Sapience Therapeutics, Inc., a US-based clinical-stage biotechnology company, announced on Thursday that it has received Orphan Drug Designation (ODD) from the US Food and Drug Administration (FDA) for ST316 for the treatment of familial adenomatous polyposis (FAP), a pre-malignant rare genetic condition that causes the formation of hundreds to thousands of polyps throughout the colon, typically starting in adolescence.

ST316 is a first-in-class antagonist of beta-catenin and its co-activator, BCL9, currently in a Phase 2 study for the treatment of CRC. ST316 is designed to selectively shut down the Wnt/beta-catenin signalling pathway, which is a key driver of FAP and more than 80% of CRCs, underscoring the importance of this pathway for therapeutic intervention.

Dr. Abi Vainstein-Haras, Sapience's chief medical officer, said: "We are thrilled that the FDA has granted Orphan Drug Designation to ST316 for the treatment of FAP. This award is an important regulatory milestone that advances our approach to targeting genetic alterations in the Wnt/beta-catenin signalling pathway, which are drivers of both pre-malignant and malignant disease. Other than high morbidity surgery and intensive colonoscopy surveillance, patients with FAP have no available treatment options to prevent their disease from progressing to CRC, the second-leading cause of cancer death in the United States. We look forward to continuing to work diligently to advance ST316 development in both FAP and CRC patient populations."