LEHI, Utah — December 8, 2025 — Leads & Copy — Halia Therapeutics presented new clinical data from its Phase 2a study of ofirnoflast (HT-6184) at the 67th American Society of Hematology (ASH) Annual Meeting, showcasing clinically meaningful and sustained hematologic responses in patients with lower-risk myelodysplastic syndromes (MDS) and symptomatic anemia.
The Stage 1 efficacy population (N=18) treated with 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.
Key Stage 1 findings included:
- 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.
David Bearss, Ph.D., CEO of Halia Therapeutics, stated that the data highlight the potential of ofirnoflast to meaningfully improve outcomes for patients with lower-risk MDS and underscores the therapeutic promise of NEK7 inhibition. He added that the company is looking forward to building on these results as they advance the program toward later-stage development.
Following the FDA Orphan Drug Designation granted in October 2025, Halia is currently communicating next steps with the FDA and is 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. EST
HT-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.
For media inquiries, contact Taylor Avei, Director of Business Development at Halia Therapeutics, at +1 (385) 355-4315 or info@haliatx.com. For investor inquiries, contact Leigh Salvo, New Street Investor Relations, at leigh@newstreetir.com.
Source: Halia Therapeutics
