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Tokai Pharmaceuticals' Galeterone Phase 1 Data in Patients with Advanced Prostate Cancer to be Presented at ASCO Annual Meeting 2012

Novel Triple Mechanism of Action Highlighted

Galeterone Demonstrated Promising Efficacy and Was Well-Tolerated with No Dose Limiting Toxicities

CAMBRIDGE, Mass. & CHICAGO--(BUSINESS WIRE)--May 17, 2012 - Tokai Pharmaceuticals, Inc., a biopharmaceutical company focused on developing new treatments for prostate cancer, today announced that additional data from a Phase 1 safety and proof-of-concept study evaluating lead candidate, galeterone (TOK-001), in patients with castration-resistant prostate cancer (CRPC) will be highlighted in a poster presentation titled, “Phase I clinical trial of galeterone (TOK-001), a multifunctional anti-androgen and CYP17 inhibitor in castration resistant prostate cancer (CRPC),” abstract number 4665, on Sunday, June 3, 2012, 8:00 a.m. to 12:00 p.m. (CDT) at the 2012 Annual Meeting of the American Society of Clinical Oncology (ASCO).

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. The study findings, presented for the first time last month at the AACR Annual Meeting, show galeterone demonstrated efficacy and was well-tolerated in patients with CRPC. Additionally, preclinical data will be presented at ASCO elucidating the mechanisms of action of galeterone.

“While other approved and experimental CRPC therapies act via a single target, galeterone is highly differentiated as the only prostate cancer drug in development that combines three distinct mechanisms of action in one compound for a unique multi-target approach,” said Martin D. Williams, president and chief executive officer, Tokai Pharmaceuticals. “Galeterone acts in three ways against the key driver of CRPC, androgen receptor signaling – it blocks ligand synthesis, blocks ligand receptor binding, and degrades the receptor itself. The efficacy observed in the galeterone ARMOR1 study, with both biochemical and radiological responses, combined with a favorable safety profile, support our Phase 2 clinical trial program to be initiated in the second half of 2012.”

Study Overview and Key Findings

The ARMOR1 study was 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, and their colleagues at leading prostate cancer research centers in the United States. The study included sites in the Prostate Cancer Clinical Trial Consortium (PCCTC). The Phase 1 proof-of-concept clinical trial, part of the ARMOR (Androgen Receptor Modulation Optimized for Response) clinical development program for the evaluation of galeterone, enrolled 49 patients and was a dose-finding study to evaluate escalating dose levels of galeterone. The study also evaluated safety and reduction in prostate-specific antigen (PSA) levels from baseline levels measured at first visit. Patients who responded to therapy had the option to continue treatment with galeterone in an extension arm of the trial.

“Cancer cells mutate and change to get around drugs and resistance to therapy is a growing concern in treating cancer,” Dr. Montgomery, lead author of the poster, commented. “Galeterone has a novel, combined mechanism of action that hits the receptor in three different ways and may help prevent resistance. These early data show it is a well-tolerated drug and suggest it could potentially be more effective than drugs we have now.”

Efficacy tests demonstrated that 24 patients had prostate-specific antigen (PSA) reductions of 30 percent or more; 11 of these patients had reductions of 50 percent or more. In addition, CT scans revealed a significant reduction in tumor size for some patients.

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 acts as a highly selective CYP17 lyase inhibitor, as an androgen receptor antagonist, and decreases androgen receptor levels in prostate tumors – the only drug in development that has been shown to exhibit all three of these properties. In galeterone, these three distinct mechanisms of action are combined in one therapeutic compound.

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. Privately held Tokai is based in Cambridge, Massachusetts and is backed by Apple Tree Partners and Novartis Venture Fund. For more information on the company and galeterone, please visit


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Posted: May 2012