Kazia Therapeutics (ASX:KZA) has announced the commencement of a US-based phase 2 clinical trial studying its investigational therapy, GDC-0084, in the deadly brain cancer glioblastoma multiforme.
GDC-0084, which targets the signalling pathway implicated in about 90 percent of cases of the cancer, is being developed as an alternative treatment option for the two-thirds of patients resistant to the mainstay of current pharmacological treatment for glioblastoma, temozolomide.
CEO and executive director Dr James Garner announced recruitment for the study 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 with approximately 12,500 patients in the US and 1,600 in Australia diagnosed every year.
Left untreated, patients live on average for 3-4 months, and even with the best available care, the median survival rate for patients is around 12-15 months.
According to the company, GDC-0084 is distinguished from other brain cancer drugs through its ability to cross the so-called blood-brain barrier, which prevents many drugs from fully affecting the brain.
GDC-0084 was granted Orphan Drug Designation by the US FDA last month, recognising the drug as a potential treatment for a rare disease with high unmet patient need.
Kazia in-licensed GDC-0084 from Genentech after a successful phase 1 safety study 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,” said Dr Garner.
“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.”