Halia Therapeutics Reports 67% Hematological Improvement Rate with Ofirnoflast in Lower-Risk MDS Phase 2 Trial
核心洞察
Halia Therapeutics (搜索) announced final Phase 2 results for ofirnoflast (HT-6184) showing 67% hematological improvement rate in 30 evaluable patients with lower-risk myelodysplastic syndrome (搜索).
The first-in-class NEK7 (搜索) inhibitor demonstrated 55% transfusion independence for at least 8 weeks among transfusion-dependent patients, with median duration of 28.5 weeks.
The trial reported no treatment-related serious adverse events, with treatment-related adverse events occurring in only 27% of patients.
Halia Therapeutics (搜索) announced final Phase 2 results for ofirnoflast (HT-6184) demonstrating a 67% hematological improvement rate in patients with lower-risk myelodysplastic syndrome (搜索) (LR-MDS), positioning the first-in-class NEK7 (搜索) inhibitor for advancement into pivotal trials. The data will be presented at the European Hematology Association (EHA) 2026 Hybrid Congress in Stockholm, Sweden, from June 11-14, 2026.
The company simultaneously announced the appointment of Han Myint, MD, FACP, as Chief Medical Officer to lead global clinical development, medical affairs, and safety strategy as ofirnoflast progresses toward pivotal development.
Phase 2 Trial Design and Patient Population
The open-label, single-arm Phase 2 trial enrolled 37 adults with IPSS-R very low- to intermediate-risk MDS (score ≤4.5) who had symptomatic anemia (搜索) or red blood cell transfusion dependence and were refractory to, intolerant of, or ineligible for erythropoiesis-stimulating agents (搜索) (ESAs). Ofirnoflast was administered orally at 2 mg once daily on a 5-days-on/2-days-off schedule for up to 32 weeks, with hematological improvement per IWG 2018 criteria serving as the primary endpoint.
Efficacy Results Across Multiple Hematologic Parameters
Among 30 evaluable patients, ofirnoflast demonstrated multilineage activity with 67% overall hematological improvement rate, including 62% HI-E (erythroid), 60% HI-P (platelet), and 50% HI-N (neutrophil) responses. The treatment achieved 55% RBC transfusion independence for at least 8 weeks among transfusion-dependent patients (10/18), with 39% sustaining independence for at least 16 weeks and a median duration of transfusion independence of 28.5 weeks.
Non-transfusion-dependent patients showed particularly strong responses, with 75% achieving HI-E (9/12 patients). Responders experienced a median hemoglobin rise of 4.5 g/dL, with individual improvements ranging from 0.1 to 7.1 g/dL. Activity was observed across WHO MDS subtypes and mutational backgrounds, including patients with and without SF3B1 (搜索) or del(5q) mutations.
Safety Profile and Tolerability
The trial reported no treatment-related serious adverse events, with treatment-related adverse events occurring in 27% of patients and only a single Grade ≥3 event (hypertension). This favorable safety profile supports the potential for long-term treatment in the target patient population.
"These final Phase 2 results validate our approach of targeting upstream innate immune biology in lower-risk MDS," said David J. Bearss, Ph.D., President, Chief Executive Officer, and co-founder of Halia Therapeutics (搜索). "Ofirnoflast has demonstrated durable transfusion independence, multilineage hematological improvement, and a favorable safety profile in a patient population with significant unmet need."
Leadership Appointment to Support Pivotal Development
Dr. Myint brings more than three decades of experience spanning academic medicine, biotechnology and global pharmaceutical organizations, with deep expertise in hematologic malignancies and oncology drug development. Previously, he served as Chief Medical Officer of NextCure, Inc., and NexImmune, Inc., and held senior leadership roles at Celgene Corporation (now part of Bristol Myers Squibb (搜索)), including Global Myeloid Disease Lead and Co-Chair of the Global Myeloid Franchise Team.
"The addition of Han to our leadership team strengthens our ability to execute the next phase of the ofirnoflast development program with clinical and regulatory execution, as we advance toward pivotal development," continued Bearss. "His experience in myeloid diseases, pivotal trial strategy, and regulatory engagement is directly aligned with where Halia is headed."
Dr. Myint commented on his appointment: "I am excited to join Halia at such a pivotal moment. The ofirnoflast Phase 2 data support a differentiated therapeutic approach in lower-risk MDS, where patients continue to need new oral options that can improve hematopoiesis and reduce transfusion burden."
Mechanism of Action and Development Strategy
Ofirnoflast is a first-in-class, oral, allosteric NEK7 (搜索) inhibitor designed to modulate NLRP3 inflammasome (搜索) activation upstream of inflammatory signaling. By targeting NEK7 before NLRP3 assembly, ofirnoflast prevents inflammasome formation and disrupts a key pathway implicated in ineffective hematopoiesis. Halia is advancing ofirnoflast as a potential disease-modifying therapy for lower-risk MDS, with broader development opportunities across inflammasome-driven diseases.
The EHA2026 presentation will include additional efficacy, safety, biomarker, and quality-of-life data. The oral presentation is scheduled for Friday, June 12, 2026, from 17:15-18:30 CEST in the A2-3 Hall at Stockholmsmässan, Stockholm, Sweden, and will be available virtually on the EHA Congress platform.