NMS-03597812

NMS-03597812 is an orally administered small molecule inhibitor targeting PERK (PKR-like endoplasmic reticulum kinase), also known as EIF2AK4. By inhibiting PERK activity, the drug blocks activation of the PERK pathway and disrupts the unfolded protein response (UPR) stress adaptation in tumor cells. This inhibition can lead to tumor cell apoptosis and suppression of tumor growth[1][3][5]. The drug was developed by Nerviano Medical Sciences for oncology indications, specifically relapsed or refractory multiple myeloma and acute myeloid leukemia. Clinical trials have evaluated its safety, pharmacokinetics, and preliminary antitumor activity in these patient populations[2][3][4][5]. As of September 2024, development for multiple myeloma has been discontinued due to strategic reasons after initial Phase I studies established reasonable pharmacokinetics and safety[7], while development continues in acute myeloid leukemia at Phase I[3][5].