Halia Therapeutics Pipeline:

Genetic Resilience-Based Drug Development

Advancing Clinical Programs in Alzheimer’s, MDS, Obesity, and More

At Halia Therapeutics, our approach to drug discovery is driven by a groundbreaking idea: genetic resilience—the natural ability of certain individuals to resist or recover from disease.

Our clinical and preclinical programs are built on this foundation, harnessing insights from real-world human biology to develop therapies that target the underlying drivers of chronic conditions. From Alzheimer’s disease and myelodysplastic syndromes (MDS) to obesity and systemic inflammation, our pipeline reflects a bold new way of thinking—turning resilience into medicine.

Clinical-Stage Drug Candidates Based on Human Biology

Every therapy in our pipeline begins with the GENMOR Platform — our proprietary, AI-powered engine that identifies protective genetic modifiers in individuals who remain healthy despite carrying high-risk genetic variants.

By decoding these natural defenses, we design therapeutics that mimic resilience at the molecular level, leading to treatments that are more precise, biologically validated, and inherently safer.

HT-6184 for Myelodysplastic Syndromes (MDS)

Disease Area: Oncology
‍Stage: Phase 2a Clinical Trial
‍Milestone: Phase 2a Completion Expected in 3Q25
‍Mechanism of Action: NEK7 modulation to suppress NLRP3 inflammasome-driven inflammation

HT-6184 is being developed for patients with lower-risk myelodysplastic syndromes (MDS), a chronic blood disorder characterized by ineffective hematopoiesis and limited non-toxic treatment options. By targeting the innate immune dysfunction that drives disease progression, HT-6184 aims to reduce inflammation in the bone marrow, potentially slowing disease progression and improving hematologic outcomes.

HT-6184 + Semaglutide for Obesity

‍Disease Area : Metabolic Disease

‍Stage : Phase 1 Completed

‍Milestone : Phase 2 Trial Begins in 2Q25

‍Differentiator : In preclinical models, HT-6184 combined with semaglutide preserves lean mass and enhances GLP-1 efficacy at lower doses.

HT-6184, when combined with semaglutide, addresses a critical gap in obesity care: maintaining muscle mass during weight loss. While GLP-1s drive significant fat loss, they often lead to depletion of lean tissue and treatment discontinuation. HT-6184 targets metaflammation—the chronic, low-grade inflammation associated with obesity—enhancing metabolic outcomes while helping patients retain muscle and stay on therapy for longer.

HT-4253 for Alzheimer’s Disease in APOE4 Carriers

Disease Area : Neurology

‍Stage : Phase 1 Completed

‍Milestone : Phase 2 Trial Begins in 3Q25

‍Origin : Based on protective RAB10 suppression seen in resilient APOE4+/+ individuals.

HT-4253 is the first therapeutic program specifically designed for early intervention in APOE4 carriers—individuals with the highest genetic risk for Alzheimer’s disease. Inspired by real-world cases of genetic resilience, HT-4253 mimics natural protective mechanisms to delay disease progression and modify the trajectory of neurodegeneration before clinical symptoms fully manifest.

Early-Stage Resilience-Based Programs

Each program builds on a shared mechanism: regulating the NLRP3 inflammasome, a key driver of chronic inflammation.

HT-6283 —
Neuroinflammation

  • Phase 1 Start: 4Q25
  • Targets inflammation pathways in neurodegenerative conditions

HT-6184 —
Ophthalmology

  • Phase 1 Start: 4Q25
  • Investigating local delivery to reduce retinal inflammation

HT-6515 —
Dermatology

  • Phase 1 Start: 1Q26
  • Designed for chronic skin conditions driven by innate immune dysregulation

Clinical-Stage Drug Candidates Based on Human Biology

Halia’s clinical team is led by scientists and doctors with deep expertise and experience in the design and execution of clinical trials to explore the activity of our product pipeline. We have translated the compounds to demonstrate their activity in assays and in vivo models, which are readying them for clinical development.

Halia’s investigational drugs are at an early stage of development. We cannot offer expanded access (“compassionate use”) to its investigational drugs outside of a clinical trial. For more information, please read our full expanded access policy.

More information about Halia’s clinical trials can be found at clinicaltrials.gov.

See MDS Clinical Trial
See HT-4253 Phase 1 Clinical Trial
See Obesity Clinical Trial
Halia Therapeutics Policy for Expanded Access to Investigational Drugs

Halia is committed to developing novel, meaningful medicines for patients with serious diseases who may benefit from new treatment options. Halia’s current policy for evaluating and responding to requests for individual patient access to investigational drugs intended to treat serious diseases is found below.

Our product pipeline consists of investigational medications currently being tested in clinical trials and have not yet been approved by the US Food and Drug Administration (FDA). Halia’s investigational drugs are currently at an early stage in development, and we are is currently focused on enrolling patients in our clinical trials and continuing to learn more about our investigational drugs’ safety and efficacy. We encourage patients to speak with their physicians about their eligibility for enrollment in any of Halia’s clinical trials whenever possible. Our clinical trials are designed, conducted, and monitored to advance the development of our investigational drugs and make them more broadly available to patients in the future. Additional information about Halia’s ongoing clinical trials is available at https://clinicaltrials.govusing the search term “Halia.”

CURRENTLY, HALIA CANNOT MAKE EXPANDED ACCESS AVAILABLE TO ITS INVESTIGATIONAL DRUGS.

Halia may revise this expanded access policy at any time in the future, and this posting will be updated should there be any policy change. For more information regarding our investigational drugs or questions about participation in one of our clinical trials, please submit a request to info@haliatherapeutics.com. Halia personnel will acknowledge receipt of any expanded access questions within ten business days of receipt. For additional information on expanded access to investigational drugs, visit the FDA’s website.