Proof Point
ABCC1 Program (C-001)
- Serves as first clinical validation of transporter activation in Alzheimer’s disease
- Demonstrated CNS target engagement and biomarker response
- Establishes mechanistic blueprint applicable across the evolutionarily conserved ABC transporter family
- De-risked biology, assays, and regulatory path
Platform Expansion Logic
Systematic expansion from ABCC1 to additional ABC transporters enables a multi-asset pipeline across neurodegeneration and other chronic diseases.
Transporter Expansion
- Systematic screening of additional ABC transporters
- Selection driven by human genetic causality, not hypothesis alone
- Each transporter treated as an independent, patentable drug class
Indication Expansion
- Alzheimer’s disease and tauopathies
- Other neurodegenerative and clearance-related disorders
- Potential extension into non-CNS indications
Pipeline Outcomes
- Multiple parallel discovery programs
- Portfolio of first-in-class or best-in-class small molecules
- Continuous generation of partnerable assets
Proof Point
ABCC1 Program (C-001)
- Serves as first clinical validation of transporter activation in Alzheimer’s disease
- Demonstrated CNS target engagement and biomarker response
- Establishes mechanistic blueprint applicable across the evolutionarily conserved ABC transporter family
- De-risked biology, assays, and regulatory path
Platform Expansion Logic
Systematic expansion from ABCC1 to additional ABC transporters enables a multi-asset pipeline across neurodegeneration and other chronic diseases.
Transporter Expansion
- Systematic screening of additional ABC transporters
- Selection driven by human genetic causality, not hypothesis alone
- Each transporter treated as an independent, patentable drug class
Indication Expansion
- Alzheimer’s disease and tauopathies
- Other neurodegenerative and clearance-related disorders
- Potential extension into non-CNS indications
Pipeline Outcomes
- Multiple parallel discovery programs
- Portfolio of first-in-class or best-in-class small molecules
- Continuous generation of partnerable assets