{"id":7161,"date":"2024-03-14T03:48:09","date_gmt":"2024-03-14T03:48:09","guid":{"rendered":"https:\/\/economicherald.net\/?p=7161"},"modified":"2024-03-14T03:48:09","modified_gmt":"2024-03-14T03:48:09","slug":"pharmaust-files-groundbreaking-mnd-als-clinical-data-with-the-fda","status":"publish","type":"post","link":"https:\/\/economicherald.net\/?p=7161","title":{"rendered":"PharmAust files groundbreaking MND\/ALS clinical data with the FDA"},"content":{"rendered":"<p>Phase 1 MEND study outcomes for MND\/ALS treatment now with FDA for review in support of PharmAust\u2019s Orphan Drug Designation request<br \/>\nClinical data shows monepantel (MPL) has the potential to delay the progression of MND\/ALS by ~58%<br \/>\nPAA on track to begin pivotal registration Phase 2\/3 study in H1 CY24<\/p>\n<p>\u00a0<\/p>\n<p><strong><em>Special Report<\/em><\/strong><strong>: Biotech company PharmAust has submitted supplementary clinical data from its recently completed Phase 1 MEND Study with the US Food and Drug Administration (FDA) <\/strong><strong>Office of Orphan Products Development in support of its recent request for Orphan Drug Designation (ODD) for monepantel (MPL) <\/strong><strong>to treat motor neurone disease (MND) and amyotrophic lateral sclerosis (ALS).<\/strong><\/p>\n<p>Positive top-line data from the study \u2013 involving 12 patients with MND\/ALS and the daily administration of MPL over a 7-12 month period \u2013 showed the drug was well-tolerated and did not result in any dose-limiting toxicities or serious side effects.<\/p>\n<p>A <a href=\"https:\/\/stockhead.com.au\/health\/pharmausts-mpl-could-offer-mnd-als-sufferers-up-to-56-5-months-additional-life-expectancy\/\">58% reduction<\/a> in disease progression was reported from Phase 1.<\/p>\n<p><a href=\"https:\/\/stockhead.com.au\/company\/pharmaust-paa\/\"><strong>PharmAust (ASX:PAA)<\/strong><\/a> says the trial is showing a meaningful therapeutic effect with biomarkers demonstrating a large reduction in cerebrospinal fluid.<\/p>\n<p>Presently, sufferers have an average life expectancy of about 27 months, with current FDA-approved treatments Riluzole and Relyvrio only able to prolong life by about 2-3 months and 10 months respectively.<\/p>\n<p>MPL indicates that life expectancy could increase patients\u2019 lives by an additional 13.5-56.5 months.<\/p>\n<p>An updated request for Orphan Drug Designation (ODD) to the FDA has now been submitted, following a request for the Phase 1 data received in January 2024.<\/p>\n<p>\u00a0<\/p>\n<h2><strong>Next phase to begin in mid-2024<\/strong><\/h2>\n<p>PAA chief executive Dr Michael Thurn is very confident the FDA will grant monepantel an ODD for the treatment of MND\/ALS \u2013 which affects over 350,000 people globally \u2013 based on the strength of the Phase 1 MEND Study outcomes.<\/p>\n<p>\u201cReceiving ODD status from the FDA will come at an opportune time for PharmAust given the recent clinical trial failures of the 2 leading FDA drugs for MND\/ALS,\u201d he says.<\/p>\n<p>\u201cAt a time of need, monepantel offers much-needed hope for patients with this severely debilitating disease.<\/p>\n<p>\u201cThe company is in the process of finalising our exciting plans for its pivotal registration Phase 2\/3 study and is on track to commence the study during the middle of this year.\u201d<\/p>\n<p>It has recently been announced that two FDA-approved treatments for MND\/ALS \u2013 the Amylyx Pharmaceuticals drug RELYVRIO\u00ae and Ferrier International S.A. drug edaravone \u2013 produced unsuccessful Phase 3 clinical trial results and are at risk of being withdrawn from the market, creating an even greater need for new solutions for patients.<\/p>\n<p>\u00a0<\/p>\n\n<p>\u00a0<\/p>\n<p><em>This article was developed in collaboration with PharmAust, a Stockhead advertiser at the time of publishing.<\/em><\/p>\n<p><em>This article does not constitute financial product advice. You should consider obtaining independent advice before making any financial decisions.<\/em><\/p>\n<p><span class=\"et_bloom_bottom_trigger\"><\/span><\/p>\n<p>The post <a href=\"https:\/\/stockhead.com.au\/health\/pharmaust-files-groundbreaking-mnd-als-clinical-data-with-the-fda\/\">PharmAust files groundbreaking MND\/ALS clinical data with the FDA<\/a> appeared first on <a href=\"https:\/\/stockhead.com.au\/\">Stockhead<\/a>.<\/p>","protected":false},"excerpt":{"rendered":"<p>Phase 1 MEND study outcomes for MND\/ALS treatment now with FDA for review in support of PharmAust\u2019s Orphan Drug Designation request Clinical data shows monepantel <a href=\"https:\/\/economicherald.net\/?p=7161\" class=\"read-more-link\">[more&#8230;]<\/a><\/p>\n","protected":false},"author":0,"featured_media":7162,"comment_status":"","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[4],"tags":[],"class_list":["post-7161","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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