Deutsche Bank 40th Annual Health Care Conference
Boston, MA May 6, 2015 NASDAQ: LGND
2
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, drug development program benefits may not be realized and there can be no assurance that Ligand will achieve its guidance in 2015 or thereafter or that third party research summarized herein is correct or complete. The forward-looking statements made in the presentation are subject to several risk factors, including, statements regarding intent, belief, or current expectations of Ligand, its internal and partnered programs, including Promacta™, Kyprolis®, and Duavee™, Ligand’s reliance on collaborative partners for milestone and royalty payments, royalty and other revenue projections based on third party research, regulatory hurdles facing Ligand's and partners’ product candidates, uncertainty regarding Ligand's and partners’ product development costs, the possibility that Ligand's and partners’ drug candidates might not be proved to be safe and efficacious and commercial performance of Ligand's and/or its partners’ products, risks related to Ligand’s internal controls, its compliance with regulations, accounting principles and public disclosure, and other risks and uncertainties described in its public filings with the Securities and Exchange Commission, available at www.sec.gov. Additional risks may apply to forward-looking statements made in this presentation. Readers are cautioned not to place undue reliance on these forward-looking statements, which reflect our good faith beliefs (or those of the indicated third parties) and speak only as of the date hereof. All forward-looking statements are qualified in their entirety by this cautionary statement, and Ligand undertakes no obligation to revise or update this presentation to reflect events or circumstances or update third party research numbers after the date hereof. This caution is made under the safe harbor provisions of Section 21E of the Securities Exchange Act of 1934. Our trademarks, trade names and service marks referenced herein include Ligand and Captisol. Each other trademark, trade name or service mark appearing in this presentation belongs to its owner. The process for reconciliation between non-GAAP financial numbers presented on slide 15, and the corresponding GAAP figures is explained in the footnotes on those slides and a full reconciliation can be found in our earnings press release dated, February 9, 2015.
Safe Harbor Statement
3
Ligand: 2015 and Beyond
• Ligand is a high-growth company with economic rights to some of the world’s most important medicines
• Largest portfolio ever and projected to continue to drive the business significantly
• Cutting-edge innovations with Captisol and LTP technology are making major drugs possible
• Well positioned for strong revenue and profitability growth
Ligand Fast Facts
4
Portfolio Size
Blockbusters
Catalysts
Outlook
Over 100 fully-funded programs
Currently 2: Promacta® and Kyprolis®
6 major programs highlighted
Over 20 revenue-generating products expected by 2020
Financials Revenue
Profits
Cash Flow
> 30% annualized growth projected
> 45% annualized growth projected
High due to low costs and low taxes
5
Recent Events are Transforming Ligand
Date Program Event
November ’14 Promacta EU submission - Severe Aplastic Anemia
December ’14 Duavive EU approval
December ’14 Promacta sNDA submission - Pediatric ITP
December ’14 CE-Melphalan NDA submission
January ‘15 Delafloxacin Phase 3 study - Positive interim results
January ’15 Sparsentan Orphan drug designation - Focal Segmental Glomerulosclerosis
January ’15 SAGE-547 Phase 1/2 study - Positive data update
January ’15 Kyprolis US and EU submissions - Relapsed Multiple Myeloma
February ’15 Lasofoxifene Sermonix licensing agreement
February ’15 Promacta EU submission - Pediatric ITP
March ’15 Kyprolis Phase 3 ENDEAVOR study - Positive results
March ’15 Promacta Promacta acquisition closed (GSK to Novartis)
March ’15 Kyprolis Priority FDA review – Relapsed Multiple Myeloma
April ‘15 SAGE-547 Phase 1/2 completion – Positive results
April ’15 SAGE-547 Phase 3 trial initiated
April ’15 IRAK-4 Positive preclinical data presented
0
2
4
6
8
2008 2014
6
Fully
-Fu
nd
ed P
rogr
ams
(“Sh
ots
-on
-Go
al”)
Ligand’s Portfolio Continues to Grow
100 +
0
20
40
60
80
100
2008 2014
9
Ligand’s Achievement: Portfolio Expansion
Partners’ Achievement: Products Generating Revenue for LGND
Excellent record as drug researcher, innovator and licensor
Our partners are doing their job getting new products to the market
1
7
Co
mm
erc
ial P
rod
uct
s G
ener
atin
g R
even
ue
for
Liga
nd
Technology and Novel R&D Drive Deal Making
Potential Launch
7
Our Platform Technologies
Our Novel R&D
LTP Technology™
Glucagon Receptor Antagonist Program for Diabetes Phase 1
Positive Phase 1a data showing robust effects after single dose Phase 1b study expected to complete in coming weeks
Change in fasting glucose(24 hr post dose)
Place
bo
2 mg
10 m
g
40 m
g
120 mg
240 mg
480 mg
-15
-10
-5
0
5
10
MeanSEM
F
astin
g G
luco
se (
mg
/dL
)
Change in fasting glucosediabetic subjects(24 hr post dose)
Place
bo
40 m
g
-80
-60
-40
-20
0
20
MeanSEM
F
astin
g G
luco
se (
mg
/dL
)
Solving solubility and stability challenges
Designed to selectively deliver broad range of pharmaceutical agents to the liver
Oral GCSF Preclinical Leveraging our technology and heritage in small molecule discovery
8
20 Products by 2020 Significant Expansion of Revenue Generating Assets Projected
• Over 20 commercial programs projected to be generating revenue for Ligand by the end of this decade
• Programs expected to come from existing portfolio; no new deals required to drive that expansion
2008 2014 2020Projected
1
7
> 20
Ligand’s Revenue Generating Assets
Biotech44%
Big Pharma34%
Generic12%
Spec Pharma10%
9
65 Different Partners
Select Big Pharma
Select Biotech Select Spec Pharma
Select Generic
Diverse Portfolio Among Drug Companies
• We estimate our partners will spend over $1.1 billion in 2015 on R&D to advance our programs
• More partnered programs and late-stage trials are pushing spending up over 30% higher than 2014
Fully-funded Partnerships Driving Growth
10
– 13 Phase 3 trials – 14 preclinical programs
– 38 Phase 2 trials – Manufacturing scale-up
– 58 Phase 1 trials – Regulatory filing fees
– 2 Phase 4 trials
• Continued strong financial performance
• Business model provides tremendous earnings leverage
— Growing total revenues
— Flat cash operating costs
— Significant estate of tax assets
• Accelerating revenue and earnings growth going forward
Financial Overview
11
Accelerating Projected Revenue Growth
$0
$30
$60
$90
$120
$150
2011 2012 2013 2014 2015 2016 2017
• Growth due to:
– New products launched
– Growth in existing brands
– Higher royalties
12
$ m
illio
ns
$0
$20
$40
$60
$80
$100
$120
$140
$160
2013 2014 2015 2016 2017
License and other
Material Sales
Royalties
Projected Revenue
$49.0
$64.5
$81-$83
$ m
illio
ns
13
>$146
>$107
14
Ligand’s Cash-Generating Power Becoming Increasingly Clear
$0
$30
$60
$90
$120
$150
2011 2012 2013 2014 2015 2016 2017
Revenue Cash Expenses
Actual Outlook
• Strong revenue growth
• Operating expense levels projected to remain similar the next few years
• Significant increase in cash-flow projected
$ M
illio
ns
Accelerating Projected Non-GAAP EPS Growth
$0.00
$0.50
$1.00
$1.50
$2.00
$2.50
$3.00
$3.50
$4.00
$4.50
2011 2012 2013 2014 2015 2016 2017
• Growth due to:
– Higher revenues
– High gross margins
– Low and flat expenses
– Lean share count
15 Note: Non-GAAP EPS excludes changes in contingent liabilities, mark-to-market adjustment for amounts owed to licensors, non-cash SBC expense and non-cash debt related costs
Promacta®
• Oral medicine that boosts platelets. Ligand owed royalties
• Long patent protection, Orange Book patent expiration in 2027
• Blockbuster commercial potential (>$1 billion) due to growing and large list of potential therapeutic indications
ITP HCV ORT Idiopathic
Thrombocytopenia
Thrombocytopenia Induced by Hepatitis C
Oncology Related
Thrombocytopenia
17
Promacta®: Blockbuster Commercial Potential
Aplastic Anemia
95 Countries
Recently filed in the EU Recent Pediatric ITP filings
53 Countries
Global filing and launch investment
Major clinical investment ongoing: MDS, AML, CLL, CIT,
others
AA Currently Approved Indications
Ongoing Development New Markets
3 Countries
Myelodysplastic Syndromes
(MDS)
Acute Myeloid Leukemia
(AML)
Chronic Lymphocytic Leukemia
(CLL)
Cancers of the Blood
Severe cytopenia, patients need frequent transfusions
Fast-progressing cancer of the blood
Slow-progressing cancer
Excess bleeding results in major complications or death for nearly
25% of patients1
Clinically, Promacta shown to increase platelets
and pre-clinically, inhibits leukemia growth
Clinical data in CLL indicates 80% response rate
in CLL-associated ITP, 55% overall response rate
Global Phase 3 studies
in progress
Abnormal red blood cells and platelets can quickly crowd out
normal cells
Focused in white blood cells
~19,000 new diagnoses
in US each year2
~14,500
new diagnoses in US each year2
~16,000 new diagnoses
in US each year2
1Expert Opinion: Thrombocytopenia & Myelodysplastic Syndrome medscape.org/viewarticle/565023 2 National Cancer Institute, SEER Cancer Review, 2012
18
Promacta®: Oncology-Related Thrombocytopenia
$ m
illio
ns
GSK reported quarterly sales. Figures converted from GBP to USD 1Growth calculations 2014 vs. 2013
19
2011
Promacta®: Regional Quarterly Revenue
$0.0
$20.0
$40.0
$60.0
$80.0
$100.0
Q1 Q2 Q3 Q4 Q1 Q2 Q3 Q4 Q1 Q2 Q3 Q4 Q1 Q2 Q3 Q4
ROW
EU
US
2012 2013 2014
ROW 26%
EU 8%
US 39%
TOTAL 25%
Expected strong growth of LGND royalties
— New territories being added — New indications being pursued — Higher royalties on higher sales
Strong year-over-year growth in all geographies1
70
85
GSK Novartis
1,300
8,000
GSK Novartis
$2.0
$11.7
GSK Novartis
GSK and Novartis: Business Unit Profiles1
Novartis has a superior oncology business, ~6 times larger than GSK’s; Promacta® transitioned to Novartis on March 1st
Oncology Employees Countries with Presence 2014 Oncology Revenue ($B)
1 GSK and Novartis company disclosures relating to revenue and business unit structures; GSK.com, Novartis.com
20
Promacta® Projections: NOVN Analysts Annual Revenue Projections
21
$0
$100
$200
$300
$400
$500
$600
$700
$800
$900
2016 2017 2018 2019 2020
$ M
illio
ns High
Low
Average
4 NOVN covering analyst reports as of 4/27/15
Kyprolis®
23
• Leading 3rd-line treatment for multiple myeloma (MM) in the US
— Viewed as best-in-class proteosome inhibitor
— 25% year-over-year growth in 2014
Kyprolis®
• Royalty rates of 1.5% to 3.0%
ASPIRE Phase 3 Data
Dr. Keith Stewart The Mayo Clinic
“We are observing an unprecedented duration of remission, without additional toxicity, in relapsed and heavily pretreated patients.”
• Amgen has submitted US and EU applications for relapsed MM
— Granted priority FDA review; July 26, 2015 PDUFA date — Also granted EU Accelerated Assessment
• Major investment by Amgen focused on further expansion of the label
— Front-Line MM: Phase 3 (CLARION) — Small-cell Lung Cancer: Phase 2
24
• Recent data and events have continued to demonstrate the significant potential of Kyprolis
Kyprolis®
• APSIRE Phase 3 trial demonstrated an unprecedented PFS (26.3 months) in relapsed MM
• ENDEAVOR Phase 3 trial demonstrated a DOUBLING of median PFS over Velcade® (18.7 vs. 9.4 months) in relapsed MM
• Significant unmet needs remain in MM, and the market is expected to double to over $13 billion by 2020
Reference AMGN March 2, 2015 corporate presentation
$0.0
$0.5
$1.0
$1.5
$2.0
$2.5
$3.0
2016 2017 2018 2019 2020
25
Kyprolis® Projections: AMGN Analysts Annual Revenue Projections
$ B
illio
ns High
Low
Average
16 AMGN covering analyst reports as of 4/27/15
The Big Six
• Certain portfolio assets stand above others, having the potential to add significantly to Ligand’s top and bottom line
• They do so as a result of a mixture of factors, including:
— Market size or therapy area addressed
— Upcoming potential milestone events
— Royalty rate or specifics of deal economics
• Major news catalysts expected over the next 6 to 24 months
• More potential programs could move into the Big Six
The Big Six: Major Pipeline Assets
27
Partner
Program (Therapy Area)
Stage
Royalty Rate
Potential Launch
Potential 2015 Events
CE- Melphalan (Oncology)
NDA
20% 2015 Approval
Delafloxacin IV (Infection)
Phase 3 Undisclosed 2016 Phase 3 data
SAGE-547 (Neurology)
Phase 2 Undisclosed 2017 Pivotal Initiation
Sparsentan (FSGS - Kidney Disease)
Phase 2 9% 2017 Enrollment Completion
MK-8931 (Alzheimer’s Disease)
Phase 3 Undisclosed 2018 Updates
IRAK-4 (Oncology)
Preclinical 6.0-9.5% 2019 Clinical Start
28
The Big Six: Major Pipeline Assets
Viking Therapeutics
VKTX: Company Overview
30
• San Diego-based biotech, developing novel, first-in-class or best-in-class drugs for metabolic disorders
— Clinical-stage programs with preliminary efficacy signals in humans
• VK5211: Entering Phase 2 development for hip fracture
— Non-steroidal selective androgen receptor modulator (SARM)
— Promising efficacy in both bone and muscle
— Phase 2 data expected 2Q16
• TR-β agonists: X-linked adrenoleukodystrophy (X-ALD)
— VK0214, VK2809: Novel, selective thyroid receptor-β agonists
— Strong scientific rationale for application in X-ALD, rare neurodegenerative disorder
— Preclinical work on-going
— Potential human POC data 1H16
— Additional opportunities in lipid disorders such as NASH and cholesterolemia
• Three additional programs targeting diabetes, anemia, lipid disorders
VKTX: Investment Overview
31
• Initial single-program partnership with Ligand in 2012
• Partnership expanded to five programs in 2014
• Viking IPO priced last week
— Equity milestone payment to Ligand initially valued at ~$27M, subject to adjustment on issuance of IPO over-allotment shares
— Ligand invested additional $9M in offering
• Benefits to Ligand
— Potential to receive significant future royalties through 5 additional Shots-on-Goal
— Equity stake in promising growth biotech
Ligand’s Novel R&D:
Glucagon Receptor Antagonist
• Novel, highly potent, oral GCGR antagonist for treatment of type 2 diabetes completed Phase 1a trial in mid-2014, and Phase 1b trial initiated late last year
— One of Ligand’s most promising un-partnered assets
— Potential best-in-class properties
• Diabetes market is expected to double to $60 billion by 20201
— Combo therapy highly prevalent and necessary to optimize management of disease
— Creates significant opportunity for novel treatment mechanisms
33
Novel R&D: Glucagon Receptor Antagonist LGD-6972 for Diabetes
1 Brinson Patrick report 12/3/12; SunTrust report 6/25/13
Existing Class
Product Profile GCGR Advantage
DPP-IV Inhibitors
Modest reduction of plasma glucose Higher glucose
reduction
GLP-1 Agonists
Only available as injectables Oral
SGLT-2 Inhibitors
Contraindicated for renally impaired patients Spares kidney
• Product profile and recent clinical data suggest significant market advantages for a safe, highly potent, oral GCGR antagonist as compared to existing classes of new mechanisms
34
Novel R&D: Glucagon Receptor Antagonist Advantages of Potent GCGR Antagonist
Novel R&D: Glucagon Receptor Antagonist Positive Phase 1a Clinical Data for LGD-6972
35
Change in fasting glucose(24 hr post dose)
Place
bo
2 mg
10 m
g
40 m
g
120 mg
240 mg
480 mg
-15
-10
-5
0
5
10
MeanSEM
F
astin
g G
luco
se (
mg
/dL
)
Change in fasting glucosediabetic subjects(24 hr post dose)
Place
bo
40 m
g
-80
-60
-40
-20
0
20
MeanSEM
F
astin
g G
luco
se (
mg
/dL
)
• Positive clinical data presented in 2014
• Excellent safety profile
• Dose-dependent decreases in fasting plasma glucose in normals
• Robust decreases in glucose in type 2 diabetics after just a single dose
• Recent scientific ripening of the field, glucagon receptor antagonism widely seen as one of the most promising novel approaches to treatment of diabetes
• Multi-dose trial currently in progress, data expected in coming weeks
36
Potential Upcoming Events
Target Date Program Event
Q2’15 Duavive EU product launch
Q2’15 LGD-6972 Phase 1b results
7/26/15 Kyprolis Multiple Myeloma sNDA PDUFA date
2H’15 Promacta Pediatric ITP NDA and MAA approvals
2H’15 Kyprolis Relapsed multiple myeloma NDA and MAA approvals
2H’15 NS-2 Phase 2 trial initiation
2H’15 Delafloxacin NDA Submission
Q4’15 Promacta Severe Aplastic Anemia MAA approval
Q4’15 CE-Melphalan NDA approval
Q4’15 Carbella NDA approval
Q4’15 Topiramate Phase 2 trial initiation
Q4’15 IRAK-4 Phase 1 trial initiation
Deutsche Bank 40th Annual Health Care Conference
Boston, MA May 6, 2015 NASDAQ: LGND