This morning’s announcement may have surprised many investors following the Pharmaxis (ASX: PXS) story because where on earth does Parkinson’s fit into the equation alongside cancer, skin scarring and cystic fibrosis?
Unpacking the Pharmaxis investment case reveals an agile company with more than one trick up their sleeves, keeping business buoyant whilst other biotechs list against the backdrop of the plummeting NASDAQ.
Specialists in fibrosis and inflammation, Pharmaxis’ pipeline addresses these key disease mechanisms underpinning numerous conditions. It’s this underlying inflammation and fibrosis-abating technology that has seen the Company delve into a broad range of conditions, with compounds undergoing clinical trials for myelofibrosis, skin scarring and liver cancer.
Today’s announcement is also a good example of the Company’s ability to access non-dilutive funding to develop its pipeline and follows the recent receipt of $7 million from Aptar.
Pharmaxis isn’t a drug developer for cancer, skin scarring or respiratory conditions, they’re a biotech optimising their expertise in the deep biochemical processes behind disease, working towards the sorts of breakthroughs that define modern medicine.
Looking at it this way, adding another condition to the mix isn’t surprising, but in true Pharmaxis fashion, they’re surprising us yet again by repurposing a drug and capitalising on an accidental discovery.
The PXS-4728 backstory
Sold to Boehringer-Ingelheim in 2015, PXS-4728 was studied extensively for the treatment of liver-related disease Nonalcoholic Steatohepatitis (NASH), and for diabetic retinopathy. The drug was returned to Pharmaxis in 2021 as it wasn’t suitable for the aforementioned indications.
Whilst investors may have thought PXS-4728 was in a holding pattern, when reviewing the substantial preclinical, safety and regulatory work already completed, Pharmaxis identified new potential, this time for neuro inflammatory conditions – an indication that wasn’t being sought by Boehringer.
Potential in Parkinson’s
Neuro inflammation is a key driver of Parkinson’s disease (PD), a condition that affects 2 in every 1000 people. According to the World Health Organisation, the prevalence of the disease has doubled in the past 25 years.
A degenerative condition of the brain with no known cause or cure, a Parkinson’s diagnosis is bleak, with up to 80% of sufferers going on to develop dementia. The average time from onset of movement problems to the development of dementia is approximately 10 years.
With the endorsement and support of leading charity, Parkinson’s UK, Pharmaxis are set to explore the potential neuroprotective characteristics of PXS-4728 in the context of isolated Rapid Eye Movement Sleep Behaviour Disorder (iRBD), which is evidenced to be a strong predictor for the development of Parkinson’s disease.
Thanks to Boehringer’s previous investment of more than $100 million to ensure the safety of the drug, Pharmaxis is able to initiate a new Phase 2 study looking into iRBD in collaboration with some of the best neurologists in the business without incurring those significant development expenses.
Professor Simon Lewis, the Director of the Parkinson’s Disease Research Clinic at the Brain & Mind Centre, University of Sydney will take the helm as the Australian lead investigator working with scientists at the University of Oxford to conduct the study.
“Currently, we have no disease modifying treatments for Parkinson’s disease and by the time patients are diagnosed they have already lost a significant number of brain cells,” said Professor Lewis.
“Therefore, targeting patients with iRBD offers us our best strategy for slowing cell death when it could be most impactful. This trial provides an unprecedented opportunity to study the effect of PXS-4728 and its potential role to act as a neuroprotective agent by reducing neuroinflammation in regions of the brain associated with progression to disease.”
40 patients with iRBD will undergo treatment with PXS-4728 to assess its ability to reduce neuroinflammation, the outcome of which will be measured via nuclear scanning.
The ultimate vote of confidence comes from Parkinson’s UK who are putting their hard won raised funds forward to drive the study. The charity will provide £2.9m (~A$5m) in the hope that Pharmaxis will make meaningful inroads towards a cure for Parkinson’s, something no other company has done yet.
The funding comes from the charity’s Parkinson’s Virtual Biotech program, which is the drug discovery arm of Parkinson’s UK who dubbed the study “ground-breaking”, due to the drug’s potential to slow the onset of Parkinson’s, putting medicine a step closer to a tangible cure.
Should the drug make it to commercialisation, Parkinson’s UK will receive a return of up to four times their initial investment from royalties on any future revenue.
Recruitment of trial participants is expected to begin next year. PXS-4728 has also been highlighted as an “ideal candidate” for other neurodegenerative diseases like Alzheimer’s and Huntington’s.
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