Aura Biosciences, Inc. · NASDAQ:AURA
Aura Biosciences — Investor Presentation (May 2025)
51 slides from Aura Biosciences's investor presentation, May 16, 2025
Slide 1: Innovating the future of cancer care to cure patients and preserve organ function
Exhibit 99.1 May 2025 Innovating the future of cancer care to cure patients and preserve organ function
Slide 2: Legal disclosure
Legal disclosure This presentation contains forward-looking statements, all of which are qualified in their entirety by this cautionary statement. Many of the forward-looking statements contained herein can be identified by the use of forward-looking words such as may , anticipate , believe , could'
Slide 3: Well positioned with multiple near-term clinical catalysts
Well positioned with multiple near-term clinical catalysts Novel class of drugs Positive clinical data in Large market opportunity Key upcoming virus-like drug conjugates multiple indications in areas of unmet need catalysts VDCs have the potential to Positive phase 2 data in early- Ocular oncology
Slide 4: Clinical pipeline across multiple solid tumor indications
Clinical pipeline across multiple solid tumor indications Program Preclinical Phase 1 Phase 2 Phase 3 Planned milestones Ocular oncology Phase 3 enrollment complete as Primary uveal melanoma early as the end of 2025 Metastases to the choroid Initial phase 2 data in 2025 Ocular surface cancers Initia
Slide 5: Virus-like drug conjugates have the potential to transform early cancer treatment
Virus-like drug conjugates have the potential to transform early cancer treatment Unique tumor selectivity Tumor and mutation-agnostic Targets a key receptor molecule expressed in the >100 cell lines early stages of malignant tumor transformation >15 animal tumor models Dual MoA High potency Targete
Slide 6: AU-011 has a novel dual mechanism of action
VLPs bind to macrophages, B cells, dendritic cells and neutrophils and are capable of stimulating antigen-presenting cells through TLR-4 engagement and NFk-β production Reactive oxygen species disrupts cell membrane and organelles AU-011 has a novel dual mechanism of action Disruption of the tumor c
Slide 7: Bel-sar's unique platform technology is potentially applicable across multiple cancers
Bel-sar’s unique platform technology is potentially applicable across multiple cancers Bladder cancer CNS cancers Choroidal melanoma NMIBC/MIBC GI cancers Ongoing phase 3 with SPA Based on positive phase 2 clinical data Positive phase 1 data in NMIBC Head and neck cancer Multiple CRs with single dos
Slide 8: Ocular Oncology
Ocular Oncology Bel-sar target indications: Primary uveal melanoma | Metastases to the choroid | Ocular surface cancers
Slide 9: Ocular oncology franchise total addressable market
~66,000 patients/year Bel-sar opportunities Ocular oncology franchise total addressable market (US/EU) in ocular oncology represent a multi- a,1–5 6 ~35,000/yr ~11,000/yr billion-dollar Ocular surface cancers Primary uveal melanoma addressable market • With only ~100 ocular oncologists in the US/EU,
Slide 10: Bel-sar is in phase 3 for primary uveal melanoma
Choroid is 90% 1 of the uvea Bel-sar is in phase 3 for primary uveal Most common primary 2,3 melanoma, the most intraocular cancer in adults 3 ~11,000/yr common primary Choroidal melanoma intraocular cancer ~80% of patients diagnosed 3 with early-stage disease in adults Iris • Primary uveal melanoma
Slide 11: Current treatment paradigm for primary uveal melanoma
1–3 Current treatment paradigm for primary uveal melanoma Small Medium Large Metastatic SIZE 1 2.5 – 3 >10 (mm): Indeterminate lesions Small melanomas Prevalence of Risk Factors Growth Small CM choroidal nevi ranges from 4.6–7.9% in 2 Enuc. Caucasians Observation Systemic chemotherapy Radiotherapy R
Slide 12: High morbidity associated with current standard of care
High morbidity associated with current standard of care 3–6 Radiotherapy Up to 87% of primary uveal melanoma patients become legally blind 1,2 Adverse Event over time in the eye treated with radiotherapy Surgeries secondary to AEs 40%+ (e.g., cataracts) Proportion of patients legally blind (BCVA Rad
Slide 13: Bel-sar has the potential to be the first approved vision-preserving therapy
Treatment Goals Bel-sar has the potential to be the Reduce metastasis Local tumor Vision No radiation- Improve safety risk with early related morbidity control preservation and quality of life first approved treatment vision-preserving In-office procedure therapy in primary uveal melanoma Delivery v
Slide 14: Global phase 3 CoMpass trial now enrolling
Bel-sar for small choroidal melanoma or indeterminate lesions: Global phase 3 CoMpass trial now enrolling Target enrollment ~100 participants globally Anticipated sites in North America, Europe, Middle East and Asia-Pacific Regions Primary endpoint 80 µg bel-sar treatment arm Time to tumor progressi
Slide 15: Phase 2 end of study data using planned phase 3 endpoints
Product-Limit Survival Estimates With Number of Subjects at Risk 11 1 .0 ..0 0 + + C e Cn en ss oo re red d Time to tumor 00 0 .8 ..8 8 progression 00 0 .6 ..6 6 P = 0.0005 Change from baseline in thickness ≥0.5 mm; or in LBD ≥1.5 mm 0.4 0 0..4 4 confirmed by at least one Phase 2 end of study repeat
Slide 16: Bel-sar has a significant commercial opportunity in ocular oncology
Bel-sar has a significant commercial opportunity in ocular oncology Bel-sar’s potential value drivers Highly favorable competitive landscape Ocular surface a,2–6 ~35,000/yr cancers Regulatory and manufacturing synergies Focused call point (~100 ocular Metastases to oncologists in US/EU) with potenti
Slide 17: Metastases to the choroid: multiple tumor types that could be impacted
Metastases to the choroid: Clinical translation of multiple tumor types that could be impacted by bel-sar Platform potential in multiple solid tumors CNS cancers Ocular surface cancers Choroidal metastasis 3 Multiple tumor types metastasize to the eye Head and Neck Uveal melanoma Cancer Retinoblasto
Slide 18: Metastases to the choroid originate from multiple primary cancers
Metastases to the choroid originate from Metastases to the 1 multiple primary cancers ~1/4 of patients have tumors choroid is a high 2 bilaterally unmet medical need Breast 40–53% GI 4% and potentially Skin 2% Lung 20–29% Kidney 2% doubles the ocular Prostate 2% oncology market opportunity Choroidal
Slide 19: Metastases to the choroid: study expanded to any systemic carcinoma
Metastases to the choroid: Study being expanded to include patients with any systemic carcinoma a,b Study Design (n=12) Cohort 1 Cohort 2 Cohort 3 Cohort 4 (N=3) (N=3) (N=3) (N=3) 80µg 160µg 200µg 200µg 1 cycle 1 cycle 1 cycle 2 cycles • Safety/dose-limiting toxicity • Patients with unilateral, unif
Slide 20: Cancers of the Ocular Surface: high unmet need
Cancers of the Ocular Surface: One of the largest ocular oncology indications, with high unmet need 1-5 Cancer Types • Conjunctival Melanoma & other Melanocytic Tumors (PAM): ~30,000 • Conjunctival Squamous Cell Carcinoma / OSSN: ~5,000 6,7 Treatment • Surgery/Excision • Neoadjuvant and/or adjuvant
Slide 21: Planned proof-of-concept study of bel-sar for ocular surface tumors
Planned Proof-of- Concept Phase 1 Study of Bel-sar for Ocular Surface Tumors • Assess feasibility, histopathologic response, Day 1 Day 8 Day 15 immune response, preliminary efficacy • TREATMENT #2:• IMAGE: • TREATMENT #1: • IMAGE: • Inject bel-sar • Measure tumor • 2-week primary endpoint, • Measure
Slide 22: Urologic Oncology
Urologic Oncology Bel-sar target indications: Intermediate-risk NMIBC | High-risk NMIBC
Slide 23: Bladder cancer: high unmet medical need for organ-sparing therapies
Bladder cancer: High unmet medical need for function-preserving organ-sparing therapies most common Conventional bladder cancer treatments are suboptimal th 1 cancer worldwide 9 • Short- and long-term side effects of patients do not • Considerable impact on QoL complete a full course 84% 6 of BCG tr
Slide 24: Current treatment paradigm based on upfront resection leads to recurrence
Current treatment paradigm based on upfront resection leads to recurrence High-risk papillary disease Low grade – low & intermediate risk High-risk CIS – BCG unresponsive a Recurrence Front line Front line Adjuvant therapy Adjuvant therapy Adjuvant therapy Intravesical gene therapy BCG TURBT BCG TUR
Slide 25: Bel-sar has an innovative dual MoA
1 2 Long-term anti-tumor Robust targeted immune memory cytotoxicity has the potential to provide designed to rapidly Bel-sar has immune surveillance, urothelial destroy cancer cells field effect, and prevent recurrence an innovative dual MoA Immune ablation offers an effective front-line therapy, le
Slide 26: New formulation for urologic therapeutic area
New formulation for urologic therapeutic area • Stable at 2–8°C with simple refrigeration New formulation of • Convenient administration in urologist office anticipated bel-sar for use in bladder cancer o No need for cold chain (–70°C) o No need for biosafety (BSL-2) AU-012 o No need for general ane
Slide 27: Bel-sar shifts the paradigm to immune-ablative front-line treatment
Bel-sar shifts the treatment paradigm from resection-based to immune-ablative front-line treatment Current SoC: TURBT + adjuvant treatment Bel-sar has an immune-mediated MoA NIR light Necrotic tumor cells No tumor cells TURBT + bel-sar immune surveillance injection DAMPS and neoantigens Tumor cells
Slide 28: Phase 1 study design in NMIBC
Bel-sar is administered before scheduled biopsy and standard of care (SoC) TURBT Clinical response data up to 21 days; safety data up to 56 days Treatment Phase: Feasibility and mechanism of action Follow-up Phase: Safety Day 9 ± 1 (Cohort A) Day 1 Day 2 Day 56 ± 7 Day 14+7 (Cohort B+C) End of follo
Slide 29: Safety data: single-dose drug with light activation
a Cohort A-C: Single-dose drug with light activation (n=11) Event Grade Number of patients Adverse events (related to study drug) Cohort A-C: Nocturia 1 1/11 Single-dose drug with Urinary urgency 1 1/11 light activation Adverse events (related to injection or laser procedure) 1 1/11 Hematuria Safety
Slide 30: Efficacy data: Ta intermediate-risk NMIBC
Efficacy data: Ta intermediate-risk NMIBC Cohorts A–C (single-dose drug with light activation) 4/5 patients demonstrated CR; 5/5 patients with immune response in target tumor c d Patient A1 Patient A3 Patient A4 Patient B2 Patient C1 Multiple Multiple Multiple Multiple Multiple (TURBT) Screening dia
Slide 31: Efficacy data: Ta high-risk NMIBC
Efficacy data: Ta high-risk NMIBC Cohorts A–C (single-dose drug with light activation) 1/5 patients demonstrated CR; 5/5 patients with immune response in target tumor d Patient A2 Patient B1 Patient B3 Patient C2 Patient C3 Single Single Multiple Multiple Multiple Screening diagnosis Ta high-grade T
Slide 32: Clinical complete response confirmed with histopathologic evaluation (Patient A3)
Clinical complete response visualized at time of TURBT confirmed with Patient A3 histopathologic evaluation 72-year-old Hispanic male Screening diagnosis: (2024) • Multiple bel-sar • Ta low-grade (<3 cm) injection • No CIS Screening AUA risk classification: Intermediate Initial diagnosis: (2019) Bio
Slide 33: Mature tertiary lymphoid structures in target lesion
Mature Tertiary Lymphoid Structures (TLS) in Target (Treated) Lesion: Active Immunosurveillance After Bel-sar Treatment CD3: T cells CD20: B cells CD23: Follicular Dendritic Cells (FDC) (Found in B cell follicles, only present in mature TLS) PNAd: Peripheral Node Addressin (Stains for high endotheli
Slide 34: Early tertiary lymphoid structures in distant non-target lesion
Early Tertiary Lymphoid Structures (TLS) in Distant Non- Target (Non-Treated) Lesion: Urothelial Immune Field Effect After Bel-sar Treatment CD3: T cells CD20: B cells CD23: Follicular Dendritic Cells (FDC) (Found in B cell follicles, only present in mature TLS) PNAd: Peripheral Node Addressin (Stai
Slide 35: Phase 1b/2 treatment schedule in NMIBC patients
Phase 1b/2 treatment schedule in intermediate and high-risk NMIBC patients Immune ablative design: Intermediate Risk Patients Cycle 1 Cycle 2 a D1 D2 D15 D16 8W 3M FU 6M FU 9M FU 12M FU V5 V6 V7 V8 inj. las. inj. las. Cystoscopy as clinically indicated V1 V2 V3 V4 st nd 1 treatment 2 treatment Respo
Slide 36: Phase 1b/2 study design
In parallel Optional high dose Phase 1b/2 study design Cohort 4e (N=5) 400 µg IT (per lesion, per treatment) Key updates 1200 µg max. total bladder dose Cohort 4h (N=6) 2-cycle immune-ablative 800 µg IT Part 1: Dose escalation Intermediate Risk (per lesion, per treatment) • 21–26 participants 2400 µ
Slide 37: Company highlights
Company highlights Corporate Ocular Oncology Therapeutic Area Primary uveal melanoma • Current Cash expected to fund operations into 1H 2027• Global phase 3 CoMpass trial actively enrolling globally; study enrollment may be • Experienced leadership team across functions completed as early as the end
Slide 38: Appendix Ocular Oncology
Appendix Ocular Oncology & aura
Slide 39: Phase 2 trial of bel-sar for choroidal melanoma
Phase 2 trial of bel-sar for choroidal melanoma: Open-label, dose-escalation with suprachoroidal administration Trial design – 22 participants enrolled Patient population representative of early-stage disease: Small choroidal melanoma and indeterminate lesions Subtherapeutic regimens Therapeutic reg
Slide 40: Baseline characteristics of study participants
All patients (n=22) Female (%) 54.5 White, not Hispanic or Latino (%) 100 Subretinal fluid at screening (%) 100 Baseline Orange pigment at screening (%) 86.4 characteristics 86.4 Documented growth prior to screening (%) (100% of therapeutic group) Mean age at screening (years, ± SD) 59.2 (±16.5) All
Slide 41: High tumor control rates with therapeutic regimen
High tumor control rates with therapeutic regimen in phase 3-eligible patients with active growth Phase 3-eligible 100% participants 80% 80% Tumor control Dose/ Regimen n rate, % High local complete response rate at 12 60% Subtherapeutic regimen months follow-up ≤2 cycles 10 20% (2/10) 40% a 80% tum
Slide 42: Rate of tumor growth with bel-sar treatment
Rate of tumor growth with bel-sar treatment P < 0.0001 In phase 3-eligible 1.0 0.5 Untreated patients, the 3-cycle projected growth rate 0.351 regimen resulted in 0.4 0.5 cessation of growth Pre-treatment 0.3 among responders (N=8) actual growth rate 0.0 0.2 Post-treatment 0.1 0.011 actual growth ra
Slide 43: Median change in BCVA with therapeutic regimen
Median change in BCVA in phase 3-eligible participants with therapeutic regimen a (N=10) Visual acuity was 5 preserved in 90% of 0 • Vision preservation in phase 3-eligible 9/10 participants • Loss of 18 letters in one patients receiving a -5 patient with progression of -5 preexisting juxtafoveal fl
Slide 44: Phase 2 safety outcomes
Phase 2 safety outcomes (bel-sar/laser-related) All treated participants (n=22)* Drug/laser-related Grade I Grade II Grade III-V Total adverse events Bel-sar treatment Anterior chamber inflammation** 4 (18.2%) 0 0 4 (18.2%) had a favorable Anterior chamber cell** 2 (9.1%) 0 0 2 (9.1%) safety profile
Slide 45: Phase 2 data support phase 3 assumptions
Phase 2 data support phase 3 assumptions Robustness analysis of tumor control rates 100% Overall rate P < 0.05 P < 0.005 in phase 2 80% 2x “worse” than phase 2 60% 2x “worse” Actual data 94% Actual data 93% than phase 2 (Δ60) >99% power (Δ60) >99% power power (Δ30) power (Δ20) 40% Overall rate Actua
Slide 46: Appendix Urologic Oncology
Appendix Urologic Oncology & aura
Slide 47: Immune-ablative and neoadjuvant/multimodal treatment approaches
Immune-ablative Neoadjuvant/multimodal (-TURBT) (+ TURBT) Treat tumor with bel-sar first Treat tumor with bel-sar first and avoid the need for TURBT ahead of TURBT Value proposition: Value proposition: • Prevent recurrence and progression by • Prevent recurrence and progression by Paradigm-shifting
Slide 48: Bel-sar as standalone immune-ablative treatment or neoadjuvant to TURBT
NIR light Necrotic tumor cells No tumor cells + bel-sar immune surveillance injection DAMPS and neoantigens 1 Immune-ablative Bel-sar has potential treatment without T cell trafficking and TURBT proliferation Activation as a standalone (LR/IR NMIBC) of APCs immune-ablative Presentation of neoantigen
Slide 49: Tumor-free survival after single dose of bel-sar
Tumor-free survival after single dose of bel-sar 100 NIR only 80 Bel-sar + NIR A single systemic 60 treatment of bel-sar Long-term tumor free survival after single 40 resulted in long-term dose of bel-sar Day 10: bel-sar tumor-free survival and single-dose 20 induction of anti-tumor 0 0 14 28 42 56
Slide 50: CD4+ and CD8+ T-cells key to durability of response
CD4+ and CD8+ T-cells are key to the long-term durability of response and protection of rechallenge with bel-sar Depletion of CD4+ and CD8+T cells Depletion of CD4+ and CD8+T cells at the time of treatment at the time of rechallenge Implant TC-1 Implant TC-1 Rechallenge with 3 3 Tumor volume: 50mm T
Slide 51: Bel-sar seeks to redefine the treatment of bladder cancer
Bel-sar seeks to redefine the treatment of bladder cancer with a novel and disruptive approach Dual Mechanism of Transformative Broad Market Action Clinical Impact Opportunity Front-line approach Targeted cytotoxicity Early clinical activity + cell mediated anti-tumor demonstrated across the Immedia