Merck and AstraZeneca announced on November 12, 2024, positive topline results from the Phase 3 KOMET trial. The study evaluated Koselugo in adults with neurofibromatosis type 1 (NF1) who have symptomatic, inoperable plexiform neurofibromas (PNs).
Koselugo demonstrated a statistically significant and clinically meaningful improvement in objective response rate (ORR), the study's primary endpoint, compared to placebo. The safety profile was consistent with observations in pediatric trials, with no new safety signals identified.
These results indicate the potential to expand Koselugo's use beyond pediatric patients to adults, for whom there are currently no approved targeted therapies. This development strengthens Merck's oncology and rare disease portfolio.
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