Halia Therapeutics Reports Positive Phase 2 Results for HT-6184 in LR-MDS Patients

LEHI, UT, USA I December 10, 2024 I Halia Therapeutics, a leading biopharmaceutical company focused on developing treatments for chronic inflammation and related disorders, has announced encouraging topline results leading to the next phase of its Phase 2 clinical trial. This trial is focused on HT-6184, an innovative oral allosteric NEK7/NLRP3 inflammasome inhibitor, for patients with lower-risk myelodysplastic syndromes (LR-MDS).

In the initial trial phase, 18 patients diagnosed with LR-MDS exhibited significant hematological improvement, specifically an erythroid response (HI-E), after 16 weeks of treatment with HT-6184. This positive outcome surpassed the initial requirement of achieving responses in at least three patients, enabling the trial to proceed to the second stage. This subsequent phase will involve enrolling an additional 8 to 10 patients to further explore HT-6184's therapeutic potential.

Dr. Alan List, a member of Halia’s Scientific Advisory Board, emphasized the importance of these findings. He noted that the high frequency of erythroid response underscores the critical role of the NLRP3 inflammasome and myddosome pathways as drivers in the ineffective hematopoiesis observed in MDS. HT-6184 promises to be a safe and effective oral treatment option for patients with LR-MDS.

Dr. David J. Bearss, President and CEO of Halia Therapeutics, highlighted that the trial marks a significant advancement in addressing the unmet needs of LR-MDS patients. HT-6184 aims to improve treatment outcomes and quality of life by targeting the underlying inflammatory signaling contributing to the condition. Currently, patients with LR-MDS have limited treatment options, making this development particularly promising.

The Phase 2 trial employs a Simon’s minimax two-stage design to assess the safety and efficacy of HT-6184 in up to 40 patients across various clinical sites in India. The primary endpoints of the study focus on hematologic improvements, including transfusion dependency and hemoglobin level changes. Secondary endpoints evaluate the impact of HT-6184 on biomarkers of inflammasome activation in MDS and the size of somatic gene mutation clones, providing a comprehensive assessment of its therapeutic potential.

The trial's conclusion is anticipated by the end of the second quarter of 2025. The findings from this study are expected to yield crucial data supporting the continued clinical development of HT-6184 and its eventual regulatory approval.

Myelodysplastic Syndromes (MDS) are a collection of blood-related cancers characterized by bone marrow dysfunction, resulting in abnormal and underdeveloped blood cells. This condition leads to complications such as anemia and increased infection risk, with some cases advancing to acute myeloid leukemia. The existing treatments for LR-MDS are limited in both effectiveness and duration, highlighting the urgent need for innovative therapies.

HT-6184 represents a novel treatment approach by targeting the NEK7 protein through an allosteric mechanism. NEK7 is crucial for the assembly and activity of the NLRP3 inflammasome. Preclinical models have demonstrated that by inhibiting NEK7's binding to NLRP3, HT-6184 disrupts inflammasome signaling, reducing inflammatory response. It prevents NLRP3 inflammasome formation and facilitates its disassembly once activated.

Halia Therapeutics, headquartered in Lehi, Utah, is advancing a pipeline of novel therapeutics aimed at improving the lives of patients with chronic inflammatory disorders and neurodegenerative diseases. The company's early-stage programs focus on NEK7 and LRRK2. HT-6184, Halia’s leading candidate, has successfully completed Phase I trials evaluating its safety and tolerability. Additionally, Halia has launched two Phase II trials to further investigate HT-6184's efficacy, including its impact on post-procedure diagnostic biomarkers of inflammation and pain.