DUBLIN, Oct. 31, 2024 /PRNewswire/ -- Jazz Pharmaceuticals plc (Nasdaq: JAZZ) today announced that eight abstracts presenting data from across its sleep portfolio were featured at the 37th annual Psych Congress, held in Boston from October 29 through November 2, 2024. The data includes the top-line results of the Phase 4 DUET (Develop hypersomnia Understanding by Evaluating low-sodium oxybate Treatment) trial, evaluating the effectiveness of low-sodium oxybate on key sleep outcomes in adults with narcolepsy or idiopathic hypersomnia (IH).

都柏林，2024年10月31日/PRNewswire/--Jazz Pharmaceuticals plc（纳斯达克：Jazz）今天宣布，在2024年10月29日至11月2日于波士顿举行的第37届年度心理大会上，展示了其睡眠组合数据的八篇摘要。数据包括第4阶段DUET（通过评估低羟丁酸钠治疗来发展嗜睡理解）试验的最重要结果，该试验评估了低羟丁酸钠对发作性睡病或特发性嗜睡（IH）成年人关键睡眠结局的有效性。

Notably, these DUET data are the first to show prospective improvements on excessive daytime sleepiness (EDS), as well as key polysomnography (PSG) outcomes of sleep disruption, among adults with narcolepsy treated with Xywav® (calcium, magnesium, potassium, and sodium oxybates) oral solution. Further, new DUET data evaluating nighttime Xywav treatment in adults with IH demonstrate clinical improvements on daytime symptoms, including sleep inertia measured by the Patient Global Impression of Change (PGIc). .

值得注意的是，这些二重奏数据首次显示，在用Xywav®（钙，镁，钾和羟丁酸钠）治疗的发作性睡病成人中，白天过度嗜睡（EDS）以及睡眠中断的关键多导睡眠图（PSG）结果的前瞻性改善口服溶液。此外，评估IH成人夜间Xywav治疗的新DUET数据显示，白天症状的临床改善，包括由患者全球变化印象（PGIc）测量的睡眠惯性。。

The DUET trial is a Phase 4, prospective, single-arm, open-label study to assess the effect of Xywav treatment on EDS, polysomnography parameters, and functional outcomes in adults with narcolepsy or IH. Observed adverse events were consistent with the known safety profile of Xywav, with the most common including nausea, dizziness and headache..

DUET试验是一项4期前瞻性单臂开放标签研究，旨在评估Xywav治疗对发作性睡病或IH成人EDS，多导睡眠图参数和功能结局的影响。观察到的不良事件与已知的Xywav安全性一致，最常见的包括恶心，头晕和头痛。。

'The new DUET data presented today demonstrate the impact of low-sodium Xywav treatment on key narcolepsy and idiopathic hypersomnia symptoms,' said Logan Schneider, MD, adjunct clinical associate professor of sleep medicine, Stanford Sleep Center and Consultant Neurologist, Stanford/VA Alzheimer's Center.

斯坦福睡眠中心睡眠医学副教授兼斯坦福/弗吉尼亚州阿尔茨海默氏病中心顾问神经学家洛根·施奈德医学博士说：“今天公布的新二重唱数据证明了低钠Xywav治疗对关键发作性睡病和特发性嗜睡症状的影响。”。

'These data build on our confidence that appropriate treatment can meaningfully impact the outcomes that matter to patients and their functioning.'.

“这些数据建立在我们的信心之上，即适当的治疗可以有意义地影响对患者及其功能至关重要的结果。”。

Presentation highlights include:

演讲亮点包括：

Xywav DUET Narcolepsy Top-line Results (P166): Novel analysis of primary and key secondary outcomes in participants with narcolepsy show improvements in daytime and nighttime symptoms with Xywav treatment compared to baseline across daytime sleepiness and PSG-based measures of sleep disruption, including sleep stage shifts from deeper to lighter stages of sleep, time spent in deep sleep, and number of awakenings.

。

Specifically, the statistically significant change (least square mean, standard error) from baseline on the Epworth Sleepiness Scale (ESS) was −7.7 (0.9), P<0.0001, while changes for total shifts from deeper to lighter stages of sleep, N3 sleep (deep sleep) duration in minutes, and number of awakenings were −13.1 (3.0), P<0.0001; 45.0 (8.8), P<0.0001; and −3.2 (0.9), P=0.0015, respectively (N=34 each)..

具体而言，Epworth嗜睡量表（ESS）与基线相比，统计学上显着的变化（最小二乘平均值，标准误差）为-7.7（0.9），P<0.0001，而从睡眠深度到较轻阶段的总变化，N3睡眠（深度睡眠）持续时间（分钟）和觉醒次数为-13.1（3.0），P<0.0001；45.0（8.8），P＜0.0001；和-3.2（0.9），P=0.0015（每个N=34）。。

Xywav DUET IH Top-line Results (P165): First presentation of primary and key secondary outcomes in participants with IH show Xywav treatment resulted in improvements in EDS, measured by ESS and Idiopathic Hypersomnia Severity Scale (IHSS). Additionally, improvements were observed across PGIc measures of overall symptoms, with new data on sleep inertia measured within the PGIc.

Xywav DUET IH顶级结果（P165）：IH参与者的主要和关键次要结果的首次介绍显示，Xywav治疗导致EDS的改善，这是通过ESS和特发性嗜睡严重程度量表（IHSS）测量的。此外，在PGIc总体症状测量中观察到改善，在PGIc内测量了有关睡眠惯性的新数据。

Statistically significant changes (least square mean, standard error) in ESS (N=40) and IHSS (N=36) from baseline to end of treatment were −8.4 (0.7), P<0.0001 and −15.5 (1.5), P<0.0001, respectively. Most participants reported improvement (very much, much, minimally) on the PGIc overall (94.6%; N=37) and PGIc sleep inertia inventory (81.1%; N=37)..

从基线到治疗结束，ESS（N=40）和IHSS（N=36）的统计学显着变化（最小二乘平均值，标准误差）分别为-8.4（0.7），P<0.0001和-15.5（1.5），P<0.0001。大多数参与者报告了PGIc总体（94.6%；N=37）和PGIc睡眠惯性量表（81.1%；N=37）的改善（非常多，很少）。。

'These DUET data are the first to show Xywav's impact on polysomnography measures of nighttime awakenings, sleep stage shifts, and deep sleep for people living with narcolepsy, as well as improvements among adults with IH in key outcomes important for daily life,' said Kelvin Tan, MBBCh, MRCPCH, chief medical affairs officer of Jazz Pharmaceuticals.

Jazz Pharmaceuticals首席医疗事务官Kelvin Tan，MBBCh，MRCPCH说：“这些二重唱数据首次显示了Xywav对嗜睡症患者夜间觉醒，睡眠阶段转换和深度睡眠的多导睡眠图测量的影响，以及IH成年人对日常生活重要结果的改善。”。

'Jazz is committed to our leadership in sleep, as exemplified by our presentations at Psych Congress of the DUET trial data, which studied the effectiveness of low-sodium Xywav on symptoms of narcolepsy and IH by demonstrating improvements in measures which are key for patients to wake up more refreshed with improved wakefulness during the day.'.

“Jazz致力于我们在睡眠方面的领导地位，例如我们在Psych大会上对DUET试验数据的介绍，该数据研究了低钠Xywav对嗜睡症和IH症状的有效性，通过证明改进措施是患者在白天醒来时更加清醒的关键。”。

The Psych Congress 2024 presentations are available on-demand through the conference mobile application. Abstracts and posters will also be published on HMP Global's Psychiatry & Behavioral Health Learning Network 30-60 days after the event ends.

Psych Congress 2024演示文稿可通过会议移动应用程序按需提供。活动结束后30-60天，文摘和海报也将在HMP Global的精神病学和行为健康学习网络上发布。

The Clinical and Humanistic Burden of Idiopathic Hypersomnia in the United States: Analysis of the National Health and Wellness Survey

美国特发性嗜睡的临床和人文负担：国家健康与福祉调查分析

Design Elements for a Switch Study From High- to Low-Sodium Oxybate Evaluating Blood Pressure in Narcolepsy (XYLO)

从高到低羟丁酸钠转换研究的设计元素评估发作性睡病（XYLO）的血压

About NarcolepsyNarcolepsy is a chronic, debilitating neurologic sleep disorder characterized by excessive daytime sleepiness (the inability to stay awake and alert during the day resulting in the irrepressible need to sleep or unplanned lapses into sleep or drowsiness), or EDS, and an inability to regulate sleep-wake cycles normally.1 Patients with EDS due to narcolepsy experience sleep attacks and, despite fighting the urge to sleep, may unintentionally fall asleep for short periods.2,3 These sleep attacks may happen at inappropriate or potentially dangerous times such as during driving, cycling, eating, or mid-conversation.4.

关于发作性睡病，发作性睡病是一种慢性，使人衰弱的神经系统睡眠障碍，其特征是白天过度嗜睡（白天无法保持清醒和警觉，导致无法抑制的睡眠需求或无计划的进入睡眠或嗜睡），或EDS，以及无法正常调节睡眠-觉醒周期[1]。由于发作性睡病导致的EDS患者会出现睡眠发作，尽管抵抗了睡眠的冲动，但可能会在短时间内无意入睡[2,3]。这些睡眠发作可能发生在不适当或潜在的危险时间，例如在驾驶，骑自行车，进食或谈话中。

There is no cure for narcolepsy, therefore this EDS is lifelong and has a substantial negative impact on a person's ability to function psychologically, socially and professionally.5 Patients with narcolepsy are at increased risk for hypertension, cardiometabolic morbidity, stroke, myocardial infarction, heart failure, cardiac arrest, and death.6,7,8,9 As narcolepsy is a chronic condition that requires lifelong, nightly treatment, early access to an effective, low-sodium treatment can transform lives and reduce the impact of narcolepsy on a person's physical and mental health.5.

5 嗜眠症患者患高血压、心脏代谢疾病、中风、心肌梗塞、心力衰竭、心脏骤停和死亡的风险增加。由于嗜睡症是一种需要终生、每晚接受治疗的慢性疾病，及早获得有效的低钠治疗可以改变患者的生活，减少嗜睡症对患者身心健康的影响。

About Idiopathic Hypersomnia Idiopathic hypersomnia is an often debilitating, neurologic sleep disorder that goes beyond chronic excessive daytime sleepiness.10,11,12,13 Idiopathic hypersomnia is a 24-hour sleep disorder, and symptoms may include a prolonged but non-restorative main (nighttime) sleep episode of more than 9 hours, or a sleep duration of 11 hours or longer over a 24-hour period; cognitive impairment; long and unrefreshing naps; brain fog, or the inability to focus for long periods of time; and severe sleep inertia (prolonged difficulty waking, with frequent reentries into sleep, confusion, and irritability).10,11,12,13,14 Although there are overlapping clinical features with narcolepsy, idiopathic hypersomnia is a condition with its own specific diagnostic criteria.13,15 .

关于特发性嗜睡特发性嗜睡是一种经常使人衰弱的神经系统睡眠障碍，超出了慢性白天过度嗜睡的范围[10,11,12,13]。特发性嗜睡是一种24小时睡眠障碍，症状可能包括长时间但非恢复性的主要（夜间）睡眠发作超过9小时，或在24小时内睡眠时间为11小时或更长；认知障碍；长时间不休息；大脑迷雾，或长时间无法集中注意力；和严重的睡眠惯性（长时间难以醒来，经常再次进入睡眠，困惑和烦躁不安）[10,11,12,13,14]。虽然发作性睡病的临床特征重叠，但特发性嗜睡症是一种有其特定诊断标准的疾病[13,15]。

Idiopathic hypersomnia is an often debilitating illness that can significantly affect social, educational, and occupational functioning.16,17 In the U.S., approximately 37,000 adult patients have been diagnosed with idiopathic hypersomnia and are actively seeking healthcare.18 This low number of people may be due to the many difficulties in identifying and diagnosing idiopathic hypersomnia, as well as distinguishing it from other similar sleep disorders.

特发性嗜睡症是一种常使人衰弱的疾病，可显着影响社会，教育和职业功能[16,17]。S、 ，大约37000名成年患者被诊断出患有特发性嗜睡，并正在积极寻求医疗保健[18]。这一人数少可能是由于在识别和诊断特发性嗜睡以及将其与其他类似睡眠障碍区分开来方面存在许多困难。

It is estimated that far fewer patients are currently receiving pharmacological treatment for their idiopathic hypersomnia.18,19,20,21.

据估计，目前接受特发性嗜睡药物治疗的患者少得多[18,19,20,21]。

About Xywav® (calcium, magnesium, potassium, and sodium oxybates) oral solutionXywav is the only low-sodium oxybate approved by the U.S. Food and Drug Administration (FDA) for the treatment of cataplexy or excessive daytime sleepiness (EDS) in patients 7 years of age and older with narcolepsy. The FDA recognized seven years of Orphan Drug Exclusivity for Xywav for the treatment of cataplexy or EDS in patients 7 years of age and older with narcolepsy.

关于Xywav®（钙，镁，钾和羟丁酸钠）口服溶液Xywav是美国唯一批准的低羟丁酸钠。S、 美国食品和药物管理局（FDA）用于治疗7岁及以上发作性睡病患者的猝倒或白天过度嗜睡（EDS）。美国食品和药物管理局（FDA）承认Xywav对7岁及以上发作性睡病患者的瘫痪或EDS治疗具有七年的孤儿药独家经营权。

The Office of Orphan Product Development (OOPD) at the FDA also published its summary of clinical superiority findings for Xywav for the treatment of cataplexy or EDS in patients 7 years of age and older with narcolepsy by means of greater cardiovascular safety compared to Xyrem® (sodium oxybate) oral solution.

美国食品和药物管理局孤儿产品开发办公室（OOPD）还发布了其关于Xywav治疗7岁及以上发作性睡病患者猝倒或EDS的临床优越性的总结，与Xyrem®（羟丁酸钠）口服溶液相比，其心血管安全性更高。

The decision of the OOPD is based on the FDA findings that Xywav provides a greatly reduced chronic sodium burden compared to Xyrem. Xywav has 131 mg of sodium at the maximum recommended nightly dose whereas other high sodium oxybates have 1640 mg at the equivalent dose. Xywav is comprised of a unique composition of cations resulting in 92% less sodium, or a reduction of approximately 1,000 to 1,500 mg/night at the recommended dose range of 6 g to 9 g/night. Xywav is the only oxybate therapy that does not carry a warning in the label related to use in patients sensitive to high sodium intake..

OOPD的决定是基于FDA的发现，即与Xyrem相比，Xywav可以大大减少慢性钠负荷。Xywav的最大推荐夜间剂量为131毫克钠，而其他高浓度的羟丁酸钠的等效剂量为1640毫克。Xywav由独特的阳离子组成，可减少92%的钠，或在推荐的6 g至9 g/夜剂量范围内减少约1000至1500 mg/夜。Xywav是唯一一种在标签上没有警告的羟丁酸盐疗法，该疗法与对高钠摄入敏感的患者有关。。

Xywav is also the first and only U.S. FDA-approved treatment option for idiopathic hypersomnia in adults. The FDA recognized seven years of Orphan Drug Exclusivity for Xywav for the treatment of idiopathic hypersomnia in adults. Xywav is the only FDA-approved treatment studied across the multiple symptoms of idiopathic hypersomnia, such as EDS, sleep inertia (severe grogginess or confusion when waking up), long sleep duration and cognitive impairment. Xywav can be administered as a twice- or once-nightly regimen for the treatment of idiopathic hypersomnia in adults..

Xywav也是第一个也是唯一一个美国公司。S、 FDA批准成人特发性嗜睡的治疗选择。美国食品和药物管理局（FDA）承认Xywav在治疗成人特发性嗜睡方面具有七年的孤儿药独家经营权。Xywav是FDA批准的唯一针对特发性嗜睡多种症状进行研究的治疗方法，例如EDS，睡眠惯性（醒来时严重昏昏欲睡或困惑），睡眠时间长和认知障碍。Xywav可以作为两次或每夜一次的方案用于治疗成人特发性嗜睡。。

The exact mechanism of action of Xywav in the treatment of adults with idiopathic hypersomnia and of cataplexy and EDS in narcolepsy is unknown. It is hypothesized that the therapeutic effects of Xywav are mediated through GABAB actions during sleep at noradrenergic and dopaminergic neurons, as well as thalamocortical neurons.1 The U.S. Drug Enforcement Agency (DEA) has designated Xywav as a Schedule III medicine.

Xywav治疗成人特发性嗜睡和发作性睡病中的猝倒和EDS的确切作用机制尚不清楚。据推测，Xywav的治疗效果是通过睡眠期间去甲肾上腺素能和多巴胺能神经元以及丘脑皮层神经元的GABAB作用介导的。S、 缉毒署（DEA）已将Xywav指定为附表III药物。

The DEA defines Schedule III drugs, substances, or chemicals as drugs with a moderate to low potential for physical and psychological dependence.1,2 Because of the risks of central nervous system (CNS) depression and abuse and misuse, Xywav is available only through a restricted program under a Risk Evaluation and Mitigation Strategy (REMS) called the XYWAV and XYREM REMS..

DEA将附表III药物、物质或化学品定义为具有中度至低身体和心理依赖可能性的药物。1,2由于中枢神经系统（CNS）抑郁以及滥用和误用的风险，Xywav只能通过风险评估和缓解策略（REMS）下的受限计划获得，称为Xywav和XYREM REMS。。

About Jazz Pharmaceuticals Jazz Pharmaceuticals plc is a global biopharma company whose purpose is to innovate to transform the lives of patients and their families. We are dedicated to developing potentially life-changing medicines for people with serious diseases — often with limited or no therapeutic options.

关于Jazz Pharmaceuticals Jazz Pharmaceuticals plc是一家全球生物制药公司，其宗旨是创新以改变患者及其家人的生活。我们致力于为患有严重疾病的人开发可能改变生命的药物-通常治疗选择有限或没有。