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NEA_Menlo_Park

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European headquarters located in Cambridge, UK. 9 EU country subsidiaries. General Managers ... DuPont, Maxim. Bill Poncy, VP US Sales & Mktg. Aventis. Terry ... – PowerPoint PPT presentation

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Title: NEA_Menlo_Park


1
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2
Pharmion Overview
  • Global Specialty Pharmaceutical Company
  • Focused on Hematology and Oncology
  • Licensed Four Products to Date
  • Commercial, Regulatory and Development
    Capabilities
  • Innovative Products with High Therapeutic Impact

3
Global Leverage
  • Pharmion Organization US, EU and Australia
  • Distributor network in 30 additional countries
  • Increases product licensing opportunities
  • Allows for regional and global partnerships
  • Global branding
  • Consistent regulatory strategy
  • Consistent product label
  • Consistent pricing
  • Consistent product positioning
  • Highly economic for hematology/oncology products

4
Pharmion Organization
  • 130 employees
  • Regulatory Affairs
  • Medical Marketing
  • Sales and Marketing
  • Commercial Development
  • Information Technology

5
Initial Focus Hematology
  • Highly responsive to scientific marketing and
    education programs
  • Well developed global network
  • Focused target audience
  • Opinion leaders
  • Community professionals
  • Limited attention from big pharma
  • Platform to enter broader oncology market

6
Pipeline Overview
  • Phase of Product Indication
    Development Licensor Territory
  • Thalomid Relapsed/refractory
    Registration Celgene EU selected multiple
    myeloma ROW ENL territories
  • Azacitidine Myelodysplastic Preregistration Phar
    macia Global syndrome
  • Refludan Heparin induced Marketed Schering
    AG EU/ROW thrombocytopenia
  • Innohep Treatment of Marketed LEO
    Pharma US DVT with or A/S without PE

7
Thalomid
  • Licensed from Celgene for all markets outside
    North America, Japan, China, Korea and Taiwan
  • License fees and royalties to Celgene
  • Validated sales potential 82M in 2001 US sales
  • Predominant use in Multiple Myeloma
  • Potential in multiple tumor types
  • Licensed S.T.E.P.S. safety program

8
Thalomid Regulatory Status
  • Licensed Thalomid from Celgene November 2001
  • Submitted MAA to EMEA March 2002
  • Seeking two indications
  • ENL
  • Relapsed/refractory multiple myeloma
  • Orphan drug designations provide 10-year
    exclusivity
  • Approval for initial indication expected 2003

9
Thalomid in Multiple Myeloma (MM)
  • Relapsed/refractory MM
  • Barlogie University of Arkansas study
  • 31 of patients responded (? 50 ? in M-protein)
  • 47 overall survival rate at 2 years
  • Initial therapy MM
  • Phase II Mayo Clinic study
  • Thalidomide dexamethasone, 50 patients 64
    response (? 50 ? in M-protein)
  • Phase III Celgene sponsored study
  • 440 patients
  • Thalidomide dexamethasone VS placebo
    dexamethasone

10
THALOMID US Oncology Usage
  • Solid Tumors 26
  • Renal cell 6
  • Brain 4
  • Melanoma 3
  • Colorectal 2
  • Prostate 2
  • Bladder 1
  • Liver 1
  • Lung 1
  • Other 6

Hematologic Malignancies 74 Multiple myeloma
67 MDS 5 Other 2
Solid Tumor
Hematologic
SOURCE S.T.E.P.S.
11
Key Elements of S.T.E.P.S.(System for Thalomid
Education and Prescribing Safety)
  • Prescriber, pharmacy, and patient registration
  • Patient education and informed consent
  • Pregnancy testing and contraception
  • Monthly surveys for prescribers and patients
  • Prospective interventions by Company to prevent
    inappropriate use
  • Company authorization for dispensing each
    prescription
  • No automatic refills or phone orders
  • Controlled pharmacy reorders and patient refills

12
Azacitidine
  • Licensed global rights from Pharmacia in June
    2001
  • Royalty-based acquisition
  • One-time option to convert to 50/50
    WWco-promotion relationship
  • Initial indication sought Myelodysplastic
    Syndromes
  • Phase III study completed and published
  • Unique Mechanism of Action Demethylation
  • Potential in other tumor types

13
Azacitidine Regulatory Status
  • Licensed Azacitidine from Pharmacia June 2001
  • Pre-NDA meeting held Q4 2002
  • NDA submission on schedule for H1 2003
  • EMEA submission on schedule for H2 2003
  • Seeking first-line MDS indication
  • Confirmatory Phase III study to begin Q1 2003
  • Orphan drug designations provide 7-year (US) and
    10-year (EU) exclusivity

14
Myelodysplastic Syndromes (MDS)
  • Bone marrow disorder characterized by immature
    blood cells with abnormal function
  • Can effect red blood cells, white blood cells,
    and platelets
  • Mortality from bleeding and/or infection
  • Transformation to acute leukemia
  • Incidence in US and Europe 35,000 patients
  • Currently no approved treatment
  • Azacitidine could be first approved treatment for
    MDS

15
Azacitidine Phase III Results
  • Azacitidine statistically superior to supportive
    care for
  • Response 60 of pts (7 CR, 16 PR, 37
    improved) on azacitidine vs 5 improved on
    supportive care (plt0.001)
  • Time to leukemic transformation or death 21
    months with azacitidine vs 13 months on
    supportive care (plt0.007)
  • Transformation to AML first event in 15 of
    azacitidine patients vs 38 with supportive care
    (plt0.001)
  • QOL Improvement (fatigue, shortness of breath,
    psychological state, etc.) (plt0.05)

Silverman et al, JCO, May 15 2002, 2429-2440
16
Azacitidine in MDS
  • The positive experience with azacitidinebrings
    new hopes to thousands of patientswith MDS and
    to investigators who dedicate their research to
    improving prognosis in this relatively common and
    lethal malignancy

- Hagop M. Kantarjian
JCO, May 15 2002, 2415-2416
17
Refludan
  • Licensed from Schering AG in June 2002 for all
    countries outside US and Canada
  • Upfront and milestone payments
  • Direct thrombin inhibitor for treatment of
    heparin-induced thrombocytopenia (HIT)
  • 2001 EU Sales 5M in Germany with no promotion
  • Pharmion launching throughout EU and ROW
  • Approved in 42 countries

18
Refludan Establishes EU/ROW Commercial
Infrastructure
  • European headquarters located in Cambridge, UK
  • 9 EU country subsidiaries
  • General Managers
  • Regulatory Specialists
  • Medical Directors
  • Sales reps
  • 15 EU sales reps in place
  • 40 EU sales reps planned for the end of 2003
  • Australia subsidiary and ROW distributor network
    for 30 countries in place
  • Refludan funds EU/ROW structure for Thalomid
    launch

19
Innohep
  • Licensed US rights from LEO Pharma in July 2002
  • Up-front payment and royalties
  • Low molecular weight heparin indicated for
    treatment of deep vein thrombosis (DVT)
  • US LMWH market is approximately 1.2 billion and
    growing
  • 2001 EU Innohep sales were 100 million
  • Pharmion re-launched Innohep in US October 2002

20
Innohep Product Positioning
  • Position as the treatment of choice for the
    hematology/oncology DVT patient
  • DVT second leading cause of death in cancer
    patients
  • Only LMWH with simple once a day dosing
  • Efficacy is supported by the only published
    double-blind DVT treatment trial compared to
    heparin
  • Unrestricted dosing in complicated patients
  • Weight-compromised
  • Renal Impairment
  • Elderly

21
U.S. Commercial Organization
  • 31 field-based professionals
  • Average oncology sales experience 13 years
  • Medical marketing team
  • Phase IIIb/IV clinical development team
  • Building relationships for azacitidine launch
  • Field sales organization of 60 professionals
    planned for azacitidine launch
  • Capacity available for additional products

22
Finances
  • 130 million raised to date
  • December 31, 2002 cash balance - 62 million
  • Current investors
  • NEA
  • Nomura
  • Bay City Capital
  • Proquest
  • Aberdare Ventures
  • Domain Associates
  • Versant Ventures
  • GE Pension Trust

23
2002 Accomplishments
  • Thalidomide European MAA submission
  • Refludan acquisition
  • Innohep acquisition
  • Established US commercial organization
  • 30 field force launching Innohep
  • Established European commercial organization
  • Launching Refludan in 15 countries
  • Completed 40 million financing

24
2003 Objectives
  • Thalidomide European approval and launch
  • Implement S.T.E.P.S. safety program
  • Additional ROW Thalidomide approvals
  • Azacitidine NDA and MAA submissions
  • 20-30 mm combined sales of Innohep and Refludan
  • Acquire at least one additional product

25
Summary
  • Global hematology/oncology company
  • Commercial, development and regulatory
    capabilities
  • Four products licensed over past 18 months
  • Innovative products responsive to scientific
    marketing
  • Creative product licensing and acquisition
    structures
  • Highly experienced management team

26
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27
Management Team
28
Global Sales and Commercialization
29
Product Portfolio
  • Building a product portfolio with a balancedrisk
    profile
  • Innovative products with high therapeutic impact
  • Thalomid
  • Azacitidine
  • Refludan
  • Innohep
  • Attractive economic terms
  • Common target audience

30
Thalomid in Other Indications
  • Metastatic renal cell carcinoma
  • 2 Phase II studies
  • 1st 16/25 - stable disease (SD) 14 alive at 1
    yr
  • 2nd 14/22 patients responded 2 PR 12 SD
  • Metastatic colorectal cancer in combination with
    irinotecan
  • Pilot trial 4/10 patients responded 2 CR, 2 PR
    with ? incidence of irinotecan-induced GI
    toxicity in all patients
  • Glioblastoma
  • Phase II trial 18/38 patients responded 2 PR,
    16 SD
  • Androgen independent prostate cancer
  • Phase II trial 27 of 63 patients had 40 ? in
    PSA levels
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