Ariad initiates Phase III trial of oral deforolimus

This pivotal trial will assess progression-free survival as the primary endpoint and overall survival as a secondary endpoint in patients with metastatic sarcomas following a favorable response to chemotherapy.

The sarcoma multi-center clinical evaluation of the efficacy of deforolimus (Succeed) trial is a randomized, double-blind, placebo-controlled Phase III study of oral deforolimus in metastatic sarcoma patients who have achieved a favorable response to chemotherapy. Continued treatment with traditional chemotherapeutic drugs has not been established to provide additional clinical benefit after such a response, according to Ariad.

This trial is designed to evaluate approximately 650 patients who will be randomized (1:1) to oral deforolimus or placebo at approximately 125 sites. The trial is 90% powered to detect a 33% increase in median PFS (corresponding to a hazard ratio of 0.75) comparing the deforolimus arm with the placebo arm. Two interim efficacy analyses are included. Complete patient enrollment and the second interim analysis are expected to take place within approximately two years of the first patient being enrolled.

George Demetri, director, the Ludwig Center at Dana-Farber Cancer Institute said: “The Succeed trial is one of the largest studies of patients with soft-tissue and bone sarcomas ever conducted – representing an expansive international collaboration of sarcoma experts to test a hypothesis that could shift the paradigm of how such patients are treated. In particular, this trial is designed to study a new treatment option vs. the current standard of ‘watchful waiting’ for disease progression. Thus, the trial will provide a unique opportunity to evaluate whether more active management of the disease prolongs progression-free survival over the traditional clinical approach.”

Harvey Berger, chairman and CEO of Ariad, added: “The start of the Succeed trial signals an important advance in our comprehensive global development of deforolimus in oncology. Deforolimus is a promising targeted oral treatment, and we look forward to obtaining further confirmation of its efficacy and safety in patients with metastatic sarcomas from this trial.”