Pipeline Insight: Hematological Malignancies - Pursuit for the next blockbuster intensifies : Market Research Report

Pipeline Insight: Hematological Malignancies - Pursuit for the next blockbuster intensifies

Published by: Datamonitor

Published: Mar. 30, 2007 - 374 Pages

CHAPTER 1 EXECUTIVE SUMMARY: Scope of the analysis; Datamonitor insight into the hematological malignancies market; Key metrics; Datamonitor pipeline assessment summary
CHAPTER 2 PIPELINE OVERVIEW AND DYNAMICS: Pipeline overview; Products in late-phase development for hematological malignancies; Products in Phase II development for hematological malignancies; Products in Phase I development for hematological malignancies; Pipeline by developmental phase and class; There are 180 different products in the clinical developmental pipeline for hematological malignancies; Segmentation of products by developmental phase reflects high attrition rate in oncology drug development; Segmentation of products by class reflects shift in oncology drug development away from cytotoxics; Pipeline by indication; Over 50% of the pipeline products are being investigated in leukemia; The 22 late-phase pipeline products target eight different hematological malignancies; Pipeline by company; Developmental pipeline dominated by small pharma/biotech players; Only 17 (12%) companies/institutes have more than two candidates in the developmental pipeline for hematological malignancies; Novartis has six candidates in clinical development; Biogen Idec draws on the success of Rituxan
CHAPTER 3 HEMATOLOGICAL MALIGNANCIES - MARKET POTENTIAL: A diverse range of disease subtypes; Genetic basis of cancer evolution; Tumorigenesis is the result of co-operative accumulated mutations; Existing pharmacotherapy approaches provide limited treatment benefit; Cytotoxic drugs lack specificity; Hormonal or endocrine therapy provides incremental benefit in selected tumors; Optimizing current treatment strategies is paramount; The emergence of targeted treatment heralds a revolution in cancer pharmacotherapy; Dynamic cancer market offers significant commercial opportunity; Ongoing sales growth drives the market; Intensive R&D produces a rich developmental pipeline; Epidemiology - Hematological Malignancies; Cancer epidemiology - an expanding patient base; Leukemia; Lymphoma; Multiple myeloma; Myelodysplastic syndrome; Disproportionate increase in prevalence results from improvements in diagnosis and treatment; Significant areas of unmet need persist; The need for more sophisticated pharmacotherapy; Long-term control of advanced tumors is suboptimal; Novel strategies required to reduce relapse rates in early-stage disease; Toxicity of existing treatments jeopardizes quality of life and rates of treatment uptake; Improvements in diagnostics and prognostic analysis will enhance cost-effectiveness of treatment; Enhanced preventative strategies will ease the disease burden; Clinical and strategic threats to the commercialization of cancer drugs; Progressively rising R&D costs threaten industry productivity; High attrition rates can be mitigated by improved strategic decision-making; Lengthening drug approval process - a consequence of increased regulatory demands; Pharmacoeconomic pressures drive payers to implement restrictive pricing and reimbursement policies; Therapeutic and generic competition reduces periods of market exclusivity; Segmentation of market will require changes in clinical trial methodology
CHAPTER 4 R&D APPROACH: Classification of pipeline products; Molecular targeted therapies; Angiogenesis inhibitors; Single-target signal transduction inhibitors; Multi-targeted inhibitors; Cell cycle and apoptosis targeted inhibitors; Epigenetic modulators; Cytotoxic Therapies; Antimetabolites; Mitotic inhibitors; DNA-interactive chemotherapeutic agents; Immunotherapeutic agents; Antibody-based technologies are an effective anticancer approach; Active, specific immunotherapy; Evolution in oncology clinical trial design; Patient selection is increasingly significant in the era of targeted treatment; Clinical trials must have sufficient follow-up to establish true clinical benefit; Diversity of targeted treatments will require an evolution in clinical trial design; Most oncology clinical trials designate multiple endpoints; Survival; Quality of life; Tumor response rates; Toxicity; Time to tumor progression; Modification of accelerated approval process may impact significantly on approval times for hematologic oncology drugs
CHAPTER 5 MOLECULAR TARGETED THERAPIES ANALYSIS AND FORECASTS: Overview of molecular targeted therapies for hematological malignancies; Pipeline summary; Late-phase pipeline of molecular targeted therapies; Phase II pipeline of molecular targeted therapies; Phase I pipeline of molecular targeted therapies; Comparative forecasts; Definition of current comparator therapy; Novartis's Gleevec/Glivec (imatinib); Tasigna (Nilotinib, AMN-107; Novartis); Drug overview; Clinical trial data; Tasigna enters preregistration in the US and EU for Gleevec-resistant CML; Promising Phase II interim data reported; Tasigna appears effective in CML patients who have failed or are intolerant to both Gleevec and Sprycel; Only one BCR-ABL mutation is insensitive to Tasigna; Datamonitor comments; Tasigna ready to challenge Bristol-Myers Squibb's already approved Sprycel; Novartis looking to expand its leading role in the CML therapy market; Forecasts to 2016; Datamonitor drug assessment summary; Ceflatonin (Myelostat; ChemGenex Pharmaceuticals); Drug overview; Clinical trial data; Ceflatonin receives Fast Track status for chronic myeloid leukemia; Ceflatonin aims to restore Gleevec sensitivity in CML patients; ChemGenex looking to expand Ceflatonin into the AML/APL market; Datamonitor comments; Despite convincing clinical benefit, Ceflatonin will face strong competition from Bristol-Myers Squibb's Sprycel and Novartis's Tasigna; Forecasts to 2016; Datamonitor drug assessment summary; Sarasar (Lonafarnib; Schering-Plough); Drug overview; Clinical trial data; Main focus of Sarasar development in MDS, where greatest antitumor activity is shown; Farnesyl transferase inhibitors predominately in hematological disorders; Mild toxicity in the majority of patients, although grade 3 events do occur; Datamonitor comments; Sarasar's chances for approval will be delayed beyond 2007; Sarasar racing against Johnson & Johnson's Zarnestra as the first farnesyl transferase inhibitor to reach the market; Presence in oncology market will aid commercialization of Sarasar; Forecasts to 2016; Datamonitor drug assessment summary; Torisel (Temsirolimus; Wyeth); Drug overview; Torisel inhibits a key pathway in tumor cell proliferation; Clinical trial data; Torisel showing promise in mantle cell lymphoma; Torisel also making headway in other NHL subtypes; Datamonitor comments; Torisel will have to face Velcade in the MCL market; Prior commercialization of Mylotarg and Neumega will provide Wyeth with valuable insight into the oncology market; Forecasts to 2016; Datamonitor drug assessment summary; Zarnestra (Tipifarnib; Janssen/Johnson & Johnson); Drug overview; Clinical trial data; Following rejection of NDA, the FDA requires Phase III data for Zarnestra in AML before regulatory approval can be considered; Results from Phase III studies have yet to be announced; Zarnestra has demonstrated a favorable profile in a variety of Phase II studies; Single-agent Zarnestra demonstrates antitumor activity in relapsed/refractory aggressive NHL; Zarnestra holding promise in juvenile myelomonocytic leukemia; Initiation of Phase II trials in large granular lymphocyte leukemia and multiple myeloma; Mild toxicity is particularly significant since Zarnestra's main indication is for elderly AML patients where quality of life is a major issue; Datamonitor comments; Schering-Plough's Sarasar catching up with Zarnestra as the first farnesyl transferase inhibitor to reach the market; Johnson & Johnson limiting Zarnestra's target population in the short term; Johnson & Johnson's experience will be invaluable to Zarnestra; Forecasts to 2016; Datamonitor drug assessment summary; Alvocidib (Flavopiridol; Sanofi-Aventis); Drug overview; Clinical trial data; Continuous infusion dosing schedules fail to demonstrate clinical activity; Modified dosing regimen drives further development in CLL; Datamonitor comments; Given alvocidib's checkered history, Sanofi-Aventis may face an uphill struggle communicating the drug's potential; Alvocidib may show more promise as part of a combination regimen; Presence in oncology field will aid commercialization of alvocidib; Enzastaurin (LY317615; Eli Lilly); Drug overview; Clinical trial data; Enzastaurin looking to make its mark in the B-Cell Lymphoma market; Enzastaurin holding promise as a maintenance therapy in mantle cell lymphoma; Datamonitor comments; Eli Lilly adopt a risky stragtegy for enzastaurin in DLBCL; Termination of Phase III trial for enzastaurin in glioma may hamper its potential in other indications; Forecasts to 2016; Datamonitor drug assessment summary; Lestaurtinib (CEP-701; Cephalon); Drug overview; Clinical trial data; Lestaurtinib emerging as a promising agent for AML patients harboring Flt-3 activating mutations; Datamonitor comments; Lestaurtinib may be the first in its class to reach the market; Cephalon's recent acquisition of Trisenox will provide invaluable experience of the leukemia market; Forecasts to 2016; Datamonitor drug assessment summary; Genasense (Oblimersen; Genta); Drug overview; Clinical trial data; FDA reject Genasense for use in combination with chemotherapy in CLL; Early-phase benefits of Genasense in AML require confirmation in Phase III clinical trial; Disappointing Phase III trial results in multiple myeloma means status of further development is unclear; Promise shown in combination with Rituxan in NHL, but randomized trials have yet to be initiated; Datamonitor comments; Approval of Genasense is looking increasingly unlikely; Termination of agreement with Sanofi-Aventis is a major setback for Genta; Forecasts to 2016; Datamonitor drug assessment summary
CHAPTER 6 CYTOTOXIC THERAPIES ANALYSIS AND FORECASTS: Overview of cytotoxic therapies for hematological malignancies; Pipeline summary; Late-phase pipeline of cytotoxic therapies; Phase II pipeline of cytotoxic therapies; Phase I pipeline of cytotoxic therapies; Comparative forecasts; Clolar/Evoltra (Clofarabine; Genzyme/Bioenvision); Drug overview; Clinical trial data; FDA and EMEA approve Clolar for ALL but further data in AML are required; Clolar and cytarabine appears to be an active and well tolerated regimen for elderly AML patients for which a Phase III trial has recently been initiated; Single-agent Clolar may provide an important treatment option for AML patients with adverse cytogenetics who are unsuitable for standard chemotherapy; Despite further data now required for approval, Clolar demonstrates activity in pediatric AML; Datamonitor comments; While approval in AML will significantly broaden Clolar's label, increased economic constraints on healthcare systems may restrict its uptake; Forecasts to 2016; Datamonitor drug assessment summary; Dacogen (decitabine; MGI Pharma); Drug overview; Clinical trial data; Elderly AML is an attractive indication for horizontal expansion of Dacogen; Dacogen as a maintenance therapy in AML; Dacogen plus Zolinza may be an effective combination 227; Datamonitor comments; Dacogen would fulfill a high unmet need in unfavorable risk AML; Dacogen may face competition from Vidaza, another approved DNA demethylating agent in development for AML; Dacogen will compete with Mylotarg in the relapsed elderly AML market; Forecasts to 2016; Datamonitor drug assessment summary; Cloretazine (VNP40101M; Vion Pharmaceuticals); Drug overview; Clinical trial data; Cloretazine will enjoy the advantages of Orphan Drug and Fast Track status for AML; Cloretazine and cytarabine appears a feasible combination for second-line AML; Single agent Cloretazine appears effective in elderly poor risk AML; High-risk MDS patients may also benefit from Cloretazine monotherapy; Cloretazine and Temodar in hematological malignancies appears to be a rational combination; Vion discontinues Cloretazine development in CLL to focus on AML; Datamonitor comments; Given persistent high unmet needs and the lack of a gold-standard in relapsed/refractory AML, Cloretazine demonstrates promise; Vion should seek a collaborative agreement with a more experienced oncology partner; Forecasts to 2016; Datamonitor drug assessment summary; Pixantrone (BBR-2778; Cell Therapeutics); Drug overview; Clinical trial data; Use of pixantrone for aggressive NHL; Use of pixantrone for indolent NHL; Datamonitor comments; Problems associated with trying to replace genericized drugs must be overcome; Patient recruitment to trials and physician awareness may be an uphill struggle; Pixantrone set to benefit from co-licensing agreement with Novartis; Forecasts to 2016; Datamonitor drug assessment summary; Marqibo (Sphingosomal vincristine; Hana Biosciences); Drug overview; Clinical trial data; Despite an FDA non-approvable letter and recommendation to initiate a Phase III study in NHL, to date no such trial has been initiated; Relapsed aggressive NHL; Marqibo as a replacement for vincristine in the R-CHOP regimen for first-line NHL appears to be a promising possibility; Marqibo is a potential candidate for the treatment of relapsed/refractory Hodgkin's disease; Trials in ALL may hold promise for Marqibo; Datamonitor comments; Hana Biosciences face a difficult strategic development plan for Marqibo in NHL and shifting the focus to ALL may offer a quicker route to market
CHAPTER 7 IMMUNOTHERAPEUTIC AGENTS ANALYSIS AND FORECASTS: Overview of immunotherapeutic agents for hematological malignancies; Pipeline summary; Late-phase pipeline of immunotherapeutic agents; Phase II pipeline of immunotherapeutic agents; Phase I pipeline of immunotherapeutic agents; Comparative forecasts; Definition of current comparator therapy; Biogen Idec/Genentech/Roche's Rituxan/MabThera (rituximab); Ceplene (Histamine dihydrochloride; Epicept); Drug overview; Clinical trial data; Ceplene enters preregistration in Europe as a maintenance therapy in first remission in patients with AML; Ceplene plus IL-2 prolongs leukemia-free survival but no significant difference in overall survival observed; Datamonitor comments; FDA requests additional Phase III trial despite primary endpoints being met; EpiCept's lack of oncology experience may hamper Ceplene's market success; Forecasts to 2016; Datamonitor drug assessment summary; Galiximab (Anti-CD80 MAb; Biogen Idec); Drug overview; Clinical trial data; Randomized Phase III trial will compare survival of galiximab plus Rituxan with Rituxan alone in relapsed/refractory follicular NHL patients; Phase II results show galiximab and Rituxan can be safely combined and can produce promising response rates in follicular NHL patients; Datamonitor comments; Biogen Idec in a strong position to successfully market galiximab alone; Biogen-Idec will need to effectively demonstrate the value of a combination of galiximab and Rituxan to payers; Forecasts to 2016; Datamonitor drug assessment summary; Lumiliximab (Anti-CD23 MAb; Biogen Idec); Drug overview; Clinical trial data; Lumiliximab receives Fast Track and Orphan Drug designation for relapsed CLL; The addition of lumiliximab to the FCR regimen may produce a higher response rate without additional toxicity; Datamonitor comments; Lumiliximab on course to become an established addition to the standard treatment for CLL; Biogen Idec should look to investigate Lumiliximab as a maintenance therapy; Cluster of Differentiation (CD) drugs have become Biogen Idec's specialty; Forecasts to 2016; Datamonitor drug assessment summary; Ofatumumab (HuMax-CD20; Genmab/GlaxoSmithKline); Drug overview; Clinical trial data; Genmab hoping ofatumumab will demonstrate a preferred efficacy profile over Rituxan in the clinic; Ofatumumab receives Fast Track status for CLL and enters a Phase III trial; Genmab initiate a pivotal Phase III trial in follicular NHL; Datamonitor comments; Ofatumumab may offer hope for Rituxan-insensitive patients; Approval of other MAbs being developed by Biogen Idec for NHL and CLL may restrict ofatumumab's potential even further; GlaxoSmithKline will offer invaluable experience to Genmab and aid commercialization of ofatumumab; Forecasts to 2016; Datamonitor drug assessment summary; Zanolimumab (HuMax-CD4; Merck Serono/Genmab); Drug overview; Clinical trial data; Zanolimumab's Orphan Drug and Fast Track status reflects the high unmet needs in CTCL; Zanolimumab may also hold promise for PTCL patients; Datamonitor comments; The T-cell lymphoma market offers zanolimumab a limited commercial potential; Depletion of CD4+ T-cells by zanolimumab may render the patient susceptible to opportunistic infections; Forecasts to 2016; Datamonitor drug assessment summary; BIOVAXID (Accentia Biopharmaceuticals); Drug overview; Clinical trial data; BIOVAXID inches closer to approval in the US and European markets for follicular NHL; Phase III trial initiated in February 2000 is ongoing and continues to show favorable survival benefits of BIOVAXID; Possible association between a specific negative chromasomal translocation following vaccination and disease free survival in follicular NHL; Phase II results of BIOVAXID in mantle cell lymphoma are promising; Datamonitor comments; BIOVAXID competing with FavId and MyVax for first-to-market status; BIOVAXID's price-tag should reflect the anticipated competition and current treatment costs; Forecasts to 2016; Datamonitor drug assessment summary; FavId (Id-KLH; Favrille); Drug overview; Clinical trial data; FavId receives Fast Track status by the FDA for follicular NHL; Single-agent FavId demonstrates an objective response in indolent B-cell NHL; Favrille initiates FavId Phase III trial in DLBCL NHL; Datamonitor comments; FavId competing with BIOVAXID and MyVax to reach the market first; Favrille's lack of commercial experience will be a barrier to optimizing market penetration; Forecasts to 2016; Datamonitor drug assessment summary; MyVax (GTOP-99; Genitope); Drug overview; Clinical trial data; MyVax received Fast Track status for follicular NHL while Phase III clinical trial approaches completion; Phase II clinical trials show greater number of immune responses among previously untreated patients; Genitope initiates Phase I/II trial for MyVax in chronic lymphocytic leukemia; Follow-up Phase II data of MyVax in mantle cell and diffuse large B-cell lymphoma warrants further investigation; Datamonitor comments; Despite competition from BIOVAXID and FavId, MyVax increases its commercial potential by targeting an earlier stage treatment; With trials ongoing in CLL, MyVax may ultimately emerge as the most adaptable anti-idiotype vaccine; Forecasts to 2016; Datamonitor drug assessment summary; Comparison of anti-idiotype vaccines
APPENDIX: List of tables; List of figures; Methodology; Datamonitor forecast methodology; Datamonitor drug assessment summary; Abbreviations; Contributing experts; Key opinion leader interview transcripts; Bibliography; About Datamonitor; About Datamonitor Healthcare; Datamonitor Healthcare's therapy area capabilities; About the Disease analysis team; Disclaimer

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Pipeline Insight: Hematological Malignancies - Pursuit for the next blockbuster intensifies

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