Top-line final data from CD-enabled Cantrixil Phase I study confirms prior positive efficacy and safety signals

Kazia Therapeutics Limited, an Australian oncology-focused biotechnology company shared top-line final data from its phase I study of Cantrixil (TRX-E-002-1) in patients with persistent or recurrent
ovarian cancer (NCT02903771).

Key Points
• 25 patients with advanced metastatic ovarian cancer received at least one dose of Cantrixil at six sites in the United States and Australia, comprising 11 patients in Part A (dose escalation) and 14 patients in Part B (dose expansion)
• Trial achieved its primary objective, determining the maximum tolerated dose (MTD) of Cantrixil to be 5 mg/kg
• Overall, 16 patients were evaluable for efficacy. One patient demonstrated a complete response (CR) and two patients experienced a partial response (PR), according to industry-standard RECIST criteria, making an overall response rate (ORR) of 19%
• The patient who experienced a complete response remains in remission some three years after her last dose of Cantrixil
• The drug was generally well-tolerated, with primarily gastrointestinal toxicities observed (abdominal pain, vomiting, and nausea)

In accordance with common practice, the full data will remain embargoed until they are formally published, in order not to prejudice the appropriate dissemination of the data, and only top-line data are discussed here.

Background
Kazia Therapeutics Limited (ASX: KZA, NASDAQ: KZIA) is an innovative oncology-focused biotechnology company, based in Sydney, Australia. Our pipeline includes two clinical-stage drug development candidates, and we are working to develop therapies across a range of oncology indications.
TRX-E-002-1 (Cantrixil) is a third generation benzopyran molecule with activity against cancer stem cells and is being developed to treat ovarian cancer. TRX-E-002-1 has completed a phase I clinical trial in Australia and the United States. Cantrixil was granted orphan designation for ovarian cancer by the US FDA in April 2015.

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.