Aura Biosciences Gears Up for Bel-Sar Filing as Pivotal Eye Cancer Trial Advances

Key Points

• Aura Biosciences is nearing completion of enrollment in its registrational CoMpass study of bel-sar for early choroidal melanoma, and management is shifting focus to data collection, BLA preparation and commercialization planning. Top-line results remain on track for the second half of 2027.
• Phase 2 data showed promising tumor control and vision preservation, with 80% tumor control and 90% visual acuity preservation at 12 months in the efficacious dose range, alongside a favorable safety profile using suprachoroidal delivery. Aura said the trial has a special protocol assessment that supports registration if results are replicated.
• The company sees a sizable unmet need and little direct competition in early choroidal melanoma, estimating about 8,000 patients per year across the U.S. and five major European markets. Aura also said its recent financing leaves it with $355 million in cash and runway into the second half of 2028.

Aura Biosciences NASDAQ: AURA is shifting its focus toward regulatory preparation and commercialization planning as enrollment in its registrational CoMpass study of bel-sar in early choroidal melanoma nears completion, newly appointed CEO Natalie Holles said during a fireside chat at TD Cowen’s seventh annual Oncology Innovation Summit.

Holles said she joined Aura because of what she described as an “underappreciated market opportunity” for bel-sar, the company’s lead therapy, in early choroidal melanoma, a rare ocular cancer with limited treatment options. She said the drug’s emerging profile, combined with a lack of competing development programs in the same setting, made the opportunity compelling.

“This was a unique opportunity,” Holles said. “What really piqued my interest was the potential, the compelling value proposition of bel-sar as a new treatment for a patient population that is really desperately in need of better treatment options.”

CoMpass Study Moves Toward Data Collection and BLA Preparation

Holles said Aura is in the process of wrapping up enrollment in CoMpass, its registrational study of bel-sar for early choroidal melanoma. With that milestone, she said the company’s attention is turning to completing data collection, preparing a biologics license application and planning for a potential commercial launch.

The CoMpass study is a three-arm trial enrolling approximately 100 patients with early choroidal melanoma. The primary efficacy analysis evaluates an 80-microgram dose of bel-sar against a sham injection, using a 15-month landmark analysis of time to tumor progression. Holles said halting tumor growth is considered a functional cure in this disease setting.

A key secondary endpoint is a composite of time to tumor progression or visual acuity failure, whichever occurs first. Holles said vision preservation is important both for the U.S. Food and Drug Administration’s ophthalmology division and for future commercial positioning, particularly against radiotherapy.

Aura has received a special protocol assessment for the trial design. Holles said the SPA covers the study’s design, size and efficacy and safety analyses needed to support registration based on CoMpass results. She added that the company remains on track for top-line data in the second half of 2027.

Holles said that if the phase 3 trial recapitulates the phase 2 result of an 80% response rate in arresting tumor growth, the primary efficacy analysis is more than 99% powered.

Phase 2 Data Highlight Tumor Control and Vision Preservation

Holles reviewed previously reported phase 2 results for suprachoroidal bel-sar in choroidal melanoma. In the efficacious dose range of the ascending-dose study, Aura observed 80% tumor control and 90% visual acuity preservation at 12 months, along with what Holles characterized as a favorable safety profile.

She contrasted those results with the current treatment landscape, saying patients are generally managed either through watchful waiting, which carries the risk of metastatic disease, or radiotherapy, which can control tumor growth but may lead to blindness in many patients.

Holles also discussed Aura’s earlier experience with intravitreal administration of bel-sar, which she said produced posterior inflammation that affected safety and efficacy. Moving to suprachoroidal delivery “largely” eliminated that posterior inflammation, she said, with mostly grade 1 adverse events that resolved with no or short-term treatment. She said there were no treatment-related serious adverse events and no grade 3 to 5 treatment-related serious adverse events.

Market Opportunity and Competitive Landscape

Holles said Aura estimates early-stage choroidal melanoma incidence at about 8,000 patients per year across the U.S. and the five largest European markets, based on current diagnosis and treatment paradigms. She said patients are typically diagnosed during routine eye exams, often after a spot is observed on the retina.

Holles said the distinction between indeterminate lesions and small choroidal melanomas often depends on treatment decisions by physicians, but she described the broader group as early choroidal melanoma patients who may be eligible for bel-sar if risk factors are present.

Asked about competition, Holles said there are no other competitors in early choroidal melanoma. She noted that some treatments are being developed or used for metastatic uveal melanoma, but said bel-sar is the only treatment currently being evaluated in the early choroidal melanoma setting.

Aura has not provided pricing guidance for bel-sar. Holles said pricing would be informed by phase 3 results, the product profile, the lack of other treatment options and the size of the patient population.

Expansion Programs in Ocular Oncology

Beyond early choroidal melanoma, Holles said Aura plans to invest more meaningfully in ongoing studies of bel-sar in choroidal metastases and ocular surface cancers following a recent financing.

For choroidal metastases, Holles described the disease as metastases from other primary tumors, often breast or lung cancer, that appear in the choroid. She said current care can require daily external beam radiation for four weeks, creating a high burden for patients who are already very sick.

Aura’s phase 2 choroidal metastases study is a standard 3+3 dose-escalation study assessing changes in tumor size and visual acuity outcomes. Holles said the study is enrolling and remains on track for early data later this year.

In ocular surface cancers, Aura is conducting an initial safety and feasibility study using intratumoral injection to the front of the eye, along with a laser directed at the anterior surface. The trial is beginning in Australia, where Holles said incidence is higher due to greater ultraviolet radiation exposure. She estimated about 35,000 patients per year are diagnosed with ocular surface cancers.

Cash Runway Extends Beyond CoMpass Readout

Holles said Aura completed a follow-on financing about a month before the conference. Including those proceeds, she said the company’s pro forma cash balance was $355 million as of March 31, providing runway into the second half of 2028.

She said the runway extends beyond the expected top-line CoMpass data readout and should fund the company through several anticipated catalysts over the next 24 months.

About Aura Biosciences NASDAQ: AURA

Aura Biosciences is a clinical‐stage biopharmaceutical company focused on the development of novel virus‐like particle (VLP) therapies for the treatment of cancer. By combining proprietary VLP technology with photoactivatable dyes, Aura aims to deliver highly selective photodynamic therapies that target and destroy tumor cells while sparing healthy tissue. The company's platform is designed to address solid tumors in both ophthalmic and non‐ophthalmic settings, leveraging precision activation via near‐infrared light to induce localized tumor cell apoptosis and stimulate anti‐tumor immune responses.

The lead product candidate, AU-011, is being evaluated in patients with choroidal melanoma, a rare but potentially sight-threatening eye cancer.

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