Halia Therapeutics Announces Positive Topline Data and Advances to Second Stage of Phase 2 Clinical Trial for HT-6184 in Patients with Low-Risk Myelodysplastic Syndromes (LR-MDS)

• Treatment with HT-6184 has shown to improve hematological response in patients enrolled to date with LR-MDS, surpassing the threshold
• Halia advances to second stage expansion of Phase 2 trial

LEHI, Utah, Dec. 10, 2024 /PRNewswire/ -- Halia Therapeutics, a biopharmaceutical company at the forefront of developing treatments for chronic inflammation and related disorders, announced today promising topline data resulting in the advancement to the second stage of its Phase 2 clinical trial evaluating HT-6184, a first-in-class allosteric NEK7/NLRP3 inflammasome inhibitor given orally, in patients with lower-risk myelodysplastic syndromes (LR-MDS).

The initial cohort of 18 patients with LR-MDS demonstrated a hematological improvement-erythroid (HI-E) response to HT-6184 treatment following 16 weeks of monotherapy, exceeding the preset requirement of at least three responders. Having achieved this critical milestone, the trial now advances to its second stage enrolling an additional 8-10 patients to further validate HT-6184’s potential.

“The high frequency of erythroid response following treatment with HT-6184 validates the key importance of the NLRP3 inflammasome and myddosome pathways as pathogenetic drivers of ineffective hematopoiesis in MDS, offering the prospect of a safe and effective oral therapeutic for LR-MDS patients,” said Alan List, MD Halia SAB Member.

“This trial represents a significant step forward in addressing the unmet needs of patients with LR-MDS,” said Dr. David J. Bearss, Ph.D., President and CEO of Halia Therapeutics. “By targeting the underlying inflammatory signaling driving this condition, HT-6184 aims to transform treatment outcomes and improve quality of life for patients who currently face limited options.”

The Phase 2 trial utilizes a Simon’s minimax two-stage design to evaluate the safety and efficacy of HT-6184 in up to 40 patients across multiple clinical sites in India. Primary endpoints include hematologic improvements such as transfusion dependency and changes in hemoglobin levels. Secondary endpoints assess HT-6184’s impact on biomarkers of inflammasome activation in MDS and the size of somatic gene mutation clones, offering a comprehensive view of its therapeutic potential.

The trial is expected to conclude by the end of Q2 2025. The results of this study are anticipated to provide pivotal data to support HT-6184’s continued clinical development and eventual regulatory submission.

About Myelodysplastic Syndromes (MDS): An Urgent Unmet Need

MDS is a group of hematologic cancers characterized by bone marrow dysfunction, leading to abnormal and underdeveloped blood cells. These conditions result in complications such as anemia, increased infection risk, and, in some cases, progression to acute myeloid leukemia. Current treatments are limited in efficacy and durability for patients with LR-MDS, underscoring the need for innovative therapeutic solutions. Novel oral agents are needed for the treatment of this disease to alleviate the need to go into the office for treatment either with an injection or needed infusion.

About HT-6184

HT-6184 is a groundbreaking drug candidate that targets the protein NEK7 through an allosteric mechanism. NEK7 plays a crucial role in the NLRP3 inflammasome, which is essential for its assembly and activity. In preclinical models, Halia demonstrated that inhibiting NEK7’s binding ability to NLRP3 disrupts inflammasome signaling and reduces the inflammatory response. HT-6184 prevents the formation of the NLRP3 inflammasome and promotes its disassembly once activated. (Halia unpublished data)

About Halia Therapeutics, Inc.

Halia Therapeutics is pioneering a pipeline of novel therapeutics to enhance the lives of patients with chronic inflammatory disorders and neurodegenerative diseases. The company’s initial programs target NEK7 and LRRK2. Halia’s lead candidate, HT-6184—a novel NEK7/NLRP3 inhibitor—has successfully completed a Phase I study (NCT05447546) assessing its safety and tolerability when administered as single or multiple oral doses at escalating levels in healthy volunteers. Furthermore, Halia has launched two Phase II trials: one evaluating HT-6184’s efficacy in treating lower-risk myelodysplastic syndromes (LR-MDS) and another examining its impact on post-procedure diagnostic biomarkers of inflammation and pain (NCT06241742). Recently, Halia initiated a Phase I trial to evaluate the safety and tolerability of its LRRK2 inhibitor, HT-4253, in healthy volunteers (NCT06537817).

Headquartered in Lehi, Utah, Halia Therapeutics invites you to learn more at www.haliatx.com.

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Russo Partners, LLC

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SOURCE Halia Therapeutics