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Exhibit 99.1
Building a Major
Ophthalmic
Pharmaceutical Company
Aerie Pharmaceuticals, Inc.
Company Overview June 2-3, 2015
Important Information Aerie Pharmaceuticals, Inc.
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 RhopressaTM relate to the results in its first Phase 3 registration trial, Rocket 1, and for RoclatanTM relate to the results in its Phase 2b clinical 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 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 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.
2
Aerie – Building a Major Ophthalmic Pharmaceutical Company Aerie Pharmaceuticals, Inc.
Current Aerie Products: Once-Daily IOP-Lowering Eye Drops for Glaucoma
Triple action RhopressaTM
Inhibits ROCK and NET, lowers EVP, targets diseased tissue
First P3 missed primary endpoint, achieved secondary
Quadruple Action RoclatanTM
Fixed combination of RhopressaTM and latanoprost
P2b achieved all clinical endpoints, P3 to start mid-2015
Potentially most efficacious IOP-lowering therapy
Full patent protection through at least 2030; Blockbuster Potential
Pre-Clinical Research Findings
RhopressaTM shows potential to modify diseased tissue
May block fibrotic response in trabecular meshwork cells
May increase perfusion of the trabecular meshwork
AR-13154 shows potential for the treatment of wet AMD
May inhibit ROCK/JAK/PDGFR-ß
Lesion size reduction in rats exceeds market-leading product
These new preclinical discoveries represent potential breakthroughs
3
Triple-Action RhopressaTM Quadruple-Action RoclatanTM Aerie Pharmaceuticals, Inc.
IOP-Lowering Mechanisms
RhopressaTM:
ROCK inhibition relaxes TM, increases outflow
NET inhibition reduces fluid production
ROCK inhibition lowers Episcleral Venous Pressure (EVP)
RoclatanTM also adds latanoprost:
PGA receptor activation increases uveoscleral outflow
RhopressaTM
RKI NET
RKI
NET
Cornea
RKI
+ Outflow
+ Outflow
Trabecular Meshwork
Schlemm’s
Canal
-inflow
+ Outflow
PGA
RKI
NET
Episcleral
Veins
Uveoscleral Outflow
Latanoprost
Ciliary Processes
4
Aerie Products Cover the IOP-lowering Spectrum Aerie Pharmaceuticals, Inc.
Decreases Fluid Inflow/Production
(Ciliary Processes)
RhopressaTM
RoclatanTM
AA, BB, CAI
PGAs
Increases Fluid Outflow:
Secondary Drain
(Uveoscleral Pathway)
Increases Fluid Outflow:
Primary Drain-Trabecular Meshwork (TM);
Lowers EVP - (Episcleral Venous Pressure)
5
~80% of U.S. Glaucoma Patients Have IOPs that are
£ 26 mmHg at Time of Diagnosis
Aerie Pharmaceuticals, Inc.
The Baltimore Eye Survey
Baseline IOP*
>26 - <35 mmHg 20%
60% £21 mmHg (Normal Tension Glaucoma)
>21 - £26 mmHg 20%
~75% of Patients with IOP £24mmHg
* 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
6
Latanoprost and Timolol Show Reduced Efficacy at Lower Baseline IOPs
aerie pharmaceuticals, inc.
Latanoprost and timolol lose efficacy as baseline IOPs decline
Timolol at least 1 mmHg less effective than latanoprost across all published baselines
Timolol is the standard comparator for glaucoma Phase 3 trials
Diurnal IOP Change Month 6 (mmHg)
0
Timolol (n=369)
-2 Latanoprost (n=460)
-4
-6
-8
-10
-12
-14
-16
16 18 20 22 24 26 28 30 32 34 36 38
Untreated Diurnal IOP (mmHg)
Pooled data from three latanoprost registration studies. Hedman and Alm; European Journal Ophthalmology; 2000
7
RhopressaTM Registration Trial Design
aerie pharmaceuticals, inc.
“Rocket 1” RhopressaTM 0.02% QD 182 patients
90-Day Efficacy
Registration Trial Timolol BID 188 patients
“Rocket 2” RhopressaTM 0.02% QD ~230 patients
One Year Safety
(3 Mo. Interim RhopressaTM 0.02% BID* ~230 patients
Efficacy) Timolol BID ~230 patients
Registration Trial
“Rocket 3” RhopressaTM 0.02% QD ~90 patients
One Year Safety RhopressaTM 0.02% BID ~90 patients
Registration Trial
Canada Timolol BID ~60 patients
* PGAs have been shown to be less effective when dosed BID 8
RhopressaTM aerie pharmaceuticals, inc.
Rocket 1 Trial Design
Patients with open angle glaucoma (OAG) or ocular hypertension (OHT) with IOP >20 mmHg and < 27 mmHg
N=411 randomized at 36 sites (370 subjects per protocol)
Patients randomized
RhopressaTM 1:1 Timolol 0.5%
0.02% QD (PM) BID
Primary endpoint: Mean IOP at Weeks 2 and 6 and Day 90
9
Rocket 1 Trial Conduct aerie pharmaceuticals, inc.
Number of Early Terminations - 44 (Total RhopressaTM plus Timolol)
31 in RhopressaTM, 13 in Timolol
Major Reasons for Early Termination (Total RhopressaTM plus Timolol)
Adverse events* (55%)
Protocol violation (18%)
Withdrawal of consent (11%)
Lack of efficacy (7%)
Investigator decision (4%)
* Adverse Events for RhopressaTM included: Allergic conjunctivitis (2); Eyelid pruritus (2); Lacrimation increased (2); Angle closure glaucoma (1); Conj. hyperemia (1); Conj. edema (1); Conj. Vasc. Disorder (1); Eye irritation (1); Eye pain (1); Eye pruritus (1); Eyelid edema (1); Iris adhesions (1); Iris bombe (1); Vision blurred (1); Decreased visual acuity (1); Diarrhea (1); Dysphagia (1); Feel abnormal (1); Instill. site pain (1); Conjunctivitis (1); Hypersensitivity (1); Upper limb fracture (1); Corneal staining present (1); Prostate cancer (1); Dermatitis (1)
10
Rocket 1 Study Endpoints aerie pharmaceuticals, inc.
Efficacy:
The primary efficacy endpoint is the mean IOP at the following time points: 08:00, 10:00, and 16:00 at the Week 2, Week 6, and Day 90 visits
Secondary efficacy endpoints include:
IOP analysis stratified by baseline IOP above and below 24 mmHg
Mean change from baseline IOP
Mean percent change from diurnally adjusted baseline IOP
Mean diurnal and change from baseline diurnal IOP
Safety:
Ocular and systemic safety measures
11
Summary Of RhopressaTM Rocket 1 Efficacy Results Based On Different Baseline IOPs
aerie pharmaceuticals, inc.
Baseline IOP (mmHg) Non-inferiority Numerical Superiority
<27* Did not meet Met 2 time points
<26 Met Met 4 time points
<25 Met Met 7 time points
<24** Met All (9 time points)
<23 Met All (9 time points)
<22 Insufficient power All (9 time points)
* Primary endpoint
** Pre-specified secondary endpoint
12
Baseline IOP < 27 mmHg At All Time Points
aerie pharmaceuticals, inc.
IOP
AR-13324 0.02% (n=182)
Timolol 0.5% (n=188)
Pre-Study
8AM Qual 1
8AM 10AM 4PM
Baseline
8AM 10AM 4PM
Week 2
8AM 10AM 4PM
Week 6
8AM 10AM 4PM
Month 3
mmHg +/- SEM
15 16 17 18 19 20 21 22 23 24 25
13
Some Observations on Rocket 1 Non-Inferiority Miss
aerie pharmaceuticals, inc.
Achieved non-inferiority at <26mmHg, did not at <27mmHg
Inferiority driven by a subset of RhopressaTM patients
At higher baseline IOPs, potential for more severely diseased trabecular meshwork, limiting Rho Kinase inhibition benefit
Highly variable IOP
Many showed IOP swings of >3 mmHg between visits while on treatment
Notable variability in baseline IOP between RhopressaTM studies (Phase 2 vs. Rocket 1)
Some evidence of noncompliance, and site concentration of poorer performers
14
Baseline IOP < 24 mmHg (Pre-specified analysis) aerie pharmaceuticals, inc.
IOP
AR-13324 0.02% (n=76)
Timolol 0.5% (n=84)
Pre-Study
8AM Qual 1
8AM 10AM 4PM
Baseline
8AM 10AM 4PM
Week 2
8AM 10AM 4PM
Week 6
8AM 10AM 4PM
Month 3
mmHg +/- SEM
15 16 17 18 19 20 21 22 23 24 25
15
RhopressaTM Efficacy In Subjects On PGA Prior To Study: Baseline IOP < 24 mmHg aerie pharmaceuticals, inc.
Diurnal Average IOP
IOP mmHg + SEM
14 15 16 17 18 19 20 21 22 23
20.9 20.8
15.2 15.9 16.7
17.1 16.8 17.1
Baseline
Week 2
Week 6
Month 3
AR-13324 0.02% (n=40)
Timolol 0.5% (n=43)
Prior PGA use produced enhanced IOP-lowering with RhopressaTM at weeks 2 and 6
IOP lowering at month 3 equivalent to IOP lowering in non-PGA subjects
Prior PGA use had no effect on timolol efficacy
16
RhopressaTM Efficacy In Subjects Not On PGA Prior To Study: Baseline IOP < 24 mmHg aerie pharmaceuticals, inc.
Diurnal Average IOP
IOP mmHg + SEM
14 15 16 17 18 19 20 21 22 23
20.9 20.9
16.8 17.1 17.1
16.9 17.2 17.2
Baseline
Week 2
Week 6
Month 3
AR-13324 0.02% (n=36)
Timolol 0.5% (n=41)
No loss of efficacy seen from week 2 to month 3 for RhopressaTM or timolol
17
RhopressaTM IOP-lowering Effect Enhanced In Subjects On PGA Therapy Prior To Study Entry aerie pharmaceuticals, inc.
Prospective (pre-specified) analysis by pre-study medication status showed that prior PGA use enhanced RhopressaTM IOP-lowering at week 2 (p=0.003)
The PGA effect is lost over time, which we believe creates a false impression that RhopressaTM loses efficacy over time
The apparent PGA synergy with RhopressaTM is greatest at week 2 and lessens over time in the absence of PGA dosing
No evidence of enhanced efficacy in timolol subjects on PGA therapy prior to study entry
Retrospective analysis of Phase 2 trial results shows prior PGA use enhanced RhopressaTM IOP-lowering by 1 mmHg (p=0.007) and 1.2 mmHg (p=0.002) at weeks 2 and 4, respectively, relative to subjects not previously on PGA
18
RhopressaTM/PGA Synergy May Reflect
Complementary Actions on TM Extracellular Matrix
aerie pharmaceuticals, inc.
ROCK inhibition relaxes trabecular meshwork (TM) cells by reducing actin stress fibers and reducing production of ECM1
PGAs lower IOP by increasing ECM turnover in uveoscleral pathway- but subtle changes in TM pathway also observed with long-term dosing2
PGAs have no obvious effect on TM outflow on their own
Subtle PGA-induced changes in ECM may sensitize TM cells to the unique IOP-lowering effects of RhopressaTM
Daily exposure to both RhopressaTM and a PGA may result in ongoing synergy – and may also explain positive RoclatanTM P2b results
References: 1. Pattabiraman P and Rao P (2010) Am J Physiol Cell Physiol 298:C749-63;
2. Richter M et al. (2003) Invest Ophthalmol Vis Sci. 44:4419-26
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RhopressaTM Synergy with PGAs May Represent a Market Opportunity
aerie pharmaceuticals, inc.
FY 2014 U.S. Glaucoma Market = $2.2B; 33M TRx Market Share in TRx
Non-PGA Market PGA Market
2-3 Times Daily CAI Bimatoprost Once Daily
8% 10%
AA 10% Travoprost
9%
Fixed Combo 15%
33%
Latanoprost
14%
BB
PGA: Prostaglandin Analogue; BB: Beta Blocker; AA: Alpha Agonist; CAI: Carbonic Anhydrase Inhibitor Source: IMS MIDAS, IMS NPA
20
RhopressaTM Next Steps: Rocket 2
aerie pharmaceuticals, inc.
Trial is under way, patients still being treated
Data base not locked yet
Endpoints currently set same as Rocket 1
Originally scheduled P3 efficacy read-out Q3 2015
Pursuing with FDA changing secondary endpoint (< 24mmHg) to primary
- Rocket 1 performance in this range was very successful
- Represents a large portion of the market (~75%)
- FDA has allowed changes in the past
Considering < 25mmHg, which also performed well in Rocket 1
If endpoint is changed and patients need to be added for adequate powering of Rocket 2, efficacy read-out expected by YE 2015/Q1 2016
21
RhopressaTM Next Steps: Rocket 4
aerie pharmaceuticals, inc.
Planning to commence Rocket 4 in Q3 2015, an additional RhopressaTM trial in the U.S.
Baseline IOPs:
- Primary <24 mmHg or <25mmHg
- Pre-specified secondary <27 mmHg
- Considering stratification
- May enroll patients up to 30 mmHg
Efficacy evaluated at 3 months (primary) and 6 months (secondary)
Final design to be reviewed with FDA
Read-out expected in approximately one year
22
RoclatanTM Phase 2b Clinical Trial Design
aerie pharmaceuticals, inc.
Phase 2b Protocol
RoclatanTM 0.01% vs.
RoclatanTM 0.02% vs.
RhopressaTM 0.02% vs.
Latanoprost
All Dosed QD PM
~300 Patients
28 Days
Primary efficacy endpoint: Mean diurnal IOP on Day 29
Two concentrations of
RoclatanTM vs. RhopressaTM
0.02% and latanoprost
Trial design follows FDA requirement for fixed-dose combination
Statistically significant difference at measured time points
Higher combo efficacy vs. components of at least 1–3 mmHg, as previously accepted by FDA for product approval
23
RoclatanTM Phase 2b Clinical Trial Performance
aerie pharmaceuticals, inc.
Achieved primary efficacy measure
Superiority over each of the components on day 29
Achieved statistical superiority over the individual components at all time points
More efficacious than latanoprost by 1.6 – 3.2 mmHg
More efficacious than RhopressaTM by 1.7 – 3.4 mmHg
Main adverse event was hyperemia (eye redness):
Reported in 40 percent of patients
Mild for the large majority of patients
No systemic drug-related adverse events
24
0.02% RoclatanTM Achieved Statistical Superiority Over
Individual Components at All Time Points (p<0.001)
aerie pharmaceuticals, inc.
Mean IOP at Each Time Point Primary Efficacy Measure
29
28
27
26
25
24
mmHg +/- SEM 23
22
21
20
19
18
17
16
15
14
Pre- 8AM 8AM 10AM 4PM 8AM 10AM 4PM 8AM 10AM 4PM 8AM 10AM 4PM 8AM
Study Qual 1 Baseline Day 8 Day 15 Day 29 Day 30
0.02% RhopressaTM (n=78) 0.005% Latanoprost (n=73) 0.02% RoclatanTM (n=72)
RoclatanTM Phase 2b, Intent to Treat
25
RoclatanTM Phase 2b, ITT
aerie pharmaceuticals, inc.
Mean IOP (mmHg)
0.02% RoclatanTM (n = 72) latanoprost 0.005% RhopressaTM (n = 73) 0.02% (n = 78)
Mean Mean Difference* Mean Difference*
Day 8
8AM 17.0 19.6 -2.6 20.0 -3.1
10AM 15.6 18.3 -2.7 18.0 -2.4
4 PM 15.6 18.6 -3.1 17.9 -2.3
Day 15
8AM 16.5 19.6 -3.2 19.6 -3.1
10AM 15.8 18.3 -2.4 18.7 -2.8
4 PM 15.7 18.3 -2.6 18.4 -2.7
Day 29
8AM 16.9 19.2 -2.4 20.3 -3.4
10AM 15.9 17.7 -1.8 18.6 -2.7
4 PM 16.8 18.4 -1.6 18.5 -1.7
RoclatanTM:
Produced lowest IOP drop in any trial
Was superior to latanoprost by 1.6–3.2 mmHg (p<0.001)
Was superior to
RhopressaTM by 1.7–3.4 mmHg (p<0.001)
Impressive RhopressaTM performance
* Difference between 0.02% RoclatanTM and latanoprost or RhopressaTM
26
RoclatanTM Next Steps
aerie pharmaceuticals, inc.
Commencing “Mercury 1” in Q3 2015 in the U.S.
- Designed for superiority to individual components, similar to P2b
- Baseline IOP range tentatively > 20mmHg to <36mmHg, with stratified enrollment
- Multiple secondary endpoints
- Efficacy trial with one year safety
“Mercury 2” expected to commence in 2016 in the U.S.
- Expect same comparators as Mercury 1
- Three month efficacy study
“Mercury 3” expected to commence in 2016 in Europe
- Comparing to a leading combo product marketed in EU
- Efficacy study, duration TBD
27
Aerie Financial Resources
aerie pharmaceuticals, inc.
As of March 31, 2015 had $179.3M of cash and investments on balance sheet
Expected to fund Aerie operations for approximately the next 3 years
Proceeding with clinical path outlined for RhopressaTM and RoclatanTM, and continue to evaluate potential for pre-clinical Aerie molecules and outside opportunities
28
Summary
aerie pharmaceuticals, inc.
Key Clinical Priorities
RhopressaTM: Rocket 2 endpoint discussion with FDA
Rocket 4 commencement Q3 2015
RoclatanTM: Mercury 1 commencement Q3 2015
Research Initiatives
RhopressaTM disease modification and neuro-protection
AR-13154 potential in wet AMD, etc.
Evaluating Aerie’s 3,000+ owned molecules
Potential Business Development Opportunities
Evaluating additions to ophthalmic product pipeline
Exploring drug delivery opportunities – front and back of eye
29
Building a Major Ophthalmic Pharmaceutical Company