2011
Arthritis
M E D I C I N E S I N D E V E L O P M E N T F O R
Report
P R E S E N T E D B Y A M E R I C A ’ S B I O P H A R M A C E U T I C A L R E S E A R C H C O M P A N I E S
America’s biopharmaceutical research companies arecurrently developing 198 medicines to help themore than 50 million Americans afflicted with at
least one of the 100 different musculoskeletal disorders,
including arthritis. All of the medicines are either in
clinical trials or awaiting review by the U.S. Food and
Drug Administration.
Arthritis is the most common cause of disability in the
United States. Each year, it is responsible for 44 million
outpatient doctor visits, nearly 1 million hospitalizations,
and nearly 10,000 deaths. And it costs the American
economy nearly $128 billion annually in direct medical
costs and indirect costs, such as lost wages and
productivity.
By 2030, it is estimated that the number of Americans
with some form of diagnosed arthritis will increase to
more than 67 million. “As the baby boomer generation
ages, advances in research and the development of more
effective and safer treatments are critical to helping
improve the quality of life for millions of people with
arthritis,” said John Howard Klippel, M.D., president and
CEO of the Arthritis Foundation.
New medicines today in the research and development
pipeline offer hope of reducing the human and economic
costs of the many musculoskeletal disorders affecting
Americans. They include:
• 67 for rheumatoid arthritis that affects about 1.3 million
American adults.
• 23 for osteoporosis that affects 10 million people, 80
percent of whom are women.
• 19 for lupus which affects an estimated 1.5 million
Americans.
• 19 for osteoarthritis, the most common form of arthritis,
affecting nearly 27 million Americans.
• 15 for musculoskeletal pain that affects the muscles,
ligaments, tendons, and bones.
• 9 for fibromyalgia which affects 3 million to 6 million
Americans.
Examples of some medicines now being tested to treat
musculoskeletal disorders include:
• A new monoclonal antibody in development for lupus
modulates B-cells that produce antibodies against the
body’s own cells and tissue, causing the immune
system to turn on itself.
• A medicine in development for rheumatoid arthritis
(RA) that inhibits two types of an enzyme that are key
components in signaling activation of cytokines and
growth factors that are elevated in patients with RA.
• A potential first-in-class medicine in development for
pain associated with osteoarthritis that is an inhibitor of
a gene-encoding protein that plays a role in inflam -
matory pain.
Researching and developing new medicines remains
a risky investment and lengthy process—costing, on
average, $1.2 billion, including the cost of failures, and
taking between 10–15 years to bring a new medicine to
patients. But advances in our understanding of diseases
and how to treat them have allowed America’s
biopharmaceutical research companies to conduct the
cutting-edge research needed to reduce the destructive
toll of arthritis and other musculoskeletal disorders and
to allow more patients to lead healthier, happier, more
productive lives.
John J. Castellani
President and CEO
PhRMA
Biopharmaceutical Research Companies are Developing
Nearly 200 Medicines for the Most Common Chronic
Health Problem in the United States
MEDICINES AND VACCINES IN DEVELOPMENT FOR ARTHRITIS*
* Some medicines are l is ted in more than one category.
Other
Spondylitis
Duchenne Muscular Dystrophy
Behcet’s Syndrome
Gout
Lupus
Muscle Disorders
Osteoarthritis
Rheumatoid Arthritis
Raynaud’s Disease
Pain
Psoriatic Arthritis
Scleroderma
Osteoporosis
6
Fibromyalgia 9
3
7
19
11
19
67
4
15
7
7
6
22
23
2 M E D I C I N E S I N D E V E L O P M E N T F O R Arthritis 2011
Medicines in Development for Arthritis
B E H C E T ’ S S Y N D R O M E
Product Name Company Indication Development Status*
apremilast Celgene Behcet’s syndrome Phase II
Summit, NJ (see also psoriatic arthritis, (908) 673-9000
rheumatoid arthritis, spondylitis)
gevokizumab XOMA Behcet’s syndrome Phase II
(XOMA052) Berkeley, CA (see also rheumatoid arthritis) (510) 204-7200
Veldona® Amarillo Biosciences Behcet’s syndrome Phase II
interferon-alpha Amarillo, TX (see also fibromyalgia) (806) 376-1741
lozenge
(Orphan Drug)
D U C H E N N E M U S C U L A R D Y S T R O P H Y
Product Name Company Indication Development Status
ACE-031 Acceleron Pharma Duchenne muscular dystrophy Phase II
(Orphan Drug) Cambridge, MA (617) 649-9200
ataluren Genzyme Duchenne muscular dystrophy Phase II/III
(PTC124) Cambridge, MA (Fast Track) (617) 252-7500
(Orphan Drug) PTC Therapeutics (908) 222-7000
South Plainfield, NJ
eteplirsen AVI BioPharma Duchenne muscular dystrophy Phase II
(AVI-4658) Bothell, WA (425) 354-5038
(Orphan Drug)
GSK2402968 GlaxoSmithKline Duchenne muscular dystrophy Phase III
(antisense Rsch. Triangle Park, NC (888) 825-5249
oligonucleotide) Prosensa
(Orphan Drug) Leiden, The Netherlands
idebenone Santhera Pharmaceuticals Duchenne muscular dystrophy Phase III
(Orphan Drug) Charlestown, MA (see also other) (617) 886-5161
muscular dystrophy Asklepios Duchenne muscular dystrophy Phase I
gene therapy BioPharmaceutical (919) 968-2727
Chapel Hill, NC
F I B R O M Y A L G I A
Product Name Company Indication Development Status
AGN-XX/YY ACADIA Pharmaceuticals fibromyalgia Phase II
(rezatomidine) San Diego, CA (858) 558-2871
Allergan (800) 433-8871
Irvine, CA
Cymbalta® Eli Lilly juvenile primary fibromyalgia Phase III
duloxetine Indianapolis, IN syndrome (800) 545-5979
droxidopa Chelsea Therapeutics fibromyalgia Phase II
Charlotte, NC (704) 341-1516
* For more information about a specific medicine in this report, please call the telephone number listed.
3M E D I C I N E S I N D E V E L O P M E N T F O R Arthritis 2011
F I B R O M Y A L G I A
Product Name Company Indication Development Status
flupirtine Adeona Pharmaceuticals fibromyalgia Phase II
Ann Arbor, MI (734) 332-7800
Meda Pharmaceuticals (732) 564-2200
Somerset, NJ
Lyrica® CR Pfizer fibromyalgia Phase II/III
pregabalin New York, NY (860) 723-5156
(controlled-release)
Neupro® UCB fibromyalgia Phase II
rotigotine Smyrna, GA (770) 970-7500
transdermal
TNX-102 TONIX Pharmaceuticals fibromyalgia syndrome Phase II
(cyclobenzaprine New York, NY (212) 980-9155
very low dose)
Veldona® Amarillo Biosciences fibromyalgia Phase II
interferon-alpha Amarillo, TX (see also Behcet’s syndrome) (806) 376-1741
lozenge
Vimpat® UCB fibromyalgia Phase II completed
lacosamide Smyrna, GA (770) 970-7500
G O U T
Product Name Company Indication Development Status
Arcalyst® Regeneron Pharmaceuticals gout Phase III
rilonacept Tarrytown, NY (877) 734-6777
BCX-4208 BioCryst Pharmaceuticals gout Phase II
Birmingham, AL (205) 444-4600
Ilaris® Novartis Pharmaceuticals gouty arthritis application submitted
canakinumab East Hanover, NJ (see also osteoarthritis, (888) 669-6682
(subcutaneous) rheumatoid arthritis)
lesinurad Ardea Biosciences gout Phase II
San Diego, CA (858) 652-6500
MBX-102 Metabolex gout Phase II
(arhalofenate) Hayward, CA (510) 293-8800
NU1618 Nuon Therapeutics gout Phase II
(tranilast/allopurinol) San Mateo, CA (650) 645-1800
pegadricase 3SBio gout Phase I
Shenyang, China www.3sbio.com
L U P U S
Product Name Company Indication Development Status
A-623 Anthera Pharmaceuticals systemic lupus erythematosus Phase II
Hayward, CA (510) 856-5600
AGS-009 Argos Therapeutics systemic lupus erythematosus Phase I
Durham, NC (919) 287-6300
AMG 557 Amgen systemic lupus erythematosus Phase I
Thousand Oaks, CA (800) 772-6436
4 M E D I C I N E S I N D E V E L O P M E N T F O R Arthritis 2011
L U P U S
Product Name Company Indication Development Status
AMG 811 Amgen systemic lupus erythematosus Phase I
Thousand Oaks, CA (800) 772-6436
atacicept EMD Serono systemic lupus erythematosus Phase III
(anti-Blys/anti-APRIL Rockland, MA (800) 283-8088
fusion protein)
BAFF antibody Eli Lilly systemic lupus erythematosus Phase III
(LY2127399) Indianapolis, IN (see also rheumatoid arthritis) (800) 545-5979
Benlysta® GlaxoSmithKline systemic lupus erythematosus Phase II
belimumab Rsch. Triangle Park, NC (see also other) (888) 825-5249
(subcutaneous) Human Genome Sciences (301) 309-8504
Rockville, MD
CDP-7657 Biogen Idec systemic lupus erythematosus Phase I
(CD40L) Cambridge, MA (617) 679-2000
CNTO-136 Centocor Ortho Biotech lupus Phase II
(sirukumab) Horsham, PA (see also rheumatoid arthritis) (610) 651-6000
DV-1179 Dynavax Technologies systemic lupus erythematosus Phase I
Berkeley, CA (877) 848-5100
epratuzumab Immunomedics systemic lupus erythematosus Phase III
Morris Plains, NJ (Fast Track) (973) 605-8200
UCB
Smyrna, GA
forigerimod Cephalon systemic lupus erythematosus Phase III
(CEP-33457) Frazer, PA (610) 344-0200
laquinimod Teva Neuroscience systemic lupus erythematosus Phase II
North Wales, PA (800) 838-2872
MEDI-545 AstraZeneca systemic lupus erythematosus Phase II
(anti-IFN-alpha Wilmington, DE (800) 236-9933
mAb) Medarex (609) 430-2880
Princeton, NJ (301) 298-0000
MedImmune
Gaithersburg, MD
MEDI-570 AstraZeneca systemic lupus erythematosus Phase I
(anti-ICOS mAb) Wilmington, DE (301) 298-0000
MedImmune
Gaithersburg, MD
Orencia® Bristol-Myers Squibb systemic lupus erythematosus Phase II/III
abatacept Princeton, NJ (see also rheumatoid arthritis) (212) 546-4000
(intravenous)
PF-04236921 Pfizer lupus Phase I
New York, NY (see also rheumatoid arthritis) (860) 732-5156
RG7415 Genentech systemic lupus erythematosus Phase II
(rontalizumab) South San Francisco, CA (800) 626-3553
SBI-087 Emergent BioSolutions systemic lupus erythematosus Phase I
(PF-05230895) Rockville, MD (see also rheumatoid arthritis) (301) 795-1800
Pfizer (860) 732-5156
New York, NY
5M E D I C I N E S I N D E V E L O P M E N T F O R Arthritis 2011
M U S C L E D I S O R D E R S
Product Name Company Indication Development Status
AMG 745 Amgen muscular atrophy Phase I
Thousand Oaks, CA (800) 772-6436
arbaclofen XenoPort muscle spasticity Phase II
placarbil Santa Clara, CA www.xenoport.com
(XP19986)
Botox® Allergan muscle spasticity application submitted
onabotulinum Irvine, CA (714) 246-4500
toxin A --------------------------------------------------------------------------------------
(Orphan Drug) muscle spasm Phase III
(714) 246-4500
CK-2017357 Cytokinetics muscle fatigue Phase II
South San Francisco, CA (650) 624-3000
Dysport® Tercica focal spasticity of upper and lower Phase III
abobotulinum Brisbane, CA limb in adults and children (650) 624-4900
toxin A
for injection
GSK2245840 GlaxoSmithKline muscular atrophy Phase I
(SIRT1 protein Rsch. Triangle Park, NC (888) 825-5249
stimulant)
GTx-024 GTx muscular atrophy Phase II completed
Memphis, TN (901) 523-9700
IPX-056 Impax Laboratories muscle spasticity in patients with Phase III
(baclofen Hayward, CA multiple sclerosis (510) 240-6000
extended-release)
LY2495655 Eli Lilly muscular atrophy Phase II
(anti-myostatin Indianapolis, IN (800) 545-5979
mAb)
nabilone Valeant Pharmaceuticals muscle spasticity Phase II
International (800) 548-5100
Irvine, CA
nabiximols Otsuka America muscle spasticity Phase II
Pharmaceutical (800) 562-3974
Rockville, MD
O S T E O A R T H R I T I S
Product Name Company Indication Development Status
Ampion™ Ampio Pharmaceuticals osteoarthritis Phase I
Greenwood Village, CO (303) 418-1000
BI 113823 Boehringer Ingelheim osteoarthritis Phase I
Pharmaceuticals (800) 243-0127
Ridgefield, CT
CG-100649 CG Pharmaceuticals inflammation associated with Phase I
Emeryville, CA osteoarthritis (510) 594-8200
fibroblast growth EMD Serono osteoarthritis Phase I
factor-18 Rockland, MA (see also other) (800) 283-8088
6 M E D I C I N E S I N D E V E L O P M E N T F O R Arthritis 2011
O S T E O A R T H R I T I S
Product Name Company Indication Development Status
IBU-PC PLx Pharma pain and inflammation associated Phase II
Houston, TX with osteoarthritis (713) 842-1249
Ilaris® Novartis Pharmaceuticals osteoarthritis Phase II
canakinumab East Hanover, NJ (see also gout, rheumatoid arthritis) (888) 669-6682
(intra-arterial)
IP-880 Iroko Pharmaceuticals osteoarthritis Phase I
Philadelphia, PA (267) 546-3003
IP-889 Iroko Pharmaceuticals osteoarthritis Phase III
Philadelphia, PA (267) 546-3003
LT-NS001 Logical Therapeutics osteoarthritis Phase II/III
(naproxen etemesil) Waltham, MA (781) 290-0900
MEDI-578 AstraZeneca osteoarthritic pain Phase I
(anti-NGF mAb) Wilmington, DE (800) 236-9933
MedImmune (301) 298-0000
Gaithersburg, MD
naproxcinod NicOx osteoarthritis application submitted
Sophia Antipolis, France www.nicox.com
osteoarthritis Eli Lilly osteoarthritis Phase II
compound Indianapolis, IN (800) 545-5979
(LY2828360)
SAR113945 sanofi-aventis osteoarthritis Phase I
(IKK-ß inhibitor) Bridgewater, NJ (800) 633-1610
SAR114137 sanofi-aventis osteoarthritic pain Phase I
(cathespin S/K Bridgewater, NJ (800) 633-1810
inhibitor)
SAR292833 sanofi-aventis osteoarthritic pain Phase I
(GRC15300) Bridgewater, NJ (800) 633-1810
TRPV3 antagonist Glenmark Pharmaceuticals
Mumbai, India
SD-6010 Pfizer osteoarthritis Phase II/III
New York, NY (860) 732-5156
SMC021 Emisphere Technologies osteoarthritis Phase III
(calcitonin oral) Tarrytown, NY (see also osteoporosis) (914) 347-2220
Novartis Pharmaceuticals (888) 669-6682
East Hanover, NJ
Synavive™ Zalicus osteoarthritis Phase II completed
prednisone/ Cambridge, MA (see also rheumatoid arthritis) (617) 301-7000
dipyridamole
TG-C TissueGene osteoarthritis Phase I
Rockville, MD (301) 921-6000
O S T E O P O R O S I S
Product Name Company Indication Development Status
ACE-011 Acceleron Pharma postmenopausal osteoporosis Phase I
(sotatercept) Cambridge, MA (617) 649-9200
Celgene (908) 673-9000
Summit, NJ
7M E D I C I N E S I N D E V E L O P M E N T F O R Arthritis 2011
O S T E O P O R O S I S
Product Name Company Indication Development Status
alendronate Nycomed US osteoporosis application submitted
effervescent Melville, NY (631) 454-7677
(EX101) EffRx www.effrx.com
Lausanne, Switzerland
AMG 785 Amgen postmenopausal osteoporosis Phase II
(anti-sclerostin Thousand Oaks, CA (see also other) (800) 772-6436
mAb)
Aprela™ Ligand Pharmaceuticals postmenopausal osteoporosis Phase III
bazedoxifene/ San Diego, CA prevention (858) 550-7500
conjugated Pfizer (860) 732-5156
estrogens New York, NY
AUS-131 Ausio Pharmaceuticals osteoporosis Phase I
Cincinnati, OH (513) 731-1600
BA-058 Radius Health osteoporosis Phase II
Cambridge, MA (subcutaneous) (617) 551-4700
--------------------------------------------------------------------------------------
osteoporosis Phase I
(transdermal) (617) 551-4700
calcitonin oral Tarsa Therapeutics postmenopausal osteoporosis Phase III completed
Philadelphia, PA treatment (267) 273-7940
--------------------------------------------------------------------------------------
postmenopausal osteoporosis Phase II
prevention (267) 273-7940
DP-001 Deltanoid Pharmaceuticals osteoporosis Phase II
Madison, WI (608) 238-7710
GSK768974 GlaxoSmithKline postmenopausal osteoporosis Phase II
(parathyroid Rsch. Triangle Park, NC (888) 825-5249
hormone receptor Unigene (973) 265-1100
agonist) Boonton, NJ
MER-103 Merrion Pharmaceuticals osteoporosis Phase II completed
(oral alendronic Wilmington, DE (910) 799-1847
acid)
MK-5442 Merck osteoporosis Phase II
(CaSR antagonist) Whitehouse Station, NJ (800) 672-6372
odanacatib Merck male osteoporosis, Phase III
(MK-0822) Whitehouse Station, NJ postmenopausal osteoporosis (800) 672-6372
osteoporosis Eli Lilly osteoporosis Phase II
compound Indianapolis, IN (800) 545-5979
(LY2541546)
PF-04840082 Pfizer osteoporosis treatment and Phase I
(DKK1 protein New York, NY prevention (860) 732-5156
inhibitor)
Prolia® Amgen male osteoporosis Phase III
denosumab Thousand Oaks, CA (see also rheumatoid arthritis) (800) 772-6436
Reclast® Novartis Pharmaceuticals corticosteroid-induced osteoporosis, application submitted
zoledronic acid East Hanover, NJ postmenopausal osteoporosis (888) 669-6682
prevention
8 M E D I C I N E S I N D E V E L O P M E N T F O R Arthritis 2011
O S T E O P O R O S I S
Product Name Company Indication Development Status
SMC021 Emisphere Technologies postmenopausal osteoporosis Phase III
(calcitonin oral) Tarrytown, NY (see also osteoarthritis) (914) 347-2220
Novartis Pharmaceuticals (888) 669-6682
East Hanover, NJ
teriparatide Marina Biotech osteoporosis Phase II
intranasal Bothell, WA (425) 908-3600
teriparatide Emisphere Technologies postmenopausal osteoporosis Phase I
oral Tarrytown, NY (914) 347-2220
(PTH134) Novartis Pharmaceuticals (888) 669-6682
East Hanover, NJ
teriparatide Zosana Pharma osteoporosis Phase II
transdermal Fremont, CA (510) 745-1200
(ZP-PTH)
ViaDor-hPTH Eli Lilly osteoporosis Phase I
(1-34) Indianapolis, IN (800) 545-5979
(teriparatide TransPharma Medical
transdermal) Lod, Israel
Viviant™ Ligand Pharmaceuticals postmenopausal osteoporosis application submitted
bazedoxifene San Diego, CA prevention and treatment (858) 550-7500
Pfizer (860) 732-5156
New York, NY
ZT-034 Zelos Therapeutics osteoporosis Phase I
(teriparatide) West Conshohocken, PA (intranasal) (610) 260-6045
--------------------------------------------------------------------------------------
osteoporosis Phase I
(subcutaneous) (610) 260-6045
P A I N
Product Name Company Indication Development Status
ABT-652 Abbott Laboratories musculoskeletal pain Phase II
Abbott Park, IL (847) 937-6100
BEMA® BioDelivery Sciences back pain, musculoskeletal pain Phase III
Buprenorphine International (919) 582-9050
buprenorphine Raleigh, NC
transmucosal
bupivacaine DURECT back pain Phase II completed
transdermal Cupertino, CA (408) 777-1417
chondrocyte tissue ISTO Technologies back pain Phase I
grafts St. Louis, MO (314) 995-6049
diclofenac Nuvo Research musculoskeletal pain Phase II
transdermal Mississauga, Canada (905) 673-6980
second-generation
EXPAREL™ Pacira Pharmaceuticals musculoskeletal pain Phase I
bupivacaine Parsippany, NJ (973) 254-3560
extended-release
liposome injection
9M E D I C I N E S I N D E V E L O P M E N T F O R Arthritis 2011
P A I N
Product Name Company Indication Development Status
fulranumab Johnson & Johnson back pain Phase II
Pharmaceutical Research (800) 817-5286
& Development
Raritan, NJ
FX005 Flexion Therapeutics musculoskeletal pain Phase I/II
Woburn, MA (781) 897-9977
IDEA-033 IDEA musculoskeletal pain Phase III
(ketoprofen Munich, Germany www.idea-ag.de
transdermal)
JNJ-39439335 Johnson & Johnson musculoskeletal pain Phase I
(TRPV1 receptor Pharmaceutical Research (800) 817-5286
antagonist) & Development
Raritan, NJ
ketoprofen Hisamitsu Pharmaceutical musculoskeletal pain Phase III
transdermal Tokyo, Japan www.hisamitsu.co.jp
Opana® ER Endo Pharmaceuticals musculoskeletal pain application submitted
oxymorphone Chadd’s Farm, PA (abuse-resistant) (610) 558-9800
oxycodone/ Mundipharma back pain Phase II
naloxone Basel, Switzerland (203) 588-8000
controlled release Purdue Pharma
Stamford, CT
tapentadol Johnson & Johnson musculoskeletal pain application submitted
controlled-release Pharmaceutical Research & (800) 817-5286
Development
Raritan, NJ
ZX002 Zogenix back pain Phase III
(hydrocodone San Diego, CA (866) 964-3649
controlled-release)
P S O R I A T I C A R T H R I T I S
Product Name Company Indication Development Status
apremilast Celgene psoriatic arthritis Phase III
Summit, NJ (see also Behcet’s syndrome, (908) 673-9000
rheumatoid arthritis, spondylitis)
Cimzia® UCB psoriatic arthritis Phase III
certolizumab pegol Smyrna, GA (see also spondylitis) (770) 970-7500
RG4934 Roche psoriatic arthritis Phase I
(anti-IL-17 mAb) Nutley, NJ (973) 235-5000
RO5310074 Roche psoriatic arthritis Phase I
Nutley, NJ (973) 235-5000
secukinumab Novartis Pharmaceuticals psoriatic arthritis Phase II
East H