Curis was founded in Lexington, MA, in 2000 with the intention of exploiting a disease-causing mechanism curiously named the hedgehog signaling pathway. Three years later, the startup attracted the attention of biotech pioneer Genentech, whose scientists believed hedgehog might offer a brand new way to fight cancer. Now the development deal that the two companies formed back then is approaching a major milestone: Armed with positive results from a pivotal trial that it released June 21, South San Francisco-based Genentech (now owned by Roche) will apply this year for FDA approval to market the drug, called vismodegib, in patients with advanced basal cell carcinoma (BCC).
Vismodegib is proving to be a promising option for patients with BCC—a common and sometimes life-threatening form of skin cancer for which there are few treatment options. In the trial, the drug healed cancerous lesions, shrank tumors, or prevented them from growing further in more than 75 percent of patients. The median duration of response ranged from eight to 13 months, and 54 percent of patients whose cancer had not spread prior to the treatment showed no residual cancer in biopsies taken afterwards. “The data was very profound in terms of the drug being able to hold this cancer at bay,” Curis CEO Daniel Passeri says. Passeri hopes the FDA will grant the drug fast-track approval status and that it will be approved in 2012.
For Curis (NASDAQ: CRIS), the FDA filing will mark a welcome milestone in a startup journey that has been far from easy. The hedgehog signaling pathway, named after the funny-looking mutant fruit fly in which it was discovered, is a network of proteins that cells use to send signals to each other. The company, which came together through the merger of three small biotechs, was founded on the idea that hedgehog could be turned either off or on to treat a wide range of conditions—from serious diseases like cancer, to less serious but intriguing market opportunities like hair loss.
Yes, that’s right—Curis once seemed to have the miracle cure for baldness. The hedgehog signaling pathway is one of the biochemical processes that controls organ generation in embryos, and promotes tissue regeneration in adults. Curis had evidence that turning the pathway on with a drug would prompt the body’s stem cells to congregate around hair follicles, which in turn would cause hair to sprout from bare heads. The evidence was so compelling that in 2005, consumer-products giant Procter & Gamble (NYSE: PG) formed an R&D partnership with Curis that could have brought the biotech as much as $100 million in milestone payments.
But hedgehog experts also suspected that turning the pathway off could be a powerful inhibitor of cancer. That begged the question: Could the quest to cure baldness by flipping the hedgehog pathway on unwittingly raise the cancer risk for millions of men? Add to that the FDA’s increasingly tough stance on the potential for side effects from lifestyle drugs—those used to treat non-life-threatening conditions—and the baldness venture proved too risky for P&G and Curis. P&G pulled out of the partnership in 2007 because “the collaboration did not demonstrate an acceptable safety profile,” according to a statement. Curis abandoned the baldness program shortly thereafter.
Passeri says the hair-loss saga drove home some important lessons for Curis. “You need to be prudent in making your investments. You need to think about the practical … Next Page »
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