Halia Therapeutics Completes First-in-Human Phase 1 Study of HT-4253

Halia Therapeutics Completes First-in-Human Phase 1 Study of HT-4253

By, Admin 17 June 2025

Overview

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 (SAD/MAD) trial

  • 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.

Words from the CEO: Halia Therapeutics

  • 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.

HT-4253: Mechanism of action

  • 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 www.haliatx.com.

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