OXU-003 is a potential first-in-class registered treatment for retinoblastoma.

OXU-003 is an innovative stable, sustained-release preparation of a proprietary chemotherapy agent. It is designed to be safely delivered in the suprachoroidal space, adjacent to primary retinoblastoma tumors using Oxulumis microcatheter. Once delivered, OXU-003 is designed to release the anti-cancer drug over prescribed treatment cycles.

The US FDA granted the OXU-003 program both Rare Pediatric Disease and Orphan drug designations, which may provide opportunities for accelerated regulatory pathways, as well as potential economic incentives upon approval.

Retinoblastoma is the most common ocular cancer in children and typically develops in children before the age of five, with an estimated 8,000 new cases each year. Half of children affected will succumb to the disease due to metastatic spread. Current treatments include intra-arterial chemotherapy and systemic chemotherapy. These treatments are known to have serious adverse effects, including retinal toxicity, and diffuse neurotoxicity presenting often as deafness, stroke, and cognitive impairment.