Jazz Pharmaceuticals Presents Positive JZP-258 Phase 3 Study Data at World Sleep 2019
JZP-258 achieves primary and key secondary endpoints demonstrating highly statistically significant differences in weekly number of cataplexy attacks and Epworth Sleepiness Scale scores compared to placebo
JZP-258 is a novel oxybate product candidate with a unique composition of cations resulting in 92% less sodium than Xyrem? (sodium oxybate)
Narcolepsy is a chronic, debilitating neurological disorder characterized by excessive daytime sleepiness, and the inability to regulate sleep-wake cycles normally.1?It affects an estimated one in 2,000 people in?the United States, with symptoms typically appearing in childhood. It is estimated that more than 50% of people with narcolepsy have not been diagnosed.2?Studies have shown it may take 10 years or more for people with narcolepsy to receive a diagnosis.3Excessive daytime sleepiness is the primary symptom of narcolepsy and is present in all people with the disorder.4Excessive daytime sleepiness is characterized by the inability to stay awake and alert during the day resulting in drowsiness and unplanned lapses into sleep.2,4,5?There are five primary symptoms of narcolepsy, including excessive daytime sleepiness, cataplexy, disrupted nighttime sleep, sleep-related hallucinations, and sleep paralysis.6?While all people with narcolepsy experience excessive daytime sleepiness, not all individuals with narcolepsy experience all five symptoms.7,8 About?Cataplexy
Cataplexy, the most specific symptom of narcolepsy, is the sudden, generally brief (<2 minutes) loss of muscle tone with retained consciousness. It is usually triggered by strong emotions, such as laughter, surprise, or anger.7,8,9?Although many emotions can potentially lead to cataplexy, those associated with mirth are usually the most potent.7?Cataplexy occurs in about 70% of people with narcolepsy.10?Presentation differs widely among people with narcolepsy, ranging from sporadic partial attacks triggered by laughter to frequent complete collapse brought about by a variety of emotions.6,7?Complete collapse is less common.8?More commonly, episodes of cataplexy involve only certain muscle groups, such as arms and legs (e.g., knees buckling), the head and neck (e.g., head dropping), or the face and jaw (e.g., sagging, slurred speech, eyelid drooping).7,8,9,10 About?JZP-258
JZP-258 is an investigational product being evaluated in adults for the treatment of cataplexy and excessive daytime sleepiness in narcolepsy, as well as for the treatment of idiopathic hypersomnia. JZP-258 is a novel oxybate product candidate with a unique composition of cations resulting in 92% less sodium than Xyrem??(sodium oxybate). While the exact mechanism of action of JZP-258 is not fully understood, it is hypothesized that the therapeutic effects of JZP-258 on sleep/wake symptoms are mediated through modulation of GABAB?during sleep. About?Jazz?Pharmaceuticals?plc
Jazz Pharmaceuticals plc (Nasdaq:?JAZZ), a global biopharmaceutical company, is dedicated to developing life-changing medicines for people with limited or no options. As a leader in sleep medicine and with a growing hematology/oncology portfolio, Jazz has a diverse portfolio of products and product candidates in development, and is focused on transforming biopharmaceutical discoveries into novel medicines. Jazz Pharmaceuticals markets Sunosi? (solriamfetol), Xyrem??(sodium oxybate) oral solution, Defitelio??(defibrotide sodium), Erwinaze??(asparaginase Erwinia chrysanthemi) and Vyxeos??(daunorubicin and cytarabine) liposome for injection in the U.S. and markets Defitelio?(defibrotide), Erwinase??and Vyxeos??44 mg/100 mg powder for concentrate for solution for infusion in countries outside the U.S. For country-specific product information, please visit?www.jazzpharmaceuticals.com/medicines. For more information, please visit?www.jazzpharmaceuticals.com?and follow us on Twitter at?@JazzPharma. "Safe?Harbor"?Statement?under?the?Private?Securities Litigation?Reform?Act?of?1995?
This press release contains forward-looking statements, including, but not limited to, statements related to the company's belief that JZP-258, if approved, will provide a clinically meaningful benefit to patients prescribed oxybate and an important and novel product candidate for people with narcolepsy, and other statements that are not historical facts. These forward-looking statements are based on the company's current plans, objectives, estimates, expectations and intentions and inherently involve significant risks and uncertainties. Actual results and the timing of events could differ materially from those anticipated in such forward-looking statements as a result of these risks and uncertainties, which include, without limitation, risks and uncertainties associated with: the uncertain regulatory approval process, including the risk that the company's planned JZP-258 new drug application may not be submitted, accepted or approved by the FDA in a timely manner or at all;? effectively commercializing JZP-258, if approved; and other risks and uncertainties affecting the company and its development programs, including those described from time to time under the caption "Risk Factors" and elsewhere in Jazz Pharmaceuticals plc's Securities and Exchange Commission filings and reports (Commission File No. 001-33500), including the company's Quarterly Report on Form 10-Q for the quarter ended?June 30, 2019?and future filings and reports by the company. Other risks and uncertainties of which the company is not currently aware may also affect the company's forward-looking statements and may cause actual results and the timing of events to differ materially from those anticipated. The forward-looking statements herein are made only as of the date hereof or as of the dates indicated in the forward-looking statements, even if they are subsequently made available by the company on its website or otherwise. The company undertakes no obligation to update or supplement any forward-looking statements to reflect actual results, new information, future events, changes in its expectations or other circumstances that exist after the date as of which the forward-looking statements were made. References:
- Thorpy?M,?Krieger?A.?Delayed diagnosis?of?narcolepsy:?characterization and impact. Sleep Medicine.?2014;15(5):502?507.
- Ahmed?I,?Thorpy,?M.?Clinical?Features,?Diagnosis?and?Treatment?of?Narcolepsy.?Clin Chest?Med.?2010;31(2):371-381.
- Morrish E,?King M,?et?al.?Factors?associated with?a delay?in the diagnosis?of?narcolepsy. Sleep Medicine.?2004;5(1):37-41.
- Sateia,?M.?(2014).?International Classification of?Sleep Disorders-Third?Edition.?Chest, 146(5),?pp.1387-1394.
- Ahmed?I,?Thorpy,?M.?Sleepiness:?Causes,?Consequences?and?Treatment,?ed. Cambridge?University?Press.?2011:36-49.
- Pelayo R,?Lopes?MC.?Narcolepsy.?In:?Lee-Chiong?TL,?ed.?Sleep:?A Comprehensive Handbook.?Hoboken,?NJ:?Wiley?and Sons,?Inc.;?2006:145-149.
- American Academy?of?Sleep Medicine.?Central?disorders?of?hypersomnolence.?In:?The International?Classification of Sleep Disorders???Third Edition?(ICSD-3).?Darien, IL:?American Academy?of?Sleep Medicine;?2014.
- Ahmed?I,?Thorpy?M.?Clinical?features,?diagnosis?and treatment?of?narcolepsy.?Clin Chest Med.?2010;31(2):371-381.
- Overeem?S,?van Nues?SJ,?van der?Zande?WL,?et?al.?The clinical?features?of?cataplexy:?a questionnaire study?in narcolepsy?patients?with and without?hypocretin-1 deficiency.?Sleep Med.?2011;12(1):12-18.
- Overeem?S.?The?clinical?features of?cataplexy.?In:?Baumann?CR,?Bassetti?CL,?Scammell TE,?eds.?Narcolepsy:?Pathophysiology,?Diagnosis,?and Treatment.?New?York,?NY: Springer?Science+Business Media;?2011:283-290.