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  • New Eye Cancer Therapy Shown to Target Cancer Cells, Spare Vision

    New study shows AU-011 may be an effective first-line therapy for early-stage choroidal melanoma

    NEW ORLEANS, La. – Choroidal melanoma is a rare cancer that affects the back of the eye. If the tumor grows, it can cause the retina to detach, leading to vison loss. And if it spreads to other parts of the body, it can be fatal. There are no approved drugs that can stop its spread, and the available treatment options commonly lead to serious vision loss or loss of the eye. But a new laser-activated nanoparticle promises to target and destroy the cancer cells and preserve vision. A study presented today at AAO 2021, the 125th annual meeting of the American Academy of Ophthalmology, shows that it may be an effective first-line therapy for early-stage choroidal melanoma.

    AU-011 (belzupacap sarotalocan) is a viral-like nanoparticle that is injected into the eye, and then activated with an ophthalmic laser. The nanoparticles are modeled on the human papillomavirus and bind specifically to heparin-sulfated proteoglycans expressed by ocular melanoma cells. After being activated by an ophthalmic laser, the nanoparticles disrupt the tumor cell membrane, which kills the cancer cells, leaving healthy surrounding tissue unharmed. It also activates the immune system to create long lasting anti-tumor immunity.

    Data from a Phase 1b/2 trial showed a significant reduction in tumor growth over the 12-month study (-0.445 mm/yr, p=0.018), while tumor control was achieved in 60 percent of patients. At the same time, 73 percent maintained their visual acuity (best corrected visual acuity loss <15 letters). Among 56 treated subjects, two with juxtafoveal tumors had treatment-related serious adverse events of vision loss. Adverse events, Intraocular inflammation and IOP increase, were transient and clinically manageable in most subjects.

    “The final safety and efficacy data from the 12-month Phase 1b/2 trial presented today, along with the Phase 2 SC data, provide a high level of confidence for the advancement of AU-011 to the pivotal program in patients with small choroidal melanoma,” said Carol Shields, MD, Chief of the Ocular Oncology Service at Wills Eye Hospital and Professor of Ophthalmology at Thomas Jefferson University. “I believe AU-011 may offer patients a safe, effective first-line therapy for early-stage choroidal melanoma that preserves vision, a critical component in patients’ quality of life often neglected with today’s current treatment options.”


    About the American Academy of Ophthalmology

    The American Academy of Ophthalmology is the world’s largest association of eye physicians and surgeons. A global community of 32,000 medical doctors, we protect sight and empower lives by setting the standards for ophthalmic education and advocating for our patients and the public. We innovate to advance our profession and to ensure the delivery of the highest-quality eye care. Our EyeSmart® program provides the public with the most trusted information about eye health. For more information, visit