Light-activated AU-011 consists of viral nanoparticles, modeled on the human papillomavirus (HPV), conjugated to infrared-activated small molecules. AU-011 is administered through an intravitreal injection into the eye. The viral nanoparticle conjugates (VNCs) then bind selectively to cancer cells in the eye. Upon activation with an ophthalmic laser, the small molecules selectively destroy the membrane of cancer cells, killing them without damaging the overlaying retina and thus, potentially enabling vision preservation for patients.
In vivo results presented at the annual meeting of the Association for Research in Vision and Ophthalmology (ARVO) in 2015 and published in the International Journal of Cancer demonstrated how its synthetic viral nanoparticles modeled on the HPV are able to bind uniquely to cancer cells while leaving healthy surrounding tissue unharmed. The authors determined that tumor cells differ from healthy cells in the over-expression and modifications of heparan sulfate proteoglycans on their cell membrane, which provides a unique binding site for HPV virions and for engineered VNCs.
Light-activated AU-011 is currently being investigated in a Phase 1b/2 study for the treatment of small-to-medium primary OM and has been granted orphan drug designation by the U.S. FDA.