Ocular Therapeutix is developing sustained-release drug therapies for ophthalmic conditions to improve patient outcomes and compliance. Their lead product, DEXTENZA, is an intracanalicular insert delivering dexamethasone for up to 30 days after cataract surgery to reduce pain and inflammation. Phase 3 trials showed DEXTENZA was well-tolerated and effective in meeting primary efficacy endpoints. If approved, DEXTENZA could become the new standard of care by replacing complex topical drop regimens. The company is also developing sustained-release therapies for glaucoma and posterior segment diseases in collaboration with Regeneron.
2. FORWARD-LOOKING STATEMENTS
2
Any statements in this presentation about future expectations, plans and prospects for the Company including the development and regulatory status of the
Company’s product candidates, such as the Company’s expectations and plans regarding regulatory submissions for and the timing and conduct of, or
implications of results from, clinical trials of DEXTENZA™ for the treatment of post-surgical ocular inflammation and pain, including our expectations regarding the
New Drug Application (“NDA”) filed with the Food and Drug Administration (“FDA”), and the resubmission of the NDA and a potential NDA supplement, DEXTENZA
for the treatment of allergic conjunctivitis, DEXTENZA for the treatment of dry eye disease and OTX-TP for the treatment of glaucoma and ocular hypertension, the
ongoing development of the Company’s sustained release hydrogel depot technology, the potential utility of any of the Company’s product candidates,
potential commercialization of the Company’s product candidates, the potential benefits and future operation of the collaboration with Regeneron
Pharmaceuticals, including any potential future payments thereunder, the sufficiency of the Company’s 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 Company’s 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 Company’s scientific approach and general development
progress, the availability or commercial potential of the Company’s 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 Company’s quarterly and annual reports on file with the Securities and
Exchange Commission. In addition, the forward-looking statements included in this presentation represent the Company’s views as of the date of this
presentation. The Company anticipates 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. These forward-
looking statements should not be relied upon as representing the Company’s views as of any date subsequent to the date of this presentation.
3. THE FOCUS IS ON PATIENTS
Our products are designed to improve patient outcomes
for the leading causes of blindness in the US
3
How Many Americans Over 40 Have An Age-Related Eye Disease? Prevalence of Adult Vision Impairment and Age-Related Eye Diseases in America, National Eye Institute, NIH.
https://nei.nih.gov/eyedata/adultvision_usa. April, 2016.
AMERICANS OVER 40 WITH AN AGE-RELATED EYE DISEASE
Diabetic
Retinopathy
7.68 M
Glaucoma
2.72 M
Age-Related
Macular
Degeneration
2.07 M
Cataract
24.41 M
4. POSITIONED FOR SUCCESS
Combining critical factors to advance sustained
release therapies in ophthalmology
4
Extensive
ophthalmic
knowledge
and
experience
Developing
products
that address
unmet needs
Proprietary
hydrogel
platform
technology
Investment
in early, mid-,
and late stage
products
candidates
Commitment
to Research &
Development
Strategic
industry
collaboration
DEXTENZA™ is an investigational new drug and is not approved by the FDA as safe or effective.
5. SUSTAINED-RELEASE OPHTHALMIC THERAPIES
Improved
control for
physicians
Compliance is
alleviated
Reduced
dose
frequency
and patient
burden
Inserts are
visible with
filtered light
for retention
monitoring
5
Intracanalicular
Insert
The next generation in drug delivery for ophthalmic care
Intravitreal
Depot
6. 6
WE HAVE A DIVERSE PIPELINE
Focused on addressing ophthalmic conditions in both
the anterior and posterior segment
7. CURRENT STANDARD OF CARE
FOLLOWING CATARACT SURGERY
Post-operative eye drop treatment represents a
significant burden for patients and their caregivers
7Dosage and Administration Information per product Prescribing Information.
Steroid
1 drop QID (to start)
with 1 drop weekly
taper
Antibiotic
1 drop QD
1 week
NSAID
1 drop QD
4 weeks
QID: Four times daily. QD: Once daily. BID: Twice daily. TID: Three times daily.
8. DEXTENZA™ - NOVEL DRUG DELIVERY
Extended release resorbable drug product
• 0.4 mg dexamethasone encapsulated within hydrogel
• A single, physician administered, intracanalicular insert
without preservatives.
• Designed to replace complex topical steroid dosing
regimen
• Provides sustained and tapered delivery for up to 30 days
8
Driscoll A, et al. Toxicity and Pharmacokinetics of Sustained-Release Dexamethasone in Beagle Dogs.
Adv Ther. 2016; 33:58-67.
NDA FOR TREATMENT OF OCULAR PAIN AFTER OPHTHALMIC SURGERY IS UNDER REVIEW WITH THE FDA
Proprietary formulation designed for insertion into the lacrimal canaliculus
• Insert softens, liquefies and is cleared through the nasolacrimal duct without removal
9. *Statistically Significant; P ≤ 0.0025 ITT-LOCF Population
80.4%
77.5%
79.6%
43.4%
58.8%
61.3%STUDY 3A* STUDY 3B* STUDY 3C*
PROPORTIONOFPATIENTSWITHA
SCOREOF0INTHESTUDYEYE
ABSENCE OF PAIN, DAY 8
33.1%
39.4%
52.3%
14.5%
31.3%
31.1%
STUDY 3A* STUDY 3B STUDY 3C*
PROPORTIONOFPATIENTSWITHA
SCOREOF0INTHESTUDYEYE
ABSENCE OF ANTERIOR CHAMBER
CELLS, DAY 14
PRIMARY EFFICACY ENDPOINTS:
Absence of Pain and Inflammation
9
Walters TR, et al. Efficacy and Safety of Sustained Release Dexamethasone for the Treatment of Ocular Pain and Inflammation after Cataract Surgery: Results from Two Phase 3 Studies. J Clin Exp Ophthalmol. 2016; 7(4): 572. Tyson S, et al. Safety and
Efficacy of an Extended-Release Dexamethasone Insert for Treatment of Ocular Inflammation and Pain after Surgery: Phase 3 Study. Accepted Abstract for the American Society of Cataract and Refractive Surgery 2017 Symposium & Congress.
Los Angeles, CA, May 5-9, 2017.
DEXTENZA met its primary efficacy endpoints in two of three Phase 3 studies
n=164
n=83
n=161
n=80
n=216
n=222
n=164
n=83
n=161
n=80
n=216
n=222
DEXTENZA Placebo
10. 10
DEXTENZA PHASE 3 SAFETY OVERVIEW
• Well-tolerated. AEs and other safety observations were consistent with the safety profile of
topical ocular steroids following cataract surgery
• Most common AEs were anterior chamber inflammation and elevated intraocular pressure
• A majority of events were graded mild or moderate
• No SAEs were considered treatment-related
Study 3A Study 3B Study 3C
# of Subjects, n (%)
DEXTENZA
N=162
Placebo
N=84
DEXTENZA
N=160
Placebo
N=80
DEXTENZA
N=216
Placebo
N=221
Any Ocular AEs 2 (1.2%) 1 (1.2%) 1 (0.6%) 1 (1.3%) 1 (0.5%) 0
Conjunctivitis 0 1 (1.2%) 0 0 0 0
Eyelid Irritation 1 (0.6%) 0 0 0 0 0
Increased
Lacrimation
1 (0.6%) 0 0 0 1 (0.5%) 0
Dacryocanaliculitis 0 0 0 1 (1.3%) 0 0
Increased Intraocular
Pressure
0 0 1 (0.6%) 0 0 0
Treatment-Related Ocular Adverse Events in the Study Eye
11. PATIENT REPORTED OUTCOMES
Evaluation of the patient experience with DEXTENZA following cataract
surgery while participating in the Phase 3 clinical trial program
11
Would recommend DEXTENZA
to family members
92%
Willing to pay more than drops84%
Rated overall experience
“very” or “extremely convenient”
96%
Based on a patient-reported outcomes survey conducted by Covance on behalf of Ocular Therapeutix
Stated insert was comfortable
100%
12. DEXTENZA MARKET RESEARCH OUTCOMES
12
79% of Ophthalmologists stated that DEXTENZA could become
new standard of care
Understanding the Opportunity for DEXTENZA™ in Ophthalmology Practices Working Insights, n=42 ophthalmologists IDIs. March 2016.
93% of Ophthalmologists believe that the core value
proposition of DEXTENZA is potential to improve patient
compliance and ultimately outcomes
• Without Preservatives
• Ease of use
• Low risk of IOP spikes
• Self-tapering nature
• Continuous drug delivery
• Physician-controlled dosing
13. 13
OTX-TP & OTX-TIC GLAUCOMA TREATMENT STRATEGY
Description
• Steady drug release rate
• Designed to replace daily
therapy
– Adherence to regimen
• Product can be visualized
• Months of sustained delivery
Potential Use
• IOP reduction for glaucoma
and ocular hypertension
– Reduce vision loss
Intracanalicular Intracameral
OTX-TP PHASE 3 OTX-TIC PRECLINICAL
3 days 3 months
14. POSTERIOR SEGMENT DELIVERY
14
High drug load Low drug load
FIBER TO COIL
FIBER TO ROD
INJECTABLE
SHAPE CHANGING
DEPOTS
In preclinical studies, demonstrated:
• Protein stability
• Tolerability
• Release profile
In Partnership with Regeneron
OTX-IVT
(Antibodies)
Internal Development
• Demonstrated in initial
preclinical PK/PD studies
safety, efficacy and
tolerability up to 26 weeks,
to be presented at ARVO
OTX-TKI
(Small Molecules)
15. LOOKING FORWARD
15
PDUFA date for DEXTENZA of 7/19/2017. If approved, anticipate full
commercial launch for post-surgical ocular pain in 1H 2018
Phase 3 OTX-TP trials for open angle glaucoma and ocular hypertension
progressing well, plan to initiate 2nd phase 3 efficacy trial Q3 2017
Phase 1 clinical trials of OTX-TIC for glaucoma planned in 2H 2017
Ongoing collaboration with Regeneron to develop a form of
extended release EYLEA®
Phase 1 clinical trials of OTX-TKI anticipated in 2H 2017