Aura Biosciences, Inc. · NASDAQ:AURA
Aura Biosciences — Investor Presentation (January 2023)
37 slides from Aura Biosciences's investor presentation, Jan 6, 2023
Slide 1: Corporate Presentation January 2023
Corporate Presentation January 2023 Exhibit 99.1
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 Cancer Developing virus-like drug conjugates (VDCs) that bind to tumor associated HSPGs* and deliver a therapeutic payload Targeting multiple solid tumor indications including ocular and bladder cancers Ocular Oncology Franchise Multi-billion-dolla
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 (Breast, lung and other cancer metastasis in the eye) Primary Choroidal Melanoma (Suprachoroidal administration) Ca
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: Choroidal Melanoma – High Unmet Medical Need with No Drugs Approved
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 common primar
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: Phase 2 Trial - Evaluating Suprachoroidal Administration
Ph 2 Trial - Evaluating Suprachoroidal Administration to Determine Optimal Administration Route Small Tumors with Active Growth Tumor thickness ≥0.5 mm and ≤2.5 mm LBD ≤10 mm Active tumor growth (>0.3mm) within 2 years of screening Same criteria as the planned Phase 3 Single Dose Cohorts – Completed
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 Definition Threshold Methodology Tumor Progression Growth in Tumor Height ≥0.5mm or ≥1.5 mm in Largest Basal Diameter (LBD) Ultrasound and Digital Photography Visual Acuity Loss Long Term Loss ≥15 letters ETDRS-B
Slide 13: Phase 2 Interim Tumor Control Rates Demonstrate a Dose Response
Ph 2 Interim Tumor Control Rates Demonstrate a Dose Response Tumor Progression: change from baseline in thickness ≥0.5mm; or in LBD ≥1.5mm confirmed by at least one repeat assessment 19-Aug-2022 cutoff, interim data Populations Total Patients (n) Tumor Control Rate Average Follow-up (months) All Dos
Slide 14: Phase 2 Interim Analysis Demonstrates Tumor Control Rate 89%-100%
Ph 2 Interim Analysis Demonstrates Tumor Control Rate 89%-100% Established Therapeutic Regimen of 3 Cycles using SC Administration Change from Baseline in Tumor Thickness Over 12 Months** **1 subject without post-baseline tumor thickness data not included in plot Tumor Progression: change from basel
Slide 15: Phase 2 Interim Analysis Shows Visual Acuity Preservation
Populations Total Patients (n) Vision Failures** (n) Vision Preservation Rate Mean Change from Baseline at Last Visit (letters) Average Follow-up (months) All Dose Cohorts All Treated Patients 20 2 90.0% -3.3 8 High Risk for Vision Loss 15 2 86.7% -4.5 7 Highest Doses/Regimens* 2 Cycles (40µg) 1 0 1
Slide 16: Phase 2 Ongoing Tolerability Evaluation Continues to Be Favorable
Ph 2 Ongoing Tolerability Evaluation Continues to Be Favorable Bel-sar – Belzupacap Sarotalocan; DLT – Dose limiting toxicities; SC – Suprachoroidal; AE – Adverse event; SAE – Serious adverse event; TR- Treatment Related Majority of AEs were transient and resolved without clinical sequelae No DLTs o
Slide 17: Phase 3 Trial
Phase 3 Trial v aura
Slide 18: Global Phase 3 Trial Design Using Suprachoroidal Administration
Global Phase 3 Trial Design Using Suprachoroidal Administration Fast Track and Orphan Designations Randomize Small Tumors Active Growth (2:1:2) Intervention Group High Dose Regimen Intervention Group Low Dose Regimen Sham Group Primary Efficacy Endpoint Analysis Time to Tumor Progression Tumor Growt
Slide 19: Clinical Endpoints to Support Approval in Alignment with Regulatory Agencies
Clinical Endpoints to Support Approval in Alignment with Regulatory Agencies Growth Rate Assumptions Vision Failure Assumptions Visual Acuity - ETDRS BCVA Bel-sar: 15% VA Failure Sham: 2% VA Failure Disease Progression Increase from baseline: TH ≥0.5mm LBD ≥1.5mm Visual Acuity Failure Decrease from
Slide 20: Choroidal Metastasis
Choroidal Metastasis * aura
Slide 21: 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 20,000/year eyes diagnosed in US 88% with choroidal location 72% unilateral and solitary Standard
Slide 22: Reduced Tumor Growth Prolonged Survival Breast Cancer In-Vivo
Reduced Tumor Growth 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 late
Slide 23: Urologic Oncology
Urologic Oncology Bel-sar INN: belzupacap sarotalocan Target Indications: Non-Muscle Invasive Bladder Cancer
Slide 24: 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 across all geographies >200,000 patients/year Rate of recurrence is high Bel-Sar’s MoA Well Suited to NMIBC Strong precedent for immune-activators i
Slide 25: 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 26: 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 27: Novel Approach using Intra-mural Administration
Novel Approach using Intra-mural Administration Ongoing Phase 1 Designed to Establish Safety, and Optimize Administration in Intermediate and High Risk NMIBC Patients Intra-mural Administration Bel-sar – Belzupacap Sarotalocan Distribution Into the Mucosal Layer Into the Bladder Wall Bel-sar will be
Slide 28: 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 – Belzupacap Sarotalocan Without Light With Light
Slide 29: Strategy & Key Milestones
Strategy & Key il Milestones | a Fi] Se » aura
Slide 30: Aura Biosciences Investment Highlights
Aura Biosciences Investment Highlights Technology Platform Clinical Data Highlights 2023 Milestones Strong Cash Position Virus-like Drug Conjugates Novel class of oncology targeted therapies Targeting multiple solid tumor indications including ocular and bladder cancers Ocular Oncology Franchise Pos
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Slide 32: Appendix: Phase 1b/2 IVT Trial
Appendix: Phase 1b/2 IVT Trial
Slide 33: 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 34: 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 35: 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 36: 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 37: 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
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