aTyr Pharma Reports Q2 2024 Results and Corporate Update

In the second quarter of 2024, aTyr Pharma, Inc., a clinical-stage biotechnology company specializing in first-in-class medicines from its tRNA synthetase platform, achieved a significant milestone by completing enrollment for its global pivotal Phase 3 EFZO-FIT™ study. This study, focusing on the efficacy and safety of efzofitimod for pulmonary sarcoidosis—a major form of interstitial lung disease (ILD)—has successfully enrolled 268 patients across 85 centers in nine countries. The large-scale study is anticipated to provide topline data in the third quarter of 2025.

Dr. Sanjay S. Shukla, President and CEO of aTyr, emphasized the importance of this study, noting it as the largest interventional study ever conducted for sarcoidosis. This could potentially introduce a transformative therapy for patients who have been awaiting new treatment options for over six decades. Participants who complete this 52-week study are eligible for an Individual Patient Expanded Access Program (EAP) for continued treatment with efzofitimod.

Additionally, the company is advancing its Phase 2 EFZO-CONNECT™ study, which aims to assess the efficacy, safety, and tolerability of efzofitimod in patients with systemic sclerosis (SSc, or scleroderma)-related ILD. This randomized, double-blind, placebo-controlled study will span 28 weeks and involve three parallel cohorts, with doses administered intravenously monthly for six cycles. The study aims to enroll up to 25 patients and has incorporated a 24-week open-label extension for ongoing treatment post-study. Interim data from this study is expected by the second quarter of 2025.

During the second quarter, aTyr also presented a poster at the American Thoracic Society (ATS) 2024 International Conference, highlighting efzofitimod’s mechanism of action. The presentation underscored the role of neuropilin-2 (NRP2) as a significant immune target in ILD, showing that efzofitimod modulates myeloid cells to provide anti-inflammatory benefits.

Furthermore, aTyr entered into a research agreement with Stanford Medicine to investigate the potential of anti-NRP2 antibodies in treating glioblastoma multiforme (GBM), a prevalent primary brain cancer. Dr. Michael Lim, Chair of the Department of Neurosurgery at Stanford Medicine, will lead this research, exploring how these antibodies, in combination with chemotherapy, might reverse immune evasion mechanisms in GBM.

The company also strengthened its leadership team by appointing Dr. Jayant Aphale as Vice President of Technical Operations. With over 30 years of experience in biotechnology and pharmaceutical manufacturing, Dr. Aphale will oversee manufacturing activities and strategies related to commercial production, supply chain management, and process development of new products.

Financially, aTyr ended the second quarter of 2024 with $81.4 million in cash, equivalents, restricted cash, and investments. Research and development expenses for the quarter were $14.0 million, driven primarily by costs associated with the Phase 3 EFZO-FIT™ and Phase 2 EFZO-CONNECT™ studies, as well as manufacturing and research expenses for the efzofitimod programs. General and administrative expenses totaled $3.3 million for the quarter.

Efzofitimod, aTyr’s lead therapeutic candidate, is a first-in-class biologic immunomodulator in development for treating ILD. This tRNA synthetase-derived therapy selectively targets activated myeloid cells through NRP2, aiming to reduce inflammation without suppressing the immune system and potentially preventing the progression of fibrosis. Given the limited therapeutic options for ILD and the need for safer, more effective treatments, efzofitimod represents a promising new approach.

aTyr Pharma continues to leverage its proprietary tRNA synthetase platform to uncover novel therapeutic pathways for treating fibrosis and inflammation-related diseases. By translating the evolutionary biology of tRNA synthetases into new therapies, the company aims to address critical unmet medical needs in various immune-mediated disorders.