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.
Expanded Access Policy
Aura Biosciences is committed to developing safe and effective therapies to transform the lives of patients with ocular cancers. Our goal is to successfully advance and seek marketing approval for innovative ocular cancer treatments and to provide patients with broad access to our future products. We are currently conducting clinical trials to assess the safety and effectiveness of our first investigational drug candidate in human subjects.
Expanded Access, also known as Compassionate Use, refers to a pathway in which patients with serious or immediately life-threatening diseases or conditions may gain access to an investigational product outside the context of participation in clinical trials. Providing an investigational product as part of an Expanded Access Program is a complex matter and is different from studying the investigational product as part of a clinical trial, where more comprehensive safety and efficacy data are collected. We understand the need for Expanded Access Programs under certain circumstances, and we recognize the importance of each clinical trial sponsor thoughtfully considering all the issues and having an appropriate policy in place.
Consistent with the Food and Drug Administration (FDA’s) and other regulatory agencies’ guidelines regarding when Expanded Access may be appropriate, several factors should be considered. Those factors include, but are not limited to:
The illness must be serious or life-threatening with no other satisfactory treatment options (such as approved products or enrolling clinical trials).
There is sufficient evidence that the potential benefit to the patient would likely outweigh the potential risks, based on available safety and efficacy information on the investigational product.
The sponsor’s ability to provide a product in a fair and equitable manner, so that there is adequate manufacturing capacity for ongoing clinical programs.
Whether granting expanded access to certain patients would potentially compromise the scientific validity of broader development programs or interfere with or delay current clinical trials or regulatory filings designed to make the therapy available to many more patients.
At this time, Aura Biosciences believes that participation in one of our clinical trials is the most appropriate way to access our investigational therapy for ocular cancer, and therefore none of our investigational products are available under expanded access programs.
Aura Biosciences may revise this policy at any time. We continue to push forward in our development programs to deliver innovative products to the largest number of patients in the shortest amount of time.
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)