STIFEL 2026 HEALTHCARE CONFERENCE - AURA BIOSCIENCES INC

Explicit Guidance:

• No explicit financial guidance (revenue, earnings, margins, capex, etc.) is provided for any period.
• Cash runway announced as $297 million, sufficient to fund operations through 2029.
• Key Milestone Timelines:
  • H2 2027: Top-line efficacy data expected from the pivotal Phase 3 COMPASS study for early choroidal melanoma.
  • Q1 2027: Company commits to providing a comprehensive update on earlier-stage programs (choroidal metastases, ocular surface cancers), including disease state unmet need, study design, and data expectations.
  • 2028: 24-month durability readout from the COMPASS study.
  • Dec 8 (Year implied as current year based on context): Event to present updated epidemiology and market research for early choroidal melanoma.
  • 2027: Submission of the peer-reviewed manuscript for the Phase 2 study data.

Business-Segment Outlook:

• Core Strategy: Deprioritization of the NMIBC (bladder cancer) program post-Phase 1/2 to focus exclusively on building a preeminent ocular oncology company centered on BELSAR.
• Primary Indication: Early choroidal melanoma (uveal melanoma); the company targets the entire spectrum of early disease, moving away from the historical distinction between "indeterminate lesions" and "small choroidal melanomas" to expand the eligible population.
• Pipeline Expansion:
  • Q1 2027 Update: Plans to reveal details on two earlier-stage programs: (1) choroidal metastases from solid tumors (specifically breast and lung) and (2) ocular surface tumors (direct intertumoral injection).
  • Long-term Vision: Aiming to expand BELSAR indications to metastases to the choroid and ocular surface cancers if early results are promising, maintaining a "first-in-class frontline therapy" positioning for these devastating diseases.

Market and Competitive Outlook:

• Competitive Landscape: No direct competition identified in the early choroidal melanoma space; the company positions itself as the only program exploring treatment in the early stage to prevent progression to metastatic disease.
• Market Dynamics: Anticipates a paradigm shift where patients are treated earlier to preserve vision, rather than the current standard of waiting until tumors are larger or opting for radiotherapy/enucleation which often results in blindness.
• Adoption Strategy: The company expects to grow the market itself rather than targeting an existing treated population, citing a lack of direct commercial precedents.
• Target Audience: A focused community of ~90% of ocular oncologists in the US and Europe are already involved in the development network, facilitating high commercial overlap and familiarity.

Macro / Industry Outlook:

• Regulatory: Successfully negotiated a Special Protocol Agreement (SPA) with the FDA Ophthalmology Division, utilizing an "active growth" enrichment criterion to increase probability of success and detect signals within 15 months, with confidence that this does not translate to a narrow label.
• Clinical Practice Trends: Industry trend moving toward treating earlier stages of disease earlier, driven by the availability of therapies that control tumors without sacrificing vision.

Strategic Initiatives:

• Commercial Launch Preparation: Developing an in-office administration workflow (two injections, 30 minutes apart, followed by laser activation 4-6 hours later) designed to integrate seamlessly into existing ocular oncology practices without requiring major infrastructure changes.
• Market Expansion Event: Scheduled December 8th event to release updated epidemiology, featuring KOLs Dr. Arun Singh and Dr. Anthony Daniels, alongside commercial research to redefine the addressable market size.
• Program Deprioritization: Completed enrollment in the NMIBC program and decided to finish the study but deprioritize the asset to allocate all resources to BELSAR in ocular oncology.

Risks and Headwinds to Outlook:

• Enrollment Challenges: The "active growth" inclusion criterion required for the Phase 3 COMPASS study enriched for success but made enrollment more difficult ("a bit of a pain").
• Execution Complexity: Management acknowledges that drug launches are "harder than you expect" and no illusions about ease of launch exist.
• Data Timing: No specific guidance on data timing for earlier-stage programs (metastases/ocular surface) beyond the Q1 2027 update; future data delivery remains contingent on full funding.
• Market Education: Significant confusion exists within the investment community regarding the addressable patient population size, requiring extensive market education to demonstrate the value of treating the full early-disease spectrum.

Management Tone on the Future:

• Confidence Level: High confidence in the unique value proposition ("exquisitely selective," "no competition," "transformative value for patients") and the ability to build a valuable company around a single asset.
• Strategic Focus: Strong emphasis on "going all-in" on a single indication to maximize execution and avoid the pitfalls of managing multiple programs simultaneously.
• Growth Mindset: Expressed excitement about the opportunity to "build a market" and grow the treatment paradigm for early choroidal melanoma, viewing the lack of comps as an opportunity rather than a risk.
• Financial Security: Reiterated confidence in the $297M balance sheet covering operations well past the critical 2027 top-line data point, ensuring full funding of current programs.

Miscellaneous:

• Safety Profile: Phase 2 data showed 80% tumor control and 90% vision preservation with a clean safety profile (Grade 1 AEs, no treatment-related SAEs or discontinuations).
• Mechanism: BELSAR is a local therapy activated only via ophthalmic laser, designed to limit cytotoxic activity to the specific tumor location to spare vision.