Phase 3 Study Update of Signifor for Cushing's Disease

Novartis announced that the results of a Phase 3 study of Signifor (SOM230, pasireotide) showed normalized cortisol levels and showed clinical benefit in patients with Cushing's disease.

PASPORT-CUSHINGS (PASireotide clinical trial PORTfolio - CUSHING'S disease) was a prospective randomized, double-blind, Phase 3 study. The study evaluated the efficacy and safety of pasireotide in 162 adult patients with persistent or recurrent Cushing's disease and UFC levels greater than 1.5 times the upper limit of normal (ULN), as well as in patients with newly diagnosed Cushing's disease who are not candidates for surgery.

In the study, patients were randomized to receive pasireotide subcutaneous injection in doses of 900μg or 600μg twice daily. For the 900μg group, the study met the primary endpoint of normalizing urinary-free cortisol (UFC) levels, the key measure of biochemical control of the disease. UFC levels were normalized in 26.3% and 14.6% of patients with Cushing's disease randomized to receive pasireotide 900μg and 600μg twice daily, respectively, at six months of treatment. After 12 months of treatment, results confirmed the durability of the effect.

Study results also showed that cortisol levels decreased quickly in the majority of patients, with a median decrease of approximately 50% by month two, and remained stable in both groups through the end of the study. On average, as UFC levels were reduced, clinical manifestations of Cushing's disease improved including reduction of blood pressure, total cholesterol, weight and body mass index.

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.