Aura Biosciences (NASDAQ:AURA) is advancing what it describes as a novel class of therapies called virus-like drug conjugates (VDCs), with its lead program in ocular oncology now in a Phase 3 trial for early choroidal melanoma. Speaking at Leerink Partners’ Global Healthcare Conference in Miami, CEO Elisabet de los Pinos said the company is approaching a key milestone of completing enrollment in the pivotal study and outlined additional ocular oncology and bladder cancer development plans that could generate multiple data readouts.
Virus-like drug conjugates and bel-sar’s dual mechanism
De los Pinos said Aura’s VDC platform uses virus-like particles targeted to cancer cell membranes and conjugated to a payload—“in this case, in our first VDC, a photosensitizer.” She added that each particle carries “200 molecules of the photosensitizer.” In ocular tumors, the VDC is injected locally and requires activation with infrared light to trigger production of reactive oxygen species at the cancer cell membrane, leading to necrosis through what she called an “ablative mechanism.”
Phase 3 in early choroidal melanoma: enrollment focus and timelines
The company’s Phase 3 program in early choroidal melanoma is Aura’s “flagship” effort to support approval, and de los Pinos emphasized the importance of finishing enrollment. She said the trial has a Special Protocol Assessment (SPA), which she framed as making enrollment completion a central milestone for the company.
According to de los Pinos, patients in Aura’s Phase 2 and Phase 3 studies are early choroidal melanoma cases that would typically be eligible for observation for a period before radiotherapy, rather than immediate treatment. She said that dynamic enabled the FDA to allow randomization against a sham control, because these patients are commonly observed for several months given the risk of vision loss from radiotherapy. She also said the trial uses an enrichment strategy requiring “a certain threshold of active growth” so that sham-controlled patients are expected to progress within “6–9 months,” which she called important for powering the study.
On timing, de los Pinos reiterated that Aura has guided to top-line data in Q4 2027. She explained the analysis is driven by a time component tied to the last patient reaching 15 months of follow-up, noting that many patients will have 15–24 months of follow-up by the time the study is read out. While she did not provide a specific enrollment completion date during the discussion, she said the company was “reaffirming guidance of end of enrollment much earlier than year-end,” describing previous guidance as conservative and citing ongoing enthusiasm and momentum in recruitment.
Market sizing and treatment adoption considerations
De los Pinos estimated the early choroidal melanoma opportunity at roughly 8,000 patients across the U.S. and Europe (about 4,000 each). She said only a portion are treated today—estimating closer to 2,000—with many managed under “watchful wait” and often coded as “high-risk indeterminate lesions” rather than melanoma due to the lack of a vision-preserving early intervention option. She characterized this as “purely semantics,” arguing that genetic mutations are present earlier than documented growth and that, with an approved therapy, physicians would treat earlier and classify more patients as early choroidal melanoma.
She also addressed referral dynamics, saying some patients can remain in observation for 9–12 months and in some cases up to two years before being referred, potentially narrowing the window for intervention. Commercially, she said Aura expects uptake could extend beyond ocular oncologists to retina specialists, particularly because the therapy is local and, in her view, has a strong safety profile. She suggested patients may prefer local treatment if retina specialists can perform the administration and light activation.
Additional ocular oncology studies and a broader ocular oncology ambition
Beyond early choroidal melanoma, de los Pinos outlined two additional ocular oncology programs:
- Metastasis to the choroid (Phase 2): A proof-of-concept study in patients whose primary cancers (she highlighted metastatic breast cancer as common) spread to the eye. She said these patients are typically treated with ocular radiation despite systemic therapy, and that Aura is aiming to preserve vision while treating tumors. She said this study is expected to provide proof-of-concept data “this year,” ahead of the 2027 melanoma readout.
- Cancers of the ocular surface: Including melanomas at the front of the eye and squamous cell carcinomas of the conjunctiva. She described current approaches as surgically challenging, sometimes involving chemotherapy eye drops such as mitomycin or 5-FU, with high recurrence rates and some patients ultimately losing the eye. Aura plans an approach that can include intralesional dosing and post-treatment surgery to assess response in a “window of opportunity” design.
De los Pinos said that, in aggregate, these ocular oncology indications represent a potential 66,000-patient opportunity, breaking out estimates of about 20,000 for metastasis to the choroid and 35,000 for ocular surface cancers, in addition to the 8,000 early choroidal melanoma estimate. She also discussed a strategy of leveraging a successful Phase 3 melanoma program and shared safety database to support expansions via supplemental filings.
Bladder cancer: early signals, neoadjuvant positioning, and mid-year update
De los Pinos said Aura has initiated development in bladder cancer, emphasizing recurrence as a key problem, particularly in intermediate-risk disease. She described a strategy centered on neoadjuvant use ahead of transurethral resection of bladder tumor (TURBT), arguing this could expose tumor neoantigens to the immune system while fitting into existing urology workflows.
She said the company has developed a formulation that does not require refrigeration, biosafety level-2 handling, or general anesthesia, and could be administered shortly after diagnosis with two administrations before a scheduled TURBT. She also highlighted that TURBT can provide confirmatory assessment and biomarker readouts via immune profiling.
In discussing early data, de los Pinos referenced a feasibility study using a single dose of drug (from the ocular program) to treat a single lesion. In intermediate-risk disease, she said four of five patients had complete responses, including resolution of untreated lesions, which she described as an abscopal effect. She also said the company observed complete responses and abscopal effects in high-risk patients, along with strong tolerability.
Looking ahead, she said Aura has optimized dosing to two cycles and is evaluating immunoablative and neoadjuvant approaches in intermediate-risk disease, with only neoadjuvant cohorts in high-risk. She said the company expects three-month data by mid-year across cohorts totaling 21 patients, with durability and longer follow-up—particularly at 12 months—viewed as important for differentiating the approach. De los Pinos said the goal is to reduce the need for “heavy adjuvant treatments” and chemotherapy exposure in intermediate-risk patients by pairing a neoadjuvant approach with TURBT to support longer durability.
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.
