• Phase 1 MEND study outcomes for MND/ALS treatment now with FDA for review in support of PharmAust’s Orphan Drug Designation request
  • Clinical data shows monepantel (MPL) has the potential to delay the progression of MND/ALS by ~58%
  • PAA on track to begin pivotal registration Phase 2/3 study in H1 CY24


Special Report: Biotech company PharmAust has submitted supplementary clinical data from its recently completed Phase 1 MEND Study with the US Food and Drug Administration (FDA) Office of Orphan Products Development in support of its recent request for Orphan Drug Designation (ODD) for monepantel (MPL) to treat motor neurone disease (MND) and amyotrophic lateral sclerosis (ALS).

Positive top-line data from the study – involving 12 patients with MND/ALS and the daily administration of MPL over a 7-12 month period – showed the drug was well-tolerated and did not result in any dose-limiting toxicities or serious side effects.

A 58% reduction in disease progression was reported from Phase 1.

PharmAust (ASX:PAA) says the trial is showing a meaningful therapeutic effect with biomarkers demonstrating a large reduction in cerebrospinal fluid.

Presently, sufferers have an average life expectancy of about 27 months, with current FDA-approved treatments Riluzole and Relyvrio only able to prolong life by about 2-3 months and 10 months respectively.

MPL indicates that life expectancy could increase patients’ lives by an additional 13.5-56.5 months.

An updated request for Orphan Drug Designation (ODD) to the FDA has now been submitted, following a request for the Phase 1 data received in January 2024.


Next phase to begin in mid-2024

PAA chief executive Dr Michael Thurn is very confident the FDA will grant monepantel an ODD for the treatment of MND/ALS – which affects over 350,000 people globally – based on the strength of the Phase 1 MEND Study outcomes.

“Receiving ODD status from the FDA will come at an opportune time for PharmAust given the recent clinical trial failures of the 2 leading FDA drugs for MND/ALS,” he says.

“At a time of need, monepantel offers much-needed hope for patients with this severely debilitating disease.

“The company is in the process of finalising our exciting plans for its pivotal registration Phase 2/3 study and is on track to commence the study during the middle of this year.”

It has recently been announced that two FDA-approved treatments for MND/ALS – the Amylyx Pharmaceuticals drug RELYVRIO® and Ferrier International S.A. drug edaravone – produced unsuccessful Phase 3 clinical trial results and are at risk of being withdrawn from the market, creating an even greater need for new solutions for patients.



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.