Caladrix’s scientific platform centers on ATP-binding cassette (ABC) transporters. For Alzheimer’s disease, a validated efflux mechanism responsible for clearing neurotoxic proteins, inflammatory molecules, and metabolites from brain tissue is in development. Unlike antibody therapies that rely on immune mechanisms and carry the risk of ARIA (Amyloid-Related Imaging Abnormalities), Caladrix enhances a specific ABC-transporter function through small molecules – most notably its lead compound, C-001. This non-immunologic, ARIA-free approach restores physiological protein clearance, reduces neuroinflammation, and reinstates cellular homeostasis. Human and animal data, along with genetic validation from over 730,000 individuals, validated the target ABC-transporter as a causal and protective factor in Alzheimer’s disease.

Caladrix’s lead therapeutic candidate, C-001, is an orally available ABC modulator targeting early-stage Alzheimer’s disease. The development program is informed by preclinical efficacy data, translational biomarker findings, and human proof-of-concept studies. C-001 is entering a Phase 1 trial in Q4 2025, followed by a pivotal Phase 2 study. Additional candidates are in development for frontotemporal dementia (FTD) and other tauopathies, including modified-release oral formulations for global scalability and patient adherence.