Table 1. NCATS's first repurposing round.

Source (unless otherwise noted): BCIQ: BioCentury Online Intelligence; National Center for Advancing Translational Science (NCATS).


Academic partner(s)



New indication

Original indication(s)B

AstraZeneca plc (LSE:AZN; NYSE:AZN)

University of Virginia

Zibotentan (ZD4054)

Endothelin A receptor antagonist

Peripheral artery disease (PAD)

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

Baylor College of Medicine

Saracatinib (AZD0530)

Src inhibitor

Lymphangioleiomyomatosis (LAM)

Phase IIb to treat ovarian, breast, prostate and renal cancer

Yale University

Alzheimer's disease (AD)

Eli Lilly and Co. (NYSE:LLY)

Indiana University


Estrogen receptor-b agonist


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

Johnson & Johnson (NYSE:JNJ)

Virginia Commonwealth University; University of Pittsburgh


Nicotinic acetylcholine receptor α7 (CHRNA7) agonist2

Smoking addiction

Phase I to treat AD and cognitive impairment in schizophrenia

Pfizer Inc. (NYSE:PFE)

The University of Rhode Island, Kingston


Ghrelin receptor (GHSR) antagonist

Alcohol addiction

Phase I to treat type 2 diabetes

Yale University


Glycine transporter type 1 (GlyT1; SLC6A9) inhibitor

Cognitive impairment in schizophrenia

Phase II to treat negative symptoms in schizophrenia

Sanofi (Euronext:SAN; NYSE:SNY)

Kennedy Krieger Institute; University of Washington


Transient receptor potential vanilloid 1
(TRPV1; VR1) antagonist3-7

Duchenne muscular dystrophy (DMD)3-5

Phase I to treat acute and chronic pain

Mayo Clinic

Aortic stenosis6,7

ANCATS did not disclose compounds and/or mechanisms for all projects; in those instances, the cited reference by the academic partner and other research groups reports a potential therapeutic target in the new indication that corresponds to the mechanism of a compound the pharma originally provided to the NCATS program, from which one can infer the identity of the compound in the project receiving the NCATS award. BDenotes the disease indication 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 endpoints or for undisclosed reasons.