Aura Biosciences, Inc. · NASDAQ:AURA
Aura Biosciences — Investor Presentation (November 2022)
41 slides from Aura Biosciences's investor presentation, Nov 30, 2022
Slide 1: Corporate Presentation November 2022
Corporate Presentation November 2022 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 ocular tumors, bladder cancer and other solid tumor indications Ocular Oncology Franchise Multi-billion-dollar marke
Slide 4: 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: Experienced Executive Team and Board
Experienced Executive Team and Board Elisabet de los Pinos, PhD Founder & Chief Executive Officer Cadmus Rich, MD Chief Medical Officer, Head of R&D Julie Feder Chief Financial Officer Mark De Rosch, PhD Chief Operating Officer 20+ average years of experience 20+ Regulatory drug and device approvals
Slide 6: Targeted Oncology Platform - Virus-Like Drug Conjugates (VDCs)
Targeted Oncology Platform - Virus-Like Drug Conjugates (VDCs) Virus-Like Particles 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, Fe
Slide 7: 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 8: Ocular Oncology Franchise
Ocular Oncology Franchise Bel-sar INN: belzupacap sarotalocan Target Indications: Early-Stage Choroidal Melanoma Choroidal Metastasis Other Ocular Cancers
Slide 9: 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 10: Current Standard of Care is Invasive with Significant Co-Morbidities
Current Standard of Care is Invasive with Significant Co-Morbidities Standard of Care Often Results in Irreversible Vision Loss and Does Not Reduce Rate of Developing Metastasis Standard of Care is Radiotherapy or Enucleation
Slide 11: 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 12: Ocular Oncology Franchise Represents a Multi-Billion Dollar Commercial Opportunity
Ocular Oncology Franchise Represents a Multi-Billion Dollar Commercial 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 major irr
Slide 13: Ocular Oncology Franchise: Early-Stage Choroidal Melanoma Clinical Program
Ocular Oncology Franchise Bel-sar INN: belzupacap sarotalocan Initial Target Indication: Early-Stage Choroidal Melanoma Clinical program
Slide 14: 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 Response to Treatment Evaluated by Local Tumor Control Baseline Measurement 3mm 3mm 3mm Many early-stage melanomas have a small component of melanoma cells within a benign nevus Bel-sar targets mostly the malignant cells
Slide 15: Study Design and Clinical Endpoints in Phase 1b/2 IVT Trial
Study Design and Clinical Endpoints in Phase 1b/2 IVT Trial Dose escalation and expansion study with up to two cycles of therapy Evaluated safety and efficacy over 12 months Additional follow up in registry trial for 4 years to evaluate vision, tumor control and onset of metastases Key Endpoints Ali
Slide 16: 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 17: Phase 1b/2 – Demonstrated Favorable Safety Profile
Phase 1b/2 – Demonstrated Favorable Safety Profile Majority of AEs Were Transient and Resolved Without Clinical Sequelae Safety 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(9):1219-1228
Slide 18: Phase 1b/2 IVT - 70% Tumor Control Rate and 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 19: Visual Acuity was Preserved in Majority of Patients with IVT Administration
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
Slide 20: Phase 2 Suprachoroidal Study
Phase 2 Suprachoroidal Study
Slide 21: Evaluating Suprachoroidal Administration to Determine Optimal Route for Phase 3
Evaluating Suprachoroidal Administration to Determine Optimal Administration Route for Phase 3 Trial 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 Single Dose Cohorts – Completed Multiple Dose Cohorts 20 μg x 1
Slide 22: Phase 2 SC - Interim Tumor Control Rates Demonstrated Dose Response
Phase 2 SC - Interim Tumor Control Rates Demonstrated 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)
Slide 23: Phase 2 SC - Interim Analysis Demonstrates Tumor Control Rate 89%-100%
Phase 2 SC - 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 Ongoing Phase 2 SC trial (AU-011-202), post-SOC data not included *1 subject without post-baseline tumor t
Slide 24: Phase 2 SC – Statistically Significant Tumor Growth Rate Reduction
n Historical Growth Rate (mm/yr) AU-011 Growth Rate (mm/yr) Growth Rate Reduction (mm/yr) p-value Average Follow up (months) Active Growth and Highest Dose/Regimens* 3 Cycles 9 0.463 0.166 -0.296 0.0007 6 3 Cycles^ 8 0.390 0.090 -0.300 0.0002 6 Tumor thickness growth rates/ slopes estimated using MM
Slide 25: Phase 2 SC – Interim Analysis Shows Visual Acuity Preservation 89%-100%
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 26: Phase 2 SC - Ongoing Tolerability Evaluation Continues to Be Favorable
Phase 2 SC - Ongoing Tolerability Evaluation Continues to Be Favorable Bel-sar – Belzupacap Sarotalocan; SC – Suprachoroidal; DLT – Dose limiting toxicities; AE – Adverse event; SAE – Serious adverse event; TR- Treatment Related Majority of AEs were transient and resolved without clinical sequelae N
Slide 27: Phase 2 Interim Data with SC Administration Supports Moving into Phase 3
Phase 2 Interim Data with SC Administration Supports Moving into Phase 3 Statistically significant reduction in tumor growth rate (p=0.0007) High tumor control rate of 90% in subjects treated with the therapeutic regimen Interim tolerability profile and clinical results in Ph2 dose escalation study
Slide 28: Phase 3 Trial
Phase 3 Trial 7 aura
Slide 29: 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 (30:15:30) Intervention Group High Dose Regimen Intervention Group Low Dose Regimen Sham Group Primary Efficacy Endpoint Analysis Time to Tumor Progression Tumor Gr
Slide 30: 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 31: Choroidal Metastasis
Choroidal Metastasis = aura
Slide 32: Choroidal Metastasis are a High Unmet Medical Need
Choroidal Metastasis are 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 Standar
Slide 33: Bel-sar has Demonstrated Dose-Dependent Activity for Cancer Types Known to Metastasize to the Choroid
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 34: Urologic Oncology
Urologic Oncology Bel-sar INN: belzupacap sarotalocan Target Indications: Non-Muscle Invasive Bladder Cancer
Slide 35: NMIBC is a High Unmet Need With No Approved Targeted Therapies
NMIBC is a High Unmet Need With No Approved Targeted Therapies Mechanism of Action Supports Bel-sar Opportunity as 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 36: 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 Pre-clinical 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) Tum
Slide 37: Ongoing Phase 1 Evaluating Bel-sar Distribution, Local Necrosis and Immune Activation
Ongoing Phase 1 Evaluating Bel-sar Distribution, Local Necrosis and Evidence of Immune Activation Ongoing Phase 1 Designed to Establish Safety, and Optimize Administration in Intermediate and High Risk NMIBC Patients Intra-mural Administration Bel-sar – Belzupacap Sarotalocan Without Light With Ligh
Slide 38: Phase 1 Trial Intends to Provide Foundation for Clinical Development
Phase 1 Trial Intends to Provide Foundation for Clinical Development and Accelerated Approval Ph 1 Study Informs Safety / feasibility of intratumoral, intramural injection +/- laser Tissue and systemic distribution Immune activation Ph 1 Study Populations Phase 1 Bel-sar Tested In: Low/Intermediate
Slide 39: Strategy & Key Milestones
Strategy & Key il Milestones | a Fi] Se 7 aura
Slide 40: 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 ocular tumors, bladder cancer and other solid tumor indications Ocular Oncology Franchise Multi-billion-dollar marke
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