St. Jude Children’s Research Hospital Completes Initial Stage of Phase I Trial Assessing GDC-0084 for Deadly Pediatric Brain Tumor

Sep 16, 2019 | Brain Cancer, Brain Tumors, Diffuse Pontine Glioma (DIPG), Pediatric Cancer

St. Jude Children’s Research Hospital Completes Initial Stage of Phase I Trial Assessing GDC-0084 for Deadly Pediatric Brain Tumor

St. Jude Children’s Research Hospital has completed the first stage of a clinical trial evaluating GDC-0084 for diffuse pontine glioma (DIPG), an aggressive and dangerous brain tumor. The commercial sponsor is Australian-based Kazia Therapeutics Limited (KZIA). The study seeks to assess the proper portion of GDC-0084 for pediatric use in cases of DIPG and other diffuse midline gliomas.

The Study

The study is sponsored by St. Jude Children’s Research Hospital and supported by investigational drug maker Kazia Therapeutics.  The clinical investigation focuses on pediatric high-grade gliomas—highly aggressive and deadly—while treatment options are limited. The purpose of the study has been to examine the safety, tolerability, and pharmacokinetics of GDC-0084 and to estimate its maximum tolerate dose (MTD) when administered to pediatric patients with DIPG or other diffuse midline H3 K27M-mutant gliomas post radiation therapy. The examination started enrollment in November 2018. Thirteen patients have received treatment thus far. The initial segment of the investigation, reported Biopharma Press, was intended to decide a most extreme endured portion (MTD) in the pediatric setting. The examination will presently continue into a development companion, intended to look for starter sigh of viability.

What is GDC-0084?

GDC-0084 is a brain-penetrant inhibitor of a growth-promoting cell signaling pathway that is dysregulated in the majority of the midline glioma tumor cells. The study is also designed to enable a preliminary assessment of the antitumor activity of single-agent GDC-0084, in the hope of enabling rational combination therapy options with systematic and/or radiation therapy (RT) in the pediatric patient population—one that is in desperate need of therapeutic advances.

Principal Investigator Comments

Dr. Christopher Tinkle, co-Lead Investigator noted, “Until this point, thirteen patients have gotten GDC-0084 in our examination. We have decided a most extreme endured portion (MTD) in the pediatric setting of 27 mg/m2, which we hope to be around identical to the dosages at present being investigated in grown up studies. The wellbeing profile of the medication in youngsters seems, by all accounts, to be extensively like that in grown-ups, and we have experienced no startling discoveries in such manner. It is untimely to reach inferences with respect to potential adequacy; however, we currently leave on an extension associate to investigate the clinical impacts of the medication in this patient gathering. My associates and I are satisfied with advancement and anticipate revealing further information from the investigation.”

Who is Kazia Therapeutics?

Kazia Therapeutics Limited, an oncology-focused biotechnology company, develops therapies for a range of oncology indications. Its lead product is GDC-0084, a small molecule inhibitor of the PI3K / AKT / mTOR pathway that is in Phase II clinical trials to treat glioblastoma multiforme. The company is also developing TRX-E-002-1 (Cantrixil), a third-generation benzopyran molecule, which is in Phase I clinical trials to treat ovarian cancer. It has collaboration agreements with The University of York, The Children’s Cancer Institute Australia, The Mater Institute in Queensland, the University of Boston, and St Jude Children’s Research Hospital, as well as Dana-Farber Cancer Institute and Alliance Oncology Foundation. The company was formerly known as Novogen Limited and changed its name to Kazia Therapeutics Limited in November 2017. Kazia Therapeutics Limited was founded in 1994 and is based in Sydney, Australia.


With a share price of $2.65 and a market capitalization of $17 million, they fall under the micro-cap stock category. The stock has been depressed for over a decade.

Focus on GDC-0084 in Australia, UK and Switzerland

Kazia focuses intensely over the last year on investigating GDC-0084 in DIPG. In addition to the Phase I clinical trial with St. Jude reported on herein, they are collaborating with Professor Matt Dun and team at the University of Newcastle. In addition to the UK effort, they are undertaking pre-clinical research with the Children’s Hospital in Zurich, Switzerland under the oversight of Dr. Javad Nazarian, Head of the DIPG Research Institute. In Switzerland, Nazarian will investigate precision medicine approaches to the potential use of GDC-0084 in DPIG.

Lead Research/Investigators

Dr. Christopher Tinkle, co-Lead Investigator

Dr. Amar Gajjar, Chair of the St. Jude Department of Pediatric Medicine (Co-pioneer of the Brain Tumor Program)

Call to Action:  We all have a vested interest in saving as many children as we can from the various forms of brain tumors that currently have no cure. Sign up for the TrialSite Newsletter for updates on this study.


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