May be a few days to go yet as there was Christmas Day and New Years Day thrown in the mix.
Certainly looks positive and a comprehensive amount of supporting material was submitted.
2025 ASX Announcements look a bit light when you check the list.....maybe that's about to fill up with a run of Announcements? the one that would rekindle interest would be a cornerstone partner coming on board?
Obviously Trial Approval , 1st Patient Recruited and 1st Patient Dosed always grabs a headline.. plus it indicates the clock has started on the Trial duration. Recruitment should be relatively swift with 72 Trial Sites.
Amazing only a year ago was the FDA PAA / NUZ Meeting FDA Grants PharmAust Pre-IND Meeting Highlights: • US FDA grants PharmAust a Pre-IND meeting for monepantel for the treatment of MND/ALS
• The meeting provides PharmAust with the opportunity to receive feedback from the FDA on the proposed ongoing development program to receive accelerated approval
• FDA has committed to provide written responses by 13 February 2024
• PharmAust remains on-track for near-term milestones, including opening an IND application and initiating the adaptive Phase 2/3 clinical study, in Q2 2024
2 January 2024 – Perth, Australia: PharmAust Limited (ASX: PAA & PAAOA) (“PharmAust” or “the Company”), a clinical-stage biotechnology company, is pleased to announce the United States (US) Food and Drug Administration (FDA) has granted PharmAust a Pre-Investigational New Drug (Pre-IND) meeting for monepantel for the treatment of motor neurone disease/amyotrophic lateral sclerosis (MND/ALS). The request was submitted on 15 December 2023, and the FDA has committed to provide written responses by 13 February 2024. The request for a pre-IND meeting formally initiates communications with the FDA regarding developing monepantel to treat MND/ALS. The pre-IND meeting aims to 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. Reviewers from the FDA’s Office of Neuroscience - Division of Neurology I, across multiple disciplines including Medical, Pharmacology/Toxicology, Statistical, Clinical Pharmacology/Biopharmaceutics, and Chemists, will provide feedback on PharmAust’s proposed development program for monepantel. Importantly, and given the stage of development of monepantel for the treatment of MND/ALS, it provides PharmAust with an opportunity to seek feedback from the FDA on the design of its planned Phase 2/3 adaptive clinical study and gain insights into the FDA’s requirements for monepantel to be potentially granted accelerated approval. With these considerations in hand, PharmAust will be able to proceed confidently with its full IND application filing in Q2 2024. PharmAust Chief Executive Officer Dr Michael Thurn commented: “We are looking forward to receiving feedback from the FDA. It’s remarkable to think that PharmAust is potentially one clinical study away from receiving accelerated approval and providing MND/ALS patients with a much-needed new therapy for a condition that is invariably fatal. Receiving feedback from the FDA in February positions us well to achieve our near-term milestone of opening an IND in Q2 2024 with the goal of initiating the planned adaptive Phase 2/3 clinical study in patients shortly after that." PharmAust’s planned Phase 2/3 study is a multicentre, randomised, placebo-controlled, adaptive clinical study evaluating the safety and efficacy of monepantel in patients with MND/ALS over 48 weeks. The primary aim will be to assess the efficacy of monepantel, as compared to placebo, on the progression of MND/ALS. This will be assessed as a change from baseline disease severity measured by the ALS Functional Rating Scale-Revised (ALSFRS-R) total score and survival. As this is an adaptive study design, an interim analysis will be performed at Week 24 by a team of unblinded statisticians for the potential to stop the study early for either success or futility.
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