PharmAust reports encouraging interim results for ALS/MND drug monepantel in ongoing study

PharmAust (ASX: PAA) has received promising interim results from a major study into monepantel (MPL), its lead amyotrophic lateral sclerosis (ALS) / motor neurone disease (MND) treatment candidate.

The clinical-stage biotechnology company revealed that treatment with MPL at 10 milligrams per kilogram of body weight daily continues to be well-tolerated, slowing disease progression and improving survival after four months of an ongoing yearlong open-label extension (OLE) study of patients with ALS.

The OLE study is evaluating ten of the original 12 patients who participated in the completed Phase 1 MEND study while using the recommended dose for the Phase 2/3 study of MPL in the HEALEY ALS platform trial, expected to commence enrolment next quarter.

Encouraging results

Patients have been treated with MPL for varying durations ranging from 11.3 to 22.7 months with a median of 18.5 months and at dosage levels varying between 2 and 10mg/kg per day.

“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,” PharmAust managing director Dr Michael Thurn said.

“After just four 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.”

The HEALEY trial is an innovative Phase 2/3 platform trial structure conducted out of Massachusetts General Hospital that allows for multiple investigational treatments to be tested simultaneously using a shared master protocol.

‘Incredible progress’

“In October, we will reach the 2-year anniversary since the first patients started treatment with MPL,” Dr Thurn added.

“We have made incredible progress over this period [and] we’re very excited about the path ahead and the potential to help more patients.”

ALS/MND, also known as Lou Gehrig’s disease, is a neurological disorder that affects motor neurons—nerve cells in the brain and spinal cord that control voluntary muscle movement and breathing.

Approximately 1,400 people in Australia are living with this disease, which has a survival rate approximately two-to-five years from the onset of symptoms and typically affects people in their mid-50s.