Pharmaceutical Research Companies Are Developing More Than...

2010

Mental IllnessesM E D I C I N E S I N D E V E L O P M E N T F O R

Report

P R E S E N T E D B Y A M E R I C A ’ S P H A R M A C E U T I C A L R E S E A R C H C O M P A N I E S

America’s pharmaceutical research and biotech-nology companies are currently developing 313medicines to help the nearly 60 million American

adults today suffering from some form of mental illness—from Alzheimer’s disease to depression and fromschizophrenia to addictive disorders, such as dependenceon alcohol or drugs. All of the medicines are either inclinical trials or awaiting review by the Food and DrugAdministration.

Over the past half century, pharmaceutical research hashelped transform mental illnesses from misunderstoodcauses of shame and fear into often highly treatable con-ditions. For example, medicines for treating depression arehelping thousands of people suffering from this disease liveproductive lives. Breakthrough schizophrenia medicinesenable most patients to be treated in the community ratherthan institutional settings, and medicines for Alzheimer’sdisease are helping elderly people maintain their inde-pendence longer.

Despite such progress, mental illnesses continue toexact a heavy human and economic toll. The NationalInstitute of Mental Health (NIMH) estimates that 1 in 4American adults suffer from a diagnosable mental disor-der. According to the World Health Organization, morethan 300 million people worldwide suffer from a mentalhealth disorder. Mental illness, including suicide, accountsfor more than 15 percent of the burden of disease inestablished market economies, which is more than thedisease burden caused by all cancers. According to astudy funded by the NIMH, serious mental illnesses costthe United States more than $317 billion annually in lostwages, health care expenditures and disability benefits.

New medicines today in the research and developmentpipeline offer hope of reducing the human and economiccosts of mental illnesses. They include: 71 for depression(mood disorders), which affects nearly 21 million Americans;33 for addictive disorders, including dependence onalcohol, tobacco or illicit drugs; 38 medicines for anxietydisorders, which affect more than 40 million adults ages18 and older; 90 for dementias, including Alzheimer’sdisease, which affects more than 5 million Americans;and 54 for schizophrenia, which strikes some 2.4 millionAmerican adults each year. Other potential medicinestarget attention-deficit/hyperactivity disorder, autism,eating disorders, personality disorders, premenstrualdisorders and sleep disorders.

Examples of some medicines now being tested to treatmental illnesses include:

• A medicine to potentially treat the varied symptomsassociated with schizophrenia, with diminished sideeffects common with current treatments.

• A medicine designed to remove beta amyloid proteinfrom the brain and prevent or reverse progression ofAlzheimer’s disease.

Researching and developing new medicines remains arisky investment and lengthy process—costing, on average,$1.3 billion and taking between 10 and 15 years tobring a new medicine to patients. But advances in ourunderstanding of mental illnesses and how to treat themhave allowed America’s pharmaceutical research andbiotechnology companies to conduct the cutting-edgeresearch needed to reduce the destructive toll of thesedisorders and to allow more patients to lead healthier,happier, more productive lives.

David E. Wheadon, M.D.Senior Vice President, Scientific & Regulatory AffairsPhRMA

Pharmaceutical Research Companies Are DevelopingMore Than 300 Medicines to Treat Mental Illnesses

Addictive Disorders

Schizophrenia

Premenstrual Disorders

Attention-Deficit/Hyperactivity Disorder

Personality Disorders

Eating Disorders

Depression

Anxiety Disorders 38

20

71

Dementias 90

33

2

2

54

33

Sleep Disorders 21

Other 3

Developmental Disorders 6

* Some medicines are in development for more than one disorder.

M E D I C I N E S I N D E V E L O P M E N T F O R M E N T A LI L L N E S S E S *

Medicines in Development for Mental IllnessesA D D I C T I V E D I S O R D E R SProduct Name Sponsor Indication Development Status*

468816 GlaxoSmithKline nicotine dependence Phase IIRsch. Triangle Park, NC (888) 825-5249

618334 GlaxoSmithKline drug dependence Phase IRsch. Triangle Park, NC (888) 825-5249

AFQ056 Novartis Pharmaceuticals smoking withdrawal Phase I completedEast Hanover, NJ (see also developmental disorders) (888) 669-6682

alcohol Eli Lilly alcohol dependence Phase Idependence Indianapolis, IN (800) 545-5979therapy

alcohol Eli Lilly alcohol dependence Phase Idependence Indianapolis, IN (800) 545-5979therapy

ALKS 29 Alkermes alcohol dependence Phase I/II(baclofen/ALKS 33 Cambridge, MA (617) 494-0171combination)

ALKS 33 Alkermes alcohol dependence Phase IICambridge, MA (617) 494-0171

--------------------------------------------------------------------------------------drug dependence, Phase Iimpulse control disorder (617) 494-0171

ARD-1600 Aradigm nicotine dependence Phase I(nicotine Hayward, CA (510) 265-9000inhalation)

CPP-109 Catalyst Pharmaceutical cocaine dependence Phase II(vigabatrin) Coral Gables, FL (Fast Track) (305) 529-2522

DOV-102677 DOV Pharmaceutical alcohol dependence Phase ILittle Falls, NJ (732) 907-3600

EMB-001 Embera NeuroTherapeutics cocaine dependence Phase I completedShreveport, LA (318) 213-0198

KRL-901 Krele Pharmaceuticals alcohol dependence in clinical trialsSaddle River, NJ (212) 923-3400

lobeline Yaupon Therapeutics methamphetamine dependence Phase IIRadnor, PA (see also ADHD) (610) 975-9290

lofexidine US WorldMeds opioid dependence Phase IIILouisville, KY (502) 753-2094

MK-0594 Merck alcohol dependence Phase IIWhitehouse Station, NJ (800) 672-6372

MS-166 MediciNova opioid dependence Phase IISan Diego, CA (858) 373-1500

2 M E D I C I N E S I N D E V E L O P M E N T F O R Mental Illnesses 2010

* For more information about a specific medicine in this report, please call the telephone number listed.

A D D I C T I V E D I S O R D E R SProduct Name Sponsor Indication Development Status

nalmefene Lundbeck alcohol dependence Phase IIIDeerfield, IL (800) 455-1141

--------------------------------------------------------------------------------------nicotine dependence Phase II

(800) 455-1141

Namenda® Forest Laboratories impulse control disorder Phase IImemantine New York, NY (compulsive gambling) (800) 947-5227

NanoBUP™ Nanotherapeutics opioid dependence Phase I completedbuprenorphine/ Alachua, FL (386) 462-9663naloxone

neboglamine Rottapharm | Madaus cocaine dependence Phase II(CR 2249) Monza, Italy www.rotta.com

NIC 002 Novartis Pharmaceuticals nicotine dependence Phase IIEast Hanover, NJ (888) 669-6682

NicVAX™ GlaxoSmithKline nicotine dependence Phase IIInicotine abuse Rsch. Triangle Park, NC (Fast Track) (888) 825-5249vaccine Nabi Biopharmaceuticals

Boca Raton, FL

OpRA Eli Lilly alcohol dependence Phase IIIndianapolis, IN (800) 545-5979

Probuphine® Titan Pharmaceuticals opioid dependence Phase IIIbupenorphine South San Francisco, CA (650) 244-4990

Risperdal® Consta® Johnson & Johnson methamphetamine dependence Phase IIrisperidone Pharmaceutical Research (see also schizophrenia) (800) 817-5286(injectable) & Development

Raritan, NJ

SCH 900435 Merck alcohol dependence Phase IIWhitehouse Station, NJ (prevention of relapse) (800) 672-6372

(see also schizophrenia)

Seroquel XR® AstraZeneca alcohol dependence Phase II completedquetiapine Wilmington, DE (see also anxiety disorders, (800) 236-9933fumarate depression, personality disorders)extended-release

SYN-117 Roche drug dependence Phase I/IINutley, NJ (see also anxiety disorders) (973) 235-5000Synosia Therapeutics (650) 244-4850South San Francisco, CA

TA-CD Celtic Pharma cocaine dependence Phase II(cocaine abuse Hamilton, Bermuda www.celticpharma.comvaccine)

TO-2060 Transcept Pharmaceuticals alcohol dependence Phase I(olanzepine/ Pt. Richmond, CA (510) 215-3500ondansetron)

Topamax® Johnson & Johnson alcohol dependence Phase IItopiramate Pharmaceutical Research (see also anxiety disorders) (800) 817-5286

& DevelopmentRaritan, NJ

3M E D I C I N E S I N D E V E L O P M E N T F O R Mental Illnesses 2010

A D D I C T I V E D I S O R D E R SProduct Name Sponsor Indication Development Status

vigabatrin Lundbeck cocaine dependence Phase IIDeerfield, IL (Fast Track) (847) 282-1000

--------------------------------------------------------------------------------------methamphetamine dependence Phase I

(847) 282-1000

Vivitrol® Alkermes opioid dependence application submittednaltrexone Cambridge, MA (617) 494-0171controlled-release

A N X I E T Y D I S O R D E R SProduct Name Sponsor Indication Development Status

163090 GlaxoSmithKline anxiety Phase IRsch. Triangle Park, NC (see also depression) (888) 825-5249



586529 GlaxoSmithKline anxiety Phase IRsch. Triangle Park, NC (see also depression) (888) 825-5249Neurocrine Biosciences (858) 617-7600San Diego, CA


ABT-436 Abbott Laboratories anxiety Phase IAbbott Park, IL (see also depression) (847) 937-6100

ADX71149 Johnson & Johnson anxiety Phase IPharmaceutical Research (see also schizophrenia) (800) 817-5286& DevelopmentRaritan, NJ

AVN101 Avineuro Pharmaceuticals anxiety Phase IISan Diego, CA (see also dementias) (858) 436-1537

AVN397 Avineuro Pharmaceuticals anxiety Phase IISan Diego, CA (see also dementias) (858) 436-1537

AZD2327 AstraZeneca anxiety Phase IIWilmington, DE (see also depression) (800) 236-9933

AZD6280 AstraZeneca anxiety Phase IWilmington, DE (800) 236-9933

BCI-540 BrainCells anxiety Phase IISan Diego, CA (see also depression) (858) 812-7700

emicerfont GlaxoSmithKline anxiety Phase IIRsch. Triangle Park, NC (see also depression) (888) 825-5249Neurocrine Biosciences (858) 617-7600San Diego, CA



KRL-104 Krele Pharmaceuticals generalized anxiety disorder in clinical trialsSaddle River, NJ (212) 923-3400

Lexapro® Forest Laboratories panic disorder, social phobia application submittedescitalopram New York, NY (800) 947-5227

Lu AA21004 Lundbeck generalized anxiety disorder Phase IIIDeerfield, IL (see also depression) (800) 455-1141

Lu AA24530 Lundbeck generalized anxiety disorder Phase IDeerfield, IL (see also depression) (800) 455-1141

Lunesta® Sepracor generalized anxiety disorder Phase IIeszopiclone Marlborough, MA (see also sleep disorders) (508) 481-6700

Lyrica® Pfizer generalized anxiety disorder application submittedpregbalin New York, NY (860) 732-5156

MDMA Multidisciplinary post-traumatic stress disorder Phase IAssociation for (831) 421-6362Psychedelic StudiesSanta Cruz, CA

NSD-721 GlaxoSmithKline social anxiety disorder Phase IRsch. Triangle Park, NC (888) 825-5249NeuroSearchBallerup, Denmark

NSD-788 GlaxoSmithKline anxiety Phase IRsch. Triangle Park, NC (see also depression) (888) 825-5249NeuroSearchBallerup, Denmark

orvepitant GlaxoSmithKline post-traumatic stress disorder Phase IIRsch. Triangle Park, NC (see also depression) (888) 825-5249

PH94B Pherin Pharmaceuticals acute anxiety disorder Phase IIRedwood City, CA (650) 568-1587

Seroquel XR® AstraZeneca generalized anxiety disorders application submittedquetiapine Wilmington, DE (see also addictive disorders, (800) 236-9933fumarate depression, personality disorders)extended-release

SPN 805 Supernus Pharmaceuticals anxiety Phase IRockville, MD (301) 838-2500

SPN 808 Supernus Pharmaceuticals anxiety Phase IRockville, MD (301) 838-2500

SRX 246 Azevan Pharmaceuticals traumatic stress disorders Phase IBethlehem, PA (610) 419-1057

SSR125543 sanofi-aventis post-traumatic stress disorder Phase IBridgewater, NJ (see also depression) (800) 633-1610

Strattera® Eli Lilly social phobia Phase II/IIIatomoxetine Indianapolis, IN (800) 545-5979

SYN-117 Roche post-traumatic stress disorder Phase IINutley, NJ (see also addictive disorders) (973) 235-5000Synosia Therapeutics (650) 244-4850South San Francisco, CA



TGWOOAA Fabre-Kramer generalized anxiety disorder, Phase IIPharmaceuticals social phobia (713) 975-6900Houston, TX

TIK-101 Tikvah Therapeutics obsessive-compulsive disorder, Phase II(cycloserine) Atlanta, GA panic disorder, phobic disorders, (404) 920-3180

post-traumatic stress disorder, social phobia

Topamax® Johnson & Johnson post-traumatic stress disorder in clinical trialstopiramate Pharmaceutical Research (see also addictive disorders) (800) 817-5286

& DevelopmentRaritan, NJ

TriRima™ CeNeRx BioPharma anxiety Phase I(CX157) Rsch. Triangle Park, NC (see also depression) (919) 234-4650

verucerfont GlaxoSmithKline post-traumatic stress disorder, Phase IIRsch. Triangle Park, NC social phobia (888) 825-5249Neurocrine Biosciences (see also depression) (858) 617-7600San Diego, CA

YKP-3089 SK Drug Development anxiety Phase IICenter (973) 227-3939Fairfield, NJ

Zoloft® Pfizer generalized anxiety disorder in clinical trialssertraline New York, NY (see also eating disorders) (860) 732-5156

A T T E N T I O N - D E F I C I T / H Y P E R A C T I V I T Y D I S O R D E RProduct Name Sponsor Indication Development Status

amfetamine Noven Pharmaceuticals attention-deficit/hyperactivity Phase Itransdermal Miami, FL disorder (ADHD) (305) 253-5099

AZD1446 AstraZeneca ADHD Phase II(TC-6683) Wilmington, DE (see also dementias) (800) 236-9933

Targacept (336) 480-2100Winston-Salem, NC

AZD3480 AstraZeneca ADHD Phase II(TC-1734) Wilmington, DE (800) 236-9933


Clonicel® Shionogi Pharma ADHD in adolescents and application submittedclonidine Atlanta, GA children (800) 461-3696(controlled release)

Daytrana® Shire ADHD in adolescents application submittedmethylphenidate Wayne, PA (800) 828-2088transdermal system --------------------------------------------------------------------------------------

ADHD in adults Phase III(800) 828-2088

droxidopa Chelsea Therapeutics ADHD Phase IICharlotte, NC (combination therapy) (704) 341-1516


A T T E N T I O N - D E F I C I T / H Y P E R A C T I V I T Y D I S O R D E RProduct Name Sponsor Indication Development Status

Intuniv™ Shire ADHD in adolescents and children application submittedguanfacine Wayne, PA (adjunctive treatment, combination (484) 595-8800(extended release) therapy)

JNJ-31001074 Johnson & Johnson ADHD Phase IIPharmaceutical Research (800) 817-5286& Development --------------------------------------------------------------------------------------Raritan, NJ ADHD in children and adolescents Phase I

(800) 817-5286

KP106 KemPharm ADHD Phase INorth Liberty, IA (319) 665-2575

KRL-401 Krele Pharmaceuticals ADHD in clinical trialsSaddle River, NJ (212) 923-3400

lobeline Yaupon Therapeutics ADHD in adults Phase IIRadnor, PA (see also addictive disorders) (610) 975-9209

NERI Eli Lilly ADHD Phase II/III(LY2216684) Indianapolis, IN (pediatric patients) (800) 545-5979

(see also depression)

OPC-34712 Otsuka America ADHD Phase IIPharmaceutical (adjunctive treatment) (800) 562-3974Rockville, MD (see also depression, schizophrenia)

SEP-228432 Sepracor ADHD Phase I/IIMarlborough, MA (508) 481-6700

sofinicline Abbott Laboratories ADHD Phase IIAbbott Park, IL (847) 937-6100

SPN 810 Supernus Pharmaceuticals ADHD in children Phase II completedRockville, MD (301) 838-2500

SPN 811 Supernus Pharmaceuticals ADHD Phase IRockville, MD (301) 838-2500

SPN 812V Supernus Pharmaceuticals ADHD Phase I/IIRockville, MD (301) 838-2500

TC5619 AstraZeneca ADHD Phase IIWilmington, DE (see also dementias) (800) 236-9933Targacept Winston-Salem, NC

Vyvanse® Shire ADHD in adolescents Phase IIIlisdexamfetamine Wayne, PA (see also depression, schizophrenia, (800) 828-2088dimesylate sleep disorders)

D E M E N T I A SProduct Name Sponsor Indication Development Status

Diseases in this category are medical conditions involving the brain and can also be categorized as neurological disorders.

18-flutemetamol GE Healthcare Alzheimer’s disease diagnosis Phase III(PET imaging agent) Waukesha, WI www.gehealthcare.com



239512 GlaxoSmithKline Alzheimer’s disease Phase IIRsch. Triangle Park, NC (see also schizophrenia) (888) 825-5249

--------------------------------------------------------------------------------------mild cognitive impairment Phase I completed

(888) 825-5249

742457 GlaxoSmithKline Alzheimer’s disease Phase IIRsch. Triangle Park, NC (888) 825-5249

933776A GlaxoSmithKline Alzheimer’s disease Phase IRsch. Triangle Park, NC (888) 825-5249

1034702 GlaxoSmithKline dementia Phase IRsch. Triangle Park, NC (see also schizophrenia) (888) 825-5249

AAB-002 Janssen Alzheimer Alzheimer’s disease Phase 0Immunotherapy (800) 817-5286South San Francisco, CA (860) 732-5156Pfizer New York, NY

AAB-003 Janssen Alzheimer Alzheimer’s disease Phase 0Immunotherapy (800) 817-5286South San Francisco, CA (860) 732-5156Pfizer New York, NY

ABT-126 Abbott Laboratories Alzheimer’s disease Phase IIAbbott Park, IL (see also schizophrenia) (847) 937-6100

--------------------------------------------------------------------------------------Alzheimer’s disease in the elderly Phase I

(847) 937-6100

ABT-288 Abbott Laboratories Alzheimer’s disease Phase IIAbbott Park, IL (see also schizophrenia) (847) 937-6100

ABT-384 Abbott Laboratories Alzheimer’s disease Phase IIAbbott Park, IL (847) 937-6100

ABT-560 Abbott Laboratories cognition disorders Phase IAbbott Park, IL (847) 937-6100

ACC-002 Janssen Alzheimer Alzheimer’s disease Phase 0Immunotherapy (800) 817-5286South San Francisco, CA (860) 732-5156Pfizer New York, NY

AD02 Affiris Alzheimer’s disease Phase IIvaccine Vienna, Austria (888) 825-5249

GlaxoSmithKlineRsch. Triangle Park, NC

ADS-8703 Adamas Pharmaceuticals dementia Phase IIEmeryville, CA (510) 450-3500

Alzheimer’s Eli Lilly agitation in Alzheimer’s disease Phase IItherapy Indianapolis, IN (800) 545-5979

AMG 747 Amgen cognition disorders associated Phase IThousand Oaks, CA with schizophrenia (805) 447-1000



ARC029 Archer Pharmaceuticals Alzheimer’s disease Phase ISarasota, FL (941) 755-6644

ARC031 Archer Pharmaceuticals Alzheimer’s disease Phase ISarasota, FL (941) 755-6644

ASP 0777 Astellas Pharma US Alzheimer’s disease Phase IDeerfield, IL (800) 695-4321

AV965 Avera Pharmaceuticals Alzheimer’s disease, Phase ISan Diego, CA cognition disorders (858) 847-0650

AVN 101 Avineuro Pharmaceuticals Alzheimer’s disease Phase IISan Diego, CA (see also anxiety disorders) (858) 436-1537

AVN 322 Avineuro Pharmaceuticals Alzheimer’s disease Phase ISan Diego, CA (858) 436-1537

AVN 397 Avineuro Pharmaceuticals Alzheimer’s disease Phase IISan Diego, CA (see also anxiety disorders) (858) 436-1537

Axona™ Accera age-associated memory Phase IIBroomfield, CO impairment (303) 439-0004

AZD1446 AstraZeneca Alzheimer’s disease Phase II(TC-6683) Wilmington, DE (see also ADHD) (800) 236-9933


bapineuzumab Janssen Alzheimer Alzheimer’s disease Phase III(AAB-001) Immunotherapy (intravenous) (800) 817-5286

South San Francisco, CA (Fast Track) (860) 732-5156Pfizer --------------------------------------------------------------------------------------New York, NY Alzheimer’s disease Phase II

(subcutaneous) (800) 817-5286(860) 732-5156

BAY 85-8101 Bayer HealthCare Alzheimer’s disease diagnosis Phase I(PET imaging agent) Pharmaceuticals (888) 842-2937

Wayne, NJ

BAY 94-9172 Avid Radiopharmaceuticals Alzheimer’s disease diagnosis Phase III(florbetaben) Philadelphia, PA (215) 298-0700(PET imaging agent) Bayer HealthCare (888) 842-2937

Pharmaceuticals Wayne, NJ

begacestat Pfizer Alzheimer’s disease Phase INew York, NY (860) 732-5156

bisnorcymserine QR Pharma advanced Alzheimer’s disease Phase I(BNC) Radnor, PA (484) 253-2296

C105 Cognition Pharmaceuticals cognitive impairment/deficit Phase IINew York, NY associated with multiple sclerosis (212) 906-7071

CAD106 CytosBiotechnology Alzheimer’s disease Phase IIPostfach, Switzerland (888) 669-6682Novartis PharmaceuticalsEast Hanover, NJ



CEP-26401 Cephalon cognition disorders Phase IFrazer, PA (610) 344-0200

CERE-110 Ceregene Alzheimer’s disease Phase II(gene therapy) San Diego, CA (858) 458-8800

CHF 5074 Chiesi Pharmaceuticals Alzheimer’s disease Phase IRockville, MD (301) 424-2661

CTS-21166 Astellas Pharma US Alzheimer’s disease Phase IDeerfield, IL (800) 695-4321CoMentis (650) 359-2600South San Francisco, CA

davunetide Allon Therapeutics Alzheimer’s disease, Phase IIintranasal Vancouver, Canada cognitive impairment (604) 736-0634

associated with schizophrenia

davunetide Allon Therapeutics mild cognitive impairment Phase IIintravenous Vancouver, Canada (604) 736-0634

DEBIO 9902 Debiopharm Alzheimer’s disease Phase ILausanne, Switzerland www.debiopharm.com

docosahexaenoic Martek Biosciences Alzheimer’s disease Phase IIIacid Columbia, MD (410) 740-0081

E2212 Eisai Alzheimer’s disease Phase IWoodcliff Lake, NJ (888) 274-2378

EGb 761 Ipsen Alzheimer’s disease Phase IIIMilford, MA (508) 478-8900

ELND005 Elan Pharmaceuticals Alzheimer’s disease Phase IISouth San Francisco, CA (Fast Track) (650) 877-0900Transition Therapeutics (416) 260-7770Toronto, Canada

ELND006 Elan Pharmaceuticals Alzheimer’s disease Phase ISouth San Francisco, CA (650) 877-0900

EVP-0334 EnVivo Pharmaceuticals Alzheimer’s disease Phase IWatertown, MA (617) 225-4250

EVP-6124 EnVivo Pharmaceuticals Alzheimer’s disease Phase IIWatertown, MA (see also schizophrenia) (617) 225-4250

Exebryl-1® ProteoTech Alzheimer’s disease Phase IKirkland, WA (425) 823-0400

florbetapir F 18 Avid Radiopharmaceuticals Alzheimer’s disease diagnosis Phase III(PET imaging agent) Philadelphia, PA (215) 298-0700

Gammagard S/D™ Baxter Healthcare Alzheimer’s disease Phase IIIimmune globulin Deerfield, IL (early-stage, mid-stage disease) (800) 422-9837(IVIG)

gamma secretase Bristol-Myers Squibb Alzheimer’s disease Phase IIinhibitor Princeton, NJ (mild to moderate), (212) 546-4000

pre-dementia Alzheimer’s disease

HPP-854 TransTech Pharma Alzheimer’s disease Phase IHigh Point, NC (336) 841-0300



HT-0712 Helicon Therapeutics cognition disorders Phase IFarmingdale, NY (631) 370-8818

HTC-867 Pfizer cognitive impairment associated Phase INew York, NY with schizophrenia (860) 732-5156

huperzine A Neuro-Hitech Alzheimer’s disease Phase IINew York, NY (212) 594-1225

intravenous Grifols USA Alzheimer’s disease in clinical trialsnormal human Los Angeles, CA (888) 474-3657immunoglobulin

latrepirdine Medivation early-stage Alzheimer’s disease Phase IIISan Francisco, CA (415) 543-3470Pfizer (860) 732-5156New York, NY --------------------------------------------------------------------------------------

Alzheimer’s disease Phase I(combination therapy) (415) 543-3470

(860) 732-5156

Lipitor® Pfizer Alzheimer’s disease Phase IIIatorvastatin New York, NY (combination therapy) (860) 732-5156

Lu AE58054 Lundbeck cognitive impairment associated Phase IIDeerfield, IL with schizophrenia (800) 455-1141

LY2886721 Eli Lilly Alzheimer’s disease Phase IIndianapolis, IN (800) 545-5979

MCD-386CR Mithridion Alzheimer’s disease Phase IMadison, WI (608) 443-2432

NIC5-15 Humanetics Alzheimer’s disease Phase II completedEden Prairie, MN (952) 937-7660

NSA-789 Pfizer Alzheimer’s disease Phase INew York, NY (see also schizophrenia) (860) 732-5156

Oxigon™ Intellect Neurosciences Alzheimer’s disease Phase Iindolepropionic New York, NY (212) 448-9300acid derivative

PF-3654746 Pfizer Alzheimer’s disease Phase INew York, NY (adjunctive treatment) (860) 732-5156

PF-3654764 Pfizer Alzheimer’s disease Phase I completedNew York, NY (860) 732-5156

PF-4360365 Pfizer Alzheimer’s disease Phase IINew York, NY (860) 732-5156


--------------------------------------------------------------------------------------Alzheimer’s disease Phase I(combination therapy) (860) 732-5156


PF-5212365 Pfizer Alzheimer’s disease Phase IINew York, NY (see also schizophrenia) (860) 732-5156




PF-5236806 Janssen Alzheimer Alzheimer’s disease Phase IIImmunotherapy (800) 934-5556South San Francisco, CA (212) 407-5740PfizerNew York, NY

Posiphen™ QR Pharma Alzheimer’s disease, Phase IIR-phenserine Radnor, PA mild cognitive impairment (484) 253-2296

RG1450 Roche Alzheimer’s disease Phase I(gantenerumab) Nutley, NJ (973) 235-5000

RG1662 Roche Alzheimer’s disease Phase INutley, NJ (973) 235-5000

RG3487 Roche Alzheimer’s disease, cognitive Phase IINutley, NJ impairment associated with (973) 235-5000

schizophrenia

RG7412 Genentech Alzheimer’s disease Phase I(anti-Abeta) South San Francisco, CA (800) 626-3553

RVX-208 Resverlogix Alzheimer’s disease Phase ICalgary, Canada (403) 254-9252

SAR110894 sanofi-aventis Alzheimer’s disease Phase IBridgewater, NJ (800) 633-1610

semagacestat Eli Lilly Alzheimer’s disease Phase IIIIndianapolis, IN (800) 545-5979

SEP-227900 Sepracor Alzheimer’s disease, Phase IMarlborough, MA cognition disorders (508) 481-6700

solanezumab Eli Lilly Alzheimer’s disease Phase IIIIndianapolis, IN (800) 545-5979

ST-101 Sonexa Therapeutics Alzheimer’s disease Phase IISan Diego, CA (858) 356-6250

SYN-114 Synosia Therapeutics cognition disorders Phase ISouth San Francisco, CA (650) 244-4850

SYN-120 Synosia Therapeutics cognition disorders Phase ISouth San Francisco, CA (650) 244-4850

T-817MA Toyama Chemical Alzheimer’s disease Phase IITokyo, Japan

TC5619 AstraZeneca cognition disorders associated Phase IIWilmington, DE with schizophrenia (800) 236-9933Targacept (see also ADHD)Winston-Salem, NC

TD-5108 Theravance Alzheimer’s disease Phase ISouth San Francisco, CA (877) 275-8479

TD-8954 Theravance Alzheimer’s disease Phase ISouth San Francisco, CA (877) 275-8479



V950 Merck Alzheimer’s disease Phase IWhitehouse Station, NJ (800) 672-6372

varenicline Pfizer Alzheimer’s disease Phase IINew York, NY (see also schizophrenia) (860) 732-5156

D E P R E S S I O NProduct Name Sponsor Indication Development Status

163090 GlaxoSmithKline depression Phase IRsch. Triangle Park, NC (see also anxiety disorders) (888) 825-5249



586529 GlaxoSmithKline depression Phase IRsch. Triangle Park, NC (see also anxiety disorders) (888) 825-5249Neurocrine Biosciences (858) 617-7600San Diego, CA


1014802 GlaxoSmithKline bipolar disorder Phase IRsch. Triangle Park, NC (888) 825-5249

ABT-436 Abbott Laboratories depression Phase IAbbott Park, IL (see also anxiety disorders) (847) 937-6100

ADX N05 Shionogi Pharma depression Phase IAtlanta, GA (800) 461-3696

AZD2327 AstraZeneca depression Phase IIWilmington, DE (see also anxiety disorders) (800) 236-9933

AZD6765 AstraZeneca depression Phase IIWilmington, DE (800) 236-9933

AZD7268 AstraZenenca depression Phase IIWilmington, DE (800) 236-9933

BCI-540 BrainCells depression Phase IISan Diego, CA (see also anxiety disorders) (858) 812-7700

BCI-952 BrainCells major depressive disorder Phase IISan Diego, CA (858) 812-7700

bupropion IntelGenx depression application submitted(controlled-release) Quebec, Canada (514) 331-7440

cariprazine Forest Laboratories bipolar disorder Phase IINew York, NY (see also schizophrenia) (800) 947-5227

--------------------------------------------------------------------------------------depression Phase II(adjunctive treatment) (800) 947-5227



Contrave™ Orexigen Therapeutics depression Phase IInaltrexone/ La Jolla, CA (see also eating disorders) (858) 436-8600bupropion combination

Corlux™ Corcept Therapeutics psychotic major depression Phase IIImifeprostone Menlo Park, CA (Fast Track) (650) 327-3270

CP-601927 Pfizer depression Phase IINew York, NY (860) 732-5156

Cymbalta® Eli Lilly major depression in children and Phase IIIduloxetide Indianapolis, IN adolescents (800) 545-5979

depression therapy Eli Lilly depression Phase IIndianapolis, IN (800) 545-5979

DOV-21947 DOV Pharmaceutical depression Phase IILittle Falls, NJ (see also eating disorders) (732) 907-3600

E2508 Eisai depression Phase IWoodcliff Lake, NJ (888) 274-2378

emicerfont GlaxoSmithKline depression Phase IIRsch. Triangle Park, NC (see also anxiety disorders) (888) 825-5249Neurocrine Biosciences (858) 617-7600San Diego, CA

EVT-101 Evotec depression Phase IIHamburg, Germany

Geodon® Pfizer bipolar disorders in adolescents Phase IIIziprasidone New York, NY and children (860) 732-5156

GLYX-13 Naurex depression Phase IEvanston, IL www.naurex.com

Invega® Johnson & Johnson bipolar disorder, manic episodes Phase IIIpaliperidone Pharmaceutical Research (see also developmental disorders, (800) 817-5286extended-release & Development schizophrenia, sleep disorders)

Raritan, NJ

Lamictal® GlaxoSmithKline bipolar disorder (elderly) Phase III completedlamotrigine Rsch. Triangle Park, NC (888) 825-5249

--------------------------------------------------------------------------------------bipolar disorder (10-17 years) Phase III

(888) 825-5249

levomilnacipran Forest Laboratories depression Phase III(F2695) New York, NY (800) 947-5227

losmapimod GlaxoSmithKline major depressive disorder Phase IIRsch. Triangle Park, NC (888) 825-5249

Lovaza® GlaxoSmithKline depression Phase IIomega-3-acis Rsch. Triangle Park, NC (888) 825-5249ethyl esters

Lu AA21004 Lundbeck major depressive disorder Phase IIIDeerfield, IL (see also anxiety disorders) (800) 455-1141Takeda Pharmaceuticals (224) 554-6500North AmericaDeerfield, IL



Lu AA24530 Lundbeck major depressive disorder Phase IDeerfield, IL (see also anxiety disorders) (800) 455-1141

lurasidone Sepracor bipolar disorder Phase IIIMarlborough, MA (see also schizophrenia) (508) 481-6700

Motiva™ Neuren Pharmaceuticals post-stroke depression Phase IInefiracetam Bethesda, MD (301) 941-1830

Nectiv™ Pfizer treatment-resistant depression Phase IItraxoprodil New York, NY (combination therapy) (860) 732-5156

nemifitide Tetragenex depression Phase II completedPharmaceuticals (201) 505-1300Park Ridge, NJ

NERI Eli Lilly depressive disorders Phase II(LY2216684) Indianapolis, IN (see also ADHD) (800) 545-5979

NMDA receptor Evotec treatment-resistant depression Phase Iantagonist Hamburg, Germany (973) 235-5000

Roche Nutley, NJ

NSD-788 GlaxoSmithKline depression Phase IRsch. Triangle Park, NC (see also anxiety disorders) (888) 825-5249NeuroSearchBallerup, Denmark

Nuvigil® Cephalon bipolar depression Phase IIIarmodafinil Frazer, PA (adjunctive treatment) (610) 344-0200

(see also eating disorders)

OPC-34712 Otsuka America depression Phase IIPharmaceutical (adjunctive treatment) (800) 562-3974Rockville, MD (see also ADHD, schizophrenia)

orvepitant GlaxoSmithKline depression Phase IIRsch. Triangle Park, NC (see also anxiety disorders) (888) 825-5249

PF-4455242 Pfizer bipolar depression Phase INew York, NY (860) 732-5156

Pristiq® Pfizer major depressive disorder Phase IIdesvenlafaxine New York, NY in children and adolescents (860) 732-5156

RG1578 Roche depression Phase INutley, NJ (973) 235-5000

RG2417 Repligen bipolar disorder Phase IIWaltham, MA (800) 622-2259

RG7090 Roche depression Phase IINutley, NJ (see also developmental disorders) (973) 235-5000






Risperdal® Johnson & Johnson bipolar disorder with anxiety or Phase IIIrisperidone Pharmaceutical Research panic disorder (monotherapy), (800) 817-5286

& Development depressionRaritan, NJ

Serdaxin™ Rexahn Pharmaceuticals depressive disorders Phase IIclavulanic acid Rockville, MD (240) 268-5300

Seroquel® AstraZeneca depression (monotherapy, application submittedquetiapine Wilmington, DE prevention of relapse) (800) 236-9933fumarate (see also other)

Seroquel XR® AstraZeneca bipolar depression in adolescents Phase IIIquetiapine Wilmington, DE and children (800) 236-9933fumarate (see also addictive disorders, extended-release anxiety disorders, personality

disorders)

SPN 802 Supernus Pharmaceuticals bipolar disorder Phase IRockville, MD (301) 838-2500

SSR125543 sanofi-aventis major depressive disorder Phase IBridgewater, NJ (see also anxiety disorders) (800) 633-1610

SSR411298 sanofi-aventis depression Phase IBridgewater, NJ (800) 633-1610

Staccato® Alexza Pharmaceuticals acute agitation in patients with application submittedloxapine Palo Alto, CA bipolar I disorder (650) 687-3900inhalation (see also schizophrenia)

SYN-111 Synosia Therapeutics bipolar disorder Phase IISouth San Francisco, CA (650) 244-4850

TC5214 AstraZeneca major depressive disorder Phase IIWilmington, DE (800) 236-9933Targacept (336) 480-2100Winston-Salem, NC

TGBA01AD Fabre-Kramer depression Phase IIPharmaceuticals (713) 975-6900Houston, TX

triple reuptake Bristol-Myers Squibb depression in clinical trialsinhibitor Princeton, NJ (212) 546-4000

TriRima® CeNeRx BioPharma depressive disorders Phase II(CX157) Rsch. Triangle Park, NC (see also anxiety disorders) (919) 234-4650

tasimelteon Vanda Pharmaceuticals depression Phase IRockville, MD (see also sleep disorders) (240) 599-4500

vabicaserin Pfizer bipolar disorder Phase IINew York, NY (see also schizophrenia) (860) 732-5156

Valdoxan® Novartis Pharmaceuticals major depressive disorder, Phase IIIagomelatine East Hanover, NJ prevention of depression relapse (888) 669-6682

venlafaxine Auspex Pharmaceuticals depression Phase Ideuterium- Vista, CA (760) 599-1800substituted(SD-254)



verucerfont GlaxoSmithKline depression Phase IIRsch. Triangle Park, NC (see also anxiety disorders) (888) 825-5249Neurocrine Biosciences (858) 617-7600San Diego, CA

vilazodone PGx Health major depression application submittedNew Haven, CT (877) 274-9432

Vyvanse® Shire bipolar disorder Phase IIIlisdexamfetamine Wayne, PA (see also ADHD, schizophrenia, (434) 595-8800dimesylate sleep disorders)

ziprasidone Lipocine bipolar disorder Phase Iextended-release Salt Lake City, UT (see also schizophrenia) (810) 994-7383

D E V E L O P M E N T A L D I S O R D E R SProduct Name Sponsor Indication Development Status

AFQ056 Novartis Pharmaceuticals fragile X syndrome Phase II completedEast Hanover, NJ (see also addictive disorders) (888) 669-6682

CM-AT CureMark autism Phase IIIRye, NY (Fast Track) (914) 925-3450

Invega® Johnson & Johnson aggression in adolescents and young Phase IIIpaliperidone Pharmaceutical Research adults with autism (800) 817-5289extended-release & Development (see also depression, schizophrenia,

Raritan, NJ sleep disorders)

RG7090 Roche fragile X syndrome Phase IINutley, NJ (see also depression) (973) 235-5000

STK107 Seaside Therapeutics fragile X syndrome Phase ICambridge, MA (617) 374-9009

STK209 Seaside Therapeutics autism in children and adolescents, Phase II(arbaclofen) Cambridge, MA fragile X syndrome (617) 374-9009

E A T I N G D I S O R D E R SProduct Name Sponsor Indication Development Status

181771 GlaxoSmithKline bulimia nervosa Phase II completedRsch. Triangle Park, NC (888) 825-5249

598809 GlaxoSmithKline compulsive over-eating Phase IRsch. Triangle Park, NC (888) 825-5249

1521498 GlaxoSmithKline obesity Phase IRsch. Triangle Park, NC (888) 825-5249

anamorelin Helsinn Therapeutics anorexia Phase IIBridgewater, NJ (Fast Track) (908) 231-1435

AZD4017 AstraZeneca obesity Phase IWilmington, DE (800) 236-9933



AZD8329 AstraZeneca obesity Phase IWilmington, DE (800) 236-9933

canagliflozin Johnson & Johnson obesity Phase IIPharmaceutical Research (800) 817-5286& DevelopmentRaritan, NJ

cetilistat Norgine obesity Phase IIAmsterdam, www.norgine.comThe Netherlands

Contrave™ Orexigen Therapeutics obesity application submittednaltrexone/ La Jolla, CA (see also depression) (858) 436-8600bupropion combination

diazoxide choline Essentialis obesity Phase I(controlled-release) Carlsbad, CA (760) 431-2646

DOV-21947 DOV Pharmaceutical obesity Phase ILittle Falls, NJ (see also depression) (732) 907-3600

Empatic™ Orexigen Therapeutics obesity Phase IIzonisamide/ La Jolla, CA (858) 875-8600bupropion

GT389255 Peptimmune obesity Phase IICambridge, MA (617) 715-8000

HHP-404 TransTech Pharma obesity Phase IHigh Point, NC (336) 841-0300

Histalean™ Obecure obesity Phase IIbetahistine Ramat Gan, Israel www.obecure.com

JNJ-16269110 Johnson & Johnson obesity Phase IIPharmaceutical Research (800) 817-5286& DevelopmentRaritan, NJ

KD-3010 Kalypsys obesity Phase ISan Diego, CA (858) 754-3300

lorcaserin Arena Pharmaceuticals obesity application submittedSan Diego, CA (858) 453-7200

NN9161 Novo Nordisk obesity Phase I/IIPrinceton, NJ (609) 987-5800

Nuvigil® Cephalon binge eating disorder Phase IIIarmodafinil Frazer, PA (see also depression) (610) 344-0200

OAP-189 Pfizer obesity Phase INew York, NY (860) 732-5156

obesity therapy Eli Lilly obesity Phase IIIndianapolis, IN (800) 545-5979

pramlintide/ Amylin Pharmaceuticals obesity Phase IImetreleptin San Diego, CA (858) 552-2200combination Takeda Pharmaceuticals (877) 587-5332

North AmericaDeerfield, IL


Qnexa™ VIVUS obesity application submittedphentermine/ Mountain View, CA (650) 934-5200topiramate

S-2367 Shionogi USA obesity Phase IIFlorham Park, NJ (973) 966-6900

S-234462 Shionogi USA obesity Phase IFlorham Park, NJ (973) 966-6900

sobetirome QuatRx Pharmaceuticals obesity Phase IAnn Arbor, MI (734) 913-9900

TKS1225 Pfizer obesity Phase INew York, NY (860) 732-5156

TTP-435 TransTech Pharma obesity Phase IHigh Point, NC (336) 841-0300

Victoza® Novo Nordisk obesity Phase IIIliraglutide Princeton, NJ (second-line therapy) (609) 987-5800

ZGN-433 Zafgen obesity Phase ICambridge, MA (617) 864-1645

Zoloft® Pfizer obesity in clinical trialssertraline New York, NY (see also anxiety disorders) (860) 732-5156

Zyprexa® Eli Lilly anorexia nervosa Phase IIolanzapine Indianapolis, IN (see also personality disorders) (800) 545-5979

P E R S O N A L I T Y D I S O R D E R SProduct Name Sponsor Indication Development Status

Seroquel XR® AstraZeneca borderline personality disorder Phase IIIquetiapine Wilmington, DE (see also addictive disorders, (800) 236-9933fumarate anxiety disorders, depression)extended-release

Zyprexa® Eli Lilly borderline personality disorder Phase III completedolanzapine Indianapolis, IN (see also eating disorders) (800) 545-5979

P R E M E N S T R U A L D I S O R D E R SProduct Name Sponsor Indication Development Status

Lybrel™ Pfizer premenstrual dysphoric disorder Phase IIIlevonorgestrel/EE New York, NY (860) 732-5156

PH80 Pherin Pharmaceuticals premenstrual dysphoric disorder, Phase II completedRedwood City, CA premenstrual syndrome (650) 568-1587


S C H I Z O P H R E N I AProduct Name Sponsor Indication Development Status

239512 GlaxoSmithKline schizophrenia Phase IIRsch. Triangle Park, NC (see also dementias) (888) 825-5249

729327 GlaxoSmithKline schizophrenia Phase IRsch. Triangle Park, NC (888) 825-5249

773812 GlaxoSmithKline schizophrenia Phase II completedRsch. Triangle Park, NC (888) 825-5249


1034702 GlaxoSmithKline schizophrenia Phase IRsch. Triangle Park, NC (see also dementias) (888) 825-5249


Abilify® IM Depot Bristol-Myers Squibb schizophrenia Phase IIIaripiprazole Princeton, NJ (860) 732-5156

Otsuka America (800) 562-3974Pharmaceutical Rockville, MD

ABT-126 Abbott Laboratories schizophrenia Phase IIAbbott Park, IL (see also dementias) (847) 937-6100

ABT-288 Abbott Laboratories schizophrenia Phase IIAbbott Park, IL (see also dementias) (847) 937-6100

ADX71149 Johnson & Johnson schizophrenia Phase IPharmaceutical Research (see also anxiety disorders) (800) 817-5286& Development Raritan, NJ

ATON005 Aton Pharma psychotic disorders Phase ILawrenceville, NJ (609) 671-9010

AVN 211 Avineuro Pharmaceuticals schizophrenia Phase IISan Diego, CA (858) 436-1537

AZD8418 AstraZeneca schizophrenia Phase IWilmington, DE (800) 236-9933

AZD8529 AstraZeneca schizophrenia Phase IIWilmington, DE (800) 236-9933

BL-1020 BioLineRx schizophrenia Phase IIJerusalem, Israel www.biolinerx.com

cariprazine Forest Laboratories schizophrenia Phase IIINew York, NY (see also depression) (800) 947-5227

CM-2395 Cenomed BioSciences schizophrenia in clinical trialsIrvine, CA (949) 838-0344

DCCCyB Merck schizophrenia Phase IWhitehouse Station, NJ (800) 672-6372

EVP-6124 EnVivo Pharmaceuticals schizophrenia Phase IIWatertown, MA (see also dementias) (617) 225-4250



Fanapt™ Vanda Pharmaceuticals schizophrenia Phase I/IIiloperidone Rockville, MD (240) 599-4500(extended-release Novartis Pharmaceutical (888) 669-6682injectable East Hanover, NJformulation)

idazoxan Potomac Pharma schizophrenia Phase IIBethesda, MD

Invega® Johnson & Johnson schizophrenia in adolescents Phase IIIpaliperidone Pharmaceutical Research (see also depression, developmental (800) 817-5286extended-release & Development disorders, sleep disorders)

Raritan, NJ

ITI-007 Intra-Cellular Therapies schizophrenia Phase INew York, NY (see also sleep disorders) (212) 923-3344

JNJ-17305600 Johnson & Johnson schizophrenia Phase IPharmaceutical Research (800) 817-5286& DevelopmentRaritan, NJ

JNJ-37822681 Johnson & Johnson schizophrenia Phase IIPharmaceutical Research (800) 817-5286& DevelopmentRaritan, NJ

JNJ-39393406 Johnson & Johnson schizophrenia Phase IPharmaceutical Research (800) 817-5286& Development Raritan, NJ

JNJ-40411813 Johnson & Johnson psychotic disorders Phase IPharmaceutical Research (800) 817-5286& DevelopmentRaritan, NJ

Lu 31-30 Lundbeck psychosis Phase IIDeerfield, IL (800) 455-1141

lurasidone Sepracor schizophrenia application submittedMarlborough, MA (see also depression) (508) 481-6700

mGlu2/3 pro II Eli Lilly schizophrenia Phase II(LY2140023) Indianapolis, IN (800) 545-5979

MK-2637 Merck schizophrenia Phase I completedWhitehouse Station, NJ (800) 672-6372

MK-8998 Merck schizophrenia Phase IIWhitehouse Station, NJ (800) 672-6372

NSA-789 Pfizer schizophrenia Phase INew York, NY (see also dementias) (860) 732-5156

OPC-34712 Otsuka America schizophrenia Phase IIPharmaceutical (see also ADHD, depression) (800) 562-3974Rockville, MD

PF-217830 Pfizer schizophrenia Phase IINew York, NY (860) 732-5156



PF-2400013 Pfizer schizophrenia Phase INew York, NY (860) 732-5156



PF-5212365 Pfizer schizophrenia Phase IINew York, NY (see also dementias) (860) 732-5156

pimavanserin ACADIA Pharmaceuticals Parkinson’s disease-associated Phase III(ACP-103) San Diego, CA psychosis (858) 558-2871

Biovail --------------------------------------------------------------------------------------Mississauga, Canada schizophrenia Phase II completed

(858) 558-2871

RG1678 Roche schizophrenia Phase II completedNutley, NJ (973) 235-5000

Risperdal® Consta® Johnson & Johnson schizophrenia Phase Irisperidone Pharmaceutical Research (see also addictive disorders) (800) 817-5286(injectable) & Development

Raritan, NJ

sabcomeline Proximagen Neuroscience schizophrenia Phase IILondon, United Kingdom

Saphris® Merck psychotic disorders in elderly Phase III completedasenapine Whitehouse Station, NJ patients (800) 672-6372

SCH 900435 Merck schizophrenia Phase IIWhitehouse Station, NJ (see also addictive disorders) (800) 672-6372

schizophrenia Eli Lilly schizophrenia Phase Itherapy Indianapolis, IN (800) 545-5979

sertindole Lundbeck schizophrenia application submittedDeerfield, IL (800) 455-1141

Staccato® Alexza Pharmaceuticals acute agitation in schizophrenia application submittedloxapine Palo Alto, CA (see also depression) (650) 687-3900inhalation

TGOF02N Fabre-Kramer schizophrenia Phase IIPharmaceuticals (713) 975-6900Houston, TX

tiprolisant Bioprojet schizophrenia Phase IIParis, France www.bioprojet.comFerrerBarcelona, Spain

vabicaserin Pfizer schizophrenia Phase INew York, NY (see also depression) (860) 732-5156

varenicline Pfizer schizophrenia Phase IINew York, NY (see also dementias) (860) 732-5156

Vyvanse® Shire schizophrenia Phase IIlisdexamfetamine Wayne, PA (see also ADHD, depression, (434) 595-8800dimesylate sleep disorders)



ziprasidone Lipocine schizophrenia Phase Iextended-release Salt Lake City, UT (see also depression) (810) 994-7383

S L E E P D I S O R D E R SProduct Name Sponsor Indication Development Status

649868 GlaxoSmithKline sleep disorders Phase IIRsch. Triangle Park, NC (888) 825-5249

ABT-652 Abbott Laboratories excessive daytime sleepiness Phase IAbbott Park, IL (847) 937-6100

almorexant Actelion insomnia Phase IIIPharmaceuticals US (650) 624-6900South San Francisco, CA (888) 825-5249GlaxoSmithKline --------------------------------------------------------------------------------------Rsch. Triangle Park, NC insomnia in elderly Phase II

(650) 624-6900(888) 825-5249

AZ-007 Alexza Pharmaceuticals insomnia Phase I completed(zaleplon Mountain View, CA (650) 944-7000inhalation)

indiplon Neurocrine Biosciences insomnia application submitted(controlled-release) San Diego, CA (858) 617-7600

indiplon Neurocrine Biosciences insomnia application submitted(immediate-release) San Diego, CA (858) 617-7600

Intermezzo® Transcept Pharmaceuticals insomnia application submittedzolpidem Pt. Richmond, CA (510) 215-3500sublingual

Invega® Johnson & Johnson insomnia Phase II completedpaliperidone Pharmaceutical Research (see also depression, developmental (800) 817-5286extended-release & Development disorders, schizophrenia)

Raritan, NJ

ITI-007 Intra-Cellular Therapies sleep maintenance insomnia Phase IINew York, NY (see also schizophrenia) (212) 923-3344

KRL-102 Krele Pharmaceuticals sleep disorders in clinical trialsSaddle Brook, NJ (212) 923-3400

Lunesta® Sepracor ADHD-associated insomnia in Phase IIIeszopiclone Marlborough, MA adolescents and children (508) 481-6700

(see also anxiety disorders)

LY2624803 Eli Lilly insomnia Phase IIIndianapolis, IN (800) 545-5979

melatonin Neurim Pharmaceuticals non-24-hour sleep-wake disorder Phase III(controlled-release) Tel-Aviv, Israel www.neurim.com(Orphan Drug)MK-4305 Merck insomnia Phase III

Whitehouse Station, NJ (800) 672-6372


S L E E P D I S O R D E R SProduct Name Sponsor Indication Development Status

MK-6096 Merck insomnia Phase IIWhitehouse Station, NJ (800) 672-6372

MN-305 MediciNova insomnia Phase IISan Diego, CA (858) 373-1500

SKP-1041 Skye Pharma US sleep disorders Phase II(zaleplon Cambridge, MA (908) 901-0300controlled-release) Somnus Therapeutics

Bedminster, NJ

tasimelteon Vanda Pharmaceuticals insomnia Phase III(Orphan Drug) Rockville, MD (see also depression) (240) 599-4500

--------------------------------------------------------------------------------------circadian rhythm sleep disorder Phase II

(240) 599-4500

TGAR01H Fabre-Kramer insomnia Phase II(triazolam Pharmaceuticals (713) 975-6900intranasal) Houston, TX

Vyvanse® Shire sleep disorders Phase Ilisdexamfetamine Wayne, PA (ADHD, depression, schizophrenia) (434) 595-8800dimesylate

zaleplon-GR Intec Pharma insomnia Phase IJerusalem, Israel www.intecpharma.com

O T H E RProduct Name Sponsor Indication Development Status

CORT 108297 Corcept Therapeutics psychiatric disorders Phase IMenlo Park, CA (650) 327-3270

MK-8368 Merck psychiatric disorders Phase IWhitehouse Station, NJ (800) 672-6372

Seroquel® AstraZeneca delirium Phase IIIquetiapine Wilmington, DE (see also depression) (800) 236-9933fumarate

The content of this report has been obtained through industry sources and the Adis “R&D Insight” databasebased on the latest information. Report current as of July 5, 2010. The information may not be comprehensive.For more specific information about a particular product, contact the individual company directly or go towww.clinicaltrials.gov. The entire series of Medicines in Development is available on PhRMA’s web site.

A publication of PhRMA’s Communications & Public Affairs Department. (202) 835-3460

www.phrma.org | www.innovation.org | www.pparx.org | www.buysafedrugs.info | www.sharingmiracles.com

Provided as a Public Service by PhRMA. Founded in 1958 as the Pharmaceutical Manufacturers Association.

Copyright © 2010 by the Pharmaceutical Research and Manufacturers of America. Permission to reprint is awarded ifproper credit is given.



G L O S S A R Y

Alzheimer’s disease—Progressiveand chronic deterioration of allmental functions. Early manifesta-tions include a decrease in attentionspan, impaired powers of concen-tration, some personality changeand forgetfulness. As the diseaseprogresses, there is a loss of com-putational ability, in addition toword-finding problems and difficultywith ordinary activities. Ultimately,there is severe memory loss, completedisorientation, social withdrawalloss of independence, and is fatal.It is the seventh leading cause ofdeath in the United States.

anxiety disorders—A group ofmental illnesses in which symp-toms of anxiety (from mild uneaseto intense fear) are the main featureand there is overlap among specificsyndromes. Generalized anxietydisorder is diagnosed if a patientexperiences unrealistic or excessiveanxiety and worry (apprehensiveexpectation) about two or morelife circumstances, e.g., worryabout something bad happeningto a loved one (who is not indanger) and worry about money(for no reason) for six months orlonger. A diagnosis of panic dis-order requires that panic attacks—sudden feelings of apprehensionor fear accompanied by physicalsymptoms such as shortness ofbreath—occur with 4 out of 12concomitant psychophysiologicalsymptoms, that the attacks aresevere enough and happen oftenenough to be disruptive or dis-tressing to the individual, andthat at least one attack was spon-taneous, i.e., occurred in theabsence of a phobic stimulus.

application submitted—An appli-cation for marketing has beensubmitted to the U.S. Food andDrug Adminis tration (FDA). Theapplication can either be an NDA(new drug application) or a BLA(biologic license application).

attention deficit/hyperactivitydisorder—ADHD is a complexneurological impairment thatresults in an overactive behaviorpattern and a difficulty concen-trating. While it primarily affectschildren, a growing number ofadults are being diagnosed withthe disorder. Boys are afflictedsome three times as often as girls.Children with ADHD are full ofenergy, fidgety, impulsive, reck-less, irritable, emotionallyimmature and aggressive. Becausetheir attention span is short, theydo not conform to orderly routine.ADHD often leads to anti-socialacts and difficulty learning,although IQ is normal. No definitecause has been established, butsome researchers now believeheredity plays a role.

autism—A complex developmentaldisorder that causes severe andpervasive impairment in thinking,feeling, language, and the ability torelate to others. It is usually firstdiagnosed in early childhood andranges from a severe form, calledautism disorder, through pervasivedevelopment disorder, to a muchmilder form, Asperger syndrome.

dementia—Degeneration ofcentral nervous system functions,such as memory and learningcapacity. The natural decline ofthese functions with age is grosslyexaggerated in dementia.

depression—A feeling of intensesadness, which may follow arecent loss or other sad event butis out of proportion to that eventand persists beyond an appropriatelength of time. Major depressivedisorder (or major depressionsyndrome) includes an episode ofdepression defined as a persistent(for at least 2 weeks) mood distur-bance, plus at least 4 of thefollowing: sleep disturbance,changes in psychomotor activity,

loss of ability to experience plea-sure and interest, fatigue, feelingsof worthlessness or guilt, difficultyin concentrating, and preoccupa-tion with death or a wish to die.Major depression is associated withimpairment in social functioning.If criteria for major depressionhave been met but in addition anepisode of mania has ever occurred,then the diagnosis becomes bipolardisorder (or manic-depressiveillness). The essential feature ofmania is a distinct period whenthe predominant mood is eitherelevated, expansive, or irritable,with associated symptoms includinghyper activity, pressure of speech,flight of ideas, inflated self-esteem,decreased need for sleep, dis-tractibility and excessiveinvolvement in activities that oftenare flamboyant, bizarre or disor-ganized.

Fast Track—Fast Track is a processdesigned to facilitate the develop-ment and expedite the review ofdrugs to treat serious diseases andfill an unmet medical need. Thestatus is assigned by the U.S. Foodand Drug Administration. Thepurpose is to get important newdrugs to the patient earlier. FastTrack addresses a broad range ofserious diseases. Generally, deter-mining factors include whether thedrug will have an impact on suchfactors as survival, day-to-day func-tioning, or the likelihood that thedisease, if left untreated, willprogress from a less severe condi-tion to a more serious one. Fillingan unmet medical need is definedas providing a therapy where noneexists or providing a therapy whichmay be potentially superior toexisting therapy. Once a drugreceives Fast Track designation,early and frequent communicationbetween the FDA and a drugcompany is encouraged throughoutthe entire drug development and


review process. The frequency ofcommunication assures that ques-tions and issues are resolvedquickly, often leading to earlierdrug approval and access bypatients.

fragile X syndrome (FXS)—Themost common cause of inheritedmental impairment. This impair-ment can range from learningdisabilities to more severe cognitiveor intellectual disabilities. FXS isthe most common known cause ofautism or “autistic-like” behaviors,but 80-85 percent of autism casesare of unknown cause. Symptomsalso can include characteristicphysical and behavioral featuresand delays in speech and languagedevelopment.

obesity—For adults, overweightand obesity ranges are determinedby using weight and height to cal-culate the “body mass index”(BMI). BMI is used because, formost people, it correlates with theiramount of body fat. Adults with aBMI between 25 and 29.9 are con-sidered overweight. An adult BMIof 30 or higher is consideredobese.

obsessive-compulsive disorder—A neurosis characterized primarilyby recurrent and persistent thoughtsthat are intense, disgusting, fright-ening, absurd or otherwise alien,accompanied by ritualized, repeti-

tive behavior that is usually irra-tional and bizarre.

Orphan Drug—A drug to treat adisease that has a patient popula-tion of 200,000 or less, or a diseasethat has a patient population ofmore than 200,000 and a develop-ment cost that will not be recoveredfrom sales in the United States.Orphan Drug status is assigned bythe U.S. Food and DrugAdministration.

Phase 0—First-in-human trialsconducted in accordance withFDA’s 2006 guidance onexploratory Investigational NewDrug (IND) studies designed tospeed up development of promis-ing drugs by establishing veryearly on whether the agentbehaves in human subjects aswas anticipated from preclinicalstudies.

Phase I—Safety testing andpharma cological profiling of newdrugs in small numbers of humans.

Phase II—Effectiveness testing andidentification of side effect profileof new drugs in humans.

Phase III—Extensive clinical trialsin humans to verify effectivenessand monitor adverse reactions ofnew drugs.

Phase IV—Additional post-market-ing testing of drugs sometimesrequired by FDA.

premenstrual syndrome(PMS)/pre menstrual dysphoricdisorder (PMDD) —PMS refers tothe variation of physical and moodsymptoms that appear during thelast one or two weeks of the men-strual cycle and disappear by the end of a full flow of menses.Psychiatrists and other mental healthworkers tend to use the term PMDDto describe a specific set of moodsymptoms that interfere with socialor role functioning that are alsopresent the week before mensesand remit a few days after the start.PMS looks more at physical symp-toms such as bloating, breasttenderness and appetite change.PMDD has as part of its definitionsymptoms such as depressed mood,anxiety or tension, irritability,concentration difficulties, overeat-ing or food cravings, and feelingoverwhelmed.

psychosis—Severe mental disor-ders in which the individual losescontact with reality. Symptomsinclude delusions, hallucinations,thought disorders, loss of emotion,mania and depression.

schizophrenia—The most commonform of psychotic illness charac-terized by disturbances in thinking,emotional reaction and behavior.It is disabling and has a prolongedcourse that almost always resultsin chronic ill health and somedegree of personality change.

G L O S S A R Y


S E L E C T E D F A C T S A B O U T M E N T A L I L L N E S S E S A N DA D D I C T I V E D I S O R D E R SOverview• Hundreds of millions of people worldwide are affected by mental, behavioral, neurological, and substance use

disorders. In 2002, the World Health Organization (WHO) estimated that globally 154 million people sufferedfrom depression and 25 million from schizophrenia. Another 91 million people were affected by alcohol usedisorders and 15 million by drug use disorders. WHO also estimates that 24 million people suffer fromAlzheimer’s disease and other dementias.1

• An estimated 26.2 percent of Americans ages 18 and older—about one in four adults—suffer from a diagnosablemental disorder in a given year. When applied to the 2004 U.S. Census residential population estimate for ages18 and older, that figure translates to 57.7 million people. Mental disorders are the leading cause of disability inthe United States and Canada for ages 15-44. Many people suffer from more than one mental disorder at a giventime. Nearly half (45 percent) of those with any mental disorder meet the criteria for two or more disorders.2

• Mental illness, including suicide, accounts for more than 15 percent of the burden of disease in establishedmarket economies, which is more than the disease burden caused by all cancers.2 Worldwide, about 877,000people die by suicide every year.1

• In 2006, suicide was the eleventh leading cause of death in the United States, accounting for 33,300 deaths. Theoverall rate was 10.9 suicide deaths per 100,000 people. An estimated 12 to 25 attempted suicides occur perevery suicide death. Suicide was the seventh leading cause of death for males and the sixteenth leading causeof death for females in 2006. Almost four times as many males as females die by suicide.2

• Serious mental illnesses cost the United States more than $317 billion in lost wages, health care expendituresand disability benefits each year.2

Addictive Disorders• Substance abuse affects an estimated 25 million Americans, and another 40 million people, such as families of

abusers and those injured or killed by intoxicated drivers, are affected indirectly. Alcoholism afflicts 10 millionadults and 3 million children. An estimated 12.5 million Americans are addicted to other drugs, such as barbitu-rates, opiates, sedatives, and hallucinogens.3

• The estimated economic cost of alcohol abuse in 1998 was $184.6 billion, or $638 for every man, woman, andchild in the United States. Alcohol-related injuries alone cost an estimated $47 billion annually.4

• The Office of National Drug Control Policy reported that in 2002, the economic cost of drug abuse to the UnitedStates was $180.9 billion.5

Alzheimer’s Disease/Dementias6

• Today, 5.3 million people are living with Alzheimer’s—every 70 seconds, someone develops the disease. Some5.1 million people with Alzheimer’s are age 65 and older, but an estimated 500,000 people younger than age65 either have Alzheimer’s or another dementia.

• Alzheimer’s disease is the most common type of dementia, accounting for 60 percent to 80 percent of cases.Vascular dementia (also called multi-infarct dementia, post-stroke dementia, or vascular cognitive impairment)is considered the second most common type after Alzheimer’s.

• In 2000, an estimated 411,000 new cases of Alzheimer’s were diagnosed. That number was expected toincrease to 454,000 new cases a year by 2010; 615,000 annually by 2030; and 959,000 new cases a year by2050. By that year, the number of people age 65 and older with Alzheimer’s could be as high as 16 millionunless researchers find a way to prevent or treat the disease.

• In 2006, Alzheimer’s was listed as the “underlying cause of death” for 72,432 Americans. In 1991, only 14,112death certificates recorded Alzheimer’s as the underlying cause. From 2000-2006, deaths from Alzheimer’sincreased by 46.1 percent, while the number one cause of death, heart disease, decreased by 11.1 percent. In2006, Alzheimer’s was the seventh leading cause of death for people of all ages and the fifth in people age 65and older.


S E L E C T E D F A C T S A B O U T M E N T A L I L L N E S S E S A N DA D D I C T I V E D I S O R D E R SAlzheimer’s Disease/Dementias6 (continued) • Some 70 percent of people with Alzheimer’s and other dementias live at home, where they are cared for by

family and friends. In 2009, nearly 11 million family members and friends provided an average of 21.9 hours ofunpaid care per week for a person with Alzheimer’s disease or another dementia, for a total of about 12.5 billionhours of care. That year, the estimated economic value of the unpaid care was $144 billion. About 60 percent offamily and other unpaid caregivers of people with Alzheimer’s disease and other dementias are women.

• People with Alzheimer’s disease and other dementias are high users of health care, long-term care and hospice.Total payments for these types of care from all sources, including Medicare and Medicaid, are three times higherfor older people with Alzheimer’s and other dementias than for other older people. Based on the average perperson payments from all sources for health care and long-term care services for people aged 65 and older withAlzheimer’s disease and other dementias in 2004, total payments for 2010 are expected to be $172 billion,including $123 billion for Medicare and Medicaid.

Anxiety Disorders2

• Anxiety disorders, which include panic disorder, obsessive-compulsive disorder, post-traumatic stress disorder,generalized anxiety disorder, and phobias, affect some 40 million adults ages 18 and older, or about 18.1 percentof people in this age group in a given year.

• About 6.8 million American adults ages 18 and older, or about 3.1 percent of people in this age group, havegeneralized anxiety disorder (GAD) each year. The median age of onset is 31.

• Some 2.2 million U.S. adults age 18 and older, or about 1 percent of people in that age group, have obsessive-compulsive disorder (OCD) in a given year. The median age of onset is 19.

• Approximately 6 million Americans ages 18 and older have panic disorder, or about 2.7 percent of people inthat age group.

• About 7.7 million U.S. adults age 18 and older, or about 3.5 percent of that age group, have post-traumaticstress disorder (PTSD), in a given year. PTSD can develop at any age, but the median age of onset is 23.

• About 15 million American adults ages 18 and older, or about 6.8 percent of people in this age group, havesocial phobia yearly. The disorder typically begins around age 13.

Attention-Deficit/Hyperactivity Disorder (ADHD) • Attention-deficit/hyperactivity disorder (ADHD) is one of the most common reasons children are referred for

mental health services. It affects as many as one in every 20 children, and boys are three to four times morelikely than girls to experience the disorder.3

• ADHD usually becomes evident in preschool or early elementary years. The median age of onset is seven years,although the disorder can persist into adolescence and occasionally into adulthood.2 Although most children withADHD have normal or above-normal intelligence, 40 percent to 60 percent have serious learning difficulties.3

Autism2

• (Estimating the prevalence of autism is difficult and controversial due to differences in the ways that cases areidentified and defined, differences in study methods, and changes in diagnostic criteria.) A recent study reportedthe prevalence of autism among 8-year-olds to be about 1 in 110. Autism and other autism spectrum disorders(ASDs) develop in childhood and generally are diagnosed by age three.

• Autism is about four times more common in boys than girls; however, girls with the disorder tend to have moresevere symptoms and greater cognitive impairment.

Depression• Mood disorders, which include major depressive disorder, dysthymic disorder, and bipolar disorder, affect

nearly 21 million U.S. adults, or about 9.5 percent of the U.S. population age 18 and older in a given year. Themedian age of onset for mood disorders is age 30. These disorders often co-occur with anxiety disorders andsubstance abuse.2


S E L E C T E D F A C T S A B O U T M E N T A L I L L N E S S E S A N DA D D I C T I V E D I S O R D E R SDepression (continued) • Bipolar disorder affects about 5.7 million American adults, or about 2.6 percent of the U.S. population age 18

and older yearly. The median age of onset for this disorder is age 25.2

• Dysthymic disorder (chronic, mild depression) affects some 1.5 percent of the U.S. population age 18 and older(about 3.3 million people) in a given year. The median age of onset is 31.2

• Major depressive disorder is the leading cause of disability in the United States for ages 15-44, affecting nearly15 million American adults (about 6.7 percent of the U.S. population age 18 and older) in a given year. The dis-order can develop at any age, but the median age at onset is 32. Major depressive disorder is more prevalent inwomen than in men.2

• Clinical depression has become one of America’s most costly illnesses. Left untreated, depression is as costly asheart disease or AIDS to the U.S. economy, costing more than $43.7 billion in absenteeism from work (morethan 200 million days lost from work each year), lost productivity and direct treatment costs. Depression tends toaffect people in their prime working years and may last a lifetime if untreated. More than 80 percent of peoplewith clinical depression can be successfully treated.3

Eating Disorders• In their lifetime, an estimated 0.6 percent of the adult population in the United States will suffer from anorexia,

1.0 percent from bulimia, and 2.8 percent from a binge-eating disorder. Women are much more likely thanmales to develop an eating disorder. They are three times as likely to experience anorexia (0.9 percent of womenvs. 0.3 percent of men) and bulimia (1.5 percent of women vs. 0.5 percent of men). They are also 75 percentmore likely to have a binge-eating disorder (3.5 percent of women vs. 2.0 percent of men).2

• The mortality rate among people with anorexia has been estimated at 0.56 percent per year, or approximately5.6 percent per decade, which is about 12 times higher than the annual death rate due to all causes of deathamong females ages 15-24 in the general population.2

• An estimated 32.7 percent of U.S. adults 20 years and older are overweight, 34.3 percent are obese, and 5.9percent are extremely obese. Compared with whites, African Americans have a 51 percent higher prevalencerate of obesity and Hispanics have a 21 percent higher rate.7

• In 2004, the U.S. Centers for Disease Control and Prevention (CDC) ranked obesity as the number one healthrisk facing America. Obesity causes an estimated 400,000 deaths a year and costs the national economy nearly$122.9 billion annually.8

Premenstrual Disorders• Premenstrual syndrome (PMS) is estimated to affect up to 75 percent of women during their childbearing years.

It occurs more often in women between their late 20s and early 40s, those with at least one child, those with afamily history of a major depressive disorder, or women with a past medical history of either postpartum depres-sion or an affective mood disorder. Up to 60 percent of women with severe PMS have an underlying psychiatricdisorder.9

• Premenstrual dysphoric disorder (PMDD), a very severe form of PMS, affects about 5 percent of menstruatingwomen.10

Schizophrenia• Schizophrenia strikes some 2.4 million American adults, or about 1.1 percent of the U.S. population age 18 and

older, in a given year, affecting men and women with equal frequency. The disorder first appears in men in theirlate teens or early 20s, while women are generally affected in their 20s or early 30s.2

• The appearance of schizophrenic symptoms before age 12 is rare—less than one-sixtieth as common as theadult-onset type. Neurodevelopmental damage seems to be greater in childhood schizophrenia than in the adult-onset type. Most schizophrenic children show delays in language and other functions long before their psychoticsymptoms (hallucinations, delusions, and disordered thinking) appear, usually at age seven or later. In the firstyears of life, about 30% of these children have transient symptoms of pervasive developmental disorder, such asrocking, posturing, and arm flapping.3


S E L E C T E D F A C T S A B O U T M E N T A L I L L N E S S E S A N DA D D I C T I V E D I S O R D E R SSchizophrenia (continued) • Schizoaffective disorder, a combination of symptoms of schizophrenia and a mood disorder, may range from

two to five in 1,000 people (i.e., 0.2 percent to 0.5 percent) and may account for one-fourth or even one-third ofall people with schizophrenia.11

Sources:1. World Health Organization, www.who.int2. National Institute of Mental Health, www.nimh.nih.gov3. Mental Health America, www.mentalhealthamerica.net, www.nmha.org4. Community Safety Series, www.be-safe.org5. U.S. Department of Justice, www.usdoj.gov6. Alzheimer’s Association, www.alz.org7. Centers for Disease Control and Prevention, www.cdc.gov8. ObesityinAmerica.org, (The Endocrine Society and The Hormone Foundation), www.obesityinamerica.org9. National Library of Medicine, www.nlm.nih.gov

10. Madison Institute of Medicine, Inc., www.pmdd.factsforhealth.org11. National Alliance on Mental Illness, www.nami.org


The U.S. system of new drug approvals is perhapsthe most rigorous in the world.

It takes 10-15 years, on average, for an experimentaldrug to travel from lab to U.S. patients, according tothe Tufts Center for the Study of Drug Development,based on drugs approved from 1994 through 1998.Only five in 5,000 compounds that enter preclinicaltesting make it to human testing. And only one of thosefive is approved for sale.

On average, it costs a company $1.3 billion to getone new medicine from the laboratory to U.S. patients,according to a 2007 study by the Tufts Center for theStudy of Drug Development.

Once a new compound has been identified in thelaboratory, medicines are developed as follows:

Preclinical Testing. A pharmaceutical company con-ducts laboratory and animal studies to show biologicalactivity of the compound against the targeted disease,and the compound is evaluated for safety.

Investigational New Drug Application (IND). Aftercompleting preclinical testing, a company files an INDwith the U.S. Food and Drug Administration (FDA) tobegin to test the drug in people. The IND shows resultsof previous experiments; how, where and by whomthe new studies will be conducted; the chemicalstructure of the compound; how it is thought to workin the body; any toxic effects found in the animalstudies; and how the compound is manufactured. Allclinical trials must be reviewed and approved by theInstitutional Review Board (IRB) where the trials willbe conducted. Progress reports on clinical trials mustbe submitted at least annually to FDA and the IRB.

Clinical Trials, Phase I. These tests usually involveabout 20 to 100 normal, healthy volunteers. The tests

study a drug’s safety profile, including the safe dosagerange. The studies also determine how a drug isabsorbed, distributed, metabolized, and excreted aswell as the duration of its action.

Clinical Trials, Phase II. In this phase, controlledtrials of approximately 100 to 500 volunteer patients(people with the disease) assess a drug’s effectivenessand determine the early side effect profile.

Clinical Trials, Phase III. This phase usually involves1,000 to 5,000 patients in clinics and hospitals.Physicians monitor patients closely to confirm efficacyand identify adverse events.

New Drug Application (NDA)/Biologic LicenseApplication (BLA). Following the completion of allthree phases of clinical trials, a company analyzes allof the data and files an NDA or BLA with FDA if thedata successfully demonstrate both safety and effec-tiveness. The applications contain all of the scientificinformation that the company has gathered.Applications typically run 100,000 pages or more.The average review time for the 25 new therapeuticsapproved by the FDA in 2009 was 13.3 months.

Approval. Once FDA approves an NDA or BLA, thenew medicine becomes available for physicians toprescribe. A company must continue to submit periodicreports to FDA, including any cases of adverse reactionsand appropriate quality-control records. For somemedicines, FDA requires additional trials (Phase IV) toevaluate long-term effects.

Discovering and developing safe and effective newmedicines is a long, difficult, and expensive process.Pharmaceutical companies invested an estimated$65.3 billion in research and development in 2009.

T H E D R U G D E V E L O P M E N T A N D A P P R O V A L P R O C E S S

T H E D R U G D I S C O V E R Y , D E V E L O P M E N T A N D A P P R O V A L P R O C E S S

It takes 10-15 years on average for an experimental drug to travel from the lab to U.S. patients. Only five in 5,000compounds that enter preclinical testing make it to human testing. One of these five tested in people is approved.

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New Medicines. New Hope.®

Pharmaceutical Research and Manufacturers of America950 F Street, NWWashington, DC 20004

www.phrma.org | www.innovation.org | www.pparx.org | www.buysafedrugs.info | www.sharingmiracles.com 7/10

T R A C K I N G T H E P H A R M A C E U T I C A L A N DB I O T E C H N O L O G Y R E S E A R C H P I P E L I N E S

Today, more than 2,900 new medicines are in devel-opment in the United States. Many of these potentialnew medicines will fail in clinical trials, but somemay represent tomorrow’s new treatments. Bringingeach new medicine to patients will require, onaverage, 10 to 15 years of testing and review.

PhRMA publishes several reports that track the pharmaceutical and biotechnology research pipelinesfor many diseases, including the leading causes ofdeath among Americans—heart disease, cancer, andstroke. The reports include medicines currently inclinical trials or at the U.S. Food and DrugAdministration (FDA) for review. Below is a summaryof our most popular reports.

• Cancer—There are few things that cause patientsmore fear and uncertainty as a cancer diagnosis.Yet today—because of a steady stream of new andimproved medicines and treatments—cancer canincreasingly be managed and even beaten. The2009 report found 861 medicines in the pipeline.

• Heart Disease and Stroke—Keeping up themomentum of drug discovery that has helped cutdeaths from heart disease and stroke in half in thepast three decades, biopharmaceutical companiesare working on new medicines for these diseases.The 2009 report found 312 medicines in thepipeline.

• Diabetes—Approximately 4,110 people are diag-nosed with diabetes every day. To help fight this

disease, pharmaceutical and biotechnologyresearchers are working on new medicines to treatit and related conditions. The 2010 report found235 medicines in the pipeline.

• Mental Illnesses—Pharmaceutical and biotech-nology researchers are testing many new medi-cines to help the more than 450 million peopleworldwide who suffer from some form of mentalillness. The 2010 report found 313 medicines inthe pipeline.

• Biotechnology—Millions of people have alreadybenefited from medicines and vaccines developedthrough biotechnology, and a new report offershope that many more will benefit in the future. The2008 report found 633 medicines in the pipeline.

• Children—Biopharmaceutical researchers aretesting medicines to meet the special needs of chil-dren. These medicines offer hope that thesignificant improvements achieved in children’shealth over the past few decades will continue andeven accelerate. The 2010 report found 234 medi-cines in clinical trials.

• Older Americans—The population of Americansover 65 is surging, and the pace will only increaseover the coming years. As life expectancy contin-ues to expand, older Americans face new andgrowing challenges to their health, productivity andindependence. The 2008 report found 1,026 medi-cines in the pipeline.

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