PharmAust’s transformative quarter sees monepantel treatment gain ground on MND/ALS

The three months to end June marked a transformative quarter in PharmAust’s (ASX: PAA) focus on monepantel (MPL) for the treatment of neurodegenerative diseases.

The company achieved numerous milestones in the development of MPL for motor neurone disease (MND) / amyotrophic lateral sclerosis (ALS).

These included the granting of final ethics committee approval for an open-label extension study, the reporting of positive survival data from a Phase 1 MEND study on MND/ALS patients who continue to receive treatment and the award of orphan drug designation by the US Food and Drug Administration (FDA).

Positive industry attention

In June, US-based Berry Consultants presented Phase 1 MEND study data to an annual meeting of the European Network to Cure ALS in Stockholm, highlighting survival benefits and reduced disease progression observed in patients treated with MPL.

The compelling data is believed to have drawn positive attention from the scientific community, underscoring the treatment potential of MPL and reinforcing collaborative efforts between PharmAust and Berry Consultants to advance the research.

HEALEY ALS trial

Subsequent to the period, MPL was accepted into the prestigious HEALEY ALS platform trial in the US.

The innovative trial is a large-scale collaboration across more than 70 clinical sites in the US to evaluate several drug candidates simultaneously with a view to increasing patient access, reducing study costs and shortening study completion timelines.

MPL’s inclusion is expected to provide independent validation by global ALS experts of the drug’s potential and enhances PharmAust’s visibility within the ALS research community.

New appointments

The company also pivoted its commercialisation strategy to focus solely on the development of MPL for the treatment of a range of neurodegenerative diseases.

The drug’s mechanism of action leads the company to believe it may also have considerable utility in Alzheimer’s, Parkinson’s and Huntington’s disease.

To implement the new strategy, PharmAust made a number of board changes during the period.

PharmAust said the quality and extensive experience of the new appointments reinforces its vision of becoming a world leader in the development of treatments for neurodegenerative diseases.