{"id":17215,"date":"2020-05-11T09:47:00","date_gmt":"2020-05-11T07:47:00","guid":{"rendered":"https:\/\/www.hollandbio.nl\/?p=17215"},"modified":"2020-05-19T09:49:40","modified_gmt":"2020-05-19T07:49:40","slug":"myokardia-announces-positive-phase-3-results","status":"publish","type":"post","link":"https:\/\/www.hollandbio.nl\/en\/nieuws\/myokardia-announces-positive-phase-3-results\/","title":{"rendered":"MyoKardia announces positive Phase 3 results"},"content":{"rendered":"\n<p class=\"wp-block-paragraph\">MyoKardia has announced positive topline data from the company\u2019s\nPhase 3 pivotal EXPLORER-HCM clinical trial of mavacamten for the treatment of\npatients with symptomatic, obstructive hypertrophic cardiomyopathy (HCM) (<a href=\"http:\/\/clinicaltrials.gov\/\">clinicaltrials.gov<\/a>&nbsp;NCT03470545).\n&nbsp;Mavacamten demonstrated a robust treatment effect: the primary and all\nsecondary endpoints of the EXPLORER trial were met with statistical\nsignificance (p\u22640.0006 for all endpoints). Mavacamten was well tolerated, and\nmeaningful improvements in symptoms, functional status and quality of life, as\nwell as reduction or elimination in obstruction of the left ventricle, were\nobserved among patients on treatment versus placebo.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u201cThe extraordinary data from the EXPLORER pivotal trial confirm\nmavacamten\u2019s ability to relieve dynamic outflow obstruction, control symptoms\nand improve quality of life in patients&nbsp;with hypertrophic\ncardiomyopathy,&#8221;&nbsp;said&nbsp;Iacopo\nOlivotto, M.D.,&nbsp;Careggi\nUniversity Hospital&nbsp;and lead clinical investigator for the\nEXPLORER-HCM clinical trial. &#8220;HCM is the most common inherited\ncardiovascular disease, and patients face an uncertain journey that all too frequently\nincludes debilitating symptoms, as well as serious complications, such as heart\nfailure, stroke and cardiac arrest.&nbsp; Mavacamten is the first drug\ndeveloped to target the specific molecular defect of the disease.\n&nbsp;EXPLORER represents a major achievement toward a precision-medicine\napproach in cardiomyopathies and should provide great hope to a community\npainfully aware of the lack of disease-specific treatment options.\u201d<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">The 30-week treatment with mavacamten resulted in a highly\nstatistically significant outcome relative to placebo (<em>p<\/em>=0.0005) for the\nprimary endpoint in the EXPLORER-HCM trial, a composite functional analysis\ndesigned to capture the treatment effect of mavacamten relative to placebo on\nboth symptoms and cardiac function.&nbsp;<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">All secondary endpoints also demonstrated statistically\nsignificant and clinically meaningful improvements for mavacamten as compared\nto placebo.&nbsp; Secondary endpoints in the EXPLORER-HCM trial evaluated\nimprovements in post-exercise left ventricular outflow tract (LVOT) peak\ngradient (p&lt;0.0001),&nbsp;New York\nHeart Association&nbsp;(NYHA) functional classification (p&lt;0.0001),\npeak VO<sub>2<\/sub>&nbsp;(p=0.0006),\nthe Kansas City Cardiomyopathy Clinical Summary Score (KCCQ-CSS) (p&lt;0.0001),\nand the HCM Symptom Questionnaire Shortness of Breath Domain Score\n(p&lt;0.0001).<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Mavacamten was well tolerated and demonstrated safety results\ncomparable to placebo, with no new findings observed. Ninety-eight percent of\npatients enrolled completed the study.&nbsp; Of the two percent who dropped\nout, none were due to reduced ejection fraction or symptoms of heart failure.\n&nbsp;Overall rates of adverse events, serious adverse events, and cardiac\nadverse events, including atrial fibrillation, were comparable for patients\ntreated with mavacamten and placebo.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u201cThe resoundingly positive data from EXPLORER bring us a\nsignificant step closer to improving the lives of people with serious\ncardiovascular conditions, starting with HCM, a debilitating disease estimated\nto affect one in every 500 people,\u201d said&nbsp;Tassos\nGianakakos, Chief Executive Officer of MyoKardia.&nbsp; \u201cThe activity\nand tolerability profile observed for mavacamten in this pivotal study\nunderscores the profound impact and potential for therapeutics that target the\nunderlying biology of disease.&nbsp; We look forward to the submission of\nMyoKardia\u2019s first New Drug Application and, importantly, to serving the many\npatients that stand to benefit from mavacamten.\u201d&nbsp;<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">The EXPLORER-HCM clinical trial is part of MyoKardia\u2019s pivotal program studying mavacamten as a treatment for symptomatic, obstructive hypertrophic cardiomyopathy.\u00a0\u00a0MyoKardia\u00a0plans to submit a New Drug Application to the\u00a0U.S. Food and Drug Administration\u00a0(FDA) in the first quarter of 2021. Results from the Phase 3 EXPLORER-HCM clinical trial will be submitted to a future professional meeting in 2020.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Source: <strong>M<a href=\"https:\/\/myokardia.gcs-web.com\/news-releases\/news-release-details\/myokardia-announces-primary-and-all-secondary-endpoints-met\">yocardia (press release)<\/a><\/strong><\/p>\n","protected":false},"excerpt":{"rendered":"<p>MyoKardia has announced positive topline data from the company\u2019s Phase 3 pivotal EXPLORER-HCM clinical trial of mavacamten for the treatment of patients with symptomatic, obstructive hypertrophic cardiomyopathy (HCM) (clinicaltrials.gov&nbsp;NCT03470545). &nbsp;Mavacamten demonstrated a robust treatment effect: the primary and all secondary endpoints of the EXPLORER trial were met with statistical significance (p\u22640.0006 for all endpoints). Mavacamten [&hellip;]<\/p>\n","protected":false},"author":6,"featured_media":0,"comment_status":"closed","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"_acf_changed":false,"footnotes":""},"categories":[39],"tags":[],"class_list":["post-17215","post","type-post","status-publish","format-standard","hentry","category-gezondheid"],"acf":[],"yoast_head":"<!-- This site is optimized with the Yoast SEO plugin v28.0 - https:\/\/yoast.com\/product\/yoast-seo-wordpress\/ -->\n<title>MyoKardia announces positive Phase 3 results - hollandbio<\/title>\n<meta name=\"robots\" content=\"index, follow, max-snippet:-1, max-image-preview:large, max-video-preview:-1\" \/>\n<link rel=\"canonical\" href=\"https:\/\/www.hollandbio.nl\/en\/nieuws\/myokardia-announces-positive-phase-3-results\/\" \/>\n<meta property=\"og:locale\" content=\"en_US\" \/>\n<meta property=\"og:type\" content=\"article\" \/>\n<meta property=\"og:title\" content=\"MyoKardia announces positive Phase 3 results - hollandbio\" \/>\n<meta property=\"og:description\" content=\"MyoKardia has announced positive topline data from the company\u2019s Phase 3 pivotal EXPLORER-HCM clinical trial of mavacamten for the treatment of patients with symptomatic, obstructive hypertrophic cardiomyopathy (HCM) (clinicaltrials.gov&nbsp;NCT03470545). &nbsp;Mavacamten demonstrated a robust treatment effect: the primary and all secondary endpoints of the EXPLORER trial were met with statistical significance (p\u22640.0006 for all endpoints). 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