Aura Biosciences, Inc. · NASDAQ:AURA
Aura Biosciences — Investor Presentation (May 2023)
41 slides from Aura Biosciences's investor presentation, May 11, 2023
Slide 1: Corporate Presentation May 2023
Corporate Presentation May 2023 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: Aura Biosciences Highlights
Aura Biosciences Highlights Novel Platform to Treat Multiple Solid Tumors Developing virus-like drug conjugates (VDCs) that bind to tumor specific HSPGs* to deliver a therapeutic payload Targeting multiple solid tumor indications including ocular and bladder cancers Ocular Oncology Franchise Multi-b
Slide 4: Pipeline Targeting Life-Threatening Cancers with High Unmet Needs
Pipeline Targeting Life-Threatening Cancers with High Unmet Needs Program OTHER SOLID TUMORS OCULAR ONCOLOGY Preclinical Phase 1 Phase 2 Phase 3 Planned Milestones Choroidal Metastasis (Multiple Primary cancers with metastasis in the eye e.g. Breast and Lung) Primary Choroidal Melanoma (Suprachoroid
Slide 5: Targeted Oncology Platform: Virus-Like Drug Conjugates (VDCs)
Targeted Oncology Platform: Virus-Like Drug Conjugates (VDCs) Virus-Like Particle Conjugated to a Cytotoxic Payload Selective Binding to Tumor Associated HSPGs* Virus-Like Particle (VLP) Virus-Like Drug Conjugate (VDC) Cx Cytotoxic Drug Kines et al; International Journal of Cancer, 138;901–911, Febr
Slide 6: Bel-sar is a VDC with a Novel Dual Mechanism of Action
Bel-sar is a VDC with a Novel Dual Mechanism of Action Light Activatable Drug Bel-sar Bel-sar is a novel VDC that consists of VLP conjugated to ~200 molecules of light activatable drug Potential Key Differentiation: Agnostic to Genetic Mutations, Less Susceptible to Resistance Mechanisms, Long Term
Slide 7: Ocular Oncology Franchise
Ocular Oncology Franchise Bel-sar INN: belzupacap sarotalocan Target Indications: Early-Stage Choroidal Melanoma Choroidal Metastasis Other Ocular Cancers
Slide 8: Primary Choroidal Melanoma – High Unmet Medical Need with No Drugs Approved
Primary Choroidal Melanoma – High Unmet Medical Need with No Drugs Approved Choroidal Melanoma is a Rare and Life-Threatening Ocular Cancer Kaliki et al; Eye (Lond) 2017 Feb; 31(2): 241–257; Clearview & Putnam & Assoc. Market Research; Source: Peddada. J Contemp Brachytherapy. August 2019 Most commo
Slide 9: Bel-sar has the Potential to be the First Approved Therapy in Primary Choroidal Melanoma
Bel-sar has the Potential to be the First Approved Therapy in Primary Choroidal Melanoma No radioactive co-morbidities Preservation of vision Local tumor control Opportunity to treat early and reduce risk of metastases Improvement in safety and quality of life intravitreal suprachoroidal Bel-sar is
Slide 10: Ocular Oncology Franchise Represents a Multi-Billion Dollar Addressable Market Opportunity
Ocular Oncology Franchise Represents a Multi-Billion Dollar Addressable Market Opportunity Ocular Oncology Franchise total addressable market ~80% of patients are diagnosed at the early stage (indeterminate lesions (ILs) and small tumors) Current radiotherapy treatment Leaves ~70% of patients with m
Slide 11: No Drugs Approved and No Known Competition in Early-Stage Disease
No Drugs Approved and No Known Competition in Early-Stage Disease Plaque Brachytherapy Proton Beam Plaque Brachytherapy Proton Beam Watch & Wait Chemotherapy Enucleation 80% ~ 9000 patients Diagnosed with Early-Stage Disease: Indeterminate Lesions and Small Melanomas CM – Choroidal Melanoma Initial
Slide 12: Goal for Bel-sar: Eliminate Malignant Cells in the Choroid and Preserve Vision
Goal for Bel-sar: Eliminate Malignant Cells in the Choroid and Preserve Vision Endpoint Endpoint Definitions Tumor Progression Growth in Tumor Height ≥0.5mm or ≥1.5 mm in Largest Basal Diameter (LBD) Visual Acuity Failure Decrease from baseline: ≥15 letters Tumor Thickness Growth Rate Change in tumo
Slide 13: Phase 2 Trial - Evaluating Suprachoroidal Administration
Ph 2 Trial - Evaluating Suprachoroidal Administration to Determine Optimal Administration Route Ph2 SC Trial Design – Enrollment Complete *2 subjects were planned; third subject was additionally enrolled due to dose error in 1 subject ClinicalTrials.gov Identifier: NCT04417530 ; AU-011-202 Interim D
Slide 14: Phase 2 Interim Tumor Control Rates Demonstrated a Dose Response
Ph 2 Interim Tumor Control Rates Demonstrated a Dose Response Tumor Progression: change from baseline in thickness ≥0.5mm; or in LBD ≥1.5mm confirmed by at least one repeat assessment Interim Data- January 10, 2023; Last two subjects enrolled after data cut not included in dataset Populations Total
Slide 15: Phase 2 Interim Analysis Demonstrated Tumor Control Rate 89%-100%
Ph 2 Interim Analysis Demonstrated Tumor Control Rate 89%-100% Ph 2 Interim Analysis Demonstrated Tumor Control Rate of 100% in Planned Ph 3 Population^ Tumor Progression: change from baseline in thickness ≥0.5mm; or in LBD ≥1.5mm confirmed by at least one repeat assessment; Interim Data- January 10
Slide 16: Phase 2 Interim Analysis Demonstrated Visual Acuity Preservation
Ph 2 Interim Analysis Demonstrated Visual Acuity Preservation ~90% Interim Data Demonstrated High Vision Preservation Rates Across All Groups Including Patients at High Risk for Vision Loss SC – Suprachoroidal Populations Total Patients (n) Vision Failures (n) Vision Preservation Rate Mean Change fr
Slide 17: Phase 2 Interim Data Demonstrated Statistically Significant Tumor Growth Rate Reduction
Ph 2 Interim Data Demonstrated Statistically Significant Tumor Growth Rate Reduction Interim Data Showed Growth Arrest in Planned Phase 3 Population with a p-value of <0.0001 p=0.0001 p= <0.0001 Statistically Significant Change in Tumor Growth Rate (mm/yr): Therapeutic Regimen (3 cycles) Bel-sar – B
Slide 18: Phase 2 Ongoing Tolerability Evaluation Continues to Be Favorable
Ph 2 Ongoing Tolerability Evaluation Continues to Be Favorable No Grade 3 AEs and Majority of AEs Were Transient and Resolved Without Clinical Sequelae Tolerability Profile Supports Indication as a First Line Treatment in Early-Stage Disease *J. Contemp Brachytherapy. J. 2019 Aug; 11(4): 392–397.; A
Slide 19: Cohort Subject with Documented Tumor Growth
Cohort 5 Subject with Documented Tumor Growth Tumor location: Superotemporal Photograph and ultrasound at baseline TT: 1.50 mm, LBD: 8.92 mm (IRC reads) Photograph and ultrasound at 12 months TT: 1.47 mm, LBD: 8.55 mm (IRC reads) Baseline Week 4 Week 8 Week 12 Week 26 Week 39 Week 52 BCVA (letter sc
Slide 20: Randomized Controlled Global Phase 3 Trial
Randomized Controlled Global Phase 3 Trial
Slide 21: Global Phase 3 Trial Design Using Suprachoroidal Administration
Global Phase 3 Trial Design Using Suprachoroidal Administration Fast Track and Orphan Designations Time to Tumor Progression Tumor Growth Rate over 52 weeks Composite time to event analysis: Tumor progression or visual acuity failure between Intervention Group (High Dose) and Sham Group Primary Endp
Slide 22: Clinical Endpoints to Support Approval in Alignment with Regulatory Agencies
Clinical Endpoints to Support Approval in Alignment with Regulatory Agencies Note: Tumor Height (TH) is synonymous with Tumor Thickness; VA – Visual Acuity; Bel-sar – Belzupacap Sarotalocan Composite Endpoint Conservative Assumptions Provide >90% Power to Support Potential for a Single Global Phase
Slide 23: Choroidal Metastasis
Choroidal Metastasis 23 aura
Slide 24: Ocular Oncology Franchise Represents a Multi-Billion Dollar Addressable Market Opportunity
Ocular Oncology Franchise Represents a Multi-Billion Dollar Addressable Market Opportunity Ocular Oncology Franchise total addressable market Same Call Point as CM ~100 Ocular Oncologists in US/EU — focused call point Same Field Force as CM <20 Field Based Team Intend to add small sales force to lau
Slide 25: Choroidal Metastasis is a High Unmet Medical Need
Choroidal Metastasis is a High Unmet Medical Need Choroidal Metastasis Cause Decreased Vision and Decreased Quality of Life in Patients Fighting Metastatic Cancer Originate from Multiple Primary Cancers (e.g. Breast and Lung) 20,000/year eyes diagnosed in US 88% with choroidal location 72% unilatera
Slide 26: Bel-sar Demonstrated Dose-Dependent Activity for Cancer Types Known to Metastasize to the Choroid
Tumor Regression Prolonged Survival Breast Cancer In-Vivo (Syngeneic Mouse Model, EMT-6) Tumor cells were implanted subcutaneously. AU-011 treatment was initiated when tumors reached approximately 50 mm3. Treatment consisted of a single intravenous administration of AU-011 followed 12 hours later by
Slide 27: Urologic Oncology
Urologic Oncology Bel-sar INN: belzupacap sarotalocan Target Indications: Non-Muscle Invasive Bladder Cancer
Slide 28: Bel-Sar is a Clinical Asset with a Multibillion Dollar Market Opportunity in NMIBC
Bel-Sar is a Clinical Asset with a Multibillion Dollar Market Opportunity in Non-Muscle Invasive Bladder Cancer NMIBC is High Unmet Need High Incidence globally >500,000 patients/year Rate of recurrence is high Bel-Sar’s MoA Well Suited to NMIBC Strong precedent for immune-activators in NMIBC (BCG)
Slide 29: NMIBC is a High Unmet Need with High Recurrence Rate
NMIBC is a High Unmet Need with High Recurrence Rate Mechanism of Action Supports Bel-sar Opportunity as Potential Front-Line Treatment Following Initial Diagnosis and/or BCG Refractory Disease Source: Putnam Associates Primary Research & Literature Review, July 2021; NMIBC – Non-Muscle Invasive Bla
Slide 30: Durable CRs with Single Administration of Bel-sar in Bladder Cancer Model
Durable CRs with Single Administration of Bel-sar in Bladder Cancer Model Data Demonstrate Robust Nonclinical Activity Supporting Development of Bel-sar as Single Agent and in Combination with Checkpoint Inhibitors Syngeneic Mouse Tumor Bladder Model (MB49 Model in C57BL/6 Mice) (N=8 -10/group) Tumo
Slide 31: Novel Approach using Intra-mural Administration
Novel Approach using Intra-mural Administration Ongoing Phase 1 Trial Designed to Establish Safety, and Optimize Administration in Intermediate and High Risk NMIBC Patients Intra-mural Administration Bel-sar, AU-011 – Belzupacap Sarotalocan 31 Distribution Into the Mucosal Layer Into the Bladder Wal
Slide 32: Ongoing Phase 1 Trial Evaluating Bel-sar Distribution, Local Necrosis and Immune Activation
Ongoing Ph 1 Trial Evaluating Bel-sar Distribution, Local Necrosis and Evidence of Immune Activation Bel-sar, AU-011 – Belzupacap Sarotalocan 32 Without Light With Light
Slide 33: Strategy & Key Milestones
Strategy & Key Milestones * aura
Slide 34: Aura Biosciences Investment Highlights
Aura Biosciences Investment Highlights Technology Platform Clinical Data Highlights 2023 Milestones Key Highlights Virus-like Drug Conjugates Novel class of cancer therapies - tumor specific cytotoxicity combined with immune activation Targeting multiple solid tumor indications initially focusing on
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Slide 36: Appendix: Phase 1b/2 IVT Trial
Appendix: Phase 1b/2 IVT Trial
Slide 37: Phase 1b/2 IVT – Key Patient Populations and Objectives
Phase 1b/2 IVT – Key Patient Populations and Objectives Enrichment Strategy to Enroll Subjects with Actively Growing Tumors Provides Important Insight into How Bel-sar May Perform in Phase 3 Trial Primary Objective: Safety Drug or treatment related adverse events (AEs) / serious adverse events (SAEs
Slide 38: Phase 1b/2 – Demonstrated Favorable Tolerability Profile
Phase 1b/2 – Demonstrated Favorable Tolerability Profile Majority of AEs Were Transient and Resolved Without Clinical Sequelae Tolerability Profile Supports Indication as a First Line Treatment in Early-Stage Disease *J. Contemp Brachytherapy. J. 2019 Aug; 11(4): 392–397.; Arch Ophthalmol. 2000;118(
Slide 39: Phase 1b/2 IVT - 70% Tumor Control Rate and Statistically Significant Growth Rate Reduction
Phase 1b/2 IVT- 70% Tumor Control Rate and Statistically Significant Growth Rate Reduction Positive Data in Two Efficacy Endpoints in Patients with Early-Stage Choroidal Melanoma Cohorts 1-9 include single dose escalation cohorts and multiple dose escalation cohorts up to 1 cycle of treatment; Cohor
Slide 40: Phase 1b/2 – 64% Patients with Active Growth Achieved Tumor Control
Phase 1b/2 – 64% Patients with Active Growth Achieved Tumor Control when Treated with Therapeutic Regimen We Believe Results Support Bel-sar as First Line Treatment to Help Many Patients Avoid the Need for Radiotherapy Change from Baseline in Tumor Thickness Over 12 Months Completed Ph1b/2 IVT trial
Slide 41: Visual Acuity was Preserved in Majority of Patients with IVT Administration of Bel-sar
Visual Acuity was Preserved in Majority of Patients with IVT Administration of Bel-sar Vision was Preserved in Majority of Patients Whereas Radiotherapy Often Leads to Irreversible and Long-Term Severe Vision Loss Populations Total Patients (n) Vision Preservation Rate Failure: Long-term loss ≥15 le