Therapeutic Targets: Hedgehog
The Hedgehog signaling pathway represents a new way of understanding and potentially attacking the progression and recurrence of cancer.
The Hedgehog pathway is normally active during embryonic development and regulates tissue and organ formation. Malignant activation of the Hedgehog pathway is responsible for a broad range of cancers through three distinct mechanisms: genetic activation of the Hedgehog pathway in tumor cells, signaling to the tumor microenvironment and signaling to tumor progenitor cells.
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We are developing saridegib (also known as IPI-926), a small molecule that inhibits Smoothened (Smo), a key component of the Hedgehog pathway. Smo inhibition represents a significant anti-cancer opportunity for addressing a number of difficult-to-treat cancers by disrupting malignant activation of the pathway.
Saridegib is currently being evaluated in two Phase 2 trials exploring distinct biological hypotheses – one in patients with metastatic or locally advanced, inoperable chondrosarcoma and the other in patients with myelofibrosis. Saridegib was well-tolerated and showed clinical activity in Phase 1 trials. These clinical trials build upon a robust set of supporting preclinical data that provide a strong rationale for evaluating the potential of saridegib for treatment of a broad range of cancers.