Halia Therapeutics, a clinical-stage biopharmaceutical company pioneering therapies inspired by genetic resilience, today announced the completion of enrollment for its open-label Phase 2a clinical trial evaluating HT-6184 (Ofirnoflast) in patients with lower-risk Myelodysplastic Syndrome (MDS) who are refractory to, intolerant of, or ineligible for erythropoiesis-stimulating agents (ESA).
The Genetic Resilience Company
The study (CTRI/2023/11/059758) is designed to evaluate the efficacy, safety, and biomarker response of HT-6184, a novel allosteric modulator of NEK7 that disrupts NEK7–NLRP3 protein interaction, thereby preventing the formation of the NLRP3 inflammasome. This mechanism also promotes the disassembly of pre-formed NLRP3 inflammasomes, targeting a key inflammatory pathway implicated in bone marrow dysfunction in myelodysplastic syndromes (MDS). The two-stage study enrolled 18 evaluable patients in Stage 1 and has now completed enrollment of an additional 15 participants in Stage 2.
« Completing enrollment in our Phase 2a MDS study is a major milestone as we continue to 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 to support the therapeutic potential of HT-6184 in reducing clonal inflammation and improving hematologic outcomes for patients with symptomatic anemia. »
The trial consists of a 16-week treatment period, followed by a response-based continuation phase. Responders may continue on therapy, while non-responders showing a greater than 30% reduction in variant allele frequency (VAF) clone size may receive up to 16 additional weeks of treatment, either as monotherapy or in combination with prior ESA therapy. 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.
An interim analysis was conducted following Stage 1, and topline results from the complete study are expected later this year.
