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Hematological Cancer Therapies:Pipelines, Markets, and Business Considerations

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Over 250 pages of market data, expert interviews, 16 tables, plus over 200 companies, including:

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Hematological Cancer Therapies:Pipelines, Markets, and Business Considerations

Lucy J. Sannes, PhD, MBA recent years, newer and more specific therapies for hematological cancers have been developed, such as targeted small-molecule drugs and biological therapies including monoclonal antibodies. However,

there remains significant need from a clinical perspective, as well as challenges and opportunities for pharmaceutical companies. This report discusses:

•Thedifferenttypesofhematologicalcancers,includinginformationaboutthespecificdisease,itsepidemiology,subtypesand/orstagingifappropriate,currenttherapies,andpatientprognosis.

•Pipelinesofemergingdrugsfortreatmentofhematologicalcancersincludingleukemia,lymphoma,myeloma,myelodysplasticsyndromes,andmyeloproliferativediseases.

•Businessandstrategicconsiderationsrelatingtopharmacologicaltherapyforhematologicalcancers,includingassessmentofthecurrentmarketaswellaspotentialmarketsforemergingnewtherapies.

•Majorchallengesandhurdlesforcompaniesdevelopinghematologicalcancertherapies.

•Interviewswithexpertsinthefieldofhematologicalcancers,whichwereconductedexclusivelyforthisreport.

JUST RELEASEDMarch 2012

O V E R V I E W ABOUT THE AUTHOR

Hematological cancers arise from and are found in the blood, bone marrow, and lymphatic systems. The first hematological cancer to be identified was Hodgkin’s disease, or Hodgkin lymphoma, in 1832. Since that time, many different hematological cancers have been identified, including the various types of leukemia, lymphoma, and myeloma, plus the myelodysplastic syndromes and myeloproliferative diseases. Altogether, hematological cancers represent a significant medical problem: The American Cancer Society estimated that there would be 140,310 new cases of leukemia, lymphoma, and myeloma, combined, in the United States in 2011, and that 53,010 people would die of these diseases that year.

In this Insight Pharma Report, Hematological Cancer Therapies: Pipelines, Markets, and Business Considerations, we focus on the many different types of leukemia, lymphoma, and myeloma, and myelodysplastic syndromes and myeloproliferative diseases. Current pharmacological therapies that are used to treat these various hematological cancers are discussed. In addition, there is significant activity on the part of pharmaceutical companies to further develop a number of the currently available therapies for additional indications.

A primary focus of this Insight Pharma Report is on pipelines of emerging therapies in development for treatment of hematological cancers. Specifically, the clinical development pipelines for leukemia, lymphoma, myeloma, myelodysplastic syndromes, and myeloproliferative diseases are reviewed. Selected emerging therapies that are in Phase II or later stages of development are discussed in the text, while the extensive accompanying tables include these emerging therapies plus many additional earlier-stage therapies that are in development.

Despite the smaller number of patients who develop hematological cancers (compared to solid tumors and certain other, non-oncology markets), it is possible to develop and commercialize hematological cancer therapies that become blockbuster drugs (i.e., with sales exceeding $1 billion per year). Hematological Cancer Therapies: Pipelines, Markets, and Business Considerations reviews the current market for hematological cancer drugs and includes sales data for selected drugs. Also addressed are major challenges and hurdles, in addition to other strategic considerations such as personalized medicine, that companies in this market must address. Full transcripts of interviews with experts in the field of hematological cancers are included in this report.

Lucy J. Sannes, PhD, MBA, is president of Sannes &

Associates, a consulting firm specializing in evaluation

and management of the biosciences. Before forming

Sannes & Associates, she held management positions

at Genetic Systems and Abbott Laboratories in product development, product support, and

technical marketing. Dr. Sannes received her PhD in

biological chemistry from the University of Michigan and her MBA from Seattle

Pacific University.

SOURCE: Sannes & Associates, Inc.

ABOUT INSIGHT PHARMA REPORTS

CHI’s Insight Pharma Reports are written by experts who

collaborate with CHI to provide a series of reports that

evaluate the salient trends in pharmaceutical technology,

business, and therapy markets. Insight Pharma Reports are used

by senior decision makers at life sciences companies to keep abreast of the latest advances in pharmaceutical R&D, their

potential applications and business impacts. Our clients

include the top 50 pharmaceutical companies, top 100 biotechnology

companies, and top 100 vendors of life science products and

services. Typical purchasers are managers, directors, and VPs in

business development, discovery research, clinical develop ment,

strategic planning, portfolio management, new product

planning, and marketing.

INSIGHT PHARMA REPORTSA division of Cambridge Healthtech Institute

250 First Avenue • Suite 300 • Needham, MA 02494 • 781-972-5444 • InsightPharmaReports.com

TABLE OF CONTENTS

EXECUTIVE SUMMARY

CHAPTER 1 INTRODUCTION

1.1. Background1.2. Scope of the Report

CHAPTER 2 REVIEW OF HEMATOLOGICAL CANCERS (BLOOD CANCERS)

2.1. LeukemiaAcute Lymphocytic Leukemia (ALL)

Chronic Lymphocytic Leukemia (CLL)

Acute Myelogenous (Myeloid) Leukemia (AML)

Chronic Myelogenous (Myeloid) Leukemia (CML)

Other Types of Leukemia

2.2. LymphomaHodgkin’s Disease

Non-Hodgkin’s Lymphoma (NHL)

2.3. Myeloma2.4. Myelodysplastic Syndromes (MDS)2.5. Myeloproliferative Diseases

CHAPTER 3 CURRENT PHARMACOLOGICAL TREATMENT OPTIONS

3.1. LeukemiaAcute Lymphocytic Leukemia (ALL)

Chronic Lymphocytic Leukemia (CLL)

Acute Myelogenous (Myeloid) Leukemia (AML)

Chronic Myelogenous (Myeloid) Leukemia (CML)

Other Types of Leukemia

3.2. LymphomaHodgkin’s Disease

Non-Hodgkin’s Lymphoma (NHL)

3.3. Myeloma3.4. Myelodysplastic Syndromes (MDS)3.5. Myeloproliferative Diseases

CHAPTER 4 EMERGING THERAPIES FOR LEUKEMIA

4.1. Abbott’s and Genentech/Roche’s Navitoclax4.2. ARIAD Pharmaceuticals’ Ponatinib4.3. Astellas Pharma’s and Ambit Biosciences’

Quizartinib4.4. AstraZeneca’s AZD11524.5. BioCryst Pharmaceutical’s Forodesine4.6. Boehringer Ingelheim’s Volasertib4.7. Celator Pharmaceutical’s CPX-3514.8. Celgene’s REVLIMID4.9. Chroma Therapeutics’ and Cell Therapeutics’

Tosedostat4.10. Clavis Pharma’s Elacytarabine4.11. Cyclacel Pharmaceuticals’ Sapacitabine4.12. CytRx’ Bafetinib4.13. Eisai’s and Janssen-Cilag’s Dacogen4.14. EpiCept’s Ceplene4.15. Genzyme’s/Sanofi’s Clolar4.16. Gilead Sciences’ GS 1101

4.17. GlaxoSmithKline’s ARZERRA4.18. Medac GmbH’s Treosulfan4.19. Memgen’s ISF354.20. Micromet’s Blinatumomab4.21. Novartis’ Midostaurin4.22. Pfizer’s Bosutinib4.23. Roche’s RG7159 (Obinutuzumab)4.24. Sunesis Pharmaceuticals’ Vosaroxin4.25. Syndax Pharmaceuticals’ Entinostat4.26. Talon Therapeutics’ Marqibo

CHAPTER 5 EMERGING THERAPIES FOR LYMPHOMA

5.1. 4SC AG’s Resminostat5.2. Abbott’s and Genentech/Roche’s Navitoclax5.3. Allos Therapeutics’ Folotyn5.4. Astellas Pharma’s YM1555.5. BioCryst Pharmaceutical’s Forodesine5.6. Biovest International/Accentia

Biopharmaceuticals’ BiovaxID5.7. Celgene’s REVLIMID5.8. Cell Therapeutics’ Pixantrone5.9. Eli Lilly’s Enzastaurin5.10. Gilead Sciences’ GS 11015.11. GlaxoSmithKline’s ARZERRA5.12. Kyowa Hakko Kirin’s KW-07615.13. Memgen’s ISF355.14. Novartis’ Afinitor5.15. Novartis’ Panobinostat5.16. Pfizer’s Inotuzumab Ozogamicin5.17. Pharmacyclics’ PCI-327655.18. Plexxikon’s (and Daiichi Sankyo’s) PLX33975.19. Roche’s RG7159 (Obinutuzumab)5.20. Sanofi’s SAR2454095.21. Spectrum Pharmaceuticals’ and Topotarget’s

Belinostat5.22. Syndax Pharmaceuticals’ Entinostat5.23. Telik’s TELCYTA5.24. Teva Pharmaceutical Industries’ and CureTech’s

CT-011

CHAPTER 6 EMERGING THERAPIES FOR MYELOMA

6.1. Æterna Zentaris’ and Keryx Biopharmaceuticals’ Perifosine (KRX-1401)

6.2. Bristol-Myers Squibb’s and Abbott’s Elotuzumab6.3. Celgene’s Actimid6.4. Geron’s Imetelstat6.5. Janssen Biotech’s Siltuximab (CNTO 328)6.6. Novartis’ BHQ8806.7. Novartis’ Panobinostat6.8. Onyx Pharmaceuticals’ Carfilzomib6.9. PharmaMar’s Aplidin

CHAPTER 7 EMERGING THERAPIES FOR MYELODYSPLASTIC

SYNDROMES7.1. Amgen’s Nplate (Romiplostim)7.2. Celgene’s REVLIMID7.3. Cyclacel Pharmaceuticals’ Sapacitabine7.4. Medac GmbH’s Treosulfan7.5. Onconova Therapeutics’ ESTYBON (Rigosertib);

SymBio Pharmaceuticals’ Rigosertib7.6. Telik’s TELINTRA

CHAPTER 8 EMERGING THERAPIES FOR MYELOPROLIFERATIVE DISEASES

8.1. Celgene’s Actimid8.2. Geron’s Imetelstat8.3. Gilead Sciences’ GS 66248.4. Incyte’s and Novartis’ Ruxolitinib8.5. Infinity Pharmaceuticals’ IPI-9268.6. Sanofi’s SAR3025038.7. YM Biosciences’ CYT387

CHAPTER 9 BUSINESS CONSIDERATIONS

9.1. Current Sales of Drugs for Treatment of Hematological Cancers

9.2. Personalized Medicine in the Field of Hematological Cancers

9.3. Selected Challenges and Hurdles Being Faced by Companies in the Field of Hematological Cancers

CHAPTER 10EXPERT INTERVIEWS

10.1. Patrick A. Baeuerle, PhD, Chief Scientific Officer and Senior Vice President, Research & Development, Micromet

10.2. Steven A. Kriegsman, President and Chief Executive Officer David J. Haen, Vice President, Business Development, CytRx Corporation

10.3. Peter Nielsen, President and Chief Executive Officer, Bio-Path Holdings

10.4. Spiro Rombotis, President and Chief Executive Officer, Cyclacel Pharmaceuticals

10.5. Dan Weinseimer, Director, Strategic Marketing and Corporate Development, Sunesis Pharmaceuticals

10.6. David E. Weng, M.D., Ph.D., Chief Medical Officer, TetraLogic Pharmaceuticals

REFERENCES

APPENDIX: SELECTED COMPANIES DEVELOPING DRUGS FOR HEMATOLOGICAL CANCERS (GENERAL)

COMPANY INDEX

Also included are extensive tables describing selected drugs in development for the various types of hematological cancer as well as drugs that have been FDA-approved, including sales data for selected drugs.