{"id":40991,"date":"2025-12-08T21:22:00","date_gmt":"2025-12-08T14:22:00","guid":{"rendered":"https:\/\/thaipropertynews.com\/feeds\/?p=40991"},"modified":"2025-12-08T21:22:00","modified_gmt":"2025-12-08T14:22:00","slug":"halia-therapeutics-announces-positive-phase-2a-data-for-ofirnoflast-in-lower-risk-mds-at-ash-2025","status":"publish","type":"post","link":"https:\/\/thaipropertynews.com\/feeds\/?p=40991","title":{"rendered":"Halia Therapeutics Announces Positive Phase 2a Data for Ofirnoflast in Lower-Risk MDS at ASH 2025"},"content":{"rendered":"<table border=\"0\" cellspacing=\"10\" cellpadding=\"5\" align=\"right\">\n<tbody>\n<tr>\n<td><img decoding=\"async\" src=\"https:\/\/mma.prnasia.com\/media2\/2702932\/Halia_Therapeutics_Logo.jpg?p=medium600\" border=\"0\" alt=\"\" title=\"logo\" hspace=\"0\" vspace=\"0\" width=\"118\" \/><\/td>\n<\/tr>\n<\/tbody>\n<\/table>\n<p><i>\u2013 New findings demonstrate a 72% HI-E response rate at Week 16 with meaningful hemoglobin improvement \u2013<br \/>\u2013 Strong activity observed across ESA-refractory and ESA-intolerant patients, and across mutation and morphology subtypes \u2013<br \/>\u2013 Favorable safety profile with no treatment-related serious adverse events \u2013<\/i><\/p>\n<p><span class=\"legendSpanClass\"><span class=\"xn-location\">LEHI, Utah<\/span><\/span>, <span class=\"legendSpanClass\"><span class=\"xn-chron\">Dec. 8, 2025<\/span><\/span> \/PRNewswire\/ &#8212; Halia Therapeutics, a clinical-stage biopharmaceutical company, today presented new clinical data from its Phase 2a study of ofirnoflast (HT-6184) at the 67th American Society of Hematology (ASH) Annual Meeting. The data show that ofirnoflast, a first-in-class oral allosteric NEK7 inhibitor, induces clinically meaningful and sustained hematologic responses in patients with lower-risk myelodysplastic syndromes (MDS) and symptomatic anemia.<\/p>\n<p>In the Stage 1 efficacy population (N=18), ofirnoflast achieved a 72% hematologic improvement-erythroid (HI-E) response rate following \u226516 weeks of therapy. Consistent improvements were observed across WHO morphologic subtypes and somatic mutation categories, supporting a broad and biology-driven mechanism of action.<\/p>\n<p><b>Key Stage 1 Findings:<\/b><\/p>\n<ul type=\"disc\">\n<li>72% of patients (13\/18) achieved HI-E at Week 16, with responders showing a\u00a0median\u00a0hemoglobin increase of 3.5 g\/dL.<\/li>\n<li>Strong activity in difficult-to-treat patients, including 91% HI-E in ESA-refractory and 75% HI-E in ESA-intolerant subjects.<\/li>\n<li>Consistent responses across disease biology, with HI-E observed across transfusion burden categories, WHO morphologic subtypes, and major mutation groups (SF3B1, TET2, DNMT3A, ASXL1, TP53).<\/li>\n<li>Favorable safety profile, with no treatment-related SAEs, no Grade \u22653 related AEs, and no evidence of treatment-emergent myelosuppression.<\/li>\n<\/ul>\n<p>These findings reinforce NEK7 inhibition as a promising strategy to address the underlying inflammatory dysregulation central to ineffective hematopoiesis in MDS.<\/p>\n<p>&#8220;These data highlight the potential of ofirnoflast to meaningfully improve outcomes for patients with lower-risk MDS,&#8221; said <span class=\"xn-person\">David Bearss<\/span>, Ph.D., CEO of Halia Therapeutics. &#8220;Achieving a 72% HI-E response rate, including strong performance in refractory and intolerant patients alongside a clean safety profile, underscores the therapeutic promise of NEK7 inhibition. We look forward to building on these results as we advance the program toward later-stage development.&#8221;<\/p>\n<p><b>Next Steps<\/b><\/p>\n<p>Following the FDA Orphan Drug Designation granted in <span class=\"xn-chron\">October 2025<\/span>, Halia is currently communicating next steps with the FDA. Halia is finalizing the dataset and preparing to initiate a global Phase 3 pivotal trial in early 2026.<\/p>\n<p><b>American Society of Hematology (ASH) Poster Details:<\/b><\/p>\n<p>Title: &#8220;The Novel Allosteric NEK7 Inhibitor Ofirnoflast (HT-6184) Demonstrates Robust and Sustained Hematologic Response in Subjects with IPSS-R Very Low, Low or Intermediate Risk Myelodysplastic Syndrome (MDS) and Symptomatic Anemia&#8221;<\/p>\n<p>Time: <span class=\"xn-chron\">Monday, December 8, 2025<\/span>; <span class=\"xn-chron\">6:00 P.M. &#8211; 8:00 P.M. EST<\/span><\/p>\n<p><b>About Halia&#8217;s Phase 2 Trial of Ofirnoflast in Lower-Risk MDS<\/b><\/p>\n<p>HT-6184-MDS-001 is a Simon&#8217;s two-stage, multicenter study evaluating hematologic improvement after 16 weeks of treatment, with an extension phase for responders and molecularly improving non-responders. Key study objectives include evaluating efficacy through hematological improvement, clonal suppression, and VAF reduction, assessing safety and patient tolerance, monitoring changes in inflammasome-related biomarkers, and measuring quality of life using patient-reported outcome tools.<\/p>\n<p><b>About Halia Therapeutics<\/b><\/p>\n<p>Halia Therapeutics is a biotechnology company developing first-in-class inflammasome inhibitors. We target the root causes of inflammation-driven diseases to create transformative therapies. For more information, visit <a href=\"https:\/\/edge.prnewswire.com\/c\/link\/?t=0&amp;l=en&amp;o=4574573-1&amp;h=1011751352&amp;u=http%3A%2F%2Fwww.haliatx.com%2F&amp;a=www.haliatx.com\" target=\"_blank\" rel=\"nofollow\">www.haliatx.com<\/a>.<\/p>\n<p><b>Media Contact<\/b><\/p>\n<p>Taylor Avei\u00a0<br \/>Director of Business Development <br \/>Halia Therapeutics<br \/>+1 (385) 355-4315<br \/><a href=\"mailto:info@haliatx.com\" target=\"_blank\" rel=\"nofollow\">info@haliatx.com<\/a><\/p>\n<p><b>Investor Contact<\/b><\/p>\n<p>Leigh Salvo\u00a0<br \/>New Street Investor Relations<br \/><a href=\"mailto:leigh@newstreetir.com\" target=\"_blank\" rel=\"nofollow\">leigh@newstreetir.com<\/a><\/p>\n<p>\u00a0<\/p>","protected":false},"excerpt":{"rendered":"<p><!-- wp:html --><\/p>\n<table border=\"0\" cellspacing=\"10\" cellpadding=\"5\" align=\"right\">\n<tbody>\n<tr>\n<td><img decoding=\"async\" src=\"https:\/\/mma.prnasia.com\/media2\/2702932\/Halia_Therapeutics_Logo.jpg?p=medium600\" border=\"0\" alt=\"\" title=\"logo\" hspace=\"0\" vspace=\"0\" width=\"118\" \/><\/td>\n<\/tr>\n<\/tbody>\n<\/table>\n<p><i>\u2013 New findings demonstrate a 72% HI-E response rate at Week 16 with meaningful hemoglobin improvement \u2013<br \/>\u2013 Strong activity observed across ESA-refractory and ESA-intolerant patients, and across mutation and morphology subtypes \u2013<br \/>\u2013 Favorable safety profile with no treatment-related serious adverse events \u2013<\/i><\/p>\n<p><span class=\"legendSpanClass\"><span class=\"xn-location\">LEHI, Utah<\/span><\/span>, <span class=\"legendSpanClass\"><span class=\"xn-chron\">Dec. 8, 2025<\/span><\/span> \/PRNewswire\/ &#8212; Halia Therapeutics, a clinical-stage biopharmaceutical company, today presented new clinical data from its Phase 2a study of ofirnoflast (HT-6184) at the 67th American Society of Hematology (ASH) Annual Meeting. The data show that ofirnoflast, a first-in-class oral allosteric NEK7 inhibitor, induces clinically meaningful and sustained hematologic responses in patients with lower-risk myelodysplastic syndromes (MDS) and symptomatic anemia.<\/p>\n<p>In the Stage 1 efficacy population (N=18), ofirnoflast achieved a 72% hematologic improvement-erythroid (HI-E) response rate following \u226516 weeks of therapy. Consistent improvements were observed across WHO morphologic subtypes and somatic mutation categories, supporting a broad and biology-driven mechanism of action.<\/p>\n<p><b>Key Stage 1 Findings:<\/b><\/p>\n<ul type=\"disc\">\n<li>72% of patients (13\/18) achieved HI-E at Week 16, with responders showing a\u00a0median\u00a0hemoglobin increase of 3.5 g\/dL.<\/li>\n<li>Strong activity in difficult-to-treat patients, including 91% HI-E in ESA-refractory and 75% HI-E in ESA-intolerant subjects.<\/li>\n<li>Consistent responses across disease biology, with HI-E observed across transfusion burden categories, WHO morphologic subtypes, and major mutation groups (SF3B1, TET2, DNMT3A, ASXL1, TP53).<\/li>\n<li>Favorable safety profile, with no treatment-related SAEs, no Grade \u22653 related AEs, and no evidence of treatment-emergent myelosuppression.<\/li>\n<\/ul>\n<p>These findings reinforce NEK7 inhibition as a promising strategy to address the underlying inflammatory dysregulation central to ineffective hematopoiesis in MDS.<\/p>\n<p>&#8220;These data highlight the potential of ofirnoflast to meaningfully improve outcomes for patients with lower-risk MDS,&#8221; said <span class=\"xn-person\">David Bearss<\/span>, Ph.D., CEO of Halia Therapeutics. &#8220;Achieving a 72% HI-E response rate, including strong performance in refractory and intolerant patients alongside a clean safety profile, underscores the therapeutic promise of NEK7 inhibition. We look forward to building on these results as we advance the program toward later-stage development.&#8221;<\/p>\n<p><b>Next Steps<\/b><\/p>\n<p>Following the FDA Orphan Drug Designation granted in <span class=\"xn-chron\">October 2025<\/span>, Halia is currently communicating next steps with the FDA. Halia is finalizing the dataset and preparing to initiate a global Phase 3 pivotal trial in early 2026.<\/p>\n<p><b>American Society of Hematology (ASH) Poster Details:<\/b><\/p>\n<p>Title: &#8220;The Novel Allosteric NEK7 Inhibitor Ofirnoflast (HT-6184) Demonstrates Robust and Sustained Hematologic Response in Subjects with IPSS-R Very Low, Low or Intermediate Risk Myelodysplastic Syndrome (MDS) and Symptomatic Anemia&#8221;<\/p>\n<p>Time: <span class=\"xn-chron\">Monday, December 8, 2025<\/span>; <span class=\"xn-chron\">6:00 P.M. &#8211; 8:00 P.M. EST<\/span><\/p>\n<p><b>About Halia&#8217;s Phase 2 Trial of Ofirnoflast in Lower-Risk MDS<\/b><\/p>\n<p>HT-6184-MDS-001 is a Simon&#8217;s two-stage, multicenter study evaluating hematologic improvement after 16 weeks of treatment, with an extension phase for responders and molecularly improving non-responders. Key study objectives include evaluating efficacy through hematological improvement, clonal suppression, and VAF reduction, assessing safety and patient tolerance, monitoring changes in inflammasome-related biomarkers, and measuring quality of life using patient-reported outcome tools.<\/p>\n<p><b>About Halia Therapeutics<\/b><\/p>\n<p>Halia Therapeutics is a biotechnology company developing first-in-class inflammasome inhibitors. We target the root causes of inflammation-driven diseases to create transformative therapies. For more information, visit <a href=\"https:\/\/edge.prnewswire.com\/c\/link\/?t=0&amp;l=en&amp;o=4574573-1&amp;h=1011751352&amp;u=http%3A%2F%2Fwww.haliatx.com%2F&amp;a=www.haliatx.com\" target=\"_blank\" rel=\"nofollow\">www.haliatx.com<\/a>.<\/p>\n<p><b>Media Contact<\/b><\/p>\n<p>Taylor Avei\u00a0<br \/>Director of Business Development <br \/>Halia Therapeutics<br \/>+1 (385) 355-4315<br \/><a href=\"mailto:info@haliatx.com\" target=\"_blank\" rel=\"nofollow\">info@haliatx.com<\/a><\/p>\n<p><b>Investor Contact<\/b><\/p>\n<p>Leigh Salvo\u00a0<br \/>New Street Investor Relations<br \/><a href=\"mailto:leigh@newstreetir.com\" target=\"_blank\" rel=\"nofollow\">leigh@newstreetir.com<\/a><\/p>\n<p>\u00a0<\/p>\n<p><!-- \/wp:html --><\/p>\n","protected":false},"author":1,"featured_media":0,"comment_status":"open","ping_status":"open","sticky":false,"template":"","format":"standard","meta":{"rop_custom_images_group":[],"rop_custom_messages_group":[],"rop_publish_now":"initial","rop_publish_now_accounts":[],"rop_publish_now_history":[],"rop_publish_now_status":"pending","footnotes":""},"categories":[5,7],"tags":[],"class_list":["post-40991","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-cision-pr-newswire","category-cision-pr-newswire-en"],"_links":{"self":[{"href":"https:\/\/thaipropertynews.com\/feeds\/index.php?rest_route=\/wp\/v2\/posts\/40991","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/thaipropertynews.com\/feeds\/index.php?rest_route=\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/thaipropertynews.com\/feeds\/index.php?rest_route=\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/thaipropertynews.com\/feeds\/index.php?rest_route=\/wp\/v2\/users\/1"}],"replies":[{"embeddable":true,"href":"https:\/\/thaipropertynews.com\/feeds\/index.php?rest_route=%2Fwp%2Fv2%2Fcomments&post=40991"}],"version-history":[{"count":0,"href":"https:\/\/thaipropertynews.com\/feeds\/index.php?rest_route=\/wp\/v2\/posts\/40991\/revisions"}],"wp:attachment":[{"href":"https:\/\/thaipropertynews.com\/feeds\/index.php?rest_route=%2Fwp%2Fv2%2Fmedia&parent=40991"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/thaipropertynews.com\/feeds\/index.php?rest_route=%2Fwp%2Fv2%2Fcategories&post=40991"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/thaipropertynews.com\/feeds\/index.php?rest_route=%2Fwp%2Fv2%2Ftags&post=40991"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}