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): January 9, 2014

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)

135 US Highway 206, Suite 15
Bedminster, New Jersey	07921
(Address of principal executive offices)	(Zip code)

Registrant’s telephone number, including area code: (908) 470-4320

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.

On January 9, 2014, Aerie Pharmaceuticals, Inc. (the “Company”) issued a press release announcing the results from the Company’s recently completed Phase 1 pharmacokinetics study. A copy of the press release is furnished as Exhibit 99.1 hereto and is hereby incorporated by reference into this Item 7.01.

On or after January 9, 2014, representatives of the Company may present to various investors the information about the Company as described in the slides attached to this report as Exhibit 99.2 hereto. Exhibit 99.2 is hereby incorporated by reference into this Item 7.01.

The information in this Item 7.01 (including Exhibits 99.1 and 99.2) are 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.

Item 9.01. Financial Statements and Exhibits.

(d) Exhibits.

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

99.1 Press Release dated January 9, 2014.

99.2 Company Overview presentation dated January 9, 2014.

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: January 9, 2014	By:	/s/ Richard J. Rubino
		Richard J. Rubino
		Chief Financial Officer

EXHIBIT INDEX

Exhibit	Description
99.1	Press Release dated January 9, 2014
99.2	Company Overview presentation dated January 9, 2014

EX-99.1

Exhibit 99.1

January 9, 2014

Aerie Pharmaceuticals Reports Positive Results for Lead Candidate AR-13324 in Normotensive Individuals

Results Also Suggest a Potential New Mechanism of Action

Aerie Reaffirms Timeline to Commence Two Phase 3 Studies by mid-2014

BEDMINSTER, N.J. & RESEARCH TRIANGLE PARK, N.C. & NEWPORT BEACH, CALIF. — (BUSINESS WIRE) — Aerie Pharmaceuticals, Inc. (NASDAQ:AERI), a clinical-stage pharmaceutical company focused on the discovery, development and commercialization of first-in-class glaucoma therapies, today announced top-line results from the Company’s recently completed Phase 1 pharmacokinetics (PK) study, in which AR-13324 eye drops were administered once-daily to 18 healthy individuals over an eight-day period to assess systemic exposure to the drug. In addition to the primary PK outcome of the study, the drug’s effect on intraocular pressure (IOP) was also measured. The completion of the PK study is an important step in preparing for two planned Phase 3 registration studies of AR-13324, which are expected to commence by mid-2014.

The PK results demonstrated very low systemic exposure to the drug, with blood levels at or below the limit of detection of 0.1 ng/mL at all time points. In addition, there were no drug-related effects on systemic safety parameters such as blood pressure and heart rate. All study subjects had IOPs in the normotensive range of 12 to 21 mmHg, with an average diurnal IOP for the group of approximately 16 mmHg prior to dosing. After eight days of dosing, once-daily administration of AR-13324 reduced the average diurnal IOP to approximately 11 mmHg, representing a decrease of approximately 5 mmHg, or over 30 percent.

Brian Levy, O.D., M.Sc., Aerie’s Chief Medical Officer, commented, “The large reduction in IOP achieved by AR-13324 in normotensive individuals distinguishes AR-13324 from currently available glaucoma drugs, which are typically less effective in subjects with low baseline IOPs.¹ Preclinical normotensive animal studies have shown similar results. These data are consistent with the results of our previous Phase 2b clinical trial in which AR-13324 demonstrated consistent IOP lowering irrespective of baseline IOP, whereas the comparator latanoprost demonstrated reduced efficacy at lower baseline IOPs, as disclosed in our recently filed registration statement.”

“As documented in the Baltimore Eye Survey², patients with low-to-moderately elevated IOPs at the time of diagnosis represent the significant majority of glaucoma patients,” stated David Epstein, M.D., Chairman, Department of Ophthalmology, Duke University School of Medicine, and Chairman of Aerie’s Scientific Advisory Board. “Very commonly, glaucoma patients require a low normal IOP to prevent further progression, and a drug that could achieve this would be a great advance. I am encouraged by the recent

¹  Hedman and Alm; European Journal Ophthalmology; 2000.

² Sommer A, Tielsch JM, Katz J et al. Relationship between intraocular pressure and primary open angle glaucoma among white and black Americans: The Baltimore eye survey. Arch Ophthalmol 1991;109:1090-1095.

1

data and previous clinical findings for AR-13324, and believe that its significant IOP-lowering effect at lower baseline IOPs could reflect an additional mechanism of action beyond the dual mechanisms of which we are already aware, one that could involve the reduction of episcleral venous pressure.”

As background, IOP is determined by the contributions of four distinct MOAs: aqueous humor (eye fluid) production, resistance to aqueous outflow via the trabecular meshwork and uveoscleral pathway, and episcleral venous pressure. Historical studies have shown that episcleral venous pressure accounts for approximately half of IOP in normotensive subjects and approximately one-third of IOP in patients with pressures of 24 to 30 mmHg. When episcleral venous pressure is lowered, aqueous humor is able to flow more freely from the eye. Aerie plans to conduct additional studies to directly measure the effect of AR-13324 on episcleral venous pressure and better understand this potential new mechanism of action.

AR-13324 Clinical Program

Aerie’s successful 28-day Phase 2b trial demonstrated that once-daily administration of AR-13324 produced significant IOP lowering in the range of 5.7 to 6.2 mmHg in patients with elevated IOP, with a differentiated efficacy profile of consistent IOP lowering across all baseline IOPs tested in the clinical trial. The multiple mechanisms of action for AR-13324 include increasing fluid outflow through the trabecular pathway or primary drain, reducing fluid production, and potentially also lowering episcleral venous pressure. Aerie plans to commence two Phase 3 registration trials of AR-13324 in mid-2014, with total expected enrollment of approximately 1,200 patients. Based on the Phase 2b clinical trial results, previous discussions with the FDA, and the recent findings on the potential new mechanism of action, the Company plans to recruit patients into the Phase 3 clinical trials with baseline IOPs ranging from 21 to 26 mmHg. The trials will measure efficacy over three months and safety over 12 months. The primary efficacy endpoint of the trials will be to demonstrate non-inferiority of IOP lowering for AR-13324 (dosed once daily) compared to timolol (dosed twice daily).

Assuming the registration trials commence on schedule, three-month efficacy results are expected to be released in mid-2015, and should the trials be successful, Aerie expects to submit an NDA filing for AR-13324 by mid-2016.

PG324 Clinical Program

PG324 is a once-daily eye drop that combines AR-13324 with latanoprost, a prostaglandin analogue that is the most widely prescribed glaucoma drug. If approved, Aerie believes that PG324 would be the first glaucoma product to lower IOP through potentially four mechanisms of action: increasing fluid outflow through the trabecular pathway or primary drain, increasing fluid outflow through the uveoscleral pathway or secondary drain, reducing fluid production in the eye, and potentially also lowering episcleral venous pressure. The Company believes that PG324, if approved, would be the only glaucoma product that covers the full spectrum of IOP-lowering mechanisms, thereby providing a greater IOP-lowering effect than any currently approved glaucoma product.

2

Aerie is preparing to bring PG324 into a 28-day Phase 2b clinical trial that is expected to include approximately 300 patients and will compare two concentrations of PG324 to latanoprost and to AR-13324, all dosed once daily. The efficacy endpoint will be superiority of PG324 to each of its components. Results of the PG324 Phase 2b trial are currently expected in mid-2014.

About Aerie Pharmaceuticals, Inc.

Aerie is a clinical-stage pharmaceutical company focused on the discovery, development and commercialization of first-in-class glaucoma therapies. The Company is preparing for two Phase 3 registration trials, where the primary efficacy endpoint will be to demonstrate non-inferiority of IOP lowering for AR-13324 (dosed once daily) compared to timolol (dosed twice daily). The Company also is preparing for a Phase 2b clinical trial of its fixed-dose combination product PG324, where the primary efficacy endpoint will be to demonstrate superiority of PG324 to each of its components.

Forward-Looking Statements

This press release contains forward-looking statements for purposes of the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. We may, in some cases, use terms such as “predicts,” “believes,” “potential,” “continue,” “estimates,” “anticipates,” “expects,” “plans,” “intends,” “may,” “could,” “might,” “will,” “should” or other words that convey uncertainty of future events or outcomes to identify these forward-looking statements. Forward-looking statements include statements regarding our intentions, beliefs, projections, outlook, analyses or current expectations concerning, among other things: the success, timing and cost of our ongoing clinical trials and anticipated Phase 3 and Phase 2b clinical trials for our current product candidates, including statements regarding the timing of initiation and completion of the trials; the timing of and our ability to obtain and maintain U.S. Food and Drug Administration or other regulatory authority approval of, or other action with respect, to our product candidates; our estimates regarding anticipated capital requirements and our needs for additional financing; our expectations regarding the clinical effectiveness of our product candidates and results of our clinical trials; the potential advantages of our product candidates; our ability to protect our proprietary technology and enforce our intellectual property rights; and our expectations related to the use of proceeds from our initial public offering. 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. We discuss many of these risks in greater detail under the heading “Risk Factors” section contained in our final prospectus from our initial public offering which is on file with the Securities and Exchange Commission (SEC), and in the quarterly and annual reports that we file with the SEC. Forward-looking statements are not guarantees of future performance and our actual results of operations, financial condition and liquidity, and the development of the industry in which we operate may differ materially from the forward-looking statements contained in this press release. Any forward-looking statements that we make in this press release speak only as of the date of this press release. We assume no obligation to update our forward-looking statements whether as a result of new information, future events or otherwise, after the date of this press release.

3

Aerie Pharmaceuticals

Richard Rubino, 908-470-4320

rrubino@aeriepharma.com

or

Burns McClellan, Inc. on behalf of Aerie Pharmaceuticals

Angeli Kolhatkar - Investors

akolhatkar@burnsmc.com

Justin Jackson - Media

jjackson@burnsmc.com

Both: 212-213-0006

Source: Aerie Pharmaceuticals, Inc.

4

EX-99.2

Company Overview

January 9, 2014

Leading Innovation in Glaucoma

Leading Innovation in Glaucoma

The Next Generation

The Next Generation

Exhibit 99.2

2

Important Information

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 data results for our

AR-13324 product candidate relate to the results in our Phase 2 clinical trials.  Our product candidate PG324

has only been tested in preclinical animal models.

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, including beliefs, expectations, estimates, projections and statements relating to our business

plans, prospects and objectives, and the assumptions upon which those statements are based.  Words such as

“may,” “will,” “should,” “would,” “could,” “believe,” “expects,” “anticipates,” “plans,” “intends,” “estimates,” “targets,”

“projects” 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 our prospectus filed with the SEC on October 28, 2013, particularly in the sections titled “Risk Factors” and

“Management’s Discussion and Analysis of Financial Condition and Results of Operation.”  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

•

Large

Market

Opportunity

-

$4.5B

US/EU/JP

Market

-

Significant

unmet

needs

-

50%

of

patients

require

more

than

one

drug

-

No PGA fixed-combination products approved in the US

•

Proprietary, Differentiated First-in-Class Glaucoma Pipeline

-

Both

ROCK

&

NET

inhibition

-

First

new

MOAs

in

two

decades

-

Potential

additional

new

MOA

–

lowering

of

EVP

•

AR-13324: Novel Dual-Action, Once-Daily Therapy

-

Phase 3 expected to begin mid-2014; efficacy data expected mid-2015

-

NDA filing expected by mid-2016

•

PG324: Breakthrough Triple-Action, Once-Daily Therapy

-

Fixed combination of AR-13324 and PGA latanoprost

-

Phase 2b data expected mid-2014; Phase 3 readiness expected mid-2015

•

All

Products

Developed

Internally

-

Own

Class

&

Compounds

-

Patent protection through at least 2030 in the US

-

All rights retained with no partnerships

Aerie –

Aerie –

Next Generation in Glaucoma Therapies

Next Generation in Glaucoma Therapies

4

Establish Commercial

Establish Commercial

Capabilities

Capabilities

Expand Commercial

Expand Commercial

Offerings

Offerings

Maximize Portfolio

Maximize Portfolio

Value Ex-US

Value Ex-US

•

Explore partnership opportunities outside US

through collaborations & licensing

•

Global reach to Europe, Japan and emerging

markets

•

Build own US commercial infrastructure with ~100 sales

representatives

•

Target

~10,000

high-prescribing

eye

care

professionals

•

Explore additional ophthalmic product candidates

Advance First-in-Class

Advance First-in-Class

Glaucoma Treatments

Glaucoma Treatments

•

AR-13324 Phase 3 efficacy results expected mid-2015;

NDA filing expected mid-2016

•

PG324 Phase 2b results expected mid-2014; Initiate

prep for Phase 3 trials mid 2014-2015

Aerie Strategy Overview

Aerie Strategy Overview

5

Aerie’s Experienced Leadership Team

Aerie’s Experienced Leadership Team

Executive Team

Vince Anido, Jr., PhD

Chairman & CEO

Thomas Mitro

President & COO

Brian Levy, OD, MSc

CMO

Casey Kopczynski, PhD

CSO

Richard Rubino

CFO

Board Experience

6

Discovered new Rho Kinase Inhibitors

and the novel     

Dual-Action

ROCK/NET

Inhibitor

drug class

-

>1,500 ROCK and ROCK/NET inhibitors screened and

characterized

Pursued parallel clinical development of two franchises to

allow data driven selection of best product candidates

Dual-Action AR-13324 franchise selected for future

development

-

Superior efficacy profile in clinic

-

Longer duration of action

-

10-160x more potent than previous clinical stage ROCK inhibitors

-

Superior safety profile in long-term ocular toxicology study

-

Consistent IOP lowering regardless of baseline IOP

Aerie is a Leader in Rho Kinase R&D

Aerie is a Leader in Rho Kinase R&D

ROCK/NET: Rho Kinase / Norepinephrine Transporter

7

Aerie’s Pipeline is Advancing Rapidly

Aerie’s Pipeline is Advancing Rapidly

Dual-Action

Dual-Action

Triple-Action

Triple-Action

P2b 2014

Dual-Action

Dual-Action

Clinical Trials for Glaucoma Recruit Quickly

and Have Clear Endpoints

Preclinical

Preclinical

IND

IND

Phase

Phase

1/2a

1/2a

Phase

Phase

2b

2b

Phase

Phase

3

3

NDA

NDA

AR-13324

ROCK/NET

Inhibitor

PG324

Combination

AR

-13324 +

Latanoprost

AR-13533

ROCK/NET   

2

nd

Generation

2016

2014

P3 Prep

To mid 2015

8

Glaucoma Market

Glaucoma Market

9

Largest Rx market in ophthalmology

-

US 28.5M Rx; annual US sales $1.9B ($4.5B US/EU/JP)

2.2M glaucoma patients in the US and growing due to

aging of population

-

Glaucoma is a leading cause of blindness in US and WW

~50% of patients use multiple medications to control

disease –

compliance and tolerability are issues

No drugs launched with new MOA in the past 20 years

Significant Glaucoma Market Opportunities

Significant Glaucoma Market Opportunities

10

Currently Prescribed Glaucoma Therapies

Currently Prescribed Glaucoma Therapies

Once Daily

2-3 Times

Daily

Non-PGA

Market

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

Half of TRx are Written for Non-PGA Products

US Glaucoma Market

IMS TRx data, FY 2012

PGA

Market

Bimatoprost,

11%

Travoprost,

12%

Latanoprost,

27%

BB, 15%

BB Fixed

Combo, 14%

AA, 14%

CAI, 6%

Once-Daily

Once-Daily

Triple-Action

Triple-Action

PG324

PG324

Once-Daily

Once-Daily

Dual-Action

Dual-Action

AR-13324

AR-13324

Inflow (fluid production)

increase

decrease

increase

Outflow

Secondary Drain

(Uveoscleral)

Outflow                  

Primary Drain

(Trabecular Meshwork)

Novel Products Address

All IOP Control Mechanisms, and May Lower EVP

AA, BB, CAI

PGAs

Triple Action

PG324

11

Dual Action

AR-13324

12

The Competitive Landscape

The Competitive Landscape

Clinical

Clinical

Efficacy

Efficacy

Dosing/

Dosing/

Day

Day

Tolerability

Tolerability

Peak Product

Peak Product

Sales*

Sales*

(US/EU)

(US/EU)

Targeting

Targeting

Diseased

Diseased

Tissue

Tissue

PGA

High

1x

Hyperemia        

Iris Color

$1.7B

No

Beta  

Blocker

Moderate

2x

Cardiopulmonary

Contraindications

>$500M

No

CAI

Low

2 -

3x

Sulfonamide

Contraindication

Bitter Taste

>$500M

No

Alpha

Agonist

Low

2 -

3x

Allergy 

Drowsiness

>$400M

No

AR-13324

High;

Moderate

1x

Hyperemia

Yes

PG324

Potentially

Highest

1x

TBD

Yes

* Peak sales for best-in-class franchise

13

New MOAs

New MOAs

AR-13324 (Aerie)

ROCK/NET inhibitor (qd)

Phase 2b

K-115 (Kowa)

ROCK inhibitor (bid)

Phase 3 (Japan)

AMA0076 (Amakem)

ROCK inhibitor (bid)

Phase 2a

INO-8875 (Inotek)

Adenosine-A1 agonist (bid)

Phase 2

LX7101 (Lexicon)

LIMK2 inhibitor (bid)

Phase 1/2

SYL040012 (Sylentis)

RNAi beta blocker (bid)

Phase 2

New PGAs -

not usable as add-on to current PGAs

Glaucoma Competitors in Pipeline

Glaucoma Competitors in Pipeline

AR-13324 is the only new MOA drug dosed once-daily

New PGAs

New PGAs

BOL-303259 (B+L)

NO donating latanoprost (qd)

Phase 3

DE-117 (Santen)

EP2 agonist (qd)

Phase 2a

ONO-9054 (Ono)

FP/EP3 agonist (qd)

Phase 1

14

Single drop, once-daily

1 drug -

2 MOAs, and may also

lower EVP

Well tolerated; no systemic drug-

related AEs

Efficacy expected to be

current

non-PGA drugs

Single drop, once-daily

Combination of 2 drugs -

3 MOAs,

and may also lower EVP

Well tolerated; no systemic      

drug-related AEs

Efficacy expected to be superior to

PGA monotherapy

AR-13324 Dual-Action

AR-13324 Dual-Action

PG324 Triple-Action

PG324 Triple-Action

Future non-PGA drug of choice

Future drug of choice for all

glaucoma patients

Aerie Franchise: Highly Differentiated            

Aerie Franchise: Highly Differentiated             

Product Profiles

Product Profiles

PGA: Prostaglandin Analogue

New Dual-Action Drug Class:

AR-13324

15

16

About Open-Angle Glaucoma

About Open-Angle Glaucoma

*Early

Manifest

Glaucoma

Trial

(Heijl, 2002; Leske, 2003); Ocular Hypertension Treatment Study (Kass, 2002)

•

Open-angle glaucoma is a progressive, irreversible and 

chronic disease of the eye

-

Typical patient age >60 years

•

Elevated intraocular pressure                                  

(IOP) can lead to loss of vision                               

and eventual blindness

•

Lowering IOP slows or halts                                    

progression

-

5 mmHg IOP reduction reduces                                   

risk of disease progression by 50%*

•

Cause of elevated IOP is degeneration of primary fluid         

drain (trabecular meshwork)

17

AR-13324 Mechanisms of Action

AR-13324 Mechanisms of Action

AR-13324

NET

RKI

NET

RKI

Trabecular

Meshwork

1.

ROCK inhibition relaxes TM

2.

NET inhibition reduces fluid production

3.

ROCK inhibition may lower Episcleral

Venous Pressure (EVP)

Episcleral

Veins

Schlemm’s

Canal

Uveoscleral

Outflow

Ciliary Processes

Cornea

18

Baseline IOP

Baseline IOP

(mmHg)

(mmHg)

Percentage of POAG

Percentage of POAG

Patients Identified

Patients Identified

Cumulative Percentage

Cumulative Percentage

<

15

13%

13%

16-18

24%

37%

19-21

22%

59%

22-24

19%

78%

25-29

10%

88%

30-34

9%

97%

35

3%

100%

10,444 individuals were

screened for the prevalence of Primary Open-Angle Glaucoma (POAG)

~80% of Glaucoma IOPs Are 

~80% of Glaucoma IOPs Are 

26 mmHg           

26 mmHg           

at Time of Diagnosis

at Time of Diagnosis

Baltimore Eye Survey

* Sommer A, Tielsch JM, Katz J et al. Relationship between intraocular pressure and primary open angle

glaucoma among white and black Americans: The Baltimore eye survey. Arch Ophthalmol 1991;109:1090-1095

19

AR-13324 Phase I PK Results Support

AR-13324 Phase I PK Results Support

Potential New MOA

Potential New MOA

In

healthy

volunteers

with

normotensive

IOP

of

12

–

21

mmHg,

AR-13324

lowered

IOP

by

over

30%

on

D8

Currently available glaucoma drugs are typically less effective

at lower IOPs

High

efficacy

at

these

baselines

suggest

a

lowering

of

EVP

–

a

potential new MOA

EVP contributes up to half of IOP

In Baltimore Eye Survey, ~60% of glaucoma patients at the

time of diagnosis had low IOPs (

<

21

mmHg)

PK analysis showed low systemic exposure and no drug-

related effects on systemic safety parameters

20

AR-13324 Demonstrated Strong IOP Lowering     

AR-13324 Demonstrated Strong IOP Lowering      

in Phase 2b

in Phase 2b

13 glaucoma NCEs advanced from Phase 2 to Phase 3

since

1970s

-

All

approved

AR-13324 Efficacy at 8 AM

Diurnal Average IOP -

Entry IOP 22-36 mmHg  (n=221)

Once-daily PM dosing of

0.02% AR-13324 is

highly effective

-

IOP -5.7 and -6.2 mmHg on

D28 and D14

-

Consistent efficacy through

D28

AR-13324 efficacy results

current

non-PGA

drugs

Favorable tolerability

profile

No systemic side effects

21

The only AR-13324 finding of note was hyperemia

-

Scored as trace, mild or moderate, and transient for majority of

patients

-

No drug-related systemic adverse events

On last day of study (Day 28 at 8 AM), mild and moderate

conjunctival hyperemia was observed in 24% of AR-13324

0.02% patients and 11% of latanoprost patients

-

Frequency of hyperemia decreased throughout the study for AR-13324

and increased for latanoprost

A 12-week study by Parrish et. al. (2003) compared the three

most highly prescribed PGAs for frequency of hyperemia

-

Latanoprost: 16% frequency

-

Travoprost:  27% frequency

-

Bimatoprost:  35% frequency

American Journal of Ophthalmology,  Vol. 135, NO. 5  (2003)

AR-13324 Phase 2b Safety and Tolerability

AR-13324 Phase 2b Safety and Tolerability

22

AR-13324 and latanoprost

clinically and statistically

equivalent in patients with

moderately elevated IOPs of      

22 -

26 mmHg

Latanoprost loses ~1 mmHg

efficacy in patients with IOPs of  

22 -

26 mmHg vs. 22 -

36 mmHg

AR-13324 maintains consistent

efficacy in patients with moderately

elevated IOPs

Phase

2b

baseline

IOP

entry

requirements:

24,

22,

22

mmHg

(8am,

10am,

4pm)

Differentiated AR-13324 Efficacy Profile          

Differentiated AR-13324 Efficacy Profile          

Informs Phase 3 Study Design

Informs Phase 3 Study Design

Full Patient Population

Moderately Elevated IOP

Latanoprost

0.02%

AR-13324

Latanoprost

0.02%

AR-13324

Baseline

22 –

36 mmHg

(n=221)

Baseline

22 –

26 mmHg

(n=106)

23

Latanoprost and Timolol Show Reduced Efficacy   

Latanoprost and Timolol Show Reduced Efficacy   

at Lower Baseline IOPs

at Lower Baseline IOPs

Latanoprost and timolol

lose 0.5 mmHg efficacy for

every 1 mmHg drop in

baseline IOP

Timolol less effective

than

latanoprost at all baselines

AR-13324 equivalent/    

non-inferior

to latanoprost  

at baselines 22 –

26 mmHg

Timolol is the standard

comparator for glaucoma

Phase 3 trials

Pooled data from three latanoprost registration studies.

Hedman and Alm; European Journal Ophthalmology;2000

0

-2

-4

-6

-8

-10

-12

-14

-16

Timolol (n = 369)

Latanoprost (n = 460)

16

18

20

22

24

26

28

30

32

34

36

38

Untreated diurnal IOP (mmHg)

24

AR-13324 Registration Trial Design

AR-13324 Registration Trial Design

Primary efficacy endpoint: IOP at all

time points through Day 90

Non-inferiority design vs. timolol

-

95% CI within 1.5 mmHg at all time points, 

within 1.0 mmHg at a majority of time points

Planned entry IOP: Minimum              

21 mmHg, maximum 26 mmHg

-

FDA has agreed to Aerie proposal for entry IOPs

with no impact on label

-

AR-13324 non-inferior to latanoprost at entry

IOPs of 22-26 mmHg in Phase 2b

Start mid-2014; 90 day safety and

efficacy data expected mid-2015;   

NDA filing expected mid-2016

Phase 3 Protocol

Phase 3 Protocol

AR-13324 0.02%

AR-13324 0.02%

dosed QD PM

dosed QD PM

vs.

vs.

Timolol                

Timolol                

dosed BID

dosed BID

~1200 patients

~1200 patients

90 days efficacy

90 days efficacy

100 patients for 1 year

100 patients for 1 year

safety

safety

25

Triple-Action Fixed Combination:

Triple-Action Fixed Combination:

PG324

PG324

26

AR-13324 FIXED COMBINATION WITH

LATANOPROST

1.

2.

3.

4.

Triple-Action PG324 Combination Product

Triple-Action PG324 Combination Product

AR-13324

Ciliary Processes

Latanoprost

Uveoscleral

Outflow

Cornea

TM

Outflow

ROCK inhibition restores TM outflow

NET inhibition reduces fluid production

ROCK inhibition may lower

Episcleral Venous Pressure

PGA receptor activation

increases uveoscleral

outflow

27

PG324: Triple-Action Fixed Combination

PG324: Triple-Action Fixed Combination

First product to lower IOP through

all three known mechanisms, and

also potentially lower EVP     

-

Once-daily: 1 drop, 2 drugs, 3 MOAs

High efficacy in predictive 

primate model

Human proof of concept

established in prior ROCKi/PGA

combination trials

-

Demonstrated significant IOP

lowering beyond PGA alone

Potential for maximal medical

therapy in a single eye drop

Latanoprost

0.02% PG324

PG324 vs. Latanoprost, Primates (n=6/group)

Dose

Dose

Dose

Day

2

-

No

IOP

measured

28

PG324: A Breakthrough Triple-Action Product Candidate

PG324: A Breakthrough Triple-Action Product Candidate

AR-13324 formulated with market-leading latanoprost

-

Latanoprost has largest prescription base in glaucoma

-

Facilitates use as first-line therapy

-

Eases switch from latanoprost monotherapy

Combinations constitute an important growth segment            

of glaucoma market internationally

-

US lags -

no PGA fixed-combination glaucoma drugs in the US

-

Powerful compliance rationale for life-long medication users

-

Beneficial to payors and patients

Expected to be first PGA fixed-combination product in the US

29

PG324 Phase 2b Clinical Trial Design

PG324 Phase 2b Clinical Trial Design

Phase 2b Protocol

Phase 2b Protocol

PG324 0.01%

PG324 0.01%

vs.

vs.

PG324 0.02%

PG324 0.02%

vs.

vs.

AR-13324 0.02%

AR-13324 0.02%

vs.

vs.

Latanoprost

Latanoprost

All dosed QD PM

All dosed QD PM

~300 patients

~300 patients

28 days

28 days

Primary efficacy endpoint: Mean

diurnal IOP on Day 28

Two concentrations of PG324 vs.

AR-13324 0.02% and latanoprost

Trial design similar to registration

trial for fixed-dose combination

-

1-3 mmHg superiority vs. components

previously accepted by FDA

Trial starts early 2014

Data expected mid-2014

30

Key Milestones

Key Milestones

H1 2014

H1 2014

H2 2014

H2 2014

H2 2015

H2 2015

H1 2015

H1 2015

H1 2016

H1 2016

Early-2014: PG324

Start Phase 2b clinical trial

Mid-2014: AR-13324

Start Phase 3

registration trials

Mid-2015: AR-13324

Efficacy results from

Phase 3 expected

Mid-2016: AR-13324

NDA filing expected

Mid-2014: PG324

Results from Phase

2b expected

Mid-2015: PG324

Phase 3-prep

31

•

Large

Market

Opportunity

-

$4.5B

US/EU/JP

Market

-

Significant

unmet

needs

-

50%

of

patients

require

more

than

one

drug

-

No PGA fixed-combination products approved in the US

•

Proprietary, Differentiated First-in-Class Glaucoma Pipeline

-

Both

ROCK

&

NET

inhibition

-

First

new

MOAs

in

two

decades

-

Potential

additional

new

MOA

–

lowering

of

EVP

•

AR-13324: Novel Dual-Action, Once-Daily Therapy

-

Phase 3 expected to begin mid-2014; efficacy data expected mid-2015

-

NDA filing expected by mid-2016

•

PG324: Breakthrough Triple-Action, Once-Daily Therapy

-

Fixed combination of AR-13324 and PGA latanoprost

-

Phase 2b data expected mid-2014; Phase 3 readiness expected mid-2015

•

All

Products

Developed

Internally

-

Own

Class

&

Compounds

-

Patent protection through at least 2030 in the US

-

All rights retained with no partnerships

Aerie –

Aerie –

Next Generation in Glaucoma Therapies

Next Generation in Glaucoma Therapies