ophthalmology innovation showcase - ocular therapeutix

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Amar Sawhney, Ph.D., CEO OIS@ASCRS New Orleans May 5, 2016 1 Transforming Ophthalmic Care with Sustained Therapies

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P011643

Amar Sawhney, Ph.D., CEO

OIS@ASCRSNew OrleansMay 5, 20161Transforming Ophthalmic Carewith Sustained Therapies

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Forward-Looking Statements2This presentation contains forward-looking statements about future expectations, plans and prospects for the Company, including statements about the development and regulatory status of the Companys 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 inflammation and pain, DEXTENZA for allergic conjunctivitis, DEXTENZA for dry eye disease and OTX T-P for glaucoma and ocular hypertension, the ongoing development of the Companys sustained released hydrogel depot technology, pre-commercial activities, the potential commercialization of DEXTENZA, the potential utility of any of the Companys product candidates, the advancement of the Company's other product candidates and earlier stage pipeline, future sales of ReSure Sealant, 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 Ocular Therapeutixs 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 and DEXTENZA, 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 of the Companys filings with the Securities and Exchange Commission, including the Companys most recent Annual Report on Form 10-K. 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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Drug-eluting intracanalicular depots and intravitreal injections are investigational new drugs and not commercially available in the United States or other geographies.

Posterior Segment Sustained Release InjectionsAnterior Segment Sustained Release Therapies

Hydrogel Sealant

Addressing Diseases in the Anterior and Posterior SegmentTransforming Ophthalmic Care with Sustained Therapies3

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Compliance is Key4

Burden of Daily Eye Drops

In an observational study, 92.6% of post-cataract patients showed improper administration technique with steroid drops:Missing eyeInstilling incorrect amountContaminating bottle tipFailing to wash handsAn JA, Kasner O, Samek DA, Levesque V. Evaluation of eyedrop administration by inexperienced patients after cataract surgery. J Cataract Refract Surg, 2014; 40:1857-1861Real World Outcomes Are Not Optimal

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Platform Value Proposition5PLATFORM BUILDING BLOCKSOcular Therapeutix sustained deliveryUnderstanding the Opportunity for DEXTENZA in Ophthalmology Practices Working Insights, n=42 ophthalmologists IDIs. Krog and Partners, March, 2016. Strategy & Message Platform Validation Research, n=50 U.S. ophthalmologists, OptiBrand Rx LLC, 2015.

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6IMS data, March 2016Roche Finance Report 2015, page 11 http://www.roche.com/fb15e.pdf; Regeneron; http://www.fiercepharma.com/story/after-big-2015-regeneron-predicts-slowing-2016-sales-launch-superstar-eylea/2016-02-09; Targeting Markets in the U.S. Currently Totaling $11Bn1,2US Sales ($Bn)Market OpportunitiesInflammatory Conditions$ 4.1

OTX-IVTAnti-VEGF$ 4.2

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DEXTENZAProduct Profile:One-time sustained release depotDesigned to replace complex topical dosing regimenPreservative-freeInitial Planned Indication:Treatment of ocular painassociated with ophthalmic surgeryDelivery Method:Physician-administered into canaliculus following surgery30-day sustained and tapered delivery

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1st Phase 3 complete 2nd Phase 3 enrollment complete sNDA expected 2H 2016Allergic ConjunctivitisDEXTENZA Label Expansion Strategy9PDUFA July 24, 2016Post-Surgical PainPhase 3 trial currently enrollingsNDA expected 1Q 2017Post-SurgicalInflammationStrong pain reliefEarly onset (Day 2)One-time seasonal prophylaxisReduced risk of overmedicationDual mechanism:Build up tear levelTreat inflammationCompliance, physician control, preservative-free

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Market Research Outcomes10Standard of CareDEXTENZA79%Other21%79% of Ophthalmologists stated that DEXTENZA could become new standard of careUnderstanding the Opportunity for DEXTENZA in Ophthalmology Practices Working Insights, n=42 ophthalmologists IDIs. Krog and Partners, March, 2016.

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OTX-TP: Sustained Release Travoprostfor the Treatment of Glaucoma11

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Sustained Release Travoprost (OTX-TP)

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Product Profile:Sustained release depotDesigned to replace daily therapyPreservative-freeProduct can be monitored by patientInitial Planned Indication:Intraocular pressure reduction for glaucoma and ocular hypertensionDelivery Method:Non-invasivePhysician-administered: inserted into canaliculus Provides sustained delivery for up to 90 days

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Multiple Phase 2 trials have demonstrated clinically meaningful 5+ mm/Hg of IOP lowering with a single placement out to 2-3 months

FDA discussions for Phase 3 plans indicate:Placebo comparator acceptableNeed to demonstrate statistical superiority to placebo and clinically meaningful reduction of IOPNo timolol comparator or validation arm required, no eye drops required (placebo or active) Two planned trials: ~550 subjects/study; 100+ to be followed out to one year for safety in one trial Expect to begin Phase 3 clinical studies in 2H 2016OTX-TP Progress and Plans13

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OTX-IVT: Sustained Release Injectable Depots for the Treatment of Retinal Diseases

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Posterior Segment Programs15

ProteinTherapeuticsDemonstrated:Protein stabilityTolerabilityRelease profile Seek partnership for anti-VEGF drug

Small MoleculeDrugsTyrosine KinaseInhibitors (TKIs)Pursuing internal developmentInitial PK/PD and tolerability demonstrated

Injectable depot6 months sustained releaseFine needle injection No interference with visionBiocompatibleAbsorbable

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Rabbit PD Screening ModelFluorescein Angiography (FA) Scores at 2, 4, 6, 9, 12 weeksBevacizumab-loaded fiber showed inhibition up to 12 weeks vs. < 6 weeks from single human Avastin dose (1.25mg)

Empty Depot Control

Avastin Pre-treatmentInhibition: Leakage score = 0No Inhibition: Leakage: score = 4Fluorescein leaking from vesselsRepresentative FA ImagesBevacizumab-loaded fiber showed continued inhibition up to 12 weeksSustained Pharmacodynamic Activity

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17Transforming Ophthalmic Carewith Sustained Therapies

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