PII ROAR study, which completed in Dec 21, recruited in the US, Canada, Japan, Republic of Korea and Europe (not UK). It included adults with with rare cancers including anaplastic thyroid cancer, biliary tract cancer, gastrointestinal stromal tumor, non-seminomatous germ cell tumour/non-geminomatous germ cell tumour, hairy cell leukaemia, World Health Organisation (WHO) Grade 1 or 2 glioma, WHO Grade 3 or 4 (high-grade) glioma, multiple myeloma, and adenocarcinoma of the small intestine, with BRAF V600E positive-mutations .
Tafinlar and Mekinist showed tumour shrinkage benefits in several cancer types in two clinical trials. Across 131 patients enrolled in the PII ROAR study and Arm H of the NCI-MATCH trial, the combination triggered responses in biliary tract cancer, high-grade and low-grade glioma, ovarian cancer, small intestine adenocarcinoma and others. Plus, in a small trial (Study X2101), the doublet showed an acceptable safety profile and some tumour shrinkage ability in paediatric patients, mainly those with pre-treated glioma .