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patients and preserve organ function
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and cost of our research and development programs and our current and future nonclinical, 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 efficiently develop our existing product candidates and discover new product candidates; 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; statements regarding our beliefs and expectations for the high unmet medical need for an effective local treatment in
ocular and urologic oncology to preserve organ function; 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 first half of 2027;
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
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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 Novel class
of drugs Positive clinical data in Large market opportunity Key upcoming virus-like drug conjugates multiple indications in areas of unmet need catalysts VDCs have the potential to Positive phase 2 data in early- Ocular oncology Multiple clinical
data 1-7 transform early cancer stage choroidal melanoma ~66,000 patients/yr (US/EU) readouts expected in 2025: treatment with phase 3 ongoing initial data in phase 1b/2 in under FDA SPA agreement Urologic oncology NMIBC and phase 2 in 8 Novel
MoA: direct tumor cell ~500,000 patients/yr (globally) metastases to the choroid killing and immune cell Multiple clinical complete activation responses with single low dose Current cash expected to fund in phase 1 trial in NMIBC operations into 1H
2027 1. Yu G-P et al. Am J Ophthalmol. 2003;135(6):800-6. 2. Triay E et al. Br J Ophthalmol. 2009;93(11):1524-8. 3. Newton R et al. Lancet. 1996;347(9013):1450-1. 4. Dalvin LA. Br J Ophthalmol. 2018;102(12):1728-1734. 5. Sun EC et al. Cancer
Epidemiol Biomarkers Prev. 1997;6(2):73-7. 6. Epidemiology analysis for choroidal melanoma and choroidal metastasis by ClearView Healthcare Partners and Putman. 7. American Cancer Society. Key statistics for retinoblastoma. Available at:
https://www.cancer.org/cancer/types/retinoblastoma/about/key-statistics.html. Accessed Sept 5, 2024. 8. Bladder cancer. Putnam & Assoc. Epidemiology Analysis. FDA, United States Food and Drug Administration; SPA, Special Protocol Assessment;
VDC, Virus-like drug conjugate, MoA, Mechanism of action; NMIBC, Non-muscle-invasive bladder cancer 3
Clinical pipeline across multiple solid tumor indications Program
Preclinical Phase 1 Phase 2 Phase 3 Planned milestones Ocular oncology Phase 3 enrollment complete as Primary uveal melanoma early as the end of 2025 Metastases to the choroid Initial phase 2 data in 2025 Ocular surface cancers Initiate phase 1
trial in 2025 Urologic oncology Non-muscle-invasive bladder cancer Initial phase 1b/2 data in 2025 (NMIBC) a Other mHSPG expressing tumors undisclosed 1 a.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 (HSPGs). 1. Kines RC, and Schiller JT. Viruses. 2022;14(8):1656. mHSPG, modified heparan sulphate proteoglycan; MIBC, muscle invasive bladder
cancer; NMIBC, non-muscle-invasive bladder cancer. 4
Virus-like drug conjugates have the potential to transform early cancer
treatment Unique tumor selectivity Tumor and mutation-agnostic Targets a key receptor molecule expressed in the >100 cell lines early stages of malignant tumor transformation >15 animal tumor models Dual MoA High potency Targeted cytotoxicity
and immune ~200 cytotoxic molecules per VLP; activation; potential to generate lasting demonstrated picomolar efficacy in anti-tumor T-cell memory multiple animal tumor models Favorable safety profile Positive clinical data in multiple early-stage
local cancers No treatment-related SAEs and no DLTs Choroidal melanoma: Positive phase 2 end of study data; phase 3 ongoing reported in phase 2 choroidal melanoma trial or phase 1 data readout in NMIBC trial NMIBC: Positive phase 1
data; phase 1b/2 ongoing DLT, dose-limiting toxicity; MoA, mechanism of action; NMIBC, non-muscle-invasive bladder cancer; SAE, serious adverse event; VLP, virus-like particle. 5 5
VLPs bind to macrophages, B cells, dendritic cells and neutrophils and
are capable of stimulating antigen-presenting cells through TLR-4 engagement and NFk- production Reactive oxygen species disrupts cell membrane and organelles AU-011 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 AU-011 treatment is designed to be cytopathic to resident Release of DAMPs induces suppressor cells, reducing the immune-suppressive
anti-tumor immunity microenvironment and contributing to anti-tumor immunity 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. DAMPs, damage-associated molecular patterns; HSPG, heparan sulfate proteoglycan; VDC, virus-like drug conjugate; VLP, virus-like particle. 6
Bel-sar's unique platform technology is potentially applicable
across multiple cancers Bladder cancer CNS cancers Choroidal melanoma NMIBC/MIBC GI cancers Ongoing phase 3 with SPA Based on positive phase 2 clinical data Positive phase 1 data in NMIBC Head and neck cancer Multiple CRs with single dose Breast
cancer Metastases to the choroid Phase 1b/2 ongoing Phase 2 initiated Lung cancer Potential to expand to MIBC Cutaneous melanoma Cancers of the ocular surface Other urologic cancers Pre-IND Next-gen combination strategies Retinoblastoma (pediatric)
Pre-clinical Other cancers Urologic oncology Ocular oncology - Rare oncology Bel-sar, belzupacap sarotalocan; CR, clinical complete response; CNS, central nervous system; GI, gastrointestinal; IND, Investigational New Drug application; MIBC,
muscle-invasive bladder cancer; NMIBC, non-muscle-invasive bladder cancer; SPA, Special Protocol Assessment. The effectiveness and safety of bel-sar have not been established or clinically evaluated in tumors outside the ocular or bladder setting,
and bel-sar is not approved for use in any jurisdiction. 7
Ocular Oncology Bel-sar target indications: Primary uveal melanoma |
Metastases to the choroid | Ocular surface cancers
~66,000 patients/year Bel-sar opportunities Ocular oncology franchise
total addressable market (US/EU) in ocular oncology represent a multi- a,1-5 6 ~35,000/yr ~11,000/yr billion-dollar Ocular surface cancers Primary uveal melanoma addressable market With only ~100 ocular oncologists in the US/EU, a
global launch may be accomplished with a small (<20) field-based team Metastases to the choroid Retinoblastoma 7 6 ~500/yr ~20,000/yr a 1-5 Includes conjunctival melanoma, primary acquired melanosis, squamous cell carcinoma and ocular surface
squamous neoplasia. 1. Yu G-P et al. Am J Ophthalmol. 2003;135(6):800-6. 2. Triay E et al. Br J Ophthalmol. 2009;93(11):1524-8. 3. Newton R et al. Lancet. 1996;347(9013):1450-1. 4. Dalvin LA. Br J Ophthalmol. 2018;102(12):1728-1734. 5. Sun EC et al.
Cancer Epidemiol Biomarkers Prev. 1997;6(2):73-7. 6. Epidemiology analysis for choroidal melanoma and choroidal metastasis by ClearView Healthcare Partners and Putman. 7. American Cancer Society. Key statistics for retinoblastoma. Available at:
https://www.cancer.org/cancer/types/retinoblastoma/about/key-statistics.html. Accessed Sept 5, 2024. Bel-sar (AU-011) is an investigational product candidate The effectiveness and safety of bel-sar have not been established, and bel-sar is not
approved for use in any jurisdiction. 9
Choroid is 90% 1 of the uvea Bel-sar is in phase 3 for primary uveal
Most common primary 2,3 melanoma, the most intraocular cancer in adults 3 ~11,000/yr common primary Choroidal melanoma intraocular cancer ~80% of patients diagnosed 3 with early-stage disease in adults Iris Primary uveal melanoma is a 50% of
patients develop high unmet medical need metastasis within 15 years Ciliary body With no currently approved 2 (metastatic uveal melanoma) vision-preserving therapies, Uvea: Choroid, ciliary body and iris the current standard-of-care is
radiotherapy - treatment 4,5 that leads to legal blindness Bel-sar has the potential to provide a treatment option that preserves vision 1. Heiting, G. Iris/uvea of the eye. Available at:
https://www.allaboutvision.com/en-gb/resources/uvea-iris-choroid/. Accessed Oct. 3, 2023. 2. Kaliki S and Shields CL. Eye (Lond). 2017;31(2):241-257. 3. Epidemiology analysis for choroidal melanoma and choroidal metastasis by ClearView Healthcare
Partners and Putman. 4. 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. 5.. 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.
1-3 Current treatment paradigm for primary uveal melanoma Small
Medium Large Metastatic SIZE 1 2.5 - 3 >10 (mm): Indeterminate lesions Small melanomas Prevalence of Risk Factors Growth Small CM choroidal nevi ranges from 4.6-7.9% in 2 Enuc. Caucasians Observation Systemic chemotherapy Radiotherapy
Radiotherapy (KIMMTRAK ) Local - Early Local - Late Metastatic (~8,000) (~2,300) (~2,000) a Each figure represents ~250 persons. 1. Shields CL et al. Choroidal and ciliary body melanoma. Available at:
https://eyewiki.aao.org/Choroidal_and_Ciliary_Body_Melanoma Accessed September 9, 2024. 2. Singh AD, et al. Ophthalmology. 2005;112(10):1784-89. 3. Epidemiology analysis for choroidal melanoma and choroidal metastasis by ClearView Healthcare
Partners and Putman. CM, choroidal melanoma; Enuc., enucleation. 11 Incidence: Patients a US/EU
High morbidity associated with current standard of care 3-6
Radiotherapy Up to 87% of primary uveal melanoma patients become legally blind 1,2 Adverse Event over time in the eye treated with radiotherapy Surgeries secondary to AEs 40%+ (e.g., cataracts) Proportion of patients legally blind (BCVA Radiation
retinopathy 40%+ 1, 2 20/200) after brachytherapy Neovascular glaucoma 10% 100% Dry eye syndrome 20% 80% Strabismus 2%+ Retinal detachment 1-2% 60% Vision loss ( 15 letters) ~70% Long-term legal blindness ~90% 40% ( 20/200)
Serious Adverse Event 20% Scleral necrosis 0-5% Enucleation/eye loss 10-15% 0% Severe vision loss ( 30 letters) ~90% in HRVL 1. Jarczak J et al. Medicina (Kaunas). 2023;59(6):1131. 2. Tsui I, et al. 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. Shields CL et al. Curr Opin Ophthalmol. 2019;30(3):206-214. 6. Kaliki S,
Shields CL. Eye. 2017;31(2):241-257. AE, adverse event; BCVA, best-corrected visual acuity; HRVL, high-risk for vision loss. 12
Treatment Goals Bel-sar has the potential to be the Reduce metastasis
Local tumor Vision No radiation- Improve safety risk with early related morbidity control preservation and quality of life first approved treatment vision-preserving In-office procedure therapy in primary uveal melanoma Delivery via Light activation
with suprachoroidal injection standard ophthalmic laser Suprachoroidal Two injections (2 min. each) 30 min. apart 10-30 min. procedure Bel-sar (AU-011) is an investigational product candidate. The effectiveness and safety of bel-sar have not been
established, and bel-sar is not approved for use in any jurisdiction. 13
Bel-sar for small choroidal melanoma or indeterminate lesions: Global
phase 3 CoMpass trial now enrolling Target enrollment ~100 participants globally Anticipated sites in North America, Europe, Middle East and Asia-Pacific Regions Primary endpoint 80 g bel-sar treatment arm Time to tumor progression (n=40)
Increase in tumor thickness 0.5 mm Participants or 1.5 mm in LBD 15-month with small 40 g bel-sar primary choroidal Randomize treatment arm efficacy melanoma or 2:1:2 First key secondary endpoint (n=20) analysis indeterminate
Time to composite endpoint: lesions Tumor progression or visual acuity failure Sham control arm Increase in tumor 15 decrease in (n=40) OR ETDRS-BCVA letter thickness 0.5 mm or 1.5 mm in LBD score from baseline Received fast
track and orphan drug designations An SPA agreement indicates concurrence by the FDA that the design of the trial can adequately support a regulatory submission BCVA, best-corrected visual acuity; ETDRS, Early Treatment Diabetic Retinopathy Study;
LBD, largest basal diameter; SPA, Special Protocol Assessment. ClinicalTrials.gov Identifier: NCT06007690; AU-011-301. 14
Product-Limit Survival Estimates With Number of Subjects at Risk 11 1
.0 ..0 0 + + C e Cn en ss oo re red d Time to tumor 00 0 .8 ..8 8 progression 00 0 .6 ..6 6 P = 0.0005 Change from baseline in thickness 0.5 mm; or in LBD 1.5 mm 0.4 0 0..4 4 confirmed by at least one Phase 2 end of study repeat
assessment 00 0 .2 ..2 2 data represented 00 0 .0 ..0 0 using planned phase 3 Therapeutic 0 0 1 10 00 0 2 20 00 0 3 30 00 0 4 40 00 0 5 50 00 0 0 100 200 300 400 500 n=10 Product-Limit Survival Estimates Treatment duration (days) endpoints t td du
ur rx x With Number of Subjects at Risk Subtherapeutic t tr rt tp p 3 3 C Cy yc cl le e 2 2 C Cy yc cl le e 11 1 .0 ..0 0 + Censored n=10 + Censored Kaplan-Meier analysis simulation 00 0 .8 ..8 8 10 10 9 9 8 0 3 Cycle 10 7 7 1 0 2 Cycle of
time-to-event Time to composite 00 0 .6 ..6 6 endpoint 00 0 .4 ..4 4 P = 0.0008 Time to tumor progression or vision acuity failure ( 15 letter loss 0.2 0 0..2 2 in ETDRS-BCVA), whichever occurs earlier 00 0 .0 ..0 0 0 0 1 10 00 0 2 20 00 0 3
30 00 0 4 40 00 0 5 50 00 0 0 100 200 300 400 500 Treatment duration (days) t td du ur rx x t tr rt tp p 3 3 C Cy yc cl le e 2 2 C Cy yc cl le e 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. ClinicalTrials.gov Identifiers: NCT04417530; AU-011-202 (phase 2); NCT06007690; AU-011-301 (phase 3). 10 10 9 8 7 0 3 Cycle Data on file, Aura Biosciences. 10 7 7 1 0 2 Cycle 15 Su S Su u rr r
vv v iv i iv v al a al l p P Prr rob o ob ba a ab b bi il lil i it tit y yy Survival probability S Su ur rv vi iv va al l P Pr ro ob ba ab bi il li it ty y
Bel-sar has a significant commercial opportunity in ocular oncology
Bel-sar's potential value drivers Highly favorable competitive landscape Ocular surface a,2-6 ~35,000/yr cancers Regulatory and manufacturing synergies Focused call point (~100 ocular Metastases to oncologists in US/EU) with potential 1
the choroid expansion to retina specialists ~20,000/yr Same centers Primary uveal 1 melanoma Small (<20) field-based team ~11,000/yr Buy-and-bill reimbursement ANTICIPATED EXPEDITED TIMELINE FOR SUBSEQUENT INDICATIONS Bel-sar has the potential to
transform the ocular oncology field as a vision-preserving therapy that alleviates patient burden and reduces risk of metastasis with early treatment a 2-6 Includes conjunctival melanoma, primary acquired melanosis, squamous cell carcinoma and
ocular surface squamous neoplasia. 1. Epidemiology analysis for choroidal melanoma and choroidal metastasis by ClearView Healthcare Partners and Putman. 2. Yu G-P et al. Am J Ophthalmol. 2003;135(6):800-6. 3. Triay E et al. Br J Ophthalmol.