• PAA has finished dosing all patients in cohort 4 of its phase 1 study in MND/ALS
  • Data from phase 1 expected in Q1 CY24 before a phase 2 trial commences
  • Phase 1 data will support US orphan drug designation application
  • MND/ALS addressable market is US$3.6b per annum


Biotech company PharmAust has completed dosing all patients in cohort 4 of its Phase 1 MEND study of the drug monepantel (MPL) in patients with motor neurone disease (MND/ALS).

Data from this phase will be used to support an Orphan Drug Designation (ODD) and to open an Investigational New Drug (IND) application with the United States (US) Food and Drug Administration (FDA) to commence a Phase 2 Study in H1 CY24.

Orphan drug designation is given to a drug or biological product to prevent, diagnose or treat a rare disease or condition – like MND which affects over 350,000 people globally and kills more than 100,000 people yearly.

For the pharmaceutical company, ODD incentives include market exclusivity for several years along with tax credits for qualified clinical trials.

Not to mention, the MND/ALS addressable market is US$3.6B per annum, with Riluzole a treatment that prolongs life on average by 2-3 months reaching annual peak sales of ~US$1B.

This bodes well for PharmAust’s (ASX:PAA) treatment if it can secure ODD.

MND/ALS is invariably fatal with the average life expectancy of someone with MND/ALS being around 27 months.  Independent studies have shown that one-third of patients die within 12 months after the first diagnosis.

Encouragingly, some patients have been treated with MPL for over 12 months now, no patients have withdrawn from the study, there have been no deaths or significant adverse events.

The study is supported by a drug development grant of $881,085 from FightMND, Australia’s largest independent non-for-profit organisation for MND research.


Data expected in Q1 CY24

Patients have one last follow-up visit to attend before the study completes and the data collation process begins verification and finalisation for analysis and release of top-line data in Q1 CY24.

“We are extremely pleased with how the study has progressed over the last year,” PAA CEO Dr Michael Turn said.

“It’s an exciting milestone to reach knowing that some patients have been receiving treatment with monepantel for over a year now and patients have elected to continue treatment with monepantel under a compassionate use program.

“Patients will also be eligible to roll-over into an Open Label Extension (OLE) Study that is currently in the final stages of preparation for submission to the Human Research Ethics Committee for approval.

“The OLE Study is expected to begin very early in the New Year and will generate valuable safety and efficacy data for a further 12 months.

“This is a fantastic outcome for the company and for the patients living with this severely debilitating and ultimately fatal disease.”


Phase 2 and OLE study to run parallel

Once data is received early next year, company plans to commence a Phase 2 Study in H1 CY24, in parallel with the OLE study.

“All of the participants have tolerated the study drug very well with no safety issues, despite a year of treatment,” principal investigator Associate Professor Susan Mathers said.

“As investigators, we are very grateful for the enthusiastic support and commitment from our patients and their families.

“Like them, we eagerly await the study results.”



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.