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(NASDAQ: OCUL) ANTONY MATTESSICH, MD PRESIDENT AND CEO OCTOBER 2019 OPHTHALMOLOGY INNOVATION SUMMIT TRANSFORMING DRUG DELIVERY LEVERAGING A NOVEL TECHNOLOGY PLATFORM

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Page 1: (NASDAQ: OCUL) TRANSFORMING DRUG DELIVERY · POSITIONING OCULAR THERAPEUTIX AS THE DRUG DELIVERY COMPANY Progress of pipeline programs • OTX-TP –discussion of Ph3A results with

(NASDAQ: OCUL)

ANTONY MATTESSICH, MD PRESIDENT AND CEO OCTOBER 2019

OPHTHALMOLOGY INNOVATION SUMMIT

TRANSFORMINGDRUG DELIVERY LEVERAGING A NOVEL TECHNOLOGY PLATFORM

Page 2: (NASDAQ: OCUL) TRANSFORMING DRUG DELIVERY · POSITIONING OCULAR THERAPEUTIX AS THE DRUG DELIVERY COMPANY Progress of pipeline programs • OTX-TP –discussion of Ph3A results with

FORWARD LOOKING STATEMENTS

2

Any statements in this presentation about future expectations, plans and prospects for the Company, including the commercialization of DEXTENZA®, ReSure® Sealant,or any of the Company’s product candidates, the development and regulatory status of the Company’s product candidates, such as the Company’s regulatorysubmissions for and the timing and conduct of, or implications of results from, clinical trials of, and the prospects of approveability for, DEXTENZA for any additionalindications, OTX-TP for the treatment of primary open-angle glaucoma and ocular hypertension, OTX-TIC for the treatment of primary open-angle glaucoma and ocularhypertension, OTX-TKI for the treatment of retinal diseases including wet AMD, and OTX-IVT as an extended-delivery formulation of the VEGF trap aflibercept for thetreatment of retinal diseases including wet AMD; the Company’s post-approval studies of ReSure Sealant; the ongoing development of the Company’s extended-delivery hydrogel depot technology; the potential utility or commercial potential of any of the Company’s product candidates; the potential benefits and futureoperation of the collaboration with Regeneron Pharmaceuticals, including any potential future payments thereunder; the sufficiency of the Company’s cash resourcesand 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 SecuritiesLitigation 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 Company’s clinical development programs, future results, performanceor achievements to differ significantly from those expressed or implied by the forward-looking statements. Such risks and uncertainties include, among others, thoserelated to the timing and costs involved in commercializing DEXTENZA, ReSure Sealant or any product candidate that receives regulatory approval, including theconduct of post-approval studies, the ability to retain regulatory approval of DEXTENZA, ReSure Sealant or any product candidate that receives regulatory approval, theability to maintain reimbursement codes for DEXTENZA, the initiation, timing and conduct of clinical trials, availability of data from clinical trials and expectations forregulatory submissions and approvals, the Company’s scientific approach and general development progress, the availability or commercial potential of the Company’sproduct candidates, the sufficiency of cash resources, the Company’s existing indebtedness, the ability of the Company’s creditors to accelerate the maturity of suchindebtedness upon the occurrence of certain events of default, the outcome of the Company’s ongoing legal proceedings and need for additional financing or otheractions and other factors discussed in the “Risk Factors” section contained in the Company’s quarterly and annual reports on file with the Securities and ExchangeCommission. In addition, the forward-looking statements included in this presentation represent the Company’s views as of the date of this presentation. The Companyanticipates that subsequent events and developments will cause the Company’s 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 except as required by law. These forward-looking statementsshould not be relied upon as representing the Company’s views as of any date subsequent to the date of this presentation.

Page 3: (NASDAQ: OCUL) TRANSFORMING DRUG DELIVERY · POSITIONING OCULAR THERAPEUTIX AS THE DRUG DELIVERY COMPANY Progress of pipeline programs • OTX-TP –discussion of Ph3A results with

POSITIONED TO DEVELOP BREAKTHROUGH TREATMENTS

3

WE BELIEVE THE APPROVAL OF DEXTENZA® IS ONLY THE BEGINNING

Obsolete immediate release injections

Leverage the hydrogel platform

Obsolete drop

therapies

Enable new delivery modalities for breakthrough technologies.

Provide unique drug delivery to the surface and anterior segment of the eye. Develop sustained release

injections for the posterior segment of the eye.

Page 4: (NASDAQ: OCUL) TRANSFORMING DRUG DELIVERY · POSITIONING OCULAR THERAPEUTIX AS THE DRUG DELIVERY COMPANY Progress of pipeline programs • OTX-TP –discussion of Ph3A results with

Glaucoma is an incurable, generally painless, chronic disease

2 million individuals are affected in the United States1 3 million+ will be affected by 20201

ADHERENCE FOR PATIENTS WITH GLAUCOMA IS PROBLEMATIC

• The primary goal of glaucoma treatment is to reduce intraocular pressure

• Various medications can significantly lower intraocular pressure and reduce the progression of glaucoma, but these are almost always life-long medications that must be taken on a daily basis

• Adherence to glaucoma therapies is particularly poor, with reported rates of non-adherence ranging from 30–80%2,3,4

• Poor adherence has been shown to be associated with disease progression and blindness5,6

• The molecules are effective but the delivery is flawed

1) Friedman DS, et al. Prevalence of open-angle glaucoma among adults in the United States. Arch Ophthalmol. 2004; 122:532–8. 2) Olthoff CM, et al. Noncompliance with ocular hypotensive treatment in patients with glaucoma or ocular hypertension an evidence-based review. Ophthalmology. 2005; 112:953–61. 3) Schwartz GF, et al. Adherence and persistence with glaucoma therapy. Surv Ophthalmol. 2008; 53(suppl1):S57–68. 4) Nordstrom BL, et al. Persistence and adherence with topical glaucoma therapy. Am J Ophthalmol. 2005; 140:598–606. 5) Rossi GC, et al. Do adherence rates and glaucomatous visual field progression correlate? Eur J Ophthalmol. 2011; 21:410–4. 6) Sleath B, et al. The relationship between glaucoma medication adherence, eye drop technique, and visual field defect severity. Ophthalmology. 2011; 118:2398–402.

POOR ADHERENCE HAS BEEN SHOWN TO BE ASSOCIATED WITH DISEASE PROGRESSION AND BLINDNESS

OPHTHALMIC DROPS

Page 5: (NASDAQ: OCUL) TRANSFORMING DRUG DELIVERY · POSITIONING OCULAR THERAPEUTIX AS THE DRUG DELIVERY COMPANY Progress of pipeline programs • OTX-TP –discussion of Ph3A results with

OTX-TP DELIVERS TRAVOPROST FOR ~90 DAYS WITH A SINGLE INSERTION

Sustained zero-order release for up to 90 days

• Little to no hyperemia (red eye) and

less staining of the skin

• Less diurnal IOP variation

• Occlusion helps maintain tear fluid levels

Provides incentive for patients and physicians

• Non-invasive physician administration

• Preservative-free

• Product can be monitored by physician

• Buy and bill product with possibility of reimbursement for insertion

• Eliminates co-pay for Medicare Part B patients with supplemental insurance

OTX-TP (travoprost insert)for intracanalicular use

Page 6: (NASDAQ: OCUL) TRANSFORMING DRUG DELIVERY · POSITIONING OCULAR THERAPEUTIX AS THE DRUG DELIVERY COMPANY Progress of pipeline programs • OTX-TP –discussion of Ph3A results with

MEAN IOP CHANGE FROM BASELINE

STATISTICALLY SIGNIFICANT GREATER MEAN IOP DECREASE FROM BASELINE WITH OTX-TP IN 8 OUT OF 9 ENDPOINTS

-7

-6

-5

-4

-3

-2

-1

0

8 AM 10 AM 4 PM 8 AM 10 AM 4 PM 8 AM 10 AM 4 PM

Me

an

IO

P c

ha

ng

e f

rom

Ba

selin

e

(mm

Hg

)

OTX-TP - Baseline (n=343) Placebo - Baseline (n=211)

2 WEEKS 12 WEEKS6 WEEKS

** *

** *

* *

*Statistically Significant; P ≤ 0.05; Full Analysis Set (FAS) Population(N=554) Least Squares Means

Confidential: Not For Distribution

Page 7: (NASDAQ: OCUL) TRANSFORMING DRUG DELIVERY · POSITIONING OCULAR THERAPEUTIX AS THE DRUG DELIVERY COMPANY Progress of pipeline programs • OTX-TP –discussion of Ph3A results with

OTX-TP PHASE 3 TRIAL CONCLUSIONS

• EFFICACY

✓ OTX-TP treated subjects had greater IOP lowering than vehicle treated subjects at all time points

✓ OTX-TP showed a statistically significant decrease in mean diurnal IOP than Placebo at Weeks 2, 6 & 12, except at 8 AM on Week 12 timepoint; Achieving 8 out of 9 primary efficacy endpoints

• SAFETY

✓ Generally well tolerated

✓ Most common ocular adverse events (>5%) in patients receiving OTX-TP were dacryocanaliculitisand lacrimal structural disorders

✓ There were no ocular serious adverse events

✓ Most TEAE’s were mild or moderate

• DURABILITY

✓ OTX-TP demonstrated the ability to lower IOP over 12 weeks with a single insert

✓ OTX-TP showed greater IOP lowering over the first 6 weeks compared with later time points

OTX-TP DEMONSTRATED EFFICACY FOR CONTROLLING IOP IN SUBJECTS WITH PRIMARY OPEN ANGLE GLAUCOMA AND OCULAR HYPERTENSION FOR UP TO 12 WEEKS

Confidential: Not For Distribution

FDA meeting scheduled for October 30th 2019 on path forward

Page 8: (NASDAQ: OCUL) TRANSFORMING DRUG DELIVERY · POSITIONING OCULAR THERAPEUTIX AS THE DRUG DELIVERY COMPANY Progress of pipeline programs • OTX-TP –discussion of Ph3A results with

OTX-TIC DELIVERS TRAVOPROST FOR AT LEAST 6 MONTHS VIA INTRACAMERAL INJECTION

Sustained zero-order release for approximately 6 months

• Pre-clinical model shows marked reduction in IOP and a matching PK/PD response

• Administered with proprietary injector (27 gauge) and delivered directly to site of action

Provides another tool in glaucoma specialists’ armamentarium

• 4-6 month duration

• Secure placement in the inferior angle

• Minimal effect on endothelial cells

• Full resorption prior to second dose

• Preservative-free

• Product can be monitored by physician

OTX-TIC (travoprost implant)for intracameral injection

Page 9: (NASDAQ: OCUL) TRANSFORMING DRUG DELIVERY · POSITIONING OCULAR THERAPEUTIX AS THE DRUG DELIVERY COMPANY Progress of pipeline programs • OTX-TP –discussion of Ph3A results with

IMPLANTS VISUALIZED IN ALL SUBJECTS AT ALL VISITS THROUGH 7 MONTHS

OTX-TIC PHASE 1 INTERIM FINDINGS

Month 4

Month 6 Month 7

Day 28• Clinically-meaningful decrease in IOPIOP values were decreased as early as two days

• Duration of therapyIOP-lowering out to 9 months

• BioresorbableImplant biodegraded by 7 months

• Implant location and movementImplant did not move and was visualized in all patients

• Corneal healthNo changes from baseline observed; preservative-free

SUMMARY FROM LOW-DOSE COHORT

Page 10: (NASDAQ: OCUL) TRANSFORMING DRUG DELIVERY · POSITIONING OCULAR THERAPEUTIX AS THE DRUG DELIVERY COMPANY Progress of pipeline programs • OTX-TP –discussion of Ph3A results with

OTX-TIC EFFICACY RESULTS: MEAN IOP CHANGE FROM BASELINE, COHORT 1

MEASUREMENTS TAKEN AT 8AM

-1.5

-7.4

-8.8 -8.7-9.1

-7.0

-5.6

-4.6

-6.1

-10.8

-9.6

-7.1-6.6

-1.4

-4.6

-7.6-8.0

-6.6

-5.0 -5.2

-4.1

-5.3

-7.6

-6.8

-7.8

-6.3

-11

-10

-9

-8

-7

-6

-5

-4

-3

-2

-1

0

Study Eye: OTX-TIC Implant

Non-Study Eye: Topical Travoprost (QD Nightly)

n = 5 n = 5 n = 4 n = 5 n = 5 n = 5 n = 5 n = 5 n = 3 n = 2 n = 2 n = 2 n = 2

Data as of mid-April 2019

Page 11: (NASDAQ: OCUL) TRANSFORMING DRUG DELIVERY · POSITIONING OCULAR THERAPEUTIX AS THE DRUG DELIVERY COMPANY Progress of pipeline programs • OTX-TP –discussion of Ph3A results with

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ANTICIPATED NEAR-TERM MILESTONES

(using pass-through reimbursement status using J-code 1096 effective October 1)

Take new product candidates into the clinic in 2019

Actively seek new business development opportunities

POSITIONING OCULAR THERAPEUTIX AS THE DRUG DELIVERY COMPANY

Progress of pipeline programs• OTX-TP – discussion of Ph3A results with FDA (October 30, 2019)• OTX-TIC – plans to evaluate 2 new formulations in the near future• OTX-TKI – third therapeutic-dose cohort has begun enrollment

2019 LAUNCH

Page 12: (NASDAQ: OCUL) TRANSFORMING DRUG DELIVERY · POSITIONING OCULAR THERAPEUTIX AS THE DRUG DELIVERY COMPANY Progress of pipeline programs • OTX-TP –discussion of Ph3A results with

THANK YOU!

(NASDAQ: OCUL)

TRANSFORMINGDRUG DELIVERY LEVERAGING A NOVEL TECHNOLOGY PLATFORM