{"id":5941,"date":"2024-02-16T02:28:12","date_gmt":"2024-02-16T02:28:12","guid":{"rendered":"https:\/\/economicherald.net\/?p=5941"},"modified":"2024-02-16T02:28:12","modified_gmt":"2024-02-16T02:28:12","slug":"fda-shows-the-way-for-accelerated-approval-of-pharmausts-monepantel-in-mnd","status":"publish","type":"post","link":"https:\/\/economicherald.net\/?p=5941","title":{"rendered":"FDA shows the way for accelerated approval of PharmAust\u2019s monepantel in MND"},"content":{"rendered":"<p>PharmAust successfully completes a Pre-IND meeting with the US FDA<br \/>\nPath outlined to potentially receive accelerated and full approval of monepantel for treatment of MND\/ALS<br \/>\nPharmAust to initiate requirements requested by FDA in preparation to open IND application for its adaptive Phase 2\/3 clinical study<\/p>\n<p>\u00a0<\/p>\n<p>Special Report: PharmAust has successfully completed a Pre-IND meeting with the US Food and Drug Administration (FDA) with a path outlined to potentially receive accelerated and full approval of its lead drug monepantel for the treatment of motor neurone disease\/amyotrophic lateral sclerosis (MND\/ALS).\u00a0<\/p>\n<p><span>Clinical-stage biotech\u00a0<\/span><a href=\"https:\/\/stockhead.com.au\/company\/pharmaust-paa\/\">PharmAust (ASX:PAA)<\/a><span>\u00a0says\u00a0the US pre-IND meeting marks a significant milestone as the company gears up for an adaptive Phase 2\/3 clinical study in Q2 2024.\u00a0<\/span><\/p>\n<p><span>PAA aims to use this study as the primary support for accelerated and\/or full approval of monepantel for MND\/ALS treatment.<\/span><\/p>\n<p><span>The pre-IND meeting focused on establishing formal communication with the FDA, confirming details and ensuring the acceptability of PAA\u2019s proposed development program, including requirements for non-clinical and clinical pharmacology, clinical chemistry and manufacturing controls.\u00a0<\/span><\/p>\n<p><span>Seeking specific guidance on the design and adequacy of the planned adaptive Phase II\/III clinical study, PAA received FDA confirmation that accelerated and\/or full approval could potentially be granted based on substantial evidence of effectiveness and a robust safety database.\u00a0<\/span><\/p>\n<p><span>Crucially, the FDA says there are no set minimum requirements for the number of patients or study sites in the US, opening the door for the adaptive Phase 2\/3 clinical study to be global and enabling clinical sites and patients from Europe and Australia, reducing recruitment timelines.\u00a0<\/span><\/p>\n<p><span>Following the completion of the adaptive study, PAA intends to seek approvals from the European Medicines Agency (EMA) and Australia\u2019s Therapeutic Goods Administration (TGA).<\/span><\/p>\n<p>\u00a0<\/p>\n<h2>Study to evaluate safety and efficacy<\/h2>\n<p><span>PAA says the Phase 2\/3 clinical study will be a multicentre, randomised, placebo-controlled, adaptive clinical study evaluating the safety and efficacy of monepantel in patients with MND\/ALS over 48 weeks.\u00a0<\/span><\/p>\n<p><span>The primary aim of the study will be to assess the efficacy of monepantel compared to placebo on the progression of MND\/ALS.\u00a0<\/span><\/p>\n<p><span>This drug\u2019s efficacy will be evaluated as a change from baseline disease severity measured by the ALS Functional Rating Scale-Revised (ALSFRS-R) total score and survival.\u00a0<\/span><\/p>\n<p><span>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.<\/span><\/p>\n<p>\u00a0<\/p>\n<h2>OLE trial underway as analysis shows drug benefit<\/h2>\n<p><span>It\u2019s been a big week for PAA, which also announced the first patient has been dosed in its 12-month <\/span><a href=\"https:\/\/stockhead.com.au\/health\/pharmaust-extension-study-begins-as-analysis-shows-mnd-als-drug-defied-the-odds\/\"><span>open-label extension (OLE) study<\/span><\/a><span> for patients with MND\/ALS.\u00a0<\/span><\/p>\n<p><span>All 12 patients from the Phase 1 MEND Study are expected to be enrolled in the OLE after meeting the minimum requirement of being able to swallow.<\/span><\/p>\n<p><span>The company also unveiled significant findings from a statistical review indicating a notable survival benefit for MND\/ALS patients receiving monepantel.\u00a0<\/span><\/p>\n<p>\u00a0<\/p>\n<h2>\u2018Highly encouraged\u2019 by positive FDA feedback<\/h2>\n<p><span>PAA chief executive Dr Michael Thurn says the company is \u201chighly encouraged\u201d by the positive feedback from the FDA.<\/span><\/p>\n<p><span>\u201cIt aligns with our expectations and provides the company with a clear understanding of the requirements to potentially receive accelerated and\/or full approval of monepantel for treating MND\/ALS,\u201d he says.<\/span><\/p>\n<p><span>\u201cThis advice truly positions us as a global play following the successful completion of our planned adaptive Phase 2\/3 clinical study.\u201d<\/span><\/p>\n<p>\u00a0<\/p>\n\n<p><span class=\"mce_SELRES_start\">\ufeff<\/span><span>\u00a0<\/span><\/p>\n<p><span>This article was developed in collaboration with PharmAust, a Stockhead advertiser at the time of publishing.\u00a0<\/span><\/p>\n<p>\u00a0<\/p>\n<p><span>This article does not constitute financial product advice. You should consider obtaining independent advice before making any financial decisions.<\/span><\/p>\n<p><span class=\"et_bloom_bottom_trigger\"><\/span><\/p>\n<p>The post <a href=\"https:\/\/stockhead.com.au\/health\/highly-encouraged-pharmaust-completes-pre-ind-meeting-with-us-fda\/\">FDA shows the way for accelerated approval of PharmAust\u2019s monepantel in MND<\/a> appeared first on <a href=\"https:\/\/stockhead.com.au\/\">Stockhead<\/a>.<\/p>","protected":false},"excerpt":{"rendered":"<p>PharmAust successfully completes a Pre-IND meeting with the US FDA Path outlined to potentially receive accelerated and full approval of monepantel for treatment of MND\/ALS <a href=\"https:\/\/economicherald.net\/?p=5941\" class=\"read-more-link\">[more&#8230;]<\/a><\/p>\n","protected":false},"author":0,"featured_media":5942,"comment_status":"","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[4],"tags":[],"class_list":["post-5941","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-finance"],"yoast_head":"<!-- This site is optimized with the Yoast SEO plugin v27.4 - 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