CHAPTER SIX: SMALL MOLECULE MS DRUGS... 37

Transcription

1 CHAPTER ONE: INTRODUCTION... 1 STUDY GOALS AND OBJECTIVES... 1 REASONS FOR DOING THIS STUDY... 2 INTENDED AUDIENCE... 2 SCOPE OF REPORT... 3 METHODOLOGY... 3 INFORMATION SOURCES... 4 AUTHOR S CREDENTIALS... 4 RELATED BCC REPORTS... 4 BCC ONLINE SERVICES... 4 DISCLAIMER... 5 CHAPTER TWO: SUMMARY... 6 SUMMARY... 6 SUMMARY TABLE GLOBAL MARKET FOR MS DISEASE- MODIFYING PRODUCTS, THROUGH 2015 ($ MILLIONS)... 7 SUMMARY FIGURE GLOBAL MARKET FOR THERAPIES TREATING MS, ($ MILLIONS)... 8 CHAPTER THREE: OVERVIEW... 9 DISEASE CHARACTERISTICS... 9 HISTORY AND DISCOVERY OF MS... 9 SYMPTOMS OF MS PREVALENCE AND INCIDENCE OF MS TABLE 1 MS STATISTICS FROM AROUND THE WORLD, DIAGNOSIS OF MS DIAGNOSIS OF MS (CONTINUED) TYPES OF MS PROGNOSIS TREATMENTS FOR MS TYPES OF MS THERAPIES TABLE 2 CURRENTLY APPROVED MS THERAPIES ON THE MARKET TABLE 3 FEATURES OF SMALL MOLECULE VERSUS BIOLOGIC MS DISEASE-MODIFYING THERAPIES TABLE 3 (CONTINUED) TABLE 4 WHOLESALE ACQUISITION COST OF SELECTED THERAPIES FOR MS, 2009 ($) TARGETS OF MS THERAPIES VLA-4 (CD49D AND CD29) S1P CD IL-2 (CD25, CD122, AND CD132) CD MYELIN BASIC PROTEIN-REACTIVE T-CELLS... 22

2 SIDE EFFECTS OF MS THERAPIES TABLE 5 COMPARISON OF SIDE EFFECT PROFILES OF MARKETED AND LATE-CLINICAL STAGE THERAPIES FOR MS MS THERAPIES IN CLINICAL DEVELOPMENT TABLE 6 MS THERAPIES IN CLINICAL TRIALS, ORDERED BY PHASE TABLE 6 (CONTINUED) TABLE 6 (CONTINUED) TABLE 7 MS THERAPIES IN DEVELOPMENT, BY PHASE (NUMBER/%) FIGURE 1 MS THERAPIES IN ACTIVE CLINICAL TRIALS BY PRIMARY TARGET (NUMBER) FIGURE 1 (CONTINUED) TABLE 8 COMPANIES WITH MORE THAN ONE MS THERAPY IN ACTIVE CLINICAL TRIALS OR ON THE MARKET* CLINICAL TRIAL EFFICACY OUTCOME MEASUREMENTS IN MS STUDIES Relapse Rate EDSS Gadolinium-Enhanced (GdE) Lesions on MRI TABLE 9 EFFICACY RESULTS OF MS DISEASE-MODIFYING THERAPIES CHAPTER FOUR: TECHNOLOGY OVERVIEW TABLE 10 GLOBAL MARKET FOR MS DISEASE-MODIFYING THERAPIES BY CLASS, THROUGH 2015 ($ MILLIONS) TABLE 11 GLOBAL SALES OF MS DISEASE-MODIFYING THERAPIES BY FORMULATION, THROUGH 2015 ($ MILLIONS) TABLE 12 ADMINISTRATION PROFILES OF THERAPIES APPROVED TO TREAT MS CHAPTER FIVE: PATENT EVALUATION PATENT LENGTH AND MARKET EXCLUSIVITY ORPHAN DRUG EXCLUSIVITY REBIF AND AVONEX S ORPHAN DRUG MARKETING EXCLUSIVITY TABLE 13 U.S. PATENT EXPIRATION DATES OF THERAPIES FOR MS ON THE MARKET OR IN PHASE III DEVELOPMENT TABLE 13 (CONTINUED) BIOGEN IDEC AND THE INTERFERON BETA PATENTS SMALL MOLECULE MS DISEASE-MODIFYING PRODUCTS AND GENERIC DRUGS CHAPTER SIX: SMALL MOLECULE MS DRUGS... 37

3 MARKET OVERVIEW TABLE 14 GLOBAL MARKET FOR SMALL MOLECULE MS DISEASE- MODIFYING DRUGS BY INDIVIDUAL DRUG, THROUGH 2015 ($ MILLIONS) SMALL MOLECULE MS DISEASE-MODIFYING DRUGS COPAXONE (GLATIRAMER ACETATE, COPOLYMER 1) TABLE 15 COPAXONE PIVOTAL STUDY RESULTS Copaxone (Continued) TABLE 16 GLOBAL MARKET FOR COPAXONE BY REGION, THROUGH 2015 ($ MILLIONS) NOVANTRONE (MITOXANTRONE) Novantrone (Mitoxantrone) (Continued) TABLE 17 GLOBAL MARKET FOR NOVANTRONE BY REGION, THROUGH 2015 ($ MILLIONS) M356 (GLATIRAMER ACETATE) M356 (Glatiramer Acetate) (Continued) GILENIA (FINGOLIMOD, FTY720) Gilenia (Fingolimod, FTY720) (Continued) TABLE 18 GILENIA PIVOTAL PHASE III STUDY RESULTS Gilenia (Fingolimod, FTY720) (Continued) TABLE 19 GLOBAL MARKET FOR GILENIA BY REGION, THROUGH 2015 ($ MILLIONS) LAQUINIMOD (ABR ) TABLE 20 LAQUINIMOD PIVOTAL PHASE III STUDIES Laquinimod (ABR ) (Continued) TABLE 21 GLOBAL MARKET FOR LAQUINIMOD BY REGION, THROUGH 2015 ($ MILLIONS) MYLINAX, MOVECTRO (ORAL CLADRIBINE) Mylinax, Movectro (Oral Cladribine) (Continued) Mylinax, Movectro (Oral Cladribine) (Continued) Mylinax, Movectro (Oral Cladribine) (Continued) TABLE 22 GLOBAL MARKET FOR MYLINAX, BY REGION, THROUGH 2015 ($ MILLIONS) TERIFLUNOMIDE (HMR1726) TABLE 23 GLOBAL MARKET FOR TERIFUNOMIDE BY REGION, THROUGH 2015 ($ MILLIONS) BG-12 (DIMETHYL FUMARATE, BG00012) TABLE 24 GLOBAL MARKET FOR BG-12 BY REGION, THROUGH 2015 ($ MILLIONS) FIRATEGRAST (SB683699, T-0047) BAF ONO ACT MASITINIB... 64

4 PLENEVA (BGC ) TRIMESTA (ESTRIOL) KETAS (MN-166, AV-411, IBUDILAST) TABLE 25 MN-166 PHASE II STUDY RESULTS ELND PI CS CDP323 (UCB-323) DIRUCOTIDE (MBP8298) CHAPTER SEVEN: BIOLOGICS FOR MS OVERVIEW TABLE 26 BIOLOGICS FOR MS BY CLASS TABLE 27 GLOBAL MARKET FOR BIOLOGIC FOR MS BY INDIVIDUAL DRUG, THROUGH 2015 ($ MILLIONS) MABS ANTIBODIES FOR MS TABLE 28 BENEFITS AND CHARACTERISTICS OF MABS TABLE 29 SELECTED ANTIBODIES IN DEVELOPMENT FOR MS TYSABRI (NATALIZUMAB) TABLE 30 TYSABRI SENTINEL AND AFFIRM STUDY RESULTS Tysabri (Natalizumab) (Continued) Tysabri (Natalizumab) (Continued) TABLE 31 GLOBAL MARKET FOR TYSABRI BY REGION, THROUGH 2015 ($ MILLIONS) CAMPATH/MABCAMPATH (ALEMTUZUMAB) Campath/MabCampath (Alemtuzumab) (Continued) TABLE 32 ALEMTUZUMAB PHASE II STUDY RESULTS Campath/MabCampath (Alemtuzumab) (Continued) TABLE 33 GLOBAL MARKET FOR CAMPATH IN MS BY REGION, THROUGH 2015 ($ MILLIONS) ZENAPAX (DACLIZUMAB) TABLE 34 DACLIZUMAB CHOICE STUDY RESULTS Zenapax (Daclizumab) (Continued) TABLE 35 GLOBAL MARKET FOR DACLIZUMAB IN MS BY REGION, THROUGH 2015 ($ MILLIONS) RITUXAN, MABTHERA (RITUXIMAB) Rituxan, MabThera (Rituximab) (Continued) OCRELIZUMAB (RG1594, PRO70769) ARZERRA (OFATUMUMAB) AIN LY ABT BIIB ANTI-CD100 MAB USTEKINUMAB (CNTO1275)... 90

5 INTERFERON BETAS INTERFERON BETAS (CONTINUED) TABLE 36 GLOBAL MARKET FOR INTERFERON BETAS IN MS BY REGION, THROUGH 2015 ($ MILLIONS) BETASERON, BETAFERON (INTERFERON BETA-1B, BAY ) TABLE 37 TWO-YEAR BETASERON RRMS STUDY RESULTS TABLE 38 THREE-YEAR BETASERON RRMS STUDY RESULTS TABLE 39 GLOBAL MARKET FOR BETASERON BY REGION, THROUGH 2015 ($ MILLIONS) AVONEX (INTERFERON BETA-1A) TABLE 40 AVONEX PIVOTAL PHASE III STUDY RESULTS TABLE 41 GLOBAL MARKET FOR AVONEX BY REGION, THROUGH 2015 ($ MILLIONS) REBIF (INTERFERON BETA-1A) Rebif (Interferon Beta-1A) (Continued) TABLE 42 GLOBAL MARKET FOR REBIF BY REGION, THROUGH 2015 ($ MILLIONS) EXTAVIA (INTERFERON BETA-1B) Extavia (Interferon Beta-1b) (Continued) TABLE 43 GLOBAL MARKET FOR EXTAVIA BY REGION, THROUGH 2015 ($ MILLIONS) CINNOVEX (INTERFERON BETA-1A) BIFERONEX (INTERFERON BETA-1A) Biferonex (Interferon Beta-1a) (Continued) PEG INTERFERON BETA-1A, BIIB017) TV THERAPEUTIC VACCINES TOVAXIN ATX-MS RTL BHT NEUROVAX XTOLL (CHAPERONIN 10, CPN10) XToll (Chaperonin 10, Cpn10) (Continued) NTX-488 (PROLACTIN) ATL1102 (FORMERLY TV-1102) ATL1102 (formerly TV-1102) (Continued) CHAPTER EIGHT: DISEASE APPLICATIONS OVERVIEW TABLE 44 LABELS OF APPROVED DISEASE-MODIFYING THERAPIES FOR MS TABLE 44 (CONTINUED) MS SUBGROUPS

6 RRMS SPMS PPMS PPMS (Continued) PRMS THE MARKET FOR MS THERAPIES BY MS DISEASE SUBGROUP CLASSIFICATION CHAPTER NINE: MAJOR COMPANIES AND MARKET SHARE MARKET SHARE TABLE 45 FORECASTED SALES AND MARKET SHARE OF COMPANIES WITH AGENTS FOR MS ON THE MARKET, 2010 AND 2015 ($ MILLIONS/%) COMPANY PROFILES AB SCIENCE AB Science USA ABBOTT LABORATORIES ACTELION ACTIVE BIOTECH ADEONA PHARMACEUTICALS ANTISENSE THERAPEUTICS APITOPE ARTIELLE IMMUNOTHERAPEUTICS BAYER AG Bayer (U.S. Location) BIOGEN IDEC BTG CBIO CINNAGEN DAIICHI SANKYO ELAN ELI LILLY FAST FORWARD, LLC FIVE PRIME THERAPEUTICS GENMAB Genmab (U.S. Location) GENZYME GLAXOSMITHKLINE GlaxoSmithKline (U.S. Location) MEDICINOVA MERCK KGAA (MERCK SERONO) MITSUBISHI TANABE PHARMA MOMENTA PHARMACEUTICALS NOVARTIS ONO PHARMA

7 OPEXA THERAPEUTICS PEPTIMMUNE PFIZER ROCHE SANOFI-AVENTIS STEM CELL THERAPEUTICS TEVA PHARMACEUTICAL INDUSTRIES CHAPTER TEN: INTERNATIONAL ASPECTS AND REGULATORY ISSUES MARKET BY REGION TABLE 46 GLOBAL FORECAST FOR MS DISEASE-MODIFYING THERAPIES BY REGION, THROUGH 2015 ($ MILLIONS) MARKET BY REGION (CONTINUED) UNITED STATES The FDA Modernization Act of Fast-Track Status Special Protocol Assessments New Surveillance and Safety Requirements Approvable Letters Consumer Confidence Medicare Other Healthcare Changes EUROPEAN UNION New Drug Approvals and Innovation Drug Reimbursement and Reference Pricing Relocation of R&D JAPAN New Drug Approvals Regulatory Climate Relationship between Academia and Industry CHAPTER ELEVEN: FUTURE DIRECTIONS THE ROLE OF BIOMARKERS AND PHARMACOGENOMICS COMBINATION THERAPIES THE EFFECT OF GENERIC MS THERAPIES ON PRICE THE EFFECT OF GENERIC MS THERAPIES ON PRICE (CONTINUED)