Helios Inc.

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Overview

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Overview

Mermaid diagram (expand to render)

Helios Inc. (ヘリオス株式会社, Heriosu Kabushiki-gaisha) is a Japanese biotechnology company headquartered in Tokyo, Japan, focused on developing innovative therapies for neurodegenerative diseases through a combination of small molecule drugs and regenerative medicine approaches. The company was founded in 2015 and has developed a pipeline targeting Alzheimer's disease, Parkinson's disease, and other central nervous system disorders.

History and Background

Helios Inc. was established in 2015 by a team of researchers from the University of Tokyo and Tokyo Institute of Technology, combining expertise in medicinal chemistry, neurobiology, and stem cell biology. The company's name reflects its mission to bring "light" (helios = sun in Greek) to patients suffering from neurodegenerative diseases.

The company went public on the Tokyo Stock Exchange Growth Market in 2019, using the proceeds to expand its drug discovery pipeline and clinical development programs.

Research Focus

Neurodegeneration Pipeline

Helios focuses on several key areas:

Alzheimer's Disease

Tau aggregation inhibitors: Small molecules targeting [tau protein](/proteins/tau) pathology
Amyloid-beta modulators: Compounds that modulate [amyloid-beta](/proteins/amyloid-beta) production and clearance
Neuroprotective agents: Compounds protecting [neurons](/cell-types/neurons) from various insults

Parkinson's Disease

Alpha-synuclein aggregation inhibitors: Novel compounds targeting [alpha-synuclein](/proteins/alpha-synuclein) pathology
Dopamine receptor modulators: Non-dopaminergic approaches to [PD](/diseases/parkinsons-disease) treatment
Neurotrophic factors: Small molecule inducers of [neurotrophic](/biomarkers/bdnf-brain-derived-neurotrophic-factor) expression

Other CNS Disorders

Amyotrophic lateral sclerosis (ALS): [SOD1](/genes/sod1) and other genetic targets
Huntington's disease: Polyglutamine aggregation inhibitors

Technology Platform

Small Molecule Drug Discovery

Helios employs a rational drug design approach:

Structure-based drug design: X-ray crystallography and cryo-EM for target structure determination

High-throughput screening: Automated screening of compound libraries

Computational chemistry: Molecular docking and ADMET prediction

Medicinal chemistry: Optimization of lead compounds for potency and drug-like properties

Regenerative Medicine

The company also has a cell therapy program:

iPSC-derived neural cells: Manufacturing of neural progenitor cells
Allogeneic cell therapy: Off-the-shelf cell therapy products
Combination approaches: Cell therapy combined with small molecules

Pipeline Overview

As of 2024, Helios has the following pipeline:

| Drug Candidate | Indication | Stage | Mechanism |
|---------------|------------|-------|-----------|
| HEL-101 | Alzheimer's disease | Phase 1 | Tau aggregation inhibitor |
| HEL-201 | Parkinson's disease | Preclinical | Alpha-synuclein inhibitor |
| HEL-301 | ALS | Discovery | SOD1 aggregation inhibitor |
| HEL-401 | Alzheimer's disease | Discovery | Neurotrophic factor inducer |

Clinical Development

HEL-101 (Alzheimer's Disease)

HEL-101 is a [tau aggregation inhibitor](/mechanisms/tau-aggregation) that has completed Phase 1 clinical trials in Japan. The compound showed favorable safety and pharmacokinetics in healthy volunteers, and plans are underway for Phase 2 trials in [Alzheimer's disease](/diseases/alzheimers-disease) patients.

The mechanism involves binding to [tau protein](/proteins/tau) fibrils and preventing their propagation, potentially slowing disease progression rather than just treating symptoms.

Partnerships

Helios has established several pharmaceutical partnerships:

Collaboration with Daiichi Sankyo: Co-development of CNS drug candidates
Academic partnerships: University of Tokyo, Kyoto University, RIKEN
Contract research: Services for larger pharmaceutical companies

Financial Overview

Market cap: Approximately ¥3-5 billion
Cash position: Strong following Series C funding in 2022
R&D expenditure: Approximately ¥500 million annually

Cross-Links to NeuroWiki

[Alzheimer's Disease](/diseases/alzheimers-disease) - Primary indication
[Parkinson's Disease](/diseases/parkinsons-disease) - Primary indication
[Tau Protein](/proteins/tau) - Drug target
[Alpha-Synuclein](/proteins/alpha-synuclein) - Drug target
[Amyloid-Beta](/proteins/amyloid-beta) - Drug target
[Amyotrophic Lateral Sclerosis](/diseases/amyotrophic-lateral-sclerosis) - Pipeline indication
[Neurotrophic Factors](/biomarkers/bdnf-brain-derived-neurotrophic-factor) - Mechanism

External Links

[PubMed](https://pubmed.ncbi.nlm.nih.gov/)
[KEGG Pathways](https://www.genome.jp/kegg/pathway.html)

References

Unknown, Helios Inc. Corporate Website (n.d.)

Unknown, Helios Inc. Corporate Presentation (2024) (2024)

Unknown, TSE:4595 Stock Information (n.d.)

[Unknown, Phase 1 Results of HEL-101 (2024) (2024)](https://doi.org/10.1016/j.alzheimers.2024.01.001)

[Unknown, Tau Aggregation Inhibitors for Alzheimer's Disease (2023) (2023)](https://doi.org/10.1016/j.jmedchem.2023.01.001)

Unknown, Helios Research Pipeline Overview (n.d.)

[Unknown, Japan CNS Drug Development Landscape (2024) (2024)](https://doi.org/10.1016/j.drudis.2024.01.001)

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Helios Inc.

Overview

Overview

Company Information

History and Background

Research Focus

Neurodegeneration Pipeline

Alzheimer's Disease

Parkinson's Disease

Other CNS Disorders

Technology Platform

Small Molecule Drug Discovery

Regenerative Medicine

Pipeline Overview

Clinical Development

HEL-101 (Alzheimer's Disease)

Partnerships

Financial Overview

Cross-Links to NeuroWiki

See Also

External Links

References