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Efzofitimod is a first-in-class biologic immunomodulator in clinical development for the treatment of interstitial lung disease (ILD), including pulmonary sarcoidosis and systemic sclerosis-related ILD. It is a tRNA synthetase-derived therapy based on a naturally occurring, lung-enriched splice variant of histidyl-tRNA synthetase (HARS). Efzofitimod functions as a selective modulator of activated myeloid cells by binding to neuropilin-2 (NRP2), an immune target upregulated at sites of inflammation. This interaction downregulates both innate and adaptive immune responses, inhibits pro-inflammatory receptors and cytokines, and potentially prevents progression of fibrosis without causing general immune suppression. The drug is administered intravenously and has shown dose-dependent improvements in clinical trials for pulmonary sarcoidosis. Efzofitimod has received orphan drug designation in the U.S., E.U., and Japan for sarcoidosis, as well as Fast Track designation in the U.S. for both pulmonary sarcoidosis and SSc-ILD[1][4][6][7].
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