Kazia Therapeutics Limited, an oncology-focused biotechnology company, announced on Friday that it has received the FDA’s Orphan Drug Designation for its investigational drug, GDC-0084, for the treatment of glioblastoma multiforme, the most aggressive form of primary brain cancer.
Sydney, Australia—based Kazia Therapeutics Limited, an oncology-focused biotechnology company, announced on Friday that it has received the FDA’s Orphan Drug Designation (ODD) for its investigational drug, GDC-0084, for the treatment of glioblastoma multiforme, the most aggressive form of primary brain cancer. The ODD grants a special status to the drug and confers on Kazia privileges including tax credits for qualified clinical testing and a waived FDA prescription drug user fee.
Kazia’s CEO, James Garner, MA, MBA, MBBS, said in a statement, “We are very pleased to have successfully completed this important regulatory step in the development of GDC-0084. We share FDA’s recognition of the need for new treatments in this very challenging disease, and we believe that GDC-0084 has great promise as a potential new therapy. We anticipate an imminent start of the phase II clinical study, and look forward to working closely with the participating clinicians.”
Kazia says that it expects to undertake the phase 2 clinical trial beginning in March or April of 2018. Initial data are expected in early 2019.
Glioblastoma multiforme, which is characterized by grade 4 astrocytomas, has a 5-year survival rate of just 3%. Current therapies include surgery to remove as much of the tumor as possible, followed by radiotherapy and treatment with temozolomide. Even among optimally treated patients, survival averages 15 months.
GDC-0084, which Kazia licensed from Genentech in 2016, is an inhibitor of the phosphoinositide-3-kinase pathway, which Kazia says appears to be disordered in the majority of cases of glioblastoma multiforme. A phase 1 study1 of the drug in 47 patients suggested that the drug crosses the blood—brain barrier, with a uniform distribution throughout the brain. Of the evaluable patients, 26 (55.3%) had a best overall response of progressive disease and 19 (40.4%) had stable disease.
In addition to GDC-0084, Kazia is also in development with TRX-E-002-1, a third-generation benzopyran molecule with activity against cancer stem cells. This drug is being developed to treat ovarian cancer and is in a phase 1 clinical trial in Australia and the United States.
1. Wen PY, Cloughesy TF, Olivero A, et al. A first-in-human phase 1 study to evaluate the brain-penetrant PI3K/mTOR inhibitor GDC-0084 in patients with progressive or recurrent high-grade glioma. Presented the 2016 ASCO Annual Meeting, June 4, 2016; Alexandria, Virginia. https://meetinglibrary.asco.org/record/125239/abstract.