Corporate Presentation
November 2015
This presentation contains forward-looking statements. All statements other than statements of historical facts contained in this
presentation, including statements regarding our short-term and long-term strategies, efforts to develop and commercialize our
products, future operations, future financial position, future revenue, projected costs, prospects, plans, objectives of management
and expected market growth are forward-looking statements. These statements involve known and unknown risks, uncertainties
and other important factors that may cause our actual results, performance or achievements to be materially different from any
future results, performance or achievements expressed or implied by the forward-looking statements. The words “anticipate,”
“believe,” “could,” “estimate,” “expect,” “intend,” “may,” “plan,” “potential,” “predict,” “project,” “should,” “target,” “will,” “would” and
similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these
identifying words. We expressly disclaim any obligation to release any updates or revisions to any forward-looking statements to
reflect any change in our expectations or projections or any changes in events, conditions or circumstances on which any such
statements are based for any reason, except as required by law, even as new information becomes available. All forward-looking
statements in this presentation are qualified in their entirety by this cautionary statement.
Certain information contained in this presentation and statements made orally during this presentation relates to or is based on
information from industry publications and research, surveys and studies conducted by third parties. Industry publications and third
party research, survey and studies generally indicate that their information has been obtained from sources believed to be reliable,
although they do not guarantee the accuracy or completeness of such information. While we believe these industry publications
and third party research, surveys and studies are reliable, we have not independently verified such data.
This presentation shall not constitute an offer to sell or the solicitation of an offer to buy any securities, nor shall there be
any sale of any securities in any state or 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 jurisdiction.
3
Disclaimers and Forward-Looking Statements
Overview
Product focused & novel technology: Tumor Targeted Viral-Like Particles
Lead product for first-line treatment of Ocular melanoma
FDA Orphan Drug Designation & strong pre-clinical validation
Rapid clinical data output in Q1-2/16
Commercial launch preparation in 2017/18 for potential Fast Track Approval
Focused call point enables direct commercialization
Blockbuster market potential
Experienced Management Team, Board of Directors, and Advisors
4
Management Team, Board Members & Advisors
Board of Directors
Alan Walts, Ph.D.
Former Managing Director, Genzyme Ventures
Raj Parekh, Ph.D.
General Partner, Advent Life Sciences
Dale Pfost, Ph.D.
General Partner, Advent Life Sciences
Art Pappas
CEO, A.M. Pappas & Associates
Joel Jean-Mairet, Ph.D.
Managing Partner, Ysios Capital
Peter Wirth
Former Corporate Secretary, Genzyme
José Baselga, M.D., Ph.D.
Physician-in-Chief, MSKCC-NYC
Javier Garcia
Former VP Business Development, Eli Lilly
Management Team
Elisabet de los Pinos, Ph.D.
Founding Chief Executive Officer
Brand Manager, Eli Lilly Oncology
Post-Doctoral Fellow, London Institute of
Cancer Research
Alison Lawton
Chief Operating Officer
COO, OvaScience
General Manager, Biosurgery Business
Sanofi/Genzyme
SVP Global Market Access, Genzyme
Michael Wyzga
Chief Financial Officer
CEO, Radius Health
CFO, Genzyme
Henri Termeer
Corporate Advisor
Former Chairman, President & CEO, Genzyme
John T. Schiller, Ph.D.
Scientific Founder
Head of Neoplastic Disease Section, National
Cancer Institute (NIH)
Clinical Advisory Board:
Carol Shields, M.D.
Director of the Oncology Service at
Wills Eye Hospital, Philadelphia
Arun D. Singh, M.D.
Director of the Department of Ophthalmic
Oncology, Cole Eye Institute, Cleveland Clinic
Evangelos Gragoudas, M.D.
Director of Retina Service, Massachusetts
Eye and Ear Infirmary
Advisors
5
Novel Technology: Tumor Targeted Viral-Like Particles
Aura is introducing a breakthrough approach to targeted
therapy based on a novel and proprietary technology
Unique targeting of cancer cells with viral-like particle
Through interactions with Heparan Sulphate Proteoglycans (HSPGs) on cell
surface1
Technology discovered by Dr. John T. Schiller at National Cancer Institute2
Enables the uptake and distribution of up to 1,000 surface-coupled
“therapeutic payloads” per particle, and encapsulated delivery of nucleic
acids and other molecules3
6
1 Human papillomavirus capsids preferentially bind and infect tumor cells; Kines et al; International Journal of Cancer , Accepted August 2015
2 Recipient of the National Medal of Technology & Innovation in 2014 – the highest honor of technological achievement, given by the President of
the United States; Head of Neoplastic Disease Section of NCI; advisor to WHO; AACR Landon Award
3 Data on file submitted for publication
Lead Indication: Primary Ocular Melanoma
Primary tumor confined to eye cavity and diagnosed by Ophthalmologists
Orphan indication with no FDA approved therapies
Ocular Oncologists: highly focused call point
Unmet medical need in all stages of disease:
Primary:
Small melanomas/melanocytic lesions: watch & wait for growth
Medium melanoma: radiation (plaque brachytherapy)
Large melanoma: enucleation
Metastatic disease: Selumetinib recently failed in Phase 3 – no good treatment options
Initial addressable market: 10,000 patients worldwide1
Expansion to earlier stage disease
7
Target
Indication
1 Based on epidemiology estimates by Health Advances (US) and Company (Europe and rest of world)
Ocular Melanoma: Current Treatment Options
Ocular Melanoma is treated with highly invasive radioactive therapies
that require multiple surgeries and cause blindness without
prevention of metastases1
1 Demonstrated by the Collaborative Ocular Melanoma Study Group (COMS) study. The COMS randomized trial of Iodine 125 brachytherapy
for choroidal melanoma, III: Initial mortality findings. COMS Report No. 18. Arch Ophthalmol 119: 969-982, 2001.
8
AU-011: Mechanism of Action
9
Non-apoptotic Cell Death:
Excitation of the drug in
proximity to cell membrane
results in disruption of the
tumor membrane and
cellular damage leading to
rapid necrosis
AU-011: Administered via
intra-vitreal (ITV) injection,
viral particle targets tumor
cells, delivers drug
(IR700DX) which is later
photo-activated with laser
2 3Laser: Irradiation with
Infrared light for <2min1
9
IR700DX
AU-011: Preclinical Data Summary in Ocular
Melanoma Rabbit Model
AU-011 evaluated in multiple independent experiments in an orthotopic
rabbit model of Uveal Melanoma1
Data on 68 rabbits in 4 independent studies showed2:
Mechanism of Action: Tumor growth arrest and necrosis after ITV treatment with
AU011+ laser 50J/cm2
Efficacy: All animals with small/medium tumors (<5mm) that received 3 injections
of AU-011/laser showed no remaining tumor cells or complete necrosis
Dose response: confirming the pharmacological activity of the drug
AU-011 has been shown to penetrate throughout the entire tumor tissue
10
1. Grossniklaus et al. Investigative Ophthalmology & Visual Science June 2013, Vol.54, 4229
2. Data submitted for publication in collaboration with researchers at McGill University and at Mass Eye and Ear Infirmary-
Harvard University
AU-011: Clinical Trial Strategy
11
AU-011 will potentially be the first approved FDA treatment for first-line
therapy of Ocular Melanoma (treatment of the primary tumor in the eye)
Rapid clinical proof of concept: safety and efficacy in Q1-2/16
Accelerated path to approval:
Two clinical trials for registration (Phase I/II dose ranging & Pivotal)
Clinical endpoints – Vision and Tumor response (does not require overall
survival benefit)
Post approval rare disease registry for long-term follow-up
Clinical plan has been reviewed with the FDA in two Pre-IND meetings
Pipeline
Product Indication Development IND Phase I/II Phase III
Ocular Oncology
AU-011
Uveal Melanoma
2nd Indication
(e.g. Retinoblastoma)
Solid Tumors
AU-021
Bladder/
Head & Neck Cancer
Discovery Platform
Immuno-Oncology
12
Summary
Product focused & novel technology: Tumor Targeted Viral-Like Particles
Lead product for life threatening rare disease: Ocular melanoma
FDA Orphan Drug Designation & strong pre-clinical validation
Rapid clinical data output in Q1-2/16
Commercial launch preparation in 2017/18 for potential Fast Track Approval
Focused call point enables direct commercialization
Blockbuster market potential
Experienced Management Team, Board of Directors, and Advisors
13
Elisabet de los Pinos
Founder & CEO
eli@aurabiosciences.com

Aura

  • 2.
  • 3.
    This presentation containsforward-looking statements. All statements other than statements of historical facts contained in this presentation, including statements regarding our short-term and long-term strategies, efforts to develop and commercialize our products, future operations, future financial position, future revenue, projected costs, prospects, plans, objectives of management and expected market growth are forward-looking statements. These statements involve known and unknown risks, uncertainties and other important factors that may cause our actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements. The words “anticipate,” “believe,” “could,” “estimate,” “expect,” “intend,” “may,” “plan,” “potential,” “predict,” “project,” “should,” “target,” “will,” “would” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. We expressly disclaim any obligation to release any updates or revisions to any forward-looking statements to reflect any change in our expectations or projections or any changes in events, conditions or circumstances on which any such statements are based for any reason, except as required by law, even as new information becomes available. All forward-looking statements in this presentation are qualified in their entirety by this cautionary statement. Certain information contained in this presentation and statements made orally during this presentation relates to or is based on information from industry publications and research, surveys and studies conducted by third parties. Industry publications and third party research, survey and studies generally indicate that their information has been obtained from sources believed to be reliable, although they do not guarantee the accuracy or completeness of such information. While we believe these industry publications and third party research, surveys and studies are reliable, we have not independently verified such data. This presentation shall not constitute an offer to sell or the solicitation of an offer to buy any securities, nor shall there be any sale of any securities in any state or 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 jurisdiction. 3 Disclaimers and Forward-Looking Statements
  • 4.
    Overview Product focused &novel technology: Tumor Targeted Viral-Like Particles Lead product for first-line treatment of Ocular melanoma FDA Orphan Drug Designation & strong pre-clinical validation Rapid clinical data output in Q1-2/16 Commercial launch preparation in 2017/18 for potential Fast Track Approval Focused call point enables direct commercialization Blockbuster market potential Experienced Management Team, Board of Directors, and Advisors 4
  • 5.
    Management Team, BoardMembers & Advisors Board of Directors Alan Walts, Ph.D. Former Managing Director, Genzyme Ventures Raj Parekh, Ph.D. General Partner, Advent Life Sciences Dale Pfost, Ph.D. General Partner, Advent Life Sciences Art Pappas CEO, A.M. Pappas & Associates Joel Jean-Mairet, Ph.D. Managing Partner, Ysios Capital Peter Wirth Former Corporate Secretary, Genzyme José Baselga, M.D., Ph.D. Physician-in-Chief, MSKCC-NYC Javier Garcia Former VP Business Development, Eli Lilly Management Team Elisabet de los Pinos, Ph.D. Founding Chief Executive Officer Brand Manager, Eli Lilly Oncology Post-Doctoral Fellow, London Institute of Cancer Research Alison Lawton Chief Operating Officer COO, OvaScience General Manager, Biosurgery Business Sanofi/Genzyme SVP Global Market Access, Genzyme Michael Wyzga Chief Financial Officer CEO, Radius Health CFO, Genzyme Henri Termeer Corporate Advisor Former Chairman, President & CEO, Genzyme John T. Schiller, Ph.D. Scientific Founder Head of Neoplastic Disease Section, National Cancer Institute (NIH) Clinical Advisory Board: Carol Shields, M.D. Director of the Oncology Service at Wills Eye Hospital, Philadelphia Arun D. Singh, M.D. Director of the Department of Ophthalmic Oncology, Cole Eye Institute, Cleveland Clinic Evangelos Gragoudas, M.D. Director of Retina Service, Massachusetts Eye and Ear Infirmary Advisors 5
  • 6.
    Novel Technology: TumorTargeted Viral-Like Particles Aura is introducing a breakthrough approach to targeted therapy based on a novel and proprietary technology Unique targeting of cancer cells with viral-like particle Through interactions with Heparan Sulphate Proteoglycans (HSPGs) on cell surface1 Technology discovered by Dr. John T. Schiller at National Cancer Institute2 Enables the uptake and distribution of up to 1,000 surface-coupled “therapeutic payloads” per particle, and encapsulated delivery of nucleic acids and other molecules3 6 1 Human papillomavirus capsids preferentially bind and infect tumor cells; Kines et al; International Journal of Cancer , Accepted August 2015 2 Recipient of the National Medal of Technology & Innovation in 2014 – the highest honor of technological achievement, given by the President of the United States; Head of Neoplastic Disease Section of NCI; advisor to WHO; AACR Landon Award 3 Data on file submitted for publication
  • 7.
    Lead Indication: PrimaryOcular Melanoma Primary tumor confined to eye cavity and diagnosed by Ophthalmologists Orphan indication with no FDA approved therapies Ocular Oncologists: highly focused call point Unmet medical need in all stages of disease: Primary: Small melanomas/melanocytic lesions: watch & wait for growth Medium melanoma: radiation (plaque brachytherapy) Large melanoma: enucleation Metastatic disease: Selumetinib recently failed in Phase 3 – no good treatment options Initial addressable market: 10,000 patients worldwide1 Expansion to earlier stage disease 7 Target Indication 1 Based on epidemiology estimates by Health Advances (US) and Company (Europe and rest of world)
  • 8.
    Ocular Melanoma: CurrentTreatment Options Ocular Melanoma is treated with highly invasive radioactive therapies that require multiple surgeries and cause blindness without prevention of metastases1 1 Demonstrated by the Collaborative Ocular Melanoma Study Group (COMS) study. The COMS randomized trial of Iodine 125 brachytherapy for choroidal melanoma, III: Initial mortality findings. COMS Report No. 18. Arch Ophthalmol 119: 969-982, 2001. 8
  • 9.
    AU-011: Mechanism ofAction 9 Non-apoptotic Cell Death: Excitation of the drug in proximity to cell membrane results in disruption of the tumor membrane and cellular damage leading to rapid necrosis AU-011: Administered via intra-vitreal (ITV) injection, viral particle targets tumor cells, delivers drug (IR700DX) which is later photo-activated with laser 2 3Laser: Irradiation with Infrared light for <2min1 9 IR700DX
  • 10.
    AU-011: Preclinical DataSummary in Ocular Melanoma Rabbit Model AU-011 evaluated in multiple independent experiments in an orthotopic rabbit model of Uveal Melanoma1 Data on 68 rabbits in 4 independent studies showed2: Mechanism of Action: Tumor growth arrest and necrosis after ITV treatment with AU011+ laser 50J/cm2 Efficacy: All animals with small/medium tumors (<5mm) that received 3 injections of AU-011/laser showed no remaining tumor cells or complete necrosis Dose response: confirming the pharmacological activity of the drug AU-011 has been shown to penetrate throughout the entire tumor tissue 10 1. Grossniklaus et al. Investigative Ophthalmology & Visual Science June 2013, Vol.54, 4229 2. Data submitted for publication in collaboration with researchers at McGill University and at Mass Eye and Ear Infirmary- Harvard University
  • 11.
    AU-011: Clinical TrialStrategy 11 AU-011 will potentially be the first approved FDA treatment for first-line therapy of Ocular Melanoma (treatment of the primary tumor in the eye) Rapid clinical proof of concept: safety and efficacy in Q1-2/16 Accelerated path to approval: Two clinical trials for registration (Phase I/II dose ranging & Pivotal) Clinical endpoints – Vision and Tumor response (does not require overall survival benefit) Post approval rare disease registry for long-term follow-up Clinical plan has been reviewed with the FDA in two Pre-IND meetings
  • 12.
    Pipeline Product Indication DevelopmentIND Phase I/II Phase III Ocular Oncology AU-011 Uveal Melanoma 2nd Indication (e.g. Retinoblastoma) Solid Tumors AU-021 Bladder/ Head & Neck Cancer Discovery Platform Immuno-Oncology 12
  • 13.
    Summary Product focused &novel technology: Tumor Targeted Viral-Like Particles Lead product for life threatening rare disease: Ocular melanoma FDA Orphan Drug Designation & strong pre-clinical validation Rapid clinical data output in Q1-2/16 Commercial launch preparation in 2017/18 for potential Fast Track Approval Focused call point enables direct commercialization Blockbuster market potential Experienced Management Team, Board of Directors, and Advisors 13
  • 14.
    Elisabet de losPinos Founder & CEO eli@aurabiosciences.com