Halia Therapeutics Completes Recruitment for Phase 2a Study of HT-6184 for Myelodysplastic Syndrome Treatment

Halia Therapeutics Completes Recruitment for Phase 2a Study of HT-6184 for Myelodysplastic Syndrome

Halia Therapeutics, a clinical-stage biopharmaceutical company, has announced the successful completion of participant recruitment for its open-label Phase 2a clinical study. This pivotal research focuses on HT-6184 (Ofirnoflast) and its application for treating patients suffering from mild myelodysplastic syndrome (MDS) who are refractory to erythropoiesis-stimulating agents (ESAs) or are unsuitable for such treatments. This achievement marks a significant milestone in the ongoing commitment of Halia Therapeutics to innovate therapies anchored in genetic resilience.

The Phase 2a study (CTRI/2023/11/059758) aims to examine the efficacy, safety, and biomarker response of HT-6184. This novel allosteric modulator of NEK7 works by interrupting the NEK7-NLRP3 protein interaction, thereby preventing the formation of the NLRP3 inflammasome, an important inflammatory pathway associated with MDS and bone marrow dysfunction. Following the completion of Phase 1, which included 18 evaluable patients, the company has now enrolled an additional 15 participants for Phase 2.

Dr. David Bearss, CEO of Halia Therapeutics, commented on the recruitment completion, stating, "This milestone is crucial as we continue to validate our mechanism, which targets the dysregulation of the innate immune system. The data we gather will provide significant proof-of-concept regarding the therapeutic potential of HT-6184 in reducing clonal inflammation and improving hematological outcomes for patients with symptomatic anemia."

The design of the study features a 16-week treatment phase, followed by a continuation phase based on patient response. Those who respond positively can continue the therapy, while non-responders showing over a 30% reduction in variant allele frequency (VAF) will have the opportunity for up to 16 additional weeks of treatment, either as monotherapy or combined with prior ESA therapy. Key objectives include assessing efficacy through blood parameter improvements, clonal suppression, VAF reduction, patient safety, and overall quality of life through patient-reported outcomes.

After Phase 1, an interim analysis was conducted, and full study results are expected later this year. Halia Therapeutics aims to redefine treatments by targeting the innate immune system and leveraging genetic resilience. Their pipeline is promising and includes:

1. HT-6184, currently in a Phase 2a trial for myelodysplastic syndrome (MDS).
2. HT-6184 combined with Semaglutide, slated to begin a Phase 2a study in obese patients and those with Type 2 Diabetes (T2D) in Q3 2025.
3. HT-4253, a candidate targeting neuroinflammation, currently undergoing a Phase 1 clinical trial (NCT06537817), expected to conclude in Q3 2025.

For more details on Ofirnoflast (HT-6184), HT-4253, or ongoing clinical trials, please visit www.haliatx.com.