Novartis reports phase 3 results of Afinitor study

More than one-third of patients treated with everolimus (Afinitor) tablets had a 50% or greater reduction in the size of non-cancerous brain tumors associated with tuberous sclerosis complex (TSC) versus 0% who were treated with placebo, according to a company statement from the drug’s manufacturer Novartis.

The phase 3 randomized, placebo-controlled, double-blind, parallel group, international, multicenter study, called EXIST-1 (Examining everolimus In a Study of TSC), examined the efficacy and safety of everolimus versus placebo for the treatment of patients with subependymal giant-cell astrocytomas (SEGAs) associated with TSC.

Patients were randomly assigned to receive either everolimus (n=78) or placebo (n=39). By the cut-off date of March 2, 2011, the median treatment duration was 9.6 months in the everolimus arm and 8.3 months in the placebo arm. The primary end point was overall SEGA response rate.

Everolimus targets the mTOR protein that acts as an important regulator of tumor cell division, blood vessel growth, and cell metabolism by inhibiting overactivity that can lead to the formation of non-cancerous tumors throughout the body.

"This study, which included SEGA patients from infancy to adulthood, provides compelling evidence of the impact of everolimus in reducing SEGA size with a tolerability profile consistent with the previous everolimus trial in this treatment setting," said Sergiusz Jozwiak, MD, PhD, a lead EXIST-1 investigator and professor, department of child neurology, The Children's Memorial Health Institute, Warsaw, Poland, in the press release. "This is very good news for patients and caregivers in many countries who currently may face brain surgery as the only treatment option for growing SEGAs."