- Drug developer, Pharmaust (ASX:PAA) announces all 12 patients are finished with its phase one MEND study using monepantel (MPL)for Motor Neuron Disease (MND)
- Each patient received increasing dosages in a staggered approach with the highest dose at 10mg/kg, with higher doses subject to meeting set safety criteria
- These patients are invited to attend a 12-month open-label extension study commencing in January 2024, following approval from the HREC
- Additionally, Pharmaust applies for orphan drug designation with the FDA with help from FIGHTMND’s $880,000 development grant
- PAA shares are up 1.08 per cent, trading at 9.4 cents at 10:45 am AEDT
Drug developer, Pharmaust (ASX:PAA) has announced that all patients have completed its phase one MEND study using monepantel (MPL) to test its functions on Motor Neuron Disease (MND).
The study compiled 12 people in two cohorts of six and was used to determine the best dosage of MPL to inform phase two trials and to test safety and efficacy.
Each patient received a progression of dosages in a staggered approach with the highest dose at 10 milligrams per kilogram (mg/kg). Progression to a higher dose of MPL is subject to meeting set safety criteria governed by a safety monitoring committee.
“We have several patients who have now received treatment with monepantel for 13 months or more, with the median treatment duration being 10.6 months for the 12 patients who participated in the study,” PAA Chief Executive Officer Dr Michael Thurn said.
“Every month a patient continues on treatment brings enormous satisfaction and excitement around the potential benefit that monepantel may bring to patients, their families and caregivers, and of course the healthcare system as a whole.”
To date, the company has experienced no serious adverse reactions, but it has treated three minor occurrences, with all patients choosing to continue the treatment.
These patients have also been invited to attend a 12-month open-label extension study, which is set to commence in January 2024, following approval from the Human Research Ethics Committee (HREC).
Additionally, Pharmaust has applied for orphan drug designation with the United States Food and Drug Administration (FDA), while preparing for phase 2 trials to take off in the first half of the 2024 calendar year.
Pharmaust is pushing ahead with support from FightMND, Australia’s largest independent not-for-profit organisation for MND research, which has supplied a development grant of more than $880,000 for MPL activities.
PAA shares were trading up 1.08 per cent, trading at 9.4 cents at 10:45 am AEDT.