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Transforming the treatment of eye diseases
with sustained release therapies
OIS @ ASCRS
April 16, 2015
Amar Sawhney, Ph.D.
Chairman, President and CEO
NASDAQ: OCUL
Forward-Looking Statements
Any statements in this presentation about future expectations, plans and prospects for the Company, including statements
about the development of the Company’s product candidates, such as plans to meet with the FDA to discuss the path
forward for OTX-DP for the treatment of post-surgical ocular inflammation and pain, the ongoing development and
potential utility of OTX-DP for post-surgical ocular inflammation and pain, the timing and conduct of the Company's Phase
2b clinical trial of OTX-TP for the treatment of glaucoma and ocular hypertension and the Company’s Phase 3 clinical trials
of OTX-DP for allergic conjunctivitis or Phase 2 clinical trials of OTX-DP for inflammatory dry eye, the advancement of the
Company's other product candidates 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, the initiation and
conduct of clinical trials, availability of data from clinical trials and expectations for regulatory 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 Annual Report on Form 10-K for the year ended December 31,
2014 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.
Products and Pipeline
Hydrogel SealantAnterior Segment
Sustained Release Therapies
Posterior Segment
Sustained Release Injections
Drug-eluting punctum plugs are investigational new drugs and not commercially available in the United States or other geographies.
Focused on the development and commercialization of innovative
therapies for diseases and conditions of the eye
Anterior Segment
Sustained Release Therapies
Product can be visualized using
a blue light and yellow filter
• Expected Benefits:
– Improves compliance
– Reduces patient burden
– Tailored and steady release specific to disease type
– Visible for retention monitoring, absorbs post therapy
– No preservatives
– Easy non-invasive placement
Market Opportunity
1. IMS data, March 2015
2. Regeneron, http://www.marketwatch.com/story/regeneron-slips-on-shortfall-in-eylea-sales-2014-11-04; Roche Media Release, Basel, 28 January 2015
Sustained Release Dexamethasone
(OTX-DP)
• Market size and dynamics
– $3.6 billion total ocular anti-inflammatory market with 26.9 million prescriptions in
the U.S. in 2014(1)
• 8.55 million prescriptions and $750 million market for single-agent steroids(1)
– Indications being developed: post-op inflammation, allergic conjunctivitis, and
inflammatory dry eye.
• Value Proposition - Strong steroid, soft delivery
– No late IOP spikes observed
– 1x administration replaces 4x/day dosing over 4-week period
– Self tapering dose
• Development Status
– Two Phase 3 studies complete for pain and inflammation. Discussions ongoing with
FDA.
– Phase 2 study completed in allergic conjunctivitis, phase 3 to start in 2H 2015
– Phase 2 study underway in inflammatory dry eye
Drug-eluting punctum plugs are investigational new drugs and not commercially available in the United States or other geographies.
1. IMS Health, March 2015
OTX-DP Program Summary
• Post-operative Inflammation and Pain Phase 3a/3b Results
– Statistically significant improvement in the reduction of inflammatory cells
in the anterior chamber of the study eye in one of the two studies
– Statistically significant reductions in pain in both trials
– Strong safety profile observed in both studies
• Positive safety data can be repackaged to support additional indications
Phase 2 Phase 3a Phase 3b
OTX-DP Placebo OTX-DP Placebo OTX-DP Placebo
Absence of AC Cells
at Day 14
34.5%* 3.4% 33.7%* 14.6% 39.4% 33.1%
Absence of Pain
at Day 8
79.3%* 31.0% 76.1%* 36.1% 77.5%* 58.8%
*Statistical superiority
achieved.
• Post-operative Inflammation and Pain Phase 3a/3b Results
OTX-DP Program Summary
Combined Phase 3a/3b Results
OTX-DP Placebo P Value
Absence of AC Cells
at Day 14
36.2% 22.7% p=0.0025
Absence of Pain
at Day 8
78.9% 50.9% p<0.0001
Phase 2 Phase 3a Phase 3b
OTX-DP Placebo OTX-DP Placebo OTX-DP Placebo
Absence of AC Cells
at Day 14
34.5%* 3.4% 33.7%* 14.6% 39.4% 33.1%
Absence of Pain
at Day 8
79.3%* 31.0% 76.1%* 36.1% 77.5%* 58.8%
*Statistical superiority
achieved.
OTX-DP Allergic Conjunctivitis
• Phase 2 Study – Day 14 Results
– OTX-DP patients demonstrated statistically significant lower ocular itching and conjunctival
redness scores than the placebo group at all three time points measured on Days 14, 28, and 42
– OTX-DP achieved a mean difference of more than 0.5 units versus placebo on a five point scale at
day 14 for both ocular itching and conjunctival redness
– OTX-DP did not achieve a mean difference compared to placebo of 1.0 unit for the majority of the
three time points measured on Day 14 for either ocular itching or conjunctival redness
• Status
– Phase 3 trial expected to commence in Q2 2015
0
1
2
3
4
Pre-CAC 3 5 7
MeanOcularItching
(0-4scale)
Time (min) Post-CAC
OTX-DP (N=35) Vehicle (N=33)
0
1
2
3
4
Pre-CAC 7 15 20
MeanConjunctivalRedness
(0-4scale)
Time (min) Post-CAC
OTX-DP (N=35) Vehicle (N=33)
Sustained Release Travoprost
(OTX-TP)
• Market size and dynamics
– $2.4 billion glaucoma market with approximately 33 million prescriptions in the U.S. in 2014(1)
• >50% were prostaglandin analogs(1)
– Glaucoma is second leading cause of blindness
– Lack of compliance with therapy is major issue
• Value Proposition
– Improved compliance
– Single-placement replaces daily drop administration up to 3 months
– 24/7 drug coverage
– Minimizes hyperemia
• Development Status
– Multiple Phase 1 and Phase 2a studies completed showing clinically meaningful IOP reduction with no
conjunctival hyperemia or epiphora
– Phase 2b multicenter U.S. study underway comparing to timolol
– Placebo plug retention studies show up to 92% retention
Drug-eluting punctum plugs are investigational new drugs and not commercially available in the United States or other geographies.
1. IMS Health, March 2015
ReSure Sealant
• First and only FDA approved
ophthalmic sealant
• Wide range of clinical applications
in cataract surgery
– Premium lens
– Femtosecond laser
– Complex cataracts
– High-risk patients
– Diabetes/autoimmune
– Alzheimer’s/dementia
Posterior Segment
Sustained Release Injections
• Market size and dynamics
– $3.5 Billion market(1) with over 2 million diagnosed in the U.S.(2)
• Value proposition:
– First in class, 6 months sustained release
– Fine needle injection
– No interference with vision
– Biocompatible
– Bioresorbable
• Current status
– Feasibility projects ongoing with pharma/biotech companies
– Demonstrated 6-month in vitro release
– Ongoing in vivo studies
solidifies post-injection
Injectable anti-VEGF depot
1. Regeneron, http://www.marketwatch.com/story/regeneron-slips-on-shortfall-in-eylea-sales-2014-11-04; Roche Media Release, Basel, 28 January 2015
2. National Eye Institute: https://www.nei.nih.gov/eyedata/amd
Sustained Release Bevacizumab
in vivo Data*
• 50μL 2.5% Bevacizumab solution (Avastin**) IVT injection
• Shows expected decreasing trend in successive tissues
• No plasma levels detected after day 1
• 50μL OTX-BI (4.3% bevacizumab) IVT injection
• Shows expected decreasing trend in successive tissues
Bolus Injection Pharmacokinetics OTX Injection Pharmacokinetics
0.1
1
10
100
1000
0 5 10 15 20 25 30
TissueBevacizumab(ug/mL)
Days in vivo
Vitreous
Retina
Choroid
Plasma
0.1
1
10
100
0 5 10 15 20 25 30
TissueBevacizumab(ug/mL)
Days in vivo
Vitreous
Retina
Choroid
*Detailed results to be presented at ARVO, May 4, 2015
**Avastin is a registered trademark of Genentech, a member of the Roche Group
Summary
• Multiple clinical indications being developed for OTX-DP
• Multiple products either approved or in late clinical
development (ReSure Sealant, OTX-DP, OTX-TP)
• Sustained delivery of anti-VEGF drugs showing promise
• Several near term milestones
• Goal of creating category dominating products across a range of
ophthalmic conditions
• Creating paradigm shift from pulsed to sustained therapies
Transforming the treatment of eye
diseases with sustained therapies
www.ocutx.com
NASDAQ: OCUL

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Ocular Therapeutix

  • 1. Transforming the treatment of eye diseases with sustained release therapies OIS @ ASCRS April 16, 2015 Amar Sawhney, Ph.D. Chairman, President and CEO NASDAQ: OCUL
  • 2. Forward-Looking Statements Any statements in this presentation about future expectations, plans and prospects for the Company, including statements about the development of the Company’s product candidates, such as plans to meet with the FDA to discuss the path forward for OTX-DP for the treatment of post-surgical ocular inflammation and pain, the ongoing development and potential utility of OTX-DP for post-surgical ocular inflammation and pain, the timing and conduct of the Company's Phase 2b clinical trial of OTX-TP for the treatment of glaucoma and ocular hypertension and the Company’s Phase 3 clinical trials of OTX-DP for allergic conjunctivitis or Phase 2 clinical trials of OTX-DP for inflammatory dry eye, the advancement of the Company's other product candidates 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, the initiation and conduct of clinical trials, availability of data from clinical trials and expectations for regulatory 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 Annual Report on Form 10-K for the year ended December 31, 2014 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. Products and Pipeline Hydrogel SealantAnterior Segment Sustained Release Therapies Posterior Segment Sustained Release Injections Drug-eluting punctum plugs are investigational new drugs and not commercially available in the United States or other geographies. Focused on the development and commercialization of innovative therapies for diseases and conditions of the eye
  • 4. Anterior Segment Sustained Release Therapies Product can be visualized using a blue light and yellow filter • Expected Benefits: – Improves compliance – Reduces patient burden – Tailored and steady release specific to disease type – Visible for retention monitoring, absorbs post therapy – No preservatives – Easy non-invasive placement
  • 5. Market Opportunity 1. IMS data, March 2015 2. Regeneron, http://www.marketwatch.com/story/regeneron-slips-on-shortfall-in-eylea-sales-2014-11-04; Roche Media Release, Basel, 28 January 2015
  • 6. Sustained Release Dexamethasone (OTX-DP) • Market size and dynamics – $3.6 billion total ocular anti-inflammatory market with 26.9 million prescriptions in the U.S. in 2014(1) • 8.55 million prescriptions and $750 million market for single-agent steroids(1) – Indications being developed: post-op inflammation, allergic conjunctivitis, and inflammatory dry eye. • Value Proposition - Strong steroid, soft delivery – No late IOP spikes observed – 1x administration replaces 4x/day dosing over 4-week period – Self tapering dose • Development Status – Two Phase 3 studies complete for pain and inflammation. Discussions ongoing with FDA. – Phase 2 study completed in allergic conjunctivitis, phase 3 to start in 2H 2015 – Phase 2 study underway in inflammatory dry eye Drug-eluting punctum plugs are investigational new drugs and not commercially available in the United States or other geographies. 1. IMS Health, March 2015
  • 7. OTX-DP Program Summary • Post-operative Inflammation and Pain Phase 3a/3b Results – Statistically significant improvement in the reduction of inflammatory cells in the anterior chamber of the study eye in one of the two studies – Statistically significant reductions in pain in both trials – Strong safety profile observed in both studies • Positive safety data can be repackaged to support additional indications Phase 2 Phase 3a Phase 3b OTX-DP Placebo OTX-DP Placebo OTX-DP Placebo Absence of AC Cells at Day 14 34.5%* 3.4% 33.7%* 14.6% 39.4% 33.1% Absence of Pain at Day 8 79.3%* 31.0% 76.1%* 36.1% 77.5%* 58.8% *Statistical superiority achieved.
  • 8. • Post-operative Inflammation and Pain Phase 3a/3b Results OTX-DP Program Summary Combined Phase 3a/3b Results OTX-DP Placebo P Value Absence of AC Cells at Day 14 36.2% 22.7% p=0.0025 Absence of Pain at Day 8 78.9% 50.9% p<0.0001 Phase 2 Phase 3a Phase 3b OTX-DP Placebo OTX-DP Placebo OTX-DP Placebo Absence of AC Cells at Day 14 34.5%* 3.4% 33.7%* 14.6% 39.4% 33.1% Absence of Pain at Day 8 79.3%* 31.0% 76.1%* 36.1% 77.5%* 58.8% *Statistical superiority achieved.
  • 9. OTX-DP Allergic Conjunctivitis • Phase 2 Study – Day 14 Results – OTX-DP patients demonstrated statistically significant lower ocular itching and conjunctival redness scores than the placebo group at all three time points measured on Days 14, 28, and 42 – OTX-DP achieved a mean difference of more than 0.5 units versus placebo on a five point scale at day 14 for both ocular itching and conjunctival redness – OTX-DP did not achieve a mean difference compared to placebo of 1.0 unit for the majority of the three time points measured on Day 14 for either ocular itching or conjunctival redness • Status – Phase 3 trial expected to commence in Q2 2015 0 1 2 3 4 Pre-CAC 3 5 7 MeanOcularItching (0-4scale) Time (min) Post-CAC OTX-DP (N=35) Vehicle (N=33) 0 1 2 3 4 Pre-CAC 7 15 20 MeanConjunctivalRedness (0-4scale) Time (min) Post-CAC OTX-DP (N=35) Vehicle (N=33)
  • 10. Sustained Release Travoprost (OTX-TP) • Market size and dynamics – $2.4 billion glaucoma market with approximately 33 million prescriptions in the U.S. in 2014(1) • >50% were prostaglandin analogs(1) – Glaucoma is second leading cause of blindness – Lack of compliance with therapy is major issue • Value Proposition – Improved compliance – Single-placement replaces daily drop administration up to 3 months – 24/7 drug coverage – Minimizes hyperemia • Development Status – Multiple Phase 1 and Phase 2a studies completed showing clinically meaningful IOP reduction with no conjunctival hyperemia or epiphora – Phase 2b multicenter U.S. study underway comparing to timolol – Placebo plug retention studies show up to 92% retention Drug-eluting punctum plugs are investigational new drugs and not commercially available in the United States or other geographies. 1. IMS Health, March 2015
  • 11. ReSure Sealant • First and only FDA approved ophthalmic sealant • Wide range of clinical applications in cataract surgery – Premium lens – Femtosecond laser – Complex cataracts – High-risk patients – Diabetes/autoimmune – Alzheimer’s/dementia
  • 12. Posterior Segment Sustained Release Injections • Market size and dynamics – $3.5 Billion market(1) with over 2 million diagnosed in the U.S.(2) • Value proposition: – First in class, 6 months sustained release – Fine needle injection – No interference with vision – Biocompatible – Bioresorbable • Current status – Feasibility projects ongoing with pharma/biotech companies – Demonstrated 6-month in vitro release – Ongoing in vivo studies solidifies post-injection Injectable anti-VEGF depot 1. Regeneron, http://www.marketwatch.com/story/regeneron-slips-on-shortfall-in-eylea-sales-2014-11-04; Roche Media Release, Basel, 28 January 2015 2. National Eye Institute: https://www.nei.nih.gov/eyedata/amd
  • 13. Sustained Release Bevacizumab in vivo Data* • 50μL 2.5% Bevacizumab solution (Avastin**) IVT injection • Shows expected decreasing trend in successive tissues • No plasma levels detected after day 1 • 50μL OTX-BI (4.3% bevacizumab) IVT injection • Shows expected decreasing trend in successive tissues Bolus Injection Pharmacokinetics OTX Injection Pharmacokinetics 0.1 1 10 100 1000 0 5 10 15 20 25 30 TissueBevacizumab(ug/mL) Days in vivo Vitreous Retina Choroid Plasma 0.1 1 10 100 0 5 10 15 20 25 30 TissueBevacizumab(ug/mL) Days in vivo Vitreous Retina Choroid *Detailed results to be presented at ARVO, May 4, 2015 **Avastin is a registered trademark of Genentech, a member of the Roche Group
  • 14. Summary • Multiple clinical indications being developed for OTX-DP • Multiple products either approved or in late clinical development (ReSure Sealant, OTX-DP, OTX-TP) • Sustained delivery of anti-VEGF drugs showing promise • Several near term milestones • Goal of creating category dominating products across a range of ophthalmic conditions • Creating paradigm shift from pulsed to sustained therapies
  • 15. Transforming the treatment of eye diseases with sustained therapies www.ocutx.com NASDAQ: OCUL