Ophthalmology Innovation Summit October 2019 · Location:Moscone Center, West 2002 October 11th...

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Ophthalmology Innovation Summit October 2019

Transcript of Ophthalmology Innovation Summit October 2019 · Location:Moscone Center, West 2002 October 11th...

Page 1: Ophthalmology Innovation Summit October 2019 · Location:Moscone Center, West 2002 October 11th AbdhishR. Bhavsar M.D. Event: American Association of Ophthalmic Oncologists and Pathologists

Ophthalmology Innovation SummitOctober 2019

Page 2: Ophthalmology Innovation Summit October 2019 · Location:Moscone Center, West 2002 October 11th AbdhishR. Bhavsar M.D. Event: American Association of Ophthalmic Oncologists and Pathologists

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

Page 3: Ophthalmology Innovation Summit October 2019 · Location:Moscone Center, West 2002 October 11th AbdhishR. Bhavsar M.D. Event: American Association of Ophthalmic Oncologists and Pathologists

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

Page 4: Ophthalmology Innovation Summit October 2019 · Location:Moscone Center, West 2002 October 11th AbdhishR. Bhavsar M.D. Event: American Association of Ophthalmic Oncologists and Pathologists

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

Page 5: Ophthalmology Innovation Summit October 2019 · Location:Moscone Center, West 2002 October 11th AbdhishR. Bhavsar M.D. Event: American Association of Ophthalmic Oncologists and Pathologists

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

Page 6: Ophthalmology Innovation Summit October 2019 · Location:Moscone Center, West 2002 October 11th AbdhishR. Bhavsar M.D. Event: American Association of Ophthalmic Oncologists and Pathologists

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

Page 7: Ophthalmology Innovation Summit October 2019 · Location:Moscone Center, West 2002 October 11th AbdhishR. Bhavsar M.D. Event: American Association of Ophthalmic Oncologists and Pathologists

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

Page 8: Ophthalmology Innovation Summit October 2019 · Location:Moscone Center, West 2002 October 11th AbdhishR. Bhavsar M.D. Event: American Association of Ophthalmic Oncologists and Pathologists

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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Page 9: Ophthalmology Innovation Summit October 2019 · Location:Moscone Center, West 2002 October 11th AbdhishR. Bhavsar M.D. Event: American Association of Ophthalmic Oncologists and Pathologists

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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

Page 10: Ophthalmology Innovation Summit October 2019 · Location:Moscone Center, West 2002 October 11th AbdhishR. Bhavsar M.D. Event: American Association of Ophthalmic Oncologists and Pathologists

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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

Page 11: Ophthalmology Innovation Summit October 2019 · Location:Moscone Center, West 2002 October 11th AbdhishR. Bhavsar M.D. Event: American Association of Ophthalmic Oncologists and Pathologists

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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Page 12: Ophthalmology Innovation Summit October 2019 · Location:Moscone Center, West 2002 October 11th AbdhishR. Bhavsar M.D. Event: American Association of Ophthalmic Oncologists and Pathologists

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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.

Page 13: Ophthalmology Innovation Summit October 2019 · Location:Moscone Center, West 2002 October 11th AbdhishR. Bhavsar M.D. Event: American Association of Ophthalmic Oncologists and Pathologists

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)

Page 14: Ophthalmology Innovation Summit October 2019 · Location:Moscone Center, West 2002 October 11th AbdhishR. Bhavsar M.D. Event: American Association of Ophthalmic Oncologists and Pathologists