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New Analysis Shows RECORLEV™ (levoketoconazole) Significantly Improved Clinician- and Patient-Reported Signs and Symptoms of Cushing’s Syndrome

DUBLIN, Ireland and TREVOSE, Pa., April 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 secondary endpoint results from the Phase 3 SONICS study of RECORLEV™ (levoketoconazole) for the potential treatment of endogenous Cushing’s syndrome at the 2019 American Association of Clinical Endocrinologists (AACE) Annual Scientific and Clinical Congress, held April 24 – 28, in Los Angeles, California.

“The SONICS data presented at AACE highlight Strongbridge’s commitment to developing effective treatments that translate into meaningful, real-world outcomes for patients,” said Fredric Cohen, M.D., chief medical officer of Strongbridge Biopharma. “People with Cushing’s syndrome often experience negative physical effects as well as emotional challenges brought on by the condition. These results are the first to demonstrate that treatment with RECORLEV significantly improves many of those symptoms, including acne, excess male-pattern hair growth in women, peripheral edema, and symptoms of depression. Further, these findings provide important insights from a patient perspective that may inform clinical decision-making for this population.”

Highlights of Newly Presented SONICS Data at AACE:

• Six-months of maintenance RECORLEV therapy led to significant improvements in the clinical signs and symptoms of Cushing’s syndrome including acne, hirsutism in women, and peripheral edema.
• A reduction in mean free-testosterone in women, consistent with improvements in clinical signs of hyperandrogenism was observed, and a modest increase in mean free-testosterone in men was observed.
• Significant mean improvements in patient-reported quality-of-life outcomes were observed and symptoms of depression improved.
• RECORLEV was generally well-tolerated, with 12.8 percent of patients discontinuing treatment due to an adverse event through the end of the six-month maintenance phase.

The poster, entitled Levoketoconazole in the Treatment of Endogenous Cushing’s Syndrome:
Improvements in Clinical Signs and Symptoms, Patient-Reported Outcomes and Associated Biochemical Markers in the Phase 3 SONICS Study, 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 Endogenous Cushing’s Syndrome
Endogenous Cushing’s syndrome (CS) is a rare but serious and potentially lethal endocrine disease caused by chronic elevated cortisol exposure. Most people with CS have a variety of signs and symptoms – many of which, when they occur by themselves, are common and do not necessarily point to an underlying disease; this makes recognition of CS difficult. Common presenting symptoms include weight gain or obesity, fatigue, muscle weakness, headaches, mood or sleep disturbances, facial rounding or redness, excess body hair growth in women or baldness in men, thinned skin with stretch marks, easy bruising and other skin changes including acne, mood or sleep disturbances and irregular periods or loss of libido. Patients are often found by their doctors to have new-onset or worsening of high blood pressure, abnormal levels of blood lipids, such as cholesterol, polycystic ovaries and abnormal blood glucose or diabetes. People with uncontrolled disease are seriously ill and have a 2- to 4-fold higher mortality rate than age- and gender-matched controls, mainly due to metabolic and cardiovascular complications. Treatment options for CS include surgery, radiation therapy, and medical treatment. CS most commonly affects adults ages 20-50 and is more prevalent in females, accounting for about 70 percent of all cases.

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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