Aussies are some of the most enthusiastic adopters of new clinical drugs. In 2019, 95,000 of us participated in nearly 2,000 clinical trials where Australia is home to many of the world’s leading scientists. In keeping with that, clinical stage biotech company Pharmaxis (ASX: PXS) has a thriving drug development pipeline set to report clinical trial results in 2023, including a skin scarring trial led by renowned medical researcher Professor Fiona Wood AM, and one aiming to treat incurable bone marrow cancer.
Here’s a summary of each drug being trialled.
PXS-5505 – Myelofibrosis
When there’s too much scar tissue growing in the bone marrow, blood cell production is reduced, causing myelofibrosis, a form of chronic cancer. Affecting 15 in every 1 million Americans, the current standard of care is not enough to help people see it through for the long-term where the average life expectancy once diagnosed is only 5 years. Pharmaxis is attempting to change that.
Its Phase 2 trials for PXS-5505, a drug targeting myelofibrosis are currently underway. Its interim data has proved positive, with five out of six patients showing either stable or improved bone marrow fibrosis scores and five out of six patients showing either stable or improved platelet and/or haemoglobin scores. The full data from the trials is expected around mid 2023.
Dr Gabriela Hobbs MD and Associate Professor of Medicine at Harvard, says, “Though still early in the dose expansion phase of the study, PXS5505 appears to be stabilising and in some cases, improving the hemoglobin and platelet counts, which has also been associated with symptom improvements in those patients that were treated to 24 weeks.”
There is a USD $1 billion market for myelofibrosis treatments at present. It’s also a competitive market. Biotech company Constellation Pharmaceuticals was acquired for USD $1.7 billion after completing Phase 2 trials for their myelofibrosis treatment. However, no product in the market has the disease modifying potential that PXS-5505 does. Others treat symptoms of the disease, PXS-5505 looks to treat the underlying cause.
PXS-6302 – Skin Scarring
In the developed world, 100 million people develop cutaneous scars as a result of trauma following burns, surgeries and other trauma. Pharmaxis’s PXS-6302 has shown the potential to improve scar appearance and elasticity without reducing tissue strength. The trials for the drug are being led by Prof Fiona Wood, a renowned Aussie surgeon and the inventor of spray-on-skin for burn victims. In 2019, the total scar treatment market was valued at about USD $20 billion, with the keloid and hypertrophic scar segment comprising $3.5 billion of it.
PXS-6302 seeks to prevent the formation or improve the appearance and function of scars on the skin. Its phase 1 trials have delivered positive interim results that attracted national attention, with patients demonstrating good tolerability. Already, the latest phase 1c drug trial in patients with established scars has been featured across mainstream media for its potential to significantly improve one’s quality of life.
Complete Phase 1c trial results are expected to be released by June 2023 and are likely to attract global interest in the drug.
PXS-4728 – Parkinson’s Disease
Parkinson’s is incurable, but there are treatments to slow down its onset. PXS-4728 is aspiring to be one such treatment. Boehringer invested over $100 million into the drug PXS-4728 for fatty liver disease, then returned it to Pharmaxis when an unexpected side effect was uncovered. On further investigation it was found that the side effect potentially targeted a disease precursor to Parkinson’s called isolated Rapid Eye Movement Sleep Behaviour Disorder (iRBD). It may not have achieved what pharmaceutical giant Boehringer wanted it to in fatty liver, but PXS-4728 did catch the eye of Parkinson’s UK.
PXS-4728 aims to treat patients with the sleep disorder that can lead to neurodegenerative diseases like Parkinson’s. So impressed by the profile of the drug Parkinson’s UK offered to fund a trial—providing about $5 million. The study will examine whether PXS-4728 can reduce inflammation in the brain of people with iRBD.
The Phase 2 trial is expected to commence in 2023, and will collaborate with experts from the University of Sydney and the University of Oxford.
PXS-5505 – Liver Cancer
Fibrosis is the underlying biological effect that causes myelofibrosis. It also plays a role in other cancers including liver cancer and pancreatic cancer.
Identifying an opportunity to be used alongside chemotherapy, PXS-5505 is being trialled as a treatment for liver cancer by researchers at the University of Rochester in New York.
Liver problems have become increasingly common over the past few years, with such malignancies being the 4th leading cause of cancer-related mortality. It is a $7 billion per annum marketing opportunity, and Pharmaxis is on its way to tapping into it.
Commenting on PXS-5505, Dr Nabeel Badri, one of the researchers, said, “By inhibiting the formation of fibrotic tissue in the tumor we hope to improve delivery and effectiveness of immunotherapy drugs which have so far had a limited impact of the survival of our patients.”
Patients for the trials are being recruited now, with the data expected in June 2024.
With so much happening, it should come as no surprise that institutional investors have been increasing their stake in Pharmaxis, as evidenced by Platinum Asset Management acquiring 5.06% for $1.9 million in October, and then again increasing their stake in November to now own 7.63% of the Company.
Now, all hopes are pegged on the trial results to prove beneficial not just for shareholders but also the patients who could benefit from this ground breaking research.
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