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 Completed; Finalized CSR expected 2Q26.
‍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-4253 for Alzheimer’s Disease in APOE4 Carriers

Disease Area: Neuroinflammation
‍Stage: Phase 2a Clinical Trial
‍Milestone: Phase 1 Completed;
‍Mechanism of Action: LRRK2 inhibition to reduce RAB10 phosphorylation (pRAB10), aligned with Halia’s genetic resilience findings linking reduced RAB10 signaling to neuroprotection in Alzheimer’s disease.

HT-4253 is being developed as a neuroprotective therapy for individuals at risk for Alzheimer’s disease, grounded in Halia’s human genetic resilience findings that reduced RAB10 signaling is associated with protection from disease. By inhibiting LRRK2 to lower RAB10 phosphorylation (pRAB10), HT-4253 is designed to modulate a genetically validated pathway implicated in disease initiation, with the goal of preserving neuronal function and slowing or preventing progression.

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-10068 —
Neuropenetrant NEK7 Inhibition

  • Phase 1 Start: 4Q26
  • Targets inflammation in metabolic disorders, including Type 2 Diabetes, MASH & Obesity.

HT-6105 —
Cardiovascular risk & Inflammatory Pain

  • Phase 1 Start: 4Q26
  • Modulates hsCRP to target cardiovascular risk reduction & inflammatory pain  

    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
    Halia Therapeutics Policy for Expanded Access to Investigational Drugs

    Halia Therapeutics, Inc. (Halia) is a clinical-stage biopharmaceutical company committed to developing novel, meaningful medicines for patients with serious diseases who may benefit from new treatment options.

    CURRENTLY, NONE OF HALIA’S INVESTIGATIONALPRODUCTS ARE AVAILABLE FOR EXPANDED ACCESS.

    In general, all new drug product candidates must be reviewed and approved by the U.S. Food and Drug Administration (FDA) before they can become commercially available in the United States for treating appropriate patients. Drugs that have not been approved by the FDA may or may not be effective as a treatment and may cause unexpected serious side effects.

    Our product development pipeline consists of investigational therapeutics currently being tested in clinical trials and have not yet been approved by the FDA. Clinical trials are controlled research studies designed to evaluate whether a new therapy is safe and effective for patients, and they provide the most direct opportunity for patients to access a new product before regulatory approval. Halia’s investigational products are currently at an early stage of development, and we are currently focused on enrolling patients who meet the eligibility criteria for our sponsored clinical trials and continuing to learn more about our investigational products’ safety and efficacy. As a general policy, Halia's investigational products are currently accessible only to subjects participating in a Halia-sponsored clinical trial.

    In contrast, expanded access, also known as “compassionate use,” is a process by which a limited number of qualifying patients may be granted access to an investigational product on a case-by-case basis. Requests for expanded access must be initiated by a treating physician and generally apply to patients who do not qualify for clinical trials, have exhausted all available treatment options, and have an immediately life-threatening condition or serious disease or condition, among other criteria. You can learn more about expanded access by visiting the FDA website at Expanded Access | Information for Patients  

    While we are mindful of the serious impact that these devastating diseases have on those affected, and we are committed to completing the regulatory review and approval process through ourongoing clinical trials, we are not in a position tooffer Sponsor-initiated expanded access to our investigational products outside of our active clinical trials. We encourage patients to speak with their physicians about their eligibility for participation in any of Halia's clinical trials whenever possible. Additional information about Halia's clinical trials is available on ClinicalTrials.gov.
     
    Although we do not currently offer sponsor-initiated expanded access to our investigational products, we may offer such access in the future. Halia may revise this expanded access policy at any time in the future. The posting of this policy does not guarantee access to any specific investigational product for any individual.

    For more information regarding our investigational products or questions about our clinical trials, please submit a request to info@haliatx.com. Halia personnel will acknowledge receipt of any expanded access questions within ten business days of receipt.

    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.