• PharmAust successfully completes a Pre-IND meeting with the US FDA
  • Path outlined to potentially receive accelerated and full approval of monepantel for treatment of MND/ALS
  • PharmAust to initiate requirements requested by FDA in preparation to open IND application for its adaptive Phase 2/3 clinical study


Special Report: PharmAust has successfully completed a Pre-IND meeting with the US Food and Drug Administration (FDA) with a path outlined to potentially receive accelerated and full approval of its lead drug monepantel for the treatment of motor neurone disease/amyotrophic lateral sclerosis (MND/ALS). 

Clinical-stage biotech PharmAust (ASX:PAA) says the US pre-IND meeting marks a significant milestone as the company gears up for an adaptive Phase 2/3 clinical study in Q2 2024. 

PAA aims to use this study as the primary support for accelerated and/or full approval of monepantel for MND/ALS treatment.

The pre-IND meeting focused on establishing formal communication with the FDA, confirming details and ensuring the acceptability of PAA’s proposed development program, including requirements for non-clinical and clinical pharmacology, clinical chemistry and manufacturing controls. 

Seeking specific guidance on the design and adequacy of the planned adaptive Phase II/III clinical study, PAA received FDA confirmation that accelerated and/or full approval could potentially be granted based on substantial evidence of effectiveness and a robust safety database. 

Crucially, the FDA says there are no set minimum requirements for the number of patients or study sites in the US, opening the door for the adaptive Phase 2/3 clinical study to be global and enabling clinical sites and patients from Europe and Australia, reducing recruitment timelines. 

Following the completion of the adaptive study, PAA intends to seek approvals from the European Medicines Agency (EMA) and Australia’s Therapeutic Goods Administration (TGA).


Study to evaluate safety and efficacy

PAA says the Phase 2/3 clinical study will be a multicentre, randomised, placebo-controlled, adaptive clinical study evaluating the safety and efficacy of monepantel in patients with MND/ALS over 48 weeks. 

The primary aim of the study will be to assess the efficacy of monepantel compared to placebo on the progression of MND/ALS. 

This drug’s efficacy will be evaluated as a change from baseline disease severity measured by the ALS Functional Rating Scale-Revised (ALSFRS-R) total score and survival. 

Being an adaptive study design an interim analysis will be performed at week 24 by a team of unblinded statisticians for the potential to receive accelerated approval from the FDA.


OLE trial underway as analysis shows drug benefit

It’s been a big week for PAA, which also announced the first patient has been dosed in its 12-month open-label extension (OLE) study for patients with MND/ALS. 

All 12 patients from the Phase 1 MEND Study are expected to be enrolled in the OLE after meeting the minimum requirement of being able to swallow.

The company also unveiled significant findings from a statistical review indicating a notable survival benefit for MND/ALS patients receiving monepantel. 


‘Highly encouraged’ by positive FDA feedback

PAA chief executive Dr Michael Thurn says the company is “highly encouraged” by the positive feedback from the FDA.

“It aligns with our expectations and provides the company with a clear understanding of the requirements to potentially receive accelerated and/or full approval of monepantel for treating MND/ALS,” he says.

“This advice truly positions us as a global play following the successful completion of our planned adaptive Phase 2/3 clinical study.”



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.