PharmAust’s pioneering monepantel MND study receives HREC green light for final phase

Clinical-stage biotechnology company PharmAust (ASX: PAA) has received approval from the human research ethics committee (HREC) at Sydney’s Macquarie University to start an open-label extension study of lead drug monepantel (MPL) on patients with motor neurone disease (MND) / amyotrophic lateral sclerosis (ALS).

The approval will allow for enrolment of the study’s final three patients.

The study commenced at Calvary Health Care Bethlehem private hospital near Melbourne in February and nine of a total 12 eligible patients have been enrolled to date.

The average time since diagnosis for all patients is 29.9 months, with a minimum of 17.3 months up to a maximum of 50.3 months.

The final three patients are expected to join the study this month.

HREC approval

PharmAust chief executive officer Dr Michael Thurn welcomed the HREC approval.

“We are excited to now be able to offer all Phase 1 MEND patients the opportunity to enrol in this extension study,” he said.

“It is incredibly rewarding to know that nine patients have already been enrolled at Calvary Health Care and the remaining three can now continue receiving benefit from treatment with monepantel.”

“We are very thankful to the patients and to our [study] investigators for their ongoing involvement as we continue working towards a much-needed new therapy for MND and ALS.”

Long-term safety

PharmAust’s 12-month extension will use a daily dose of MPL equating to 10 milligrams per kilogram body weight and is primarily designed to assess the long-term safety and tolerability of the drug.

It will also assess biomarkers and efficacy endpoints (including disease severity, cognitive, respiratory and quality of life assessments) using industry-standard measures such as ALS Functional Rating Scale-Revised, Edinburgh Cognitive & Behavioural ALS Screen, Slow Vital Capacity and ALSSQOL-R.

The extension will further test the hypotheses that MPL administration to individuals living with MND and ALS can safely reduce disease-associated protein accumulation in motor neurons and provide therapeutic benefits.

Patients have been treated continuously for up to 19 months without any safety concerns to date.

Relyvrio withdrawal

The study approval comes at a critical juncture for MND/ALS sufferers, with news that controversial drug Relyvrio has been withdrawn from sale in North America.

The drug’s manufacturer Amylyx Pharmaceuticals has acknowledged that their product made no improvement in the condition, a huge disappointment for those urging the US Food and Drug Administration and other federal agencies to back more experimental therapies for the fatal disease.

MND statistics

Research shows MND affects over 350,000 people globally and kills more than 100,000 people each year.

The disease is invariably fatal, with the average life expectancy of someone with MND being around 27 months.

The annual addressable market for MND and ALS is $5.43 billion, with the current standard of care treatment drug Riluzole prolonging life on average by three months.

MND and ALS have no known cure, and there is currently no effective treatment to reverse the progression.