2. 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 2012 or thereafter.
The forward‐looking statements made in the presentation are subject to several risk factors, including, but not
limited to, statements regarding intent, belief, or current expectations of the Ligand, its internal and partnered
programs, including Promacta, Kyprolis (Carfilzomib), Melphalan, Ligand’s reliance on collaborative partners for
milestone and royalty payments, regulatory hurdles facing Ligand's and partner's product candidates,
uncertainty regarding Ligand's and partner's product development costs, the possibility that Ligand'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.
2
3. Overview of Ligand
Heritage
• Founded in 1987 in La Jolla, CA
• Rich heritage of drug discovery, development and partnering
• Contributed to the development of over 40 novel clinical
candidates and a dozen approved medicines
3
4. Overview of Ligand
Today
• Leveraged history into a new business model centered
around partnering and acquisitions
• Large portfolio of over 60 partnered programs
• 5 acquisitions in last 4 years have added significantly to
our portfolio and increased depth of partnerships with
many
• Most recent acquisition (CyDex Pharmaceuticals)
added enabling technology to our portfolio
4
7. Ligand’s Portfolio ‐ Partnered Programs
Over 60 fully funded partnered programs
Nearly 50% in Phase 2 or Later
Among others, includes::
Carbamazepine‐IV (Captisol‐enabled carbamazepine)
CNS
Phase 3
Est. Primary Completion Date: Dec. 20121
Aprela (SERM+Premarin)
Menopause related
Est. NDA 2012
Phase 2 Dinaciclib (CDK Inhibitor)
30% Oncology, multiple
Est. Phase 3 Initiation 2012
Delafloxacin (fluoroquinolone)
Infection
Est. Phase 3 Initiation 2H 2012
7 1 http://clinicaltrials.gov/ct2/show/NCT01128959?term=Captisol&rank=7
8. Ligand’s Currently Unpartnered Projects
Internal R&D Assets Muscle Wasting
SARM
Phase 2 Ready
Infectious Disease Diabetes
HepDirect® Technology Glucagon Receptor
Est. IND 2013 Antagonist
Est. IND 2013
Oncology Blood Disorders
Melphalan IV EPO Receptor Agonist
Pivotal Trial 2012 Preclinical
GCSF
Seizure Preclinical
Topiramate IV
Preclinical
8
9. Illustrative Revenue Growth
Potential
"Shots on Goal" 2015
Vision
Promacta
Turning into Reality Kyprolis
2012 Conbriza
Nexterone
Avinza
Promacta ®
Aprela
Kyprolis ®
Dinaciclib
Avinza ®
CE‐Clopidogrel
Conbriza®
CE‐Melphalan
Nexterone®
CE‐Carbamazepine
Captisol Material Sales
Lilly CE Program
License/Milestone Fees
Hospira CE Program
Captisol Material Sales
$30 million License/Milestone Fees
$150 million
9
10. Projected Financial Highlights
2012 Financial Guidance 2012 Revenue Composition
• $30M in total revenue
~ 25%
• $25M in operating expenses Material
Includes $6M non‐cash expense Sales
~ 50%
Royalty
~ 25%
License
Fees
NOLs exceed $450 million
19.9 million shares outstanding
10
11. Promacta and Kyprolis
Primetime
• Ligand has valuable partnerships with GSK and Onyx for
two potential blockbuster drugs
• Estimated peak sales for Promacta and Kyprolis combined
could achieve $4 billion annually
• Annual royalty revenue for these two products could
approach $200 million combined
11
13. Promacta
A Potential Blockbuster Driver for Ligand Growth
Oral Medicine that Boosts Platelets
to Treat Thrombocytopenia
• Marketed by GSK for ITP
• Significant royalty interest
• Major upcoming 2012 catalysts
• Long patent protection through 2027
• Potential for major label expansion
• Strong partner significantly investing, 25+ active clinical trials
13
14. The Blood Business has Three Main Markets
Disease Main Products 2011 ~Revenues
Red Blood Cells Anemia Epogen® $9 Billion
Aranesp®
Procrit®
White Blood Cells Neutropenia Neupogen® $5.8 Billion
Neulasta®
Platelets Thrombocytopenia Promacta® $0.4 Billion
Nplate®
14
15. Blood Market Overview 2011 ~Revenues
$9
$9 Billion Markets have been
$8 around ~20 years
• Anemia and Neutropenia
markets have been well $7
served by blockbuster drugs
$6
for about 20 years. These are
large, will established
$ Billions
$5.8 Billion
$5
markets
$4
• Thrombocytopenia is a very Infant market,
young high‐growth market huge growth
$3
potential
with the introduction of two
medicines approximately 3 $2
years ago
$1
$0.4 Billion
$0
Anemia Neutropenia Thrombocytopenia
15
16. Thrombocytopenia: Major Need & Opportunity
Thrombocytopenia contains a number of “sub‐markets” of rare
thrombocytopenia‐inducing diseases, similar to anemia, neutropenia markets
Medical Need is Real, Life Threatening, and Unmet
1800
1600 400 1505
1400
1200
Patient #s (000s)
340
1000
60 Patients
800 120 needed to
250 120 potentially
600
reach $1B
400 20 15 in sales
140
120 20
200
35 120
0
Sources: Aplastic Anemia (<1000 patients): (N Engl J Med 2012; 367:11‐19)
16
17. Promacta Market Opportunity
Estimated Peak
Marketed Annual Revenue
Potential
ITP $500 Million
sNDA Submitted
HCV $1.5 Billion
Phase 2
HORT and other $2.0 Billion
17
20. Promacta
A Potential Blockbuster Driver for Ligand Growth
$140
Annual ITP Sales ($M)
$120
Other
EU
$100 US
$80
• Dramatic Revenue Acceleration
$ millions
252% growth in 2011
$60
• Promacta is Gaining ITP Market Share vs. Nplate
19% in 4Q10
$40
33% in 4Q11
$20 • Ligand is entitled to a net 8.3% royalty
on $1 billion in annual sales
$‐
2009 2010 2011
20
21. Current Niche Indication: ITP
Strong Quarterly Revenue Growth for Ligand
$2.5
$2.0
• Product in infancy – patents and
Ligand Promacta Revenue ($ millions)
$1.5 royalties through 2027
• With higher sales, Ligand earns higher
royalty rates
$1.0
• Multiple major indications still in
development
$0.5
$0 Q1 Q2 Q3 Q4 Q1 Q2 Q3 Q4 Q1 Q2 Q3 Q4 Q1 Q2
2009 2010 2011 2012
21
22. Promacta
Hepatitis C‐related Thrombocytopenia
• Next big potential indication for Promacta is
thrombocytopenia in Hep C patients
• GSK submitted sNDA for US and Europe in May on basis of
positive Phase 3 trials
• Granted 6‐month accelerated review period (PDUFA date
November 2012)
• Potential blockbuster market opportunity for Hep C indication
alone
22
23. HCV: Significant Promacta Opportunity Before
AND After "All‐Oral” Regimens Arrive
• Alpha interferon and Ribavirin are still mainstay of HCV
treatment. "All Oral" has not become a commercial reality yet.
• If “All Oral" regimens are approved, timing outlook by third parties is
between two to four years
• "All Oral" regimens treat HCV, but they do not boost platelets or treat
thrombocytopenia. The sickest HCV patients with cirrhosis may still
require platelet intervention during treatment
• Developing countries have a higher rate of HCV genotypes other than
genotype 1. Due to lower cost and the disease strain, interferon‐
based regimens may still be a treatment standard in the large
markets in the developing countries
23
24. Promacta
Development in Oncology‐related Thrombocytopenia
Oncology‐related Thrombocytopenia
Significant unmet medical need
Worldwide patient population of ~100,000 annually
Chemotherapy Cancers of the Blood
‐induced (CIT)
Bone Marrow Myelodysplastic Acute Myeloid
Suppression Syndrome (MDS) Leukemia (AML)
Damage to platelet Patients develop
forming cells in bone severe cytopenia, Fast‐growing cancer
marrow require frequent
transfusions Abnormal red blood cells
Can limit treatment
and platelets can quickly
Excess bleeding crowd out normal cells
Leads to transfusions
results in major
and transplants
complications or
death for nearly 25%
of patients1
1 Expert Opinion: Thrombocytopenia & Myelodysplastic Syndrome http://www.medscape.org/viewarticle/565023
24
25. Jefferies Published Revenue Outlook for
Promacta/Revolade
• If Promacta revenue
reaches $667 million in
2016, Ligand will earn
approximately $50 million
$ millions
in royalties
Jefferies Equity Research Report for GSK dated May 3, 2012
BUY rating, sales figures converted from GBP to US Dollars
25
28. The Need for an Enabling Solubility Technology
4 out of 5 Drug candidates
have poor solubility
2 out of 5 Marketed drugs
have poor solubility
Sources: Chemical & Engineering News, 2010, American Pharmaceutical Review ‐ Solubility Roundtable, 2011
28
29. The Need for an Enabling Solubility Technology
4 out of 5 Drug candidates
have poor solubility
Kyprolis™
2 out of 5 Marketed drugs
have poor solubility
Sources: Chemical & Engineering News, 2010, American Pharmaceutical Review ‐ Solubility Roundtable, 2011
29
30. Primetime Highlight
Kyprolis™ for Multiple Myeloma
• Kyprolis (carfilzomib) is formulated with Ligand’s Captisol
• Improved solubility with Captisol enabled significantly
reduced drug load
• Kyprolis™ has shown compelling efficacy data
• 20%‐range response rates across patients
resistant/not responsive to 5 classes of currently
approved multiple myeloma drugs
• Strong potential for improvements over Velcade®
forms
• Despite important strides in the last decade, multiple
myeloma remains uniformly fatal
30
39. 12 in 2012 Highlight
Dinaciclib for Oncology
• Merck has studied dinaciclib in multiple Phase
2 studies for cancer
• Dinaciclib is cyclin dependent kinase (CDK)
inhibitor‐ inhibiting CDK blocks cell‐cycle
progression and promotes apoptosis
• Merck is making a significant commitment to
increase its oncology presence
• Ligand is entitled to receive milestones and
royalties from the dinaciclib program
39
40. Dinaciclib (CDK Inhibitor)
• What is CDK and why is it important?
• Family of kinases that are critical regulators of cell
cycle Progression
• Cell cycle dysregulation is hallmark of cancer
inhibiting CDK should block cell‐cycle progression
and promote apoptosis
• Dinaciclib inhibits CDK‐1, ‐2, ‐5 and ‐9
• In what diseases has Dinaciclib been studied?
• Phase II studies have been completed in ALL/AML,
CLL/CML, lymphoma, advanced breast/lung cancer
and mantle cell lymphoma/B‐cell CLL
• NCI conducting studies in Stage IV melanoma and
multiple myeloma
CDK Control of the Cell Cycle
• Why is Dinaciclib interesting? (sandwalk.blogspot.com‐2009)
• Novel kinase inhibitors are the next wave of
therapeutics in the oncology space
40 • Novel NME with strong patent protection
42. Partnership Highlight
IV Carbamazepine for Epilepsy
• I.V. carbamazepine is formulated with Ligand’s
Captisol
• Carbamazepine used in:
• Management of complex partial seizures
• Treatment of generalized tonic‐clonic seizures (the
most common type of generalized seizure)
• Adjunct in patients with secondary or partial epilepsy
• Captisol enabled the IV form of widely used oral
sodium channel blocker
• IV improvement in onset of action
• Ideal for hospital/emergency settings
• Currently in Phase 3 development by Lundbeck
• Estimated primary completion date December 2012
42
