Halia Therapeutics Announces Positive Phase 2a Data for Ofirnoflast in Lower-Risk MDS at ASH 2025

– 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 Steps

Following 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. EST

About Halia's Phase 2 Trial of Ofirnoflast in Lower-Risk MDS

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.

About Halia Therapeutics

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 www.haliatx.com.

Media Contact

Taylor Avei 
Director of Business Development
Halia Therapeutics
+1 (385) 355-4315
info@haliatx.com

Investor Contact

Leigh Salvo 
New Street Investor Relations
leigh@newstreetir.com

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