PharmAust (ASX:PAA) has announced that all patients in Cohort 4 have completed dosing in its Phase 1 MEND study of monepantel (MPL) in patients with motor neurone disease (MND/ALS).
The company said data from the Phase 1 MEND Study will be used to support an Orphan Drug Designation (ODD) and to open an Investigational New Drug (IND) application with the US FDA to commence a Phase 2 Study in the first half of the calendar year 2024.
PharmAust CEO Dr Michael Thurn said, “We are extremely pleased with how the study has progressed over the last 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.
"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."