Our Pipeline

Five programs originating from the CompoundAI platform, at stages from discovery through Phase I clinical trials.

2
Phase I Programs
1
IND-Enabling
2
Preclinical Programs
4
Therapeutic Areas

Clinical Programs

Phase I
CAI-001 - Solid Tumor Oncology (KRAS G12C)

First-in-class small molecule KRAS G12C inhibitor for solid tumors with confirmed KRAS G12C mutation. The target was identified through CompoundAI's multi-omics target scoring system, which flagged the mutant protein as both highly disease-relevant and druggable following recent structural insights. Dose escalation is ongoing at 4 clinical sites across the US. Initial pharmacokinetic and safety data expected Q3 2026.

IND Filed: March 2025 | Sites: 4 | Status: Dose escalation | Data readout: Q3 2026
Phase I
CAI-007 - Neurodegeneration (Tau Aggregation)

Brain-penetrant tau aggregation inhibitor targeting early Alzheimer's disease. The compound emerged from a structure-based design campaign and demonstrated exceptional selectivity in the off-target profiling panel. The Phase I safety evaluation cohort is fully enrolled. Cognitive biomarker sub-study is ongoing. Primary safety data readout expected Q4 2026.

IND Filed: August 2025 | Cohort: Fully enrolled | Data readout: Q4 2026

Preclinical Programs

IND-Enabling
CAI-014 - Metabolic Disease (NASH / FGF21)

Novel FGF21 receptor agonist for non-alcoholic steatohepatitis (NASH). Robust efficacy data in two preclinical models. GLP toxicology studies currently underway. IND submission targeted for Q1 2027.

GLP Tox: In progress | IND target: Q1 2027
Lead Optimization
CAI-022 - Rare Disease (Lysosomal Storage Disorder)

Enzyme replacement therapy candidate identified through CompoundAI's generative chemistry module. Lead series shows potent enzyme activity restoration in patient-derived cell lines. Optimization ongoing to improve selectivity index before candidate nomination.

Lead series: Identified | Stage: Selectivity optimization
Discovery
CAI-031 - Autoimmune (Inflammatory Bowel Disease)

Undisclosed mechanism. Target validation studies complete. High-throughput phenotypic screening initiated. Hit-to-lead progression expected to begin Q2 2026.

Target: Validated | HTS: Initiated

Pipeline information reflects the current status as of April 2026. Clinical stage data is preliminary and subject to update. Nothing on this page constitutes an investment recommendation or clinical advice. Programs may be discontinued, modified, or out-licensed at any time.