According to the Securities Times APP, today, Astrazeneca (AZN.US) and Merck (MRK.US) jointly announced the positive top-line results of the KOMET Phase 3 clinical trial. The analysis shows that Koselugo (selumetinib), jointly developed by the two companies, can significantly reduce the tumor volume in adult patients with symptomatic, inoperable plexiform neurofibromas (PN) of type 1 neurofibromatosis (NF1), achieving the primary endpoint of the trial.

NF1 is a rare, incurable genetic disease. NF1 is caused by mutations in the NF1 gene encoding the neurofibromin protein. This gene mutation can disrupt 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 to restore the disrupted signaling pathway, thereby alleviating the condition of NF1 patients.