PharmAust gears for major clinical milestones in 2024, following strong start and FDA feedback

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By Colin Hay - 
PharmAust ASX PAA quarterly report MND ALS MPL

PharmAust (ASX: PAA) is preparing to undertake a number of key activities in its treatment development schedule after completing a strong start to the year.

The clinical-stage biotechnology company highlighted a number of milestones in releasing its quarterly activities report for the period ended 31 March 2024.

“The March quarter was particularly important and successful for PharmAust, headlined by the positive Phase 1 trial top-line data for monepantel (MPL) in motor neurone disease (MND)/amyotrophic lateral sclerosis (ALS),” interim chief executive officer John Clark said.

“This gives us a strong foundation to push forward with both the open-label extension study and the pivotal adaptive Phase 2/3 clinical trial.”

“The positive Food and Drug Administration (FDA) feedback has provided us with a clear path towards potential accelerated approval,” he added.

Executive appointments

PharmAust also expanded its highly-experienced leadership group with key appointments to both the management team and the board.

“This has us well equipped to continue advancing the clinical development of MPL as planned,” Mr Clark said.

“We thank those involved in the Phase 1 study for their efforts to date and moving forward, including the trial participants, trial sites and the principal investigators.”

“Alongside Associate Professor Susan Mathers and one of our trial participants, I was very pleased to speak to Channel 7 on MPL and its impact on MND patients in a story that received national coverage.”

“We remain on schedule for the commencement of our pivotal adaptive Phase 2/3 clinical trial starting in CY2024.”

“I’m looking forward to leading the company through this exciting time, and we’ll be communicating further updates to shareholders as they become available.”

Numerous highlights

In a very active reporting period, PharmAust achieved a number of significant highlights including impressive Phase 1 trial results in safety and tolerability for its MPL treatment.

Preliminary efficacy data found there was a 58% reduction in the rate of disease progression for Cohort 2 (high dose) using the FDA primary efficacy endpoint, ALSFRS-R.

PharmAust received positive FDA feedback and directions for the path required to potentially receive accelerated and full approval of MPL.

The importance of this advance was magnified by the recent removal from sale in North America of previously FDA-approved MND/ALS drug Relyvrio.

Busy period ahead

PharmAust has started the current quarter off by signing manufacturing process development agreements with Syngene International and Catalent Pharma Solutions to commence validation and registration batches of MPL at scale, to support regulatory approval and commercialisation.

Under these agreements, Syngene will manufacture 60 kilograms of good manufacturing practice (GMP)-quality MPL consisting of one 15kg engineering batch followed by three 15 kg process validation batches designed to validate the GMP manufacturing process, support product registration and prepare the company for commercial supply.

Catalent will be responsible for the GMP manufacture of three registration batches totaling more than a million tablets, which will be required to support product registration and facilitate commercial scale-up activities.

The product manufactured will be used to support an upcoming pivotal registration Phase 2/3 clinical study.

It is expected that the manufacturing process development agreements will be followed by a commercial supply agreement.