Aura Biosciences NASDAQ: AURA highlighted progress across its ocular oncology pipeline and provided updates on a parallel effort in bladder cancer during a fireside chat at TD Cowen’s 46th Annual Healthcare Conference. Founder and CEO Eli de los Pinos said the company’s lead program in early-stage choroidal melanoma is enrolling well in Phase 3, while upcoming data in bladder cancer and additional ocular indications are expected to broaden the company’s opportunity.
Phase 3 choroidal melanoma: enrollment on track, top-line data targeted for Q4 2027
De los Pinos said Aura’s Phase 3 trial of bel-sar in early-stage choroidal melanoma is “enrolling very well,” and he reiterated guidance for top-line data in Q4 2027. He added that the company has “a high degree of confidence” it will complete enrollment in 2026.
He described the company’s Phase 2 dataset as a major strength, citing results in patients with actively growing lesions. In those treated with what he characterized as the full therapeutic regimen, he said 80% achieved complete cessation of tumor growth. By contrast, he said patients who received subtherapeutic dosing (one or two injections versus three cycles) showed the opposite pattern, with 80% progressing—what he described as a “20–80 delta” and a “60% delta in response” in Phase 2. He also emphasized that adverse events in that study were “literally grade 1.”
SPA details: time to tumor progression as primary endpoint and a masked, three-arm design
The CEO said Aura has a Special Protocol Assessment (SPA) from the FDA that was important because the program uses endpoints common to ocular oncologists but less familiar to regulators. Under the SPA, the Phase 3 primary endpoint is time to tumor progression, which he described as a “clean oncology endpoint” comparing progression events in bel-sar arms versus a sham (observation) arm.
He also explained that the Phase 3 includes three arms. A middle arm was included to support masking, because he said the FDA wanted a design where the treating physician would not know whether a patient received high-dose or low-dose bel-sar. He noted that the middle arm is not part of the statistical analysis.
On trial power, de los Pinos said the Phase 2 data would imply very high statistical power for the primary endpoint, but he said Aura took a more conservative approach when powering the Phase 3 by assuming a smaller treatment effect than observed in Phase 2. He also discussed a key secondary endpoint designed to capture visual acuity preservation alongside tumor control, noting that approval is driven primarily by the time-to-progression endpoint.
Market dynamics: a shift toward earlier treatment if vision-preserving therapy becomes available
De los Pinos argued that the addressable market in choroidal melanoma could expand if physicians have an option that preserves vision, because today many patients are observed rather than treated until disease progression forces intervention. He said the frequently cited figure of about 2,000 ocular melanomas reflects treated cases, while in large practices a larger group may be observed (he described this as roughly “3x” observed versus “1/3” treated).
He outlined a current pathway in which patients are first identified by ophthalmologists or optometrists, then referred to ocular oncologists, where treatment often involves radiotherapy that can lead to vision loss. He said bel-sar’s proposed role is to avoid radiotherapy rather than reduce its use.
Competition and pricing commentary
Asked about other development efforts, de los Pinos contrasted Aura’s strategy with IDEAYA’s, describing IDEAYA as positioning a systemic treatment as neoadjuvant to radiotherapy in more advanced disease, while Aura is aiming to replace radiotherapy in earlier-stage patients. He also noted Immunocore’s role in metastatic disease, which he characterized as a separate setting with different prescribers (medical oncologists versus ocular oncologists).
On pricing, he said analyst models generally assume an ultra-orphan pricing corridor. He referenced Immunocore’s launch price of $1.5 million per patient as a “bookend,” and suggested there is room to price competitively within an ultra-orphan framework, particularly if bel-sar extends across multiple ocular oncology indications.
Bladder cancer: mid-year update expected from neoadjuvant-focused study
De los Pinos identified bladder cancer as an area where the data are strong but still early due to sample size. He argued Aura is differentiated by pursuing a neoadjuvant approach, while he said other companies focus on adjuvant treatment. He said the company expects a data update mid-year designed to show durability and support the positioning of bel-sar in the treatment landscape.
Discussing prior Phase 1 findings, he said a single dose feasibility study allowed the company to biopsy tumors before and after complete responses to evaluate mechanism and potential reasons for an abscopal effect. He highlighted immune profiling results that included:
- A reported 40x increase in NK cell population
- A reported 7x increase in cytolytic CD4 T-cells
- Evidence consistent with tertiary lymphoid structure formation, which he associated with adaptive immune response
He also emphasized tolerability, saying there were no grade 2 or grade 3 adverse events and only grade 1 toxicities, and argued this could support combination use without adding significant systemic toxicity.
On the ongoing study design, he said Aura moved quickly to dose escalation after discussions with the FDA and is now treating multiple lesions and using multiple cycles (day 1 and day 7) to “prime-boost” immune response. He described three key cohorts running in parallel, including 200 micrograms per lesion and 400 micrograms per lesion dosing, with two cycles, and plus/minus surgery. He said the mid-year release could include 15–20 patients of data across intermediate- and high-risk cohorts.
He described a proof-of-concept bar as demonstrating an impact on recurrence-free survival at 12 months in 15–20 patients, and said the company expects three-month and 12-month readouts to be important for assessing competitiveness and differentiation. On market size, he cited about 80,000 intermediate-risk patients and noted a smaller funnel in higher-risk subgroups, mentioning about 4,000 patients in CIS.
Beyond choroidal melanoma and bladder cancer, de los Pinos also said Aura expects data this year in choroidal metastases and cancers of the ocular surface. He described the choroidal metastases study as a dose escalation effort where proof of concept would be tumor shrinkage and visual acuity preservation in a small number of patients, and he said those data could align with a future BLA timeline and potentially support a “very quick second indication” via a supplemental filing strategy.
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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