How Monash University scientists helped a homegrown cancer drug become an FDA-approved global success story
21 September 2023
Professor Susan Charman
It has been announced this week that ‘Momelotinib’ - a drug discovered by Australian researchers for the treatment of bone marrow cancer - has been approved by the US Food and Drug Administration (FDA).
The drug will be used to treat myelofibrosis - a rare and devastating type of cancer that leads to scar tissue in the bone marrow and severe anaemia. Momelotinib is the first and only FDA-approved medicine for both newly diagnosed and previously treated myelofibrosis patients with anaemia that addresses the key manifestations of the disease.
The program that ultimately delivered Momelotinib began in the late 1990s when Professor Andrew Wilks and Dr Christopher Burns launched the Australian biotech, Cytopia. Monash University drug researchers became involved in the project in the early 2000s shortly after Professors William Charman and Susan Charman first established the Centre for Drug Candidate Optimisation (CDCO) which now is one of five research Themes within the Monash Institute of Pharmaceutical Sciences.
The Cytopia program was among the CDCO’s first projects, with Professor Susan Charman and her team collaborating with Professor Wilks and Dr Burns to understand the biopharmaceutical properties of the series.
The CDCO, established through seed funding from the Victorian State Government and Monash University, plays a critical role in the drug discovery pipeline by profiling the physicochemical, metabolic and pharmacokinetic properties of candidate compounds to inform medicinal chemistry design strategies for compound optimisation. These properties are essential to ensure that candidates are suitable for progression into preclinical and clinical development, and ultimately that they are safe and effective when given to patients.
Professor Charman said collaboration and the bringing together of expertise is a key component of the “bench to bedside” drug development process.
“Simply put, the majority of drug discoveries are unsuccessful so getting to the stage of FDA approval, particularly with a treatment for a rare and lethal disease such as myelofibrosis, is an enormous accomplishment,” Professor Charman said.
“Australia is home to world-leading scientists and facilities. But more importantly, it’s the collaborative spirit of our scientific community that is critical in catapulting a drug such as Momelotinib from an early stage discovery to a commercial success story with the potential to significantly improve the lives of patients.
“Congratulations to Andrew, Chris and their team for this incredible achievement.”
Professor Wilks says FDA approval for Momelotinib is not only a personal victory, but a triumph for the many scientists and clinicians with whom he worked across Australia.
“Getting a drug to FDA approval is a huge undertaking involving many steps in a long journey, particularly in the early phase of discovery when you’re working to get a drug off the ground,” Professor Wilks said.
“In the early 2000s, when the CDCO was first established, the timing was right for us to collaborate with Professor Charman and her team to help progress the compound series that ultimately led to Momelotinib; these were the necessary first steps in its voyage to becoming a treatment for patients living with myelofibrosis - a rare and poorly understood disease.
"Our work with the CDCO was an early catalyst propelling the discovery programme towards its stellar clinical success.”
Director of the Monash Institute of Pharmaceutical Sciences, Professor Chris Porter, said FDA-approval of Momelotinib is testament to the perseverance of the team behind it.
“This is a truly outstanding Australian science success story, which has been decades in the making. Thanks to the tenacity of Andrew, Chris, the CDCO team and all of the other scientists and clinicians involved along the way, there is now hope for patients living with myelofibrosis,” Professor Porter said.
Since 2003, the CDCO has worked with collaborators to progress 42 new drug candidates into clinical development across disease indications including cancer, infectious diseases, and central nervous system disorders, among others.
ENDS