September 27, 2010
Novartis reported positive results from a phase III trial of pasireotide for the treatment of Cushings disease. This global, randomized, double blind trial, PASPORT-CUSHINGS (PASireotide clinical trial PORTfolio - CUSHING'S disease), enrolled 162 subjects with persistent or recurrent Cushing's disease, as well as those with newly diagnosed Cushing's disease who were not candidates for surgery. The subjects received subcutaneous injections of placebo or pasireotide in doses of 600ug or 900ug twice daily for six months. The primary endpoint was the proportion of subjects who achieved normalization of urinary free cortisol (UFC) after six months. The primary endpoint was reached in the higher dose group: UFC levels were normalized in 26% of this dosing arm. After 12 months, the proportion of responders regardless of dose up-titration was 13.4% and 25.0%, respectively, for the 600ug and 900ug groups. The median reduction in UFC after six months was 47.9% for both groups. The median reduction in UFC after 12 months was 67.6% (600ug) and 62.4% (900ug). The treatment was generally well tolerated.