Title: NEA_Menlo_Park
1(No Transcript)
2Pharmion 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
3Global 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
4Pharmion Organization
- 130 employees
- Regulatory Affairs
- Medical Marketing
- Sales and Marketing
- Commercial Development
- Information Technology
5Initial 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
6Pipeline 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
7Thalomid
- 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
8Thalomid 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
9Thalomid 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
10THALOMID 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.
11Key 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
12Azacitidine
- 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
13Azacitidine 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
14Myelodysplastic 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
15Azacitidine 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
16Azacitidine 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
17Refludan
- 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
18Refludan 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
19Innohep
- 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
20Innohep 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
21U.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
22Finances
- 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
232002 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
242003 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
25Summary
- 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
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27Management Team
28Global Sales and Commercialization
29Product 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
30Thalomid 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