AstraZeneca PLC Sponsored ADR (AZN.US) and Merck & Co., Inc. (MRK.US) jointly announced positive top-line results from the KOMET Phase 3 clinical trial.

Today, Astrazeneca PLC Sponsored ADR (AZN.US) and Merck & Co., Inc. (MRK.US) jointly announced positive top-line results from the KOMET Phase 3 clinical trial. Analysis showed that Koselugo (selumetinib), developed by both companies, significantly reduced tumor volume in adult patients with symptomatic, inoperable plexiform neurofibromas (PN) associated with type 1 neurofibromatosis (NF1), meeting the trial's primary endpoint. NF1 is a rare and incurable genetic disorder caused by mutations in the NF1 gene, which encodes the neurofibromin protein. This gene mutation disrupts the RAS/MAPK signaling pathway (RAS-RAF-MEK-ERK), leading to tumor growth. MEK is a key protein kinase in the RAS/MAPK signaling pathway. Koselugo selectively inhibits MEK1 and MEK2, restoring the disrupted signaling pathway and alleviating the condition of NF1 patients.