Aura Biosciences, Inc. · NASDAQ:AURA
Aura Biosciences — Investor Presentation (August 2024)
35 slides from Aura Biosciences's investor presentation, Aug 8, 2024
Slide 1: Corporate Presentation August 2024
Corporate Presentation August 2024 Exhibit 99.2
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", "co
Slide 3: Well Positioned with Multiple Near-Term Clinical Catalysts
Well Positioned with Multiple Near-Term Clinical Catalysts Precision Therapy Platform Developing a novel class of drugs called virus-like drug conjugates (VDCs) Direct tumor cell killing and immune activation Focal treatment approach to deliver durable response Large Market Opportunity In Areas of U
Slide 4: Clinical Pipeline Across Multiple Solid Tumor Indications
Clinical Pipeline Across Multiple Solid Tumor Indications Program OTHER SOLID TUMORS OCULAR ONCOLOGY Preclinical Phase 1 Phase 2 Phase 3 Planned Milestones Metastases to the Choroid (Multiple primary cancers with metastasis to the choroid, e.g., Breast and Lung) Primary Uveal Melanoma Ocular Surface
Slide 5: Bel-sar is a Potential First-in-Class Therapy for Multiple Solid Tumors
Bel-sar is a Potential First-in-Class Therapy for Multiple Solid Tumors
Slide 6: Bel-sar (AU-011) is a VDC Designed with Dual Specificity
Bel-sar (AU-011) is a VDC Designed with Dual Specificity to Reduce Potential for Off-target Effects: Selectively binds to tumor cells (not to local healthy tissue) Activated only at site of laser administration Fleury MJJ et al. Mol Biotechnol. 2014;56(5):479-86. Kines RC, et al. Int J Cancer. 2016;
Slide 7: Bel-sar is a VDC with a novel dual mechanism of action
Disruption of the tumor cell membrane and pro-immunogenic cell death by necrosis leads to T cell activation and immune-mediated tumor cell killing Bel-sar is a VDC with a novel dual mechanism of action Kines RC, et al. Int J Cancer. 2016;138(4):901–11. Kines RC, et al. Mol Cancer Ther. 2018;17(2):56
Slide 8: Ocular Oncology Therapeutic Area
Ocular Oncology Therapeutic Area Bel-sar Target Indications: Primary Uveal Melanoma Metastases to the Choroid Ocular Surface Cancers
Slide 9: Bel-sar Opportunities in Ocular Oncology Represent a Multi-billion-dollar Addressable Market
Bel-sar Opportunities in Ocular Oncology Represent a Multi-billion-dollar Addressable Market Ocular Oncology Total Addressable Market (US/EU) ~66,000 patients/year Ocular Surface Cancers Retinoblastoma Primary Uveal Melanoma Metastases to the Choroid ~11,000 patients/year1 ~20,000 patients/year1 ~50
Slide 10: Primary Uveal Melanoma—High Unmet Medical Need
Primary Uveal Melanoma—High Unmet Medical Need Primary Uveal Melanoma is a Rare and Life-Threatening Ocular Cancer with No Drugs Approved 1. Heiting, G. Iris/uvea of the eye. Accessed Oct. 3, 2023. https://www.allaboutvision.com/en-gb/resources/uvea-iris-choroid/ 2. Kaliki S, Shields CL. Uveal melan
Slide 11: Current Treatment Paradigm for Uveal Melanoma
Current Treatment Paradigm for Uveal Melanoma Indeterminate Lesions Small Melanomas Risk Factors Growth Small CM Observation Incidence: Patients US/EU1 Local – Early (~8,000) Local – Late (~2,300) Metastatic (~2,000) SIZE (mm): Small Medium Large Metastatic Radiotherapy Radiotherapy 1 2.5–3 >10 Enuc
Slide 12: High Morbidity Associated with Current Standard of Care
High Morbidity Associated with Current Standard of Care 1. Jarczak J, Karska-Basta I, Romanowska-Dixon B. Deterioration of visual acuity after brachytherapy and proton therapy of uveal melanoma, and methods of counteracting this complication based on recent publications. Medicina (Kaunas). 2023;59(6
Slide 13: Bel-sar has the Potential to be the First Approved Therapy in Primary Uveal Melanoma
c Bel-sar has the Potential to be the First Approved Therapy in Primary Uveal Melanoma No radiation-related morbidity Preservation of vision Local tumor control Opportunity to treat early and reduce risk of metastases Improvement in safety and quality of life Bel-sar is Delivered by Simple Suprachor
Slide 14: Phase 2 Trial – Dose Escalation and Expansion with Suprachoroidal Administration
Phase 2 Trial – Dose Escalation and Expansion with Suprachoroidal Administration Patient Population Representative of Early-Stage Disease: Small Choroidal Melanoma and Indeterminate Lesions Trial Design – Enrollment Complete (n=22) One Cycle = Doses on days 1, 8 and 15 N=10 1- 2 Doses (n=9); 2 cycle
Slide 15: Phase 2 Interim Data Demonstrates Tumor Control, Vision Preservation and Favorable Safety
Phase 2 Interim Data Demonstrates Tumor Control, Vision Preservation and a Favorable Safety Profile Subtherapeutic Therapeutic Tumor Progression: change from baseline in thickness ≥0.5mm; or in LBD ≥1.5mm confirmed by at least one repeat assessment August 3, 2023, data on file Aura Biosciences Visio
Slide 16: SPA Agreement with FDA Supports Global Phase 3 Trial Design
SPA Agreement with FDA Supports Global Phase 3 Trial Design Fast Track and Orphan Drug Designations Time to Composite Endpoint: Tumor Progression or Visual Acuity Failure Time to Tumor Progression Primary Endpoint First Key Secondary Endpoint Enrollment (N=~100) Randomize (2:1:2) 80 µg bel-sar treat
Slide 17: Kaplan-Meier simulation of Phase 2 interim data supports Phase 3 success assumptions
Kaplan-Meier analysis simulation of Phase 2 interim data support assumptions for the potential success of Phase 3 trial with high statistical significance Progression-free probability Days Progression-free probability Log-rank test P = 0.0012 Log-rank test P = 0.0023 Time to tumor progression Time t
Slide 18: Phase 2 Interim Data Support Phase 3 Assumptions
Phase 3 trial design P < 0.005 93% power (Δ20) Actual data (Δ60) >99% power P < 0.05 Robustness Analysis of Phase 2 interim tumor control rates Overall rate in Phase 2 2x “worse” than Phase 2 2x “worse” than Phase 2 Actual rate with documented growth in Phase 2 Overall rate in Phase 2 94% power (Δ30
Slide 19: Bel-sar Opportunities in Ocular Oncology Represent a Multi-billion-dollar Addressable Market
Bel-sar Opportunities in Ocular Oncology Represent a Multi-billion-dollar Addressable Market Ocular Oncology Total Addressable Market (US/EU) ~66,000 patients/year Ocular Surface Cancers Retinoblastoma Primary Uveal Melanoma Metastases to the Choroid ~11,000 patients/year1 ~20,000 patients/year1 ~50
Slide 20: Metastases to the Choroid – Phase 2 Trial Expected to Begin in 2024
Metastases to the Choroid – Phase 2 Trial Expected to Begin in 2024 Cohort 1 N=3 patients 80µg 1 cycle Cohort 2 N=3 patients 160µg 1 cycle Cohort 3 N=3 patients 200µg 1 cycle Cohort 4 N=3 patients 200µg 2 cycles Safety/Dose-limiting Toxicity Efficacy Change in Tumor Size Change in Vision Letter Scor
Slide 21: Urologic Oncology Therapeutic Area
Urologic Oncology Therapeutic Area Bel-sar Target Indications: Non-muscle invasive bladder cancer Muscle invasive bladder cancer
Slide 22: Bladder Cancer is a Global High Unmet Medical Need
Bladder Cancer is a Global High Unmet Medical Need ~500,000 New cases/ year globally1 >200,000 NMIBC New cases/year US, Europe & Asia1 New cases/year US, Europe & Asia1 >60,000 MIBC Non-Muscle Invasive Bladder Cancer Muscle Invasive Bladder Cancer Recurrence, multiple TURBT surgeries, Progression of
Slide 23: Current Treatment Paradigm for Non-Muscle Invasive Bladder Cancer
Current Treatment Paradigm for Non-Muscle Invasive Bladder Cancer Prevalence Patients (US)1,2 (~80,000) (~20,000) Progression Low grade – Low & intermediate risk High risk papillary disease CIS – BCG unresponsive BCG >6 tx Intravesical Chemotherapy (~4,000) TURBT recurrence Intravesical gene therapy
Slide 24: Bel-sar as Potential Front-Line Therapy in NMIBC
Bel-sar as Potential Front-Line Therapy in NMIBC may be Optimized for In Office-based Procedure Goals of Treatment with Bel-sar Reduce Risk of Recurrence Stimulate Anti-tumor Specific T Cell Response Focal Treatment with Direct Tumor Cell Killing Avoid TURBT /Operating Room No Virus Replication or V
Slide 25: Phase 1 trial for bladder cancer designed to evaluate safety, feasibility and MoA
Phase 1 trial for bladder cancer designed to evaluate safety, feasibility and MoA 21 participants Bel-sar alone Bel-sar + focal light activation Part 1 (n=5) Part 2 (n=16) Study objectives TURBT and cystectomy patients NMIBC and MIBC patients Safety & dose-limiting toxicity Focal distribution of bel
Slide 26: Clinical Complete Response with Immune Activation after Single Dose
Clinical Complete Response with Immune Activation after Single Dose Confirmed by Histopathology DAY 1 Biopsy TURBT Biopsy Day 1 Day 1 Day 2 Day 3-7 Bel-sar (AU-011) TURBT- transurethral resection of bladder tumor Day 9 Ph 1- Preliminary Data Light Activated Cohort (n=1) Day 1 Diagnostic biopsy shows
Slide 27: Clinical Complete Response with Immune Activation in Part 2 First Patient
Clinical Complete Response with Immune Activation after Single Dose Confirmed by Histopathology in Part 2 First Patient Papillary urothelial carcinoma Pre-injection bladder biopsy demonstrating low-grade papillary urothelial carcinoma; non-invasive. Post-treatment TURBT demonstrating necrosis, infla
Slide 28: Company Highlights
Company Highlights Ocular Oncology Therapeutic Area Primary Uveal Melanoma – Global Phase 3 CoMpass Trial: Trial actively enrolling Special Protocol Assessment (SPA) Agreement with FDA Phase 3 assumptions supported by Phase 2 data Metastases to the Choroid – Phase 2 trial planned to initiate in 2024
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Slide 30: Appendix: Phase 2 Primary Uveal Melanoma Trial – Interim Data
Appendix: Phase 2 Primary Uveal Melanoma Trial – Interim Data
Slide 31: High Local Complete Response Rate at 12 Months Follow-up
High Local Complete Response Rate at 12 months Follow-up* Dose/Regimen Total Patients (n) Total Patients (n) Tumor Control Rate Subtherapeutic Regimens Single dose up to 2 cycles 10 20% (2/10) Therapeutic Regimen 3 Cycles (n=11) 11 73% (8/11) 3 Cycles and Phase 3 eligible (n=10)* 10 80% (8/10) * One
Slide 32: High Tumor Control Rates Observed in Phase 2 Population Treated with Therapeutic Regimen
High Tumor Control Rates Observed in Phase 3 Population Treated with Therapeutic Regimen in Phase 2 Progression Definition based on Tumor Thickness (Increase ≥0.5mm) Change from Baseline in Tumor Thickness Over 12 Months Treatment Timeframe Follow-up Timeframe Change from Baseline in Tumor Thickness
Slide 33: Bel-sar Therapeutic Regimen Achieved High Tumor Control Rates in Phase 3-eligible Tumors
Bel-sar Therapeutic Regimen in Phase 2 Interim Data Achieved High Tumor Control Rates, with Complete Cessation of Growth Among Responders with Phase 3-eligible Tumors Tumor progression defined as change from baseline in thickness ≥0.5 mm; or in LBD ≥1.5 mm confirmed by at least one repeat assessment
Slide 34: Visual Acuity was Preserved in 90% of Participants Receiving a Bel-sar Therapeutic Regimen
34 80% (8/10) of these trial participants were at high risk of vision loss with tumors < 3 mm to the fovea or optic nerve BCVA change from baseline (ETDRS letter score) Median change in BCVA in Phase 3-eligible participants with therapeutic regimen (n=10)a Study month >90% of participants completed
Slide 35: Phase 2 Interim Safety Data Supports Potential to be First Line Treatment
Phase 2 Interim Safety Data Supports Potential to be First Line Treatment in Primary Uveal Melanoma Adverse Event Radiotherapy* Bel-Sar+ Surgeries secondary to AEs+ (e.g., Cataracts) 40%+ 0% Radiation Retinopathy 40%+ 0% Neovascular Glaucoma 10% 0% Dry Eye Syndrome 20% 0% Strabismus 2%+ 0% Retinal D
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