aTyr Pharma Reports Q3 2024 Results and Corporate Update

SAN DIEGO, Nov. 07, 2024 - aTyr Pharma, Inc., a clinical-stage biotechnology company focused on innovative therapies derived from its tRNA synthetase platform, has announced its third-quarter 2024 financial results and a significant corporate update.

The company has reached a critical milestone by completing the enrollment for their Phase 3 EFZO-FIT™ study of efzofitimod in patients with pulmonary sarcoidosis. The study, which is designed to evaluate the efficacy and safety of efzofitimod, is a randomized, double-blind, placebo-controlled trial. It includes 268 patients from 85 centers across nine countries. The participants are divided into three groups, receiving either 3.0 mg/kg or 5.0 mg/kg of efzofitimod, or a placebo. Each group receives monthly intravenous doses for a year. The topline data is expected in the third quarter of 2025. Patients completing the trial and opting for continued treatment can join an Individual Patient Expanded Access Program (EAP).

In addition to the Phase 3 study, aTyr continues to enroll patients in their Phase 2 EFZO-CONNECT™ study, which focuses on systemic sclerosis-related interstitial lung disease (SSc-ILD). This study involves 25 patients who receive either 270 mg or 450 mg of efzofitimod or a placebo over six monthly doses. It aims to assess the efficacy, safety, and tolerability of efzofitimod in these patients. An open-label extension (OLE) is available for those wishing to continue the treatment after the study. Interim data from this trial is expected by the second quarter of 2025.

The publication of a post hoc analysis in the European Respiratory Journal highlighted the effectiveness of efzofitimod in treating pulmonary sarcoidosis. The study showed a statistically significant improvement in the time-to-first relapse for corticosteroid use and a reduction in the corticosteroid relapse rate. This research, presented under the title "Therapeutic Doses of Efzofitimod Demonstrate Efficacy in Pulmonary Sarcoidosis," generated considerable interest within the medical community. Further, the findings were showcased at the CHEST 2024 annual meeting during the Best of CHEST Journals session, reflecting the growing recognition of efzofitimod's potential.

Financially, aTyr Pharma reported having $68.9 million in cash, cash equivalents, restricted cash, and investments as of September 30, 2024. After the quarter ended, the company raised approximately $19.4 million in gross proceeds through an at-the-market (ATM) offering with Jefferies LLC. The company believes that its current cash runway will suffice to fund operations through the filing of a Biologics License Application (BLA) for efzofitimod in pulmonary sarcoidosis.

For the third quarter of 2024, aTyr incurred research and development expenses amounting to $14.8 million, primarily due to clinical trial expenses for the EFZO-FIT™ and EFZO-CONNECT™ studies, as well as manufacturing and other R&D costs related to the efzofitimod program. General and administrative expenses stood at $3.3 million.

Efzofitimod is a pioneering biologic immunomodulator under development for treating interstitial lung disease (ILD). It functions by selectively modulating activated myeloid cells through neuropilin-2 to resolve inflammation without immune suppression, potentially preventing the progression of fibrosis. Given the limited treatment options available for ILD, efzofitimod could become a critical therapeutic tool in managing this condition.

aTyr Pharma continues to utilize evolutionary knowledge to transform tRNA synthetase biology into new therapeutic interventions targeting fibrosis and inflammation. Their focus on unveiling the signaling pathways influenced by tRNA synthetases has led to the development of efzofitimod as their leading therapeutic candidate. By targeting immune-mediated disorders that lead to lung inflammation and fibrosis, aTyr aims to provide effective, disease-modifying treatments that could significantly improve patient outcomes.