Company: AZN
Filing Date: 2025-11-20
Form Type: 6-K
Source: 0001654954-25-013317
Chunk: 2

Company: ASTRAZENECA PLC
Filing Date: 2025-11-20
Form: 6-K
Chunk 2
---
 rate (ORR) of 20% (n=14/71, 95% confidence interval [CI]: 11, 31) compared to 5% with placebo (n=4/74, 95% CI: 2, 13; p=0.011) by cycle 16, with 86% of patients on Koselugo having an observed duration of response (DOR) of at least 6 months. After 12 cycles, patients on placebo were switched to Koselugo and patients on Koselugo remained on treatment for an additional 12 cycles. 1

The safety of Koselugo in the KOMET Phase III trial was consistent with its known profile and established use in paediatric patients. 2

Koselugo has been recently approved in the EU, Japan and other countries for the treatment of adult patients with NF1 who have symptomatic, inoperable PN based on data from the KOMET Phase III trial, and additional regulatory reviews are ongoing. In the US, Koselugo granules have recently been approved for paediatric patients one year of age and older with NF1 PN.

#### Notes

#### NF1
NF1 is a rare, progressive, genetic condition that is caused by a spontaneous or inherited mutation in the NF1 gene. 3,4 NF1 is associated with a variety of symptoms, including soft lumps on and under the skin (cutaneous neurofibromas) and, in up to 50% of patients, tumours called plexiform neurofibromas (PN) may develop on the nerve sheaths. 4,5 These PN can cause clinical issues such as disfigurement, motor dysfunction, pain, airway dysfunction, visual impairment and bladder or bowel dysfunction. 4,5

#### KOMET
KOMET is a global Phase III randomised, double-blind, placebo-controlled, multicentre trial designed to evaluate the efficacy and safety of Koselugo in adults with NF1 who have symptomatic, inoperable PN. The trial enrolled 145 adults from 13 countries across North America, South America, Europe, Asia and Australia, with participants' baseline characteristics, including gender and distribution of PN, reflective of the global adult NF1 patient population. Patients were enrolled and randomised to receive Koselugo or placebo (1:1) for 12 28-day cycles. Participants were required to have diagnosis of NF1, at least one symptomatic, inoperable PN measurable by