Aura Biosciences, Inc. · NASDAQ:AURA
Aura Biosciences — Investor Presentation (June 2024)
33 slides from Aura Biosciences's investor presentation, Jun 4, 2024
Slide 1: Corporate Presentation June 2024
Exhibit 99.1 Corporate Presentation June 2024
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 , could'
Slide 3: Well Positioned with Multiple Near-Term Clinical Catalysts
Well Positioned with Multiple Near-Term Clinical Catalysts Precision Late-Stage Large Market Opportunity Key Upcoming Catalysts Therapy Clinical Development In Areas of Unmet Need Platform • Direct tumor cell killing • Phase 3 in Primary • Ocular Oncology• Multiple clinical data and immune activatio
Slide 4: Clinical Pipeline Across Multiple Solid Tumor Indications
Clinical Pipeline Across Multiple Solid Tumor Indications Planned Program Preclinical Phase 1 Phase 2 Phase 3 Milestones OCULAR ONCOLOGY 2024 – Phase 3 enrollment ongoing Primary Uveal Melanoma YE 2024 – Phase 2 12-month data Metastases to the Choroid 2024 – Phase 2 initiation (Multiple primary canc
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 has a novel dual mechanism of action
Reactive oxygen species disrupts cell membrane and organelles Bel-sar has 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 Kines RC, et al. Int J Cancer. 2016;138(4):90
Slide 7: Bel-sar Ocular Oncology Target Indications
Bel-sar Ocular Oncology Target Indications: Therapeutic Area • Primary Uveal Melanoma • Metastases to the Choroid • Ocular Surface Cancers
Slide 8: 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 ~66,000 patients/year ~80% Ocular Oncology Total Addressable Market (US/EU) of patients are diagnosed at the early stage (indeterminate lesions (ILs) and small tumors) Current radiotherapy Primary Uveal Mel
Slide 9: Primary Uveal Melanoma—High Unmet Medical Need
Primary Uveal Melanoma—High Unmet Medical Need Most common primary 2 Uvea: Choroid, Ciliary Body and Iris intraocular cancer in adults Choroid is 90% 1 of the uvea choroid Impacts ~11,000 3 50% of patients patients in US/EU per year develop metastasis within 15 years (metastatic uveal ~80% patients
Slide 10: Current Treatment Paradigm for Uveal Melanoma
Current Treatment Paradigm for Uveal Melanoma Small Medium Large Metastatic SIZE 1 2.5–3 >10 Indeterminate Small (mm): Lesions Melanomas Risk Factors Growth Small CM Enuc. Observation Systemic Chemotherapy Radiotherapy Radiotherapy ® (KIMMTRAK ) Local – Early (~8,000) Local – Late (~2,300) Metastati
Slide 11: High Morbidity Associated with Current Standard of Care
High Morbidity Associated with Current Standard of Care 3–7 Radiotherapy Up to 87% of Primary Uveal Melanoma Patients Become Legally Blind Over Time in the Eye Adverse Event Proportion of patients 1,2 Treated with Radiotherapy Surgeries secondary to AEs legally blind (BCVA 40%+ (e.g., cataracts) ≤20
Slide 12: Bel-sar has the Potential to be the First Approved Therapy in Primary Uveal Melanoma
Bel-sar has the Potential to be the First Approved Therapy in Primary Uveal Melanoma Bel-sar is Delivered by Simple Light Activation with Goals of Suprachoroidal Injection Standard Ophthalmic Laser Treatment Local tumor control Preservation of vision c No radiation-related morbidity Opportunity to t
Slide 13: 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) Endpoint Endpoint Definitions Growth in Tumor Height 6-9 doses- 40 μ
Slide 14: Phase 2 Interim Data Demonstrates Tumor Control, Vision Preservation and Excellent Safety
Phase 2 Interim Data Demonstrates Tumor Control, Vision Preservation and an Excellent Safety Profile 80% Tumor Control Rate 90% Visual Acuity Preservation Rate <20% Grade 1 AEs Aura Biosciences, Inc. Protocol AU-011-202 Exctraction 9/18/2023 Data Cut 3 Aug 2023 Open-Label Data Review Median Change i
Slide 15: 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 80 µg bel-sar treatment arm (n=40) SPA Agreement 15 Month Primary Efficacy Analysis Randomize 40 µg bel-sar treatment arm (n=20) (2:1:2) Sham control arm (n=40) Enrollment (N=~100) Primary Endpoint Fi
Slide 16: Kaplan-Meier Simulation of Phase 3 Based on Phase 2 Data
1.0 + Censored Time to tumor 0.8 Kaplan-Meier analysis progression simulation of Phase 2 0.6 Change from baseline in thickness Log-rank test interim data support ≥0.5 mm or in LBD ≥1.5 mm 0.4 confirmed by at least one repeat P = 0.0012 assumptions for the assessment potential success of 0.2 Phase 3
Slide 17: Phase 2 Interim Data Support Phase 3 Assumptions
Phase 2 Interim Data Support Phase 3 Assumptions Robustness Analysis of Phase 2 interim tumor control rates 100% Overall rate P < 0.05 P < 0.005 in Phase 2 80% 2x “worse” than Phase 2 Actual data Actual data 60% 2x “worse” 94% 93% (Δ60) (Δ60) than Phase 2 power power >99% >99% (Δ30) (Δ20) power 40%
Slide 18: 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 ~66,000 patients/year Majority originate from breast and Ocular Oncology Total Addressable Market (US/EU) lung cancer Burdensome Standard Primary Uveal Melanoma Ocular Surface Cancers of Care with Radiation
Slide 19: Metastases to the Choroid – Phase 2 Trial Expected to Begin in 2024
Metastases to the Choroid – Phase 2 Trial Expected to Begin in 2024 Planned Study Design (n=12*) Cohort 1 Cohort 2 Cohort 3 Cohort 4 N=3 patients N=3 patients N=3 patients N=3 patients 80µg 160µg 200µg 200µg 1 cycle 1 cycle 2 cycles 1 cycle Study Objectives Study Population • Patients with Unilatera
Slide 20: Bel-sar Urologic Oncology Target Indications
Bel-sar Urologic Oncology Target Indications: Therapeutic Area • Non-muscle invasive bladder cancer • Muscle invasive bladder cancer
Slide 21: Bladder Cancer is a Global High Unmet Medical Need
Bladder Cancer is a Global High Unmet Medical Need Non-Muscle Invasive Bladder Cancer Muscle Invasive Bladder Cancer ~500,000 1 New cases/ year globally >200,000 NMIBC 1 New cases/year US, Europe & Asia >60,000 Unmet Need Unmet Need MIBC 1 New cases/year US, Europe & Asia Recurrence, multiple TURBT
Slide 22: Current Treatment Paradigm for Non-Muscle Invasive Bladder Cancer
Current Treatment Paradigm for Non-Muscle Invasive Bladder Cancer Low grade – Low & intermediate risk High risk papillary disease CIS – BCG unresponsive Progression Adjuvant therapy Intermediate risk High risk adjuvant therapy adjuvant therapy Intravesical gene therapy ® (Adstiladrin ) BCG >6 tx BCG
Slide 23: 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 Bel-sar’s Local Administration Goals of Treatment Aligned with with Bel-sar Current Urologic Oncology Practice Focal Treatment with Direct Tumor Cell Killing No Virus Replication or Viral Shedding Stimula
Slide 24: Phase 1 Trial for Bladder Cancer
21 participants TURBT and cystectomy patients NMIBC and MIBC patients Phase 1 trial for bladder cancer designed to Part 1 (n=5) Part 2 (n=16) evaluate safety, Bel-sar alone Bel-sar + focal light activation feasibility and MoA NMIBC MIBC n=10 n=6 Completed No treatment-related AEs, SAEs, or dose-limi
Slide 25: Clinical Complete Response with Immune Activation after Single Dose
Clinical Complete Response with Immune Activation after Single Dose Confirmed by Histopathology in Part 2 First Patient Evidence of complete Immune response by absence of tumor infiltrate cells, as well as immune activation, after single dose Example of papillary treatment in first patient carcinoma
Slide 26: Company Highlights
Company Highlights Ocular Oncology Therapeutic Area Urologic Oncology Therapeutic Area • Primary Uveal Melanoma – Global Phase 3 • Bladder Cancer – Phase 1 Trial CoMpass Trial: • Clinical complete response in first patient with • Trial actively enrolling single dose • Special Protocol Assessment (SP
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Slide 28: Appendix: Phase 2 Primary Uveal Melanoma Trial – Interim Data
Appendix: Phase 2 Primary Uveal Melanoma Trial – Interim Data
Slide 29: High Local Complete Response Rate at 12 Months Follow-up
High Local Complete Response Rate at 12 months Follow-up* Dose Response: >90% Completed 12 Months Subtherapeutic vs Therapeutic Regimen Total Patients Tumor Control 100% Dose/Regimen Subtherapeutic Therapeutic (n) Rate 80% Subtherapeutic Regimens 80% Single dose up to 2 cycles 10 20% (2/10) 60% Ther
Slide 30: High Tumor Control Rates Observed in Phase 3 Population
High Tumor Control Rates Observed in Phase 3 Population Treated with Therapeutic Regimen in Phase 2* Aura Biosciences, Inc. Protocol AU-011-202 Exctraction 9/18/2023 Data Cut 3 Aug 2023 Subtherapeutic Regimens (n=10) Active Growth and 3 Cycle Regimens (n=10) Open-Label Data Review Figure Tumor Thick
Slide 31: Phase 2 Interim Data Demonstrated Complete Cessation of Growth Among Responders
Phase 2 Interim Data Demonstrated Complete Cessation of Growth Among Responders Successful Treatment with 3 Cycle Regimen in Phase 3 Eligible Tumors with Active Growth Change in Tumor Growth (mm/yr) (n=8) P < 0.0001 + 0.383 - 0.001 August 3, 2023, data on file Aura Biosciences Tumor thickness growth
Slide 32: 90% Visual Acuity Preservation
90% Visual Acuity Preservation Despite 80% of These Phase 2 Patients Being at High Risk for Vision Loss* >90% Patients Completed 12 months Aura Biosciences, Inc. Protocol AU-011-202 Exctraction 9/18/2023 Data Cut 3 Aug 2023 Open-Label Data Review Total Vision Vision Median Change in BCVA in Phase 3
Slide 33: 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 Ongoing Phase 2 Safety Outcomes with SC Administration + Surgeries secondary to AEs (e.g., 40%+ 0% Cataracts) All Treated Subjects (n=22) Grade III Total Drug/Las