Ophthalmology Innovation SummitOctober 2019
Pipeline withLong Term Value
Sustainable Growth• Strong IP portfolio• Ocular oncology pipeline• Life cycle management
suprachoroidal delivery
First to Market
AttractiveCommercial Opportunity
• No competition• Potential to be first FDA
approved drug for primary Choroidal Melanoma
• Focused call point
Late Stage ClinicalAsset
Orphan & Fast Track• Well tolerated with clear
drug effect in Phase 1b/2 • FDA agreed on Phase 3
study design and endpoints for approval
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Choroidal MelanomaRare Disease
Focus
No Approved Drugs• 8,000 patients diagnosed
annually with Indeterminate Lesions and Choroidal Melanoma in US/ EU
• Opportunity to transform the treatment paradigm
Aura is Bringing Innovation to Ocular Oncology
Provides local tumor control
Often results in irreversible visual impairment due to radiation retinopathy and papillopathy
High comorbidities: cataracts, diplopia, keratitis and dry eye
Enucleation due to side effects (e.g. neovascular glaucoma)
Operating room procedure that requires two surgeries
There are No FDA Approved Therapies for Choroidal MelanomaRadiation is the Current Treatment Option
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Early Diagnosis but no Vision Preserving Therapy Available80% of patients are diagnosed early without clinically evident metastatic disease
Plaque Brachytherapy
Proton Beam Therapy
Good local tumor controlPreservation of visionNo anterior segment complicationsPossibility of earlier intervention and potential impact on reduction of metastasisConvenient office procedure
Unmet Medical Need for a Vision Preserving First Line Treatment Option
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Potential to be the First FDA Approved Therapy for the Primary Treatment of Choroidal Melanoma
Viral-Like Particles: A Novel Tumor-Targeted Approach
Tumor Targeted Platform:Technology discovered at National Cancer Institute (NIH) by Dr. J. T. Schiller1
Synthetic viral-like particles: recombinantly derivedTumor targeting: Leverage viral evolution to target heparan sulphate proteoglycans (HSPGs) modified by tumor cells2
Platform technology to treat multiple solid tumors
5 1. Lasker Award 2017.2. Human papillomavirus capsids preferentially bind and infect tumor cells; Kines et al; International Journal of Cancer ,138;901–911, February 2016.
AU-011: VLP bioconjugate (VPB)IRDye® 700DX molecules covalently conjugated to capsid protein without interfering with tumor targetingFirst-in-class light-activated therapyNovel MoA: Acute cellular necrosis with pro-immunogenic anti-tumor response
Value Drivers in Multiple Ophthalmology & Oncology Indications
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- Primary Choroidal Melanoma
AU-011
AU-011
Preclinical IND Ph I Ph IIIPh II
AU-011
AU-011
- Cancers of the Ocular Surface
- Suprachoroidal Delivery(Clearside micro-injector)
- Choroidal Metastases
Program Status
Granted Fast Track & Orphan Phase 1b/2 expansion ongoingPhase 3 initiation 2H/2020
Commercial Rights
Worldwide rights
IND amendment and clinical trial 1H/2020
Worldwide rights
Worldwide rights
Pre-clinical
Worldwide rights
Pre-clinical
- Primary Bladder Carcinoma (NMIBC) Worldwide rightsPre-clinicalAU-012
Single Dose Cohorts - Completed
Dose Expansion80μg x2 lasers QW3
1 cycle withPotential for Retreatment
Multiple Dose Cohorts - Completed
20 μg x 1 Laser
40 μg x 1 Laser
80 μg x 1 Laser
3 subjects per cohort (12 total)
1st Expansion Cohort - Completed
3 subjects per cohort (12 total)
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40 μg x 1 Laser (QW2)
40 μg x 1 Laser(QW3)
80 μg x 1 Laser(QW3)
80 μg x 2 Lasers(QW3)
2nd Expansion Cohorts - OngoingDose Expansion
2 Cycles
Phase 1b/2 Study Design in Choroidal Melanoma
80 μg x 2 Lasers
8 sites completed 1st Expansion; 6 more sites added for 2nd Expansion – 14 sites total
12 subjects
Up to 20 subjects – 7 subjects left to complete the trial
All enrolled subjects with clinical diagnosis of choroidal melanoma
Phase 1b/2 Study Results Continue to be Supportive of Phase 3 Registration Plan
• Multiple doses continue to be well tolerated with AEs managed with standard of care treatments (steroids and topical anti-hypertensives)
• Demonstrated vision preservation in majority of patients with long term follow up
• Statistically significant results in subjects with prior documented growth followed by arrest in growth after treatment
• Data will be presented by Jay S. Duker M.D., Amy C. Schefler M.D. and Abdhish R. Bhavsar M.D. at AAO and AAOOP meetings
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Phase 3 Trial Design Agreed with FDA
Eligibilityscreening
Trial Endpoints
• •
Primary Secondary• Composite endpoint at 12 months:
• Disease progression, or• Visual acuity failure
• Disease progression at 12 months • Change from baseline in tumor thickness at 12 months
RandomizationN=62(2:1:2)
Intervention GroupTherapeutic Dose
N=25
Intervention GroupLow Dose
N=12
Sham GroupN=25
Primary Efficacy
endpoint at 12 months
Cycle 1 Cycle 2
≥ 12 weeks
End of studySafety
24 months
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Market Opportunity and Commercialization
An estimated 4,000 patients in US and 3,900 patients in EU diagnosed with Indeterminate Lesions and Small Choroidal Melanomas
Received Orphan Drug Designation and Fast Track Status
BLA designation agreed with FDA
100% worldwide rights retained by Aura
Focused call point: Ocular Oncologists (50-US / 50-EU)
Clear regulatory path to approval and commercialization
Opportunity to Pioneer a First to Market Drug in Ocular OncologyNo competition
Source: Clearview commissioned market research
Clinical
Operational Strength
Pipeline
üDosed a total of 46 patients to date üAgreement with FDA on Phase 3 design and study endpointsüInitiated second expansion cohort with anticipated Phase 3 dose
and identical patient population
üClosed Series D to support late stage clinical developmentüSignificant organizational growth in key functional areas
üSigned License Agreement with Clearside Biomedical üInitiated non-clinical studies for suprachoroidal deliveryüPlan for IND Amendment and first clinical trial in 1H’20
Key Milestones in the Last 12 Months
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Title: Aura Biosciences Presenter: Jay S. Duker, MD, New England Eye Center, Tufts Medical CenterSession: SYM22 The Innovators SymposiumDate and time: Sunday, October 13, 2019 from 2:47 – 2:54 PM PTLocation: Moscone Center, Esplanade Room
Title: Virus-Like Particles for Uveal MelanomaPresenter: Amy C. Schefler, MD, Weill Cornell Medical College/Retina Consultants of HoustonSession: SYM50 Delivery of Therapeutics to the Posterior Ocular SegmentDate and time: Tuesday, October 15, 2019 from 9:35 – 9:45 AM PTLocation: Moscone Center, West 2002
October 11th
Abdhish R. Bhavsar M.D.
Event: American Association of Ophthalmic Oncologists and Pathologists (AAOOP) 2019 Annual MeetingTitle: Study Update of an Ongoing Phase 1b/2 Open-label Clinical Trial of AU-011 for the Treatment of Small to Medium Choroidal MelanomaPresenter: Abdhish R. Bhavsar, MD, The Retina CenterDate and time: Sunday, October 11, 2019 Location: Grand Hyatt San Francisco, 345 Stockton Street
Multiple Presentations at the Academy this Year
October 13th
Jay S. Duker M.D.
October 15th
Amy C. Schefler M.D.
Experienced Board & Strong Investor Syndicate
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Investor Syndicate
• Giovanni Mariggi, Ph.D.
• Casper Breum
• Mark Chin
• Joel Jean-Mairet, Ph.D.
• Arthur Pappas
• Raj Parekh, Ph.D.
Board of Directors
Series C/D - $70M (2018-2019)