Aura Biosciences (AURA) Evercore ISI 8th Annual HealthCONx Conference summary

Pipeline overview and development strategy

• Advancing a novel class of Virus-like Drug Conjugates with direct cytotoxicity and robust immune activation, targeting ocular oncology and bladder cancer as key areas of focus.

• Ongoing phase III trial in early-stage choroidal melanoma, aiming for organ preservation and prevention of metastasis, with a unique frontline positioning and minimal competition.

• Phase II and proof-of-concept studies in additional ocular indications (metastatic and ocular surface cancers) and bladder cancer, with multiple near-term catalysts expected.

Ocular oncology program details

• Phase III trial (CoMpass) in early choroidal melanoma is enrolling patients with growing tumors to ensure a highly powered study and clear differentiation between treatment and sham.

• Enrollment bottleneck due to need for tumor progression confirmation, but consistent patient conversion now supports guidance for study completion in 2026 and top-line data in Q4 2027.

• Safety profile is favorable, with only Grade 1 local adverse events and no systemic exposure, supporting frontline use.

• Secondary composite endpoint includes visual acuity preservation, designed with FDA input to strengthen the value proposition.

• Proof-of-concept studies in metastatic and ocular surface cancers expected to support supplemental BLA filings with minimal additional data.

Bladder cancer program and market positioning

• Focused on neoadjuvant treatment of early-stage tumors, leveraging the drug's mechanism that requires tumor presence for efficacy and immune activation.

• No direct competitors in the neoadjuvant NMIBC space; most others are in adjuvant settings.

• Using recurrence-free survival (RFS) as a validated endpoint, aiming to demonstrate improved outcomes with a highly tolerable, urologist-friendly formulation.

• Neoadjuvant approach may complement, not replace, adjuvant therapies, with the sequence of therapy being a key differentiator.