Special Report: PharmAust has achieved a major milestone after its lead investigational drug, monepantel, was selected for inclusion in the prestigious HEALEY ALS Platform Trial in the US under a Clinical Research Support Agreement with Massachusetts General Hospital.
The milestone signifies a crucial progression for PharmAust (ASX:PAA) in its efforts to gain approval from the US FDA for monepantel (MPL) as a treatment for amyotrophic lateral sclerosis (ALS).
By being selected for inclusion in the HEALEY ALS Platform Trial, MPL’s efficacy and potential therapeutic benefits can now be independently recognised and validated within the ALS research community.
The trial is a groundbreaking Phase 2/3 initiative designed to evaluate multiple ALS treatments. The trial design allows for multiple investigational treatments to be tested simultaneously using a shared master protocol.
This model, successfully utilised in oncology, aims to expedite the study of multiple therapies, allowing investigators to test more potential therapies, increase patient access, reduce costs, and shorten development timelines.
With over 70 trial sites across the United States, the platform aims to enrol between 160 to 240 participants per regimen, offering an optimised 3:1 active drug to placebo ratio. In other words, for every three participants receiving the investigational drug, only one receives a placebo.
This approach allows researchers to more accurately assess the effectiveness and safety of the investigational treatments against a standard baseline, ultimately speeding up the evaluation process.
Next step – trial candidates
The next steps are for the HEALEY ALS Platform Trial design team to work with PharmAust to develop a regimen-specific protocol with information specific to monepantel.
“We are excited to partner with the HEALEY ALS Platform Trial, a prestigious and well-recognised initiative in the field of ALS research, providing invaluable exposure within the ALS research community,” said PharmAust’s CEO, Dr Michael Thurn.
“This partnership marks a significant step forward in our efforts to develop monepantel as a viable treatment for ALS.”
Thurn added that the collaboration with leading ALS experts and the streamlined regulatory support will accelerate the company’s progress towards delivering a much-needed therapy for patients with ALS.
“While we had been exploring the potential to conduct the adaptive Phase 2/3 STRIKE study globally, including at sites in Australia, the opportunity to be part of the HEALEY ALS Platform Trial via their US network of 72 clinical sites is substantial.
“We remain committed to supporting the ALS community in Australia, and ultimately the HEALEY ALS Platform Trial will provide us with the most efficient and safest way to bring monepantel to all patients.
“This collaboration with the HEALEY ALS Platform Trial, with its streamlined regulatory support and engagement with leading ALS experts, is a critical advancement in our mission to offer a viable treatment for ALS,” said Thurn.
Huge market potential
There is no cure for the ultimately fatal ALS disease, which is also known as MND (motor neurone disease) or Lou Gehrig’s disease.
The disease is marked by the gradual breakdown of nerve cells in the spinal cord and brain, impairing voluntary muscle control of the limbs, which can ultimately result in respiratory difficulties and death.
Every 90 minutes someone is diagnosed and dies with MND/ALS, and 90% of cases occur without a family history. Onset is usually between the ages of 40 and 70 years, while life expectancy on average is just over two years.
By 2040 the incidence of MND/ALS is expected to increase by 70%, according to the latest data.
In June, PharmAust reported outstanding progress on monepantel, suggesting the drug could potentially extend life expectancy and improve quality of life for those with MND/ALS.
PAA said that new data provided by Berry Consultants had revealed some significant findings – patients who took MPL had a 91% lower risk of death from MND/ALS compared with ‘untreated matched-controls’.
Read more on that here: PharmAust’s monepantel drug could be a game-changer for MND/ALS patients, new data shows
Significant progress
Meanwhile, PharmAust has made significant progress on its MND/ALS research over the past quarter, highlighted by positive outcomes from its Phase 1 MEND study.
The study demonstrated MPL’s superior safety profile and tolerability compared to the FDA-approved ALS drug, Relyvrio.
Moreover, preliminary efficacy data from the study indicated a significant 58% reduction in disease progression rates among participants receiving the high-dose regimen of MPL.
A pivotal aspect of the findings was the detection of MPL and its active metabolite in the cerebrospinal fluid, demonstrating the compound’s ability to effectively penetrate the blood-brain barrier – a crucial factor in treating neurological conditions such as ALS.
These promising results have enabled PharmAust to identify an optimal dosage for monepantel, laying the groundwork for its upcoming Phase 2/3 clinical trial slated to commence later this year.
In May, PharmAust was granted orphan drug designation (ODD) by the FDA for MPL.
Read more on that here: ‘Outstanding milestone’: FDA grants PharmAust orphan drug designation for monepantel.
This article was developed in collaboration with PharmAust, a Stockhead advertiser at the time of publishing.
This article does not constitute financial product advice. You should consider obtaining independent advice before making any financial decisions.
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