Clinical stage drug development company, Pharmaxis (ASX: PXS) is making a name for themselves in the oncology sector, with their lead asset oncology drug yielding promising results in numerous studies.
PXS-5505, a drug designed for the treatment of rare blood cancer myelofibrosis is built on Pharmaxis’ proprietary platform, harnessing the power of enzyme inhibition to reduce fibrosis of the bone marrow and modify the course of the disease.
The experienced biotech celebrated a milestone last month announcing the progression of PXS-5505 to Phase 2 trials in myelofibrosis patients. Having gained attention from notable researchers in the scientific community, researchers at the University of Rochester in New York decided to initiate a study of the drug in models of liver cancer.
A study led by Dr. Roberto Hernandez-Alejandro yielded promising results in August of this year, demonstrating for the first time that the drug has an effect on cancers other than myelofibrosis. Results presented at the Americas Hepato-Pancreato-Biliary Association conference in Miami communicated that PXS-5505 successfully delayed tumour growth and increased survival rates in preclinical models of the disease.
Now, the drug has gained Investigational New Drug status for the new indication of liver cancer with the FDA clearing the drug for a Phase 2 study to support the addition of PXS-5505 to the current chemotherapy standard of care to treat unresectable hepatocellular carcinomas– the most common type of primary liver cancer.
Currently only 20-30% of hepatocellular carcinomas are resectable by surgery at presentation and many patients rely heavily on chemotherapy to tackle the disease. However, with just a 21% chance of surviving more than 5 years, there is an urgent need for a better treatment course.
Dr. Roberto Hernandez-Alejandro said: “At the University of Rochester Wilmot Cancer Centre, we are excited about the prospect of combining PXS-5505 with standard first line therapy for our unresectable hepatocellular carcinoma patients. The incidence of hepatocellular carcinoma is rising in part due to increasing incidence of cirrhosis and non-alcoholic steatohepatitis. Beyond resection, effective systemic therapies for this disease are lacking, thus new treatment regiments are of significant clinical need.”
Another researcher at the University, Dr. Nabeel Badri also commented: “PXS-5505 is a potent inhibitor of lysyl oxidase, a key enzyme in collagen crosslinking. By inhibiting the formation of fibrotic tissue in the tumour we hope to improve delivery and effectiveness of immunotherapy drugs which have so far had a limited impact on the survival of our patients. Through preclinical testing and translational research, we have developed a promising clinical trial design that has the potential to benefit these patients and improve our understanding of hepatocellular carcinoma.”
The trial design includes a dose escalation phase where the safety profile of the drug will be evaluated alongside current standard of care chemotherapy. The dose will be assessed to determine the impact of PXS-5505 on fibrosis and drug perfusion rates.
CEO of Pharmaxis, Gary Philips is enthusiastic for the future of the partnership with the University, saying: “We highly value our collaboration with the research team at University of Rochester. The rapid progression from the compelling pre-clinical work presented for the first time in August to a successful IND submission is very encouraging and we look forward to concluding arrangements for them to commence the dose escalation study in 2022. Whilst PXS-5505 has recently progressed to a Phase 2 clinical trial looking for evidence of disease modifying effects in bone cancer myelofibrosis as a monotherapy, exploring its potential to address cancers where fibrosis is limiting the clinical benefit of current chemotherapy such as liver and pancreatic cancer would be significantly value adding.”
The nigh on untouchable chemotherapy market is valued at a conservative USD $50 billion. The side effects and shortfalls of chemo are well known, yet they remain one of the most effective cancer treatments we have. Adjunct therapies like PXS-5505 could piggyback on the huge market size, carving out their own market share in a rapidly changing space.
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