{"id":388795,"date":"2023-12-19T12:15:00","date_gmt":"2023-12-19T17:15:00","guid":{"rendered":"https:\/\/platohealth.ai\/investors-still-arent-sold-on-uniqures-gene-therapy-for-huntingtons\/"},"modified":"2023-12-19T22:30:20","modified_gmt":"2023-12-20T03:30:20","slug":"investors-still-arent-sold-on-uniqures-gene-therapy-for-huntingtons","status":"publish","type":"post","link":"https:\/\/platohealth.ai\/investors-still-arent-sold-on-uniqures-gene-therapy-for-huntingtons\/","title":{"rendered":"Investors still aren\u2019t sold on UniQure\u2019s gene therapy for Huntington\u2019s","gt_translate_keys":[{"key":"rendered","format":"text"}]},"content":{"rendered":"<div><img decoding=\"async\" src=\"https:\/\/platohealth.ai\/wp-content\/uploads\/2023\/12\/investors-still-arent-sold-on-uniqures-gene-therapy-for-huntingtons.jpg\" class=\"ff-og-image-inserted\"><\/div>\n<h3>Dive Brief:<\/h3>\n<ul>\n<li>Shares of UniQure dipped more than 10% Tuesday morning after the biotechnology company released more data from a small study testing an experimental gene therapy for Huntington\u2019s disease.<\/li>\n<li>The new results include up to 30 months of follow-up from 39 patients enrolled in the study, which is evaluating two doses of UniQure\u2019s therapy, called AMT-130. The company said there\u2019s evidence both doses are generally safe and can preserve or improve neurological function in patients with the disease, which progressively impairs movement and cognition.<\/li>\n<li>While UniQure leadership describes the results seen so far as \u201cvery promising,\u201d analysts on Wall Street have found them puzzling. The low dose, for example, has appeared more effective and less variable than the high dose. How long either stays effective is also unclear. Nevertheless, UniQure intends to engage with regulators early next year to talk through the data it\u2019s collected and \u201cpotential strategies for ongoing development of AMT-130.\u201d<\/li>\n<\/ul>\n<h3>Dive Insight:<\/h3>\n<p>Huntington\u2019s is caused by mutations in the gene responsible for making the similarly named \u201chuntingtin\u201d protein. While helpful to brain function in its normal form, the protein, when mutated, bunches together into toxic clumps that damage nerve cells.<\/p>\n<p>AMT-130 is designed to silence that malfunctioning gene, and has become one of UniQure\u2019s most closely watched programs.<\/p>\n<p>In its update, UniQure said neurological function in patients given the high dose of AMT-130 was preserved or improved 18 months after treatment, compared to the start of the study. That was measured by a scoring system known in short as cUHDRS, which assesses the motor function, brain function and behavior of people with Hungtinton\u2019s.<\/p>\n<p>In the low dose arm, two other scales found neurological function was preserved at 30 months, according to UniQure.<\/p>\n<p>The company added that, across all three measurements, AMT-130 showed \u201cfavorable trends\u201d on the expected rate of decline compared to a natural history cohort. The cohort, which UniQure created with <a rel=\"noopener\" target=\"_blank\" href=\"https:\/\/chdifoundation.org\/\">assistance from a nonprofit research organization<\/a>, includes 31 patients with early Huntington\u2019s disease who were similar to the participants in the AMT-130 study. Drugmakers use natural history data to better understand how a disease progresses without intervention.<\/p>\n<p>Given the design of AMT-130, the study has been measuring the amount of mutant huntingtin present in the fluid surrounding the brain and spine.<\/p>\n<p>There, average changes \u201ccontinue to be variable,\u201d according to UniQure, which claims that, because AMT-130 is administered deep in the brain, such measurements \u201care not believed to be materially representative\u201d of mutant huntingtin levels in targeted brain regions. The company previously disclosed that mutant huntingtin levels varied across the studied two doses. In the low dose arm, they had declined an average of 8% two years after treatment, while in the high dose arm, they had increased an average of 40% one year after treatment.<\/p>\n<p>UniQure\u2019s study has also been charting levels of \u201cneurofilament light chain,\u201d a molecule that research suggests is indicative of brain and nervous system damage. It has become an increasingly important measure for drugmakers trying to prove their therapies can protect nerve cells. UniQure said Tuesday that, 30 months after treatment, average levels of the molecule had declined 6.6%. And for the high dose arm, they were \u201cnear baseline\u201d at month 18.<\/p>\n<p>UniQure said there have been four serious adverse events in the high dose arm that were deemed related to AMT-160. Three were central nervous system inflammation and one was severe headache later also attributed to central nervous system inflammation.<\/p>\n<p>UniQure said patients with symptoms of this inflammation improved after receiving glucocorticoid medication. Six patients in the high-dose arm received perioperative steroids when they got AMT-130 to reduce the risk of inflammation.<\/p>\n<p>UniQure shares were trading below $7 apiece late Tuesday morning.<\/p>\n<ul class=\"plato-post-bottom-links\">\n<li class=\"plato-post-bottom-link-amplifi\">SEO Powered Content &amp; PR Distribution. <a rel=\"noopener\" target=\"_blank\" href=\"https:\/\/www.amplifipr.com\">Get Amplified Today.<\/a><\/li>\n<li class=\"plato-post-bottom-link-platodata-network\">PlatoData.Network Vertical Generative Ai. Empower Yourself. <a rel=\"noopener\" target=\"_blank\" href=\"https:\/\/platodata.network\">Access Here.<\/a><\/li>\n<li class=\"plato-post-bottom-link-platoaistream\">PlatoAiStream. Web3 Intelligence. 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Biotech and Clinical Trials Intelligence. <a href=\"https:\/\/platohealth.ai\" target=\"_blank\" rel=\"noopener\">Access Here.<\/a><\/li>\n<li class=\"plato-post-bottom-link-source\"><span>Source:<\/span> <a rel=\"noopener\" target=\"_blank\" href=\"https:\/\/www.biopharmadive.com\/news\/uniqure-huntington-gene-therapy-amt-130-study-results\/702969\/\">https:\/\/www.biopharmadive.com\/news\/uniqure-huntington-gene-therapy-amt-130-study-results\/702969\/<\/a><\/li>\n<\/ul>\n","protected":false,"gt_translate_keys":[{"key":"rendered","format":"html"}]},"excerpt":{"rendered":"<p>Dive Brief: Shares of UniQure dipped more than 10% Tuesday morning after the biotechnology company released more data from a small study testing an experimental gene therapy for Huntington\u2019s disease. The new results include up to 30 months of follow-up from 39 patients enrolled in the study, which is evaluating two doses of UniQure\u2019s therapy, [&hellip;]<\/p>\n","protected":false,"gt_translate_keys":[{"key":"rendered","format":"html"}]},"author":2,"featured_media":388798,"comment_status":"closed","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"_acf_changed":false,"footnotes":""},"categories":[50],"tags":[],"acf":[],"gt_translate_keys":[{"key":"link","format":"url"}],"_links":{"self":[{"href":"https:\/\/platohealth.ai\/wp-json\/wp\/v2\/posts\/388795"}],"collection":[{"href":"https:\/\/platohealth.ai\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/platohealth.ai\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/platohealth.ai\/wp-json\/wp\/v2\/users\/2"}],"replies":[{"embeddable":true,"href":"https:\/\/platohealth.ai\/wp-json\/wp\/v2\/comments?post=388795"}],"version-history":[{"count":1,"href":"https:\/\/platohealth.ai\/wp-json\/wp\/v2\/posts\/388795\/revisions"}],"predecessor-version":[{"id":388797,"href":"https:\/\/platohealth.ai\/wp-json\/wp\/v2\/posts\/388795\/revisions\/388797"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/platohealth.ai\/wp-json\/wp\/v2\/media\/388798"}],"wp:attachment":[{"href":"https:\/\/platohealth.ai\/wp-json\/wp\/v2\/media?parent=388795"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/platohealth.ai\/wp-json\/wp\/v2\/categories?post=388795"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/platohealth.ai\/wp-json\/wp\/v2\/tags?post=388795"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}