PharmAust’s monepantel selected for prestigious HEALEY ALS platform trial in the US

PharmAust’s (ASX: PAA) lead amyotrophic lateral sclerosis (ALS)/motor neurone disease (MND) treatment candidate monepantel (MPL) has been selected for inclusion in the prestigious HEALEY ALS Phase 2/3 platform trial.

The HEALEY trial is being conducted in the United States under a clinical research support agreement with Massachusetts General Hospital that allows multiple investigational treatments to be tested simultaneously using a shared master protocol.

The trial model aims to expedite the study of multiple therapies, allowing investigators to test more potential therapies, increase patient access, reduce costs and shorten development timelines.

Independent validation

Managing Director Dr Michael Thurn said the selection is another milestone as PharmAust progresses MPL towards seeking US Food and Drug Administration approval for the treatment and provides independent validation of its potential as an ALS treatment.

“This partnership marks a significant step forward in our efforts to develop MPL as a viable treatment for ALS,” Dr Thurn said.

“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.”

The trial design team will work with PharmAust to develop a regimen-specific protocol with information specific to MPL.

Study biostatisticians and PharmAust will adapt the regimen-specific statistical analysis plans as necessary to incorporate considerations specific to the MPL study regimen.

Streamlined pathway

Dr Thurn said the trial will provide the company 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.”

More than 70 trial sites are utilised across the US with the platform aiming to enrol 160-240 participants per regimen, offering an optimised 3:1 active drug to placebo ratio.

Drug candidates that enter the platform trial are chosen by a group of expert ALS scientists and members of the Healey & AMG Centre science advisory committee.