Strongbridge Biopharma’s pivotal phase 3 SONICS study evaluating levoketoconazole (Recorlev) for the treatment of endogenous Cushing’s syndrome achieved statistical significance of its pre-specified primary endpoint.
According to the company, 30% of patients achieved normalization of mean urinary free cortisol (UFC) after 6 months of maintenance treatment with levoketoconazole, without a dose increase (P<.25). The treatment was also associated with statistically significant and clinically meaningful improvements from baseline in fasting blood glucose, hemoglobin A1C, total cholesterol, low density lipoprotein (LDL)-cholesterol, body weight and body mass index (P<.0001 for each), all key secondary endpoints. A total of 12 patients (12.8%) discontinued the trial due to adverse events. Treatment-emergent adverse events included nausea (32%), headache (28%), peripheral edema (19%), hypertension (17%), and fatigue (16%), diarrhea (15%) and increased alanine aminotransferase (ALT; 15%) .
“The robust effect of Recorlev in normalizing or decreasing UFC levels was complemented by key secondary endpoint data showing significant improvements in multiple markers for cardiovascular risk,” said Maria Fleseriu, MD, FACE, professor of Neurological Surgery and Medicine, and director of the Oregon Health Sciences University Northwest Pituitary Center. “SONICS efficacy and safety results, including a low frequency (3.2%) of significant ALT elevations (more than 5 times above the upper limit of normal) without clinical sequelae and fully reversible, positions Recorlev, if approved, to play an important role in individualized medical therapy for Cushing’s syndrome, including consideration as a first-line treatment.”
Based on these findings, Strongbridge Biopharma plans on seeking accelerated approval of Recorlev with the Food and Drug Administration.
