smoothened inhibitor

The smoothened inhibitor from Dark Blue Therapeutics (acquired by Amgen in January 2026) is a novel small molecule targeting the Smoothened (SMO) receptor, a key G-protein coupled receptor in the developmental Hedgehog signaling pathway. Dysregulation of Hedgehog signaling is a known driver in various malignancies, promoting tumor growth, survival, and metastasis. This therapeutic candidate is being developed for the treatment of solid tumors, including basal cell carcinoma, pancreatic cancer, and esophageal cancer. It is designed to overcome the limitations of first-generation SMO inhibitors, such as challenging safety profiles and the emergence of drug-resistant mutations. The program is currently in the preclinical hit discovery phase, leveraging cancer biology insights from Oxford University to develop precision oncology therapies.

Other names

SMO inhibitorSmoothened antagonist