Kazia Therapeutics Ltd (ASX: KZA) is expecting multiple value-driving data read-outs from its clinical trials in 2020 and believes these may provide “high potential” for partnering with a big pharma.
The company has six ongoing clinical trials across two of its assets – paxalisib (formerly GDC-0084) in glioblastoma multiforme (GBM), the most common and aggressive form of primary brain cancer in adults, and Cantrixil for ovarian cancer.
Paxalisib is expected to transition to a pivotal study in the second half of 2020.
Describing 2020 as an exciting period for the company and “a crucial inflection point for our programs”, chief executive officer Dr James Garner noted that the partnering market for new oncology drugs "is active and driven by emerging data".
He highlighted the following pending developments:
- Interim data from the Phase I study of paxalisib in diffuse intrinsic pontine glioma (DIPG) (provisional) – third quarter;
- Interim data from phase II study of paxalisib in BCBM (provisional) – third quarter;
- Start of the recruitment of GBM AGILE pivotal study of paxalisib – second half;
- Interim data from phase II study of paxalisib in GBM – fourth quarter; and
- Final data from phase I study of Cantrixil in ovarian cancer – fourth quarter.
The lead program for paxalisib covers the full range of brain cancers. GDC-0084 inhibits the PI3K pathway that is thought to be critical to the development of certain kinds of tumours.
The drug is currently involved in five clinical trials, covering a range of brain cancer including:
- DIPG, a highly aggressive childhood brain tumour; and secondary brain cancer;
- Breast cancer brain metastises (BCBM); and
- Two separate trials on cancer which has spread to the brain from any primary source.
The drug is being developed for the around 65% of newly-diagnosed GBM patients who will not respond to temozolomide, the only US Food and Drug Administration (FDA) approved drug for GBA.
For patients who do not respond to temozolomide, there is no effective pharmacological treatment currently available in the market.
New phase II data compares favourably to historical data for temozolomide, with the study (n=9) showing a median overall survival (OS) of 17.7 months, which compares favourably with temozolomide, with a reported median OS of 12.7 months in this patient population.
Similarly, in the progression-free survival (PFS) study (n=30), paxalisib shows a median PFS of 8.5 months, again comparing favourably to the existing therapy of 5.3 months.
Kazia highlighted that GDC-0084 is unique in its ability to cross the blood-brain barrier and appears generally safe and well-tolerated thus far.
A total of around 133,000 cases of GBM cases are reported per annum worldwide, offering an indicative market opportunity of US$1.5 billion.
Cantrixil or TRE-E-002-1 is a third-general benzopyran that targets slower dividing ‘tumour-initiating cells’ by helping combat the problems of resistance and recurrence that occur with chemotherapy.
The drug is being trialled in Australia and the US under an Investigational New Drug (IND) application which was approved by the US FDA in 2016.
The phase I trial is being carried out in two parts: Part A, the dose-escalation component seeks to understand the safety profile of the drug and to determine the maximum tolerated dose while Part B, the dose-expansion cohort, is aimed at exploring initial signals of efficacy.
Kazia is in a phase I study in ovarian cancer and has fully recruited 13 patients for Part B of the trial.
Ovarian cancer is the seventh most common cancer in women and has the lowest survival rate of any women’s cancer.
About 240,000 new cases are reported each year with the global market for ovarian cancer therapies expected to grow to US$1 billion over the next five years.
Kazia said that the recent institutional placement which saw the company raise A$7.2 million, placed it on a very good footing, leaving it well-funded through the current economic uncertainty.
Further to the placement in April, the company raised A$1.8 million via a share purchase plan in May 2020.