{"id":2517,"date":"2023-12-01T02:19:39","date_gmt":"2023-12-01T02:19:39","guid":{"rendered":"https:\/\/economicherald.net\/?p=2517"},"modified":"2023-12-01T02:19:39","modified_gmt":"2023-12-01T02:19:39","slug":"pharmaust-completes-phase-1-motor-neurone-disease-trial-applies-for-orphan-drug-designation","status":"publish","type":"post","link":"https:\/\/economicherald.net\/?p=2517","title":{"rendered":"PharmAust completes Phase 1 motor neurone disease trial, applies for Orphan Drug Designation"},"content":{"rendered":"<p>PharmAust completes Phase 1 MEND study for MPL in patients with MND\/ALS<br \/>\nTop line results on track for release in Q1 2024<br \/>\nCompany has applied for Orphan Drug Designation with the US FDA<\/p>\n<p>\u00a0<\/p>\n<p><strong><em>Special Report:<\/em> Excitement is brewing at biotech company PharmAust after it wrapped up the Phase 1 MEND study of the drug monepantel (MPL) in patients with motor neurone disease (MND\/ALS).<\/strong><\/p>\n<p>The company is on track to release top line results from the study in Q1 2024 and has applied for an <a href=\"https:\/\/stockhead.com.au\/health\/pharmaust-eyes-us-orphan-drug-designation-after-successful-phase-1-motor-neurone-disease-trial\/\"><strong>Orphan Drug Designation (ODD)<\/strong>\u00a0<\/a>with the US Food and Drug Administration (FDA), which incentivises the development of drugs or biological products to treat rare diseases or conditions by offering market exclusivity for several years and tax credits.<\/p>\n<p><a href=\"https:\/\/stockhead.com.au\/company\/pharmaust-paa\/\"><strong>PharmAust\u2019s (ASX:PAA)<\/strong><\/a> Phase 1 MEND study is an open-label, multicentre study involving 12 patients with MND\/ALS \u2013 a nervous system disease that weakens muscles and impacts physical function.<\/p>\n<p>It affects over 350,000 people globally and is invariably fatal with the average life expectancy of someone with MND\/ALS being around 27 months.<\/p>\n<p>The study design involves two cohorts of six patients, each progressively receiving higher dose levels of MPL \u2014 which could reduce the rate of degeneration and loss of motor neurons in the brainstem\u2019s anterior horns and motor nuclei \u2014 in a staggered design approach over time.<\/p>\n<p>Some patients have been treated for more than 13 months and none have withdrawn from the study with no deaths or significant adverse events to date.<\/p>\n<p>Promisingly, the company <a href=\"https:\/\/stockhead.com.au\/health\/results-from-11-out-of-12-patients-in-pharmaust-trial-point-to-stabilised-mnd-progression\/\"><strong>noted in August<\/strong><\/a> that only one of the 12 patients showed an increase in Neurofilament Light Chain (Nfl) protein concentrations in their plasma.<\/p>\n<p>This is significant as research suggests a correlation between Nfl increases and MND progression.<\/p>\n<p>Success is likely to be hugely lucrative as the total MND\/ALS addressable market is US$3.6bn per annum. Riluzole \u2013 a treatment that prolongs life on average by\u00a0just 2-3 months \u2013 has already achieved annual peak sales of ~US$1bn.<\/p>\n<p>\u00a0<\/p>\n<h2><strong>\u201cExcitement is growing\u201d<\/strong><\/h2>\n<p>With the completion of the Phase 1 MEND study, PAA notes that all patients, who are continuing to receive MPL under a compassionate use program, remain able to swallow and breathe unassisted.<\/p>\n<p>All patients have also be invited to move to a 12-month open-label trial.<\/p>\n<p>\u201cExcitement is growing knowing that we are only months away from releasing top-line data,\u201d chief executive officer Dr Michael Thurn said.<\/p>\n<p>\u201cWe have several patients who have now received treatment with monepantel for 13 months or more, with the median treatment duration being 10.6 months for the 12 patients who participated in the study.<\/p>\n<p>\u201cEvery month a patient continues on treatment brings enormous satisfaction and excitement around the potential benefit that monepantel may bring to patients, their families and caregivers, and of course the healthcare system as a whole.\u201d<\/p>\n<p>What\u2019s of particular note is that the patients who were diagnosed 24 months before the trial have now survived for 34 months.<\/p>\n<p>\u201cThis is longer than the \u201caverage survival\u201d of 27 months and nearly matches the current survival-leading drug, which extends life by 9 months (36 months average survival),\u201d Thurn told <em>Stockhead<\/em>.<\/p>\n<p>\u00a0<\/p>\n\n<p><span class=\"mce_SELRES_start\">\ufeff<\/span>\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>\u00a0<\/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-completes-phase-1-motor-neurone-disease-trial-applies-for-orphan-drug-designation\/\">PharmAust completes Phase 1 motor neurone disease trial, applies for Orphan Drug Designation<\/a> appeared first on <a href=\"https:\/\/stockhead.com.au\/\">Stockhead<\/a>.<\/p>","protected":false},"excerpt":{"rendered":"<p>PharmAust completes Phase 1 MEND study for MPL in patients with MND\/ALS Top line results on track for release in Q1 2024 Company has applied <a href=\"https:\/\/economicherald.net\/?p=2517\" class=\"read-more-link\">[more&#8230;]<\/a><\/p>\n","protected":false},"author":0,"featured_media":2518,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[4],"tags":[],"class_list":["post-2517","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-finance"],"yoast_head":"<!-- This site is optimized with the Yoast SEO plugin v27.5 - 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