PanOptica's "Wet AMD Treatment" Presentation for OIS@AAO 2015

1. A New Wave of Innovation
Paul Chaney
President and CEO
OIS@AAO
November 12, 2015

2. 2
Creating a new wave
of innovation in
wet AMD treatment
one drop at a time

3. 3
Game-Changing Opportunity:
Safe, Effective Anti-VEGF Eye Drop
Potential Benefits in Chronic Neovascular Eye Diseases
• Support life-long treatment for optimal outcomes
• Eliminate injection-related risks
• Compatible with any combination therapy
• Empower patients and caregivers

4. 4
Game-Changing Opportunity:
Safe, Effective Anti-VEGF Eye Drop
Broad Potential in Chronic Neovascular Eye Diseases
• Wet AMD
• Diabetic retinopathy (DR)
• Diabetic macular edema (DME)
• Proliferative diabetic retinopathy (PDR)
• Retinal vein occlusion (RVO)
• Prevent progression to wet AMD in high-risk patients with dry AMD

5. 5
PAN-90806:
Potent, Selective, Small-Molecule VEGFR Antagonist
Unusually Favorable Characteristics for Topical Delivery to Back of Eye
• pK supports circumferential distribution route
o Cornea > choroid > retina > aqueous > vitreous
o Reproducible findings in rabbit, dog, primate
• Excellent target tissue distribution
o Central choroid ≈ 400-2,500 fold over kD at VEGFR2
o Central retina ≈ 200-1,000 fold over kD at VEGFR2
• Concentrations sustained at 17 hours post-dose
• Extremely low systemic exposure on topical dosing
• Performs as well as IVT anti-VEGF AB in animal models
o P. Campochiaro, Johns Hopkins Univ.; J. Penn, Vanderbilt Univ.

6. 6
PAN-01-
101
Stage 1 Stage 2
# of
Patients
40 10
Treatment
PAN-90806
monotherapy
up to 8 weeks
Single injection of ranibizumab
followed by PAN-90806 up to
12 weeks
Status Recently completed
Currently enrolling (6 pts to
date)
Preliminary
Results
Reported today
4 pts completed 12 weeks
with no intravitreal rescue
Full Results Expected Q1-Q2 2016 Expected Q1-Q2 2016
Phase 1/2 Dose-Ranging Study of PAN-90806 in Treatment-Naïve Patients
with Wet AMD
Stage 1 Monotherapy Arm Completed

7. 7
Phase 1/2 Dose-Ranging Study of PAN-90806
Preliminary Results from Stage 1 Monotherapy Arm
Signals of Anti-VEGF Biological Activity Across All Dose Arms
• Results confirmed by independent panel of retina experts
• Maximum tolerated dose (MTD) established over 8 weeks
• No treatment-related systemic AEs to date
• AEs limited to reversible ocular surface findings in higher dose/frequency arms
• Ongoing safety assessment by Safety Monitoring Committee
• Full results expected Q1-Q2 2016

8. 8
Phase 1/2 Dose-Ranging Study of PAN-90806
Patient 1174 from Stage 1 Monotherapy Arm
Occult Lesion w/SRF & Moderate Thickening, Vision Loss at Baseline
Week 1 Week 2 Week 4 VA: 67 CST: 219
Week 8 VA: 71 CST: 213 1 Week Post-Tx 1 Month Post-Tx
Day 1 VA: 66 CST: 286

9. 9
Phase 1/2 Dose-Ranging Study of PAN-90806
Patient 1188 from Stage 1 Monotherapy Arm
Active Extrafoveal Classic Lesion with Significant Retinal Edema
Week 1 Week 2 Week 4 VA: 75 CPT: 282
Week 8 VA: 74 CPT: 242 1 Week Post-Tx 1 Month Post-Tx
Day 1 VA: 67 CPT: 381

10. 10
Phase 1 Dose-Ranging Study of PAN-90806 in
Patients with Proliferative Diabetic Retinopathy (PDR)
PAN-01-201
# of Patients
10 – 20 pts with
PDR with or without
DME
Treatment
PAN-90806
monotherapy up to
8 weeks
Status
Currently enrolling
(3 pts to date)
Results Q1-Q2 2016

11. 11
PAN-90806:
Advanced, Next-Generation Formulation
Strong Potential To Expand Dose Range (non-human primate study)
• Uniformly low corneal concentrations
o Decrease in ocular surface exposure by 0.75-1.5 log vs current formulation
o No dose relationship between 1.0 mg/ml and 10 mg/ml
• Enhances distribution of PAN-90806 to central choroid and central retina
o Dose dependently achieves between 200-2,000 fold over kD at VEGFR2
o ~2x-10x above concentration achieved with current formulation 1 mg/ml

12. 12
Back to the Clinic in 2016 – 2017
With a Chance To Do Something Special
Enable Amended IND with New Next-Generation Formulation
• Confirm safety, tolerability, biological response in wet AMD
o Dose-ranging monotherapy study
o Select dose/dose-range for later-stage trials
• Then demonstrate safety, efficacy and therapeutic utility in wet AMD
maintenance treatment in well-powered controlled Phase 2b study
• Expand exploratory work in relevant indications (PDR, DME, RVO)

13. 13
PanOptica Board of Directors
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.
*Observer

14. 14
Highly Capable Team Hand-Picked to Lead, Plan, Execute
Ophthalmology Drug Evaluation & Development
David P. Bingaman, DVM, PhD
Sr. Dir. & Head, Retina Development
Paul G. Chaney
President & CEO
• 35 years of industry experience
• 20 years in ophthalmology
• OSI Eyetech, Eyetech, Pharmacia
(Pfizer)
• Launches of Xalatan, Xalcom, Macugen
Kristine Curtiss
Executive Director of Clinical Operations
Lori Forrest
Executive Director of Finance, Controller
Angela Kothe, OD, PhD
Regulatory Affairs Consultant
Martin B. Wax, MD
Chief Medical Officer
EVP Product Development
• Former VP of R&D at Alcon
• Led discovery & development in glaucoma, retina,
allergy, dry eye, inflammation, anti-infectives
• Recognized glaucoma clinician-scientist with
200+ publications

15. PanOpticaPharma.com
PanOptica, Inc.
150 Morristown Rd, Suite 205
Bernardsville, NJ 07924