Ligand Pharmaceuticals Incorporated · NASDAQ:LGND

Ligand Pharmaceuticals — Investor Day (December 2012)

88 slides from Ligand Pharmaceuticals's investor day presentation, Dec 4, 2012

  1. Slide 1: 2013 and Beyond Analyst Day

    2013 and Beyond Analyst Day New York, NY December 4, 2012 NASDAQ: LGND

  2. Slide 2: Safe Harbor Statement

    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

  3. Slide 3: Ligand... 2013 and Beyond

    3 Ligand . . . 2013 and Beyond • Today Ligand is at a major corporate inflection point • Company projected to produce significant growth of • Revenue • Profitability • Operating Cash Flow • Our “Shots-on-Goal” business model is projected to continue to drive the business for years to come

  4. Slide 4: Agenda

    4 1) General Overview John Higgins • Business Model • Major Inflection Point • Growth Drivers 2) Financial Overview and Outlook John Sharp 3) R&D Model and Captisol Matt Foehr 4) Antiplatelet Market Simona Skerjanec and MDCO-157 The Medicines Company 5) “Shots-on-Goal” and Select Nishan de Silva Por

  5. Slide 5: "Shots-on-Goal" Business Model

    “Shots-on-Goal” Business Model

  6. Slide 6: Biotech Performance and Value Creation

    6 Biotech Performance and Value Creation Perceived Value Positive Data NDA Actions Product Launch Real Value Sustainable Cash Flow Positive events might impact a stock price, but a company has not created real value unless it generates sustainable cash flow Event Value Created Ligand’s Top Priority

  7. Slide 7: Ligand: A Unique Biotech Investment Company

    7 Strong Financials Diversified Risk Robust Growth Engine Ligand: A Unique Biotech Investment Company Projects Above-Average Growth, Below-Average Risk

  8. Slide 8: "Shots-on-Goal" Business Model

    8 • Most programs fail in biotech/pharma R&D • BUT, not all • Ligand’s business model is premised on these basic principals — The more programs in development the more likely you will have success — A company does not need to retain full program rights to generate significant revenue and cash flow “

  9. Slide 9: "Shots-on-Goal" Business Model

    9 • Ligand has two primary business objectives: — Drive R&D to earliest inflection point for partnering — Acquire assets efficiently to further build portfolio • The RESULT: — High-margin revenue — Lower costs — Portfolio diversity “Shots-on-Goal” Business Model

  10. Slide 10: Bold Expansion of Partnered Portfolio

    10 Six years ago, Ligand had 9 programs Today, Ligand has 70 Partnered Programs Bold Expansion of Partnered Portfolio

  11. Slide 11: Business Model: Focused 22-Person Team

    11 Business Model: Focused 22-Person Team Business Development Investment Banking Private Equity Venture Capital Health Care Management Consulting Big Pharma Biotech Research Chemistry Biology Pharmacology Formulation Sciences Operations Supply Chain Technical Operations and Quality Administration L

  12. Slide 12: Business Model: Core Competencies

    12 1) Hunting for new drugs – research 2) Licensing – finding, creating and managing deals 3) Acquiring and integrating assets/companies 4) Serving partners and customers for drug formulations Business Model: Core Competencies

  13. Slide 13: Major Inflection Point

    Major Inflection Point Ligand

  14. Slide 14: Ligand... 2013 and Beyond

    14 Ligand is uniquely positioned to generate significant cash flow per share – High-quality revenue – High gross margins – Low operating costs – Significant tax assets – Low share count Ligand . . . 2013 and Beyond

  15. Slide 15: Strong Financials

    Strong Financials Revenue: 4 major drivers of revenue Expenses: Low cost structure. Revenue expected to grow even without additional investments Tax Assets: More than half a billion dollars Share Count: 20 million Given major positive developments for revenue drivers, low costs and significant tax a

  16. Slide 16: Promacta: Accelerating Royalty Revenue

    $ m ill io n s Promacta: Accelerating Royalty Revenue 16 $2.5 $2.0 $1.5 $1.0 $0.5 $0 Q1 Q2 Q3 Q4 Q1 Q2 Q3 Q4 Q1 Q2 Q3 Q4 Q1 Q2 Q3 Q4 2009 2010 2011 2012 Ligand royalties based on prior quarter product sales Q4 2012 royalty to be reported in Ligand’s year-end results $3.0 $3.5 Royalty revenue acceler

  17. Slide 17: Promacta Projections: GSK Sell-Side Analysts

    17 $ m ill io n s Revenue Projections … …Yield these Royalty Projections $ m ill io n s High Low Average Sales figures converted from GBP to USD Conversion rate = 1.6 Values calculated on a one quarter lag 5 GSK covering analysts reports as of 11/9/12 Promacta Projections: GSK Sell-Side Analysts

  18. Slide 18: Kyprolis Projections: ONYX Sell-Side Analysts

    Kyprolis Projections: ONYX Sell-Side Analysts 18 11 ONYX covering analysts reports as of 11/9/12 Values calculated on a one quarter lag Revenue Projections … …Yield these Royalty Projections $ bi lli o n s $ bi lli o n s High Low Average

  19. Slide 19: Major Inflection Point: Projected Royalties

    $0 $10 $20 $30 $40 $50 $60 $70 $80 $90 $100 2010 2011 2012 2013 2014 2015 2016 2017 Other Conbriza Nexterone Avinza Kyprolis Promacta Actual Projections 19 *One quarter lag Promacta: 5 GSK covering analysts reports as of 11/9/12 Kyprolis: 11 ONYX covering analysts reports as of 11/9/12 $ m ill io n

  20. Slide 20: Two-Year Financial Outlook and Projections

    20 • 40% annual revenue growth projected for next two years — 2013: $41 million to $44 million — 2014: > $60 million • 5% annual expense growth — Annual operating expenses $26 million to $28 million • 200% earnings per share growth 2014 over 2013 — 2013 > $0.35 — 2014 > $1.05 • Pay down debt and bui

  21. Slide 21: What Does Ligand Plan To Do with Its Excess Cash?

    21 1) Acquire assets or companies to continue building fully-funded “Shots-on-Goal” portfolio or 2) Return capital to shareholders through share repurchases and/or dividends What Does Ligand Plan To Do with Its Excess Cash?

  22. Slide 22: Growth Drivers

    Growth Drivers Ligand

  23. Slide 23: Significant Growth Potential from New Products

    23 • 70 partnered programs • Realistically, not all will make it to market considering standard biotech success rates • But, given current program status, here is how new products could enter the market through 2020 Significant Growth Potential from New Products Year 2013 2014 2015 2016 2017 2018 20

  24. Slide 24: Significant Growth Potential from New Products

    24 • Clear evidence the “Shots-on-Goal” model is working — Multiple late-stage events over past 12 months with new products advancing to market — Continued further expansion of “Shots-on-Goal” portfolio Significant Growth Potential from New Products

  25. Slide 25: Financial Overview and Outlook

    Financial Overview and Outlook John Sharp Vice President, Finance and Chief Financial Officer

  26. Slide 26: Efficient Operating Costs

    Efficient Operating Costs 26 $ m ill io n s Combined R&D and G&A Do not need to increase costs to grow revenue Reduced headcount and shut down of non-core facility

  27. Slide 27: 2012 Cost Structure

    2012 Cost Structure 27 • Annual costs split into four main categories with one-fourth of expenses non-cash • Efficient cost structure that supports activities to further expand the “Shots-on-Goal” portfolio Fixed costs Facilities, insurance, taxes and public company expenses Personnel costs Variable

  28. Slide 28: Tax Assets

    Tax Assets • Ligand has three types of tax assets — Net operating loss carryforwards (NOLs) — Tax credits — Future tax deductions 28

  29. Slide 29: Tax Assets: NOLs

    Tax Assets: NOLs Net operating loss carryforwards (NOLs) • Prior-year tax losses that may be carried forward to offset future taxable income • $480 million of federal NOLs (~$435 million unrestricted) • $140 million of state NOLs 29

  30. Slide 30: Tax Assets: Credits

    Tax Assets: Credits Tax Credits • Credits for R&D expenses generated in prior years can be used to offset future tax payments due • $19.5 million of federal R&D tax credits • $17.5 million of state R&D tax credits 30

  31. Slide 31: Tax Assets: Future Tax Deductions

    Tax Assets Future tax deductions • Deductions already recorded for book purposes but will be deductible for tax purposes in the future • Significant amount relates to R&D expenses capitalized in prior years and will be amortized as expensed over the subsequent 5 to 10 years • $90 million of future f

  32. Slide 32: Summary of Tax Assets

    Summary of Tax Assets • NOLs: — $480 million of federal NOLs (~$435 million unrestricted) — $140 million of state NOLs • Tax credits: — $19.5 million of federal R&D tax credits — $17.5 million of state R&D tax credits • Future deductions: — $90 million of future federal NOLs (capitalized R&D) 32 Bas

  33. Slide 33: Projected Tax Expense

    Projected Tax Expense • Ligand currently has a full “valuation allowance” against its deferred $190 million tax assets – Until “valuation allowance” is released, annual book tax expense is equal to $0.5 million plus actual taxes paid • Upon reaching consistent, multi-year profitability, Ligand will

  34. Slide 34: Projected Revenue Composition

    Projected Revenue Composition 34 $30-$31 $41-$44 > $60 By 2014, royalties projected to make up > 70% of total revenues $ m ill io n s

  35. Slide 35: 2013-2014 Revenue Buildup

    2013 – 2014 Revenue Buildup • Royalty revenue: — Based on sell-side analyst projections for Promacta and Kyprolis • Captisol sales: — Projected to be between $13 and $16 million per year • License/milestones: — Projected to be between $3 and $5 million per year based on potential milestones for exis

  36. Slide 36: Gross Margins

    Gross Margins • Royalties – 100% gross margin • License and Milestone payments – 100% gross margin • Captisol Material Sales – Projected 60% gross margin for 2013 and 2014 — Cost of goods % dependent on type of sale — Clinical: ~80% gross margin — Commercial: ~50% gross margin • For 2013 and 2014, m

  37. Slide 37: 2012 Financial Guidance

    2012 Financial Guidance 37 ($ in millions) Revenue Royalties 4.7$ - 4.9$ 14.0$ - 14.2$ Material sales 5.2 - 5.8 9.4 - 10.0 License and other 2.3 - 2.5 6.6 - 6.8 12.2$ - 13.2$ 30.0$ - 31.0$ Cost of goods sold 40% 35% Combined R&D and G&A 5.9$ - 6.4$ 26.0$ - 26.5$ EPS - Continuining operations 0.14$ -

  38. Slide 38: Ligand's R&D Model

    Ligand’s R&D Model Matt Foehr Executive Vice President and Chief Operating Officer

  39. Slide 39: Ligand's R&D Model

    39 • Steady, thoughtful and selective R&D investment • Partnering-driven, diversified, backed by strong science ― Drive R&D to earliest inflection point for partnering • Focus on de-risking, through internal or external milestones, with a goal of partnering ― Leverage ripening science and landscape

  40. Slide 40: Ligand's Unpartnered Assets

    Program Indication Pre-clinical Phase l Phase Il Captisol-enabled Melphalan SARM Glucagon Recept Antagonist Oral GCSF ASCT HepDirect® Nuc Delivery HCV Captisol-enabled Topiramate Seizure Muscle Wasting Diabetes Neutropenia Captisol-enabled Lamotrigene Seizure Additional programs covering Pain, Oncol

  41. Slide 41: LGD-6972: Glucagon Receptor Antagonist

    • Obtained through Metabasis acquisition ― Differentiated novel agent with strong IP position ― Highly potent and selective ― Proven effective in multiple animal models ― Robust PK, predicting once-daily oral dosing ― IND-enabling safety studies completed to support IND • Glucagon receptor antagonis

  42. Slide 42: A Platform for Continued Growth

    A Platform for Continued Growth

  43. Slide 43: Platform for Continued Growth

    43 • Meets enduring and growing industry need ― Formulation solubility and stability • The right technology at the right time ― Combinatorial chemistry and high-throughput screening advances greatly increased the number of promising compounds that have solubility issues ― High quality manufacture an

  44. Slide 44: Increasing Needs

    10,000 8,000 6,000 4,000 2,000 0 1980 1985 1990 1995 2000 2005 2010 Year Nu m b e r of p u bl ic at ion s Solubility publications High-throughput screening publications Introduction of HT screening in the mid 1990's accelerated drug discovery and the need for proven solubility technologies Increasin

  45. Slide 45: Platform for Continued Growth

    45 • Makes major drugs possible ― Kyprolis™, V-FEND i.v. • Makes existing drugs better ― Clopidogrel i.v. (MDCO-157), Nexterone®, Melphalan Platform for Continued Growth

  46. Slide 46: Platform for Continued Growth

    46 • Paying us today ― Captisol royalties positioned for growth • Existing deals already in partner pipelines ― Over 30 Captisol “Shots-on-Goal” • New deals and customers being added ― Anticipated source of future deals Platform for Continued Growth

  47. Slide 47: Multiple Ways to Create Value

    47 Single NCE Multiple compounds Oral-I.V. switch, lifecycle management, with clinical data Partner cannot formulate candidate Partner has pipeline of insoluble compounds Partner needs late-stage product with clear development path License fees Milestones Royalties • Illustrative deal structures. Mu

  48. Slide 48: Kyprolis: Case Study

    • Use of Captisol – Active ingredient is more soluble, better absorption – Less active used, better efficacy/safety profile, ultimately lower drug cost 48 • Captisol is critical to the Kyprolis formulation – Solubility issues led to unacceptably high drug loads during early development – Captisol so

  49. Slide 49: Illustrative Proportional Quantity: Captisol-to-API

    49 Illustrative Proportional Quantity: Captisol-to-API in a finished drug dose 60 mg 3,000 mg Captisol API

  50. Slide 50: Kyprolis: Case Study

    50 • Q3 2012 Commercial Performance – Early launch performance exceeded analyst expectations, $18.6 million in first partial quarter – Onyx beginning to “unlock the significant potential for Kyprolis” • Ligand’s Royalty Up to, and including $250 million $251 million to $500 million $501 million to $

  51. Slide 51: Kyprolis Revenue: ONYX Analyst Projections

    51 Kyprolis Revenue: ONYX Analyst Projections $ b ill io n s High Low Average 11 ONYX covering analysts reports as of 11/9/12

  52. Slide 52: Lilly: Case Study

    52 • Executed Platform license deal in Q4 2011 • Lilly permitted to nominate an unlimited number of Captisol-enabled candidates from their internal pipeline – $1 million up-front – Milestones and royalties undisclosed • Significant player putting major resource behind Captisol- enabled programs Lill

  53. Slide 53: MDCO-157: Case Study

    53 • Ligand licensed worldwide exclusive rights to The Medicines Company for Captisol-enabled i.v. Clopidogrel (active ingredient in Plavix®) • Novel I.V. form - onset of action, important in critical care setting • Ligand completed CMC and early clinical PK studies, enabling higher economics in lic

  54. Slide 54: Value growth in hospital acute intensive medicine

    & Value growth in hospital acute intensive medicine Simona Skerjanec, VP, Global Competitive Strategy December 4, 2012

  55. Slide 55: Legal notices

    THE MEDICINES COMPANY Acute | Intensive Care Medicine NASDAQ: MDCO Slide 55 Legal notices Forward looking statements Statements contained in these slides about The Medicines Company (the “Company”), the Company's products and product candidates, the timing of clinical trial results, regulatory submi

  56. Slide 56: The Medicines Company: Acute Intensive Care Hospital Focus

    THE MEDICINES COMPANY Acute | Intensive Care Medicine NASDAQ: MDCO Slide 56 The Medicines Company Acute | intensive care hospital focus To save lives, alleviate suffering and improve the economic efficiency of the ~3,000 leading acute | intensive care hospitals in the world Purpose Lead in customer

  57. Slide 57: Large acute care market

    THE MEDICINES COMPANY Acute | Intensive Care Medicine NASDAQ: MDCO Slide 57 Large acute care market Worldwide, 68 million deaths projected in 2030 World Health Statistics 2008. WHO: http://www.who.int/whosis/whostat/EN_WHS08_Full.pdf Top-5 causes Deaths (M) (%) Heart attack 9.6 (14.2) Stroke 8.2 (12

  58. Slide 58: Growing demand

    THE MEDICINES COMPANY Acute | Intensive Care Medicine NASDAQ: MDCO Slide 58 Growing demand Lifestyle changes are driving a shift in geography Murray CJL, Lopez AD, eds. The Global Burden of Disease: Boston, Mass: Harvard School of Public Health; 1996 1990 2020 India N. America | W. Europe Middle Eas

  59. Slide 59: Angiomax: Market leading solution

    THE MEDICINES COMPANY Acute | Intensive Care Medicine NASDAQ: MDCO Slide 59 ~5m patients 35k lives saved 50k transfusions 5k hospital years $2.5b+ savings Angiomax® (bivalirudin) Market leading solution: PCI anticoagulation Sources: Rassen et al EJC | Cohen et al JACC | Pinto et al. JACC | Kessler e

  60. Slide 60: Indication and Important Safety Information

    THE MEDICINES COMPANY Acute | Intensive Care Medicine NASDAQ: MDCO Slide 60 Indication and Important Safety Information Angiomax® (bivalirudin) for Injection is a direct thrombin inhibitor indicated for use as an anticoagulant in patients undergoing percutaneous coronary intervention (PCI) with prov

  61. Slide 61: Large acute care pipeline

    THE MEDICINES COMPANY Acute | Intensive Care Medicine NASDAQ: MDCO Slide 61 Pipeline Large acute care pipeline Compound Current Proposed patient population Oritavancin Ph. III Acute infections MDCO-157 505(b)2 ACS/PCI Cangrelor Ph. III ACS/PCI/bridge to surgery MDCO-216 Ph. I ACS

  62. Slide 62: MDCO-157 Captisol-enabled IV clopidogrel

    THE MEDICINES COMPANY Acute | Intensive Care Medicine NASDAQ: MDCO Slide 62 MDCO-157* Captisol-enabled® IV clopidogrel • Potential indication IV option in situations where administration of oral platelet inhibitors is not feasible or desirable • Regulatory pathway First patient enrolled dose respons

  63. Slide 63: Oral agent challenges in ACS

    THE MEDICINES COMPANY Acute | Intensive Care Medicine NASDAQ: MDCO Slide 63 Oral agent challenges in ACS Patient Type Prevalence ( % ACS) Reference Pre-hospital resuscitation 8.7% PREMIER Investigators AJC(2012) International Journal of Cardiology (2008) Intubated on admission 4.0% PREMIER Investiga

  64. Slide 64: Oral clopidogrel in AMI

    THE MEDICINES COMPANY Acute | Intensive Care Medicine NASDAQ: MDCO Slide 64 Oral clopidogrel in AMI Comparison of changes in platelet aggregation induced by 5 umol/l (A) between STEMI patients (n=9) and health controls (n=10) after administration of a 600 mg clopidogrel loading dose. ( Haastermans,

  65. Slide 65: Oral clopidogrel in PCI patient

    THE MEDICINES COMPANY Acute | Intensive Care Medicine NASDAQ: MDCO Slide 65 Oral clopidogrel in PCI patient

  66. Slide 66: MDCO-157 Captisol-enabled IV clopidogrel

    THE MEDICINES COMPANY Acute | Intensive Care Medicine NASDAQ: MDCO Slide 66 MDCO-157* Captisol-enabled® IV clopidogrel • Significant unmet need Bioavailability of oral agents in acute ACS is not ideal • Opportunity The Medicines Company has the right vision, know how and global customer reach to dev

  67. Slide 67: "Shots-on-Goal" Business Model and Select Portfolio Assets

    “Shots-on-Goal” Business Model and Select Portfolio Assets Nishan de Silva, MD Vice President Corporate Development

  68. Slide 68: Ligand's Business Model: "Shots-on-Goal"

    • Most drugs fail, but not all • Large and diverse portfolio of opportunities maximizes odds of business success • Ligand has a robust portfolio of partnered programs Ligand’s Business Model: “Shots-on-Goal” 68

  69. Slide 69: Ligand's Portfolio: "Shots-on-Goal"

    11% 5% 27%41% 15% 70 Partnered Programs 69 Preclinical Marketed NDA/ Phase 3 Phase 2 Phase 1 Key programs include: • Promacta (GSK) • Kyprolis (Onyx) • Aprela (Pfizer) • Dinaciclib (Merck) • BACE (Merck) • Clopidogrel-IV (MDCO) • Carbamazepine- IV (Lundbeck) Ligand’s Portfolio: 70 “Shots-on-Goal”

  70. Slide 70: Promacta

    70 • Marketed by GSK for ITP since late 2008 • Recently approved for chronic hepatitis C • Long patent protection through 2027 • Potential for major label expansion- over 25 active clinical trials • Significant royalty rates: — Less than $100 million: 4.7% — $100 to $200 million: 6.6% — $200 to $400

  71. Slide 71: Promacta/Revolade Approved Countries

    71 Global brand with significant momentum Approved in 91 countries Promacta/Revolade

  72. Slide 72: Promacta: Regional Quarterly Revenue

    $ m ill ion s *Sales figures converted from GBP to USD Note: “EMAP” = Emerging Markets Asia Pacific 72 Continued strong growth across geographies 2011 2012 % Growth YoY Prior Q ROW 50% 33% EMAP 150% 0% EU 56% 17% US 50% 14% TOTAL 62%* 15%* Promacta: Regional Quarterly Revenue

  73. Slide 73: Promacta Launch in HCV

    • Approved November for “the treatment of thrombocytopenia in patients with chronic hepatitis C to allow initiation and maintenance of interferon-based therapy” • GSK has commercial and scientific presence focused on hepatologists and infectious disease specialists • GSK focused on launch excellence

  74. Slide 74: Promacta and HCV "All Oral" Landscape

    • Major industry investment to achieve interferon-free regimen • However, significant opportunities remain for Promacta — Interferon off patent in coming years, likely to become low-cost therapy and remain standard-of-care in larger emerging markets — Drugs in development not equally effective again

  75. Slide 75: Promacta: HCV Opportunity

    75 150+ million • 100,000 patients represents only ~2% of worldwide HCV patients with low platelets • Feasible for Promacta to capture 100,00 patients worldwide over 3-5 years after launch — U.S. alone has 147,000 eligible patients (per GSK) • Represents estimated $2 billion total revenue opportunit

  76. Slide 76: Promacta: Potential Label Expansion

    76 ITP Oncology-Related Thrombocytopenias • MDS • AML • CIT Others Chronic hepatitis C Approved 2008 Approved 2012 Future Promacta: Potential Label Expansion

  77. Slide 77: Promacta: Development in MDS

    • MDS patients develop severe cytopenia, require frequent transfusions • Excess bleeding results in major complications or death for nearly 25% of patients* • ~17,000 MDS patients in the U.S. • Data from Phase 2 placebo-controlled study to be presented at ASH 77 * Expert Opinion: Thrombocytopenia &

  78. Slide 78: Promacta: Development in AML

    • AML is fast-growing cancer • Abnormal red blood cells and platelets can quickly crowd out normal cells • Pre-clinically, Promacta inhibits leukemia cell growth • ~14,000 AML patients in the U.S. • Data from Phase 1 dose-escalation study to be presented at ASH 78 Promacta: Development in AML

  79. Slide 79: Promacta: Aplastic Anemia

    • Rare disease where bone marrow does not produce enough new cells • Patients have low red cells, white cells and platelets • Promacta data published in New England Journal of Medicine July 5, 2012 suggest potential use beyond supportive care — NIH–sponsored Phase 2 study — Patients refractory to im

  80. Slide 80: Highlights of Select Portfolio Assets

    Highlights of Select Portfolio Assets

  81. Slide 81: BACE: Alzheimer's Opportunity

    BACE: Alzheimer’s Opportunity 81 • Elevated beta amyloid found in Alzheimer’s patients’ brains • BACE inhibitors reduce beta amyloid levels • Merck reported single doses associated with up to 92% mean reduction in beta amyloid levels from baseline • Merck’s remarks from Q3 2012 call: “We are also ex

  82. Slide 82: Dinaciclib: Late-Stage Oncology Asset

    • Cyclin dependent kinase (CDK) inhibitor • Merck recently initiated a Phase 2b/3 adaptive study in refractory Chronic Lymphocytic Leukemia (CLL) • Novel molecule with patent protection • Ligand entitled to milestones and royalties Dinaciclib: Late-Stage Oncology Asset CDK Control of the Cell Cycle

  83. Slide 83: Aprela: Late-Stage Women's Health Asset

    Aprela: Late-Stage Women's Health Asset 83 • Once-daily, oral, tissue-selective estrogen complex containing bazedoxifene (SERM) and conjugated estrogens (Premarin®) • Bazedoxifene discovered by Ligand, licensed to Wyeth (now Pfizer) in 1990s • Targeted Indication: Treatment of symptoms associated wi

  84. Slide 84: Captisol-Enabled Melphalan Opportunity

    • Ligand advancing development of proprietary program for stem cell transplant conditioning • Program integrates Captisol into an existing product to create new, high-value transplant offering • Low-risk, late-stage opportunity • Rare combination of 505(b)(2) pathway and Orphan designation • Evaluat

  85. Slide 85: Melphalan: Multiple Myeloma Overview

    Stem Cell Transplant Used Throughout Long Continuum of Multiple Myeloma Treatment Sources: NCCN guidelines, UpToDate, Physician interviews, Survey results (1) Other conditioning regimens include irradiation, fludarabine, cyclophosphamide, and busulfan M y e lo ma B u rd e n Frontline Therapy 2nd and

  86. Slide 86: Multiple Myeloma Stem Cell Transplants Will Continue to Grow

    • Multiple myeloma incidence growing 1.7% annually in U.S. • Stem cell transplants growing at nearly double that rate • Current IV Melphalan market $130 million, with predominant use in stem cell transplants  IV Melphalan market well- positioned for future growth 5.2K 5.3K 5.4K 5.6K 5.7K 5.8K 1.9K

  87. Slide 87: Melphalan: The Next Generation

    87 Melphalan: The Next Generation Alkeran® (Reference Listed Drug) includes Propylene Glycol Captisol-enabled® Melphalan, Propylene Glycol-Free (LGD-353) • 2 vial system • 60 min infusion (stability) • 1 vial system • Up to 24 hour infusion • Potential for higher dosing over longer duration Product

  88. Slide 88: Potential News and Catalysts

    88 Potential News and Catalysts Milestone Partner Ligand Estimated Timing 2 0 1 2 Melphalan pivotal trial initiation Unpartnered December Promacta HCV commercial launch GSK December Promacta data in MDS, AML at ASH GSK December 2 0 1 3 Oncology Phase 2 initiation MEI Pharma 1Q CE-Carbamazapine Phase

Ligand Pharmaceuticals — Investor Day (December 2012) on 6ix