Roche tees up new app on promising Ph2 cancer drug

Lexington, MA-based Curis ($CRIS) is eagerly spreading the news that its partners at Roche/Genentech have wrapped a positive mid-stage trial for an experimental new treatment for advanced basal cell carcinoma. And the biotech adds that Roche plans to file at least one marketing application for the hedgehog pathway inhibitor, GDC-0449, later this year on the "pivotal" data.

Genentech is holding on to the data from the study for a scientific meeting to be announced, but Curis touted the fact that the therapy hit its primary endpoint of achieving a target overall response rate, "showing that GDC-0449 (now dubbed vismodegib) shrank advanced BCC tumors in a pre-defined percentage of people in the study." Vismodegib is designed to inhibit signaling in the Hedgehog pathway by targeting the Smoothened protein.

"We are extremely pleased by the highly encouraging outcome of this study, particularly since there is currently no standard of care for patients with this serious disease," said Curis CEO Dan Passeri. "We look forward to Genentech's presentation of the study data in more detail in the near future, and to its planned regulatory submissions for vismodegib."

The prospect of using Phase II data from a single-arm study of 104 patients to support a regulatory filing in the U.S. could prove problematic, though, considering Roche's experience with the highly promising "armed antibody" T-DM1. In a surprisingly conservative move, U.S. regulators handed back Roche's application on that breast cancer therapy last summer, despite highly promising mid-stage data on patients with no other treatment options. Roche, though, may go straight to Europe to see if it can garner a regulatory win at an early stage.

- here's the Curis release