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Amar Sawhney, Ph.D., CEO
OIS@ASCRS
New Orleans
May 5, 2016
1
Transforming Ophthalmic Care
with Sustained Therapies
Forward-Looking Statements
2
This presentation contains forward-looking statements about future expectations, plans and prospects for the
Company, including statements about 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 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 Company’s sustained released hydrogel depot technology, pre-commercial activities, the
potential commercialization of DEXTENZA, the potential utility of any of the Company’s product candidates, the
advancement of the Company's other product candidates and earlier stage pipeline, future sales of ReSure®
Sealant, 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 Ocular
Therapeutix’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 and
DEXTENZA, 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 of
the Company’s filings with the Securities and Exchange Commission, including the Company’s most recent
Annual Report on Form 10-K. 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.
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
Injections
Anterior Segment
Sustained
Release
Therapies
Hydrogel Sealant
Addressing Diseases in the
Anterior and Posterior Segment
Transforming Ophthalmic Care with
Sustained Therapies
3
Compliance is Key
4
Burden of Daily Eye Drops In an observational study,
92.6% of post-cataract patients
showed improper
administration technique with
steroid drops:
• Missing eye
• Instilling incorrect amount
• Contaminating bottle tip
• Failing to wash hands
An JA, Kasner O, Samek DA, Levesque V. Evaluation of
eyedrop administration by inexperienced patients after cataract
surgery. J Cataract Refract Surg, 2014; 40:1857-1861
Real World Outcomes Are Not Optimal
Platform Value Proposition
5
COMPLIANCE
PHYSICIAN CONTROL
PRESERVATIVE-FREE
Ocular Therapeutix
sustained delivery
Understanding 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.
6
1. IMS data, March 2016
2. Roche 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 $11Bn1,2
US Sales ($Bn)
Market Opportunities
OTX-TP
Glaucoma
$ 2.7
Allergy
$ 1.0
Dry eye
$ 1.6
Pain & Inflammation
(Anti-Inflammatories)
$ 1.5
Inflammator
y
Conditions
$ 4.1
OTX-IVT
Anti-VEGF
$ 4.2
7
7
8
DEXTENZA™
Product Profile:
• One-time sustained release depot
• Designed to replace complex topical
dosing regimen
• Preservative-free
Initial Planned Indication:
• Treatment of ocular pain
associated with ophthalmic surgery
Delivery Method:
• Physician-administered into canaliculus
following surgery
• 30-day sustained and tapered delivery
• 1st Phase 3 complete
• 2nd Phase 3 enrollment
complete
• sNDA expected 2H 2016
Allergic Conjunctivitis
DEXTENZA Label Expansion Strategy
9
PDUFA July 24, 2016
Post-Surgical Pain
• Phase 3 trial currently
enrolling
• sNDA expected 1Q 2017
Post-Surgical
Inflammation
• Strong pain relief
• Early onset (Day 2)
• One-time seasonal
prophylaxis
• Reduced risk of
overmedication
Dual mechanism:
• Build up tear level
• Treat inflammation
Compliance, physician control, preservative-free
Market Research Outcomes
10
Standard of Care
DEXTENZA
79%
Other
21%
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. Krog and Partners, March,
OTX-TP: Sustained Release Travoprost
for the Treatment of Glaucoma
11
Sustained Release Travoprost (OTX-TP)
12
Product Profile:
• Sustained release depot
• Designed to replace daily therapy
• Preservative-free
• Product can be monitored by patient
Initial Planned Indication:
• Intraocular pressure reduction for glaucoma and ocular hypertension
Delivery Method:
• Non-invasive
• Physician-administered: inserted into canaliculus
• Provides sustained delivery for up to 90 days
• 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 acceptable
– Need to demonstrate statistical superiority to placebo and
clinically meaningful reduction of IOP
– No 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 2016
OTX-TP Progress and Plans
13
OTX-IVT: Sustained Release Injectable
Depots for the Treatment of Retinal Diseases
14
Posterior Segment Programs
15
Protein
Therapeutics
• Demonstrated:
– Protein stability
– Tolerability
– Release profile
• Seek partnership for
anti-VEGF drug
Small Molecule
Drugs
Tyrosine Kinase
Inhibitors (TKIs)
• Pursuing internal
development
• Initial PK/PD and
tolerability demonstrated
Injectable depot
• 6 months sustained release
• Fine needle injection
• No interference with vision
• Biocompatible
• Absorbable
Rabbit PD Screening Model
Fluorescein Angiography (FA) Scores at 2, 4, 6, 9, 12 weeks
Bevacizumab-loaded fiber showed inhibition up to 12 weeks
vs. < 6 weeks from single human Avastin dose (1.25mg)
Empty Depot Control
Avastin Pre-treatment
Inhibition: Leakage score = 0
No Inhibition: Leakage: score = 4
Fluorescein leaking from vessels
Representative FA Images
Bevacizumab-loaded fiber showed continued inhibition up to 12 weeks
Sustained Pharmacodynamic Activity
17
Transforming Ophthalmic Care
with Sustained Therapies

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OPHTHALMOLOGY INNOVATION SHOWCASE - Ocular Therapeutix

  • 1. Amar Sawhney, Ph.D., CEO OIS@ASCRS New Orleans May 5, 2016 1 Transforming Ophthalmic Care with Sustained Therapies
  • 2. Forward-Looking Statements 2 This presentation contains forward-looking statements about future expectations, plans and prospects for the Company, including statements about 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 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 Company’s sustained released hydrogel depot technology, pre-commercial activities, the potential commercialization of DEXTENZA, the potential utility of any of the Company’s product candidates, the advancement of the Company's other product candidates and earlier stage pipeline, future sales of ReSure® Sealant, 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 Ocular Therapeutix’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 and DEXTENZA, 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 of the Company’s filings with the Securities and Exchange Commission, including the Company’s most recent Annual Report on Form 10-K. 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. 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 Injections Anterior Segment Sustained Release Therapies Hydrogel Sealant Addressing Diseases in the Anterior and Posterior Segment Transforming Ophthalmic Care with Sustained Therapies 3
  • 4. Compliance is Key 4 Burden of Daily Eye Drops In an observational study, 92.6% of post-cataract patients showed improper administration technique with steroid drops: • Missing eye • Instilling incorrect amount • Contaminating bottle tip • Failing to wash hands An JA, Kasner O, Samek DA, Levesque V. Evaluation of eyedrop administration by inexperienced patients after cataract surgery. J Cataract Refract Surg, 2014; 40:1857-1861 Real World Outcomes Are Not Optimal
  • 5. Platform Value Proposition 5 COMPLIANCE PHYSICIAN CONTROL PRESERVATIVE-FREE Ocular Therapeutix sustained delivery Understanding 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.
  • 6. 6 1. IMS data, March 2016 2. Roche 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 $11Bn1,2 US Sales ($Bn) Market Opportunities OTX-TP Glaucoma $ 2.7 Allergy $ 1.0 Dry eye $ 1.6 Pain & Inflammation (Anti-Inflammatories) $ 1.5 Inflammator y Conditions $ 4.1 OTX-IVT Anti-VEGF $ 4.2
  • 7. 7 7
  • 8. 8 DEXTENZA™ Product Profile: • One-time sustained release depot • Designed to replace complex topical dosing regimen • Preservative-free Initial Planned Indication: • Treatment of ocular pain associated with ophthalmic surgery Delivery Method: • Physician-administered into canaliculus following surgery • 30-day sustained and tapered delivery
  • 9. • 1st Phase 3 complete • 2nd Phase 3 enrollment complete • sNDA expected 2H 2016 Allergic Conjunctivitis DEXTENZA Label Expansion Strategy 9 PDUFA July 24, 2016 Post-Surgical Pain • Phase 3 trial currently enrolling • sNDA expected 1Q 2017 Post-Surgical Inflammation • Strong pain relief • Early onset (Day 2) • One-time seasonal prophylaxis • Reduced risk of overmedication Dual mechanism: • Build up tear level • Treat inflammation Compliance, physician control, preservative-free
  • 10. Market Research Outcomes 10 Standard of Care DEXTENZA 79% Other 21% 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. Krog and Partners, March,
  • 11. OTX-TP: Sustained Release Travoprost for the Treatment of Glaucoma 11
  • 12. Sustained Release Travoprost (OTX-TP) 12 Product Profile: • Sustained release depot • Designed to replace daily therapy • Preservative-free • Product can be monitored by patient Initial Planned Indication: • Intraocular pressure reduction for glaucoma and ocular hypertension Delivery Method: • Non-invasive • Physician-administered: inserted into canaliculus • Provides sustained delivery for up to 90 days
  • 13. • 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 acceptable – Need to demonstrate statistical superiority to placebo and clinically meaningful reduction of IOP – No 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 2016 OTX-TP Progress and Plans 13
  • 14. OTX-IVT: Sustained Release Injectable Depots for the Treatment of Retinal Diseases 14
  • 15. Posterior Segment Programs 15 Protein Therapeutics • Demonstrated: – Protein stability – Tolerability – Release profile • Seek partnership for anti-VEGF drug Small Molecule Drugs Tyrosine Kinase Inhibitors (TKIs) • Pursuing internal development • Initial PK/PD and tolerability demonstrated Injectable depot • 6 months sustained release • Fine needle injection • No interference with vision • Biocompatible • Absorbable
  • 16. Rabbit PD Screening Model Fluorescein Angiography (FA) Scores at 2, 4, 6, 9, 12 weeks Bevacizumab-loaded fiber showed inhibition up to 12 weeks vs. < 6 weeks from single human Avastin dose (1.25mg) Empty Depot Control Avastin Pre-treatment Inhibition: Leakage score = 0 No Inhibition: Leakage: score = 4 Fluorescein leaking from vessels Representative FA Images Bevacizumab-loaded fiber showed continued inhibition up to 12 weeks Sustained Pharmacodynamic Activity