Aura Biosciences (AURA) TD Cowen 46th Annual Health Care Conference summary

Company strengths and strategic positioning

• Leading in ocular oncology with three indications and strong phase II data; phase III enrollment progressing well, with top-line data expected in Q4 2027 and high probability of success.

• Unique positioning in neoadjuvant bladder cancer treatment, with no direct competitors in this setting and a focus on immune activation before surgery.

• Strong safety and feasibility profile in bladder cancer, with minimal adverse events and potential for combination with other therapies.

• Large addressable markets in both intermediate-risk bladder cancer (80,000 patients/year) and ocular oncology, with potential for expansion into broader early-stage populations.

• Ultra-orphan pricing strategy supported by market consensus, with potential for multi-billion dollar market across multiple ocular indications.

Clinical development updates and milestones

• Bladder cancer phase I/II study uses a prime-boost approach, treating multiple lesions and cycles, with data from 15-20 patients expected mid-year.

• Proof of concept in bladder cancer defined as 15-20 patients at 12 months showing improved recurrence-free survival; three- and twelve-month endpoints are key for regulatory and clinical differentiation.

• Ocular melanoma phase III trial is highly powered, fully masked, and uses time to tumor progression as the primary endpoint, with top-line data expected in Q4 2027.

• SPA agreement with FDA ensures endpoints are registration-supportive; secondary endpoint focuses on visual acuity preservation.

• Additional studies underway for choroidal metastases and ocular surface cancers, with proof-of-concept data expected this year and potential for rapid supplemental BLA.

Market opportunity and competitive landscape

• Ocular melanoma market is larger than current estimates, as many patients are observed rather than treated; vision-preserving therapy expected to expand the treatable population.

• Drug is tumor-agnostic, with potential applications in metastases to the choroid and ocular surface cancers, broadening the market.

• Differentiation from competitors centers on avoiding radiotherapy and preserving vision, unlike systemic or neoadjuvant approaches from other companies.

• Ultra-orphan pricing corridor is supported by precedent, with Immunocore’s launch at $1.5M per patient as a benchmark.

• Market expansion anticipated as new indications are validated and the drug’s safety and efficacy profile is established across ocular oncology.