Halia Therapeutics, a clinical-stage biopharmaceutical company pioneering the development of genetic resilience-based therapies, today announced the completion of enrollment in its open-label, Phase 2a clinical trial evaluating HT-6184 ( ofirnoflast ) in patients with lower-risk myelodysplastic syndromes (MDS) who are refractory to, intolerant to, or ineligible for erythropoiesis-stimulating agents (ESAs).
The study ( CTRI/2023/11/059758 ) is designed to investigate the efficacy, safety, and biomarker response of HT-6184, a novel allosteric modulator of NEK7 that disrupts the NEK7-NLRP3 protein interaction, thereby preventing the formation of the NLRP3 inflammasome. This mechanism also promotes the degradation of pre-formed NLRP3 inflammasomes, thereby targeting a key inflammatory pathway associated with bone marrow dysfunction in myelodysplastic syndromes (MDS). The two-stage study enrolled 18 evaluable patients in Phase 1 and has now completed recruitment of an additional 15 participants for Phase 2.
""Completing enrollment in our Phase 2a MDS study is an important milestone as we further validate our mechanism of action targeting innate immune dysregulation,"" said Dr. David Bearss , CEO of Halia Therapeutics. ""This study provides important proof-of-concept data supporting the therapeutic potential of HT-6184 in reducing clonal inflammation and improving hematological outcomes in patients with symptomatic anemia.""
The study consists of a 16-week treatment period followed by a response-based extension phase. Responders can continue therapy, while non-responders who demonstrate a greater than 30% reduction in the variant allele frequency (VAF) clone size can receive up to 16 additional weeks of treatment, either as monotherapy or in combination with prior ESA therapy. Key study objectives include evaluating efficacy based on improvements in blood counts, clone suppression, and VAF reduction; assessing patient safety and tolerability; monitoring changes in inflammasome-related biomarkers; and measuring quality of life using patient-reported outcome tools.
An interim analysis was conducted following Phase 1, and initial results from the full study are expected later this year.
About Halia Therapeutics:
Halia Therapeutics is setting new standards in treatment by targeting the innate immune system and harnessing genetic resilience. Halia's therapies are based on groundbreaking research identifying protective mutations in individuals genetically predisposed to serious disease and aim to restore immune balance in inflammatory and neurodegenerative diseases.
The company’s pipeline includes:
HT-6184 , currently in a Phase 2a study for myelodysplastic syndrome (MDS)
HT-6184 in combination with semaglutide , a planned Phase 2a study in obese patients and patients with type 2 diabetes (T2D), is expected to start in the third quarter of 2025.
HT-4253 , a candidate targeting neuroinflammation, is currently in an ongoing Phase 1 clinical trial ( NCT06537817 ) that is expected to be completed in Q3 2025
