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Leukemia Lymphoma Society: An Invitation to Innovation

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Leukemia Lymphoma Society: An Invitation to Innovation - as presented at the Partnering for Cures meeting, December 2, 2009, New York

Leukemia Lymphoma Society: An Invitation to Innovation - as presented at the Partnering for Cures meeting, December 2, 2009, New York

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  • The following graph is a 5 year survival curve of newly diagnosed CML patients. On standard chemotherapy only 55% survive to the 5 yr mark. However on Gleevec, a targeted therapy, nearly 95% of patients survive. The shift in this graph represents lives saved. And, the Society supported Dr. Brian Druker and others as they developed this revolutionary new “targeted” drug, and continues to support further improvements ~ 20,000 patients diagnosed with chronic phase CML over this period Without Gleevec: expect ~ 4,700 deaths With Gleevec: ~ 700 deaths ~ 4,000 lives saved (and counting)
  • Discovery = find the root cause of the disease and a potential therapy Development = carrying out the studies required by Regulatory Agencies (FDA, Health Canada) in order to get a new therapy approved/ In addition to discovery and development drugs need to be commercialized in order to get to patients. This is in the hands of the biotech & pharmaceutical industries. LLS now has a comprehensive suite of research programs that covers the discovery (our research grant program) and development (the Therapy Acceleration Program) steps for finding new therapies and is partnering with the pharma industry to get new therapies to the health care providers.
  • An Academic Concierge Project: The testing of 5000 existing, FDA approved, generic drugs for activity against acute leukemia by an academic investigator receiving one of our grants led to the discovery that a drug used to treat toe nail fungus, CPX, was active in blocking the growth of leukemia cell lines, leukemia in animal (mouse) models and AML cells from patients. Our goal: test the drug in a clinical trial against acute leukemia Process: LLS has assembled a development team to create an oral formulation of the existing product and an application to Health Canada to open a clinical trial Status: The oral formulation is complete. A Clinical Trial Application has been submitted to Health Canada. We expect a positive review and to be able to open a Phase I clinical trial in September. We moved this project forward with remarkable speed: concept to trial in 8 months!
  • A high level review of the 400 active projects shows that LLS is supporting research across some of the most important areas for the development of targeted therapies, into fundamental cellular processes that regulate cell growth and survival and in virtually every therapeutic modality that applies to treatment of the blood cancers. Now lets focus on the 60 projects that are in the development stage.
  • The bars and numbers above each step indicate the number of projects at each step in the drug development pathway (small molecule) in the LLS project portfolio. As might be expected, given our large discovery investment and previous neglect of development, most projects are at the earliest steps of this pathway. Given our desire to impact patient outcomes we want to better realize and maximize the value of this pipeline and have instituted the Therapy Acceleration Program to do so.
  • Transcript

    • 1. An Invitation to Innovation! Louis J DeGennaro, PhD Executive Vice President Chief Scientific Officer The Leukemia & Lymphoma Society
    • 2. Anyone can get blood cancer In 2009: One million North Americans
    • 3. The Leukemia & Lymphoma Society
      • Mission: Cure leukemia, lymphoma and myeloma while improving the quality of life for patients and their families.
      • Advocacy-Patient Services-Research
      • LLS is largest private funder of blood cancer research. FY2009 Research spend = $72 million ($250 million committed to 400 active projects)
      • Our Vision: blood cancer is eradicated
    • 4. Goal & Strategy
      • Goal: significantly improve the standard of care
      • Multi-pronged strategy:
      • Raise & deploy capital
        • Fund discovery research in academia
        • Advance development-stage projects to Phase I
        • Alliances with pharma & biotech companies
      • Partner our pipeline
        • Alliances with pharma & biotech
        • Share risk and cost with partners
    • 5. Research Value Proposition: CML & Gleevec 5yr survival 55% Lives Saved 90%
    • 6. The Pathway to Therapies New Therapies! Research Grant Program Therapy Acceleration Program Discovery Development Commercialize 7 - 10 years ?
    • 7. Grant Portfolio Overview by Disease $68 MM; 400 active projects $21.2 MM (32.4)% $4.3 MM (6.6)% $8.5 MM (13.0%) $3.6 MM (5.4%) $28 MM (42.7%) Leukemia Lymphoma Myeloma Basic Biology Gen IM/SCT
    • 8. CPX Project - AML
      • Test a panel of 5000 FDA approved, generic drugs
      • CPX
        • kills AML cells in patient samples
      • Our Challenge:
      • Create an oral formulation – done
      • Regulatory approval for clinical trial – opened in October
      • Accelerate it – 9 months from concept to trial
      • Toe Nail Fungus Cream
    • 9. LLS Therapeutic Pipeline Discovery Preclinical Phase I Phase II Phase III Patients Lymphoma - PTLD Acute Leukemia - AML Lymphoma – CLL, FL Lymphoma - CLL Acute Leukemia Lymphoma AML Multiple Trials Novel Drug Delivery Multiple Project Analysis Repurposed drug novel indication Antisense Inhibitor Small Molecule Engineered Immunotherapy Vaccine
    • 10. Budget
      • FY09 Audited Financials
        • Revenue: $288 million
        • Expenses: $280 million
        • 76% ($213 million) deployed to mission
        • We are not a bank: we raise and deploy these dollars each year
    • 11. Why donate to, or partner with, LLS?
      • Proven research strategy
        • 400 cutting-edge research projects
        • A 50-project drug development pipeline
      • Industry-quality approach
        • Competitive intelligence
        • Project management expertise
        • Smart, non-dilutive capital
        • Access to Key Opinion Leaders
      • A sense of urgency
    • 12. An Invitation to Innovation! Louis J DeGennaro, PhD Executive Vice President Chief Scientific Officer The Leukemia & Lymphoma Society
    • 13. Understanding the unmet needs 27 86% 1,300 8,500 HL 70 60% Children 15% Adults 9,000 12,800 AML 104 69% 19,000 63,000 NHL 56 35% 10,600 20,500 MM 28 90% Children ~50% Adults 1,400 5,800 ALL 55 79% 4500 15,500 CLL 42 89% 500 5,000 CML Drugs in development 5 year overall survival rate Deaths per year New cases per year
    • 14. Significant Market Opportunity in Blood Cancers Global Sales by Indication (US$ Billions) Current Reported Sales Followed by Estimates for 2010 (US$ Billions) Per Espicom Business Intelligence 22.728 15.485 7.626 Total 1.265 .995 .493 ALL, MDS, Hodgkin’s 5.585 3.674 1.661 CML 3.814 2.943 .965 Multiple Myeloma .941 .262 .152 AML 5.538 3.937 2.695 NHL 5.585 3.674 1.661 CLL 2012 est. 2009 est. 2005 Indication 1.203 WW sales 4.646 WW sales 7 3.2 .265 US only 2.852 .102 2.6 Velcade Rituxan Sprycel Gleevec
    • 15. In The Historical Record
      • 1995: Stem Cell IP
      • > licensed to Athersys
      • 1996: “Hedgehog IP
      • > partnered with Genentech, compounds in trials
      • 1997: Uses of Inorganic Arsenicals
      • > marketed as Trisenox by Cephalon
      • 1998: Second generation Proteosome Inhibitor
      • > licensed to Proteolix , compound in Phase II, myeloma
      • 1998: Vertebrate Apoptosis Gene
      • > led to formation of Idun, acquired by Pfizer
    • 16. Portfolio by Target, Mechanism & Modality 0 10 20 30 40 50 60 70 80 Tyrosine Kinase Kinase (Not Tyrosine) Methyl/Acetyl Anti-sense Diagnostic/Biomarker Transcription Cell Cycle Cell Division Apoptosis Small Molecule Antibody Vaccine Immunotherapy Transplant Stem Cell Cell Processes Targeted Therapy Treatment Modality
    • 17. Portfolio Disease Detail HL NHL CLL, SLL General LY AML CML ALL OM General L Myeloma Gen IM/SCT Basic Biology Dollars Funded in Millions $40 $35 $25 $15 $20 $5 $10 $0 $30 44.9% 34.2% 15% 5.9% % $ Disease $5 $10 $15 $20 $25 $30 $35 $40
    • 18. Development Project Portfolio 21 2 1 2 2 3 Scale Up IND Prep Tox Form Dev PK & ADME Med Chem IND Sub In vivo POP 21 Clinical Trials 10 1 62 Pipeline projects 28 on Watch List 26 Grants in 2009 with potential to move into development ~350 Discovery Projects THERAPIES