One Platform.
Multiple Assets.
Scalable Innovation.
Onconox is redefining medicine development,
creating safer, more effective therapies for patients with cancer and neurodegeneration.
Our Pipeline
We are building a next-generation pipeline driven by the BIG Platform, our modular innovation engine for precision drug delivery.
By reformulating validated small molecules and pairing them with advanced delivery or targeting technologies, we create therapies designed to reach the right tissue, at the right concentration, with fewer systemic side effects.
Our pipeline spans oncology and CNS disorders, united by a single philosophy:
local delivery, improved tolerability, and faster translational progress.
Pipeline Overview
Our pipeline is structured around three precision delivery approaches:
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Inhaled Delivery :
For targeted lung exposure in respiratory and thoracic cancers. -
Intranasal Delivery :
For direct nose-to-brain access in CNS tumors and
neurological disease. -
Direct-to-Tumor Delivery :
For local administration into or around solid tumors.
Pipeline Snapshot
Program | Delivery | Indication | MoA | Stage |
|---|---|---|---|---|
Pipeline Snapshot
Program | Delivery | Indication | MoA | Stage |
|---|---|---|---|---|
The BIG Platform
Onconox’s BIG Platform is the foundation of our innovation model. It enables us to create multiple therapeutic candidates from a single, well-characterized small molecule, enhanced through selective delivery technologies or targeting mechanisms.
At its core, the BIG Platform provides:
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A unified pharmacological backbone
Allowing predictable biology, established safety, and strong translational rationale.
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Precision delivery options
Including inhaled, intranasal, and direct-to- tumor routes designed to maximize local tissue exposure.
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Optional targeted enhancements
Using molecular tags or tumor-associated features to improve selectivity and activity within diseased tissue.
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A scalable development engine
Where formulation learnings, regulatory frameworks, and CMC processes are reused across programs, reducing timelines and development risk.
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Built-in optionality across therapeutic areas
From NSCLC to GBM to neurodegeneration, the BIG Platform enables a multi-asset pipeline from one backbone. This model gives us platform-level efficiency, multiple clinical “shots on goal”, and a sustainable path
to long-term growth.
Program | Formulations / Indications | Discovery | Preclincial | IND-Enabling | Ph I | Ph II | Ph III |
|---|---|---|---|---|---|---|---|
The Pipeline
AI at the Core of Smarter, Faster Therapeutics
Artificial intelligence plays a central role across all our development programs. While we do not disclose the specifics of our models or proprietary workflows, AI is embedded into the BIG Platform as a strategic enabler at every stage — helping us move faster, make better decisions, and reduce the inherent risks of drug development.
Enhancing Prioritization and Early Insight
AI supports the way we identify and evaluate opportunities, rapidly analyzing scientific literature, biological pathways, and emerging evidence to help us focus on the most promising disease targets and delivery routes.
Strengthening Mechanistic Foundations
We use AI to deepen our understanding of disease biology, therapeutic mechanisms, and delivery relevance. This helps us validate the scientific rationale behind each program and build a stronger foundation ahead of IND-enabling studies.
Mapping Indications with Platform Potential
AI helps us explore new oncology and CNS indications that share molecular or pathway characteristics suited to our precision delivery approaches, allowing the BIG Platform to scale into areas of high unmet need.
Informing Combination Strategy Discovery
By analyzing large pharmacology datasets and mechanistic patterns, AI supports the identification of potential therapeutic combinations that may enhance or complement our precision-delivered assets.
Accelerating Evidence Generation
AI enables faster, more efficient synthesis of available data, helping us identify contradictions, gaps, and key questions early. This ensures our scientific team focuses on the highest-value work from the outset.
The Pipeline
OCN001 – Our Lead Program
OCN001 is being developed as a first-in- class inhaled therapy designed to enhance the effectiveness of radiotherapy for patients with non–small cell lung cancer.
As the first program emerging from the BIG Platform, it reflects the full strength of ourdevelopment philosophy: targeted delivery, improved tolerability, and rapid clinical translation.
Addressing a Critical Need in Lung Cancer
Lung cancer remains one of the most challenging and lethal cancers worldwide, and radiotherapy is a core component of treatment for the majority of patients. Despite this, there are few therapeutic options specifically designed to improve the effectiveness of radiation, and current systemic agents often bring significant toxicity burdens.
OCN001 aims to address this gap by delivering therapy directly to the lungs, where it can act at the site of disease while minimizing systemic exposure.
Our Approach: Precision Inhaled Delivery
OCN001 uses targeted inhalation to achieve high local concentrations within the tumor microenvironment, supporting enhanced response to radiotherapy. By focusing therapy where it is needed most, this approach is designed to:
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Increase local therapeutic activity
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Reduce off-target toxicity
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Improve treatment tolerability
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Support better adherence during radiotherapy
This targeted route underpins the rationale for progressing OCN001 as the first clinical candidate within our portfolio.
Why OCN001 Leads Our Portfolio
OCN001 was selected as our first clinical program because it is uniquely positioned to demonstrate the strengths of the BIG Platform:
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Clear unmet need in a large global cancer population
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Strong biological rationale for combining with radiotherapy
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Highly localized route of administration
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Favorable safety expectations from platform pharmacology
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Straightforward pathway into early clinical studies
Success with OCN001 is expected to accelerate the progression of our follow-on assets, providing a template for platform-wide execution.