Curis Inc., a drug development company seeking to develop next generation targeted small molecule drug candidates for cancer treatment, and its collaborator Roche has submitted an application for marketing registration for Erivedge (vismodegib) to Australia's Therapeutic Goods Administration (TGA).

The application is currently under review by the TGA for the treatment of adults with advanced basal cell carcinoma (BCC) for whom surgery is inappropriate. Erivedge is a first-in-class oral medicine designed to selectively inhibit signaling in the Hedgehog pathway and is being developed by Roche and Genentech, under a collaboration agreement between Curis and Genentech, a member of the Roche Group.

Curis earned a $4 million milestone payment as a result of the submission of this application to the TGA. If Roche receives approval to commercialize Erivedge in Australia, Curis will also be entitled to receive an additional milestone payment as well as royalties on any future net sales of Erivedge in Australia.

“The Australian marketing application for Erivedge is further evidence of Roche's commitment to providing Erivedge to patients with advanced BCC around the world. In addition to Australia, Erivedge is currently under review for approval in Europe, Canada and Switzerland,” said Dan Passeri, president and CEO, Curis. “Approvals in these territories are expected to meaningfully expand patient access to Erivedge outside of the United States.”

The achievement of several key Erivedge regulatory objectives by Genentech and Roche, including the launch of Erivedge in the United States, has resulted in Curis earning $28 million in milestone payments in the previous six months. Curis currently expects to receive royalty payments on US sales and is eligible for milestone payments should Erivedge be approved in Europe or Australia. Curis will also receive royalty payments on net sales of Erivedge in Europe, Australia and any other country where Erivedge is approved and commercialized.

Erivedge is approved in the US for the treatment of adults with BCC that has spread to other parts of the body or that has come back after surgery or that their healthcare provider decides cannot be treated with surgery or radiation.

The application to the TGA is based on clinical data from ERIVANCE BCC/SHH4476g, a pivotal phase II study of vismodegib in patients with advanced BCC. The results were presented at the Seventh European Association of Dermato-Oncology (EADO) Congress in July 2011 as well as at the European Multidisciplinary Cancer Congress (EMCC) in September 2011.

BCC is the most common type of skin cancer in Europe, Australia and the United States. The disease is generally considered curable if the cancer is restricted to a small area of the skin. In advanced BCC, if the disease is left untreated or recurs in the same location after surgery or radiotherapy, it may advance further into surrounding areas such as sensory organs (ears, nose and eyes), bone, or other tissues. Depending on the location of the lesion, some cases of advanced BCC can be disfiguring, and treatment with surgery or radiation can lead to the loss of sensory organs and their functions such as eyesight or hearing.

The Hedgehog signaling pathway plays an important role in regulating proper growth and development in the early stages of life and becomes less active in adults. Abnormal Hedgehog signaling is implicated in more than 90 percent of BCC cases.

Under the ongoing collaboration agreement between Genentech, a wholly owned member of the Roche Group, and Curis, Erivedge (vismodegib) was discovered by Genentech and was jointly validated by the parties through a series of preclinical studies. Pursuant to this collaboration, Genentech and Roche are responsible for clinical development, and Genentech (US), Roche (Ex-US excluding Japan) and Chugai Pharmaceuticals (Japan) are responsible for commercialization of Erivedge. Curis is eligible to receive cash payments upon the successful achievement of specified clinical development and regulatory approval milestones, as well as royalties assuming successful commercialization of Erivedge by Genentech and its sublicensees, which include Roche and Chugai.

Roche and Genentech are also evaluating Erivedge in a Phase II trial in people with operable forms of BCC. Furthermore, the potential of Erivedge is being evaluated by third-party investigators in a number of other disease areas.