AstraZeneca (AZN.US) and Merck (MRK.US) jointly announced positive top-line results from the KOMET Phase 3 clinical trial.

Today, AstraZeneca (AZN.US) and Merck (MRK.US) jointly announced positive top-line results from the KOMET clinical Phase 3 trial. The analysis showed that the companies' jointly developed drug, Koselugo (selumetinib), significantly reduced tumor volume in adult patients with neurofibromatosis type 1 (NF1) who have symptomatic, unresectable plexiform neurofibromas (PNs), meeting the primary endpoint of the trial.

NF1 is a rare, incurable genetic disease caused by mutations in the NF1 gene, which encodes the neurofibromin protein. The 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.