What Halia Therapeutics does

Halia Therapeutics is a clinical-stage biopharmaceutical company founded in 2017 and headquartered in Lehi, Utah, that develops small-molecule therapies targeting chronic inflammation across hematologic, neurologic, metabolic, and oncologic diseases. The company's scientific thesis — branded "genetic resilience" — inverts traditional drug discovery by studying individuals who remain healthy despite carrying high-risk genetic variants, using population-scale pedigrees and genomic data (most notably the Utah Population Database) to identify protective mutations such as the RAB10 variant found in APOE4-carrying families that did not develop Alzheimer's disease. This insight underpins the LRRK2 → RAB10 → NEK7 → NLRP3 inflammasome axis that anchors Halia's pipeline.

The pipeline is led by ofirnoflast (HT-6184), a first-in-class oral allosteric NEK7 modulator that prevents NLRP3 inflammasome assembly. Ofirnoflast has demonstrated a 72% hematologic improvement-erythroid (HI-E) response rate and 67% overall hematologic improvement in Phase 2 lower-risk MDS, with FDA Orphan Drug Designation (Oct 2025) and Fast Track designation (Jun 2026); a global Phase 3 is planned for early 2026. Adjacent programs include HT-4253 (LRRK2 inhibitor for Alzheimer's in APOE4 carriers), HT-10068 (neuropenetrant NEK7 inhibitor for neuroinflammation/metabolic disease), and HT-6105 (hsCRP modulator for cardiovascular risk and inflammatory pain), supported by the GENMOR/GENMOR-AI discovery platform that the company claims reduces discovery timelines by ≥50% and time-to-Phase 2B to one-third the industry average.

Halia is pre-revenue. Its business model is to advance candidates through FDA-regulated clinical trials, secure regulatory approval, and commercialize either independently or via partnership/licensing deals. Near-term commercial pathway is centered on ofirnoflast in lower-risk MDS, with expansion optionality into obesity (HT-6184 + GLP-1 combinations) and Alzheimer's. Distribution today is limited to clinical trial sites (US and India), with a strategic international expansion underway through a partnership with the Department of Health – Abu Dhabi leveraging the Emirati Genome and through a Novo Nordisk Golden Ticket – BioLabs European Award for European lab presence.

Name

Halia Therapeutics

Legal name

Halia Therapeutics, Inc.

Website

https://haliatx.com

Company type

Private

Founded year

2017

Operating status

Operating

Headcount range

11–50 employees

Short description

Halia Therapeutics is a clinical-stage biopharmaceutical company developing small-molecule therapies that target the LRRK2 → RAB10 → NEK7 → NLRP3 inflammasome axis to treat inflammation-driven diseases including lower-risk MDS, Alzheimer's in APOE4 carriers, and obesity.

Ownership category

akta.pro rank

Product category

Clinical-Stage Biopharmaceuticals (Inflammation / Inflammasome Therapeutics)

NAICS

Research and Development in Biotechnology (except Nanobiotechnology) (541714), Biological Product (except Diagnostic) Manufacturing (325414)

SIC

Biological Products, (No Disgnostic Substances) (2836)

akta.pro primary industry

Translational & Pharmacodynamic Biomarkers (MOA, target engagement, PD/PK-linked markers) (HLAAAMAD)

akta.pro secondary industry

Predictive Biomarkers for Therapy Selection (response/non-response stratification) (HLAAAMAE)

Keywords

• Inflammasome inhibitor therapeutics
• Genetic resilience drug discovery
• Biopharmaceutical clinical development
• NLRP3 inflammasome modulation
• Human genetics drug discovery

Headquarters

HQ city

Lehi

HQ country

United States

HQ region

North America

Offices1 record

Markets served

GTM type

B2B

Offering type

Hardware or Manufacturing

Cost components

Technology or R&D, Personnel, Operations, Marketing or Sales, Others

Revenue model

1. Pharmaceutical Product Revenue: As a clinical-stage biopharmaceutical company, Halia Therapeutics does not currently generate revenue. The company's business model is to advance its pipeline through clinical development, secure regulatory approvals (FDA and international equivalents), and commercialize therapeutic products. Revenue will be generated through product sales following regulatory approval. The company is also pursuing partnership and licensing deals as a potential near-term revenue mechanism.

Go-to-market motion1 record

Distribution channels1 record

Marketing channels6 records

Core offering

Halia Therapeutics is a clinical-stage biopharmaceutical company developing resilience-based drug candidates that target the genetically validated LRRK2 → RAB10 → NEK7 → NLRP3 inflammasome axis to treat chronic inflammation-driven diseases. Its lead asset, ofirnoflast (HT-6184), is a first-in-class oral allosteric NEK7 modulator in Phase 2/3 development for lower-risk myelodysplastic syndromes, with additional programs in Alzheimer's (HT-4253), obesity, neuroinflammation (HT-10068), and cardiovascular/inflammatory pain (HT-6105). Discovery is powered by the proprietary GENMOR and GENMOR-AI platforms, which identify protective genetic modifiers in resilient individuals using the Utah Population Database.

Product overview

Halia Therapeutics is a clinical-stage biopharmaceutical company developing a pipeline of resilience-based therapies targeting inflammation-driven diseases. The company's core offerings include lead drug candidates Ofirnoflast (HT-6184) for myelodysplastic syndromes and HT-4253 for Alzheimer's disease in APOE4 carriers, supported by two AI-powered discovery platforms: the GENMOR Platform for identifying protective genetic modifiers and GENMOR-AI for accelerating target discovery. Additional programs include HT-10068 for neuroinflammatory/metabolic disorders and HT-6105 for cardiovascular risk and inflammatory pain. The company leverages human genetic resilience insights—studying individuals who remain healthy despite high-risk genetic variants—to develop more precise and safer therapeutics.

Differentiator

Problem solved

Functional benefit

Brands

• GENMOR Platform: Proprietary AI-powered discovery engine that identifies protective genetic modifiers in individuals who remain healthy despite carrying high-risk genetic variants
• GENMOR-AI™

Products and services

• Ofirnoflast (HT-6184) First-in-class selective oral allosteric NEK7 modulator designed to prevent formation and promote disassembly of the NLRP3 inflammasome complex. Targets underlying inflammatory drivers of hematologic diseases (lower-risk MDS) and is being evaluated in preclinical and clinical studies for obesity in combination with GLP-1 receptor agonists such as semaglutide. Administered orally at 2mg once daily, 5 days on / 2 days off.
• HT-4253 LRRK2 inhibitor designed for early intervention in APOE4 carriers — the highest genetic risk population for late-onset Alzheimer's disease. Based on protective RAB10 suppression observed in resilient APOE4+/+ individuals identified through the Utah Population Database; designed to reduce RAB10 phosphorylation (pRAB10) and provide neuroprotection before clinical symptoms manifest.
• HT-10068 Neuropenetrant NEK7 inhibitor targeting inflammation in neuroinflammatory conditions and metabolic disorders including Type 2 diabetes, MASH, and obesity. Phase 1 start targeted for 4Q26.
• HT-6105 Cardiovascular risk reduction and inflammatory pain program that modulates hsCRP (high-sensitivity C-reactive protein) to target cardiovascular inflammatory risk and inflammatory pain pathways. Currently in discovery.

Quantifiable outcome

• 72% hematologic improvement-erythroid (HI-E) response rate at Week 16 in lower-risk MDS patients (N=18 evaluable Stage 1 population)
• +4 more outcomes

Segments4 records

Ideal customer profiles4 records

Technology focussed Yes

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

AI capability3 records

Feature5 records

Partnerships

Two partnerships are on record, tiered core.

• Department of Health – Abu DhabicoreStrategic or Co-development Partner · 6 October 2025Strategic partnership leveraging the Emirati Genome dataset to discover genetic resilience factors that protect individuals from disease. Paul Jones, newly appointed as Chief Strategy Officer & GM International Markets, is key to advancing this partnership. The collaboration supports Halia's APOE4 Alzheimer's program by studying individuals who carry the highest-risk genetic variant but never develop the condition.
• Novo Nordisk / BioLabscoreGTM or Marketing Partner · 11 March 2025Awarded the Novo Nordisk Golden Ticket – BioLabs European Award for companies working on transformational cardiometabolic and obesity therapies. The award provides fully sponsored BioLabs lab space (one bench, one desk, one year membership) at BioLabs Heidelberg or Paris, with priority admission, access to shared research facilities, and engagement with Novo Nordisk's scientific and business executives. Supports Halia's expansion into the European biotech ecosystem.

Scale indicators10 records

Recent moves6 records

Expansion highlights7 records

Emerging players

• Alector: Clinical-stage neuroscience biotech developing immuno-neurology therapies for neurodegenerative diseases including Alzheimer's. Comparable to Halia's HT-4253 program targeting APOE4 carriers via genetically validated mechanisms.
• Denali Therapeutics: Biotech developing therapies for neurodegenerative diseases using genetics-driven target selection and engineered biological molecules. Comparable human-genetics-first approach to drug discovery, overlapping with HT-4253 in Alzheimer's.

Direct peers

• Exscientia: AI-driven precision medicine company using machine learning to accelerate small-molecule drug discovery. Comparable platform-based business model and inflammation/oncology therapeutic focus areas.
• Recursion Pharmaceuticals: AI-driven drug discovery platform combining high-throughput biology, machine learning, and genomics. Closely comparable to Halia's GENMOR-AI approach of integrating population-scale data with ML for target identification and prioritization.
• NodThera: UK-based clinical-stage biotech developing small-molecule NLRP3 inflammasome inhibitors for inflammatory and neurodegenerative diseases. Most direct mechanistic competitor to Halia's NEK7/NLRP3 axis, targeting overlapping indications including neurological and metabolic conditions.
• Inflazome (Roche): Acquired by Roche in 2020, Inflazome developed small-molecule NLRP3 inflammasome inhibitors. Represents a direct mechanistic competitor now backed by Roche's commercial and clinical development capabilities across inflammation-driven indications.
• Olatec Therapeutics: Clinical-stage biotech developing dapansutril and related NLRP3 inflammasome inhibitors for inflammatory diseases. Comparable mechanistic approach and clinical-stage profile to Halia.
• Insitro: AI/ML-driven drug discovery platform integrating human genetics, genomics, and cellular phenotyping to identify therapeutic targets. Highly comparable platform paradigm to Halia's GENMOR-AI genetic resilience approach.
• IFM Tre (Bristol Myers Squibb): Subsidiary of BMS (formerly IFM Therapeutics), developing NLRP3 and cGAS-STING pathway inhibitors. Direct mechanistic competitor with significantly greater capital and commercial infrastructure backing.

Broad incumbents

• Bristol Myers Squibb: Large pharmaceutical company with established MDS franchise (Reblozyl/luspatercept) and NLRP3 inflammasome assets via IFM Tre subsidiary. Represents both a potential commercial competitor in MDS and a likely partnership/licensing counterparty for Halia.

Market position

Strengths5 records

Weaknesses5 records

Competitive moat5 records

Key risks7 records

Key highlights7 records

Customer concentration

• Xx.com/Halia_Tx
• LinkedInlinkedin.com/company/halia-therapeutics-inc

Compliance2 records

Revenue estimate

Valuation estimate

Number of profiles

Profiles11 records

Funding overview

Funding rounds5 records

Investors

Funding detail is available on the Subscription and Enterprise plan.Contact sales →

M&A

Investments

M&A and investment is available on the Subscription and Enterprise plan.Contact sales →

Frequently asked questions about Halia Therapeutics