First patients dosed in Phase 2 clinical trials to tackle Parkinson’s disease

Parkinson’s UK, in collaboration with Australian researchers, has taken a major step towards preventing the development of Parkinson’s disease with the first Australian patient in a Phase 2 clinical trial having been dosed with PXS-4708, a novel drug aimed at tackling  Parkinson’s before its onset, along with other neurodegenerative disorders.

The multi-national trial was initiated in Sydney and is primarily funded by the Parkinson’s Virtual Biotech fund, with the drug being supplied by Syntara (soon to be renamed from Pharmaxis, ASX: PXS). 

Recruitment for the trial focused on patients with isolated Rapid Eye Movement Sleep Behavior Disorder (iRBD), a condition known to be a strong precursor to Parkinson’s. 

It is hypothesised by Parkinson’s UK and Syntara, based on encouraging preclinical and Phase 1 clinical data, that PXS-4728 can significantly slow the onset of Parkinson’s by targeting inflammation in the brains of iRBD patients. This effect would serve as a neuroprotective strategy to prevent the progression of these conditions.

iRBD is characterised by individuals, who are otherwise healthy, acting out their dreams during sleep with historical data research indicating that iRBD is a significant predictor of Parkinson’s disease. 

Lead investigator of the trial, Professor Simon Lewis, Director of the Parkinson’s Disease Research Clinic at the Brain & Mind Centre, is particularly drawn to the groundbreaking potential of PXS-4728. 

“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 as a neuroprotective agent.”

The first patient dosed in the Phase 2 trial is part of a collaborative design study where experts from the University of Sydney and the University of Oxford are aiming to enrol 40 patients with iRBD for a 3-month study. These patients will undergo state-of-the-art nuclear scanning techniques to assess whether PXS-4728 can effectively reduce neuroinflammation. 

PXS-4728 was originally discovered by Syntara which is supplying the drug for these trials, a decision Syntara CEO, Gary Phillips says will create new hope for those predisposed to the life-altering disease. 

“iRBD patients have very few treatment options available, so this study provides hope for an effective treatment with the potential to move towards the longer-term goal of stopping neurodegeneration,” said Phillips. 

Syntara has strategically structured its funding for the Phase 2 clinical trial through an agreement with the Parkinson’s Virtual Biotech. Up to $5.5 million will be provided to Syntara to support the Phase 2 trial, with advance payments received as the trial progresses. 

The total cost of the trial is expected to be approximately $5.8 million with Parkinson’s Virtual Biotech planning to recoup its investment through future royalties on revenue generated from the commercialization of PXS-4728. 

Recruitment in a UK centre is set to begin later this year, with the trial expected to continue throughout 2024 and results anticipated in the first half of 2025. 

This collaborative effort between Syntara, leading research institutions, and Parkinson’s UK represents a crucial step in the quest to combat Parkinson’s disease and is part of Syntara’s clinical-stage drug development portfolio.