Positive top line results reported from SONICS study of Recorlev to treat Cushing's syndrome.- Strongbridge BioPharma

Strongbridge Biopharma plc, announced top-line results from the multinational, pivotal Phase III SONICS study evaluating Recorlev (levoketoconazole) for the treatment of endogenous Cushing�s syndrome.The open-label, single-arm SONICS study achieved statistical significance of its pre-specified primary endpoint, with 30 percent of patients achieving normalization of mean urinary free cortisol (UFC) following six months of maintenance treatment with Recorlev without a dose increase (p<.025).>

Sensitivity analyses as well as secondary and exploratory endpoints of UFC response were supportive of the primary endpoint. For key secondary endpoints of cardiovascular risk, including fasting blood glucose, hemoglobin A1C, total cholesterol, low density lipoprotein (LDL)-cholesterol, body weight and body mass index (BMI), Recorlev demonstrated statistically significant and clinically meaningful improvements from baseline (p<.0001 for each). safety and tolerability findings based upon data collected through the six-month maintenance phase indicate that recorlev was generally well tolerated.>

12 patients (12.8 percent) discontinued treatment with Recorlev due to adverse events (AEs). For the two most commonly reported AEs, no patients discontinued treatment due to nausea and one patient discontinued treatment due to headache. 14 patients (14.9 percent) reported one or more serious adverse events (SAEs), of whom four had SAEs deemed drug related by investigators. One patient death was reported and not considered drug related (colon cancer).

Comment: Ketaconazole an approved anti fungal can reduce cortisol levels and consequently has an off label use for Cushing's syndrome but the drug carries a boxed warning. Surgery to remove benign tumors is standard. Aside from ketoconazole, drug treatment options for controlling Cushing's include mitotane (HRA Pharma SA's Lysodren and generics) and Corcept Therapeutics Inc.'s Korlym (mifepristone). More recently, Novartis AG's Signifor LAR (pasireotide), received a supplementary FDA approval in June 2018 for Cushing's disease in patients for whom pituitary surgery is not an option or has not been curative.