Clinical-stage biotechnology company PharmAust (ASX:PAA) has provided a four-month progress update on the ongoing 12-month Open-Label Extension (OLE) study in patients with Amyotrophic Lateral Sclerosis (ALS).
The OLE study is evaluating ten of the original twelve patients who participated in the completed Phase 1 MEND study.
PharmAust said treatment with monepantel (MPL) daily continues to be well-tolerated, slowed disease progression and improved survival.
Managing director Dr Michael Thurn said, "These results are extremely encouraging and continue to emphasise the excellent long-term safety profile and potential of MPL to increase the life expectancy of this very vulnerable patient population. After just 4 months in the OLE study, the rate of decline in ALSFRS-R was 0.41 points/month with daily treatment of MPL.
"Studies have shown that rates of decline in ALSFRS-R scores between -0.25 to -0.45 points/month translate to a median survival of 3.7 years. The results also provide us with confidence on dose selection for the HEALEY ALS Platform Trial."
"In October, we will reach the 2-year anniversary since the first patients started treatment with MPL," continued Dr Thurn.
"We have made incredible progress over this period, including engaging with the US Food and Drug Administration regarding the development pathway for MPL via a Pre-Investigational New Drug meeting, being granted Orphan Drug Designation from the US FDA and more recently, monepantel being selected for inclusion in the prestigious HEALEY ALS Platform Trial at Massachusetts General Hospital, the largest teaching hospital of Harvard Medical School. We're very excited about the path ahead and the potential to help more patients."
