PLK1 Cancer, Treatment Acute Myeloid Leukemia | Trovagene

Integrating a Predictive Clinical Biomarker Approach to Develop Oncology Therapeutics that Target Cell Division

Identifying sub-populations of patients most likely to respond to treatment

Our Focus – Precision Cancer MedicineTM (PCM)

We are a clinical-stage oncology therapeutics company, taking a PCMTM approach to develop drugs that target cell division (mitosis) for the treatment of leukemias, lymphomas and solid tumor cancers.

Identifying Patients Most Likely to Respond

Onvansertib is a first-in-class, 3rd generation Polo-like Kinase 1 (PLK1) Inhibitor. PLK1 is essential for precisely regulating the cell division and maintaining genome stability in mitosis (cell division), spindle assembly, and DNA damage response. Studies have shown that PLK1 is highly expressed in most cancers, and its over-expression is associated with poor prognosis in patients.

Data has shown that blocking the expression of PLK1 by kinase inhibitors can effectively inhibit the proliferation of and induce apoptosis (death) of tumor cells. Development of onvansertib, as part of a combination regimen with already approved drugs, has the potential to bring new treatment options to patients across a wide array of cancers.

We believe that integrating a predictive clinical biomarker approach into our onvansertib clinical development program may enable us to tailor treatment to specific sub-populations of patients who are most likely to respond and have a positive clinical impact. PLK1 uniquely phosphorylates translational control tumor protein (TCTP) to form pTCTP and inhibition of this enzymatic activity by onvansertib appears to be predictive of patient response to treatment.

Trovagene has filed a U.S. patent application with the United States Patent and Trademark Office (USPTO) to protect its method for evaluating responsiveness of a cancer to a Polo-like Kinase 1 (PLK1) inhibitor by determining its ability to inhibit phosphorylation of a unique target of PLK1 in cells of the cancer.

Developing onvansertib as Part of a Combination Regimen

Combination therapy is at the center of Precision Cancer MedicineTM and onvansertib has demonstrated synergy with chemotherapies and targeted therapeutics to potentially enhance the efficacy of, and increase the duration of response to, approved drugs in indications where this is a significant need for new treatment options.

Onvansertib is a first-in-class, 3rd generation Polo-like Kinase 1 (PLK1) inhibitor with best-in-class attributes. Onvansertib was developed to have high selectivity for PLK1, only, which is over-expressed in most types of cancer. It has ideal pharmacokinetics – oral administration and a half-life of ~24 hours. Onvansertib has demonstrated synergy in combination with chemotherapies and targeted therapeutics, including Zytiga® (abiraterone acetate)/prednisone, Beleodaq® (belinostat), Quizartinib (AC220), a development stage FLT3 inhibitor, and Velcade® (bortezomib), in in-vitro and in-vivo preclinical models.


Synergistic in Combination with SOC Therapies




Non-Small Cell Lung



Leukemias (Acute
Myeloid Leukemia)




Ovarian Bladder


Non-Small Cell Lung



Multiple Myeloma



Acute Myeloid


T-Cell Lymphoma






Acute Myeoid Leukemia
Chronic Lymphocytic Leukemia



Non-Small Cell Lung



Currently, we have three active clinical trials:

Phase 1b/2 open-label trial of onvansertib in combination with either low-dose cytarabine (LDAC) or decitabine for the treatment of Acute Myeloid Leukemia (AML) – (NCT03303339);

Phase 2 open-label trial of onvansertib in combination with Zytiga® (abiraterone acetate)/prednisone for the treatment of metastatic Castration-Resistant Prostate Cancer (mCRPC) – (NCT03414034);

Phase 1b/2 open-label trial of onvansertib in combination with FOLFIRI + Avastin® for second-line treatment of metastatic Colorectal Cancer (mCRC) with a KRAS mutation – (NCT03829410).

A phase 1 trial in solid tumor cancers established the safety and tolerability of onvansertib and identified the recommended Phase 2 dose of 24 mg/m2 (Phase 1 Study).