PharmAust reports positive top-line results from Phase 1 MEND study

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PharmAust (ASX:PAA) has announced that it has met its primary safety and tolerability endpoints with monepantel (MPL) in the Phase 1 MEND study.

The company said MPL demonstrated a positive potential efficacy signal. Rates of disease progression in patients with Motor Neurone Disease (MND)/Amyotrophic Lateral Sclerosis (ALS) measured by changes in ALSFRS-R may be slowed by 58 per cent for cohort two compared to an external control cohort.

The Phase 1 MEND study was a multicentre, open-label study comprising a 24-hour escalating single-dose PK study and a four-week repeated escalating dose study to establish the safety, tolerability and pharmacokinetic (PK) parameters of MPL administered orally to patients with MND/ALS.

PharmAust said the safety and tolerability profile of MPL included no treatment-related deaths, with all 12 patients completing the study, and no dose-limiting toxicities experienced. A total of 56 treatment-emergent Adverse Events (AEs) were reported. Only 3 AEs, graded mild, were considered possibly related to the study drug.

Patients have remained on daily treatment of MPL for 10 to 16 months. Upon completing the study, all participants continued receiving MPL via a special access scheme and opted to enrol on a 12-month open-label extension (OLE) study.

PharmAust CEO Dr Michael Thurn, “The release of the top-line Phase 1 MEND study results is an exciting milestone for PharmAust as we take a significant step towards helping people diagnosed with this rare and incurable disease.

"The 58% slowing in ALSFRS-R decline amongst Cohort 2 participants clearly demonstrates the potential to provide meaningful clinical benefit to people living with MND/ALS. To know that we have potentially prolonged the lives of 12 patients is extremely satisfying and humbling.

"We now look forward to advancing discussions with strategic partners who share our vision for monepantel. I want to thank the trial participants, their caregivers, and families, as well as the sites' principal investigators, Associate Professor Susan Mathers and Professor Dominic Rowe, and their study coordinators for their tremendous ongoing contribution to the MEND study.”