Rare Daily Staff

Strongbridge Biopharma reported positive top-line findings from the extended evaluation phase of the pivotal late-stage study of its experimental drug Recorlev for the treatment of endogenous Cushing’s syndrome.

Endogenous Cushing’s syndrome is typically caused by pituitary adenomas that causes sustained, elevated levels cortisol, a hormone that plays an essential role in the body’s response to stress. The condition can cause a wide range of health problems including heart attack, stroke, bone loss, high blood pressure, depression, memory loss, and diabetes. About 65,000 people in the United States and European Union have the condition.

Recorlev is a cortisol synthesis inhibitor. It has received orphan drug designation from the U.S. Food and Drug Administration and the European Medicines Agency for the treatment of endogenous Cushing’s syndrome.

The six-month extended evaluation phase is part of the SONICS study and was intended to evaluate the long-term safety, tolerability and benefit-risk during chronic use of Recorlev. SONICS is an open-label, phase 3 study of Recorlev as a treatment for endogenous Cushing’s syndrome that enrolled 94 patients at centers in North America, Europe and the Middle East.

A total of 60 out of 61 study participants who completed the maintenance phase elected to participate in the extended evaluation phase. Of the 60 patients that entered the extended evaluation phase, 46 patients completed it. Data were collected twice, at three-month intervals, which is common practice for the long-term follow-up of chronic medical therapy for endogenous Cushing’s syndrome.

At the end of the extended evaluation phase, normalization of mUFC was observed in 41 percent of patients, and normalization of, or at least 50 percent improvement in, mUFC was observed in 68 percent of patients

Clinically meaningful improvements were also seen in key cardiovascular risk markers (hemoglobin A1c, fasting glucose, total and LDL-cholesterol) throughout the extended evaluation phase, the company said. 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. Four patients (6.7 percent) discontinued due to adverse events.

“We are encouraged by the top-line results from the SONICS extended evaluation phase. Recorlev (levoketoconazole) 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, or mUFC, as well as key cardiovascular risk markers such as weight and LDL-cholesterol,” said Fredric Cohen, chief medical officer of Strongbridge Biopharma.

Cohen said the company expects to meet with the FDA in the first quarter of 2019 to seek guidance on the regulatory path forward to obtain marketing approval for Recorlev for the treatment of endogenous Cushing’s syndrome. The company expects to provide an update in the second quarter of 2019.

Photo: Fredric Cohen, chief medical officer of Strongbridge Biopharma

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