Aura Biosciences, Inc. · NASDAQ:AURA
Aura Biosciences — Corporate Update (November 2024)
43 slides from Aura Biosciences's corporate update, Nov 12, 2024
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 November 2024 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
*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; MIB
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: 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 6 Positive clinical data in multiple early-stage local cancers Cho
Slide 7: 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 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, the most common primary intraocular cancer in adults
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 using planned phase 3 endpoints
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: Non-muscle-invasive bladder cancer | Muscle-invasive bladder cancer
Slide 20: 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 21: NMIBC treatment paradigm and prevalence
aEach figure represents 1000 persons. 1. Holzbeierlein JM et al. J Urol. 2024;212(1):3–10. 2. Holzbeierlein JM et al. J Urol. 2024 Apr;211(4):533-538. 3. Internal Aura epidemiology of market size; data on file. 4. Shalata AT, et al. Cancers (Basel). 2022;14(20):5019. 5. van Rhijn BWG, et al. Eur Uro
Slide 22: AU-011 as a potential front-line immune ablative therapy in NMIBC
AU-011 as a potential front-line immune ablative therapy in NMIBC AU-011 has a dual mechanism of action and can potentially reduce the treatment burden NMIBC, non–muscle-invasive bladder cancer; TURBT, transurethral resection of bladder tumor. Treatment goals Focal treatment with direct tumor cell k
Slide 23: AU-011 administration and activation may be optimized for the urology clinic
AU-011 administration and activation may be optimized for the urology clinic Local administration of AU-011 is aligned with current practice in urology offices TURBT, transurethral resection of bladder tumor. In-office procedure Local cystoscopic administration of drug with standard cystoscopy needl
Slide 24: Window of opportunity study design
Window of opportunity study: AU-011 administered between scheduled biopsy and standard TURBT TURBT, transurethral resection of bladder tumor. Clinicaltrials.gov identifier: NCT05483868; AU-011-102. Day 1 Cystoscopy + biopsy AU-011 injection Day 2 Cystoscopy Laser light activation Day 9 ± 1 (Cohort A
Slide 25: Phase 1 trial of AU-011 for bladder cancer
Phase 1 trial of AU-011 for bladder cancer designed to evaluate safety, feasibility, and mechanism of action Safety Review Board completed after each cohort. Patients followed for safety after TURBT to 56 days. NMIBC, non-muscle-invasive bladder cancer; MoA, mechanism of action; TURBT, transurethral
Slide 26: Cohorts A–C: Early efficacy data in Ta low-grade
Cohorts A–C: Single-dose drug with light activation 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-
Slide 27: Cohorts A + B: Early efficacy data in Ta high-grade
Cohorts A + B: Single-dose drug with light activation 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
Slide 28: Cohort A: Patient A3 case
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 29: Patient A3: focal distribution, necrosis, and positive immune staining
Patient A3: AU-011 focal distribution, necrosis, and positive immune staining (target lesion) Cohort A: Single-dose drug with light activation H&E CD3 CD4 CD8 Pre-treatment Post-treatment H&E, hematoxylin and eosin. Clinicaltrials.gov identifier: NCT05483868; AU-011-102. *Early data reported October
Slide 30: Light-activated cohorts: Evidence of immune-mediated mechanism of action
Light-activated cohorts (A + B): Strong evidence of immune-mediated mechanism of action aPatients for which biopsies were available. bOrganized aggregates of immune cells. MOA, mechanism of action Clinicaltrials.gov identifier: NCT05483868; AU-011-102. *Early data reported October 17, 2024. 100% (7/
Slide 31: Favorable safety profile with robust clinical and immunological response
AU-011 demonstrated a favorable safety profile with robust clinical and immunological response in early data readout of ‘all-comers’ NMIBC patients AE, adverse event; DLT, dose-limiting toxicity; DOR, duration of response; MOA, mechanism of action; NMIBC, non-muscle-invasive bladder cancer; SAE, ser
Slide 32: 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 33: Appendix Ocular Oncology
Appendix Ocular Oncology & aura
Slide 34: 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 35: 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 36: High local complete response rate at 12 months follow-up
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 37: 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 38: Visual acuity preserved in 90% of phase 3-eligible patients
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 39: 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 40: 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 41: Appendix Urologic Oncology
Appendix Urologic Oncology & aura
Slide 42: Patient population: AUA risk classification and grade at screening
AUA, American Urological Association. Clinicaltrials.gov identifier: NCT05483868; AU-011-102. *Early data reported October 17, 2024. Patient population: AUA risk classification and grade at screening Low-grade High-grade Low-risk Intermediate- risk High-risk Drug only: Patient 3 Drug only: Patient 4
Slide 43: Patient A3: Post-treatment generation of secondary lymphoid follicles
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