8-K

UNITED STATES

SECURITIES AND EXCHANGE COMMISSION

WASHINGTON, D.C. 20549

FORM 8-K

CURRENT REPORT

Pursuant to Section 13 or 15(d)

of the Securities Exchange Act of 1934

Date of Report (Date of earliest event reported): September 21, 2016

Aerie Pharmaceuticals, Inc.

(Exact name of registrant as specified in its charter)

Delaware	001-36152	20-3109565
(State or other jurisdiction of incorporation)	(Commission File Number)	(I.R.S. Employer Identification Number)

2030 Main Street, Suite 1500

Irvine, California 92614

(Address of principal executive offices) (Zip code)

Registrant’s telephone number, including area code: (949) 526-8700

Check the appropriate box below if the Form 8-K filing is intended to simultaneously satisfy the filing obligation of the registrant under any of the following provisions (see General Instruction A.2. below):

¨ Written communications pursuant to Rule 425 under the Securities Act (17 CFR 230.425)

¨ Soliciting material pursuant to Rule 14a-12 under the Exchange Act (17 CFR 240.14a-12)

¨ Pre-commencement communications pursuant to Rule 14d-2(b) under the Exchange Act (17 CFR 240.14d-2(b))

¨ Pre-commencement communications pursuant to Rule 13e-4(c) under the Exchange Act (17 CFR 240.13e-4(c))

Item 7.01. Regulation FD Disclosure.

Subsequent to June 30, 2016, Aerie Pharmaceuticals, Inc. (the “Company”) issued and sold a total of 4,067,733 shares of common stock under separate at-the-market sales agreements with RBC Capital Markets, LLC and Cantor Fitzgerald & Co. and received net proceeds of approximately $94.3 million, after deducting fees and other offering expenses. In addition, the Company issued and sold 2,542,373 shares of common stock pursuant to the underwriting agreement, dated September 15, 2016, between the Company and Cantor Fitzgerald & Co., and received net proceeds of approximately $71.3 million, after deducting underwriting discounts and commissions and other offering expenses.

After giving effect to the foregoing issuances of shares and the net proceeds therefrom, the Company expects total shares outstanding and cash and cash equivalents as of September 30, 2016 to be approximately 33 million shares and $250 million, respectively.

Based upon current business plans, the Company has updated full-year 2016 cash burn guidance to approximately $85 million, an increase from the previous guidance of $75 million. The updated guidance reflects new initiatives such as start-up efforts for clinical trials in Japan and commencement of construction of a manufacturing plant in Ireland. With this updated full-year 2016 cash burn guidance, the Company expects cash and cash equivalents to total approximately $230 million as of December 31, 2016.

From time to time after September 21, 2016, representatives of the Company may present to various investors the information described in the slides attached to this report as Exhibit 99.1 hereto, which is hereby incorporated by reference into this Item 7.01.

The information in this Item 7.01 (including Exhibit 99.1) is being furnished, not filed, pursuant to Regulation FD. Accordingly, the information in this Item 7.01 will not be incorporated by reference into any registration statement filed by the Company under the Securities Act of 1933, as amended, unless specifically identified therein as being incorporated therein by reference. The furnishing of the information in this Item 7.01 is not intended to, and does not, constitute a determination or admission by the Company that this information is material or complete, or that investors should consider this information before making an investment decision with respect to any security of the Company.

Forward-Looking Statements

This report contains forward-looking statements for purposes of the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. The Company may, in some cases, use terms such as “predicts,” “believes,” “potential,” “proposed,” “continue,” “estimates,” “anticipates,” “expects,” “plans,” “intends,” “may,” “could,” “might,” “will,” “should,” “exploring,” “pursuing” or other words that convey uncertainty of future events or outcomes to identify these forward-looking statements. Forward-looking statements include statements regarding the Company’s intentions, beliefs, projections, outlook, analyses or current expectations concerning, among other things: the success, timing and cost of its ongoing and anticipated preclinical studies and clinical trials for its current product candidates, including statements regarding the timing of initiation and completion of the studies and trials; its expectations regarding the clinical effectiveness of its product candidates and results of its clinical trials; the timing of and its ability to request, obtain and maintain U.S. Food and Drug Administration or other regulatory authority approval of, or other action with respect to, its product candidates; its expectations regarding the commercialization of its product candidates; its expectations related to the share issuances discussed in this report, including the expected use of proceeds therefrom and its expected total shares outstanding and cash and cash equivalents after giving effect to these share issuances; its updated full-year 2016 cash burn guidance, the new initiatives reflected herein and expected cash and cash equivalents as of December 31, 2016; the potential advantages of its product candidates; and its plans to pursue development of its product candidates for additional indications and other therapeutic opportunities. By their nature, forward-looking statements involve risks and uncertainties because they relate to events, competitive dynamics and industry change, and depend on regulatory approvals and economic circumstances that may or may not occur in the future or may occur on longer or shorter timelines than anticipated. The Company discusses many of these risks in greater detail under the heading “Risk Factors” in the quarterly and annual reports that it files with the Securities and Exchange Commission. Forward-looking statements are not guarantees of future performance and the Company’s actual results of operations, financial condition and liquidity, and the development of the industry in which it operates may differ materially from the forward-looking statements contained in this report. Any forward-looking statements that the Company makes in this report speak only as of the date of this report. The Company assumes no obligation to update its forward-looking statements whether as a result of new information, future events or otherwise, after the date of this report.

Item 9.01. Financial Statements and Exhibits.

(d) Exhibits.

The following exhibit relating to Item 7.01 shall be deemed to be furnished, and not filed:

99.1 Company Presentation dated September 21, 2016.

SIGNATURES

Pursuant to the requirements of the Securities Exchange Act of 1934, the registrant has duly caused this report to be signed on its behalf by the undersigned hereunto duly authorized.

	AERIE PHARMACEUTICALS, INC.
Date: September 21, 2016	By:	/s/ Richard J. Rubino
		Richard J. Rubino
		Chief Financial Officer

EXHIBIT INDEX

Exhibit	Description
99.1	Company Presentation dated September 21, 2016.

EX-99.1

Company Overview

September 21, 2016

Exhibit 99.1

2

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. In addition, any discussion of clinical trial results for Rhopressa™ (netarsudil

ophthalmic solution) 0.02% relate to the results in its first Phase 3 registration trials, Rocket 1 and Rocket 2, and for

Roclatan™ (netarsudil/latanoprost ophthalmic solution) 0.02%/0.005% relate to the results in its Phase 3 registration trial.

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, including the updated guidance presented herein, 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. In particular, the topline Mercury 1

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

additional information about the results may be disclosed at any time, including at our Investor Day on October 5, 2016. In

addition, the preclinical research discussed in this presentation is preliminary and the outcome of such preclinical studies

may not be predictive of the outcome of later trials. Any future clinical trial results may not demonstrate safety and efficacy

sufficient to obtain regulatory approval related to the preclinical research findings discussed in this presentation. 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.” 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.

3

Aerie Late Stage IOP–Lowering Products

Pre-Clinical Research

•

Rhopressa™

•

Potential for disease modification and neuroprotection

•

AR-13154

•

Significant

lesion size reduction in wet AMD

•

Drug Delivery -

Front and back of the eye

•

Rhopressa™ (netarsudil ophthalmic solution) 0.02%

•

Inhibits ROCK, NET, lowers EVP, targets diseased tissue

•

NDA filed Q3 2016

•

Roclatan™ (netarsudil/latanoprost

ophthalmic solution) 0.02%/0.005%

•

Fixed combination of Rhopressa™ and latanoprost

•

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

Aerie –

Building a Major Ophthalmic

Pharmaceutical Company

Data on file

FY 2015 U.S. Glaucoma Market = $2.5B; 34M TRx

Market Share in TRx

PGA Market

Non-PGA Market

2015 US GLAUCOMA MARKET

Once Daily

2-3 Times Daily

Bimatoprost

Travoprost

Latanoprost

BB

Fixed Combo

AA

CAI

PGA: Prostaglandin Analogue; BB: Beta Blocker; AA: Alpha Agonist; CAI: Carbonic Anhydrase Inhibitor

Source: IMS MIDAS. IMS NPA

13%

15%

8%

10%

9%

35%

4

8%

5

Rhopressa™

and Roclatan™

Expected Aerie Market Positioning

Commercialization

Strategy

–

Rhopressa

TM

Now

Entering

Launch

Mode

North America : ~100 sales reps targeting ~10,000 high prescribers

Europe and Japan: Currently exploring opportunities

Rhopressa™

Roclatan™

Potential drug of choice as

adjunctive therapy to PGAs

when additional IOP lowering is

desired

Potential drug of choice for

patients requiring maximal IOP

lowering

6

Rhopressa™ Trials for Q3 2016 NDA Submission

“Rocket 1”

90-Day Efficacy

Registration Trial

U.S.

Rhopressa™ 0.02% QD

~200 patients

timolol BID

~200 patients

(Total enrollment: 411 patients)

“Rocket 2”

One Year Safety

(3 Mo. Interim

Efficacy)

Registration Trial

U.S.

Rhopressa™ 0.02% QD

~230 patients

Rhopressa™ 0.02% BID

~230 patients

timolol BID

~230 patients

(Total enrollment: 756 patients)

ClinicalTrials.gov Identifier: NCT02207491, NCT02207621

7

Rhopressa™ Trials for EU Filing;

Not Needed for NDA Filing

ClinicalTrials.gov Identifier: NCT02246764, NCT02558374

“Rocket 4”

3 Month Efficacy

6 Month Safety

Rhopressa™ 0.02% QD

~350 patients

timolol BID

~350 patients

“Rocket 3”

One Year Safety 

Trial  Canada

Rhopressa™ 0.02% QD

~90 patients

Rhopressa™ 0.02% BID

~90 patients

timolol BID

~60 patients

8

Rocket 2 and Rocket 1 Performance

Maximum Baseline IOP <25 mmHg

Rocket 2

Rocket 1

Data on file

9

Rocket 2: 12 Month 8am IOP Efficacy

Safety Population, Completed Patients (<27 mmHg)

Difference in 8am mean IOP from

Day 90 (Month 3) to Month 12:

Rhopressa

TM

QD:

Full population: +0.1 mmHg

Subset analyses: -0.3 to +0.3 mmHg

Timolol

BID:

Full population: +0.5 mmHg

Subset analyses: +0.1 to +1.4 mmHg

Day 90

Month 12

AR-13324 0.02% q.d. (n=118)

BL

W2

W6

M3

M6

M9

M12

Data on file:  AR-13324 –

CS302

10

Rocket 2: Safety/Tolerability Overview of

Rhopressa

TM

QD (Interim 12-Month)

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

**

The

updated

term

for

these

deposits

based

on

recent

MedDRA

coding

revisions

There

were

no

drug-related

serious

adverse

events

(SAEs)

No

new

adverse

events

introduced

over

the

twelve-month

period

The

most

common

adverse

event

was

conjunctival

hyperemia

with

~50%

incidence*,

the

majority

mild

Other

ocular

AEs

AEs

occurring

in

~5-23%

of

patients

included:

conjunctival

hemorrhage,

corneal

deposits

(verticillata**),

blurry

vision

and

reduced

visual

acuity

11

Rhopressa

Adverse Events Summary

Data on File:  AR-13324-CS302

* The

updated

term

for

these

deposits

based

on

MedDRA

coding

revisions

Hyperemia –

absent or sporadic for 90% of patients                                        

-

only 10% of patients had hyperemia AE at all 6 study

Conjunctival

Hemorrhage

–

sporadic

subconjunctival

petechiae

-

none

noted at month 12 visit       

Corneal

Deposits

(verticillata*)

–

asymptomatic

non-toxic

lipid

deposits      

-

high resolution rate         

Visual

Acuity

Reduced

–

sporadic,

mostly

single

visit,

only

one

eye        

-

incidence reduced over time

Vision

Blurred

–

sporadic

and

significantly

reduced

over

12

months

TM

visits

12

When Present, 80% of Rhopressa

QD

Hyperemia Graded as Mild

Grade

Image

Description

0

None/Normal

1

Mild

2

Moderate

3

Severe

Data on File: AR-13324

–

CS302

For illustrative purposes only

TM

13

Conjunctival Hemorrhage Using Biomicroscopy

Evaluation

•

Subconjunctival petechiae

seen sporadically in Rhopressa

Rocket studies

•

MedDRA

coded to conjunctival hemorrhage

•

No conjunctival hemorrhages noted at month 12 visit

Data

on

File:

An

example

of

conjunctival

hemorrhage

from

AR-13324

–

CS302 

TM

14

Aerie Perspective on Potential Rhopressa

Advantages

*Data on file:  AR-13324 –

CS301 and AR-13324 –

CS302 **Anti-fibrotic Effects of AR-13324 in a 3D Bioengineered Human Trabecular Meshwork Model.

Cheng-Wen Lin, Feryan

Ahmed, Dhruba

Bharali, Karen Torrejon, Casey Kopczynski; The Mechanisms of Increasing Outflow Facility by AR-13324M in Human

Eyes. Haiyan Gong, Ruiyi

Ren, Guorong

Li, Casey Kopczynski, Daniel Stamer; and Visualization of conventional outflow tissue responses to netarsudil

in living

mouse eyes. Li G, Mukherjee D, Navarro I, Ashpole

NE, Sherwood JM, Chang J, Overby

DR, Yuan F, Gonzalez P, Kopczynski CC, Farsiu

S, Stamer

WD.

Eur

J Pharmacol. 2016. 787:20-31. ***Topical administration of a Rock/Net inhibitor promotes retinal ganglion cell survival and axon regeneration after optic

nerve injury. Shaw PX, Sang A, Wang Y, Ho D, Douglas C, Dia

L, Goldberg JL.

Exp

Eye Res. 2016 Jul 18. pii: S0014-4835(16)30181-6. [Epub

ahead of print]

Clinical*:

Demonstrated once-daily IOP lowering in Phase 3 trials

Three mechanisms of action

Potential PGA synergy

More consistent IOP-lowering across baselines than PGAs and timolol

No systemic side effects

Research:

Targets diseased trabecular meshwork in glaucoma

Potential to preserve health of trabecular outflow pathway**

Potential to promote retinal ganglion cell survival and axon regeneration***

TM

15

Roclatan™ Registration Trial Design

“Mercury 1”*

One Year Safety

(3 Mo. Interim

Efficacy)

Registration Trial

U.S.

“Mercury 2”**

90-Day Efficacy

Registration Trial

U.S. and Canada

“Mercury 3”

6 Mo. Efficacy and

Safety

Registration Trial

Europe

Roclatan™ QD

~230 patients

Rhopressa™ 0.02% QD

~230 patients

latanoprost QD

~230 patients

Roclatan™ QD

~230 patients

Comparator (TBD)

~230 patients

*    ClinicalTrials.gov Identifier: NCT02558400

**   ClinicalTrials.gov Identifier: NCT02674854

Roclatan™ QD

~230 patients

Rhopressa™ 0.02% QD

~230 patients

latanoprost QD

~230 patients

16

•

Roclatan

TM

met the criteria for demonstrating superiority over both

latanoprost

and

Rhopressa

TM

for the primary efficacy analysis

–

statistical superiority of Roclatan™ was demonstrated at all 9

time points versus latanoprost and versus Rhopressa™

(p<0.0001)

•

IOP-lowering effect of Roclatan™ was greater (1-3 mmHg) than

monotherapy with either latanoprost or Rhopressa™ throughout the

duration of the study (i.e., Week 2, Week 6, Month 3)

•

Roclatan

TM

reduced mean diurnal IOPs to 16 mmHg or lower in 61

percent of patients, a significantly higher percentage than observed

in the comparator arms

•

The main adverse event for Roclatan

TM

was conjunctival hyperemia,

which was reported in ~50% of patients and was scored as mild for

~80% of these patients

•

There were no drug-related serious adverse events and no evidence

of treatment-related systemic effects

Mercury 1: Roclatan

TM

Achieves Primary

Clinical Endpoint

Data on file and PG324 -

CS301 Top Line Data

17

Mercury 1: Study Design And Analysis

•

Trial design follows FDA requirement for fixed dose combination

–

Superiority of combination over each individual component

–

Statistically significant difference at each measured time

point

–

Higher combo efficacy vs. components at ~1-3 mmHg, as

previously accepted by FDA for product approval

(Simbrinza

®

)

•

The primary statistical modeling of the primary efficacy analysis

was agreed with the FDA using the intent to treat (ITT)

population with imputation for any missing data

Data on file

18

Roclatan

TM

Achieved Statistical Superiority Over

Individual Components at All Time Points

Mean IOP at Each Time Point  (ITT)

Data on file and PG324 -

CS301 Top Line Data

***p<0.0001

vs

Latanoprost

and

Rhopressa

TM

19

Mercury 1: Roclatan

TM

Phase 3, ITT

Roclatan

TM

superior to latanoprost by 1.3–2.5 mmHg (p<0.0001)

Roclatan

TM

superior to Rhopressa

TM

by 1.8-3.0 mmHg (p<0.0001)

8:00 AM

24.8

24.8

24.6

10:00 AM

23.7

23.5

23.4

4:00 PM

22.6

22.6

22.4

Mean Diurnal

23.7

23.6

23.5

8:00 AM

15.6

18.6

17.8

-3.0

(-3.6, -2.5)

-2.2

(-2.8, -1.7)

10:00 AM

14.9

17.8

17.4

-2.9

(-3.5, -2.3)

-2.5

(-3.1, -1.9)

4:00 PM

14.8

17.2

17.2

-2.4

(-2.9, -1.9)

-2.3

(-2.9, -1.8)

Mean Diurnal

15.1

17.9

17.5

-2.8

(-3.3, -2.3)

-2.4

(-2.9, -1.9)

8:00 AM

16.0

19.0

17.7

-3.0

(-3.6, -2.4)

-1.7

(-2.3, -1.1)

10:00 AM

15.3

18.0

17.1

-2.7

(-3.3, -2.2)

-1.9

(-2.5, -1.3)

4:00 PM

15.3

17.5

17.0

-2.2

(-2.7, -1.6)

-1.7

(-2.2, -1.1)

Mean Diurnal

15.5

18.2

17.3

-2.7

(-3.1, -2.2)

-1.8

(-2.3, -1.3)

8:00 AM

16.2

18.9

17.6

-2.7

(-3.4, -2.1)

-1.5

(-2.1, -0.9)

10:00 AM

15.3

18.2

16.9

-2.9

(-3.5, -2.3)

-1.6

(-2.2, -1.0)

4:00 PM

15.4

17.2

16.7

-1.8

(-2.4, -1.2)

-1.3

(-2.0, -0.7)

Mean Diurnal

15.6

18.1

17.1

-2.5

(-3.0, -2.0)

-1.5

(-2.0, -1.0)

Day 90

Mean IOP

mmHg

Rhopressa™

Day 43

Day 15

Latanoprost

Difference from Roclatan™

(95% CI)

Roclatan™

N=238

Rhopressa™

N=244

Latanoprost

N=236

Baseline

Data on file and PG324 -

CS301 Top Line Data

20

Mercury 1: Roclatan

TM

Phase 3 Responder

Analysis

Day 90:  % of Patients

with IOP Reduced to 18 mmHg or Lower

***p<0.0001 vs Latanoprost and Rhopressa

TM

###

p<0.0001 vs

Rhopressa

TM

, p<0.05 vs Latanoprost

Data on file and PG324 -

CS301 Top Line Data

14%

23%

32%

42%

54%

15%

25%

39%

54%

69%

33%

44%

61%

71%

82%

Rhopressa

TM

(n=198)

Latanoprost

(n=223)

Roclatan

TM

(n=200)

IOP on Treatment

21

Mercury 1: Safety/Tolerability Overview of

Roclatan

TM

•

There were no drug-related serious adverse events

(SAEs)

•

There was no evidence of treatment-related systemic

effects

•

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 Roclatan

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 Top Line Data

TM

22

Mercury 1: Roclatan

TM

Phase 3 Safety Profile

Adverse Events                     

(

5.0% in any group)

Roclatan

n=238

Rhopressa

n=244

Latanoprost

n=236

Eye Disorders

Conjunctival Hyperemia

126 (52.9%)

99 (40.6%)

33 (14.0%)

Conjunctival Hemorrhage

25 (10.5%)

34 (13.9%)

1 ( 0.4%)

Eye Pruritus

18 ( 7.6%)

17 ( 7.0%)

3 ( 1.3%)

Lacrimation Increased

14 ( 5.9%)

15 ( 6.1%)

1 ( 0.4%)

Cornea Verticillata

12 ( 5.0%)

9 ( 3.7%)

0 (0.0%)

Administration Site Conditions

Instillation site pain

45

(18.9%)

51 ( 20.9%)

15 ( 6.4%)

Patients with known contraindications or hypersensitivity to latanoprost

were excluded

Data on file and PG324 -

CS301 Top Line Data

TM

TM

23

Mercury 1: Roclatan™ Summary

•

Demonstrated

superiority

over

both

latanoprost

and

Rhopressa™

for

the

primary

efficacy

analysis

at

all

9

time

points

(p<0.0001)

•

IOP-lowering

effect

was

greater

(1-3

mmHg)

than

monotherapy

with

either

latanoprost

or

Rhopressa™

throughout

the

duration

of

the

study

•

There

were

no

drug-related

serious

adverse

events

and

was

no

evidence

of

treatment-related

systemic

effects

•

The

main

adverse

event

was

conjunctival

hyperemia,

~50%

of

patients

and

was

scored

as

mild

for

~80%

of

these

patients

Next

Steps:

•

Mercury 2: Topline 3-month expected H1 2017

•

Mercury 3 (Europe): Efficacy study, comparing to a leading combo product

marketed in Europe, expected to commence in H1 2017

•

Roclatan

NDA filing expected near year-end 2017

Aerie will present additional details at Investor Day in NYC, Oct 5

Data on file and PG324 -

CS301 Top Line Data

TM

24

Mercury 1: Rhopressa™ Arm Performance

Summary

•

Demonstrated non-inferiority to latanoprost in patients

with baseline IOP < 25 mmHg

•

Efficacy relative to latanoprost improves as baseline IOP

declines

•

Rhopressa

maintained consistent IOP lowering across

all baseline IOPs including > 25 mmHg

•

Stable efficacy from week 2 to month 3

•

Adverse event profile consistent with previous studies

Data on file and PG324 -

CS301 Top Line Data

TM

25

Expanding Aerie Franchise to Europe and Japan

Europe

Current clinical plan expected to satisfy EU regulatory requirements

(including Rocket 4 for Rhopressa

and Mercury 3 for Roclatan

)

Establishing KOL relationships in top 5 countries

Targeting completion of EU commercialization strategy by YE16

Expect to file EU MAA for Rhopressa™ by approximately mid-2017

Japan

Discussions regarding potential Rhopressa™

out-licensing are ongoing

Also preparing to advance clinical development on our own

Conferring with PMDA to confirm path to approval

Prepared to secure a CRO relationship in 2016 to conduct trials

TM

TM

26

Enhancing the Aerie Pipeline

Rhopressa

preclinical findings*

Disease

modification

potential

–

anti-fibrotic

and

increased

perfusion

Neuroprotection potential

From our proprietary molecule library –

preclinical AR-13154 for

wet AMD and diabetic retinopathy

Drug Delivery opportunities –

front of the eye (Glaucoma) and

back of the eye (e.g., AR-13154 for wet AMD)

* Data on file

TM

27

*    Active

ingredient

of

Rhopressa™;

TGF

2:

Transforming

growth

factor

2;

SMA:

Smooth

muscle

actin;

FSP1:

Fibroblast-specific

protein

1

•

Pattabiraman,

Padmanabhan

P.,

et.

Al.,

(2015

March)

“Effects

of

Rho

Kinase

inhibitor

AR-13324

on

actin

cytoskeleton

and

TGF

2

and

CTGF-

induced fibrogenic activity in Human Trabecular Meshwork Cells”.

AR-13324* May Have Anti-Fibrotic Activity

in Human Trabecular Meshwork Cells

P. Vasantha

Rao, Duke University

Control 

TGF

2

(8ng/ml)

TGF  2 (8 ng/ml) +

AR-13324 (500nM)

AR-13324 Blocks TGF-beta-Induced Expression of Fibrosis

Proteins in Human TM Cells

28

Netarsudil* Causes Expansion of TM Tissue,

Opening Spaces for Increased Outflow

Dan Stamer (Duke), Haiyan Gong (Boston University)

Control

+ Netarsudil

TM: Trabecular Meshwork

SC: Schlemm’s

Canal

Control = buffered saline solution

Increasing Trabecular Outflow, Reducing Fibrosis Could Stop

Degeneration of Outflow Tissues in Glaucoma

*Active ingredient of Rhopressa™

Source:

The

Mechanisms

of

Increasing

Outflow

Facility

by

AR-13324M

in

Human

Eyes.

Haiyan

Gong,

Ruiyi

Ren,

Guorong

Li,

Casey

Kopczynski, Daniel Stamer

29

Rhopressa

TM

Promotes RGC Survival and Axon

Regeneration Following Optic Nerve Injury

Jeffrey Goldberg (Stanford University)*

RGC Survival

Retina Location

Rat optic nerve crush model

*Topical administration of a Rock/Net inhibitor promotes retinal ganglion cell survival and axon regeneration after optic nerve injury.

Shaw

PX,

Sang

A,

Wang

Y,

Ho

D,

Douglas

C,

Dia

L,

Goldberg

JL.

Exp

Eye

Res.

2016

Jul

18.

pii:

S0014-4835(16)30181-6.

[Epub

ahead

of

print]

AR-13324 (n=3)

Placebo  (n=3)

Axon Regeneration

Distance from crush site (

m)

AR-13324 (n=3)

Placebo  (n=3)

30

AR-13154 vs. Eylea in Preclinical AMD Model

20000

30000

40000

50000

60000

70000

80000

90000

100000

110000

Saline

n=49

0.06 ug/mL

AR-13154

n=28

0.6 ug/mL

AR-13154

n=25

6 ug/mL

AR-13154

n=25

800 ug/mL

Eylea

n=20

Total CNV Lesion Area (Day 21)

**

*

*

p<0.05 vs. Saline

**

p<0.001

Laser-induced

choroidal

neovascularization

(CNV) in rats

Compounds delivered

by intravitreal injection

ROCK/JAK2/PDGFR

Inhibitor AR-13154 Numerically More Effective than

Eylea

®

in Rat Model of AMD

Data on file; Effectiveness of AR-13154 Monotherapy and Combination Therapy in Animal Models of Wet Age-related Macular

Degeneration and Proliferative Diabetic Retinopathy. Cheng-Wen Lin, Jill M. Sturdivant, Mitchell A. deLong and Casey C. Kopczynski

®

31

Topical AR-13154(S) Provides Added Efficacy to

Eylea

in Proliferative Diabetic Retinopathy Model

-37%

-34%

-57%

**

***

***

***

Oxygen-induced

retinopathy model of

PDR (mouse)

0.06% AR-13154(S)

delivered topically

from P12 to P17

Eylea

delivered IP

Confirms AR-13154(S)

potential as effective

adjunct to anti-VEGF

therapies

***   p < 0.0001 vs. vehicle control

**    p < 0.001 vs. monotherapy

Data on file; Effectiveness of AR-13154 Monotherapy and Combination Therapy in Animal Models of Wet Age-related Macular

Degeneration and Proliferative Diabetic Retinopathy. Cheng-Wen Lin, Jill M. Sturdivant, Mitchell A. deLong and Casey C. Kopczynski

0%

20%

40%

60%

80%

100%

120%

Vehicle Control

(n=55)

AR

-13154(S)

topical

(n=28)

Eylea

1mg/kg IP (n=26)

AR

-13154(S) +

Eylea (n=18)

Total Neovascular Area

32

Financial Summary

•

Cash Balance as of June 30, 2016 = $112M

•

26.6M shares outstanding

•

Expected Cash Balance as of September 30, 2016 of ~$250M

•

~33M shares outstanding

•

Expected Cash Balance as of December 31, 2016 of ~$230M

•

Updated Full-Year 2016 Cash Burn Guidance of ~$85M

•

Use of Proceeds from Recent Financings

•

General Corporate Purposes and Working Capital Requirements

•

Complete Funding of Rhopressa™ Commercialization Costs

•

Execution of Clinical Trials in Japan

•

Commencement of Construction of a Manufacturing Plant in Ireland

33

Summary

•

Key Clinical Priorities

•

Rhopressa™: NDA filing submitted Q3 2016                               

Rocket 4 in process (not required for NDA filing)

•

Roclatan™:    Mercury 1 and Mercury 2 completion

Mercury 3 commencement 1H 2017 (EU)

•

Research Initiatives

•

Rhopressa™ disease modification, neuroprotection, sustained release    

•

AR-13154 potential in wet AMD, etc.

•

Evaluating Aerie’s 3,000+ proprietary molecules

•

Business Development and Expansion Opportunities

•

Drug delivery opportunities for front and back of the eye

•

EU/JP clinical path and commercialization strategy

•

Ireland Manufacturing Facility

34

Rhopressa™ and Roclatan™ Key Milestones

Q3-2017: Roclatan™

P3 Mercury 1

Topline safety (12 mos)

Near YE 2017:

Roclatan™ NDA filing

expected

1H-2017: Roclatan™

P3 Mercury 3 (EU)

to be initiated

Q1-2016: Rhopressa™

Rocket 2 Topline safety (12 mos)

Q3-2016: Rhopressa™

NDA filed

Q4-2016: Rhopressa™

Rocket 4 Topline efficacy (3 mos)

Q3-2016: Roclatan™

P3 Mercury 1

Topline efficacy (3 mos)

Q2-2017: Roclatan™

P3 Mercury 2

Topline efficacy (3 mos)

Q1-2016: Roclatan™

P3

Mercury

2

initiated

2016

2017