Tokai Pharmaceuticals, Inc., a biopharmaceutical company focused on developing new treatments for prostate cancer, today announced that the company raised $35.5 million in a Series E financing based on encouraging clinical progress and investor support for galeterone (TOK-001), Tokai's lead prostate cancer drug candidate. The proceeds will be used to expand the ongoing ARMOR2 Phase 2 clinical trial of galeterone in patients with castration-resistant prostate cancer (CRPC) and prepare for registration studies. The financing round included both current investors, Apple Tree Partners and Novartis Venture Funds, as well as undisclosed angel investors.

"We are extremely encouraged by the maturing data we are seeing from the ARMOR2 clinical trial," said Seth Harrison, M.D., chairman of the Tokai Board of Directors and managing general partner, Apple Tree Partners. "This additional funding will allow Tokai to complete the ARMOR2 study in broad CRPC patient populations to streamline Phase 3 clinical development path for galeterone."

"Based on its highly differentiated clinical profile, unique triple mechanism of action and the ARMOR2 clinical results to date, we believe that galeterone may be a promising new treatment option for all stages of CRPC," commented Reinhard J. Ambros, Ph.D., global head of Novartis Venture Funds. "We are pleased to continue to support Tokai in the development of this important new prostate cancer therapy."

ARMOR2 is the second study in Tokai's ARMOR (Androgen Receptor Modulation Optimized for Response) clinical development program for the evaluation of galeterone. ARMOR2 is a Phase 2 clinical trial evaluating the efficacy and safety of a new oral formulation of galeterone in distinct populations of CRPC patients, including both metastatic and non-metastatic treatment-naïve and those who have progressed while taking Zytiga® (abiraterone acetate) or Xtandi® (enzalutamide). The primary endpoints of the study are reduction in prostate-specific antigen (PSA) levels, and safety. The secondary endpoints include tumor responses by RECIST. Patients who respond to therapy will have the opportunity to continue treatment in an extension arm.

ARMOR2 follows the successfully completed ARMOR1 Phase 1 clinical trial in which an early formulation of galeterone demonstrated clinical activity and was well tolerated in patients with CRPC. The 49 patient dose-finding study was completed in 2012 and results were presented at the 2012 ASCO Annual Meeting. At the highest dose tested, significant PSA responses and tumor reduction rates were seen. There were no cases of secondary mineralocorticoid excess (ME), a clinical syndrome that is commonly associated with other CYP17 lyase inhibitors, observed and no concomitant prednisone therapy was required in patients in ARMOR1.

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 distinct properties. Results from the ARMOR1 Phase 1 clinical trial showed that galeterone demonstrated clinical activity and was well tolerated in patients with castration-resistant prostate cancer (CRPC). Galeterone is currently being evaluated in a Phase 2 study, ARMOR2, in patients with CRPC as part of the ARMOR (Androgen Receptor Modulation Optimized for Response) clinical development program.

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. Galeterone is currently being evaluated in a Phase 2 study, ARMOR2, in patients with castration-resistant prostate cancer (CRPC). Based in Cambridge, Massachusetts, Tokai was founded by Apple Tree Partners and is backed by Apple Tree and Novartis Venture Funds. For more information on the company and galeterone, please visit www.tokaipharma.com.