FIGURE 2 GLOBAL MARKET FORECAST FOR ALS THERAPEUTICS, ($ MILLIONS) THE U.S. MARKETS (CONTINUED) ALS: BACKGROUND INFORMATION...

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1 INTRODUCTION... xix STUDY GOALS AND OBJECTIVES... xix REASONS FOR DOING THE STUDY... xix AUDIENCE FOR THE REPORT... xx SCOPE AND FORMAT... xx METHODOLOGY AND INFORMATION SOURCES... xxi ABOUT THE AUTHOR... xxi BCC INFORMATION AND RELATED BCC STUDIES... xxi DISCLAIMER... xxii BCC RESEARCH ONLINE SERVICES... xxii BCC ONLINE SERVICES... xxii DISCLAIMER... xxiii EXECUTIVE SUMMARY... xxiv SUMMARY TABLE GLOBAL MARKET FORECAST FOR BRANDED MEDICATIONS FOR NEURODEGENERATIVE DISORDERS, THROUGH 2012 ($ MILLIONS)... XXIV SUMMARY FIGURE GLOBAL MARKET FORECAST FOR BRANDED MEDICATIONS FOR NEURODEGENERATIVE DISORDERS, ($ MILLIONS... XXV OVERVIEW... 1 GOVERNMENT REGULATIONS... 1 INTRODUCTION... 1 DRUG DISCOVERY... 2 PRECLINICAL TESTING AND SUBMISSION OF AN IND... 3 CLINICAL TRIALS... 4 TIMELINE AND COSTS... 5 SUBMISSION OF AN NDA OR BLA... 5 POST-FDA APPROVAL... 6 NEW DRUG APPROVALS... 7 TABLE 1 DRUGS APPROVED BY THE FDA, TABLE 2 DRUGS APPROVED BY THE FDA, TABLE 3 NEW MOLECULAR/BIOLOGICAL ENTITIES APPROVED BY THE FDA, CONCLUSIONS... 9 TABLE 4 NME APPROVALS FOR NEUROLOGICAL DISORDERS, Conclusions (Continued) THE U.S. MARKETS FOR ALZHEIMER S DISEASE MEDICATIONS ABOUT ALZHEIMER S DISEASE TABLE 5 GLOBAL MARKET FORECAST FOR ALZHEIMER S DISEASE PHARMACEUTICALS, THROUGH 2012 ($ MILLIONS)... 11

2 FIGURE 1 GLOBAL MARKET FORECAST FOR ALZHEIMER S DISEASE PHARMACEUTICALS, ($ MILLIONS) ABOUT ALZHEIMER S DISEASE (CONTINUED) CURRENT BRANDED PHARMACEUTICALS THAT TREAT ALZHEIMER S DISEASE ARICEPT (DONEPEZIL HCL) TABLE 6 GLOBAL MARKET FORECAST FOR ARICEPT SALES, THROUGH 2012 ($ MILLIONS) Acetylcholinesterase Inhibitors Generic Competition COGNEX (TACRINE HYDROCHLORIDE) TABLE 7 GLOBAL MARKET FORECAST FOR COGNEX SALES, THROUGH 2012 ($ MILLIONS) EBIXA, NAMENDA, AND AXURA (MEMANTINE HYDROCHLORIDE) TABLE 8 GLOBAL MARKET FORECAST FOR MEMANTINE SALES, THROUGH 2012 ($ MILLIONS) NMDA Antagonists Patent Protection and Second-Generation NMDA Antagonists TABLE 9 NMDA ANTAGONISTS IN CLINICAL TRIALS EXELON (RIVASTIGMINE TARTRATE) TABLE 10 GLOBAL MARKET FORECAST FOR AD-RELATED EXELON SALES, THROUGH 2012 ($ MILLIONS) RAZADYNE (GALANTAMINE HYDROBROMIDE) TABLE 11 GLOBAL MARKET FORECAST FOR RAZADYNE SALES, THROUGH 2012 ($ MILLIONS) COMPOUNDS IN CLINICAL TRIALS FOR AD TABLE 12 POTENTIAL AD AGENTS IN PHASE III CLINICAL TRIALS Alzhemed Flurizan Neramexane Phenserine Failed AD Candidate Drugs COMPANY PROFILES FOREST LABORATORIES MEDIVATION MERZ PHARMA GMBH & CO. KGAA NEUROCHEM, INC SCIELE PHARMA, INC THE U.S. MARKETS FOR MEDICATIONS DIRECTED AT AMYOTROPHIC LATERAL SCLEROSIS TABLE 13 GLOBAL MARKET FORECAST FOR ALS THERAPEUTICS, THROUGH 2012 ($ MILLIONS)... 27

3 FIGURE 2 GLOBAL MARKET FORECAST FOR ALS THERAPEUTICS, ($ MILLIONS) THE U.S. MARKETS (CONTINUED) ALS: BACKGROUND INFORMATION ALS: BACKGROUND (CONTINUED) RILUTEK: THE SOLE FDA-APPROVED DRUG FOR ALS TABLE 14 U.S. MARKET FORECAST FOR RILUTEK, THROUGH 2012 ($ MILLIONS) ANIMAL MODELS OF ALS AND PRECLINICAL STUDIES STRENGTHS OF RODENT ALS MODELS LIMITATIONS OF RODENT ALS MODELS TABLE 15 ALS/SOD1 RODENT MODELS ALS DRUG CANDIDATES IN CLINICAL TRIALS SURVIVAL TABLE 16 A SAMPLE OF ALS DRUG CANDIDATES IN CLINICAL TRIALS TABLE 17 ALS DRUG CANDIDATES FOR WHICH NDAS HAVE BEEN FILED WITH THE FDA TABLE 18 ALS DRUG CANDIDATES THAT PEFORMED WELL IN CLINICAL TRIALS TABLE 19 A SAMPLE OF RECENT ALS DRUG CANDIDATES THAT DID NOT PEFORM WELL IN CLINICAL TRIALS TABLE 19 (CONTINUED) CURRENT COLLABORATIONS AND DRUG DISCOVERY PROGRAMS IN ALS DRUG DISCOVERY OR DEVELOPMENT TABLE 20 ALS DRUG CANDIDATES IN PRECLINICAL DEVELOPMENT AEOLUS PHARMACEUTICALS BIOGEN IDEC AND AMORFIX LIFE SCIENCES ISIS PHARMACEUTICALS METABOLON POWER3 MEDICAL PRODUCTS DIAGNOSTIC PRODUCTS FOR ALS TABLE 21 U.S. MARKETFORECAST FOR ALS DIAGNOSTIC PRODUCTS, THROUGH 2012 ($ MILLIONS) TABLE 22 RESEARCH EFFORTS AIMED AT BIOMARKERS FOR ALS COMPANY PROFILES AEOLUS PHARMACEUTICALS AMORFIX LIFE SCIENCES AVICENA GROUP, INC CYTRX CORP ISIS PHARMACEUTICALS, INC METABOLON POWER3 MEDICAL PRODUCTS... 48

4 MARKETS FOR MEDICATIONS DIRECTED AT HUNTINGTON S DISEASE TABLE 23 GLOBAL MARKET FORECAST FOR HUNTINGTON S DISEASE THERAPEUTIC AGENTS, THROUGH 2012 ($ MILLIONS) FIGURE 3 GLOBAL MARKET FORECAST FOR HUNTINGTON S DISEASE THERAPEUTIC AGENTS, ($ MILLIONS) HUNTINGTON S DISEASE: BACKGROUND INFORMATION TETRABENAZINE LIKELY THE FIRST PRODUCT TO GAIN FDA APPROVAL FOR HUNTINGTON S DISEASE TABLE 24 GLOBAL MARKET FORECAST FOR THE U.S. AND WORLDWIDE SALES FOR TETRABENAZINE, THROUGH 2012 ($ MILLIONS) HD DRUG CANDIDATES IN CLINICAL TRIALS TABLE 25 HD DRUG CANDIDATES IN CLINICAL TRIALS MIRAXION DIMEBON CREATINE/HD MINOCYCLINE OTHER AGENTS IN CLINICAL TRIALS FOR HD COMPANY PROFILES AMARIN CORP., PLC AVICENA GROUP, INC MEDIVATION PRESTWICK PHARMACEUTICALS Prestwick Pharmaceuticals (Continued) MARKETS FOR MEDICATIONS DIRECTED AT MULTIPLE SCLEROSIS TABLE 26 GLOBAL MARKET FORECAST FOR MULTIPLE SCLEROSIS THERAPEUTICS, THROUGH 2012 ($ MILLIONS) FIGURE 4 GLOBAL MARKET FORECAST FOR MULTIPLE SCLEROSIS THERAPEUTICS, ($ MILLIONS) ABOUT MS TABLE 27 OVERALL MARKET LEADERS IN MS THERAPEUTIC AGENTS, 2005 AND 2006 ($ MILLIONS) TABLE 28 U.S. MARKET LEADERS IN MS THERAPEUTICS, 2006 AND 2006 ($ MILLIONS) CURRENT PHARMACEUTICAL AGENTS THAT TREAT MULTIPLE SCLEROSIS AVONEX TABLE 29 GLOBAL MARKET FORECAST FOR AVONEX SALES, THROUGH 2012 ($ MILLIONS) TABLE 30 U.S. AVONEX SALES AS A PERCENTAGE OF TOTAL SALES, (PERCENT (%) ROUNDED TO THE NEAREST WHOLE VALUE) Factors Affecting Avonex Sales... 65

5 BETASERON TABLE 31 GLOBAL MARKET FORECAST FOR BETASERON, THROUGH 2012 ($ MILLIONS) TABLE 32 U.S. BETASERON SALES AS A PERCENTAGE OF TOTAL SALES, (ROUNDED TO THE NEAREST WHOLE VALUE) Factors Affecting Sales of Betaseron Generic competition Cross-Border Sales in the European Union Tysabri COPAXONE TABLE 33 GLOBAL MARKET FORECAST FOR COPAXONE, THROUGH 2012 ($ MILLIONS) TABLE 34 U.S. COPAXONE SALES AS A PERCENTAGE OF TOTAL SALES, (PERCENT (%) ROUNDED TO THE NEAREST WHOLE VALUE) Factors Affecting Copaxone s Future Revenues NOVANTRONE (MITOXANTRONE) TABLE 35 MARKET FORECAST FOR BRAND-NAME NOVANTRONE, THROUGH 2012 ($ MILLIONS) REBIF TABLE 36 GLOBAL MARKET FORECAST FOR REBIF, THROUGH 2012 ($ MILLIONS) TABLE 37 U.S. REBIF SALES AS A PERCENTAGE OF TOTAL SALES, (PERCENT (%) ROUNDED TO THE NEAREST WHOLE VALUE) Factors Affecting Rebif s Future Revenues TYSABRI TABLE 38 GLOBAL MARKET FORECAST FOR TYSABRI, THROUGH 2012 ($ MILLIONS) Tysabri (Continued) MS DRUG CANDIDATES IN CLINICAL TRIALS TABLE 39 A SAMPLE OF MS DRUG CANDIDATES IN CLINICAL TRIALS EARLY STAGE COLLABORATIONS IN MS Early Stage Collaborations (Continued) COMPANY PROFILES BIOGEN IDEC CHEMOCENTRYX, INC PARATEK PHARMACEUTICALS PDL BIOPHARMA, INC SERONO INTERNATIONAL SA Serono, Inc SYNTONIX PHARMACEUTICALS, INC TEVA PHARMACEUTICALS... 86

6 Teva North America Teva (Continued) MARKETS FOR MEDICATIONS DIRECTED AT PARKINSON S DISEASE TABLE 40 GLOBAL MARKET FORECAST FOR PARKINSON S DISEASE THERAPEUTICS, THROUGH 2012 ($ MILLIONS) FIGURE 5 GLOBAL MARKET FORECAST FOR PARKINSON S DISEASE THERAPEUTICS, ($ MILLIONS) CHARACTERISTICS OF THE PARKINSON S DISEASE THERAPEUTICS INDUSTRY ABOUT PARKINSON S DISEASE SHORTCOMINGS OF LEVODOPA THERAPY AND NEW MEDICATIONS IMPROVEMENTS TO LEVODOPA MONOTHERAPY Dopamine Agonists Anticholinergics MAO-B inhibitors COMT Inhibitors CURRENT BRANDED PHARMACEUTICAL AGENTS THAT TREAT PD APOKYN/APOMORPHINE TABLE 41 GLOBAL MARKET FORECAST FOR APOKYN/APOMORPHINE, THROUGH 2012 ($ MILLIONS) INJECTABLE APOMORPHINE: BARRIERS TO MARKET PENETRATION AZILECT (RASAGILINE) TABLE 42 GLOBAL MARKET FORECAST FOR AZILECT, THROUGH 2012 ($ MILLIONS) MIRAPEX (PRAMIPEXOLE) TABLE 43 GLOBAL MARKET FORECAST FOR MIRAPEX, THROUGH 2012 ($ MILLIONS) MIRAPEX AND COMPULSIVE BEHAVIORS MIRAPEX AND RESTLESS LEGS SYNDROME REQUIP (ROPINIROLE HYDROCHLORIDE) TABLE 44 GLOBAL MARKET FORECAST FOR REQUIP FOR PARKINSON S DISEASE, THROUGH 2012 ($ MILLIONS) TABLE 45 U.S. PD-RELEATED REQUIP SALES AS A PERCENTAGE OF TOTAL SALES, (PERCENT (%) ROUNDED TO THE NEAREST WHOLE VALUE) REQUIP AND COMPULSIVE BEHAVIORS STALEVO AND COMTAN TABLE 46 GLOBAL MARKET FORECAST FOR COMTAN/STALEVO, THROUGH 2012 ($ MILLIONS) TASMAR (TOLCAPONE)

7 TABLE 47 GLOBAL MARKET FORECAST FOR TASMAR, THROUGH 2012 ($ MILLIONS) ZELAPAR (SELEGILINE HCL) TABLE 48 GLOBAL MARKET FORECAST FOR ZELAPAR, THROUGH 2012 ($ MILLIONS) PD DRUG CANDIDATES IN CLINICAL TRIALS TABLE 49 A SAMPLE OF PD DRUG CANDIDATES IN CLINICAL TRIALS E Direct-to-the-Brain Drug Delivery COMPANY PROFILES ORION PHARMACEUTICALS TEVA PHARMACEUTICALS Teva North America VALEANT PHARMACEUTICALS VERNALIS, PLC MARKETS FOR MEDICATIONS DIRECTED AT MUSCULAR DYSTROPHY AND RARE NEURODEGENERATIVE DISORDERS TABLE 50 GLOBAL MARKET FORECAST FOR MUSCULAR DYSTROPHY-RELATED THERAPEUTIC AGENTS, THROUGH 2011 ($ MILLIONS) FIGURE 6 GLOBAL MARKET FORECAST FOR MUSCULAR DYSTROPHY-RELATED THERAPEUTIC AGENTS, ($ MILLIONS) THE MUSCULAR DYSTROPHIES BACKGROUND INFORMATION MD DRUG CANDIDATES IN CLINICAL TRIALS TABLE 51 MD DRUG CANDIDATES IN CLINICAL TRIALS PTC Stamulumab Other Agents in Clinical Trials for MD RARE NEURODEGENERATIVE DISORDERS TABLE 52 GLOBAL MARKET FORECAST FOR RARE NEURODEGENERATIVE DISORDERS, THROUGH 2012 ($ MILLIONS) FIGURE 7 GLOBAL MARKET FORECAST FOR RARE NEURODEGENERATIVE DISORDERS, ($ MILLIONS) CANDIDATES FOR RARE NEURODEGENERATIVE DISORDERS IN CLINICAL TRIALS TABLE 53 CANDIDATES IN CLINICAL TRIALS FOR RARE DISORDERS Batten Disease Niemann-Pick Disease Adrenoleukodystrophy

8 Lorenzo s Oil COMPANY PROFILES GENZYME Genzyme (Continued) PTC THERAPEUTICS STEM CELLS, INC WYETH PHARMACEUTICALS Wyeth Pharmaceuticals (Continued) U.S. PATENT ANALYSIS INTRODUCTION AMYOTROPHIC LATERAL SCLEROSIS (ALS) TABLE 54 ALS-RELATED TECHNOLOGY: U.S. PATENT TRENDS, * TABLE 55 ALS-RELATED PATENTS BY USE CATEGORY GENERAL TRENDS TRENDS BY ASSIGNEE TABLE 56 ALS-RELATED PATENTS BY ASSIGNEE CATEGORY TABLE 56 (CONTINUED) TABLE 57 ALS-RELATED PATENTS BY COMPANY PATENT APPLICATION TRENDS TABLE 58 ALS-RELATED PATENT APPLICATIONS BY USE CATEGORY TABLE 59 ALS-RELATED TECHNOLOGY: U.S. PATENT APPLICATION TRENDS, TRENDS BY COUNTRY HUNTINGTON S DISEASE (HD) TABLE 60 HUNTINGTON S DISEASE-RELATED TECHNOLOGY: U.S. PATENT TRENDS, TABLE 61 HD-RELATED PATENTS BY USE CATEGORY TRENDS BY ASSIGNEE TABLE 62 HD-RELATED PATENTS BY ASSIGNEE CATEGORY TABLE 63 HD-RELATED PATENTS BY COMPANY ALZHEIMER S DISEASE (AD) TABLE 64 ALZHEIMER S DISEASE-RELATED TECHNOLOGY: U.S. PATENT TRENDS, TABLE 65 AD-RELATED PATENTS BY USE CATEGORY TRENDS BY ASSIGNEE TABLE 66 AD-RELATED PATENTS BY ASSIGNEE CATEGORY TABLE 67 AD-RELATED PATENTS BY COMPANY PARKINSON S DISEASE (PD) TABLE 68 PARKINSON S DISEASE-RELATED TECHNOLOGY: U.S. PATENT TRENDS, TABLE 69 PD-RELATED PATENTS BY USE CATEGORY TRENDS BY ASSIGNEE

9 TABLE 70 PD-RELATED PATENTS BY ASSIGNEE CATEGORY TABLE 71 PD-RELATED PATENTS BY COMPANY MULTIPLE SCLEROSIS (MS) TABLE 72 MULTIPLE SCLEROSIS-RELATED TECHNOLOGY: U.S. PATENT TRENDS, TABLE 73 MS-RELATED PATENTS BY USE CATEGORY TRENDS BY ASSIGNEE TABLE 74 MS-RELATED PATENTS BY ASSIGNEE CATEGORY TABLE 75 MS-RELATED PATENTS BY COMPANY MUSCULAR DYSTROPHY (MD) TABLE 76 MUSCULAR DYSTROPHY-RELATED TECHNOLOGY: U.S. PATENT TRENDS, TABLE 77 MD-RELATED PATENTS BY USE CATEGORY TRENDS BY ASSIGNEE TABLE 78 MD-RELATED PATENTS BY ASSIGNEE CATEGORY TABLE 79 MD-RELATED PATENTS BY ORGANIZATION MD-RELATED PATENT APPLICATIONS TABLE 80 MUSCULAR DYSTROPHY-RELATED TECHNOLOGY: U.S. PATENT APPLICATION TRENDS, TABLE 81 MD-RELATED PATENT APPLICATIONS BY USE CATEGORY A RARE DISORDER: ATAXIA-TELANGIECTASIA TABLE 82 RARE DISORDER PATENT APPLICATIONS BY DISEASE TABLE 83 ATAXIA-TELANGIECTASIA-RELATED PATENTS BY USE CATEGORY