Glioblastoma Drug Granted Orphan Drug Designation

Mathew Shanley

This morning, it was announced that Kazia Therapeutics Limited received orphan drug designation for its investigational new drug, GDC-0084, by the United States Food and Drug Administration (FDA).

GDC-0084 is being developed to treat glioblastoma multiforme (GBM), the most common and most aggressive variation of primary brain cancer.

GBM is characterized by headaches, changes in personality, nausea, and stroke-like symptoms. It can be the result of genetic disorders like neurofibromatosis or prior radiation therapy. According to the Mayo Clinic, the disease is very difficult to treat and current standard of care consists of therapies include those that slow the progression of cancer and alleviate signs and symptoms.

The cancer type is more frequently diagnosed during a neurological exam or via imaging tests. Approximately 130,000 patients are diagnosed worldwide annually, and median survival on best available care is typically 12-15 months.

“We are very pleased to have successfully completed this important regulatory step in the development of GDC-0084,” said Dr James Garner, Kazia CEO in a press release. “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."

GDC-0084 is designed to be a small molecule inhibitor of phosphatidylinositol 3-kinase (PI3K) – which is frequently associated with tumorigenesis – with potential antineoplastic activity. By inhibiting the PI3K in the PI3K/AKT kinase signaling pathway, the drug is expected to be able to obstruct cell growth in susceptible tumor cell populations.

The drug was licensed from Genentech in late 2016 after demonstrating favorable results in a Phase 1 study of 47 patients with advanced brain tumors, including many who had GBM specifically. Results from the trial were presented at the American Society of Clinical Oncology (ASCO) Annual Meeting in 2016, and the data exhibited GDC-0084’s acceptable tolerability, as well as its ability to reduce tumor size and activity in some patients via its experimental imaging technology known as FDG-PET.

A multicenter, international Phase 2 study is expected to be initiated sooner than later, and will evaluate the efficacy of GDC-0084. Kazia is aiming to provide definitive clinical proof-of-concept data by the first half of 2019.

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