PYR001

PYR001 is a first-in-class small molecule inhibitor of the E1 ubiquitin-activating enzyme (UBA1), currently under development by Reach Pharmaceuticals. UBA1 is the apex enzyme of the ubiquitin-proteasome system (UPS), responsible for the first step of the ubiquitination cascade. By selectively inhibiting UBA1, PYR001 prevents the tagging of proteins with ubiquitin, leading to a global disruption of protein homeostasis. This mechanism triggers significant endoplasmic reticulum (ER) stress and activates apoptotic pathways, particularly in cancer cells that are highly dependent on the UPS for survival. Reach Pharmaceuticals is advancing PYR001 through IND-enabling studies, targeting indications in hematologic malignancies, including multiple myeloma and acute myeloid leukemia, as well as various solid tumors.

Other names

UBA1 inhibitorUBA-1 inhibitorUBA 1 inhibitor