22
Pharmion Overview
• Global Specialty Pharmaceutical Company
• Focused on Hematology and Oncology
• Licensed Four Produc...
33
Global Leverage
• Pharmion Organization: US, EU and Australia
• Distributor network in 30 additional countries
• Increa...
44
Pharmion Organization
• 130 employees
• Regulatory Affairs
• Medical Marketing
• Sales and Marketing
• Commercial Devel...
55
Initial Focus: Hematology
• Highly responsive to scientific marketing and
education programs
• Well developed global ne...
66
Pipeline Overview
Phase of
Product Indication Development Licensor Territory
Thalomid®
• Relapsed/refractory Registrati...
77
Thalomid®
• Licensed from Celgene for all markets outside
North America, Japan, China, Korea and Taiwan
• License fees ...
88
Thalomid Regulatory Status
• Licensed Thalomid from Celgene November 2001
• Submitted MAA to EMEA March 2002
• Seeking ...
99
Thalomid in Multiple Myeloma (MM)
• Relapsed/refractory MM:
– Barlogie University of Arkansas study
• 31% of patients r...
1010
THALOMID®
US Oncology Usage
Solid Tumor
Hematologic
Solid Tumors 26%
Renal cell 6%
Brain 4%
Melanoma 3%
Colorectal 2%...
1111
Key Elements of S.T.E.P.S.®
(System for Thalomid Education and Prescribing Safety)
• Prescriber, pharmacy, and patien...
1212
Azacitidine
• Licensed global rights from Pharmacia in June 2001
• Royalty-based acquisition
– One-time option to con...
1313
Azacitidine Regulatory Status
• Licensed Azacitidine from Pharmacia June 2001
• Pre-NDA meeting held Q4 2002
• NDA su...
1414
Myelodysplastic Syndromes (MDS)
• Bone marrow disorder characterized by immature
blood cells with abnormal function
–...
1515
Azacitidine Phase III Results
• Azacitidine statistically superior to supportive care for:
– Response: 60% of pts (7%...
1616
Azacitidine in MDS
“…The positive experience with azacitidine…
brings new hopes to thousands of patients
with MDS and...
1717
Refludan
• Licensed from Schering AG in June 2002 for all
countries outside US and Canada
• Upfront and milestone pay...
1818
Refludan: Establishes EU/ROW Commercial
Infrastructure
• European headquarters located in Cambridge, UK
• 9 EU countr...
1919
Innohep
• Licensed US rights from LEO Pharma in July 2002
• Up-front payment and royalties
• Low molecular weight hep...
2020
Innohep Product Positioning
• Position as the treatment of choice for the
hematology/oncology DVT patient
• DVT secon...
2121
U.S. Commercial Organization
• 31 field-based professionals
– Average oncology sales experience 13 years
• Medical ma...
2222
Finances
• $130 million raised to date
• December 31, 2002 cash balance - $62 million
• Current investors
– NEA
– Nom...
2323
2002 Accomplishments
• Thalidomide European MAA submission
• Refludan acquisition
• Innohep acquisition
• Established...
2424
2003 Objectives
• Thalidomide European approval and launch
• Implement S.T.E.P.S. safety program
• Additional ROW Tha...
2525
Summary
• Global hematology/oncology company
• Commercial, development and regulatory
capabilities
• Four products li...
2727
Management Team
Patrick Mahaffy, President & CEO NeXstar
Judith Hemberger, PhD, Exec VP & COO HMR
Barrie Alioth, VP S...
2828
Global Sales and Commercialization
2929
Product Portfolio
• Building a product portfolio with a balanced
risk profile
• Innovative products with high therape...
3030
Thalomid in Other Indications
• Metastatic renal cell carcinoma
– 2 Phase II studies
• 1st: 16/25 - stable disease (S...
Pharmion Overview Global Specialty Pharmaceutical Company
Pharmion Overview Global Specialty Pharmaceutical Company
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Pharmion Overview Global Specialty Pharmaceutical Company

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  • RCC trials:
    1st: Motzer RJ et al. Phalse II trial of thalidomide for patients with advanced renal cell carcinoma. J Clin Oncol 2002; 20:302-306.
    2nd: Stebbing J et al. The treatment of advanced renal cell cancer with high-dose thalidomide. Br J Cancer 2001;85:953-958.
    Metastatic colorectal cancer:
    Govindarajan R. Irinotecan and thalidomide in metastatic colorectal cancer. Oncology 2000;14:29-32.
    Glioblastoma:
    Marx GM et al. Phase II study of thalidomide in the treatment of recurrent glioblastoma multiforme. J Neurooncol 2001;54:31-38.
    AIPC:
    Figg WD et al. A randomized phase II trial of thalidomide, an angiogenesis inhibitor, in patients with androgen-independent prostate cancer. Clin Cancer Res 2001;7:1888-1893.
  • Pharmion Overview Global Specialty Pharmaceutical Company

    1. 1. 22 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
    2. 2. 33 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
    3. 3. 44 Pharmion Organization • 130 employees • Regulatory Affairs • Medical Marketing • Sales and Marketing • Commercial Development • Information Technology
    4. 4. 55 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
    5. 5. 66 Pipeline Overview Phase of Product Indication Development Licensor Territory Thalomid® • Relapsed/refractory Registration Celgene EU & selected multiple myeloma ROW • ENL territories Azacitidine • Myelodysplastic Preregistration Pharmacia 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
    6. 6. 77 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
    7. 7. 88 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
    8. 8. 99 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
    9. 9. 1010 THALOMID® US Oncology Usage Solid Tumor Hematologic 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% SOURCE: S.T.E.P.S.®
    10. 10. 1111 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
    11. 11. 1212 Azacitidine • Licensed global rights from Pharmacia in June 2001 • Royalty-based acquisition – One-time option to convert to 50/50 WW co-promotion relationship • Initial indication sought: Myelodysplastic Syndromes – Phase III study completed and published • Unique Mechanism of Action – Demethylation • Potential in other tumor types
    12. 12. 1313 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
    13. 13. 1414 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
    14. 14. 1515 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 (p<0.001) – Time to leukemic transformation or death: 21 months with azacitidine vs 13 months on supportive care (p<0.007) – Transformation to AML: first event in 15% of azacitidine patients vs 38% with supportive care (p<0.001) – QOL Improvement (fatigue, shortness of breath, psychological state, etc.) (p<0.05) Silverman et al, JCO, May 15 2002, 2429-2440
    15. 15. 1616 Azacitidine in MDS “…The positive experience with azacitidine… brings new hopes to thousands of patients with 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
    16. 16. 1717 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
    17. 17. 1818 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
    18. 18. 1919 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
    19. 19. 2020 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
    20. 20. 2121 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
    21. 21. 2222 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
    22. 22. 2323 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
    23. 23. 2424 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
    24. 24. 2525 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
    25. 25. 2727 Management Team Patrick Mahaffy, President & CEO NeXstar Judith Hemberger, PhD, Exec VP & COO HMR Barrie Alioth, VP Strategic Planning Aventis Alan Cooke, VP Europe Sales & Mktg NeXstar/Gilead Michael Cosgrave, VP ROW Sales & Mktg NeXstar/Gilead Jeff Davis, VP Information Technology NeXstar/Gilead Pam Herriott, VP Human Resources HMR Gillian Ivers-Read, VP Development, Regulatory Affairs Aventis Dave Katterhenrich, VP Science & Educ Erle Mast,VP and Chief Financial Officer Aventis Dura,Elan Bill Poncy, VP US Sales & Mktg DuPont, Maxim Terry Shelton, VP Business Development Aventis
    26. 26. 2828 Global Sales and Commercialization
    27. 27. 2929 Product Portfolio • Building a product portfolio with a balanced risk profile • Innovative products with high therapeutic impact – Thalomid – Azacitidine – Refludan – Innohep • Attractive economic terms • Common target audience
    28. 28. 3030 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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