PharmAust Limited (ASX: PAA) has confirmed that all patients have completed its Phase 1 MEND Study of monepantel (MPL) for motor neurone disease (MND/ALS) and that the release of top-line results remains on track for Q1 CY24.
“Excitement is growing knowing that we are only months away from releasing top-line data.
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,” PharmAust CEO 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.”
Phase 1 MEND Study
The Phase 1 MEND Study is an open-label, multicentre study involving 12 patients with MND/ALS to determine the recommended Phase 2 dose based on safety and preliminary efficacy. The study design involves two cohorts of six patients, each 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. The highest dose evaluated was 10 mg/kg.
To date, there have been no deaths or treatment-related serious adverse events. Only three adverse events possibly related to treatment with monepantel have been recorded. There have been no reports of difficulty in swallowing tablets or patients needing assistance with breathing, which are common clinical signs of late-stage disease progression. All patients are continuing treatment with monepantel under a compassionate use program and will be invited to participate in a 12-month open-label extension study due to commence in or around January 2024.
The study is supported by a drug development grant of $881,085 from FightMND, Australia’s largest independent not-for-profit organisation for MND research. The final instalment payment of $150,142.80 (plus GST) was received from FightMND in November 2023.
Future Directions
PharmAust has successfully submitted for approval the protocol for the 12-month Open Label Extension (OLE) Study to the Human Research Ethics Committee (HREC). In addition, the Company has applied for an Orphan Drug Designation with the United States (US) Food and Drug Administration (FDA) and remains on track to commence a Phase 2 Study for MND/ALS in H1 CY24.
The FDA has the authority to grant orphan drug designation (ODD) to a drug to prevent, diagnose or treat a rare disease or condition, defined as any disease or condition that affects less than 200,000 persons in the US. An ODD qualifies sponsors for incentives, including tax credits for qualified clinical trials, exemption from user fees, and the potential for seven years of market exclusivity after approval.
