EX-99.2 3 d914487dex992.htm EX-99.2 EX-99.2
1
Rhopressa
TM
(ROCK-NET Inhibitor)
Rho Kinase Elevated Intraocular Pressure Treatment Trial
(Rocket 1) Phase 3 Topline Results
1
Exhibit 99.2

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.
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. 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.

3
Background
There are three Phase 3 registration trials for Rhopressa
TM
, “Rocket 1,”
a 90-day efficacy trial, the results of which are reported in this
presentation, “Rocket 2,”
a 12-month safety trial with a 90-day interim
efficacy readout expected third quarter 2015, and “Rocket 3,”
a safety-
only study being conducted in Canada.
Rocket 1 clinical trial evaluated
•
the
ocular
hypotensive
efficacy
of
Rhopressa
TM
0.02%,
dosed
QD,
versus Timolol, 0.5%, dosed BID, in patients with elevated
intraocular pressure (OAG and OHT) for 90 days
•
the
ocular
and
systemic
safety
of
Rhopressa
TM
,
0.02%
for
90
days

4
4
Patients randomized
1:1
Primary endpoint: Mean IOP at
Weeks 2 and 6 and Day 90
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)
Timolol 0.5%
BID
Rhopressa
TM
Rocket 1 Trial Design
Rhopressa
TM
0.02% QD (PM)

5
Study Endpoints
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

6
Trial Conduct
Number
of
Patients
Randomized
-
411
Number
of
Early
Terminations
–
44
(Combined
Rhopressa
TM
and
Timolol)
31 in Rhopressa
TM
, 13 in Timolol
Major
Reasons
for
Early
Termination
(Combined
Rhopressa
TM
and
Timolol)
Adverse events (55%)
Protocol violation (18%)
Withdrawal of consent (11%)
Lack of efficacy (7%)
Investigator decision (4%)

7
Primary Endpoint: Topline Summary
Per Protocol population (baseline IOP < 27 mmHg)
Rhopressa
TM
did
not
meet
criteria
for
non-inferiority
to
Timolol
Rhopressa
TM
mean
difference
from
Timolol
ranged
from
–0.4
to
+1.3 mmHg
Inferiority
was
driven
by
a
subset
of
Rhopressa
TM
patients
losing
efficacy over time (~ 20%)

8
Rhopressa
TM
Rocket 1, Per Protocol
* Missed upper 95% CI criteria (<1.5 mmHg)
Baseline IOP < 27 mmHg at all time points
TM
Baseline
8 AM
23.4
23.4
10 AM
22.3
21.9
4 PM
21.8
21.5
Day 15
8 AM
18.7
18.3
0.4
(-0.3, 1.0)
10 AM
17.3
17.6
-0.3
(-0.9, 0.4)
4 PM
17.2
17.7
-0.5
(-1.1, 0.2)
Day 43
8 AM
19.4
18.2
1.1
(0.4, 1.8)*
10 AM
18.1
17.4
0.7
(0.0, 1.4)
4 PM
17.9
17.7
0.2
(-0.5, 0.8)
Day 90
8 AM
19.8
18.5
1.3
(0.6, 2.0)*
10 AM
18.9
18.0
1.0
(0.3, 1.7)*
4 PM
18.5
17.7
0.7
(0.1, 1.4)
(95% CI)
Timolol
Rhopressa
–
Mean IOP
Rhopressa
TM
N=182
Timolol
N=188
Mean
Difference
95% CI

9
Mean Baseline IOP < 27 mmHg at All Time Points

10
Rhopressa
TM
Subjects With Decreasing Efficacy
Over Time
(Decreasing efficacy over time defined as greater than 3 mmHg)
Correlation With Entry Baseline IOP
Possible explanations for “drifter”
subjects
n= number of subjects per protocol
•
Severe trabecular meshwork damage (e.g. duration of disease)
•
Dosing compliance
•
Concomitant medications
•
<27 mmHg –
36 drifters (n=182, 19.8%)
•
<26 mmHg –
18 drifters (n=133, 13.5%)
•
<24 mmHg –
5 drifters  (n=76, 6.6%)

11
Lower baseline populations (IOP < 26 mmHg and IOP < 24 mmHg)
IOP
<
26
mmHg
cohort
(n=277),
Rhopressa
TM
met
non-inferiority
criteria at all 9 time points and was numerically superior to Timolol
at the majority of time points
IOP
<
24mmHg
cohort
(n=160),
Rhopressa
TM
met
non-inferiority
criteria at all 9 time points and was numerically superior to Timolol
at all 9 time points (Pre-specified analysis)
Subjects with loss of efficacy reduced by 50% in baseline IOP < 26
mmHg cohort and by 86% in baseline IOP < 24 mmHg
Topline Summary (Lower Baseline IOP)

12
Rhopressa
TM
Rocket 1, Baseline IOP < 26 mmHg
Rhopressa
TM
met non-inferiority criteria at all time points and
numerical superiority at majority of time points
Baseline
8 AM
22.7
22.8
10 AM
21.7
21.3
4 PM
21.0
20.8
Day 15
8 AM
17.8
18.0
-0.2
(-0.8,0.4)
10 AM
16.5
17.1
-0.6
(-1.2,0.0)
4 PM
16.4
17.3
-0.9
(-1.5,-0.2)
Day43
8 AM
18.3
17.9
0.4
(-0.3,1.1)
10 AM
17.3
17.1
0.2
(-0.5,0.8)
4 PM
17.0
17.4
-0.4
(-1.1,0.3)
Day 90
8 AM
18.8
18.3
0.5
(-0.2,1.3)
10 AM
17.9
17.7
0.2
(-0.5,0.9)
4 PM
17.4
17.4
-0.01
(-0.7,0.6)
Mean IOP
Rhopressa
TM
N=133
Timolol
N=144
Rhopressa
(95% CI)
Timolol
–
TM
Mean
Difference
95% CI

13
Baseline IOP < 26 mmHg

14
Rhopressa
TM
Rocket 1, Baseline IOP < 24 mmHg
Rhopressa
TM
met
non-inferiority
criteria
at
all
9
time
points
was
numerically superior to Timolol at all 9 time points
(Pre-specified analysis)
Baseline
8 AM
21.8
21.9
10 AM
20.7
20.6
4 PM
20.2
20.1
Day 15
8 AM
16.7
17.3
-0.6
(-1.3, 0.1)
10 AM
15.6
16.8
-1.2
(-1.9, -0.4)
4 PM
15.6
16.9
-1.4
(-2.1, -0.7)
Day 43
8 AM
17.2
17.4
-0.2
(-0.9, 0.6)
10 AM
16.3
16.8
-0.4
(-1.2, 0.3)
4 PM
15.9
16.8
-0.9
(-1.7, -0.1)
Day 90
8 AM
17.3
17.6
-0.3
(-1.0, 0.5)
10 AM
16.6
16.9
-0.3
(-1.1, 0.5)
4 PM
16.7
17.0
-0.3
(-1.1, 0.5)
Mean IOP
Rhopressa
TM
N=76
Timolol
N=84
Mean
Difference
95% CI
(95% CI)
Timolol
–
TM
Rhopressa

15
Baseline IOP < 24 mmHg (Pre-specified analysis)

16
Safety/Tolerability Overview of Rhopressa
TM
(Days 15-90)
There were no drug-related serious adverse events (SAEs)
The most common adverse event was conjunctival hyperemia
•
Conjunctival hyperemia measured by biomicroscopy at 8am was
~35% of which 80% was mild
Adverse events occurring in approximately 5-13% of the subjects
receiving
Rhopressa
TM
included:
conjunctival
hemorrhage,
erythema
of
the eyelid, blurry vision and corneal deposits

17
Next Steps
Rocket 2 efficacy results (expected in Q3 2015) important in
determining the next steps, which may include the need for an
additional
Rhopressa
TM
registration
trial
Expect
to
file
the
Rhopressa
TM
NDA
by
the
end
of
2016,
if
additional
trial is required
Expect
to
commence
Roclatan
TM
Phase
3
registration
trials
by
the
end
of 2015, after review of Rocket 2 results
Over $179 million on the balance sheet as of the end of Q1 2015.
We
are well financed to execute our strategies