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 promising early data.

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

• Ultra-orphan pricing corridor anticipated, with potential for multi-billion dollar markets as indications expand.

• Broad market opportunity in ocular oncology, with potential expansion to other eye cancers and tumor-agnostic applications.

Clinical development updates and milestones

• Phase III ocular melanoma trial is highly powered, with primary endpoint of time to tumor progression and secondary endpoint of visual acuity preservation; enrollment on track for completion in 2026.

• Bladder cancer program advancing with mid-year data readout expected; study design includes multiple cohorts and cycles, targeting both intermediate and high-risk patients.

• Proof of concept in bladder cancer defined as 15-20 patients at 12 months showing improved recurrence-free survival.

• Ocular melanoma phase III trial uses a sham comparator, with FDA SPA agreement on endpoints and masking; top-line data expected Q4 2027.

• Additional studies in choroidal metastases and ocular surface cancers underway, with proof-of-concept data expected this year.

Market opportunity and competitive landscape

• Intermediate-risk bladder cancer population estimated at 80,000 patients, with significant unmet need for safer, office-based therapies.

• Ocular melanoma market currently estimated at 2,000 treated patients, but true addressable population may be 3x larger due to under-treatment.

• Drug positioned to avoid radiotherapy in ocular cancers, differentiating from competitors focused on reducing or combining with radiotherapy.

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

• Expansion into choroidal metastases and ocular surface cancers could further increase market size and value.