Posterior Segment Company Showcase - PanOptica at OIS@AAO 2016. Presenter: Paul Chaney, Co-Founder, President & CEO Powered by: Healthegy For more ophthalmology innovation Visit us at www.ois.net

1. Creating a new wave
of innovation in
wet AMD treatment
one drop at a time
Paul Chaney
President and CEO
OIS@AAO Chicago
October 13, 2016

2. 2
Stepping forward with confidence
PAN-90806: Topical eye drop for neovascular AMD
Clinical data and New Suspension Formulation development
• Phase 1/2 study completed in treatment naïve patients with neovascular AMD (former
solution formulation)
- Monotherapy up to 2 months (n=40)
- Maintenance therapy for 3 months following single IVT Lucentis injection (n=10)
• New suspension formulation shows significant risk reduction
- Only consistent adverse event reported: Dose-dependent reversible keratopathy noted at higher
doses in animals and humans
• Regulatory requirements established for development as maintenance therapy
• Intellectual property recognized with US Patent issued Sept. 20
• Financing closed (Third Rock, SVLS, Novo Ventures) through amended IND/CTAs
• Clinic ready 2Q 2017 with potential for financing, partnership, risk share prior to study start

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Effects observed on vascular leakage, lesion morphology, and vision
PAN-90806: Encouraging Phase 1/2 results slated for
late breaking developments at Retina Subspecialty Day
Week 8 VA: 71 CST: 213
Day 1 VA: 66 CST: 286Day 1 VA: 67 CPT: 381
Week 8 VA: 74 CPT: 242
Patient 1188 Patient 1174
Conclusions of Independent Retina
Specialist Review:
~50% of treated patients had
anti-VEGF responses that provided
patient benefit within the range of
current approved anti-VEGF therapies
and outside the expected course of
natural disease history.
PAN-90806 – a novel topical
treatment for neovascular AMD
Friday Oct 14, 4:37 pm, North Hall B
Scott W. Cousins, MD

4. 4
Physical chemistry is key:
• Aqueous solubility
• Lipophilicity
• Free drug fraction
• Sustained target tissue concentration
• Highest initial drug concentration observed transiently in cornea
• Partially inhibits signaling through a kinase receptor essential to
corneal epithelial homeostasis
• Produces reversible dose-dependent corneal keratopathy
PAN-90806: Consistent exposure to the back of the eye
regardless of formulation
Cornea >> Choroid > Retina > Aqueous > Vitreous

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PAN-90806: New suspension formulation significantly
and favorably alters the cornea pK profile for safety
Total daily exposure of topical PAN-90806 (ug)
0
10
20
30
40
50
60
0 50 100 150 200 250 300 350 400
PrimateCorneaconcentration(uM)
1hrfollowinglastdoseonday21
Former Phase 1 Solution Formulation
New Suspension Formulation
Cornea Concentration (C-max) as a Function of Total Daily Dose
on day 21 in primate study

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While maintaining consistently low, constant cornea concentrations across dose range
0
200
400
600
800
1000
1200
1400
1600
1800
2000
0
10
20
30
40
50
60
240 360 720 600
CentralChoroidConc(nM)
Corneaconcentration(µM)
Total Daily Exposure of Topical PAN-90806 (µg)
Central Choroid Conc (nM)
New Suspension Formulation
Cornea Concentration (µM)
New Suspension Formulation
IC50 at VEGFR2 = 1.27 nM
PAN-90806: New suspension formulation showed
excellent dose-dependent bioavailability at the target
tissues in the central choroid and retina
Tissue levels Cmax 1-hour post dose on day 21 as a function of
total daily dose

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PAN-90806: Primate toxicity studies suggest new
suspension formulation can favorably alter therapeutic
index with expanded safe dose range
• No adverse findings have been observed to date with new suspension
formulation in exploratory primate studies at up to 20 mg/ml for up to 28 days
• Safe PAN-90806 clinical dose range with new suspension formulation expected
to be ≥ 6-10x human MTD with previous solution formulation
• Will enable amended IND and CTAs for New Suspension Formulation 1Q 2017

8. 8
Dose-ranging as monotherapy in treatment naïve nAMD
(safety, efficacy, dose selection)
• Up to 60 pts, 3-5 dose cohorts in Europe and US
• Safety, anatomical & functional endpoints (SD-OCT, FA, VA); assess dose-
response
Select dose-range for chronic (6mo) GLP toxicity studies when approximately half of
planned patients have completed 3 months treatment
• Reduces risk for expensive / critical chronic GLP studies
• Enables IND amendment for next, later phase trial by end of this trial (1H 2018)
Demonstrate improved tolerability, higher dose range, and more
robust anti-VEGF activity and dose response to inform design of
Ph2+ maintenance trial(s)
PAN-90806: Phase 1b/2a trial in 2017 will confirm
benefit of suspension formulation as monotherapy and
inform later stage trials with data 1H 2018

9. 9
Staged development program designed to minimize
risk, achieve PoC, create value
2016
JUL AUG SEP NOVOCT DECJUN APR MAY JUNJAN FEB MAR
2017
Mfg + Release GMP BFS supplies
3mo Rabbit GLP Tox: monoRx
• Dosing - Jul’16; Audited Draft Report – early Feb ’17
3mo Primate GLP Tox: monoRx + adjunct/maintenanc
• Dosing - Jul’16; Audited Draft Report – late Jan’17 Ph1b therapeutic window: GO
GMP Ph1b supply platform: GO
Submit
IND Amend/CTAs
Ph1b PoC
open US
enrollment
EU Scientific Advice
Mtgs for Ph1b
Phase 1/2 Results
Clin Results monotx +
maint, Susp primate
tox/pK
Primate Tox & pK
EU
Clin/Re
g Prep
AAO ph1/2 pres,
OIS pres, GLP tox
in-life, Clin Supply
update
IND & CTAs
Filed, Site
Prep, Clinic
Ready Trial Initiation
US&EUClinical & Non-Clin Dev
GLP Tox initiation
EU Reg &
Investigator
Meetings
GLP Tox complete,
GMP clin supply
Campaign
Negotiations
& Diligence
Financing/Partner
Communication
Tox results
Incl histopath
In-life tox
complete

10. 10
Potential for:
• Early partnership participation (option, cost-share, financing) 1Q 2017
• NDA filings on primary efficacy analysis in 2022-25 as maintenance tx
Setting the stage for significant commercial opportunity
2016 20182017 20242019 2020 2021 2022 2023
Phase 2/3 – 285-380 patients
$25-31 Mil
Pivotal Phase 3 – 933 patients; $74 Mil
Phase 2/3 – 285-380 patients; $25-31 Mil
Phase 2/3 – 285-380 patients; $25-31 Mil
Accelerated
Devt NDA filing
2022-3
Base Case
NDA Filing 2025
*Range based on trial design, potential initiation of chronic GLP tox for ph2+ trial
Phase
1/2 – 30-50 pts
$2-2.5 Mil
$8-15M to PoC*

11. 11
Accomplished ophthalmology-experienced management
team, board of directors, and investor syndicate
Paul G. Chaney - President & CEO
• 20 years of experience in ophthalmology
• OSI Eyetech, Eyetech, Pharmacia (Pfizer)
• Launches of Xalatan, Xalcom, Macugen
Martin B. Wax, MD - Chief Medical Officer & EVP,
Product Development
• Former VP of R&D at Alcon
David P. Bingaman, DVM, PhD - Sr. Dir. & Head,
Retina Development
Kristine Curtiss - Executive Director, Clinical
Operations
Lori Forrest - Executive Director, Finance & Controller
Angela Kothe, OD, PhD - Regulatory Affairs Consultant
David Guyer, MD - CEO, Ophthotech Corporation
Colin Goddard, PhD - CEO & Executive Chair, Coferon
Bruce Peacock - Venture Partner, SV Life Sciences
Mike Ross - Managing Partner, SV Life Sciences
Kevin Starr - Partner, Third Rock Ventures
Thomas Dyrberg, MD - Senior Partner, Novo Ventures
Paul Chaney - President & CEO, PanOptica Inc.
Management Team Board of Directors

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