Merck and AstraZeneca announce results of Koselugo in neurofibromatosis trial

AstraZeneca (AZN) Rare Disease, Alexion and Merck (MRK) announced positive topline results from the Phase 3 KOMET trial, a Phase 3 trial in adults with neurofibromatosis type 1 – NF1 – who have symptomatic, inoperable plexiform neurofibromas, or PN. Topline results showed that Koselugo demonstrated a statistically significant and clinically meaningful improvement in objective response rate, the study’s primary endpoint, versus placebo, in these adult patients. Neurofibromatosis type 1 is a rare, progressive genetic condition affecting an estimated 1.7 million people worldwide, approximately 70% of whom are adults. There are no approved treatments for adults. 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 Rare Disease will share these data with regulatory authorities and present these data at a forthcoming medical meeting. AstraZeneca and Merck are jointly developing and commercializing Koselugo globally.