Health

Kazia Gets Orphan Drug Designation for Investigational Glioblastoma Multiforme Drug

February 23, 2018

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2 min read

Kazia Therapeutics (Australia: KZA; Nasdaq: KZIA) announced today that the FDA has granted Orphan Drug Designation (ODD) to Kazia’s lead investigational new drug, GDC-0084, for the treatment of glioblastoma multiforme (GBM), the most common and most aggressive form of primary brain cancer.

GBM is an area of significant unmet medical need with 133,000 cases diagnosed worldwide each year. Prognosis is extremely poor among those afflicted, with three to four months of typical untreated survival time. Even among patients treated with existing therapies (commonly, temozolomide after surgery and radiotherapy), median survival of only 12 to 15 months is expected with best available care. Further, existing drug treatments are largely ineffective in almost two-thirds of patients.

Kazia licensed GDC-0084 from Genentech, part of Roche Holding (Switzerland: ROG; OTCQX: RHHBY) in late 2016, after the drug demonstrated favorable results in a phase I study of 47 patients with advanced brain cancer. Kazia intends to shortly commence an international phase II clinical study to provide definitive evidence of clinical efficacy. This phase II study will initially be conducted predominantly at leading centers in the US, with initial data available in early 2019.

In addition to GDC-0084, Kazia is in the clinic with its investigational ovarian cancer therapy, Cantrixil.

Source: Kazia Therapeutics

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.

– Dr James Garner, CEO, Kazia Therapeutics

The FDA accords ODD status to drugs considered to be promising potential treatments for rare diseases, generally defined as those which affect less than 200,000 cases per year in the US. ODD can provide drug developers with up to seven years of Orphan Drug Exclusivity (ODE), extending the effective life of a commercial product. It also provides opportunities for grant funding, protocol assistance and financial benefits, such as a waiver of New Drug Application fees, and tax credits.

Kazia’s common stock trades on the Australian Stock Exchange with ADRs on Nasdaq. Market cap at today’s midday price is about US$25 million, which seems to present a fair risk-reward proposition given the potential of GDC-0084.