Novel MEK blocker shows promise in rare genetic disorder

12 November 2024

AstraZeneca (LSE: AZN) and Merck & Co (NYSE: MRK) have announced that their jointly developed drug Koselugo (selumetinib) has shown positive outcomes in the KOMET Phase III trial.

The gold standard KOMET trial, which tests Koselugo in certain adults with neurofibromatosis type 1 (NF1), is the largest and most comprehensive global study of its kind.

Results indicate a statistically significant improvement in objective response rate (ORR) compared to placebo, achieving the primary endpoint. Koselugo is currently the only approved targeted therapy for NF1-related tumors in children, and the data suggest a possible expansion into adult care.

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