Harmony Biosciences’ Genetic Disorder Drug Disappoints In Phase 3 Study <\/p>\n
Harmony Biosciences Holdings, Inc. (NASDAQ:HRMY<\/a>) released topline results from its Phase 3 registrational clinical trial (the RECONNECT Study) of ZYN002 in Fragile X syndrome (FXS)<\/a>.<\/p>\n Fragile X syndrome is a rare genetic disorder that is the leading known cause of both inherited intellectual disability and autism spectrum disorder.<\/p>\n The disorder negatively affects synaptic function, plasticity and neuronal connections, and results in a spectrum of intellectual disabilities and behavioral symptoms, such as social avoidance and irritability.<\/p>\n Also Read: Harmony Biosciences Stock Falls On FDA Setback For Pitolisant In Sleeping Disorder<\/a><\/p>\n There is a significant unmet medical need in patients living with FXS as there are currently no FDA-approved treatments for this disorder.<\/p>\n The RECONNECT Study did not meet the primary endpoint of improvement in social avoidance primarily due to a higher-than-expected placebo response rate<\/a>.<\/p>\n Kumar Budur, Chief Medical and Scientific Officer at Harmony Biosciences, said, \u201cWe will conduct a comprehensive analysis of the full dataset to better understand the results…\u201d<\/p>\n The RECONNECT Study was a Phase 3 randomized, double-blind, placebo-controlled, multiple-center study, to assess the efficacy and safety of ZYN002, a pharmaceutically manufactured cannabidiol administered as a transdermal gel to patients with FXS ages 3 to under 30 years old.<\/p>\n \u201cWe have a late-stage, catalyst-rich pipeline with multiple Phase 3 programs in the clinic and continue to be on track to initiate our Phase 3 trials for pitolisant HD in narcolepsy and idiopathic hypersomnia in the fourth quarter of this year. Harmony\u2019s unique profile as a profitable self-funding biotech company with strong cash-generation positions us well to drive future growth,\u201d said Jeffrey Dayno, President and CEO of Harmony Biosciences.<\/p>\n Price Action: HRMY stock was trading lower by 14.55% to $27.41\u00a0at last check Wednesday.<\/p>\n Read Next:<\/p>\n Photo via Shutterstock<\/p>\n Up Next: Transform your trading with Benzinga Edge’s one-of-a-kind market trade ideas and tools. Click now to access unique insights<\/a> that can set you ahead in today’s competitive market.<\/p>\n Get the latest stock analysis from Benzinga?<\/p>\n This article Harmony Biosciences’ Genetic Disorder Drug Disappoints In Phase 3 Study<\/a> originally appeared on Benzinga.com<\/a><\/p>\n \u00a9 2025 Benzinga.com. Benzinga does not provide investment advice. All rights reserved.<\/p>\n","protected":false},"excerpt":{"rendered":"Harmony Biosciences’ Genetic Disorder Drug Disappoints In Phase 3 Study Harmony Biosciences Holdings, Inc. (NASDAQ:HRMY) released topline results…\n","protected":false},"author":2,"featured_media":166734,"comment_status":"","ping_status":"","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[25],"tags":[12330,49,48,85292,14432,316,85975,66,85976],"class_list":{"0":"post-166733","1":"post","2":"type-post","3":"status-publish","4":"format-standard","5":"has-post-thumbnail","7":"category-genetics","8":"tag-autism-spectrum-disorder","9":"tag-ca","10":"tag-canada","11":"tag-fragile-x-syndrome","12":"tag-genetic-disorder","13":"tag-genetics","14":"tag-harmony-biosciences","15":"tag-science","16":"tag-topline-results"},"_links":{"self":[{"href":"https:\/\/www.newsbeep.com\/ca\/wp-json\/wp\/v2\/posts\/166733","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/www.newsbeep.com\/ca\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/www.newsbeep.com\/ca\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/www.newsbeep.com\/ca\/wp-json\/wp\/v2\/users\/2"}],"replies":[{"embeddable":true,"href":"https:\/\/www.newsbeep.com\/ca\/wp-json\/wp\/v2\/comments?post=166733"}],"version-history":[{"count":0,"href":"https:\/\/www.newsbeep.com\/ca\/wp-json\/wp\/v2\/posts\/166733\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/www.newsbeep.com\/ca\/wp-json\/wp\/v2\/media\/166734"}],"wp:attachment":[{"href":"https:\/\/www.newsbeep.com\/ca\/wp-json\/wp\/v2\/media?parent=166733"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/www.newsbeep.com\/ca\/wp-json\/wp\/v2\/categories?post=166733"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/www.newsbeep.com\/ca\/wp-json\/wp\/v2\/tags?post=166733"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}