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

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8-K - FORM 8-K - AERIE PHARMACEUTICALS INC	d653928d8k.htm
EX-99.1 - EX-99.1 - AERIE PHARMACEUTICALS INC	d653928dex991.htm

Company Overview

January 9, 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

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.

•

Large

Market

Opportunity

$4.5B

US/EU/JP

Market

Significant

unmet

needs

50%

patients

require

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

two

decades

Potential

additional

new

MOA

–

lowering

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 –

Next Generation in Glaucoma Therapies

Establish Commercial

Capabilities

Expand Commercial

Offerings

Maximize Portfolio

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

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’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

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

ROCK/NET: Rho Kinase / Norepinephrine Transporter

Aerie’s Pipeline is Advancing Rapidly

Dual-Action

Triple-Action

P2b 2014

Dual-Action

Clinical Trials for Glaucoma Recruit Quickly

and Have Clear Endpoints

Preclinical

IND

Phase

1/2a

Phase

NDA

AR-13324

ROCK/NET

Inhibitor

PG324

Combination

-13324 +

Latanoprost

AR-13533

ROCK/NET

Generation

2016

2014

P3 Prep

To mid 2015

Glaucoma Market

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

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

Triple-Action

PG324

Once-Daily

Dual-Action

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

Dual Action

AR-13324

The Competitive Landscape

Clinical

Efficacy

Dosing/

Day

Tolerability

Peak Product

Sales*

(US/EU)

Targeting

Diseased

Tissue

PGA

High

Hyperemia

Iris Color

$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;

Moderate

Hyperemia

Yes

PG324

Potentially

Highest

TBD

Yes

* Peak 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

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

AR-13324 is the only new MOA drug 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

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

PG324 Triple-Action

Future non-PGA drug of choice

Future drug of choice for all

glaucoma patients

Aerie Franchise: Highly Differentiated

Product Profiles

PGA: Prostaglandin Analogue

New Dual-Action Drug Class:

AR-13324

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)

AR-13324 Mechanisms of Action

AR-13324

NET

RKI

NET

RKI

Trabecular

Meshwork

ROCK inhibition relaxes TM

NET inhibition reduces fluid production

ROCK inhibition may lower Episcleral

Venous Pressure (EVP)

Episcleral

Veins

Schlemm’s

Canal

Uveoscleral

Outflow

Ciliary Processes

Cornea

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% 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

AR-13324 Phase I PK Results Support

Potential New MOA

healthy

volunteers

with

normotensive

IOP

–

mmHg,

AR-13324

lowered

IOP

over

30%

Currently available glaucoma drugs are typically less effective

at lower IOPs

High

efficacy

these

baselines

suggest

lowering

EVP

–

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 (

mmHg)

PK analysis showed low systemic exposure and no drug-

related effects on systemic safety parameters

AR-13324 Demonstrated Strong IOP Lowering

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

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 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

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

Baseline

22 –

36 mmHg

(n=221)

Baseline

22 –

26 mmHg

(n=106)

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 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

AR-13324 0.02%

dosed QD PM

vs.

Timolol

dosed BID

~1200 patients

90 days efficacy

100 patients for 1 year

safety

Triple-Action Fixed Combination:

PG324

AR-13324 FIXED COMBINATION WITH

LATANOPROST

Triple-Action PG324 Combination Product

AR-13324

Ciliary Processes

Latanoprost

Uveoscleral

Outflow

Cornea

Outflow

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

Potential for maximal medical

therapy in a single eye drop

Latanoprost

0.02% PG324

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

Dose

Day

IOP

measured

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

PG324 Phase 2b Clinical Trial Design

Phase 2b Protocol

PG324 0.01%

vs.

PG324 0.02%

vs.

AR-13324 0.02%

vs.

Latanoprost

All dosed QD PM

~300 patients

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

Key Milestones

H1 2014

H2 2014

H2 2015

H1 2015

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

•

Large

Market

Opportunity

$4.5B

US/EU/JP

Market

Significant

unmet

needs

50%

patients

require

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

two

decades

Potential

additional

new

MOA

–

lowering

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 –

Next Generation in Glaucoma Therapies

AERIE PHARMACEUTICALS INC - FORM 8-K - EX-99.2 - January 9, 2014

Attached files

AERIE PHARMACEUTICALS INC Reports