A Phase III study of Afinitor® (everolimus) in patients with angiomyolipomas, associated with tuberous sclerosis complex (TSC), met its primary endpoint of best overall response rate.
Findings from the trial, known as EXIST-2, were presented recently at the International TSC Research Conference in Belfast, Northern Ireland.
Results of the 118-patient, randomised, placebo-controlled Phase III EXIST-2 (EXamining everolimus In a Study of TSC) trial showed 42% of patients (33 of 79) experienced a response in the everolimus arm versus 0% of patients (0 of 39) on placebo (p<0.0001) based on best overall response rate.
These results add to the recent positive Phase III data from a separate trial in patients with TSC, which demonstrated reduction in the size of non-cancerous brain tumours (SEGAs) with everolimus.
“For the first time, a large placebo-controlled study has focused specifically on angiomyolipomas in TSC patients, an area with clear unmet need,” said Dr. John Bissler, lead study author and Clark D. West Endowed Chair of Nephrology at Cincinnati Children’s Hospital Medical Centre, Cincinnati, Ohio, USA.
“In addition to the tumour reduction seen with everolimus in this trial, significant improvement in skin lesions including facial angiofibromas was observed, which can be a key concern for people living with TSC.”