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

Halia Therapeutics Completes Enrollment in Phase 2a Clinical Trial of HT-6184 for Myelodysplastic Syndrome (MDS)

Halia Therapeutics, a pioneering biopharmaceutical company focused on genetic resilience therapies, has announced a significant milestone: the completion of patient enrollment in its Phase 2a clinical trial. The trial evaluates HT-6184 (also known as Ofirnoflast) for patients suffering from low-risk myelodysplastic syndrome (MDS) who are either refractory to, intolerant of, or not eligible for erythropoiesis-stimulating agents (ESAs).

The clinical trial, listed under the identifier CTRI/2023/11/059758, has been structured to assess the efficacy, safety, and biomarker responses associated with HT-6184. This drug is designed as a new allosteric modulator of NEK7, acting to disrupt the interaction between NEK7 protein and NLRP3. By doing so, it impedes the formation of the NLRP3 inflammasome, a critical pathway implicated in bone marrow dysfunction in MDS.

The study comprises two stages, having already enlisted 18 evaluable patients in Stage 1 and an additional 15 in Stage 2. The CEO of Halia Therapeutics, Dr. David Bearss, expressed his enthusiasm about this achievement, emphasizing the importance of validating the company’s action mechanism targeting innate immune dysregulation. He stated, “Completing the enrollment for our Phase 2a study on MDS is a critical step in demonstrating our therapeutic potential, aimed at reducing clonal inflammation and improving hematological outcomes for patients with symptomatic anemia.”

The trial incorporates a treatment period extending across 16 weeks, followed by a continuation phase based on the patient’s response. Participants experiencing positive responses can maintain the treatment, while those who do not see a response, with over a 30% reduction in the variant allele frequency (VAF) of the clonotype size, may be offered up to 16 additional weeks of therapy, either as monotherapy or combined with prior ESA treatment. Key study objectives include measuring efficacy via hematological improvement and clonal suppression, assessing patient safety and tolerance, monitoring changes in inflammasome-related biomarkers, and evaluating quality of life using patient-reported outcomes.

A preliminary analysis was conducted post-Stage 1, with main results from the complete study anticipated by year-end.

About Halia Therapeutics

Founded on cutting-edge research identifying protective mutations in those genetically predisposed to severe medical conditions, Halia Therapeutics is actively redefining therapeutic approaches. The company’s focus is on restoring immune balance in inflammatory and neurodegenerative diseases. Its current project portfolio includes:

• - HT-6184: Currently undergoing a Phase 2a trial for myelodysplastic syndrome (MDS).
• - HT-6184 combined with Semaglutide: A planned Phase 2a trial starting in Q3 2025 aimed at patients with obesity and type 2 diabetes (T2D).
• - HT-4253: Targeting neuroinflammation, currently in a Phase 1 clinical trial (NCT06537817), expected to conclude in Q3 2025.

For further details about Ofirnoflast (HT-6184), HT-4253, or ongoing clinical trials, visit www.haliatx.com.