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Heart 2013
1. 2013REPORT
Biopharmaceutical research companies
are developing 215 medicines for two of
the leading causes of death in Ameri-
cans—heart disease and stroke. Medi-
cines have helped cut deaths from these
diseases by 30 percent between 2001
and 2011, and in 2008 stroke dropped to
the fourth leading cause of death after
being the third for more than 50 years.
This progress is in large part due to
innovative medicines.
Despite this progress, heart disease and
stroke persist as key public health chal-
lenges. According to the American Heart
Association, every 39 seconds an Ameri-
can dies from cardiovascular disease, and
more than 83 million Americans have
at least one type of the disease. These
diseases cost our society more than $312
billion a year.
The medicines in development include:
30 for heart failure, 29 for lipid disorders
(such as high cholesterol), 19 for stroke,
and 17 each for high blood pressure and
ischemic disorders. Many of the potential
medicines use cutting-edge technologies
and new scientific approaches, such as:
• A gene therapy that uses a patient’s
own cells to treat heart failure.
• A medicine that blocks the transfer of
good (HDL) cholesterol to bad (LDL).
• A genetically-engineered medicine that
dissolves clots to treat stroke.
All of the medicines are either in clinical
trials or awaiting review by the Food and
Drug Administration. A glossary of terms
begins on page 24. Links to sponsor
company websites provide more informa-
tion on the potential products.
For information on the current innovative
products in the pipeline for cardiovascular
disease and the value of medicines for
patients and our healthcare system, please
see Medicines in Development for Heart
Disease and Stroke 2013—Overview.
Biopharmaceutical Research Companies Are
Developing More Than 200 Medicines for
Cardiovascular Disease
Medicines in Development
Heart Disease and Stroke
A Report on Cardiovascular Disease
presented by america’s biopharmaceutical research companies
LipidDisorders
Hypertension
30
17 17
IschemicDisorders
29
HeartFailure
Stroke
19
Application
Submitted
Phase III
Phase II
Phase I
Medicines in Development
For Heart Disease and Stroke
83.6 Million
Americans Suffer
From Cardiovascular
Disease
More Than
700,000
Die Each Year
2. Medicines in Development Heart Disease and Stroke 20132
Medicines in Development for Heart Disease and Stroke
*For more information about a specific medicine or company in the report, please click on the provided link.
Acute Coronary Syndrome
Product Name Sponsor Indication Development Phase*
cangrelor The Medicines Company
Parsippany, NJ
acute coronary syndrome
(see also coronary artery disease,
thrombosis)
Phase I
www.themedicinescompany.com
CER-001
(recombinant HDL)
CerenisTherapeutics
Ann Arbor, MI
acute coronary syndrome
(see also lipid disorders)
Phase II
www.cerenis.com
gevokizumab Servier
Paris, France
XOMA
Berkeley, CA
acute coronary syndrome Phase II
www.servier.com
www.xoma.com
inclacumab
(RG1512)
Roche
Nutley, NJ
acute coronary syndrome
(see also heart attack, peripheral
vascular disease)
Phase II
www.roche.com
losmapimod
(p38 kinase inhibitor)
GlaxoSmithKline
Rsch. Triangle Park, NC
acute coronary syndrome Phase II
www.gsk.com
PF-06282999 Pfizer
New York, NY
acute coronary syndrome Phase I
www.pfizer.com
REG1
(anivamersen-pegnivacogin
intravenous)
Regado Biosciences
Basking Ridge, NJ
acute coronary syndrome Phase II
www.regadobio.com
RVX-208
(BET protein inhibitor)
Resverlogix
Calgary, Canada
acute coronary syndrome
(see also atherosclerosis)
Phase II
www.resverlogix.com
vorapaxar
(thrombin/PAR-1 receptor
antagonist)
Merck
Whitehouse Station, NJ
acute coronary syndrome
(prevention) (FastTrack)
(see also thrombosis)
Phase III
www.merck.com
VT-111a VironTherapeutics
Ontario, Canada
acute coronary syndrome Phase II
www.vironinc.com
Vytorin®
ezetimibe/simvastatin
fixed-dose combination
Merck
Whitehouse Station, NJ
acute coronary syndrome Phase III
www.merck.com
Xarelto®
rivaroxaban
Bayer HealthCare Pharmaceuticals
Wayne, NJ
Janssen Research Development
Raritan, NJ
acute coronary syndrome
(FastTrack)
(see also thrombosis, other)
application submitted
www.bayerpharma.com
www.janssenrnd.com
3. Medicines in Development Heart Disease and Stroke 2013 3
Medicines in Development for Heart Disease and Stroke
Adjunctive Therapies, Revascularization
Product Name Sponsor Indication Development Phase
ATryn®
antithrombin
(recombinant)
(Orphan Drug)
rEVO Biologics
Framingham, MA
heparin resistance in patients
undergoing coronary artery bypass
graft (CABG) surgery
(see also hypertension)
--------------------------------------
heparin resistance in patients
undergoing CABG surgery in
neonates
Phase III
www.gtc-bio.com
-----------------------------------------
Phase I
www.gtc-bio.com
CMX-2043
(proto-oncogene protein C
AKT modulator)
Ischemix
Maynard, MA
prevention of ischemia-reperfusion
injury in patients undergoing
percutaneous coronary
intervention (PCI)
Phase II
www.ischemix.com
myolimus-eluting stent Elixir Medical
Sunnyvale, CA
coronary artery restenosis
(prevention)
Phase I
www.elixirmedical.com
novolimus-eluting coronary stent Elixir Medical
Sunnyvale, CA
coronary artery restenosis
(prevention)
in clinical trials
www.elixirmedical.com
PRT-201 ProteonTherapeutics
Waltham, MA
vascular access for hemodialysis
(see also peripheral vascular
disease)
Phase II
www.proteontherapeutics.com
ranolazine Gilead Sciences
Foster City, CA
incomplete revascularization
in patients who undergo PCI
Phase III
www.gilead.com
sirolimus-eluting coronary stent REVA Medical
San Diego, CA
coronary artery restenosis in clinical trials
www.teamreva.com
sirolimus-eluting coronary stent Svelte Medical Systems
New Providence, NJ
ischemic heart disorder Phase I/II
www.sveltemedical.com
SRM003
(endothelial cell therapy)
Shire Pharmaceuticals
Wayne, PA
prevention of peripheral vascular
complications in patients
undergoing arteriovenous access
procedures for hemodialysis
Phase II
www.shire.com
4. Medicines in Development Heart Disease and Stroke 20134
Medicines in Development for Heart Disease and Stroke
Arrhythmia, Atrial Fibrillation
Product Name Sponsor Indication Development Phase
ARM036 ARMGO Pharma
Tarrytown, NY
catecholaminergic polymorphic
ventricular tachycardia type 1
(see also heart failure)
Phase II
www.armgo.com
azimilide Forest Laboratories
New York, NY
ventricular arrhythmia Phase III
www.frx.com
BMS-919373
(Ikur antagonist)
Bristol-Myers Squibb
Princeton, NJ
atrial fibrillation Phase I
www.bms.com
danegaptide
(ZP1609)
Zealand Pharma
Copenhagen, Denmark
atrial fibrillation Phase I
www.zealandpharma.com
GS-6615
(sodium channel antagonist)
Gilead Sciences
Foster City, CA
arrhythmia
(see also ischemic disorders)
Phase I
www.gilead.com
ISIS-CRPRx
(C-reactive protein inhibitor)
Isis Pharmaceuticals
Carlsbad, CA
paroxysmal atrial fibrillation Phase I
www.isispharm.com
OPC-108459 Otsuka America Pharmaceutical
Rockville, MD
paroxysmal and persistent atrial
fibrillation
Phase I
www.otsuka.com
ranolazine/dronedarone
fixed-dose combination
Gilead Sciences
Foster City, CA
paroxysmal atrial fibrillation Phase II
www.gilead.com
vanoxerine ChanRx
Cleveland, OH
atrial fibrillation Phase II
www.chanrx.com
vernakalant
(oral)
Cardiome Pharma
Vancouver, Canada
atrial fibrillation Phase II
www.cardiome.com
Atherosclerosis
Product Name Sponsor Indication Development Phase
ACP-501
(rhLCAT)
AlphaCore Pharma
Ann Arbor, MI
atherosclerosis
(see also coronary artery disease)
Phase I
www.alphacorepharma.com
ACZ885
(canakinumab)
Novartis Pharmaceuticals
East Hanover, NJ
atherosclerosis in patients with
type 2 diabetes
(see also other)
Phase II
www.novartis.com
anacetrapib
(MK-0859)
Merck
Whitehouse Station, NJ
atherosclerosis
(see also lipid disorders)
Phase III
www.merck.com
BMS-852927
(LXR modulator)
Bristol-Myers Squibb
Princeton, NJ
atherosclerosis Phase I
www.bms.com
5. Medicines in Development Heart Disease and Stroke 2013 5
Medicines in Development for Heart Disease and Stroke
Atherosclerosis
Product Name Sponsor Indication Development Phase
BMS-962476
(PCSK9 adnectin)
Bristol-Myers Squibb
Princeton, NJ
atherosclerosis Phase I
www.bms.com
CSL-112
(cholesterol modulator)
CSL
Victoria, Australia
atherosclerosis Phase II
www.csl.com.au
darapladib
(Lp-PLA2 inhibitor)
GlaxoSmithKline
Rsch. Triangle Park, NC
atherosclerosis Phase III
www.gsk.com
ISIS-APOARx
(antisense RNA inhibitor)
Isis Pharmaceuticals
Carlsbad, CA
atherosclerosis Phase I
www.isispharm.com
rilapladib GlaxoSmithKline
Rsch. Triangle Park, NC
atherosclerosis Phase II completed
www.gsk.com
RVX-208
(BET protein inhibitor)
Resverlogix
Calgary, Canada
atherosclerosis
(see also acute coronary syndrome)
Phase II
www.resverlogix.com
Tekturna®
aliskiren
Novartis Pharmaceuticals
East Hanover, NJ
atherosclerosis in coronary
artery disease
(see also heart failure,
hypertension)
Phase III
www.novartis.com
VB201
(IL-12 subunit p40 inhibitor)
VBLTherapeutics
Or Yehuda, Israel
atherosclerosis Phase II
www.vblrx.com
Coronary Artery Disease
Product Name Sponsor Indication Development Phase
ACP-501
(rhLCAT)
AlphaCore Pharma
Ann Arbor, MI
coronary artery disease
(see also atherosclerosis)
Phase I
www.alphacorepharma.com
cangrelor The Medicines Company
Parsippany, NJ
coronary artery disease in patients
requiring percutaneous coronary
intervention (PCI)
(see also acute coronary syndrome,
thrombosis)
Phase III
www.themedicinescompany.com
cardiovascular therapy
releasing balloon
(drug-coated Angiosculpt®)
AngioScore
Fremont, CA
coronary artery restenosis Phase II
www.angioscore.com
coronary artery disease
gene therapy
BioCardia
San Carlos, CA
coronary artery disease Phase II
www.biocardia.com
6. Medicines in Development Heart Disease and Stroke 20136
Medicines in Development for Heart Disease and Stroke
Coronary Artery Disease
Product Name Sponsor Indication Development Phase
fibroblast growth factor-1 (FGF-1) CardioVascular BioTherapeutics
Las Vegas, NV
severe coronary heart disease Phase II
www.cvbt.com
LT-1951
(oligo-L-arginine)
LumenTherapeutics
Menlo Park, CA
coronary artery restenosis Phase I/II
www.lumentherapeutics.com
pradigastat
(LCQ908)
Novartis Pharmaceuticals
East Hanover, NJ
coronary artery disease
(see also lipid disorders)
Phase II
www.novartis.com
RG7652
(PCSK9 protein inhibitor)
Genentech
South San Francisco, CA
coronary heart disease Phase II
www.gene.com
Heart Attack (Myocardial Infarction)
Product Name Sponsor Indication Development Phase
abciximab
(intracoronary infusion)
Atrium Medical Corporation
Hudson, NH
myocardial infarction
(new delivery system)
Phase III
www.atriummed.com
adipose-derived regenerative cell
(ADRC) therapy
CytoriTherapeutics
San Diego, CA
acute myocardial infarction
(see also ischemic disorders)
Phase II
www.cytori.com
AMR-001
(stem cell therapy)
Amorcyte (NeoStem)
New York, NY
myocardial infarction Phase I
www.neostem.com
BB3
(HGF mimetic)
Angion Biomedica
Uniondale, NY
acute myocardial infarction Phase II
www.angion.com
Brilinta®
ticagrelor
AstraZeneca
Wilmington, DE
prior myocardial infarction
(see also peripheral vascular
disease)
Phase III
www.astrazeneca.com
CAP-1002
(allogeneic cardiosphere-derived
stem cell therapy)
Capricor
Beverly Hills, CA
myocardial infarction Phase I/II
www.capricor.com
inclacumab Roche
Nutley, NJ
myocardial infarction
(see also acute coronary syndrome,
peripheral vascular disease)
Phase II
www.roche.com
ischemic tolerant allogeneic
mesenchymal stem cell therapy
Stemedica CellTechnologies
San Diego, CA
acute myocardial infarction Phase II
www.stemedica.com
mesenchymal stem cell therapy Mesoblast
New York, NY
heart attack
(see also heart failure)
Phase II
www.mesoblast.com
7. Medicines in Development Heart Disease and Stroke 2013 7
Medicines in Development for Heart Disease and Stroke
Heart Attack (Myocardial Infarction)
Product Name Sponsor Indication Development Phase
MultiStem®
stem cell therapy
Athersys
Cleveland, OH
acute myocardial infarction
(see also stroke)
Phase I completed
www.athersys.com
NEU 2000 GNT Pharma
Yongin, South Korea
myocardial infarction
(see also stroke)
Phase I completed
www.gntpharma.com
Prochymal®
remestemcel-L
OsirisTherapeutics
Columbia, MD
acute myocardial infarction Phase II
www.osiris.com
Heart Failure (Congestive Heart Failure)
Product Name Sponsor Indication Development Phase
albiglutide
(GSK716155)
GlaxoSmithKline
Rsch. Triangle Park, NC
heart failure Phase II completed
www.gsk.com
ANX-042
(natriuretic-peptide)
Anexon
Cambridge, MA
acute decompensated heart failure Phase I
www.anexonrx.com
ARM036 ARMGO Pharma
Tarrytown, NY
chronic heart failure
(see also arrhythmia)
Phase II
www.armgo.com
BAY 868050
(vasopressin receptor antagonist)
Bayer HealthCare Pharmaceuticals
Wayne, NJ
heart failure Phase I
www.bayerpharma.com
BAY 948862
(mineralocorticoid receptor [MR]
antagonist)
Bayer HealthCare Pharmaceuticals
Wayne, NJ
chronic heart failure Phase II
www.bayerpharma.com
BAY 1021189
(guanylate cyclase stimulant)
Bayer HealthCare Pharmaceuticals
Wayne, NJ
heart failure Phase I
www.bayerpharma.com
BAY 1067197
(partial adenosine A1 agonist)
Bayer HealthCare Pharmaceuticals
Wayne, NJ
heart failure Phase I
www.bayerpharma.com
BMS-986046
(PEG-relaxin)
Ambrx
La Jolla, CA
Bristol-Myers Squibb
Princeton, NJ
heart failure Phase I
www.ambrx.com
www.bms.com
bucindolol companion diagnostic ARCA biopharma
Broomfield, CO
LabCorp
Burlington, NC
heart failure (diagnosis) in clinical trials
www.arcabiopharma.com
www.labcorp.com
8. Medicines in Development Heart Disease and Stroke 20138
Medicines in Development for Heart Disease and Stroke
cenderitide NileTherapeutics
San Mateo, CA
acute heart failure
(FastTrack)
--------------------------------------
chronic heart failure
(FastTrack)
Phase II
www.nilethera.com
-----------------------------------------
Phase I
www.nilethera.com
CLP-1001
(sodium-potassium-chloride
symporter inhibitor)
SorbentTherapeutics
Sunnyvale, CA
congestive heart failure in patients
with chronic kidney disease
Phase II
www.sorbent.com
CXL-1020
(nitroxyl donor)
Cardioxyl Pharmaceuticals
Chapel Hill, NC
acute decompensated heart failure Phase II
www.cardioxyl.com
Gencaro™
bucindolol
ARCA biopharma
Broomfield, CO
genotype-defined heart failure
(FastTrack)
application submitted
www.arcabiopharma.com
GGF2
(recombinant neuregulin-1)
AcordaTherapeutics
Ardsley, NY
heart failure
(FastTrack)
Phase I
www.acorda.com
GSK2849466
(selective androgen receptor
modulator)
GlaxoSmithKline
Rsch. Triangle Park, NC
heart failure Phase I
www.gsk.com
JNJ-39588146 Janssen Research Development
Raritan, NJ
heart failure Phase II
www.janssenrnd.com
JVS-100
(stromal cell-derived factor-1)
JuventasTherapeutics
Cleveland, OH
heart failure
(see also ischemic disorders)
Phase II
www.juventasinc.com
LCZ696
(ARB/NEP inhibitor)
Novartis Pharmaceuticals
East Hanover, NJ
heart failure
(see also hypertension)
Phase III
www.novartis.com
mesenchymal stem cell therapy Mesoblast
New York, NY
congestive heart failure
(see also heart attack)
Phase II
www.mesoblast.com
Mydicar®
SERCA 2a gene therapy
Celladon
San Diego, CA
advanced (Class III/IV) heart failure
(FastTrack)
--------------------------------------
chronic heart failure
Phase II
www.celladon.net
-----------------------------------------
Phase II completed
www.celladon.net
MyoCell®
stem cell therapy
Bioheart
Sunrise, FL
heart failure Phase II/III
www.bioheartinc.com
MyoCell® SDF1
muscle stem cell therapy
(second-generation)
BioHeart
Sunrise, FL
congestive heart post myocardial
infarction
Phase I
www.bioheartinc.com
Heart Failure (Congestive Heart Failure)
Product Name Sponsor Indication Development Phase
9. Medicines in Development Heart Disease and Stroke 2013 9
Medicines in Development for Heart Disease and Stroke
omecamtiv mecarbil Amgen
Thousand Oaks, CA
Cytokinetics
South San Francisco, CA
heart failure Phase II
www.amgen.com
www.cytokinetics.com
PL-3994
(natriuretic-peptide)
PalatinTechnologies
Cranbury, NJ
congestive heart failure
(see also hypertension)
Phase II
www.palatin.com
recombinant human
neuregulin-1 beta
Zensun
Shanghai, China
stable chronic heart failure Phase II
www.zensun.com
serelaxin
(RLX030)
Novartis Pharmaceuticals
East Hanover, NJ
acute heart failure
(FastTrack)
--------------------------------------
chronic heart failure
Phase III
www.novartis.com
-----------------------------------------
Phase II
www.novartis.com
Tekturna®
aliskiren
Novartis Pharmaceuticals
East Hanover, NJ
acute decompensated heart failure
(see also atherosclerosis,
hypertension)
--------------------------------------
reduction of cardiovascular death/
hospitalization in chronic heart
failure patients
Phase III completed
www.novartis.com
-----------------------------------------
Phase III
www.novartis.com
TRV027
(angiotensin type 1 receptor
antagonist)
Forest Laboratories
New York, NY
Trevena
King of Prussia, PA
acute heart failure
(late-stage disease)
Phase II
www.frx.com
www.trevenainc.com
ularitide Cardiorentis
Zug, Switzerland
acute heart failure Phase III
www.cardiorentis.ch
urocortin 2 Neurocrine Biosciences
San Diego, CA
congestive heart failure Phase II
www.neurocrine.com
Hypertension (High Blood Pressure)
Product Name Sponsor Indication Development Phase
AHU377
(neprilysin inhibitor)
Novartis Pharmaceuticals
East Hanover, NJ
essential hypertension Phase II
www.novartis.com
ATryn®
antithrombin
(recombinant)
rEVO Biologics
Framingham, MA
preeclampsia
(see also adjunctive therapies)
Phase II
www.gtc-bio.com
Heart Failure (Congestive Heart Failure)
Product Name Sponsor Indication Development Phase
10. Medicines in Development Heart Disease and Stroke 201310
Medicines in Development for Heart Disease and Stroke
candesartan cilexetil/nifedipine
fixed-dose combination
Bayer HealthCare Pharmaceuticals
Wayne, NJ
essential hypertension Phase III
www.bayerpharma.com
digoxin immune Fab (DIF)
(Orphan Drug)
Glenveigh Pharmaceuticals
Chattanooga, TN
preeclampsia
(FastTrack)
Phase II
www.glenveigh.com
Edarbi®
azilsartan medoxomil
Takeda Pharmaceuticals
Deerfield, IL
hypertension (pediatric) Phase I
www.takeda.com
GSK2944406
(amlodipine/losartan
fixed-dose combination)
GlaxoSmithKline
Rsch. Triangle Park, NC
hypertension Phase I
www.gsk.com
HL-040XC
(atorvastatin/losartan
fixed-dose combination)
HanAll Biopharma
Seoul, South Korea
hypertension
(see also lipid disorders)
Phase II
www.hanall.co.kr
HT-101
(amiloride/spironolactone)
HemodynamicTherapeutics
Durham, NC
obesity-related resistant
hypertension
Phase II
www.hemodynamictx.com
KD027
(PDE5 inhibitor)
Kadmon Pharmaceuticals
Warrendale, PA
hypertension Phase I
www.kadmon.com
LCZ696
(ARB/NEP inhibitor)
Novartis Pharmaceuticals
East Hanover, NJ
essential hypertension
(see also heart failure)
Phase II
www.novartis.com
MK-7145 Merck
Whitehouse Station, NJ
hypertension Phase I completed
www.merck.com
MK-8150 Merck
Whitehouse Station, NJ
hypertension Phase I
www.merck.com
nebivolol/valsartan
fixed-dose combination
Forest Laboratories
New York, NY
essential hypertension Phase III
www.frx.com
PB1046
(Vasomera)
PhaseBio Pharmaceuticals
Malvern, PA
essential hypertension Phase I/II
www.phasebio.com
perindopril/amlodipine
fixed-dose combination
XOMA
Berkeley, CA
hypertension Phase III
www.xoma.com
PL-3994
(natriuretic-peptide)
PalatinTechnologies
Cranbury, NJ
hypertension
(see also heart failure)
Phase II
www.palatin.com
Tekturna®
aliskiren
Novartis Pharmaceuticals
East Hanover, NJ
hypertension in children age
6-17 years of age
(see also atherosclerosis, heart
failure)
Phase III
www.novartis.com
Hypertension (High Blood Pressure)
Product Name Sponsor Indication Development Phase
11. Medicines in Development Heart Disease and Stroke 2013 11
Medicines in Development for Heart Disease and Stroke
99m-Tc-EC-G CellPoint
Centennial, CO
myocardial ischemia (diagnosis) Phase II
www.cellpointweb.com
Amiscan™
Tc-99m-glucarate
MolecularTargetingTechnologies
West Chester, PA
acute coronary syndrome
(diagnosis)
Phase II
www.mtarget.com
BFPET™
(myocardial perfusion imaging
agent)
FluoroPharma
Montclair, NJ
coronary artery disease (diagnosis) Phase I
www.fluoropharma.com
CardioPET™
cardiac PET imaging agent
FluoroPharma
Montclair, NJ
coronary artery disease (diagnosis) Phase II
www.fluoropharma.com
fluorine-18-ML-10 IBA Molecular US
Dulles, VA
stroke (diagnosis) Phase II
www.iba-molecular.com
flurpiridaz F-18 Lantheus Medical Imaging
N. Billerica, MA
coronary artery disease (diagnosis) Phase III
www.lantheus.com
Imagify™
perflubutane
Acusphere
Lexington, MA
coronary artery disease (diagnosis) Phase III
www.acusphere.com
LMI 1195
(PET imaging agent)
Lantheus Medical Imaging
N. Billerica, MA
heart failure (diagnosis) Phase I
www.lantheus.com
ThromboView®
Tc-99m 3B6/22 anti-fibrin mAb
AGEN Biomedical
(Agenix)
Victoria, Australia
deep vein thrombosis (diagnosis),
pulmonary thrombosis (diagnosis)
Phase II
www.agenix.com
Ischemic Disorders
Product Name Sponsor Indication Development Phase
ACY001
(autologous AC133+ selected adult
bone marrow-derived stem cell
therapy)
Compass Biomedical
Cleveland, OH
chronic coronary ischemia Phase I
www.compassbiomedical.com
adipose-derived regenerative cell
(ADRC) therapy
CytoriTherapeutics
San Diego, CA
chronic myocardial ischemia
(see also heart attack)
Phase I/II
www.cytori.com
Imaging Agents
Product Name Sponsor Indication Development Phase
12. Medicines in Development Heart Disease and Stroke 201312
Medicines in Development for Heart Disease and Stroke
Ischemic Disorders
Product Name Sponsor Indication Development Phase
ALD-201
(bone marrow-derived adult
stem cell therapy)
Cytomedix
Gaithersburg, MD
ischemic heart failure Phase I
www.cytomedix.com
ALD-301
(bone marrow-derived adult
stem cell therapy)
Cytomedix
Gaithersburg, MD
critical limb ischemia Phase I/II
www.cytomedix.com
ALO212
(stem cell therapy)
Compass Biomedical
Cleveland, OH
critical limb ischemia Phase I
www.compassbiomedical.com
allogeneic/autologous adult
mesenchymal stem cell therapy
BioCardia
San Carlos, CA
ischemic heart disorder Phase I/II
www.biocardia.com
autologous stem cell therapy Baxter Healthcare
Deerfield, IL
myocardial ischemia Phase III
www.baxter.com
Bendavia™
mitochondrial-targeting
short peptide
Stealth Peptides
Newtown Centre, MA
ischemic reperfusion in acute
myocardial infarction and heart
failure
Phase II
www.stealthpeptides.com
Collategene®
beperminogene perplasmid
(HGF plasmid)
AnGes
Bethesda, MD
MitsubishiTanabe Pharma America
Jersey City, NJ
------------------------------------------------
AnGes
Bethesda, MD
critical limb ischemia
(FastTrack)
--------------------------------------
ischemic heart disorder
Phase III
www.anges-mg.com
www.mt-pharma-america.com
-----------------------------------------
Phase I
www.anges-mg.com
ERC-124
(endometrial blood stem cell
therapy)
MediStem
San Diego, CA
critical limb ischemia in clinical trials
www.medisteminc.com
Generx®
alferminogene tadenovec
(Ad5FGF-4)
CardiumTherapeutics
San Diego, CA
myocardial ischemia
(FastTrack)
Phase III
www.cardiumthx.com
GS-6615
(sodium channel antagonist)
Gilead Sciences
Foster City, CA
ischemic heart disease
(see also arrhythmia)
Phase I
www.gilead.com
JVS-100
(stromal cell-derived factor-1)
JuventasTherapeutics
Cleveland, OH
critical limb ischemia
(see also heart failure)
Phase II
www.juventasinc.com
MESENDO
combination autologous
stem cell therapy
TCA CellularTherapy
Covington, LA
coronary ischemia, lower limb
ischemia
Phase II
www.tcacellulartherapy.com
13. Medicines in Development Heart Disease and Stroke 2013 13
Medicines in Development for Heart Disease and Stroke
Ischemic Disorders
Product Name Sponsor Indication Development Phase
MP4OX Sangart
San Diego, CA
ischemia Phase II completed
www.sangart.com
PLX-PAD
(stem cell therapy)
PluristemTherapeutics
Haifa, Israel
critical limb ischemia
(see also peripheral vascular
disease)
Phase I
www.pluristem.com
VM202
(modified hepatocyte growth
factor gene therapy)
ViroMed
Seoul, South Korea
myocardial ischemia
(see also peripheral vascular
disease)
Phase I
www.viromed.co.kr
Lipid Disorders
Product Name Sponsor Indication Development Phase
ALN-PCS
(PCSK9 protein inhibitor)
Alnylam Pharmaceuticals
Cambridge, MA
The Medicines Company
Parsippany, NJ
hypercholesterolemia Phase I
www.alnylam.com
www.themedicinescompany.com
AMG 145
(PCSK9 inhibitor mAb)
Amgen
Thousand Oaks, CA
heterozygous familial
hypercholesterolemia,
hypercholesterolemia
Phase I
www.amgen.com
AMR-102
(icosapent ethyl/statin
fixed-dose combination)
Amarin
Bedminster, NJ
hyperlipidemia Phase I
www.amarincorp.com
anacetrapib
(MK-0859)
Merck
Whitehouse Station, NJ
heterozygous familial
hypercholesterolemia,
hypercholesterolemia
(see also atherosclerosis)
Phase III
www.merck.com
ARI-3037MO
(niacin analog)
Arisaph Pharmaceuticals
Boston, MA
dyslipidemia Phase I
www.arisaph.com
CAT-2003
(eicosapentaenoic acid/niacin)
Catabasis Pharmaceuticals
Cambridge, MA
severe hypertriglyceridemia Phase I
www.catabasis.com
14. Medicines in Development Heart Disease and Stroke 201314
Medicines in Development for Heart Disease and Stroke
Lipid Disorders
Product Name Sponsor Indication Development Phase
CER-001
(recombinant HDL)
CerenisTherapeutics
Ann Arbor, MI
homozygous familial
hypercholesterolemia
(see also acute coronary syndrome)
Phase II
www.cerenis.com
CER-002
(PPAR-delta agonist)
CerenisTherapeutics
Ann Arbor, MI
dyslipidemia with low HDL Phase I
www.cerenis.com
CER-627
(low flushing niacin)
CerenisTherapeutics
Ann Arbor, MI
dyslipidemia with low HDL Phase I
www.cerenis.com
diazoxide choline
controlled-release
Essentialis
Carlsbad, CA
hypertriglyceridemia Phase II
www.essentialistherapeutics.com
Epanova™
eicosapentaenoic acid/
docosahexaenoic acid
Omthera Pharmaceuticals
Princeton, NJ
hypertriglyceridemia
(combination therapy)
--------------------------------------
hypertriglyceridemia
(monotherapy)
Phase III
www.omthera.com
-----------------------------------------
Phase II/III
www.omthera.com
ETC-1002
(ATP citrate [pro-S]-lyase inhibitor/
adenylate kinase stimulator)
EsperionTherapeutics
Plymouth, MI
dyslipidemia Phase II
www.esperion.com
fenofibrate/simvastatin
fixed-dose combination
AbbVie
North Chicago, IL
hyperlipidemia Phase III
www.abbvie.com
GSK1292263
(bombesin receptor agonist)
GlaxoSmithKline
Rsch. Triangle Park, NC
hyperlipidemia Phase II completed
www.gsk.com
HL-040XC
(atorvastatin/losartan
fixed-dose combination)
HanAll Biopharma
Seoul, South Korea
hyperlipidemia
(see also hypertension)
Phase II
www.hanall.co.kr
HPP593
(PPAR-delta agonist)
High Point Pharmaceuticals
High Point, NC
dyslipidemia Phase I
www.highpointpharma.com
ISIS-APOCIIIRx
(apolipoprotein C-III inhibitor)
Isis Pharmaceuticals
Carlsbad, CA
hypertriglyceridemia Phase I
www.isispharm.com
K-877
(PPAR-alpha agonist)
Kowa Pharmaceuticals America
Montgomery, AL
dyslipidemia Phase I
www.kowa.co.jp
KD026
(MTP inhibitor)
Kadmon Pharmaceuticals
Warrendale, PA
dyslipidemia Phase II
www.kadmon.com
Kynamro®
mipomersen
Genzyme
Cambridge, MA
severe homozygous familial
hypercholesterolemia
Phase III
www.genzyme.com
15. Medicines in Development Heart Disease and Stroke 2013 15
Medicines in Development for Heart Disease and Stroke
Lipid Disorders
Product Name Sponsor Indication Development Phase
MBX-8025
(PPAR-delta agonist)
Metabolex
Hayward, CA
dyslipidemia Phase II
www.metabolex.com
MGL-3196
(liver-directed thyroid hormone
receptor-ß agonist)
Madrigal Pharmaceuticals
Fort Washington, PA
dyslipidemia,
hypercholesterolemia
Phase I
www.madrigalpharma.com
pradigastat
(LCQ908)
Novartis Pharmaceuticals
East Hanover, NJ
familial chylomicronemia
syndrome
(see also coronary artery disease)
--------------------------------------
hypertriglyceridemia
Phase III
www.novartis.com
-----------------------------------------
Phase II
www.novartis.com
RN316
(PF-04950615)
Pfizer
New York, NY
hypercholesterolemia Phase II
www.pfizer.com
RN317
(PF-05335810)
Pfizer
New York, NY
hypercholesterolemia Phase I
www.pfizer.com
SAR236553
(anti-PCSK-9 mAb)
Regeneron Pharmaceuticals
Tarrytown, NY
Sanofi US
Bridgewater, NJ
heterozygous familial
hypercholesterolemia,
hypercholesterolemia
Phase III
www.regeneron.com
www.sanofi.com
SCH-900271 Merck
Whitehouse Station, NJ
hyperlipidemia Phase II completed
www.merck.com
TAP311 Novartis Pharmaceuticals
East Hanover, NJ
dyslipidemia Phase I completed
www.novartis.com
XZK-monascus
(HMG-CoA reductase inhibitor)
Beijing Peking University
WBL Biotech
Beijing, China
hyperlipidemia Phase II
Peripheral Vascular Disease
Product Name Sponsor Indication Development Phase
Benlysta®
belimumab
GlaxoSmithKline
Rsch. Triangle Park, NC
vasculitis Phase III
www.gsk.com
Brilinta®
ticagrelor
AstraZeneca
Wilmington, DE
peripheral arterial disease
(see also heart attack)
Phase III
www.astrazeneca.com
16. Medicines in Development Heart Disease and Stroke 201316
Medicines in Development for Heart Disease and Stroke
Peripheral Vascular Disease
Product Name Sponsor Indication Development Phase
defibrotide Gentium
Villa Guardia, Italy
Sigma-Tau Pharmaceuticals
Gaithersburg, MD
treatment of hepatic venous
occlusive disease after stem cell
transplantation
Phase III
www.gentium.com
www.sigmatau.com
human plasma-derived fibrinolysin Grifols
Barcelona, Spain
peripheral vascular occlusive
disease
Phase I
www.grifols.com
inclacumab Roche
Nutley, NJ
prevention of saphenous vein
graft disease
(see also acute coronary syndrome,
heart attack)
Phase II
www.roche.com
K-134
(PDE3 inhibitor)
Kowa Pharmaceuticals America
Montgomery, AL
arteriosclerosis obliterans Phase II
www.kowa.co.jp
MABp1
(IL-1α mAb)
XBiotech
Austin, TX
vascular restenosis
(FastTrack)
Phase II
www.xbiotech.com
MultiGeneAngio
peripheral arterial disease cell
therapy
MultiGeneVascular Systems
Nesher, Israel
peripheral arterial disease Phase I/II
www.mgvs.co.il
paclitaxel coated balloon Lutonix
Minneapolis, MN
peripheral arterial disease Phase II
www.lutonix.com
paclitaxel-eluting balloon catheter MEDRAD
(Bayer HealthCare)
Warrendale, PA
stenotic peripheral artery lesions application submitted
www.medrad.com
PLX-PAD
(stem cell therapy)
PluristemTherapeutics
Haifa, Israel
intermittent claudication
(see also ischemic disorders)
Phase II
www.pluristem.com
PRT-201 ProteonTherapeutics
Waltham, MA
peripheral arterial disease
(see also adjunctive therapies)
Phase I
www.proteontherapeutics.com
TSC
(trans-sodium crocetinate)
Diffusion Pharmaceuticals
Charlottesville, VA
peripheral arterial disease Phase II completed
www.diffusionpharma.com
TV1001
(sodium nitrite oral)
Theravasc
Cleveland, OH
peripheral arterial disease Phase II
www.theravasc.com
VM202
(modified hepatocyte growth
factor gene therapy)
ViroMed
Seoul, South Korea
peripheral arterial disease
(see also ischemic disorders)
Phase II
www.viromed.co.kr
17. Medicines in Development Heart Disease and Stroke 2013 17
Medicines in Development for Heart Disease and Stroke
Pulmonary Vascular Disease
Product Name Sponsor Indication Development Phase
Adcirca™
tadalafil
Eli Lilly
Indianapolis, IN
pulmonary hypertension
(pediatric)
Phase I/II
www.lilly.com
APD811
(prostanoid receptor agonist)
Arena Pharmaceuticals
San Diego, CA
pulmonary arterial hypertension Phase I
www.arenapharm.com
beraprost 314d
(single isomer version)
UnitedTherapeutics
Silver Spring, MD
pulmonary arterial hypertension Phase II
www.unither.com
DA-8159
(PDE5 inhibitor)
Dong-A Pharmaceutical
Seoul, South Korea
pulmonary arterial hypertension Phase II
en.donga.co.kr
INOmax®
nitric oxide inhalation
(Orphan Drug)
Ikaria
Hampton, NJ
pulmonary arterial hypertension Phase I
www.ikaria.com
Letairis®
ambrisentan
(Orphan Drug)
Gilead Sciences
Foster City, CA
pulmonary hypertension
(pediatric)
Phase II
www.gilead.com
macitentan
(endothelin A receptor antagonist)
(Orphan Drug)
Actelion Pharmaceuticals
South San Francisco, CA
pulmonary arterial hypertension application submitted
www.actelion.com
nitric oxide inhalation GeNO
Waltham, MA
pulmonary arterial hypertension Phase II
www.genollc.com
nitrite inhalation
(Orphan Drug)
Aires Pharmaceuticals
San Diego, CA
pulmonary arterial hypertension Phase II
www.airespharma.com
riociguat Bayer HealthCare Pharmaceuticals
Wayne, NJ
chronic thromboembolic
pulmonary hypertension
application submitted
www.bayerpharma.com
SAR407899
(Rho kinase inhibitor)
Sanofi US
Bridgewater, NJ
pulmonary hypertension Phase I
www.sanofi.com
selexipag Actelion Pharmaceuticals
South San Francisco, CA
pulmonary arterial hypertension Phase III
www.actelion.com
Tracleer®
bosentan
Actelion Pharmaceuticals
South San Francisco, CA
pulmonary arterial hypertension
(pediatric)
Phase III
www.actelion.com
treprostinil (oral)
(sustained-release)
UnitedTherapeutics
Silver Spring, MD
pulmonary arterial hypertension application submitted
www.unither.com
18. Medicines in Development Heart Disease and Stroke 201318
Medicines in Development for Heart Disease and Stroke
Stroke
Product Name Sponsor Indication Development Phase
3K3A-APC
(recombinant human
activated protein C)
ZZ Biotech
Houston, TX
stroke Phase I
www.zzbiotech.com
ALD-401
(bone marrow-derived adult
stem cell therapy)
Cytomedix
Gaithersburg, MD
stroke Phase II
www.cytomedix.com
allogeneic mesenchymal
bone marrow cell therapy
Stemedica CellTechnologies
San Diego, CA
ischemic stroke Phase I/II
www.stemedica.com
Ampyra™
fampridine sustained-release
AcordaTherapeutics
Ardsley, NY
post-stroke deficits Phase II
www.acorda.com
betrixaban Portola Pharmaceuticals
South San Francisco, CA
stroke (prevention) in patients
with atrial fibrillation
(see also thrombosis)
Phase II
www.portola.com
CNTO-0007
(cell therapy)
Janssen Research Development
Raritan, NJ
ischemic stroke Phase I
www.janssenrnd.com
desmoteplase Lundbeck
Deerfield, IL
ischemic stroke
(FastTrack)
Phase III
www.lundbeck.com
GM602
(nervous system modulator)
Genervon Biopharmaceuticals
Pasadena, CA
acute ischemic stroke Phase II
www.genervon.com
GSK249320
(myelin-associated glycoprotein
mAb)
GlaxoSmithKline
Rsch. Triangle Park, NC
stroke Phase II
www.gsk.com
metamfetamine Sinapis Pharma
Jacksonville, FL
stroke Phase I completed
www.sinapispharma.com
MP-124
(PARP inhibitor)
MitsubishiTanabe Pharma America
Jersey City, NJ
acute ischemic stroke Phase I
www.mt-pharma.co.jp
MultiStem®
stem cell therapy
Athersys
Cleveland, OH
stroke
(see also heart attack)
Phase II
www.athersys.com
NA-1
(signal transduction
pathway inhibitor)
NoNO Inc
Toronto, Canada
acute ischemic stroke Phase II completed
www.nonoinc.ca
NEU 2000 GNT Pharma
Yongin, South Korea
stroke
(see also heart attack)
Phase I completed
www.gntpharma.com
19. Medicines in Development Heart Disease and Stroke 2013 19
Medicines in Development for Heart Disease and Stroke
Stroke
Product Name Sponsor Indication Development Phase
PF-03049423 Pfizer
New York, NY
ischemic stroke Phase II
www.pfizer.com
RP-1127
(NCCA-ATP channel inhibitor)
Remedy Pharmaceuticals
New York, NY
stroke Phase II
www.remedypharmaceuticals.com
SAR126119
(TAFIa inhibitor)
Sanofi US
Bridgewater, NJ
acute ischemic stroke Phase I
www.sanofi.com
SB623
(stem cell therapy)
SanBio
Mountain View, CA
stroke Phase I/II
www.san-bio.com
TS01
(recombinant complement
C1-inactivator-protein)
Thrombolytic Science International
Cambridge, MA
stroke Phase I
www.tsillc.net
Thrombosis
Product Name Sponsor Indication Development Phase
betrixaban Portola Pharmaceuticals
South San Francisco, CA
thromboembolism (prevention)
(see also stroke)
Phase III
www.portola.com
cangrelor The Medicines Company
Parsippany, NJ
prevention of coronary thrombosis
prior to coronary artery bypass
graft (CABG)
(see also acute coronary syndrome,
coronary artery disease)
Phase II
www.themedicinescompany.com
clopidogrel intravenous
(MDCO-157)
The Medicines Company
Parsippany, NJ
coronary thrombosis (prevention) Phase III
www.themedicinescompany.com
desirudin IV
(intravenous)
Canyon Pharmaceuticals
Columbia, MD
intravenous bolus-only
administration in patients
undergoing coronary artery
stenting
in clinical trials
www.canyonpharma.com
edoxaban
(oral factor Xa inhibitor)
Daiichi Sankyo
Parsippany, NJ
venous thromboembolism
(prevention), embolism/stroke
in patients with atrial fibrillation
(prevention)
Phase III
www.daiichisankyo.com
20. Medicines in Development Heart Disease and Stroke 201320
Medicines in Development for Heart Disease and Stroke
Eliquis®
apixaban
Bristol-Myers Squibb
Princeton, NJ
Pfizer
New York, NY
prevention of venous
thromboembolism, treatment of
venous thrombosis
Phase III
www.bms.com
www.pfizer.com
GCC-4401 Green Cross
Yongin, South Korea
coronary thrombosis Phase I completed
www.greencross.co.kr
Iprivask®
desirudin SC
(subcutaneous)
Canyon Pharmaceuticals
Columbia, MD
thromboembolism in patients with
suspected heparin-induced
thrombocytopenia (prevention and
treatment), venous
thromboembolism in high-risk
surgical and medical patients
(prevention)
in clinical trials
www.canyonpharma.com
ISIS-FXIRx
(factor XI inhibitor)
Isis Pharmaceuticals
Carlsbad, CA
venous thromboembolism Phase I
www.isispharm.com
Pradaxa®
dabigatran etexilate
Boehringer Ingelheim Pharmaceuticals
Ridgefield, CT
acute treatment and reduction
in the risk of recurrence of deep
venous thrombosis and pulmonary
embolism
Phase III
www.boehringer-ingelheim.com
REG2
(pegnivacogin subcutaneous)
Regado Biosciences
Basking Ridge, NJ
venous thrombosis Phase I
www.regadobio.com
vorapaxar
(thrombin/PAR-1 receptor
antagonist)
Merck
Whitehouse Station, NJ
arterial thrombosis (prevention)
(FastTrack)
(see also acute coronary syndrome)
Phase III
www.merck.com
Xarelto®
rivaroxaban
Bayer HealthCare Pharmaceuticals
Wayne, NJ
Janssen Research Development
Raritan, NJ
venous thromboembolism
(pediatric)
(see also acute coronary syndrome,
other)
Phase I
www.bayerpharma.com
www.janssenrnd.com
Other
Product Name Sponsor Indication Development Phase
ACZ885
(canakinumab)
Novartis Pharmaceuticals
East Hanover, NJ
secondary prevention of
cardiovascular events
(see also atherosclerosis)
Phase III
www.novartis.com
Thrombosis
Product Name Sponsor Indication Development Phase
21. Medicines in Development Heart Disease and Stroke 2013 21
Medicines in Development for Heart Disease and Stroke
aleglitazar
(PPAR alpha/gamma co-agonist)
Roche
Nutley, NJ
prevention of cardiovascular
disorders in patients with type 2
diabetes
Phase III
www.roche.com
ALN-TTRsc
(prealbumin inhibitor)
Alnylam Pharmaceuticals
Cambridge, MA
familial amyloidotic
cardiomyopathy
Phase I
www.alnylam.com
Bydureon®
exenatide extended-release
for injectable suspension
AstraZeneca
Wilmington, DE
Bristol-Myers Squibb
Princeton, NJ
cardiovascular outcomes Phase III
www.astrazeneca.com
www.bms.com
caplacizumab
(Orphan Drug)
Ablynx
Ghent, Belguim
thrombotic thrombocytopenic
purpura
Phase II
www.ablynx.com
CardiaPill®
aspirin/lisinopril/lovastatin
CardioPharma
Wilmington, NC
cardiovascular disorders in clinical trials
www.cardio-pharma.com
cardiovascular disease medicine Eli Lilly
Indianapolis, IN
cardiovascular disease Phase I
www.lilly.com
droxidopa
(Orphan Drug)
ChelseaTherapeutics
Charlotte, NC
orthostatic hypotension
(FastTrack)
application submitted
www.chelseatherapeutics.com
evacetrapib Eli Lilly
Indianapolis, IN
prevention of cardiovascular events
in high-risk vascular disease
Phase III
www.lilly.com
Forxiga®
dapagliflozin
AstraZeneca
Wilmington, DE
Bristol-Myers Squibb
Princeton, NJ
cardiovascular outcomes Phase III
www.astrazeneca.com
www.bms.com
ixmyelocel-T
(stem cell therapy)
Aastrom Biosciences
Ann Arbor, MI
dilated cardiomyopathy Phase II
www.aastrom.com
MEK162
(MEK inhibitor)
Novartis Pharmaceuticals
East Hanover, NJ
Noonan syndrome hypertrophic
cardiomyopathy
Phase II
www.novartis.com
Onglyza®
saxagliptin
AstraZeneca
Wilmington, DE
Bristol-Myers Squibb
Princeton, NJ
cardiovascular outcomes Phase III
www.astrazeneca.com
www.bms.com
PA8140
(aspirin 81mg/omeprazole 40mg)
POZEN
Chapel Hill, NC
secondary prevention of
cardiovascular and cerebrovascular
disease in patients at risk for
aspirin-induced ulcers
application submitted
www.pozen.com
Other
Product Name Sponsor Indication Development Phase
23. Medicines in Development Heart Disease and Stroke 2013 23
Glossary
adjunctive therapy—Auxiliary treatment
that is secondary to the main treatment.
angina pectoris—Chest pain, usually
caused by “myocardial ischemia,” a low
supply of oxygen to the heart muscle
resulting from hardening, narrowing,
and sometimes spasm of the coronary
arteries.
application submitted—An application
for marketing has been submitted by the
company to the Food and Drug Admin-
istration (FDA).
arrhythmia—Abnormal heart rhythm,
usually detected by an electrocardio-
gram. Arrhythmias can be caused by
several factors, such as coronary artery
disease, heart valve problems or hyper-
thyroidism.
arteriosclerosis obliterans—An alter-
native term for peripheral vascular
disease.
atherosclerosis—A common disease
in which deposits of plaque containing
calcium and fatty substances, such as
cholesterol, are formed within the inner
layers of the arteries. It is a condition
that progresses over decades, chiefly
affecting the arteries of the heart, brain
and extremities. Its complications include
heart attacks and strokes.
atrial fibrillation—Very fast electrical
discharge patterns that make the heart’s
atria contract extremely rapidly, which
causes the ventricles to contract faster
and less efficiently than normal. As a
result, inadequate amounts of blood are
pumped out of the heart, blood pressure
falls, and heart failure may occur.
bypass graft—A vein or artery graft that
bypasses blockage in an artery.
cardiomyopathy—A type of heart
disease in which the heart muscle is
abnormally enlarged, thickened and/or
stiffened. As a result, the heart muscle’s
ability to pump blood is usually impaired.
cardiovascular—Of or relating to the
heart and blood vessels.
coronary artery disease—A condition
caused by atherosclerosis of the arteries
that supply the heart.
critical limb ischemia (CLI)—CLI is a
severe obstruction of the arteries that
seriously decreases blood flow to the
extremities (arms, hands, legs, feet) and
has progressed to the point of severe
pain and even skin ulcers or sores. The
pain, called “rest pain,” caused by CLI
can wake up a person at night. CLI is
a very severe condition of peripheral
arterial disease and needs comprehen-
sive treatment by a vascular surgeon or
specialist.
deep vein thrombosis—Blood clotting
within the deep-lying veins, often in the
legs or pelvic veins.
dyslipidemia—A condition marked by
abnormal concentrations of lipids or
lipoproteins in the blood.
embolism—The obstruction of a blood
vessel by a foreign substance or a blood
clot. Foreign substances that can cause
embolism include an air bubble, amni-
otic fluid, a globule of fat, a clump of
bacteria, chemicals, and drugs. Blood
clots are the most common cause of
embolism. The term “embolus” refers to
the substance or clot that is obstructing
the blood vessel, while “embolism” refers
to the process by which that happens.
Fast Track—A process designed to
facilitate the development and expedite
the review of drugs to treat serious
diseases and fill an unmet medical need.
The status is assigned by the U.S. Food
and Drug Administration. The purpose
is to get important new drugs to the
patient earlier. Fast Track addresses a
broad range of serious diseases. Gener-
ally, determining factors include whether
the drug will have an impact on such
factors as survival, day-to-day function-
ing, or the likelihood that the disease, if
left untreated, will progress from a less
severe condition to a more serious one.
Filling an unmet medical need is defined
as providing a therapy where none exists
or providing a therapy which may be
potentially superior to existing therapy.
Once a drug receives Fast Track designa-
tion, early and frequent communication
between the FDA and a drug company is
encouraged throughout the entire drug
development and review process. The
frequency of communication assures that
questions and issues are resolved quickly,
often leading to earlier drug approval
and access by patients.
genotype—The genetic constitution
(genome) of a cell, an individual, or an
organism. The genotype of a person is
her or his genetic makeup. It can pertain
to all genes or to a specific gene.
HDL—High-density lipoprotein, some-
times called “good cholesterol.”
heart attack (myocardial infarction)—A
part of the heart muscle (myocardium)
dies as a result of blood and oxygen
deprivation.
heart failure—The end result of many
different types of heart disease. The
heart cannot pump blood out normally.
This results in congestion (water and salt
retention) in the lungs, swelling in the
extremities, and reduced blood flow to
body tissues.
hemodialysis—A medical procedure that
uses a special machine (a dialysis ma-
chine) to filter waste products from the
blood and to restore normal constituents
to it.
hypercholesterolemia—The presence of
an abnormally large amount of cho-
lesterol in the cells and plasma of the
circulating blood.
hyperlipidemia—A group of metabolic
disorders characterized by high levels of
lipids (fatty substances, including choles-
terol) in the blood. Hyperlipidemia is a
risk factor for accelerated atherosclerosis
and premature heart attacks.
hypertension (high blood pressure)—
Persistent elevation of blood pressure
above the normal range while the heart
is in systolic (contracting) or diastolic
(relaxed) mode. Uncontrolled, chronic
24. Medicines in Development Heart Disease and Stroke 201324
Glossary
hypertension strains the heart, damages
arteries and creates a greater risk of
heart attack, stroke and kidney problems.
hypertriglyceridemia—An elevated
triglyceride concentration in the blood.
hypotension—A sudden fall in blood
pressure. It may be caused by hypovo-
lemia resulting from the excessive use
of diuretics, vasodilators, or other types
of drugs, dehydration, or prolonged bed
rest. The disorder may be associated
with Addison’s disease, atherosclerosis
(build-up of fatty deposits in the arter-
ies), diabetes, and certain neurological
disorders. Symptoms, which generally
occur after sudden standing, include diz-
ziness, light-headedness, blurred vision,
and the temporary loss of consciousness.
imaging agent—A substance used to
enhance images of organs and spaces in
the body.
intermittent claudication—The most
prominent symptom of peripheral arte-
rial disease (PAD). It occurs in one-third
to one-half of PAD patients. Claudica-
tion refers to the pain that occurs in PAD
patients when they exercise, particularly
during walking, which is relieved only by
rest. Leg pain occurs in one leg in 40
percent of patients and in both legs in
60 percent of patients.
ischemia—Insufficient supply of blood to
an organ or tissue, which can cause or-
gan damage such as an ischemic stroke.
lipids—A group of fatty substances that
includes triglycerides (the principal forms
of fat in body fat), phospholipids (im-
portant constituents of cell membranes),
and sterols (such as cholesterol).
peripheral vascular disease—The
obstruction of blood supply to the ex-
tremities, particularly the legs, caused by
atherosclerosis.
PET imaging—Positron emission tomog-
raphy.
Phase I—Safety testing and pharmaco-
logical profiling in humans.
Phase II—Effectiveness testing in
humans.
Phase III—Extensive clinical trials in
humans.
preeclampsia—A condition in pregnant
women characterized by high blood
pressure and high levels of protein in
urine.
pulmonary hypertension—High blood
pressure in the arteries supplying the
lungs due to increased resistance to
blood flow through the lungs.
Raynaud’s disease—A circulatory disor-
der caused by insufficient blood supply
to the hands and feet, resulting in cya-
nosis, numbness, pain, and, in extreme
cases, gangrene.
reperfusion injury—Refers to myocar-
dial, vascular, or electro-physiological
dysfunction that is induced by the
restoration of blood flow to previously
ischemic tissue.
restenosis—A condition where an artery
plugs up again following treatment to
open it up.
stent—A metal device that is used to
hold tissue in place. For example, a stent
can keep blood vessels open after a sur-
gical procedure or heart catheterization.
stroke—Usually caused by atheroscle-
rosis. It results in death or serious brain
damage, such as paralysis or loss of
speech. An ischemic stroke is caused
by blocked or narrowed arteries that
prevent sufficient blood and oxygen from
reaching the brain.
thromboembolism—Blockage of a blood
vessel by a fragment that has broken
off and been carried from a thrombus
(blood clot) elsewhere in the circulation.
thrombosis—The formation of a blood
clot within the heart or a blood vessel.
25. The Drug Discovery, Development and Approval Process
The U.S. system of new drug approvals is
perhaps the most rigorous in the world.
It takes 10-15 years, on average, for an experi-
mental drug to travel from lab to U.S. patients,
according to the Tufts Center for the Study of
Drug Development. Only five in 5,000 com-
pounds that enter preclinical testing make it to
human testing. And only one of those five is
approved for sale.
On average, it costs a company $1.2 billion,
including the cost of failures, to get one new
medicine from the laboratory to U.S. patients,
according to a recent study by the Tufts Center
for the Study of Drug Development.
Once a new compound has been identified in
the laboratory, medicines are usually developed
as follows:
Preclinical Testing. A pharmaceutical company
conducts laboratory and animal studies to show
biological activity of the compound against the
targeted disease, and the compound is evalu-
ated for safety.
Investigational New Drug Application (IND).
After completing preclinical testing, a com-
pany files an IND with the U.S. Food and Drug
Administration (FDA) to begin to test the drug
in people. The IND shows results of previous
experiments; how, where and by whom the new
studies will be conducted; the chemical structure
of the compound; how it is thought to work in
the body; any toxic effects found in the animal
studies; and how the compound is manufac-
tured. All clinical trials must be reviewed and ap-
proved by the Institutional Review Board (IRB)
where the trials will be conducted. Progress
reports on clinical trials must be submitted at
least annually to FDA and the IRB.
Clinical Trials, Phase I—Researchers test the
drug in a small group of people, usually between
20 and 80 healthy adult volunteers, to evaluate
its initial safety and tolerability profile, deter-
mine a safe dosage range, and identify potential
side effects.
Clinical Trials, Phase II—The drug is given
to volunteer patients, usually between 100 and
300, to see if it is effective, identify an optimal
dose, and to further evaluate its short-term
safety.
Clinical Trials, Phase III—The drug is given to a
larger, more diverse patient population, often
involving between 1,000 and 3,000 patients
(but sometime many more thousands), to gener-
ate statistically significant evidence to confirm
its safety and effectiveness. They are the lon-
gest studies, and usually take place in multiple
sites around the world.
New Drug Application (NDA)/Biologic License
Application (BLA). Following the completion
of all three phases of clinical trials, a company
analyzes all of the data and files an NDA or BLA
with FDA if the data successfully demonstrate
both safety and effectiveness. The applications
contain all of the scientific information that the
company has gathered. Applications typically
run 100,000 pages or more.
Approval. Once FDA approves an NDA or BLA,
the new medicine becomes available for physi-
cians to prescribe. A company must continue
to submit periodic reports to FDA, including
any cases of adverse reactions and appropriate
quality-control records. For some medicines,
FDA requires additional trials (Phase IV) to
evaluate long-term effects.
Discovering and developing safe and effective
new medicines is a long, difficult, and expensive
process. PhRMA member companies invested an
estimated $48.5 billion in research and develop-
ment in 2012.
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
PRE-DISCOVERY
DRUG DISCOVERY PRECLINICAL CLINICAL TRIALS FDA REVIEW LG-SCALE MFG
3 – 6 YEARS 6– 7 YEARS
0.5– 2
YEARS
100–300 1,000–3,00020–80
PHASE
2
PHASE
3
PHASE
1
INDSUBMITTED
NDASUBMITTED
PHASE4:POST-MARKETINGSURVEILLANCE
NUMBER OF VOLUNTEERS
ONE FDA-
APPROVED
DRUG
5,000 –10,000
COMPOUNDS
250 5
Drug Discovery and Development: A LONG, RISKY ROAD
