Salute e Benessere
Halia Therapeutics Announces Positive Phase 2a Data for Ofirnoflast in Lower-Risk MDS at ASH 2025
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.
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."
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.
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.
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.
Taylor Avei
Director of Business Development
Halia Therapeutics
+1 (385) 355-4315
info@haliatx.com
Leigh Salvo
New Street Investor Relations
leigh@newstreetir.com
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