Apterna’s pipeline is built around precision delivery and clinically meaningful outcomes. Our lead programme is advancing toward first-in-human readiness in severe liver disease, with the goal of restoring hepatic resilience and supporting real-world access and durability for established therapies, including GLP-1R+ regimens. In parallel, we are progressing a second platform designed for systemic delivery across the blood-brain barrier to address CNS-involved oncology and neurological indications. Every step is milestone-led, data-driven, and focused on high-impact unmet need.
From Discovery to Translation: Building the Next Generation of RNA Therapeutics
A Portfolio Anchored in Precision Delivery
Apterna’s programmes are built around two validated, organ-targeted delivery platforms:
Liver-targeted platform
Focused on restoring hepatic resilience and improving metabolic health, with the goal of expanding real-world eligibility and durability for GLP-1R+ therapy in patients whose liver dysfunction limits treatment options.
Brain-targeted platform
Designed for systemic, non-invasive transport across the blood-brain barrier to deliver RNA payloads to the CNS, supporting partnering opportunities in CNS oncology and brain metastases.
Both platforms are non-viral, chemically defined, and engineered for scalable development and safety. Our objective is to progress from robust preclinical validation to IND and CTA readiness and early clinical translation.
Hepatocyte Uptake
GalNAc liver delivery pathway
BBB Transport
Receptor-guided brain delivery pathway
Apterna's Development Programmes
Current outline of our active and emerging programmes. Timelines reflect planned progression from preclinical to early clinical development.
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Apterna Development Programmes
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| Programme | Target Organ | Therapeutic Focus | Indication | Stage | Next Milestone |
|---|---|---|---|---|---|
| APX-101 | Liver | Restoration of liver function, supporting GLP-1R+ eligibility and durability | Severe liver disease, including advanced hepatic impairment (GLP-1R+ enablement) | Late Preclinical | IND and CTA-enabling studies |
| APX-102 | Liver | Metabolic modulation designed for combination potential with GLP-1R+ therapies | MASLD, MASH and NASH (GLP-1R+ combination and durability) | Preclinical Discovery |
Candidate selection |
| APX-201 | Brain | RNA delivery across BBB | Brain metastases | Preclinical Validation |
Biodistribution studies |
| APX-202 | Brain | Neuroprotection | CNS indications | Discovery | Platform optimisation |
| APX-301 | Multisystem | Platform co-development | Partner-led indications | Strategic | Out-licensing |
APX-101: Liver Restoration in Advanced Liver Disease
Pioneering a Functional Rescue Approach
Rationale:
- Severe liver failure remains largely untreatable beyond transplantation
- Many systemic oncology regimens cannot be safely administered in patients with compromised hepatic function
- Even modest improvements in liver function markers may restore treatment eligibility and expand access to established therapies, including GLP-1R+ regimens where liver status limits real-world use
Programme Objectives:
- Demonstrate safe, hepatocyte-selective delivery with low systemic exposure
- Improve key liver function markers in relevant preclinical models
- Generate IND and CTA-ready regulatory packages to support first-in-human evaluation
Development Process
- Organ-selective delivery
- Liver function rescue
- IND and early clinical translation
Programme Objectives:
- GLP toxicology and biodistribution package (2025 to 2026)
- IND and CTA submission (2027)
- First-in-human study with liver function endpoints and GLP-1R+ enablement hypothesis (2027 to 2028)
Metabolic Intervention
Lipid accumulation → RNA intervention → Restored balance
APX-102: Fatty Liver and Metabolic Dysfunction
Addressing the Global Epidemic
Fatty liver disease is highly prevalent and continues to rise alongside obesity and metabolic dysfunction. APX-102 is designed to modulate hepatocyte pathways that drive lipid accumulation and inflammatory signalling, with the goal of reducing hepatic fat burden and slowing progression across the MASLD to MASH spectrum, including settings where liver status limits the real-world safety and durability of GLP-1R+ therapy.
Scientific Rationale:
Apterna’s liver delivery platform enables selective targeting of metabolic pathways within hepatocytes, helping minimise systemic exposure that has constrained many prior metabolic liver programmes.
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Demonstrate meaningful reduction in hepatic lipid content and inflammatory markers
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Evaluate dosing durability, repeat-dose tolerability, and longer-term liver safety
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Position for expansion into obesity-associated liver dysfunction and GLP-1R+ combination strategies
Current Stage: Early preclinical exploration with candidate optimisation ongoing. Mechanistic validation planned for 2025–2026.
APX-201: Reaching the Brain Without Surgery
Our second technological pillar — a receptor-guided RNA delivery platform capable of crossing the blood–brain barrier after systemic administration.
Non-Viral System
Fully synthetic with proven brain penetration
RNA Modalities
Compatible with silencing, activating, coding
Partnership Ready
Extensive licensing potential
Key Highlights:
- Utilises a fully synthetic, non-viral system with proven in-vivo brain penetration
- Compatible with multiple RNA modalities (silencing, activating, coding)
- Offers extensive potential for licensing and co-development in oncology and neurology
Current Work:
- Ongoing preclinical validation with academic partners
- Biodistribution and brain uptake studies
- Expanding platform data package for partnerships in 2026
Strategic Roadmap Toward First-in-Human Studies
Preclinical Validation (APX-101, APX-201)
IND/CTA-Enabling Studies & Manufacturing Scale-up
Regulatory Submission & Phase 1b Study Initiation
Early Clinical Readout & Platform Licensing
Collaborate With Us to Advance Precision RNA Medicine
Apterna offers multiple engagement models for pharmaceutical and academic partners
Co-Development
Joint work on specific disease indications
Platform Licensing
Access to liver or brain delivery systems for proprietary RNA payloads
Collaborative Research
Shared preclinical studies to accelerate validation
Our goal is to build partnerships that expand the therapeutic reach of RNA medicine while maintaining rigorous scientific and ethical standards.
From Precision Science to Real-World Impact
Apterna’s pipeline is built to deliver measurable patient benefit through safe, targeted RNA delivery. By combining validated chemistry, scalable design, and translational expertise, we aim to move beyond theoretical potential — toward a new generation of clinically meaningful RNA therapeutics.