Aura Biosciences (AURA) investor relations material

Pipeline and clinical progress

• Lead drug is a virus-like drug conjugate (VDC) in phase III for early choroidal melanoma, with additional development in two other ocular oncology indications and bladder cancer.

• Phase III trial for early choroidal melanoma is the flagship program, with end of enrollment as a key milestone and top-line data expected in Q4 2027.

• The VDC platform uses a dual mechanism: direct tumor ablation via light-activated photosensitizer and immune activation, showing abscopal effects in both preclinical and early clinical settings.

• Additional phase II studies are ongoing in metastasis to the choroid and cancers of the ocular surface, with proof-of-concept data expected before the melanoma readout.

• Bladder cancer program targets intermediate and high-risk patients, focusing on neoadjuvant and immunoablative approaches, with promising early results and further data expected mid-year.

Market opportunity and commercial strategy

• Early choroidal melanoma represents 8,000 patients in the US and Europe, with the potential to expand as new therapies enable earlier intervention.

• The total addressable market in ocular oncology is estimated at 66,000 patients, including 20,000 with metastasis to the choroid and 35,000 with cancers of the ocular surface.

• Current standard of care for early choroidal melanoma is radiotherapy, which often leads to blindness; the new therapy aims to preserve vision and expand treatment to patients currently under observation.

• Commercial success depends on educating retina specialists and ocular oncologists to adopt early intervention, leveraging a buy-and-bill model and rare disease pricing.

• Regulatory strategy leverages enrichment trial design, with confidence that FDA approval will allow a broad label beyond actively growing lesions, based on precedent.

Forward-looking statements and milestones

• End of enrollment for the phase III melanoma trial is expected much earlier than year-end, with top-line data in Q4 2027 and a 15-month follow-up period.

• Proof-of-concept data for metastasis to the choroid and ocular surface cancers anticipated this year, potentially supporting supplemental BLA filings in subsequent years.

• Bladder cancer program will report three-month data for 21 patients by mid-year, with longer-term durability and biomarker data to follow.

• The company aims to own the ocular oncology space by expanding indications and leveraging the same drug and physician network.

• Investor attention is expected to increase as key milestones approach, particularly end of enrollment and upcoming data readouts.