FDA Accepts Curis' NDA; Grants Priority Review

Shares of Curis, Inc. CRIS rose in early trading on Wednesday after the firm announced that the U.S. Food and Drug Administration (FDA) has accepted and filed for review Genentech’s new drug application for Vismodegib for the treatment of adults with advanced basal cell carcinoma for whom surgery is considered inappropriate.

As part of a milestone payment agreement from Genentech, Curis earned an $8 million upon the FDA’s acceptance of the NDA submission. If vismodegib receives FDA approval, Curis will also be entitled to receive an additional milestone payment as well as royalties on any future sales.

This particular application is based on clinical data from a pivotal Phase II study of vismodegib in patients with advanced basal cell carcinoma. 

The drug candidate, which is first-in-class, investigational, oral medicine designed to selectively inhibit signaling in the Hedgehog pathway and is being developed by Genentech, a member of the Roche group, under a collaboration agreement with Curis, has been granted priority review by the FDA. U.S. regulators grant priority review status to products that, if approved, would address an unmet medical need or are considered to be potentially significant therapeutic advancements over existing approved therapies in the treatment, diagnosis or prevention of a disease. 

The drug candidate presents a substantial market opportunity for the pharmaceutical firms since basal cell carcinoma is the most common cancer in the United States and the most common type of skin cancer, accounting for approximately two million new cases annually. The disease is generally considered curable when the cancer is restricted to a small area of the skin. However, in a small group of people, if the disease is left untreated or does not respond to treatment, the cancer may advance further into the skin, bones or other tissues, or spread to other parts of the body. In such rare cases, the disease can become difficult to treat and life-threatening.

We are noting on our FDA Calendar and Worldwide Regulatory Tracker database that the FDA has now established March 8, 2012 as the action date under the Prescription Drug User Fee Act (PDUFA) to complete its review of the NDA and potentially grant marketing approval of vismodegib. As previously noted under the same calendar database entrees for Curis, an SEC filing in March indicated that "assuming that submissions were filed by Roche and accepted by the applicable regulatory agency, they will be eligible to receive milestone payments for the U.S. and Europe territories." 

"We are extremely pleased that the FDA has accepted the vismodegib NDA for review and that the submission will receive priority review," said Dan Passeri, Curis President and Chief Executive Officer. "With no currently approved treatments for advanced basal cell carcinoma, this brings vismodegib one step closer to being an important treatment and the only approved pharmacologic treatment for those suffering from this serious disease."

Curis is engaged in utilizing medicinal chemistry and biological expertise to develop a series of proprietary targeted cancer drug programs. These programs are primarily focused on developing a number of proprietary, small molecule, single-agent, multi-targeted inhibitor drug compounds. Each proprietary compound is designed to inhibit biologically- or clinically-validated cancer targets, including EGFR, Her2, Bcr-Abl tyrosine kinase and phosphatidylinositol-3-kinase (PI3k), in combination with inhibition of HDAC, which is a validated non-kinase cancer target. HDAC inhibition is a core component in each of its multi-targeted inhibitors. 

Curis' product pipeline includes: GDC-0449 indicated for the treatment of advanced and operable basal cell carcinoma; CUDC-101, a multi-target first-in-class small molecule of the quinazoline class indicated for the treatment of advanced, refractory solid tumors; Debio 0932, a small molecule inhibitor of Heat Shock Protein 90 (Hsp90) indicated for the treatment of cancer; CUDC-907, an orally available, multi-targeted small molecule indicated for the treatment of cancer; etc. 

Curis has collaborations with Genentech, Inc., Debiopharm SA, Stryker Corp SYK, Amylin Pharmaceuticals, Inc AMLN, AGY Therapeutics, and others.

Nearly 6 million of the firm's 70.6 million outstanding shares are short the stock whose shares are trading only 14.5% from their 52-Wk High ($ 4.42 ).

A substantial number (53.70%) of the Company's shares are held by "smart money" institutional investors including: