varoglutamstat

Varoglutamstat is an orally administered, first-in-class small molecule inhibitor of human glutaminyl cyclases (QPCT and QPCTL), developed primarily by Vivoryon Therapeutics. It was originally designed to treat early Alzheimer's disease (AD) by blocking the formation of N3pE amyloid, a particularly neurotoxic variant of Abeta peptides implicated in AD pathology. The drug acts upstream of other anti-Abeta therapies by inhibiting the enzyme responsible for generating this toxic peptide, thereby aiming to prevent plaque formation rather than removing existing plaques. Additionally, varoglutamstat inhibits QPCTL-mediated conversion of CCL2 into its proinflammatory form pE‑CCL2, potentially reducing neuroinflammation and tau pathology—both key features in AD progression. Recent clinical data have shown that while it did not meet primary endpoints in AD trials, varoglutamstat demonstrated statistically significant improvements in kidney function (eGFR) in elderly patients and those with diabetes or diabetic kidney disease (DKD). As a result, development focus has shifted toward kidney diseases such as DKD[3][4][5][6][7][8].

Brand names

varoglutamstat

Other names

varoglutamstatPQ912PQ-912PQ 9121276021-65-8UNII-E3DSH8LL4LUNII-E-3DSH8LL4LUNII-E 3DSH8LL4LE3DSH8LL4LE-3DSH8LL4LE 3DSH8LL4L