aTyr Pharma announced a publication in Science Translational Medicine detailing the mechanism of action of efzofitimod, its lead therapeutic candidate for interstitial lung disease (ILD). The research demonstrates efzofitimod’s unique anti-inflammatory properties, working by targeting the neuropilin-2 (NRP2) receptor on macrophages. This action disrupts inflammatory pathways and potentially fibrosis.
This research is crucial for advancing ILD treatment. Current therapeutic options for ILD are limited, and many carry significant risks. Efzofitimod’s targeted mechanism, which avoids general immune suppression, offers a potentially safer and more effective approach to managing these chronic inflammatory conditions, potentially improving patient outcomes and quality of life. Clarifying the drug’s mechanism of action builds confidence in its therapeutic potential and may encourage further research into similar tRNA synthetase-based therapies.
The preclinical data revealed that efzofitimod, derived from a splice variant of histidyl-tRNA synthetase, binds specifically to the NRP2 receptor. This binding inhibits the expression of pro-inflammatory receptors and cytokines in macrophages, effectively downregulating inflammation and potentially preventing the progression of fibrosis. The research supports the ongoing Phase 3 EFZO-FIT™ study in pulmonary sarcoidosis and the Phase 2 EFZO-CONNECT™ study in systemic sclerosis-related ILD. Efzofitimod has received orphan drug designation in the U.S., EU, and Japan for sarcoidosis and Fast Track designation in the U.S. for both pulmonary sarcoidosis and systemic sclerosis-related ILD.
This publication strengthens the foundation for efzofitimod’s development as a novel ILD treatment. Positive results from the ongoing clinical trials could lead to a much-needed new therapy for patients suffering from these debilitating lung diseases. The novel mechanism of action may also open doors for the development of other tRNA synthetase-based therapeutics targeting chronic inflammatory conditions.
Jon Napitupulu is Director of Media Relations at The Clinical Trial Vanguard. Jon, a computer data scientist, focuses on the latest clinical trial industry news and trends.
