- PharmAust (ASX:PAA) wraps up cohort four of its phase one MEND study using MPL at 10 mg/kg to treat MND
- The study is supported by Australia’s largest independent not-for-profit organisation for MND research, FightMND
- PAA is using the results to support orphan drug designation and open an investigational new drug application with the US FDA
- This process will allow the company to proceed to a phase two study in H1 CY24
- Shares last traded at 7.5 cents
PharmAust (ASX:PAA) has completed dosing all patients in cohort four of its phase one MEND study using monepantel (MPL) at 10 milligrams per kilogram to treat Motor Neurone Disease (MND).
The study is supported by a drug development grant of $881,085 from FightMND, Australia’s largest independent not-for-profit organisation for MND research.
The company reported it will use the results, which have been collected over 12 months, to support orphan drug designation (ODD) and to open an investigational new drug (IND) application.
These processes await approval from the United States Food and Drug Administration (FDA) in order for Pharmaust to commence a phase two study in the first half of the 2024 calendar year.
“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,” PAA CEO Dr Michael Thurn said.
“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.”
PharmAust expects to begin the OLE study early in the new year to conduct a second round of 12-month analysis. Meanwhile, patients from phase one will undergo a final follow-up appointment before data is collated for release early next year.
PAA shares last traded at 7.5 cents.