Biotechnology company Kazia Therapeutics (ASX: KZA) has reported early data from a phase 2 study into the effects of lead candidate GDC-0084 on newly-diagnosed brain glioblastomas in adults has shown the drug may delay the cancer’s progression.
The study enrolled patients with an unmethylated MGMT promotor, which is a genetic marker associated with near total resistance to temzolomide – the only FDA-approved pharmacological treatment for newly-diagnosed glioblastoma.
Approximately two-thirds of all glioblastoma patients have an unmethylated MGMT promotor.
Data from the first nine patients in the study showed a median progression-free survival rate of 8.4 months when using GDC-0084, compared to 5.3 months on temozolomide.
While Kazia said it was too early to calculate overall survival, 75% of evaluable patients are reported to be still alive at the cut-off date for analysis providing a strong signal that GDC-0084 may provide clinical benefits.
The study also established a maximum tolerated dose of 60mg, which is higher than the 45mg dose determined in an earlier phase 1 study in late-stage patients.
The safety and tolerability of GDC-0084 was broadly consistent with phase 1 endpoints, with hyperglycaemia (raised blood sugar), oral mucositis (mouth ulcers) and rash among the most common drug-related toxicities.
Aggressive brain cancer
GDC-0084 is a small molecule inhibitor of the PI3K / AKT / mTOR pathway, and is being developed to treat glioblastoma multiforme – the most common and most aggressive form of primary brain cancer in adults.
The drug was licensed from Genentech in late-2016 and entered Phase 2 trial stage last year.
The trial comes at a time where there is an acknowledged and urgent need for new therapies in glioblastoma.
Chief executive officer Dr James Garner said GDC-0084 could potentially be an important new addition to the treatment of a challenging disease.
“[Although] this is early ‘first look’ data from the study, representing around a third of the total patients to be enrolled, it has already exceeded our expectations,” he said.
“The fact that the majority of patients in the first stage of the study remain alive more than a year after diagnosis suggests that a meaningful overall survival benefit may emerge as the study matures … that would be a remarkable finding.”
GDC-0084 is also the subject of four other ongoing clinical trials in diffuse intrinsic pontine glioma and brain metastases.
Dr Garner said these trials are expected to report interim data during the early part of 2020.
At mid-afternoon, shares in Kazia were trading 70.45% higher at $0.75.