On October 3, 2022 Aura Biosciences Inc. (NASDAQ: AURA), a clinical-stage biotechnology company developing a novel class of virus-like drug conjugate (VDC) therapies for multiple oncology indications, reported that interim Phase 2 data evaluating the safety and efficacy of suprachoroidal (SC) administration using its first VDC product candidate, belzupacap sarotalocan (AU-011), for the first-line treatment of patients with early-stage choroidal melanoma (indeterminate lesions and small choroidal melanoma (IL/CM)), were presented at the American Academy of Ophthalmology (AAO) 2022 Annual Meeting held September 30-October 3, 2022, in Chicago, IL (Press release, Aura Biosciences, OCT 3, 2022, View Source [SID1234621632]).

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"The Phase 2 interim safety and efficacy data that was presented at AAO is very encouraging for patients with primary choroidal melanoma, as the majority of patients are diagnosed with early-stage disease and have no vision-preserving treatment options. Interim data showed a statistically significant reduction in tumor growth rate and a robust tumor control response with a high rate of visual acuity preservation at the therapeutic regimen," said Dr. Ivana Kim, Director of the Ocular Melanoma Center, Massachusetts Eye and Ear. "Belzupacap sarotalocan offers a favorable safety profile supporting the potential to become the first vision-preserving treatment for early-stage choroidal melanoma, where patients have had to rely on radiotherapy for the last few decades."

"Preliminary analysis of the data from the Phase 2 trial using suprachoroidal administration supports tolerability up to three cycles of therapy and shows a dose-dependent anti-tumor response. The results provide further clinical evidence to support the potential use of belzupacap sarotalocan as a novel targeted therapy in patients with early-stage disease with this targeted route using suprachoroidal administration," said Dr. Cadmus Rich, Chief Medical Officer and Head of R&D of Aura Biosciences. "We believe that the data to date provides proof of concept for an additional intraocular route of administration and further supports belzupacap sarotalocan’s target product profile."

The presentation can be accessed on the Company’s website: link

Interim Safety and Efficacy Data from the Ongoing Phase 2 Trial with SC Administration

This Phase 2 trial (NCT04417530) is assessing the safety and preliminary efficacy of single- and multiple ascending-doses of belzupacap sarotalocan up to three cycles of treatment via SC administration for the first-line treatment of early-stage choroidal melanoma (IL/CM). A total of 20 adult patients have been enrolled in the trial including the single dose Cohorts 1-3 (n=6) and multiple dose escalation Cohorts 4-6 (n=14). Cohorts 5 and 6 received up to three cycles of therapy, which was considered the therapeutic regimen for evaluation. One patient in Cohort 5 (n=3) received two cycles of therapy and two patients in Cohort 5 received three cycles of therapy (40 µg/dose). All patients from Cohort 6 (n=8) received three cycles of therapy at the highest dose (80 µg/dose). One patient from Cohort 6, who discontinued after

one cycle due to unrelated serious adverse events (SAEs), is not included. All patients in Cohorts 5 and 6 had active growth at study entry, as an enrichment strategy to evaluate preliminary efficacy. This group of patients with active growth treated at the therapeutic regimen of three cycles was evaluated for tumor growth rate, tumor control, and visual acuity preservation as the defined clinical endpoints to evaluate preliminary efficacy. These endpoints have been discussed with the U.S. Food and Drug Administration and are planned to be used in the pivotal program. The results, with an average of six months follow up in patients that received three cycles of therapy in Cohorts 5 and 6, showed a statistically significant reduction in the tumor growth rate (-0.296 mm/yr, p = 0.0007) compared to each patient’s documented growth rate at study entry, and an 88.9% (8/9) tumor control rate. In addition, the visual acuity preservation rate was 88.9% (8/9) in these cohorts, with the majority of patients being at high-risk for vision loss with tumors close to fovea or optic disk. The overall safety profile of belzupacap sarotalocan was generally favorable, with no dose-limiting toxicities or treatment-related SAEs reported as of August 19, 2022. There was no posterior inflammation and only mild anterior inflammation (Grade 1) in 20% of the patients. Treatment-related AEs were predominantly mild and resolved without sequalae. We believe these interim results indicate that belzupacap sarotalocan may offer a targeted vision preserving therapy for the first-line treatment of primary CM, where 80% of patients are diagnosed early and have no approved therapies to date.

Details for the Virtual Investor Day:

The Company will host a virtual Investor Day today at 11:30 a.m. Eastern Time to discuss belzupacap sarotalocan, its first VDC product candidate, for the first-line treatment of patients with early-stage choroidal melanoma. The Company’s executive management team will be joined by three distinguished ocular oncology thought leaders:

Carol Shields, MD, Chief of the Ocular Oncology Service at Wills Eye Hospital and Professor of Ophthalmology at Thomas Jefferson University (USA)

Ivana Kim, MD, MBA, Director of the Ocular Melanoma Center, Massachusetts Eye and Ear & Associate Professor of Ophthalmology, Harvard Medical School (USA)

Martine Jager, MD, PhD, Professor of Ophthalmology, Leiden University (Netherlands) & Past President of the International Society of Ocular Oncology and the Association for Research in Vision and Ophthalmology

To access the virtual Investor Day, please dial (888) 660-6585 (U.S. and Canada) or (929) 203-0858 (international) at least 10 minutes prior to the start time and refer to conference ID 9748492. A live video webcast will be available in the Investor section of the Company’s website at View Source A webcast replay will also be available on the corporate website at the conclusion of the call.