Phase 3 study of pasireotide for the treatment of Cushing's disease

Novartis announced that the results of a Phase 3 study of pasireotide (also known as SOM230) showed a reduction in cortisol levels in patients with Cushing's disease.  This trial, also known as PASPORT-CUSHINGS, evaluated the efficacy and safety of SOM230 in patients with persistent or recurrent Cushing's disease, as well as in patients with newly diagnosed Cushing's disease who are not candidates for surgery. The primary endpoint was the proportion of patients who achieved normalization of UFC after six months without dose up-titration relative to randomized dose. UFC is typically used to diagnose and monitor Cushing's disease. The primary endpoint was met for the higher dose tested (900μg sc twice daily). Secondary endpoints included safety, time to response, response duration and changes from baseline in clinical signs, symptoms, tumor volume and health-related quality of life.

After six months, the primary efficacy responder rate was 26.3% (95% confidence interval [CI], 16.6 to 35.9) and 14.6% (95% CI, 7.0 to 22.3), respectively, for the 900μg and 600μg groups. Based on pre-specified criteria (lower bound of 95% CI >15%), the 900μg group met the primary endpoint and the 600μg group did not meet the primary endpoint. After 12 months, the proportion of responders regardless of dose up-titration was 13.4% and 25.0%, respectively, for the 600μg and 900μg 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% (600μg) and 62.4% (900μg).

Pasireotide is an investigational pituitary-directed therapy that targets the cause of Cushing's disease, with the aim to control excess cortisol secretion and its debilitating complications. Pasireotide targets multiple subtypes of the receptor for somatostatin (sst), a hormone that controls the pituitary gland. Its highest affinity is to sst5, a receptor subtype frequently expressed by the pituitary tumors associated with Cushing's disease.

For more information call (800) 693-9993 or visit www.us.novartis.com.