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2013 REPORT - E E DEMENTIA S S

CATARACTS AG AG E S E E I S S R OS U EA EA S S I I VER VER AIL D D

OPOR S ' T F ETES R TE R B NEY OS D EA IA H D KI CENT OF ALL CENT OF ALL C S or chronic kidney disease, where where disease, kidney or chronic LZHEIME or osteoporosis, a major health or osteoporosis, NI A ITI E 20 f with older age. grows prevalence 15 f 44 estimated an million for threat Americans. PER BENEFICIARIES RO PER BENEFICIARIES R

ARE RECIPIENT S ARE RECIPIENT • • for selected terms can be Definitions the sponsor A link to on page 36. found de- more site provides web company’s behind on the science information tailed potential product. each of medicines, value on the information For an in-depth innovation look at current benefit breakthroughs medical and key ing older Americans, please see Medi- see please ing older Americans, Ameri- for Older cines in Development . cans 2013—Overview hope in this reportThe medicines provide these from who suffer older Americans to and are diseases debilitating chronic independent more longer, live to seeking lives. and healthier H C TH EA R

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VE PRESCRIPTION DRUG CO VE PRESCRIPTION DRUG CO or depression, which affects an which affects or depression, or COPD, which affects Americans which affects or COPD,

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ORE i ORE RO A EMENTI D and ischemic heart heart8 for failure 2 for Alzheimer’s disease, which disease, Alzheimer’s 2 for 2 for rheumatoid arthritis rheumatoid 2 for and osteo- t  at a higher rate. age 65 over 34 f million Americans age 6.5 estimated and older. 65 million Americans age 65 and older. million Americans age 65 9 arthritis, 12.4 million which affects and older. 65 age people, 8 4 dis- cardiovascular of many two disease, million Americans, 80.7 affecting eases 60 and older. age half of which are 40 f 142 f by afflict 8 million people nearly could is or prevention 2030 unless a cure found. EPRE H eading Chronic Diseases Diseases Chronic eading

THAN M THAN COPD HA C D M eu HA • • • • iopharmaceutical research companies companies research Biopharmaceutical and medicines 465 developing are vac diseases chronic the 10 leading cines for This beneficiaries.* Medicare affecting in report and vaccines lists medicines by human clinical trials or under review and Drug Administration Food the U.S. (FDA). include: in development The medicines • • More Than 400 Medicines in Development forThan 400 in Development Medicines More Older Americans Affecting Diseases Chronic Leading *Centers for Medicare and Medicaid Services (CMS), Chronic Condition Data Warehouse (CCW) Medicare 5% Sample, 2009 5% Sample, Medicare (CCW) Warehouse Data Condition Chronic Services and Medicaid (CMS), Medicare for *Centers mac r t a en ph o m p 48

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Medicines in Development in Development Medicines Chronic Leading For Affecting Diseases

presen Older Americans Older The Medicare Me Alzheimer Medicines in Development for Older Americans

Alzheimer’s Disease and Dementia

Product Name Sponsor Indication Development Phase*

18F-florbetaben Piramal Healthcare Alzheimer’s disease (diagnosis) Phase III (BAY 94-9172) Mumbai, India piramalenterprises.com

AAB-003/PF-05236812 Janssen Alzheimer Immunotherapy Alzheimer’s disease Phase I (amyloid beta protein inhibitor) South San Francisco, CA janimm.com pfizer.com New York, NY

ABT-126 AbbVie mild to moderate Alzheimer’s Phase II (α7-NNR antagonist) North Chicago, IL disease .com

ABT-288 AbbVie Alzheimer’s disease Phase II completed (neurotransmitter receptor North Chicago, IL abbvie.com modulator)

ABT-384 AbbVie Alzheimer’s disease Phase II completed North Chicago, IL abbvie.com

ABT-957 AbbVie Alzheimer’s disease Phase I (calpain inhibitor) North Chicago, IL abbvie.com

AC-1204 Accera Alzheimer’s disease Phase II/III Broomfield, CO accerapharma.com

ACC-001/PF-05236806 Janssen Alzheimer Immunotherapy Alzheimer’s disease Phase II South San Francisco, CA janimm.com Pfizer pfizer.com New York, NY

AD02 vaccine Affiris treatment of Alzheimer’s disease Phase II Vienna, Austria gsk.com GlaxoSmithKline Rsch. Triangle Park, NC

AD03 vaccine Affiris treatment of Alzheimer’s disease Phase I Vienna, Austria gsk.com GlaxoSmithKline Rsch. Triangle Park, NC

AD4833/TOMM40 Takeda Pharmaceuticals International Alzheimer’s disease prevention Phase I Deerfield, IL takeda.com

APH-0703 Aphios Alzheimer’s disease Phase I/II (protein kinase C activator) Woburn, MA aphios.com

*For more information about a specific medicine or company in the report, please click on the provided link.

2 Medicines in Development Older Americans 2013 Medicines in Development for Older Americans

Alzheimer’s Disease and Dementia

Product Name Sponsor Indication Development Phase

ARC029 Archer Pharmaceuticals Alzheimer’s disease Phase I (nilvadipine) Sarasota, FL archerpharma.com

ARC031 Archer Pharmaceuticals Alzheimer’s disease Phase I (soluble amyloid reducing/clearing Sarasota, FL archerpharma.com agent)

ARC031 SR Archer Pharmaceuticals Alzheimer’s disease Phase I (soluble amyloid reducing/clearing Sarasota, FL archerpharma.com agent)

AVN 101 Avineuro Pharmaceuticals Alzheimer’s disease Phase II ( 5-HT6 receptor San Diego, CA avineuro.com antagonist)

AVN 322 Avineuro Pharmaceuticals Alzheimer’s disease Phase I (serotonin 5-HT6 receptor San Diego, CA avineuro.com antagonist)

AVN 397 Avineuro Pharmaceuticals Alzheimer’s disease Phase II San Diego, CA avineuro.com

AZD1446 AstraZeneca Alzheimer’s disease Phase I (alpha4/beta2 neuronal nicotinic Wilmington, DE .com receptor agonist) Targacept Winston-Salem, NC

AZD2184 Navidea Biopharmaceuticals Alzheimer’s disease (diagnosis) Phase I (PET imaging) Dublin, OH navidea.com

AZD2995 Navidea Biopharmaceuticals Alzheimer’s disease (diagnosis) Phase I (PET imaging) Dublin, OH navidea.com

AZD3293 AstraZeneca Alzheimer’s disease Phase I (beta secretase) Wilmington, DE astrazeneca.com Astex Pharmaceuticals Dublin, CA

AZD5213 AstraZeneca Alzheimer’s disease Phase II (histamine-3 ) Wilmington, DE astrazeneca.com

ß-secretase inhibitor Eli Lilly Alzheimer’s disease Phase II (LY2886721) Indianapolis, IN (slow disease progression) lilly.com

BAN2401 Eisai early-stage Alzheimer’s disease Phase II Woodcliff Lake, NJ eisai.com

BAY 85-8101 Piramal Healthcare Alzheimer’s disease (diagnosis) Phase I completed (18F-labeled radiopharmaceutical) Mumbai, India piramalenterprises.com

Medicines in Development Older Americans 2013 3 Medicines in Development for Older Americans

Alzheimer’s Disease and Dementia

Product Name Sponsor Indication Development Phase

BIIB037 Idec Alzheimer’s disease Phase I (anti-ß-amyloid antibody) Weston, MA biogenidec.com

bisnorcymserine QR Pharma Alzheimer’s disease Phase I (BNC) Berwyn, PA qrpharma.com

BMS-241027 Bristol-Myers Squibb Alzheimer’s disease, Phase I (microtubule stabilizer) Princeton, NJ senile dementia bms.com

CAD106 Novartis Pharmaceuticals Alzheimer’s disease Phase II East Hanover, NJ novartis.com

CERE-110 Ceregene Alzheimer’s disease Phase II (AAV-NGF gene therapy) San Diego, CA ceregene.com

crenezumab Alzheimer’s disease Phase II (anti-Abeta) South San Francisco, CA gene.com

davunetide intranasal Allon Therapeutics Alzheimer’s disease Phase II Vancouver, Canada allontherapeutics.com

donepezil/memantine Adamas Pharmaceuticals moderate to severe Alzheimer’s Phase II extended-release fixed-dose Emeryville, CA disease adamaspharma.com combination Forest Laboratories frx.com New York, NY

DSP-8658 Sunovion Pharmaceuticals Alzheimer’s disease Phase I (PPAR alpha/gamma agonist) Marlborough, MA (see also diabetes) sunovion.com

E2609 Eisai Alzheimer’s disease Phase I completed Woodcliff Lake, NJ eisai.com

ELND005 Elan Alzheimer’s disease Phase II (amyloid beta-protein inhibitor) Dublin, Ireland elan.com

EVP-0962 EnVivo Pharmaceuticals Alzheimer’s disease Phase II (gamma secretase modulation) Watertown, MA envivopharma.com

EVP-6124 EnVivo Pharmaceuticals Alzheimer’s disease Phase II (nicotine A7 agonist) Watertown, MA envivopharma.com

Exebryl-1® ProteoTech Alzheimer’s disease Phase I Alzheimer’s disease therapeutic Kirkland, WA proteotech.com

F-18 T808 Siemens Molecular Imaging Alzheimer’s disease (diagnosis) Phase 0 (PET imaging agent) Malvern, PA medical.siemens.com

F18-flutemetamol GE Healthcare Alzheimer’s disease (diagnosis) application submitted (PET imaging agent) Waukesha, WI gehealthcare.com

4 Medicines in Development Older Americans 2013 Medicines in Development for Older Americans

Alzheimer’s Disease and Dementia

Product Name Sponsor Indication Development Phase

Gammagard® early-stage Alzheimer’s disease, Phase III immune globulin Deerfield, IL mid-stage Alzheimer’s disease baxter.com

gamma secretase modulator, Bristol-Myers Squibb Alzheimer’s disease, in clinical trials A-beta modulator Princeton, NJ senile dementia bms.com

gantenerumab Roche early-stage Alzheimer’s disease Phase II/III (amyloid beta-protein inhibitor) Nutley, NJ roche.com

GSK742457 GlaxoSmithKline dementia Phase II (5-HT6 receptor antagonist) Rsch. Triangle Park, NC gsk.com

GSK2647544 GlaxoSmithKline Alzheimer’s disease Phase I (Lp-PLA2 inhibitor) Rsch. Triangle Park, NC gsk.com

HPP 854 High Point Pharmaceuticals Alzheimer’s disease Phase I (BACE1 protein inhibitor) High Point, NC highpointpharma.com

human immunoglobulin Grifols Alzheimer’s disease Phase III (intravenous) Los Angeles, CA grifols.com

KU-046 Kareus Therapeutics Alzheimer’s disease Phase I (amyloid-ß protein modulator) La Chaux-de-Fonds, Switzerland kareustherapeutics.com

Lu AE58054 Lundbeck USA Alzheimer’s disease Phase II Deerfield, IL lundbeck.com

masitinib AB Science USA Alzheimer’s disease Phase III completed (AB-1010) Short Hills, NJ ab-science.com

MCD-386 Mithridion Alzheimer’s disease Phase I Madison, WI mithridion.com

MK-3328 Merck Alzheimer’s disease (diagnosis) Phase I completed (PET imaging) Whitehouse Station, NJ merck.com

MK-8931 Merck Alzheimer’s disease Phase II/III (BACE1 protein inhibitor) Whitehouse Station, NJ merck.com

MSDC-0160 Metabolic Solutions Development Alzheimer’s disease Phase II Kalamazoo, MI (see also diabetes) msdrx.com

NAV4694 Navidea Biopharmaceuticals Alzheimer’s disease (diagnosis) Phase III (fluorine-18 labeled PET Dublin, OH navidea.com imaging agent)

NIC5-15 Humanetics Alzheimer’s disease Phase II Minneapolis, MN humaneticscorp.com

Medicines in Development Older Americans 2013 5 Medicines in Development for Older Americans

Alzheimer’s Disease and Dementia

Product Name Sponsor Indication Development Phase

PF-05212377 Pfizer Alzheimer’s disease Phase II (SAM-760) New York, NY pfizer.com

Posiphen® QR Pharma Alzheimer’s disease, Phase II R-phenserine Berwyn, PA mild cognitive impairment qrpharma.com

PRX-3140 Nanotherapeutics Alzheimer’s disease Phase II (serotonin 4 receptor agonist) Alachua, FL nanotherapeutics.com

RG1577 Roche Alzheimer’s disease Phase II (MAO-B inhibitor) Nutley, NJ roche.com

RG7129 Roche Alzheimer’s disease Phase I (BACE-1 inhibitor) Nutley, NJ roche.com

rilapladib GlaxoSmithKline Alzheimer’s disease Phase II (Lp-PLA2 inhibitor) Rsch. Triangle Park, NC gsk.com

RVX-208 Resverlogix Alzheimer’s disease Phase I Calgary, Canada resverlogix.com

SAR110894 Sanofi US Alzheimer’s disease Phase II (H3 antagonist) Bridgewater, NJ sanofi.com

SAR228810 Sanofi US Alzheimer’s disease Phase I (anti-protofibrillar AB mAb) Bridgewater, NJ sanofi.com

sGC-1061 sGC Pharma Alzheimer’s disease Phase I Wellesley, MA sgcpharma.com

solanezumab Eli Lilly Alzheimer’s disease Phase III (LY2062430) Indianapolis, IN lilly.com

ST101 Sonexa Therapeutics Alzheimer’s disease Phase II San Diego, CA sonexa.com

T-817MA Toyota Chemical mild to moderate Alzheimer’s Phase II completed (amyloid beta-protein inhibitor) Tokyo, Japan disease toyama-chemical.co.jp

TAK-357 Takeda Pharmaceuticals International Alzheimer’s disease Phase I (oral cognitive enhancer) Deerfield, IL takeda.com

TC-1734 Targacept Alzheimer’s disease Phase II (alpha4/beta2 neuronal nicotinic Winston-Salem, NC targacept.com receptor agonist)

TC-5619 Targacept Alzheimer’s disease Phase I (alpha7 nAChR) Winston-Salem, NC targacept.com

6 Medicines in Development Older Americans 2013 Medicines in Development for Older Americans

Alzheimer’s Disease and Dementia

Product Name Sponsor Indication Development Phase

TRx-0237 TauRx Therapeutics Alzheimer’s disease, dementia Phase III (LMTX) Singapore, Republic of Singapore taurx.com

TTP4000 TransTech Pharma Alzheimer’s disease Phase I High Point, NC ttpharma.com

TTP488 TransTech Pharma Alzheimer’s disease Phase II High Point, NC ttpharma.com

UB-311 United Biomedical mild to moderate Alzheimer’s Phase I (amyloid beta protein inhibitor Hauppauge, NY disease unitedbiomedical.com vaccine)

V950 Merck Alzheimer’s disease Phase I Whitehouse Station, NJ merck.com

velusetrag Theravance Alzheimer’s disease Phase I completed (TD-5108) South San Francisco, CA theravance.com

VI-1121 VIVUS Alzheimer’s disease Phase II Mountain View, CA vivus.com

XEL-001HP Xel Pharmaceuticals Alzheimer’s disease Phase I (transdermal patch) Draper, UT xelpharmaceuticals.com

Arthritis

Product Name Sponsor Indication Development Phase

2ME2 EntreMed rheumatoid arthritis Phase I Rockville, MD entremed.com

ABT-122 AbbVie rheumatoid arthritis Phase I (anti-TNF/anti-IL-17 mAb) North Chicago, IL abbvie.com

ABT-413 AbbVie rheumatoid arthritis Phase I (sphingosine 1 phosphate receptor North Chicago, IL abbvie.com modulator)

ABT-494 AbbVie rheumatoid arthritis Phase I (JAK-1, 2, 3 inhibitor) North Chicago, IL abbvie.com

Medicines in Development Older Americans 2013 7 Medicines in Development for Older Americans

Arthritis

Product Name Sponsor Indication Development Phase

ABT-652 AbbVie osteoarthritis of the knee Phase II (histamine H3 receptor modulator) North Chicago, IL abbvie.com

ABT-981 AbbVie osteoarthritis Phase I (anti-IL-1 α and β mAb) North Chicago, IL abbvie.com

Actemra® Genentech rheumatoid arthritis application submitted South San Francisco, CA (first-line therapy) gene.com Roche roche.com Nutley, NJ ------rheumatoid arthritis Phase III (early-stage disease) gene.com roche.com

biosimilar Boehringer Ingelheim Pharmaceuticals rheumatoid arthritis Phase I completed Ridgefield, CT boehringer-ingelheim.com

AF-219 Afferent Pharmaceuticals osteoarthritis Phase II (purinoceptor P2X3 antagonist) San Mateo, CA afferentpharma.com

AIN457 Novartis Pharmaceuticals rheumatoid arthritis Phase III () East Hanover, NJ novartis.com

ARG301 arGentis Pharmaceuticals rheumatoid arthritis Phase I Memphis, TN argentisrx.com

ARRY-797 Array BioPharma moderate to severe pain associated Phase II (p38 MAPK inhibitor) Boulder, CO with osteoarthritis of the knee arraybiopharma.com

ASP015K Astellas Pharma US rheumatoid arthritis Phase II (JAK-3 inhibitor) Northbrook, IL astellas.com

ASP2408 Perseid Therapeutics rheumatoid arthritis Phase I (CTLA4-Ig fusion protein) Redwood City, CA perseidtherapeutics.com

baricitinib Eli Lilly rheumatoid arthritis Phase III (LY3009104) Indianapolis, IN (see also chronic kidney) lilly.com

BI-695500 Boehringer Ingelheim Pharmaceuticals rheumatoid arthritis Phase III (rituximab biosimilar) Ridgefield, CT boehringer-ingelheim.com

BMS-817399 Bristol-Myers Squibb osteoarthritis, Phase II (CCR1 antagonist) Princeton, NJ rheumatoid arthritis bms.com

BT-061 AbbVie rheumatoid arthritis Phase II North Chicago, IL abbvie.com Biotest biotest.de Dreieich, Germany

8 Medicines in Development Older Americans 2013 Medicines in Development for Older Americans

Arthritis

Product Name Sponsor Indication Development Phase

buprenorphine transmucosal BioDelivery Sciences International chronic pain associated with Phase III Raleigh, NC osteoarthritis bdsi.com Endo Pharmaceuticals endo.com Malvern, PA

Novartis Pharmaceuticals gouty arthritis application submitted (ACZ885) East Hanover, NJ (see also diabetes) novartis.com ------osteoarthritis Phase II novartis.com

cebranopadol Forest Laboratories pain due to osteoarthritis of the Phase II New York, NY knee frx.com

CEP-33237 osteoarthritis pain Phase III (hydrocodone extended-release) Frazer, PA cephalon.com

CF101 Can-Fite BioPharma rheumatoid arthritis Phase II Waltham, MA canfite.com

Chondrogen™ Osiris Therapeutics osteoarthritis (prevention) Phase II mesenchymal stem cell therapy Columbia, MD osiris.com

Cimzia® UCB early rheumatoid arthritis Phase III Smyrna, GA ucb.com

Bristol-Myers Squibb osteoarthritis, Phase II (anti-IL6-mAb) Princeton, NJ rheumatoid arthritis bms.com

CNTO-1959 rheumatoid arthritis Phase II Horsham, PA janssenbiotech.com

COV-155 osteoarthritis Phase III Hazelwood, MO mallinckrodt.com

COV-795 Mallinckrodt osteoarthritis Phase III Hazelwood, MO mallinckrodt.com

CT-1578 Cell Therapeutics rheumatoid arthritis Phase I (JAK2/FLT-3/c-Fms inhibitor) Seattle, WA celltherapeutics.com

Dekavil Pfizer rheumatoid arthritis Phase I (F8-IL10) New York, NY pfizer.com Philogen philogen.com Sovicille, Italy

DMI-9523 Ampio Pharmaceuticals osteoarthritis Phase I Greenwood Village, CO ampiopharma.com

Medicines in Development Older Americans 2013 9 Medicines in Development for Older Americans

Arthritis

Product Name Sponsor Indication Development Phase

etoricoxib Merck rheumatoid arthritis Phase III Whitehouse Station, NJ merck.com

fostamatinib AstraZeneca rheumatoid arthritis Phase III (SYK inhibitor) Wilmington, DE astrazeneca.com Rigel Pharmaceuticals rigel.com South San Francisco, CA

FX005 Flexion Therapeutics osteoarthritis of the knee Phase I/II (p38 MAPK inhibitor) Woburn, MA flexiontherapeutics.com

FX006 Flexion Therapeutics osteoarthritis of the knee Phase II (triamcinolone acetonide Woburn, MA flexiontherapeutics.com intra-articular sustained-release)

XOMA osteoarthritis of the hand Phase II (XOMA 052) Berkeley, CA xoma.com

GLPG-0555 Galapagos osteoarthritis Phase I Mechelen, Belgium gsk.com GlaxoSmithKline Rsch. Triangle Park, NC

GLPG-0634 AbbVie rheumatoid arthritis Phase II North Chicago, IL abbvie.com Galapagos Mechelen, Belgium

GSK2941266 GlaxoSmithKline rheumatoid arthritis Phase II (CCR1 chemokine receptor Rsch. Triangle Park, NC gsk.com antagonist)

HE3286 Harbor Biosciences rheumatoid arthritis Phase II San Diego, CA (see also diabetes) harbortx.com

INCB39110 Incyte active rheumatoid arthritis Phase II (JAK1 inhibitor) Wilmington, DE incyte.com

IP045 Iroko Pharmaceuticals pain associated with osteoarthritis Phase I (meloxicam) Philadelphia, PA of the knee iroko.com

IP-880 Iroko Pharmaceuticals osteoarthritis pain Phase III (low dose diclofenac) Philadelphia, PA iroko.com

10 Medicines in Development Older Americans 2013 Medicines in Development for Older Americans

Arthritis

Product Name Sponsor Indication Development Phase

ISIS-CRPRx Isis Pharmaceuticals rheumatoid arthritis Phase II (C-reactive protein inhibitor) Carlsbad, CA isispharm.com

JNJ-38518168 Janssen Research & Development rheumatoid arthritis Phase II (histamine H4 receptor Raritan, NJ janssenrnd.com antagonist)

JNJ-39439335 Janssen Research & Development chronic pain associated with Phase I (mavatrep) Raritan, NJ osteoarthritis of the knee janssenrnd.com

JNJ-40346527 Janssen Research & Development rheumatoid arthritis Phase II (M-CSF receptor antagonist) Raritan, NJ janssenrnd.com

LT-NS001 Logical Therapeutics osteoarthritis Phase II/III (naproxen etemesil) Foxboro, MA logicaltx.com

LX2931 rheumatoid arthritis Phase II (S1P lyase inhibitor) The Woodlands, TX lexicon-genetics.com

AstraZeneca rheumatoid arthritis Phase II (anti-GM-CSFR mAb) Wilmington, DE astrazeneca.com MedImmune medimmune.com Gaithersburg, MD

MEDI-5117 AstraZeneca rheumatoid arthritis Phase I (anti-IL-6 mAb) Wilmington, DE astrazeneca.com MedImmune medimmune.com Gaithersburg, MD

mesenchymal stem cell therapy for Medipost osteoarthritis Phase I/II cartilage repair Seoul, South Korea medi-post.com

MK-8457 Merck rheumatoid arthritis Phase II Whitehouse Station, NJ merck.com

MK-8808 Merck rheumatoid arthritis Phase I Whitehouse Station, NJ merck.com

MORAb-022 Eisai rheumatoid arthritis Phase I (IgG1 mAb) Woodcliff Lake, NJ eisai.com

Takeda Pharmaceuticals International rheumatoid arthritis Phase I Deerfield, IL takeda.com

naproxcinod NicOx osteoarthritis application submitted Sophia Antipolis, France nicox.com

Medicines in Development Older Americans 2013 11 Medicines in Development for Older Americans

Arthritis

Product Name Sponsor Indication Development Phase

NN8209 Novo Nordisk rheumatoid arthritis Phase II (anti-C5aR-151) Princeton, NJ novonordisk.com

NN8210 Novo Nordisk rheumatoid arthritis Phase I (anti-C5aR-215) Princeton, NJ novonordisk.com

NN8226 Novo Nordisk rheumatoid arthritis Phase II (anti-IL20 mAb) Princeton, NJ novonordisk.com

NN8765 Novo Nordisk rheumatoid arthritis Phase I (anti-NKG2A) Princeton, NJ novonordisk.com

NN8828 Novo Nordisk rheumatoid arthritis Phase II (anti-IL21 mAb) Princeton, NJ novonordisk.com

UCB rheumatoid arthritis Phase II Smyrna, GA ucb.com

OLT-1177 Olatec Industries pain associated with osteoarthritis Phase II New York, NY of the knee

osteoarthritis medicine Eli Lilly osteoarthritis pain Phase I (NCE) Indianapolis, IN lilly.com

GlaxoSmithKline rheumatoid arthritis Phase I (CD3 mAb) Rsch. Triangle Park, NC gsk.com

Otrexup™ Antares Pharma rheumatoid arthritis application submitted methotrexate subcutaneous Ewing, NJ antarespharma.com auto-injection

ozoralizumab Ablynx rheumatoid arthritis Phase II Ghent, Belgium ablynx.com

PDA-001 Celgene Cellular Therapeutics rheumatoid arthritis Phase I/II (stem cell therapy) Summit, NJ celgene.com

Pennsaid® 2% Mallinckrodt osteoarthritis pain and stiffness application submitted diclofenac transdermal Hazelwood, MO mallinckrodt.com

PF-04171327 Pfizer rheumatoid arthritis Phase II (SGRM) New York, NY pfizer.com

PF-04236921 Pfizer rheumatoid arthritis Phase I (mAb) New York, NY pfizer.com

PF-05280586 Pfizer rheumatoid arthritis Phase I (rituximab biosimilar) New York, NY pfizer.com

12 Medicines in Development Older Americans 2013 Medicines in Development for Older Americans

Arthritis

Product Name Sponsor Indication Development Phase

PLX5622 Plexxikon rheumatoid arthritis Phase I (FGF-1 antagonist) Berkeley, CA plexxikon.com

Prolia® rheumatoid arthritis Phase II denosumab Thousand Oaks, CA amgen.com

PRTX-100 Protalex rheumatoid arthritis Phase I (staphylococcal protein A) Summit, NJ protalex.com

Ravax™ Immune Response BioPharma rheumatoid arthritis Phase III rheumatoid arthritis vaccine New York, NY immuneresponsebiopharma.com

RX-10001 Resolvyx Pharmaceuticals rheumatoid arthritis Phase I (synthetic resolvin) Cambridge, MA resolvyx.com

SAN-300 Santarus rhematoid arthritis Phase I (anti-VLA-1 antibody) San Diego, CA santarus.com

SAR113945 Sanofi US osteoarthritis Phase II (IKK-ß inhibitor) Bridgewater, NJ sanofi.com

SAR292833 Sanofi US osteoarthritis pain Phase II (TRVP3 antagonist) Bridgewater, NJ sanofi.com

Sanofi US rheumatoid arthritis Phase III (anti-IL-6R mAb) Bridgewater, NJ sanofi.com

Simponi® Janssen Biotech moderate to severe rheumatoid application submitted (intravenous) Horsham, PA arthritis janssenbiotech.com

GlaxoSmithKline rheumatoid arthritis Phase III (anti-IL-6 mAb) Rsch. Triangle Park, NC gsk.com Janssen Biotech janssenbiotech.com Horsham, PA

sprifermin EMD Serono osteoarthritis Phase I (FGF-18) Rockland, MA emdserono.com

TG-C TissueGene osteoarthritis Phase II (allogeneic cell therapy) Rockville, MD tissuegene.com

Stelara® Janssen Research & Development rheumatoid arthritis Phase II Raritan, NJ janssenrnd.com

Medicines in Development Older Americans 2013 13 Medicines in Development for Older Americans

Arthritis

Product Name Sponsor Indication Development Phase

V116517 pain associated with osteoarthritis Phase II Stamford, CT of the knee purduepharma.com

veltuzumab Immunomedics rheumatoid arthritis Phase II subcutaneous Morris Plains, NJ immunomedics.com Takeda Pharmaceuticals International takeda.com Deerfield, IL

VX30 Vaccinex rheumatoid arthritis Phase I Rochester, NY vaccinex.com

VX-509 rheumatoid arthritis Phase II Cambridge, MA vrtx.com

Cataracts

Product Name Sponsor Indication Development Phase

dexamethasone ophthalmic EyeGate Pharma cataracts Phase II (EGP-437) Waltham, MA eyegatepharma.com

Chronic Kidney Disease

Product Name Sponsor Indication Development Phase

Acthar® Gel Questcor Pharmaceuticals diabetic nephropathy Phase II corticotropin gel Anaheim Hills, CA questcor.com

AbbVie diabetic nephropathy Phase II North Chicago, IL abbvie.com

AZD1722 Ardelyx chronic kidney disease Phase I (NHE3 inhibitor) Fremont, CA ardelyx.com AstraZeneca astrazeneca.com Wilmington, DE

baricitinib Eli Lilly diabetic nephropathy Phase II (LY3009104) Indianapolis, IN (see also arthritis) lilly.com

14 Medicines in Development Older Americans 2013 Medicines in Development for Older Americans

Chronic Kidney Disease

Product Name Sponsor Indication Development Phase

BB-3 Angion chronic kidney fibrosis Phase I Uniondale, NY angion.com

BMS-813160 Bristol-Myers Squibb chronic kidney disease Phase I (dual CCR2/CCR5 antagonist) Princeton, NJ bms.com

CCX140 ChemoCentryx diabetic nephropathy Phase I (CCR2 receptor antagonist) Mountain View, CA chemocentryx.com

chronic kidney disease therapeutic Eli Lilly chronic kidney disease Phase I Indianapolis, IN lilly.com

CLP-1004 Sorbent Therapeutics chronic kidney disease Phase I (potassium channel modulator) Sunnyvale, CA sorbent.com

CTP-499 Concert Pharmaceuticals diabetic nephropathy Phase I Lexington, MA concertpharma.com

INV-144 InVasc Therapeutics chronic kidney disease Phase II (losartan/thioctic acid) Tucker, GA invasc.net

linagliptin Boehringer Ingelheim Pharmaceuticals diabetic nephropathy Phase III Ridgefield, CT boehringer-ingelheim.com Eli Lilly lilly.com Indianapolis, IN

LY3016859 Eli Lilly diabetic nephropathy Phase I Indianapolis, IN lilly.com

MP-146 Mitsubishi Tanabe Pharma chronic kidney disease Phase III Osaka, Japan mt-pharma.co.jp

MR antagonist Eli Lilly chronic kidney disease Phase II (LY2623091) Indianapolis, IN lilly.com

PF-00489791 Pfizer diabetic nephropathy Phase II (PDE5 inhibitor) New York, NY pfizer.com

PF-04634817 Pfizer diabetic nephropathy Phase II (CCR2/CCR5 receptor antagonist) New York, NY pfizer.com

Pyridorin™ NephroGenex diabetic nephropathy Phase II pyridoxamine Rsch. Triangle Park, NC (Fast Track) nephrogenex.com

TGF-ß mAb Eli Lilly diabetic nephropathy Phase II (LY2382770) Indianapolis, IN lilly.com

VTP-27999 Vitae Pharmaceuticals chronic kidney disease Phase I (renin inhibitor) Fort Washington, PA vitaepharma.com

Medicines in Development Older Americans 2013 15 Medicines in Development for Older Americans

Chronic Obstructive Pulmonary Disease (COPD)

Product Name Sponsor Indication Development Phase

aclidinium bromide/formoterol Forest Laboratories chronic obstructive pulmonary Phase III combination New York, NY disease (COPD) frx.com

AZD2115 AstraZeneca COPD Phase II (MABA) Wilmington, DE astrazeneca.com Pulmagen Therapeutics Slough, United Kingdom

AZD5423 AstraZeneca COPD Phase II (inhaled SGRM) Wilmington, DE astrazeneca.com

AZD7594 AstraZeneca COPD Phase I (inhaled SGRM) Wilmington, DE astrazeneca.com

BCT197 Novartis Pharmaceuticals COPD Phase II East Hanover, NJ novartis.com

bedoradrine MediciNova exacerbations of COPD Phase II (MN-221) San Diego, CA medicinova.com

AstraZeneca COPD Phase II (anti-IL-5R mAb) Wilmington, DE (see also asthma) astrazeneca.com MedImmune medimmune.com Gaithersburg, MD

BI-137882 Boehringer Ingelheim Pharmaceuticals COPD Phase I Ridgefield, CT boehringer-ingelheim.com

BIO-11006 BioMarck Pharmaceuticals COPD Phase II Durham, NC biomarck.com

Dulera® Merck COPD application submitted mometasone/formoterol Whitehouse Station, NJ merck.com

fluticasone furoate/vilanterol/ GlaxoSmithKline COPD Phase I umeclidinium combination Rsch. Triangle Park, NC gsk.com

formoterol/fluticasone propionate Specialty COPD Phase II completed fixed-dose combination Basking Ridge, NJ mylanspecialty.com

glycopyrrolate Sunovion Respiratory Development COPD Phase II (SUN-101) Marlborough, MA sunovion.com

GSK225629 GlaxoSmithKline COPD Phase I (soluble epoxide hydrolase Rsch. Triangle Park, NC gsk.com inhibitor)

16 Medicines in Development Older Americans 2013 Medicines in Development for Older Americans

Chronic Obstructive Pulmonary Disease (COPD)

Product Name Sponsor Indication Development Phase

GSK1325756 GlaxoSmithKline COPD Phase I (CXCR2 chemokine receptor Rsch. Triangle Park, NC gsk.com antagonist)

GSK2269557 GlaxoSmithKline COPD Phase I (phosphoinositide 3 kinase Rsch. Triangle Park, NC gsk.com inhibitor)

GSK961081 GlaxoSmithKline COPD Phase II (muscarinic antagonist/beta2 Rsch. Triangle Park, NC gsk.com agonist) Theravance theravance.com South San Francisco, CA

losmapimod GlaxoSmithKline COPD Phase II (oral p38 kinase inhibitor) Rsch. Triangle Park, NC gsk.com

MEDI-7814 AstraZeneca COPD Phase I (anti-C5/C5a mAb) Wilmington, DE astrazeneca.com MedImmune medimmune.com Gaithersburg, MD

MEDI-8968 AstraZeneca COPD Phase II (nati-IL-1R mAb) Wilmington, DE astrazeneca.com MedImmune medimmune.com Gaithersburg, MD

NVA237 Novartis Pharmaceuticals COPD Phase III (glycopyrrolate inhalation) East Hanover, NJ novartis.com

olodaterol/tiotropium bromide Boehringer Ingelheim Pharmaceuticals COPD Phase III fixed-dose combination Ridgefield, CT boehringer-ingelheim.com

OPC-6535 Otsuka America Pharmaceutical COPD Phase II (tetomilast) Rockville, MD otsuka.com

PF-03715455 Pfizer COPD Phase I (MAPK-14 inhibitor) New York, NY pfizer.com

PH-797804 Pfizer COPD Phase II (p38 MAP kinase inhibitor) New York, NY pfizer.com

Prochymal™ Osiris Therapeutics COPD Phase II remestemcel-L Columbia, MD (see also diabetes) osiris.com

PT001 Pearl Therapeutics COPD Phase II (glycopyrrolate inhalation aerosol) Redwood City, CA pearltherapeutics.com

PT003 Pearl Therapeutics COPD Phase II (glycopyrrolate/formoterol Redwood City, CA pearltherapeutics.com inhalation aerosol)

Medicines in Development Older Americans 2013 17 Medicines in Development for Older Americans

Chronic Obstructive Pulmonary Disease (COPD)

Product Name Sponsor Indication Development Phase

PT005 Pearl Therapeutics COPD Phase II (formoterol inhalation aerosol) Redwood City, CA pearltherapeutics.com

PUR118 Pulmatrix COPD Phase I Lexington, MA pulmatrix.com

QMF149 Novartis Pharmaceuticals COPD Phase II (indacaterol/mometasome East Hanover, NJ novartis.com fixed-dose combination)

QVA149 Novartis Pharmaceuticals COPD Phase III (glycopyrrolate/indacaterol East Hanover, NJ novartis.com fixed-dose combination)

salbutamol dry-powder Teva Pharmaceutical COPD Phase II inhalation North Wales, PA tevapharm.com

Striverdi Respimat™ Boehringer Ingelheim Pharmaceuticals COPD application submitted olodaterol Ridgefield, CT boehringer-ingelheim.com

Merck COPD Phase I completed (dual receptor antagonist) Whitehouse Station, NJ merck.com

TD-4208 Theravance COPD Phase II (LAMA) South San Francisco, CA theravance.com

umeclidinium GlaxoSmithKline COPD Phase III (muscarinic acetylcholine Rsch. Triangle Park, NC gsk.com antagonist)

umeclidinium/vilanterol GlaxoSmithKline COPD application submitted fixed-dose combination Rsch. Triangle Park, NC gsk.com

vilanterol GlaxoSmithKline COPD Phase III (long-acting beta2 agonist) Rsch. Triangle Park, NC gsk.com Theravance theravance.com South San Francisco, CA

vilanterol/fluticasone furoate GlaxoSmithKline COPD application submitted fixed-dose combination Rsch. Triangle Park, NC gsk.com Theravance theravance.com South San Francisco, CA ------COPD (mortality claim) Phase III gsk.com theravance.com

18 Medicines in Development Older Americans 2013 Medicines in Development for Older Americans

Depression

Product Name Sponsor Indication Development Phase

ABT-436 AbbVie major depressive disorder Phase II (selective North Chicago, IL abbvie.com antagonist)

ALKS-5461 major depressive disorder Phase II (buprenorphine/samidorphan Waltham, MA alkermes.com fixed-dose combination)

Euthymics major depressive disorder Phase II/III Cambridge, MA euthymics.com

ARA 290 Araim Pharmaceuticals depressive disorders Phase II Ossining, NY araim.org

Teva Pharmaceutical major depressive disorder Phase III Tikvah, Israel tevapharm.com

AZD6765 AstraZeneca major depressive disorder Phase II (NMDA receptor antagonist) Wilmington, DE astrazeneca.com

BCI -632 BrainCells major depressive disorder Phase I (mGluR2/3 antagonist) San Diego, CA braincellsinc.com

BCI-838 BrainCells major depressive disorder Phase I (mGluR2/3 antagonist) San Diego, CA braincellsinc.com

BMS-820836 Bristol-Myers Squibb major depressive disorder Phase II (triple reuptake inhibitor) Princeton, NJ bms.com

brexpiprazole Otsuka Pharmaceutical adjunct therapy for major Phase III (OPC-34712) Rockville, MD depressive disorder otsuka.com

Brintellix™ Lundbeck major depressive disorder application submitted Deerfield, IL lundbeck.com Takeda Pharmaceuticals International takeda.com Deerfield, IL

cariprazine Forest Laboratories major depressive disorder Phase III New York, NY frx.com

CX157 CeNeRx BioPharma major depressive disorder Phase II completed (monoamine oxidase A inhibitor) Cary, NC cenerx.com

DSP-1053 Sunovion depression Phase I (serotin uptake inhibitor) Marlborough, MA sunovion.com

Medicines in Development Older Americans 2013 19 Medicines in Development for Older Americans

Depression

Product Name Sponsor Indication Development Phase

Eli Lilly major depressive disorder Phase III (LY2216684) Indianapolis, IN lilly.com

GLYX-13 Naurex major depressive disorder Phase II Evanston, IL naurex.com

HT-2157 Dart NeuroScience major depressive disorder Phase I/II (GALR3 antagonist) San Diego, CA dartneuroscience.com

JNJ-40411813 Janssen Research & Development major depressive disorder with Phase II (mGluR2 modulator) Raritan, NJ anxiety symptoms janssenrnd.com

Forest Laboratories major depressive disorder application submitted (F2695) New York, NY frx.com

lisdexamfetamine Shire major depressive disorder Phase III Wayne, PA shire.com

lurasidone Sunovion bipolar depression application submitted Marlborough, MA sunovion.com

LY2940094 Eli Lilly major depressive disorder Phase II Indianapolis, IN lilly.com

Corcept Therapeutics psychotic major depression Phase III Menlo Park, CA (Fast Track) corcept.com

MK-6096 Merck major depressive disorder Phase II ( 1/2 antagonist) Whitehouse Station, NJ merck.com

nefiracetam Neuren Pharmaceuticals major depressive disorder Phase II Bethesda, MD post-stroke neurenpharma.com

NSI-189 Neuralstem major depressive disorder Phase I (stimulating neurogenesis) Rockville, MD neuralstem.com

PNB01 PharmaNeuroBoost severe major depressive disorder Phase III (/pipamperone Alken, Belgium pharmaneuroboost.com fixed-dose combination)

RG1578 Roche major depression disorder Phase II (mGluR2 modulator) Nutley, NJ roche.com

RG7090 Roche treatment-resistent depression Phase II (mGluR5 antagonist) Nutley, NJ roche.com

RO4995819 Roche major depressive disorder Phase II Nutley, NJ roche.com

20 Medicines in Development Older Americans 2013 Medicines in Development for Older Americans

Depression

Product Name Sponsor Indication Development Phase

SEP-228432 Sunovion major depressive disorder Phase I Marlborough, MA sunovion.com

SKL-DEP SK Biopharmaceuticals major depressive disorder Phase I Seoul, South Korea skbp.com

Lundbeck major depressive disorders Phase II Deerfield, IL lundbeck.com Takeda Pharmaceuticals International takeda.com Deerfield, IL

TGBA01AD Fabre-Kramer Pharmaceuticals major depressive disorder Phase II Houston, TX fabrekramer.com

Diabetes

Product Name Sponsor Indication Development Phase

11ß-HSD1 inhibitor Eli Lilly type 2 diabetes Phase II (LY2523199) Indianapolis, IN lilly.com

AC-201 TWi Pharmaceuticals type 2 diabetes Phase II Taipei, Taiwan twipharma.com

Afrezza® Mannkind type 1 diabetes, type 2 diabetes Phase III inhalation Valencia, CA mannkindcorp.com

GlaxoSmithKline type 2 diabetes application submitted Rsch. Triangle Park, NC (see also heart) gsk.com

aleglitazar Genentech type 2 diabetes Phase III South San Francisco, CA gene.com Roche roche.com Nutley, NJ

alpha-1 antitrypsin (AAT) OmniBio Pharmaceuticals type 1 diabetes Phase I/II Greenwood Village, CO omnibiopharma.com

AMG 151 Amgen type 2 diabetes Phase II Thousand Oaks, CA amgen.com

anagliptin Kowa Research Institute type 2 diabetes Phase II (DPP-4 inhibitor) Morrisville, NC kowaus.com

Medicines in Development Older Americans 2013 21 Medicines in Development for Older Americans

Diabetes

Product Name Sponsor Indication Development Phase

analog insulin-PH20 type 1 diabetes, type 2 diabetes Phase II San Diego, CA halozyme.com

ARI-2243 Arisaph Pharmaceuticals type 2 diabetes Phase I (DPP-4 inhibitor) Boston, MA arisaph.com

BGP-15 N-Gene Research Laboratories type 2 diabetes Phase II New York, NY ngene.us

BI-135585 Boehringer Ingelheim Pharmaceuticals type 2 diabetes Phase I (11ß-HSD1 inhibitor) Ridgefield, CT boehringer-ingelheim.com

BIOD-123 Biodel type 1 diabetes Phase II (ultra-rapid-acting recombinant Danbury, CT biodel.com human insulin)

BIOD-125 Biodel type diabetes Phase I completed (ultra-rapid-acting recombinant Danbury, CT biodel.com human insulin)

BIOD-238 Biodel diabetes Phase I (ultra-rapid-acting recombinant Danbury, CT biodel.com human insulin)

BIOD-250 Biodel diabetes Phase I (ultra-rapid-acting recombinant Danbury, CT biodel.com human insulin)

BMS-770767 Bristol-Myers Squibb type 2 diabetes Phase II (11ßHSD inhibitor) Princeton, NJ bms.com

canagliflozin Janssen Research & Development type 2 diabetes application submitted Raritan, NJ janssenrnd.com

canagliflozin/metformin Janssen Research & Development type 2 diabetes Phase III extended-release formulation Raritan, NJ janssenrnd.com

canagliflozin/metformin Janssen Research & Development type 2 diabetes application submitted immediate-release formulation Raritan, NJ janssenrnd.com

canakinumab Novartis Pharmaceuticals type 2 diabetes Phase II/III (ACZ885) East Hanover, NJ (see also arthritis) novartis.com

CAT 1004 Catabasis Pharmaceuticals type 2 diabetes Phase I Cambridge, MA catabasis.com

22 Medicines in Development Older Americans 2013 Medicines in Development for Older Americans

Diabetes

Product Name Sponsor Indication Development Phase

cetilistat Norgine type 2 diabetes in clinically obese Phase II Amsterdam, Netherlands patients norgine.com

CJC-1134-PC ConjuChem type 2 diabetes Phase II Los Angeles, CA conjuchem.com

COV 096 Pfizer type 2 diabetes Phase I (PF-04856883) New York, NY pfizer.com

dapagliflozin AstraZeneca type 2 diabetes application submitted Wilmington, DE astrazeneca.com Bristol-Myers Squibb bms.com Princeton, NJ

dapagliflozin/metformin AstraZeneca type 2 diabetes Phase III fixed-dose combination Wilmington, DE astrazeneca.com Bristol-Myers Squibb bms.com Princeton, NJ

DC-9703 Obio Pharmaceutical type 2 diabetes Phase I Kowloon, Hong Kong obiopharma.com

diabetes medicine Eli Lilly diabetes Phase I (biologic) Indianapolis, IN lilly.com

diabetes medicine Eli Lilly diabetes Phase I (biologic) Indianapolis, IN lilly.com

diabetes medicine Eli Lilly diabetes Phase I (biologic) Indianapolis, IN lilly.com

diabetes medicine Eli Lilly diabetes Phase I (biologic) Indianapolis, IN lilly.com

diabetes medicine Eli Lilly diabetes Phase I (NCE) Indianapolis, IN lilly.com

diabetes medicine Eli Lilly diabetes Phase I (NCE) Indianapolis, IN lilly.com

DiaPep277® Andromeda Biotech type 1 diabetes Phase III Yavne, Israel andromedabio.com

DS-7250 Daiichi Sankyo diabetes Phase I (DGAT1 inhibitor) Parsippany, NJ dsi.com

Medicines in Development Older Americans 2013 23 Medicines in Development for Older Americans

Diabetes

Product Name Sponsor Indication Development Phase

DSP-8658 Sunovion Pharmaceuticals type 2 diabetes Phase I (PPAR alpha/gamma agonist) Marlborough, MA (see also Alzheimer’s) sunovion.com

Eli Lilly type 2 diabetes Phase III (LY2189265) Indianapolis, IN lilly.com

EGT-0001442 Theracos type 2 diabetes Phase II (SGLT2 inhibitor) Marlborough, MA theracos.com

EGT-0001474 Theracos type 2 diabetes Phase I completed (SGLT2 inhibitor) Marlborough, MA theracos.com

empagliflozin Boehringer Ingelheim Pharmaceuticals type 2 diabetes Phase III (BI-10773) Ridgefield, CT boehringer-ingelheim.com Eli Lilly lilly.com Indianapolis, IN

empagliflozin/linagliptin Boehringer Ingelheim Pharmaceuticals type 2 diabetes Phase III fixed-dose combination Ridgefield, CT boehringer-ingelheim.com Eli Lilly lilly.com Indianapolis, IN

empagliflozin/metformin Boehringer Ingelheim Pharmaceuticals type 2 diabetes Phase I fixed-dose combination Ridgefield, CT boehringer-ingelheim.com Eli Lilly lilly.com Indianapolis, IN

emricasan Conatus Pharmaceuticals type 1 diabetes Phase I/II San Diego, CA conatuspharma.com

enclomifene type 2 diabetes in men with Phase II The Woodlands, TX secondary hypogonadism reprosrx.com

fasiglifam Takeda Pharmaceuticals International type 2 diabetes Phase III (TAK-875) Deerfield, IL takeda.com

GK1-399 Forest Laboratories type 2 diabetes Phase II (glucokinase activator) New York, NY frx.com

glimepiride extended-release/ GlaxoSmithKline type 2 diabetes Phase I metformin extended-release Rsch. Triangle Park, NC gsk.com fixed-dose combination

-R antagonist Eli Lilly type 2 diabetes Phase II (LY2409021) Indianapolis, IN lilly.com

GPR40 Bristol-Myers Squibb type 2 diabetes in clinical trials Princeton, NJ bms.com

24 Medicines in Development Older Americans 2013 Medicines in Development for Older Americans

Diabetes

Product Name Sponsor Indication Development Phase

GPR119 agonist Bristol-Myers Squibb type 2 diabetes in clinical trials Princeton, NJ bms.com

GSK1070806 GlaxoSmithKline type 2 diabetes Phase II (anti-IL18 mAb) Rsch. Triangle Park, NC gsk.com

GSK1614235/GSK2330672 GlaxoSmithKline type 2 diabetes Phase I (SGLT1 inhibitor/iBAT inhibitor) Rsch. Triangle Park, NC gsk.com

HE3286 Harbor Biosciences type 2 diabetes Phase II San Diego, CA (see also arthritis) harbortx.com

HM-11260C Hanmi Pharmaceuticals type 2 diabetes Phase II ( long-acting) Seoul, South Korea hanmipharm.com

HPP404 High Point Pharmaceuticals diabetes Phase I (histamine H3 receptor antagonist) High Point, NC highpointpharma.com

HPP593 High Point Pharmaceuticals diabetes Phase I (PPAR-delta agonist) High Point, NC highpointpharma.com

human proislet CureDM type 2 diabetes Phase I (HIP2B) Wilmington, DE curedm.com

IDegLira Novo Nordisk type 2 diabetes Phase III / Princeton, NJ novonordisk.com

insulin B-chain vaccine Orban Biotech type 1 diabetes Phase I completed Brookline, MA orbanbiotech.com

Sanofi US type 2 diabetes Phase III (new formulation) Bridgewater, NJ sanofi.com

Eli Lilly type 1 diabetes, type 2 diabetes Phase I Indianapolis, IN lilly.com

INT131 InteKrin Therapeutics type 2 diabetes Phase II Los Altos, CA intekrin.com

ISIS-GCCRRx Isis Pharmaceuticals type 2 diabetes Phase I Carlsbad, CA isispharm.com

ISIS-GCGRRx Isis Pharmaceuticals type 2 diabetes Phase I Carlsbad, CA isispharm.com

ISIS-PTP1BRx Isis Pharmaceuticals type 2 diabetes Phase I Carlsbad, CA isispharm.com

Medicines in Development Older Americans 2013 25 Medicines in Development for Older Americans

Diabetes

Product Name Sponsor Indication Development Phase

ISIS-SGLT2Rx Isis Pharmaceuticals type 2 diabetes Phase I Carlsbad, CA isispharm.com

ITCA 650 Intarcia Therapeutics type 2 diabetes Phase III (exenatide subcutaneous implant) Hayward, CA intarcia.com

Januvia® Merck type 2 diabetes in elderly patients Phase III sitagliptin Whitehouse Station, NJ with inadequate glycemic control merck.com

JNJ-16269110 Janssen Research & Development type 2 diabetes Phase II completed (usistapide) Raritan, NJ janssenrnd.com

JNJ-41443532 Janssen Research & Development type 2 diabetes Phase II completed Raritan, NJ janssenrnd.com

JTT-851 Akros Pharma type 2 diabetes Phase II Princeton, NJ akrospharma.com

KD026 Kadmon Pharmaceuticals type 2 diabetes Phase II (MTP inhibitor) New York, NY kadmon.com

KRP-104 ActivX Biosciences type 2 diabetes Phase II La Jolla, CA activx.com

Lantus®/lixisenatide Sanofi US type 2 diabetes Phase II insulin glargine and Bridgewater, NJ sanofi.com fixed-ratio combination

Lantus®/lixisenatide Sanofi US type 2 diabetes Phase I insulin glargine and lixisenatide Bridgewater, NJ sanofi.com fix-flex combination

LCQ908 Novartis Pharmaceuticals type 2 diabetes Phase II (DGAT 1 inhibitor) East Hanover, NJ (see also heart) novartis.com

LEZ763 Novartis Pharmaceuticals type 2 diabetes Phase II East Hanover, NJ novartis.com

LIK066 Novartis Pharmaceuticals type 2 diabetes Phase I/II (SGLT 1/2 inhibitor) East Hanover, NJ novartis.com

linagliptin/pioglitazone Boehringer Ingelheim Pharmaceuticals type 2 diabetes Phase III fixed-dose combination Ridgefield, CT boehringer-ingelheim.com Eli Lilly lilly.com Indianapolis, IN

26 Medicines in Development Older Americans 2013 Medicines in Development for Older Americans

Diabetes

Product Name Sponsor Indication Development Phase

luseogliflozin Taisho Pharmaceuticals type 2 diabetes Phase I (TS-071) Tokyo, Japan taisho.co.jp

LX4211 Lexicon Pharmaceuticals type 1 diabetes, type 2 diabetes Phase II (SLGT1/SLGT2 inhibitor) The Woodlands, TX lexicon-genetics.com

Lyxumia® Sanofi US type 2 diabetes application submitted lixisenatide Bridgewater, NJ sanofi.com (GLP-1 agonist)

MABp1 XBiotech type 2 diabetes Phase II Austin, TX xbiotech.com

MBX-2982 Metabolex type 2 diabetes Phase II Hayward, CA metabolex.com

mesenchymal stem cell therapy Mesoblast type 2 diabetes Phase I/II New York, NY (see also heart) mesoblast.com

mitiglinide Elixir Pharma type 2 diabetes Phase III Gujarat, India elixirpharma.in

MK-8655 Merck type 2 diabetes Phase I Whitehouse Station, NJ merck.com

MSDC-0160 Metabolic Solutions Development type 2 diabetes Phase II (MTOR protein inhibitor) Kalamazoo, MI (see also Alzheimer’s) msdrx.com

MSDC-0602 Metabolic Solutions Development type 2 diabetes Phase II (MTOR protein inhibitor) Kalamazoo, MI msdrx.com

new insulin glargine product Boehringer Ingelheim Pharmaceuticals type 2 diabetes Phase III (LY2963016) Ridgefield, CT boehringer-ingelheim.com Eli Lilly lilly.com Indianapolis, IN

NewMet™ Elcelyx Therapeutics type 2 diabetes Phase II delayed-release metformin San Diego, CA elcelyx.com

NGM-282 NGM Biopharmaceuticals diabetes Phase I South San Francisco, CA ngmbio.com

NN1218 Novo Nordisk type 1 diabetes, type 2 diabetes Phase I ( fast-acting) Princeton, NJ novonordisk.com

Medicines in Development Older Americans 2013 27 Medicines in Development for Older Americans

Diabetes

Product Name Sponsor Indication Development Phase

NN1436 Novo Nordisk type 1 diabetes, type 2 diabetes Phase I (LAI287) Princeton, NJ novonordisk.com

NN1953 Novo Nordisk type 1 diabetes, type 2 diabetes Phase I (OI338GT) Princeton, NJ novonordisk.com

NN1954 Novo Nordisk type 1 diabetes, type 2 diabetes Phase I (OI362GT) Princeton, NJ novonordisk.com

NN9924 Novo Nordisk type 2 diabetes Phase I (OG217SC) Princeton, NJ novonordisk.com

NN9926 Novo Nordisk type 2 diabetes Phase I (OG987GT) Princeton, NJ novonordisk.com

NN9927 Novo Nordisk type 2 diabetes Phase I (OG987SC) Princeton, NJ novonordisk.com

novel basal insulin/insulin Eli Lilly type 2 diabetes Phase III peglispro Indianapolis, IN lilly.com (LY2605541) ------type 1 diabetes Phase I lilly.com

omarigliptin Merck type 2 diabetes Phase III (MK-3102) Whitehouse Station, NJ merck.com

Onglyza® AstraZeneca type 2 diabetes Phase III saxagliptin Wilmington, DE (combination therapy) astrazeneca.com Bristol-Myers Squibb bms.com Princeton, NJ

Oral-lyn™ Generex Biotechnology type 1 diabetes, type 2 diabetes Phase III oral insulin Toronto, Canada generex.com

Merck type 2 diabetes Phase I Whitehouse Station, NJ merck.com

PAZ320 Boston Therapeutics type 2 diabetes Phase II Manchester, NH bostonti.com

PB1023 PhaseBio Pharmaceuticals type 2 diabetes Phase II Malvern, PA phasebio.com

PEG-FGF21 Bristol-Myers Squibb type 2 diabetes in clinical trials Princeton, NJ bms.com

28 Medicines in Development Older Americans 2013 Medicines in Development for Older Americans

Diabetes

Product Name Sponsor Indication Development Phase

PF-04937319 Pfizer type 2 diabetes Phase II New York, NY pfizer.com

PF-04971729 Pfizer type 2 diabetes Phase II (ertugliflozin) New York, NY pfizer.com

PF-05175157 Pfizer type 2 diabetes Phase I New York, NY pfizer.com

PF-05231023 Pfizer type 2 diabetes Phase I New York, NY pfizer.com

PF-06291874 Pfizer type 2 diabetes Phase I New York, NY pfizer.com

PF-06342674 Pfizer type 1 diabetes Phase I New York, NY pfizer.com

Prochymal® Osiris Therapeutics type 1 diabetes Phase II remestemcel-L Columbia, MD (see also COPD) osiris.com

Qsymia™ VIVUS type 2 diabetes Phase II phentermine/ Mountain View, CA vivus.com

ranolazine extended-release type 2 diabetes Phase III (late sodium current inhibitor) Foster City, CA gilead.com

RG7697 Roche type 2 diabetes Phase I (GLP-1/GIP receptors) Nutley, NJ roche.com

RM-493 Rhythm Pharmaceuticals diabetes Phase I (MC4R peptide) Boston, MA rhythmtx.com

RO6811135 Roche type 2 diabetes Phase I Nutley, NJ roche.com

roflumilast Takeda Pharmaceuticals International type 2 diabetes Phase I Deerfield, IL takeda.com

Ryzodeg® Novo Nordisk type 1 diabetes, type 2 diabetes application submitted insulin degludec/insulin aspart Princeton, NJ novonordisk.com

S-707106 Shionogi type 2 diabetes Phase II Florham Park, NJ shionogi.com

SaxaDapa AstraZeneca diabetes Phase III saxagliptin/dapagliflozin Wilmington, DE astrazeneca.com fixed-dose combination Bristol-Myers Squibb bms.com Princeton, NJ

Medicines in Development Older Americans 2013 29 Medicines in Development for Older Americans

Diabetes

Product Name Sponsor Indication Development Phase

Novo Nordisk type 2 diabetes Phase II (NN9535) Princeton, NJ novonordisk.com

SRT2104 Sirtris type 2 diabetes Phase II (SIRT1 protein stimulant) Cambridge, MA sirtrispharma.com

SRT2379 Sirtris type 2 diabetes Phase I (SIRT1 protein stimulant) Cambridge, MA sirtrispharma.com

SRT3025 Sirtris type 2 diabetes Phase I (SIRT1 protein stimulant) Cambridge, MA sirtrispharma.com

SYR-472 Takeda Pharmaceuticals International type 2 diabetes Phase II (trelagliptin) Deerfield, IL takeda.com

TAK-329 Takeda Pharmaceuticals International type 1 diabetes Phase I (glucokinase stimulant) Deerfield, IL takeda.com

teneligliptin Mitsubishi Tanabe Pharma type 2 diabetes Phase I (MP-513) Osaka, Japan mt-pharma.co.jp

MacroGenics type 1 diabetes Phase III (anti-CD3 mAb) Rockville, MD macrogenics.com

tofogliflozin Chugai Pharmaceutical type 2 diabetes Phase II (CSG452) Tokyo, Japan chugai-pharm.co.jp Genentech gene.com South San Francisco, CA

Tresiba® Novo Nordisk type 1 diabetes, type 2 diabetes application submitted insulin degludec Princeton, NJ novonordisk.com

TTP054 TransTech Pharma type 2 diabetes Phase II (GLP-1 receptor agonist) High Point, NC ttpharma.com

TTP814 TransTech Pharma type 2 diabetes Phase I/II (PTP-1B inhibitor) High Point, NC ttpharma.com

U-Strip Transdermal Global type 1 diabetes, type 2 diabetes Phase I (insulin transdermal) Norwalk, CT transdermalspecialties.com

vildagliptin Novartis Pharmaceuticals type 2 diabetes Phase III East Hanover, NJ novartis.com

vildagliptin/metformin Novartis Pharmaceuticals type 2 diabetes Phase III combination East Hanover, NJ novartis.com

VRS-859 Diartis Pharmaceuticals type 2 diabetes Phase I Redwood City, CA diartispharma.com

30 Medicines in Development Older Americans 2013 Medicines in Development for Older Americans

Diabetes

Product Name Sponsor Indication Development Phase

VVP808 Verva Pharmaceuticals type 2 diabetes Phase II (insulin sensitizer) Carlsbad, CA vervapharma.com

ZP2929 Boehringer Ingelheim Pharmaceuticals type 2 diabetes Phase I Ridgefield, CT boehringer-ingelheim.com Zealand Pharma zealandpharma.com Copenhagen, Denmark /Ischemic Heart Disease

Product Name Sponsor Indication Development Phase

ACP-501 AlphaCore Pharma coronary artery disease Phase I (rhLCAT) Ann Arbor, MI alphacorepharma.com

ACY001 Compass Biomedical cardiac ischemia Phase I (stem cell therapy) (Arteriocyte) compassbiomedical.com Cleveland, OH

adult mesenchymal stem cell BioCardia ischemic heart disorders Phase I/II therapy San Carlos, CA biocardia.com

albiglutide GlaxoSmithKline heart failure Phase II Rsch. Triangle Park, NC (see also diabetes) gsk.com

ALD-201 Cytomedix ischemic heart disorder Phase I (stem cell therapy) Gaithersburg, MD cytomedix.com

ANX-042 Anexon acute decompensated heart failure Phase I () Cambridge, MA anexonrx.com

BAY 10-21189 Bayer HealthCare Pharmaceuticals heart failure Phase I (sGC stimulator) Wayne, NJ bayerpharma.com

BAY 10-67197 Bayer HealthCare Pharmaceuticals heart failure Phase I (partial adenosin A1 agonist) Wayne, NJ bayerpharma.com

BAY 86-8050 Bayer HealthCare Pharmaceuticals heart failure Phase I (vasopressin receptor) Wayne, NJ bayerpharma.com

BAY 94-8862 Bayer HealthCare Pharmaceuticals chronic heart failure Phase II (MR antagonist) Wayne, NJ bayerpharma.com

Medicines in Development Older Americans 2013 31 Medicines in Development for Older Americans

Heart Failure/Ischemic Heart Disease

Product Name Sponsor Indication Development Phase

beperminogene perplasmid AnGes MG ischemic heart disorders Phase I Osaka, Japan anges-mg.com

BFPET™ FluoroPharma coronary artery disease (diagnosis) Phase II blood flow imaging agent Montclair, NJ fluoropharma.com

BR-4628 Bayer HealthCare Pharmaceuticals heart failure Phase II (MR antagonist) Wayne, NJ bayerpharma.com

bucindolol ARCA Biopharma heart failure application submitted Broomfield, CO (Fast Track) arcabiopharma.com

bucindolol companion diagnostic ARCA Biopharma heart failure (diagnosis) in clinical trials Broomfield, CO arcabiopharma.com LaBCorp labcorp.com Burlington, NC

cangrelor The Medicines Company coronary artery disease Phase III Parsippany, NJ themedicinescompany.com

CardioPET™ FluoroPharma fatty acid uptake in coronary artery Phase II muscle state imaging agent Montclair, NJ disease (diagnosis) fluoropharma.com

cenderitide Nile Therapeutics acute heart failure Phase II (CD-NP) San Mateo, CA (Fast Track) nilethera.com ------chronic heart failure Phase I (Fast Track) nilethera.com

CLP-1001 Sorbent Therapeutics heart failure in patients with Phase II Sunnyvale, CA chronic kidney disease sorbent.com

combination autologous stem cell TCA Cellular Therapy ischemic heart disorders Phase II therapy Covington, LA tcacellulartherapy.com

CXL-1020-IV Cardioxyl Pharmaceuticals acute heart failure Phase II Chapel Hill, NC cardioxyl.com

endometrial blood stem cell MediStem heart failure Phase II therapy San Diego, CA medisteminc.com

epicardial angiogenesis patch Theregen heart failure, ischemic heart Phase I San Francisco, CA disorders theregen.com

GGF 2 Acroda Therapeutics heart failure Phase I (glial growth factor 2) Ardsley, NY acroda.com

32 Medicines in Development Older Americans 2013 Medicines in Development for Older Americans

Heart Failure/Ischemic Heart Disease

Product Name Sponsor Indication Development Phase

GSK2849466 GlaxoSmithKline heart failure Phase I (selective androgen receptor Rsch. Triangle Park, NC gsk.com modulator)

JNJ-39588146 Janssen Research & Development heart failure Phase II completed Raritan, NJ janssenrnd.com

JVS-100 Juventas Therapeutics heart failure Phase II Cleveland, OH juventasinc.com

LCQ908 Novartis Pharmaceuticals coronary artery disease Phase II (DGAT1 inhibitor) East Hanover, NJ (see also diabetes) novartis.com

LCZ696 Novartis Pharmaceuticals heart failure Phase III (NEP inhibitor/ARB) East Hanover, NJ novartis.com

LMI 1195 Lantheus Medical Imaging heart failure (diagnosis) Phase I (cardiac neuronal agent) N. Billerica, MA lantheus.com

mesenchymal stem cell therapy Mesoblast heart failure Phase II New York, NY (see also diabetes) mesoblast.com

MTP-131 Stealth chronic heart failure Phase I Newton Centre, MA stealthpeptides.com

Mydicar® Celladon advanced (Class III/IV) heart failure Phase II SERCA 2a gene therapy San Diego, CA (Fast Track) celladon.net

MyoCell® Bioheart heart failure Phase II/III cell-based heart damage therapy Sunrise, FL bioheartinc.com

MyoCell® SDF-1 Bioheart heart failure Phase I cell-based heart damage therapy Sunrise, FL bioheartinc.com

omecamtiv mecarbil Amgen heart failure Phase II Thousand Oaks, CA amgen.com

PCSK9 adnectin Bristol-Myers Squibb ischemic heart disease in clinical trials Princeton, NJ bms.com

PEG-relaxin Bristol-Myers Squibb heart failure in clinical trials Princeton, NJ bms.com

PL-3994 Palatin Technologies heart failure Phase II (NP receptor A agonist) Cranbury, NJ palatin.com

recombinant human Zensun chronic heart failure Phase II neuregulin-1 beta Shanghai, China zensun.com

Medicines in Development Older Americans 2013 33 Medicines in Development for Older Americans

Heart Failure/Ischemic Heart Disease

Product Name Sponsor Indication Development Phase

RLX030 Novartis Pharmaceuticals acute heart failure Phase III (serelaxin) East Hanover, NJ (Fast Track) novartis.com ------chronic heart failure Phase II novartis.com

SAR126119 Sanofi US acute ischemic stroke Phase I (TAFIa inhibitor) Bridgewater, NJ sanofi.com

-eluting coronary stent Svelte Medical Systems ischemic heart disorder Phase II New Providence, NJ sveltemedical.com

Tekturna® Novartis Pharmaceuticals acute heart failure in the elderly, Phase III aliskiren East Hanover, NJ chronic heart failure novartis.com

TRV027 Trevena acute heart failure Phase II (AT1R) King of Prussia, PA trevena.com

ularitide Cardiorentis acute heart failure Phase III Zug, Switzerland cardiorentis.com

2 Neurocrine Biosciences heart failure Phase II San Diego, CA neurocrine.com

VM202 Cordis myocardial ischemia Phase I/II (modified hepatocyte growth Bridgewater, NJ cordis.com factor gene therapy) ViroMed viromed.com Minneapolis, MN Osteoporosis

Product Name Sponsor Indication Development Phase

AUS-131 Ausio Pharmaceuticals osteoporosis Phase I completed ( receptor beta agonist) Cincinnati, OH ausiopharma.com

BA-058 SC Radius Health postmenopausal osteoporosis Phase III (human parathyroid Cambridge, MA radiuspharm.com hormone-related protein)

BA-058 TD Radius Health postmenopausal osteoporosis Phase II (human parathyroid Cambridge, MA radiuspharm.com hormone-related protein)

bazedoxifene/conjugated Pfizer postmenopausal osteoporosis application submitted combination New York, NY (prevention) pfizer.com

34 Medicines in Development Older Americans 2013 Medicines in Development for Older Americans

Osteoporosis

Product Name Sponsor Indication Development Phase

blosozumab Eli Lilly postmenopausal osteoporosis Phase II (LY2541546) Indianapolis, IN lilly.com

BPS804 Novartis Pharmaceuticals postmenopausal osteoporosis Phase II (anti-sclerostin IgG2 antibody) East Hanover, NJ novartis.com

oral Tarsa Therapeutics postmenopausal osteoporosis Phase II (recombinant) Philadelphia, PA tarsatherapeutics.com

DP-001 Deltanoid Pharmaceuticals osteoporosis Phase II Madison, WI deltanoid.com

Fablyn® Ligand Pharmaceuticals postmenopausal osteoporosis application submitted lasofoxifene La Jolla, CA ligand.com

odanacatib Merck postmenopausal osteoporosis, Phase III Whitehouse Station, NJ male osteoporosis merck.com

romosozumab Amgen postmenopausal osteoporosis Phase III (anti-sclerostin mAb) Thousand Oaks, CA amgen.com UCB ucb.com Brussels, Belgium

sotatercept Acceleron Pharma postmenopausal osteoporosis Phase I (ACE-011) Cambridge, MA acceleronpharma.com Celgene celgene.com Summit, NJ

Viviant™ Pfizer osteoporosis treatment and application submitted bazedoxifene New York, NY prevention pfizer.com

ZT-034 Azelon Pharmaceuticals osteoporosis Phase II ( nasal spray West Conshohocken, PA formulation)

ZP-PTH Zosano Pharma osteoporosis Phase II (teriparatide transdermal) Fremont, CA zosanopharma.com

Medicines in Development Older Americans 2013 35 Glossary

adjunctive therapy—Auxiliary treatment natural decline of these functions with (a condition in which the body fails to that is secondary to the main treatment. age is grossly exaggerated in dementia. properly use insulin), combined with relative insulin deficiency. Most Ameri- Alzheimer’s disease—The most com- depression—A mental illness character- cans who are diagnosed with diabetes mon form of dementia, characterized ized by an intense feeling of sadness, have type 2, which in most cases can by progressive and chronic deteriora- which is of greater proportion and be controlled if treated properly by a tion of cognitive functions, including duration than expected by objective combination of dietary measures, weight memory, thinking and reasoning. Early reason (i.e., recent loss or other sad loss, and oral . manifestations include forgetfulness, event). Major depressive disorder (or impaired ability to focus, and changes major depression) includes an episode of diabetic nephropathy—Kidney disease in mood and personality. As the disease depression defined as a persistent (for or damage that can occur in people with progresses, there is a loss of computa- at least 2 weeks) mood disturbance, plus diabetes. The kidneys have many tiny tional ability, in addition to word-finding at least four of the following symptoms: blood vessels that filter waste from the problems and difficulty with ordinary sleep disturbance, changes in psychomo- blood. High blood sugar from diabetes activities. Ultimately, the disease leads tor activity, loss of ability to experience can destroy those blood vessels. Over to severe memory loss, complete dis- pleasure and interest, fatigue, feelings time, the kidney isn’t able to do its job orientation, social withdrawal, loss of of worthlessness or guilt, difficulty in as well and may stop working com- independence, and death. concentrating, and/or suicidal thoughts. pletely, which is called kidney failure. Major depression is associated with im- Out of 100 people with diabetes, as application submitted—An application pairment in social functioning. If criteria many as 40 will develop kidney dam- for marketing has been submitted to for major depression have been met but age. Currently, diabetic nephropathy the U.S. Food and Drug Administration in addition an episode of mania has ever is the leading cause of chronic kidney (FDA). The application can either be an occurred, then the diagnosis becomes disease in the United States and other NDA (new drug application) or a BLA bipolar disorder (or manic-depressive ill- Western societies. It is also one of the (biologic license application). ness). The essential feature of mania is a most significant long-term complications distinct period when the mood is either in terms of morbidity and mortality for chronic obstructive pulmonary disease elevated, expansive, or irritable, with as- individual patients with diabetes, which (COPD)—A group of lung diseases, sociated symptoms including hyperactiv- is responsible for up to 40 percent of all including chronic bronchitis and em- ity, flight of ideas, inflated self-esteem, end-stage renal disease (ESRD) cases in physema, in which there is a persistent decreased need for sleep, distractibility the United States. disruption of airflow out of the lungs and excessive involvement in activities and eventual hypoxemia (low level of that often are flamboyant, bizarre or Fast Track—Fast Track is a process oxygen in the blood). disorganized. designed to facilitate the development and expedite the review of drugs to cognitive disorders—Disorders of the diabetes—A chronic disease in which treat serious diseases and fill an unmet higher mental processes, including the body does not produce or properly medical need. The status is assigned by understanding, reasoning, knowledge, use insulin, a hormone that is needed the U.S. Food and Drug Administration. and intellectual capacity. A person with to convert sugar, starches and other The purpose is to get important new a cognitive disorder, such as Alzheimer’s food into energy needed for daily life. drugs to the patient earlier. Fast Track disease, does not process information Symptoms may include excessive thirst, addresses a broad range of serious correctly within the brain, resulting in hunger, urination and weight loss. The diseases. Generally, determining fac- impaired awareness and judgment, dif- cause of diabetes continues to be a tors include whether the drug will have ficulty reasoning and focusing, loss of mystery, although both genetics and an impact on such factors as survival, memory and abnormal mental capacity. environmental factors such as obesity day-to-day functioning, or the likelihood People with cognitive disorders have and lack of exercise appear to play roles. that the disease, if left untreated, will problems acquiring, mentally organizing Type 1 diabetes, the more severe form, progress from a less severe condition and responding to information, which results from the body’s failure to pro- to a more serious one. Filling an unmet results in an inability to function nor- duce insulin, which “unlocks” the cells of medical need is defined as providing a mally in everyday life situations. the body, allowing glucose to enter and therapy where none exists or providing a dementia—Loss of mental ability that fuel them. It is estimated that 5 percent therapy which may be potentially supe- interferes with normal daily activities. to 10 percent of Americans who are rior to existing therapy. Once a drug re- It lasts more than six months, is not diagnosed with diabetes have type 1, ceives Fast Track designation, early and present at birth and is not associated which requires insulin treatment. Type 2 frequent communication between the with loss or altered consciousness. The diabetes results from insulin resistance FDA and a drug company is encouraged

36 Medicines in Development Older Americans 2013 Glossary

throughout the entire drug development loss, significant changes in personal- Drug (IND) studies designed to speed and review process. The frequency of ity, and the need for assistance with up development of promising drugs by communication assures that questions basic daily activities. These changes establishing very early on whether the and issues are resolved quickly, often are related to damage in areas of the agent behaves in human subjects as was leading to earlier drug approval and ac- brain that control language, reasoning, anticipated from preclinical studies. cess by patients. sensory processing, and conscious think- ing. At this stage, patients may have Phase I—Researchers test the drug in a heart failure—The end result of many problems recognizing family and friends, small group of people, usually between different types of heart disease. The experience hallucinations, delusions, and 20 and 80 healthy adult volunteers, to heart cannot pump blood out normally. paranoia, and may behave impulsively. evaluate its initial safety and tolerability This results in congestion (water and salt profile, determine a safe dosage range, retention) in the lungs, swelling in the osteoarthritis—The most common form and identify potential side effects. extremities, and reduced blood flow to of joint disease, characterized by degen- body tissues. eration of the cartilage that lines joints Phase II—The drug is given to volun- and by the formation of reactive bony teer patients, usually between 100 and ischemia—Insufficient supply of blood outgrowths at the boundary of a joint. 300, to see if it is effective, identify an to an organ or tissue, which can cause optimal dose, and to further evaluate its organ damage such as an ischemic osteoporosis—The most common short-term safety. stroke. metabolic bone disease in older people in which a reduction in bone mass leads Phase III—The drug is given to a larger, mild Alzheimer’s disease—A stage of to fractures, especially of the vertebrae, more diverse patient population, often Alzheimer’s disease characterized by a hips and wrists. involving between 1,000 and 3,000 series of changes in cognitive abilities patients (but some time many more that may include memory loss for recent PET imaging—Positron emission to- thousands), to generate statistically events, difficulty with problem solving, mography (PET), a noninvasive medical significant evidence to confirm its safety changes in personality, difficulty orga- imaging technique that utilizes a ra- and effectiveness. They are the longest nizing and expressing thoughts, getting dioactive agent (“tracer”) incorporated studies and usually take place in multiple lost or misplacing belongings. This is the in a biologically active molecule and a sites around the world. stage at which the disease is often first scanner to produce three-dimensional diagnosed. images of the body. rheumatoid arthritis—A type of arthritis that particularly attacks the small joints moderate Alzheimer’s disease—A stage Phase 0—First-in-human trials conduct- of the hands, wrists and feet. The joints of Alzheimer’s disease characterized by ed in accordance with FDA’s 2006 guid- become painful, swollen, stiff and, in increased confusion, greater memory ance on exploratory Investigational New severe cases, deformed.

The content of this report has been obtained through public, government and industry sources, and the Adis “R&D Insight” data- base based on the latest information. Report current as of March 10, 2013. The medicines in this report include medicines being developed by U.S. based companies conducting trials in the United States and abroad, PhRMA-member companies conducting trials in the United States and abroad and foreign companies conducting clinical trials in the United States. The information in this report 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 Provided as a Public Service by PhRMA. Founded in 1958 as the Pharmaceutical Manufacturers Association. Copyright © 2013 by the Pharmaceutical Research and Manufacturers of America. Permission to reprint is awarded if proper credit is given.

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Medicines in Development Older Americans 2013 37 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.

Drug Discovery and Development: A LONG, RISKY ROAD

DRUG DISCOVERY PRECLINICAL CLINICAL TRIALS FDA REVIEW LG-SCALE MFG Y

5,000–10,000 250 5 VEILLANCE ONE FDA-

VER COMPOUNDS APPROVED O DRUG

PHASE PHASE PHASE ARKETING SUR 1 2 3 -M ST NUMBER OF VOLUNTEERS PRE-DISC 20–80 100–300 1,000–3,000 A SUBMITTED 0.5–2 IND SUBMITTE D 3–6 YEARS6–7 YEARS ND

YEARS PHASE 4: PO The Drug Development and Approval Process The U.S. system of new drug approvals is per- in people. The IND shows results of previous ate statistically significant evidence to confirm haps the most rigorous in the world. experiments; how, where and by whom the new its safety and effectiveness. They are the lon- studies will be conducted; the chemical structure gest studies, and usually take place in multiple It takes 10-15 years, on average, for an experi- of the compound; how it is thought to work in sites around the world. mental drug to travel from lab to U.S. patients, the body; any toxic effects found in the animal according to the Tufts Center for the Study of New Drug Application (NDA)/Biologic License studies; and how the compound is manufac- Drug Development. Only five in 5,000 com- Application (BLA). Following the completion tured. All clinical trials must be reviewed and ap- pounds that enter preclinical testing make it to of all three phases of clinical trials, a company proved by the Institutional Review Board (IRB) human testing. And only one of those five is analyzes all of the data and files an NDA or BLA where the trials will be conducted. Progress approved for sale. with FDA if the data successfully demonstrate reports on clinical trials must be submitted at both safety and effectiveness. The applications On average, it costs a company $1.2 billion, least annually to FDA and the IRB. contain all of the scientific information that the including the cost of failures, to get one new Clinical Trials, Phase I—Researchers test the company has gathered. Applications typically medicine from the laboratory to U.S. patients, drug in a small group of people, usually between run 100,000 pages or more. according to a recent study by the Tufts Center 20 and 80 healthy adult volunteers, to evaluate for the Study of Drug Development. Approval. Once FDA approves an NDA or BLA, its initial safety and tolerability profile, deter- the new medicine becomes available for physi- Once a new compound has been identified in mine a safe dosage range, and identify potential cians to prescribe. A company must continue the laboratory, medicines are usually developed side effects. to submit periodic reports to FDA, including as follows: Clinical Trials, Phase II—The drug is given any cases of adverse reactions and appropriate Preclinical Testing. A pharmaceutical company to volunteer patients, usually between 100 and quality-control records. For some medicines, conducts laboratory and animal studies to show 300, to see if it is effective, identify an optimal FDA requires additional trials (Phase IV) to biological activity of the compound against the dose, and to further evaluate its short-term evaluate long-term effects. targeted disease, and the compound is evalu- safety. Discovering and developing safe and effective ated for safety. Clinical Trials, Phase III—The drug is given to a new medicines is a long, difficult, and expensive Investigational New Drug Application (IND). larger, more diverse patient population, often process. PhRMA member companies invested an After completing preclinical testing, a com- involving between 1,000 and 3,000 patients estimated $49.5 billion in research and develop- pany files an IND with the U.S. Food and Drug (but sometime many more thousands), to gener- ment in 2011. Administration (FDA) to begin to test the drug