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~ Subgroup Analysis of Patients with Comorbid Diabetes Mellitus and Cushing’s Syndrome Presented at Annual Meeting of the Endocrine Society (ENDO) Shows Potential Clinical Benefit of Treatment with RECORLEV™ (levoketoconazole) ~

~ Additional Analyses of RECORLEV Secondary Endpoints Presented at 16^th Annual International Pituitary Congress Continue to Demonstrate Clinically Meaningful Improvements in Cortisol Control ~

DUBLIN, Ireland and TREVOSE, Pa., March 25, 2019 (GLOBE NEWSWIRE) -- Strongbridge Biopharma plc, (Nasdaq: SBBP), a global commercial-stage biopharmaceutical company focused on the development and commercialization of therapies for rare diseases with significant unmet needs, today announced the presentation of new data analyses from the Phase 3 SONICS study of RECORLEV™ (levoketoconazole) for the potential treatment of endogenous Cushing’s syndrome. Secondary endpoint results were presented at the 16^th Annual International Pituitary Congress (IPC), held March 20 – 22, and a subgroup analysis was presented at the Annual Meeting of the Endocrine Society (ENDO), held March 23 – 26, in New Orleans, Louisiana.

“The SONICS study continues to generate important findings that help characterize the potential clinical benefit and role of levoketoconazole in the treatment of patients with endogenous Cushing’s syndrome,” said Maria Fleseriu, M.D., FACE, professor of Medicine and Neurological Surgery and director of the Oregon Health Sciences University Northwest Pituitary Center, who presented the data. “The subgroup analysis of Cushing’s syndrome patients with diabetes mellitus – among the more common comorbidities associated with the disease – not only shows similar levels of cortisol control compared to patients without, but also more pronounced improvements in key glycemia measures, such as hemoglobin A1c and fasting blood glucose, and other cardiovascular risk markers, such as LDL-cholesterol. In both treatment groups, the overall safety profile was acceptable.”

Highlights of Newly Presented SONICS Data at ENDO:

• Mean urinary-free cortisol (mUFC) normalization with RECORLEV was similar in patients with and without diabetes.
• Improvements in hemoglobin A1c (HbA1c) and fasting blood glucose (FBG) in the maintenance phase were more pronounced among patients with comorbid diabetes mellitus, while anti-diabetic medications were more often decreased than increased.
• Significant improvements in cardiovascular risk markers of low-density lipoprotein (LDL)-cholesterol, weight, body mass index (BMI), and waist circumference were seen in patients with and without diabetes mellitus. Additionally, improvement in LDL-cholesterol occurred without any new use of statins or increases in statin dose.
• The most common treatment-emergent adverse events (TEAEs) in patients with diabetes mellitus were nausea (58%), vomiting (19%), and urinary tract infection (17%); headache (36%), peripheral edema (22%) and hypertension (19%) were most common among patients without diabetes mellitus.

The poster, entitled Results from the Phase 3 Multicenter SONICS Study of Levoketoconazole: Subgroup Analysis of Cushing’s Syndrome in Patients with Diabetes Mellitus, can be accessed here.

Highlights of Newly Presented SONICS Data at IPC:

• RECORLEV treatment led to sustained reductions in both mUFC and late night salivary cortisol (LNSC) levels over a six-month maintenance treatment period.     
  • LNSC measures the free cortisol in the saliva at the time point when cortisol should be at its lowest level and helps detect the loss of diurnal rhythm. In this study, LNSC was much less commonly normalized than mUFC, indicating improvement in, but generally incomplete normalization, of cortisol diurnal rhythmicity. The incomplete return of diurnal rhythm is potentially clinically beneficial and represents the first data to show LNSC outcomes with RECORLEV. 
• Adrenocorticotrophic hormone (ACTH) levels in the subset of patients with Cushing’s disease increased about two-fold from baseline to the end of the maintenance phase.
• RECORLEV was generally well-tolerated with no unexpected safety signals observed during the study.

The poster, entitled Levoketoconazole in the Treatment of Endogenous Cushing’s Syndrome: Secondary Endpoint Results From a Phase 3 Study (SONICS), can be accessed here.

About the SONICS Study

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. Following a screening phase, SONICS has three treatment phases:

(1) Dose Titration Phase: Patients started RECORLEV at 150 mg twice daily (300 mg total daily dose) and titrated in 150 mg increments with the goal of achieving a therapeutic dose – a dose resulting in mean UFC normalization – at which point titration was stopped; (2) Maintenance Phase: The dose was fixed and should not have been changed other than for safety reasons or loss of efficacy. At the end of the six-month maintenance phase, the mean UFC response rate was measured; and (3) Extended Evaluation Phase: Patients continued on RECORLEV for another six months to evaluate long-term safety and tolerability and explore efficacy durability.

About Strongbridge Biopharma

Strongbridge Biopharma is a global commercial-stage biopharmaceutical company focused on the development and commercialization of therapies for rare diseases with significant unmet needs. Strongbridge’s commercial portfolio within its rare endocrine franchise includes RECORLEV™ (levoketoconazole), a cortisol synthesis inhibitor currently being studied in Phase 3 clinical studies for the treatment of endogenous Cushing’s syndrome, and veldoreotide extended release, a pre-clinical next-generation somatostatin analog being investigated for the treatment of acromegaly and potential additional applications in other conditions amenable to somatostatin receptor activation. Both RECORLEV and veldoreotide have received orphan drug designation from the FDA and the European Medicines Agency. The Company’s rare neuromuscular franchise includes KEVEYIS^® (dichlorphenamide), the first and only FDA-approved treatment for hyperkalemic, hypokalemic, and related variants of primary periodic paralysis. KEVEYIS has orphan drug exclusivity in the United States.

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Source: Strongbridge Biopharma plc