URB597 was previously approved for a phase I clinical trial by Health Canada and Exxel Pharma is working towards running a phase I clinical trial. The lead clinical indication is PTSD, an area with a significant unmet medical need and a market size projected to reach $1.7 billion by 2019. The Company is exploring additional clinical indications, including cannabis and opioid use disorders (dependence and addiction).
URB597 blocks the FAAH enzyme, similar to URB937, thereby preventing degradation of the endocannabinoid anandamide and leading to increased cannabinoid receptor signaling. In contrast to URB937, URB597 readily enters the CNS, which enables treatment of neurological disorders such as PTSD, addiction, anxiety and insomnia. Importantly, URB597 is non-addictive in preclinical models of addiction, which distinguishes the molecule from all other FAAH inhibitors in development. Both URB597 and follow-on molecules in this drug family are patented, and ready for commercialization. The molecules are very potent (nanomolar IC50) with good oral bioavailability (35-87%).