Our goal in ocular
melanoma is early diagnosis and treatment
Our lead product candidate, light-activated AU-011, has the potential to be the first targeted therapy ever developed for the primary treatment of ocular melanoma (OM), also known as choroidal or uveal melanoma, the most common primary cancer of the eye.
Our goal is to create a safe and effective way of eliminating cancer cells early in the disease course with the selectivity of molecular surgery, preserving vision and transforming the treatment of ocular melanoma into a routine outpatient procedure.
Unlike most other cancers, ocular melanoma is often detected in its earliest stages of growth, through a routine ophthalmological exam. Just as the timely detection and removal of skin lesions has prevented countless cases of skin cancer from advancing, so too can an effective early intervention radically reshape the treatment of OM.
Today, people diagnosed with OM confront an array of poor treatment options, which often result in severe vision loss, removal of the eye, and in about half of all cases, metastasis to the liver, where the disease is nearly always fatal.
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.
We are committed to supporting patients with ocular melanoma (OM) and encourage patients to stay connected with each other as well as with the broader medical community:
- National Cancer Institute, National Institutes of Health
- CURE OM (Community United for Research and Education of Ocular Melanoma)
- OMF (Ocular Melanoma Foundation)
- A Cure in Sight
- National Organization for Rare Diseases (NORD)