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| 8-K - FORM 8-K - AERIE PHARMACEUTICALS INC | d707759d8k.htm |
 Leading Innovation in Glaucoma
The Next Generation
Company Overview
April 9, 2014
Exhibit 99.1 |
 2
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
TM
product candidate relate to the results in our Phase 2 clinical trials. Our
product candidate Roclatan
TM
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.
Important Information |
 3
Rhopressa
TM
Triple Action
All Products Fully
Owned by Aerie
Roclatan
TM
Quadruple Action
•
Fixed combination of Rhopressa
TM
and latanoprost
•
Potential for maximal IOP lowering
•
Expect P2b results early 3Q 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
Aerie –
Next Generation in Glaucoma Therapies |
 4
•
Largest Rx market in ophthalmology: $4.5B US/EU/JP
•
US Glaucoma patients: 2.7M growing to 4.3M by 2030
1
•
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
•
show consistent efficacy across a broad range of
Glaucoma Market
1
National Eye Institute
Expanding Market
Unmet Needs
pressures |
 5
Fluid Inflow / Production:
Ciliary Processes
AA, BB, CAI
Decrease
Fluid
Production
Increase
Fluid
Outflow
Via
Uveoscleral
Pathway
Aerie Covers the IOP-lowering Spectrum
Fluid Outflow:
Secondary Drain
(Uveoscleral)
Fluid
Outflow:
Primary Drain (TM);
Lower EVP
(Episcleral Venous Pressure)
EVP
Decrease
Increase
Fluid
Outflow
Via
Trabecular
Meshwork
(TM)
Rhopressa
TM
Roclatan
TM
PGAs |
 6
Once Daily
2-3 Times
Daily
Non-PGA
Market
PGA: Prostaglandin Analogue; BB: Beta Blocker; AA: Alpha Agonist; CAI: Carbonic
Anhydrase Inhibitor PGA
Market
Current Product Dashboard
FY 2013 US Glaucoma Market = $2.0B; 31.5M TRx
Market Share in TRx
Bimatoprost,
11%
Travoprost,
10%
Latanoprost,
32%
BB, 15%
BB Fixed
Combo, 14%
AA, 10%
CAI, 8% |
 7
Aerie Competitive Advantage
Clinical Efficacy
Doses
Per
Day
Tolerability
Peak Product
Sales*
(US/EU)
Targeting
Diseased
Tissue
Rhopressa
TM
High; Consistent
1
Hyperemia
Yes
Roclatan
TM
Potentially
Highest
1
TBD
Yes
PGA
High
1
Hyperemia
Iris Color Change
Other Cosmetic
$1.7B
No
Beta
Blocker
Moderate
2
Cardiopulmonary
Contraindications
>$500M
No
CAI
Low
2 -
3
Sulfonamide
Contraindication
Bitter Taste
>$500M
No
Alpha
Agonist
Low
2 -
3
Allergy
Drowsiness
>$400M
No
* Peak sales for best-in-class franchise |
 New PGAs -
not usable as add-on to current PGAs
Glaucoma Competitors in Pipeline
Rhopressa
TM
is the only new MOA product 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
OPA-6566
(Acucela
)
Adenosine
-A2a agonist
(bid)
Phase 1/2
SYL040012 (Sylentis)
RNAi
beta
blocker (bid)
Phase 2
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 MOAs
New PGAs
8 |
 9
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
TM
Quadruple-Action Roclatan
TM
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
TM
Rhopressa
TM
Trabecular
Meshwork
1.
ROCK inhibition relaxes TM, increases outflow
2.
NET inhibition reduces fluid production
3.
ROCK inhibition lowers EVP
Episcleral
Veins
Schlemm’s
Canal
Mechanisms of Action:
Ciliary Processes
Uveoscleral
Outflow
Cornea
10 |
 11
Rhopressa
TM
:
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%
Rhopressa
TM
is
highly effective
-
IOP -5.7 and -6.2 mmHg on
D28 and D14
-
Consistent efficacy through D28
Rhopressa
TM
efficacy
results within ~1 mmHg of
latanoprost
Favorable tolerability profile
No systemic side effects
Rhopressa
TM
Efficacy 8 AM
Rhopressa
TM
0.01% (n=74)
Rhopressa
TM
0.02% (n=71)
Latanoprost
(n=76) |
 12
Rhopressa
TM
and latanoprost
clinically and statistically
equivalent in patients with
moderately elevated
IOPs of 22
- 26 mmHg
Latanoprost loses ~1 mmHg
efficacy
Rhopressa
TM
maintains consistent
efficacy
Latanoprost does not target the
diseased tissue –
Rhopressa
TM
does
Phase
2b
baseline
IOP
entry
requirements:
24,
22,
22
mmHg
(8am,
10am,
4pm)
Differentiated Rhopressa
TM
Efficacy Profile
Baseline
22 –
26 mmHg
(n=106)
Baseline
22 –
36 mmHg
(n=221)
Full Patient Population
Moderately Elevated IOP |
 13
Consistent efficacy across baseline IOPs
Lowered average IOP by over 30%
from 16 to 11 mmHg
Lowered EVP by 35%
Rhopressa
TM
EVP-Lowering Breakthrough
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
|
 14
Baseline IOP
(mmHg)
Percentage of POAG
Patients Identified
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
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 |
 15
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
TM
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
Timolol (n = 369)
Latanoprost (n = 460)
Untreated diurnal IOP (mmHg) |
 16
Rhopressa
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
-
Rhopressa
TM
non-inferior to latanoprost at entry
IOPs of 22-26 mmHg in Phase 2b
Phase 3 Protocol
Rhopressa
TM
0.02%
dosed QD PM
vs.
Timolol
dosed BID
~1200 patients
90 days efficacy
100+
patients for
1 year safety
TM |
 Quadruple-Action Roclatan
TM
RHOPRESSA
TM
FIXED COMBINATION WITH
LATANOPROST
1.
2.
3.
4.
Rhopressa™
MOAs:
Ciliary Processes
Cornea
Uveoscleral
Outflow
Trabecular
Meshwork
Episcleral
Veins
Schlemm’s
Canal
Latanoprost
ROCK inhibition relaxes TM, increases outflow
NET inhibition reduces fluid production
ROCK inhibition lowers EVP
PGA receptor activation
increases uveoscleral
outflow
|
 18
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
Dose
Dose
0.02% Roclatan
vs. Latanoprost
Primates (n=6/group)
Latanoprost
Roclatan™
TM
TM |
 19
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
Roclatan™
Phase
2b
Clinical
Trial
Design |
 20
Early-2014: Roclatan™
Started Phase 2b clinical trial
H1 2014
H1 2014
H2 2014
H2 2014
H2 2015
H2 2015
H1 2015
H1 2015
Early 3Q 2014: Rhopressa™
Start Phase 3 registration
trials
Early 3Q 2014: Roclatan™
Results from Phase 2b
expected
Mid-2015: Rhopressa™
Efficacy results from
Phase 3 expected
H1 2016
H1 2016
Mid-2015: Roclatan™
Phase 3 prep
Mid-2016: Rhopressa™
NDA filing expected
Key Milestones Financed by IPO Proceeds |
 21
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
Financial Summary
Expected
NDA
Approval
for
Rhopressa™
Expected
NDA
Filing
for
Roclatan™
Targeted Mid-2017: |
 22
Market
Cap
(dollars
in
millions)
10/31/13
-
$243.7
12/31/13
-
$418.2
4/4/14
-
$444.6
Aerie Stock & Market Cap Performance |