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2012 Repo r t

Medicines in Development Mental Illnesses

presented by america’s biopharmaceutical research companies

Pharmaceutical Research Companies Are Developing Nearly 200 Medicines to Treat Mental Illnesses and Addictive Disorders

ADDICTIVE DISORDERS ANXIETY DISORDERS ATTENTION-DEFICIT/HYPERACTIVITY DISORDERER DEPRESSION DEVELOPMENTAL DISORDERS EATING DISORDERS PERSONALITY DISORDERS Medicines in Development For Selected Mental Illnesses SSLEEEP DISORDERS And Addictive Disorders 1 in 4 American adults suffer from a 52 diagnosable mental illness

America’s biopharmaceutical research com- annually in lost wages, health care expendi- panies are developing 187 medicines to help tures, and disability benefits. the nearly 60 million American adults now suf- 36 Examples of some medicines now being fering from some form of mental illness—from tested to treat mental illnesses include: anxiety to depression and from schizophrenia to addictive disorders, such as dependence • A medicine to potentially treat the various on or . All of the medicines are symptoms associated with schizophrenia, 26 26 either in clinical trials or awaiting review by with diminished side effects. the Food and Administration. Over the • An intranasal medicine for the treatment past half century, biopharmaceutical research of anxiety which has shown to improve has helped transform mental illnesses from symptoms within several minutes of misunderstood causes of shame and fear into administration. often highly treatable conditions. For example, Researching and developing new medicines medicines for treating depression are helping remains a risky investment and lengthy thousands of people live productive lives and process. But advances in our understanding breakthrough schizophrenia medicines have of mental illnesses and how to treat them have enabled patients to be treated in the commu- allowed America’s biopharmaceutical com- nity rather than being institutionalized. panies to conduct the cutting-edge research Despite such progress, mental illnesses con- needed to reduce the destructive toll of these

tinue to exact a heavy human and economic disorders and to allow more patients to lead s s toll. The National Institute of Mental Health healthier, happier, more productive lives. (NIMH) estimates that 1 in 4 American adults Anxiety Addictive Disorder Disorder suffer from a diagnosable mental disorder. Ac- Depression cording to the NIMH, serious mental illnesses Schizophrenia cost the United States more than $317 billion Medicines in Development for Mental Illnesses

Medicines in Development for Mental Illnesses*

Addictive Disorders 26

Anxiety Disorders 26

Attention-Deficit/ Hyperactivity Disorder 20

Cognition Disorders 10

Depression 52

Developmental Disorders 10

Eating Disorders 3

Schizophrenia 36

Sleep Disorders 22

Other 9

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

Addictive Disorders

Product Name Sponsor Indication Development Status*

ALKS33 Alkermes drug dependence Phase I (samidorphan) Cambridge, MA (617) 494-0171

ALKS5461 Alkermes dependence Phase I (/samidorphan) Cambridge, MA (see also depression) (617) 494-0171

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

buprenorphine/naloxone Orexo dependence Phase III fixed-dose combination Uppsala, Sweden www.orexo.com

buprenorphine/naloxone BioDelivery Sciences International opioid dependence Phase I transmucosal Raleigh, NC (919) 582-9050

Ch-mAb7F9 InterveXion drug dependence Phase I Little Rock, AR (501) 554-2377

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

2 Medicines in Development Mental Illnesses 2012 Medicines in Development for Mental Illnesses

Addictive Disorders

Product Name Sponsor Indication Development Status

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

Psyadon Pharmaceuticals pathological gambling Phase II (PSYRX101) Germantown, MD (see also other) (301) 919-2020

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

GSK561679 GlaxoSmithKline stress-related alcohol abuse Phase II () Rsch. Triangle Park, NC (see also anxiety) (888) 825-5249

ibudilast MediciNova dependence Phase I (MN-166/AV11) San Diego, CA (858) 373-1500

INT-0003/2005 Cary Pharmaceuticals nicotine dependence Phase I Great Falls, VA (703) 759-7460 IntelGenx (514) 331-7440 Quebec, Canada

KRL-901 Krele Pharmaceuticals alcohol dependence in clinical trials (TONIX Pharmaceuticals) (212) 908-9155 New York, NY

US WorldMeds opioid dependence Phase III Louisville, KY (502) 753-2094

MT-7716 Mitsubishi Tanabe Pharma alcohol dependence Phase I Development America (908) 607-1950 Warren, NJ

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

neboglamine Rottapharm | Madaus cocaine dependence Phase II (CR-224) Monza, Italy www.rotta.com

nepicastat oral Biotie Therapies cocaine dependence Phase II completed (SYN117) South San Francisco, CA (see also anxiety) (650) 244-4850

NIC002 Novartis Pharmaceuticals nicotine dependence Phase II East Hanover, NJ (888) 669-6682

NicVAX™ GlaxoSmithKline nicotine dependence (Fast Track) Phase III nicotine abuse vaccine Rsch. Triangle Park, NC (888) 825-5249 Nabi Biopharmaceuticals (800) 685-5579 Rockville, MD

Medicines in Development Mental Illnesses 2012 3 Medicines in Development for Mental Illnesses

Addictive Disorders

Product Name Sponsor Indication Development Status

OMS-403 Omeros nicotine dependence, Phase II Seattle, WA opioid dependence (206) 676-5000 New York State Psychiatric Institute New York, NY

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

Sabril® cocaine dependence (Fast Track) Phase II vigabatrin Deerfield, IL (866) 337-6996 ------methamphetamine addiction Phase I (866) 337-6996

SEL-068 Selecta Biosciences smoking dependence Phase I Watertown, MA (617) 923-1400

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

X-22 22nd Century Group smoking dependence Phase II Westport, CT (203) 222-7399

Anxiety Disorders

Product Name Sponsor Indication Development Status

ABT-436 Abbott Laboratories anxiety Phase I (-1b Abbott Park, IL (see also depression) (847) 937-6100 antagonist)

sublingual Pfizer anxiety Phase I completed New York, NY (860) 732-5156

alprazolam transdermal Nuvo Research panic disorder Phase I Mississauga, Canada (905) 673-6980

AVN 101 Avineuro Pharmaceuticals anxiety Phase II ( 6 receptor San Diego, CA (858) 436-1537 antagonist)

AVN 397 Avineuro Pharmaceuticals anxiety Phase II San Diego, CA (858) 436-1537

BNC210 Bionomics anxiety Phase I completed Adelaide, Australia (617) 621-7722 Ironwood Pharmaceuticals Cambridge, MA

4 Medicines in Development Mental Illnesses 2012 Medicines in Development for Mental Illnesses

Anxiety Disorders

Product Name Sponsor Indication Development Status

Columbia Northwest Pharmaceuticals post-traumatic stress disorder Phase II Bellevue, WA (877) 453-0404

CX157 CeNeRx Pharma anxiety Phase I Cary, NC (see also depression) (919) 234-4072

CXB722 CeNeRx BioPharma anxiety Phase II ( prodrug) Cary, NC (see also depression) (919) 234-4072

Cymbalta® Eli Lilly generalized anxiety disorder Phase III Indianapolis, IN (pediatric) (800) 545-5979 (see also depression)

ganaxolone Marinus Pharmaceuticals post-traumatic stress disorder Phase II Branford, CT (203) 315-0566

GSK561679 GlaxoSmithKline post-traumatic stress disorder Phase II (verucerfont) Rsch. Triangle Park, NC (see also addictive) (888) 825-5249

INTUNIV™ Shire Pharmaceuticals anxiety disorders (pediatric), Phase II Wayne, PA generalized anxiety disorder www.shire.com extended-release (pediatric), social phobia (pediatric)

KRL-104 Krele Pharmaceuticals generalized anxiety disorder in clinical trials (TONIX Pharmaceuticals) (212) 908-9155 New York, NY

nepicastat oral Biotie Therapies post-traumatic stress disorder Phase II (SYN117) South San Francisco, CA (see also addictive) (650) 244-4850

PH94B Pherin Pharmaceuticals generalized social phobia Phase II Los Altos, CA (650) 961-2703

PRX-3140 Nanotherapeutics post-traumatic stress disorder Phase II Alachua, FL (386) 462-9663

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

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

SRX246 Azevan Pharmaceuticals traumatic stress disorder Phase I Bethlehem, PA (610) 419-1057

Medicines in Development Mental Illnesses 2012 5 Medicines in Development for Mental Illnesses

Anxiety Disorders

Product Name Sponsor Indication Development Status

Lundbeck generalized anxiety disorder Phase I Deerfield, IL (see also depression) (866) 337-6996 Takeda Pharmaceuticals U.S.A. (877) 825-3327 Deerfield, IL

TGFK08AA Fabre-Kramer Pharmaceuticals generalized anxiety disorder Phase II Houston, TX (713) 975-6900

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

TO-2061 Transcept Pharmaceuticals obsessive-compulsive disorder Phase II (/) Pt. Richmond, CA (510) 215-3500

Lundbeck generalized anxiety disorder Phase III Deerfield, IL (see also depression) (866) 337-6996 Takeda Pharmaceuticals U.S.A. (877) 825-3327 Deerfield, IL ------anxiety disorders (pediatric) Phase II (866) 337-6996 (877) 825-3327

YKP-3089 SK Life Science anxiety Phase I Fair Lawn, NJ (see also depression) www.sklsi.com

Attention-Deficit/Hyperactivity Disorder

Product Name Sponsor Indication Development Status

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

AZD5213 AstraZeneca ADHD Phase I (histamine-3 receptor Wilmington, DE (800) 236-9933 antagonist)

bavisant Johnson & Johnson Pharmaceutical ADHD Phase II (JNJ-31001074) Research & Development (800) 817-5286 Raritan, NJ ------ADHD (pediatric) Phase II (800) 817-5286

COL-171 Collegium Pharmaceuticals ADHD in clinical trials Cumberland, RI (401) 762-2000

6 Medicines in Development Mental Illnesses 2012 Medicines in Development for Mental Illnesses

Attention-Deficit/Hyperactivity Disorder

Product Name Sponsor Indication Development Status

CX1739 Cortex Pharmaceuticals ADHD Phase I completed Irvine, CA (949) 727-3157

EB-1020 Euthymics Bioscience ADHD Phase I (triple ) Cambridge, MA (617) 758-0300

Eli Lilly ADHD (pediatric) Phase II (LY2216684) Indianapolis, IN (see also depression) (800) 545-5979

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

KRL-401 Krele Pharmaceuticals ADHD in clinical trials (TONIX Pharmaceuticals) (212) 908-9155 New York, NY

Purdue Pharma ADHD Phase III extended-release Stamford, CT (203) 588-8000

NWP06 NextWave Pharmaceuticals ADHD application submitted (methylphenidate Cupertino, CA (408) 342-1300 extended-release suspension)

NWP09 NextWave Pharmaceuticals ADHD Phase II (methylphenidate Cupertino, CA (408) 342-1300 extended-release chewable tablets)

OPC-34712 Lundbeck ADHD Phase II () Deerfield, IL (see also depression, schizophrenia) (866) 337-6996 Otsuka America Pharmaceutical (800) 562-3974 Rockville, MD

ORADUR-ADHD DURECT ADHD Phase I (sustained-release oral therapy) Cupertino, CA (408) 777-1417

sofinicline Abbott Laboratories ADHD Phase II Abbott Park, IL (847) 937-6100 NeuroSearch Ballerup, Denmark

SPN-810 Supernus Pharmaceuticals conduct disorder and impulsive Phase II () Rockville, MD aggression in ADHD (pediatric) (301) 838-2500

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

SPN-812 Supernus Pharmaceuticals ADHD (adults) Phase I/II Rockville, MD (301) 838-2500

Medicines in Development Mental Illnesses 2012 7 Medicines in Development for Mental Illnesses

Attention-Deficit/Hyperactivity Disorder

Product Name Sponsor Indication Development Status

TC-5619 Targacept ADHD Phase II Winston-Salem, NC (see also cognition) (336) 480-2100

TD-9855 Theravance ADHD Phase II South San Francisco, CA (877) 275-8479

Cognition Disorders

Product Name Sponsor Indication Development Status

AMG 747 Amgen cognitive deficits and negative Phase II (GlyT-1 inhibitor) Thousand Oaks, CA symptoms associated with (800) 772-6436 schizophrenia

AQW051 Novartis Pharmaceuticals cognition disorders associated with Phase II East Hanover, NJ schizophrenia (888) 669-6682

AV965 Avera Pharmaceuticals cognition impairment associated with Phase I (serotonin-1A antagonist) San Diego, CA schizophrenia (858) 847-0650

davunetide intranasal Allon Therapeutics mild cognitive impairment associated Phase II completed Vancouver, Canada with schizophrenia (604) 736-0634

irdabisant Cephalon cognition disorders associated with Phase I (CEP-26401) Frazer, PA schizophrenia (610) 344-0200

LU AE58054 Lundbeck cognitive impairment associated Phase II Deerfield, IL with schizophrenia (800) 455-1141

RG1662 Roche cognitive disorders in patients with Phase I (GABA receptor) Nutley, NJ Down’s syndrome (973) 235-5000

sabcomeline BrainCells cognitive decline associated with Phase I (BCI-224) San Diego, CA schizophrenia (858) 812-7700

SYN-120 Biotie Therapies cognition disorders associated with Phase I South San Francisco, CA schizophrenia (650) 244-4850

TC-5619 Targacept cognition disorders associated with Phase II Winston-Salem, NC schizophrenia (336) 480-2100 (see also ADHD)

8 Medicines in Development Mental Illnesses 2012 Medicines in Development for Mental Illnesses

Depression

Product Name Sponsor Indication Development Status

ABT-436 Abbott Laboratories major depressive disorder Phase I (vasopressin-1b receptor Abbott Park, IL (see also anxiety) (847) 937-6100 antagonist)

ADX N05 Aerial BioPharma depressive disorders Phase I Morrisville, NC (adjunctive treatment) (919) 460-9500 (see also sleep)

Euthymics Bioscience major depressive disorder Phase II/III (EB-1010) Cambridge, MA (617) 758-0300

ARA 290 Araim Pharmaceuticals depression Phase II Ossining, NY (914) 762-7586

ALKS5461 Alkermes major depressive disorder Phase II (buprenorphine/samidorphan) Cambridge, MA (see also addictive) (617) 494-0171

/ Otsuka America Pharmaceutical major depressive disorder Phase III fixed-dose combination Rockville, MD (800) 562-3974

Cephalon bipolar depression Phase III Frazer, PA (see also eating) (610) 344-0200

AZD6765 AstraZeneca major depressive disorder Phase II (NMDA ) Wilmington, DE (800) 236-9933

BCI-632 BrainCells major depressive disorder Phase I (mGluR2/3 antagonist) San Diego, CA (858) 812-7700

BCI-838 BrainCells major depressive disorder Phase I (mGluR2/3 antagonist) San Diego, CA (858) 812-7700

BCI-952 BrainCells major depressive disorder Phase II (/melatonin) San Diego, CA (858) 812-7700

bipolar disorder NCE Eli Lilly bipolar disorder Phase I Indianapolis, IN (800) 545-5979

BMS-820836 Bristol-Myers Squibb major depressive disorder Phase II (triple reuptake inhibitor) Princeton, NJ (800) 332-2056

Forest Laboratories bipolar disorder Phase III New York, NY (see also schizophrenia) (800) 947-5227 Gedeon Richter ------Budapest, Hungary bipolar depression, Phase II major depressive disorder (800) 947-5227

Medicines in Development Mental Illnesses 2012 9 Medicines in Development for Mental Illnesses

Depression

Product Name Sponsor Indication Development Status

CX157 CeNeRx Pharma treatment-resistant depression Phase I Cary, NC (see also anxiety) (919) 234-4072

CXB722 CeNeRx BioPharma depressive disorders Phase II (pivagabine prodrug) Cary, NC (see also anxiety) (919) 234-4072

Cymbalta® Eli Lilly major depressive disorder (pediatric) Phase III duloxetine Indianapolis, IN (see also anxiety) (800) 545-5979

depression NCE Eli Lilly depression Phase I Indianapolis, IN (800) 545-5979

depression NCE Eli Lilly depression Phase I Indianapolis, IN (800) 545-5979

DSP-1053 Sunovion Pharmaceuticals major depressive disorder Phase I Marlborough, MA (508) 481-6700

edivoxetine Eli Lilly major depressive disorder Phase III (LY2216684) Indianapolis, IN (see also ADHD) (800) 545-5979

elpetrigine Jazz Pharmaceuticals bipolar disorder Phase I Dublin, Ireland www.jazzpharma.com

GLYX-13 Naurex major depressive disorder Phase II Evanston, IL (847) 871-0377

HT-2157 Dart NeuroScience major depressive disorder Phase I/II San Diego, CA (858) 736-3060

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

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

Latuda® Sunovion Pharmaceuticals bipolar disorder, major depressive Phase III Marlborough, MA disorder (508) 481-6700

Forest Laboratories major depressive disorder Phase III New York, NY (800) 947-5227

10 Medicines in Development Mental Illnesses 2012 Medicines in Development for Mental Illnesses

Depression

Product Name Sponsor Indication Development Status

Columbia Northwest Pharmaceuticals suicide ideation in patients with Phase II Bellevue, WA depressive mood disorders (877) 453-0404

Motiva™ Neuren Pharmaceuticals major depressive disorder post Phase II nefiracetam Auckland, New Zealand stroke www.neurenpharma.com

Tetragenex Pharmaceuticals major depressive disorder Phase II completed Park Ridge, NJ (201) 505-1300

NSI-189 Neuralstem major depressive disorder Phase I Rockville, MD (301) 366-4960

OPC-34712 Lundbeck major depressive disorder Phase III (brexpiprazole) Deerfield, IL (see also ADHD, schizophrenia) (866) 337-6996 Otsuka America Pharmaceutical (800) 562-3974 Rockville, MD

PNB01 PharmaNeuroBoost major depressive disorder Phase III (/) Alken, Belgium www.pharmaneuroboost.com

Pristiq® Pfizer major depressive disorder Phase III New York, NY (pediatric) (860) 732-5156

RG1578 Roche major depressive disorder Phase II (mGluR2 modulator) Nutley, NJ (973) 235-5000

RG7090 Roche major depressive disorder Phase II (mGluR5 antagonist) Nutley, NJ (see also developmental) (973) 235-5000

RO4995819 Roche major depressive disorder Phase II Nutley, NJ (973) 235-5000

Rozerem® Takeda Pharmaceuticals U.S.A. bipolar depression, bipolar disorder Phase II ramelteon Deerfield, IL (see also sleep) (877) 825-3327

sabcomeline BrainCells major depressive disorder Phase I (BCI-224) San Diego, CA (858) 812-7700

Saphris® Merck bipolar disorder (pediatric) Phase III Whitehouse Station, NJ (see also schizophrenia) (800) 672-6372

SEP-228432 Sunovion Pharmaceuticals depressive disorders Phase I Marlborough, MA (508) 481-6700

Medicines in Development Mental Illnesses 2012 11 Medicines in Development for Mental Illnesses

Depression

Product Name Sponsor Indication Development Status

Seroquel XR® AstraZeneca bipolar depression (pediatric) Phase III completed fumarate Wilmington, DE (800) 236-9933 extended-release

SKL-10406 SK Life Science major depressive disorder Phase I Fair Lawn, NJ www.sklsi.com

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

tasimelteon Vanda Pharmaceuticals major depressive disorder Phase I/II Washington, DC (see also sleep) (202) 734-3400

tedatioxetine Lundbeck major depressive disorder Phase I Deerfield, IL (see also anxiety) (866) 337-6996 Takeda Pharmaceuticals U.S.A. (877) 825-3327 Deerfield, IL

TGBA01AD Fabre-Kramer Pharmaceuticals major depressive disorder Phase II Houston, TX (713) 975-6900

Auspex Pharmaceuticals major depressive disorder Phase I deuterium-substituted La Jolla, CA (858) 558-2400

vortioxetine Lundbeck major depressive disorder Phase III Deerfield, IL (see also anxiety) (866) 337-6996 Takeda Pharmaceuticals U.S.A. (877) 825-3327 Deerfield, IL ------depressive disorders (pediatric) Phase II (866) 337-6996 (877) 825-3327

Vyvanse® Shire Pharmaceuticals bipolar depression, major depressive Phase III lisdexamfetamine Wayne, PA disorder www.shire.com (see also eating, schizophrenia, sleep)

YKP-3089 SK Life Science bipolar disorder Phase I Fair Lawn, NJ (see also anxiety) www.sklsi.com

12 Medicines in Development Mental Illnesses 2012 Medicines in Development for Mental Illnesses

Developmental Disorders

Product Name Sponsor Indication Development Status

AFQ056 Novartis Pharmaceuticals fragile X syndrome Phase II/III East Hanover, NJ (888) 669-6682

AT001 Autism Therapeutics autism (Fast Track) Phase III (-rapid dissolve) London, United Kingdom www.autismtherapeutics.com (Orphan Drug)

AT002 Autism Therapeutics fragile X syndrome Phase I/II (fenobam) London, United Kingdom www.autismtherapeutics.com (Orphan Drug)

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

CYP2001 Cypress Bioscience autism (Fast Track) Phase I ( intranasal) (Royalty Pharma) (212) 883-0200 New York, NY

Namenda® Forest Laboratories Asperger syndrome (pediatric), Phase II New York, NY autism (pediatric) (800) 947-5227

RG7090 Roche fragile X syndrome Phase II (mGluR5 antagonist) Nutley, NJ (see also depression) (973) 235-5000

RG7314 Roche autism Phase I (vasopressin-1 receptor Nutley, NJ (973) 235-5000 antagonist)

STX107 Seaside Therapeutics fragile X syndrome Phase II (mGluR5 antagonist) Cambridge, MA (617) 374-9009

STX209 Seaside Therapeutics fragile X syndrome, Phase III (GABA-B receptor ) Cambridge, MA fragile X syndrome (pediatric) (617) 374-9009 (Orphan Drug) ------autism (pediatric) Phase II (617) 374-9009

Medicines in Development Mental Illnesses 2012 13 Medicines in Development for Mental Illnesses

Eating Disorders

Product Name Sponsor Indication Development Status

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

armodafinil Cephalon binge-eating disorder Phase III Frazer, PA (see also depression) (610) 344-0200

Vyvanse® Shire Pharmaceuticals binge-eating disorder Phase II lisdexamfetamine Wayne, PA (see also depression, schizophrenia, www.shire.com sleep)

Schizophrenia

Product Name Sponsor Indication Development Status

Abilify® IM Depot Lundbeck schizophrenia application submitted aripiprazole Deerfield, IL (866) 337-6996 (once-monthly) Otsuka America Pharmaceutical (800) 562-3974 Rockville, MD

ABT-126 Abbott Laboratories schizophrenia Phase II (alpha-7 neuronal nicotinic Abbott Park, IL (847) 937-6100 receptor antagonist)

ABT-288 Abbott Laboratories schizophrenia Phase II (neurotransmitter receptor Abbott Park, IL (847) 937-6100 modulator)

ALKS9070/ALKS9072 Alkermes schizophrenia Phase III (aripiprazole injectable) Cambridge, MA (617) 494-0171

AM-831 ACADIA Pharmaceuticals schizophrenia Phase I San Diego, CA (858) 558-2871

AMG 579 Amgen schizoaffective disorder, Phase I (PDE10A inhibitor) Thousand Oaks, CA schizophrenia (800) 772-6436

ATON-005 ATON Pharma psychotic disorders Phase I Lawrenceville, NJ (609) 671-9010

AVN 211 Avineuro Pharmaceuticals schizophrenia Phase II completed (serotonin 6 receptor San Diego, CA (858) 436-1537 antagonist)

14 Medicines in Development Mental Illnesses 2012 Medicines in Development for Mental Illnesses

Schizophrenia

Product Name Sponsor Indication Development Status

bitopertin Roche schizophrenia Phase III (RG1678) Nutley, NJ (973) 235-5000

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

cariprazine Forest Laboratories schizophrenia Phase III New York, NY (see also depression) (800) 947-5227 Gedeon Richter Budapest, Hungary

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

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

EVP-6124 EnVivo Pharmaceuticals schizophrenia Phase II (α7-nAChR agonist) Watertown, MA (617) 225-4250

Fanapt® Novartis Pharmaceuticals schizophrenia Phase I/II East Hanover, NJ (888) 669-6682 injectable

GSK239512 GlaxoSmithKline Phase II completed Rsch. Triangle Park, NC (888) 825-5249

Invega® Johnson & Johnson Pharmaceutical schizoaffective disorder Phase III Research & Development (prevention of relapse) (800) 817-5286 Raritan, NJ

ITI-007 Intra-Cellular Therapies schizophrenia Phase II (high-dose formulation) New York, NY (see also sleep) (212) 923-3344

JNJ-17305600 Johnson & Johnson Pharmaceutical schizophrenia Phase I Research & Development (800) 817-5286 Raritan, NJ

JNJ-37822681 Johnson & Johnson Pharmaceutical schizophrenia Phase II Research & Development (800) 817-5286 Raritan, NJ

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

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

Medicines in Development Mental Illnesses 2012 15 Medicines in Development for Mental Illnesses

Schizophrenia

Product Name Sponsor Indication Development Status

INVENT Pharmaceuticals psychotic disorders Phase I completed oral disintegrating tablet Del Mar, CA (858) 945-8576

OPC-34712 Lundbeck schizophrenia Phase III (brexpiprazole) Deerfield, IL (see also ADHD, depression) (866) 337-6996 Otsuka America Pharmaceutical (800) 562-3974 Rockville, MD

PF-02545920 Pfizer schizophrenia Phase II New York, NY (860) 732-5156

PF-04958242 Pfizer schizophrenia Phase I New York, NY (860) 732-5156

PF-05180999 Pfizer schizophrenia Phase I New York, NY (860) 732-5156

pitolisant Bioprojet schizophrenia Phase II Paris, France www.bioprojet.fr Ferrer www.ferrergrupo.com Barcelona, Spain

pomaglumetad Eli Lilly schizophrenia (monotherapy) Phase III (LY2140023) Indianapolis, IN (800) 545-5979 ------schizophrenia (add-on therapy) Phase II (800) 545-5979

RP5063 Reviva Pharmaceuticals schizoaffective disorder, Phase II San Jose, CA schizophrenia (408) 960-2209

Saphris® Merck schizophrenia (pediatric) Phase I asenapine Whitehouse Station, NJ (see also depression) (800) 672-6372

SB773812 GlaxoSmithKline schizophrenia Phase II completed Rsch. Triangle Park, NC (888) 825-5249

SLV 354 Abbott Laboratories schizophrenia Phase I Abbott Park, IL (847) 937-6100

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

Vyvanse® Shire Pharmaceuticals schizophrenia Phase II lisdexamfetamine Wayne, PA (see also depression, eating, sleep) www.shire.com

Lundbeck schizophrenia Phase II Deerfield, IL (866) 337-6996

16 Medicines in Development Mental Illnesses 2012 Medicines in Development for Mental Illnesses

Sleep Disorders

Product Name Sponsor Indication Development Status

ABT-652 Abbott Laboratories excessive daytime sleepiness Phase I completed (histamine H3 receptor Abbott Park, IL (847) 937-6100 modulator)

ADX N05 Aerial BioPharma excessive daytime sleepiness Phase II Morrisville, NC (see also depression) (919) 460-9500

E2006 Eisai insomnia Phase I Woodcliff Lake, NJ (888) 274-2378

GSK649868 GlaxoSmithKline Phase II ( antagonist) Rsch. Triangle Park, NC (888) 825-5249

ITI-007 Intra-Cellular Therapies sleep maintenance insomnia Phase II (low-dose formulation) New York, NY (see also schizophrenia) (212) 923-3344

KRL-103 Krele Pharmaceuticals sleep disorders in clinical trials (TONIX Pharmaceuticals) (212) 980-9155 New York, NY

Lunesta® Sunovion Pharmaceuticals ADHD-associated insomnia Phase III completed eszopiclone Marlborough, MA (pediatric) (508) 481-6700

melatonin controlled-release Neurim Pharmaceuticals sleep disorders Phase III (Orphan Drug) Tel-Aviv, Israel www.neurim.com

MK-3697 Merck insomnia Phase II Whitehouse Station, NJ (800) 672-6372

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

MK-7288 Merck excessive daytime sleepiness Phase I Whitehouse Station, NJ (800) 672-6372

Neu-P11 Neurim Pharmaceuticals insomnia Phase II Tel-Aviv, Israel www.neurim.com

Rozerem® Takeda Pharmaceuticals U.S.A. insomnia (pediatric) Phase I completed ramelteon Deerfield, IL (see also depression) (877) 825-3327

SKP-1041 Somnus Therapeutics sleep disorders Phase II completed (zaleplon controlled-release) Branchburg, NJ (908) 901-0300

Merck insomnia Phase III (MK-4305) Whitehouse Station, NJ (800) 672-6372

tasimelteon Vanda Pharmaceuticals insomnia Phase III Washington, DC (see also depression) (202) 734-3400

Medicines in Development Mental Illnesses 2012 17 Medicines in Development for Mental Illnesses

Sleep Disorders

Product Name Sponsor Indication Development Status

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

Vimpat® UCB sleep disorders Phase I lacosamide Smyrna, GA (770) 970-7500

Vyvanse® Shire Pharmaceuticals excessive daytime sleepiness Phase II lisdexamfetamine Wayne, PA (see also depression, eating, www.shire.com schizophrenia)

zaleplon Intec Pharma insomnia Phase II controlled-release Jerusalem, Israel www.intecpharma.com

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

Zolpimist™ NovaDel Pharma sleep maintenance insomnia Phase I zolpidem buccal spray Bridgewater, NJ (908) 203-4640

Other

Product Name Sponsor Indication Development Status

Abilify® Bristol-Myers Squibb Gilles de la Tourette’s syndrome Phase III aripiprazole Princeton, NJ (800) 332-2056 (once-weekly tablet) Otsuka America Pharmaceutical (800) 562-3974 Rockville, MD

ADX71149 Addex Pharmaceuticals psychiatric disorders Phase I (mGluR2-positive allosteric Geneva, Switzerland (800) 817-5286 modulator) Johnson & Johnson Pharmaceutical Research & Development Raritan, NJ

BI-409306 Boehringer Ingelheim psychiatric disorders Phase I Pharmaceuticals (800) 243-0127 Ridgefield, CT

BMS-933043 Bristol-Myers Squibb psychiatric disorders Phase I (a-7 ) Princeton, NJ (800) 332-2056

18 Medicines in Development Mental Illnesses 2012 Medicines in Development for Mental Illnesses

Other

Product Name Sponsor Indication Development Status

CORT-108297 Corcept Therapeutics weight gain associated with Phase I/II Menlo Park, CA (650) 327-3270 ------psychiatric disorders Phase I (650) 327-3270

ecopipam Psyadon Pharmaceuticals Gilles de la Tourette’s syndrome Phase I/II (PSYRX101) Germantown, MD (see also addictive) (301) 919-2020 ------psychiatric disorders Phase I (301) 919-2020

Alexza Pharmaceuticals acute agitation associated with application submitted inhalation Mountain View, CA bipolar disorder or schizophrenia (650) 944-7000 (AZ-004)

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

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

The content of this report has been obtained through industry sources and the Adis “R&D Insight” database based on the latest information. Report current as of June 14, 2012. The information may not be comprehensive. For more specific information about a particular product, contact the individual company directly or go to www.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 © 2012 by the Pharmaceutical Research and Manufacturers of America. Permission to reprint is awarded if proper credit is given.

Pharmaceutical Research and Manufacturers of America • 950 F Street, NW, Washington, DC 20004

Medicines in Development Mental Illnesses 2012 19 Glossary

anxiety disorders—A group of mental ill- called autism disorder, to a much milder form a drug company is encouraged throughout the nesses in which symptoms of anxiety (from called Asperger syndrome. entire drug development and review process. mild unease to intense fear) are the main The frequency of communication assures that feature. Generalized anxiety disorder is diag- depression—A mental illness characterized questions and issues are resolved quickly, of- nosed if a patient experiences unjustifiable or by an intense feeling of sadness, which is of ten leading to earlier drug approval and access excessive anxiety and worry, e.g., worry about greater proportion and duration than expected by patients. something bad happening to a loved one (who by objective reason (i.e., recent loss or other is not in danger), or worry about money (for sad event). Major depressive disorder (or fragile X syndrome (FXS)—The most com- no reason) on two or more life circumstances major depression) includes an episode of mon inherited mental illness. Impairment can and for six months or longer. A diagnosis of depression defined as a persistent (for at least range from learning disabilities to more severe cognitive or intellectual disabilities. FXS is panic disorder requires occurrence of “panic 2 weeks) mood disturbance, plus at least four the most common known cause of autism or attacks”—sudden feelings of apprehension or of the following symptoms: sleep disturbance, “autistic-like” behaviors, but up to 85 percent of fear accompanied by physical symptoms such changes in psychomotor activity, loss of ability autism cases are of unknown cause. Symp- as shortness of breath—in association with a to experience pleasure and interest, fatigue, toms can also include characteristic physical few concomitant psychological symptoms, that feelings of worthlessness or guilt, difficulty in and behavioral features and delays in speech the attacks are severe enough and happen concentrating, and/or suicidal thoughts. Major and language development. often enough to be disruptive or distressing depression is associated with impairment in social functioning. If criteria for major depres- to the individual, and that at least one attack NCE—New chemical entity. occurs spontaneously (i.e., in the absence of a sion have been met but in addition an episode fearful stimulus). of mania has ever occurred, then the diagnosis obsessive-compulsive disorder—A psychi- becomes bipolar disorder (or manic-depres- atric condition characterized by recurrent and application submitted—An application for sive illness). The essential feature of mania persistent thoughts that are intense, frighten- marketing has been submitted to the U.S. Food is a distinct period when the mood is either ing, absurd or otherwise alien, accompanied and Drug Administration (FDA). The application elevated, expansive, or irritable, with associ- by ritualized, repetitive behavior that is usually can either be an NDA (new drug application) or ated symptoms including hyperactivity, flight of irrational and bizarre. a BLA (biologic license application). ideas, inflated self-esteem, decreased need for sleep, distractibility and excessive involvement Orphan Drug—A drug to treat a disease that attention deficit/hyperactivity disorder in activities that often are flamboyant, bizarre has a patient population of 200,000 or less, or (ADHD)—ADHD is a complex neurological im- or disorganized. a disease that has a patient population of more pairment that results in an overactive behavior than 200,000 and a development cost that pattern and a difficulty concentrating. While it Fast Track—Fast Track is a process designed will not be recovered from sales in the United primarily affects children, a growing number of to facilitate the development and expedite the States. Orphan Drug status is assigned by the adults are being diagnosed with the disorder. review of drugs to treat serious diseases and U.S. Food and Drug Administration. Boys are affected about three times as often as fill an unmet medical need. The status is as- girls. Children with ADHD are fidgety, impul- signed by the U.S. Food and Drug Administra- Phase 0—First-in-human trials conducted sive, reckless, irritable, emotionally immature tion. The purpose is to get important new drugs in accordance with FDA’s 2006 guidance on and sometimes aggressive. Because their to the patient earlier. Fast Track addresses a exploratory Investigational New Drug (IND) attention span is short, they do not conform to broad range of serious diseases. Generally, studies designed to speed up development orderly routine. ADHD often leads to anti-social determining factors include whether the drug of promising drugs by establishing very early acts and difficulty learning, although IQ is nor- will have an impact on such factors as survival, whether the tested compound behaves in hu- mal. No definite cause has been established, day-to-day functioning, or the likelihood that man subjects as was anticipated from preclini- but some researchers now believe genetics the disease, if left untreated, will progress cal studies. plays a role. from a less severe condition to a more serious Phase I—Safety testing and pharmacologi- one. Filling an unmet medical need is defined cal profiling of new drugs in small numbers of autism—A complex developmental disorder as providing a therapy where none exists or humans. that causes severe and pervasive impairment providing a therapy which may be potentially in thinking, feeling, language, and the ability to superior to existing therapy. Once a drug Phase II—Effectiveness testing and identi- relate to others. It is usually first diagnosed in receives Fast Track designation, early and fication of side effect profile of new drugs in early childhood and ranges from a severe form, frequent communication between the FDA and humans.

20 Medicines in Development Mental Illnesses 2012 Glossary

Phase III—Extensive clinical trials in humans before menses and remit a few days after the schizophrenia—The most common form of to verify effectiveness and monitor adverse start. PMS looks more at physical symptoms psychotic illness characterized by disturbances reactions of new drugs. such as bloating, breast tenderness and appe- in thinking, emotional reaction and behavior. It tite change. PMDD has as part of its definition is disabling and has a prolonged course that premenstrual syndrome (PMS)/premenstru- symptoms such as depressed mood, anxiety almost always results in chronic ill health and al dysphoric disorder (PMDD)—PMS refers or tension, irritability, concentration difficul- some degree of personality change. to the variation of physical and mood symp- ties, overeating or food cravings, and feeling toms that appear during the last one or two overwhelmed. weeks of the menstrual cycle and disappear by the end of a full flow of menses. Psychiatrists psychosis—Severe mental disorders in and other mental health workers tend to use which the individual loses contact with reality. the term PMDD to describe a specific set of Symptoms include delusions, hallucinations, mood symptoms that interfere with social or thought disorders, loss of emotion, mania and role functioning that are also present the week depression.

Medicines in Development Mental Illnesses 2012 21 Selected Facts about Mental Illnesses and Addictive Disorders

Overview

• An estimated 450 million people worldwide have a mental disorder. At any given time, approximately 10 percent of adults are experiencing a current mental disorder, and 25 percent will develop one at some point during their lifetimes.1

• Mental disorders account for 13 percent of the global burden of disease, and that figure will rise to nearly 15 percent by 2030. Depression alone is likely to be the second highest contributor to the global burden of disease by that date.1

• Suicide is among the top 20 leading causes of death globally for all ages. Mental disorders are associated with more than 90 percent of the 1 million suicides that occur annually. On average, almost 3,000 people commit suicide daily.1

• Mental disorders are common both in the United States and internationally. An estimated 26.2 percent of Americans ages 18 and older—about 1 in 4 adults—suffer from a diagnosable mental disorder in a given year. That’s 57.7 million people with a diagnosable mental disorder in a year. In addition, an estimated 4 million American children and adolescents suffer from a severe mental illness. While mental disorders are widespread in the population, the main burden of illness is concentrated in a much smaller proportion of the population; about 6 percent, or 1 in 17 Ameri- cans, suffer from a serious mental illness.2

• Mental disorders are the leading cause of disability in the United States and Canada for people ages 15 to 44. Many people suffer from more than one mental disorder at a given time. Nearly half (45 percent) of those with any mental disorder meet criteria for two or more disorders.2

• Suicide is a major, preventable public health problem. In 2007, it was the tenth leading cause of death in the United States, accounting for 34,598 deaths. The overall rate was 11.3 suicide deaths per 100,000 people. Risk factors for suicide include depression and other mental dis- orders or a substance-abuse disorder (often in combination with other mental disorders). More than 90 percent of people who die by suicide have those risk factors.2

• An estimated 11 attempted suicides occur for every suicide death. Men are more likely to die by suicide than women, but women are more likely to attempt suicide. Men are more likely to use deadlier methods, such as firearms or suffocation, while women are more likely to attempt suicide by poisoning.2

• Year after year, suicide remains one of the top three leading causes of death for young people ages 15 to 24. Older adults are at risk for suicide, too. White males age 85 and older consistently have the highest suicide rate than any other age and ethnic group.2

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

Addictive Disorders

• Slightly more than half (51.8 percent) of Americans ages 12 or older reported being current drinkers of alcohol in the 2010 National Survey on Drug Use and Health (NSDUH), an annual survey sponsored by the Substance Abuse and Mental Health Services Administration (SAMHSA). That translates to an estimated 131.3 million people. In 2010, nearly one-quarter (23.1 percent) of people ages 12 or older—about 58.6 million people--participated in binge drinking. Also in 2010, heavy drinking was reported by 6.7 percent of that population, or 16.9 million people.3

• In 2010, an estimated 22.6 million Americans ages 12 or older were current (past month) illicit drug users. That estimate represents 8.9 percent of the population ages 12 or older. Illicit drugs include marijuana/hashish, cocaine (including crack), heroin, hallucinogens, inhalants, or prescrip- tion-type psychotherapeutics used non-medically.3

• In 2010, an estimated 22.1 million people (8.7 percent of the population ages 12 or older) were classified with substance dependence or abuse in the past year. Of those, 2.9 million were classified with dependence or abuse of both alcohol and illicit drugs, 4.2 million had dependence or abuse of illicit drugs but not alcohol, and 15.0 million had dependence or abuse of alcohol but not illicit drugs.3

22 Medicines in Development Mental Illnesses 2012 Selected Facts about Mental Illnesses and Addictive Disorders

Addictive Disorders (continued)

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

• Illicit drug use in the United States is estimated to have cost the U.S. economy more than $193 billion in 2007, according to a study produced by the National Drug Intelligence Center (NDIC).5

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 percent of people in that age group in a given year.

• Generalized anxiety disorder (GAD) affects about 6.8 million American adults, including twice as many women as men. The disorder develops gradually and can begin at any point in life, although the years of highest risk are between childhood and middle age.

• Obsessive-compulsive disorder (OCD) affects about 2.2 million American adults, striking men and women in roughly equal numbers. OCD usually appears in childhood, adolescence, or early adulthood. One-third of adults with OCD develop symptoms as children, and research indi- cates that OCD might run in families.

• Panic disorder affects about 6 million American adults and is twice as common in women as men. Panic attacks often begin in late adolescence or early adulthood.

• Post-traumatic stress disorder (PTSD) affects about 7.7 million American adults, but it can occur at any age, including childhood. Women are more likely to develop PTSD than men, and there is some evidence that susceptibility to the disorder may run in families.

• Social phobia affects about 15 million American adults. Women and men are equally likely to develop the disorder, which usually begins in childhood or early adolescence.

Attention-Deficit/Hyperactivity Disorder (ADHD)6

• 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 more likely than girls to experience the disorder. Although most children with ADHD have normal or above-normal intelligence, 40 percent to 60 percent have serious learning difficulties.

• Children and adolescents with ADHD are more likely than children without the disorder to suffer from other mental disorders. About one-half of all young people with ADHD have oppositional defiant disorder; about one-quarter have an anxiety disorder; as many as one-third have depression; and one-fifth have bipolar disorder. Adolescents with untreated ADHD are at risk for substance abuse disorders.

Autism7

• Autism spectrum disorders (ASDs) are a group of developmental disabilities that can cause significant social, communication and behavioral challenges. The U.S. Centers for Disease Control and Prevention (CDC) estimates that about 1 in 88 children has been identified with an ASD. ASDs are reported in all racial, ethnic, and socioeconomic groups. They are almost five times more common among boys (1 in 54) than among girls (1 in 252).

Medicines in Development Mental Illnesses 2012 23 Selected Facts about Mental Illnesses and Addictive Disorders

Depression

• Mood disorders, which include major depressive disorder, dysthymic disorder, and bipolar disorder, affect nearly 21 million adults, or about 9.5 percent of the U.S. population age 18 and older in a given year.2

• Bipolar disorder often develops in a person’s late teens or early adult years. At least half of all cases start before age 25. Some people have their first symptoms during childhood, while others may develop symptoms late in life.2

• Depression can strike anyone regardless of age, ethnic background, socioeconomic status, or gender; however, studies have found that depression is about twice as common in women as in men. In any given one year period, depressive illnesses affect 12 percent of women (more than 12 million women) and nearly 7 percent of men (more than 6 million men).2

• Dysthymic disorder, which is characterized by chronic low-level depression, affects approximately 1.5 percent of the adult population in the United States.2

• Both dysthymic disorder and major depressive disorder together have affected approximately 11.2 percent of 13- to 18-year olds in the United States at some point during their lives. Girls are more likely than boys to experience depressive disorders. Additionally, 3.3 percent of 13- to 18-year olds have experienced a seriously debilitating depressive disorder.2

• Major depressive disorder is, by itself, the leading cause of disability among Americans ages 15-44. It affects 6.7 percent of the U.S. adult population.2

• Bipolar disorder costs twice as much in lost productivity as major depressive disorder, according to a study by the National Institute of Mental Health (NIMH). Each U.S. worker with bipolar disorder averaged 65.5 lost workdays in a year, compared to 27.2 for major depression. Even though major depression is more than six times as prevalent, bipolar disorder costs the U.S. workplace nearly half as much—a disproportionate- ly high $14.1 billion in lost salary-equivalent productivity compared to $36.6 billion for major depression annually. Researchers traced the higher toll mostly to bipolar disorder’s more severe depressive episodes rather than to its agitated manic periods.2

• Clinical depression has become one of America’s most costly illnesses. Left untreated, depression is as costly as heart disease or AIDS to the U.S. economy, costing over $51 billion in absenteeism from work and lost productivity and $26 billion in direct treatment costs. Depression tends to affect people in their prime working years and may last a lifetime if untreated. More than 80 percent of people with clinical depression can be successfully treated.6

eating Disorders2

• Eating disorders are more common in women. The lifetime rate for anorexia nervosa among women is estimated at 0.9 percent compared to 0.3 percent among men. The lifetime rate among women for bulimia nervosa is 0.5 percent compared to 0.1 percent among men. And the life- time rate among women for binge-eating disorder is 3.5 percent compared to 2 percent among men. Some researchers believe those numbers are under-estimates, in part because hospitalization rates for eating disorders continue to rise, increasing 18 percent between 1999 and 2006.

premenstrual Disorders2

• 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 a family history of major depression, or women with a past medi- cal history of either postpartum depression or an affective . Up to 60 percent of women with severe PMS have an underlying psychiatric disorder.

• Premenstrual dysphoric disorder (PMDD), a very severe form of PMS, affects between 3 percent to 8 percent of menstruating women.

24 Medicines in Development Mental Illnesses 2012 Selected Facts about Mental Illnesses and Addictive Disorders

Schizophrenia

• Schizophrenia affects about 1 percent of the American population and affects men and women equally. It occurs at similar rates in all ethnic groups around the world. Symptoms such as hallucinations and delusions usually start between the ages of 16 and 30. Men tend to experience symptoms a little earlier than women. Most of the time, people do not get schizophrenia after age 45.2

• The appearance of schizophrenic symptoms before age 12 is rare—less than one-sixtieth as common as the adult-onset type. Neurodevel- opmental 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 psychotic symptoms (hallucinations, delusions, and disordered thinking) appear, usually at age 7 or later. In the first years of life, about 30 percent of those children have transient symptoms of pervasive developmental disorder, such as rock- ing, posturing, and arm flapping.6

• Schizoaffective disorder, a combination of symptoms of schizophrenia and a mood disorder, may range from 2 to 5 in 1,000 people (i.e., 0.2 percent to 0.5 percent) and may account for one-fourth or even one-third of all people with schizophrenia.8

Sources:

1. World Health Organization, www.who.int 2. National Institute of Mental Health, www.nimh.nih.gov 3. Substance Abuse and Mental Health Services Administration, www.samhsa.gov 4. Community Safety Services, www.be-safe.org 5. U.S. Department of Justice, www.usdoj.gov 6. Mental Health America, www.mentalhealthamerica.net 7. U.S. Centers for Disease Control and Prevention, www.cdc.gov 8. National Alliance on Mental Illness, www.nami.org

Medicines in Development Mental Illnesses 2012 25 The Drug Discovery, Development and Approval Process

Developing a new medicine takes an average of 10-15 years; For every 5,000-10,000 compounds in the pipeline, only 1 is approved.

The Drug Development and Approval Process The U.S. system of new drug approvals is Administration (FDA) to begin to test the drug hospitals. Physicians monitor patients closely to perhaps the most rigorous in the world. in people. The IND shows results of previous confirm efficacy and identify adverse events. experiments; how, where and by whom the It takes 10-15 years, on average, for an New Drug Application (NDA)/Biologic new studies will be conducted; the chemical experimental drug to travel from lab to U.S. License Application (BLA). Following the structure of the compound; how it is thought patients, according to the Tufts Center for the completion of all three phases of clinical trials, to work in the body; any toxic effects found in Study of Drug Development. Only five in 5,000 a company analyzes all of the data and files an the animal studies; and how the compound compounds that enter preclinical testing make NDA or BLA with FDA if the data successfully is manufactured. All clinical trials must be it to human testing. And only one of those five demonstrate both safety and effectiveness. reviewed and approved by the Institutional is approved for sale. The applications contain all of the scientific Review Board (IRB) where the trials will be information that the company has gathered. On average, it costs a company $1.2 billion, conducted. Progress reports on clinical trials Applications typically run 100,000 pages or including the cost of failures, to get one new must be submitted at least annually to FDA and more. medicine from the laboratory to U.S. patients, the IRB. according to a 2007 study by the Tufts Center Approval. Once FDA approves an NDA or Clinical Trials, Phase I. These tests usually for the Study of Drug Development. BLA, the new medicine becomes available involve about 20 to 100 healthy volunteers. The for physicians to prescribe. A company must Once a new compound has been identified in tests study a drug’s safety profile, including the continue to submit periodic reports to FDA, the laboratory, medicines are usually devel- safe dosage range. The studies also determine including any cases of adverse reactions and oped as follows: how a drug is absorbed, distributed, metabo- appropriate quality-control records. For some lized, and excreted as well as the duration of Preclinical Testing. A pharmaceutical com- medicines, FDA requires additional trials its action. pany conducts laboratory and animal studies (Phase IV) to evaluate long-term effects. to show biological activity of the compound Clinical Trials, Phase II. In this phase, Discovering and developing safe and effective against the targeted disease, and the com- controlled trials of approximately 100 to 500 new medicines is a long, difficult, and expensive pound is evaluated for safety. volunteer patients (people with the disease) process. PhRMA member companies invested assess a drug’s effectiveness and determine Investigational New Drug Application (IND). an estimated $49.5 billion in research and the early side effect profile. After completing preclinical testing, a company development in 2011. files an IND with the U.S. Food and Drug Clinical Trials, Phase III. This phase usually involves 1,000 to 5,000 patients in clinics and