– New results demonstrate HI-E response rate of 72% at week 16 with significant improvement in hemoglobin –
– High activity observed in patients refractory and intolerant to ESA, as well as in mutation and morphology subtypes –
– Favorable safety profile with no treatment-related serious adverse events –
LEHI, Utah, December 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 Annual Meeting of the American Society of Hematology (ASH). Data show that ofirnoflast, a first-in-class oral allosteric NEK7 inhibitor, induces clinically meaningful and sustained hematologic responses in patients with low-risk myelodysplastic syndromes (MDS) and symptomatic anemia.
In the stage 1 efficacy population (N=18), ofirnoflast achieved a hematologic-erythroid improvement (HI-E) response rate of 72% after ≥16 weeks of treatment. Consistent improvements were observed across morphological subtypes and WHO somatic mutation categories, supporting a broad, biology-driven mechanism of action.
Key results from step 1:
- 72% of patients (13/18) achieved HI-E at week 16, with responders having a median increase in hemoglobin of 3.5 g/dL.
- High activity in difficult-to-treat patients, including 91% HI-E in ESA-refractory subjects 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 AEs, and no evidence of treatment-related myelosuppression.
These results reinforce NEK7 inhibition as a promising strategy to combat the underlying inflammatory dysregulation central to ineffective hematopoiesis in MDS.
“These data highlight the potential for ofirnoflast to significantly improve outcomes for patients with low-risk MDS,” said David Bearss, Ph.D., CEO of Halia Therapeutics. “Achieving a HI-E response rate of 72%, including strong performance in refractory and intolerant patients as well as a clean safety profile, highlights the therapeutic promise of NEK7 inhibition. We look forward to building on these results as we advance the program to a later stage of development.
Next steps
Following FDA orphan drug designation granted in October 2025, Halia is currently communicating next steps with the FDA. Halia is finalizing the data set and preparing to launch a global pivotal Phase 3 trial in early 2026.
American Society of Hematology (ASH) Poster Details:
Title: “The novel allosteric NEK7 inhibitor Ofirnoflast (HT-6184) demonstrates a 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
About Halia’s Phase 2 Trial of Ofirnoflast in Low-Risk MDS
HT-6184-MDS-001 is a two-stage multicenter study by Simon evaluating hematologic improvement after 16 weeks of treatment, with an extension phase for molecularly enhanced responders and non-responders. The primary study objectives include evaluating efficacy through hematologic improvement, clonal deletion and reduction of VAF, assessing patient safety and tolerability, monitoring changes in inflammasome-related biomarkers, and measuring quality of life using patient-reported outcomes tools.
About Halia Therapeutics
Halia Therapeutics is a biotechnology company developing first-in-class inflammasome inhibitors. We target the root causes of inflammatory diseases to create transformative therapies. For more information, visit www.haliatx.com.
Media contact
Taylor Avei
Director of Business Development
Halia Therapeutic
+1 (385) 355-4315
info@haliatx.com
Investor contact
Leigh Salvo
New relationships with street investors
leigh@newstreetir.com
SOURCE Halia Therapeutics
