Sarcomas are a group of aggressive cancers of connective tissue in the body for which there are currently limited treatment options. ARIAD is conducting a Phase 3 clinical trial of ridaforolimus (formerly called deforolimus) in patients with metastatic soft-tissue and bone sarcomas.
In 2009, the American Cancer Society estimates that approximately 10,600 new cases of soft-tissue sarcomas will be diagnosed in the United States, and more than 3,800 Americans will die of the disease. In addition, approximately 2,600 new cases of bone sarcomas will be diagnosed and nearly 1,500 deaths are estimated.* Sadly, there has been no new approved therapy in the U.S. for patients with soft-tissue or bone sarcomas in more than 25 years.
Ridaforolimus has been designated both as a fast-track and orphan drug product by the U.S. Food and Drug Administration (FDA) and as an orphan drug by the European Medicines Agency (EMEA) for the treatment of soft-tissue and bone sarcomas. ARIAD is pursuing this indication as the initial registration path for ridaforolimus.
Phase 3 SUCCEED clinical trial in sarcoma
The SUCCEED trial, a global Phase 3 study of oral ridaforolimus in patients with metastatic soft-tissue and bone sarcomas, is now fully enrolled. This pivotal trial, which ARIAD is conducting in collaboration with Merck & Co., Inc., will assess progression-free survival (PFS) as the primary endpoint and overall survival as a secondary endpoint in patients with metastatic sarcomas following a favorable response to chemotherapy.
We have reached agreement on a Special Protocol Assessment (SPA) with the FDA for the SUCCEED clinical trial. The EMEA has provided protocol advice consistent with that of the FDA regarding the trial design as part of its Protocol Assistance program.
Positive clinical results from ridaforolimus Phase 2 trial in sarcoma
In a multi-center Phase 2 trial of 212 patients with advanced sarcomas, at least 90 percent of whom had progressive disease, ridaforolimus demonstrated efficacy and was well tolerated. The trial achieved its primary endpoint—evidenced by clinical-benefit response (CBR) rates—in the three most prevalent types of sarcoma (bone sarcoma, leiomyosarcoma and liposarcoma). Treatment with ridaforolimus more than doubled progression-free survival when compared with historical control data published by the European Organization for Research and Treatment of Cancer (EORTC).
At the 2007 annual meeting of the American Society of Clinical Oncology (ASCO), we announced that further analysis of the Phase 2 trial of ridaforolimus demonstrated that documented disease stabilization and/or tumor regression with single-agent ridaforolimus is a strong predictor of improved overall survival. Specifically, patients with a ridaforolimus CBR—tumor regression or disease stabilization for at least 16 weeks—had a median overall survival of approximately 17 months, nearly double that of the overall trial population (approximately 9 months).
* Source: American Cancer Society, Cancer Facts & Figures 2008.
