PharmAust ASX PAA major milestone motor neurone disease treatment study dosing patients
October 13, 2023
PharmAust hits major milestone in motor neurone disease treatment study

Perth-headquartered clinical-stage biotechnology company PharmAust (ASX: PAA) has achieved a significant milestone with the final dosing of patients participating in the assessment of a treatment for the fatal motor neurone disease (MND/ALS).

The study has concluded that all patients participating in Cohort 4 of the in-depth trial have successfully completed dosing in its Phase 1 MEND study of monepantel (MPL) in patients with motor neurone disease.

PharmAust chief executive officer, Dr Michael Thurn, said data from the Phase 1 MEND study will now be used in an orphan drug designation (ODD) and to open an investigational new drug (IND) application with the United States Food and Drug Administration (FDA) to commence a Phase 2 study early next 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,” Dr Thurn said.

What is MND?

Motor neurone disease (MND) is a term used to describe a group of diseases that affect nerve cells called “motor neurones”.

Motor neurones normally carry messages from the brain to the muscles via the spinal cord. The messages allow people to make voluntary movements like walking, swallowing, talking and breathing.

With MND, however, the nerves become damaged and start to die so muscles gradually get weaker and waste away.

Currently, there is no cure for MND and it is life shortening.

The speed at which MND progresses and survival times vary for each person.

Recent statistics estimate there are over 2,000 people in Australia who are currently diagnosed with MND and every day 2 Australians are diagnosed.

According to the International Alliance of ALS/MND Associations, MND affects over 350,000 people globally and kills more than 100,000 people yearly. The disease is invariably fatal with the average life expectancy of someone with MND being around 27 months.

Pharmaust study rolls on

Patients participating in the PharmAust study will now 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,” Dr Thurn said.

Phase 1 MEND study

The Phase 1 MEND Study is an open label, multicentre study involving 12 patients with MND/ALS with the goal of determining the recommended Phase 2 dose based on safety and preliminary efficacy.

The study design involves 2 groups of 6 patients with each group progressively receiving higher dose levels of MPL in a staggered design approach over time.

Progression to a new dose level of MPL was subject to meeting set safety criteria governed by a safety monitoring committee.

In the next stage, open label extension (OLE) study, the 12 patients currently receiving treatment with monepantel will continue treatments for a further 12 months.

Principal Investigator associate professor Susan Mathers, said all of the participants have tolerated the study drug very well with no safety issues, despite a year of treatment.

“As investigators, we are very grateful for the enthusiastic support and commitment from our patients and their families. Like them, we eagerly await the study results.”

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