The Curis/Genentech drug (GDC-0449) is a drug that targets the hedgehog pathway.
The cancer is caused via either a MUTATION-driven mechanism of the HH pathway, (OR) it's LIGAND-driven.
In the case of Basal Cell Carcinoma and Medulloblastoma, these two are specifically MUTATION driven. GDC-0449 is EXTREMELY EFFECTIVE in mutation-driven cancers which the HH pathway is involved. It's almost, literally, a sure shot on goal (seriously, look at the NEJM pictures of the BCC trial)(as well as scans in medulloblastoma).
THE DRUG, IMO, is JUNK, in ligand-driven cancers (as seen in two recent Phase 2 trials - colon cancer and ovarian cancer). Colon was an outright failure. Ovarian, looks not much better. Hence, the junk penny stock price. LIGAND driven is a MUCH bigger market.
So, basically, in mutation driven cancers targeting HH pathway, they have a legitimate product that really hits the target hard, literally has incredible results (like the drug Plexxikon is working on).
