Tokai Pharmaceuticals Announces Expansion to Collaboration with Qiagen on the Development of an AR-V7 Companion Diagnostic

Tokai and Qiagen Expect to Develop AR-V7 Clinical Trial Assay Prior to Opening ARMOR3-SV in 1H’15

Tokai Granted Exclusive Right to Qiagen’s Circulating Tumor Cell (CTC) Enrichment Technology for use with a Companion Diagnostic Paired with Galeterone

CAMBRIDGE, Mass.--()--Tokai Pharmaceuticals, Inc. (NASDAQ:TKAI), a clinical-stage biopharmaceutical company focused on developing novel therapies for prostate cancer and other hormonally driven diseases, today announced an expansion of the agreement with Qiagen (NASDAQ: QGEN; Frankfurt Prime Standard: QIA) for the development and commercialization of an AR-V7 companion diagnostic for use with galeterone.

Under the expanded agreement, Tokai will receive the exclusive right from Qiagen to its newly acquired circulating tumor cell (CTC) enrichment technology for use with galeterone, which will be incorporated into the companion diagnostic already under development by Qiagen.

“We are pleased to be expanding our partnership with Qiagen with exclusive access to their newly acquired CTC enrichment technology for use with the AR-V7 companion diagnostic for galeterone,” stated Jodie Morrison, president and chief executive officer of Tokai Pharmaceuticals. “This access adds further support to our growing IP portfolio for galeterone for AR-V7 positive castration-resistant prostate cancer (CRPC). Qiagen is a global leader in liquid biopsy-based solutions for precision medicine and has the expertise to commercialize the companion diagnostic around the world. This global capability will be critical as we work to bring galeterone to prostate cancer patients who test positive for AR-V7, which has been linked to non-responsiveness to commonly used oral therapies. It is our belief that future availability of this companion diagnostic for AR-V7 will allow prostate cancer patients and their physicians to make more informed decisions regarding their care.”

Development of an AR-V7 companion diagnostic for use with galeterone has been underway since October 2014. Qiagen’s newly acquired CTC technology was utilized in the AR-V7 assay methods developed by the Johns Hopkins University licensed by Tokai in January. Tokai and Qiagen expect that development of the AR-V7 clinical trial assay will be completed in the first half of 2015 prior to the start of the ARMOR3-SV AR-V7 metastatic CRPC registration clinical trial.

AR-V7 positive prostate tumors express a truncated form of the androgen receptor (AR). These truncated ARs are missing the C-terminal end of the receptor that contains the ligand binding domain. In 2014, the Johns Hopkins University, in a prospective study using the Qiagen technology to isolate and enrich CTCs and an assay to determine AR-V7 status, demonstrated poor responsiveness to Zytiga® (abiraterone acetate) and Xtandi® (enzalutamide), two commonly used oral therapies for metastatic CRPC. The Company believes that galeterone has the potential to treat patients with AR-V7 based on data from preclinical studies and retrospective data in patients with C-terminal loss, the most common form of which is AR-V7, from the Company’s Phase 2 ARMOR2 trial of galeterone.

“Our partnership with Tokai Pharmaceuticals, one of the collaborations which we are pursuing with pharma in this area, is expected to result in a liquid biopsy, CTC-based test for the AR-V7 splice variant in the first half of this year, with the potential to enhance outcomes for prostate cancer patients,” said Peer M. Schatz, chief executive officer of Qiagen. “Following the success of the first-ever regulated companion diagnostic for solid tumors based on molecular biomarkers from a liquid biopsy in Europe, we are expanding our portfolio of highly accurate tests that analyze samples of body fluids that are non-invasive and more accessible than traditional tissue biopsies. Our liquid biopsy portfolio holds potential to create valuable insights and improve outcomes for patients.”

About Galeterone

Galeterone is a highly selective, multi-targeted, oral small molecule drug candidate being developed for the treatment of CRPC that acts by actively disrupting AR signaling, the key driver of prostate cancer growth, via multiple mechanisms of action. Galeterone combines the mechanisms of action of CYP17 inhibition and androgen receptor antagonism with an additional mechanism of androgen receptor degradation. To Tokai’s knowledge, galeterone is the only drug candidate currently in clinical trials that disrupts the AR signaling pathway at multiple points and includes a mechanism of AR degradation.

Preclinical evidence suggests galeterone’s mechanism of androgen receptor degradation does not require a functional ligand binding domain to disrupt the activation of the pathway and tumor growth and thus galeterone may have activity even in the presence of C-terminal loss, such as AR-V7. In a retrospective analysis of the Phase 2 ARMOR2 trial, galeterone was associated with clinical responses in patients identified as having C-terminal loss, with 6 of 7 patients achieving a 12-week PSA50 response (the one non-responder discontinued therapy due to an adverse event unrelated to galeterone and did not receive the full treatment regimen).

About the ARMOR3-SV Clinical Trial

ARMOR3-SV is a randomized, open label Phase 3 clinical trial that will compare galeterone to Xtandi® (enzalutamide) in 148 metastatic CRPC treatment-naïve patients whose prostate tumors express the AR-V7 splice variant. The primary endpoint of the trial will be radiographic progression-free survival measured from the time of patient randomization to the time of radiographic evidence of disease progression or time of death from any cause. The Company expects to commence the trial in the first half of 2015.

About Tokai Pharmaceuticals

Tokai Pharmaceuticals is a biopharmaceutical company focused on developing novel therapies for prostate cancer and other hormonally-driven diseases. The company’s lead drug candidate, galeterone, is a highly selective, multi-targeted, oral small molecule drug candidate being developed for the treatment of patients with castration-resistant prostate cancer. The Company’s ARDA drug discovery program is focused on the identification and evaluation of compounds that are designed to disrupt androgen receptor signaling through enhanced androgen receptor degradation and are targeted to patients with androgen receptor signaling diseases, including prostate cancer. For more information on the company and galeterone, please visit www.tokaipharma.com.

Forward-looking Statements

Any statements in this press release about future expectations, plans and prospects for the Company, including statements about the Company’s strategy, future operations, intellectual property, and other statements containing the words “believes,” “anticipates,” “plans,” “expects,” and similar expressions, constitute forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including: whether the Company’s cash resources will be sufficient to fund the Company’s continuing operations for the period anticipated; whether data from early clinical trials will be indicative of the data that will be obtained from future clinical trials; whether galeterone will advance through the clinical trial process on the anticipated timeline, including whether ARMOR3-SV will be initiated when anticipated; whether a companion diagnostic can be developed successfully and on a timely basis; whether the results of ARMOR3-SV will warrant submission for regulatory approval of galeterone and whether such submission will receive approval from the United States Food and Drug Administration or equivalent foreign regulatory agencies; whether, if galeterone obtains such approval, it will be successfully distributed and marketed; and other factors discussed in the “Risk Factors” section of the Company’s quarterly report on Form 10-Q for the quarter ended September 30, 2014. In addition, the forward-looking statements included in this press release represent the Company’s views as of March 16, 2015. The Company anticipates that subsequent events and developments may cause the Company’s views to change. However, while the Company may elect to update these forward-looking statements at some point in the future, the Company specifically disclaims any obligation to do so. These forward-looking statements should not be relied upon as representing the Company’s views as of any date subsequent to March 16, 2015.

Contacts

Investor Contact:
Tokai Pharmaceuticals
Lee Kalowski, 617-225-4305
Chief Financial Officer
or
Media Contact:
Pure Communications, Inc.
Susan Heins, 864-286-9597

Release Summary

Tokai Pharmaceuticals Announces Expansion to Collaboration with Qiagen on the Development of an AR-V7 Companion Diagnostic

Contacts

Investor Contact:
Tokai Pharmaceuticals
Lee Kalowski, 617-225-4305
Chief Financial Officer
or
Media Contact:
Pure Communications, Inc.
Susan Heins, 864-286-9597