Glioblastoma is one of the most debilitating and deadly cancers, with limited treatment options that are currently effective for only a third of patients, says Kazia Therapeutics (ASX: KZA) CEO Dr James Garner.
“The entire team has been working hard to design and implement the GDC-0084 clinical study. We are very pleased to now have the trial underway. The need for new therapies in this disease remains immense, and we believe that GDC-0084 may have a valuable role to play in improving outcomes for patients with glioblastoma,” says Dr Garner.
The move to conduct a phase 2 trial is part of what Kazia Chairman Iain Ross calls, “a significant transformation of the organization, to optimally support this clinical-stage portfolio, and we are now a lean, cost-effective, and highly-focused biotech.”
Given the absence of meaningful drug development over the past 20 years or the existence of currently viable treatments, there is a high demand for new treatment options and Kazia hopes to provide a viable solution in the form of its GDC-0084 drug candidate that’s just been put forward for an international Phase 2 clinical trial with the Food and Drug Administration (FDA) in the US.
Commencement of the clinical trial follows the decision of the US FDA to grant orphan drug designation to GDC-0084 in glioblastoma last month.
Kazia is keen to achieve product registration as soon as possible and hopes to set up its phase 2 trial effectively so that phase 3 can be avoided altogether, as some drug candidates are able to do under current FDA rules.
To date, Kazia has been active to obtain extensive regulatory consultation, has re-manufactured over 40kg of drug product into capsules to be used in the trial, optimised its intellectual property portfolio and factored in supplementary animal studies to support the development of GDC-0084.
GDC-0084 is being developed for glioblastoma multiforme (GBM), the most common and most aggressive form of primary brain cancer.
According to Dr Garner, the mainstay of current pharmacological treatment, temozolomide, is effective only in about one-third of patients, and median survival is approximately 12 to 15 months from diagnosis.
In recent years, glioblastoma has been highlighted by several high-profile cases, all hailing from the US political scene.
US Senator John McCain, Beau Biden, son of former US Vice President Joe Biden, and Ted Kennedy, brother to John F Kennedy and a serving Massachusetts Senator for over 40 years, have all been diagnosed with glioblastomas.
According to Kazia, its phase 2 study will initially be conducted at leading US-based centres, in collaboration with specialist clinicians in the neuro-oncology field, and under an Investigational New Drug (IND) filing with the FDA.
Currently, the study is awaiting registration with ‘clinicaltrials.gov’ and will commence screening patients after the Easter holiday period.
According to Kazia, the company anticipates that the study will provide an initial data read-out in early calendar 2019.