22

Pharmion OverviewPharmion 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

• 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

33

Global LeverageGlobal 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

• 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

44

Pharmion OrganizationPharmion Organization

• 130 employees

• Regulatory Affairs

• Medical Marketing

• Sales and Marketing

• Commercial Development

• Information Technology

• 130 employees

• Regulatory Affairs

• Medical Marketing

• Sales and Marketing

• Commercial Development

• Information Technology

55

Initial Focus: Hematology 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

• 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

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Pipeline OverviewPipeline 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 USDVT with or A/Swithout PE

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 USDVT with or A/Swithout PE

77

Thalomid®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

• 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

88

Thalomid Regulatory StatusThalomid 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

• 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

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Thalomid in Multiple Myeloma (MM)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

• 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

1010

THALOMID® US Oncology UsageTHALOMID® 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.®

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Key Elements of S.T.E.P.S.®

(System for Thalomid Education and Prescribing Safety)

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

• 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

1212

AzacitidineAzacitidine

• 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

• 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

1313

Azacitidine Regulatory StatusAzacitidine 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

• 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

1414

Myelodysplastic Syndromes (MDS)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

• 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

1515

Azacitidine Phase III ResultsAzacitidine 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)

• 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, 2429-2440Silverman et al, JCO, May 15 2002, 2429-2440

1616

Azacitidine in MDSAzacitidine in MDS

“…The positive experience with azacitidine…brings new hopes to thousands of patientswith MDS and to investigators who dedicate their research to improving prognosis in this relatively common and lethal malignancy…”

“…The positive experience with azacitidine…brings 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- Hagop M. Kantarjian

JCO, May 15 2002, 2415-2416JCO, May 15 2002, 2415-2416

1717

RefludanRefludan

• 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

• 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

1818

Refludan: Establishes EU/ROW Commercial InfrastructureRefludan: 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

• 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

1919

InnohepInnohep

• 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

• 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

2020

Innohep Product PositioningInnohep 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

• 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

2121

U.S. Commercial OrganizationU.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

• 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

2222

FinancesFinances

• $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

• $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

2323

2002 Accomplishments2002 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

• 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

2424

2003 Objectives2003 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

• 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

2525

Summary 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

• 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

2727

Management TeamManagement 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

2828

Global Sales and CommercializationGlobal Sales and Commercialization

2929

Product PortfolioProduct 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

• Building a product portfolio with a balancedrisk profile

• Innovative products with high therapeutic impact– Thalomid

– Azacitidine

– Refludan

– Innohep

• Attractive economic terms

• Common target audience

3030

Thalomid in Other IndicationsThalomid 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

• 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