AERIE PHARMACEUTICALS INC - FORM 8-K - EX-99.1

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8-K - FORM 8-K - AERIE PHARMACEUTICALS INC	d664273d8k.htm

Leading Innovation in Glaucoma

The Next Generation

Company Overview

January 27, 2014

Exhibit 99.1

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. Know

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

and 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 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.

Aerie –

Next Generation in Glaucoma Therapies

AR-13324

Novel Dual Action

All Products Fully

Owned by Aerie

PG324 Breakthrough

Triple Action

•

Fixed combination of AR-13324 and latanoprost

•

Potential for maximal IOP lowering

•

Expect

P2b

results

mid-2014,

readiness

mid-2015

•

Inhibits ROCK and NET, may also lower EVP

•

Targets diseased tissue, consistent IOP lowering

•

Expect

efficacy

data

mid-2015,

NDA

filing

mid-2016

Large Market

Opportunity

•

$4.5B US/EU/JP Market with significant unmet needs

•

Multiple MOAs, once daily, high efficacy and safety

•

Late-stage/potential blockbuster revenue opportunity

•

Full

patent

protection

through

least

2030

•

Will retain US rights and plan to partner in

JP/EU

Currently Prescribed Glaucoma Therapies

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

Once Daily

2-3 Times

Daily

Non-PGA

Market

US Glaucoma Market

IMS TRx data, FY 2012

PGA

Market

Bimatoprost,

11%

Travoprost,

12%

Latanoprost,

27%

BB, 15%

BB Fixed

AA, 14%

CAI, 6%

Combo, 14%

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 expected to grow to

over

2020

•

~50% of patients use multiple medications to control

disease

•

No drugs with new MOA launched in 20 years

•

None of the existing drugs target the diseased tissue, or

Trabecular Meshwork (primary drain)

•

None of the existing drugs have shown consistent

efficacy across a broad range of pressures

Significant Glaucoma Market Opportunities

National Eye Institute

Inflow (fluid production)

AA, BB, CAI

decrease

increase

Aerie’s Novel Products Address

All IOP Control Mechanisms and May Lower EVP

Triple Action

PG324

Outflow

Secondary Drain

(Uveoscleral)

Outflow

Primary Drain

(Trabecular Meshwork)

Dual Action

AR-13324

increase

PGAs

The Competitive Landscape

Clinical

Efficacy

Dosing/

Day

Tolerability

Peak Product

Sales*

(US/EU)

Targeting

Diseased

Tissue

PGA

High

Hyperemia

Iris Color Change

Other Cosmetic

$1.7B

Beta

Blocker

Moderate

Cardiopulmonary

Contraindications

>$500M

CAI

Low

2 -

Sulfonamide

Contraindication

Bitter Taste

>$500M

Alpha

Agonist

Low

2 -

Allergy

Drowsiness

>$400M

AR-13324

High;

Consistent

Hyperemia

Yes

PG324

Potentially

Highest

TBD

Yes

* Peak sales for best-in-class franchise

New PGAs -

not usable as add-on to current PGAs

Glaucoma Competitors in Pipeline

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

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 MOAs

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

New PGAs

Once-daily, consistent IOP lowering

1 drug -

2 MOAs, and may also

lower EVP, targets diseased tissue

Well tolerated; no systemic

drug-related AEs

Efficacy expected to be

current

drugs for majority of patients

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 all

current drugs

AR-13324 Dual-Action

PG324 Triple-Action

Future drug of choice for 80%

of patients -

IOP

26mmHg

Future drug of choice for

patients requiring maximal

IOP lowering

Aerie Franchise: Highly Differentiated

Product Profiles

Uveoscleral

Outflow

AR-13324

Trabecular

Meshwork

ROCK inhibition relaxes TM, increases outflow

NET inhibition reduces fluid production

ROCK inhibition may lower Episcleral

Venous Pressure (EVP)

Episcleral

Veins

Schlemm’s

Canal

Mechanisms of Action:

AR-13324

RKI

NET

Ciliary Processes

RKI

NET

Cornea

AR-13324 Demonstrated Strong IOP Lowering

in Phase 2b

13 glaucoma NCEs advanced from Phase 2 to Phase 3

since 1970s -

All approved

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

within ~1 mmHg of

latanoprost

Favorable tolerability profile

No systemic side effects

Diurnal Average IOP -

Entry IOP 22-36 mmHg (n=221)

AR-13324 Efficacy at 8 AM

AR-13324 and latanoprost

clinically and statistically

equivalent in patients with

moderately elevated IOPs

of 22 -

26 mmHg

Latanoprost loses ~1 mmHg

efficacy

AR-13324 maintains consistent

efficacy

Latanoprost does not target the

diseased tissue –

AR-13324 does

Baseline

22 –

26 mmHg

(n=106)

Baseline

22 –

36 mmHg

(n=221)

Phase

baseline

IOP

entry

requirements:

24,

22,

mmHg

(8am,

10am,

4pm)

Differentiated AR-13324 Efficacy Profile

Informs Phase 3 Study Design

Full Patient Population

Moderately Elevated IOP

Latanoprost

0.02%

AR-13324

Latanoprost

0.02%

AR-13324

Potential New Additional MOA

May Represent Breakthrough

In healthy volunteers with normotensive IOPs 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 baseline IOPs suggests AR-13324 may

lower EVP –

a potential new MOA

No currently marketed products are known to lower EVP,

which contributes up to half of IOP in normotensive subjects

Aerie Phase 1 PK Study Press Release dated January 9, 2014

Average IOP dropped 5 mmHg – from 16 to 11 mmHg

Baseline IOP

(mmHg)

Percentage of POAG

Patients Identified

Cumulative Percentage

13%

16-18

24%

37%

19-21

22%

59%

22-24

19%

78%

25-29

10%

88%

30-34

97%

100%

10,444 individuals were

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

~80%

Glaucoma

IOPs

Are

mmHg

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

Latanoprost and Timolol Show Reduced Efficacy

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

-2

-4

-6

-8

-10

-12

-14

-16

Timolol (n = 369)

Latanoprost (n = 460)

Untreated diurnal IOP (mmHg)

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 expected impact on label

AR-13324 non-inferior to latanoprost at entry

IOPs of 22-26 mmHg in Phase 2b

Phase 3 Protocol

AR-13324 0.02%

dosed QD PM

vs.

Timolol

dosed BID

~1200 patients

90 days efficacy

100 patients for 1 year

safety

Potential

drug

choice

for

80%

patients

–

those

with

IOPs

26mmHg

Once daily, high efficacy, consistent IOP lowering, good

tolerability and safety profile through Phase 2

The only drug to target the diseased tissue, with added MOAs

of lowering fluid production and potentially lowering EVP

Next Steps:

Phase 3 expected to commence in mid-2014

Phase 3 90-day efficacy and short-term safety results expected mid-2015

NDA filing expected by mid-2016

AR-13324 Summary

AR-13324 FIXED COMBINATION WITH

LATANOPROST

Triple-Action PG324

MOAs:

Ciliary Processes

Uveoscleral

Outflow

Latanoprost

RKI

NET

Cornea

Outflow

RKI

NET

AR-13324

ROCK inhibition restores TM outflow

NET inhibition reduces fluid production

ROCK inhibition may lower

Episcleral Venous Pressure

PGA receptor activation

increases uveoscleral

outflow

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

Latanoprost

0.02% PG324

PG324 Phase 2b Clinical Trial Design

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

Phase

Protocol

PG324 0.01%

vs.

PG324 0.02%

vs.

AR-13324 0.02%

vs.

Latanoprost

All dosed QD PM

~300 patients

28 days

Potential for use by patients requiring maximal IOP lowering

and/or patients with advanced disease state

Potential to be the most efficacious IOP-lowering drug in

glaucoma with at least three and potentially four MOAs

Expected to be the first PGA fixed combination product in the

US, and dosed as a single drop once daily

Next Steps:

Phase 2b first patient dosed early 1Q 2014

Phase 2b results expected mid-2014

Phase 3 preparatory work commences second half of 2014

Phase 3 readiness expected mid 2015

PG324 Summary

Key Milestones Financed by IPO Proceeds

Early-2014: PG324

Start Phase 2b clinical trial

H1 2014

H2 2014

H2 2015

H1 2015

Mid-2014: AR-13324

Start Phase 3

registration trials

Mid-2014: PG324

Results from Phase

2b expected

Mid-2015: AR-13324

Efficacy results from

Phase 3 expected

H1 2016

Mid-2015: PG324

Phase 3-prep

Mid-2016: AR-13324

NDA filing expected

Financial Summary

AR-13324

Phase 3 registration trials

Direct clinical -

$25.0M

Non-clinical -

$12.4M

PG324

Phase 2b clinical trial

Direct clinical -

$2.5M

Non-clinical -

$1.0M

Phase 3 enabling activities

Non-clinical -

$6.3M

G&A and working capital -

$20.8M

Use of IPO Proceeds:

$68M through Mid-2016

Additional PG324 Needs:

$40M through Mid-2017

PG324

Phase 3 registration trials

Direct clinical -

$27.5M

12 Months G&A and

working capital -

$12.5

Targeted Mid-2017:

Expected NDA Approval

for AR-13324

Expected NDA Filing for PG324

Stock & Market Cap Performance

10/31/13

$243.7

11/30/13

$261.8

12/31/13

$418.2

1/15/14

$499.9

(dollars

millions)

Market

Cap

Aerie –

Next Generation in Glaucoma Therapies

AR-13324

Novel Dual Action

All Products Fully

Owned by Aerie

PG324 Breakthrough

Triple Action

•

Fixed combination of AR-13324 and latanoprost

•

Potential for maximal IOP lowering

•

Expect P2b results mid-2014, P3 readiness

mid-2015

•

Inhibits ROCK and NET, may also lower EVP

•

Targets diseased tissue, consistent IOP lowering

•

Expect P3 efficacy data mid-2015, NDA filing mid-

2016

Large Market

Opportunity

•

$4.5B US/EU/JP Market with significant unmet needs

•

Multiple MOA’s, once daily, high efficacy and safety

•

Late-stage/potential blockbuster revenue opportunity

•

Full patent protection through at least

2030

•

Will retain US rights and plan to partner in

JP/EU

AERIE PHARMACEUTICALS INC - FORM 8-K - EX-99.1 - January 27, 2014

Attached files

AERIE PHARMACEUTICALS INC Reports