Halia Therapeutics, Inc.

LEHI, UT, USA I December 10, 2024 I 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 . Connect with us on LinkedIn and Twitter (X) for the latest updates. SOURCE: Halia Therapeutics

Halia Therapeutics is moving its lead asset into the second part of a mid-stage trial following initial positive data in patients with an early form of blood cancer that affects the bone marrow. The Utah-based biotech is investigating its oral NEK7/NLRP3 inflammasome inhibitor, dubbed HT-6184, in patients with lower-risk myelodysplastic syndromes. Patients with MDS have abnormal and underdeveloped blood cells, which can lead to anemia, a heightened risk of infections, and progression to acute myeloid leukemia, according to the company. In the first small cohort of 18 participants taking the inhibitor as a monotherapy, the drug demonstrated hematological improvement in all of the patients’ erythroid response after 16 weeks of treatment, meaning they saw an improvement in the number of healthy red blood cells. The requirement to move HT-6184 into the second stage of the trial was at least three patients responding to treatment. The study is expected to finish in the middle of next year. The primary endpoints include transfusion dependency and changes in hemoglobin levels. “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,” Halia CEO David Bearss said in a statement. The Phase 2 trial will enroll up to 40 patients in India, with enrollment to be completed by February. The drug is also in another trial investigating its impact on inflammation and pain in patients following a surgical procedure. If Halia’s drug is approved, it will compete against Bristol Myers Squibb’s Reblozyl, which was approved for the treatment of anemia in patients with the disorder last year, and Geron’s Rytelo, which scored FDA approval in June for patients who are dependent on blood transfusions.

LEHI, Utah, Dec. 5, 2024 /PRNewswire/ -- Halia Therapeutics, Inc. (Halia), a clinical-stage biopharmaceutical company at the forefront of developing innovative treatments for chronic inflammatory diseases, today announced that David J. Bearss, Ph.D., Co-Founder, President and CEO of Halia Therapeutics, will present cutting-edge data on oral obesity therapeutics at the upcoming Obesity Innovation Therapeutics Summit. The conference will be held in San Diego, December 10-12, 2024. In addition, Dr. Bearss will also participate in a panel discussion addressing supply chain challenges in the obesity therapeutics landscape. The Obesity Innovation Therapeutics Summit highlights groundbreaking advancements in obesity treatment, including novel mechanisms of action, combination therapies, and strategies to enhance efficacy, durability, and muscle preservation. Presentation Details: Title: Patient-Centric Development: Exploring Oral Obesity Therapeutics to Move Away from Injectable Peptides Date: December 11, 2024 Time: 5 p.m. PT Presenter: David J. Bearss, Ph.D., Co-Founder & CEO, Halia Therapeutics Panel Details: Title: The Obesity Race on the Manufacturing Floor – Exploring Supply Challenges with Innovations in Discovery Date: December 11, 2024 Time: 2:40 p.m. PT Panelists: David J. Bearss, Ph.D., Co-Founder & CEO, Halia Therapeutics Feng Liu, Executive Director, Research & Development, Regor Pharmaceuticals Adam Mendelsohn, Chief Executive Officer, Vivani Medical About Halia Therapeutics, Inc. Halia Therapeutics is transforming the treatment landscape for chronic inflammatory and neurodegenerative diseases with a pipeline of groundbreaking therapeutics. The company's programs target NEK7 and LRRK2, key drivers of inflammation and cellular dysfunction. Halia's lead candidate, HT-6184—a novel NEK7/NLRP3 inhibitor—has demonstrated safety and tolerability in Phase I trials (NCT05447546) with single and multiple escalating oral doses in healthy volunteers. It is now being evaluated in two Phase II studies: one assessing its efficacy in treating lower-risk myelodysplastic syndromes (LR-MDS) and another exploring its role in mitigating post-procedural inflammation and pain (NCT06241742). Halia recently initiated a Phase I trial for HT-4253, an innovative LRRK2 inhibitor targeting neurodegenerative diseases, in healthy volunteers (NCT06537817). Based in Lehi, Utah, Halia Therapeutics is dedicated to advancing patient care through science-driven innovation. Learn more at and follow us on LinkedIn and Twitter (X). Company Contact Halia Therapeutics [email protected] +1 (385) 355-4315 Media Contact: Ignacio Guerrero-Ros, Ph.D. Russo Partners, LLC +1 (646) 942-5604 [email protected] SOURCE Halia Therapeutics WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED