Attached files
| file | filename |
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| 8-K - 8-K - AERIE PHARMACEUTICALS INC | d636249d8k.htm |
| EX-99.1 - EX-99.1 - AERIE PHARMACEUTICALS INC | d636249dex991.htm |
 Leading Innovation in Glaucoma
Leading Innovation in Glaucoma
The Next Generation
The Next Generation
Company Overview
December 4, 2013
Exhibit 99.2 |
 2
Important Information
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. |
 3
•
Large Market Opportunity -
$4.5B US/EU/JP Market
-
Significant unmet needs -
50% of patients require more 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 in two decades
•
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 –
Aerie –
Next Generation in Glaucoma Therapies
Next Generation in Glaucoma Therapies |
 4
Aerie Strategy Overview
Aerie Strategy Overview
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 |
 5
Aerie’s Experienced Leadership Team
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
Investors |
 6
•
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
Aerie is a Leader in Rho Kinase R&D
ROCK/NET: Rho Kinase / Norepinephrine Transporter |
 7
Aerie’s Pipeline is Advancing Rapidly
Dual-Action
2014
Triple-Action
P2b 2014
P3 Prep
To mid 2015
2016
Dual-Action
Clinical Trials for Glaucoma Recruit Quickly
and Have Clear Endpoints |
 8
Glaucoma Market |
 9
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 |
 10
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 |
 11
increase
Inflow (fluid production)
AA, BB, CAI
decrease
increase
Novel Dual-
and Triple-Action Products Address
All IOP Control Mechanisms
Triple Action
PG324
Outflow
Secondary Drain
(Uveoscleral)
Outflow
Primary Drain
(Trabecular Meshwork) |
 12
The Competitive Landscape
Clinical
Efficacy
Dosing/
Day
Tolerability
Peak Product
Sales*
(US/EU)
Targeting
Diseased
Tissue
PGA
High
1x
Hyperemia
Iris Color
$1.7B
No
Beta
Blocker
Moderate
2x
Cardiopulmonary
Contraindications
>$500M
No
CAI
Low
2 -
3x
Sulfonamide
Contraindication
Bitter Taste
>$500M
No
Alpha
Agonist
Low
2 -
3x
Allergy
Drowsiness
>$400M
No
AR-13324
High;
Moderate
1x
Hyperemia
Yes
PG324
Potentially
Highest
1x
TBD
Yes
* Peak sales for best-in-class franchise |
 13
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
|
 14
Single drop, once-daily
1 drug -
2 MOAs
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
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 |
 15
New Dual-Action Drug Class:
AR-13324 |
 16
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) |
 17
~80% of Glaucoma IOPs Are 26
mmHg at Time
of Diagnosis 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)
Baltimore Eye Survey |
 18
AR-13324 Demonstrated Strong IOP Lowering
AR-13324 Demonstrated Strong IOP Lowering
in Phase 2b
in Phase 2b
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
Diurnal Average IOP -
Entry IOP 22-36 mmHg (n=221)
13 glaucoma NCEs advanced from Phase 2 to Phase 3
since 1970s -
All approved
AR-13324 Efficacy at 8 AM |
 19
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 |
 20
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
2b
baseline
IOP
entry
requirements:
24,
22,
22
mmHg
(8am,
10am,
4pm)
Differentiated AR-13324 Efficacy
Profile
Differentiated AR-13324 Efficacy
Profile Informs
Phase 3 Study Design Informs Phase 3 Study Design
Baseline
Baseline
22 36 mmHg
22 36 mmHg
(n=221)
(n=221)
Baseline
Baseline
22 26 mmHg
22 26 mmHg
(n=106)
(n=106)
Full Patient Population
Moderately Elevated IOP
0.02%
AR-13324
0.02%
AR-13324
Latanoprost
Latanoprost |
 21
Latanoprost and Timolol Show Reduced Efficacy
Latanoprost and Timolol Show Reduced Efficacy
at Lower Baseline IOPs
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
0
-2
-4
-6
-8
-10
-12
-14
-16
Timolol (n = 369)
Latanoprost (n = 460)
16
18
20
22
24
26
28
30
32
34
36
38
Untreated diurnal IOP (mmHg) |
 22
AR-13324 Registration Trial Design
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 26-30 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
Phase 3 Protocol
AR-13324 0.02%
AR-13324 0.02%
dosed QD PM
dosed QD PM
vs.
vs.
Timolol
Timolol
dosed BID
dosed BID
~1200 patients
~1200 patients
90 days efficacy
90 days efficacy
100 patients for 1 year
100 patients for 1 year
safety
safety |
 23
Triple-Action Fixed Combination:
Triple-Action Fixed Combination:
PG324
PG324 |
 24
Triple-Action PG324 Combination Product
Triple-Action PG324 Combination Product
Ciliary Processes
Cornea
Uveoscleral
Outflow
Latanoprost
TM
Outflow
AR-13324 FIXED COMBINATION WITH
LATANOPROST
1.
ROCK inhibition restores TM outflow
2.
NET inhibition reduces fluid production
3.
PGA receptor
activation
increases
uveoscleral
outflow
AR-13324
NET
NET
RKI
RKI
NET
NET
RKI
RKI |
 25
PG324: Triple-Action Fixed Combination
PG324: Triple-Action Fixed Combination
First product to lower IOP through
all three known mechanisms
-
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
Day 2 -
No IOP
measured
Dose
Dose
Dose
Latanoprost
0.02% PG324
PG324 vs. Latanoprost, Primates (n=6/group) |
 26
PG324: A Breakthrough Triple-Action Product Candidate
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
|
 27
PG324 Phase 2b Clinical Trial Design
PG324 Phase 2b Clinical Trial Design
Phase 2b Protocol
Phase 2b Protocol
PG324 0.01%
PG324 0.01%
vs.
vs.
PG324 0.02%
PG324 0.02%
vs.
vs.
AR-13324 0.02%
AR-13324 0.02%
vs.
vs.
Latanoprost
Latanoprost
All dosed QD PM
All dosed QD PM
~300 patients
~300 patients
28 days
28 days |
 28
Key Milestones
Key Milestones
Early-2014: PG324
Start Phase 2b clinical trial
H1 2014
H1 2014
H2 2014
H2 2014
H2 2015
H2 2015
H1 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
H1 2016
Mid-2015: PG324
Phase 3-prep
Mid-2016: AR-13324
NDA filing expected |
 29
•
Large Market Opportunity -
$4.5B US/EU/JP Market
-
Significant unmet needs -
50% of patients require more 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 in two decades
•
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 –
Aerie –
Next Generation in Glaucoma Therapies
Next Generation in Glaucoma Therapies |