Aptabio Therapeutics, Inc. focuses on research and development of diabetic complications and intractable cancer drugs. We are developing drugs for major indications based on the NOX inhibitor discovery platform and Apta-DC platform technology. The company's platform technology and pipeline status are as follows

Diabetic Complications

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Diabetic Nephropathy (APX-115)

  • The conventional diabetic nephropathy drugs where mostly based on inflammation or fibrosis relief and since it was only the level of expanded indication for diabetes treatments and currently it was stopped using due to insufficient efficacy and severe adverse effects. Rather APX-115 is a new concept of first-in-class, which inhibits production of reactive oxygen species by inhibition of NADPH oxidase (NOX) and thus is a fundamental treatment to prevent damage to glomerular cells and tubular epithelial cells caused by oxidative stress.


  • Our product is a first-in-class innovative drug that is different from other NASH candidates being developed by several competitors. It is a fundamental treatment that simultaneously suppress inflammation improvement and fibrosis progression via inhibition of NADPH oxidase (NOX) which suppresses the production of reactive oxygen species (ROS)
  • We have compared with competing substances such as Obeticholic acid (Phase 3) in various pharmacodynamic and biomarker tests, the company has expanded its indications for APX-115 compound (in case of conducting Phase 2 clinical trial the project was named APX-311 after completion of Phase 1 clinical trial and the indication was expanded to NASH) was confirmed to be superior, and it is at the completion of Phase 1 clinical trial.

Diabetes Retinopathy(APX-1004)

  • Lucentis and Eylea, drugs for the treatment of diabetic retinopathy, are protein drugs targeting VEGF and PDGF. However, it is known that it is difficult to block the main cause of retinopathy. APX-1004, on the other hand, inhibits NOX which is the cause of diabetic retinopathy and prevents the onset of diabetic retinopathy from early on. It is expected to be a competitive new drug with excellent therapeutic effects. GLP toxicity study (non-clinical trial) is in progress.

Macular Degeneration(APX-1004F)

  • APX-1004F effectively inhibits neovascularization through Nox inhibition, and we believe that wet-AMD can be treated fundamentally. We are currently conducting non-clinical studies at a global GLP organization and are planning to starta phase 1/2 clinical study in 2019. By inhibiting Nox, we expect to develop a new therapeutic agent for wet-AMD by blocking the direct cause of the disease from an early stage.


  • The main cause of Atherosclerosis is the formation of ‘foam cells’ by oxidized low-density lipoprotein (LDL) which is a main risk factor. Currently, treatment for hyperlipidemia moderates the production of Foam cells, but APX-5278 treatsatherosclerosis by fundamentally blocks the‘foam cell formation’.

Pancreatic • Bladder(Apta-12)

  • Apta-12 is a novel therapeutic drug that is a G-quadruplex structured anti-nucleolin aptamer and is directly conjugated with gemcitabine. A comparisonresultwith gemcitabine and AS1411 (anti-nucleolin aptamer) independently, Apta-12 has shown excellent anticancer efficacy against pancreatic cancer and overcome gemcitabine resistance.

AML • MDS(Apta-16)

  • Apta-16 has proven to be a competitive anticancer drug in the field of AML or MDS therapy by securing superior efficacy as compared to cytarabine, as well as overcoming resistance to cytarabine, decitabine, and azacytidine. Currently, pre-clinical studieshave been completed for Phase 1 clinical study IND application and proven to be safe without any toxicity issues.