Aura Biosciences, Inc. · NASDAQ:AURA
Aura Biosciences — Investor Presentation (March 2025)
59 slides from Aura Biosciences's investor presentation, Mar 24, 2025
Slide 1: Innovating the future of cancer care to cure patients and preserve organ function
Innovating the future of cancer care to cure patients and preserve organ function March 2025 Exhibit 99.2
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: Well positioned with multiple near-term clinical catalysts
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 anal
Slide 4: Clinical pipeline across multiple solid tumor indications
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 1. Kines RC, and Schiller JT. Viruses. 2022;14(8):1656. mHSPG, modified heparan sulphate proteoglycan; MI
Slide 5: Virus-like drug conjugates have the potential to transform early cancer treatment
DLT, dose-limiting toxicity; MoA, mechanism of action; NMIBC, non-muscle-invasive bladder cancer; SAE, serious adverse event; VLP, virus-like particle. Virus-like drug conjugates have the potential to transform early cancer treatment 5 Positive clinical data in multiple early-stage local cancers Cho
Slide 6: AU-011 has a novel dual mechanism of action
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 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
Slide 7: Bel-sar's unique platform technology is potentially applicable across multiple cancers
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. Bel-sar’s unique platform
Slide 8: Ocular Oncology
Ocular Oncology Bel-sar target indications: Primary uveal melanoma | Metastases to the choroid | Ocular surface cancers
Slide 9: 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 With only ~100 ocular oncologists in the US/EU, a global launch may be accomplished with a small (<20) field-based team aIncludes conjunctival melanoma, primary acquired melanosis, squamous cell carcinoma an
Slide 10: Primary uveal melanoma is a high unmet medical need
Bel-sar is in phase 3 for primary uveal melanoma, the most common primary intraocular cancer in adults Primary uveal melanoma is a high unmet medical need With no approved vision-preserving therapies, the current standard-of-care is radiotherapy – treatment that leads to legal blindness4,5 1. Heitin
Slide 11: Current treatment paradigm for primary uveal melanoma
aEach 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 September 9, 2024. Singh AD, et al. Ophthalmology. 2005;112(10):1784–89. Epidemiology analysis for choroidal melanoma and ch
Slide 12: High morbidity associated with current standard of care
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
Slide 13: Bel-sar has the potential to be the first approved vision-preserving therapy
Bel-sar has the potential to be the first approved vision-preserving therapy in primary uveal melanoma No radiation- related morbidity Vision preservation Local tumor control Reduce metastasis risk with early treatment Improve safety and quality of life Treatment Goals In-office procedure Two inject
Slide 14: Global phase 3 CoMpass trial now enrolling
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, Spec
Slide 15: Phase 2 end of study data
Phase 2 end of study data represented using planned phase 3 endpoints Kaplan-Meier analysis simulation of time-to-event 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 assign
Slide 16: 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 With only ~100 ocular oncologists in the US/EU, a global launch may be accomplished with a small (<20) field-based team aIncludes conjunctival melanoma, primary acquired melanosis, squamous cell carcinoma an
Slide 17: Metastases to the choroid is a high unmet medical need
Metastases to the choroid is a high unmet medical need and potentially doubles the ocular oncology market opportunity Metastases to the choroid decrease vision and quality of life in patients fighting metastatic cancer 1. Mathis T et al. Prog Ret Eye Res. 2019;68:144-176. 2. Shields CL et al. Ophtha
Slide 18: Metastases to the choroid: first sites activated
Highlights: Primary endpoint at one-month post-treatment; possibility to see tumor shrinkage and vision improvement *3+3 Design. Each cohort to have a minimum of 3 and a maximum of 6 patients **Simplified schema of study design. Metastases to the choroid: first sites activated Study Population Study
Slide 19: Urologic Oncology
Urologic Oncology Bel-sar target indications: Intermediate-risk NMIBC | High-risk NMIBC
Slide 20: Bel-sar seeks to redefine the treatment of bladder cancer
20 Bel-sar seeks to redefine the treatment of bladder cancer with a novel and disruptive approach
Slide 21: Bladder cancer: high unmet medical need for organ-sparing therapies
1. GLOBOCAN 2022. Bladder. Available at: https://gco.iarc.who.int/media/globocan/factsheets/cancers/30-bladder-fact-sheet.pdf. [Accessed October 1, 2024]. 2. Sung H, et al. CA Cancer J Clin. 2021;71(3):209–49. 3. Burger M, et al. Eur Urol. 2013;63(2):234–41. 4. Flaig TW, et al. J Natl Compr Canc Net
Slide 22: Bel-sar has an innovative dual MoA
Bel-sar has an innovative dual MoA Long-term anti-tumor immune memory has the potential to provide immune surveillance, urothelial field effect, and prevent recurrence Robust targeted cytotoxicity designed to rapidly destroy cancer cells 1 2 Focal administration treats the tumor, not the entire urot
Slide 23: Current treatment paradigm for NMIBC
~80,000 ~20,000 Low grade – low & intermediate risk High-risk papillary disease High-risk CIS – BCG unresponsive BCG Intravesical chemotherapy ~4,000 TURBT recurrence Intravesical gene therapy Systemic immunotherapy TURBT recurrence Adjuvant therapy Adjuvant therapy Intravesical immunotherapy BCG In
Slide 24: Bel-sar shifts the treatment paradigm to immune-ablative front-line treatment
A, adjuvant treatment; APC, antigen-presenting cell; B, bel-sar; bel-sar, belzupacap sarotalocan; DAMPs, damage-associated molecular patterns; M, month; MOA, mechanism of action; NIR, near-infrared; SoC, standard-of-care; T, TURBT; TURBT, transurethral resection of bladder tumor. Bel-sar shifts the
Slide 25: Current treatment paradigm for NMIBC
~80,000 ~20,000 Low grade – low & intermediate risk High-risk papillary disease High-risk CIS – BCG unresponsive BCG Intravesical chemotherapy ~4,000 TURBT recurrence Intravesical gene therapy Systemic immunotherapy TURBT recurrence Adjuvant therapy Adjuvant therapy Intravesical immunotherapy BCG In
Slide 26: Standalone immune-ablative treatment or neoadjuvant to TURBT
Bel-sar has potential as a standalone immune-ablative treatment or as a neoadjuvant to TURBT Immune-ablative approach could eliminate the need for TURBT, or be used prior to resection to improve treatment outcomes APC, antigen-presenting cell; bel-sar, belzupacap sarotalocan; DAMPs, damage-associate
Slide 27: Current treatment paradigm for NMIBC
~80,000 ~20,000 Low grade – low & intermediate risk High-risk papillary disease High-risk CIS – BCG unresponsive BCG Intravesical chemotherapy ~4,000 TURBT recurrence Intravesical gene therapy Systemic immunotherapy TURBT recurrence Adjuvant therapy Adjuvant therapy Intravesical immunotherapy BCG In
Slide 28: Phase 1 data: phase 1 trial of bel-sar in bladder cancer
Phase 1 data Phase 1 trial of bel-sar in bladder cancer
Slide 29: Phase 1 bladder cancer trial treatment schema
Bel-sar, belzupacap sarotalocan; TURBT, transurethral resection of bladder tumor. Clinicaltrials.gov identifier: NCT05483868; AU-011-102. Bel-sar is administered before scheduled biopsy and standard of care (SoC) TURBT Day 1 Cystoscopy + biopsy bel-sar injection Day 2 Cystoscopy Laser light activati
Slide 30: Phase 1 trial designed to evaluate safety, feasibility, and mechanism of action
Bel-sar, belzupacap sarotalocan; NMIBC, non-muscle-invasive bladder cancer; TURBT, transurethral resection of bladder tumor. Clinicaltrials.gov identifier: NCT05483868; AU-011-102. Phase 1 trial of bel-sar for bladder cancer designed to evaluate safety, feasibility, and mechanism of action Safety Re
Slide 31: Cohort A-C safety data
Cohort A-C: Single-dose drug with light activation Safety data No serious adverse events No dose limiting toxicities ACompiled safety data includes all completed light-activated cohorts (A, B, and C), including one patient treated but not efficacy evaluable. Data cutoff date of March 3, 2025. TEAE,
Slide 32: Efficacy data: high-risk and intermediate-risk NMIBC
aFor purposes of this analysis, Clinical complete response defined as absence of tumor cells on histopathologic evaluation. bBladder urothelial field effect: absence of tumor cells in non-target lesions. cPreviously treated tumor demonstrated high-grade disease but pathology at time of treatment rev
Slide 33: Efficacy data: Ta high-risk NMIBC cohorts A-C
aClinical complete response defined as absence of tumor cells on histopathologic evaluation. bBladder urothelial field effect: absence of tumor cells in non-target lesions. cImmune response is defined by immunocyte infiltration on post-treatment histopathology. dTwo tumors in target tumor field with
Slide 34: Cohort A: Patient A3 clinical complete response
Cohort A: Single-dose drug with light activation Patient A3 72-year-old Hispanic male Screening diagnosis: (2024) Multiple Ta low-grade (<3 cm) No CIS Screening AUA risk classification: Intermediate Initial diagnosis: (2019) Ta high-grade <3 cm No CIS Intermediate risk Prior TURBT: 2019, 2020 (x2),
Slide 35: Patient A3: secondary lymphoid follicles and T cell infiltration
Cohort A: Single-dose drug with light activation Patient A3: Post-treatment generation of secondary lymphoid follicles and increase in CD3, CD4, and CD8 infiltration H&E: Pre-treatment H&E: Post-treatment CD4: Pre-treatment CD4: Post-treatment H&E, hematoxylin and eosin. Clinicaltrials.gov identifie
Slide 36: Multiplex immunofluorescence: mature tertiary lymphoid structures in treated lesion
Multiplex Immunofluorescence: Patient A3 (Intermediate-Risk NMIBC) TLS Not Present in Lesion Prior to Treatment Mature Tertiary Lymphoid Structures (TLS) in Target (Treated) Lesion: Active Immunosurveillance After Bel-sar Treatment CD3: T cells CD20: B cells CD23: Follicular Dendritic Cells (FDC) (F
Slide 37: Early tertiary lymphoid structures in non-target lesion
Early Tertiary Lymphoid Structures (TLS) in Distant Non-Target (Non-Treated) Lesion: Urothelial Immune Field Effect After Bel-sar Treatment Multiplex Immunofluorescence: Patient A3 (Intermediate-Risk NMIBC) CD3: T cells CD20: B cells CD23: Follicular Dendritic Cells (FDC) (Found in B cell follicles,
Slide 38: Clinical complete responses and immune responses after single low dose
Light-activated cohorts (A–C) after single, low-dose of bel-sar Clinical complete responses and immune responses demonstrated induction of effector immunity, relief from immunosuppression, and development of active immunosurveillance aTotal of 7 patients with non-target tumors. bOrganized aggregates
Slide 39: Single systemic treatment resulted in long-term tumor-free survival in murine model
A single systemic treatment of bel-sar resulted in long-term tumor-free survival and induction of anti-tumor responses in TC-1 murine tumor model Long-term tumor-free survival and protection from tumor re-challenge CD4+ and CD8+ T-cells are required both at the time of treatment and at the time of r
Slide 40: CD4+ and CD8+ T-cells key to durability of response
Kines RC, et al. Cancer Immunol Res. 2021;9(6):693–706. Bel-sar, belzupacap sarotalocan; NIR, near-infrared. CD4+ and CD8+ T-cells are key to the long-term durability of response and protection of rechallenge with bel-sar +1 9 11 Day: 0 100 Implant TC-1 tumor cells Tumor volume: 50mm3 7 10 13 20 Day
Slide 41: Phase 1b/2 trial of bel-sar in non-muscle invasive bladder cancer
Phase 1b/2 trial of bel-sar in non-muscle invasive bladder cancer
Slide 42: Paradigm-shifting treatment approach
Paradigm-shifting treatment approach Treat the tumor first to generate cell-mediated immunity (CMI) BCG, Bacillus Calmette-Guérin; bel-sar, belzupacap sarotalocan; CMI, cell-mediated immunity; MIBC, muscle-invasive bladder cancer; NMIBC, non-muscle-invasive bladder cancer; TURBT, transurethral resec
Slide 43: Phase 1b/2 treatment schedule
D, day; FU, follow-up; inj, injection; las, laser; M, month; W, week; NMIBC, non-muscle-invasive bladder cancer, . Clinicaltrials.gov identifier: NCT05483868; AU-011-102. Each cycle = 2 weeks Phase 1b/2 treatment schedule in intermediate and high-risk NMIBC patients Immune ablative design: Intermedi
Slide 44: Phase 1b/2 study design
IT, intratumoral; LG IR, low-grade intermediate risk; TURBT, transurethral resection of bladder tumor. Clinicaltrials.gov identifier: NCT05483868; AU-011-102. Phase 1b/2 study design Key updates Part 1: Dose escalation 21–26 participants Recurrent LG IR (4d + 4e + 4g) High risk (4f) Up to 3 tumors p
Slide 45: Bel-sar seeks to redefine the treatment of bladder cancer
BCG, Bacillus Calmette-Guérin; Bel-sar, belzupacap sarotalocan; CIS, carcinoma in situ; MIBC, muscle-invasive bladder cancer; MIBC, muscle-invasive bladder cancer; NMIBC, non-muscle-invasive bladder cancer; TURBT, transurethral resection of bladder tumor. 45 Targeted cytotoxicity + cell mediated ant
Slide 46: Company highlights
1. ClearView & Putnam & Assoc. Epidemiology Analysis Choroidal Melanoma and Choroidal Metastasis. FDA, United States Food and Drug Administration. NMIBC, non-muscle-invasive bladder cancer. Bel-sar (AU-011) is an investigational product candidate. The effectiveness and safety of bel-sar have not bee
Slide 47: Appendix Ocular Oncology
Appendix Ocular Oncology & aura
Slide 48: Phase 2 trial of bel-sar for choroidal melanoma
Goal: To determine safety, optimal dose and therapeutic regimen with suprachoroidal administration One cycle = Doses on days 1, 8, and 15. a12 patients enrolled, 1 patient who discontinued after 1 cycle due to unrelated SAEs is not included in data analysis (n=11). bCohort 2: 2 participants were pla
Slide 49: Baseline characteristics
Baseline characteristics All study participants aHigh risk for vision loss defined as tumor edge within either 3 mm of foveal center or 3 mm of optic disc edge. BCVA, best-corrected visual acuity; ETDRS, Early Treatment Diabetic Retinopathy Study; LBD, largest basal diameter. Data on file, Aura Bios
Slide 50: High local complete response rate at 12 months
High local complete response rate at 12 months follow-up 80% tumor control ratea at 12 months among the 10 phase 3-eligible patients in the 3-cycle cohorts aLocal complete response, or CR, in early-stage choroidal melanoma is described as tumor control and complete arrest of tumor growth by ocular o
Slide 51: Rate of tumor growth with bel-sar treatment
Rate of tumor growth ± SE, mm/yr P < 0.0001 Rate of tumor growth with bel-sar treatment In phase 3-eligible patients, the 3-cycle regimen resulted in cessation of growth among responders (N=8) Tumor thickness growth rates/slopes estimated using Mixed Models for Repeat Measures (MMRM); random interce
Slide 52: Visual acuity preservation
Vision loss threshold (−15 letters) Populations Patients (n) Vision failuresb (n) Vision preservation rate (%) All dose cohorts All treated patients 22 1 95% Subtherapeutic ≤2 cycles 10 0 100% Therapeutic 3 cycles and phase 3-eligiblea 10 1 90% BCVA change from baseline (ETDRS letter score) Median c
Slide 53: Bel-sar treatment had a favorable safety profile
Bel-sar treatment had a favorable safety profile No posterior inflammation No treatment-related SAEs No grade 3–5 treatment-related AEs * Table presents participants with AEs related to bel-sar or laser by severity and overall; participants with >1 AE are counted in the highest severity group AE, ad
Slide 54: Phase 2 data support phase 3 assumptions
ClinicalTrials.gov Identifier: NCT06007690; AU-011-301. Phase 2 data support phase 3 assumptions Phase 3 trial design P < 0.005 93% power (Δ20) Actual data (Δ60) >99% power P < 0.05 Robustness analysis of tumor control rates Overall rate in phase 2 2x “worse” than phase 2 2x “worse” than phase 2 Act
Slide 55: Appendix Urologic Oncology
Appendix Urologic Oncology & aura
Slide 56: Patient population: AUA risk classification and grade at screening
aBCG failure in patient C3 qualifying as high risk by AUA criteria. AUA, American Urological Association. Clinicaltrials.gov identifier: NCT05483868; AU-011-102. Patient population: AUA risk classification and grade at screening Low-grade High-grade Low-risk Intermediate- risk High-risk Drug only: P
Slide 57: Phase 1 trial designed to evaluate safety, feasibility, and mechanism of action
Bel-sar, belzupacap sarotalocan; NMIBC, non-muscle-invasive bladder cancer; TURBT, transurethral resection of bladder tumor. Clinicaltrials.gov identifier: NCT05483868; AU-011-102. Phase 1 trial of bel-sar for bladder cancer designed to evaluate safety, feasibility, and mechanism of action Safety Re
Slide 58: Drug only: treatment schedule
Drug only: Treatment schedule Total dose: 100 µg 50 µg into base of tumor 50 µg into lamina propria Bel-sar, belzupacap sarotalocan; H&E, hematoxylin and eosin; TURBT, transurethral resection of bladder tumor. Clinicaltrials.gov identifier: NCT05483868; bel-sar-102. Drug only without light activatio
Slide 59: Drug only (no light activation): safety cohort
Drug only (no light activation): First in human in bladder – safety cohort as required by FDA No signs of clinical activity in the absence of light activation as anticipated FDA, Food and Drug Administration. ClinicalTrials.gov Identifier: NCT06007690; bel-sar-301. Drug only without light activation
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