Halia Therapeutics to Present Groundbreaking Data on Novel Allosteric NEK7 Inhibitor Ofirnoflast at the 2025 American Society of Hematology Annual Meeting

– Data demonstrates ofirnoflast’s efficacy in myelodysplastic syndrome (MDS) with robust and sustained hematologic responses –

– A novel mechanism targeting NEK7 and NLRP3 inflammasomes shows promise across multiple myeloid neoplasms –

– Preclinical data reveal potential applications in hemolytic anemias and obesity-induced inflammatory anemia –

LEHI, Utah, Nov. 10, 2025 /PRNewswire/ — Halia Therapeutics, a clinical-stage biopharmaceutical company developing first-in-class anti-inflammatory therapies, today announced six poster presentations and one oral presentation at the 67^th American Society of Hematology (ASH) Annual Meeting & Exposition taking place in Orlando, Florida, from December 6-9, 2025.

Ofirnoflast is a novel, oral, allosteric NEK7 inhibitor designed to block the formation and promote disassembly of the NLRP3 inflammasome complex. The NLRP3 inflammasome is a key driver of inflammation and plays a critical role in the pathogenesis of anemia and disease progression in myelodysplastic syndrome and other hematologic conditions. By selectively targeting NEK7, an essential component required for inflammasome assembly, ofirnoflast provides a precise mechanism to interrupt inflammatory cascades while potentially avoiding broader immunosuppressive effects. The safety and efficacy of ofirnoflast is currently being evaluated in clinical studies for patients with myelodysplastic syndrome and other inflammatory conditions.

“The comprehensive data we’re presenting at ASH 2025 represents a significant milestone for Halia Therapeutics and, more importantly, for patients with myelodysplastic syndrome and other inflammatory hematologic conditions who have limited therapeutic options,” said David Bearss, Chief Executive Officer of Halia Therapeutics. “Ofirnoflast’s unique mechanism of targeting the NEK7-NLRP3 inflammasome axis offers a fundamentally different approach to treating these challenging conditions. The robust and sustained hematologic responses we’re observing in MDS patients, combined with the favorable safety profile, reinforce our commitment to advancing this innovative therapy through clinical development.”

ASH Presentations:

Ofirnoflast, a Novel Inflammasome Inhibitor, Rewires Transcriptional Programs and Shows Clinical and Preclinical Efficacy and Myeloid Neoplasms
Saturday, December 6, 2025; 4:00 P.M. – 5:30 P.M. EST

Ofirnoflast Suppresses Heme-induced Inflammation, Representing a Novel Therapeutic Strategy for Hemolytic Anemias
Saturday, December 6, 2025; 5:30 P.M. – 7:30 P.M. EST

Targeting NLRP3 with Ofirnoflast, Alone or in Combination with Semaglutide, Ameliorates Inflammatory Anemia Associated with Obesity-Induced Inflammation in Rodent Models

Saturday, December 6, 2025; 5:30 P.M. – 7:30 P.M. EST

Ofirnoflast The Novel Allosteric NEK7 Inhibitor Ofirnoflast (HT-6184) Suppresses IL-8 and Other Pro-Inflammatory Cytokines in Patients with Low-Risk Myelodysplastic Syndrome and Symptomatic Anemia
Saturday, December 6, 2025; 5:30 P.M. – 7:30 P.M. EST

The Novel Allosteric NEK7 Inhibitor Ofirnoflast (HT-6184) Reduces Oxidized Mitochondrial DNA in Patients with Low-Risk Myelodysplastic Syndrome
Saturday, December 6, 2025; 5:30 P.M. – 7:30 P.M. EST

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
Monday, December 8, 2025; 6:00 P.M. – 8:00 P.M. EST

Monoclonal Antibodies Against Pathogenic S100A8/9 Alarmins can Restore Hematopoiesis and Result in Immunomodulation in Myelodysplasia and Leukemia Models
Monday, December 8, 2025; 6:00 P.M. – 8:00 P.M. EST

The presentations will feature comprehensive clinical and preclinical data on orfinoflast (HT-6184), a novel allosteric NEK7 inhibitor targeting the NLRP3 inflammasome, demonstrating its potential across multiple hematologic conditions, including myelodysplastic syndrome, hemolytic anemias, and inflammatory conditions.

Clinical data from the ongoing study of orfinoflast demonstrate robust and sustained hematologic responses in patients with IPSS-R very low, low, or intermediate risk myelodysplastic syndrome (MDS) and symptomatic anemia. Ofirnoflast represents a breakthrough therapeutic approach through its innovative mechanism of action, selectively targeting NEK7 to inhibit NLRP3 inflammasome activation, addressing the underlying inflammatory pathways that drive anemia and disease progression in MDS patients.

Additional clinical findings reveal that ofirnoflast showed a trend toward reducing oxidized mitochondrial DNA levels, IL-8, and other pro-inflammatory cytokines in patients with low-risk MDS and symptomatic anemia. Furthermore, comprehensive analyses demonstrate that ofirnoflast rewires transcriptional programs in myeloid neoplasms, providing mechanistic insights into its therapeutic efficacy across multiple hematologic malignancies.

Preclinical studies presented at ASH showcase ofirnoflast’s broader therapeutic potential, including its ability to suppress heme-induced inflammation, representing a novel therapeutic strategy for hemolytic anemias. Additional research demonstrates that targeting NLRP3 with ofirnoflast, alone or in combination with semaglutide, ameliorates inflammatory anemia associated with obesity-induced inflammation in rodent models. Collaborative research also highlights the therapeutic potential of targeting pathogenic S100A8/9 alarmins to restore hematopoiesis in models of myelodysplasia and leukemia.

About Halia Therapeutics

Halia Therapeutics is an innovative biotechnology company focused on developing first-in-class inflammasome inhibitors for inflammatory diseases and hematologic malignancies. The company is committed to addressing significant unmet medical needs through novel mechanisms of action that target the root causes of inflammation-driven diseases. Halia’s lead program, ofirnoflast, represents a breakthrough approach to treating conditions where the NLRP3 inflammasome drives disease pathology. With a focus on precision medicine and patient-centric drug development, Halia Therapeutics aims to bring transformative therapies to patients suffering from serious inflammatory and hematologic conditions.

Contact Information

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

Leigh Salvo
New Street Investor Relations
leigh@newstreetir.com

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