Novel Australian drug GDC-0084 for glioblastoma brain cancer starts Phase II clinical trial in US



  • Newly-diagnosed patients resistant to existing standard treatment (temozolomide) targeted for recruitment
  • Adaptive study design will see initial data read out on optimal dose in early 2019, followed by definitive evidence of clinical efficacy
  • Recruitment has started at the Stephenson Cancer Center at the University of Oklahoma; Australian sites expected to open latter half of study

A phase II clinical trial of a drug that targets the signaling pathway implicated in about 90% of cases of the deadly brain cancer glioblastoma multiforme has started in the US.

The drug, GDC-0084, is being developed by the Australian biotech Kazia Therapeutics Limited (ASX: KZA; NASDAQ: KZIA) as an alternative treatment option for the two thirds of patients resistant to the mainstay of current pharmacological treatment for glioblastoma, temozolomide.1

Kazia Therapeutics CEO and Executive Director Dr James Garner announced today that recruitment for the international phase II study of GDC-0084 had opened at the Stephenson Cancer Center at the University of Oklahoma. More sites in the US will open for recruitment in coming weeks.

Glioblastoma is the most common and aggressive form of primary brain cancer.

About 12,500 patients in the US2 and 1600 patients in Australia3 are diagnosed with glioblastoma each year.

Left untreated, patients will only live on average for 3-4 months4, and even with the best available care, the median survival rate for patients is around 12-15 months.5

GDC-0084 is being developed as an inhibitor of the PI3K signaling pathway, which is implicated in most cases of glioblastoma.

The drug is distinguished from other brain cancer drugs being developed by its ability to cross the so called blood-brain barrier, which prevents many drugs from fully affecting the brain.

Last month, GDC-0084 was granted Orphan Drug Designation by the US Food and Drug Administration, recognising the drug as a potential treatment for a rare disease with high unmet patient need.

Kazia in-licenced GDC-0084 from drug development giant Genentech after a successful Phase I safety study also showed signs of efficacy.

“The need for new therapies for glioblastoma remains immense - clinical trials of a variety of potential new therapies are ongoing, but we haven’t yet seen a very convincing new treatment option emerge,” Dr Garner said.

“One reason is that many of the drugs being tried are really intended for use in other tumour types, and are only tested in glioblastoma via small and speculative clinical trials.

“GDC-0084 was designed specifically as a treatment for brain cancer, so it has been very carefully optimised for this disease area. The work on the drug to date has been of an exceptionally high standard.

“We are excited to now have the trial underway, and look forward to working with the participating clinicians.”


Read the ASX announcement

Watch: Why GDC-0084 for glioblastoma: Q&A with Kazia CEO Dr James Garner


Watch: GDC-0084 and glioblastoma


1 ME Hegi, A-C Diserens, T Gorlia, et al. (2005). N Engl J Med 352:997-1003’

2 American Brain Tumour Association,, accessed 28 March 2018

3 Brain Foundation, accessed 14.02.2018

4  Schapira, Anthony H.V. (2007). Neurology and clinical neuroscience. Philadelphia: Mosby Elsevier. p. 1336

5  Gallego, O (August 2015). "Nonsurgical treatment of recurrent glioblastoma". Current oncology (Toronto, Ont.). 22 (4): e273–81


About Kazia Therapeutics Limited

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.