InSite Vision's (INSV) Corporate Presentation "DOUBle Study Results"

1. DOUBle Study ResultsDOUBle Study ResultsDOUBle Study ResultsDOUBle Study Results
Tim Ruane CEOTim Ruane, CEO
InSite Vision
J l 2013July, 2013

2. BlepharitisBlepharitis: Acute and/or Chronic : Acute and/or Chronic
Inflammation of the EyelidsInflammation of the Eyelids
BlepharitisBlepharitis: Acute and/or Chronic : Acute and/or Chronic
Inflammation of the EyelidsInflammation of the Eyelids
Signs &
• Redness
• Flaking skin
• Gritty sensation
• Itching
Signs &
Symptoms
g
• Crusting
• Cysts
• Irritation
g
• Vision impairment
• Discomfort
Possible
Causes
• Bacteria
• Viruses
• Allergy
E i t l diti
Blepharitis
(also known as Lid Margin Disease)
• Environmental conditions
• Systemic disease
• Estimated 34 million people in the
Prevalence
• Estimated 34 million people in the
U.S. alone
• Widely considered both
under-diagnosed and misdiagnosed
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Image Source: WebMD.com

3. Special Protocol Assessment (SPA) Approval: Special Protocol Assessment (SPA) Approval:
DOUBleDOUBle Phase 3 for Phase 3 for AzaSiteAzaSite Plus & Plus & DexaSiteDexaSite
Special Protocol Assessment (SPA) Approval: Special Protocol Assessment (SPA) Approval:
DOUBleDOUBle Phase 3 for Phase 3 for AzaSiteAzaSite Plus & Plus & DexaSiteDexaSite
DOUBle (Dual Ophthalmic agents Used in Blepharitis)
Simultaneous evaluations of AzaSite Plus, AzaSite, DexaSite and
DuraSite (vehicle)DuraSite (vehicle)
• Valuable data on our two agents ‐ and AzaSite ‐ in one Phase 3 study
Four arm Study of 900 Patients Design and Statistical Analysis PlanFour‐arm Study of 900 Patients Design and Statistical Analysis Plan
N= 907
Randomized
AzaSite Plus
N= 300
AzaSite Plus
N= 300
Randomized
Blinded
AzaSite
N= 150
AzaSite
N= 150
DexaSiteDexaSite
Powered at 80%
2‐sided Fisher’s Exact Test
DexaSite
N= 300
DexaSite
N= 300
DuraSite (vehicle)
N 150
DuraSite (vehicle)
N 150
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N= 150N= 150

4. Special Protocol Assessment (SPA) Approval: Special Protocol Assessment (SPA) Approval:
DOUBleDOUBle Phase 3 for Phase 3 for AzaSiteAzaSite Plus & Plus & DexaSiteDexaSite
Special Protocol Assessment (SPA) Approval: Special Protocol Assessment (SPA) Approval:
DOUBleDOUBle Phase 3 for Phase 3 for AzaSiteAzaSite Plus & Plus & DexaSiteDexaSite
DOUBle (Dual Ophthalmic agents Used in Blepharitis)
SPA Agreed Primary Endpoints: May 2011SPA Agreed Primary Endpoints: May 2011
Traditional Endpoint
Complete (100%) resolution of all
clinical signs & symptoms (cure)
New Endpoint #1
Time to recurrence:
Patients with complete (100%)
Other Endpoints
Improvement in clinical signs &
symptoms: All other patientsclinical signs & symptoms (cure) Patients with complete (100%)
resolution of clinical signs & symptoms
(cure)
symptoms: All other patients
Exacerbation of clinical signs &
symptoms: All other patients
A Sit PlA Sit Pl A Sit PlA Sit Pl A Sit PlA Sit Pl
vs.
AzaSite PlusAzaSite Plus
AzaSiteAzaSite
vs.
AzaSite PlusAzaSite Plus
DexaSiteDexaSite
vs.
AzaSite PlusAzaSite Plus
AzaSiteAzaSite
vs.
DexaSiteDexaSite
DuraSite (Vehicle)DuraSite (Vehicle)
vs.
DexaSiteDexaSite
DuraSite (Vehicle)DuraSite (Vehicle)
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5. DOUBle Study ResultsDOUBle Study ResultsDOUBle Study ResultsDOUBle Study Results
Screening Enrollment Safety Post‐Dose
(Day ‐7) (Day 0) (Day 8) (Day 15)
AzaSite Plus
Severe Disease 56 ( 18.4%) 51 ( 16.7%) 16 ( 5.3%) 4 ( 1.3%) ~18/80/2 % distribution of disease severity
across arms at initial screening (Day 7)Moderate Disease 244 ( 80.0%) 254 ( 83.3%) 158 ( 52.7%) 83 ( 27.8%)
Mild Disease 5 ( 1.6%) 0 123 ( 41.0%) 187 ( 62.5%)
Disease Free 0 0 3 ( 1.0%) 25 ( 8.4%)
DexaSite
across arms at initial screening (Day -7)
BID lid margin wipes & hygiene effective in <7%
of patients (Day –7 to Day 0 pre-enrollment period)
~16/84 % distribution of disease severity across
arms at initial screening (Day 0)
Severe Disease 51 ( 17.1%) 42 ( 14.1%) 16 ( 5.5%) 3 ( 1.0%)
Moderate Disease 246 ( 82.6%) 256 ( 85.9%) 168 ( 57.3%) 70 ( 24.1%)
Mild Disease 1 ( 0.3%) 0 109 ( 37.2%) 201 ( 69.1%)
Disease Free 0 0 0 17 ( 5.8%)
Clear disease severity reductions at Day 8 & Day
15 when Rx agents are combined with lid
wipes/hygiene
Disease severity reductions are statistically
significant at Day 15 for AzaSite Plus & DexaSite
AzaSite
Severe Disease 28 ( 18.1%) 30 ( 19.4%) 15 ( 9.8%) 7 ( 4.6%)
Moderate Disease 125 ( 80.6%) 124 ( 80.0%) 89 ( 58.2%) 52 ( 34.4%)
Mild Disease 2 ( 1.3%) 1 ( 0.6%) 49 ( 32.0%) 84 ( 55.6%)
significant at Day 15 for AzaSite Plus & DexaSite
Disease Free 0 0 0 8 ( 5.3%)
Vehicle
Severe Disease 30 ( 20.1%) 25 ( 16.8%) 14 ( 9.7%) 9 ( 6.3%)
Moderate Disease 115 ( 77.2%) 124 ( 83.2%) 84 ( 57.9%) 48 ( 33.3%)
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Moderate Disease 115 ( 77.2%) 124 ( 83.2%) 84 ( 57.9%) 48 ( 33.3%)
Mild Disease 4 ( 2.7%) 0 45 ( 31.0%) 80 ( 55.6%)
Disease Free 0 0 2 ( 1.4%) 7 ( 4.9%)

6. DOUBle Study ResultsDOUBle Study ResultsDOUBle Study ResultsDOUBle Study Results
Screening Enrollment Safety Post‐Dose
(Day ‐7) (Day 0) (Day 8) (Day 15)
AzaSite Plus
Severe Disease 56 ( 18.4%) 51 ( 16.7%) 16 ( 5.3%) 4 ( 1.3%) Raw Scores @ Day 15 (Post-treatment)
Moderate Disease 244 ( 80.0%) 254 ( 83.3%) 158 ( 52.7%) 83 ( 27.8%)
Mild Disease 5 ( 1.6%) 0 123 ( 41.0%) 187 ( 62.5%)
Disease Free 0 0 3 ( 1.0%) 25 ( 8.4%)
DexaSite
Overall p = 0.0059
Arm Comparisons
AzaSite Plus DexaSite Azasite
Vehicle p = 0.0298 p = 0.0116 p = 0.8476
Severe Disease 51 ( 17.1%) 42 ( 14.1%) 16 ( 5.5%) 3 ( 1.0%)
Moderate Disease 246 ( 82.6%) 256 ( 85.9%) 168 ( 57.3%) 70 ( 24.1%)
Mild Disease 1 ( 0.3%) 0 109 ( 37.2%) 201 ( 69.1%)
Disease Free 0 0 0 17 ( 5.8%)
Azasite p = 0.0152 p = 0.0055
DexaSite p = 0.6935
Groups Scores @ Day 15 (Post-treatment)
AzaSite
Severe Disease 28 ( 18.1%) 30 ( 19.4%) 15 ( 9.8%) 7 ( 4.6%)
Moderate Disease 125 ( 80.6%) 124 ( 80.0%) 89 ( 58.2%) 52 ( 34.4%)
Mild Disease 2 ( 1.3%) 1 ( 0.6%) 49 ( 32.0%) 84 ( 55.6%)
Overall p = 0.0013
Arm Comparisons
AzaSite Plus DexaSite Azasite
Vehicle p = 0.0086 p = 0.0017 p = 0.8206
A it 0 0154 0 0033
Disease Free 0 0 0 8 ( 5.3%)
Vehicle
Severe Disease 30 ( 20.1%) 25 ( 16.8%) 14 ( 9.7%) 9 ( 6.3%)
Moderate Disease 115 ( 77.2%) 124 ( 83.2%) 84 ( 57.9%) 48 ( 33.3%)
Azasite p = 0.0154 p = 0.0033
DexaSite p = 0.6282
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Moderate Disease 115 ( 77.2%) 124 ( 83.2%) 84 ( 57.9%) 48 ( 33.3%)
Mild Disease 4 ( 2.7%) 0 45 ( 31.0%) 80 ( 55.6%)
Disease Free 0 0 2 ( 1.4%) 7 ( 4.9%)

7. DOUBle Study Results SummaryDOUBle Study Results SummaryDOUBle Study Results SummaryDOUBle Study Results Summary
1st controlled study to quantify effects of BID lid margin wipes & hygiene
alone (<7%) in moderate to severe blepharitis
Acute care endpoint (Score of zero) completely missed/unachievableAcute care endpoint (Score of zero) completely missed/unachievable
AzaSite Plus & DexaSite, in combination with BID lid margin wipes &
hygiene, significantly reduce disease severity at Day 15
No safety issues
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8. Next StepsNext StepsNext StepsNext Steps
FDA briefing package submitted Friday
FDA meeting scheduled August 19th
EU Health Authority meeting(s) planned
Additional data analyses ongoing (e.g., Quality of Life, follow‐up period)
No additional investments (other than time/analysis) in blepharitis
program until further clarity is gained on current data sets from both
FDA and EU health authorities
Continue focus on BromSite confirmatory Phase 3 study enrollment
• Enrollment currently at 107/240 patients (45% complete)
Continue focus on other corporate initiatives (debt, partnering, etc.)p p g
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