PharmAust inks approval from Macquarie University for Open-Label MND extension study

Macquarie University has given ethics approval to PharmAust allowing the final 3 patients to be enrolled in the Open-Label Extension study of monepantel
The study started at Calvary Health Care Bethlehem in February and 9 of the 12 eligible patients have enrolled on the study to date
The Phase 1 MEND study, compassionate-use program, and OLE study have now seen patients treated continuously with monepantel for up to 19 months

Special Report: Macquarie University has given PharmAust the green light to conduct an Open-Label extension study of monepantel (MPL) in patients with Motor Neurone Disease.

The study’s overarching objective is to assess the long-term safety and tolerability of MPL as well as the biomarkers and efficacy endpoints of 12 patients who took part in the Phase 1 MEND study.

Using a daily dose of 10mg/kg body weight of MPL, the 12-month Open Label Extension (OLE) study will collect ongoing data and further test the hypotheses that MPL administration to individuals living with MND/ALS will safely reduce disease-associated protein accumulation in motor neurons and provide therapeutic benefits.

The study kicked off at Calvary Health Care Bethlehem in February with 9 of the 12 eligible patients enrolled to date.

Patients have now been treated continuously for up to 19 months without safety concerns. For these 12 patients the average time since diagnosis is now 29.9 months, contrasting with population-based prospective registries which report 1 year mortality rates after diagnosis ranging from 22% to 34%.

Remaining 3 patients can now benefit from MPL

“We are delighted and excited to receive ethics approval from Macquarie University, which allows us to offer all Phase 1 MEND patients the opportunity to enrol in the Open-Label Extension study,” PAA CEO Dr Michael Thurn says.

“It’s incredibly rewarding to know that 9 patients have already been enrolled at Calvary Health Care Bethlehem and the remaining 3 patients can now continue receiving benefit from treatment with monepantel.”

A highly significant market opportunity

Motor neurone disease (MND), or amyotrophic lateral sclerosis (ALS), presents a major market opportunity for PharmAust should MPL be approved in major regulatory jurisdictions such as the United States and Europe.

In the US and western Europe it is estimated there are more than 37,000 and 56,000 patients with MND/ALS, respectively, with more than 268,000 globally.

The MND/ALS treatment market was more than US$9 billion in 2022 and is estimated to each USD $23 billion by the end of 2035. In 2021, the median treatment cost was US$218,872 for orphan drugs (ranging from US$237 to US$1,272,021).

The last three drugs (Qalsody, Radicava, and Relyvrio) that received US Food and Drug Administration approval for the treatment of MND/ALS each have an annual cost of between US$150,000 to US$200,000, with Relyvrio having generated US$381m in sales during 2023, its first year after launch.

This article was developed in collaboration with PharmAust, a Stockhead advertiser at the time of publishing.

This article does not constitute financial product advice. You should consider obtaining independent advice before making any financial decisions.