Tokai Pharmaceuticals Doses First Patient in a Phase 2 Study of Galeterone in Advanced Prostate Cancer

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Tokai Pharmaceuticals Doses First Patient in a Phase 2 Study of Galeterone in Advanced Prostate Cancer
Galeterone Offers Unique Multi-Target Approach to Treating Prostate Cancer

December 13, 2012

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Tokai Pharmaceuticals, Inc., a biopharmaceutical company focused on developing new treatments for prostate cancer, today announced the treatment of the first patient in the ARMOR2 trial, a Phase 2 clinical study of its lead candidate galeterone (TOK-001) in patients with castration-resistant prostate cancer (CRPC). ARMOR2 is the second study in Tokai's ARMOR (Androgen Receptor Modulation Optimized for Response) clinical development program for the evaluation of galeterone. Dosing occurred at the Urology Cancer Center and GU Research Network in Omaha, Neb. under the direction of Luke Nordquist, M.D., F.A.C.P.

CRPC is an advanced, difficult-to-treat form of prostate cancer that occurs when the disease progresses, despite the use of androgen deprivation therapy. Galeterone is a proprietary small molecule, oral drug for the treatment of CRPC that disrupts the growth and survival of prostate cancer cells via a novel triple mechanism of action. Results from the ARMOR1 Phase 1 proof-of-concept clinical trial demonstrated that galeterone had clinical activity and was well tolerated in patients with CRPC; these data were presented at the American Association for Cancer Research (AACR) Annual Meeting 2012 and the 2012 Annual Meeting of the American Society of Clinical Oncology (ASCO).

"Galeterone is unique in that it is the first and only single agent therapeutic that combines three distinct approaches to attack prostate cancer, acting as a selective CYP17 lyase inhibitor, an androgen receptor antagonist and by decreasing overall androgen receptor levels in prostate cancer tumors cells in preclinical studies," said Philip Kantoff, M.D., chief clinical research officer, chief, division of solid tumor oncology, and director, Lank Center for Genitourinary Oncology at the Dana-Farber Cancer Institute; professor of medicine, Harvard Medical School. "We are excited to further explore the clinical utility of galeterone in CRPC, a cancer for which additional new therapies with improved tolerability and novel mechanisms of action are needed to address the emergence of resistance of existing agents."

ARMOR2 is a Phase 2 clinical trial to evaluate the efficacy and safety of a new oral formulation of galeterone in distinct populations of CRPC patients, including those who are treatment-naïve and those who have progressed while taking the androgen-directed hormonal therapy, Zytiga® (abiraterone acetate). Up to 196 patients will be enrolled in the trial. The primary endpoints of the study are reduction in prostate-specific antigen (PSA) levels and safety. Patients who respond to therapy will have the opportunity to continue treatment in an extension arm of the trial.

"The initiation of ARMOR2 comes on the heels of several important milestones for Tokai this year, including the presentation of successful results from our ARMOR1 study at ASCO and AACR, and Fast Track designation for galeterone from the FDA, which reinforces the unmet need in CRPC and validates the potential for galeterone as a new treatment option for this patient population," said Martin D. Williams, president and chief executive officer, Tokai Pharmaceuticals. "With our improved new tablet formulation, ARMOR2 will provide us with additional important insights on the clinical utility of galeterone in several distinct CRPC patient populations, informing us for our Phase 3 planning."

ARMOR2 is being conducted globally at leading prostate cancer treatment centers. The study is being led by co-principal investigators Bruce Montgomery, M.D., associate professor of medical oncology at the University of Washington School of Medicine and Mary-Ellen Taplin, M.D., associate professor of medicine, director of genitourinary clinical research, Dana-Farber Cancer Institute, Harvard Medical School. Please refer to www.clinicaltrials.gov for additional clinical trial details and enrollment information.

About Galeterone (TOK-001)

Galeterone is a proprietary small molecule, oral drug for the treatment of prostate cancer that disrupts androgen receptor signaling via a novel triple mechanism of action. In preclinical studies, galeterone acted as a highly selective CYP17 lyase inhibitor, as a potent androgen receptor antagonist, and decreased androgen receptor levels in prostate tumors – the only drug in development that has been shown to exhibit all three of these properties. Galeterone combines these three distinct mechanisms of action in one therapeutic compound. Results from the ARMOR1 Phase 1 clinical trial showed that galeterone demonstrated clinical activity and was well tolerated in patients with CRPC.

About Tokai Pharmaceuticals

Tokai Pharmaceuticals is a U.S. biopharmaceutical company focused on developing new treatments for prostate cancer. The company's lead drug candidate, galeterone (TOK-001), is the first investigational new drug that can decrease overall androgen receptor levels in prostate tumors and in which three distinct mechanisms of action are combined to disrupt androgen receptor signaling in one oncotherapeutic. Based in Cambridge, Massachusetts, Tokai is backed by Apple Tree Partners and Novartis Venture Fund. For more information on the company and galeterone, please visit www.tokaipharma.com.

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