Cantrixil (TRX-E-002-1)

Cantrixil is currently being developed for treatment of ovarian cancer and is being trialled in hospitals across Australia and the United States under an Investigational New Drug application which was approved by the US FDA in 2016.

  • Market Need

    Ovarian cancer is the seventh most common cancer in women and has the lowest survival rate of any women's cancer.  It affects about one in 100 women worldwide, with about 240,000 new cases each year.
    The Global market for ovarian cancer therapies is around US$ 500M, projected to grow to US$ 1B over next 5 years (Decision Resources).

    The survival rate for this disease is poor because of the high rate of relapse after standard-of-care treatment and the late stage at which the disease tends to be diagnosed. When ovarian cancer relapses the disease is often not responsive to standard chemotherapy agents.

    Cantrixil is thought to target chemotherapy-resistant tumor-initiating cells that are thought to be responsible for disease relapse.

    • Most women are diagnosed with late-stage disease (stage III & IV), for which the prognosis remains poor
    • The front-line platinum and taxane-based treatment of this disease has not changed significantly in two decades and there is a high incidence of recurrent drug-resistant disease after chemotherapy
    • Targeted therapies for recurrent ovarian cancer have yielded mixed success due to the heterogeneous nature of the disease.
    The Science Behind It


    Cantrixil consists of the active molecule, a third-generation benzopyran named TRXE-002-01, encapsulated in a cyclodextrin.

    Cantrixil is a first-in-class development candidate which targets the entire spectrum of cancer cells, including tumour-initiating cells cells thought to cause cancer recurrence.

    The novelty of this agent is its ability to kill the cancer stem cell / tumor-initiating cells and their daughter cancel cells that are responsible for cancers originating, metastasizing and relapsing. These slower-growing tumour-initiating cells are often resistant to other types of chemotherapies. Cantrixil has the potential to revive the use of intra-peritoneal (IP) chemotherapy and invigorate the use of this mode of administration for ovarian cancer patients. It has the opportunity to become a standard front-line agent, complementing the use of platinum therapy for a group of gynecological cancer. Cantrixil is currently in phase I clinical trials at six trial sites across Australia and the United States.


    Timing and Market Implications

    Cantrixil has been successfully taken into an international phase I clinical trial under a US IND, only three years after discovery. The phase I trial is being conducted in two parts; a dose escalation component (Part A), which seeks to understand the safety profile of the drug and to determine the maximum tolerated dose, and a dose expansion cohort (Part B), which seeks to explore initial signals of efficacy.

    Preliminary data from Part A has been released; in general the drug has encountered few dose-limiting toxicities. Part A concluded in October 2018 and Part B commenced at that time. More information about the trial and recruitment can be found at:


    Mode of administration

    Intra-peritoneal (IP) administration of first-line chemotherapy to women with advanced stage ovarian cancer immediately after their debulking surgery has been recommended by the National Institute of Health in the USA since 2006. This mode of administration exposes residual tumor cells after surgery to higher doses of chemotherapy for longer periods compared with chemotherapy administered intravenously. Improvements in survival time with IP chemotherapy have lead national health and consumer advisory groups to continue to recommend this mode of administration for eligible groups of ovarian cancer patients.

  • News


    More News

    Publications and Presentations


    More Publications and Presentations




    • Recruitment to the Phase I trial was completed during August.  Part A of the study collected data from 14 patients, and Part B has now fully recruited 13 patients. 
    • In April Kazia presented Cantrixil Phase I data at AACR



    • Part A of the Phase I trial was completed in October
    • Preliminary data was released, and on the basis of 10 enrolled patients, few dose-limiting toxicities were encountered.  Of those patients, 3 of 5 evaluable for efficacy have shown stable disease, while 1 exhibited a partial response.



    • On 1st August 2017, we announced that the Cantrixil study had successfully progressed through a number of dose levels and participating patients were being carefully monitored for safety
    • More drug substance was being manufactured to support the trial
    • Five hospitals were reported as participating in the study, and all sites were open to recruitment, after approval by their respective Human Research Ethics Committees
    • Two new patents were granted to further extend Cantrixil’s intellectual property library


    Second Half of 2016

    • Trial officially opened in December 2016
    • Successful completion of safety studies and human research ethics committee approval.
    • Complete Investigator’s brochure.
    • Complete clinical trial protocol.
    • Submit proposal to Human Research Ethics Committee.
    • Assessment of toxicity studies by independent external toxicologists.



    November 2015 - May 2016

    Formulation and stability testing of GMP product for Phase 1 clinical trial.


    January - December 2015

    Formal toxicity studies to assess safety of Cantrixil.


    August - November 2015

    Manufacture of cGMP grade Cantrixil drug substance for Phase 1 clinical trial – completed.


    January -August 2015

    Scale-up of manufacture of GLP-grade drug substance and drug product for toxicity studies – completed.


    November 2014

    Cantrixil proves highly effective in preclinical tests of refractory ovarian cancer.


    June 2014

    Researchers at Yale University confrm potency in ovarian stem cell model.