Investing in the Future of Precision Cancer Medicine

Developing a first-in-class, 3rd generation Polo-like Kinase 1 (PLK1) Inhibitor to treat leukemias, lymphomas
and solid tumor cancers

Trovagene Presents Data at AACR Meeting 2018 Showing Synergy of PCM-075 in Combination with FLT3 Inhibitors in Acute Myeloid Leukemia (AML)

Combination of PCM-075, a highly-selective Polo-like Kinase 1 (PLK1) inhibitor, and Quizartinib resulted in 97% tumor growth inhibition and regression in FLT3 AML model

Apr 16, 2018

SAN DIEGO, April 16, 2018 /PRNewswire/ -- Trovagene, Inc. (NASDAQ: TROV), a clinical-stage oncology therapeutics company, developing targeted therapeutics for the treatment of hematologic and solid tumor cancers, today announced the presentation of data showing that PCM-075 exhibits synergistic activity when combined with FLT3 inhibitors in a human xenograft acute myeloid leukemia (AML) model, at the American Association for Cancer Research (AACR) Annual Meeting in Chicago, IL.

A molecular diagnostics company with unique intellectual property around the discovery that DNA and RNA, present in urine, can be diagnostic of cancer and infectious disease. (PRNewsFoto/Trovagene, Inc.)

The poster entitled Selective Polo-like Kinase 1 (PLK1) Inhibitor PCM-075 is Highly Active Alone and Shows Synergy When Combined with FLT3 Inhibitors in Models of Acute Myeloid Leukemia (AML) presents data demonstrating that PCM-075 in combination with quizartinib (Daiichi-Sankyo) resulted in 97.3% tumor growth inhibition (TGI), compared to 77.9% with quizartinib and 80.2% with PCM-075 as monotherapy.

Additionally, in other in vitro data presented, PCM-075 was found to have synergistic effects when combined with therapies used routinely in many hematologic and solid tumor cancers, including paclitaxel, sorafenib, doxorubicin and cytarabine.

"We are encouraged by the synergy we are seeing when PCM-075 is combined with targeted and standard-of-care chemotherapies in preclinical studies," said Mark Erlander, PhD, Chief Scientific Officer of Trovagene. "The specificity of PCM-075 to PLK1 may complement the mechanism of action of other oncology therapeutics when used in combination which could improve and extend the duration of response."

About Trovagene, Inc.

Trovagene is a clinical-stage, oncology therapeutics company. The Company's primary focus is to develop targeted cancer therapeutics for improved patient care and to optimize drug development by leveraging its proprietary technology in tumor genomics. Trovagene has broad intellectual property and proprietary technology to analyze circulating tumor DNA (ctDNA) and clinically actionable biomarkers to identify patients most likely to respond to specific cancer therapies. The Company plans to continue to vertically integrate its tumor genomics technology with targeted cancer therapeutics.  For more information, please visit https://www.trovagene.com.

About PCM-075

PCM-075 is a highly-selective adenosine triphosphate (ATP) competitive inhibitor of the serine/threonine polo-like-kinase 1 (PLK 1) enzyme, which is over-expressed in multiple hematologic and solid tumor cancers. Studies have shown that inhibition of polo-like-kinases can lead to tumor cell death, including a Phase 2 study in Acute Myeloid Leukemia (AML) where response rates up to 31% were observed when used in conjunction with a standard therapy for AML (low-dose cytarabine-LDAC) versus treatment with LDAC alone with a 13.3% response rate. A Phase 1 open-label, dose escalation safety study of PCM-075 has been completed in patients with advanced metastatic solid tumor cancers, and published in Investigational New Drugs. Trovagene has an ongoing Phase 1b/2 clinical trial with PCM-075 in AML that was accepted by the National Library of Medicine (NLM) and is now publicly viewable on www.clinicaltrials.gov. The NCT number assigned by clinicaltrials.gov for this study is NCT03303339. PCM-075 has been granted Orphan Drug Designation by the FDA for the treatment of patients with AML.

PCM-075 only targets PLK1 isoform (not PLK2 or PLK3), is oral, has a 24-hour drug half-life with reversible on-target hematologic toxicities. Trovagene believes that targeting only PLK1 with reversible on-target activity and an improved dose/scheduling protocol can significantly improve on the long-term outcome observed in previous studies with a PLK inhibitor in AML.

PCM-075 has demonstrated synergy in preclinical studies with over 10 chemotherapeutic and target agents used in hematologic and solid tumor cancers, including FLT3 and HDAC inhibitors, taxanes, and cytotoxins. Trovagene believes the combination of its targeted PLK1 inhibitor, PCM-075, with other compounds has the potential for improved clinical efficacy in Acute Myeloid Leukemia (AML), metastatic Castration-Resistant Prostate Cancer (mCRPC), Non-Hodgkin Lymphoma (NHL), Triple Negative Breast Cancer (TNBC) and Adrenocortical Carcinoma (ACC).

Forward-Looking Statements

Certain statements in this press release are forward-looking within the meaning of the Private Securities Litigation Reform Act of 1995. These statements may be identified by the use of words such as "anticipate," "believe," "forecast," "estimated" and "intend" or other similar terms or expressions that concern Trovagene's expectations, strategy, plans or intentions. These forward-looking statements are based on Trovagene's current expectations and actual results could differ materially.  There are a number of factors that could cause actual events to differ materially from those indicated by such forward-looking statements.  These factors include, but are not limited to, our need for additional financing; our ability to continue as a going concern; clinical trials involve a lengthy and expensive process with an uncertain outcome, and results of earlier studies and trials may not be predictive of future trial results; our clinical trials may be suspended or discontinued due to unexpected side effects or other safety risks that could preclude approval of our product candidates; uncertainties of government or third party payer reimbursement; dependence on key personnel; limited experience in marketing and sales; substantial competition; uncertainties of patent protection and litigation; dependence upon third parties; our ability to develop tests, kits and systems and the success of those products; regulatory, financial and business risks related to our international expansion and risks related to failure to obtain FDA clearances or approvals and noncompliance with FDA regulations.  There are no guarantees that any of our technology or products will be utilized or prove to be commercially successful, or that Trovagene's strategy to design its liquid biopsy tests to report on clinically actionable cancer genes will ultimately be successful or result in better reimbursement outcomes.  Additionally, there are no guarantees that future clinical trials will be completed or successful or that any precision medicine therapeutics will receive regulatory approval for any indication or prove to be commercially successful.  Investors should read the risk factors set forth in Trovagene's Form 10-K for the year ended December 31, 2017, and other periodic reports filed with the Securities and Exchange Commission.  While the list of factors presented here is considered representative, no such list should be considered to be a complete statement of all potential risks and uncertainties.  Unlisted factors may present significant additional obstacles to the realization of forward-looking statements.  Forward-looking statements included herein are made as of the date hereof, and Trovagene does not undertake any obligation to update publicly such statements to reflect subsequent events or circumstances.

Trovagene Contact:

Vicki Kelemen
VP, Corporate Communications
858-952-7652
vkelemen@trovagene.com

SOURCE Trovagene, Inc.