Full Press Release Details
Corporate Presentation January 2023
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 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; the effect of the COVID-19 pandemic, including mitigation efforts and economic effects,
on any of the foregoing or other aspects of our business operations, including but not limited to our preclinical studies and clinical trials and any future studies or trials; our ability to commercialize our products, if approved; and the
implementation of our business model, and 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 filed with the Securities and Exchange Commission, as well as discussions of potential
risks, uncertainties, and other important factors in our other subsequent filings with the Securities and Exchange Commission. 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 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.
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-dollar addressable market opportunity to treat early-stage choroidal melanoma (CM) and other ocular tumors Standard of care is invasive and may lead to blindness and loss of eye Clinical proof of concept with two routes of
administration Advancing to global Phase 3 trial Strong Cash Position Expected to fund operations into 2025 *VDCs bind to a subset of modified tumor associated glycosaminoglycans (GAGs) that are part of the heparan sulphate chain of HSPGs.
Schiller et al. Viruses 2022, 14(8), 1656
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) Cancers of the Ocular Surface Non-Muscle Invasive Bladder Cancer Other HSPG* Expressing Tumors (e.g., Cutaneous Melanoma, HNSCC) 2023 - Phase 2 data 2023 - FPI Phase 3 trial 2023 - Initiate Phase 2
trial 2024 - Phase 2 data 2023 - Phase 1 data Global Commercial Rights for All Product Candidate Indications *VDCs bind to a subset of modified tumor associated glycosaminoglycans (GAGs) that are part of the heparan sulphate chain
of HSPGs. Schiller et al. Viruses 2022, 14(8), 1656
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, February 2016; Kines et al; Molecular Cancer Therapeutics, 17(2) February 2018; Kines et al; Cancer Immunology Research, May 2021 Potential Key Differentiation: Potency, Binding and Selectivity Potential Treatment of Multiple
Solid Tumors *VDCs bind to a subset of modified tumor associated glycosaminoglycans (GAGs) that are part of the heparan sulphate chain of HSPGs. Schiller et al. Viruses 2022, 14(8), 1656
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 Anti-tumor Immunity Kines et al; Cancer Immunology Research, May 2021 VDCs bind to a subset of modified tumor associated glycosaminoglycans (GAG) that are part of the heparan sulphate chain of HSPGs. Schiller et al.
Viruses 2022, 14(8), 1656 Bel-sar - Belzupacap Sarotalocan
Ocular Oncology Franchise Bel-sar INN:
belzupacap sarotalocan Target Indications: Early-Stage Choroidal Melanoma Choroidal Metastasis Other Ocular Cancers
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 primary intraocular cancer in adults Impacts 11,000 patients in US/Europe per year ~80% patients diagnosed with early-stage disease Melanoma cells Melanocytic Lesion Benign nevus cells Standard of Care is
Radiotherapy or Enucleation Blindness, Eye Loss, and Disfiguration The choroid is the part of the uvea that is behind the retina
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 Delivered by Simple Intravitreal or Suprachoroidal Injection Goals of Treatment Light Activation with Standard Ophthalmic Laser Bel-sar - Belzupacap Sarotalocan
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 major irreversible vision loss within 5-10 years ~100 Ocular Oncologists in US/EU - focused call point <20 Field Based Team Intend to add small sales force to launch globally 33,500 ClearView & Putnam & Assoc.
Epidemiology Analysis Choroidal Melanoma and Choroidal Metastasis American Cancer Society- Retinoblastoma statistics Batsi et al Cornea 2003 Ocular Surface squamous neoplasia: a review Multibillion $ addressable market opportunity Cancers of the
Ocular Surface Retinoblastoma Choroidal Melanoma Choroidal Metastasis 11,000 patients/year 20,000 patients/year 500 patients/year 2,000 patients/year 11,000 Choroidal Melanoma patients diagnosed each year (US/EU) Choroidal Melanoma - Initial
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 Multiple Dose Cohorts 20 g x 1 Laser 40 g x 1 Laser 40 g x 2 Lasers 40 g x 2 Lasers QWx2 40 g x 2 Lasers QWx3 Up to 3 cycles Cohort 1 (n=1) Cohort 2 (n=3*) Cohort 3
(n=2) Cohort 4 (n=3) Cohort 5 (n=3) 80 g x 2 Lasers QWx3 Up to 3 cycles Cohort 6 (n=10) ONGOING Ph2 SC Trial Design: Dose Escalation Phase *2 subjects were planned; third subject was additionally enrolled due to dose error in 1 subject
ClinicalTrials.gov Identifier: NCT04417530 suprachoroidal Goal: To Determine Safety, Optimal Dose and Therapeutic Regimen with Suprachoroidal Administration Patient Population Representative of Early-Stage Disease (IL/CM) SC - Suprachoroidal;
IL - Indeterminate Lesion; CM- Choroidal Melanoma; LBD - Largest Basal Diameter
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-BCVA Tumor Thickness Growth Rate Tumor Thickness Growth over 12 Months Ultrasound Bel-sar - Belzupacap Sarotalocan
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 Doses/Regimens All Treated Patients 20 55.0% (11/20) 8 Lower Doses/Regimens+ Less than 1 cycle (20 g-40 g) 9 22.2% (2/9) 11 2 Cycles (40 g) 1 0% (0/1) 6 Highest Doses/Regimens*++ 3 Cycles (n=9) 40 g
(n=2)/80 g (n=7) 9 88.9% (8/9) 6 Average 6 Months of Follow Up *One subject in C6 who discontinued after 1 cycle due to unrelated SAEs is not included +Assigned regimens- up to two cycles: with doses of 20 g x 1 Laser or 40 g x
1 or 2 Lasers ++ Assigned regimens- 3 cycles, each cycle comprised of 3 once/week treatments of 40 g x 2 Laser or 80 g x 2 Laser Dose Response and Interim Tumor Control Rates Demonstrate Meaningful Clinical Benefit 3 Cycle Regimens
vs. Lower Regimens LBD - Largest Basal Diameter Up to 2 cycles regimens (n=10) 3 cycle regimens (n=9)
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 baseline in thickness 0.5mm; or in LBD 1.5mm confirmed by at least one repeat assessment; Interim data cutoff August 19, 2022 Progression Definition based on Tumor Thickness (Increase 0.5mm) Subject 015-2029 had
circumpapillary tumor - similar subjects will be excluded from Phase 3 trial 89%-100% Tumor Control Rate: Active Growth and Therapeutic Regimen (3 cycles) Statistically Significant Change in Tumor Growth (mm/yr): Therapeutic Regimen (3 cycles)
p=0.0007 p=0.0002 0.390 0.090 0.463 0.166 Tumor thickness growth rates/ slopes estimated using MMRM (random intercept and slope model for Hx and Study periods) SC - Suprachoroidal; LBD - Largest Basal Diameter *One subject in C6 who
discontinued after 1 cycle due to unrelated SAEs is not included ^One subject in C6 with circumpapillary tumor not included (similar subjects not planned in Phase 3 trial)
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 (40 g) 1 0 100% -3.0 6 3 Cycles (40 g-80 g) 40 g (n=2)/80 g (n=7) 9 1 88.9% -3.9 6 Vision Preservation Rates *One subject in C6 who discontinued after 1 cycle due to unrelated SAEs is not included
**Confirmed loss 15 letters at Week 39; post-SOC data not included Interim data cutoff August 19, 2022 Ph 2 Interim Analysis Shows Visual Acuity Preservation 89%-100% Interim Data Showed High Vision Preservation Rates
Across All Groups Including Subjects at High Risk for Vision Loss SC - Suprachoroidal
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 or treatment related SAEs No significant vitritis to date through 3 cycles with 80 g of AU-011 No pigmentary changes observed at edge of tumor treatment Table presents percentage of subjects with AEs
related to bel-sar or laser by severity and overall; subjects with more than 1 AE are counted in the highest severity group Interim Data cutoff Aug 19, 2022 All Treated Subjects (n=20) Drug/Laser Related Adverse Events 10% Subjects Grade I
Grade II Grade III Total Anterior Chamber Cell/ Inflammation 25% 0 0 25% Conjunctival hyperemia 15% 0 0 15% Eye Pain 5% 5% 0 10% Punctate Keratitis 10% 0 0 10% Interim SC Data Showed No Posterior Inflammation and No TR-SAEs Supportive of
Superior Tolerability Profile vs IVT
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 Growth Rate over 52 weeks Composite time to event analysis: Tumor progression or visual acuity failure between Intervention Group (High Dose) and Sham Group Primary Endpoint Key Secondary Endpoints Adaptive Assessment Adapt trial
Add Subjects Don't Adapt Adaptive Design Optimizes Probability of Success Patient population the same as the Ph2 SC study
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 baseline: 15 letters Tumor Height (TH): Ultrasound Largest Basal Diameter (LBD): fundus photography Bel-sar: 35% Tumor Progression Sham: 85% Tumor Progression Fundus photography Ultrasound Tumor Progression
Assumptions Change in Tumor Height (TH) over time: Ultrasound Bel-sar vs Sham : -0.28mm/year reduction Growth Rate Change in tumor height over time Note: Tumor Height (TH) is synonymous with Tumor Thickness; VA - Visual Acuity; Bel-sar -
Belzupacap Sarotalocan Composite Endpoint Conservative Assumptions Provide >90% Power to Maximize Probability of Success with Single Phase 3 Trial
Choroidal Metastasis
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 of Care is Radiotherapy Blindness, Eye Loss, and Disfiguration Breast 40-53% Lung 20-29% GI 4% Kidney 2% Prostate 2% Skin 2% Mathis et al. New concepts in choroidal metastasis, Progress in retinal and eye research (2019), Cohen,
Ocular metastasis, Eye (2014), Shields et al. Survey of 520 eyes with uveal metastases. Ophthalmology (1997), Namad et al. Bilateral choroidal metastasis from non-small lung cancer, Case reports in oncological medicine (2014).
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