Full Press Release Details
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', expect , should ,
plan , intend , estimate , will , potential and ongoing , among others, although not all forward-looking statements contain these identifying words. These forward-looking statements include statements about the initiation, timing, progress, results
and cost of our research and development programs and our current and future preclinical studies and clinical trials, including statements regarding the timing of initiation and completion of studies or trials and related preparatory work, the
period during which the results of the trials will become available and our research and development programs; our ability to successfully manufacture our drug substances and product candidates for preclinical use, for clinical trials and on a
larger scale for commercial use, if approved; the ability and willingness of our third-party strategic collaborators to continue research and development activities relating to our development candidates and product candidates; our ability to
commercialize our products, if approved; our ability to obtain funding for our operations necessary to complete further development and commercialization of our product candidates; our ability to obtain and maintain regulatory approval of our
product candidates; the size and growth potential of the markets for our product candidates and our ability to serve those markets; our financial performance; our expected cash runway into the second half of 2026; and the implementation of our
business model, including strategic plans for our business and product candidates. Except as otherwise noted, these forward-looking statements speak only as of the date of this presentation, and we undertake no obligation to update or revise any of
such statements to reflect events or circumstances occurring after this presentation. Because forward-looking statements are inherently subject to risks and uncertainties, some of which cannot be predicted or quantified and some of which are beyond
our control, you should not rely on these forward-looking statements as predictions of future events. For a discussion of these and other risks and uncertainties, and other important factors, any of which could cause our actual results to differ
from those contained in the forward- looking statements, see the section entitled Risk Factors in our most recent Annual Report on Form 10-K and Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission (SEC), as well as
discussions of potential risks, uncertainties, and other important factors in our other subsequent filings with the SEC, which are available on the SEC's website at www.sec.gov. The events and circumstances reflected in our forward-looking
statements may not be achieved or occur and actual results could differ materially from those projected in the forward-looking statements. We caution you not to place undue reliance on the forward-looking statements contained in this presentation.
This presentation discusses product candidates that are under preclinical or clinical evaluation and that have not yet been approved for marketing by the U.S. Food and Drug Administration (FDA) or any other regulatory authority. Until finalized in a
clinical study report, clinical trial data presented herein remain subject to adjustment as a result of clinical site audits and other review processes. No representation is made as to the safety or effectiveness of these product candidates for the
use for which such product candidates are being studied. This presentation shall not constitute an offer to sell or the solicitation of an offer to buy, nor shall there be any sale of these securities in any state or other jurisdiction in which such
offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such state or other jurisdiction. 2
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
activation Uveal Melanoma >60,000 patients/yr readouts expected within 2 Ongoing (US/EU) next 6-12 months, Focal treatment approach including early Phase 1 1 FDA SPA Agreement to deliver durable Urologic Oncology bladder
data response ~500,000 patients/yr 3 (globally) Cash expected to fund operations into 2H 2026 1. Special Protocol Assessment (SPA). 2. See sources on slide 8 of this presentation. 3 3 3. Bladder cancer. Putnam & Assoc. Epidemiology
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
cancers with metastasis to YE 2024 - Initial Phase 2 data the choroid, e.g., Breast and Lung) Ocular Surface Cancers OTHER SOLID TUMORS Bladder Cancer (Non-Muscle Invasive (NMIBC) and Muscle Mid-2024 - Early Phase 1 data Invasive (MIBC))
Other HSPG* Expressing Tumors *Virus-like drug conjugates (VDCs) bind to a subset of modified tumor associated glycosaminoglycans (GAGs) that are part of the heparan sulphate chain of heparan sulfate proteoglycans 4 (HSPGs). Schiller et al. Viruses
Bel-sar is a Potential First-in-Class Therapy for Multiple Solid
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):901-11. Kines
RC, et al. Mol Cancer Ther. 2018;17(2):565-74. Kines RC, et al. Cancer Immunol Res. 2021;9:693-706. 6 DAMPs, damage-associated molecular patterns; HSPG, heparan sulfate proteoglycan; VDC, virus-like drug conjugate; VLP, virus-like
particle. Bel-sar, AU-011
Bel-sar Ocular Oncology Target Indications: Therapeutic Area
Primary Uveal Melanoma Metastases to the Choroid Ocular Surface Cancers
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 Melanoma Ocular
Surface Cancers treatment 3 1 ~35,000 patients/year ~11,000 patients/year Leaves up to 87% of patients with major irreversible vision loss ~100 Ocular Oncologists in US/EU - focused call 4 point opportunity <20 Metastases to the Choroid
Field Based Team - Retinoblastoma Intend to add small sales 4 2 1 force to launch globally ~500 patients/year ~20,000 patients/year 1. ClearView & Putnam & Assoc. Epidemiology Analysis Choroidal Melanoma and Choroidal Metastasis 2.
American Cancer Society- Retinoblastoma statistics 3. Includes Conjunctival Melanoma, Primary Acquired Melanosis, Squamous Cell Carcinoma and Ocular Surface Squamous Neoplasia (https://pubmed.ncbi.nlm.nih.gov/12788119/;
https://pubmed.ncbi.nlm.nih.gov/19628487/; https://pubmed.ncbi.nlm.nih.gov/8676629/; https://pubmed.ncbi.nlm.nih.gov/29511061/; https://pubmed.ncbi.nlm.nih.gov/9037556/) 8 4. Bel-sar is an investigational product candidate. Subject to regulatory
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 diagnosed 2
melanoma) 3 with early-stage disease Primary Uveal Melanoma is a Rare and Life-Threatening 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. Uveal melanoma: relatively rare but deadly cancer. Eye (Lond). 2017;31(2):241-257. doi:10.1038/eye.2016.275 3. Clearview & Putnam & Assoc. Market
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) Metastatic (~2,000) 1. Each figure represents ~250 persons. Shields CL et al. Choroidal and ciliary body melanoma. Available at: https://eyewiki.aao.org/Choroidal_and_Ciliary_Body_Melanoma. Accessed May 2, 2024. Epidemiology analysis for
choroidal melanoma and choroidal metastasis by ClearView Healthcare Partners and Putman. 10 10 Enuc., enucleation. CM, Choroidal Melanoma. Incidence: Patients 1 US/EU
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)
20/200) after Brachy Radiation retinopathy 40%+ 1, 2 Therapy Neovascular glaucoma 10% 100% Dry eye syndrome 20% 80% Strabismus 2%+ 60% Retinal detachment 1-2% 40% Vision loss ( 15 letters) ~70% 20% Long-term legal blindness ~90%
( 20/200) 0% Serious Adverse Event Baseline Year 1 Year 2 Year 3 Long Term Scleral necrosis 0-5% Enucleation/eye loss 10-15% Severe vision loss ( 30 ~90% letters) in HRVL 1. 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). 2023;59(6):1131. 2. Tsui I, Beardsley RM, McCannel TA, Oliver SC, et al. Visual
acuity, contrast sensitivity and color vision three years after iodine-125 brachytherapy for choroidal and ciliary body melanoma. Open Ophthalmol J. 2015;9:131-5. 3. Shields CL et al. Arch Ophthalmol. 2000;118(9):1219-1228. 4. Peddada KV et al. J
Contemp Brachytherapy. 2019;11(4):392-397. 5. Jarczak J et al. Medicina (Kaunas). 2023;59(6):1131. 6. Shields CL et al. Curr Opin Ophthalmol. 2019;30(3):206-214. 7. Kaliki S, Shields CL. Eye 2017;31(2):241-257. AE, adverse event; BCVA,
best-corrected 11 visual acuity; HRVL, high-risk for vision loss.
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 treat early and
reduce risk of metastases Two ~2 minute Two ~5 minute Injections Lasers Improvement in safety and quality of life In-Office Procedure Bel-sar is an investigational product candidate. Subject to regulatory approval. 12
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 g 9
doses- 80 g 2 doses- 40 g x 2 Lasers 1 dose- 20 g 1 dose- 40 g 1 dose- 40 g x 2 Lasers Tumor Progression 0.5mm or 1.5 mm in Largest x 2 Lasers QWx3 x 1 Laser x 1 Laser x 2 Lasers QWx3 QWx2 Up to 3 cycles
Basal Diameter (LBD) 3 cycles Cohort 1 Cohort 2 Cohort 3 Cohort 4 Cohort 5 Cohort 6 (n=1) (n=3*) (n=2) (n=3) 2 Cycles (n=1) (n=up to 10) Decrease from Baseline: 3 Cycles (n=2) Visual Acuity Loss 15 letters Change in Rate of Growth
Subtherapeutic Regimens Therapeutic Regimen Tumor Thickness Growth Rate of Tumor Thickness N=10 N=11** 3 Cycles (9 doses) 1- 2 Doses (n=9); 2 cycles-6 doses (n=1) One Cycle = Doses on days 1, 8 and 15 Goal: To Determine Safety, Optimal Dose and
Therapeutic Regimen with Suprachoroidal Administration *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 unrelated SAEs
is not included in data analysis (n=11). Data that follows will be based on a cohort of 11 13 ClinicalTrials.gov Identifier: NCT04417530 ; AU-011-202
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 in BCVA in Phase
3 Eligible Median Vision Graph Change Over Time Cycle 3 N=10 Patients with Therapeutic Regimen (n=10) Safety Analysis Set new window 100% Subtherapeutic Therapeutic 3 80% 1 80% -1 60% -3 -5 40% -7 20% 20% -9 -11 0% -13 Up to 2 Cycle Regimens 3 Cycle
Regimens (n=10) (n=10) -15 0 3 6 9 12 Tumor Progression: change from baseline in thickness 0.5mm; or in LBD Months Vision Loss (15 letters) 1.5mm confirmed by at least one repeat assessment wind August 3, 2023, data on file Aura
Biosciences Vision acuity loss definition based on ETDRS BCVA letter Change from Baseline score ( 15 letters from baseline) ' PROGRAM fVA2chgm.sas Outputs are not QCed Programmatically or by Prometrika 14 % Subjects with Tumor Control Change
SPA Agreement with FDA Supports Global Phase 3 Trial Design Fast Track
and Orphan Drug Designations 80 g bel-sar treatment arm (n=40) SPA Agreement 15 Month Primary Efficacy Analysis Randomize 40 g bel-sar treatment arm (n=20) (2:1:2) Sham control arm (n=40) Enrollment (N=~100) Primary Endpoint First Key
Secondary Endpoint Time to Tumor Progression Time to Composite Endpoint: Tumor Progression or Visual Acuity Failure An SPA Indicates Concurrence by the FDA that the Design of the Trial can Adequately Support a Regulatory Submission 15
SPA - Special Protocol Assessment
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
trial with high 0.0 statistical significance Subtherapeutic 0 100 200 300 400 500 ( 2 cycles), n=10 3 cycles, n=10 1.0 + Censored 0.8 Time to composite 0.6 endpoint 0.4 Time to tumor progression or vision Log-rank test acuity failure
( 15 letter loss in P = 0.0023 ETDRS-BCVA), whichever occurs 0.2 earlier 0.0 0 100 200 300 400 500 Days Study duration 12 months. Participants either had an event or were censored at the last visit; some had their Week 52 visit after 365 days.
Any events at the final visit are assigned to the actual time of that visit. Log-rank test p-value based on unsimulated original Kaplan-Meier curves. BCVA, best-corrected visual acuity; ETDRS, Early Treatment Diabetic Retinopathy Study; LBD, largest
basal diameter. August 3, 2023 data on file, Aura 16 Biosciences. ClinicalTrials.gov Identifier: NCT04417530; AU-011-202. Progression-free probability Progression-free probability
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% ( 60) ( 60) than Phase 2 power power >99%
>99% ( 30) ( 20) power 40% Overall rate power in Phase 2 Actual rate with 20% documented growth in Phase 2 0% Sham Bel-sar Same dose, regimen, route of administration, range of tumor sizes and reading center as Phase 2 trial Phase 3
Similar population to Phase 2 participants receiving the therapeutic regimen trial design Enriching for early documented growth; Phase 3 randomization stratified by growth rate 17 ClinicalTrials.gov Identifier: NCT06007690;
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 3 1 ~35,000
patients/year ~11,000 patients/year Morbidities Daily treatments of radiotherapy for 4 weeks ~100 Ocular Oncologists in US/EU - focused call 4 point opportunity <20 Metastases to the Choroid Field Based Team - Retinoblastoma Intend to
add small sales 4 2 1 force to launch globally ~500 patients/year ~20,000 patients/year 1. ClearView & Putnam & Assoc. Epidemiology Analysis Choroidal Melanoma and Choroidal Metastasis 2. American Cancer Society- Retinoblastoma statistics 3.
Includes Conjunctival Melanoma, Primary Acquired Melanosis, Squamous Cell Carcinoma and Ocular Surface Squamous Neoplasia (https://pubmed.ncbi.nlm.nih.gov/12788119/; https://pubmed.ncbi.nlm.nih.gov/19628487/;
https://pubmed.ncbi.nlm.nih.gov/8676629/; https://pubmed.ncbi.nlm.nih.gov/29511061/; https://pubmed.ncbi.nlm.nih.gov/9037556/) 18 4. Bel-sar is an investigational product candidate. Subject to regulatory approval.
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 80 g 160 g 200 g 200 g 1 cycle 1 cycle 2 cycles 1 cycle Study Objectives Study Population Patients
with Unilateral, Unifocal Safety/Dose-limiting Toxicity Metastases to the Choroid Efficacy Breast or Lung Primary Change in Tumor Size No Changes in Concurrent Systemic Change in Vision Letter Score
Medications Planned Highlights: Primary Endpoint at One-month Post-treatment; Possibility to See Tumor Shrinkage and Vision Improvement 19 *3+3 Design. Each cohort to have a minimum of 3 and a maximum of 6 patients.
Bel-sar Urologic Oncology Target Indications: Therapeutic Area
Non-muscle invasive bladder cancer Muscle invasive bladder cancer
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
surgeries, Progression of Disease, Loss of Bladder/Cystectomy, Progression of Disease, Loss of Metastasis and Survival Bladder/Cystectomy 1. Bladder cancer. Putnam & Assoc. Epidemiology Analysis. 21
Current Treatment Paradigm for Non-Muscle Invasive Bladder Cancer Low