Australian oncology company, Kazia Therapeutics, has uncovered an exciting new breakthrough for its drug ‘paxalisib’, that could offer new hope for thousands of Australian melanoma patients.

Kazia scientists, in collaboration with the Huntsman Cancer Institute at the University of Utah in Salt Lake City, USA, have released promising new data for the drug in the treatment of metastatic melanoma, the most aggressive and deadliest form of skin cancer.

 Until now, the drug has mostly been known for its potential to treat brain cancer.

Melanoma affects more than 17,000 Australians every year, and the country has the highest melanoma rates in the world. Most outcomes for patients often involve aggressive and disfiguring surgery to remove the cancer.

Kazia CEO Dr James Garner hopes paxalisib can one day make treatment more effective and less invasive, and is excited by the surprise research discovery that demonstrates the drug’s versality across a range of cancer types.

“Although brain cancer, in various forms, has been the primary focus of paxalisib’s clinical development thus far, we have always recognised that the drug has significant potential to cancers that metastasize to the brain, and indeed cancers outside the central nervous system,” Dr Garner said.

“This very promising data from one of the leading US melanoma research centers points towards an important new opportunity for paxalisib. We now plan to step up our research in this area with the aim of moving towards human clinical trials.”

Around 50% of all melanoma cases have a genetic mutation known as BRAF. These patients are often treated with a combination of two drugs - MEK inhibitor and BRAF inhibitors. However, for most patients the cancer eventually progresses due to the body building up resistance to these therapies by activating the PI3K / Akt / mTOR pathway. Kazia’s drug paxalisib works by inhibiting this pathway, and therefore potentially provides an additional therapy that will improve survival rates.

 The research showed paxalisib used alone resulted in substantial success against the cancer.  However, even greater outcomes were found when used in combination with MEK and BRAF inhibitors.

Lead investigator for the study, Professor Sheri Holmen said this is among the most promising single data finding the team has come across in their research.

The study is being conducted in the lab with the early findings from in vitro and in vivo preclinical models of metastatic melanoma.

“Despite the widespread adoption of immunotherapy in recent years, there remains substantial unmet need in melanoma, particularly in those patients who develop brain metastases,” Professor Holmen said.

“We look forward to exploring the potential of paxalisib further in our research, and hopefully seeing the drug transition to a clinical trial in the near future.”

The Kazia/University of Utah team presented their research at the 19th International Congress of the Society for Melanoma Research, held in Edinburgh, Scotland.

Melanoma in Australia

• Australia has the highest melanoma rates in the world.
• It is expected that 17,700 Australians will be diagnosed with melanoma in 2022.
• 1 Australian is diagnosed with melanoma every 30 minutes.
• It is estimated 1,281 Australians will die from melanoma this year.
• Melanoma is the most common cancer for Australians aged 20 – 39.
• Melanoma is the second most common cancer for men, after prostate cancer.
• Melanoma is the third most common cancer for women after breast and colorectal cancer.

    Source: Melanoma Institute Australia and Cancer Australia