ophthalmology innovation showcase 1 - aerie pharmaceuticals

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Company Overview OIS @ ASCRS May 4, 2017 MLR-0035 New Investigational Drug OIS @ ASCRS 2017

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Page 1: Ophthalmology Innovation Showcase 1 - Aerie Pharmaceuticals

Company Overview

OIS @ ASCRS

May 4, 2017MLR-0035 New Investigational Drug OIS @ ASCRS 2017

Page 2: Ophthalmology Innovation Showcase 1 - Aerie Pharmaceuticals

Important Information

Any discussion of the potential use or expected success of our product candidates is subject to our product candidates

being approved by regulatory authorities.

The information in this presentation is current only as of its date and may have changed or may change in the future.

We undertake no obligation to update this information in light of new information, future events or otherwise. We are not

making any representation or warranty that the information in this presentation is accurate or complete.

Certain statements in this presentation are “forward-looking statements” within the meaning of the federal securities

laws. Words such as “may,” “will,” “should,” “would,” “could,” “believe,” “expects,” “anticipates,” “plans,” “intends,”

“estimates,” “targets,” “projects,” “potential” or similar expressions are intended to identify these forward-looking

statements. These statements are based on the Company’s current plans and expectations. Known and unknown risks,

uncertainties and other factors could cause actual results to differ materially from those contemplated by the

statements. In evaluating these statements, you should specifically consider various factors that may cause our actual

results to differ materially from any forward-looking statements. These risks and uncertainties are described more fully

in the quarterly and annual reports that we file with the SEC, particularly in the sections titled “Risk Factors” and

“Management’s Discussion and Analysis of Financial Condition and Results of Operations.” In particular, the topline

Rocket 4 data presented herein is preliminary and based solely on information available to us as of the date of this

press release and additional information about the results may be disclosed at any time. Such forward-looking

statements only speak as of the date they are made. We undertake no obligation to publicly update or revise any

forward-looking statements, whether because of new information, future events or otherwise, except as otherwise

required by law.

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Page 3: Ophthalmology Innovation Showcase 1 - Aerie Pharmaceuticals

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Aerie Current Glaucoma Products – Patented thru 2030

Pre-Clinical Research

• Rhopressa™

• Improved aqueous outflow via the trabecular meshwork

• Anti-fibrotic activity in cultured human trabecular meshwork cells

• AR-13154

• Significant lesion size reduction in wet AMD model

• Rhopressa™

(netarsudil ophthalmic solution) 0.02%

• Inhibits ROCK, NET, lowers EVP, targets diseased tissue

• NDA resubmitted in February 2017; entering launch mode

• Roclatan™

(netarsudil / latanoprost ophthalmic solution) 0.02% / 0.005%

• Fixed combination of Rhopressa™

and latanoprost

• Two Phase 3’s in process; first P3 achieved primary efficacy endpoint

Aerie – Building a Major Ophthalmic Pharmaceutical Company

Data on file OIS @ ASCRS 2017

Page 4: Ophthalmology Innovation Showcase 1 - Aerie Pharmaceuticals

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RhopressaTM Once-Daily Efficacy Performance: Summary To Date

Well researched by 200+ eye doctors in over 2,000 clinical patients

Once-daily efficacy demonstrated in 4 Phase 3 trials achieving non-inferiority vs B.I.D. timolol (Rocket 1, 2, 4) and latanoprost (Mercury 1) in subjects with baseline IOP < 25 mmHg

Stable IOP lowering through 12 months, no evidence of “IOP drift”

Initial evidence of 24-hour IOP control

Additional differentiating attributes e.g., trabecular meshwork outflow and anti-fibrotic effects

OIS @ ASCRS 2017

Page 5: Ophthalmology Innovation Showcase 1 - Aerie Pharmaceuticals

Rocket 4: RhopressaTM Achieved Non-Inferiority in the Primary

Efficacy Analysis (Baseline IOP < 25 mmHg)

Mean IOP at Each Time Point (PP) – Topline 6-Month

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• Rhopressa™

performance remained within the non-inferiority range

through 6 months++Data on File

Based on Rocket 4 Topline 6-month safety

Page 6: Ophthalmology Innovation Showcase 1 - Aerie Pharmaceuticals

Rocket 4: RhopressaTM Efficacy Stable through 6 Months

(8 AM IOP)

Baseline < 27 mmHg

Rhopressa™ (n=xx)

BL W2 W6 M3 M4 M5 M6++Data on File

Based on Rocket 4 Topline 6-month 8am IOP6

BL W2 W6 M3 M4 M5 M6

Baseline < 25 mmHgRhopressa™ (n=186) Rhopressa™ (n=240)

Page 7: Ophthalmology Innovation Showcase 1 - Aerie Pharmaceuticals

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Rhopressa™ 24-hour IOP Pilot StudyDemonstrates Effective Nocturnal Efficacy

Rhopressa™(n=8)

Baseline

(n=8)

****

*****

****

*** ***

** p<0.01

*** p<0.001

• Rhopressa™ equally effective during nocturnal and diurnal periods

• Current glaucoma medications either have no efficacy at night (beta blockers, alpha agonists) or reduced efficacy at night (PGAs, CAIs)

1 - 6

Data on File: AR-13324-CS204

1. Liu JH, et al. Am J Ophthalmol. 2004; 138:389-395. 2. Gulati V, et al. Arch Ophthalmol. 2012; 130:677-684. 3. Liu JH, et al. Ophthalmology. 2009; 116:449-454. 4. Liu JH, et al.

Ophthalmology. 2010; 117:2075-9. 5. Fan S et al. J Glaucoma. 2014; 23:276-81. 6. Liu JH, et al. Am J Ophthalmol. 2016;169:249-257.

Pre-dose

Post-dose (Day 8/9)

Page 8: Ophthalmology Innovation Showcase 1 - Aerie Pharmaceuticals

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RhopressaTM Once-Daily Safety Performance: Summary To Date

Well tolerated with no evidence of treatment-related serious or systemic AEs

Most common AE: conjunctival hyperemia, ~48% incidence

- mild for ~75% of patients and sporadic; only 10% of subjects had hyperemia at each study visit.

- 4% discontinued due to hyperemia.

20% of subjects had hyperemia at post-washout baseline prior to initiating treatment

AEs occurring in ~5-25% of subjects included: cornea verticillata, conjunctival hemorrhage (petechiae), lacrimation increased, erythema of eyelid and vision blurred

Page 9: Ophthalmology Innovation Showcase 1 - Aerie Pharmaceuticals

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RoclatanTM Achieved Statistical Superiority Over Individual

Components At All 9 Time Points

Mean IOP at Each Time Point (ITT)

***p<0.0001 vs Latanoprost and RhopressaTM

Data on File

In Mercury 1, Roclatan™ IOP lowering was 1-3 mmHg greater than

monotherapy with latanoprost or Rhopressa™

Mean IOP at Each Time Point (ITT)

Page 10: Ophthalmology Innovation Showcase 1 - Aerie Pharmaceuticals

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Mercury 1: RoclatanTM Phase 3 Responder Analysis

Day 90: % of Patients with IOP Reduced to 18 mmHg or Lower

******

***

14%

23%

32%

42%

54%

15%

25%

39%

54%

69%

33%44%

61%

71%

82%

0%

20%

40%

60%

80%

100%

≤ 14 mmHg ≤ 15 mmHg ≤ 16 mmHg ≤ 17 mmHg ≤ 18 mmHg

% o

f P

ati

en

ts

IOP on Treatment

Rhopressa™ (n=198) Latanoprost (n=223) Roclatan™ (n=200)

###

###

***p<0.0001 vs Latanoprost and RhopressaTM

###p<0.0001 vs RhopressaTM, p<0.05 vs Latanoprost

Data on file and PG324 - CS301

Page 11: Ophthalmology Innovation Showcase 1 - Aerie Pharmaceuticals

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Mercury 1: Safety/Tolerability Overview of RoclatanTM

• There were no drug-related serious or systemic adverse events

• The most common adverse event was conjunctival hyperemia with

~50% incidence*, ~80% mild on biomicroscopy

• Other ocular AEs

– AEs occurring in ~5-11% of subjects receiving RoclatanTM

included: conjunctival hemorrhage, eye pruritus, lacrimation

increased and cornea verticillata.

* Incidence of conjunctival hyperemia ~50% including baseline at ~20%

Data on file and PG324 - CS301

Page 12: Ophthalmology Innovation Showcase 1 - Aerie Pharmaceuticals

Aerie Corporate Advertisements

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