EX-99.1 2 dex991.htm SLIDE PRESENTATION FOR INVESTOR AND ANALYST DAY Slide Presentation for Investor and Analyst Day
Investor and Analyst Day
June 23, 2011
Nasdaq: LGND
Exhibit 99.1


Investor and Analyst Day
John Higgins
President and Chief Executive Officer
2


3
Safe Harbor Statement
The following presentation contains forward-looking statements regarding Ligand’s
prospects, plans and strategies, drug development programs and collaborations.  Forward-
looking statements include financial projections, expectations regarding research and
development programs, and other statements including words such as “will,”
“should,”
“could,”
“plan,”
etc. Actual events or results may differ from Ligand’s expectations. For
example, expense reductions and drug development programs may not be realized.  In
addition there can be no assurance that Ligand will achieve its guidance in 2011.
The forward-looking statements made in the presentation are subject to several risk
factors, including, but not limited to, Ligand’s reliance on collaborative partners for
milestone and royalty payments, regulatory hurdles facing Ligand's, CyDex's and partner's
product candidates, uncertainty regarding Ligand's, CyDex's and partner's product
development costs, the possibility that Ligand's, CyDex's and partner's drug candidates
might not be proved to be safe and efficacious and commercial performance of Ligand's
and/or its partner's products.  Additional risks may apply to forward-looking statements
made in this presentation.
The risk factors facing Ligand are explained in greater detail in Ligand’s filings with the
SEC, including the most recently filed annual reports on Form 10-K and quarterly reports
on Form 10-Q, as well as other public filings.
While forward-looking statements reflect our good faith beliefs (or those of the indicated
third parties), they are not guarantees of future performance. We disclaim any obligation to
update or revise any forward-looking statements, whether as a result of new information,
future events or otherwise.


4
Investor and Analyst Day Agenda
Welcome and Company Overview
John Higgins
Captisol
®
Technology
Matt Foehr
Melphalan Program
Rick White
Promacta Highlights
Rob McKay
Thrombocytopenia in Hep C
Nezam H. Afdhal, M.D.
Chief of Hepatology, Director of Liver Center,
Beth Israel Deaconess Medical Center
Financial Highlights
John Sharp
Small Group Workshops
Captisol –
Powerful Enabling Technology
“Shots on Goal”
Portfolio
Questions and Answers / Reception


5
Small-Group Workshops
1.
Captisol
®
Powerful Enabling Technology
Matt
Foehr
Executive
Vice
President,
Chief
Operating
Officer
JD Pipkin –
Senior Director, New Product Development
Vince Antle –
Senior Director, Technical Operations and Quality Assurance
2.
“Shots-on-Goal”
Portfolio
Rob McKay
Senior Director, Business Development and Investor Relations
Syed Kazmi
VP, Business Development and Strategic Planning
Rick White
VP, Business Development and Marketing
Room
3
2


6
Ligand’s Business Model
Ligand's focus is to build a large portfolio of high
quality pharmaceutical assets that can drive
substantial cash flow and profitability. We operate
the
business with an emphasis on focused drug
development
and
partnerships,
with
a
disciplined
and
highly selective cost structure.


7
Revenue Outlook
Robust Pipeline
Captisol
®
Enabling Technology
High Quality Partners
The Foundation of the Ligand Opportunity


8
Ligand is Doing Great
Significant expansion (doubling of portfolio to over 60 programs) in
past 6 months as a result of Cydex acquisition
A number of positive news events by partners in 2011
Important new additions to senior management and Board of Directors
Most revenue generating assets ever on lowest cost structure
Positive clinical data announced in the past three months
Promacta
®
is a “now”
story
Directors have personally purchased over $1.2 million of Ligand stock
(131,125 shares) over the past 6 months. Directors now own 10% of
Ligand.
Over past year, daily trading volume has increased by ~30%


9
Ligand’s Potential Downside
Individual project set-backs
Slower growth
Partners drop programs
Risk
Reward
The Investment Proposition: Risk vs. Reward
Ligand’s Potential Upside
Potential blockbuster product approved/        
in-development
Near-term profitability
Well funded/financially disciplined
More
royalty
partnerships
than
any
peer
co
Attractive fully-owned
pipeline
Substantial calendar of news flow
Large NOLs
We believe the upside reward is substantially greater than the downside risk


10
Ligand’s Potential Upside
List presented last summer (2010)
Updated Outlook
Potential blockbuster product approved/
in development
Data
for
Promacta
®
expected
over
next
6
months
Near-term profitability
Projected to turn profitable by year-end 2011
Well funded/financially disciplined
Lowest cost structure in company’s history
More royalty partnerships than any peer co
Large portfolio of assets, doubled in over last 12 months
Attractive fully-owned pipeline
SARM, diabetes, JAK 3, and melphalan programs
Substantial calendar of news flow
Continued news flow
Large NOLs
Continue to be ready to use following profitability


11
Potential Significant Revenue Expansion Over Next Several Years
*Plus license fees
Illustrative Growth
2015
2012
"Shots on Goal" Vision                                          
Turning into Reality
Promacta
Avinza
Conbriza
Promacta
Nexterone
Avinza
Carfilzomib
Conbriza
Aprela
Nexterone
CXCR2
Carfilzomib
Clopidogrel
>$30 million revenue*
Melphalan
Carbamazepine
Merck Captisol
®
Program
>$200 million revenue*


12
Worldwide Quarterly Promacta
®
Revenue Growth
$0.0
$2.0
$4.0
$6.0
$8.0
$10.0
$12.0
$14.0
$16.0
$18.0
$20.0
4Q08
1Q09
2Q09
3Q09
4Q09
1Q10
2Q10
3Q10
4Q10
1Q11
New markets and
new indications
should help
accelerate
revenue growth
Ligand and GSK reports
Ligand expects to
receive royalties
for another         
14 years


13
Over 50 Partnered Programs
17%
9%
30%
39%
5%
Over half of the partnered portfolio is Phase II or later
Portfolio is highly diversified across many partners, stages of development and
therapeutic areas
Merck,
GSK
and
Pfizer
are
our
partners
with
the
most
programs
Marketed /
Approved
Phase III / NDA
Phase II
Phase I
PreClin
Ligand Portfolio: Partnered Programs


14
19%
12%
7%
26%
35%
Programs are highly diversified across more than 10 therapeutic areas
The therapeutic areas most represented in the portfolio are oncology,
inflammation, neurology and metabolic disease
Ligand Portfolio: Total Portfolio Snapshot
Total Portfolio (Over 60 programs)
Marketed /
Approved
Phase III / NDA
Phase II
Phase I
PreClin


15
Ligand's Internal Pipeline Focus


16
Timing*
Projected Event
3Q11
Carfilzomib NDA filing
Platform Captisol Partnership
4Q11
Promacta Phase III HepC Results
Aprela NDA filing
SARM program update
1Q12
Initiate pivotal Melphalan study
Top Line IL-9 Phase II Results
Clopidogrel 505(b)(2) study initiation
2Q12
Merck CXCR2/COPD Study Completion
Carfilzomib NDA Approval
Promacta sNDA filing for HepC
*Ligand internal estimates
Ligand Portfolio:
Potential Upcoming News Flow



18
High return late-stage
internal development
programs
New opportunities
New
Shots
High quality
relationships
Significant
expansion of “shots
on goal”
Deals
Powerful
enabling
technology
meeting key
needs
Captisol
Captisol
®
®
CyDex Acquisition:
What has it brought to Ligand?


19
Captisol
®
:
The Need


20
Patented, chemically-modified cyclodextrin
Increase drug solubility, reduce site reactions
Versatile across molecule families and sizes
Safe, inactive, inert
Over 100 clinical studies
Type V Drug Master File
Captisol
®
:
Enabling New Drugs


21
Strong manufacturing technology and IP estate
Hovione Partnership
50 metric ton capacity for Captisol
®
, ability to double
Exclusive relationship with built-in site redundancy
Intellectual Property
8 patent families covering technology, 12 patent families for products
Formulation and process-based patent portfolio providing protection
through 2029
Trade secrets, Drug Master File
Continuing internal innovation
Captisol
®
:
Enabling New Drugs


22
Captisol
®
:
The Value


23
Approved or
pending
products
Broader
platform
relationships
with partners
who can
leverage
Captisol
®
in
more ways
Internal
Captisol-
enabled
®
development
programs
with potential
for high
returns
Captisol
®
:
The Value


24
Reformulation can bring meaningful innovation to
established medicines
Late-stage internal development can create significant
value in exchange for relatively modest investment
505(b)(2) pathway leverages existing data
Lower development costs
Shorter timelines
Smaller infrastructure requirements
Captisol-enabled
®
Internal Development Programs


25
High dose conditioning treatment prior to
hematopoietic progenitor (stem) cell transplantation
for multiple myeloma
Orphan Drug Status
Market exclusivity, PDUFA fee waiver
Phase II dosing now completed
Interim results presented at ASCO
Pivotal trial projected to begin early 2012
Captisol-enabled
®
PG-Free Melphalan



27
Continuing need for new
and innovative therapies
Multiple Myeloma
Second most common
hematologic cancer
50,000 patients
in the U.S., 20,000
diagnosed annually
Area of growing
industry interest
Transplant remains
a centerpiece in
managing disease
progression


28
Multiple
Myeloma:
Disease
Treatment
and
Progression


29
Deep Freeze
Thawing and infusion of
patient stem cells
Autologous
Stem
Cells
Autologous
Stem
Cells
Autologous
Stem
Cells
High Dose
Chemotherapy
Cryopreservation of
patient stem cells
Mobilization and
leukapheresis of
patient stem cells
Multiple Myeloma:
Stem Cell Transplantation


30
Captisol-enabled
®
Melphalan for
Injection
Ligand
Internal Development Asset
One vial system
Propylene glycol-free
24-hr infusion window
Treatment Flexibility
Alkeran
®
Launched in 1993
GlaxoSmithKline
Two vial system
Propylene glycol diluent
60-min infusion window
Per label limitations
Melphalan Product Comparison


31
Captisol-enabled
®
Melphalan for
Injection
Ligand
Internal Development Asset
One vial system
Propylene glycol-free
24-hr infusion window
Treatment Flexibility
Product Advantages:
Improved stability and use time in an all
aqueous formulation
Longer administration durations, slower
infusion rates
Elimination of two-vial system
Physicians expected to:
Safely achieve higher dose intensity
Easily deliver concomitant meds in high-
dose regimens
Modulate dose to patient tolerability
Higher dosage
potential for improved
response rates
Melphalan Product Comparison


32
IMS Reports Sales of $85MM for Rolling 12 Month Period
Majority of the Current Usage as a High Dose Conditioning Agent Prior to
Autologous Stem Cell Transplant in Multiple Myeloma Patients (currently off
label)
Potential Label, and Orphan Designation, for Captisol-enabled
®
Propylene
Glycol-Free Melphalan Addresses this Market
Unmet Medical Need with the Current Standard of Care:
Stability following reconstitution (60 minutes)
Limits on Absolute Daily Dosing
Limits on Duration of Infusion
Potential Adverse Reactions Due to Co-Solvent (propylene glycol)  
Melphalan Market


33
A Marketing and Sales effort could be relatively lean and efficient
Melphalan therapy is well understood and entrenched, with few
influential bone-marrow-transplant hospitals
“Ultra-niche”
call universe
Benefits of Captisol-enabled
®
Melphalan formulation are easily
positioned
Effective promotion with lean sales organization achievable
Orphan Indication has favorable payer landscape (public and private)
Compass/Defined Health survey, 2009
Captisol-enabled
®
Melphalan: Opportunity


34
Captisol-enabled
®
Melphalan: Projected Timeline



36
The Shots on Goal Model
8 Assets Currently
Generating Commercial
Revenue
Over 50
Partnered Programs
Over 60
Total Programs
Over 25
Different Partners
Over 10
Therapeutic Areas
Minimizing Risk Through Portfolio Size and Diversification
10 Potential New
Approvals in
the Next 4 Years
-
-


37
The Value Pyramid For Ligand Assets
The Top of the Value Pyramid for Ligand is Promacta
Avinza
Carfilzomib
Aprela  Nexterone
CXCR2 Viviant
Over 50 Additional Programs
from Preclinical to PIII
There are numerous programs in
the Ligand portfolio today which
add significant value to the
business
But, Ligand’s royalty interest in the
Promacta franchise at GSK is the
most valuable single asset Ligand
owns


38
Promacta  Background
What is Promacta
Promacta is a once-daily oral medicine that activates the thrombopoietin
(TPO) receptor
Activation of the TPO receptor causes platelets to increase, relieving
conditions of low platelets known as thrombocytopenia
History
Ligand and GSK jointly discovered Promacta as part of a thrombopoietin
(TPO) receptor agonist research collaboration started in 1995
GSK later licensed from Ligand the follow-on TPO receptor agonist LGD-4665
in 2008


39
Approved for ITP
Marketed by GSK
PIII HepC Data Release in 3Q/4Q11
Promacta Follow-On : GSK-5921
5-10%, blended 9% on $1B in Sales
An Approved Drug in All Major Markets 
Promacta
®
: The Foundation of the Ligand Growth Story
Aspects Of The Promacta Franchise Make It An Ideal
Foundation For Ligand’s Financial Growth Story
Significant Royalty Interest 
Major Upcoming Catalyst Events 
Long Patent Protection 
Major Potential for Label Expansion 
Marketed by a Premier Pharma Company 
Life-Cycle Management Opportunity 
ITP
HepC
Oncology
Others
Patented until July 2025


40
Promacta Program Recent Developments (1 of 2)
New ITP Launches Around the World
EU, Japan, South America
Increased ITP Sales
World-wide ITP sales in 1Q2011 increased 109% over 1Q2010
The Promacta ITP Franchise Has Continued To
Generate Positive News In The Past Year


41
Promacta Program Recent Developments (2 of 2)
Full ITP Approval Granted by FDA
GSK completed post-approval commitment of generating long-term safety
data
Label now includes efficacy and safety data from RAISE, 6-month ITP study
Since 2008, GSK has been working with FDA. Studies submitted:
6-month efficacy and safety data
2-year safety data in chronic ITP
Updates from chronic liver disease (ELEVATE) trial
The Promacta ITP Franchise Has Continued To
Generate Positive News In The Past Year


42
Idiopathic Thrombocytopenia Purpura (ITP)
Phase I
Phase II
Phase III
Approved
Hepatitis C-Related Thrombocytopenia
Oncology-Related
Thrombocytopenia
Clinical Studies
2 P-III Studies
GSK Is Investing Significantly In The
Growth Of The Promacta Franchise
11 PI and PII Studies
Approved
(Label Expansion
Studies Ongoing)
GSK Investment in Promacta Franchise Expansion


43
Patients
needed
to potentially
reach
$1B
in sales
Thrombocytopenia Is Comprised Of Multiple Sub-Markets of
Thrombocytopenia-Inducing Diseases, Similar to Anemia and Neutropenia
Wall Street Research on GSK published Nov. 21, 2005
Thrombocytopenia: A Multibillion Dollar Platelet Potential


44
Significant Revenue Growth Potential for Ligand
Territory Expansion
Indication Expansion
HepC
Oncology
Others
+
+
Long IP Protection
2025
Expansion of the Promacta Franchise
Translates Into Significant Revenue Growth for Ligand
Promacta Royalty
Illustrative Ligand Revenue
Annual Sales
Royalty
<$100M
4.70%
$100M-$200M
6.60%
$200M-$400M
7.5%
$400M-$1.5B
9.40%
>1.5B
9.30%
Annual
Sales
Blended
Royalty
Ligand
Revenue
$500M
7.1%
$36M
$1B
8.3%
$83M
$1.5B
8.6%
$130M


45
45
The Role for Eltrombopag (Promacta) in the
Treatment of HepC-Related Thrombocytopenia
N. Afdhal M.D
Beth Israel Deaconess Liver Center
Harvard Medical School


46
46
Agenda
Eltrombopag and thrombocytopenia
Current status of HCV therapy
The ENABLE 1 and ENABLE 2 P-III Studies
ELEVATE Study –
conclusions on Eltrombopag safety


47
47
The Unmet Medical Need for Thrombocytopenia
Thrombocytopenia is a major factor in patients being unable to
achieve a desired clinical outcome in dozens of diseases
Currently estimated to be nearly two million patients annually in the
US who need to be treated for thrombocytopenia
Current non-drug techniques used to increase platelets (i.e. platelet
transfusion, splenectomy) are costly, risky, and inconvenient.
The need for a more convenient and effective method for combating
thrombocytopenia is clear


48
48
Liver DiseaseAssociated Thrombocytopenia
Platelet counts may be as low as
20,000–40,000/
L
Prevalence of thrombocytopenia
increases with severity of liver disease
Degree of thrombocytopenia correlated
with severity of liver disease
Thrombocytopenia predictive of
reduced 5-year survival
Thrombocytopenia may develop or
worsen with interferon-based therapy
4% in recent DAA trials
Degree of Liver
Damage
Thrombocytopenia
Prevalence
Normal liver
2.3%
Fatty liver
5.1%
Chronic hepatitis
20.3%
Advanced liver
disease
31.8%


49
49
Eltrombopag
Thrombopoietin receptor agonist
Oral, once-daily tablet
Induces megakaryocyte
proliferation and differentiation
Increases platelet counts in
patients with HCV¹
1. McHutchison J, et al.N Engl J Med.
2007;357:2227–2236.


50
50
Current Status of HCV therapy
and the Role for Eltrombopag


51
Thrombocytopenia, HepC, and Eltrombopag
Nearly 10% of HepC
patients in the US suffer from treatment-limiting
thrombocytopenia
These patients have significantly worse outcomes due to their
underlying liver disease and inability to complete antiviral therapies
Eltrombopag
PII data demonstrates that this drug may be useful for
these patients to overcome thrombocytopenia limitations and
complete antiviral therapy
Key
question
arising
around
the
eltrombopag
HepC
opportunity
How will new HepC
therapies impact the need for eltrombopag?
Is
there
a
long-term
safety
concern
for
eltrombopag
because of
the ELEVATE study results?


52
HCV Pipeline* by MOA and Stage of Development
3/2/2010 –
selected
compounds only.
*Publicly available information via press release, corporate
presentations, and assumptions based on patent filings.


53
Target “Regimen”
Outcomes
Today’s Regimen
2015+
Target
IFN + Ribavirin
(R)
G1-Naive
SVR = 40%
Duration = 48 weeks
G2-3 Naïve
SVR = 75%
Duration = 24 weeks
G1-Experienced
No meaningful option
Frequent dosing and
poor tolerability
New Triple / Quad
Combo Regimens
Highest possible SVR
No / low resistance
Better tolerability
Shorter durations
Simplified delivery
1.
IFN + R + DAA
2.
IFN + DAA + DAA
3.
IFN + R + DAA + DAA
4.
DAA + DAA (+ DAA)
IFN = Interferon
R = Ribavirin
DAA = Novel Direct Acting Antiviral agents
Regimen 4. High risk / low probability / far away / no PoC


54
SVR Rates in Telaprevir-Treated Patients Compared with
Peginterferon/Ribavirin
Alone
Jacobson IM et al. Hepatology.
2010;52(Suppl 1):Abstract 211.
Telaprevier
12 weeks +
Peg/Riba
48 weeks
Telaprevier
8 weeks +
Peg/Riba
48 weeks
Peg/Riba
48 weeks


55
The Impact of New HepC
Therapies on the Use
of Eltrombopag
in HepC
Underlying thrombocytopenia is still an issue for many HepC
patients
The new protease inhibitors do not change that fact
New protease inhibitor therapies slightly increase the rate of
thrombocytopenia
Due to increased SVR seen with new cocktails, the need for
eltrombopag
should actually increase
Higher SVR rates may encourage doctors to treat more
aggressively those patients with thrombocytopenia


56
56
Eltrombopag
Clinical Studies
HepC
Studies


57
Eltrombopag
PII HepC
Study Design
4 wk
12 wk
PEG-IFN + ribavirin
+ eltrombopag
Pre-antiviral
Eltrombopag
4 wk
4 wk
4 wk
12 wk +
Eltrombopag
12 wk +
Eltrombopag
12 wk +
Eltrombopag
INITIATE
antiviral Rx if platelets
>70–100 K/µL
30 mg
50 mg
75 mg
PART 1                                         PART 2
4 wk
4 wk
4 wk
4 wk
Follow-up


PLACEBO
30 mg
50 mg
75 mg
0.0007
0.0003
< 0.0001
0%
74%
75%
95%
0%
20%
40%
60%
80%
100%
McHutchison, NEJM 2007
58
Phase II: Primary Efficacy Endpoint
Platelet count
100,000/uL at Week 4


59
59
PLACEBO
30 mg
50 mg
75 mg
6%
53%
36%
65%
0%
20%
40%
60%
80%
100%
Phase II:
Subjects Completing 12 Weeks of  PEG-IFN Therapy


Phase II:
Median Platelet Count > 200K without thrombotic events
0
50
100
150
200
250
0
14
28
42
56
70
84
98
112
Placebo
30 mg
50 mg
75 mg
INITIATION
MAINTENANCE
Study Day
McHutchison, AASLD 2006
60


61
Treatment Group, n (%)
PBO
N=18
30mg
N=14
50mg
N=19
75mg
N=23
Any AE
10 (56)
11 (79)
10 (53)
13 (57)
Headache
3 (17)
5 (36)
3 (16)
4 (17)
Dry mouth
1 (6)
2 (14)
2 (11)
2 (9)
Pruritus
0
0
0
2 (9)
Nausea
0
1 (7)
2 (11)
1 (4)
Fatigue
0
0
2 (11)
1 (4)
Upper abdominal pain
0
2 (14)
2 (11)
0
Insomnia
0
0
2 (11)
0
Arthralgia
0
2 (14)
1 (5)
0
No thromboembolic
or elevated LFT events of concern
Phase
II:
Adverse
Events
Pre-Antiviral
Phase


62
62
Phase II: Conclusions
Eltrombopag increased platelet counts in subjects in all dose groups
A significant number of subjects achieved the primary endpoint (Week 4)
in all dose groups compared to placebo
Eltrombopag enabled 45/56 subjects to initiate IFN therapy
31 subjects completed 12 weeks of IFN therapy
Preliminary PK findings in general indicate exposure increases with dose
with wide variability
No safety signals of concern in this initial short term study
Safety and efficacy data supports further investigation of eltrombopag in
this patient population


Two parallel global Phase III studies
ENABLE 1 and 2
63
peginterferon
alfa-2a
(PEGASYS)
plus
ribavirin
ENABLE 1
peginterferon
alfa-2b
(PEG-Intron)
plus
ribavirin
ENABLE 2
ltrombopag
to
INitiate
and
Maintain
Interferon 
ntiviral
Treatment
to Benefit Subjects with Hepatitis C related Liver DiseasE
E
A


Pre-Antiviral Phase
Open-label eltrombopag
(2 –
9 weeks)
Antiviral Phase
Randomized
(up to 48 weeks)
platelets
>90,000/uL (E1)
or
100,000/uL (E2)
25mg>50mg>75mg>100mg
2:1 randomization eltrombopag:placebo
Dose titration of eltrombopag allowed throughout
Primary endpoint = proportion of patients achieving SVR (6M off –Tx)
N=750 dosed/675 randomized study
3 regions, 26 countries, >250 centres
SVR
SVR
6 months
off-Tx
Randomized Withdrawal Design
open label
eltrombopag
Eltrombopag
+ PEG-IFN/Rib
Placebo
+ PEG-IFN/Rib
64


65
65
Screening
45
Days
Platelets
<75K/µL
Pre-Antiviral Treatment Phase
Open-label eltrombopag dosed for
up to 9wks until platelet count
increases to enable initiation of
antiviral therapy.
Part 1 (2-9 Wks)
Open-Label
Platelets
90K/µL*
Platelets
90K/µL*
Platelets
90K/µL*
*Platelets
<90K/µL
*Platelets
<90K/µL
*Platelets
<90K/µL
WD
START
*Platelets
<90K/µL
Platelets
90K/µL*
*90K/µL (ENABLE 1)
100K/µL (ENABLE 2)
2:1 Randomisation (Eltrombopag:Placebo)
Eltrombopag
plus
Antiviral Therapy
Antiviral Treatment Phase
Randomised either to
maintain dose of
eltrombopag from Part
1 or to matched
placebo.
Part 2 (
48 Wks)
Double-Blind
Placebo
plus
Antiviral Therapy
or
a
24 Week FU
Ocular/SVR
a
Post-last dose of
investigational
product.
ENABLE 1 and 2 Study Design
25mg
2 Weeks
50mg
1-2 Weeks
75mg
1-2 Weeks
100mg
1-3 Weeks


66
SVR rate defined as percentage of subjects with non-detectable
HCV-RNA at 24 weeks post-completion of the planned treatment
period
Platelet count
90-100,000/
L in Part 1
Dose modifications
Safety and tolerability
Platelet counts
PK
RVR,
EVR and ETR
Health-related quality of life
Safety
modified
to
include
risk
of
thrombotic
events
both
studies
completed without DSMB concerns
Endpoints


Eltrombopag Safety Summary
ELEVATE Study and Safety
67


68
Eltrombopag in Chronic Liver Disease Patients
with Thrombocytopenia Undergoing an Elective
Invasive Procedure: Results from ELEVATE,
a Randomised Clinical Trial
N.
Afdhal,
E.
Giannini,²
G.N.
Tayyab,
A.
Mohsin,
4
J-W.
Lee,
5
A. Andriulli,
6
L. Jeffers,
7
J. McHutchison,
8
F. Campbell,
9
N. Blackman,
10
D. Hyde,
9
A. Brainsky,
11
D. Theodore
12
1. Division of Gastroenterology/Liver Center, Beth Israel Deaconess Medical Center, Boston, MA, USA;  2. Gastroenterology Unit, Department of Internal
Medicine, University of Genoa, Genoa, Italy;  3. Department of Medicine, Gastroenterology and Hepatology, Post Graduate Medical Institute, and Lahore
General Hospital, Lahore, Pakistan;  4. Department of Gastroenterology, Services Hospital Lahore, Services Institute of Medical Sciences, Lahore,
Pakistan;  5. Department of Internal Medicine, Inha University Hospital and
School of Medicine, Incheon, Korea;  6. Department of Internal Medicine,
Division of Gastroenterology, Casa Sollievo Sofferenza Hospital,
San Giovanni Rotondo, Italy;  7. Center for Liver Diseases, University of Miami, Miller
School of Medicine, Miami, FL, USA;  8. Division of Gastroenterology, Duke University Medical Center, Durham, NC, USA;  9. Clinical Development,
GlaxoSmithKline, Stockley Park, Uxbridge, UK;  10. Biometrics and Epidemiology, GlaxoSmithKline, Collegeville, PA, USA;  11. Clinical Development,
GlaxoSmithKline, Collegeville, PA, USA;  12. Clinical Development, GlaxoSmithKline, Research Triangle Park, NC, USA
1
3


Difference 53% (95% CI: 43, 62)
p
< 0.0001
0
10
20
30
40
50
60
80
100
Placebo
(N = 147)
Eltrombopag
(N = 145)
70
90
72%
19%
n = 28
n = 104
Primary Endpoint: Avoiding Platelet
Transfusions with Elective Invasive Procedure
69


Placebo
(N = 145)
Eltrombopag
(N = 143)
n (%)
n (%)
Bleeding
25 (17)
19 (13)
Thrombotic event
2 (1)
6 (4)
Ocular (focus on cataracts / visual
acuity decrease)
6 (4)
6 (4)
Malignancies*
1 (<1)
1 (<1)
Selected Adverse Events
70
* Basal cell carcinoma (Grade 2) reported for one patient receiving placebo and B cell lymphoma (Grade 4) reported
for one patient receiving eltrombopag; neither was considered to be related to treatment by the investigator.


71
71
Thrombotic event
Temporal
relationship to last
dose
Temporal
relationship to
procedure
Platelet count at
event (Gi/L)
Procedure
PV / SMV thrombosis
+1
–6 days
417
Brain tumour
resection
PV thrombosis
+5
+4 days
288
Oesophageal
variceal
ligation
SMV thrombosis
+8
+7 days
235
Dental extraction
SMV / mesenteric
thrombosis
+9
+7 days
289
HCC ablation
SPV thrombosis
+14
+13 days
241
TACE
PV thrombosis
+38
+34 days
33
Oesophageal
variceal
ligation
Acute MI
+20
+19 days
83
Colon resection
Non-occlusive PV and
mesenteric thrombosis
+128
+128 days
Unknown
Oesophagoduo-
denoscopy
PV, portal vein; SMV, superior mesenteric vein; SPV, spleno-portal vein; MI, myocardial infarction
Eltrombopag
Placebo
Summary of Thrombotic Events


72
72
Platelet count >200,000/µL
YES
NO
47 patients
(16%)
241 patients
(84%)
YES = 5 (10.6%)
NO = 42 (89.4%)
YES = 3 (1.2%)
NO = 238 (98.8%)
TEs
Thrombotic Events and Platelet Count


73
73
ELEVATE Conclusions
Eltrombopag 75 mg for 14 days
Reduced the need for platelet transfusions in CLD patients
with thrombocytopenia undergoing elective invasive
procedures
Increased platelets during treatment period and up to 2
weeks following treatment
Similar incidence of adverse events and serious adverse
events
More thrombotic events in the eltrombopag group
Relationship demonstrated for elevation of platelets
Procedure with endovascular inflammation essential feature


74
74
ENABLE studies are expected to confirm role of eltrombopag in HCV
therapy in 2011
Eltrombopag
is
expected
to
be
more
widely
used
in
HCV
increased
SVR –
increased treatment
Long term safety of eltrombopag  continues to be better understood
and  continued expansion of the eltrombopag franchise is warranted
Eltrombopag Summary


75
Financial Highlights
John Sharp
Vice President, Finance
and Chief Financial Officer
Ligand
Pharmaceuticals
Incorporated


76
2011 Revenue Outlook
Total 2011 revenue currently projected to
be $22 -
$24 million
$11
-
$12
million
from
Captisol
sales
$8 million from royalties
$3 -
$4 million from license and other
Potential for additional sources of
revenue and cash in 2011 above these
projections based on new license
agreements
2011 Revenue Breakdown*
License And Other
Royalties
Material Sales
*Excludes revenue from new deals, if any
~15%
~35%
~50%


77
Financial Guidance
2011 Guidance:
Revenue of $22 -
$24 million
Operating expenses projected to be ~$20 million
Projecting turning profitable on an operating basis and
having positive cash flow from operations by the 4
th
quarter of 2011
Cash at year-end projected to be ~$20 million


78
Low Cost Structure
$0.0
$10.0
$20.0
$30.0
$40.0
$50.0
$60.0
$70.0
$80.0
2007
2008
2009
2010
2011*
G&A
R&D
* High end of expense guidance range
Ligand
has significantly reduced expenses over the last several years
During the same period, the company closed 5 acquisitions while
significantly expanding its asset base
Pharmacopeia Acquisition
Neurogen
Acquisition
Metabasis
Acquisition
CyDex
Acquisition
IL9 Account Acquisition


79
Multiple Future Revenue Sources
8 programs currently generating royalty revenues
Over 50 partnered programs
Numerous internal programs
Growing Captisol
business


80
Expected Revenue Growth
*Excludes revenue from new deals, if any
$0.0
$5.0
$10.0
$15.0
$20.0
$25.0
$30.0
$35.0
$40.0
$45.0
2011 Guidance*
2013 Illustrative
License & Other
Material Sales
Royalty
Revenue could potentially double within two years on similar cost structure
Recurring and high-growth royalty revenue projected to drive the majority of revenue in 2013
*


81
High Quality Revenue
Future revenue drivers:
Increased royalties from multiple commercial products
(Promacta,Conbriza, Carfilzomib, Nexterone, etc…)
Steadily
increasing
Captisol
sales
Less
dependence
on
“one-time”
license/milestone
events
Continued potential for additional sources of revenue and cash
from new license agreements


82
Value of Net Operating Loss Carryforwards
(NOL)
Ligand
has
accumulated
substantial
NOL’s
through
our
operating
history and acquisitions
NOL’s
should provide significant relief on taxable income if the
company turns profitable
NOL’s
as of December 31, 2010 = $438 million
Due
to
tax
code,
NOL’s
are
limited
in
the
quantity
and
the
timing
in
which
they
can
be
used,
so
we
do
not
expect
to
get
a
full
offset
on
taxable income immediately
Estimated
net
present
value
of
NOL’s
=
~$100M
In near-term, the NOL tax should reduce federal tax rate from 34%
down to 2% (AMT)
Additionally, Ligand
has ~$16 million of federal R&D Tax Credits


Ligand Pharmaceuticals Incorporated
83
83
83
83
“Shots on Goal”
Portfolio
2011 Ligand Analyst Day Workshop


The “Shots on Goal”
Model at Ligand
The low ROI for biopharma R&D suggests that sharing the cost and
risk of R&D through partnering is a preferred route to meaningful ROI
Ligand has assembled the largest portfolio of partnered assets of any
company in its biopharma peer group
Now over 50 assets fully funded by partners
A focus on assembling partnered assets allows Ligand to run a very
lean cost structure without  significantly high R&D costs
Coupled with our cost structure,
this portfolio of assets gives
Ligand a reasonable probability of
becoming a sustainably profitable
biopharma business
84


85
Industry Unique Partnered Asset Portfolio
Over 25 different partners
Over 10 different
therapeutic areas
Over half of the portfolio is
PII or later
Ligand estimates that
partners spend over
$150M per year on this
portfolio
Significant  quarterly news
flow


86
Portfolio Asset Highlights
PIII/NDA
Captisol-enabled
proteasome
inhibitor for multiple
myeloma
Ligand eligible to
receive milestones,
royalties and
material sales
revenue
Potential 2012
launch
Beyond Promacta, there are multiple-late stage programs in the portfolio
that offer Ligand opportunities for substantial future revenue growth
Onyx
Carfilzomib
Merck
CXCR2
Pfizer
Aprela
Merck
Captisol Program
PII
Novel CXCR2
antagonist for
COPD
Ligand eligible to
receive milestones
and royalties
Potential 2015
launch
PIII
Combination of
Viviant and
Premarin for
menopausal
symptoms
Ligand eligible to
receive milestones
and royalties
Potential 2013
launch
PII
Captisol-enabled
undisclosed
program
Ligand  eligible to
receive milestones
and material sales
revenue
Potential 2013
launch
Over 40 Other Programs from Preclinical to Phase III Represent
Another Potential Revenue Wave


87
Quality Portfolio News Flow
New Territory Launches
New Product Launches
NDA Approvals
NDA Filings
3Q11
4Q11
1Q12
2Q12
3Q12
4Q12
The
Size
of
the
Portfolio
Generates
Regular,
Meaningful,
Quarterly
News
Flow
PIII Data Releases
PIII Trial Initiations
PII Data Releases
Promacta
Nexterone
Viviant
Aprela
Carfilzomib
CXCR2
IL-9
CDK
Merck Captisol
Clopidogrel
Others


88
Deep Partner Relationships
PII CXCR2 (COPD)
PII CDK Inhibitor
(oncology)
PI BACE (Alzheimers)
Captisol Program
Promacta ITP
Promacta HepC
Promacta Oncology
LGD 4665/GSK 3521
Viviant
Aprela
Marketed Captisol
Products
Ligand has Multiple Broad and Diverse Relationships
With Premier Pharma Companies


89
Ligand Business Development
LIGAND
REVENUE DRIVERS
LIGAND
PROPRIETARY PIPELINE
LIGAND
BUSINESS
DEVELOPMENT
Ligand business development is the core engine that drives the shots on goal model
-Acquisitions –
Licensing –
Spin Offs -
Alliance Management-