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Ophthalmology Innovation Summit October 2018 [Option 1] Focused on Cornea Therapeutics

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Page 1: Focused on Cornea Therapeutics - ois.net · 4 The Endothelial Opportunity FRONT OF CORNEA Epithelium •Epithelial defects lead to corneal opacity and glare and induce severe pain

Ophthalmology Innovation Summit

O c t o b e r 2 0 1 8

[Option 1]

Focused on Cornea Therapeutics

Page 2: Focused on Cornea Therapeutics - ois.net · 4 The Endothelial Opportunity FRONT OF CORNEA Epithelium •Epithelial defects lead to corneal opacity and glare and induce severe pain

2

Restoring visionlost to corneal disease using an enhanced form of naturally occurring FGF-1 proteinto regenerate corneal tissue

Corneal Disease

TTHX1114(enhanced FGF-1)

BACK OF CORNEA

Endothelial Disease(e.g. Fuchs)

FRONT OF CORNEA

Epithelial Disorder(e.g. Corneal Ulcers)

IntracameralInjection Topical

No Current Pharmacologic Treatment

Page 3: Focused on Cornea Therapeutics - ois.net · 4 The Endothelial Opportunity FRONT OF CORNEA Epithelium •Epithelial defects lead to corneal opacity and glare and induce severe pain

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InvestmentHighlights

Two first in class products to treat indications in the front and back of the cornea

Innovative treatment – No current pharmacologic therapy for corneal endothelial dystrophy or most corneal epithelial diseases

> $2.5bn combined revenue projections

Next value inflection point – Clinical PoC in Fuchs endothelial corneal dystrophy and IND in corneal surface indication

Page 4: Focused on Cornea Therapeutics - ois.net · 4 The Endothelial Opportunity FRONT OF CORNEA Epithelium •Epithelial defects lead to corneal opacity and glare and induce severe pain

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The Endothelial Opportunity

FRONT OF CORNEA

Epithelium• Epithelial defects lead to corneal

opacity and glare and inducesevere pain

• Disease process (Herpetic keratopathy) and generalized keratitis

BACK OF CORNEA

Endothelium• Fuchs corneal dystrophy and related

diseases cause a decline in vision driven by endothelial dysfunction

• Leading cause of corneal transplantation

Page 5: Focused on Cornea Therapeutics - ois.net · 4 The Endothelial Opportunity FRONT OF CORNEA Epithelium •Epithelial defects lead to corneal opacity and glare and induce severe pain

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CornealEndothelialDystrophy

Corneal Endothelial Cells(Loss of cells from organic disease or surgical trauma)

NORMAL CORNEA DISEASED CORNEA

Corneathickens

Corneabecomes opaque

Normal Worsening Diseased

TTHX1114

Normal focus Poor focus –> Poor visual acuity

Reduced Edema | Restoration of Visual Acuity

Regenerate Corneal Endothelial CellsProliferation Migration

Page 6: Focused on Cornea Therapeutics - ois.net · 4 The Endothelial Opportunity FRONT OF CORNEA Epithelium •Epithelial defects lead to corneal opacity and glare and induce severe pain

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z

Corneal Endothelial Market Opportunity: US and EU

FUCHS

~200KLate stage Fuchs

(Transplant within 24 months)

~600KTotal diagnosed/high risk

CATARACT/GLAUCOMA SURGERY-ENABLING

POPULATION

~900KMild endothelial dystrophy makes

cataract surgery risky

12 million

~4% of Populationwith Endothelial Disease

TodayCorneal transplantationonly treatment option

OpportunityPharmacologic therapy

dramaticallyincreases diagnosis and

treatment rates

Page 7: Focused on Cornea Therapeutics - ois.net · 4 The Endothelial Opportunity FRONT OF CORNEA Epithelium •Epithelial defects lead to corneal opacity and glare and induce severe pain

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0%

2%

4%

6%

8%

10%

12%

control TTHX1114

4X increase

TTHX1114 Stimulates Proliferation in Dystrophic Human Corneas

Untreated Fuchs Cornea + TTHX1114

Green = Proliferating Cells

% of Cells with EdU,

+/- sem

P = 0.0055

Page 8: Focused on Cornea Therapeutics - ois.net · 4 The Endothelial Opportunity FRONT OF CORNEA Epithelium •Epithelial defects lead to corneal opacity and glare and induce severe pain

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Endothelial Dystrophy/Fuchs Program Summary

• Proliferation, acceleration of healing of corneal endothelial cells in vitro

• Corneal clearing in rabbit cryoinjury model

• Proliferation of endothelial cells in human dystrophic (Fuchs) corneas ex vivo

Multiple validating efficacy studies

Page 9: Focused on Cornea Therapeutics - ois.net · 4 The Endothelial Opportunity FRONT OF CORNEA Epithelium •Epithelial defects lead to corneal opacity and glare and induce severe pain

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BACK OF CORNEA

Endothelium• Fuchs corneal dystrophy and related

diseases cause a decline in visiondue to corneal edema (swelling) and opacity

• Result of decreased corneal endothelial cell count

The Epithelial Opportunity

FRONT OF CORNEA

Epithelium• Epithelial defects lead to corneal

opacity and glare and inducesevere pain

• Disease process (Herpetic keratopathy) and generalized keratitis

Page 10: Focused on Cornea Therapeutics - ois.net · 4 The Endothelial Opportunity FRONT OF CORNEA Epithelium •Epithelial defects lead to corneal opacity and glare and induce severe pain

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Untreated

Treated

Topical TTHX1114 Reduces Corneal Damage

300

400

500

600

700

800

0 1 3 5 8

*

** *

*

Corneal Thickness, mean µm +/- sem

Reduction in Edema FollowingSulphur Mustard Challenge

* p < 0.05

Reduction in Keratitis in Herpetic Keratopathy

Days Days

Keratitis Score+/- sem

Drug Treatment

0

1

2

3

4

0 10 20 30

vehicle400ng/ml

**

* p < 0.05

vehicle500pg/ml25ng/ml

Page 11: Focused on Cornea Therapeutics - ois.net · 4 The Endothelial Opportunity FRONT OF CORNEA Epithelium •Epithelial defects lead to corneal opacity and glare and induce severe pain

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Revenue Overview >$2.5B (US/EU)FRONT OF CORNEA

Epithelial

$0.8B

HZO

HSV

UlcerativeKeratitis

Other

Ocular Surface

Post Cataract

Cataract

Glaucoma

Other

BACK OF CORNEAEndothelial

Fuchs Endothelial Corneal Dystrophy

Surgery-EnablingTherapy

$1.0B $0.7B

Fuchs

Other

Page 12: Focused on Cornea Therapeutics - ois.net · 4 The Endothelial Opportunity FRONT OF CORNEA Epithelium •Epithelial defects lead to corneal opacity and glare and induce severe pain

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Development Timeline and Financing Plan2019 2020 2021 2022 2023

Q1 Q2 Q3 Q4 Q1 Q2 Q3 Q4 Q1 Q2 Q3 Q4 Q1 Q2 Q3 Q4 Q1 Q2 Q3 Q4

POC studyIND enabling Phase 2b/3

IND PoC NDAEndothelial

IND

HK POCPhase 1HK IND enabling

PoCHK

NDA2025

Epithelial

Series A financing delivers Proof of Conceptin 1 indication and IND in 2nd indication

Page 13: Focused on Cornea Therapeutics - ois.net · 4 The Endothelial Opportunity FRONT OF CORNEA Epithelium •Epithelial defects lead to corneal opacity and glare and induce severe pain

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Experienced Management

David Eveleth, PhDChief Executive Officer

• 30+ years experience in ophthalmology, Allergan and Santen• Roles in finance, marketing, corporate strategy, business development• Independent advisor to numerous early stage start-up companies

Ben F. McGraw, III, PharmDExecutive Chairman

• 20+ years in biotech and pharma (Pfizer, Pharmacia, Bayer, Cortex Pharma)• Gained approval for 3 NCEs; 7 INDs / Phase IIs• Managed Pfizer ophthalmic R&D group, peak sales ~1.7 billion

• Chairman and CEO of TheraVida, Inc.• Board member Aerie Pharmaceuticals• Chairman/CEO numerous early stage companies• Previous senior executive positions at Valentis, Allergan, Marion Merrell Dow

Schalon Newton, DMChief Business Officer

Page 14: Focused on Cornea Therapeutics - ois.net · 4 The Endothelial Opportunity FRONT OF CORNEA Epithelium •Epithelial defects lead to corneal opacity and glare and induce severe pain

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Summary Two First-in-Class products Innovative regenerative approach for corneal disease

US/EU revenue potential >$2.5B Conservative market share assumptions with demographics driving growth

Efficient development plan Potential for proof of concept in two indications with two products within 3 years

Experienced Ophthalmic Management and investors

Page 15: Focused on Cornea Therapeutics - ois.net · 4 The Endothelial Opportunity FRONT OF CORNEA Epithelium •Epithelial defects lead to corneal opacity and glare and induce severe pain

[Option 1]

Focused on Cornea Therapeutics