Biotech firm Aura Biosciences has received orphan drug designation from the US Food and Drug Administration (FDA) for its drug, AU-011, to treat patients with uveal melanoma.

Uveal melanoma is also referred to as ocular melanoma (OM) and is an aggressive form of cancer that develops in the uvea or uveal tract of the eye.

The company has also presented the AU-011 pre-clinical research in-vivo data at the Association for Research in Vision and Ophthalmology Annual Meeting.

The study demonstrated that the drug is efficacious for treating uveal melanoma tumours in an orthotopic xenograft model.

“There are currently no approved drug therapies for the treatment of uveal melanoma, which is a rare but life-threatening disease.”

Aura Biosciences founder and CEO Elisabet de los Pinos said: “There are currently no approved drug therapies for the treatment of uveal melanoma, which is a rare but life-threatening disease.

“We are thrilled to receive this orphan drug designation that, together with the positive preclinical data, is enabling us to move this drug one step closer to the clinic.”

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The AU-011 includes viral-like Nanoparticle that show highly selective targeting of solid tumours while leaving normal epithelium untouched.

Aura’s drug integrates its viral-like Nanoparticle conjugated with a potent cell-killing laser-activated molecule that is delivered efficiently and selectively to cancerous cells, allowing to reduce or eliminate the risk of non-specific activity and undesirable toxicity.

According to the company, the molecule is activated by a standard ophthalmologic laser and treatment will be provided in an outpatient visit.

Image: Photograph of an iris melanoma. Photo: courtesy of Jonathan Trobe, MD, University of Michigan Kellogg Eye Center.