Belnacasan (VX-765) is a drug developed by

Vertex Pharmaceuticals Vertex Pharmaceuticals is an American biopharmaceutical company based in Boston, Massachusetts. It was one of the first biotech firms to use an explicit strategy of rational drug design rather than combinatorial chemistry. It maintains headqu ...

which acts as a potent and selective inhibitor of the enzyme

caspase 1 Caspase-1/Interleukin-1 converting enzyme (ICE) is an evolutionarily conserved enzyme that proteolytically cleaves other proteins, such as the precursors of the inflammatory cytokines interleukin 1β and interleukin 18 as well as the pyroptosi ...

. This enzyme is involved in

inflammation Inflammation (from la, inflammatio) is part of the complex biological response of body tissues to harmful stimuli, such as pathogens, damaged cells, or irritants, and is a protective response involving immune cells, blood vessels, and molec ...

and cell death, and consequently blocking its action may be useful for various medical applications, including treatment of

epilepsy Epilepsy is a group of non-communicable neurological disorders characterized by recurrent epileptic seizures. Epileptic seizures can vary from brief and nearly undetectable periods to long periods of vigorous shaking due to abnormal electrica ...

, arthritis, aiding recovery from

heart attack A myocardial infarction (MI), commonly known as a heart attack, occurs when blood flow decreases or stops to the coronary artery of the heart, causing damage to the heart muscle. The most common symptom is chest pain or discomfort which ma ...

and slowing the progression of Alzheimer's disease. Belnacasan is an orally active prodrug, being converted in the body to the active drug VRT-043198 (''O''-desethyl-belnacasan). However while belnacasan has proved well tolerated in human

clinical trial Clinical trials are prospective biomedical or behavioral research studies on human participants designed to answer specific questions about biomedical or behavioral interventions, including new treatments (such as novel vaccines, drugs, diet ...

s, it has not shown sufficient efficacy to be approved for use for any of the applications suggested to date, though research continues into possible future uses of this or similar drugs.

References

Enzyme inhibitors {{pharm-stub