitimod is currently under investigation in the global Phase 3 EFZO-FIT™ study for pulmonary sarcoidosis and the Phase 2 EFZO-CONNECT™ study for SSc-ILD. These diseases have limited treatment options, underscoring the need for safer, more effective therapies.

aTyr continues to harness its tRNA synthetase platform to develop new treatments for fibrosis and inflammation. The company aims to unlock novel therapeutic pathways by exploring the extracellular roles of tRNA synthetases, essential proteins with diverse regulatory functions.