aTyr Pharma's ATYR0101 Reaches IND Candidate Stage for Pulmonary Fibrosis

aTyr Pharma, a biotechnology enterprise at the forefront of developing innovative medicines, has announced a significant advancement in their research pipeline with the progress of ATYR0101. This investigational new drug candidate will be highlighted in an oral presentation at the American Thoracic Society’s 2025 Respiratory Innovation Summit in San Francisco. Scheduled for May 16-17, the summit will serve as a platform to share compelling preclinical data surrounding ATYR0101, a fusion protein derived from the extracellular domain of aspartyl-tRNA synthetase (DARS).

The key mechanism of ATYR0101 involves its interaction with latent transforming growth factor beta binding protein 1 (LTBP-1), triggering apoptosis in myofibroblasts. These cells are largely implicated in the advancement of fibrosis, a condition that significantly contributes to morbidity and mortality in various diseases, especially those affecting the lungs. Dr. Leslie A. Nangle, aTyr’s Vice President of Research, emphasized the potential of ATYR0101 to reverse fibrosis, especially in pulmonary contexts where current treatments merely decelerate disease progression.

Sanjay S. Shukla, the President and CEO of aTyr, highlighted the promising trajectory of efzofitimod, a lead therapy derived from their tRNA synthetase platform, which has shown positive clinical outcomes in interstitial lung disease. Building on this success, Shukla expressed enthusiasm for advancing ATYR0101 into studies that enable Investigational New Drug (IND) applications. This next step underscores the transformative promise of aTyr’s drug discovery platform and its dedication to developing therapies targeting inflammation and fibrosis. The company aims to submit an IND application for ATYR0101 in the latter half of 2026.

The upcoming presentation, titled “ATYR0101: A New Approach to Fibrosis,” will be delivered by Ryan Adams, Ph.D., Senior Director of In Vitro Biology at aTyr, during the showcase session on ILD/Fibrosis. Attendees at the San Francisco Marriott Marquis on May 17 will gain insights into the novel approach ATYR0101 represents in targeting fibrotic diseases.

ATYR0101, as detailed by aTyr, derives from a unique extracellular domain of DARS, interacting distinctly with LTBP-1 to induce apoptosis in myofibroblasts. The protein’s efficacy has been demonstrated in preclinical models, suggesting broad therapeutic applicability across various fibrotic diseases beyond the lungs, including potential benefits in kidney fibrosis. Currently undergoing studies to enable IND application, ATYR0101 illustrates the potential of aTyr’s innovative approach to drug development.

aTyr Pharma specializes in the translation of tRNA synthetase biology into novel therapies targeting fibrosis and inflammation. The company leverages evolutionary intelligence to unlock hidden therapeutic intervention points, with a focus on signaling pathways driven by its proprietary library derived from all 20 tRNA synthetases. Their flagship therapeutic candidate, efzofitimod, is in clinical development for treating interstitial lung disease—a group of disorders causing lung inflammation and fibrosis.

The company’s research reflects a commitment to pioneering first-in-class medicines that address significant unmet needs in fibrotic and inflammatory conditions. By exploring the unique functionalities of tRNA synthetases, aTyr continues to push the boundaries of drug discovery and development, aiming to make significant strides in the fight against pulmonary and systemic fibrotic diseases.