Strongbridge Biopharma plc Announces Publication of Secondary Endpoints Data from Phase 3 SONICS Study of RECORLEV™ (levoketoconazole) for the Treatment of Endogenous Cushing’s Syndrome in the Journal, Pituitary

~ RECORLEV™ (levoketoconazole) Treatment Resulted in Significantly Improved Clinician-Assessed Signs and Symptoms and Patient-Reported Outcomes of Cushing’s Syndrome ~

DUBLIN, Ireland and TREVOSE, Pa., Nov. 24, 2020 (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 that secondary endpoint results from the Phase 3 SONICS study of RECORLEV™ (levoketoconazole) for the potential treatment of endogenous Cushing’s syndrome were published online in advance of print publication in the peer-reviewed journal, Pituitary, the official publication of the Pituitary Society and the Growth Hormone Research Society.

“People with Cushing’s syndrome often experience a variety of problematic signs, symptoms, and comorbidities related to excess cortisol, testosterone or both,” said Fredric Cohen, M.D., chief medical officer of Strongbridge Biopharma. “The SONICS study provided important evidence that RECORLEV treatment was associated with durable improvements in several of the more common and bothersome signs and symptoms of the syndrome, including those that most commonly affect women, such as hirsutism and acne, which are related to increases in testosterone produced by the adrenal glands.”

The manuscript, entitled “Levoketoconazole improves clinical signs and symptoms and patient-reported outcomes in patients with Cushing’s syndrome,” includes analyses demonstrating that treatment with RECORLEV led to significant improvements in Cushing’s syndrome signs and symptoms, patient-reported quality of life (QoL) outcomes, depression symptoms, and testosterone levels.

• Significant mean improvements from baseline were noted at end of the Maintenance phase (Month 6) for acne, hirsutism (females only), and peripheral edema.
• These improvements were observed as early as Day 1 of Maintenance for hirsutism, the end of Month 1 of Maintenance for acne, and Month 4 of Maintenance for peripheral edema.
• By Month 3 of Maintenance, significant mean improvements in patient-reported QoL outcomes were observed and by Month 6, symptoms of depression had improved.
• 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.
• RECORLEV was generally well-tolerated; the most commonly reported adverse events during the dose-titration and maintenance phases were nausea (32%) and headache (28%).

The full manuscript can be accessed online at: https://www.strongbridgebio.com/therapeutic-focus/key-publications-posters/related-investigational-agent-recorlev/#heading_4

About Cushing’s Syndrome
Endogenous Cushing’s syndrome is a rare, serious and potentially lethal endocrine disease caused by chronic elevated cortisol exposure – often the result of a benign tumor of the pituitary gland. This benign tumor tells the body to overproduce high levels of cortisol for a sustained period of time, and this often results in undesirable physical changes. The disease is most common among adults between the ages of 30 to 50, and it affects women three times more often than men. Women with Cushing’s syndrome may experience a variety of health issues including menstrual problems, difficulty becoming pregnant, excess male hormones (androgens), primarily testosterone which can cause hirsutism (growth of coarse body hair in a male pattern), oily skin, and acne. Additionally, the internal manifestations of the disease are potentially life threatening. These include metabolic changes such as high blood sugar, or diabetes, high blood pressure, high cholesterol, fragility of various tissues including blood vessels, skin, muscle and bone, and psychologic disturbances such as depression, anxiety and insomnia. Untreated, the five-year survival rate is only approximately 50 percent.

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 mUFC 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 mUFC 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 RECORLEV
RECORLEV^® (levoketoconazole) is an investigational cortisol synthesis inhibitor in development for the treatment of patients with endogenous Cushing’s syndrome, a rare but serious and potentially lethal endocrine disease caused by chronic elevated cortisol exposure. RECORLEV is the pure 2S,4R enantiomer of ketoconazole, a steroidogenesis inhibitor. RECORLEV has demonstrated in two successful Phase 3 studies to significantly suppress serum cortisol and has the potential to be a next-generation cortisol inhibitor.

The Phase 3 program for RECORLEV includes SONICS and LOGICS: two multinational studies designed to evaluate the safety and efficacy of RECORLEV when used to treat endogenous Cushing’s syndrome. The SONICS study met its primary and secondary endpoints, demonstrating a statistically significant normalization rate of urinary free cortisol at six months. The LOGICS study, which met its primary endpoint, is a double-blind, placebo-controlled randomized-withdrawal study of RECORLEV that is designed to supplement the long-term efficacy and safety information supplied by SONICS. The ongoing long-term open label OPTICS study will gather further useful information related to the long-term use of RECORLEV.

RECORLEV has received orphan drug designation from the FDA and the European Medicines Agency for the treatment of endogenous Cushing’s syndrome.

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 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.

Forward-Looking Statements
This press release contains forward-looking statements within the meaning of the federal securities laws. The words “anticipate,” “estimate,” “expect,” “intend,” “may,” “plan,” “potential,” “project,” “target,” “will,” “would,” or the negative of these terms or other similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. All statements, other than statements of historical facts, contained in this press release, are forward-looking statements, including statements related to the secondary endpoint data from the SONICS study, the potential advantages of RECORLEV,  Strongbridge’s strategy, plans, outcomes of product development efforts and objectives of management for future operations. Forward-looking statements involve risks and uncertainties that could cause actual results to differ materially from those expressed in such statement, including risks and uncertainties associated with clinical development and the regulatory approval process, the reproducibility of any reported results showing the benefits of RECORLEV, the adoption of RECORLEV by physicians, if approved, as treatment for any disease and the emergence of unexpected adverse events following regulatory approval and use of the product by patients.  Additional risks and uncertainties relating to Strongbridge and its business can be found under the heading “Risk Factors” in Strongbridge’s Annual Report on Form 10-K for the year ended December 31, 2019 and its subsequent Quarterly Reports on Form 10-Q, as well as its other filings with the SEC. These forward-looking statements are based on current expectations, estimates, forecasts and projections and are not guarantees of future performance or development and involve known and unknown risks, uncertainties and other factors. The forward-looking statements contained in this press release are made as of the date of this press release, and Strongbridge Biopharma does not assume any obligation to update any forward-looking statements except as required by applicable law.

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