PharmAust’s monepantel study for MND treatment on fast track after FDA meeting

PharmAust (ASX: PAA) has achieved another significant milestone along the path to gaining approval for the use of monepantel (MPL) in the treatment of motor neurone disease/amyotrophic lateral sclerosis (MND/ALS).

The company has received positive feedback from a pre-investigational new drug (pre-IND) meeting with the United States Food and Drug Administration (FDA), which has provided important guidance for the next stages of the approvals process.

PharmAust says guidelines provided by the FDA indicate it might only be required to undertake an adaptive Phase 2/3 clinical study in Q2 2024 to support accelerated and/or full approval of MPL for treating MND/ALS.

PharmAust chief executive officer Dr Michael Thurn said this is a significant development for the company.

“We are highly encouraged by the positive feedback from the FDA.”

“It aligns with our expectations and provides the company with a clear understanding of the requirements to potentially receive accelerated and/or full approval of MPL for treating MND/ALS,” Dr Thurn said.

“This advice truly positions us as a global play[er] following the successful completion of our planned adaptive Phase 2/3 clinical study.”

It being an adaptive study design, an interim analysis will be performed at Week 24 by a team of unblinded statisticians for the potential to receive accelerated approval from the FDA.

FDA Discussions

The pre-IND meeting initiated formal communication with the FDA to help confirm the details and acceptability of PharmAust’s proposed ongoing development program, including the requirements for non-clinical and clinical pharmacology, clinical chemistry and manufacturing controls.

PharmAust was looking for particular guidance from the FDA on the design and overall adequacy of the planned adaptive Phase 2/3 clinical study.

Significantly, the meeting confirmed that PharmAust may potentially receive accelerated and/or full approval from this single clinical study subject to demonstrating substantial evidence of effectiveness and an adequate database supporting safety.

The FDA also advised that there were no minimum requirements for the number of patients and study sites located in the US, paving the way for the adaptive Phase 2/3 clinical study to be global in nature.

International studies

PharmAust is now planning to utilise clinical sites and patients from Europe and Australia to reduce recruitment timelines, while also allowing it to seek approval from the European Medicines Agency and Australia’s Therapeutic Goods Administration following completion of the Phase 2/3 study.

The Phase 2/3 clinical study will be a multicentre, randomised, placebo-controlled, adaptive clinical study evaluating the safety and efficacy of MPL in MND/ALS patients over 48 weeks.

The primary aim will be to assess the efficacy of monepantel on the progression of MND/ALS compared to a placebo.

This will be evaluated as a change from baseline disease severity measured by the ALS functional rating scale-revised total score and survival.

First patient dosed

Earlier this week, PharmAust dosed the first patient in an open-label extension (OLE) study follow-up to a program that has obtained promising initial results.

Being conducted at the Calvary Health Care Bethlehem in Melbourne, the OLE study is investigating the long-term safety, tolerability and efficacy of MPL in MND/ALS patients who previously completed the Phase 1 MEND Study.

In this next stage, patients will receive a daily dose of 10 mg MPL per kilogram of body weight to further test if MPL will safely reduce disease-associated protein accumulation in motor neurons and provide therapeutic benefits for MND/ALS sufferers.