Breakthrough Findings with CQ-001

Validated In-Vivo Efficacy
Completed rodent studies at Western University and MES seizure modeling at the Ontario Brain Institute (OBI), demonstrating robust anticonvulsant activity in validated epilepsy models.

Increased Efficacy
Approximately 35% greater seizure protection compared to isolated cannabidiol (CBD) in preclinical refractory epilepsy models.

Improved Tolerability Profile
Designed to achieve therapeutic effect at lower cannabidiol exposure, supporting improved tolerability and reduced adverse event burden.

Translational De-Risking Strategy
Integrated animal efficacy data (Western University, OBI MES modeling), real-world neurological datasets, and AI-driven modeling to bridge preclinical findings to human clinical trials.

Focused Rare Pediatric Neurology Strategy

CQ-001 is a differentiated multi-pathway small-molecule neurotherapeutic for Developmental and Epileptic Encephalopathies (DEEs), initially targeting refractory drop and generalized seizures within genetically defined pediatric populations.

The formulation is engineered to address multiple mechanisms central to refractory epilepsies, including excitatory/inhibitory imbalance, neuroinflammation, and synaptic dysfunction.

Regulatory & Strategic Advantages

Capital-Efficient 505(b)(2) Pathway
Leverages established cannabidiol safety data to reduce development risk and compress timelines relative to traditional CNS programs.

Phase IIb Value Inflection
Current development plan is structured to reach Phase IIb readiness within ~12–15 months, representing the first proprietary human efficacy milestone.

Rare Pediatric Regulatory Positioning
Pursuing Rare Pediatric Disease designation with potential eligibility for a Priority Review Voucher (PRV), a historically monetizable non-dilutive asset with nine-figure precedent transactions.

AI-Driven Development Optimization
AI modeling supports formulation optimization and precision dosing as part of a disciplined, data-informed clinical strategy.

CNS: Central Nervous System