SOUTH SAN FRANCISCO, Calif., Sep 07, 2011 (BUSINESS WIRE) -- Exelixis, Inc. /quotes/zigman/82395/quotes/nls/exel EXEL +6.65% today announced that the pre-specified number of progression-free survival (PFS) events required for un-blinding of the data for the ongoing phase 3 pivotal trial of cabozantinib in patients with medullary thyroid cancer (MTC), known as the EXAM trial, has been reached. Final data analysis in preparation for un-blinding is underway, and the company expects to report top-line data early in the fourth quarter of 2011.
"We are pleased to have reached this important milestone in the EXAM trial and to now be finalizing our internal work to report top-line results of this important phase 3 trial for Exelixis," said Michael Morrissey, president and chief executive office of Exelixis. "We view MTC as the first building block in the cabozantinib franchise, which we expect to complement with the planned initiation of our pivotal trial in castration-resistant prostate cancer later this year."
EXAM Trial Design
EXAM is an international, randomized, placebo-controlled, double-blinded study of cabozantinib in patients with unresectable, locally advanced, or metastatic MTC. Patients with documented progressive disease were randomized in a 2:1 ratio to receive cabozantinib or placebo administered at a daily dose of 175 mg. The study does not allow for cross-over from the placebo arm to cabozantinib. With an enrollment target of 315 patients and a planned event-driven analysis, the trial provides 90% power to detect a 75% increase in progression-free survival, the primary endpoint of the study. Additionally, the study is designed to assess overall survival at a later time point once those events have been achieved, and is powered to detect a 50% improvement in survival compared with placebo. Exelixis is conducting this trial under a Special Protocol Assessment from the FDA, which allows for full approval on the basis of PFS if the data are supportive. EXAM completed enrollment in the first quarter of 2011.
Exelixis, Inc. is a biotechnology company committed to developing small molecule therapeutics for the treatment of cancer. Exelixis is focusing its resources and development efforts exclusively on cabozantinib, its most advanced solely-owned product candidate, in order to maximize the therapeutic and commercial potential of this compound. Exelixis believes cabozantinib has the potential to be a high-quality, differentiated pharmaceutical product that can make a meaningful difference in the lives of patients. Exelixis has also established a portfolio of other novel compounds that it believes have the potential to address serious unmet medical needs. For more information, please visit the company's web site at www.exelixis.com .