Abstract
Overview
In 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:
- The different types of hematological cancers, including information about
the specific disease, its epidemiology, subtypes and/or staging if
appropriate, current therapies, and patient prognosis.
- Pipelines of emerging drugs for treatment of hematological cancers
including leukemia, lymphoma, myeloma, myelodysplastic syndromes, and
myeloproliferative diseases.
- Business and strategic considerations relating to pharmacological therapy
for hematological cancers, including assessment of the current market as well
as potential markets for emerging new therapies.
- Major challenges and hurdles for companies developing hematological cancer
therapies.
- Interviews with experts in the field of hematological cancers, which were
conducted exclusively for this report.
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
this 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,
including 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 seven interviews with experts in the field of hematological
cancers are included in this report.
About the Author
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.
Table of Contents
Executive Summary
Chapter 1 INTRODUCTION
- 1.1. Background
- 1.2. Scope of the Report
Chapter 2 REVIEW OF HEMATOLOGICAL CANCERS (BLOOD CANCERS)
- 2.1. Leukemia
- Acute Lymphocytic Leukemia (ALL)
- Chronic Lymphocytic Leukemia (CLL)
- Acute Myelogenous (Myeloid) Leukemia (AML)
- Chronic Myelogenous (Myeloid) Leukemia (CML)
- Other Types of Leukemia
- 2.2. Lymphoma
- Hodgkin's Disease
- Non-Hodgkin's Lymphoma (NHL)
- 2.3. Myeloma
- 2.4. Myelodysplastic Syndromes (MDS)
- 2.5. Myeloproliferative Diseases
Chapter 3 CURRENT PHARMACOLOGICAL TREATMENT OPTIONS
- 3.1. Leukemia
- Acute Lymphocytic Leukemia (ALL)
- Chronic Lymphocytic Leukemia (CLL)
- Acute Myelogenous (Myeloid) Leukemia (AML)
- Chronic Myelogenous (Myeloid) Leukemia (CML)
- Other Types of Leukemia
- 3.2. Lymphoma
- Hodgkin's Disease
- Non-Hodgkin's Lymphoma (NHL)
- 3.3. Myeloma
- 3.4. Myelodysplastic Syndromes (MDS)
- 3.5. Myeloproliferative Diseases
Chapter 4 EMERGING THERAPIES FOR LEUKEMIA
- 4.1. Abbott's and Genentech/Roche's Navitoclax
- 4.2. ARIAD Pharmaceuticals' Ponatinib
- 4.3. Astellas Pharma's and Ambit Biosciences' Quizartinib
- 4.4. AstraZeneca's AZD1152
- 4.5. BioCryst Pharmaceutical's Forodesine
- 4.6. Boehringer Ingelheim's Volasertib
- 4.7. Celator Pharmaceutical's CPX-351
- 4.8. Celgene's REVLIMID
- 4.9. Chroma Therapeutics' and Cell Therapeutics' Tosedostat
- 4.10. Clavis Pharma's Elacytarabine
- 4.11. Cyclacel Pharmaceuticals' Sapacitabine
- 4.12. CytRx' Bafetinib
- 4.13. Eisai's and Janssen-Cilag's Dacogen
- 4.14. EpiCept's Ceplene
- 4.15. Genzyme's/Sanofi's Clolar
- 4.16. Gilead Sciences' GS 1101
- 4.17. GlaxoSmithKline's ARZERRA
- 4.18. Medac GmbH's Treosulfan
- 4.19. Memgen's ISF35
- 4.20. Micromet's Blinatumomab
- 4.21. Novartis' Midostaurin
- 4.22. Pfizer's Bosutinib
- 4.23. Roche's RG7159 (Obinutuzumab)
- 4.24. Sunesis Pharmaceuticals' Vosaroxin
- 4.25. Syndax Pharmaceuticals' Entinostat
- 4.26. Talon Therapeutics' Marqibo
Chapter 5 EMERGING THERAPIES FOR LYMPHOMA
- 5.1. 4SC AG's Resminostat
- 5.2. Abbott's and Genentech/Roche's Navitoclax
- 5.3. Allos Therapeutics' Folotyn
- 5.4. Astellas Pharma's YM155
- 5.5. BioCryst Pharmaceutical's Forodesine
- 5.6. Biovest International/Accentia Biopharmaceuticals' BiovaxID
- 5.7. Celgene's REVLIMID
- 5.8. Cell Therapeutics' Pixantrone
- 5.9. Eli Lilly's Enzastaurin
- 5.10. Gilead Sciences' GS 1101
- 5.11. GlaxoSmithKline's ARZERRA
- 5.12. Kyowa Hakko Kirin's KW-0761
- 5.13. Memgen's ISF35
- 5.14. Novartis' Afinitor
- 5.15. Novartis' Panobinostat
- 5.16. Pfizer's Inotuzumab Ozogamicin
- 5.17. Pharmacyclics' PCI-32765
- 5.18. Plexxikon's (and Daiichi Sankyo's) PLX3397
- 5.19. Roche's RG7159 (Obinutuzumab)
- 5.20. Sanofi's SAR245409
- 5.21. Spectrum Pharmaceuticals' and Topotarget's Belinostat
- 5.22. Syndax Pharmaceuticals' Entinostat
- 5.23. Telik's TELCYTA
- 5.24. Teva Pharmaceutical Industries' and CureTech's CT-011
Chapter 6 EMERGING THERAPIES FOR MYELOMA
- 6.1. Aterna Zentaris' and Keryx Biopharmaceuticals' Perifosine (KRX-1401)
- 6.2. Bristol-Myers Squibb's and Abbott's Elotuzumab
- 6.3. Celgene's Actimid
- 6.4. Geron's Imetelstat
- 6.5. Janssen Biotech's Siltuximab (CNTO 328)
- 6.6. Novartis' BHQ880
- 6.7. Novartis' Panobinostat
- 6.8. Onyx Pharmaceuticals' Carfilzomib
- 6.9. PharmaMar's Aplidin
Chapter 7 EMERGING THERAPIES FOR MYELODYSPLASTIC SYNDROMES
- 7.1. Amgen's Nplate (Romiplostim)
- 7.2. Celgene's REVLIMID
- 7.3. Cyclacel Pharmaceuticals' Sapacitabine
- 7.4. Medac GmbH's Treosulfan
- 7.5. Onconova Therapeutics' ESTYBON (Rigosertib); SymBio Pharmaceuticals'
Rigosertib
- 7.6. Telik's TELINTRA
Chapter 8 EMERGING THERAPIES FOR MYELOPROLIFERATIVE DISEASES
- 8.1. Celgene's Actimid
- 8.2. Geron's Imetelstat
- 8.3. Gilead Sciences' GS 6624
- 8.4. Incyte's and Novartis' Ruxolitinib
- 8.5. Infinity Pharmaceuticals' IPI-926
- 8.6. Sanofi's SAR302503
- 8.7. 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 10 EXPERT 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. Carlos F. Santos, Ph.D.
- Senior Vice President, Product Development & Regulatory Affairs
- Douglas W. Calder
- Vice President, Strategic Planning & Capital Markets
- Biovest International and Accentia Biopharmaceuticals
- 10.6. Dan Weinseimer
- Director, Strategic Marketing and Corporate Development
- Sunesis Pharmaceuticals
- 10.7. David E. Weng, M.D., Ph.D.
- Chief Medical Officer
- TetraLogic Pharmaceuticals
References
Appendix: Selected Companies Developing Drugs for Hematological Cancers (General)
Company Index
TABLES
- Table 2.1. Incidence and Mortality of Hematological Cancers in the United
States, 2011
- Table 2.2. Updated REAL/WHO Classification of Lymphoproliferative Neoplasms
- Table 2.3. PDQ Modification of REAL Classification of Lymphoproliferative
Diseases
- Table 3.1. Selected Chemotherapy Drugs that are FDA-Approved for Treatment
of Cancer
- Table 3.2. Selected Drugs Approved by the FDA for Treatment of Leukemia
- Table 3.3. Selected Drugs Approved by the FDA for Treatment of Lymphoma,
Including Hodgkin's Disease and Non-Hodgkin's Lymphoma (NHL)
- Table 3.4. Selected Drugs Approved by the FDA for Treatment of Multiple
Myeloma
- Table 3.5. Selected Drugs Approved by the FDA for Treatment of
Myelodysplastic Syndrome (MDS)
- Table 3.6. Selected Drugs Approved by the FDA for Treatment of
Myeloproliferative Diseases
- Table 4.1. Selected Companies Developing Drugs for Leukemia
- Table 5.1. Selected Companies Developing Drugs for Lymphomas
- Table 6.1. Selected Companies Developing Drugs for Myeloma
- Table 7.1. Selected Companies Developing Drugs for Myelodysplastic
Syndromes
- Table 8.1. Selected Companies Developing Drugs for Myeloproliferative
Diseases
- Table 9.1. Sales of Selected Drugs Used to Treat Hematological Cancers
- Appendix: Selected Companies Developing Drugs for Hematological Cancers
(General)
