12 October 2021 - - US-based clinical-stage oncology company Aura Biosciences has presented interim Phase 2 data with seven months average follow up evaluating the safety of suprachoroidal administration of AU-011, the company's lead product candidate for the first-line treatment of primary choroidal melanoma, as a part of the American Society of Retina Specialists 2021 annual meeting, the company said.

There have been no related serious adverse events, dose limiting toxicities, or grade 3 adverse events observed during the study.

The Phase 2 trial is comprised of an open-label, dose escalation phase and a randomized, masked dose expansion phase that is assessing the safety and efficacy of ascending single- and repeat-doses of AU-011 via SC administration, followed by one or two laser applications per treatment.

The randomized, dose expansion portion will be masked, sham-controlled and is designed to evaluate the safety and efficacy of the highest dose regimen of AU-011. Cohorts 1-5 have been fully enrolled (13 patients) and cohort 6 is currently enrolling in the Phase 2 study.

The primary objective of the study is to assess safety and efficacy of AU-011 via SC administration for purposes of treating primary indeterminate lesions and choroidal melanoma.

The randomized phase of the trial is planned to begin in the second half of 2022 in patients with documented growth to establish the safety and efficacy of AU-011 and serve as the first pivotal trial for the treatment of indeterminate lesions and choroidal melanoma.

The maximum treatment regimen anticipated for the randomized phase is three cycles of three weekly treatments of AU-011 at a dose of 80µg with 2 laser administrations.

AU-011 is a first-in-class virus-like drug conjugate therapy in clinical development for the first line treatment of choroidal melanoma.

The virus-like component of the VDC selectively binds unique heparin sulphate proteoglycans (HSPGs), which are modified and overexpressed on the tumor cell surface of malignant cells in the choroid and AU-011 delivers a potent cytotoxic drug that is activated with infrared light.

Upon activation with an ophthalmic laser, the cytotoxic drug rapidly and specifically disrupts the cell membrane of malignant cells with a pro-immunogenic cell death that can activate the immune system generating long term anti-tumor immunity.

The unique specificity of tumor binding by the VDC enables the preservation of key eye structures, which may allow for the potential of preserving patients' vision and reducing other long-term complications of radiation treatment.

AU-011 can be delivered using equipment commonly found in an ophthalmologist's office and does not require a surgical procedure, pointing to a potentially less invasive, more convenient therapy for patients and physicians.

AU-011 for the treatment of choroidal melanoma is currently in Phase 2 clinical development and the company plans to expand the clinical program into choroidal metastasis.

The suprachoroidal space injection treatment approach offers unprecedented access to the back of the eye where sight-threatening disease often occurs.

Aura believes that delivering AU-011 into SCS within the eye, has the potential to offer certain advantages, including higher bioavailability at the tumor site and reduced exposure of non-targeted tissues, which may lead to an improved therapeutic index for AU-011.

Collectively, these features could allow for the treatment of a wider range of tumor sizes, and, therefore, a larger number of patients.

The company is partnered with Clearside Biomedical for use of Clearside's SCS Microinjector for administration of AU-011 into the SCS.

In preclinical research presented as part of the ARVO 2020 virtual program, AU-011 showed excellent distribution in the SCS, complete necrosis of tumors following laser activation in an animal model of choroidal melanoma and no clinical signs of anterior segment or posterior segment inflammation.

Aura Biosciences, Inc. is a clinical-stage oncology company developing a novel technology platform based on virus-like drug conjugates to target and destroy cancer cells selectively while activating the immune system to create long lasting anti-tumor immunity.