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Drug intelligence / Profile preview
ORIN1001 is a first-in-class, orally bioavailable small molecule that selectively inhibits the endoribonuclease activity of inositol-requiring enzyme 1 alpha (IRE1α), a key mediator of the unfolded protein response (UPR) in the endoplasmic reticulum. By blocking IRE1α RNase activity, ORIN1001 prevents activation of X-Box Binding Protein 1 (XBP1), disrupting cellular stress responses that promote tumor cell survival and fibrosis. The drug has demonstrated preclinical anti-tumor and anti-fibrotic efficacy both as monotherapy and in combination with standard-of-care agents across multiple cancer types—including breast, prostate, lung, liver, pancreatic, brain, colon, ovarian, esophageal cancers—and idiopathic pulmonary fibrosis (IPF). ORIN1001 is under clinical investigation for advanced solid tumors and metastatic breast cancer as well as IPF. It has received FDA Fast Track Designation[3][4][5][6][7].
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