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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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
Medicines in Development Heart Disease and Stroke 201322
PA32540
(aspirin 325mg/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
RX-10001
(lipid modulator)
Resolvyx Pharmaceuticals
Cambridge, MA
cardiovascular disorders Phase I completed
www.resolvyx.com
T89
(herbal medicine product)
Tasly Pharmaceuticals
Rockville, MD
angina pectoris Phase III
www.tasly.com
Vascana™
nitroglycerin topical
MediQuestTherapeutics
Maple Valley, WA
Raynaud’s disease Phase III
www.mqti.com
Vyndaqel®
tafamidis meglumine
Pfizer
New York, NY
transthyretin familial amyloid
cardiomyopathy
Phase III
www.pfizer.com
Xarelto®
rivaroxaban
Bayer HealthCare Pharmaceuticals
Wayne, NJ
Janssen Research  Development
Raritan, NJ
prevention of cardiovascular events
secondary to chronic heart failure,
coronary or peripheral artery
disease, or atrial fibrillation
(FastTrack)
(see also acute coronary syndrome,
thrombosis)
Phase III
www.bayerpharma.com
www.janssenrnd.com
Medicines in Development for Heart Disease and Stroke
Other
Product Name Sponsor Indication Development Phase
The content of this report has been obtained through public, government and industry sources, and the Adis “RD Insight” data-
base based on the latest information. Report current as of May 22, 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 website.
A publication of PhRMA’s Communications  Public Affairs Department. (202) 835-3460
www.phrma.org | www.innovation.org | www.pparx.org
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.
Pharmaceutical Research and Manufacturers of America • 950 F Street, NW, Washington, DC 20004
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
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.
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

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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
  • 22. Medicines in Development Heart Disease and Stroke 201322 PA32540 (aspirin 325mg/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 RX-10001 (lipid modulator) Resolvyx Pharmaceuticals Cambridge, MA cardiovascular disorders Phase I completed www.resolvyx.com T89 (herbal medicine product) Tasly Pharmaceuticals Rockville, MD angina pectoris Phase III www.tasly.com Vascana™ nitroglycerin topical MediQuestTherapeutics Maple Valley, WA Raynaud’s disease Phase III www.mqti.com Vyndaqel® tafamidis meglumine Pfizer New York, NY transthyretin familial amyloid cardiomyopathy Phase III www.pfizer.com Xarelto® rivaroxaban Bayer HealthCare Pharmaceuticals Wayne, NJ Janssen Research Development Raritan, NJ prevention of cardiovascular events secondary to chronic heart failure, coronary or peripheral artery disease, or atrial fibrillation (FastTrack) (see also acute coronary syndrome, thrombosis) Phase III www.bayerpharma.com www.janssenrnd.com Medicines in Development for Heart Disease and Stroke Other Product Name Sponsor Indication Development Phase The content of this report has been obtained through public, government and industry sources, and the Adis “RD Insight” data- base based on the latest information. Report current as of May 22, 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 website. A publication of PhRMA’s Communications Public Affairs Department. (202) 835-3460 www.phrma.org | www.innovation.org | www.pparx.org 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. Pharmaceutical Research and Manufacturers of America • 950 F Street, NW, Washington, DC 20004
  • 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