Corporate PresentationOIS, October 13th 2016

Elisabet de los Pinos, Ph.D.Founding Chief Executive Officer

Disclaimers and forward-looking statements

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.

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Working to transform the treatment of patients with choroidal melanoma

Targeted therapy with unique mechanism of action

Orphan Drug Designation granted May 2015

Potential for early clinical readout and accelerated regulatory path forward

Platform technology for non-ocular cancers

Management team with expertise in orphan drug development and market launch

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1 Based on epidemiology estimates by Health Advances (US) and Company (Europe and rest of world)2 COMS_2005. Collaborative Ocular Melanoma Study Group Report No. 26. Development of metastatic disease after enrollment into the COMS trials for treatment of choroidal

melanoma. Arch Ophthalmol 2005;123(12):1639-43

High unmet medical need with no FDA-approved therapies

Choroidal melanoma is a rare disease.

Diagnosed and treated by ocular oncologists (retina specialists)

54 ocular oncologist in the U.S.

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ocular oncologist in Europe

10,000

patients worldwide1

Early diagnosis ofprimary tumor

High unmet medical need for

the treatment of disease with vision preservation

25% of patients develop metastatic disease after five years2

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Current radioactive treatment options with high association of comorbidities

1. Medium melanomas: Plaque brachytherapy and proton beam radiotherapy - Requires multiple surgeries- Causes severe vision loss (>5 lines)1

- Does not prevent metastases2

- Cost of treatment: $50,0003

2. Large melanomas: Enucleation

1 Shields CL et al. Arch Ophthalmol. 2000 Sep;118(9):1219-282 COMS Report No. 18. Arch Ophthalmol 119: 969-982, 2001.3 Harbour et al. Investigative Ophthalmology & Visual Science March 2012, Vol.53, 3400

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AU-011: A first-in-class targeted therapy Targeted cytotoxicity upon light activation

Small molecule is conjugated to the primary amines in the surface of the viral nanoparticle. The drug is inactive without irradiation.

Viral nanoparticle conjugates (VNC) are delivered by intra-vitreal injection and target tumor cells in the choroid.

VNCs bind specifically to HSPGs on the tumor cell surface (multivalent binding).

Ophthalmic laser 689nm activates the drug. The light-activated drug disrupts the tumor cell membrane, leading to necrosis.

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Efficient outpatient treatment protocol

Routine administration by intra-vitreal injection – same as Avastin®, Eylea®, Lucentis®

Activated by ophthalmic laser within six hours in standard outpatient setting

689nm laser activation – same wavelength and light fluence as used for Visudyne®

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Significant response to AU-011 in tumor model with high correlation to human disease

Rabbit orthotopic tumor model with human choroidal melanoma cells1

Administration schedule: once a week for two or three weeks

All animals with small tumors had complete responses to the therapy2

Small number of incomplete responders had large tumors that extended beyond the treatment field of the laser

Data replicated in multiple academic labs3

AU-011 Dose Response

% Complete ResponseDose

0%Control

0%5 ug

20%20 ug

50 ug 80%

1 Grossniklaus et al. Investigative Ophthalmology & Visual Science June 2013, Vol.54, 42292 Complete Responses defined by >90% tumor necrosis or absence of tumor cells after treatment3 Dr. Grossniklaus/Emory University; Dr. Burnier/McGill University; BRM with oversight from Dr. Vavvas/ MEEI

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Efficacy results validated by histopathology

Complete responses after intra-vitreal injections of AU-011 once a week for two weeks in medium-large tumors

Original location of treated posterior tumor

Data: Fundoscopy and Histopathology images provided by Ora Clinical-BRM. Tumor implants performed by Dr. Vavvas Harvard Medical School. Data reviewed by Dr. Grossniklaus (Certified Ocular Pathologist at Emory University)

Histopathology (H&E): Posterior choroidal tumor with extra-ocular extension (untreated control)

Histopathology (H&E): No signs of posterior tumor after administration of AU-011 once a week for two weeks and light activation

A B

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Near-term clinical milestones

AU-011 could be the first FDA-approved therapy for primary choroidal melanoma.

Phase II/III Clinical TrialPhase I Clinical Trial

PIND agreement on clinical endpoints: - Safety / vision- Tumor response

IND

NDA

Robust preclinical data with complete responses in multiple independent animal model studies

Preclinical IND-Enabling Studies

Safety

Preliminary efficacy

Registration trialPhase II/III

2014 2015 2016 2017 2018

Fast Track /Breakthrough Designations

Clinical operations underway:- Multiple U.S. sites

qualified- Laser system setup

and training complete at a number of sites

2019

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Carol Shields, M.D.

Sara Selig, M.D.

A strong rare disease community of patients, foundations and ocular oncologists

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Rare disease with large market potential

Choroidal melanoma represents a unique rare disease commercial opportunity.

Unmet medical need with no FDA-approved therapies

Orphan drug market access and reimbursement value

Focused call points enable efficient commercialization opportunity

Initiating corporate and financing discussions to develop drug through approval

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Pipeline

Ocular Oncology

- Primary choroidal melanoma

- Additional rare ocular cancers (e.g. choroidal metastases) AU-011

AU-011

Preclinical

Additional Indications

AU-012- Primary bladder carcinoma

Technology Platform AU-014

AU-013- Immuno-oncology / CAR-T

IND Phase I Phase II/III

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Working to transform the lives of patients with choroidal melanoma

First-in-class targeted cancer therapy

Strong preclinical data in animal model with high correlation to human disease

Granted FDA Orphan Drug Designation

Phase I 2017

Rare disease with potential accelerated regulatory path forward

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