AERIE PHARMACEUTICALS INC - FORM 8-K - EX-99.2

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8-K - FORM 8-K - AERIE PHARMACEUTICALS INC	d724124d8k.htm
EX-99.1 - EX-99.1 - AERIE PHARMACEUTICALS INC	d724124dex991.htm

Aerie Pharmaceuticals, Inc.

Company Overview

May 12, 2014

Leading

Innovation in

Glaucoma

The Next

Generation

Exhibit 99.2

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 data

results for our Rhopressa™ product candidate relate to the results in our Phase 2 clinical trials. Our

product candidate Roclatan™ is currently in Phase 2 clinical trials.

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 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

Rhopressa

Triple Action

All Products Fully

Owned by Aerie

Roclatan

Quadruple Action

•

Fixed

combination

Rhopressa

and

latanoprost

•

Potential for maximal IOP lowering

•

Expect P2b results late June or early July 2014,

P3 readiness mid-2015

•

Inhibits ROCK and NET, targets diseased tissue

•

Consistent IOP lowering,

lowers EVP

•

Expect P3 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 at least 2030

•

Will retain US rights and plan to partner in

JP/EU

Largest Rx market in ophthalmology: $4.5B US/EU/JP

US Glaucoma patients: 2.7M growing to 4.3M by 2030

Patients on more than one drug to control disease: 50%

No commonly prescribed drugs:

target the diseased tissue

relax the main fluid drain and lower EVP

Glaucoma Market

National Eye Institute

show consistent efficacy across a broad range of

pressures

Expanding Market

Unmet Needs

increase

Decreases Fluid

Inflow/Production

(Ciliary Processes)

Aerie Covers the IOP-lowering Spectrum

Increases Fluid Outflow:

Secondary Drain

(Uveoscleral Pathway)

Increases Fluid Outflow:

Primary

Drain

(Trabecular

Meshwork);

Lowers EVP

(Episcleral Venous Pressure)

Rhopressa™

Roclatan™

AA, BB, CAI

PGAs

Once Daily

2-3 Times

Daily

Non-PGA

Market

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

PGA

Market

FY 2013 US Glaucoma Market = $2.0B; 31.5M TRx

Market Share in TRx

Current Product Dashboard

Bimatoprost,

11%

Travoprost,

10%

Latanoprost,

32%

BB, 15%

BB Fixed

Combo, 14%

AA, 10%

CAI, 8%

Aerie Competitive Advantage

Clinical Efficacy

Doses

Per

Day

Tolerability

Peak Product

Sales*

Targeting

Diseased

Tissue

Rhopressa

High; Consistent

Hyperemia

Yes

Roclatan

Potentially

Highest

TBD

Yes

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

* Peak worldwide sales for best-in-class franchise.

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

OPA-6566 (Acucela)

Adenosine-A2a agonist

(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

Rhopressa

the

only

new

MOA

product

dosed

once-daily

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

Future drug of choice for the

80% of patients with IOP of

26 mmHg or less

Future product of choice for

patients requiring maximal IOP

lowering

Triple-Action

Rhopressa

Quadruple-Action

Roclatan

Aerie Market Positioning

Also for PGA users as add-on

therapy

Also for PGA non-responders and

those with tolerability concerns

Also for patients with low-tension

glaucoma

Efficacy potentially superior to all

currently marketed drugs

For patients with IOPs above

26 mmHg

Also for patients at any IOP with

significant disease progression

Triple-Action Rhopressa

Cornea

Uveoscleral

Outflow

Rhopressa

Trabecular

Meshwork

ROCK inhibition relaxes TM, increases outflow

NET inhibition reduces fluid production

ROCK inhibition lowers EVP

Episcleral

Veins

Schlemm’s

Canal

Mechanisms of Action:

RKI

NET

Ciliary Processes

RKI

NET

RKI

13 glaucoma NCEs advanced from Phase 2 to Phase 3

since 1970s -

All approved

Once-daily PM dosing of

0.02% Rhopressa

is highly

effective

IOP -5.7 and -6.2 mmHg on D28

and D14

Consistent efficacy through D28

Rhopressa

efficacy results

within ~1 mmHg of

latanoprost

Favorable tolerability profile

No systemic side effects

Rhopressa

Efficacy 8 AM

Diurnal Average IOP -

Entry IOP 22-36 mmHg (n=221)

Rhopressa

0.01% (n=74)

Rhopressa

0.02% (n=71)

Latanoprost

(n=76)

Rhopressa

: Strong IOP Lowering in Phase 2b

Rhopressa

0.02% q.d.

Rhopressa

and latanoprost

clinically and statistically

equivalent in patients with

moderately elevated IOPs

of 22 -

26 mmHg

Latanoprost loses ~1 mmHg

efficacy

Rhopressa

maintains consistent

efficacy

Latanoprost does not target the

diseased tissue –

Rhopressa

does

Phase

baseline

IOP

entry

requirements:

24,

22,

mmHg

(8am,

10am,

4pm)

Differentiated Rhopressa

Efficacy Profile

Latanoprost

Baseline

22 –

26 mmHg

(n=106)

Latanoprost

0.02%

Rhopressa™

Baseline

22 –

36 mmHg

(n=221)

Full Patient Population

Moderately Elevated IOP

0.02%

Rhopressa™

Phase 2b data provided

first sign of EVP-lowering:

Phase 1 study in low baseline IOP subjects:

Preclinical in vivo study:

Note: EVP contributes up to half of IOP in normotensive subjects

Consistent efficacy across baseline IOPs

Lowered average IOP by over 30%

from 16 to 11 mmHg

Lowered EVP by 35%

Rhopressa

™

EVP-Lowering

Breakthrough

10,444 individuals were

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

~80% of Glaucoma IOPs Are

26 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

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%

Latanoprost and Timolol Show Reduced Efficacy

at Lower Baseline IOPs

Latanoprost and timolol

lose efficacy as baseline

IOPs decline

Timolol at least 1 mmHg

less effective

than

latanoprost across all

published baselines

Rhopressa

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)

™

Rhopressa

Registration Trial Overview

Primary efficacy endpoint: IOP at all time points through Day 90

Non-inferiority design vs. timolol

95%

within

1.5

mmHg

all

time

points,

within

1.0

mmHg

majority

time points

Planned entry IOP: Over 20 mmHg and less than 27 mmHg

FDA

has

agreed

Aerie

proposal

for

entry

IOPs

with

expected

impact

label

Rhopressa

non-inferior to latanoprost at entry IOPs of 22-26 mmHg in

Phase 2b

•

FDA discussions on Phase 3 design now complete

Combined trials to include approximately 1,300 total patients

100 patients of 12 month safety data needed for NDA filing

Rhopressa

Registration Trial Design

“Rocket 1”

90-Day Efficacy Registration Trial:

Rhopressa

0.02%

~200

patients

Timolol

BID

~200

patients

“Rocket 2”

One Year Safety (3 mo. interim efficacy) Registration Trial:

Rhopressa

0.02%

Rhopressa

0.02%

BID

~230

patients

Timolol

BID

~230

patients

PGAs have been shown to be less effective when dosed BID

“Rocket 3”

One Year Safety-Only Study –

Canada:

Rhopressa

0.02%

~90

patients

Rhopressa

0.02%

BID

~90

patients

Timolol

BID

~60

patients

~230 patients

Ciliary Processes

Cornea

Uveoscleral

Outflow

NET

RKI

NET

RKI

Trabecular

Meshwork

Episcleral

Veins

Schlemm’s

Canal

Latanoprost

Quadruple-Action Roclatan

Rhopressa

MOAs:

RKI

PGA

RHOPRESSA

FIXED

COMBINATION

WITH

LATANOPROST

ROCK

inhibition

relaxes

TM,

increases

outflow

NET

inhibition

reduces

fluid

production

ROCK

inhibition

lowers

EVP

PGA receptor activation

increases uveoscleral

outflow

Roclatan

: Quadruple-Action Fixed Combination

First product to lower IOP through

all three known mechanisms, and

also lowers 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

Day 2 -

No IOP

measured

Dose

0.02% Roclatan

vs. Latanoprost

Primates (n=6/group)

Latanoprost

Roclatan

Roclatan

Phase 2b Clinical Trial Design

Primary efficacy endpoint: Mean

diurnal IOP on Day 28

Two concentrations of Roclatan

vs.

Rhopressa

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 2b Protocol

Roclatan

0.01%

vs.

Roclatan

0.02%

vs.

Rhopressa

0.02%

vs.

Latanoprost

All dosed QD PM

~300 patients

28 days

June or early July 2014

Results expected to be released late

Key Milestones Financed by IPO Proceeds

Started Phase 2b clinical trial

H1 2014

H2 2014

H2 2015

H1 2015

Start Phase 3 registration

trials

Late June or Early July 2014:

Roclatan

Results from Phase 2b

expected

Efficacy results from

Phase 3 expected

H1 2016

Phase 3 prep

NDA filing expected

Early-2014: Roclatan

Early 3Q 2014: Rhopressa

Mid-2015: Rhopressa

Mid-2016: Rhopressa

Mid-2015: Roclatan

Rhopressa

Phase 3 registration trials

Direct clinical -

$25.0M

Non-clinical -

$12.4M

Roclatan

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 Roclatan

Needs:

$40M through Mid-2017

Roclatan

Phase 3 registration trials

Direct clinical -

$25.5M

Non-clinical -

$2.0M

12 Months G&A and

working capital -

$12.5

Targeted

Mid-2017:

Expected

NDA

Approval

for

Rhopressa

Expected

NDA

Filing

for

Roclatan

Financial Summary

Market

Cap

(dollars

millions)

10/31/13

$243.7

12/31/13

$418.2

5/9/14

$348.0

Aerie Stock & Market Cap Performance

AERIE PHARMACEUTICALS INC - FORM 8-K - EX-99.2 - May 12, 2014

Attached files

AERIE PHARMACEUTICALS INC Reports