Vision Medicines is developing two drug candidates, VM100 and VM200, to treat retinal diseases with high unmet medical need and large market opportunities. VM100 is in Phase 2/3 development for geographic atrophy and intermediate AMD, with a combined $20B market. VM200 is in preclinical development for Stargardt disease, an orphan indication with no approved therapies and a $6B market opportunity. Both drugs address the underlying disease mechanisms and have demonstrated preclinical efficacy in preserving retinal structure and function. Vision Medicines aims to develop these assets and consolidate the fragmented ophthalmology space, which has fewer products per company than other therapeutic areas.

1. Vision Medicines Inc. – Proprietary & Confidential
2015 Annual Ophthalmology Innovation Summit at AAO
Vision Medicines, Inc.
Science to See Tomorrow™
Chris Varma, PhD
Chairman & CEO
November 2015

2. Vision Medicines Inc. – Proprietary & Confidential
Vision Medicines Inc.
Focused on retina, the highest growth area in ophthalmology
Overview
• Treatments for severe retinal degenerative diseases; high unmet medical need
• Multi-billion dollar market opportunities; strong IP
• Proven management team and world-class scientific advisory board
Strategy
• Develop assets and drive consolidation in the ophthalmology marketplace
Programs
Phase 2/3 ready, VM100:
- Geographic atrophy (GA) and intermediate AMD (iAMD)
- Combined $20B market opportunity
IND-enabling studies ongoing, VM200 for Stargardt Disease (orphan disease):
- No current therapies; $6B market opportunity US/EU5/Japan
- $7.5M non-dilutive co-funding from Foundation Fighting Blindness
- Additional orphan indications: SLS, SSADH, and BEST Disease (~$1B)
2
Sources: RBC Capital Markets Research Report, Back of the Eye Preview, 12-2-14 and Vision Medicines internal research

3. Vision Medicines Inc. – Proprietary & Confidential
Vision Medicines Inc.
Pipeline addresses high unmet medical needs and
multi-billion dollar market opportunities
Product MOA Indication
Stage of Development
Market opportunity
Preclin Ph 1 Ph2 Ph 3
VM100
(mAb)
Clear
retinal
Ab &
comple-
ment
Geographic
Atrophy
Intermediate
AMD
$20B WW for GA and iAMD
- 2016 – Initiate P2/3 study
- Dark adaptation data at 3
months
- 6 mo interim data
- 2017 – PoC in GA
VM200
(small
molecule)
Alde-
hyde
trap
Stargardt
Disease
(Orphan)
SLS*
(Orphan)
SSADH*
(Orphan)
Best
Disease
(Orphan)
Stargardt Disease $6B
US/EU5/JPN
- 2016 –Initiate P1/2 study
- Autofluorescence
biomarker data at 3 months
- 2018 – PoC in Stargardt
Additional indications: SLS,
SSADH, Best Disease
~$1B WW
2018
PoC
3
Sources: RBC Capital Markets Research Report, Back of the Eye Preview, 12-2-14 and Vision Medicines internal research
2017
PoC
Phase 2/3 ready
*SLS=Sjogrren
Larson Syndrome,
SSADH=Succininc
semi-aldheyde
dehydrogenase
deficiency

4. Vision Medicines Inc. – Proprietary & Confidential
Vision Medicines Inc.
VM100: Key points of differentiation
4
• Systemic treatment
• Treats both eyes simultaneously
• Less invasive than intravitreal (IVT) injections into each eye
• No safety signal; 61 patients treated in 2 Phase 1 trials
• Potential for less frequent dosing
• Potential for every other month or quarterly dosing
• Better convenience and compliance
• Effects multiple targets
• Beta amyloid (Aβ)
• Complement
• VEGF/PEDF

5. Vision Medicines Inc. – Proprietary & Confidential
Vision Medicines Inc.
VM100: Why we believe
Target Validation
• Biogen has clinically validated Aβ as a driver of Alzheimer's disease
Mechanism
• Drusen are a biomarker for risk of progression to advanced AMD; drusen
contain toxic Aβ and activated complement
• In post-mortem studies, ~100% of eyes with high drusen load also have retinal
Aβ and Aβ oligomers are only present in eyes with drusen
• Aβ pathophysiology links to clinically-validated mechanistic pathways in
advanced AMD (wet AMD and GA)
Data
• Anti-Aβ mAbs administered systemically decrease retinal Aβ and complement
in two mouse models of dry AMD using several antibodies
• Improvement in retinal function was observed with VM100
5
Luibl et al. Journal of Clinical Investigation. 116 (2006) 378-385
Anderson et al. Experimental Eye Research. 78 (2004) 243-256
Wang et al. Journal of Immunology. 181 (2008) 712-720
Wang et al. Journal Cell Physiol. 220 (2009) 119-128.
Yoshida et al. The Journal of Clinical Investigation. 115 (2005) 2793-2800
Moreth et al. Immunity & Ageing 2013, 10:18
Johnson PNAS 99:11830 (2002) – and replicated by 3 other groups

6. Vision Medicines Inc. – Proprietary & Confidential
Vision Medicines Inc.
Goal: Treat GA and iAMD to prevent progression; $20B market opportunity
6
Intermediate AMD
Advanced AMD
(geographic atrophy)
Geographic atrophy (GA)Wet AMD
Advanced AMD
(Blinding)
~10%/year
Intermediate AMD (dry)
Sources:
EDPRG Arch Ophth 2004;122:564
AREDS Rep 18 Arch Ophth 2005;123:1570
Our focus
1 million in US
No therapies
1.2 million in US
Main treatment anti-VEGF
• 7.3 million in US
• High risk of vision loss
• No current treatments
Advanced AMD
(Blinding)
~10%/year
Our focusIntermediate AMD (iAMD)

7. Vision Medicines Inc. – Proprietary & Confidential
Vision Medicines Inc.
VM100 clears harmful Aβ and complement to preserve retinal
structure and function in GA and iAMD
7
VM100
VEGF, CFB
PEDF, CFI

8. Vision Medicines Inc. – Proprietary & Confidential
Vision Medicines Inc.
VM100 prevents Aβ and complement deposition in the aged/APOE/HFC
diet mouse model of dry AMD, preserving retinal structure and function
VMI - Proprietary &
8
Mouse model of dry AMD:
Human ApoE KI mice,
Aged > 1 year,
fed high cholesterol diet
Source: Ding PNAS 108:E279 (2011)
Preservation of function (ERG)
with VM100 (red line)
Normal diet (ND) (Red=Aβ)
High fat diet (HFC) (Red=Aβ)
HFC + VM100 (Red=Aβ)
RetinaImmunohistochemistry
VM100 prevents deposition
of Aβ
VM100 prevents deposition of
activated complement
Preserves structure Preserves function
VM100

9. Vision Medicines Inc. – Proprietary & Confidential
Vision Medicines Inc.
Phase 1a
(Single ascending dose)
Phase 1b
(Multiple ascending dose)
Design Single IV dose: 0.3, 1.0, 3.0, 10.0,
20.0, or 40 mg/kg, placebo
6 monthly IV doses: 5,10, or 15
mg/kg, or placebo
Size,
Population
N=57 total
N=36 treatment group
dry AMD patients, including GA
N=24 total
N=18 treatment group
dry AMD patients, including GA
Safety • No clinically meaningful
differences in AEs across
treatment groups.
• 1 SAE (1 mg group, unrelated):
carotid stenosis
• No deaths
• Transient, low level ADAs in 8
with no clinical sequelae
• No clinically meaningful
differences in AEs across
treatment groups.
• 2 SAE (15 mg group,
unrelated): syncope, anemia
• No deaths
• No ADA
9
VM100 safety data: 61 treated GA/iAMD patients in 2 trials with no
drug-related safety signals; Ready for Phase 2/3

10. Vision Medicines Inc. – Proprietary & Confidential
Vision Medicines Inc.
VM200: Key points of differentiation
10
• Oral, systemic drug that treats both eyes
• Non-invasive application
• Treats the whole retina
• $7.5M non-dilutive co-funding from Foundation Fighting Blindness
• Prevalence estimated to be 100K in US, EU5 and Japan
• Enantiomer of an approved drug with established safety profile
• Suitable for all stages of disease
• Directly targets toxic all-trans retinal
• Strong pre-clinical animal PoC data in Stargardt disease
• Preserves retinal structure and function in mouse model
• General aldehyde trap mechanism provides option for multiple orphan
indications including SLS and SSADH. Potential also in Best disease.

11. Vision Medicines Inc. – Proprietary & Confidential
Vision Medicines Inc.
VM200 traps toxic all-trans retinal until it can be neutralized
11
Normal state
with intact
transporter
Disease state
without
transporter
VM200
neutralizes
all-trans
retinal (toxic)
VM200Treated
VM200
VM200

12. Vision Medicines Inc. – Proprietary & Confidential
Vision Medicines Inc.
VM200 prevents retinal degeneration in the Abca4-/- Rdh8-/- mouse
model of Stargardt disease
VM200 administered to Abca4-/-/Rdh8-/- mice 2 hours prior to light damage
preserves retinal structure and function in a dose-dependent manner
12
11-cis-retinal is a biochemical marker of intact
photoreceptors
OCT is an in vivo retinal structure imaging
technique
Source: Unpublished Data from Palcziewski
Lab, Case Western Reserve University
Source: OCT data from Maeda et al. Nature
Chem Biology. Feb 2012
0
20
40
60
80
100
0.05 0.2 0.5 2
11-cisretinal(%) VM200 Dose (mg)
0
1
2
3
4
0 0.5 2
OCTScore
VM200 Dose (mg)
No damage
Max damage
No dysfunction
High
Dysfunction
VM200 preserves retinal structure VM200 preserves retinal function

13. Vision Medicines Inc. – Proprietary & Confidential
Vision Medicines Inc.
Ophthalmology therapeutic area is ripe for consolidation
13
< 2
Products /
Company
35%
2-3
Products /
Company
29%
3+
Products /
Company
36%
Disease
Category
(All Companies)
# of
Companies
# of
Products
Products Per
Company
Mix of Total
Companies
Mix of
Total
Products
Oncology 1108 5046 4.55 22.5% 34.3%
Neurology 737 2179 2.96 15.0% 14.8%
Internal Medicine 974 2586 2.66 19.8% 17.6%
Cardiology 372 968 2.60 7.6% 6.6%
Endocrinology / Diabetes / Metabolism 489 1259 2.57 9.9% 8.6%
Allergy / Immunology 513 1232 2.40 10.4% 8.4%
Ophthalmology 245 561 2.29 5.0% 3.8%
Dermatology 248 487 1.96 5.0% 3.3%
Urology 236 403 1.71 4.8% 2.7%
Mean (All Companies) 547 1,636 2.63 N/A N/A
Median (All Companies) 489 1,232 2.57 N/A N/A
Products per company breakdown
Source: RW Baird investment banking data and Vision Medicines research. Note: Selection of AMA recognized specialty areas most
relevant to pharmaceutical drugs. Neurology includes Psychiatric and Neurology for simplicity. Pediatric not included as many
pediatric drugs are repurposed from other specialty areas. Per AMA classification, Internal Medicine includes specialty areas such
as Hematology, Infectious Disease, Pulmonary, and Gastroenterology.