- The document discusses Ophthotech's development of Fovista, a potential new treatment for wet age-related macular degeneration (AMD). - Fovista is currently in Phase 3 clinical trials and aims to show superiority over anti-VEGF monotherapy by inhibiting platelet-derived growth factor (PDGF) in addition to VEGF. Topline Phase 3 data is expected in 2016. - Ophthotech has an ex-US licensing agreement with Novartis for Fovista that could provide over $1 billion in payments. Ophthotech will independently commercialize Fovista in the US if approved.

1. David R. Guyer, M.D.
Chief Executive Officer and Chairman of the Board
Developing the Next Generation of Science-driven AMD Therapies
October 2014NASDAQ: OPHT

2. Forward-Looking Statements
2
Any statements in this presentation about Ophthotech’s future expectations, plans and prospects constitute forward-
looking statements for purposes of the safe harbor provisions under the Private Securities Litigation Reform Act of
1995. Forward-looking statements include any statements about Ophthotech’s strategy, future operations and future
expectations and plans and prospects for Ophthotech, and any 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. In this presentation, Ophthotech’s forward looking
statements include statements about potential receipt of milestone payments and royalties under its ex-US licensing
and commercialization agreement, the conduct of the Fovista Phase 3 clinical program, including obtaining initial,
top-line data from the Fovista Phase 3 clinical program and seeking marketing approval for Fovista, the potential of
Fovista as a wet AMD combination therapy, the initiation of additional clinical trials for Fovista and Zimura and
obtaining data from these additional planned trials. Such forward-looking statements involve substantial risks and
uncertainties that could cause Ophthotech’s clinical development programs, future results, performance or
achievements to differ significantly from those express or implied by the forward-looking statements. Such risks and
uncertainties include, among others, those related to the initiation and conduct of clinical trials, availability of data
from clinical trials and expectations for regulatory approvals or other actions and other factors discussed in the “Risk
Factors” section contained in the quarterly and annual reports that Ophthotech files with the SEC. Any forward-
looking statements represent Ophthotech’s views only as of the date of this presentation. Ophthotech anticipates
that subsequent events and developments will cause its views to change. While Ophthotech may elect to update
these forward-looking statements at some point in the future, Ophthotech specifically disclaims any obligation to do
so except as required by law.

3. 3
Fovista®
First-in-class anti-PDGF agent for wet AMD combination therapy
Worldwide commercialization rights; multi-billion dollar market
• Phase 3 program
• On-track; Top-line data expected in 2016
• OPHT to independently commercialize Fovista in the US; ex-US partnership with
Novartis
• Demonstrated statistically significant superiority in a large (N=449) randomized,
controlled, Phase 2b trial
• 62% comparative benefit from baseline over Lucentis® monotherapy
• No imbalances in safety profile
• Potential dual MOA
• Neovascular regression
• Anti-fibrosis; New data at AAO
: Developing the Next Generation of
Science-driven AMD Therapies

4. 4
• Expanding the Pipeline
• Additional Fovista trials initiated and/or planned to address other unmet
needs for wet AMD
• Initial results starting in 2015
• Zimura™ (ARC-1905, C5 complement inhibitor) trials planned
• Phase 2/3: monotherapy for Geographic Atrophy (a form of dry AMD); no
approved therapy, multi-billion dollar market opportunity
• Phase 2: wet AMD in combination with Fovista and anti-VEGF therapy (anti-
VEGF failures)
• Management team with successful track record in the development and
commercialization of ophthalmology products
: Developing the Next Generation of
Science-driven AMD Therapies

5. Ophthotech’s Ex-US Fovista Deal with Novartis
One of the Largest Single Product Partnering Deals
• Deal totals over $1 billion in upfront and potential milestone payments
• Immediate payment and near-term milestones totaling $330 million
• Received upfront fee of $200 million
• Phase 3 enrollment-based milestones of $130 million
• Received $50 million milestone payment
• Eligible for contingent future milestones totaling up to $700 million
• Marketing approval milestones of $300 million
• Sales milestones of $400 million
• A mid-30 percent range royalty on ex- US net sales of Fovista as a stand
alone product
• Approximately equal value on ex-US co-formulated Fovista products
• Ophthotech remains responsible for existing royalties owed to third parties
5

6. Fovista: Licensing and Commercialization Strategy
Phase 3 Program Remains Unchanged
• Fovista Phase 3 program
• Ophthotech continues to lead global clinical program
• US registration managed by OPHT; collaborate on ex-US regulatory with NVS
• Fovista development strategy remains anti-VEGF agnostic
• Separate injections provide choice of preferred anti-VEGF agent in
combination with Fovista
• Delivery treatment alternatives; flexibility for physicians
• Novartis seeks co-formulation of Fovista with a NVS proprietary anti-VEGF
and pre-filled syringe for Fovista
• OPHT granted options to US rights
6

7. Fovista: Global Commercialization Strategy
Ophthotech to Build US Sales Force; Novartis Ex-US Partner
• US commercialization
• Ophthotech to independently commercialize Fovista in the US
• ~2,000 retinal specialists in the US
• Build own specialty sales/marketing team of <100
• Ex-US commercialization
• Novartis to commercialize Fovista in ex-US territories
• Far-reaching presence within international retinal community
• Extensive pricing and reimbursement experience on a country by country basis
• An unparalleled global reach; wide distribution capabilities including EU, Asia
and South America
7

8. Current Anti-VEGF Market:
Expanding Multi-Billion Dollar Opportunity
8
Annual Global Branded Anti-VEGF Sales*($Bn)
Lucentis®
**Eylea®
Sales in First 12 Months Following Launch
**Available sales data is for 13 months 10 days following launch
$800MM
$862MM
*Ophthotech internal estimate
0.0
1.0
2.0
3.0
4.0
5.0
6.0
2006 2007 2008 2009 2010 2011 2012 2013
0.4
1.2
1.8
2.3
2.9
3.8
4.8
6.1

9. VA
Letters
Lost
Monotherapy Anti-VEGF: Visual Outcome (Onset to 7 Years)
Significant Unmet Medical Need
9
Vision: Pre-Wet AMD Diagnosis
At Presentation
0
-7
-15
-3
2- 3 Years
• Majority do not gain significant vision
• ~20-30% lose additional vision
After 4-7 Years
• No improvement in vision compared to
acute visual loss at presentation
UNMET MEDICAL NEED
PERSISTENT VISUAL LOSS
Ophthalmology 2013; 120-2292-2299

10. VA
Letters
Lost
Monotherapy Anti-VEGF: Visual Outcome (Onset to 7 Years)
Significant Unmet Medical Need
10
Vision: Pre-Wet AMD Diagnosis
At Presentation
0
-7
-15
-3
2- 3 Years
• Majority do not gain significant vision
• ~20-30% lose additional vision
After 4-7 Years
• No improvement in vision compared to
acute visual loss at presentation
UNMET MEDICAL NEED
PERSISTENT VISUAL LOSS
Fovista*
Phase 3 Program Ongoing
* In combination with standard of care
Wet AMD Anti-
VEGF Treatment
Failure Trials
Wet AMD Treatment
Burden Reduction
Trial
Wet AMD Prevention of
Long Term Visual Loss
(Subretinal Fibrosis)
Trials
Ophthalmology 2013; 120-2292-2299

11. Fovista (1.5mg anti-PDGF) Combination Therapy:
Potential Dual Mechanism of Visual Benefit
11
0"
2"
4"
6"
8"
10"
12"
wk"0"
wk"4"
wk"8"
wk"12"
wk"16"
wk"20"
wk"24"
Neovascular++
Regression*+
An23Fibrosis+
Reduced+Neovascular+
Growth*+
BASELINE+ WK+24+
BASELINE+ WK+24+
1.5mg+Fovista/Lucen2s"
Lucen2s"
Neovascular Regression!Neovascular+Regression+
BASELINE+ WK+24+
BASELINE+ WK+24+1.5mg+Fovista/Lucen2s+
Lucen2s+
An23Fibrosis+
* Company hypothesis with respect to MOA

12. Fovista Phase 3 Program in Wet AMD
• Strategy – replicate Phase 2b – two trials vs. Lucentis®
– Inclusion/exclusion criteria consistent with Phase 2b study
– Endpoint: Mean change in visual acuity from baseline at 1-year
• Third trial; combination with Eylea® or Avastin®
• Phase 3 – On track
• Top-line Phase 3 data – expected 2016
12
Overview

13. Fovista Phase 3 Program in Wet AMD
13
Fovista 1.5mg + Lucentis (N=311) Consistent w/Lucentis dosing schedule
Q4W approved in EU*
Lucentis 0.5mg (N=311) Consistent w/Lucentis dosing schedule
Q4W approved in EU*
Trial 1
N = 622
Year 1
Fovista 1.5mg + Lucentis (N=311) Q8W (pre-specified PRN therapy during
Q4W non-treating months)
Lucentis 0.5mg (N=311) Q8W (pre-specified PRN therapy during
Q4W non-treating months)
Fovista 1.5mg + Avastin 1.25mg /Eylea 2mg (N=311) Avastin: Q4W / Eylea: Q8W
Avastin Q4W / Eylea Q4W for 3 months, then Q8W
Avastin 1.25mg / Eylea 2mg (N=311) Avastin Q4W / Eylea Q8W
Avastin Q4W / Eylea Q4W for 3 months, then Q8W
Primary Endpoint: Mean Change in Visual Acuity from Baseline at 1-Year
PrimaryEndpoint–1-Year
Year 2
Trial 2
N = 622
Trial 3
N = 622
*Approved 2013 label

14. Early and Sustained Improvement Over Time
14
0
3
6
9
12
wk 0 wk 4 wk 8 wk 12 wk 16 wk 20 wk 24
MeanChangeVA(Letters)
Fovista 1.5mg
(Anti-PDGF) + Lucentis®
Fovista 0.3mg
(Anti-PDGF) + Lucentis®
Lucentis
0.5mg Lucentis 0.3mg Fovista + Lucentis 1.5mg Fovista + Lucentis
Intent to Treat population using Last Observation Carried Forward (ITT LOCF)
Classic dose response curve
Phase 2b study

15. Fovista Phase 2b: Fovista (1.5mg Anti-PDGF)
Combination Met the Pre-Specified Primary Endpoint
15
Note
1. The Hochberg procedure.
2. Not statistically significant.
10.6
8.8
6.5
0.0
2.0
4.0
6.0
8.0
10.0
12.0
1.5mg Fovista™ + Lucentis® 0.3mg Fovista™ + Lucentis® Lucentis®
MeanChangeVA(Letters)
Mean Change in VA (Baseline to Week 24)
P = 0.0190 (1)
1.5mg
Fovista
+
Lucentis
0.3mg
Fovista
+
Lucentis
Lucentis
N=151 N=147 N=147
(2)
62% Comparative Benefit from Baseline Over Monotherapy Lucentis®
Intent to Treat population using Last Observation Carried Forward (ITT LOCF)

16. Fovista Phase 2b: No Imbalances in Safety Profile
16
# of Patients (%)
Monotherapy
Lucentis®
N = 148
0.3mg Fovista
+ Lucentis®
N = 149
1.5mg Fovista
+ Lucentis®
N = 152
Ocular Serious Adverse Events – Eye Disorders 1 (0.7%) 1 (0.7%) 1 (0.7%)
Corneal Erosion 0 (0.0%) 0 (0.0%) 1 (0.7%)
Uveitis 0 (0.0%) 1 (0.7%) 0 (0.0%)
Visual Acuity Reduced 1 (0.7%) 0 (0.0%) 0 (0.0%)
Patients With ≥ 1 Systemic SAE 11 (7.4%) 13 (8.7%) 9 (5.9%)
MedDRA System Organ Class (1)
Cardiac Disorders 2 (1.4%) 2 (1.3%) 2 (1.3%)
Gastrointestinal Disorders 1 (0.7%) 2 (1.3%) 3 (2.0%)
Infections 1 (0.7%) 2 (1.3%) 0 (0.0%)
Musculoskeletal Disorders 1 (0.7%) 0 (0.0%) 2 (1.3%)
Neoplasms 3 (2.0%) 3 (2.0%) 1 (0.7%)
Nervous System Disorders 3 (2.0%) 1 (0.7%) 0 (0.0%)
Respiratory Disorders 0 (0.0%) 3 (2.0%) 2 (1.3%)
Any APTC Event(2) 3 (2.0%) 1 (0.7%) 0 (0.0%)
Non-Fatal Myocardial Infarction 0 (0.0%) 0 (0.0%) 0 (0.0%)
Non-Fatal Stroke 2 (1.4%) 1 (0.7%) 0 (0.0%)
Vascular Death 1 (0.7%) 0 (0.0%) 0 (0.0%)
1. Data are listed only for system organ classes with 3 or more events.
2. Antiplatelet Trialists’ Collaboration
3. Table omits patients with one or more systemic adverse events

17. Increased Reduction in CNV Size in Small and Large
Baseline CNV in Fovista (1.5mg) Combination Arm
17
Decrease in Neovascular Size
1.5mg Fovista + Lucentis 0.5mg Lucentis
Mean Baseline CNV Size = 1.62 DA
-0.12
-0.06
-0.12
-0.09
-0.06
-0.03
0.00
ChangeinCNVSize:Disc
Area(DA)
-1.73
-1.59
-2.4
-2.0
-1.6
-1.2
-0.8
-0.4
0.0
N=104 N=84 N=43 N=60Small CNV ≤ 1.62 DA Large CNV >1.62 DA
Ad-hoc analysis; not pre-specified
-0.10
-0.01
-0.12
-0.10
-0.08
-0.06
-0.04
-0.02
0.00
-2.33
-0.24
-2.4
-2.0
-1.6
-1.2
-0.8
-0.4
0.0
Week 24
Mean Baseline CNV Size = 1.62 DA
Small CNV ≤1.62 DA Large CNV >1.62 DA
Visual Outcome: >3-linesVisual Outcome: ALL Patients
N=104 N=84 N=43 N=60
N=43 N=32 N=10 N=10
Week 24
Phase 2b study

18. Increased Resolution of Subretinal Hyper-Reflective Material
18
Ad-hoc analysis; not pre-specified
1.5mg Fovista / Lucentis 0.5mg Lucentis
Baseline
Presence of
Subretinal Hyper-Reflective Material
Week 24
Absence of
Subretinal Hyper-Reflective Material
92.8% 93.2%
1.5mg Fovista
+
Lucentis
Lucentis
32.4%
21.5%
0%
10%
20%
30%
% Patients
All Patients
Fovista (1.5mg Anti-PDGF) Combination
Phase 2b study

19. Potential Dual Mechanism of Action
of Fovista
19
Pericyte
• Neovascular regression
• Anti-fibrosis

20. Anti-PDGF Therapy as an Anti-Fibrotic Agent
• PDGF is a mediator of fibrosis in multiple pre-
clinical models of organ fibrosis and retinal scarring
• Fovista monotherapy was shown to be anti-fibrotic
in a preclinical model of retinal scarring and
fibrosis
! Akiyama et al., (2006) Intraocular injection of an aptamer that binds PDGF-B: a
potential treatment for proliferative retinopathies: J Cell Physiol. May;207(2):407-12
20

21. Subretinal Fibrosis is the End Stage of Wet AMD
• Urgent, unmet medical need -
prevention of fibrosis in wet AMD
• Fibrotic scarring of the retina is
observed in up to 45% of
patients on monotherapy
Lucentis® by 2 years*
• Fibrotic scarring - biomarker for
predicting poor final visual acuity
21
*Daniel, E., et al. Comparison of Age-related Macular Degeneration Treatments Trials Research, G. (2013). Risk of Scar in the Comparison of Age-related Macular Degeneration Treatments Trials.
Ophthalmology.
*Bloch, S. B., et al. (2013). Subfoveal fibrosis in eyes with neovascular age-related macular degeneration treated with intravitreal ranibizumab. Am J Ophthalmol, 156(1), 116-124 e111

22. National Eye Institute’s Comparison of Age-related
Macular Degeneration Treatments Trials (CATT)
22
Risk of Scar in the Comparison of Age-related
Macular Degeneration Treatments Trials
Ebenezer Daniel, MBBS, PhD,1 Cynthia A. Toth, MD,2 Juan E. Grunwald, MD,1 Glenn J. Jaffe, MD,2 Daniel F. Martin, MD,3 Stuart L. Fine, MD,4 Jiayan Huang, MS,1
Gui-shuang Ying, MD, PhD,1 Stephanie A. Hagstrom, PhD,3 Katrina Winter, BS,2 Maureen G. Maguire, PhD,1 for the Comparison of Age-related Macular
Degeneration Treatments Trials Research Group*"
December 2013 by the American Academy of Ophthalmology
Quote Regarding Fovista (Anti-PDGF ) in CATT Trial
“The CATT included eyes treated only with anti-VEGF monotherapy. However, in a
recent phase II study, eyes were treated with ranibizumab, with or without anti-platelet-
derived growth factor therapy (Dugal PU, Reichel E, Boyer DS, et al. Phase 2b trial
results show effectiveness of combination therapy. Retina Times. Fall 2012).The results
showed that combination therapy more effectively than monotherapy improved visual
acuity and better eliminated SHRM. In the present study, we found that SHRM was
associated with scar formation. Accordingly, in the future, it will be of interest to
determine whether combination therapy similarly reduced the rate of fibrotic or
nonfibrotic scar as one explanation for the better visual acuity observed in that study.”

23. BASELINE WK 24
Lucentis Monotherapy
BASELINE WK 24
Fovista + Lucentis
Combination

24. Breaking News… AAO Abstract*
Masked retrospective analysis by independent reading center of fundus
images at baseline and 24 weeks in a subset (70 eyes) from the Fovista
Phase 2b study with >0 ETDRS letter loss.
• Mean change in fibrosis: 0.97 vs. 2.0 (P = 0.003)
• 27% of eyes (Fovista + Lucentis) vs. 54% (Lucentis
monotherapy) had ≥ 2 step worsening of fibrosis (2x
difference)
• In eyes with no fibrosis, 10% (Fovista + Lucentis) vs.
51% (Lucentis monotherapy) developed fibrosis
24
* Chakravarthy U, Jaffe GJ, Dual Antagonism of Platelet Derived Growth Factor (Fovista 1.5mg) and Vascular Endothelial Growth Factor (Lucentis 0.5mg) Results in Reduced Sub-retinal
Fibrosis and Neovascular Growth. Paper presentation at the 2014 American Academy of Ophthalmology Annual Meeting, October 21, 2014. Abstract PA092.

25. Subfoveal Fibrosis Develops at ~ 3 to 6 months
Following Initiation of Anti-VEGF Therapy
25
Kaplan-Meier fibrosis-specific curves!
Bloch SB et al. Am J Ophthalmol 2013;156:116–124!
All Forms of Subfoveal Fibrosis Subfoveal Fibrosis with Retinal Atrophy
Neovascular AMD!

26. Fovista (1.5mg anti-PDGF) Combination Therapy:
Potential Dual Mechanism of Visual Benefit
26
0"
2"
4"
6"
8"
10"
12"
wk"0"
wk"4"
wk"8"
wk"12"
wk"16"
wk"20"
wk"24"
Neovascular++
Regression*+
An23Fibrosis+
Reduced+Neovascular+
Growth*+
BASELINE+ WK+24+
BASELINE+ WK+24+
1.5mg+Fovista/Lucen2s"
Lucen2s"
Neovascular Regression!Neovascular+Regression+
BASELINE+ WK+24+
BASELINE+ WK+24+1.5mg+Fovista/Lucen2s+
Lucen2s+
An23Fibrosis+
* Company hypothesis with respect to MOA

27. Pipeline (Parallel Trials to Fovista Phase 3)
Data Events Expected to Begin 2015*
27
*Data event dependent on timing of study initiation and enrollment rate.
Fovista
Other wet AMD trials
- Anti-fibrosis
- Reduced treatment burden
- Anti-VEGF treatment non-responders
Proliferative Vitreoretinopathy
Von Hippel-Lindau – NEI
Zimura
Wet AMD (Anti-VEGF failures due to complement
mediated inflammation)
Expanding the Fovista Franchise (Fovista Expansion Studies)
Anti Complement C5 Program for AMD
Dry AMD (Geographic Atrophy)

28. Ophthotech’s Anti-Complement Dry AMD (GA)
Program
• OPHT’s Zimura hits C5 pathway
• Phase 2a trial with Zimura monotherapy completed
• Recent third party data supports complement
inhibition as a target in dry AMD (GA)
• Phase 2/3 planning underway
• All worldwide commercialization rights retained for
Zimura
28

29. Financing Overview
• Cash Resources
– $452.5M cash, cash equivalents and marketable securities as of 6/30/14
• $125M royalty purchase financing led by Novo A/S in May 2013; Two of three equal
tranches have been received totaling $83.4M (May 2013 and January 2014) with
$41.7M potential funding remaining
• Completed $192 Million IPO in September 2013
• Completed $59.9M follow-on public offering in February 2014
– Ex-US licensing and commercialization agreement with Novartis in May 2014
• Over $1 Billion, inclusive of upfront fee and potential milestone payments
• Immediate payment and near-term milestone totaling up to $330M
• Received an upfront fee of $200M
• Enrollment-based milestones of up to $130M ($50M received Sept. 2014)
• Milestone due to third-party as a result of entry into agreement and potential tax
implications
29

30. Strong Senior Management with Significant
Ophthalmology Domain Experience
30
Name / Title Experience
David Guyer, MD
CEO, Chairman and Co-founder
Eyetech Pharmaceuticals – Co-Founder, CEO
SV Life Sciences – Partner
NYU School of Medicine – Professor and Chairman, Department of
Ophthalmology
Samir Patel, MD
President, Vice-Chair and Co-founder
Ophthotech – CEO
Eyetech Pharmaceuticals – Co-Founder, CMO
University of Chicago – Director of Retina Service. Univ of Chicago
Michael G. Atieh
EVP, Chief Financial & Business Officer
Merck – SVP Merck Medco Managed Care / Various Senior Level Positions /
Treasurer
OSI Pharmaceuticals – EVP & Chief Financial Officer
Eyetech Inc. – Executive Chairman
Todd N. Smith
SVP, Chief Commercial Officer
Horizon Pharma – EVP, Chief Commercial Officer
Bayer, Abbott, Agouron and Fenwal Inc. – Various Senior Level Marketing,
Strategy & Commercial Positions

31. 31
Fovista®
First-in-class anti-PDGF agent for wet AMD combination therapy
Worldwide commercialization rights; multi-billion dollar market
• Phase 3 program
• On-track; Top-line data expected in 2016
• OPHT to independently commercialize Fovista in the US; ex-US partnership with
Novartis
• Demonstrated statistically significant superiority in a large (N=449) randomized,
controlled, Phase 2b trial
• 62% comparative benefit from baseline over Lucentis® monotherapy
• No imbalances in safety profile
• Potential dual MOA
• Neovascular regression
• Anti-fibrosis; New data at AAO
: Developing the Next Generation of
Science-driven AMD Therapies

32. 32
• Expanding the Pipeline
• Additional Fovista trials initiated and/or planned to address other unmet
needs for wet AMD
• Initial results starting in 2015
• Zimura™ (ARC-1905, C5 complement inhibitor) trials planned
• Phase 2/3: monotherapy for Geographic Atrophy (a form of dry AMD); no
approved therapy, multi-billion dollar market opportunity
• Phase 2: wet AMD in combination with Fovista and anti-VEGF therapy (anti-
VEGF failures)
• Management team with successful track record in the development and
commercialization of ophthalmology products
: Developing the Next Generation of
Science-driven AMD Therapies