• PharmAust engages globally renowned experts in MND/ALS to form a scientific advisory board (SAB)
  • The SAB will provide expert advice for the development of monepantel to treat MND/ALS
  • The SAB will also support the planning stages for PharmAust’s adaptive Phase 2/3 clinical study


Special Report: PharmAust has engaged globally renowned experts in MND/ALS and rare neurodegenerative diseases to form a scientific advisory board (SAB) for the next stage of development of monepantel (MPL).

Monepantel is PharmAust’s (ASX:PAA) lead candidate for the treatment of Motor Neurone Disease (MND) / Amyotrophic Lateral Sclerosis (ALS) and pipeline expansion activities in additional neurodegenerative diseases.

MPL is a potent and safe inhibitor of the mTOR pathway, which plays a central role in cell growth, proliferation of cancer cells and degenerating neurons, and regulates the cellular “cleaning process” where toxic protein is broken down into macromolecules to be reused.

This autophagic process is disrupted in most neurodegenerative diseases, including motor neurone disease (MND/ALS).

PAA is currently completing a Phase 1 study in patients with MND/ALS with results expected to be announced sometime this quarter.

The clinical stage biotech company anticipates starting an adaptive Phase 2/3 clinical study in the first half of 2024. This could lead to accelerated approval with the US Food and Drug Administration in 2026.


SAB board members

PAA has also formed a scientific advisory board (SAB) for the provision of expert advice on the development of monepantel as well as to explore other neurodegenerative diseases.

Members of the SAB include Prof Leonard van den Berg, who holds a chair in experimental neurology of motor neuron diseases at the University Medical Center Utrecht in the Netherlands, and Dr Sabrina Paganoni who is co-director of the Neurological Clinical Research Institute at the Massachusetts General Hospital, assistant professor at Harvard Medical School and co-Principal Investigator of the HEALEY ALS Platform Trial, the world’s first platform trial for ALS.

Other members include Dr Melanie Quintana, director and senior statistical scientist at Berry Consultants in Austin, Texas and Dr Christian Freitag, who brings more than 20 years of experience in the pharmaceutical industry.


‘Clear need for more effective treatment options’

“We are honoured to work with this prestigious and accomplished group of thought leaders as our scientific advisory board,” PAA chief executive Dr Michael Thurn says.

“These innovative individuals have pioneered breakthroughs in MND/ALS, and together they bring a wealth of knowledge and experience to PharmAust.”

Prof van den Berg says she is looking forward to being a part of PharmAust’s SAB.

“There is a clear need for more effective treatment options for MND/ALS,” he says.

“The survival data produced to date with monepantel is encouraging and compounds that enhance autophagy have the potential to treat a wide range of neurodegenerative diseases”.



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.