Cowen Research Report

Pharmaceuticals/Specialty
Ohr Pharmaceutical
Initiating Coverage
March 2, 2015
Initiation: OHR-102 Success In Wet

AMD Likely To Create Tremendous Value
Price: $7.69 (02/27/2015)

Price Target: $25.00
OUTPERFORM (1)
The Cowen Insight
Tyler Van Buren, M.Sc.

646.562.1338
tyler.vanburen@cowen.com
We are initiating on Ohr with an Outperform and $25 PT. Our rating is predicated
on the potential for OHR-102 to be successfully developed in wet AMD. If it has a
competitive efficacy profile and is ultimately approved, our consultants suggest that
the vast majority of treated wet AMD patients will be on OHR-102 therapy as it has a
superior product profile relative to other development candidates.
Ken Cacciatore
646.562.1305
ken.cacciatore@cowen.com
Sal Rais, M.D.
646.562.1420
sal.rais@cowen.com
OHR-102 Could Become The First Eye Drop For The Treatment Of Wet AMD
Key Data
Symbol
NASDAQ: OHRP
52-Week Range:
$20.00 - 6.01
Market Cap (MM):
$195.3
Net Debt (MM):
$(13.2)
Cash/Share:
$0.52
Dil. Shares Out (MM):
33.5
The Companys lead program is OHR-102, which is a first-in-class anti-angiogenic

compound that inhibits growth factors VEGF, PDGF, and bFGF, and is being developed
as a combination therapy with anti-VEGFs for wet AMD. OHR-102 is currently in a
Phase II clinical development program, which has already reported positive interim
visual acuity data, and final data is expected to readout by mid-to-late March. If the
final data are positive which we and our consultants believe has a good probability
of occurring Ohr plans to move OHR-102 into Phase III clinical trials in Q2:2015.
Ultimately, our consultants suggest that the vast majority of treated AMD patients will
want better visual acuity and therefore go on PDGF treatment.
Enterprise Value (MM): $184.8

ROIC:
NA
ROE (LTM):
NA
BV/Share:
$0.90
Dividend:
NA
FY (Sep)
2014A
Consultants Believe Early Visual Acuity Data Are Surprising And Intriguing
2015E
2016E
$(0.41)
$(1.00)
$(1.85)
NM
NM
NM
$0.0
$0.0
$0.0
Earnings Per Share

Year
P/E
Revenue (MM)
Year
The ongoing Phase II IMPACT study has enrolled 142 patients and is designed to
measure the impact of twice-daily OHR-102 on visual acuity in combination with
PRN Lucentis injections versus placebo drops plus PRN Lucentis. At interim (n=62),
the mean change in visual acuity at the end of 9 months for OHR-102 eye drops
plus Lucentis PRN was +10.4 letters versus +6.3 letters for placebo plus Lucentis
PRN a mean +4.1 letter improvement in visual acuity (p=0.18). Additionally, 48.3%
of OHR-102-treated patients showed BCVA gains of >3 lines (Phase III primary
endpoint confirmed with the FDA) compared with 21.2% in the placebo arm, which
was statistically significant (p=0.025). This result simply needs to be repeated to be
successful. Overall, our consultants suggest that the visual acuity results look very
much like Ophthotech's and the curves and outcomes look remarkably similar.
A Risk/Reward Play Skewed Towards The Upside
We assume a potential US OHR-102 approval and launch in early 2019 with a peak
penetration of treated wet AMD patients eligible for anti-PDGF treatment just above
35% by year 7, which assumes other PDGF competition. This results in a peak US
sales potential of $1B+. Ex-US, we assume an early 2020 launch and an ultimate peak
penetration of 25% by year 6-7 factoring in other PDGF competition. This results in
a peak Ex-US sales potential of $750MM+. Providing a necessary high discount rate
given the still early stage of development to these estimates yields an equity value of
$800MM+ or $25 per fully-diluted share, which is the basis of our price target. If the
final Phase II data are successful, our discount rate would correspondingly decrease
resulting in a higher equity value. Worth noting, our valuation does not give any credit
to OHR-102s follow-on indications of RVO, PVR, and DME, or the Alcon-partnered
sustained-release programs.
www.cowen.com
Please see addendum of this report for important disclosures.
This report is intended for world-cowen-morningnotesdistribution@cowen.com. Unauthorized redistribution of this report is prohibited.
Equity Research
Cowen and Company
Ohr Pharmaceutical
Equity Research
March 2, 2015
Our Investment Thesis
Forthcoming Catalysts
We assume a potential US OHR-102 approval and launch in early 2019 with a peak
penetration of treated wet AMD patients eligible for anti-PDGF treatment just above
35% by year 7, which assumes other PDGF competition. This results in a peak US
sales potential of $1B+. Ex-US, we assume an early 2020 launch and an ultimate peak
penetration of 25% by year 6-7 factoring in other PDGF competition. This results in
a peak Ex-US sales potential of $750MM+. Providing a necessary high discount rate
given the still early stage of development to these estimates yields an equity value of
$800MM+ or $25 per fully-diluted share, which is the basis of our price target. If the
final Phase II data are successful, our discount rate would correspondingly decrease
resulting in a higher equity value. Worth noting, our valuation does not give any credit
to OHR-102s follow-on indications of RVO, PVR, and DME, or the Alcon-partnered
sustained-release programs.
Mid-to-late March Final topline
Base Case Assumptions
Upside Scenario
Downside Scenario
$25 on positive OHR-102 Phase II

IMPACT data
$40 on better than expected IMPACT

data and potential buyout or licensing
deal
$2-3 on Phase II IMPACT failure
Price Performance
Company Description
data readout of the OHR-102 Phase II

IMPACT study for wet AMD
Q2:2015 Potential Phase III global
program initiation for OHR-102 in wet
AMD
H1:2015 Potential IND filing for one
of four sustained-release programs
partnered with Alcon
2015 (potentially by the end of H1)
OHR-102 RVO and PVR data readouts
Ohr Pharma is a development-stage specialty pharmaceutical company with multiple

programs in development for various ophthalmic indications. The Companys lead
program is OHR-102, which is in Phase II and being developed as a combination
therapy with anti-VEGFs for wet AMD.
$20
18
16
14
12
Analyst Top Picks
10
Ohr Pharmaceutical
8
6
May-14
Aug-14
Nov-14
Feb-15
Source: Bloomberg
www.cowen.com
Ticker
Price (02/27/2015)
Price Target
Rating
OHRP
$7.69
$25.00
Outperform
At A Glance
Cowen and Company
Ohr Pharmaceutical
Equity Research
March 2, 2015
OHR-102 is currently in Phase II development,

which has already reported positive interim data,
and final data is expected to readout by mid-tolate March. If the final data are positive, which
we and our consultants believe has a good
probability of occurring, Ohr plans to move OHR102 into Phase III clinical trials in Q2:2015.
OHR-102 has a composition of matter patent on

OHR-102 lactate salt to 2029 and an additional
broad-base formulation patent to 2030 that is still
pending. A couple new filings are expected and
we would also note that these expirations do not
consider any potential extensions from HatchWaxman. Nonetheless, OHR-102 appears to be a
long-duration drug.
OHR-102 is a first-in-class anti-angiogenic

compound that inhibits multiple growth factors:
vascular endothelial growth factor (VEGF),
platelet-derived growth factor (PDGF), and basic
fibroblast growth factor (bFGF).
Ohr Pharma is a development-stage specialty pharmaceutical company with multiple

programs in development for various ophthalmic indications. The Companys lead
program is OHR-102, which is being developed as a combination therapy with antiVEGFs for wet AMD. OHR-102 is currently in a Phase II clinical development program,
which has already reported positive interim visual acuity data, and final data is
expected to readout by mid-to-late March. If the final data are positive, which we and
our consultants believe has a good probability of occurring, Ohr plans to move OHR102 into Phase III clinical trials in Q2:2015. Follow-on indications of PVR, RVO, and
DME are anticipated next for OHR-102 and the approach appears logical given the
success of on-market wet AMD treatments in these areas. For RVO and PVR, OHR102 is being tested in investigator-sponsored studies with Phase II data to readout
later this year potentially by the end of H1:2015. For DME, the Company is running
its own Phase II study with a final data to readout around mid-2016. OHR-102 has a
composition of matter patent on OHR-102 lactate salt to 2029 and an additional
broad-base formulation patent to 2030 that is still pending. A couple new filings are
expected and we would also note that these expirations do not consider any potential
extensions from Hatch-Waxman. Nonetheless, OHR-102 appears to be a long-duration
drug. Additionally, the Company has a research agreement with Alcon a Novartis
company which is one of the largest ophthalmology players in the world with over
$10B in annual revenues. Clearly, this provides a source of validation for Ohrs
programs and expertise in ophthalmology. This collaboration is testing Ohrs
sustained-release hydrogel platform technology and encompasses four active
research programs in glaucoma, steroid-induced glaucoma, eye allergy, and retinal
disease. Depending on the outcome of these early programs with partner Alcon, Ohr
anticipates potentially filing an IND for one program by the end of H1:2015. Finally, we
believe Ohr has assembled an impressive management team and group of experts.
Both Dr. Jason Slakter, the CMO, and Dr. Peter Kaiser, the SVP of product
development, are world-renowned retinal specialists. Dr. Avner Ingerman, another
accomplished ophthalmologist who ran the Eylea clinical development program at
Regeneron, just joined as the Companys Chief Clinical Officer. Ohr also has several
other thought leaders involved and consulting on the program including Dr. David
Boyer who presented the recent abicipar pegol/DARPin data and Dr. Jeff Heier, who
has presented PDGF data from the Regeneron and Ophthotech programs, among
others. Needless to say, this is an impressive group of individuals.
One consultant believes that despite previous topical failed programs for wet AMD, it
will only be a matter of time before an eye drop potent enough is found with the right
dosage that reaches the retina. We believe Ohrs OHR-102 (squalamine) might be the
one. OHR-102 is a first-in-class anti-angiogenic compound that inhibits multiple
growth factors: vascular endothelial growth factor (VEGF), platelet-derived growth
factor (PDGF), and basic fibroblast growth factor (bFGF). VEGFs have revolutionized
the treatment of wet AMD, but they produce most of their improvement in about 4
months and then there is a plateau in efficacy. Moreover, patients must be
continuously dosed with frequent anti-VEGF to maintain the plateau; data from several
large trials were shown to suggest that switching from monthly dosing to PRN results
in a loss of efficacy. The biological explanation for this may be that anti-VEGFs cause
regression of tip cells at the distal end of neovascularization, but the bulk of the
vessel outgrowths are protected from the anti-VEGF by a lining of pericytes. AntiPDGF agents, in combination with the anti-VEGFs, denude the overgrown vessels of
pericytes and cause regression of the outgrowth. Current development of anti-VEGF
agents is aimed at extending the duration of treatment thereby allowing for a fewer
www.cowen.com
Investment Thesis: OHR-102 Success In Wet AMD Likely To Create

Tremendous Value
Ohr Pharmaceutical
Equity Research
March 2, 2015
However, this dogma appears to be changing

with advent of anti-PDGF agents. PDGF agents
in the case of OHR-102 and Fovista have
demonstrated significant visual acuity gains on
top of anti-VEGF treatments when used in
combination. We would remind investors that for
wet AMD, a disease where patients ultimately
lose their vision, increased visual acuity is
paramount. Ultimately, our consultants suggest
that the vast majority of treated AMD patients
(2/3 and up) will want better visual acuity and
therefore go on PDGF treatment.
number of injections a year, or better compliance as recent agents have largely failed
to meaningfully increase visual acuity in long-term studies. However, this dogma
appears to be changing with advent of anti-PDGF agents. PDGF agents in the case
of OHR-102 and Fovista have demonstrated significant visual acuity gains on top of
anti-VEGF treatments when used in combination. We would remind investors that for
wet AMD, a disease where patients ultimately lose their vision, increased visual acuity
is paramount. Ultimately, our consultants suggest that the vast majority of treated
AMD patients (2/3 and up) will want better visual acuity and therefore go on PDGF
treatment. Lastly, our consultants note that as many as 10-20% of their patients are
not adequately managed by the current anti-VEGF options, and therefore there is also
a need for more potent alternatives to help better manage the disease in refractory
patients
Consultants Believe Early OHR-102 Visual Acuity Data Are Surprising And
Intriguing; Final Phase II Data Due Mid-to-Late March
While there is still substantial risk to any wet

AMD data readout, OHR-102 appears to have a
strong possibility of repeating the interim data. If
this occurs, not only would the perception of the
program be strengthened even more, but it
would be significantly de-risked as its head into
Phase III with the same exact clinical endpoint.
However, when analysis was conducted on the

more clinically relevant visual acuity endpoints,
the data suggested a strong visual acuity benefit
across the two most common endpoints, mean
visual acuity and the proportion of >3 line
gainers. The mean change in visual acuity at the
end of 9 months for OHR-102 eye drops plus
Lucentis PRN was +10.4 letters versus +6.3
letters for placebo plus Lucentis PRN a mean
+4.1 letter improvement in visual acuity. Our
consultants note that this 65% visual acuity
benefit is impressive, especially considering the
potential ceiling effect for enrolling a good
amount of patients with relatively mild disease.
In June 2014, Ohr announced positive interim topline clinical results of a Phase II
study in wet AMD. Prior to the positive data release, few had any expectations for the
program much less an observed visual acuity benefit. However, since the data
which produced significant visual acuity benefits and our consultants believe is very
surprising and intriguing that perception has changed and we now approach the
final Phase II data readout in mid-to-late March with the expectation of a visual acuity
benefit. While there is still substantial risk to any wet AMD data readout, OHR-102
appears to have a strong possibility of repeating the interim data. If this occurs, not
only would the perception of the program be strengthened even more, but it would be
significantly de-risked as its head into Phase III with the exact same clinical endpoint.
The ongoing Phase II IMPACT study has enrolled 142 patients (completed in April
2014) and is designed to measure the impact of twice-daily OHR-102 on visual acuity
in combination with PRN (as needed) Lucentis versus placebo drops plus PRN
Lucentis. Per the study plan, a pre-specified interim analysis was conducted with
~50% of the planned total patient enrollment completing the study protocol. 62
patients 29 and 33 in the OHR-102 and placebo arms, respectively completed the 9
month treatment period at interim. Not surprisingly, the primary endpoint, the
difference in the frequency of Lucentis injections, did not meet statistical significance
at interim. Given our knowledge of PRN studies (no difference in year 2 of the VIEW
studies with Eylea/Lucentis) and the fact that no study has shown the ability to reduce
Lucentis injections including Ophthotechs Phase II program we were not
surprised by this result. Furthermore, the panelists at our March 2014 Health Care
Conference stated that a fewer injections endpoint largely isnt clinically relevant.
Thus, based on the data (6.2 Lucentis injections for the OHR-102 arm and 6.4 for the
placebo arm), we find it improbable that this endpoint will meet statistical significance
in the final data set nor do we care. Sure a reduction in injections would be an
improvement but ultimately what matters to clinicians is improving visual acuity.
However, when analysis was conducted on the more clinically relevant visual acuity
endpoints, the data suggested a strong visual acuity benefit across the two most
common endpoints mean visual acuity and the proportion of >3 line gainers. The
mean change in visual acuity at the end of 9 months for OHR-102 eye drops plus
Lucentis PRN was +10.4 letters versus +6.3 letters for placebo plus Lucentis PRN a
mean +4.1 letter improvement in visual acuity. Our consultants note that this 65%
visual acuity benefit is impressive, especially considering the potential ceiling effect for
enrolling a good amount of patients with relatively mild disease. However, it was not
statistically significant (p=0.18) given the small study size. With that in mind, our
www.cowen.com
Cowen and Company
Cowen and Company
Ohr Pharmaceutical
Equity Research
March 2, 2015
Our consultants suggest that this endpoint is also

has a good chance of reaching statistical
significance when the larger, final 142-patient
Phase II data set reads out by the end of March
2015 as the data in 62 patients gives us a good
feel for OHR-102s efficacy. One consultant
remarked that it would be shocking if the final
data showed no visual acuity benefit as the first
62 patients are very typical looking and he
would put the odds of success on the >3 line
gainers endpoint pretty high.
Since the interim readout, Ohr has met with the FDA and received confirmation that
the Phase III endpoint will be the proportion of patients with >3 line visual acuity
gains, which was the exact secondary endpoint that hit statistical significance at the
interim readout per above in only 62 patients. Moreover, our consultants suggest that
this endpoint also has a good chance of reaching statistical significance when the
larger, final 142-patient Phase II data set reads out by the end of March 2015 as the
data in 62 patients gives us a good feel for OHR-102s efficacy. One consultant
remarked that it would be shocking if the final data showed no visual acuity benefit
as the first 62 patients are very typical looking and he would put the odds of success
on the >3 line gainers endpoint pretty high. If it does, our consultants would also
consider the final Phase III program employing the exact same endpoint to be
significantly de-risked. In fact, they believe that upon a successful readout, OHR-102
would have the same probability of success in Phase III as Ophthotech which per
previous physician surveys has been pegged at around 75%. One consultant
believes that successful Phase II wet AMD results tend to have an 80% chance of
translating to Phase III. At the very least, we believe the early interim results suggest
that OHR-102 treats the back of the eye and may cause a significant increase in visual
acuity in wet AMD patients. Overall, our consultants suggest that the mean change in
visual acuity and >3 line gainer results look very much like Ophthotechs and the
curves and outcomes look remarkably similar. While some skepticism will always
remain through the final Phase III data, consultants appear to be looking at the
program very differently now following the interim data.
As we head towards the final results, it is also important to consider that while 62
patients wasnt enough to hit on the mean visual acuity endpoint, 142 patients with
the observed +4.1 letter increase in visual acuity is just reaching the boundary
where there might be enough powering to reach statistical significance, per our
consultants. Of course, a 300-400 patient study would be ideally powered, but the
potential to hit statistical significance on mean visual acuity in the final dataset is a
source of additional upside and would further help to dispel any skeptics. However, it
is important to keep in mind that even if mean visual acuity doesnt hit which could
be a perceived negative all that matters is that the >3 line gainers Phase III endpoint
hits.
Upon potential successful final Phase II data, Ohr

has stated that they would be able to initiate the
Phase III program by the end of Q2:2015. We
estimate that the time to topline data could be 22.5 years accounting for 12-18 months of
enrollment and the 9 month primary endpoint.
Upon potential successful final Phase II data, Ohr has stated that they would be able
to initiate the Phase III program by the end of Q2:2015. We estimate that the time to
topline data could be 2-2.5 years accounting for 12-18 months of enrollment and the 9
month primary endpoint. 2.5 years would put the topline data readout at late 2017.
Since OHR-102 has Fast Track status, it could begin filing a rolling NDA submission
during the trial and then submit the final topline 9 month primary endpoint data last in
early 2018. In essence, Ohr could receive approval even before the ultimate 2 year
study time point just like Eylea. This would allow for a potential approval and launch
by early 2019. Lastly, we would note that this timeline could potentially be conservative
by 6 months.
For Europe, Ohr plans to meet with regulators soon to nail down the requirements for
the Phase III program. However, we would note that Ohr will be measuring the primary
www.cowen.com
Additionally, 48.3% of OHR-102-treated patients

showed BCVA gains of >3 lines (>15 letters) on
a standard ETDRS eye chart compared with
21.2% in the placebo arm (p=0.025) a
statistically significant observation. Overall, our
consultants view both the mean visual acuity and
>3 line gain results as impressive particularly if
they hold up when the final data reads out in the
second half of March.
consultants note that it is still too early and in too few patients to believe that
OHR-102 wont reach statistical significance on visual acuity once the final data reads
out. Additionally, 48.3% of OHR-102-treated patients showed BCVA gains of >3 lines
(>15 letters) on a standard ETDRS eye chart compared with 21.2% in the placebo arm
(p=0.025) a statistically significant observation. Our consultants view both the mean
visual acuity and >3 line gain results as impressive particularly if they hold up when
the final data reads out in the second half of March.
Cowen and Company
Ohr Pharmaceutical
Equity Research
March 2, 2015
Anticipated Upcoming Milestones For Ohr Pharma Are:

2015
Mid-to-late March Final topline data readout of the OHR-102 Phase II IMPACT
study for wet AMD
Q2:2015 Potential Phase III global program initiation for OHR-102 in wet AMD
H1:2015 Potential IND filing for one of four sustained-release programs partnered
with Alcon
2015 (potentially by the end of H1) OHR-102 RVO and PVR data readouts from
investigator-sponsored studies
2016
Mid-2016 OHR-102 Phase II DME study readout
2017
Late 2017 Potential OHR-102 Phase III program data readout
2018
Early 2018 Potential OHR-102 NDA filing for wet AMD
2019
Early 2019 Potential approval of OHR-102 for wet AMD
Valuation Analysis: A Risk/Reward Play Skewed Towards The Upside

For our OHR-102 global market build, we assume that 2/3 of treated wet AMD
patients will be eligible for anti-PDGF combination treatment, which will most likely
prove to be conservative per our consultants. In the US, we assume a per bottle net
price for OHR-102 of $750, which could also be conservative considering that branded
wet AMD agents command just under $2,000 per injection over either one or two
months. We are pricing OHR-102 to capture significant market uptake and to
potentially be used prophylactically, or in the larger patient population, since it is an
eye drop vs. an injection. We assume a potential US approval and launch in early 2019
with a peak penetration of eligible patients just above 35% by year 7, which assumes
other PDGF competition. This results in a peak US sales potential of $1B+. Ex-US, we
assume an early 2020 launch. We were generally more conservative for the EU and
assume a net price of $500 with an ultimate peak penetration of 25% by year 6-7 this
too assumes other PDGF competition. This results in a peak Ex-US sales potential of
$750MM+.
On the following pages, we provide our US and Ex-US wet AMD market models for
OHR-102.
www.cowen.com
endpoint out to 12 months in Phase III in case they require the standard 12 month
endpoint. Additionally, it will be a global trial with clinical sites in the US and EU. It is
quite possible that the EU submission could come shortly after the US submission, but
we conservatively assume a year delay in the European launch.
Cowen and Company
Ohr Pharmaceutical
Equity Research
March 2, 2015
2013A
2014A
2015E
2016E
2017E
2018E
2019E
2020E
2021E
2022E
2023E
2024E
U.S. We t A MD
Prevalence of moderate-advanced wet AMD pt.s ('000)
% diagnosed
# diagnosed patients ('000)
% of diagnosed pt.s Tx with anti-VEGFs
# treated patients ('000)
% growth
454
80%
361
95%
343
+6%
476
80%
381
96%
364
+6%
500
80%
400
96%
384
+6%
525
80%
420
96%
403
+5%
552
80%
441
96%
424
+5%
579
80%
463
96%
445
+5%
608
80%
486
96%
467
+5%
632
80%
506
96%
486
+4%
658
80%
526
96%
505
+4%
684
80%
547
96%
525
+4%
711
80%
569
96%
546
+4%
740
80%
592
96%
568
+4%
769
80%
615
96%
591
+4%
Avastin
% Avastin patient penetration
# patients receiving Avastin each year ('000)
Average # vials per patient per year
Avastin price per dose
U.S. A va s tin Sa le s In We t A MD ($MM)
26%
89
5.6
$50
$2 5
33%
120
5.5
$50
$3 5
37%
142
5.5
$50
$4 0
40%
161
5.5
$50
$4 5
40%
169
5.5
$50
$4 5
41%
182
5.5
$50
$5 0
42%
196
5.5
$50
$5 5
42%
204
5.5
$50
$5 5
42%
212
5.5
$50
$6 0
42%
221
5.5
$50
$6 0
42%
229
5.5
$50
$6 5
42%
239
5.5
$50
$6 5
42%
248
5.5
$50
$7 0
+757%
+40%
+14%
+13%
+0%
+11%
+10%
+0%
+9%
+0%
+8%
+0%
+8%
35%
120
5.6
$1,950
$1 ,3 1 0
23%
84
5.5
$1,950
$9 0 0
17%
65
5.5
$1,950
$7 0 0
16%
65
5.5
$1,950
$6 9 0
15%
64
5.5
$1,950
$6 8 0
15%
67
5.5
$1,950
$7 1 5
15%
70
5.5
$1,950
$7 5 0
15%
73
5.5
$1,950
$7 8 0
15%
76
5.5
$1,950
$8 1 5
15%
79
5.5
$1,950
$8 4 5
15%
82
5.5
$1,950
$8 8 0
15%
85
5.5
$1,950
$9 1 5
15%
89
5.5
$1,950
$9 5 0
+4%
-31%
-22%
-1%
-1%
+5%
+5%
+4%
+4%
+4%
+4%
+4%
+4%
39%
53%
134
5.4
$1,850
$1 ,3 3 4
44%
66%
160
5.2
$1,850
$1 ,5 4 0
46%
73%
177
5.2
$1,850
$1 ,7 0 0
45%
74%
182
5.2
$1,850
$1 ,7 4 5
45%
75%
191
5.2
$1,850
$1 ,8 3 5
44%
75%
196
5.2
$1,850
$1 ,8 8 0
43%
72%
201
5.2
$1,850
$1 ,9 3 0
43%
66%
209
5.2
$1,850
$2 ,0 1 0
43%
59%
217
5.2
$1,850
$2 ,0 9 0
43%
53%
226
5.2
$1,850
$2 ,1 7 5
43%
50%
235
5.2
$1,850
$2 ,2 6 0
43%
48%
244
5.2
$1,850
$2 ,3 5 0
43%
46%
254
5.2
$1,850
$2 ,4 4 5
+64%
+15%
+10%
+3%
+5%
+2%
+3%
+4%
+4%
+4%
+4%
+4%
+4%
66%
226
66%
240
66%
254
66%
266
66%
280
66%
294
66%
308
2%
6
11.0
$750
66%
321
8%
24
10.7
$750
66%
333
15%
50
10.5
$750
66%
347
23%
78
10.3
$750
66%
361
28%
99
10.0
$750
66%
375
32%
120
10.0
$750
66%
390
36%
140
10.0
$750
% growth
Lucentis
% Lucentis patient penetration
# patients receiving Lucentis each year ('000)
Lucentis price per dose
U.S. Luc e ntis Sa le s In We t A MD ($MM)
% growth
Eylea
% Eylea patient penetration
% penetration of non-Avastin market
# patients receiving Eylea each year ('000)
Eylea price per dose
U.S. Ey le a Sa le s In We t A MD ($MM)
% growth
OHR-1 0 2
% treated patients eligible for anti-PDGF treatment
# treated patients eligible for anti-PDGF treatment ('000)
% OHR-102 patient penetration
# patients receiving OHR-102 each year ('000)
Average # bottles per patient per year
OHR-102 price per dose
U.S. OHR-1 0 2 Sa le s In We t A MD ($MM)
$5 0
% gr ow th
$1 9 5
$3 9 5
$6 0 0
$7 4 5
$9 0 0
+2 9 0 %
+1 0 3 %
+5 2 %
+2 4 %
+2 1 %
2 0 2 5 E 5 Y CG R Com m e nts
$1 ,0 5 5
$1,310
$900
$700
$690
$680
$715
$750
$780
$815
$845
$880
$915
$950
% growth
Eylea Sales ($MM)
+4%
$1,334
-31%
$1,540
-22%
$1,700
-1%
$1,745
-1%
$1,835
+5%
$1,880
+5%
$1,930
+4%
$2,010
+4%
$2,090
+4%
$2,175
+4%
$2,260
+4%
$2,350
+4%
$2,445
+64%
+15%
+10%
+3%
+5%
+2%
+3%
$50
+4%
$195
+4%
$395
+4%
$600
+4%
$745
+4%
$900
+4%
$1,055
% growth
T OT A L U.S. WET A MD SA LES ($MM)
% gr ow th
- Low cost due to compounding

+7% - Recognized by compounders
- 4 week treatment duration; 6-7 weeks in practice
+2%
- 8 week treatment duration

- Very rapid uptake upon launch
+3% - Ma r k e t le a de r
- Per consultants; most will want better vision

- Potential launch in early 2019
- Assumes PDGF competition
- Assumes 2.5 years from start-finish for PIII
- Assume it stabilizes around 10 bottles per year
- Priced between $500-$1,000 for market uptake
+ 78% - Should e xpe r ie nc e r a pid upta k e
+1 7 %
U.S. We t A MD
Lucentis Sales ($MM)
% growth
OHR-102 Sales ($MM)

- Increased payor pressure to drive growth
$2 ,6 4 4
$2 ,4 4 0
$2 ,4 0 0
$2 ,4 3 5
$2 ,5 1 5
$2 ,5 9 5
$2 ,7 3 0
+290%
$2 ,9 8 5
+103%
$3 ,3 0 0
+52%
$3 ,6 2 0
+24%
$3 ,8 8 5
+21%
$4 ,1 6 5
+17%
$4 ,4 5 0
+2 7 %
-8 %
-2 %
+1 %
+3 %
+3 %
+5 %
+9 %
+1 1 %
+1 0 %
+7 %
+7 %
+7 %
+2%
+3%
+78% - Assumes PDGF competition
+ 4%
Source: Cowen and Company; PriceRx
www.cowen.com
Figure 1 US Wet AMD Market Model
Cowen and Company
Ohr Pharmaceutical
Equity Research
March 2, 2015
2013A
2014E
2015E
2016E
2017E
2018E
2019E
2020E
2021E
2022E
2023E
2024E
Ex-U.S. We t A MD
% diagnosed
% growth
681
80%
541
95%
514
+6%
715
80%
572
96%
546
+6%
750
80%
600
96%
576
+6%
788
80%
630
96%
605
+5%
827
80%
662
96%
635
+5%
869
80%
695
96%
667
+5%
912
80%
730
96%
700
+5%
949
80%
759
96%
728
+4%
986
80%
789
96%
758
+4%
1,026
80%
821
96%
788
+4%
1,067
80%
854
96%
819
+4%
1,110
80%
888
96%
852
+4%
1,154
80%
923
96%
886
+4%
- We estimate 50% larger than U.S.
Avastin
Ex-U.S. A va s tin Sa le s In We t A MD ($MM)
55%
282
5.6
$50
$7 9
48%
262
5.5
$50
$7 0
44%
254
5.5
$50
$7 0
43%
260
5.5
$50
$7 0
43%
273
5.5
$50
$7 5
43%
287
5.5
$50
$8 0
43%
301
5.5
$50
$8 5
43%
313
5.5
$50
$8 5
43%
326
5.5
$50
$9 0
43%
339
5.5
$50
$9 5
43%
352
5.5
$50
$9 5
43%
366
5.5
$50
$1 0 0
43%
381
5.5
$50
$1 0 5

-11%
-11%
+0%
+0%
+7%
+7%
+6%
+0%
+6%
+6%
+0%
+5%
+5%
38%
195
5.6
$1,950
$2 ,1 3 3
36%
197
5.5
$1,950
$2 ,1 1 0
34%
196
5.5
$1,950
$2 ,1 0 0
32%
194
5.5
$1,950
$2 ,0 7 5
30%
191
5.5
$1,950
$2 ,0 4 5
29%
193
5.5
$1,950
$2 ,0 7 5
28%
196
5.5
$1,950
$2 ,1 0 5
27%
197
5.5
$1,950
$2 ,1 1 0
26%
197
5.5
$1,950
$2 ,1 1 5
25%
197
5.5
$1,950
$2 ,1 1 5
24%
197
5.5
$1,950
$2 ,1 1 0
23%
196
5.5
$1,950
$2 ,1 0 0
22%
195
5.5
$1,950
$2 ,0 9 0
+10%
-1%
-0%
-1%
-1%
+1%
+1%
+0%
+0%
+0%
-0%
-0%
-0%
7%
16%
37
5.4
$1,850
$3 7 0
16%
31%
87
4.7
$1,850
$7 6 0
22%
39%
127
4.3
$1,850
$1 ,0 1 0
25%
44%
151
4.3
$1,850
$1 ,2 0 5
27%
47%
172
4.3
$1,850
$1 ,3 6 5
28%
49%
187
4.3
$1,850
$1 ,4 8 5
29%
51%
203
4.3
$1,850
$1 ,6 1 5
30%
52%
219
4.3
$1,850
$1 ,7 4 0
31%
49%
235
4.3
$1,850
$1 ,8 7 0
32%
46%
252
4.3
$1,850
$2 ,0 0 5
33%
44%
270
4.3
$1,850
$2 ,1 5 0
34%
43%
290
4.3
$1,850
$2 ,3 0 5
35%
43%
310
4.3
$1,850
$2 ,4 7 0
+1847%
+105%
+33%
+19%
+13%
+9%
+9%
+8%
+7%
+7%
+7%
+7%
+7%
66%
339
66%
360
66%
380
66%
399
66%
419
66%
440
66%
462
66%
481
1%
5
11.0
$500
$2 5
66%
500
6%
30
10.8
$500
$1 6 0
66%
520
13%
65
10.5
$500
$3 4 0
66%
541
18%
97
10.3
$500
$5 0 0
66%
562
22%
124
10.0
$500
$6 2 0
66%
585
25%
146
10.0
$500
$7 3 0
+5 4 0 %
+1 1 3 %
+4 7 %
+2 4 %
+1 8 %
% growth
Lucentis
Ex-U.S. Luc e ntis Sa le s In We t A MD ($MM)
% growth
Eylea
Ex-U.S. Ey le a Sa le s In We t A MD ($MM)
% growth
OHR-1 0 2
Ex-U.S.OHR-1 0 2 Sa le s In We t A MD ($MM)
% gr ow th
Ex-U.S. We t A MD
% growth
Eylea Sales ($MM)
% growth
OHR-102 Sales ($MM)
% growth
T OT A L Ex-U.S. WET A MD SA LES ($MM)
% gr ow th
+ 0% - Ma r k e t le a de r

- Rapid uptake, but less so than the U.S.
+ 11%

- Potential launch in early 2020; could be conserv.
- Assume price 2/3 of U.S.
+ 96% - Quic k upta k e , but s lowe r tha n U.S.
$2,110
$2,100
$2,075
$2,045
$2,075
$2,105
$2,110
$2,115
$2,115
$2,110
$2,100
$2,090
+0%
+10%
$370
-1%
$760
-0%
$1,010
-1%
$1,205
-1%
$1,365
+1%
$1,485
+1%
$1,615
+0%
$1,740
+0%
$1,870
+0%
$2,005
-0%
$2,150
-0%
$2,305
-0%
$2,470
+11%
+1847%
+105%
+33%
+19%
+13%
+9%
+9%
+8%
$25
$2 ,5 0 3
$2 ,8 7 0
$3 ,1 1 0
$3 ,2 8 0
$3 ,4 1 0
$3 ,5 6 0
$3 ,7 2 0
$3 ,8 7 5
+7%
$160
+7%
$340
+7%
$500
+7%
$620
+7%
$730
+540%
$4 ,1 4 5
+113%
$4 ,4 6 0
+47%
$4 ,7 6 0
+24%
$5 ,0 2 5
+18%
$5 ,2 9 0
+15%
+8%
+5%
+4%
+4%
+4%
+4%
+7%
+8%
+7%
+6%
+5%
W.W. We t A MD
$3,443
$3,010
$2,800
$2,765
$2,725
$2,790
$2,855
$2,890
$2,930
$2,960
$2,990
$3,015
$3,040
% growth
Eylea Sales ($MM)
+7%
$1,704
-13%
$2,300
-7%
$2,710
-1%
$2,950
-1%
$3,200
+2%
$3,365
+2%
$3,545
+1%
$3,750
+1%
$3,960
+1%
$4,180
+1%
$4,410
+1%
$4,655
+1%
$4,915
% growth
OHR-102 Sales ($MM)
+105%
+35%
+18%
+9%
+8%
+5%
+5%
$50
+6%
$220
+6%
$555
+6%
$940
+6%
$1,245
+6%
$1,520
+6%
$1,785
+340%
$6 ,8 6 0
+152%
$7 ,4 4 5
+69%
$8 ,0 8 0
+32%
$8 ,6 4 5
+22%
$9 ,1 9 0
+17%
$9 ,7 4 0
+6%
+9%
+9%
+7%
+6%
+6%
% gr ow th
$2,133
+27%
% growth
T OT A L W.W. WET A MD SA LES ($MM)

$5 ,1 4 7
+27%
$5 ,3 1 0
+3%
$5 ,5 1 0
+4%
$5 ,7 1 5
+4%
$5 ,9 2 5
+4%
$6 ,1 5 5
+4%
$6 ,4 5 0
+5%

+ 4%
+1%
+7%
+ 4%
For our DCF valuation, we included the peak sales potentials from above and assumed
gross margins of 90%+. We also assumed the Phase III program will cost $75MM and
include SG&A expense within the range of normal industry standards. We expect that
Ohr will begin hiring commercial employees in mid-2016 and hire an initial OHR-102
US sales force of 50 reps upon approval, which will be later expanded. Similar
assumptions are made for an EU launch, although we suspect that Ohr may look to
partner Ex-US, similarly to the Fovista/Novartis deal, which resulted in ~$1B in
potential milestones and royalties on Ex-US sales. Providing a necessary high discount
rate given the still early stage of development to these estimates yields in an equity
value of $800MM+ or $25 per fully-diluted share, which is the basis of our price
target. If the final Phase II data are successful, our discount rate would
correspondingly decrease resulting in a higher equity value. Additionally, OHR-102 is
the only late-stage, unpartnered anti-PDGF program and we expect it to receive a lot
of interest from large ophthalmology players if the results are successful in March. Put
simply, as OHR-102 moves through the clinic, we expect the valuation gap between
OHRP and OPHT (~$1.6B currently) will narrow. We also note that our valuation does
not give any credit to OHR-102s follow-on indications of RVO, PVR, or DME or the
Alcon-partnered sustained-release programs.
www.cowen.com
Figure 2 Ex-US Wet AMD Market Model
Cowen and Company
Ohr Pharmaceutical
Equity Research
March 2, 2015
On the following pages, we provide our Ohr Pharma annual P&L and DCF.
Figure 3 Ohr Pharma Annual P&L

OHR PHA RMA - 2 0 1 5 -2 0 2 5 EST IMA T ED A NNUA L EPS BUILDUP ($MM)
F iscal Year E nded S ept em ber 30
2014
2015E
2016E
2017E
2018E
2019E
2020E
2021E
2022E
2023E
2024E
2025E
$50.0
$195.0
$395.0
$600.0
$745.0
$900.0
$1,055.0
+290%
+103%
+52%
+24%
+21%
+17%
$25.0
$160.0
$340.0
$500.0
$620.0
$730.0
$ 220. 0
$ 555. 0
5YR C GR C om m ent s
O HR- 102
U.S. Wet AMD Sales ($MM)
% Growth
Ex-U.S. Wet AMD Sales ($MM)
% Growth
Tot al O hr Rev enues
+540%
NM
NM
NM
NM
NM
+ 340%
+ 152%
+ 69%
+ 32%
+ 22%
+ 17%
$0.0
$0.0
$0.0
$0.0
$5.0
$22.0
$50.0
$84.6
$99.6
$121.6
$125.0
Gross Profit
$0.0
$0.0
$0.0
$0.0
$0.0
$45.0
$198.0
$505.1
$855.4
$1,145.4
$1,398.4
$1,660.1
NM
NM
NM
NM
NM
90.0%
90.0%
91.0%
91.0%
92.0%
92.0%
93.0%
$5.1
$7.5
$15.0
$20.0
$25.0
$50.0
$90.0
$150.0
$240.0
$290.0
$340.0
$390.0
% of Revs
% of Revs
$ 940. 0
$ 1, 245. 0
$ 1, 520. 0
$ 1, 785. 0
NM
NM
NM
NM
NM
100.0%
40.9%
27.0%
25.5%
23.3%
22.4%
21.8%
$4.0
$20.0
$40.0
$30.0
$20.0
$15.0
$15.0
$10.0
$10.0
$10.0
$10.0
$10.0
NM
NM
NM
NM
NM
30.0%
6.8%
1.8%
1.1%
0.8%
0.7%
0.6%
$9.1
$27.5
$55.0
$50.0
$45.0
$65.0
$105.0
$160.0
$250.0
$300.0
$350.0
$400.0
NM
NM
NM
NM
NM
130.0%
47.7%
28.8%
26.6%
24.1%
23.0%
22.4%
($9.1)
($27.5)
($55.0)
($50.0)
($45.0)
($20.0)
$93.0
$345.1
$605.4
$845.4
$1,048.4
$1,260.1
NM
NM
NM
NM
NM
NM
42.3%
62.2%
64.4%
67.9%
69.0%
70.6%
Interest Income
$0.0
$0.0
$0.0
$0.0
$0.0
$0.0
$0.0
$0.0
$0.0
$0.0
$0.0
$0.0
Interest Expense
(0.0)
0.0
0.0
0.0
0.0
0.0
0.0
0.0
0.0
0.0
0.0
0.0
Other Income
(0.0)
(2.0)
0.0
0.0
0.0
0.0
0.0
0.0
0.0
0.0
0.0
0.0
Non-Operating Income
($0.0)
($2.0)
$0.0
$0.0
$0.0
$0.0
$0.0
$0.0
$0.0
$0.0
$0.0
$0.0
Pretax Income
($9.1)
($29.5)
($55.0)
($50.0)
($45.0)
($20.0)
$93.0
$345.1
$605.4
$845.4
$1,048.4
$1,260.1
NM
NM
NM
NM
NM
NM
42.3%
62.2%
64.4%
67.9%
69.0%
70.6%
$32.6
$120.8
$211.9
$295.9
$366.9
$441.0
35.0%
35.0%
35.0%
35.0%
35.0%
35.0%
Operating Expenses
% of Revs
Operating Income
% Operating Margin
- C urrently being tested in other retina disorders

+96% - COM patent to 2029; formulation to 2030
+18%
$0.0
R&D
$ 50. 0
+24%
C ost of Goods
SG&A
$ 0. 0
+47%
$ 0. 0
Gross Margin
$ 0. 0
+113%
$ 0. 0
% Grow t h
$ 0. 0
+78% - Topical anti-angiogenic eye drop for wet AMD
- Final Phase II data by end of March; Potential Q2:2015 Phase III start
+ 98% - Rapid upt ake ex pect ed; assum es P DGF com pet it ion
- 90%+ margins; easy to manufacture

+64% - Assumes an initial sales force of 50 reps
- C ommercial hiring to begin in mid-2016
-6% - Phase III wet AMD program expected to cost $75MM; bell curve-like
+31%
NM
% of Revs
Income Taxes
Income Tax Rate
Net Income - Operations
% Net Margin
($9.1)
($29.5)
($55.0)
($50.0)
($45.0)
($20.0)
$60.5
$224.3
$393.5
$549.5
$681.5
$819.0
NM
NM
NM
NM
NM
NM
27.5%
40.4%
41.9%
44.1%
44.8%
45.9%
($9.1)
($29.5)
($55.0)
($50.0)
($45.0)
($20.0)
$60.5
$224.3
$393.5
$549.5
$681.5
$819.0
$0.0
$0.0
$0.0
$0.0
$0.0
$0.0
$0.0
$0.0
$0.0
$0.0
$0.0
$0.0
($ 0. 41)
($ 1. 00)
($ 1. 85)
($ 1. 60)
($ 1. 40)
($ 0. 60)
$ 1. 80
$ 6. 40
$ 10. 95
$ 14. 85
$ 17. 95
$ 21. 00
NM
NM
NM
NM
NM
NM
NM
+256%
NM
NM - Assumes no NOLs
- Standard U.S. corporate tax rate
NM
Extraordinary Items
Adjusted Net Income
Interest Add-Back
E P S (N on- GAAP ) - B ef ore E x . It em s
% Growth
EPS - Extraordinary Items
EPS - Adjusted
Shares Outstanding (MM)
+71%
36%
21%
NM
N M - True prof it abilit y reached in 2020
17%
$0.00
$0.00
$0.00
$0.00
$0.00
$0.00
$0.00
$0.00
$0.00
$0.00
$0.00
$0.00
($0.41)
($1.00)
($1.85)
($1.60)
($1.40)
($0.60)
$1.80
$6.40
$10.95
$14.85
$17.95
$21.00
22.1
29.0
30.0
31.0
32.0
33.0
34.0
35.0
36.0
37.0
38.0
39.0
NM
+3% - Aassuming some onward dilution from options
Source: Cowen and Company
www.cowen.com
Cowen and Company
Ohr Pharmaceutical
Equity Research
March 2, 2015
Assumptions:
Output:
Increase in WC
Discount Rate
5.0% Equity Value
$829.1
19.5% E s t. Sha r e Pr i ce
Fully Diluted Shares
$2 5 .0
33.5 Debt
$0.0
Cash
$35.0
Enterprise Value
Notes:
Netted $26-27MM from Feb 11 offering
Burned ~$2.8MM Q1; expected to be higher in Q2
$794.1
OHR PHA RMA DCF
Total Revenues
% Change
Cost of Goods
Gross Profit
Gross Margin - Total
SG&A
% of Revs
R&D
% of Revs
Operating Expenses
2014
2015E
2016E
2017E
2018E
2019E
$0.0
$0.0
$0.0
$0.0
$0.0
$50.0
NM
-50%
+0%
$125.0
$142.8
$159.3
$175.0
$149.8
$87.5
$38.5
$14.0
$7.0
$7.0
$7.0
$0.0
$0.0
$0.0
$0.0
$0.0
$45.0
$198.0
$505.1
$855.4
$1,145.4
$1,398.4
$1,660.1
$1,897.2
$2,115.8
$2,325.0
$1,990.2
$1,162.5
$511.5
$186.0
$93.0
$93.0
$93.0
93.0%
93.0%
93.0%
93.0%
$50.0
$25.0
$20.0
$20.0
NM
$4.0
NM
$9.1
NM
$20.0
NM
$27.5
NM
$40.0
NM
$55.0
NM
$30.0
NM
$50.0
NM
$20.0
NM
$45.0
90.0%
$50.0
$90.0
100.0%
40.9%
27.0%
25.5%
23.3%
22.4%
21.8%
21.1%
20.9%
20.0%
21.0%
20.0%
22.7%
25.0%
25.0%
20.0%
20.0%
$15.0
$15.0
$10.0
$10.0
$10.0
$10.0
$10.0
$10.0
$10.0
$10.0
$10.0
$10.0
$7.5
$5.0
$5.0
$5.0
$5.0
1.4%
2.5%
5.0%
5.0%
30.0%
$65.0
6.8%
$105.0
NM
NM
NM
NM
NM
130.0%
47.7%
($9.1)
($27.5)
($55.0)
($50.0)
($45.0)
($20.0)
$93.0
NM
NM
NM
NM
NM
NM
(0.0)
(2.0)
0.0
0.0
0.0
0.0
($9.1)
($29.5)
($55.0)
($50.0)
($45.0)
($20.0)
NM
NM
NM
NM
NM
NM
Adjusted EBIT
% of Revs
42.3%
1.8%
$160.0
28.8%
$345.1
62.2%
$240.0
1.1%
$250.0
26.6%
$605.4
64.4%
$290.0
0.8%
$300.0
24.1%
$845.4
67.9%
$340.0
0.7%
$350.0
23.0%
$1,048.4
69.0%
$390.0
0.6%
$400.0
22.4%
$1,260.1
70.6%
93.0%
$430.0
0.5%
$440.0
21.6%
$1,457.2
71.4%
93.0%
$475.0
0.4%
$485.0
21.3%
$1,630.8
71.7%
93.0%
$500.0
0.4%
$510.0
20.4%
$1,815.0
72.6%
93.0%
$450.0
0.5%
$460.0
21.5%
$1,530.2
71.5%
93.0%
$250.0
0.8%
$260.0
20.8%
$902.5
72.2%
93.0%
$125.0
$132.5
24.1%
$379.0
68.9%
5.0%
$55.0
$30.0
$25.0
$25.0
27.5%
30.0%
25.0%
25.0%
$63.0
$68.0
$68.0
63.0%
68.0%
68.0%
$131.0
65.5%
0.0
0.0
0.0
0.0
0.0
0.0
0.0
0.0
0.0
0.0
0.0
0.0
0.0
0.0
0.0
0.0
$93.0
$345.1
$605.4
$845.4
$1,048.4
$1,260.1
$1,457.2
$1,630.8
$1,815.0
$1,530.2
$902.5
$379.0
$131.0
$63.0
$68.0
$68.0
65.5%
63.0%
68.0%
68.0%
$45.9
$22.1
$23.8
$23.8
35.0%
35.0%
35.0%
35.0%
$85.2
$41.0
$44.2
$44.2
($3.0)
($3.0)
($3.0)
($3.0)
42.3%
Taxes
$32.6
Income Tax Rate
35.0%
NOPAT
$150.0
93.0%
+0%
90.0%
% of Revenues
Other Income
2035E
$100.0
$121.6
Operating Income
% Operating Margin
2034E
$100.0
$99.6
92.0%
-64%
2033E
$100.0
+32%
92.0%
-56%
2032E
$200.0
$84.6
91.0%
-42%
2031E
$550.0
+69%
91.0%
-14%
2030E
$1,250.0
$50.0
NM
+10%
2029E
$2,140.0
+152%
$25.0
+12%
2028E
$2,500.0
$22.0
NM
+14%
2027E
$2,275.0
+340%
$20.0
+17%
2026E
$2,040.0
$5.0
NM
+22%
2025E
$1,785.0
$0.0
$15.0
NM
2024E
$1,520.0
$0.0
NM
NM
2023E
$1,245.0
$0.0
$7.5
NM
2022E
$940.0
$0.0
NM
NM
2021E
$555.0
$0.0
$5.1
NM
2020E
$220.0
62.2%
$120.8
35.0%
$224.3
64.4%
$211.9
35.0%
$393.5
67.9%
$295.9
35.0%
$549.5
69.0%
$366.9
35.0%
$681.5
70.6%
$441.0
35.0%
$819.0
71.4%
$510.0
35.0%
$947.2
71.7%
$570.8
35.0%
$1,060.0
72.6%
$635.3
35.0%
$1,179.8
71.5%
$535.6
35.0%
$994.6
72.2%
$315.9
35.0%
$586.6
68.9%
$132.7
35.0%
($9.1)
($29.5)
($55.0)
($50.0)
($45.0)
($20.0)
$60.5
$246.4
Capex
($0.1)
($0.3)
($0.5)
($1.0)
($2.0)
($3.0)
($3.0)
Depreciation & Amortization
($0.5)
($1.0)
($1.0)
($1.0)
($1.0)
($1.0)
($1.0)
($1.0)
($1.0)
($1.0)
($1.0)
($1.0)
($1.0)
($1.0)
($1.0)
($1.0)
($1.0)
($1.0)
($1.0)
($1.0)
($1.0)
($1.0)
$3.4
$5.0
$2.5
$0.0
($5.0)
($10.0)
($10.5)
($11.0)
($11.6)
($12.2)
($12.8)
($13.4)
($14.1)
($14.8)
($15.5)
($16.3)
($17.1)
($18.0)
($18.9)
($19.8)
($20.8)
($21.8)
($6.3)
($25.8)
($54.0)
($52.0)
($53.0)
($34.0)
$46.0
$209.3
$377.9
$533.4
$664.7
$801.6
$929.1
$974.3
$565.5
$224.4
$62.3
$17.2
$19.4
$18.4
Adjustments:
Change In Working Capital

Free Cash Flow
Terminal
($3.0)
($3.0)
10
www.cowen.com
($3.0)
($3.0)
($3.0)
($3.0)
($3.0)
$1,041.2
($3.0)
$1,160.2
($3.0)
($3.0)
($3.0)
$94.21
Figure 4 Ohr Pharma DCF Suggests $25 Per Share
Cowen and Company
Ohr Pharmaceutical
Equity Research
March 2, 2015
Ohr Pharma Is Going All In On Ophthalmology

The Companys lead program is OHR-102, which is being developed as a combination
therapy with anti-VEGFs for wet AMD. OHR-102 is currently in a Phase II clinical
development program, which has already reported positive interim data, and final data
is expected to readout by mid-to-late March. If the final data is positive, which we and
our consultants believe has a good probability of occurring, Ohr plans to move OHR102 into Phase III clinical trials in Q2:2015. Worth noting, Ohr has already met with the
FDA and confirmed potential Phase III plans for the program. Follow-on indications of
PVR, RVO, and DME are anticipated next for OHR-102 and the approach appears
logical given the success of on-market wet AMD treatments in these areas. For RVO
and PVR, OHR-102 is being tested in investigator-sponsored studies with Phase II data
to readout later this year, potentially by the end of H1:2015. For DME, the Company is
running its own Phase II study with a final data to readout around mid-2016.
Additionally, the Company has a research agreement with Alcon a Novartis company
which is one of the largest ophthalmology players in the world with over $10B in
annual revenues. Clearly, this provides a source of validation for Ohrs programs and
expertise in ophthalmology. This collaboration is testing Ohrs sustained-release
hydrogel platform technology and encompasses four active research programs in
glaucoma, steroid-induced glaucoma, eye allergy, and retinal disease. Depending on
the outcome of these early programs with partner Alcon, Ohr anticipates potentially
filing an IND for one program by the end of H1:2015.
Figure 5 Ohr Pharmaceuticals Clinical Development Pipeline
Source: Ohr Pharma
OHR-102 Has The Potential To Be The First Topical Eye Drop For Retinal
Disease
Our consultants believe that despite previous
topical failed programs for wet AMD, it will only
be a matter of time before a drop potent enough
is found with the right dosage that reaches the
retina. We believe Ohrs OHR-102 (squalamine)
might be the one.
Our consultants believe that despite previous topical failed programs for wet AMD, it
will only be a matter of time before an eye drop potent enough is found with the right
dosage that reaches the retina. We believe Ohrs OHR-102 (squalamine) might be the
one. OHR-102 is a first-in-class molecule anti-angiogenic compound that inhibits
multiple growth factors: vascular endothelial growth factor (VEGF), platelet-derived
growth factor (PDGF), and basic fibroblast growth factor (bFGF). VEGF is a signaling
www.cowen.com
11
If the final data is positive, which we and our

consultants believe has a good probability of
occurring, Ohr plans to move OHR-102 into
Phase III clinical trials in Q2:2015. Worth noting,
Ohr has already met with the FDA and confirmed
potential Phase III plans for the program.
Cowen and Company
Ohr Pharmaceutical
Equity Research
March 2, 2015
Figure 6 Mechanism Comparison Of Wet AMD Agents
Source: Ohr Pharma
The OHR-102 eye drop formulation 0.2% squalamine lactate has emollient
properties to soothe the ocular surface, which results in increased comfort for
12
www.cowen.com
Alternatively, OHR-102 is a small molecule and it

therefore is able to enter the intracellular space
of vascular endothelial cells. Our consultants
note that steroids work by an intracellular
mechanism and are of course, very effective. Not
only do intracellular drugs tend to work well, but
our consultants note that they also tend to have a
longer duration of effect well beyond what the
kinetics suggest.
protein that stimulates vasculogenesis and angiogenesis. In other words, VEGF

promotes the growth and formation of blood vessels. PDGF has a similar function in
blood vessel formation as it helps regulate cell growth and division. bFGF is present in
basement membranes and in the subendothelial extracellular matrix of blood vessels
and when activated, it is thought to also aid in the formation of new blood vessels.
Thus, OHR-102 has a three-pronged anti-angiogenic approach/mechanism. Inhibiting
VEGF has been well-validated by the market leading treatments for retinal disease,
Eylea, Lucentis, and Avastin. Targeting PDGF has largely gained credibility over the
past few years as Ophthotech and now Ohr has reported significant visual acuity
benefits in Phase II. Also, several large players in the ophthalmology space, such as
Regeneron and Allergan have early PDGF programs earlier in clinical development.
Interestingly, elevated bFGF has been implicated in the pathophysiology of
proliferative diabetic retinopathy (PVR) and retinal vein occlusions (RVO). Additionally,
Eylea, Lucentis, Avastin, and Fovista are all large biologic molecules (recombinant
proteins; monoclonal antibodies; aptamers) and are therefore too large to enter the
cell. They remain in the extracellular space and work by neutralizing freely diffusible
proteins. Alternatively, OHR-102 is a small molecule and it therefore is able to enter
the intracellular space of vascular endothelial cells. Our consultants note that steroids
work by an intracellular mechanism and are of course, very effective. Not only do
intracellular drugs tend to work well, but our consultants note that they also tend to
have a longer duration of effect well beyond what the kinetics suggest.
Cowen and Company
Ohr Pharmaceutical
Equity Research
March 2, 2015
Figure 7 OHR-102 Reaches The Posterior

Sclera/Choroid
Source: Ohr Pharma
Interestingly, these supratherapeutic levels of

OHR-102 are reached rapidly in 15 minutes after
administration and maintained up to 24 hours
resulting in significant residence time.
Per below and as presented at ARVO 2012, a preclinical single-dose biodistribution

study was conducted by administering OHR-102 to the front of the eye in Dutch
belted rabbits. We would note that all the large ophthalmology players depend on
these standardized preclinical models and our consultants believe results in these
models to be fairly predictive of drug effects in the human eye. In this study, OHR-102
was shown to achieve supratherapeutic levels of ~90ng/g in sclera/choroid tissue. Of
note, Ohr estimates that approximate levels of 12ng/g in the sclera/chroid tissue are
the threshold drug concentration required to inhibit neovascularization. Interestingly,
these supratherapeutic levels of OHR-102 are reached rapidly in 15 minutes after
administration and maintained up to 24 hours resulting in significant residence time.
We would note that this is just a single dose, not the BID dosing regimen that is being
tested in clinical trials. Therefore, BID dosing of OHR-102 appears to deliver more than
sufficient drug levels to the eye.
www.cowen.com
13
OHR-102 has been shown to have improved

residence time on the ocular surface along with
minimized uptake into the aqueous humor.
Additionally, OHR-102 has demonstrated transscleral permeability into the choroid with
increased retention time in the posterior
sclera/choroid.
patients, particularly in the elderly patient population which is the vast majority of wet
AMD patients. Moreover, OHR-102 has been shown to have improved residence time
on the ocular surface along with minimized uptake into the aqueous humor.
Additionally, OHR-102 has demonstrated trans-scleral permeability into the choroid
with increased retention time in the posterior sclera/choroid. The sclera is known as
the white of the eye and it is the opaque, fibrous, protective outer layer of the eye. Just
inside that is the choroid, which is the vascular layer of the eye, contains connective
tissue, and lies between the retina and the sclera. Hence, OHR-102 has been shown
penetrate the tissue immediate to the retina, which is the primary target of treatment
for wet AMD and other retinal diseases.
Cowen and Company
Ohr Pharmaceutical
Equity Research
March 2, 2015
Source: Ohr Pharma
The BID dosing regimen achieved drugs level at

day 14 around 70ng/g approximately 30ng/g
higher than QD dosing and appeared to taper
off around day 14 approximately 55ng/g above
the threshold level. The most important takeaway
from these studies are that OHR-102 drug
concentrations well above the threshold level are
reached quickly as peak levels are almost
reached after 7 days with the BID dosing
regimen.
Ohr also measured multi-dose biodistribution of OHR-102 in rabbit posterior

sclera/choroid tissue. Trough level drug concentrations of OHR-102 were measured
over 14 days and the data was presented at ARVO 2012. Both once-daily and twicedaily dosing regimens were employed. At day 7, supratherapeutic and statistically
significant (p<0.01) OHR-102 drug trough levels were observed with both regimens.
Even higher OHR-102 drug trough concentrations were observed at day 14 and were
statistically significant (p<0.001). The BID dosing regimen achieved drugs level at day
14 around 70ng/g approximately 30ng/g higher than QD dosing and appeared to
taper off around day 14 approximately 55ng/g above the threshold level. The most
important takeaway from these studies are that OHR-102 drug concentrations well
above the threshold level are reached quickly as peak levels are almost reached after
7 days with the BID dosing regimen.
Figure 9 Posterior Sclera/Choroid Tissue Trough Levels Of OHR-102 Over 14 Days
Source: Ohr Pharma
14
www.cowen.com
Figure 8 Single Dose Biodistribution: OHR-102 Concentrations In The

Sclera/Choroid
Cowen and Company
Ohr Pharmaceutical
Equity Research
March 2, 2015
AMD Is The Leading Cause Of Blindness In People Over 55

According to the National Eye Institute, more
than 1MM people are diagnosed with AMD
annually and the incidence is expected to grow
as the population ages. Approximately 15M
people in the U.S. have macular degeneration, of
which 10-15% have active blood vessel growth
and leakage (wet AMD). The medical literature
suggests there are approximately 1.5M people
with wet AMD in the U.S., although
Roche/Genentech estimates the size of the
treatable market is at least a third of that.
Age-related Macular Degeneration (AMD) is the leading cause of blindness in people

over the age of 55. Legal blindness is defined as a person who is only able to see
20/200 or less with glasses. According to the National Eye Institute, more than 1MM
people are diagnosed with AMD annually and the incidence is expected to grow as
the population ages. Approximately 15M people in the U.S. have macular
degeneration, of which 10-15% have active blood vessel growth and leakage (wet
AMD). The medical literature suggests there are approximately 1.5M people with wet
AMD in the U.S., although Roche/Genentech estimates the size of the treatable
market is at least a third of that. We estimate the U.S. AMD market opportunity at
$3B+, with a similar opportunity ex-U.S. The market opportunity for AMD drugs may
be further expanded by penetration into other back-of-the-eye conditions such as
retinal vein occlusion or diabetic retinopathy and adjuncts/improvement to existing
therapies such as the new PDGF drugs in development.
What Is AMD?
AMD is subdivided into wet lesions (characterized by excessive new blood vessel
formation in the retina and fluid buildup) and dry AMD (a precursor of the wet form;
thinning of the macular tissues and disturbances in its pigmentation). AMD gradually
destroys a persons central vision function. The early stages of the disease may be
barely noticeable to some, but symptoms can vary. Sometimes only one eye loses
vision and the other maintains good vision for many years. Some patients have milder
symptoms in both eyes that may not impair vision significantly for many years. Other
frequent symptoms include distortion, or when straight lines look wavy, such as the
lines on an Amsler grid (an ophthalmic diagnostic tool in the picture below) or if a
doorframe or blinds look bent. Sometimes colors don't look quite right or there may be
a purple or gray spot in the center vision. (See picture below.)
www.cowen.com
15
We acknowledge that despite this data, many are still skeptical on the ability for drops
to get to the back of the eye. We believe this is largely because previous topical wet
AMD eye drop programs including GSKs pazopanib or Votrient have failed.
Additionally, there was an old IV formulation of squalamine that was terminated in wet
AMD, but this was due to its short half-life, which didnt provide adequate coverage
when dosed weekly, and the systemic dosing, which led to subtherapeutic drug levels
in the eye and systemic safety concerns. With that said, the IV formulation did show a
dose response and positive visual and anatomical benefits. Per the data above, OHR102 is clearly not systemic and certainly has an adequate coverage of 24 hours.
Whether or not investors believe eye drops can reach the back of the eye, the clinical
data with OHR-102 should ultimately win out and put this controversy to bed and we
believe that the interim data was a solid start in the right direction.
Cowen and Company
Ohr Pharmaceutical
Equity Research
March 2, 2015
Source: Cowen and Company; Macular Degeneration Foundation
Upon onset of macular degeneration, many people have trouble adjusting quickly
between bright sunlight or dim light or shadows. This may be especially dangerous
when driving in bright sunlight and then entering the shade or vice versa. Whereas a
normal retina takes 3-5 minutes to adjust from bright light to dim (when entering a
movie theater, for example), a person with macular degeneration may take 8-12
minutes or longer.
Figure 11 The Progression Of Wet AMD
Source: Molecular Partners
16
www.cowen.com
Figure 10 AMD & Amsler Grid Diagnostic Tool
Cowen and Company
Ohr Pharmaceutical
Equity Research
March 2, 2015
Etiology Of AMD Not Well Understood, But VEGF Is Clearly An Important Player
There Are Three Categories Of Wet AMD: Occult, Minimally Classic, and Classic
Based on the appearance of the blood vessels at the back of the eye when visualized
using fluorescein angiography, wet AMD can be subdivided into predominantly classic
(25%), minimally classic (35%) and occult (40%). In predominantly classic choroidal
neovascularization (CNV), greater than 50% of the neovascular AMD lesions can be
clearly defined, and this is generally regarded as the most aggressive subtype. In
minimally classic choroidal neovascularization, 1% to 50% of the neovascular AMD
lesions can be clearly defined, and this is generally regarded as the intermediate
subtype in terms of progression. In occult choroidal neovascularization, none of the
neovascular lesions can be clearly defined, and this is generally regarded as being the
least aggressive subtype. It is helpful to think of the AMD lesion as an expanding
vascular membrane which penetrates into an area underneath the retina. Angiography
lights up the blood vessels in this membrane, and physicians use the angiogram to
discern the outline of the membrane. However, leakage of dye from these blood
vessels can obscure a physicians ability to clearly see the membrane, and the extent
to which this visibility is obscured determines its classification. While it is important to
identify lesion subtypes to determine potential eligibility for photodynamic therapy
with Visudyne, the intra-vitreous anti-VEGF therapies have utility across wet AMD
subtypes, making differentiation less critical.
The Wet AMD Treatment Paradigm Has And Is Expected To Continue To Evolve
Rapidly
Current treatment methodologies for wet AMD include three options: anti-VEGF
therapy, photodynamic therapy (PDT), and laser photocoagulation. Retina specialists
are quick to adopt new therapies, often before they have undergone rigorous clinical
testing or have received FDA approval. The wet AMD treatment paradigm therefore
evolves rapidly and sometimes unexpectedly.
The First Wave Of AMD Treatment Used Lasers
Until March 2000, the only primary treatment for neovascular wet AMD was laser
coagulation. This technique involves the use of a strong light source targeted on
extrafoveal and juxtafoveal CNV lesions to coagulate the diseased tissue. The laser
produces a thermal effect within the lesion that causes necrosis of its cellular
components. In the 1980s, the National Eye Institute conducted the Macular
Degeneration Photocoagulation Studies (MPS), and the results demonstrated that
photocoagulation of well-demarcated CNV membranes effectively prevented large
decreases in visual acuity compared with observation for CNV. However, the thermal
effect of the laser can damage viable photoreceptor cells, resulting in retinal scarring
and loss of visual acuity. As such, use of laser coagulation in the current practice
setting is rare, as superior treatment options discussed below have emerged.
www.cowen.com
17
Any number of factors, including genetics, age, race, gender, menopause, nutrition,
smoking, and exposure to sunlight, may cause AMD. Based on the original striking
results from pan-VEGF inhibition therapy with Roches Lucentis, it is clear that VEGF is
an important driver of new blood vessel formation in the retina and conversion from
dry to wet AMD. Research is ongoing to identify additional molecules that drive the
wet AMD process and to better understand the pathophysiologic processes that result
in VEGF overexpression.
Cowen and Company
Ohr Pharmaceutical
Equity Research
March 2, 2015
Visudyne: An Outdated Procedure For Wet AMD
Anti-Angiogenesis Treatments Dominate The Wet AMD Market

Eyetechs Macugen, the first anti-VEGF therapy, was approved for wet AMD in 2004
and rapidly gained widespread adoption with an outstanding commercial launch.
Macugen is an aptamer that binds to and inhibits activity of the VEGF165 isoform and
was shown to decrease the rate of visual acuity loss in wet AMD patients versus
placebo in the Phase II/III VISION trials. Macugen revenue plummeted in 2005-2007 as
pan-VEGF antibodies emerged as a superior treatment option for preserving or
restoring vision of wet AMD patients. Roches/Novartiss Lucentis is a monoclonal
antibody fragment targeting all VEGF isoforms and has set a new bar for wet AMD
therapies. Data from the Phase II/III MARINA trial of Lucentis demonstrated an
impressive ability to improve visual acuity in wet AMD patients, a goal never before
achieved in advanced clinical trials for this indication. Lucentis secured U.S. approval
in June 2006 and EMEA approval in January 2007. The drugs U.S. launch was very
impressive (Roche/Genentech estimated that Lucentis achieved 55% penetration of
the wet AMD market just 6 months after FDA approval), relegating Macugen and
photodynamic therapy to niche market roles. While waiting for Lucentis to reach the
market, retina specialists began to use off-label, compounded intravitreous Avastin, a
cheap and commercially available anti-VEGF option. Even before there was Phase III
data to support its use, intravitreous Avastin nonetheless captured the majority of the
remaining (non-Lucentis treated) market and is the most commonly used anti-VEGF
for the treatment of wet AMD.
In turn, the growth of Regenerons Eylea is the
most recent example of the rapid market
transformation in this market and has expanded
upon advances made by the earlier agents by
offering a less frequent injection schedule (every
8 weeks vs. every 4 weeks).
18
In June 2011, the FDAs Dermatologic and Ophthalmic Drugs Advisory Committee
voted unanimously in favor of approving Eylea for wet AMD at a dose of 2mg Q8W,
following three initial doses given monthly. The panel was benign with no real points
of contention. After a minor hiccup in which the PDUFA date was pushed back due to
clarifying questions from the FDA regarding the CMC section, Eylea was FDA
approved in November 2011. In line with the panels conclusions, Eyleas
recommended dose is 2mg Q8W, following three initial loading doses given monthly.
In turn, the growth of Regenerons Eylea is the most recent example of the rapid
market transformation in this market and has expanded upon advances made by the
earlier agents by offering a less frequent injection schedule (every 8 weeks vs. every 4
weeks). Following the success of anti-VEGF therapies Lucentis, Eylea, and Avastin, the
most effective treatment for wet AMD is acknowledged to be angiogenesis inhibition.
Given its vascular and progressive nature, AMD thrives on new blood vessel growth.
www.cowen.com
Visudyne was originally approved in 2000 and was most recently acquired by Valeant
(from QLT) in September 2012 for $125MM suggesting that its use is very limited.
Visudyne is a photosensitizer that attaches to lipoproteins, which tend to accumulate
in rapidly proliferating cells. Visudyne traps energy from light and converts oxygen in
cells to a highly energized state that results in cell death. Visudyne therefore must be
activated at the site of desired effect by light. Photodynamic therapy (PDT) with
Visudyne consists of administering the light-activated drug by intravenous infusion
and then shining a light to the back of the eye to activate Visudyne in the retina.
Clinicians must therefore purchase the machinery to administer the light (up to $40K
in fixed costs), and hire personnel to administer the intravenous infusion. Therapy is
repeated every three months if patients continue to have active lesions, and our
consultants estimate that, on average, patients receive three to four treatments before
the CNV lesion is converted to a quiescent scar. Side effects of Visudyne include
transient skin sensitivity to bright light, acute onset and transient back pain, and
rarely, acute visual deterioration, which may not be reversible. Successful pan-VEGF
therapies Lucentis, Avastin, and Eylea have since significantly eroded Visudynes
commercial presence.
Cowen and Company
Ohr Pharmaceutical
Equity Research
March 2, 2015
Over the past 1-2 years, surveyed physicians

believe that the number of wet AMD patients in
their practice has grown modestly by 5-10% and
that approximately 50% of patients are treated
with Roches Avastin. The physician panelists
believe that the slow increase in wet AMD
patients is due to the chronic nature of the
disease.
Over the past 1-2 years, surveyed physicians believe that the number of wet AMD
patients in their practice has grown modestly by 5-10% and that approximately 50% of
patients are treated with Roches Avastin. The physician panelists believe that the slow
increase in wet AMD patients is due to the chronic nature of the disease. Additionally,
the amount of Avastin use depends on the size of the practice as larger groups are
more familiar with billing and will therefore use more Avastin (perhaps more than
50%), while smaller private groups may be closer to using Avastin in 25% of patients.
Avastin substitution has and continues to be a barrier to Lucentiss commercial
success in the U.S. Compounding pharmacies reconstitute the significantly cheaper
Avastin ($50 versus just under $2000 a dose) for intraocular injection despite its lack
of FDA approval for AMD. However, the current main competition is from Eylea, which
our Biotech team estimates will reach approximately $2.2B and $2.8B in 2015 and 2019
U.S. sales, respectively. In the U.S., our Pharma team estimates Lucentis sales of $1.7B
in 2015, decreasing to $1.5B in 2019 due to increased competition and as U.S. patents
begin to expire. Our Pharma team estimates ex-U.S. Lucentis sales of $2.5B in 2015,
declining to $1.8B in 2019 as the ex-US patents expire and competition continues to
increase.
Combination Therapy With Anti-PDGFs To Come Next For Retinal Disease
For wet AMD, a disease where patients ultimately

lose their vision, increased visual acuity is
paramount. Ultimately, our consultants suggest
that the vast majority of treated AMD patients
(2/3 and up) will want better visual acuity and
therefore go on PDGF treatment. Lastly, our
consultants note that as many as 10-20% of their
patients are not adequately managed by the
current anti-VEGF options, and therefore there is
also a need for more potent alternatives to help
better manage the disease in refractory patients
Fovista, an anti-PDGF aptamer, is the PDGF program that is furthest along in clinical
development. It is used in combination with anti-VEGF therapy (rather than in place of
it). OHR-102 due to potentially enter Phase III by the end of H1:2015 upon successful
final Phase II data is the second drug furthest along in clinical development and is
catching up to Fovista quickly. Allergan/Molecular Partners and Regeneron also have
PDGF programs, but they are much earlier-stage than Fovista and OHR-102. AntiVEGFs have revolutionized the treatment of wet AMD, but they produce most of their
improvement in about 4 months and then there is a plateau in efficacy. Moreover,
patients must be continuously dosed with frequent anti-VEGF to maintain the plateau;
data from several large trials were shown to suggest that switching from monthly
dosing to PRN results in a loss of efficacy. The biological explanation for this may be
that anti-VEGFs cause regression of tip cells at the distal end of neovascularization,
but the bulk of the vessel outgrowths are protected from the anti-VEGF by a lining of
pericytes. Anti-PDGF agents, in combination with the anti-VEGFs, denude the
overgrown vessels of pericytes and cause regression of the outgrowth. Current
development of anti-VEGF agents is aimed at extending the duration of treatment
thereby allowing for a fewer number of injections a year, or better compliance as
recent agents have largely failed to meaningfully increase visual acuity in long-term
studies. However, this dogma appears to be changing with advent of anti-PDGF
agents. PDGF agents in the case of Fovista and OHR-102 have demonstrated
significant visual acuity gains on top of anti-VEGF treatments when used in
combination. For wet AMD, a disease where patients ultimately lose their vision,
increased visual acuity is paramount. Ultimately, our consultants suggest that the vast
majority of treated AMD patients (2/3 and up) will want better visual acuity and
therefore go on PDGF treatment. Lastly, our consultants note that as many as 10-20%
of their patients are not adequately managed by the current anti-VEGF options, and
therefore there is also a need for more potent alternatives to help better manage the
disease in refractory patients.
www.cowen.com
19
By blocking the development of new blood vessels, supply of oxygen and nutrients is
cut off and, therefore, the diseases proliferation throughout the back of the eye.
Cowen and Company
Ohr Pharmaceutical
Equity Research
March 2, 2015
OHR-102 appears to have a strong possibility of

repeating the interim data. If this occurs, not only
would the perception of the program be
strengthened even more, but it would be
significantly de-risked as its head into Phase III
with the same exact clinical endpoint.
In June 2014, Ohr announced positive interim topline clinical results of a Phase II
study in wet AMD. Prior to the positive data release, few had any expectations for the
program much less an observed visual acuity benefit. However, since the data
which produced significant visual acuity benefits and our consultants believe is very
surprising and intriguing that perception has changed and we now approach the
final Phase II data readout in mid-to-late March with expectations of a visual acuity
benefit. While there is still substantial risk to any wet AMD data readout, OHR-102
appears to have a strong possibility of repeating the interim data. If this occurs, not
only would the perception of the program be strengthened even more, but it would be
significantly de-risked as it heads into Phase III with the same exact clinical endpoint.
Upon potential successful final Phase II data, Ohr has stated that they would be able
to initiate the Phase III program by the end of Q2:2015. We estimate that the time to
topline data could be 2-2.5 years accounting for 12-18 months of enrollment and the 9
month primary endpoint. 2.5 years would put the topline data readout at late 2017.
Since Ohr has Fast Track status, it could begin filing a rolling NDA submission during
the trial and then submit the final topline 9 month primary endpoint data in early 2018.
In essence, Ohr could receive approval even before the final 2-year study time point
just like Eylea. This would allow for a potential approval and launch by early 2019.
Lastly, we would note that this timeline could potentially be conservative by 6 months.
For Europe, Ohr plans to meet with regulators soon to nail down the requirements for
the Phase III program. However, we would note that Ohr will be measuring the primary
endpoint out to 12 months in case they require the standard 12 month endpoint.
Additionally, it will be a global program with clinical sites in the US and EU. It is quite
possible that the EU submission could come shortly after the US submission, but we
conservatively assume a year delay in the European launch.
There are a few points worth making with

respect to the enrollment criteria: (1) including all
lesions is representative of the overall patient
population and includes patients with both mild
and aggressive wet AMD. Ophthotech Ohrs
closest comp enrolled patients with only classic
containing CNV lesions. Essentially, these
patients should have more aggressive disease
and therefore larger improvements in visual
acuity may be seen with treatment; (2) Ohr
enrolled patients with visual acuity up to 20/40,
which also encompasses patients with fairly mild
disease and is where most wet AMD studies
start. In contrast, Ophthotech enrolled patients
with more advanced disease up to 20/63 visual
acuity; (3) Ophthotech restricted disc size, while
Ohr did not. (1), (2), and (3) would theoretically
have provided a relative ceiling effect of
response, which is impressive considering the
interims results
20
The ongoing Phase II IMPACT study has enrolled 142 patients (completed in April
2014) and is designed to measure the impact of twice-daily OHR-102 on visual acuity
in combination with PRN (as needed) Lucentis versus placebo drops plus PRN
Lucentis. Lucentis retreatment is mandated if any of the following present on SD-OCT:
retinal cystic changes, retinal fluid, subretinal fluid, or meaningful RPE elevation. The
primary endpoint was the reduction in frequency of Lucentis injections at 9 months.
Secondary visual acuity endpoints included mean visual acuity and the proportion of
patients gaining >3 lines (>15 letters; >3 line gainers) of vision acuity at 9 months.
The study was randomized, double-masked, and placebo controlled and enrolled
patients at 23 sites across the US. Enrollment criteria allowed for treatment nave wet
AMD patients with all lesion compositions (classic and occult), <12 disc areas in size,
and visual acuity ranging from 20/40 to 20/320. Diabetic patients without retinopathy
were also included. There are a few points worth making with respect to the
enrollment criteria: (1) including all lesions is representative of the overall patient
population and includes patients with both mild and aggressive wet AMD. Ophthotech
Ohrs closest comp enrolled patients with only classic containing CNV lesions.
Essentially, these patients should have more aggressive disease and therefore larger
improvements in visual acuity may be seen with treatment; (2) Ohr enrolled patients
with visual acuity up to 20/40, which also encompasses patients with fairly mild
disease and is where most wet AMD studies start. In contrast, Ophthotech enrolled
patients with more advanced disease up to 20/63 visual acuity; (3) Ophthotech
restricted disc size, while Ohr did not. (1), (2), and (3) would theoretically have
provided a relative ceiling effect of response, which is impressive considering the
interim results discussed in previous sections; and finally, (4) Ophthotech excluded
diabetic patients in their study because Fovista has been shown to almost completely
www.cowen.com
Consultants Believe Early OHR-102 Visual Acuity Data Are Surprising

And Intriguing; Final Phase II Data Due Mid-to-Late March
Cowen and Company
Ohr Pharmaceutical
Equity Research
March 2, 2015
Figure 12 Phase II IMPACT Study Design
Most importantly, the mean baseline vision of the

overall study patient population was 7-10 letters
better than most other wet AMD programs,
including the Fovista Phase II program. In
general, this means that Ohr enrolled relatively
healthier patients with better vision. Again, this
could have provided a ceiling effect.
Source: Ohr Pharma
The baseline demographics for the IMPACT study were consistent with the enrollment
criteria described above, which called for enrollment of a wide swath of patients with
varying disease severity mild, moderate, and severe patients. Most importantly, the
mean baseline vision of the overall study patient population was 7-10 letters better
than most other wet AMD programs, including the Fovista Phase II program. In
general, this means that Ohr enrolled relatively healthier patients with better vision.
Again, this could have provided a ceiling effect. Similarly, 47% of patients had occult
CNV, which is more moderate disease, while Ophthotechs Phase II studies enrolled
patients with only classic CNV or more aggressive disease. Average lesion size was
also on the larger end of the spectrum for the population and 16% of patients were
diabetic.
www.cowen.com
21
strip pericytes, which can cause serious health consequences. We wonder if Ohrs
topical formulation as opposed to Ophthotechs intravitreal injection may have a
less destructive effect on pericytes and ultimately be safer for diabetic patients. This
could have significant implications for the DME indication for which Ohr has a Phase
II program ongoing and is arguably the largest follow-on indication.
Cowen and Company
Ohr Pharmaceutical
Equity Research
March 2, 2015
Source: Ohr Pharma; modified by Cowen and Company
The Reduction Of Lucentis Injection Frequency Endpoint Is Not A Point Of Focus For
Us
Per the study plan, a pre-specified interim analysis was conducted with ~50% of the
planned total patient enrollment completing the study protocol. 62 patients 29 and
33 in the OHR-102 and placebo arms, respectively completed the 9 month treatment
period at interim. Not surprisingly, the primary endpoint, the difference in the
frequency of Lucentis injections, did not meet statistical significance at interim. Given
our knowledge of PRN studies (no difference in year 2 of the VIEW studies with
Eylea/Lucentis) and the fact that no study has shown the ability to reduce Lucentis
injections including Ophthotechs Phase II program we were not surprised by this
result. Furthermore, our panelists at our March 2014 Health Care Conference stated
that a fewer injections endpoint largely isnt clinically relevant. Thus, based on the
data (6.2 Lucentis injections for the OHR-102 arm and 6.4 for the placebo arm), we
find it improbable that this endpoint will meet statistical significance in the final data
set nor do we care. Sure a reduction in injections would be a positive outcome, but
ultimately what matters to clinicians is improving visual acuity. To Ohrs defense, this
primary endpoint was picked almost 4 years ago, where the role of anti-PDGFs in wet
AMD was significantly less defined. This will not be the primary endpoint in Phase III.
22
www.cowen.com
Figure 13 Baseline Demographics Of Phase II Impact Study
Cowen and Company
Ohr Pharmaceutical
Equity Research
March 2, 2015
Visual Acuity Endpoints Are All That Matter

When analysis was conducted on secondary, more clinically relevant visual acuity
endpoints, the data suggested a strong visual acuity benefit across the two most
common endpoints, mean visual acuity and the proportion of >3 line gainers. The
mean change in visual acuity at the end of 9 months for OHR-102 eye drops plus
Lucentis PRN was +10.4 letters versus +6.3 letters for placebo plus Lucentis PRN a
mean +4.1 letter improvement in visual acuity. Our consultants note that this 65%
visual acuity benefit is impressive, especially considering the potential ceiling effect,
described previously. However, it was not statistically significant (p=0.18) given the
small study size. With that in mind, our consultants note that it is too early and in too
few patients to believe that OHR-102 wont reach statistical significance on visual
acuity once the final data reads out. Ohr notes that meaningful visual acuity
improvements were seen as early as four weeks and the relative difference in visual
acuity appeared to increase throughout the study as depicted by the figure below. At
all times points evaluated from 4 to 38 weeks, visual acuity gains in the OHR-102
group relative to placebo were observed.
Figure 14 Mean Change In Visual Acuity For OHR-102 +Lucentis vs. Placebo + Lucentis
Source: Ohr Pharma
Additionally, 48.3% of OHR-102-treated patients

showed BCVA gains of >3 lines (>15 letters) on
a standard ETDRS eye chart compared with
21.2% in the placebo arm (p=0.025) a
statistically significant observation. Additionally,
the effect was sustained throughout the end of
the study with no saw tooth-like effects that
can be observed with anti-VEGF agent like
Lucentis. Overall, our consultants view both the
mean visual acuity and >3 line gain results as
impressive particularly if they hold up when the
final data reads out in the second half of March.
OHR-102 Hit On The Planned Phase III Primary Endpoint In Few Patients
Additionally, 48.3% of OHR-102-treated patients showed BCVA gains of >3 lines (>15
letters) on a standard ETDRS eye chart compared with 21.2% in the placebo arm
(p=0.025) a statistically significant observation. Additionally, the effect was
sustained throughout the end of the study with no saw tooth-like effects that can be
observed with anti-VEGF agent like Lucentis. Overall, our consultants view both the
mean visual acuity and >3 line gain results as impressive particularly if they hold up
when the final data reads out in the second half of March. Since the interim readout,
Ohr has met with the FDA and received confirmation that the Phase III endpoint will
be the proportion of patients with >3 line visual acuity gains, which was the exact
secondary endpoint that hit statistical significance at the interim readout per above in
www.cowen.com
23
The mean change in visual acuity at the end of 9

months for OHR-102 eye drops plus Lucentis
PRN was +10.4 letters versus +6.3 letters for
placebo plus Lucentis PRN a mean +4.1 letter
improvement in visual acuity. Our consultants
note that this 65% visual acuity benefit is
impressive, especially considering the potential
ceiling effect, described previously.
Cowen and Company
Ohr Pharmaceutical
Equity Research
March 2, 2015
As we head towards the final results, it is also important to consider that while 62
patients wasnt enough to hit on the mean visual acuity endpoint, 142 patients with
the observed +4.1 letter increase in visual acuity is just reaching the boundary
where there might be enough powering to reach statistical significance, per our
consultants. Of course, a 300-400 patients study would be ideally powered, but the
potential to hit statistical significance on mean visual acuity in the final dataset is a
source of additional upside and would further help to dispel any skeptics. However, it
is important to keep in mind that even if mean visual acuity doesnt hit which could
be a perceived negative all that matters is that the >3 line gainers Phase III endpoint
hits.
Figure 15 Percentage of > 3 Line VA Gainers For OHR-102 +Lucentis vs. Placebo + Lucentis
Source: Ohr Pharma
24
www.cowen.com
Our consultants suggest that this endpoint is also

has a good chance of reaching statistical
significance when the larger, final 142-patient
Phase II data set reads out by the end of March
2015 as the data in 62 patients gives us a good
feel for OHR-102s efficacy. One consultant
remarked that it would be shocking if the final
data showed no visual acuity benefit as the first
62 patients are very typical looking and he
would put the odds of success on the >3 line
gainers endpoint pretty high.
only 62 patients. Moreover, our consultants suggest that this endpoint also has a good
chance of reaching statistical significance when the larger, final 142-patient Phase II
data set reads out by the end of March 2015 as the data in 62 patients gives us a
good feel for OHR-102s efficacy. One consultant remarked that it would be
shocking if the final data showed no visual acuity benefit as the first 62 patients are
very typical looking and he would put the odds of success on the >3 line gainers
endpoint pretty high. If it does, our consultants would also consider the final Phase
III program employing the exact same endpoint and study design to be
significantly de-risked. In fact, they believe that upon a successful readout, OHR-102
would have the same probability of success in Phase III as Ophthotech which per
previous physician surveys has been pegged at around 75%. One consultant
believes that successful Phase II wet AMD results tend to have an 80% chance of
translating to Phase III. At the very least, we believe the early interim results
demonstrate that OHR-102 treats the back of the eye and may cause a significant
increase in visual acuity in wet AMD patients. Overall, our consultants suggest that
the mean change in visual acuity and >3 line gainer results look very much like
Ophthotech and the curves and outcomes look remarkably similar. While some
skepticism will always remain through the final Phase III data, consultants appear to
be looking at the program very differently now following the interim data.
Cowen and Company
Ohr Pharmaceutical
Equity Research
March 2, 2015
Figure 16 The Percentage Of Patients With >4, 5 Line Visual Acuity Gains
Source: Ohr Pharma
Per the figure below, an interesting trend was observed with OHR-102 combination
therapy. It resulted in patients transitioning to a better vision category with almost 50%
of patients gaining >3 lines (>15 letters per the chart) of visual acuity. Lucentis
performed as expected and most patients gained letters, but <3 lines of visual acuity
gain was observed in most patients (0-14 letters with a mean gain of 6-7 letters).
www.cowen.com
25
Further to the >3 line gainer results above, the Company also analyzed the proportion
of patients with >4 and >5 line visual acuity gains. 28% and 6% of OHR-102 and
placebo patients achieved >4 line visual acuity gains, respectively. This result reached
statistical significance with a p-value of 0.022. For >5 line visual acuity gains, 17% and
3% of OHR-102 and Lucentis patients met the endpoint, respectively (p=0.059).
Cowen and Company
Ohr Pharmaceutical
Equity Research
March 2, 2015
Source: Ohr Pharma
A Subgroup Analysis And Comparison To The Ophthotech Phase II Dataset

OHR-102 performed even better with mean visual
acuity gains of +13.8 letters over the +6.7 letters
in the placebo arm. With respect to >3 line
gainers, 67% of OHR-102 patients achieved the
endpoint, while only 20% of placebo patients did.
Our consultants believe this delta is really big
so big that it almost doesnt make sense.
Clearly, we have interpret these statements
positively.
As discussed previously, there are some significant differences between the patient
population that Ohr enrolled in the IMPACT study and the Ophthotech Phase II
program. Therefore, the Company completed an exercise or a subgroup analysis
looking at patients with classic containing CNV and up to 12 disc areas only a
population similar to the Ophthotech Phase II study. In this patient population, OHR102 performed even better with mean visual acuity gains of +13.8 letters over the +6.7
letters in the placebo arm. With respect to >3 line gainers, 67% of OHR-102 patients
achieved the endpoint, while only 20% of placebo patients did. Our consultants believe
this delta is really big so big that it almost doesnt make sense. Clearly, we have
interpreted these statements positively. With that said, given that the analysis includes
only includes 33 patients, we would not expect either endpoint to be statistically
significant (as they werent), but nonetheless we do find it impressive that OHR-102
once again reached statistical significance on >3 line gainers. Furthermore, the
magnitude of the mean visual acuity results is even more impressive than that
observed in the overall patient population and substantially higher (an estimated +2.5
letter difference when comparing both at 6 months) than that observed with Fovista in
Phase II. With that said, we would be remiss if we did not caveat this comparison by
noting that cross study comparisons using subgroup analyses typically cannot be
relied on too much. Nonetheless, while we dont place any reliance on this analysis,
we do find it interesting.
26
www.cowen.com
Figure 17 Visual Acuity Distribution: Placebo + Lucentis vs. OHR-102 + Lucentis
Cowen and Company
Ohr Pharmaceutical
Equity Research
March 2, 2015
Source: Ohr Pharma
Positive SD-OCT results were also observed with OHR-102 and presented at the ASRS
2014 meeting. The mean change in central retinal thickness for OHR-102 and the
placebo arms was -139 microns and -117 microns, respectively a meaningful benefit
of 22 microns at 9 months. Our consultants believe that these anatomical results
correlate with and help validate the positive visual acuity results discussed above.
Figure 19 OHR-102 + Lucentis vs. Placebo + Lucentis SD-OCT Changes
Source: Ohr Pharma
www.cowen.com
27
Figure 18 Subgroup Analysis: Mean Visual Acuity And >3 Line Gainers In Patients With Classic Containing CNV And Up To 12 Disc Areas
Cowen and Company
Ohr Pharmaceutical
Equity Research
March 2, 2015
An Additional Anatomical Analysis: SHRM Reduction
In a total of 54 patients (27 per arm) with

measurable SHRM at baseline, average
reductions of 75% and 56% were observed in the
OHR-102 and placebo arms, respectively.
Ohr has presented some individual case studies on SHRM reduction in the IMPACT
study via a quantitative analysis of SHRM via OCT. Two independent masked readers
at the Digital Angiography Reading Center (DARC) conducted the analysis by
measuring the greatest width and height observed on all scans. Only patients with
measurable SHRM at baseline were included in the analysis. In the case study below,
when comparing the baseline and end of treatment pictures, a clear reduction in
SHRM (indicated by the red arrow) does appear to be correlated with a 26 letter
increase in visual acuity. In a total of 54 patients (27 per arm) with measurable SHRM
at baseline, average reductions of 75% and 56% were observed in the OHR-102 and
placebo arms, respectively.
Figure 20 Subretinal Hyper-Reflective Material Analysis In A Single Patient
Source: Ohr Pharma
Across patients with measurable SHRM at baseline, the proportion of subject with
total SHRM reduction was 59% and 44% in the OHR-102 and placebo arms,
respectively. In classic containing CNV, or more aggressive disease, an even greater
relative SHRM reduction was observed (74% for OHR-102 vs. 43% for placebo; n=31).
The proportion of patients with total SHRM reduction was also enhanced in this
patient population (56% for OHR-102 vs. 31% for placebo). Furthermore, in the overall
28
www.cowen.com
Additional anatomical analysis was conducted by analyzing the presence of SHRM, or

subretinal hyper-reflective material. While SHRM is a recent academic discovery and
not widely accepted yet in the medical community, it does appear to be gaining some
traction. Additionally, Ophthotech has also shown total SHRM resolution data. It
appears to potentially be a marker of CNV disease activity by indicating neovascular
tissue, pre-fibrotic material, and other subretinal debris. Ohr subjects that SHRM
reduction may lead to better visual acuity due to normalization of the retinal
architecture via: (1) restoration of contact between the RPE and photoreceptors in
areas where SHRM has resolved; and (2) decreased development of subretinal
fibrosis.
Cowen and Company
Ohr Pharmaceutical
Equity Research
March 2, 2015
patient population and classic containing CNV patients, a relationship was observed
between increase in visual acuity and SHRM reduction. We believe it is possible that
these observations are related to the data announced by Ophthotech at AAO 2014,
which suggested that Fovista has the potential to reduce the severity of subretinal
fibrosis relative to Lucentis (mean change in severity of SRF at 24 weeks = 0.97 vs. 2.0;
p=0.003). Our consultants have noted that the subretinal fibrosis data appears very
interesting and clinically meaningful and it appears that there is a clear and
compelling anatomical benefit. The scientific rationale is that a decrease in PDGF
decreases fibroblast migration, which then may lead to a decrease in fibrosis.
Figure 21 An Observed Relationship Between SHRM Reduction And Improved Visual Acuity
Source: Ohr Pharma
Interim safety results suggest that OHR-102 eye drops were well tolerated and had a
comparable safety profile both in terms of ocular or systemic AEs to placebo eye
drops. Furthermore, there were no differences in intraocular pressure, no cataract
formation, and no corneal changes.
We Believe The Phase III Program Is Employing A Meaningfully De-Risked Endpoint
The Company will be required to complete the
standard two identical Phase III studies (one
primarily US-based and one EU-based) with 2
years of follow-up for safety. The design will be
almost identical to the Phase II program as it will
compare twice-daily OHR-102 + Lucentis with
placebo drops + Lucentis with 1:1 randomization
and 300-350 patients per arm. Between both
studies, there will be a total of 1,200-1,400
patients. It will be double-masked and placebocontrolled and include 125-150 clinical sites.
As previously discussed, Ohr received guidance from the FDA on September 16, 2014,
which allowed the Company to go ahead and proceed with the global Phase III
program once Phase II wraps up. The Company will be required to complete the
standard two identical Phase III studies (one primarily US-based and one EU-based)
with 2 years of follow-up for safety. The design will be almost identical to the Phase II
program as it will compare twice-daily OHR-102 + Lucentis with placebo drops +
Lucentis with 1:1 randomization and 300-350 patients per arm. Between both studies,
there will be a total of 1,200-1,400 patients. It will be double-masked and placebocontrolled and include 125-150 clinical sites. However, both arms will dose Lucentis
once-monthly as opposed to PRN in Phase II, which resulted from Lucentis injection
www.cowen.com
29
Across patients with measurable SHRM at

baseline, the proportion of subject with total
SHRM reduction was 59% and 44% in the OHR102 and placebo arms, respectively. In classic
containing CNV, or more aggressive disease, an
even greater relative SHRM reduction was
observed (74% for OHR-102 vs. 43% for placebo;
n=31). The proportion of patients with total
SHRM reduction was also enhanced in this
patient population (56% for OHR-102 vs. 31% for
placebo). Furthermore, in the overall patient
population and classic containing CNV patients,
a relationship was observed between increase in
visual acuity and SHRM reduction.
Cowen and Company
Ohr Pharmaceutical
Equity Research
March 2, 2015
Most importantly and as previously discussed,

the primary endpoint that the FDA agreed to is
the proportion of >3 line visual acuity gainers at
9 months, which is virtually identical to the Phase
II endpoint that reached statistical significance at
the interim readout.
Most importantly and as previously discussed, the primary endpoint that the FDA
agreed to is the proportion of >3 line visual acuity gainers at 9 months, which is
virtually identical to the Phase II endpoint that reached statistical significance at the
interim readout. The study will be 90% powered to detect low double-digit visual
acuity gains, which is a low bar when being compared to the interim data. Therefore,
Ohr ultimately just needs to replicate the final Phase II IMPACT study if successful in
late March. This is also similar, but perhaps more clinically relevant than the outdated
primary endpoint that Eylea and Lucentis used in registration, which was the
proportion of patients losing >3 lines of visual acuity. For perspective, one consultant
expects 90% of patients to not have >3 line vision loss using current standard of care
and treatment techniques. Our consultants note that achieving statistical significance
by actually gaining vision is more meaningful. Moreover, while recent registration
programs in wet AMD have tended to use a 12 month time point, clearly the FDA
doesnt see a meaningful difference and our consultants certainly dont as they note
that the vast majority of visual acuity is gained in the first 9 months and maintained
through 2 years. Furthermore, they note that Lucentis looked at >3 line gainers at 9
months in the historical ANCHOR, HARBOR, MARINA, and CATT studies (discussed
further in the Appendix) and that any visual acuity endpoint beyond 6 months is
adequate for the wet AMD space. Additionally, our consultants note that hitting
statistical significance on a >3 line gainer endpoint is probably somewhat easier to do
and safer than mean visual acuity due to the nature of what the clinical endpoint is
measuring. It measures the amount of patients with a strong, meaningful visual acuity
improvement as opposed to a mean measure, which may include outliers or noise.
For all of these reasons, we and our consultants believe that this choosing this
endpoint was an intelligent decision by Ohr and was an incremental de-risking event
for the Phase III program.
Figure 22 Planned OHR-102 Phase III Wet AMD Trial Design
Source: Ohr Pharma
30
www.cowen.com
every 5.5-6 weeks. The Company can change the Lucentis dosing to PRN any time
after the 9 month primary endpoint at its discretion.
Cowen and Company
Ohr Pharmaceutical
Equity Research
March 2, 2015
It is important to note that we do not expect the

various anti-PDGF drugs in development to
directly compete with the anti-VEGFs on market
or in development. The goal of anti-PDGFs is to
use them in combination and improve total visual
acuity. Therefore, one can assume that the two
are in fact complimentary.
As previously stated, we expect anti-PDGF therapy to be the next major class of drugs
for wet AMD. For that reason, several of the large players are exploring their own
proprietary programs. But before we discuss the PDGF competitive landscape, it is
important to note that we do not expect the various anti-PDGF drugs in development
to directly compete with the anti-VEGFs on market or in development. The goal of
anti-PDGFs is to use them in combination and improve total visual acuity. Therefore,
one can assume that the two are in fact complementary. We believe Ophthotech is
clearly in the lead with their PDGF program as the Phase III program is ongoing with
topline data to readout during 2016. If successfully developed, we expect them to
make it to market ~1-1.5 years before OHR-102, which would give Fovista a firstmover advantage. However, there are dramatic differences in the Fovista and OHR-102
product profiles that one should consider.
A second injection would require a second

preparation and most likely, ultimate a second
waiting room for many doctors. This may
ultimately cause an increase in time of 45-60
minutes per wet AMD patient. Therefore, the
Fovista approach does not seem to jive well with
consultants desires for therapeutics that can
manage the condition with fewer injections
and/or better potency, but we believe OHR-102
fits this profile.
Due to the fact that Fovista is an injection and a certain volume of drug will be
injected into the intravitreal space following an anti-VEGF injection, 30 minutes will be
required between both injections in order to avoid adverse increases in intraocular
pressure. This is a very meaningful difference per our consultants. Many
ophthalmologists already feel a huge burden as it is to shuffle patients through their
clinic to receive injections. Our consultants say that they and their staff already spend
much of their time giving anti-VEGF injections and would like to be free to perform
other (perhaps more interesting or lucrative) procedures. In fact, some physicians
have dedicated injection days where they just inject patients one after another.
Others note that patient volume continues to increase with sometimes up to 60
patients a day. Many have streamlined the process by ensuring that patients get
imaged immediately after dilating, then prepped for injection immediately, and treated
right away. A second injection would require a second preparation and most likely, a
second waiting room for many doctors. This may ultimately cause an increase in time
of 45-60 minutes per wet AMD patient. Therefore, the Fovista approach does not seem
to jive well with consultants desires for therapeutics that can manage the condition
with fewer injections and/or better potency, but we believe OHR-102 fits this profile.
We expect some physicians may adapt their practice to administer two injections, but
once OHR-102 potentially reaches the market not long after a potential Fovista launch,
our consultants anticipate that a significant portion of doctors would prefer to just
simply write a prescription for OHR-102 topical eye drops and send the patient home
with it after their initial anti-VEGF injection. Therefore, we would expect a significant
amount of Fovista patients to switch over to OHR-102 therapy. One consultant
believes that OHR-102 could completely cannibalize Fovista; although we believe this
is an aggressive assumption and that there is plenty of room for both in the market. In
general, our consultants expect OHR-102s product profile to be viewed more
favorably than Fovista and that many patients will simply opt for the drops.
The other two visible potential competitors in development are Regeneron and
Allergan/Molecular Partners and they are at least an estimated two years behind.
Regeneron plans to enter Phase II in H1:2015 and Allergan/Molecular Partners plans
to enter the clinic this year. Both are developing anti-VEGF/PDGF co-formulations,
which we and our consultants think will ultimately provide stiffer competition for
OHR-102 than Fovista (assuming all are successfully developed and approved) for the
following reasons: (1) they are co-formulated, so they will require a single injection
and wont significantly affect physicians practices; and (2) they would potentially be
launching on the back of successful anti-VEGF franchises in the case of Eylea (and
potentially Allergan/Molecular Partners abicipar pegol) which will give them a
significant advantage in terms of brand loyalty and market uptake.
www.cowen.com
31
Anticipated Competitive Dynamics For Anti-PDGF Programs In

Development
Cowen and Company
Ohr Pharmaceutical
Equity Research
March 2, 2015
Source: Ohr Pharma
Opthotechs Fovista Succeeded In Phase IIb; Phase III Under Way With Data By 2016
In June 2012, Opthotech reported top-line Phase IIb data showing statistical
superiority of the combination of Fovista and Lucentis over Lucentis alone. The 449patient trial demonstrated a 10.6-letter vision gain on the combination vs. 6.5 letters on
Lucentis alone at 24 weeks (p = 0.019). Full data were presented at the AAO meeting
in November 2012. Fovistas trial was randomized and blinded, and had three arms,
comparing 1.5 mg Fovista to 0.3 mg Fovista to placebo, all on a background of
Lucentis (n=152, n=149, and n=148, respectively). The full data revealed that there
were no baseline imbalances in randomization. Both drug arms achieved statistical
significance on the primary endpoint of superiority over Lucentis on visual acuity at 6
months (10.76 letters gained for 1.5 mg Fovista+Lucentis and 8.76 letters gained for
0.3mg Fovista+Lucentis vs. 6.52 letters gained for Lucentis alone). There was a clear
dose response across the entire duration of the trial, and the separation between
curves continued to grow through 6 months, suggesting longer-term follow-up could
reveal greater benefit. Unlike anti-VEGF treatment alone, in the drug arms there was
no evidence of plateauing efficacy after four months. There was a consistent, doseresponsive benefit across all subgroups examined, including stratification by baseline
lesion size, baseline vision, baseline fluid level (OCT reading), and proportion of
patients gaining at least 3 lines, 4 lines, and 5 lines. Moreover, there was also a dose
dependent benefit on proportion of patients losing visual acuity (patients losing >5
letters was 8.3% on high drug plus Lucentis vs. 21.5% on Lucentis alone). There were
no imbalances in AEs or SAEs (ocular or systemic) and no difference in intraocular
pressure between arms.
32
www.cowen.com
Figure 23 Wet AMD Competitive Landscape
Cowen and Company
Ohr Pharmaceutical
Equity Research
March 2, 2015
Source: Ophthotech S-1
An interesting wrinkle was that Fovista had no apparent effect on OCT. The presenter
and panelists seemed to agree that this was likely because the anti-VEGF treatment
cleared up the vascular leakage and fluid buildup, while anti-PDGF would affect other
potential biomarkers. Some possible biomarkers were examined in the trial, but
consultants told us they were largely still in the validation stage.
All the consultants we have spoken with are convinced that Fovistas Phase IIb data
reflects genuine additive efficacy over Lucentis alone, the first time this has ever been
seen in a clinical trial prior to the OHR-102 interim results. Consultants expressed
extreme optimism that Fovistas Phase IIb results would lead to success in Phase III,
assuming the Phase IIb trial design is simply replicated (with a one-year efficacy
follow-up). Their optimism was based on: (1) a robust, clinically and statistically
significant, dose-dependent treatment effect that increased with time and was present
in all subgroups; (2) blinded trial design; (3) large patient number in the trial; (4) no
imbalance in baseline randomization; and (5) clean safety. In fact, consultants appear
to estimate the chance of success in the Phase III trial at approximately 75%. Some
consultants were disdainful of any suggestion, based on cross-trial comparisons, that
the Lucentis arm performed unusually poorly, mainly because it is scientifically
unsound to make cross-trial comparisons, but also because 6 letters gained in 6
months actually struck them as about the expected level of efficacy for Lucentis. Also,
the loss of vision in the Lucentis arm of Ophthotechs trial was less than they would
have expected, suggesting to them that if anything these Lucentis patients did better
than usual. However, other consultants are less convinced and come to the conclusion
that the Lucentis arm behaved unusually poorly as the ANCHOR studies, which
enrolled all classic CNV patients similar to Ophthotech, showed a much larger +10
letter visual acuity benefit with Lucentis.
Fovista Partnered With Novartis; The $1B+ Deal Validates The Anti-PDGF Approach
In May 2014, Ophthotech signed a collaboration agreement with Novartis for Fovistas
ex-U.S. rights. Under the agreement, Ophthotech received a $200MM upfront
payment, and is further entitled to receive up to $130MM in Phase III enrollmentbased milestones, up to $300MM in ex-U.S. approval milestones, and another
$400MM in ex-U.S. sales milestones. In addition, Ophthotech will receive royalties on
ex-U.S. sales. The total deal value is estimated over $1B. We view these terms as very
www.cowen.com
33
Figure 24 Fovistas Efficacy On Top Of Lucentis In Wet AMD
Cowen and Company
Ohr Pharmaceutical
Equity Research
March 2, 2015
Fovistas IP Is An Issue To Be Considered. According to Ophthotechs S-1, filed in

August 2013 in conjunction with its IPO, Fovistas composition of matter patents expire
in 2017 (U.S.) and 2018 (E.U.) The company does have a method of use patent for
treatment in combination with Avastin or Lucentis, expiring in 2024. Still, with
Opthotech projecting a possible NDA/MAA filing around YE:16, it appears possible
that the drug will launch with little or no composition patent protection remaining.
Fovista Phase III Development Under Way, Data Expected In 2016
Ophthotech began its Phase III program in August 2013. The program consists of
three Phase III trials enrolling nearly 1,900 patients at 225 centers worldwide. The first
trial is a 622-patient study comparing monthly Lucentis + Fovista to Lucentis alone.
The second is an identical, duplicate study. The third study will randomize patients to
receive either monthly Avastin or bimonthly Eylea, with or without monthly Fovista. The
endpoint of all the studies is the change in visual acuity from baseline at 12 months.
The first two studies began U.S. enrollment in August 2013, with enrollment in the last
study planned to begin shortly. Ophthotech has guided that the Phase III data will be
available in 2016 and it is planning to submit regulatory applications in the U.S./E.U.
before YE:16.
Physicians Remain Very Optimistic About Fovistas Phase III Program; We Believe This
Bodes Well For OHR-102
In the past, physicians have been impressed by Fovistas efficacy, and believed the
magnitude is clinically meaningful and the duration and size of the trial is robust. In
our October 2014 survey, the majority of surveyed physicians (n=39) continue to
believe that Fovista has a 75% probability of success in the ongoing Phase III trial. In
fact, surveyed physicians in October 2014 were even more optimistic than they were in
March 2014. As noted previously, our consultants believe that upon OHR-102 success
in >3 line gainers in the final 142-patient data readout, that the program will have a
similar probability of success in Phase III. The majority of surveyed physicians also
believe that there is a 50-75% likelihood that Fovista, when combined with anti-VEGF
treatment in wet AMD, will demonstrate a reduction in the number of annual
intraocular wet AMD injections required during a future Phase II study. We are less
convinced of this outcome given that this has not been observed in a study to date.
34
www.cowen.com
lucrative for Ophthotech, particularly, given the fact that the company was able to
secure up to $330MM in near-term milestones, while retaining full U.S. rights. Under
the agreement, Novartis will also develop a co-formulation of Fovista with its antiVEGF candidates (likely with ESBA-1008). We view this partnership to have strategic
importance for Novartis, who also has exclusive ex-U.S. rights to Lucentis. In light of
this recent collaboration, and keeping in mind Novartis goal to co-formulate Fovista
with an anti-VEGF therapy.
Cowen and Company
Ohr Pharmaceutical
Equity Research
March 2, 2015
March 2014
October 2014
Source: Cowen and Company March 2014 Health Care And October 2014 Therapeutics Conferences; Vistacom
Consultants Think Anti-PDGFs Have The Potential To Impact Branded Anti-VEGF

Long-Term Sales
Consultants agree that they would use anti-PDGFs widely if approved, offering it to all
their new wet AMD patients and a significant fraction of their continuing patients who
had not achieved satisfactory efficacy and/or dose interval. Several doctors emphasize
that their patients are clamoring for more effective agents and they expected demand
to be brisk. Interestingly, the introduction of anti-PDGFs to the marketplace may prove
problematic for branded anti-VEGF sales even if it is not coformulated. While some
ophthalmologists expect to use anti-PDGFs as labeled initially, others may be more
experimental and plan to attempt to optimize dosing of the two agents. Given the
efficacy of these combinations, it would also seem that the anti-VEGF dosing interval
could be extended, although that has yet to be seen. When asked how the treatment
paradigm is likely to evolve over time, our consultants say they expect a shift in
treatment more toward an induction-maintenance paradigm. Specifically, they expect
patients to be initially treated with several doses of simultaneous anti-VEGF and antiPDGF agents until their neovascularizations regressed. At that point, the physicians
expect to maintain the remission with just one of the agents. While they admit they
are not sure whether the maintenance agent will be an anti-PDGF or an anti-VEGF,
both strongly suspect the former based on the biologic rationale. Thus, the physicians
expect that approval of anti-PDGFs would be a net negative for all anti-VEGF sales.
However, we note that this dynamic remains to be observed in clinical trials. In
addition, Ophthotechs management has suggested that its market research indicates
that physicians would be reluctant to use two expensive branded drugs together, so
that approval of Fovista could drive a shift toward Avastin for financial reasons. If true,
this would of course also be a negative for branded anti-VEGFs. However, this point of
view is not shared by academic physicians, who would continue to use their anti-VEGF
of choice following approval of anti-PDGFs regardless of cost. We tend to agree with
the specialists on this point, and note that Eyleas perceived superiority to Avastin has
allowed Eylea to capture some Avastin patients despite the substantially higher price.
We believe physicians will continue using the drug they perceive as most effective,
unless patients insurance will not cover it. Visual acuity is paramount.
www.cowen.com
35
Figure 25 Probability Of Fovista Phase III Success
Cowen and Company
Ohr Pharmaceutical
Equity Research
March 2, 2015
Regenerons Own Anti-PDGF To Enter Phase II Studies In H1:15
Related Retina Disorders Are Natural Label Expansion Opportunities

DME, RVO And Relatively Smaller Commercial Retina Opportunities
Consultant commentary on three other retinal indications in which anti-angiogenic
agents are used highlights the relatively smaller commercial opportunity vs. wet AMD:
these include diabetic macular edema (DME), diabetic retinopathy or proliferative
diabetic retinopathy (PDR), and two retinal vein occlusion types: branch retinal vein
occlusion (BRVO) and central retinal vein occlusion (CRVO). Wet AMD is the most
commonly treated of these conditions: our consultants estimate that for each wet
AMD patient in his practice, there is one patient with either DME or one of the RVOs.
Of the patients with DME or RVOs, 80-85% have DME while 15-20% have an RVO.
Among the RVO patients, 70% are BRVO and 30% are CRVO. Roche estimates that
there may be 190K RVO patients in the U.S. Our consultant commented that RVO
patients are often asymptomatic and self-resolving and frequently lend themselves to
a watch-and-wait approach. We estimate about one-third of RVO patients may be
treated by a physician, and of these, about 30% may receive an anti-VEGF in any given
year and a majority of those would be eligible for an anti-PDGF. Cowen Biotech teams
2019 U.S. RVO estimate for Eylea is just $120MM.
DME is a much more common condition than CRVO, though we still believe it is a
relatively small market opportunity compared to wet AMD. Diabetic retinopathy is the
most prevalent cause of vision loss in working-aged adults. Diabetic macular edema
(DME), the most common complication of diabetic retinopathy, refers to a swelling of
the retina caused by fluid leakage from capillaries in the macula. DME results from
chronically elevated blood sugar levels, which cause structural changes in the
endothelium of retinal blood vessels, leading to vascular dysfunction and eventually
vascular occlusion. It is believed that upregulation of growth factors in the retina, such
as IL-6 and VEGF, induce vascular permeability. That vascular permeability leads to a
breakdown of the blood-retina barrier, allowing fluid and proteins to leak into the
retina, thickening the macula and distorting nerve cells. The result is a reversible
decrease in visual acuity which becomes permanent if left untreated.
Though we believe that the U.S. DME population may be nearly as large as the wet
AMD population, the opportunity for anti-VEGFs in this indication appears to be
somewhat smaller. We estimate that there are nearly 400K DME patients in the U.S.
today. However, Roche estimates that only about half of patients have clinically
significant disease, such that only about 190K DME patients are under an
ophthalmologists care. On its Q2:13 call, Regeneron gave comparable estimates,
suggesting that there might by 570K total DME patients in the U.S. today, with 40%
36
www.cowen.com
Regeneron entered the clinic with a co-formulation of its own human anti-PDGFR
antibody (REGN2176) and Eylea in Q1:2014 under an agreement with Bayer.
Regeneron will conduct the first proof of concept trial, after which Bayer will have an
opt-in right. Should Bayer opt in, it will gain full ex-U.S. commercialization rights and
share the cost of development (25% of global development costs and 50% of
development costs exclusively for territory outside the U.S.). Regeneron will retain full
rights in the U.S., and be entitled to a 50% profit share ex-U.S. In February 2015,
Regeneron announced that initial data from a Phase I trial of REGN2176-3 showed
that the combination is safe and well-tolerated. Based on Phase I data, REGN has also
identified two doses that it plans to move forward in Phase II studies in H1:15.
Cowen and Company
Ohr Pharmaceutical
Equity Research
March 2, 2015
As previously discussed, OHR-102 is being tested in investigator-sponsored studies

for RVO and PDR with Phase II data for both studies to readout later this year. For the
PDR indication, an open-label monotherapy study has been initiated in 5 treatment
nave patients who have not undergone laser or anti-VEGF therapy. The study (003) is
being run by Dr. Michael Elman and an initial case report entitled Regression of
Retinal Neovascularization in Proliferative Diabetic Retinopathy Using Squalamine
Lactate Eye Drops was presented at the Macular Society Annual Meeting on
February 19, 2014. In the case study, the first patient to complete treatment had
observed regression of retinal vessels within two months and maintained it through six
months of QID monotherapy treatment. Interestingly vessel regrowth was observed at
one and two months after therapy was stopped.
The ongoing study 004 in RVO is a single-site, prospective clinical trial in 20 patients
with a primary endpoint of ETDRS letters gained at week 10. Patients are being treated
for 10 weeks with OHR-102 and also receiving Lucentis injections at week 2 and 6.
Patients were initially randomized 1:1 to receive OHR-102 or discontinue drops until
week 38 with rescue Lucentis injections based on OCT criteria and laser treatment
was not permitted. Per the table below, an average increase of +20 letters was
observed across all RVO patients and 80% of patients gained >3 lines. Worth noting,
only 1 of 20 eyes qualified for a third injection of Lucentis at week 10.
Figure 26 Visual Acuity At Week 10 From RVO Study 004
Source: Ohr Pharma
www.cowen.com
37
(230K) treated with anti-VEGFs. As an alternative to anti-VEGFs, though, laser

photocoagulation therapy directs light into the eye where heat is used to cauterize
abnormal blood vessels to cut off sites of leakage. Our consultant treats DME initially
by laser, which he finds to be adequately effective in 50-60% of cases. In laserrefractory patients, he uses adjunctive anti-VEGFs or steroids, such as triamcinolone.
Patients given anti-VEGFs do not typically receive lifelong continuous therapy:
treatment is more acute, ranging from one or two injections to several months of
therapy. Overall, our consultants estimate that the number of VEGF injections given
each year for DME is 10-25% of the number given for wet AMD. Thus, both number of
treated patients and duration of therapy appear to limit the commercial opportunity for
anti-VEGFs in DME to perhaps 10-25% of the wet AMD market. Regeneron has
suggested that it has data showing that patients receiving laser up front do not
subsequently do as well on anti-VEGF therapy, and expects the proportion of patients
treated with anti-VEGFs to grow upon introduction of Eylea.
Cowen and Company
Ohr Pharmaceutical
Equity Research
March 2, 2015
Early Sustained-Release Programs Partnered With Alcon

Ohrs research agreement with Alcon clearly provides a source of validation for Ohrs
programs and expertise in ophthalmology. This collaboration employing Ohrs
sustained-release platform technology encompasses four active research programs in
glaucoma, steroid-induced glaucoma, eye allergy, and retinal disease. Depending on
the outcome of these early programs with partner Alcon, Ohr anticipates potentially
filing an IND for one program by the end of H1:2015. While little is known about these
early programs, the technology involves producing microfabricated microparticles
using a dissolvable gelatin hydrogel template. A silicon wafer master template is used
to from the hydrogel template and ultimate mold, which is then used to create
microparticles. The hydrogel mold is loaded with polymer and drug and eventually the
mold dissolves gently in water leaving behind finished microparticles. Ohr believes
that the drug loading capacity of these microparticles is 30% higher than conventional
methods and that it has significant advantages over currently available microparticle
drug delivery systems prepared using emulsion methods.
Figure 27 Sustained-Release Platform Technology Schematic
Source: Ohr Pharma
The only data that has been shared for this technology by Ohr is some in vitro data
with the sustained-release protein, SKS1002. The current SKS1002 formulation has
demonstrated sustained-protein release up to 3 months. The ultimate goal is to
achieve duration up to 3-6 months. We would note that this technology appears to be
very similar to Ocular Therapeutix sustained-release programs and hydrogel
38
www.cowen.com
For DME, the Company is running its own Phase II study (005) with final data to
readout in mid-2016. The topline data will include several visual acuity endpoints,
most likely mean visual acuity and >3 line gainers. Based on the interim IMPACT data,
this study has been expanded to 90 patients looking at visual acuity parameters over
24 weeks.
Cowen and Company
Ohr Pharmaceutical
Equity Research
March 2, 2015
Figure 28 Percentage Of Cumulative SKS1002 Protein Released In Vitro
Source: Ohr Pharma
www.cowen.com
39
technology, which has piqued investors interest as of late. In particular, a 3 month

release profile appears attractive for the glaucoma and allergic conjunctivitis
indications where compliance is very poor and results in suboptimal clinical outcomes.
We believe a product candidate with potential 6 month duration in these indication
would be game changing.
Cowen and Company
Ohr Pharmaceutical
Equity Research
March 2, 2015
2013A
2014A
2015E
2016E
2017E
2018E
2019E
2020E
2021E
2022E
2023E
2024E
U.S. We t A MD
% diagnosed
% growth
454
80%
361
95%
343
+6%
476
80%
381
96%
364
+6%
500
80%
400
96%
384
+6%
525
80%
420
96%
403
+5%
552
80%
441
96%
424
+5%
579
80%
463
96%
445
+5%
608
80%
486
96%
467
+5%
632
80%
506
96%
486
+4%
658
80%
526
96%
505
+4%
684
80%
547
96%
525
+4%
711
80%
569
96%
546
+4%
740
80%
592
96%
568
+4%
769
80%
615
96%
591
+4%
Avastin
U.S. A va s tin Sa le s In We t A MD ($MM)
26%
89
5.6
$50
$2 5
33%
120
5.5
$50
$3 5
37%
142
5.5
$50
$4 0
40%
161
5.5
$50
$4 5
40%
169
5.5
$50
$4 5
41%
182
5.5
$50
$5 0
42%
196
5.5
$50
$5 5
42%
204
5.5
$50
$5 5
42%
212
5.5
$50
$6 0
42%
221
5.5
$50
$6 0
42%
229
5.5
$50
$6 5
42%
239
5.5
$50
$6 5
42%
248
5.5
$50
$7 0
+757%
+40%
+14%
+13%
+0%
+11%
+10%
+0%
+9%
+0%
+8%
+0%
+8%
35%
120
5.6
$1,950
$1 ,3 1 0
23%
84
5.5
$1,950
$9 0 0
17%
65
5.5
$1,950
$7 0 0
16%
65
5.5
$1,950
$6 9 0
15%
64
5.5
$1,950
$6 8 0
15%
67
5.5
$1,950
$7 1 5
15%
70
5.5
$1,950
$7 5 0
15%
73
5.5
$1,950
$7 8 0
15%
76
5.5
$1,950
$8 1 5
15%
79
5.5
$1,950
$8 4 5
15%
82
5.5
$1,950
$8 8 0
15%
85
5.5
$1,950
$9 1 5
15%
89
5.5
$1,950
$9 5 0
+4%
-31%
-22%
-1%
-1%
+5%
+5%
+4%
+4%
+4%
+4%
+4%
+4%
39%
53%
134
5.4
$1,850
$1 ,3 3 4
44%
66%
160
5.2
$1,850
$1 ,5 4 0
46%
73%
177
5.2
$1,850
$1 ,7 0 0
45%
74%
182
5.2
$1,850
$1 ,7 4 5
45%
75%
191
5.2
$1,850
$1 ,8 3 5
44%
75%
196
5.2
$1,850
$1 ,8 8 0
43%
72%
201
5.2
$1,850
$1 ,9 3 0
43%
66%
209
5.2
$1,850
$2 ,0 1 0
43%
59%
217
5.2
$1,850
$2 ,0 9 0
43%
53%
226
5.2
$1,850
$2 ,1 7 5
43%
50%
235
5.2
$1,850
$2 ,2 6 0
43%
48%
244
5.2
$1,850
$2 ,3 5 0
43%
46%
254
5.2
$1,850
$2 ,4 4 5
+64%
+15%
+10%
+3%
+5%
+2%
+3%
+4%
+4%
+4%
+4%
+4%
+4%
66%
226
66%
240
66%
254
66%
266
66%
280
66%
294
66%
308
2%
6
11.0
$750
66%
321
8%
24
10.7
$750
66%
333
15%
50
10.5
$750
66%
347
23%
78
10.3
$750
66%
361
28%
99
10.0
$750
66%
375
32%
120
10.0
$750
66%
390
36%
140
10.0
$750
% growth
Lucentis
U.S. Luc e ntis Sa le s In We t A MD ($MM)
% growth
Eylea
U.S. Ey le a Sa le s In We t A MD ($MM)
% growth
OHR-1 0 2
U.S. OHR-1 0 2 Sa le s In We t A MD ($MM)
$5 0
% gr ow th
$1 9 5
$3 9 5
$6 0 0
$7 4 5
$9 0 0
+2 9 0 %
+1 0 3 %
+5 2 %
+2 4 %
+2 1 %
$1 ,0 5 5
$1,310
$900
$700
$690
$680
$715
$750
$780
$815
$845
$880
$915
$950
% growth
Eylea Sales ($MM)
+4%
$1,334
-31%
$1,540
-22%
$1,700
-1%
$1,745
-1%
$1,835
+5%
$1,880
+5%
$1,930
+4%
$2,010
+4%
$2,090
+4%
$2,175
+4%
$2,260
+4%
$2,350
+4%
$2,445
+64%
+15%
+10%
+3%
+5%
+2%
+3%
$50
+4%
$195
+4%
$395
+4%
$600
+4%
$745
+4%
$900
+4%
$1,055
% growth
T OT A L U.S. WET A MD SA LES ($MM)
% gr ow th
$2 ,6 4 4
$2 ,4 4 0
$2 ,4 0 0
$2 ,4 3 5
$2 ,5 1 5
$2 ,5 9 5
$2 ,7 3 0
+290%
$2 ,9 8 5
+103%
$3 ,3 0 0
+52%
$3 ,6 2 0
+24%
$3 ,8 8 5
+21%
$4 ,1 6 5
+17%
$4 ,4 5 0
+2 7 %
-8 %
-2 %
+1 %
+3 %
+3 %
+5 %
+9 %
+1 1 %
+1 0 %
+7 %
+7 %
+7 %
40
www.cowen.com

+2%

- Very rapid uptake upon launch
+3% - Ma r k e t le a de r

- Potential launch in early 2019
- Priced between $500-$1,000 for market uptake
+ 78% - Should e xpe r ie nc e r a pid upta k e
+1 7 %
U.S. We t A MD
% growth
OHR-102 Sales ($MM)

+2%
+3%
+ 4%
Figure 29 US Wet AMD Market Model
Cowen and Company
Ohr Pharmaceutical
Equity Research
March 2, 2015
2013A
2014E
2015E
2016E
2017E
2018E
2019E
2020E
2021E
2022E
2023E
2024E
Ex-U.S. We t A MD
% diagnosed
% growth
681
80%
541
95%
514
+6%
715
80%
572
96%
546
+6%
750
80%
600
96%
576
+6%
788
80%
630
96%
605
+5%
827
80%
662
96%
635
+5%
869
80%
695
96%
667
+5%
912
80%
730
96%
700
+5%
949
80%
759
96%
728
+4%
986
80%
789
96%
758
+4%
1,026
80%
821
96%
788
+4%
1,067
80%
854
96%
819
+4%
1,110
80%
888
96%
852
+4%
1,154
80%
923
96%
886
+4%
- We estimate 50% larger than U.S.
Avastin
Ex-U.S. A va s tin Sa le s In We t A MD ($MM)
55%
282
5.6
$50
$7 9
48%
262
5.5
$50
$7 0
44%
254
5.5
$50
$7 0
43%
260
5.5
$50
$7 0
43%
273
5.5
$50
$7 5
43%
287
5.5
$50
$8 0
43%
301
5.5
$50
$8 5
43%
313
5.5
$50
$8 5
43%
326
5.5
$50
$9 0
43%
339
5.5
$50
$9 5
43%
352
5.5
$50
$9 5
43%
366
5.5
$50
$1 0 0
43%
381
5.5
$50
$1 0 5

-11%
-11%
+0%
+0%
+7%
+7%
+6%
+0%
+6%
+6%
+0%
+5%
+5%
38%
195
5.6
$1,950
$2 ,1 3 3
36%
197
5.5
$1,950
$2 ,1 1 0
34%
196
5.5
$1,950
$2 ,1 0 0
32%
194
5.5
$1,950
$2 ,0 7 5
30%
191
5.5
$1,950
$2 ,0 4 5
29%
193
5.5
$1,950
$2 ,0 7 5
28%
196
5.5
$1,950
$2 ,1 0 5
27%
197
5.5
$1,950
$2 ,1 1 0
26%
197
5.5
$1,950
$2 ,1 1 5
25%
197
5.5
$1,950
$2 ,1 1 5
24%
197
5.5
$1,950
$2 ,1 1 0
23%
196
5.5
$1,950
$2 ,1 0 0
22%
195
5.5
$1,950
$2 ,0 9 0
+10%
-1%
-0%
-1%
-1%
+1%
+1%
+0%
+0%
+0%
-0%
-0%
-0%
7%
16%
37
5.4
$1,850
$3 7 0
16%
31%
87
4.7
$1,850
$7 6 0
22%
39%
127
4.3
$1,850
$1 ,0 1 0
25%
44%
151
4.3
$1,850
$1 ,2 0 5
27%
47%
172
4.3
$1,850
$1 ,3 6 5
28%
49%
187
4.3
$1,850
$1 ,4 8 5
29%
51%
203
4.3
$1,850
$1 ,6 1 5
30%
52%
219
4.3
$1,850
$1 ,7 4 0
31%
49%
235
4.3
$1,850
$1 ,8 7 0
32%
46%
252
4.3
$1,850
$2 ,0 0 5
33%
44%
270
4.3
$1,850
$2 ,1 5 0
34%
43%
290
4.3
$1,850
$2 ,3 0 5
35%
43%
310
4.3
$1,850
$2 ,4 7 0
+1847%
+105%
+33%
+19%
+13%
+9%
+9%
+8%
+7%
+7%
+7%
+7%
+7%
66%
339
66%
360
66%
380
66%
399
66%
419
66%
440
66%
462
66%
481
1%
5
11.0
$500
$2 5
66%
500
6%
30
10.8
$500
$1 6 0
66%
520
13%
65
10.5
$500
$3 4 0
66%
541
18%
97
10.3
$500
$5 0 0
66%
562
22%
124
10.0
$500
$6 2 0
66%
585
25%
146
10.0
$500
$7 3 0
+5 4 0 %
+1 1 3 %
+4 7 %
+2 4 %
+1 8 %
% growth
Lucentis
Ex-U.S. Luc e ntis Sa le s In We t A MD ($MM)
% growth
Eylea
Ex-U.S. Ey le a Sa le s In We t A MD ($MM)
% growth
OHR-1 0 2
Ex-U.S.OHR-1 0 2 Sa le s In We t A MD ($MM)
% gr ow th
Ex-U.S. We t A MD
% growth
Eylea Sales ($MM)
% growth
OHR-102 Sales ($MM)
% growth
T OT A L Ex-U.S. WET A MD SA LES ($MM)
% gr ow th
+ 0% - Ma r k e t le a de r

- Rapid uptake, but less so than the U.S.
+ 11%

- Potential launch in early 2020; could be conserv.
- Assume price 2/3 of U.S.
+ 96% - Quic k upta k e , but s lowe r tha n U.S.
$2,110
$2,100
$2,075
$2,045
$2,075
$2,105
$2,110
$2,115
$2,115
$2,110
$2,100
$2,090
+0%
+10%
$370
-1%
$760
-0%
$1,010
-1%
$1,205
-1%
$1,365
+1%
$1,485
+1%
$1,615
+0%
$1,740
+0%
$1,870
+0%
$2,005
-0%
$2,150
-0%
$2,305
-0%
$2,470
+11%
+1847%
+105%
+33%
+19%
+13%
+9%
+9%
+8%
$25
$2 ,5 0 3
$2 ,8 7 0
$3 ,1 1 0
$3 ,2 8 0
$3 ,4 1 0
$3 ,5 6 0
$3 ,7 2 0
$3 ,8 7 5
+7%
$160
+7%
$340
+7%
$500
+7%
$620
+7%
$730
+540%
$4 ,1 4 5
+113%
$4 ,4 6 0
+47%
$4 ,7 6 0
+24%
$5 ,0 2 5
+18%
$5 ,2 9 0
+15%
+8%
+5%
+4%
+4%
+4%
+4%
+7%
+8%
+7%
+6%
+5%
W.W. We t A MD
$3,443
$3,010
$2,800
$2,765
$2,725
$2,790
$2,855
$2,890
$2,930
$2,960
$2,990
$3,015
$3,040
% growth
Eylea Sales ($MM)
+7%
$1,704
-13%
$2,300
-7%
$2,710
-1%
$2,950
-1%
$3,200
+2%
$3,365
+2%
$3,545
+1%
$3,750
+1%
$3,960
+1%
$4,180
+1%
$4,410
+1%
$4,655
+1%
$4,915
% growth
OHR-102 Sales ($MM)
+105%
+35%
+18%
+9%
+8%
+5%
+5%
$50
+6%
$220
+6%
$555
+6%
$940
+6%
$1,245
+6%
$1,520
+6%
$1,785
+340%
$6 ,8 6 0
+152%
$7 ,4 4 5
+69%
$8 ,0 8 0
+32%
$8 ,6 4 5
+22%
$9 ,1 9 0
+17%
$9 ,7 4 0
+6%
+9%
+9%
+7%
+6%
+6%
% gr ow th
$2,133
+27%
% growth
T OT A L W.W. WET A MD SA LES ($MM)

$5 ,1 4 7
+27%
$5 ,3 1 0
+3%
$5 ,5 1 0
+4%
$5 ,7 1 5
+4%
$5 ,9 2 5
+4%
$6 ,1 5 5
+4%
$6 ,4 5 0
+5%

+ 4%
+1%
+7%
+ 4%
www.cowen.com
41
Figure 30 Ex-US Wet AMD Market Model
Cowen and Company
Ohr Pharmaceutical
Equity Research
March 2, 2015
OHR PHA RMA - 2 0 1 5 -2 0 2 5 EST IMA T ED A NNUA L EPS BUILDUP ($MM)

F iscal Year E nded S ept em ber 30
2014
2015E
2016E
2017E
2018E
2019E
2020E
2021E
2022E
2023E
2024E
2025E
$50.0
$195.0
$395.0
$600.0
$745.0
$900.0
$1,055.0
+290%
+103%
+52%
+24%
+21%
+17%
$25.0
$160.0
$340.0
$500.0
$620.0
$730.0
$ 220. 0
$ 555. 0
5YR C GR C om m ent s
O HR- 102
U.S. Wet AMD Sales ($MM)
% Growth
Ex-U.S. Wet AMD Sales ($MM)
% Growth
Tot al O hr Rev enues
+540%
NM
NM
NM
NM
NM
+ 340%
+ 152%
+ 69%
+ 32%
+ 22%
+ 17%
$0.0
$0.0
$0.0
$0.0
$5.0
$22.0
$50.0
$84.6
$99.6
$121.6
$125.0
Gross Profit
$0.0
$0.0
$0.0
$0.0
$0.0
$45.0
$198.0
$505.1
$855.4
$1,145.4
$1,398.4
$1,660.1
NM
NM
NM
NM
NM
90.0%
90.0%
91.0%
91.0%
92.0%
92.0%
93.0%
$5.1
$7.5
$15.0
$20.0
$25.0
$50.0
$90.0
$150.0
$240.0
$290.0
$340.0
$390.0
% of Revs
% of Revs
$ 940. 0
$ 1, 245. 0
$ 1, 520. 0
$ 1, 785. 0
NM
NM
NM
NM
NM
100.0%
40.9%
27.0%
25.5%
23.3%
22.4%
21.8%
$4.0
$20.0
$40.0
$30.0
$20.0
$15.0
$15.0
$10.0
$10.0
$10.0
$10.0
$10.0
NM
NM
NM
NM
NM
30.0%
6.8%
1.8%
1.1%
0.8%
0.7%
0.6%
$9.1
$27.5
$55.0
$50.0
$45.0
$65.0
$105.0
$160.0
$250.0
$300.0
$350.0
$400.0
NM
NM
NM
NM
NM
130.0%
47.7%
28.8%
26.6%
24.1%
23.0%
22.4%
($9.1)
($27.5)
($55.0)
($50.0)
($45.0)
($20.0)
$93.0
$345.1
$605.4
$845.4
$1,048.4
$1,260.1
NM
NM
NM
NM
NM
NM
42.3%
62.2%
64.4%
67.9%
69.0%
70.6%
Interest Income
$0.0
$0.0
$0.0
$0.0
$0.0
$0.0
$0.0
$0.0
$0.0
$0.0
$0.0
$0.0
Interest Expense
(0.0)
0.0
0.0
0.0
0.0
0.0
0.0
0.0
0.0
0.0
0.0
0.0
Other Income
(0.0)
(2.0)
0.0
0.0
0.0
0.0
0.0
0.0
0.0
0.0
0.0
0.0
($0.0)
($2.0)
$0.0
$0.0
$0.0
$0.0
$0.0
$0.0
$0.0
$0.0
$0.0
$0.0
Pretax Income
($9.1)
($29.5)
($55.0)
($50.0)
($45.0)
($20.0)
$93.0
$345.1
$605.4
$845.4
$1,048.4
$1,260.1
NM
NM
NM
NM
NM
NM
42.3%
62.2%
64.4%
67.9%
69.0%
70.6%
$32.6
$120.8
$211.9
$295.9
$366.9
$441.0
35.0%
35.0%
35.0%
35.0%
35.0%
35.0%
Operating Expenses
% of Revs
Operating Income
% Operating Margin
- C urrently being tested in other retina disorders

+96% - COM patent to 2029; formulation to 2030
+18%
$0.0
R&D
$ 50. 0
+24%
C ost of Goods
SG&A
$ 0. 0
+47%
$ 0. 0
Gross Margin
$ 0. 0
+113%
$ 0. 0
% Grow t h
$ 0. 0
+78% - Topical anti-angiogenic eye drop for wet AMD
- Final Phase II data by end of March; Potential Q2:2015 Phase III start
+ 98% - Rapid upt ake ex pect ed; assum es P DGF com pet it ion
- 90%+ margins; easy to manufacture

+64% - Assumes an initial sales force of 50 reps
- C ommercial hiring to begin in mid-2016
-6% - Phase III wet AMD program expected to cost $75MM; bell curve-like
+31%
NM
% of Revs
Income Taxes
Income Tax Rate
Net Income - Operations
% Net Margin
($9.1)
($29.5)
($55.0)
($50.0)
($45.0)
($20.0)
$60.5
$224.3
$393.5
$549.5
$681.5
$819.0
NM
NM
NM
NM
NM
NM
27.5%
40.4%
41.9%
44.1%
44.8%
45.9%
($9.1)
($29.5)
($55.0)
($50.0)
($45.0)
($20.0)
$60.5
$224.3
$393.5
$549.5
$681.5
$819.0
$0.0
$0.0
$0.0
$0.0
$0.0
$0.0
$0.0
$0.0
$0.0
$0.0
$0.0
$0.0
($ 0. 41)
($ 1. 00)
($ 1. 85)
($ 1. 60)
($ 1. 40)
($ 0. 60)
$ 1. 80
$ 6. 40
$ 10. 95
$ 14. 85
$ 17. 95
$ 21. 00
NM
NM
NM
NM
NM
NM
NM
+256%
NM
NM - Assumes no NOLs
- Standard U.S. corporate tax rate
NM
Extraordinary Items
Adjusted Net Income
Interest Add-Back
E P S (N on- GAAP ) - B ef ore E x . It em s
% Growth
EPS - Extraordinary Items
EPS - Adjusted
Shares Outstanding (MM)
36%
21%
$0.00
$0.00
$0.00
$0.00
$0.00
$0.00
$0.00
$0.00
$0.00
$0.00
$0.00
$0.00
($1.00)
($1.85)
($1.60)
($1.40)
($0.60)
$1.80
$6.40
$10.95
$14.85
$17.95
$21.00
22.1
29.0
30.0
31.0
32.0
33.0
34.0
35.0
36.0
37.0
38.0
39.0
www.cowen.com
N M - True prof it abilit y reached in 2020
17%
($0.41)
42
+71%
NM
NM
+3% - Aassuming some onward dilution from options
Figure 31 Ohr Pharma Annual P&L
Cowen and Company
Ohr Pharmaceutical
Equity Research
March 2, 2015
Assumptions:
Output:
Increase in WC
Discount Rate
5.0% Equity Value
$829.1
19.5% E s t. Sha r e Pr i ce
Fully Diluted Shares
$2 5 .0
33.5 Debt
$0.0
Cash
$35.0
Enterprise Value
Notes:
Netted $26-27MM from Feb 11 offering
Burned ~$2.8MM Q1; expected to be higher in Q2
$794.1
OHR PHA RMA DCF
Total Revenues
% Change
Cost of Goods
Gross Profit
Gross Margin - Total
SG&A
% of Revs
R&D
% of Revs
Operating Expenses
2014
2015E
2016E
2017E
2018E
2019E
$0.0
$0.0
$0.0
$0.0
$0.0
$50.0
NM
-50%
+0%
$125.0
$142.8
$159.3
$175.0
$149.8
$87.5
$38.5
$14.0
$7.0
$7.0
$7.0
$0.0
$0.0
$0.0
$0.0
$0.0
$45.0
$198.0
$505.1
$855.4
$1,145.4
$1,398.4
$1,660.1
$1,897.2
$2,115.8
$2,325.0
$1,990.2
$1,162.5
$511.5
$186.0
$93.0
$93.0
$93.0
93.0%
93.0%
93.0%
93.0%
$50.0
$25.0
$20.0
$20.0
NM
$4.0
NM
$9.1
NM
$20.0
NM
$27.5
NM
$40.0
NM
$55.0
NM
$30.0
NM
$50.0
NM
$20.0
NM
$45.0
90.0%
$50.0
$90.0
100.0%
40.9%
27.0%
25.5%
23.3%
22.4%
21.8%
21.1%
20.9%
20.0%
21.0%
20.0%
22.7%
25.0%
25.0%
20.0%
20.0%
$15.0
$15.0
$10.0
$10.0
$10.0
$10.0
$10.0
$10.0
$10.0
$10.0
$10.0
$10.0
$7.5
$5.0
$5.0
$5.0
$5.0
1.4%
2.5%
5.0%
5.0%
30.0%
$65.0
6.8%
$105.0
NM
NM
NM
NM
NM
130.0%
47.7%
($9.1)
($27.5)
($55.0)
($50.0)
($45.0)
($20.0)
$93.0
NM
NM
NM
NM
NM
NM
(0.0)
(2.0)
0.0
0.0
0.0
0.0
($9.1)
($29.5)
($55.0)
($50.0)
($45.0)
($20.0)
NM
NM
NM
NM
NM
NM
Adjusted EBIT
% of Revs
42.3%
1.8%
$160.0
28.8%
$345.1
62.2%
$240.0
1.1%
$250.0
26.6%
$605.4
64.4%
$290.0
0.8%
$300.0
24.1%
$845.4
67.9%
$340.0
0.7%
$350.0
23.0%
$1,048.4
69.0%
$390.0
0.6%
$400.0
22.4%
$1,260.1
70.6%
93.0%
$430.0
0.5%
$440.0
21.6%
$1,457.2
71.4%
93.0%
$475.0
0.4%
$485.0
21.3%
$1,630.8
71.7%
93.0%
$500.0
0.4%
$510.0
20.4%
$1,815.0
72.6%
93.0%
$450.0
0.5%
$460.0
21.5%
$1,530.2
71.5%
93.0%
$250.0
0.8%
$260.0
20.8%
$902.5
72.2%
93.0%
$125.0
$132.5
24.1%
$379.0
68.9%
5.0%
$55.0
$30.0
$25.0
$25.0
27.5%
30.0%
25.0%
25.0%
$63.0
$68.0
$68.0
63.0%
68.0%
68.0%
$131.0
65.5%
0.0
0.0
0.0
0.0
0.0
0.0
0.0
0.0
0.0
0.0
0.0
0.0
0.0
0.0
0.0
0.0
$93.0
$345.1
$605.4
$845.4
$1,048.4
$1,260.1
$1,457.2
$1,630.8
$1,815.0
$1,530.2
$902.5
$379.0
$131.0
$63.0
$68.0
$68.0
65.5%
63.0%
68.0%
68.0%
$45.9
$22.1
$23.8
$23.8
35.0%
35.0%
35.0%
35.0%
$85.2
$41.0
$44.2
$44.2
($3.0)
($3.0)
($3.0)
($3.0)
42.3%
Taxes
$32.6
Income Tax Rate
35.0%
NOPAT
$150.0
93.0%
+0%
90.0%
% of Revenues
Other Income
2035E
$100.0
$121.6
Operating Income
% Operating Margin
2034E
$100.0
$99.6
92.0%
-64%
2033E
$100.0
+32%
92.0%
-56%
2032E
$200.0
$84.6
91.0%
-42%
2031E
$550.0
+69%
91.0%
-14%
2030E
$1,250.0
$50.0
NM
+10%
2029E
$2,140.0
+152%
$25.0
+12%
2028E
$2,500.0
$22.0
NM
+14%
2027E
$2,275.0
+340%
$20.0
+17%
2026E
$2,040.0
$5.0
NM
+22%
2025E
$1,785.0
$0.0
$15.0
NM
2024E
$1,520.0
$0.0
NM
NM
2023E
$1,245.0
$0.0
$7.5
NM
2022E
$940.0
$0.0
NM
NM
2021E
$555.0
$0.0
$5.1
NM
2020E
$220.0
62.2%
$120.8
35.0%
$224.3
64.4%
$211.9
35.0%
$393.5
67.9%
$295.9
35.0%
$549.5
69.0%
$366.9
35.0%
$681.5
70.6%
$441.0
35.0%
$819.0
71.4%
$510.0
35.0%
$947.2
71.7%
$570.8
35.0%
$1,060.0
72.6%
$635.3
35.0%
$1,179.8
71.5%
$535.6
35.0%
$994.6
72.2%
$315.9
35.0%
$586.6
68.9%
$132.7
35.0%
($9.1)
($29.5)
($55.0)
($50.0)
($45.0)
($20.0)
$60.5
$246.4
Capex
($0.1)
($0.3)
($0.5)
($1.0)
($2.0)
($3.0)
($3.0)
Depreciation & Amortization
($0.5)
($1.0)
($1.0)
($1.0)
($1.0)
($1.0)
($1.0)
($1.0)
($1.0)
($1.0)
($1.0)
($1.0)
($1.0)
($1.0)
($1.0)
($1.0)
($1.0)
($1.0)
($1.0)
($1.0)
($1.0)
($1.0)
$3.4
$5.0
$2.5
$0.0
($5.0)
($10.0)
($10.5)
($11.0)
($11.6)
($12.2)
($12.8)
($13.4)
($14.1)
($14.8)
($15.5)
($16.3)
($17.1)
($18.0)
($18.9)
($19.8)
($20.8)
($21.8)
($6.3)
($25.8)
($54.0)
($52.0)
($53.0)
($34.0)
$46.0
$209.3
$377.9
$533.4
$664.7
$801.6
$929.1
$974.3
$565.5
$224.4
$62.3
$17.2
$19.4
$18.4
Adjustments:
Change In Working Capital

Free Cash Flow
Terminal
($3.0)
($3.0)
($3.0)
($3.0)
($3.0)
($3.0)
($3.0)
$1,041.2
($3.0)
$1,160.2
($3.0)
($3.0)
($3.0)
$94.21
www.cowen.com
43
Figure 32 Ohr Pharma DCF Suggests $25 Per Share
Cowen and Company
Ohr Pharmaceutical
Equity Research
March 2, 2015
Appendix: Additional Background On Marketed Anti-VEGF Agents
About 10-15% of people who start out with the dry form progress to the wet form of
AMD. As dry AMD worsens, new, fragile blood vessels grow from the outer part of the
eye towards the center of the retina, known as the macula. The formation of new
blood vessels (choroidal neovascularization or CNV) is characteristic of wet AMD and
these vessels often leak blood and fluid, causing rapid damage to the macula and
quickly leading to a loss of central vision. Wet AMD typically presents with acuteonset visual deterioration super-imposed on a background of more gradual visual
deterioration characteristic of dry AMD.
AMD Progression
Source: Avalanche Biotechnologies
The Blood-Eye Barrier Can Be Bypassed With Direct Injection

The eye, like the brain, is protected from the systemic blood circulation; therefore, it is
very difficult to deliver drugs into the posterior segment of the eye, particularly the
retina, with sufficient concentration and reduced side effects. This is a primary reason
that diseases of the posterior segment of the eye are treated by intravitreal injections
44
www.cowen.com
The Conversion From Dry To Wet AMD Can Result In Abrupt Deterioration
Cowen and Company
Ohr Pharmaceutical
Equity Research
March 2, 2015
Regenerons Eylea Has Had A Great Launch In Wet AMD, And Is Now The
Market-Leading Branded Anti-VEGF Therapy
Eylea (aflibercept) is a potent VEGF/PlGF inhibitor that is FDA approved in Macular
Degeneration (AMD) and Diabetic Macular Edema (DME) where it is having a
successful launch, competing with Roches/Novartiss Lucentis and Avastin by virtue
of a less frequent injection schedule. Eylea is now the market leader in the branded
anti-VEGF market and appears to be on track to be a $5B+ product worldwide. Eylea
was initially approved for wet AMD by the FDA in November 2011, in the EU in
November 2012, in Japan in September 2012, and in other countries including Brazil,
Colombia, and Australia in 2012. Since then, its label has been expanded to include
DME, RVO (CRVO/BRVO), and CNV indications, with approvals in other indications
including diabetic retinopathy in patients with DME expected globally throughout
2015 (several of these opportunities are discussed later in the chapter). With
Roches/Novartiss standard-of-care Lucentis having achieved 2014 global sales
greater than $4B, the market opportunity for branded wet AMD treatments is sizable
(we estimate likely in the $7B+ area). Regenerons foray into this market was based
on the hope that a more convenient dosing profile (less frequent injections than
Lucentis) would provide Eylea with a competitive commercial advantage. That hope
was born out as Eyleas initial uptake was rapid, well ahead of expectations.
Regeneron reported Q4:11 U.S. Eylea net sales of $25MM, several multiples ahead of
Street expectations, followed by Q1:12 sales of $124MM, again well ahead of then
consensus of about $60MM, Q2:12 sales of $194MM, besting consensus of $143MM,
then $244MM in Q3:12, yet again beating consensus of $225MM. Though Eyleas
launch is maturing somewhat in its initial indication, growth has continued to be quite
robust. Eylea recorded U.S. sales of $838MM in 2012, $1.4B in 2013 (+68% Y/Y), and
$1.7B in 2014 (+23% Y/Y). In fact, in February 2015, Regeneron said that Eylea is now
the market-leading branded anti-VEGF therapy for retinal diseases in the U.S., with
53% dollar share. The company estimates that branded anti-VEGF therapies hold
~56% share of the overall wet AMD market.
In February 2015, Regeneron issued 2015 Eylea guidance of 25-30% growth, implying
revenue of $2.1B-2.3B. This was just below the period consensus estimate of $2.315B
(+33%). On its Q4 call, Regeneron said that it expects "very gradual" uptake in DME,
but did note a few elements that could further augment Eylea growth in 2015: (1)
Protocol T data could favorably impact uptake in DME, (2) an approval in diabetic
retinopathy in patients with DME will provide access to new patient base (PDUFA in
March), and (3) continued growth in macular edema following RVO.
www.cowen.com
45
(directly into the eye), intravenous, or latero-bulbar injections, exposing the patient to
pain and potential side effects. Thus, the mere location of AMD and other back-ofthe-eye diseases makes it difficult for pharmaceutical/device companies to target with
therapies.
Cowen and Company
Ohr Pharmaceutical
Equity Research
March 2, 2015
$600
$518
$500
$445
$415
$402
$400
$363
$314
$300
$359
$330
$276
$244
$194
$200
$124
$100
$25
$Q4:11A
Q1:12A
Q2:12A
Q3:12A
Q4:12A
Q1:13A
Q2:13A
Q3:13A
Q4:13A
Q1:14A
Q2:14A
Q3:14A
Q4:14A
Eylea Partnered With Bayer Ex-U.S.

In 2006, Regeneron entered a partnership with Bayer for ex-U.S. development and
commercialization of Eylea. In June 2011, Bayer submitted regulatory applications in
the EU, Japan, and other countries. Eylea won EMA approval for wet AMD in
November 2012. Bayer launched Eylea ex-U.S. in Q4:12, when Eyleas ex-U.S. sales as
reported by Bayer were $19MM. In 2014, ex-US sales grew to $1.04B (+121% Y/Y).
Eylea currently has only 34% share of ex-U.S. "branded" anti-VEGF market. Regeneron
expects continued growth in ex-U.S. sales, and that its share will ultimately approach
that of the U.S. market.
Bayer markets Eylea ex-U.S., and Regeneron and Bayer share global development
costs and profits equally. The ex-U.S. market is at least comparable to that in the U.S.;
we model $2.6B in ex-U.S. sales by 2019 vs. $2.8B in the U.S. Regeneron must
reimburse Bayer for 50% of the development cost that it has incurred, though such
payments need never be more than the lesser of (1) 5% of the outstanding repayment
obligation or (2) Regenerons share of collaboration profits. At the end of 2013,
Regenerons repayment obligation under the Bayer deal stood at $272MM. In Q3:14,
Regenerons repayment obligation to Bayer was about $14.4MM, meaning that
$85.4MM of Regenerons $99.8MM share of Eylea profits was booked as net profit to
Regeneron. Going forward, Regeneron expects that its repayment obligation will
remain roughly flat each quarter, while Eylea profit will grow, such that net profit to
Regeneron will grow also.
In May 2012, Regeneron and Bayer announced that Santen, a leading Japanese
ophthalmology company, would co-promote Eylea in Japan. In conjunction with this,
Bayer and Regeneron amended the existing global partnership to give Regeneron a
33.5% - 40.0% royalty for Japan instead of the 50/50 profit split, which Regeneron has
said represents approximately similar economics. Eylea was approved for wet AMD in
Japan in September 2012.
Phase III VIEW Studies Differentiate On Convenience, But Not Efficacy
In February 2011, one-year data from the Phase III VIEW 1 and 2 studies (comparing
three different regimens of Eylea to monthly Lucentis) were presented at the Bascom
46
www.cowen.com
Eylea U.S. Quarterly Sales Performance ($MM)
Cowen and Company
Ohr Pharmaceutical
Equity Research
March 2, 2015
Phase III VIEW 1 and 2 Study Results
1 Year Endpoint
VEGFT 0.5mg Q4W
VIEW-1 (U.S.)
(n=1,217)
VEGFT 2mg Q4W
VEGFT 2mg Q8W
Lucentis 0.5mg Q4W
n=
n=
n=
n=
Mean # doses administered over 1-year
~12
~12
~8
~12
% losing <15 letters
95.9%
95.1%
95.1%
94.4%
Mean change in VA (letters)
6.9
10.9
7.9
8.1
1 Year Endpoint
VEGFT 0.5mg Q4W
VIEW-2 (ex-U.S.)
(n=1,240)
VEGFT 2mg Q4W
VEGFT 2mg Q8W
Lucentis 0.5mg Q4W
n=
n=
n=
n=
Mean # doses administered over 1-year
~12
~12
~8
~12
% losing <15 letters
96.3%
95.6%
95.6%
94.4%
Mean change in VA (letters)
9.7
7.6
8.9
9.4
Average change in VA (VIEW 1& 2)*
8.3
9.3
8.4
8.8
Source: Cowen and Company, Regeneron
VIEW 1 and 2 Mean Change in Visual Acuity
www.cowen.com
47
Palmer Angiogenesis Meeting in Miami. In aggregate, the studies suggested

differentiation vs. Lucentis by virtue of convenience (less frequent injections) but not
efficacy.
Cowen and Company
Ohr Pharmaceutical
Equity Research
March 2, 2015
1. Is Eylea More Efficacious Than Lucentis? Although the VIEW-1 study demonstrated
a statistically superior VA change for monthly VEGFT (2mg) vs. Lucentis (10.9 vs. 8.1
letters), VIEW-2 trended lower in this regard (7.6 vs. 9.4 letters; no statistically
significant difference). Aggregate results appear modestly better for Eylea, though
the margin of difference (~0.5 letters) appears too small to be deemed clinically
meaningful. Therefore when dosed monthly, the efficacy of these two agents
appears to be relatively similar.
2. Can Eylea Produce Similar VA Benefits To Lucentis At A Lower Injection Frequency?
Aggregate results suggest similar results for Lucentis (dosed 12x in year 1) and
Eylea (dosed 8x in year 1), with 8.8 vs. 8.4 letters VA change, respectively. When the
two Eylea arms are compared (monthly vs. Q8W), monthly dosing appears to
provide a modest numerical improvement in VA (~1 letter). One key clinically
relevant secondary endpoint was the rate of so-called 3-line gainers. Consistent
with the VA data, bi-monthly Eylea looked comparable to Lucentis on this endpoint
at week 52 (31% vs. 31% in VIEW-1; 31% vs. 34% in VIEW-2). Nonetheless, since
neither Lucentis nor Eylea were dosed as needed (PRN) during the first year of
the study, and the number of injections patients received was mandated by the
protocol, Eyleas true advantage in dosing frequency was not definitively answered.
3. How Does Eyleas Safety Compare To Lucentis? Eylea looked very clean relative to
Lucentis on both ocular and non-ocular AEs. Ocular SAEs occurred at a rate of
1.6% vs. 3.0% (VIEW-1) and 2.3% vs. 3.1% (VIEW-2) for all Eylea arms vs. Lucentis.
Rates of death (1.3% vs. 1.6%, and 0.8% vs. 0.7%) and thromboembolic (APTC)
events (1.6% vs. 1.6%, and 1.9% vs. 1.7%) were also well balanced, suggesting Eylea
is not associated with any increased risk of systemic VEGF-related side effects.
Perhaps the one fly in the ointment for Eylea related to OCT readings for bi-monthly
Eylea. In the studies in which OCTs were conducted monthly (VIEW-2 and the Phase II
DME study), bi-monthly Eylea was associated with a saw-tooth pattern on OCT, with
retinal thickness increasing at the end of Eylea off-months. The amplitude of these
changes (17 microns maximum) was relatively small, the pattern declined over the
course of treatment, and it was not associated with any changes in visual acuity.
However the issue was a source of some debate as the pattern was not seen in
monthly Eylea or Lucentis arms. With VA changes generally thought to lag OCT
findings, one physician on the VIEW review panel also suggested this pattern could
have significant negative implications for patients who miss one (bi-monthly)
physician visit.
48
www.cowen.com
The studies primary endpoint (maintenance of vision at oneyear) was non-inferior in

all arms, as the majority of patients lost <15 letters of vision at one year (95-96% Eylea
vs. 94% for Lucentis). Meanwhile secondary endpoints (including change in visual
acuity, and % of patients with >15 letters visual gain at 52 weeks) were also similar
between all study arms. Key questions about Eyleas profile that these data have
helped answer, include:
Cowen and Company
Ohr Pharmaceutical
Equity Research
March 2, 2015
Eylea And Lucentis Looked Undifferentiated In Year 2 Of VIEW Trials

In December 2011, Regeneron announced the top-line integrated analysis of the
second-year results of the pivotal VIEW-1 and -2 trials of Eylea. In the first year of the
VIEW trials, Eylea was dosed at 2mg every 8 weeks (the approved dose), as well as
two other regimens, and Lucentis was dosed at 0.5mg every 4 weeks (its labeled
dose). In the second year of VIEW, all 4 arms switched to PRN treatment with their
respective drug and dose, including monthly monitoring visits. The Year 2 data reveal
that during the second year, visual acuity gains were sustained in all groups (7.6
letters gained from baseline to week 96 for Eylea, versus 7.9 letters for Lucentis).
However, Eylea showed a very modest numerical advantage in dosing frequency vs.
Lucentis during the second year (4.2 doses vs. 4.7 doses). Safety was similar between
groups.
Eyleas Year Two VIEW-1 and -2 Efficacy Data
Dose Group
Lucentis Q4W -> PRN
Eylea 2.0mg Q4W -> PRN
Eylea 0.5mg Q4wk -> PRN
Eylea 2.0mg Q8W ->PRN
% Maintaining VA
92%
92%
91%
92%
Mean change in BCVA

7.9
7.6
6.6
7.6
Avg injections over 2 years

16.5
16.0
16.2
11.2
Avg injections, Year 2

4.7
4.1
4.6
4.2
Source: Company data, Cowen and Company
Eylea Won FDA Approval In Wet AMD In 2011

In June 2011, the FDAs Dermatologic and Ophthalmic Drugs Advisory Committee
voted unanimously in favor of approving Eylea for wet AMD at a dose of 2mg Q8W,
following three initial doses given monthly. The panel was benign with no real points
of contention. After a minor hiccup in which the PDUFA date was pushed back due to
clarifying questions from the FDA regarding the CMC section, Eylea was FDA
approved in November 2011. In line with the panels conclusions, Eyleas
recommended dose is 2mg Q8W, following three initial doses given monthly.
www.cowen.com
49
View 1 & 2 Mean Change in Retinal Thickness
Cowen and Company
Ohr Pharmaceutical
Equity Research
March 2, 2015
How big a discount Eyleas price actually is in practice depends on dosing frequency.
Eyleas label implies that patients will receive on average 7.5 doses during their first
year of treatment, and therefore they will go to their physicians office an average of
7.5 times. Assuming each dose of Eylea is $1,850, and each visit costs $250 (in
addition to the cost of Eylea), which translates into an annual cost of $15,750. Our
consultants suggest that on average they evaluate Lucentis patients every 5-6 weeks
or 8.5-10 times per year. Moreover they suggest that Lucentis patients get an average
of 7-9 doses per year. Taking the midpoint of these ranges, on average Lucentis costs
$17,912 per year, suggesting that Eylea could actually cost about 12% less than
Lucentis. This is less dramatic than the 45% discount suggested by Regeneron.
Physicians Expect Eylea To Maintain Majority Share Of Branded Market
We conducted a survey of ophthalmologists in conjunction with our October 2014
Annual Therapeutics Conference to assess the future use of Eylea. Surveyed
physicians (n=39) believe that the portion of branded wet AMD patients treated with
Eylea is currently somewhere in between 50-75%, but close to 75%, while the
remaining patients are being treated with Lucentis. In three years time, surveyed
physicians expect Eylea to peak at around 75% branded share with Lucentis
remaining at around 25% most likely due to switching between the therapies as some
patients become refractory. Eylea continues to have a modestly longer dosing interval
than Lucentis in clinical practice. Lucentis treatment is observed to be effective up to
9 weeks, while Eylea has a +1-2 week benefit. While established patients represent a
mix of Avastin, Lucentis, and Eylea, our consultants note that most new wet AMD
patients are being started on Eylea.
50
www.cowen.com
Perhaps the biggest controversy heading into Eyleas PDUFA date was Regenerons
pricing strategy, and whether the company would choose to price at a premium to, on
par with, or at a discount to, Roches Lucentis. Regeneron chose to price Eylea at a
small discount. Eylea is priced at $1,850/dose, while Lucentiss WAC is $1,950/dose,
implying a 5% discount per dose. Interestingly, these prices have remained since
Eyleas launch. Regeneron management has suggested that the annual cost of Eylea
will be approximately $16K/patient, compared to an annual cost of Lucentis of
$29K/patient. Regenerons calculations assume that Eylea is dosed Q8W following the
three loading doses, while Lucentis is dosed Q4W, in line with its label. Moreover,
Regenerons calculations also assume that physicians monitor Lucentis patients
monthly, while Eylea patients are monitored bimonthly after the loading doses.
Cowen and Company
Ohr Pharmaceutical
Equity Research
March 2, 2015
Estimated Eylea vs. Lucentis Use In Three Years, March 2014
Source: Cowen and Company October 2014 Therapeutics Conference; Vistacom
Docs View Eylea As Better Than Lucentis On Dose Interval

We conducted a survey in October 2013 to assess physicians perception of Eyleas
clinical profile in wet AMD and their usage patterns. Of the physicians surveyed, the
majority (58%) continue to believe that Eylea has a modestly longer dosing interval
than Lucentis.
Perception Of Eyleas Dosing Interval
Source: Cowen and Company October 2013 Therapeutics Conference
www.cowen.com
51
Cowen and Company
Ohr Pharmaceutical
Equity Research
March 2, 2015
And Eylea Seemingly Better On Efficacy Than Lucentis, Too
Eylea is seemingly beginning to be perceived as having differential efficacy vs.

Lucentis. To explore this phenomenon, we asked physicians to estimate the
percentage of patients who responded to Eylea after having an inadequate response
to a prior anti-VEGF. Over three surveys we conducted in 2012, the doctors
consistently indicated that about 40% of prior anti-VEGF failures responded when
treated with Eylea.
Reasons For Starting Eylea
Ad eq uat e Resp onse I n Nonr esp ond er s
Per cent Wit h Adequat e Response
100%
75%
50%
25%
0%
March
May
August
Source: Cowen and Company Eylea Surveys
These survey results are more or less in line with our expert consultants commentary
regarding their clinical experience with Eylea. The physicians perception of Eylea has
improved with clinical experience. Ahead of our October 2011 Therapeutics
Conference, held approximately a month before Eyleas approval, the majority of our
surveyed physicians (60%) said they believed Eyleas clinical profile was not
differentiated from that of Lucentis. At the October 2012 conference, physicians firsthand experience had evidently improved their view of the drug, with the majority
(57%) of surveyed doctors saying Eylea is a modest, incremental advance over
Lucentis. Interestingly, our consultants believe Eylea is differentiated from Lucentis not
so much by dosing frequency, where they agree it confers an approximate 1-week
advantage vs. Lucentis, but rather by efficacy. The consultants indicate that in about
5-15% of their patients, Eylea has shown better efficacy than Lucentis. Of course, they
admit that there is some selection bias inherent in this observation, in that only
patients who are not doing well on Lucentis would generally be switched. The
52
www.cowen.com
To gain insight into the reasons that the patients already on therapy were switched to
Eylea, we asked the physicians to estimate the percent of patients who were switched
because (1) the patient had had an inadequate response to the prior agent, or (2) the
physician hoped to reduce the frequency of dosing. The physicians indicated that a
mean 20% of new Eylea patients were switched in order to reduce dosing frequency,
while 33% were switched due to inadequate efficacy of a prior agent. (The remaining
47% of Eylea patients were switched for another reason or were treatment nave).
Cowen and Company
Ohr Pharmaceutical
Equity Research
March 2, 2015
Perceptions Of Eyleas Efficacy
Source: Cowen and Company Health Care Conference, March 2013
Indeed, we have heard numerous anecdotal reports from our retinal specialist
consultants that a subset of their patients who have had an inadequate response to
Lucentis or Avastin may be well-controlled if switched to Eylea. However, it has been
unclear to us to what degree this represents a genuine superiority in efficacy for Eylea
in these Lucentis/Avastin-refractory patients, as opposed to a simple selection bias
resulting from patients being switched from the older agents to the newer but not
(yet) vice versa. We had a conversation with a retinal specialist at the November 2012
AAO meeting that raises our confidence that Eylea may have a genuine efficacy
benefit in some patients. This specialist was an investigator on Eyleas Phase II trial,
and so he has performed the experiment of switching patients doing well on Eylea to
Lucentis/Avastin (when the Phase II trial ended) and saw many of them do much
worse on Lucentis/Avastin. While ideally one would like to know whether there have
been examples of patients doing poorly on Eylea who then improved upon switching
to Lucentis, it appears to be still too early for much of this type of experience to have
accumulated. Nevertheless, our consultants experience moderately increases our
confidence that there may be a legitimate efficacy benefit for Eylea in some patients.
In our view, this bolsters Eyleas prospects for capturing substantial share from
Lucentis in the near term, as well as some from Avastin. In fact, our consultant said his
28,000-injection per year practice has now completely stopped ordering Lucentis and
that he would put all his patients on Eylea if only their insurance covered it.
Regeneron Settled With Roche On Patents For U.S. Ophthalmic Use
In January 2012, Regeneron announced a partial settlement with Genentech in their
intellectual property dispute over patents relating to VEGF receptor proteins and U.S.
ophthalmic sales of Eylea. Under the terms of the agreement, litigation relating to U.S.
ophthalmic sales of Eylea will end and Regeneron will make payments to Genentech
based on U.S. Eylea sales through May 7, 2016. Regeneron paid a $60MM sales
milestone to Roche when cumulative U.S. Eylea sales reached $400MM, and will also
www.cowen.com
53
consultants views were consistent with physicians we surveyed ahead of our Health
Care conference in March 2013. 70% of surveyed physicians said Eylea had a
modestly better dose interval than Lucentis and 80% said Eylea had modestly better
efficacy. Most consultants say they would put all their new wet AMD patients on Eylea
if they could, though some patients still receive Avastin or Lucentis due to insurance
considerations.
Cowen and Company
Ohr Pharmaceutical
Equity Research
March 2, 2015
Competition Pressuring Lucentis Despite DME Approval

Roche/Novartiss Lucentis is a pan anti-VEGF antibody fragment, and while the
VEGF165 isoform is thought to account for most VEGF activity in AMD, blocking all
VEGF activity in the retina appears to translate into superior clinical activity. Lucentis
data have been stellar. In June 2003, Novartis licensed ex-North American rights for
Lucentis from Roche/Genentech. Final FDA approval was received in June 2006 with
the European approval in January 2007. Roche/Genentech estimates that the average
wet AMD patient will require five to seven injections per year.
Avastin substitution has been a barrier to Lucentiss commercial success in the U.S.
Compounding pharmacies reconstitute the significantly cheaper Avastin ($50 versus
$2000 a dose) for intraocular injection despite its lack of FDA approval for AMD.
However, the current main competition is from Eylea, which we estimate will reach
approximately $2.2B and $2.8B in 2015 and 2019 U.S. sales, respectively. In the U.S.,
we estimate Lucentis sales of $1.7B in 2015, decreasing to $1.5B in 2019 from
increased competition and as U.S. patents begin to expire. We forecast ex-U.S.
Lucentis sales of $2.5B in 2015, declining to $1.8B in 2019 as the ex-US patents expire
and competition continues to increase. We discuss Lucentiss opportunity in DME and
RVO later in this chapter.
US Claims Database Data Suggest The Risk Of Endophthalmitis Is 65% Higher
Following Treatment With Eylea When Compared To Lucentis
At the EURETINA congress in London September, 2014, Novartis presented a
retrospective analysis of data from a US claims database. Novartis claims that the
database offers the first direct comparison of the risk of developing endophthalmitis
for wet AMD patients treated with Lucentis or Eylea. A total of 253,647 Lucentis
injections in 54,551 patients and 179,147 Eylea injections in 39,389 patients were
eligible for analysis. Novartis reported that the results suggest that the risk of
endophthalmitis is 65% higher following treatment with Eylea than treatment with
Lucentis. While these results appear interesting, we would interpret them with caution
as this analysis was retrospective and not a head-to-head comparison study. Also, we
have yet to discern whether or not this data will affect Eylea or Lucentis use in clinical
practice.
MARINA Data Off The Charts
At the July 2005 ASRS meeting in Montreal, one-year results from the ongoing twoyear MARINA trial of Lucentis were presented. The study evaluated 716 patients with
minimally classic and occult wet AMD lesions. Impressively, the rate of 3-line vision
gain in the MARINA study was 33.8%/24.8%/4.6% for the Lucentis 0.5 mg, Lucentis
0.3 mg and sham arms, respectively. Average vision change at one year was
+7.2/+6.5/(-10.5) letters for the same trial arms, respectively. Subset analysis showed
a sizable clinical benefit from Lucentis was achieved regardless of lesion size or
subtype. Side effects were in an acceptable range. Based on these compelling
findings, the retina community has embraced Lucentis with open arms.
54
www.cowen.com
pay a royalty of 4.75% on cumulative U.S. sales of Eylea between $400MM and $3B,
and a 5.5% royalty on cumulative sales over $3B. In May 2013, the settlement was
updated to include a royalty obligation on ex-U.S. revenues under the same terms,
though this portion of the obligation will be shared by Bayer. All of Regeneron's royalty
obligations end on May 7, 2016.
Cowen and Company
Ohr Pharmaceutical
Equity Research
March 2, 2015
Sham
Study
MARINA
No. patients
Lesion size (DA)
Occult
Minimally Classic
Predominantly Classic
238
4.41
63.4%
36.6%
0%
Lucentis
0.3 mg
Lucentis
0.5 mg
Macugen
0.3 mg
MARINA MARINA
VISION
Baseline Demographics
238
240
298
4.26
4.47
4.2
63.5%
61.7%
40%
36.1%
37.9%
34%
0.40%
0.40%
26%
# of injections/total possible
Received PDT
10.8/12
10.50%
11.5/12
0.40%
Treatment
11.3/12
0%
3 line vision loss or better

3 line vision gain or better
Change in visual acuity from baseline (letters)
62.20%
4.6%
(10.5)
94.50%
24.8%
6.5
Results
94.60%
33.8%
7.2
0%
0%
0%
0%
9.7%
0.4%
0.4%
0%
0.4%
0.4%
0.8%
8.4%
0.4%
0.4%
Culture-positive endophthalmitis
Culture-negative endophthalmitis
Total endophthalmitis
Uveitis
Hypertension
Myocardial infarction
Cerebrovascular event
Sham
Adverse Events
0%
0.8%
0.8%
0.4%
8.4%
0.4%
1.3%
VISION
295
3.7
38%
38%
24%
8.5/9
13%-30%
N/A
11%-22%
55%
2%
(15.0)
70%
6%
(7.0)
N/A
N/A
0%
N/A
5%
1%
1%
N/A
N/A
2%
N/A
7%
1%
<1%
Source: Cowen and Company, FDA
ANCHOR Confirms Lucentis Potency

Results of the 423-patient Phase III ANCHOR study were presented in January 2006 at
Macula 2006 in New York by Dr. Peter Kaiser. The ANCHOR study compared
intravitreous Lucentis to photodynamic therapy with Visudyne in patients with
predominantly classic wet AMD lesions, and further established Lucentis as the most
potent therapy ever to be developed for wet AMD. After 12 months of therapy, the 0.3
mg arm of Lucentis beat Visudyne by 18 letters while the 0.5 mg arm of Lucentis beat
Visudyne by 21 letters. Patients receiving 0.3 mg of Lucentis gained an average of 8.5
letters after one year, while patients receiving 0.5 mg of Lucentis gained an average of
11 letters. Among patients receiving 0.3 mg of Lucentis, 94% lost 3 lines or less of
visual acuity while 36% gained 3 or more lines of vision. Among patients receiving 0.5
mg of Lucentis, 96% lost 3 lines or less of visual acuity while 40% gained 3 or more
lines of vision. These groups were both clearly superior to the Visudyne arm, in which
only 64% of patients lost 3 lines or less of visual acuity and only 6% gained 3 or more
lines of vision. Adverse ocular events in this study were consistent with those in other
Lucentis trials. There was a slight increase in myocardial infarction rate in the highdose Lucentis arm (2.1%) compared to the low dose arm (0.7%) and the Visudyne arm
(0.7%), although the absolute number of patients with events was quite low.
Roche/Genentech also indicated that the combined rate of cerebral vascular events
and myocardial infarctions was 1.3% in the control arm, 1.3% in the low-dose Lucentis
arm and 2.9% in the high-dose Lucentis arm. Roche/Genentech received approval for
the high-dose (0.5 mg) option.
Two-Month Data, ANCHOR Study
Outcome
Visudyne (n =
143)
Lucentis 0.5 mg
(n=140)
Difference
Loss of < 15 letters in visual

acuity
64%
96%
33%
Gain of > 15 letters in visual

acuity
6%
40%
35%
Mean change in visual acuity

(letters)
-9.5
11.3
21.1
Source: Cowen and Company, Lucentis package insert
www.cowen.com
55
Comparison Of One-Year Results From Lucentis And Macugen Pivotal Studies
Cowen and Company
Ohr Pharmaceutical
Equity Research
March 2, 2015
Lucentis FOCUS Data Also Impressive
One-Year Data From Lucentis FOCUS Study
No. patients
Lesion size (DA)
Sham
56
2.56
3 line vision loss or better

3 line vision gain or better
Change in visual acuity from baseline (letters)
67.9%
5.4%
(8.2)
90.5%
23.8%
4.9
3.4
1.3
0%
0%
0%
0%
7.1%
3.6%
0.0%
1.0%
3.8%
4.8%
8.6%
12.4%
0.0%
3.8%
Average number of PDT administrations

Culture-positive endophthalmitis
Culture-negative endophthalmitis
Total endophthalmitis
Uveitis
Hypertension
Myocardial infarction
Cerebrovascular event
Lucentis 0.5 mg
106
2.51
p-value
p=0.0003
p=0.0033
While there was an increase in inflammatory adverse ocular events seen with PDT plus
Lucentis, our consultants do not believe this represents a significant concern. The
presenting investigator noted that the risk of ocular inflammation was likely related to
the Lucentis formulation used in the trial (lyophilized powder, switched to pre-mixed
formulation for the Phase II/III studies) and to the timing of Lucentis and PDT
administration (which prompted a change in the study protocol to avoid administering
both drugs simultaneously). Furthermore, visual outcomes even in the group of
patients who had inflammatory ocular complications were better than seen in the
PDT-alone arm. Additionally, there was a trend of increased hypertension and
cerebrovascular events seen with Lucentis plus PDT, although this was offset by a
trend of increased cardiovascular events seen with PDT alone. These findings seem to
have had little impact on Lucentiss adoption.
PIER, PrONTO Provide Clarity On Lucentis Dosing Requirements
In June 2006, results of the Phase IIIb PIER study, which evaluated a less frequent
dosing regimen for Lucentis, were presented at the Retinal Physician Symposium in
the Bahamas. While the MARINA and ANCHOR trials evaluated monthly intravitreous
administration through the course of therapy, the PIER study treated patients with
monthly injections for the first three months after which injections were administered
every three months. After three months of therapy, patients receiving 0.3 mg Lucentis
gained 2.9 letters of visual acuity and patients receiving 0.5 mg Lucentis gained 4.3
letters of visual acuity, while patients receiving sham injections lost 8.7 letters of visual
acuity. However, vision gains were not sustained, and by month 12, patients treated
with Lucentis had returned to baseline while those receiving sham injections
deteriorated further.
56
www.cowen.com
One-year data from the ongoing Phase I/II FOCUS study were also presented at ASRS
in July 2006. The FOCUS trial was a single-masked study that randomized patients
with predominantly classic wet AMD lesions to receive either PDT alone or PDT plus
Lucentis (0.5 mg). The addition of Lucentis to PDT resulted in substantial efficacy
benefit and greatly reduced the need for subsequent PDT administrations. Benefit was
seen regardless of prior PDT therapy. Data from this study are presented in the table
below.
Cowen and Company
Ohr Pharmaceutical
Equity Research
March 2, 2015
Lucentis 0.3 mg (n
= 60)
Outcome
Lucentis 0.5 mg
(n=61)
Sham (n= 63)
Loss of < 15 letters in visual acuity
83%
90%
49%
Gain of > 15 letters in visual acuity
12%
13%
0%
Mean change in visual acuity

(letters)
-1.6
-0.2
-16.3
Source: Company data
Roche/Genentech reported that in the first year of Lucentis therapy, the average
patient had 3-4 Lucentis injections in the first four months of treatment, followed by
an average of 3 injections in the remaining 8 months. The second year is 4-5 doses
per year.
CATT Data Played Out According To Physicians Expectations And Did Not Change
Clinical Practice
CATT (Comparison of Wet AMD Treatments Trial) was an NIH-sponsored trial that
tested four different treatment strategies as outlined in the table below. The trials
primary endpoint was mean visual acuity change at 12-months, on which the study
was is 90% powered to demonstrate non-inferiority of monthly Lucentis to monthly
Avastin (non-inferiority margin of 5-letters on ETDRS). 12-month data from CATT were
presented at the ARVO meeting in May 2011, followed by two-year data at ARVO
2012.
Lucentis vs. Avastin CATT Study Design
Arm
Schedule
Endpoints
(note follow-up for all arms every 4 weeks)
Primary Endpoint
A
(n=300)
Lucentis Q4W
(re-randomize at 1 year: Q4W or variable dosing)
B
(n=300)
Avastin Q4W
(re-randomize at 1 year: Q4W or variable dosing)
C
(n=300)
Lucentis PRN
(prn after 1 initial treatment)
D
(n=300)
Avastin PRN
(prn after 1 initial treatment)
Mean change in visual acuity (VA) at 1 year*
Secondary Endpoints
1)
2)
3)
4)
5)
6)
3-line change in VA (% patients gaining or losing 15 letters)

Change in sub/intra-retinal fluid on OCT
Change in lesion size on fluorescein angiography
Incidence of endophthalmitis, retinal detachement, cataract, uveitis
Incidence of AE's
Cost
* non-inferiority limit of 5 letters
Source: Cowen and Company, Adapted from www.clinicaltrials.gov
A Brief History of the Lucentis/Avastin Controversy: Off-label intravitreous Avastin for

AMD initially gained traction in 2005 prior to FDA approval of Lucentis. Despite scant
clinical data, adoption was driven by highly positive anecdotal experiences and a
retina community willing to experiment with new therapies. Genentechs rationale in
www.cowen.com
57
12-Month Data From PIER Study
Cowen and Company
Ohr Pharmaceutical
Equity Research
March 2, 2015
Numerous investigator-initiated studies have now associated Avastin with

improvements in retinal edema and VA, and a safety profile considered by most
physicians to be indistinguishable from Lucentis. Several rabbit studies have also
since demonstrated full penetration of Avastin through the retina. With Avastin having
gained widespread use in AMD, the NEI authorized CATT as a way of providing
physicians with a definitive body of prospective head-to-head data on Avastin.
According to the trial manual, additional rationale for conducting CATT relates to cost
(implications of Lucentis vs. Avastin and monthly vs. PRN dosing), and the treatment
burden on patients/physicians of regular monthly injections. After an initial delay
related to trial funding, enrollment in the study began in early 2008.
Factors Influencing Physicians Choice Between Lucentis/Avastin In AMD
Factors Driving Use of Lucentis
Comment
Availability of controlled Phase III data and

FDA endorsement
Lucentis remains the more proven and thoroughly-tested drug, and has a larger and better-established safety
database. All else equal, most patients would naturally opt for Lucentis vs. Avastin given that Avastin has not
been thoroughly evaluated by the FDA in wet AMD. Academic centers have tended to migrate toward Lucentis
on this basis.
Financial incentives
At a cost of roughly $2K/injection (vs. $50 for Avastin), ASP+6% economics allow high volume private practices
to prosper significantly from dosing Lucentis. This is in addition to other financial perks (e.g. credit card
rewards) physicians might receive.
Potential Safety Risk Of Repackaged Avastin
Compounding pharmacies separate single vials of Avastin (intended for cancer treatment) into multiple singledose syringes. Occasional lapses in sterility have resulted in sporadic cases of infection and vision loss.
Factors Driving Use Of Avastin
Comment
Favorable anecdotal off-label experience
As anecdotal experience has built, physicians increasingly view Avastin and Lucentis as indistinguishable in
terms of efficacy and safety. As a result, other factors (financial risk/patient co-pay) have tended to drive
treatment decisions for many patients.
Financial risk of Lucentis inventory
Because lower-volume practices are not set up to track ultra expensive drugs, even one or two lost vials can tip
Lucentis economics from modestly profitable to negative. Many practices must devote additional resources
(clerical time) to prevent this from happening.
Patient co-pay
Depending on the level of insurance/Medicare coverage, many patients are required to pay Lucentis co-pays
(often 20% of the drug cost), driving the decision to use Avastin. To qualify for Genentechs co-pay
reimbursement, patients income must be less than $75K.
Discontent toward Roche/Genentech
Several policies enacted by Roche/Genentech since Lucentis launch (many since reversed) have led to a
degree of anti-Genentech sentiment within the retina community. Examples include reduced drug payment
timelines and restriction of purchases to drug wholesalers only (i.e. detracting from +6% economics). CMSs
2009 decision to dramatically cut Avastin reimbursement in AMD (reversed in 2010) also fueled discontent.
58
www.cowen.com
developing Lucentis (a Fab fragment of Avastin that has 140x the VEGF-binding
affinity) had been based on preclinical data suggesting that a 149 kD antibody
(Avastin) could not penetrate the retina. Genentech has consistently pointed to several
differences between the two drugs that render Avastin unsuitable for intravitreal use
(e.g., lack of preclinical tox data, longer intravitreal half-life, lower VEGF binding
affinity, potential for full length antibodies to cause ocular inflammation, differing
manufacturing standards for ocular and systemic drugs).
Cowen and Company
Ohr Pharmaceutical
Equity Research
March 2, 2015
One-year data from the CATT study were published in the New England Journal of
Medicine in April 2011.
Key points from the data are as follows:
Monthly treatment: Avastin and Lucentis looked similar in terms of efficacy in the
monthly arms at one year, with 8.0 and 8.5 letters gained at 12 months, respectively.
The mean decrease in central retinal thickness was greater in the Lucentis-monthly
group (196 m) than in the other groups (152 to 168 m, P=0.03).
PRN treatment: Avastin administered as needed was equivalent to Lucentis as
needed, with 5.9 and 6.8 letters gained, respectively. Lucentis PRN was equivalent to
monthly Lucentis, although the comparison between Avastin PRN and monthly
Avastin was inconclusive. At the ARVO presentation, Dr. Daniel Martin (the studys
Principal Investigator) expressed positive surprise at the performance of the PRN
dosing arms, indicating the strong results could likely be attributed to 1) a greater
number of injections given vs. previous studies (7 injections per year for Lucentis
PRN arm), 2) a much lower threshold of disease activity for re-treatment, and 3)
PRN injections that were more evenly distributed over the course of the year relative
to other PRN trials (which typically include three upfront monthly doses).
Safety: Rates of death, MI, and stroke were similar for patients receiving either
Avastin or Lucentis (P>0.20). At the ARVO meeting, updated data on deaths and
arteriothrombotic events through 2 years were presented, with similar rates between
Lucentis and Avastin at this time (4.3% vs. 5.5% deaths, respectively; 4.3% vs. 4.1%
arteriothrombotic events, respectively).The proportion of patients with systemic
SAEs (primarily hospitalizations) was higher with Avastin than with Lucentis (24.1%
vs. 19.0%; risk ratio, 1.29; 95CI: 1.01-1.66). Dr. Daniel Martin, the studys principal
investigator described the higher rate of SAEs for Avastin was viewed as intriguing
and worthy of follow-up, but not concerning at present. This reflects: 1) differences
in SAEs that were in areas not previously associated with Avastin in cancer trials
(e.g. sepsis, pneumonia, surgical procedures), with a broad distribution across
organ groups; 2) a higher rate of SAEs in PRN groups; and 3) higher baseline age,
hypertension, diabetes, smoking MI history, arrhythmia and emphysema in Avastintreated patients.
CATT Has Had Little Effect On Thought Leaders Use Of Lucentis/Avastin In Wet AMD
We checked in with several thought leaders at our Therapeutics conference in
October 2011 to get their thoughts on the impact of the CATT data on clinical practice.
Our panelists believed that the CATT results support the view that Lucentis and
Avastin are essentially identical in efficacy and safety. According to the panelists,
CATTs results provide the first evidence from a controlled trial that PRN Avastin or
Lucentis are as effective as the monthly protocol. Since they have been using both
agents PRN since 2005 anyway, CATT has had no impact on their prescribing
practices. Our surveyed specialists were in agreement, with almost all indicating they
would not change their practices as a result of CATT.
www.cowen.com
59
NEJM CATT Publication Highlights Similarities Between Avastin And Lucentis

Treatment
Cowen and Company
Ohr Pharmaceutical
Equity Research
March 2, 2015
Source: Cowen and Company 2011 Therapeutics Conference, Vistacom
Second-Year CATT Data Also Expected To Have Little Effect On Relative Share of
Avastin or Lucentis...
In April 2012, the journal Ophthalmology published the two-year results of the CATT
trial. We believe there are three key findings in the two-year results: (1) Mean visual
acuity gain was similar for Avastin and Lucentis through year two (Avastin-Lucentis
difference of -1.4 letters, p=0.21); (2) The mean gain was greater for monthly
treatment than for as-needed treatment (difference -2.4 letters, p=0.046), although the
magnitude of the difference at 2.4 letters was less than what our consultants have in
the past said was borderline clinical significance; (3) The proportion of patients who
had SAEs was again higher for Avastin than Lucentis (39.9% vs 31.7%, p=0.009),
although similar to the one year data, the excess events have not been associated
previously with systemic anti-VEGF therapy. Moreover, while the cumulative incidence
of SAEs on Avastin compared to Lucentis increased, the annualized frequency
difference actually decreased in year 2 compared to year 1.
We did not expect the results of CATT Year 2 to have a dramatic effect on practice
patterns and it appears that has been the case. In general, it appears experts can all
find some aspect of the data that supports their particular preferred practice. For
example, two consultants we checked in with regarding the CATT data had
diametrically opposed views of the impact of CATT on Lucentis and Avastin. The
physician who predominantly uses Lucentis today thinks the data strongly support
Lucentis dosed monthly, while the physician who uses Avastin as-needed in the
majority of his practice thinks the same about Avastin PRN. The Lucentis user thinks
the 3.8 mean letter improvement of Lucentis monthly compared to Avastin PRN is a
substantial, meaningful difference in visual acuity. He is worried by the increase in
systemic serious adverse events in Avastin-treated patients compared to Lucentistreated patients. He believes therefore the totality of the data recommend monthly
Lucentis of the regimens tested as it produced the best efficacy with the fewest
serious adverse events. This physician is skeptical that the increase in geographic
atrophy seen with monthly Lucentis is a real finding, as in his experience this is a
much less frequent event than suggested by the data. Meanwhile, the Avastin user
thinks the data support the use of PRN Avastin. While he acknowledges the difference
60
www.cowen.com
CATT Trial Results and Use of Avastin for Wet AMD
Cowen and Company
Ohr Pharmaceutical
Equity Research
March 2, 2015
Responses from surveyed physicians regarding CATT Year 2 are consistent with the
mixed feedback from consultants. Specifically, we asked which of the four treatment
regimens tested in CATT is the best. There was little agreement on the interpretation
of CATT. About a third (32%) of the physicians said none of the tested regimens could
be said to be best, while another 16% were unfamiliar with the CATT data. The
remaining 52% of physicians were comprised of 24% who said Lucentis monthly is the
best regimen, 12% in favor of Avastin monthly, 12% in favor of Avastin PRN, and 4% in
favor of Lucentis PRN.
Roches HARBOR Study Failed To Demonstrate A Benefit To High-Dose Lucentis
Efficacy data from Roche's Phase III HARBOR trial in wet AMD failed to make the case
for conducting further investigations to use a higher 2mg dose of Lucentis. The
HARBOR studies evaluated once-monthly and as-needed dosing regimens of the
higher dose and the approved 0.5 mg dose, which is indicated for once-monthly use.
As the pivotal MARINA and ANCHOR trials both demonstrated a dose-response curve
favoring 0.5mg Lucentis over 0.3mg dosing, HARBOR was designed to test whether a
higher Lucentis dose could provide better clinical benefits (improved VA, or less
frequent dosing).
Avastin Remains Part Of The Treatment Paradigm; Compounding Quality Act Passed
In November 2013, Not Materially Affecting Use
A couple years ago, the deadly outbreak of meningitis associated with steroid
injections from unregulated compounding pharmacies may have led to some
physicians foregoing the use of compounded Avastin. Since then, these compounded
pharmacies have been under increased scrutiny from federal authorities and
lawmakers. In November 2013, a new law was passed (The Drug Quality And Security
Act) that codified FDAs authority over compounding pharmacies, which had been a
source of debate previously because states oversee pharmacies as well. The law
created a class of compounding pharmacies called outsourcing facilities, referring to
those that compound in bulk and ship across state lines, which will need to be
inspected and certified by the FDA. Outsourcing facilities are exempt from obtaining
FDA approval for their products and from labeling them fully, but must comply with
cGMP practices. Regeneron believes that, according to FDA regulations, all bulk
biologics compounding is in fact illegal, including Avastin for off-label ophthalmic use
(meaning Avastin can only be compounded in response to a prescription for a specific
patient). While FDA now has the clear authority to enforce that interpretation and
block compounded Avastin from being bulk produced, it remains to be seen if in fact
this will come to pass. Our physician consultants have not found access to Eylea any
more difficult following the passage of the bill, and have also not noted any changes
in the behavior of the compounding pharmacies thus far.
www.cowen.com
61
in letter improvement, he thinks the magnitude of the difference is modest. However,

he is more concerned by the rates of geographic atrophy observed in the trial, and
notes that the lowest incidence occurs in the Avastin PRN arm. He worries that it is
logical that long-term, chronic suppression of VEGF could cause such a side effect. He
also points out that monthly injections lead to about 1 case of endophthalmitis per 100
patient-years, ten times more frequent than found in the as-needed arm. This
physician is not worried by the rate of systemic adverse events attributed to Avastin,
as most of the excess events have not been previously associated with anti-VEGF
therapy. Moreover, the trial reports adverse events at a much higher frequency than
his experience suggests is reasonable. Therefore he looks on the increase in Avastin
AEs as an anomaly related to trials.
Cowen and Company
Ohr Pharmaceutical
Equity Research
March 2, 2015
Over the past several months, there have been rumblings of payors increasing
pressure on the use of Avastin. Interestingly, when the panelists were queried, they
did admit that it might be possible that they get more step-throughs for Avastin in the
future, but this largely remains to be seen. They do already see this for secondary
insurance though. Additionally, the concerns over the past 2 years of the deadly
outbreak of meningitis associated with steroid injections from unregulated
compounding pharmacies appear to have subsided and certainly do not have any
effect on physicians foregoing the use of compounded Avastin at the moment. Our
panelists noted that these concerns were probably overdone in the first place. Our
panelists believe that the only practices affected were those directly impacted by the
outbreak or connected to the compounders in some way.
62
www.cowen.com
Avastin Remains Part Of The Treatment Paradigm; Payor Pressure Might Increase, But
Its Still Too Early To Tell
Cowen and Company
Ohr Pharmaceutical
Equity Research
March 2, 2015
Valuation Methodology
For our valuation methodology, we arrive at fair value utilizing a discounted cash flow
(DCF) approach to derive our 12-month price target.
Investment Risks
Risks include: (1) growing competitive dynamics in the specialty pharmaceuticals
space; (2) the ability of management to execute on external growth by successfully
acquiring new strategic, accretive products; (3) the ability to grow organically and
keep the product pipeline robust; (4) potential regulatory delays, rejections, or failures
of pipeline products; (5) economic sensitivity of any self-pay products or weakening
consumer demand; (6) domestic or international pricing pressures for marketed
products; and (7) failure to execute on new product launches.
Risks To The Price Target

Ohr is a development-stage company and with that carries substantial risk. The
valuation is almost entirely predicated on OHR-102 and if the Company fails to
successful develop the product, there will be substantial downside to the current
valuation.
www.cowen.com
63
Valuation Methodology And Risks
Cowen and Company
Ohr Pharmaceutical
Equity Research
March 2, 2015
Stocks Mentioned In Important Disclosures

Ticker
Company Name
OHRP
Ohr Pharmaceutical
Analyst Certification
Each author of this research report hereby certifies that (i) the views expressed in the research report accurately reflect his or her personal views about any and all of the subject
securities or issuers, and (ii) no part of his or her compensation was, is, or will be related, directly or indirectly, to the specific recommendations or views expressed in this report.
Important Disclosures
Cowen and Company, LLC and or its affiliates make a market in the stock of Ohr Pharmaceutical securities.
Ohr Pharmaceutical has been client(s) of Cowen and Company, LLC in the past 12 months.
Cowen and Company, LLC and/or its affiliates expect to receive, or intend to seek, compensation for investment banking services in the next 3 months from Ohr Pharmaceutical.
Ohr Pharmaceutical is or was in the past 12 months a client of Cowen and Company, LLC; during the past 12 months, Cowen and Company, LLC provided IB services.
Cowen and Company, LLC and/or its affiliates received in the past 12 months compensation for investment banking services from Ohr Pharmaceutical.
Cowen and Company, LLC and/or its affiliates managed or co-managed a public offering of Ohr Pharmaceutical within the past twelve months.
Cowen and Company, LLC compensates research analysts for activities and services intended to benefit the firm's investor clients. Individual compensation determinations for
research analysts, including the author(s) of this report, are based on a variety of factors, including the overall profitability of the firm and the total revenue derived from all sources,
including revenues from investment banking. Cowen and Company, LLC does not compensate research analysts based on specific investment banking transactions.
Disclaimer
This research is for our clients only. Our research is disseminated primarily electronically and, in some cases, in printed form. Research distributed electronically is available
simultaneously to all Cowen and Company, LLC clients. All published research can be obtained on the Firm's client website, https://cowenlibrary.bluematrix.com/client/library.jsp.
Further information on any of the above securities may be obtained from our offices. This report is published solely for information purposes, and is not to be construed as an offer
to sell or the solicitation of an offer to buy any security in any state where such an offer or solicitation would be illegal. Other than disclosures relating to Cowen and Company, LLC,
the information herein is based on sources we believe to be reliable but is not guaranteed by us and does not purport to be a complete statement or summary of the available data.
Any opinions expressed herein are statements of our judgment on this date and are subject to change without notice.
For important disclosures regarding the companies that are the subject of this research report, please contact Compliance Department, Cowen and Company, LLC, 599 Lexington
Avenue, 20th Floor, New York, NY 10022. In addition, the same important disclosures, with the exception of the valuation methods and risks, are available on the Firm's disclosure
website at https://cowen.bluematrix.com/sellside/Disclosures.action.
Price Targets: Cowen and Company, LLC assigns price targets on all covered companies unless noted otherwise. The price target for an issuer's stock represents the value that
the analyst reasonably expects the stock to reach over a performance period of twelve months. The price targets in this report should be considered in the context of all prior
published Cowen and Company, LLC research reports (including the disclosures in any such report or on the Firm's disclosure website), which may or may not include price
targets, as well as developments relating to the issuer, its industry and the financial markets. For price target valuation methodology and risks associated with the achievement of
any given price target, please see the analyst's research report publishing such targets.
Notice to UK Investors: This publication is produced by Cowen and Company, LLC which is regulated in the United States by FINRA. It is to be communicated only to persons
of a kind described in Articles 19 and 49 of the Financial Services and Markets Act 2000 (Financial Promotion) Order 2005. It must not be further transmitted to any other person
without our consent.
Copyright, User Agreement and other general information related to this report
2015 Cowen and Company, LLC. Member NYSE, FINRA and SIPC. All rights reserved. This research report is prepared for the exclusive use of Cowen clients and may not be
reproduced, displayed, modified, distributed, transmitted or disclosed, in whole or in part, or in any form or manner, to others outside your organization without the express prior
written consent of Cowen. Cowen research reports are distributed simultaneously to all clients eligible to receive such research reports. Any unauthorized use or disclosure is
prohibited. Receipt and/or review of this research constitutes your agreement not to reproduce, display, modify, distribute, transmit, or disclose to others outside your organization
the contents, opinions, conclusion, or information contained in this report (including any investment recommendations, estimates or price targets). All Cowen trademarks displayed
in this report are owned by Cowen and may not be used without its prior written consent.
Cowen and Company, LLC. New York (646) 562-1000 Boston (617) 946-3700 San Francisco (415) 646-7200 Chicago (312) 577-2240 Cleveland (440) 331-3531 Atlanta
(866) 544-7009 London (affiliate) 44-207-071-7500
COWEN AND COMPANY RATING DEFINITIONS
Cowen and Company Rating System effective May 25, 2013
Outperform (1): The stock is expected to achieve a total positive return of at least 15% over the next 12 months
Market Perform (2): The stock is expected to have a total return that falls between the parameters of an Outperform and Underperform over the next 12 months
Underperform (3): Stock is expected to achieve a total negative return of at least 10% over the next 12 months
Assumption: The expected total return calculation includes anticipated dividend yield
Cowen and Company Rating System until May 25, 2013
Outperform (1): Stock expected to outperform the S&P 500
Neutral (2): Stock expected to perform in line with the S&P 500
Underperform (3): Stock expected to underperform the S&P 500
Assumptions: Time horizon is 12 months; S&P 500 is flat over forecast period
Cowen Securities, formerly known as Dahlman Rose & Company, Rating System until May 25, 2013
Buy The fundamentals/valuations of the subject company are improving and the investment return is expected to be 5 to 15 percentage points higher than the general market
return
64
www.cowen.com
Addendum
Cowen and Company
Ohr Pharmaceutical
Equity Research
March 2, 2015
Hold The fundamentals/valuations of the subject company are neither improving nor deteriorating and the investment return is expected to be in line with the general market
return
Cowen And Company Rating Definitions

Distribution of Ratings/Investment Banking Services (IB) as of 12/31/14
Rating
Count
Ratings Distribution
Count
IB Services/Past 12 Months
Buy (a)
461
60.50%
109
23.64%
Hold (b)
288
37.80%
14
4.86%
Sell (c)
13
1.71%
0.00%
(a) Corresponds to "Outperform" rated stocks as defined in Cowen and Company, LLC's rating definitions. (b) Corresponds to "Market Perform" as defined in Cowen and Company,
LLC's ratings definitions. (c) Corresponds to "Underperform" as defined in Cowen and Company, LLC's ratings definitions.
Note: "Buy", "Hold" and "Sell" are not terms that Cowen and Company, LLC uses in its ratings system and should not be construed as investment options. Rather, these ratings
terms are used illustratively to comply with FINRA and NYSE regulations.
Ohr Pharmaceutical Rating History as of 02/27/2015

powered by: BlueMatrix
20
15
10
5
0
Apr 2012
Jul 2012
Oct 2012
Jan 2013
Apr 2013
Jul 2013
Closing Price
Oct 2013
Jan 2014
Apr 2014
Jul 2014
Oct 2014
Jan 2015
Target Price
Legend for Price Chart:

I = Initiation | 1 = Outperform | 2 = Market Perform | 3 = Underperform | UR = Price Target Under Review | T = Terminated Coverage | $xx = Price Target | NA = Not Available |
S=Suspended
www.cowen.com
65
Sell The fundamentals/valuations of the subject company are deteriorating and the investment return is expected to be 5 to 15 percentage points lower than the general market
return
Cowen and Company
Ohr Pharmaceutical
Equity Research
March 2, 2015
Analyst Profiles
Tyler Van Buren, M.Sc.
Ken Cacciatore
Sal Rais, M.D.
New York
New York
New York
646.562.1338
646.562.1305
646.562.1420
tyler.vanburen@cowen.com
ken.cacciatore@cowen.com
sal.rais@cowen.com
Tyler Van Buren is a vice president

covering specialty pharma. He joined
Cowen in 2013, after working at LifeSci
Advisors and Amylin.
Ken is a senior analyst covering

specialty pharmaceuticals. He has been
with Cowen for 17 years in health care
banking & equity research.
Sal Rais is an associate covering

specialty pharmaceuticals. He previously
worked at Campbell Alliance and has an
M.D. and MBA from UVA.
Reaching Cowen
Main U.S. Locations
New York
Boston
Cleveland
San Francisco
599 Lexington Avenue

New York, NY 10022
646.562.1000
800.221.5616
Two International Place

Boston, MA 02110
617.946.3700
800.343.7068
20006 Detroit Road

Suite 100
Rocky River, OH 44116
440.331.3531
555 California Street, 5th Floor

San Francisco, CA 94104
415.646.7200
800.858.9316
Atlanta
Chicago
3399 Peachtree Road NE

Suite 417
Atlanta, GA 30326
866.544.7009
181 West Madison Street

Suite 3135
Chicago, IL 60602
312.577.2240
International Locations
Cowen International
Limited
Cowen and Company (Asia)

Limited
London
Hong Kong
1 Snowden Street - 11th Floor

London EC2A 2DQ
United Kingdom
44.20.7071.7500
Suite 1401 Henley Building

No. 5 Queens Road Central
Central, Hong Kong
852 3752 2333
@CowenResearch
66
Cowen and Company

www.cowen.com
Points Of Contact

Cowen Research Report

Uploaded by

Document Information

Copyright

Available Formats

Share this document

Share or Embed Document

Sharing Options

Did you find this document useful?

Is this content inappropriate?

Copyright:

Available Formats

Cowen Research Report

Uploaded by

Copyright:

Available Formats

Pharmaceuticals/Specialty

Initiation: OHR-102 Success In Wet

Price: $7.69 (02/27/2015)

The Cowen Insight

Tyler Van Buren, M.Sc.

Market Cap (MM):

Net Debt (MM):

Dil. Shares Out (MM):

The Companys lead program is OHR-102, which is a first-in-class anti-angiogenic

Enterprise Value (MM): $184.8

Earnings Per Share

Please see addendum of this report for important disclosures.

Cowen and Company

Our Investment Thesis

Mid-to-late March Final topline

Base Case Assumptions

$25 on positive OHR-102 Phase II

$40 on better than expected IMPACT

$2-3 on Phase II IMPACT failure

data readout of the OHR-102 Phase II

Ohr Pharma is a development-stage specialty pharmaceutical company with multiple

Analyst Top Picks

Cowen and Company

OHR-102 is currently in Phase II development,

OHR-102 has a composition of matter patent on

OHR-102 is a first-in-class anti-angiogenic

Ohr Pharma is a development-stage specialty pharmaceutical company with multiple

Investment Thesis: OHR-102 Success In Wet AMD Likely To Create

However, this dogma appears to be changing

While there is still substantial risk to any wet

However, when analysis was conducted on the

Cowen and Company

Cowen and Company

Our consultants suggest that this endpoint is also

Upon potential successful final Phase II data, Ohr

Additionally, 48.3% of OHR-102-treated patients

Cowen and Company

Anticipated Upcoming Milestones For Ohr Pharma Are:

Valuation Analysis: A Risk/Reward Play Skewed Towards The Upside

Cowen and Company

- Low cost due to compounding

- 4 week treatment duration; 6-7 weeks in practice

- 8 week treatment duration

- Per consultants; most will want better vision

- 4 week treatment duration; 5-6 weeks in practice

Source: Cowen and Company; PriceRx

Figure 1 US Wet AMD Market Model

Cowen and Company

- We estimate 50% larger than U.S.

- 4 week treatment duration; 5-6 weeks in practice

- 8 week treatment duration

- Per consultants; most will want better vision

- 4 week treatment duration; 6-7 weeks in practice

- Low cost due to compounding

+96% - Assumes PDGF competition

Source: Cowen and Company; PriceRx

Figure 2 Ex-US Wet AMD Market Model

Cowen and Company

Figure 3 Ohr Pharma Annual P&L

- C urrently being tested in other retina disorders

+78% - Topical anti-angiogenic eye drop for wet AMD

- 90%+ margins; easy to manufacture

N M - True prof it abilit y reached in 2020

Source: Cowen and Company