GDC-0084 is a novel targeted therapy that modulates the PI3K pathway. Kazia has licensed GDC-0084 from Genentech and is developing it as a potential treatment for glioblastoma. Investigator led clinical studies are also underway for patients with DIPG and breast cancer which has metastasised to the brain.

  • Market Need

    Glioblastoma multiforme (GBM) is the most common and most aggressive form of brain cancer. Despite all efforts, there have been few significant advances in treatment over the last decade, and the prognosis remains poor. The five-year survival rate is around 3%, compared with approximately 89% for patients with breast cancer.

    It is estimated that approximately 2 out of every 100,000 people will develop GBM each year, and there are approximately 12,500 new cases per annum in the United States. The disease commonly begins with innocuous symptoms such as headache and nausea, but progresses rapidly if untreated.

    Newly-diagnosed patients typically undergo surgery to remove as much of the tumour as possible, and are then treated with radiotherapy and a drug named temozolomide in order to delay recurrence. However, the vast majority of patients soon experience disease progression and survival, even in optimally-treated patients, averages approximately fifteen months.

    The Science Behind It

    The phosphoinositide-3-kinase (PI3K) signalling pathway is one of the central control mechanisms for cells in the human body. It is disordered in many types of cancer, and for some years researchers have experimented with drugs that inhibit this pathway as an approach to treating cancer. At present, one drug, idelalisib, is approved for use in certain kinds of leukaemia and lymphoma.

    The PI3K pathway is disordered in many types of cancer, and has been well validated in clinical trials as a target for new treatments.

    The PI3K pathway appears to be disordered in more than 85% of cases of GBM, and so this appears to be a high-potential target for new GBM therapies.

    GDC-0084 is a potent inhibitor of the PI3K pathway, and has been shown to have an anti-tumour effect in animal models of GBM. GDC-0084 is distinguished by its ability to cross the so-called blood-brain barrier, which prevents many drugs from fully affecting brain tissue.

    In a phase I clinical trial conducted by Genentech, GDC-0084 was shown to have acceptable tolerability in a group of patients with advanced brain tumours, including a majority of patients with GBM. GDC-0084 also showed an ability to reduce tumour size in some patients, and demonstrated a reduction in the activity of some tumours using an experimental imaging technology known as FDG-PET. These data were presented at the ASCO Annual Meeting in June 2016.



    Timing and Market Implications

    Kazia Therapeutics commenced a clinical study of GDC-0084 in GBM in early 2018. This study will aim to provide definitive clinical proof-of-concept data for GDC-0084.

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