Aura Biosciences, Inc. · NASDAQ:AURA
Aura Biosciences — Investor Presentation (January 2024)
41 slides from Aura Biosciences's investor presentation, Jan 8, 2024
Slide 1: Corporate Presentation January 2024
Corporate Presentation January 2024 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 – Developing a Novel Class of Drugs in Oncology
Aura Biosciences – Developing a Novel Class of Drugs in Oncology Ocular Oncology Franchise Choroidal Melanoma – Global Ph 3 CoMpass Trial: FPI was Q4 2023 Most US sites activated; Trial actively enrolling Special Protocol Assessment (SPA) Agreement with FDA Ph 3 assumptions supported by Ph 2 data Ch
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 (Suprachoroi
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
Primary Choroidal Melanoma—High Unmet Medical Need Choroidal Melanoma is a Rare and Life-Threatening Primary 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. Uve
Slide 9: Radiotherapy is the Standard of Care for Early-Stage Disease
Radiotherapy is the Standard of Care for Early-Stage Disease Surgical treatment of large choroidal melanoma with ruthenium 106 plaque brachytherapy2 1. Up To Date. Plaque brachytherapy for uveal melanoma. Accessed September 20, 2023. https://ykhoa.org/d/image.htm?imageKey=ONC/116591 2. Kim M, Lee SM
Slide 10: Up to 87% of Patients Will be Blinded when Treated with Radiotherapy
Up to 87% of Patients Will be Blinded when Treated with Radiotherapy 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).
Slide 11: Benefit/Risk of Radiotherapy Drives "Watch and Wait" in Early-Stage Disease
Benefit/Risk of Radiotherapy Drives “Watch and Wait” in Patients with Early-Stage Disease Brachytherapy: Vision Loss/Blindness Clearview & Putnam & Assoc. Market Research Kaliki S, Shields CL. Uveal melanoma: relatively rare but deadly cancer. Eye (Lond). 2017;31(2):241-257. doi:10.1038/eye.2016.275
Slide 12: 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 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 Suprach
Slide 13: 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 (US/EU) ~80% of patients are diagnosed at the early stage (indeterminate lesions (ILs) and small tumors) Current radiotherapy treatment Leaves ~87% of patient
Slide 14: Bel-Sar Potential Vision Preserving Therapy for Indeterminate Lesions and Small Choroidal Melanoma
Bel-Sar has the Potential to be an Effective, Vision Preserving Therapy for the Treatment of Indeterminate Lesions and Small Choroidal Melanoma Potential Future Patient Treatment Journey Treatment First Line Treatment with Bel-sar Brachytherapy: Vision Loss/Blindness (~8,000)1 (~2,300)1 (~2,000)2 In
Slide 15: Adverse Event Comparison: KIMMTRAK, Darovasertib + Crizotinib, Bel-Sar
Adverse Event or Adverse Reactions KIMMTRAK1 Darovasertib + Crizotinib Combination2 Bel-Sar Any Grade Any Grade Any Grade* Cytokine release syndrome 89% Not Disclosed 0% Rash 83% Not Disclosed 0% Pyrexia 76% Not Disclosed 0% Pruritus 69% Not Disclosed 0% Fatigue 64% 40% 0% Nausea 49% 79% 0% Vomiting
Slide 16: Choroidal Melanoma Ph 2 Data
Choroidal Melanoma Ph 2 Data
Slide 17: Ph 2 Trial – Dose Escalation and Expansion with Suprachoroidal Administration
Ph 2 Trial – Dose Escalation and Expansion with Suprachoroidal Administration Trial Design – Enrollment Complete (n=22) *Cohort 2: 2 subjects were planned; third subject was additionally enrolled due to dose error in 1 subject **12 patients enrolled, 1 subject who discontinued after 1 cycle due to u
Slide 18: High Tumor Control Rates Observed – Durable at 12 Months Follow Up
High Tumor Control Rates Observed – Durable at 12 Months Follow Up Dose Response: Subtherapeutic vs Therapeutic Regimen 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 Dose/Regimen
Slide 19: High Tumor Control Rates Observed in Ph 3 Population Treated with Therapeutic Regimen
High Tumor Control Rates Observed in Ph 3 Population Treated with Therapeutic Regimen August 3, 2023, data on file Aura Biosciences Active Growth and 3 Cycle Regimens (n=10) Progression Definition based on Tumor Thickness (Increase ≥0.5mm) Change from Baseline in Tumor Thickness Over 12 Months Treat
Slide 20: Ph 2 Interim Data Demonstrated a Negative Growth Rate Post-Treatment
Ph 2 Interim Data Demonstrated a Negative Growth Rate Post-Treatment Successful Treatment with 3 Cycle Regimen in Ph 3 Eligible Tumors with Active Growth Change in Tumor Growth (mm/yr) (n=8) August 3, 2023, data on file Aura Biosciences Tumor thickness growth rates/ slopes estimated using Mixed Mode
Slide 21: 90% Visual Acuity Preservation
. Populations Total Patients (n) Vision Failures (n) Vision Preservation Rate All Dose Cohorts All Treated Patients 22 1 96% Subtherapeutic Single dose up to 2 cycles 10 0 100% Therapeutic Regimens 3 Cycles (n=11) 11 1 91% 3 Cycles and Ph 3 eligible (n=10)* 10 1 90% >90% Patients Completed 12 months
Slide 22: Ph 2 Safety Data Supports Potential to be First Line Treatment
Ph 2 Safety Data Supports Potential to be First Line Treatment in Early-Stage Disease No Posterior Inflammation, No SAEs and No Grade 3 Related Adverse Events *J. Contemp Brachytherapy. J. 2019 Aug; 11(4): 392–397.; Arch Ophthalmol. 2000;118(9):1219-1228; Curr. Opin. Ophthalmol. 2019 May;30(3):206-2
Slide 23: Randomized Controlled Global Ph 3 Trial
Randomized Controlled Global Ph 3 Trial
Slide 24: SPA Agreement with FDA Supports Global Ph 3 Trial Design
SPA Agreement with FDA Supports Global Ph 3 Trial Design Fast Track and Orphan 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 treatment arm
Slide 25: Time to Composite Endpoint and Time to Tumor Progression
Note: Subjects either had an event or were censored at the last visit. Some subjects had Week 52 visit after 365 days. Time to Composite Endpoint is defined as time to tumor progression or vision acuity failure, whichever occurs earlier. Tumor progression is defined as a change from baseline in thic
Slide 26: Clinical Endpoints to Support Potential Approval in Alignment with Regulatory Agencies
Clinical Endpoints to Support Potential Approval in Alignment with Regulatory Agencies Note: Tumor Height is synonymous with Tumor Thickness; VA – Visual Acuity; Bel-sar – Belzupacap Sarotalocan Conservative Assumptions Provide Support for Potential for Regulatory Submission (Phase 3 Trial Powered a
Slide 27: Choroidal Metastasis
Choroidal Metastasis
Slide 28: 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% unilater
Slide 29: Choroidal Metastasis – Ph 2 Trial Design
Choroidal Metastasis – Ph 2 Trial Design Cohort 1a N=3 patients 80µg x 2L 1 cycle Cohort 2a N=3 patients 140µg x 2L 1 cycle Cohort 3a N=3 patients 200µg x 2L 1 cycle Cohort 3b N=3 patients 200µg x 2L 2 cycles Safety/DLT Efficacy Choroidal Tumor Progression Choroidal Tumor Growth rate Vision improvem
Slide 30: Urologic Oncology
Urologic Oncology Bel-sar INN: belzupacap sarotalocan Target Indications: Bladder Cancer
Slide 31: Bladder Cancer is a High Unmet Medical Need
Bladder Cancer is a High Unmet Medical Need Non-Muscle Invasive Bladder Cancer Muscle Invasive Bladder Cancer ~500,000 New cases/ year globally1 61,000 New cases/year in the US1 New cases/year in the US2 20,000 Putnam & Assoc. Epidemiology Analysis Campbell-Walsh-wein Urology, 2021, 12th edition ; C
Slide 32: Bel-Sar has the Potential to be Front Line in Conjunction with TURBT
Intravesical gene therapy (Adstiladrin) Systemic Immunotherapy (Keytruda) INTRAVESICAL CHEMOTHERAPY Bel-Sar has the Potential to be Front Line in Conjunction with TURBT Presents with Blood in urine TURBT BCG >6 tx INTRAVESICAL CHEMOTHERAPY Adding Bel-sar can potentially: Prevent Recurrence after TUR
Slide 33: Bel-Sar has a Unique MoA Designed for the Local Treatment
Bel-Sar has a Unique MoA Designed for the Local Treatment Bel-sar’s Local Administration is Aligned with Clinical Practice and has a Unique MoA that may be Complementary to Other Drugs Local Administration and Light Activation with Standard Cystoscope Bel-sar, AU-011 – Belzupacap Sarotalocan MoA – M
Slide 34: Ph 1 Trial Designed to Evaluate Safety, Feasibility and MoA
Ph 1 Trial Designed to Evaluate Safety, Feasibility and MoA Ph 1 Data is Anticipated to Support the Future Development of Bel-sar for the Treatment of Both NMIBC and MIBC Study Design and Objectives (n=19) Bel-sar Alone Part 1 (n=5) Part 2 (n=14) Bel-sar + Focal Light Activation Completed Ongoing Sa
Slide 35: Clinical Complete Response with Immune Activation after Single Dose
Clinical Complete Response with Immune Activation after Single Dose Confirmed by Histopathology Day 1 Diagnostic biopsy shows non- invasive, low grade urothelial carcinoma Injection of Bel-sar (100ug) performed within tumor and below tumor (Aura present w/ Urologist) DAY 1 Biopsy Day 2 Urologist per
Slide 36: Pre-Treatment Biopsy (Day 1) shows Papillary Urothelial Carcinoma
Pre-Treatment Biopsy (Day 1) shows Papillary Urothelial Carcinoma Papillary urothelial carcinoma Example of papillary carcinoma (Ta) H&E Stain Pre-injection bladder biopsy demonstrating low-grade papillary urothelial carcinoma; non-invasive.
Slide 37: Clinical Complete Response Confirmed by Histopathology after Single Dose of Bel-sar
Clinical Complete Response Confirmed by Histopathology after Single Dose of Bel-sar Disappearance of all cancer cells after treatment and immune activation Post-treatment TURBT demonstrating necrosis, inflammatory infiltrate, and no residual carcinoma. Circled region shows area of necrosis; arrow in
Slide 38: 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 Demonstrated 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) Tum
Slide 39: Strategy & Key Milestones
Strategy & Key Milestones
Slide 40: Aura Biosciences – Developing a Novel Class of Drugs in Oncology
Aura Biosciences – Developing a Novel Class of Drugs in Oncology Ocular Oncology Franchise Choroidal Melanoma – Global Ph 3 CoMpass Trial: FPI was Q4 2023 Most US sites activated; Trial actively enrolling Special Protocol Assessment (SPA) Agreement with FDA Ph 3 assumptions supported by Ph 2 data Ch
Slide 41: aura
aura
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