#USA #Lehi, Utah #Halia Therapeutics #HT-6184 #Myelodysplastic Syndrome

Halia Therapeutics Advances MDS Treatment Research

Halia Therapeutics, a clinical-stage biopharmaceutical firm located in Lehi, Utah, has officially completed the enrollment phase for its pivotal Phase 2a clinical trial investigating the effectiveness and safety of HT-6184 (Ofirnoflast) for individuals suffering from low-risk Myelodysplastic Syndrome (MDS). This critical milestone indicates Halia's commitment to exploring innovative treatments that leverage genetic resilience in combating this complex condition.

Myelodysplastic Syndrome is a group of disorders caused by poorly formed or dysfunctional blood cells. It can lead to severe symptoms, including debilitating fatigue and increased risk of infections. Patients typically manage MDS symptoms through erythropoiesis-stimulating agents (ESAs), but challenges arise for those who do not respond to, cannot tolerate, or are contraindicated from using these treatments. HT-6184 enters the fray as a potential new therapeutic agent.

The study (clinical trial registration number CTRI/2023/11/059758) is not only observing the safety profile of HT-6184 but also aims to dissect its biomarker responses. This novel therapeutic agent acts as an allosteric modulator of the NEK7 protein, showcasing a unique mechanism that interrupts the NEK7–NLRP3 protein interaction. This pivotal interaction is known to be involved in the formation of the inflammatory NLRP3 inflammasome, a key player in the dysregulation of the innate immune response often seen in myelodysplastic syndromes.

With the Phase 2a trial structured in two stages, it initially enrolled 18 patients in Stage 1, followed by an additional 15 participants concluded in Stage 2. Dr. David Bearss, the CEO of Halia Therapeutics, heralded this accomplishment, stating, “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.” He emphasized the importance of the study as a proof-of-concept for HT-6184’s ability to mitigate clonal inflammation, thereby potentially enhancing hematologic outcomes for patients battling symptomatic anemia.

Patients involved in the trial will undergo treatments over a 16-week period. Participants demonstrating a favorable response may continue their treatment, while those exhibiting more than a 30% reduction in variant allele frequency (VAF) clone size could receive additional treatment, extendable up to 16 weeks. This approach not only intends to improve hematological metrics but also seeks to evaluate the therapy’s overall safety and patient tolerance, emphasizing quality of life through patient-reported outcomes.

As the clinical trials progress, Halia’s research team conducted an interim analysis after Stage 1. Topline results from the complete study are eagerly awaited and are scheduled to be released later this calendar year. This promising research signifies an encouraging step in expanding treatment options for MDS, a condition often burdened with limited therapeutic choices.

About Halia Therapeutics

Founded on groundbreaking research into protective mutations, Halia Therapeutics aims to redefine therapeutic approaches to diseases marked by inflammatory and neurodegenerative processes. The company's pipeline illustrates their innovative focus:

• - HT-6184: The current Phase 2a trial candidate targeting MDS.
• - HT-6184 with Semaglutide: A planned Phase 2a trial for obese and Type 2 diabetes patients expected to launch in the latter part of 2025.
• - HT-4253: A candidate aimed at neuroinflammation currently undergoing a Phase 1 clinical trial, expected to conclude by 2025.

To explore the company’s clinical trials, including HT-6184 and HT-4253, more information can be found on their official website at www.haliatx.com.