Genentech announced that a Phase 2 clinical study showed positive results of its investigational Hedgehog Pathway Inhibitor, vismodegib (RG3616/GDC-0449) in people with advanced basal cell carcinoma, a particularly severe and debilitating form of skin cancer. The study met its primary endpoint of achieving a target overall response rate, showing that vismodegib shrank tumors in a predefined percentage of people in the study. A preliminary safety assessment showed the most common adverse events were consistent with previous experience with vismodegib. A detailed safety assessment is ongoing.

The Hedgehog signaling pathway plays an important role in regulating proper growth and development in the early stages of life and then becomes less active in adults. However, mutations in the pathway that reactivate Hedgehog signaling are seen in several different types of cancer. Abnormal signaling in the Hedgehog pathway is implicated in the majority of basal cell carcinoma cases.

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