A Randomized, Double-Blind, Placebo-Controlled Multicenter Phase 2 Study to Evaluate the Safety, Efficacy and Tolerability of Ofirnoflast (HT-6184) and Semaglutide in Obese or Overweight Participants With Type 2 Diabetes Mellitus (T2DM)

The purpose of this study is to evaluate the efficacy and safety of ofirnoflast in combination with semaglutide in comparison to semaglutide alone in obese participants with T2DM.

开始日期2026-01-01

申办/合作机构

Halia Therapeutics, Inc.

NCT06241742

/ Completed临床2期

A Phase-2 Study to Evaluate the Ability of HT-6184 to Attenuate Diagnostic Biomarkers of Post-procedure Acute Inflammation and Manage Pain Following Third Molar Extraction

The goal of this clinical trial is to test the ability of HT-6184 to decrease inflammation and pain in patients after third molar removal.
The main question it aims to answer are:
* Does HT-6184 decrease inflammation in patients after third molar removal?
* Does HT-6184 decrease pain in patients after third molar removal?
Participants will be asked to do the following during the clinical trial:
* Take a single oral dose of HT-6184 or placebo
* Allow a oral surgeon remove their third molar teeth
* Blood draws on 5 occurrences
* Rate their pain intensity
* Attend two follow-up appointments on day 1 and day 2 after third molar removal
* Participate in one follow-up phone call 5-7 days after third molar removal

开始日期2024-02-12

申办/合作机构

Halia Therapeutics, Inc.

NCT07052006

/ Active, not recruiting临床2期

A Phase 2a Study of HT-6184 in Subjects With IPSS-R Very Low, Low or Intermediate Risk Myelodysplastic Syndrome (MDS) and Symptomatic Anemia

This research is being conducted to asses if HT-6184 is effective in the treatment of Very Low, Low, or Intermediate Risk Myelodysplastic Syndrome (MDS) and Symptomatic Anemia.
The study includes a 28-day Screening Period followed by a 16- or 32-week Treatment Period.
Participants will be monitored at each cycle for drug tolerance, safety, and hematological response. A response assessment will occur after 16 weeks of study treatment.

开始日期2023-12-09

申办/合作机构

Halia Therapeutics, Inc.

100 项与 Ofirnoflast 相关的临床结果

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100 项与 Ofirnoflast 相关的转化医学

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100 项与 Ofirnoflast 相关的专利（医药）

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项与 Ofirnoflast 相关的文献（医药）

JOURNAL OF DRUG TARGETING

Ofirnoflast: A First-in-Class NEK7-Targeted Inhibitor of the NLRP3 Inflammasome

Article

作者: Burnett, William ; Bearss, Benjamin ; Mollard, Alexis ; Bursey, Devan ; Bearss, Jared ; Avei, Taylor ; Ramesh, Subbiah ; Bijpuria, Shipra ; Chaugule, Viduth K. ; Bearss, David ; Janat-Amsbury, Margit ; Teichert, Russ ; Davis, Chadwick ; Srinivas Tripuraneni, Naga

Ofirnoflast is a first-in-class, orally bioavailable NEK7 inhibitor currently undergoing Phase 2 clinical evaluation. It disrupts NLRP3 inflammasome assembly by targeting NEK7's scaffolding function-blocking complex formation independently of NLRP3 activation status, upstream of caspase activation, pyroptosis, and inflammatory cytokine release. This mechanism offers a novel therapeutic approach for chronic inflammation. Unlike NSAIDs, corticosteroids, cytokine-neutralising biologics, and NLRP3-directed small molecules-which are frequently limited by off-target effects, immunosuppression, or incomplete efficacy-ofirnoflast provides a targeted approach with fewer anticipated liabilities. We demonstrate that ofirnoflast engages an allosteric site adjacent to NEK7's ATP-binding pocket, inducing conformational shifts that impair its scaffolding function. In THP-1 macrophages and iPSC-derived microglia, ofirnoflast suppresses ASC specks, IL-1β release, and pyroptotic cell death. Biophysical assays and molecular dynamics simulations confirm that ofirnoflast stabilises NEK7 in a unique conformation and suggest a type-2 kinase-inhibitor binding mode. In vivo, ofirnoflast exhibits oral bioavailability, achieving systemic exposures well above cellular potency thresholds. In a DSS-induced colitis model, treatment significantly reduces cytokine levels and improves phsyiological outcomes. Collectively, these findings validate NEK7 as a druggable checkpoint for NLRP3 inflammasome control and position Ofirnoflast as a mechanistically distinct, clinically advanced candidate for treating inflammation driven by aberrant inflammasome activation.

项与 Ofirnoflast 相关的新闻（医药）

2025-12-08

·PRNewswire

Halia Therapeutics Announces Positive Phase 2a Data for Ofirnoflast in Lower-Risk MDS at ASH 2025

– New findings demonstrate a 72% HI-E response rate at Week 16 with meaningful hemoglobin improvement – – Strong activity observed across ESA-refractory and ESA-intolerant patients, and across mutation and morphology subtypes – – Favorable safety profile with no treatment-related serious adverse events – LEHI, Utah, Dec. 8, 2025 /PRNewswire/ -- Halia Therapeutics, a clinical-stage biopharmaceutical company, today presented new clinical data from its Phase 2a study of ofirnoflast (HT-6184) at the 67th American Society of Hematology (ASH) Annual Meeting. The data show that ofirnoflast, a first-in-class oral allosteric NEK7 inhibitor, induces clinically meaningful and sustained hematologic responses in patients with lower-risk myelodysplastic syndromes (MDS) and symptomatic anemia. In the Stage 1 efficacy population (N=18), ofirnoflast achieved a 72% hematologic improvement-erythroid (HI-E) response rate following ≥16 weeks of therapy. Consistent improvements were observed across WHO morphologic subtypes and somatic mutation categories, supporting a broad and biology-driven mechanism of action. Key Stage 1 Findings: 72% of patients (13/18) achieved HI-E at Week 16, with responders showing a median hemoglobin increase of 3.5 g/dL. Strong activity in difficult-to-treat patients, including 91% HI-E in ESA-refractory and 75% HI-E in ESA-intolerant subjects. Consistent responses across disease biology, with HI-E observed across transfusion burden categories, WHO morphologic subtypes, and major mutation groups (SF3B1, TET2, DNMT3A, ASXL1, TP53). Favorable safety profile, with no treatment-related SAEs, no Grade ≥3 related AEs, and no evidence of treatment-emergent myelosuppression. These findings reinforce NEK7 inhibition as a promising strategy to address the underlying inflammatory dysregulation central to ineffective hematopoiesis in MDS. "These data highlight the potential of ofirnoflast to meaningfully improve outcomes for patients with lower-risk MDS," said David Bearss, Ph.D., CEO of Halia Therapeutics. "Achieving a 72% HI-E response rate, including strong performance in refractory and intolerant patients alongside a clean safety profile, underscores the therapeutic promise of NEK7 inhibition. We look forward to building on these results as we advance the program toward later-stage development." Next Steps Following the FDA Orphan Drug Designation granted in October 2025, Halia is currently communicating next steps with the FDA. Halia is finalizing the dataset and preparing to initiate a global Phase 3 pivotal trial in early 2026. American Society of Hematology (ASH) Poster Details: Title: "The Novel Allosteric NEK7 Inhibitor Ofirnoflast (HT-6184) Demonstrates Robust and Sustained Hematologic Response in Subjects with IPSS-R Very Low, Low or Intermediate Risk Myelodysplastic Syndrome (MDS) and Symptomatic Anemia" Time: Monday, December 8, 2025; 6:00 P.M. - 8:00 P.M. EST About Halia's Phase 2 Trial of Ofirnoflast in Lower-Risk MDS HT-6184-MDS-001 is a Simon's two-stage, multicenter study evaluating hematologic improvement after 16 weeks of treatment, with an extension phase for responders and molecularly improving non-responders. Key study objectives include evaluating efficacy through hematological improvement, clonal suppression, and VAF reduction, assessing safety and patient tolerance, monitoring changes in inflammasome-related biomarkers, and measuring quality of life using patient-reported outcome tools. About Halia Therapeutics Halia Therapeutics is a biotechnology company developing first-in-class inflammasome inhibitors. We target the root causes of inflammation-driven diseases to create transformative therapies. For more information, visit . Media Contact Taylor Avei Director of Business Development Halia Therapeutics +1 (385) 355-4315 [email protected] Investor Contact Leigh Salvo New Street Investor Relations [email protected] SOURCE Halia Therapeutics 21% more press release views with Request a Demo

临床结果临床2期ASH会议孤儿药临床3期

2025-06-16

·PRNewswire

Halia Therapeutics Completes First-in-Human Phase 1 Study of HT-4253, a Novel LRRK2 Inhibitor Targeting Neuroinflammation

– Completion of SAD/MAD trial marks advancement toward further clinical development of HT-4253 in Alzheimer's and other neurodegenerative diseases – LEHI, Utah, June 16, 2025 /PRNewswire/ -- Halia Therapeutics, Inc., a clinical-stage biopharmaceutical company redefining treatment paradigms through inflammation-targeted and resilience-driven therapies, today announced the successful completion of its first-in-human Phase 1 clinical trial evaluating HT-4253 in healthy adult volunteers. The randomized, double-blind, placebo-controlled single- and multiple-ascending-dose (SAD/MAD) trial (ClinicalTrials.gov Identifier: NCT06537817) was conducted at CMAX Clinical Research in Adelaide, Australia. A total of 80 participants were dosed across 10 cohorts. The primary objectives of the trial were safety and tolerability, with secondary endpoints assessing pharmacokinetics (PK) and pharmacodynamics (PD. HT-4253 was generally well tolerated across all dose levels, with no serious adverse events reported. A comprehensive Clinical Study Report (CSR) is anticipated in Q4 2025. "The successful completion of this first-in-human study marks a significant milestone for Halia and provides compelling validation of HT-4253's safety, tolerability, and its potential to address a critical unmet need in neurodegenerative diseases, where dysregulated immune responses play a central role," said Dr. David Bearss, Chief Executive Officer of Halia Therapeutics. "These results show that HT-4253 can be safely administered at pharmacologically active doses, positioning us to move forward into patient populations with confidence. Backed by robust preclinical data and a mechanism inspired by naturally resilient individuals, HT-4253 represents a potentially transformative approach to modulating neuroinflammation in diseases such as Alzheimer's. Our team is now focused on preparing for the next phase of clinical development and identifying strategic opportunities to accelerate the program. Ultimately, our goal is to harness the protective biology observed in genetically resilient individuals and translate it into medicines that can delay or prevent disease in at-risk populations." About HT-4253 HT-4253 is a selective, orally administered LRRK2 kinase inhibitor that pharmacologically reduces phosphorylated Rab10 (pRab10), a biomarker associated with neuroinflammatory processes. Preclinical, genetic findings have suggested that some individuals who carry the high-risk APOE4 allele for Alzheimer's disease, but exhibit naturally reduced Rab10 activity, may correlate with cognitive resilience, highlighting the potential therapeutic rationale for targeting this pathway. This natural resilience suggests that dampening the LRRK2–Rab10 pathway may protect against the progression of neurodegeneration. By pharmacologically inhibiting LRRK2, HT-4253 reduces pRab10 levels, helping to suppress innate immune overactivation and lysosomal dysfunction. The therapeutic goal is to replicate the resilience seen in genetically protected individuals and apply it to patients at risk of Alzheimer's and other chronic neuroinflammatory conditions. About Halia Therapeutics Halia Therapeutics focuses on harnessing the biology of naturally resilient individuals to redefine treatment paradigms, aiming to restore immune balance in inflammatory and neurodegenerative conditions. Founded on breakthrough research identifying protective mutations in individuals genetically predisposed to severe diseases, Halia's therapies aim to restore immune balance in inflammatory and neurodegenerative conditions. The company's expanding pipeline includes: HT-6184 (Ofirnoflast): currently in a Phase 2a trial for Myelodysplastic Syndrome (MDS); enrollment is complete, with the study expected to conclude in late 2025. For more details, visit the clinical trial record: CTRI/2023/11/059758) HT-6184+Semaglutide: planned Phase 2a trial in patients with Obesity and Type 2 diabetes (T2D), expected to initiate in Q3 2025; HT-4253: neuroinflammation-targeted candidate now completed Phase 1, with a CSR expected in Q4 2025. For more information about HT-4253, Ofirnoflast (HT-6184), or ongoing clinical trials, please visit . Media Contact Taylor Avei Director of Business Development Halia Therapeutics [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

临床1期临床2期

2025-01-08

·PRNewswire

Halia Therapeutics to Attend the 43rd J.P. Morgan Healthcare Conference and Present at BioPharma Obesity Innovation Forum in San Francisco

LEHI, Utah, Jan. 8, 2025 /PRNewswire/ -- Halia Therapeutics, a biopharmaceutical company at the forefront of developing treatments for chronic inflammation and related disorders, announced today that its leadership team will attend and participate in a panel discussion during the 43nd J.P. Morgan Healthcare Conference (January 13 – 16, 2025), and will also present at the 1st Annual BioPharma Obesity Innovation Forum (January 11, 2025), both taking place in San Francisco. At The BioPharma Obesity Innovation Forum, Dave J. Bearss, Ph.D., President and CEO of Halia will be presenting data on its lead candidate, HT-6184, the first drug to target the protein NEK7 to inhibit NLRP3 inflammasome activity to resolve chronic inflammation for treating multiple diseases including the chronic inflammatory effects of obesity. Presentation details at BioPharma Obesity Innovation Forum: Title: Targeting Inflammation in Obesity: HT-6184 as a Synergistic Partner to GLP-1 Agonists for Enhanced Glucose Control and Weight Loss Date: January 11, 2025 Time: 11:40 a.m. PST Presenter: David J. Bearss, Ph.D., President and Chief Executive Officer, Halia Therapeutics Location: Marines' Memorial Club, San Francisco During the J.P. Morgan Healthcare Conference, Dave J. Bearss, Ph.D., President and CEO of Halia will be participating in an Executive Roundtable discussion at the 13th annual WuXi Global Forum 2025. Roundtable discussion details: Date: January 14, 2025 Time: 5:00 – 6:00 p.m. PST Presenter: David J. Bearss, Ph.D., President and Chief Executive Officer, Halia Therapeutics Location: Continental Ballroom 5, Ballroom Level of Tower 3, Hilton San Francisco Union Square, San Francisco David J. Bearss, CEO of Halia, alongside the management team, will be available for one-on-one meetings with investors and strategic partners at each of these conferences. Please contact Logan Berrett ([email protected]) for scheduling. 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 . Connect with us on LinkedIn and Twitter (X) for the latest updates. Company Contact Halia Therapeutics [email protected] +1 (385) 355-4315 Media Contact: Ignacio Guerrero-Ros, Ph.D. Russo Partners, LLC [email protected] +1 (646) 942-5604 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

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100 项与 Ofirnoflast 相关的药物交易

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研发状态

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适应症	最高研发状态	国家/地区	公司	日期
术后疼痛	临床2期	美国	Halia Therapeutics, Inc.	2024-02-12
术后炎症	临床2期	美国	Halia Therapeutics, Inc.	2024-02-12
贫血	临床2期	印度	Halia Therapeutics, Inc.	2023-12-09
骨髓增生异常综合征	临床2期	印度	Halia Therapeutics, Inc.	2023-12-09
炎症	临床1期	美国	Halia Therapeutics, Inc.	2022-06-16
眼部疾病	临床前	美国	Halia Therapeutics, Inc.	2024-10-11
血液疾病	临床前	美国	Halia Therapeutics, Inc.	2023-12-11

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研究	分期	人群特征	评价人数	分组	结果	评价	发布日期


NCT07052006 (ASH2025)	临床2期	贫血 \| 骨髓增生异常综合征	18	Ofirnoflast (Low-risk myelodysplastic syndrome with symptomatic anemia)	鹽餘選齋廠築獵淵鹹鬱(衊範鬱網憲遞構鹽膚範) = 齋壓衊獵網壓憲鹹範蓋構製鹽衊鑰餘簾醖獵糧 (獵膚窪窪鹹廠築鹽夢餘 )	积极	2025-12-06
NCT07052006 (HT-6184-MDS-001, ASH2025)	临床2期	骨髓增生异常综合征	23	Ofirnoflast	壓淵蓋夢願糧範憲遞壓(積範鹹夢衊構築艱蓋壓) = n=2 觸遞餘膚製衊鑰築餘壓 (壓醖餘鬱鏇觸願築獵膚 ) 更多	积极	2025-12-06
NCT07052006 (ASH2025)	临床2期	骨髓增生异常综合征	18	Ofirnoflast (HT-6184) 2 mg	膚觸壓醖觸簾餘製顧憲(繭鹹觸糧糧淵簾築獵獵) = 鹽廠廠繭觸鏇簾願構顧鬱構鬱蓋鹹範觸網醖範 (廠鏇網膚遞餘觸鬱膚襯 ) 更多	积极	2025-12-06
NCT05447546 (PRNewswire) 人工标引	临床1期	-	32	HT-6184 (SAD)	鹹夢顧齋觸餘夢醖壓獵(簾齋製憲遞廠蓋網蓋餘) = 襯壓獵餘壓鏇齋艱鑰艱顧簾製鑰壓製願窪齋繭 (選淵簾醖積簾膚鹽鬱製 ) 更多	积极	2023-11-29
NCT05447546 (PRNewswire) 人工标引	临床1期	-	32	placebo (SAD)	鹹夢顧齋觸餘夢醖壓獵(簾齋製憲遞廠蓋網蓋餘) = 鑰獵網選鹽齋繭廠鏇範顧簾製鑰壓製願窪齋繭 (選淵簾醖積簾膚鹽鬱製 )	积极	2023-11-29