Aura Biosciences (AURA) Status Update summary

Clinical trial results and safety profile

• Early phase I data for AU-011/bel-sar in NMIBC show a favorable safety profile, with only grade 1 drug-related adverse events in less than 10% of patients and no serious or dose-limiting toxicities reported.

• Four out of five low-grade NMIBC patients achieved complete clinical response in target tumors after a single low dose with light activation, with evidence of immune response in both target and non-target lesions.

• Immune cell infiltration (CD4, CD8 T cells) was observed in all treated patients, supporting a dual mechanism of action: direct tumor ablation and immune activation.

• Visual tumor shrinkage and immune activation were also observed in high-grade NMIBC patients and in some untreated tumors, indicating a bladder urothelial field effect.

• The procedure is feasible in a clinic setting without general anesthesia, with no significant safety differences between light-activated and non-light-activated cohorts.

Mechanism of action and clinical implications

• AU-011/bel-sar is a virus-like drug conjugate activated by infrared light, causing targeted cytotoxicity and robust immune activation.

• The treatment induces both local tumor necrosis and adaptive immune responses, including the formation of lymphoid follicles, suggesting potential for durable immunity.

• Immune-mediated effects were observed in non-injected, non-target lesions, indicating a bladder urothelial field effect.

• The focal nature of AU-011/bel-sar minimizes toxicity to healthy tissue compared to traditional intravesical therapies.

• Preclinical models show AU-011 induces durable immunological memory and prevents tumor growth after rechallenge; combination with checkpoint inhibitors enhances efficacy and survival.

Patient population and clinical workflow

• NMIBC, especially low-grade, intermediate-risk patients, represents a large unmet need due to high recurrence rates and treatment burden.

• AU-011/bel-sar administration is compatible with standard urology clinic procedures, requiring no general anesthesia and minimal additional training.

• The therapy is particularly suitable for elderly patients or those with comorbidities who may not tolerate surgery.

• Experts anticipate broad applicability in the clinic, potentially reducing the need for TURBT and shifting care from the OR to office-based settings.

• 13 patients enrolled in the ongoing phase I trial: 10 with low-grade and 3 with high-grade disease, most with recurrent bladder cancer and prior treatments.