Strongbridge Biopharma plc to Present KEVEYIS® (dichlorphenamide) Primary Periodic Paralysis Data at the 2018 American Academy of Neurology Annual Meeting
~ Post-Hoc Analysis of Phase 3 Placebo-Controlled Study of KEVEYIS Showed Adolescents had Similar Improvements in Short-Term Attack Rates as Adults ~
~ Pooled Analysis of Two Phase 3 Studies Further Demonstrate the Clinical Benefit and Use of KEVEYIS in Patients with Hypokalemic and Hyperkalemic Primary Periodic Paralysis ~
DUBLIN, Ireland and TREVOSE, Pa., March 05, 2018 (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 new clinical analyses for KEVEYIS® (dichlorphenamide), the first and only U.S. Food and Drug Administration (FDA) approved treatment for hyperkalemic, hypokalemic, and related variants of primary periodic paralysis, will be presented at the 70th American Academy of Neurology (AAN) Annual Meeting from April 21-27 in Los Angeles, California.
“These new analyses help to validate the overall clinical profile of KEVEYIS in a variety of patient populations and disease variants,” said Fredric Cohen, M.D., chief medical officer of Strongbridge Biopharma. “Primary periodic paralysis is a rare genetic neuromuscular condition with significant unmet need and we are encouraged by the consistency of positive data for KEVEYIS and the potential value it can bring to the underserved periodic paralysis community.”
In a poster presentation entitled, “Efficacy and Safety of Dichlorphenamide in Adolescent Patients with Primary Periodic Paralysis,” Emma Ciafaloni, M.D., lead investigator and professor of neurology and pediatrics at the University of Rochester Medical Center, will present new findings from a post-hoc analysis of a Phase 3, placebo-controlled, cross-over trial comparing the effects of dichlorphenamide in adolescent and adult patients with primary periodic paralysis. The results show that adolescents had similar improvements in short-term attack rates and similar side effects as adults.
In an oral poster presentation entitled, “Efficacy of Dichlorphenamide in Primary Periodic Paralysis: Pooled-Data Analysis of Two Phase 3 Clinical Trials,” Perry B. Shieh, M.D., Ph.D., lead investigator and associate professor of the neuromuscular program at the University of California at Los Angeles, David Geffen School of Medicine, will present a pooled analysis of efficacy data from two Phase 3 placebo-controlled trials evaluating dichlorphenamide in 138 patients with primary periodic paralysis. The results add to the growing body of clinical evidence for KEVEYIS and supports its efficacy across disease variants by measuring changes from baseline in weekly attack rates.
• Poster #955 – Efficacy and safety of dichlorphenamide in adolescent patients with primary periodic paralysis; Emma Ciafaloni; Poster Session P3, April 24, 2018 – 05:30 p.m.-7:00 p.m. PT
Detailed findings include:
– Dichlorphenamide treatment resulted in median changes from baseline in weekly attack rate of −0.96 [CI: ‒1.63, ‒0.88] in adolescents vs ‒0.83 [CI: ‒2.75, ‒0.26] in adults.
– Median changes from baseline in severity-weighted weekly attack rate were ‒2.25 [CI: −3.50, −1.38] in adolescents vs ‒1.17 [CI: −3.38, ‒0.63] in adults.
– Three of six adolescents with safety data reported ≥1 solicited side effect with dichlorphenamide treatment; no side effects were dose-limiting or resulted in study withdrawal.
– The frequency of each side effect reported by the adolescents was similar in adults.
• Oral Poster #958 – Efficacy of Dichlorphenamide in Primary Periodic Paralysis: Pooled-Data Analysis of Two Phase 3 Clinical Trials; Perry B. Shieh; Poster Session P2, April 23, 2018 – Oral Presentation: 12:25 p.m. – 12:30 p.m. PT / Poster Presentation: 05:30 p.m.-7:00 p.m. PT
Detailed findings include:
– Median weekly attack rate improved with dichlorphenamide vs placebo: Median change from baseline was −0.75 vs +0.13, P=0.0004; treatment-effect size −1.22 [CI: −3.15, −0.38]. Median relative change from baseline was −51.67% for dichlorphenamide and 0.00% for placebo, P<0.0001; treatment-effect size −65.31% [CI: −93.17%, −34.40%].
– Median severity-weighted attack rate also improved with dichlorphenamide: Median change from baseline was −0.67 vs +0.55, P=0.0049; treatment-effect size −1.63 [CI: −4.47, −0.09]. Median relative change from baseline was −45.18% vs +16.05%, P=0.0002; treatment-effect size −60.85% [CI: −88.46%, −23.53%].
Primary periodic paralysis is a rare inherited disorder characterized by recurrent, progressive, and debilitating episodes of muscle weakness and temporary paralysis. Primary periodic paralysis is very rare, affecting about 5,000 to 6,000 individuals in the United States.
About Strongbridge Biopharma plc
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 neuromuscular and rare endocrine franchises includes KEVEYIS®(dichlorphenamide), the first and only FDA-approved treatment for hyperkalemic, hypokalemic, and related variants of Primary Periodic Paralysis, and MACRILEN™ (macimorelin), the first and only FDA-approved oral drug indicated for the diagnosis of Adult Growth Hormone Deficiency. KEVEYIS has orphan drug exclusivity status in the U.S. to August 2022. MACRILEN has been granted orphan drug designation in the U.S. and has patents with expiration dates through late 2027. The Company’s rare endocrine franchise also includes a clinical-stage pipeline of therapies: RECORLEV™ (levoketoconazole), a cortisol synthesis inhibitor currently being studied for the treatment of endogenous Cushing’s syndrome, and veldoreotide, a next-generation somatostatin analog being investigated for the treatment of acromegaly, with potential additional applications in other conditions amenable to somatostatin receptor activation. Both RECORLEV and veldoreotide have received orphan designation from the U.S. Food and Drug Administration and the European Medicines Agency. For more information, visit www.strongbridgebio.com.
KEVEYIS® (dichlorphenamide) is indicated for the treatment of primary hyperkalemic periodic paralysis, primary hypokalemic periodic paralysis, and related variants. In clinical studies, the most common side effects of KEVEYIS were a numbness or tingling, difficulty thinking and paying attention, changes in taste, and confusion. These are not all of the possible side effects that you may experience with KEVEYIS. Talk to your doctor if you have any symptoms that bother you or do not go away. You are encouraged to report side effects to Strongbridge Biopharma at 1-855-324-8912, or to the FDA at 1-800-FDA-1088 or visit www.fda.gov/medwatch/. For additional KEVEYIS important safety information and the full prescribing information visit www.keveyis.com.
This press release contains forward-looking statements that involve substantial risks and uncertainties. All statements, other than statements of historical facts, contained in this press release, are forward-looking statements. These statements relate to future events and involve known and unknown risks, including, without limitation, uncertainties regarding Strongbridge’s strategy, plans, anticipated investments, costs and results, product development and commercialization efforts and objectives of management for future operations. 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. 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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Source: Strongbridge Biopharma