Strongbridge Biopharma announced positive top-line results this week demonstrating the safety and efficacy of levoketoconazole, a steroidogenesis inhibitor used to treat endogenous Cushing’s syndrome, according to a company press release.
The findings from the 6-month, extended evaluation phase of the phase 3 SONICS study of levoketoconazole (Recorlev) demonstrated that the treatment was well-tolerated and no new drug-related safety signals were observed. At the end of the extended evaluation phase, 41% of patients achieved a normalization of mean urinary free cortisol, whereas 68% of patients achieved normalization or at least a 50% improvement in mean urinary free cortisol, according to the release.
“We are encouraged by the top-line results from the SONICS extended evaluation phase,” Fredric Cohen, MD, chief medical officer of Strongbridge Biopharma, said in the release. “Recorlev treatment was associated with no new clinically relevant liver-related findings or other new safety signals, while demonstrating long-term efficacy to reduce mean urinary free cortisol, as well as key cardiovascular risk markers, such as weight and LDL cholesterol.”
The single-arm, open-label SONICS study included 94 patients with Cushing’s syndrome who received an initial dose of levoketoconazole 150 mg twice per day, titrated in 150-mg increments with the goal of achieving a therapeutic dose. Once patients achieved mean urinary free cortisol normalization, titration was stopped. At the end of a 6-month maintenance phase, researchers measured mean urinary free cortisol response rate; patients continued in an extended evaluation phase for another 6 months to allow researchers to assess long-term safety and tolerability, as well as explore efficacy durability.
No patients experienced an increase in either alanine aminotransferase or aspartate aminotransferase greater than three times the upper limit of normal. There were no adverse events of special interest related to liver injury reported, according to the release. The most commonly reported treatment-emergent adverse events during the extended evaluation phase were arthralgia (7%), headache (7%), hypokalemia (7%) and nasopharyngitis (5%).
Cohen said the company is meeting with the FDA in the first quarter of 2019 to seek guidance on the regulatory path forward to obtain marketing approval for levoketoconazole. – by Regina Schaffer
Disclosure: Cohen is chief medical officer of Strongbridge Biopharma.