MEDIA RELEASE: Milestone for Australian scientists working collaboratively to fight cancer

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 In an exciting achievement for the Australian biotechnology industry, a potential cancer therapy invented in Australia has entered clinical trials. The drug, if successful, could be used to treat patients with solid tumours such as breast, prostate or lung cancer which affects an estimated five million people worldwide per year.1 

13 December 2020, joint CTxONE and Cancer Therapeutics CRC Press Release MEDIA RELEASE, Melbourne Australia: Australian biotech company, CTxONE Pty Ltd, today announced that an investigational drug from Pfizer Inc. known as PF-07248144 has entered Phase 1 clinical trials for the potential treatment of patients with advanced or metastatic breast, prostate or lung cancer. 

CTxONE, a commercial partner of the Cancer Therapeutics Cooperative Research Centre (CRC), licensed the chromatin-targeting technology to Pfizer in 2018 in a deal worth up to US$475M in development milestones, with additional royalties on sales if the technology is successful. The deal culminated more than a decade of research and development towards the PF-07248144 therapy from a team led by Cancer Therapeutics CRC including researchers from WEHI, Peter MacCallum Cancer Centre, Australia’s national science agency, CSIRO, Monash Institute of Pharmaceutical Sciences, Griffith University, St Vincent’s Institute of Medical Research, and Children’s Cancer Institute. 

PF-07248144 is a first-in-class therapy targeting a MYST histone acetyltransferase called MOZ (KAT6A), which plays an important role in cell growth regulation. The new class of drugs is the first to target MYST MOZ, one of the most commonly amplified genes in solid tumours, and these new drugs are designed to stop MOZ-dependent cancers by switching off the signals that trigger the cancer cells to divide and grow – a novel and exciting mechanism of action. 

The opportunity to discover drugs for KAT6A emerged from research conducted by Professor Anne Voss and Associate Professor Tim Thomas based at WEHI. 

“The KAT6A project was brought into Cancer Therapeutics as a novel target idea, and through collaboration between our experienced drug development researchers and the original investigators, we were able to develop potential therapies that were subsequently licensed to Pfizer. It is exciting to see this project enter clinical trials and take one step closer to becoming a treatment,” said Brendon Monahan, Chief Scientific Officer at Cancer Therapeutics CRC 

“We are delighted to share this clinical development milestone today. Commencing the Phase 1 trial for PF-07248144 is a strong example of how partners contributing to the Cancer Therapeutics CRC have worked collaboratively to transform Australian medical research into potentially world-first treatments for cancer,” said CTxONE Director and Scientific Advisor, Dr Ian Street. 

“CTxONE’s role as a commercialisation partner of this incredible brains trust is to provide access to funding and to support tangible progress for potentially life-saving cancer treatments – and ultimately, to ensure they reach real people. Today represents a significant step forward in meeting that goal.” 

The clinical trial started on 19 November 2020 and will test PF-07248144 for safety and effectiveness in breast, prostate and lung cancer patients with advanced or metastatic tumours. 

Jeff Settleman, Senior Vice President and Head of Pfizer’s Oncology Research & Development Group said: “We are pleased with the progress made on this program to date, and believe that PF-07248144 represents a very promising, differentiated program that has the potential to provide a new treatment option for oncology patients.” 

Breast cancer, lung cancer and prostate cancer are among the top five most common cancers which affect Australians.2 While the survival rates for these cancers have improved in recent years, new and effective therapies are still required for patients who do not respond to standard of care treatments. 

“We are optimistic that PF-07248144 will one day be a strong line of attack against these all too common and devastating diseases. This is a big first step in the clinical development process,” said Dr Street. 

“CTxONE is committed to empowering Australian scientists to not only discover life-changing treatments, but to work collaboratively with commercial partners and incubators like the Cancer Therapeutics CRC, to ensure our world-class medical research can be translated from the lab, to life.” 

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