Phase III extension study Recorlev meets objectives in Cushing's syndrome

Strongbridge Biopharma announced the top-line findings from the extended evaluation phase of the pivotal Phase III SONICS study of Recorlev (levoketoconazole) for the potential treatment of endogenous Cushing’s syndrome. The purpose of the six-month extended evaluation phase was to evaluate the long-term safety, tolerability and benefit-risk during chronic use of Recorlev. In this exploratory evaluation, an observed-case analysis of completers was used to evaluate Mean Urinary Free Cortisol (mUFC) responders. At the end of the extended evaluation phase, normalization of mUFC was observed in 41% of patients, and normalization of, or at least 50% improvement in, mUFC was observed in 68% of patients. Clinically meaningful improvements in key cardiovascular risk markers (hemoglobin A1c, fasting glucose, total and LDL-cholesterol) were observed throughout the extended evaluation phase. Weight loss and reduction in body mass index (BMI) continued throughout the extended evaluation phase. Recorlev was generally well-tolerated during the extended evaluation phase and no new drug-related safety signals were observed.

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.

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