– New findings demonstrate a 72% HI-E response rate at Week 16 with meaningful hemoglobin improvement –– Strong activity observed across ESA-refractory and ESA-intolerant patients, and across mutation and morphology subtypes –– Favorable safety profile with no treatment-related serious adverse events –LEHI, Utah, Dec. 8, 2025 /PRNewswire/ — 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.
In the Stage 1 efficacy population (N=18), ofirnoflast achieved a 72% hematologic improvement-erythroid (HI-E) response rate following ≥16 weeks of therapy. Consistent improvements were observed across WHO morphologic subtypes and somatic mutation categories, supporting a broad and biology-driven mechanism of action.Key Stage 1 Findings:72% of patients (13/18) achieved HI-E at Week 16, with responders showing a median hemoglobin increase of 3.5 g/dL.Strong activity in difficult-to-treat patients, including 91% HI-E in ESA-refractory and 75% HI-E in ESA-intolerant subjects.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).Favorable safety profile, with no treatment-related SAEs, no Grade ≥3 related AEs, and no evidence of treatment-emergent myelosuppression.These findings reinforce NEK7 inhibition as a promising strategy to address the underlying inflammatory dysregulation central to ineffective hematopoiesis in MDS.”These data highlight the potential of ofirnoflast to meaningfully improve outcomes for patients with lower-risk MDS,” said David Bearss, Ph.D., CEO of Halia Therapeutics. “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.”Next StepsFollowing the FDA Orphan Drug Designation granted in October 2025, 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.American Society of Hematology (ASH) Poster Details:Title: “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”Time: Monday, December 8, 2025; 6:00 P.M. – 8:00 P.M. ESTAbout Halia’s Phase 2 Trial of Ofirnoflast in Lower-Risk MDSHT-6184-MDS-001 is a Simon’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.About Halia TherapeuticsHalia 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 www.haliatx.com.Media ContactTaylor Avei Director of Business Development Halia Therapeutics+1 (385) 355-4315info@haliatx.comInvestor ContactLeigh Salvo New Street Investor Relationsleigh@newstreetir.comLogo – https://www.newsoutnow.com//wp-content/uploads/2025/12/Halia_Therapeutics_Logo.jpg
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