PowerPoint Presentation

Amar Sawhney, Ph.D.CEO1Transforming Ophthalmic Care with Sustained TherapiesPosterior Segment Company Showcase OIS@AAO October 13, 2016

Forward looking statements2Any statements in this presentation about future expectations, plans and prospects for the Company, including statements about the potential benefits and future operation of the collaboration with Regeneron, including any potential future payments thereunder, the ongoing development of the Companys sustained release hydrogel depot technology, the development and regulatory status of the Companys other product candidates, such as the Companys expectations and plans regarding regulatory submissions for and the timing and conduct of clinical trials of DEXTENZA for post-surgical ocular inflammation and pain, including our expectations regarding the NDA filed with the FDA, DEXTENZA for the treatment of allergic conjunctivitis, DEXTENZA for dry eye disease and OTX-TP for the treatment of glaucoma and ocular hypertension, the potential utility of any of the Companys product candidates, potential commercialization of the Companys product candidates, the sufficiency of the Companys cash resources and other statements containing the words "anticipate," "believe," "estimate," "expect," "intend", "goal," "may", "might," "plan," "predict," "project," "target," "potential," "will," "would," "could," "should," "continue," and similar expressions, constitute forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors. Such forward-looking statements involve substantial risks and uncertainties that could cause the Companys clinical development programs, future results, performance or achievements to differ significantly from those expressed or implied by the forward-looking statements. Such risks and uncertainties include, among others, those related to the timing and costs involved in commercializing ReSure Sealant or any product candidate that receives regulatory approval, the initiation and conduct of clinical trials, availability of data from clinical trials and expectations for regulatory submissions and approvals, the Companys scientific approach and general development progress, the availability or commercial potential of the Companys product candidates, the sufficiency of cash resources and need for additional financing or other actions and other factors discussed in the Risk Factors section contained in the Companys quarterly and annual reports on file with the Securities and Exchange Commission. In addition, the forward-looking statements included in this presentation represent the Companys views as of the date of this presentation. The Company anticipates that subsequent events and developments will cause the Companys views to change. However, while the Company may elect to update these forward-looking statements at some point in the future, the Company specifically disclaims any obligation to do so. These forward-looking statements should not be relied upon as representing the Companys views as of any date subsequent to the date of this presentation.

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Intracanalicular InsertExtended and tapered delivery for 30 daysPreservative-freeAbsorbable no need for removalDelivery MethodNon-invasivePhysician-administered with forcepsDEXTENZA (dexamethasone insert) For Intracanalicular Use

Potential IndicationTreatment of ocular pain occurring after ophthalmic surgeryTarget MarketApproximately 5.3 million ocular surgeries in the U.S. in 2015; 27 million worldwide1One-time insert designed to replace complex steroid eye drop topical dosing regimen1 Source: Market Scope

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As seen in this short animation, the Dextenza depot hydrates and swells on contact with fluid, releasing preservative free dexamethasone steroid medication in a slowly tapered fashion tailored to last 1month, mimicking a standard drop tapering regimen requiring 70 separate drop administrations that need occur with varying and decreasing frequency as the weeks go by. Because the depot is placed by the physician, there is no concern that the patient may be getting too much or too little medication. In fact, the amount of steroid needed to achieve therapeutic effect with Dextenza is only about 7% of what the eye would be exposed to if drops were used for this same course of therapy.

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DEXTENZA (dexamethasone insert) For Intracanalicular UsePlanned NDA re-submission under discussion with FDAData read-out of Phase 3 study for potential indication expansion to obtain inflammation by end of Q4, 2016

OTX-TP (extended release travoprost)1st Phase 3 clinical study enrollment initiated for the treatment of glaucoma and ocular hypertensionPotential to be first non-invasive treatment of glaucoma and ocular hypertension for up to 3 months with one dose

Anterior segment program status4

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Product pipeline5

Regeneron collaboration6Goal: to advance current standard of care by reducing injection frequency in the treatment of wet AMDGlobal market for anti-VEGF drugs >$7.5 billion per year

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Dual posterior segment development strategy

TKIs

ProteinTherapeuticsPartnership with RegeneronDemonstrated pre-clinical:Protein stabilityRelease profileHydrogel tolerability

SmallMoleculeDrugsTyrosine KinaseInhibitors (TKIs)Pursuing internal developmentDemonstrated pre-clinical:PKPDHydrogel tolerability7

Antibody drugs act to intercept VEGF, PDGF to prevent engagement with their receptors: VEGFR2, PDGFRTKIs inhibit the activity of kinases associated with VEGFR2, PDGFR

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Ocular Therapeutixintravitreal depot technology

Biocompatible with known anti-angiogenesis drugs and absorbableFine needle deliveryLittle to no visual field impactUp to 6-month sustained delivery

Depots to scaleOcular Therapeutixs depot technology has been tested with a range of anti-angiogenesis drugs in multiple preclinical models8

Ocular Therapeutix technology provides a sustained release vehicle designed to work well with common intravitreal injection practice and with known anti-angiogenesis drugs

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Free floating within the vitreousAbsorbs after 6-7 months Allows for high drug capacityProtein anti-VEGF depot9Depot presents a smooth, hydrophilic, biocompatible surface to tissues

In vivo IR images of depot at 2, 4 and 8 weeksDepot hydrates and coils as it exits needleDepot is soft, lubricious biodegradable hydrogel composed of >50% drug

Ocular Therapeutix has recently developed a proprietary technology for a coiling fiber depot that forms a compact shape in situ after ejection from a needle. This is a soft, smooth and lubricious hydrogel, containing embedded drug particles.

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Depot EyeInhibitionLeakage score = 1

Control EyeNo Inhibition Leakage score = 4Fluorescein leaking from vesselsBevacizumab depot showed continued inhibition up to 12 weeks vs. < 6 weeks from single human dose of Avastin (1.25mg) in pre-clinical VEGF Challenge Model.Continued inhibitionProtein anti-VEGF depot suppressed vascular leakage for 3 monthsRepresentative FA images

We have used a modification of a VEGF angiogenesis induction model in dutch belted rabbits to determine if the anti-angiogenic activity can be sustained beyond the normal Avastin formulation. This model uses repeated challenges at various time points following the single injection of the test article - as long as the rabbits remain protected from VEGF. This particular bevacizumab depot was designed to deliver for 3 months.

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Target 100% release 4-6 months11Stable sustained protein anti-VEGF deliveryThree anti-VEGF agents showed active release out to 5 months (SEC) in a pre-clinical modelDELIVERY: Meets 4-6 month targetAGGREGATION: Highly stable in hydrogel

We have worked with several antibodies, including anti-angiogenic antibody drugs. The hydrogel has been found to work with a wide molecular weight range from Fab fragments to full antibodies. Aggregation is the first form of protein change seen, in which antibody molecules reversibly join to form mostly dimers, and some trimers. These aggregates are referred to collectively as high molecular weight species. They are not formed from the PEG vehicle. The anti-VEGF molecules we have tested have shown good stability, as evidenced by this low aggregation, which has been minimal and does not result in loss of protein activity.11

Fine needle deliveryBiocompatible, hydrophilic Little to no visual field impactAbsorbs after 6-7 months TKI depot121 mm

18 mm

Depot is soft and forms compact depot in situ

Pre-filled ready-to-use applicator

Hydrated depot

Ocular Therapeutix has recently developed a proprietary technology for a coiling fiber depot that forms a compact shape in situ after ejection from a needle. This is a soft, smooth and lubricious hydrogel, containing embedded drug particles.

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TKI depot in vivo ocular tolerability13Score ScaleNo changeMinimalMildModerateMarkedSevere

Hydrogel residueNo inflammatory cellsNo inflammation at 26 weeksTolerability demonstrated at 26 weeks

Normal retinal morphology preserved over 26 weeksTKI depot retinal tolerability4 weeksDrug loaded12 weeksPartially released26 weeksMostly absorbed

Normal retinal morphologyNormal retinal morphologyNormal retinal morphology

in vivo IR/OCT drug-loaded depot imaging14

TKI depot efficacy in DL-AAA model for AMD15Pre- injection(Baseline)2 weeks8 weeks

LeakageLeakageLeakageLeakageNo LeakageNo LeakageNo LeakageNo LeakageContinuous leakage modelTKI depotAvastin injection

10 weeks

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TKI depot in VEGF challenge model for AMD

16PKPDHigh sustained concentrationsContinued effectin vivo PK/PD challenge model over 6-month duration

Posterior SegmentAnti-VEGF protein depots demonstrated:in vivo tolerability through 26 weeks in rabbitsin vivo effect (PD) in VEGF-induced leakage model through 12 weeksin vitro sustained release of three (3) anti-VEGFs for 4-6 monthsTKI depot demonstrated to 26 weeks:in vivo tolerabilityin vivo high sustained retinal concentration (PK) in vivo effect (PD) in DL-AAA and VEGF-induced leakage models

Anterior SegmentDEXTENZA (dexamethasone insert) Planned NDA re-submission under discussion with FDAOTX-TP (extended release travoprost)Phase 3 underway

Ocular Therapeutix program summary17

18Transforming Ophthalmic Care with Sustained Therapies

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