America’s biopharmaceutical research companies are developing 221 medicines to treat diabetes and related conditions. All of the medicines in this report are either in clinical trials or awaiting approval by the U.S. Food and Drug Administration. Diabetes affects nearly 26 million Americans —8.3 percent of the U.S. population—and about one-quarter are unaware they have the disease.

1. 2012 Report
Medicines in Development
Diabetes
presented by america’s biopharmaceutical
research companies
Biopharmaceutical Research Companies Are
Developing More Than 220 Medicines to Treat
Diabetes and Related Conditions
Each day
Medicines in Development more than
For Diabetes*
7 million 5,000
American
Americans 79 million
130 18.8 million are American
adults are
Americans diagnosed
have been undiagnosed adults with
diagnosed have diabetes
with prediabetes
diabetes
America’s biopharmaceutical research com- type 2, in which relative insulin deficiency
panies are developing 221 medicines to treat combines with the body failing to properly use
diabetes and related conditions. All of the insulin. Between 5 percent and 10 percent
64 medicines in this report are either in clinical of Americans with diabetes have type 1, in
trials or awaiting approval by the U.S. Food which the body fails to produce insulin.
and Drug Administration. Diabetes affects The medicines in the pipeline today offer
nearly 26 million Americans —8.3 percent of hope of reducing the human toll and eco-
the U.S. population—and about one-quarter nomic costs of diabetes. Examples of some
are unaware they have the disease. medicines now being tested include:
32 Unfortunately, while the death rate due to • medicine that improves
A
diabetes is declining, the rate of new cases glucose-dependent insulin secretion.
has been rising. The number of Americans
diagnosed with diabetes has more than tripled • medicine designed to inhibit an enzyme
A
14
since 1980, according to the U.S. Centers linked to diabetic neuropathy.
for Disease Control and Prevention (CDC). • medicine to treat type 2 diabetes that
A
Lifestyle choices can affect this increase. may allow for once-weekly dosing.
The CDC-led National Diabetes Prevention
While diabetes remains a formidable foe,
s
s
ns
s
ete
ete
Program found that weight loss and increased
ete
itio
America’s biopharmaceutical research com-
iab
iab
ab
physical activity in people at high risk for
nd
1D
2D
panies are continuing their efforts to develop
Di
Co
diabetes reduced the development of type
d
novel and more effective therapies to treat the
pe
pe
ifie
ted
2 diabetes by 58 percent in a 3-year period.
Ty
Ty
ec
ela
disease and increase patients’ quality of life.
sp
Medicines can also help reduce the risk of type
s-R
Un
ete
2 diabetes. For example, one medicine was
ab
found in studies to lower the risk by 31 percent.
Di
* Some medicines are listed in more According to the American Diabetes Asso-
than one category ciation, most Americans with diabetes have

2. Medicines in Development for Diabetes
Diabetes, Type 1 and Type 2
Product Name Sponsor Indication Development Status*
11ß-HSD inhibitor Bristol-Myers Squibb diabetes Phase II
Princeton, NJ (800) 332-2056
AC 201 Twi Pharmaceuticals type 2 diabetes Phase II
(interleukin-1ß inhibitor) Taipei, Taiwan www.twipharma.com
AC 165198 Amylin Pharmaceuticals diabetes Phase I
(fusion protein) San Diego, CA (858) 552-2200
Biocon
Bangalore, India
Afrezza® MannKind type 1 diabetes, type 2 diabetes application submitted
insulin inhalation Valencia, CA (661) 775-5300
aleglitazar Roche type 2 diabetes Phase III
(PPAR a/g agonist) Nutley, NJ (973) 235-5000
-------------------------------------------------- -------------------------------------------
type 2 diabetes Phase I
(combination therapy) (973) 235-5000
alogliptin Takeda Pharmaceuticals U.S.A. type 2 diabetes application submitted
(DPP-IV inhibitor) Deerfield, IL (877) 825-3327
-------------------------------------------------- -------------------------------------------
type 2 diabetes in adolescents and Phase I
children (877) 825-3327
alogliptin/metformin Takeda Pharmaceuticals U.S.A. type 2 diabetes application submitted
(fixed-dose combination) Deerfield, IL (877) 825-3327
alogliptin/pioglitazone Takeda Pharmaceuticals U.S.A. type 2 diabetes application submitted
(fixed-dose combination) Deerfield, IL (877) 825-3327
alogliptin/roflumilast Takeda Pharmaceuticals U.S.A type 2 diabetes Phase I
Deerfield, IL (877) 825-3327
alpha-1 antitrypsin Omni Bio Pharmaceutical type 1 diabetes Phase I/II
Greenwood Village, CO www.omnibiopharma.com
AMG 151 Amgen type 2 diabetes Phase II
(glucokinase stimulant) Thousand Oaks, CA (800) 772-6436
AMG 876 Amgen type 2 diabetes Phase I
(fusion protein) Thousand Oaks, CA (800) 772-6436
*For more information about a specific medicine in this report, please call the telephone number listed.
2 Medicines in Development Diabetes 2012

3. Medicines in Development for Diabetes
Diabetes, Type 1 and Type 2
Product Name Sponsor Indication Development Status
anagliptin Kowa Research Institute type 2 diabetes Phase II
(DPP-IV antagonist) Morrisville, NC (919) 433-1600
analog insulin-PH20 Halozyme Therapeutics type 1 diabetes, type 2 diabetes Phase II
San Diego, CA (858) 794-8889
ARI-2243 Arisaph Pharmaceuticals type 2 diabetes Phase I
(DPP-IV antagonist) Boston, MA (617) 986-4500
ASP-1941 Astellas Pharma US type 2 diabetes Phase II
(SGLT2 inhibitor) Deerfield, IL (800) 695-4321
AV 133 Avid Radiopharmaceuticals diabetes (diagnosis) Phase I
(imaging agent) Philadelphia, PA (215) 298-0700
BGP-15 N-Gene Research Laboratories type 2 diabetes Phase II
(JNK inhibitor/insulin sensitiser) New York, NY (212) 605-0225
BI-135585 Boehringer Ingelheim type 2 diabetes Phase I
(11ß-HSD1 inhibitor) Pharmaceuticals (800) 243-0127
Ridgefield, CT
BIOD-123 Biodel type 1 diabetes Phase II
(ultra-rapid-acting recombinant Danbury, CT (203) 796-5000
human insulin)
BIOD-125 Biodel type 1 diabetes Phase I completed
(ultra-rapid-acting recombinant Danbury, CT (203) 796-5000
human insulin)
Byetta® Amylin Pharmaceuticals type 2 diabetes in adolescents Phase III
exenatide San Diego, CA (858) 552-2200
CA-18C3 XBiotech type 2 diabetes Phase II
Austin, TX (512) 386-2900
canagliflozin Janssen Research Development type 2 diabetes application submitted
(SGLT2 inhibitor) Raritan, NJ www.janssenrnd.com
canagliflozin/metformin Depomed type 2 diabetes Phase III
extended-release Menlo Park, CA (650) 462-5900
(fixed-dose combination) Janssen Research Development www.janssenrnd.com
Raritan, NJ
canagliflozin/metformin Janssen Research Development type 2 diabetes Phase III
immediate-release Raritan, NJ www.janssenrnd.com
(fixed-dose combination)
Medicines in Development Diabetes 2012 3

4. Medicines in Development for Diabetes
Diabetes, Type 1 and Type 2
Product Name Sponsor Indication Development Status
CAT 1004 Catabasis Pharmaceuticals type 2 diabetes Phase I
Cambridge, MA (617) 349-1971
CCX140 ChemoCentryx type 2 diabetes Phase II
Mountain View, CA (see also diabetes-related) (650) 210-2900
cetilistat Norgine type 2 diabetes in clinically obese Phase II
(lipase inhibitor) Amsterdam, Netherlands patients www.norgine.com
CJC-1134-PC ConjuChem type 2 diabetes Phase II
(GLP-1 stimulant) Los Angeles, CA www.conjuchem.com
CVX 096 Pfizer type 2 diabetes Phase I
(PF-04856883) New York, NY (800) 879-3477
(GLP-1 receptor agonist)
dapagliflozin AstraZeneca type 2 diabetes application submitted
(SGLT2 inhibitor) Wilmington, DE (800) 236-9933
Bristol-Myers Squibb (800) 332-2056
Princeton, NJ ----------------------------------------
-----------------------------------------------
type 2 diabetes Phase III
(combination therapy), (800) 236-9933
type 2 diabetes (800) 332-2056
(second-line therapy)
dapagliflozin/metformin AstraZeneca type 2 diabetes Phase III
(fixed-dose combination) Wilmington, DE (800) 236-9933
Bristol-Myers Squibb (800) 332-2056
Princeton, NJ ------------------------------------------------ ---------------------------------
type 2 diabetes Phase I
(with extended-release metformin) (800) 236-9933
(800) 332-2056
DB 959 DARA BioSciences type 2 diabetes Phase I
(PPAR d/g agonist) Raleigh, NC (919) 872-5578
DC9703 Obio Pharmaceutical type 2 diabetes Phase I
(Omega Bio-Pharma) www.obiopharma.com
Wilmington, DE
diabetes compound Eli Lilly diabetes Phase I
Indianapolis, IN (800) 545-5979
diabetes compound Eli Lilly diabetes Phase I
Indianapolis, IN (800) 545-5979
diabetes compound Eli Lilly diabetes Phase I
Indianapolis, IN (800) 545-5979
4 Medicines in Development Diabetes 2012

5. Medicines in Development for Diabetes
Diabetes, Type 1 and Type 2
Product Name Sponsor Indication Development Status
diabetes compound Eli Lilly diabetes Phase I
Indianapolis, IN (800) 545-5979
DSP-8658 Sunovion Pharmaceuticals type 2 diabetes Phase I
(PPAR a/g agonist) Marlborough, MA (508) 481-6700
dulaglutide Eli Lilly type 2 diabetes Phase III
(GLP-1 analog) Indianapolis, IN (800) 545-5979
EGT-0001442 Theracos type 2 diabetes Phase II
(SGLT2 inhibitor) Marlborough, MA (508) 688-4221
EGT-0001474 Theracos type 2 diabetes Phase I completed
(SGLT2 inhibitor) Marlborough, MA (508) 688-4221
empagliflozin Boehringer Ingelheim type 2 diabetes Phase III
(SGLT2 inhibitor) Pharmaceuticals (800) 243-0127
Ridgefield, CT (800) 545-5979
Eli Lilly
Indianapolis, IN
empagliflozin/linagliptin Boehringer Ingelheim type 2 diabetes Phase III
(fixed-dose combination) Pharmaceuticals (800) 243-0127
Ridgefield, CT
empagliflozin/metformin Boehringer Ingelheim type 2 diabetes Phase I
(fixed-dose combination) Pharmaceuticals (800) 243-0127
Ridgefield, CT
emricasan Conatus Pharmaceuticals type 1 diabetes after islet cell Phase I/II
San Diego, CA transplantation www.conatuspharma.com
encapsulated islet cell ViaCyte type 1 diabetes Phase I/II
transplantation therapy San Diego, CA (858) 455-3708
enclomifene Repros Therapeutics type 2 diabetes in men with second- Phase II
The Woodlands, TX ary hypogonadism or adult-onset (281) 719-3400
idiopathic hypogonadotropic
hypogonadism
ertugliflozin/PF-04971729 Pfizer type 2 diabetes Phase II
(SGLT2 inhibitor) New York, NY (800) 879-3477
exenatide long-acting Hanmi Pharmaceutical type 2 diabetes Phase II
Seoul, South Korea www.hanmipharm.com
exenatide suspension Amylin Pharmaceuticals type 2 diabetes Phase II
San Diego, CA (858) 552-2200
Medicines in Development Diabetes 2012 5

6. Medicines in Development for Diabetes
Diabetes, Type 1 and Type 2
Product Name Sponsor Indication Development Status
gevokizumab XOMA type 1 diabetes, type 2 diabetes Phase II
(IL-1B inhibitor mAb) Berkeley, CA (510) 204-7200
GK1-399 Forest Laboratories type 2 diabetes Phase I/II
(TTP-399) New York, NY (800) 947-5227
(glucokinase activator) TransTech Pharma (336) 841-0300
High Point, NC
GPR 119 agonist Bristol-Myers Squibb diabetes Phase I
Princeton, NJ (800) 332-2056
GSK256073 GlaxoSmithKline type 2 diabetes Phase II
(HM74A agonist) Rsch. Triangle Park, NC (888) 825-5249
GSK1070806 GlaxoSmithKline type 2 diabetes Phase I
(IL-18 mAb) Rsch. Triangle Park, NC (888) 825-5249
GSK1614235 GlaxoSmithKline type 2 diabetes Phase I
(SGLT1 inhibitor) Rsch. Triangle Park, NC (888) 825-5249
GSK2330672 GlaxoSmithKline type 2 diabetes Phase I
(ileal bile acid transfer inhibitor) Rsch. Triangle Park, NC (888) 825-5249
HDV-insulin Diasome Pharmaceuticals type 2 diabetes Phase II
Conshohocken, PA (609) 923-9443
HE3286 Harbor BioSciences type 2 diabetes Phase II
San Diego, CA (858) 587-9333
HIP-2B CureDM type 1 diabetes, type 2 diabetes Phase I
(human pro-islet peptide) Wilmington, DE (302) 288-0620
HPP-404 High Point Pharmaceuticals diabetes Phase I
(histamine H3 receptor High Point, NC www.highpointpharma.com
antagonist)
HPP-593 High Point Pharmaceuticals diabetes Phase I
(PPAR d agonist) High Point, NC www.highpointpharma.com
Humalog® Eli Lilly type 1 diabetes, type 2 diabetes Phase III
insulin lispro Indianapolis, IN (800) 545-5979
IBC-VS01 Orban Biotech type 1 diabetes Phase I
(insulin B-chain vaccine) Brookline, MA (774) 571-2626
6 Medicines in Development Diabetes 2012

7. Medicines in Development for Diabetes
Diabetes, Type 1 and Type 2
Product Name Sponsor Indication Development Status
Ilaris® Novartis type 2 diabetes Phase II/III
canakinumab East Hanover, NJ (888) 669-6682
------------------------------------------------ ---------------------------------
type 1 diabetes Phase II
(888) 669-6682
indeglitazar Plexxikon type 2 diabetes Phase II
(PLX-204) Berkeley, CA (510) 647-4000
INGAP peptide Exsulin type 1 diabetes, type 2 diabetes Phase II
Minneapolis, MN www.exsulin.com
insulin adjustable basal Biodel diabetes Phase I
Danbury, CT (203) 796-5000
insulin glargine Sanofi US type 1 diabetes, type 2 diabetes Phase III
(new formulation) Bridgewater, NJ (800) 981-2491
insulin oral sublingual Biodel type 1 diabetes Phase I
Danbury, CT (203) 796-5000
insulin transdermal Transdermal Global type 1 diabetes, type 2 diabetes Phase I
Broomall, PA www.transdermalglobal.com
INT131 InteKrin Therapeutics type 2 diabetes Phase II
(PPAR g modulator) Los Altos, CA (650) 941-5501
ISIS-GCCRrx Isis Pharmaceuticals type 2 diabetes Phase I
(glucocorticoid receptor Carlsbad, CA (800) 679-4747
antagonist)
ISIS-GCGRrx Isis Pharmaceuticals type 2 diabetes Phase I
(glucagon receptor antagonist) Carlsbad, CA (800) 679-4747
ISIS-PTP1Brx Isis Pharmaceuticals type 2 diabetes Phase I
(PTP-1B gene inhibitor) Carlsbad, CA (800) 679-4747
ISIS-SGLT2rx Isis Pharmaceuticals type 2 diabetes Phase I
Carlsbad, CA (800) 679-4747
ITCA 650 Intarcia Therapeutics type 2 diabetes Phase III
(exenatide subcutaneous Hayward, CA (510) 782-7800
implant)
Janumet® Merck type 2 diabetes in adolescents and Phase III
sitagliptin/metformin Whitehouse Station, NJ children (800) 672-6372
Medicines in Development Diabetes 2012 7

8. Medicines in Development for Diabetes
Diabetes, Type 1 and Type 2
Product Name Sponsor Indication Development Status
Januvia® Merck type 2 diabetes in the elderly and Phase III
sitagliptin Whitehouse Station, NJ adolescents (800) 672-6372
JNJ-16269110 Janssen Research Development type 2 diabetes Phase II completed
(usistapide) Raritan, NJ www.janssenrnd.com
JNJ-41443532 Janssen Research Development type 2 diabetes Phase II completed
Raritan, NJ www.janssenrnd.com
KRP-104 ActivX Biosciences type 2 diabetes Phase II
(CD26 antigen inhibitor) La Jolla, CA (858) 558-5558
LCQ908 Novartis type 2 diabetes Phase II
(DGAT1 inhibitor) East Hanover, NJ (888) 669-6682
LEZ763 Novartis type 2 diabetes Phase II
East Hanover, NJ (888) 669-6682
LIK066 Novartis type 2 diabetes Phase I/II
East Hanover, NJ (888) 669-6682
LIM-0705 Limerick Biopharma type 2 diabetes Phase I
South San Francisco, CA (650) 742-0110
linagliptin/pioglitazone Boehringer Ingelheim type 2 diabetes Phase III
(fixed-dose combination) Pharmaceuticals (800) 243-1027
Ridgefield, CT
lisofylline DiaKine Therapeutics type 1 diabetes Phase I
(lysophosphatidic acid Norfolk, VA (434) 981-4777
transferase inhibitor)
lixisenatide Sanofi US type 2 diabetes Phase III
(AVE0010) Bridgewater, NJ (800) 981-2491
lixisenatide/Lantus® insulin Sanofi US type 2 diabetes Phase II
glargine Bridgewater, NJ (800) 981-2491
(fixed-dose combination)
LX-4211 Lexicon Pharmaceuticals type 2 diabetes Phase II
(SLGT1/SLGT2 inhibitor) The Woodlands, TX (281) 863-3000
LY2409021 Eli Lilly type 2 diabetes Phase II
(glucagon-R antagonist) Indianapolis, IN (800) 545-5979
8 Medicines in Development Diabetes 2012

9. Medicines in Development for Diabetes
Diabetes, Type 1 and Type 2
Product Name Sponsor Indication Development Status
LY2523199 Eli Lilly type 2 diabetes Phase II
(11ß-HSD1 inhibitor) Indianapolis, IN (800) 545-5979
LY2605541 Boehringer Ingelheim type 1 diabetes, type 2 diabetes Phase III
novel basal insulin/insulin Pharmaceuticals (800) 243-1027
peglispro Ridgefield, CT (800) 545-5979
Eli Lilly
Indianapolis, IN
LY2963016 Boehringer Ingelheim type 1 diabetes, type 2 diabetes Phase III
(new insulin glargine product) Pharmaceuticals (800) 243-1027
Ridgefield, CT (800) 545-5979
Eli Lilly
Indianapolis, IN
MBX-2982 Metabolex type 2 diabetes Phase II
(G-protein coupled receptor) Hayward, CA (510) 293-8800
mesenchymal precursor Mesoblast type 2 diabetes Phase II
cell (MPC) product New York, NY (212) 880-2060
(stem cell therapy)
metformin delayed-release Elcelyx Therapeutics type 2 diabetes Phase I
formulation San Diego, CA (858) 876-1814
(gut sensory modulator)
metreleptin Amylin Pharmaceuticals diabetes and/or hypertriglyceridemia application submitted
San Diego, CA in patients with inherited or acquired (858) 552-2200
lipodystrophy
-------------------------------------------------- -------------------------------------------
type 1 diabetes Phase I
(858) 552-2200
mitiglinide Kissei America type 2 diabetes Phase III completed
Parsippany, NJ (973) 334-4400
Mitoglitazone™ Metabolic Solutions Development type 2 diabetes Phase II
MSDC-0160 Kalamazoo, MI (269) 343-6732
(insulin sensitizer)
MK-3102 Merck type 2 diabetes Phase III
(DPP-IV inhibitor) Whitehouse Station, NJ (800) 672-6372
MK-5823 Merck type 2 diabetes Phase I
Whitehouse Station, NJ (800) 672-6372
MSDC-0602 Metabolic Solutions Development type 2 diabetes Phase II
(insulin sensitizer) Kalamazoo, MI (269) 343-6732
Medicines in Development Diabetes 2012 9

10. Medicines in Development for Diabetes
Diabetes, Type 1 and Type 2
Product Name Sponsor Indication Development Status
NN1218 Novo Nordisk type 1 diabetes, type 2 diabetes Phase I
(insulin aspart fast-acting) Princeton, NJ (800) 727-6500
NN1953 Novo Nordisk type 1 diabetes, type 2 diabetes Phase I
(insulin oral) Princeton, NJ (800) 727-6500
NN1954 Novo Nordisk type 1 diabetes, type 2 diabetes Phase I
(insulin oral) Princeton, NJ (800) 727-6500
NN9068 Novo Nordisk type 2 diabetes Phase III
(insulin degludec/liraglutide) Princeton, NJ (800) 727-6500
NN9924 Novo Nordisk type 2 diabetes Phase I
(GLP-1 receptor agonist) Princeton, NJ (800) 727-6500
NN9926 Novo Nordisk type 2 diabetes Phase I
(GLP-1 stimulant) Princeton, NJ (800) 727-6500
NP-500 Napo Pharmaceuticals type 2 diabetes Phase I
San Francisco, CA (see also diabetes-related) (415) 371-8300
OAP-189 Pfizer type 2 diabetes Phase I
(PF-05212389) New York, NY (800) 879-3477
Onglyza™ AstraZeneca type 2 diabetes in adolescents and Phase III
saxagliptin Wilmington, DE children (800) 236-9933
Bristol-Myers Squibb (800) 332-2056
Princeton, NJ
Oral-lyn™ Generex Biotechnology type 1 diabetes Phase III
insulin oral Toronto, Canada (416) 364-2551
oxyntomodulin Merck type 2 diabetes Phase I completed
Whitehouse Station, NJ (800) 672-6372
PAZ-320 Boston Therapeutics type 2 diabetes Phase II
Manchester, NH (978) 886-0421
PB-1023 PhaseBio Pharmaceuticals type 2 diabetes Phase II
(GLP-1 receptor agonist) Malvern, PA (610) 891-6500
PEG-FGF-21 Bristol-Myers Squibb diabetes in clinical trials
Princeton, NJ (800) 332-2056
10 Medicines in Development Diabetes 2012

11. Medicines in Development for Diabetes
Diabetes, Type 1 and Type 2
Product Name Sponsor Indication Development Status
peptide p277 Andromeda Biotech type 1 diabetes (newly diagnosed) Phase III
(Orphan Drug) Ness Ziona, Israel www.andromedabio.com
PF-04937319 Pfizer type 2 diabetes Phase II
New York, NY (800) 879-3477
PF-05175157 Pfizer type 2 diabetes Phase I
New York, NY (800) 879-3477
PF-05231023 Pfizer type 2 diabetes Phase I
New York, NY (800) 879-3477
Prochymal® Osiris Therapeutics type 1 diabetes Phase II
remestemcel-L Columbia, MD (443) 545-1800
Qnexa® VIVUS type 2 diabetes in obese patients Phase II
phentermine/topiramate Mountain View, CA (650) 934-5200
Ranexa® Gilead Sciences type 2 diabetes Phase III
ranolazine extended-release Foster City, CA (800) 445-3235
RG4929 Roche type 2 diabetes Phase II
(11ß-HSD inhibitor) Nutley, NJ (973) 235-5000
RG7201 Roche type 2 diabetes Phase III
(SGLT2 inhibitor) Nutley, NJ (973) 235-5000
RG7426 Bayhill Therapeutics type 1 diabetes Phase I completed
(BHT-3021) Palo Alto, CA (650) 320-2800
Genentech (800) 626-3553
South San Francisco, CA
RG7685 Roche type 2 diabetes Phase II
(GLP-1/GIP dual agonist) Nutley, NJ (973) 235-5000
RM-493 Rhythm Pharmaceuticals diabetes Phase I
(MC4R peptide) Boston, MA (857) 264-4280
Ryzodeg® Novo Nordisk type 1 diabetes, type 2 diabetes application submitted
insulin degludec plus Princeton, NJ (800) 727-6500
insulin aspart
S-707106 Shionogi type 2 diabetes Phase II
Florham Park, NJ (973) 966-6900
Medicines in Development Diabetes 2012 11

12. Medicines in Development for Diabetes
Diabetes, Type 1 and Type 2
Product Name Sponsor Indication Development Status
semaglutide Novo Nordisk type 2 diabetes Phase II
(NN9535) Princeton, NJ (800) 727-6500
sitagliptin/atovastatin Merck type 2 diabetes Phase III
(fixed-dose combination) Whitehouse Station, NJ (800) 672-6372
SLX-4090 Kadmon Pharmaceuticals diabetes Phase II
(MTP inhibitor) Warrendale, PA (724) 778-6100
SRT2104 Sirtris (GlaxoSmithKline) type 2 diabetes Phase II
(SIRT1 protein stimulant) Cambridge, MA (617) 252-6920
SRT2379 Sirtris (GlaxoSmithKline) type 2 diabetes Phase I
(SIRT1 protein stimulant) Cambridge, MA (617) 252-6920
SRT3025 Sirtris (GlaxoSmithKline) type 2 diabetes Phase I
(SIRT1 protein stimulant) Cambridge, MA (617) 252-6920
Syncria® GlaxoSmithKline type 2 diabetes Phase III
albiglutide Rsch. Triangle Park, NC (888) 825-8249
(GLP-1 agonist)
T2 18C3 XBiotech type 2 diabetes Phase I
(mAb) Austin, TX (512) 386-2900
TAK-329 Takeda Pharmaceuticals U.S.A. type 1 diabetes Phase I
(glucokinase stimulant) Deerfield, IL (877) 825-3327
TAK-472 Takeda Pharmaceuticals U.S.A. type 2 diabetes Phase II
(DPP-IV antagonist) Deerfield, IL (877) 825-3327
TAK-875 Takeda Pharmaceuticals U.S.A. type 2 diabetes Phase III
(GPR40 agonist) Deerfield, IL (877) 825-3327
taurocholic acid Satiogen Pharmaceuticals type 2 diabetes Phase I/II
San Diego, CA www.satiogen.com
teneligliptin Mitsubishi Pharma America type 2 diabetes Phase I
(DPP-IV antagonist) Warren, NJ www.mt-pharma-america.com
teplizumab MacroGenics type 1 diabetes Phase III
(Orphan Drug) Rockville, MD (301) 251-5172
12 Medicines in Development Diabetes 2012

13. Medicines in Development for Diabetes
Diabetes, Type 1 and Type 2
Product Name Sponsor Indication Development Status
Tresiba® Novo Nordisk type 1 diabetes, type 2 diabetes application submitted
insulin degludec Princeton, NJ (800) 727-6500
------------------------------------------------ ---------------------------------
type 1 diabetes in adolescents and Phase III
children (800) 727-6500
TT-401 Transition Therapeutics type 2 diabetes Phase I
(GLP-1 receptor agonist) Toronto, Canada (416) 260-7770
TTP054 TransTech Pharma type 2 diabetes Phase II
(GLP-1 receptor agonist) High Point, NC (336) 841-0300
TTP355 Forest Laboratories type 2 diabetes Phase I
(glucokinase stimulant) New York, NY (800) 947-5227
TransTech Pharma (336) 841-0300
High Point, NC
TTP814 TransTech Pharma type 2 diabetes Phase I/II
(PTP-1ß inhibitor) High Point, NC (336) 841-0300
Victoza® Novo Nordisk type 2 diabetes in adolescents and Phase III
liraglutide Princeton, NJ children (800) 727-6500
------------------------------------------------ ---------------------------------
type 1 diabetes Phase II
(800) 727-6500
vildagliptin Novartis type 2 diabetes Phase III
East Hanover, NJ (888) 669-6682
------------------------------------------------ ---------------------------------
type 2 diabetes in treatment-naïve Phase II/III
patients (888) 669-6682
vildagliptin/metformin Novartis type 2 diabetes Phase III completed
(fixed-dose combination) East Hanover, NJ (888) 669-6682
VRS-859 Diartis Pharmaceuticals type 2 diabetes Phase I
(GLP-1 agonist) Redwood City, CA (650) 963-8595
VVP808 Verva Pharmaceuticals type 2 diabetes Phase II
(insulin sensitizer) Carlsbad, CA (760) 271-4783
ZP2929 Boehringer Ingelheim type 2 diabetes Phase I
(GLP-1 receptor agonist) Pharmaceuticals (800) 243-0127
Ridgefield, CT www.zealandpharma.com
Zealand Pharma
Copenhagen, Denmark
Medicines in Development Diabetes 2012 13

14. Medicines in Development for Diabetes
Diabetes-Related Conditions
Product Name Sponsor Indication Development Status
ABT-335 Abbott Laboratories diabetic macular edema Phase II completed
(choline fenofibrate) Abbott Park, IL (847) 937-6100
ABT-639 Abbott Laboratories diabetic neuropathy Phase II
(T-type calcium channel Abbott Park, IL (847) 937-6100
antagonist)
ABT-652 Abbott Laboratories diabetic neuropathy Phase II
(histamine H3 receptor Abbott Park, IL (847) 937-6100
modulator)
ACT-1 topical gel FirstString Research diabetic foot ulcers Phase II
Mount Pleasant, SC (843) 860-8372
AKB-9778 Aerpio Therapeutics diabetic macular edema Phase I
(HPTPß inhibitor/Tie-2 receptor Cincinnati, OH (513) 985-1920
activator)
AL-39,324 Alcon diabetic retinopathy Phase I/II
(tyrosine kinase inhibitor) Fort Worth, TX (800) 862-5266
ALG-1001 Allegro Ophthalmics diabetic macular edema Phase I/II
(small peptide therapy) San Juan Capistrano, CA (949) 940-8130
AmiKet™ EpiCept diabetic neuropathy Phase II
amitriptyline/ketamine Tarrytown, NY (914) 606-3500
ARA 290 Araim Pharmaceuticals diabetic neuropathy Phase II
(erythropoietin receptor agonist) Ossining, NY (914) 762-7586
ARC-4558 Aricon Therapeutics diabetic neuropathy Phase II completed
(clonidine topical gel) Baltimore, MD (Fast Track) (410) 522-8701
atrasentan Abbott Laboratories diabetic nephropathy Phase II
(selective endothelin A receptor Abbott Park, IL (847) 937-6100
antagonist)
baricitinib Eli Lilly diabetic nephropathy Phase II
(JAK1/JAK2 inhibitor) Indianapolis, IN (800) 545-5979
Incyte
Wilmington, DE
BVS857 Novartis Pharmaceuticals insulin resistance Phase I
East Hanover, NJ (888) 669-6682
C-peptide long-acting Cebix diabetic neuropathy Phase I/II
La Jolla, CA (Fast Track) (858) 729-6500
14 Medicines in Development Diabetes 2012

15. Medicines in Development for Diabetes
Diabetes-Related Conditions
Product Name Sponsor Indication Development Status
carisbamate Janssen Research Development diabetic neuropathy Phase II completed
Raritan, NJ www.janssenrnd.com
CCX140 ChemoCentryx diabetic nephropathy Phase II
Mountain View, CA (see also diabetes) (650) 210-2900
Cogenzia™ Innocoll diabetic foot ulcers Phase III
gentamicin-collagen sponge Ashburn, VA www.innocollinc.com
CORT001 Cortendo metabolic syndrome Phase II
Halmstad, Sweden www.cortendo.com
CTP-499 Concert Pharmaceuticals diabetic nephropathy Phase II
Lexington, MA (781) 860-0045
darapladib GlaxoSmithKline diabetic macular edema Phase II
Rsch. Triangle Park, NC (888) 825-5249
dextromethorphan/quinidine Avanir Pharmaceuticals diabetic neuropathy Phase III
(fixed-dose combination) Aliso Viejo, CA www.avanir.com
DSC-127 Derma Sciences diabetic foot ulcers Phase II
Princeton, NJ (609) 514-4744
EVK-001 Evoke Pharma diabetic gastroparesis Phase II
(intranasal dopamine-receptor San Diego, CA (858) 967-5454
antagonist)
Eylea® Bayer HealthCare Pharmaceuticals diabetic macular edema Phase III
afilibercept Wayne, NJ (888) 842-2937
Regeneron Pharmaceuticals (914) 847-7000
Tarrytown, NY
GRT 6005/06 Forest Laboratories diabetic neuropathy Phase II
New York, NY (800) 947-5227
GSK962040 GlaxoSmithKline diabetic gastroparesis Phase II
(motilin receptor agonist) Rsch. Triangle Park, NC (888) 825-5249
HO-0303 HealOr diabetic foot ulcers Phase II/III
(protein kinase C inhibitor/ Ness Ziona, Israel www.healor.com
stimulant)
ICO-007 iCo Therapeutics diabetic macular edema Phase II
(antisense oligonucleotide) Vancouver, Canada (604) 602-9414
Medicines in Development Diabetes 2012 15

16. Medicines in Development for Diabetes
Diabetes-Related Conditions
Product Name Sponsor Indication Development Status
Iluvien® Alimera Sciences diabetic macular edema application submitted
fluocinolone acetonide Alpharetta, GA (Fast Track) (678) 990-5740
pSivida (617) 926-5000
Watertown, MA
iroxanadine Orphazyme diabetic foot ulcers, Phase I
Copenhagen, Denmark diabetic neuropathy www.orphazyme.com
losartan/thioctic acid InVasc Therapeutics diabetic nephropathy Phase II
(INV-144) Tucker, GA (678) 736-5900
Lucentis® Genentech diabetic macular edema Phase I
ranibizumab implant South San Francisco, CA (800) 626-3553
(sustained delivery) ForSight Vision4
Menlo Park, CA
LY2382770 Eli Lilly diabetic nephropathy Phase II
(TGF-b mAb) Indianapolis, IN (800) 545-5979
LY2623091 Eli Lilly renal disease associated with Phase II
(MR antagonist) Indianapolis, IN diabetes (800) 545-5979
Macugen® Eyetech diabetic macular edema (Fast Track), Phase III
pegaptanib Palm Beach Gardens, FL diabetic retinopathy www.eyetech.com
MK-6096 Merck diabetic neuropathy Phase II
(dual orexin receptor Whitehouse Station, NJ (800) 672-6372
antagonist)
modified hepatocyte growth ViroMed diabetic nephropathy Phase I/II
factor gene therapy Seoul, South Korea www.viromed.com
NanoDOX® Nanotherapeutics diabetic foot ulcers Phase II
doxycycline topical gel Alachua, FL (386) 462-9663
nemonoxacin TaiGen Biotechnology infections associated with diabetic Phase II completed
Taipei, Taiwan foot ulcers www.taigenbiotech.com
Nexagon® CoDa Therapeutics diabetic foot ulcers Phase II
antisense oligonucleotide San Diego, CA (858) 677-4074
Nova63035 Novagali Pharma diabetic macular edema Phase I/II
(dexamethasone prodrug Evry, France www.novagali.com
emulsion)
NP-500 Napo Pharmaceuticals metabolic syndrome Phase I
San Francisco, CA (see also diabetes) (415) 371-8300
16 Medicines in Development Diabetes 2012

17. Medicines in Development for Diabetes
Diabetes-Related Conditions
Product Name Sponsor Indication Development Status
ocriplasmin ThromboGenics diabetic retinopathy Phase II
Iselin, NJ (732) 590-2900
Optina™ Ampio Pharmaceuticals diabetic macular edema Phase II
danazol low-dose Greenwood Village, CO (720) 437-6500
Ozurdex® Allergan diabetic macular edema Phase III
dexamethasone intravitreal Irvine, CA (714) 246-4500
implant
pexiganan Dipexium Pharmaceuticals diabetic foot ulcers application submitted
New York, NY (212) 618-1415
PF-655 Quark Pharmaceuticals diabetic macular edema Phase II
(RTP801i14) Fremont, CA (510) 402-4020
PF-00489791 Pfizer diabetic nephropathy Phase II
(phosphodiesterase 5 inhibitor) New York, NY (800) 879-3477
PF-03882845 Pfizer diabetic nephropathy Phase I
New York, NY (800) 879-3477
Pyridorin™ Nephrogenex diabetic nephropathy Phase II
pyridoxamine Rsch. Triangle Park, NC (Fast Track) (609) 986-1780
Qutenza® Astellas Pharma US diabetic neuropathy Phase III
capsaicin dermal patch Deerfield, IL (800) 695-4321
NeurogesX (650) 358-3300
San Mateo, CA
ranirestat Eisai diabetic neuropathy Phase III
(AS-3201) Woodcliff Lake, NJ (888) 274-2378
RM-131 Rhythm Pharmaceuticals diabetic gastroparesis Phase II
(ghrelin peptide agonist) Boston, MA (857) 264-4280
safotibant (FOV2304) Sanofi US diabetic macular edema Phase II
(bradykinin ß1 antagonist) Bridgewater, NJ (800) 981-2491
SAR164653 Sanofi US cardiovascular-related complications Phase I
(cathepsin A inhibitor) Bridgewater, NJ and death in diabetic patients (800) 981-2491
SAR407899 Sanofi US diabetic nephropathy Phase I
(Rho kinase inhibitor) Bridgewater, NJ (800) 981-2491
Medicines in Development Diabetes 2012 17

18. Medicines in Development for Diabetes
Diabetes-Related Conditions
Product Name Sponsor Indication Development Status
sirolimus ophthalmic MacuSight diabetic macular edema Phase II
Union City, CA (subconjunctival) (Fast Track) (510) 400-1440
------------------------------------------------ ---------------------------------
diabetic macular edema Phase I
(intravitreous) (510) 400-1440
SKL-NP SK Life Science diabetic neuropathic pain Phase II
Fair Lawn, NJ (201) 421-3864
TAK-428 Takeda Pharmaceuticals U.S.A. diabetic neuropathy Phase II
(nerve growth factor agonist) Deerfield, IL (877) 825-3327
TTPABC TransTech Pharma diabetic nephropathy Phase I
High Point, NC (336) 841-0300
TV1001 TheraVasc diabetic angiopathy Phase II
(low-dose sodium nitrate) Cleveland, OH (318) 349-3851
TZP-102 Tranzyme Pharma diabetic gastroparesis Phase II/III
(ghrelin agonist) Durham, NC (Fast Track) (919) 474-0020
Vasaloc™ Ampio Pharmaceuticals diabetic nephropathy Phase I
danazol low-dose Greenwood Village, CO (720) 437-6500
white blood cell therapy Macrocure diabetic foot ulcers Phase III
(intralesional) Tikva, Israel www.macrocure.com
M
edicines in Development for Diabetes and
Diabetes-Related Conditions by Phase of Development*
Phase I 85
Phase II 94
Phase III 42
Application Submitted 11
* Some medicines are listed in more than one phase of development.
18 Medicines in Development Diabetes 2012

19. Glossary
application submitted—An application for and biological tissues (called ischemia) and It is also one of the most significant long-
marketing has been submitted by the company leads to cellular death (necrosis and gangrene, term complications in terms of morbidity and
to the Food and Drug Administration (FDA). which in turn may require amputation). Thus, mortality for individual patients with diabetes,
tissues which are very sensitive to oxygen which is responsible for up to 40 percent of all
diabetes—A chronic disease in which the levels, such as the retina, develop microan- end-stage renal disease (ESRD) cases in the
body does not produce or properly use insulin, giopathy and may cause blindness (so-called United States.
a hormone that is needed to convert sugar, proliferative diabetic retinopathy). Damage to
starches and other food into energy needed nerve cells may cause peripheral neuropathy, diabetic neuropathy—Nerve damage in
for daily life. Symptoms may include excessive and to kidney cells, diabetic nephropathy. the arms, hands, legs, and feet caused by
thirst, hunger, urination and weight loss. The diabetes. The condition develops slowly and
cause of diabetes continues to be a mystery, diabetic gastroparesis—A disorder affecting worsens over time. Depending on the types
although both genetics and environmental fac- people with both type 1 and type 2 diabetes of nerves involved, one or more signs and
tors such as obesity and lack of exercise ap- in which the stomach takes too long to empty symptoms may be present in diabetic periph-
pear to play roles. Type 1 diabetes, the more its contents (delayed gastric emptying). The eral neuropathy. Sensory neuropathy results
severe form, results from the body’s failure to vagus nerve controls the movement of food in numbness or tingling in the feet or pain or
produce insulin, which “unlocks” the cells of the through the digestive tract. If the vagus nerve discomfort in the feet or legs, including prickly,
body, allowing glucose to enter and fuel them. is damaged or stops working, the muscles of sharp pain or burning feet. Motor neuropathy
It is estimated that 5 percent to 10 percent of the stomach and intestines do not work nor- involves muscle weakness and loss of muscle
Americans who are diagnosed with diabetes mally, and the movement of food is slowed or tone in the feet and lower legs, loss of balance,
have type 1, which requires insulin treatment. stopped. Just as with other types of neuropathy or changes in foot shape that can lead to areas
Type 2 diabetes results from insulin resistance (nerve damage), diabetes can damage the of increased pressure. Autonomic neuropathy
(a condition in which the body fails to properly vagus nerve if blood glucose levels remain results in dry feet and cracked skin. The loss
use insulin), combined with relative insulin high over a long period of time. High blood glu- of sensation and other problems associated
deficiency. Most Americans who are diagnosed cose causes chemical changes in nerves and with nerve damage make a patient prone to
with diabetes have type 2, which in most cases damages the blood vessels that carry oxygen developing skin ulcers (open sores) that can
can be controlled if treated properly by a com- and nutrients to the nerves. Gastroparesis can become infected and may not heal. This seri-
bination of dietary measures, weight loss, and make diabetes worse by making it more dif- ous complication of diabetes can lead to the
oral medication. ficult to manage blood glucose. When food that loss of a foot, a leg, or even a life.
has been delayed in the stomach finally enters
diabetic angiopathy—Similar to most com- the small intestine and is absorbed, blood diabetic retinopathy—The most common
plications brought about by diabetes, diabetic glucose levels rise. If food stays too long in the diabetic eye disease and a leading cause of
angiopathy is primarily due to hyperglycemia stomach, it can cause problems like bacterial blindness in American adults. It is caused by
or the high levels of blood sugar known as overgrowth because the food has fermented. changes in the blood vessels of the retina, the
glucose. Angiopathy can occur in any part Also, the food can harden into solid masses, light sensitive tissue at the back of the eye that
of the body where the effect of high glucose called bezoars, that may cause nausea, vomit- is necessary for good vision. In some people
levels result in the build up of plaque in the ing, and obstruction in the stomach. Bezoars with diabetic retinopathy, blood vessels may
inner walls of the vessels. There are two types can be dangerous if they block the passage of swell and leak fluid. In others, abnormal new
of the disease—macroangiopathy or microan- food into the small intestine. blood vessels grow on the surface of the retina.
giopathy. In macroangiopathy, atherosclerosis Over time, diabetic retinopathy, which usually
and a resultant blood clot forms on the large diabetic nephropathy—Kidney disease or affects both eyes, can worsen and cause
blood vessels, sticks to the vessel walls, and damage that can occur in people with diabetes. vision loss. The condition has four stages: 1)
blocks the flow of blood. Macroangiopathy may The kidneys have many tiny blood vessels that mild nonproliferative retinopathy, the earliest
cause other complications, such as ischemic filter waste from the blood. High blood sugar stage during which microaneurysms occur
heart disease, stroke, and peripheral vascular from diabetes can destroy those blood vessels. in the retina’s tiny blood vessels; 2) moder-
disease, which contributes to the diabetic foot Over time, the kidney isn’t able to do its job as ate nonproliferative retinopathy, during which
ulcers and the risk of amputation. In microan- well and may stop working completely, which some blood vessels that nourish the retina are
giopathy, the walls of the smaller blood vessels is called kidney failure. Out of 100 people with blocked; 3) severe nonproliferative retinopathy,
become so thick and weak that they bleed, leak diabetes, as many as 40 will develop kidney when many more blood vessels are blocked
protein, and slow the flow of blood through the damage. Currently, diabetic nephropathy is and deprive several areas of the retina with
body. The decrease of blood flow through clot the leading cause of chronic kidney disease in their blood supply; and 4) proliferative retinopa-
formation impairs the flow of oxygen to cells the United States and other Western societies. thy, the advanced stage when signals sent by
Medicines in Development Diabetes 2012 19

20. Glossary
the retina for nourishment trigger the growth predisposed to insulin resistance. Acquired Phase I—Researchers test the drug in a small
of new blood vessels, which grow along the factors, such as excess body fat and physical group of people, usually between 20 and 80
retina and the surface of the clear, vitreous inactivity, also can elicit insulin resistance. Most healthy adult volunteers, to evaluate its initial
gel that fills the inside of the eye. If those thin, people with insulin resistance have abdomi- safety and tolerability profile, determine a
fragile blood vessels leak blood, severe vision nal obesity. The biologic mechanisms at the safe dosage range, and identify potential side
loss and even blindness can result. Up to 45 molecular level between insulin resistance and effects.
percent of Americans diagnosed with diabetes metabolic risk factors aren’t fully understood
have some stage of diabetic retinopathy. and appear to be complex. Phase II—The drug is given to volunteer
patients, usually between 100 and 300, to see
diabetic ulcers—The most common foot macular edema—A condition in which fluid if it is effective, identify an optimal dose, and to
injuries leading to lower extremity amputation. can leak into the center of the macula, the part further evaluate its short-term safety.
The vast majority of diabetic foot complications of the eye where sharp, straight-ahead vision
resulting in amputation begin with the forma- occurs. The fluid makes the macula swell, thus Phase III—The drug is given to a larger, more
tion of skin ulcers. The three main causes blurring vision. It can occur at any stage of diverse patient population, often involving be-
of diabetic foot ulcers are: neuropathy, poor diabetic retinopathy, although it is more likely tween 1,000 and 3,000 patients (but sometime
blood supply, and infection. Up to 15 percent to occur as the disease progresses. About half many more thousands), to generate statistically
of diabetics are likely to develop a foot ulcer of the people with proliferative retinopathy also significant evidence to confirm its safety and
at some stage in their lives. Early detection have macular edema. effectiveness. They are the longest studies,
and appropriate treatment of those ulcers may and usually take place in multiple sites around
prevent up to 85 percent of amputations. The metabolic syndrome—A group of meta- the world.
risk of lower extremity amputation is 15 to 46 bolic risk factors that raise the risk of type 2
times higher in diabetics than in people who do diabetes in one person, including: abdominal
not have the disease. obesity (excessive fat tissue in and around the
abdomen); atherogenic dyslipidemia (blood fat
insulin resistance—A generalized metabolic disorders—high triglycerides, low HDL choles-
disorder caused by the body’s inability to use terol, and high
insulin efficiently. Some people are genetically
The content of this report has been obtained through industry sources and the Adis “RD Insight” database based on the latest information. Report
current as of October 9, 2012. The information may not be comprehensive. For more specific information about a particular product, contact the
individual company directly or go to www.clinicaltrials.gov. The entire series of Medicines in Development is available on PhRMA’s web site.
A publication of PhRMA’s Communications Public Affairs Department. (202) 835-3460
www.phrma.org | www.innovation.org | www.pparx.org | www.buysafedrugs.info
Provided as a Public Service by PhRMA. Founded in 1958 as the Pharmaceutical Manufacturers Association.
Copyright © 2012 by the Pharmaceutical Research and Manufacturers of America. Permission to reprint is awarded if proper credit is given.
Pharmaceutical Research and Manufacturers of America • 950 F Street, NW, Washington, DC 20004
20 Medicines in Development Diabetes 2012

21. Selected Facts About Diabetes in the United States*
Diabetes1
• In the United States, 25.8 million people, or nearly 8.3 percent of the population, have diabetes. An estimated 18.8 million have been diagnosed,
but 7 million people are not aware that they have the disease. Another 79 million have pre-diabetes.
• In 2010, 1.9 million new cases of diabetes were diagnosed in people ages 20 and older. In that age group, 11.3 percent, or 25.6 million people,
have diabetes—13 million men and 12.6 million women.
• In young people under the age of 20, 215,000, or 0.26 percent, have diabetes. About 1 in every 400 children and adolescents has diabetes.
Non-Hispanic white youth have the highest rate of new cases of type 1 diabetes (24.8 per 100,000 per year among those younger than age 10
and 22.6 per 100,000 per year among those ages 10–19).
• Among those people age 65 years and older, 10.9 million, or 26.9 percent, have diabetes. In 2004, heart disease was noted on 68 percent and
stroke on 16 percent of diabetes-related death certificates among that age group.
• The 2007-2009 national survey data for people diagnosed with diabetes ages 20 and older found the following prevalence of diabetes by race and
ethnicity: 7.1 percent of non-Hispanic whites, 8.4 percent of Asian Americans, 12.6 percent of non-Hispanic blacks, and 11.8 percent of Hispanics.
Among Hispanics, the prevalence rates were 7.6 percent for Cubans, 13.3 percent for Mexican Americans, and 13.8 percent for Puerto Ricans.
•
Diabetes is the seventh leading cause of death in the United States. In 2007, diabetes was listed as the underlying cause on 71,382 death certifi-
cates and as a contributing factor on an additional 160,022 death certificates, which means that diabetes contributed to a total of 231,404 deaths.
Diabetes-Related Conditions
• Each year, about 1 percent to 4 percent of people with diabetes develop a foot ulcer, and 10 percent to 15 percent of those with diabetes will
have at least one foot ulcer during their lifetimes. Lower extremity amputations (LEAs) are an extreme complication associated with diabetes
and foot ulcers. In the United States, nearly 80,000 LEAs are performed on diabetics each year. In 2005, the overall rate of hospital discharges
for new LEAs was about 4.3 per 1,000 people with diabetes compared with a rate of about 0.3 per 1,000 in the general population.2
• Adults with diabetes have heart disease death rates and stroke risk at about 2 to 4 times higher than adults without diabetes. In 2004, heart
disease was noted on 68 percent of diabetes-related death certificates among people ages 65 years and older, while stroke was noted on 16
percent of diabetes-related death certificates among that age group.1
•
Diabetes is the leading cause of kidney failure, accounting for 44 percent of new cases in 2008. That year, 48,374 people with diabetes
began treatment for end-stage kidney disease, and another 202,290 people with end-stage kidney disease due to diabetes were living on
chronic dialysis or with a kidney transplant in the United States.1
• About 60 percent to 70 percent of people with diabetes have mild to severe forms of nervous system damage (diabetic neuropathy).1
• Diabetes is the leading cause of new cases of blindness among adults ages 20–74. From 2005-2008, 4.2 million people with diabetes (28.5
percent) age 40 and older had diabetic retinopathy, and of those, almost 0.7 million (4.4 percent) had advanced diabetic retinopathy that
could lead to severe vision loss.1
Costs1
• The total annual economic cost of diabetes in 2007 was $174 billion. Direct medical costs totaled $116 billion, while indirect costs (including
disability, work loss, and premature mortality) came to $58 billion. If the additional costs of undiagnosed diabetes ($18 billion), prediabetes
($25 billion), and gestational diabetes ($623 million) were factored in, the total cost of diabetes in the United States adds up to $218 billion.
• Average medical expenditures among people with diagnosed diabetes are 2.3 times higher than expenditures for people who don’t have diabetes.
Sources:
1. American Diabetes Association, www.diabetes.org
2. Agency for Healthcare Research and Quality, www.ahrq.gov
Medicines in Development Diabetes 2012 21

22. The Drug Discovery, Development and Approval Process
Developing a new medicine takes an average of 10-15 years;
For every 5,000-10,000 compounds in the pipeline, only 1 is approved.
The Drug Development and Approval Process
The U.S. system of new drug approvals is in people. The IND shows results of previous statistically significant evidence to confirm its
perhaps the most rigorous in the world. experiments; how, where and by whom the safety and effectiveness. They are the longest
new studies will be conducted; the chemical studies, and usually take place in multiple sites
It takes 10-15 years, on average, for an
structure of the compound; how it is thought around the world.
experimental drug to travel from lab to U.S.
to work in the body; any toxic effects found in
patients, according to the Tufts Center for the New Drug Application (NDA)/Biologic
the animal studies; and how the compound
Study of Drug Development. Only five in 5,000 License Application (BLA). Following the
is manufactured. All clinical trials must be
compounds that enter preclinical testing make completion of all three phases of clinical trials,
reviewed and approved by the Institutional
it to human testing. And only one of those five a company analyzes all of the data and files an
Review Board (IRB) where the trials will be
is approved for sale. NDA or BLA with FDA if the data successfully
conducted. Progress reports on clinical trials
demonstrate both safety and effectiveness.
On average, it costs a company $1.2 billion, must be submitted at least annually to FDA and
The applications contain all of the scientific
including the cost of failures, to get one new the IRB.
information that the company has gathered.
medicine from the laboratory to U.S. patients,
Clinical Trials, Phase I—Researchers test Applications typically run 100,000 pages or
according to a 2007 study by the Tufts Center
the drug in a small group of people, usually more.
for the Study of Drug Development.
between 20 and 80 healthy adult volunteers, to
Approval. Once FDA approves an NDA or
Once a new compound has been identified in evaluate its initial safety and tolerability profile,
BLA, the new medicine becomes available
the laboratory, medicines are usually devel- determine a safe dosage range, and identify
for physicians to prescribe. A company must
oped as follows: potential side effects.
continue to submit periodic reports to FDA,
Preclinical Testing. A pharmaceutical com- Clinical Trials, Phase II—The drug is given including any cases of adverse reactions and
pany conducts laboratory and animal studies to volunteer patients, usually between 100 and appropriate quality-control records. For some
to show biological activity of the compound 300, to see if it iseffective, identify an optimal medicines, FDA requires additional trials
against the targeted disease, and the com- dose, and to further evaluate its short-term (Phase IV) to evaluate long-term effects.
pound is evaluated for safety. safety.
Discovering and developing safe and effective
Investigational New Drug Application (IND). Clinical Trials, Phase III—The drug is given to new medicines is a long, difficult, and expensive
After completing preclinical testing, a company a larger, more diverse patient population, often process. PhRMA member companies invested
files an IND with the U.S. Food and Drug involving between 1,000 and 3,000 patients (but an estimated $49.5 billion in research and
Administration (FDA) to begin to test the drug sometime many more thousands), to generate development in 2011.