The trial enrolled 145 individuals with baseline characteristics, such as gender and distribution of PNs, representative of the global adult NF1 patient population. Credit: TimeImage Production/Shutterstock.

Merck & Co (MSD), Alexion, and AstraZeneca Rare Disease have reported positive topline outcomes from the Phase III KOMET trial, assessing the efficacy and safety of oral Koselugo for treating adults with neurofibromatosis type 1 (NF1) with symptomatic inoperable plexiform neurofibromas (PN).

The randomised, global, multi-centre, double-blind, placebo-controlled trial enrolled 145 individuals with baseline characteristics, such as gender and distribution of PNs, representative of the global adult NF1 patient population.

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Participants were enrolled from 13 countries across Australia, Asia, South America, Europe, and the US.

The trial’s primary endpoint, the objective response rate (ORR), showed a statistically significant and meaningful improvement clinically in patients treated with Koselugo, compared to those receiving a placebo.

All the subjects were required to have at least one symptomatic and inoperable PN, measurable by volumetric magnetic resonance imaging (MRI) analysis.

The ORR was determined using the percentage of patients with a confirmed complete response or partial response by cycle 16, as per independent central review (ICR) based on response evaluation in neurofibromatosis and schwannomatosis (REiNS) criteria.

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In the trial, the therapy’s safety profile was consistent with previous clinical trials among paediatric and adolescent patients, with no new safety signals observed.

AstraZeneca and MSD are co-developing and commercialising the therapy across the globe.

Koselugois has already been approved in the US, European Union, China, and Japan, and has received orphan drug status in these regions for the treatment of paediatric patients with NF1 PN.

Alexion CEO Marc Dunoyer said: “These promising results demonstrate that Koselugo, the first and only approved targeted therapy for certain children with NF1 plexiform neurofibromas, now has the potential to benefit adult patients, for whom there are no approved targeted therapies.

“As the largest and only global placebo-controlled Phase III trial in adults with NF1 plexiform neurofibromas, KOMET reinforces our leadership in advancing potential treatment options for people living with this debilitating disease. We look forward to sharing these findings with regulatory authorities.”

MSD reported the discontinuation of its two Phase III clinical trials for Keytruda (pembrolizumab) in August 2024.