Table 1. Repurposing pharma compounds.

Source: BCIQ: BioCentury Online Intelligence; National Center for Advancing Translational Sciences (NCATS) website

Company

Compound

Mechanism

Original indication(s)A

In commercial
developmentB

Literature-based indication(s)C

Sanofi (Euronext:SAN; NYSE:SNY)

Canosimibe (AVE5530)

Sterol O-acyltransferase (SOAT; ACAT) inhibitor

Phase III to treat hypercholesterolemia

1 to treat atherosclerosis

Alzheimer's disease (AD)

Pfizer Inc. (NYSE:PFE)

Senicapoc (PF-05416266; ICA-17043)

Potassium channel KCa3.1 (KCNN4) inhibitor

Phase IIa to treat asthma, sickle cell disease (SCD)

Not available

Inflammatory bowel disease (IBD), renal fibrosis

Abbott Laboratories (NYSE:ABT)

ABT-639

Calcium channel voltage-dependent T type-α 1H subunit (CACNA1H; Cav3.2) inhibitor

Phase II to treat diabetic neuropathic pain (DNP)

1 to treat neuropathic pain

Hypertension, obesity

Pfizer

Otenabant (CP-945598)

Cannabinoid CB1 receptor (CNR1) antagonist

Phase III to treat obesity in type 2 diabetes

4 to treat diabetes, obesity, dyslipidemia, liver disease

Pain

Eli Lilly and Co. (NYSE:LLY)

LY2828630

Cannabinoid CB2 receptor (CNR2) agonist

Phase II to treat pain in osteoarthritis (OA)

6 to treat pain, IBD, autoimmune disease, inflammatory disease

Brain inflammation, cocaine addiction, bone marrow transplant (BMT)

AstraZeneca plc (LSE:AZN; NYSE:AZN)

AZD1981

Prostaglandin D2 receptor subtype DP2 antagonist

Phase II to treat asthma, chronic obstructive pulmonary disease (COPD)

8 to treat asthma, COPD, allergy, inflammation

Alopecia (prostaglandin D2 receptor (CRTH2; GPR44; CD294) subtype unspecified)

AstraZeneca

AZD2423

CC chemokine receptor 2 (CCR2; CD192) antagonist

Phase IIa to treat pain and COPD

4 to treat diabetes, diabetic neuropathy, HIV/AIDS, inflammatory disease

Metastatic breast cancer, AD

Pfizer

PF-04136309

Phase II to treat HCV infection, pain in OA

Pfizer

CE-326597

Cholecystokinin A receptor (CCKAR; CCK1R) agonist

Phase II to treat obesity in type 2 diabetes

1 to treat IBD

Hyperglycemia

Bristol-Myers Squibb Co. (NYSE:BMY)

Pexacerfont (BMS-562086)

Corticotropin-releasing factor 1 (CRF1) antagonist

Phase II to treat major depressive disorder (MDD), generalized anxiety disorder (GAD), irritable bowel syndrome (IBS)

2 to treat depression, post-traumatic stress disorder (PTSD), glioblastoma multiforme (GBM), breast cancer, colon cancer

AD

AstraZeneca

Zibotentan (ZD4054)

Endothelin A receptor antagonist

Phase III to treat castration-resistant prostate cancer (CRPC); Phase II to treat non-small cell lung cancer (NSCLC), ovarian cancer; Phase I to treat solid tumors

5 to treat prostate cancer, pulmonary arterial hypertension (PAH), renal disease, shock/trauma

SCD, tumor imaging

Pfizer

Prinaberel (PF-00913086; ERB-041)

Estrogen receptor-b agonist

Phase II to treat endometriosis, rheumatoid arthritis (RA); Phase I to treat Crohn's disease

9 to treat menopause, pain, Parkinson's disease (PD), multiple sclerosis (MS), cancer, endocrine disease

Not available

Eli Lilly

LY500307

Phase II to treat lower urinary tract symptoms (LUTS) in benign prostatic hyperplasia (BPH)

Sanofi

Riferminogene pecaplasmid (temusi; NV1FGF; XRP0038)

Nonviral DNA plasmid encoding fibroblast growth factor 1 (FGF1)

Phase III to treat critical limb ischemia

6 to treat spinal cord injury (SCI), coronary artery disease (CAD), peripheral vascular disease (PVD), dermal ulcers, wounds, Huntington's disease (HD)

Diabetes, peripheral neuropathy

AstraZeneca

AZD7325

GABAA receptor agonist

Phase IIa to treat GAD

16 to treat insomnia, seizures, epilepsy, cognitive impairment in both schizophrenia and AD

Not available

AstraZeneca

Lesogaberan (AZD3355)

GABAB receptor agonist

Phase IIb to treat gastroesophageal reflux disease (GERD)

5 to treat incontinence, spasticity, autism, fragile X syndrome

Not available

Pfizer

PF-05190457

Ghrelin receptor (GHSR) antagonist

Phase I to treat type 2 diabetes

2 to treat obesity, diabetes

Alcohol dependence

AstraZeneca

AZD1656

Glucokinase (GCK; GK) activator

Phase IIb to treat type 2 diabetes

8 to treat diabetes

Not available

Bristol-Myers Squibb

BMS-820132

Phase I to treat type 2 diabetes

Pfizer

PF-03463275

Glycine transporter type 1 (GlyT1; SLC6A9) inhibitor

Phase II to treat schizophrenia

4 to treat schizophrenia, cognitive dysfunction

Not available

Sanofi

Ataciguat (HMR1766)

Soluble guanylyl cyclase (sGC) activator

Phase II to treat peripheral artery disease (PAD), neuropathic pain, angina pectoris

2 to treat heart failure, hypertension

Attention deficit hyperactivity disorder (ADHD)

Abbott

ABT-288

Histamine H3 receptor (HRH3) antagonist

Phase II to treat cognitive impairment in both schizophrenia and AD

8 to treat cognitive dysfunction, AD, schizophrenia, narcolepsy, cataplexy, rhinitis, obesity

Not available

Pfizer

PF-03654746

Phase II to treat ADHD, narcolepsy, rhinitis; Phase I to treat cognitive impairment in both schizophrenia and AD

GlaxoSmithKline plc (LSE:GSK; NYSE:GSK)

GSK1004723

Dual HRH1 and HRH3 antagonist

Phase II to treat allergic rhinitis

19 to treat allergy, rhinitis, conjunctivitis, urticaria, insomnia, emesis

Malaria

GSK

GSK835726

Pfizer

CE-210666

Serotonin (5-HT1B) receptor antagonist

Phase III to treat MDD

7 to treat migraine, ADHD

Not available

Pfizer

Elzasonan (CP-448187)

Phase II to treat MDD

Pfizer

Deramciclane (EGIS-3886; PH-670187)

Dual serotonin (5-HT2A) receptor and serotonin (5-HT2C) receptor antagonist

Phase III to treat GAD

6 to treat schizophrenia, psychosis, bipolar disorder, mania, depression, arterial thrombosis

Not available

1 to treat OA

Post-SCI muscle spasticity

Pfizer

PF-04995274

Serotonin (5-HT4) receptor agonist

Phase I to treat cognitive impairment in AD

12 to treat GERD, constipation, gastroparesis, IBS, gastrointestinal dysfunction, gastrointestinal motility disorder, cognitive dysfunction, AD

Not available

AstraZeneca

MEDI2338

IL-18 inhibitor

Phase I to treat COPD

1 to treat metabolic syndrome

MS, neuropathic pain

Pfizer

PF-04191834

5-Lipoxygenase (ALOX5; 5-LO) inhibitor

Phase II to treat asthma, pain in OA

1 to treat asthma

Inflammation, chronic myelogenous leukemia (CML)

Pfizer

SD-7300
(SC-81490)

Inhibitor of matrix metalloproteinase 2 (MMP2), MMP9 and MMP13

Phase I to treat myocardial infarction (MI), OA

Not available

Cancer, congestive heart failure (CHF), thrombosis, OA, neuropathic pain, acute myelogenous leukemia (AML), tuberculosis (TB), vaginal prolapse

AstraZeneca

AZD1236

Dual MMP9 and MMP12 inhibitor

Phase IIa to treat COPD

1 to treat COPD, MS, liver fibrosis

Asthma, emphysema, bacterial infection

Bristol-Myers Squibb

BMS-830216

Melanin-concentrating hormone receptor 1 (MCHR1; GPR24) antagonist

Phase I/II to treat obesity

Not available

IBD

AstraZeneca

AZD5904

Myeloperoxidase (MPO) inhibitor

Phase I to treat COPD and MS

1 to treat PD

Atrial fibrillation (AF)

Abbott

ABT-089

Nicotinic acetylcholine receptor a4b2 agonist

Phase II to treat ADHD, AD

2 to treat AD, ADHD

Not available

AstraZeneca

AZD0328

Nicotinic acetylcholine receptor α7 (CHRNA7) agonist

Phase IIa to treat cognitive impairment in schizophrenia

3 to treat ADHD, AD, schizophrenia, cognitive dysfunction

Epilepsy, brain damage associated with cardiac arrest/stroke

Johnson & Johnson (NYSE:JNJ)

JNJ-39393406

Phase I to treat AD, cognitive impairment in schizophrenia

GSK

GW274150

Inducible nitric oxide synthase 2 (NOS2; iNOS) inhibitor

Phase II to treat RA, migraine; Phase I to treat asthma

3 to treat neuropathic pain, postherpetic neuralgia (PHN), mucositis

Brain cancer, arthritis, inflammation, genetic association with psoriasis

Pfizer

SD-6010 (SC-84250)

Phase III to treat pain in OA, asthma

AstraZeneca

AZD7268

Opioid receptor d1 (OPRD1; DOR) agonist

Phase II to treat MDD

1 to treat pain in OA

Not available

Sanofi

AVE8134

Peroxisome proliferation-activated receptor-a (PPARA; PPARa) agonist

Phase II to treat type 2 diabetes

13 to treat diabetes, dyslipidemia, hyperlipidemia, hypertriglyceridemia, hypercholesterolemia, metabolic syndrome, cardiovascular disease

Not available

Sanofi

AVE0847

Dual PPARa and PPARg (PPARG) agonist

Phase I to treat type 2 diabetes

6 to treat diabetes, dyslipidemia, AD, cardiovascular disease

Not available

Pfizer

PF-05019702 (PRA-27)

Progesterone receptor antagonist

Phase I to treat endometriosis

1 to treat depression, Cushing's disease

Not available

AstraZeneca

AZD9056

Purinergic receptor P2X ligand-gated ion channel 7 (P2RX7; P2X7) antagonist

Phase II to treat COPD, RA, Crohn's disease

5 to treat pain, RA, OA, MS

Cancer, MI, heart failure, arterial thrombosis, graft-versus-host disease (GvHD), SCI, PD, neuroinflammatory diseases

Eli Lilly

LY2245461

Selective estrogen receptor modulator (SERM)

Phase unknown, to treat hot flashes in postmenopausal women

10 to treat osteoporosis, menopause, androgen deficiency, hypogonadism, breast cancer, pain

Cervical cancer

Sanofi

SAR115740

Transient receptor potential vanilloid 1 (TRPV1; VR1) antagonist

Phase I to treat acute and chronic pain

7 to treat pain, itch, rhinitis, incontinence

Not available

AstraZeneca

Recentin cediranib (AZD2171)

Inhibitor of VEGF receptor 1 (FLT1; VEGFR-1), VEGFR-2 (KDR/Flk-1) and VEGFR-3 (FLT4)

Phase II or III to treat multiple solid tumor types

4 to treat multiple solid tumors, lymphoma, sarcoma, age-related macular degeneration (AMD)

Not available

ADenotes the disease indication(s) in which the company has already tested the compound in the clinic; in most cases, the company discontinued development of the compound because of lack of efficacy, failure to meet trial endpoint(s) or for undisclosed reasons. BDenotes the number of compounds with the same mechanism of action that at least one company has or had in preclinical and/or clinical development to treat the listed indication(s). CDenotes additional disease indications in which a compound with the same mechanism of action could have efficacy, based on preclinical results reported in selected scientific literature.