AstraZeneca announces Koselugo met primary endpoing in KOMET Phase III trial

High-level results of KOMET, the largest, global randomised double-blind placebo-controlled multicentre Phase III trial in adults with neurofibromatosis type 1 who have symptomatic, inoperable plexiform neurofibromas showed that Koselugo, an oral, selective MEK inhibitor, met its primary endpoint, demonstrating a statistically significant and clinically meaningful objective response rate versus placebo in these adult patients. NF1 is a rare, progressive genetic condition affecting an estimated 1.7 million individuals worldwide, approximately 70% of whom are adults. In 30-50% of patients, tumours develop on the nerve sheaths and may cause debilitating symptoms. NF1 is usually diagnosed in early childhood, however, NF1 often progresses into adulthood. There are no approved treatments for adults, leaving many to experience disfigurement, dysfunction, persistent pain or endure multiple surgeries. In the trial, ORR was defined as the percentage of patients with confirmed complete response or partial response by cycle 16 as determined by independent central review per response evaluation in neurofibromatosis and schwannomatosis criteria. The safety profile of Koselugo in this study was consistent with that observed in clinical trials among children and adolescents. No new safety signals were identified. Alexion, AstraZeneca (AZN) Rare Disease will share these data with regulatory authorities and present at a forthcoming medical meeting. AstraZeneca and MSD are jointly developing and commercialising Koselugo globally.