SAN DIEGO--(BUSINESS WIRE)--Acadia Pharmaceuticals Inc. (Nasdaq: ACAD) today announced the initiation of the Phase 3 COMPASS PWS study evaluating the efficacy and safety of carbetocin nasal spray (ACP-101) for the treatment of hyperphagia in Prader-Willi syndrome (PWS). PWS is a rare, neurobehavioral genetic disorder that is estimated to affect 8,000 to 10,000 patients in the United States.1-4 The most common symptom is hyperphagia, which is an unrelenting lack of satiety.

圣地亚哥-（商业线）-Acadia制药公司（纳斯达克股票代码：ACAD）今天宣布启动第3阶段COMPASS PWS研究，评估卡贝托星鼻喷雾剂（ACP-101）治疗的有效性和安全性。Prader-Willi综合征（PWS）的食欲过盛。PWS是一种罕见的神经行为遗传疾病，估计在美国影响8000至10000名患者.1-4最常见的症状是食欲过盛，这是一种持续缺乏饱腹感。

Other defining features of PWS may include altered metabolism, developmental delays, behavioral challenges and moderate cognitive deficits.5.

PWS的其他定义特征可能包括代谢改变，发育迟缓，行为挑战和中度认知缺陷。

“Currently there is no FDA-approved treatment for hyperphagia in PWS, which presents serious challenges for those living with this condition and their families,” said Shawn McCandless, M.D., Chair, Department of Genetics and Metabolism, Children’s Hospital Colorado and COMPASS PWS study investigator.

科罗拉多州儿童医院遗传与代谢系主席Shawn McCandless博士和COMPASS PWS研究调查员说：“目前，PWS尚无FDA批准的治疗食欲亢进的方法，这对患有这种疾病的人及其家人提出了严峻的挑战。

“Essentially all experience hyperphagia, feeling a near-constant state of hunger, as though their body is telling them that they are starving, despite being actually well-nourished. As a result, individuals with PWS often exhibit food-seeking behaviors that require constant supervision to prevent life-threatening risks, including gastric rupture, irregular swallowing and choking.

“基本上所有人都会经历食欲亢进，感觉饥饿状态接近持续，好像他们的身体告诉他们饥饿，尽管实际上营养良好。因此，PWS患者经常表现出寻求食物的行为，需要不断监督预防危及生命的风险，包括胃破裂，不规则吞咽和窒息。

I look forward to the outcome of this Phase 3 clinical trial, and the potential of having a treatment option to offer those living with PWS.”.

我期待这个3期临床试验的结果，以及为PWS患者提供治疗选择的潜力。

COMPASS PWS is a 12-week, double-blind, randomized, placebo-controlled global Phase 3 trial evaluating the efficacy and safety of carbetocin nasal spray 3.2 mg three times daily (TID) in approximately 170 children and adults aged five to 30 years with PWS. The primary efficacy endpoint of the study is change from baseline to week 12 on the hyperphagia questionnaire for clinical trials (HQ-CT) score, a caregiver assessment for hyperphagia-related behaviors.

COMPASS PWS是一项为期12周，双盲，随机，安慰剂对照的全球3期临床试验，评估约170名5至30岁儿童和成人每日三次卡贝霉素鼻腔喷雾剂3.2 mg（TID）的疗效和安全性PWS。该研究的主要疗效终点是从基线到第12周的hyperphagia临床试验问卷（HQ-CT）评分的变化，该评分是对食欲过盛相关行为的护理人员评估。

Participants who complete the Phase 3 study will be eligible to enroll in a long-term, open-label extension study designed to investigate the safety and tolerability of long-term treatment with ACP-101..

完成3期研究的参与者将有资格参加长期开放标签扩展研究，旨在调查ACP-101长期治疗的安全性和耐受性。。

“The launch of the Phase 3 COMPASS PWS trial will build on previous Phase 3 clinical trial experience, where carbetocin nasal spray 3.2 mg was observed to reduce hyperphagia-related behaviors,” said Ponni Subbiah, M.D., M.P.H., Senior Vice President, Global Head of Medical Affairs and Chief Medical Officer.

Ponni Subbiah博士，M.P.H.博士，全球医疗事务高级副总裁兼首席医疗官说：“第三阶段COMPASS PWS试验的启动将建立在以前的第三阶段临床试验经验的基础上，观察到3.2毫克的卡贝托星鼻腔喷雾剂可减少与食欲过盛有关的行为。”。

“We look forward to working with the Prader-Willi community and clinical research sites as we continue to advance the ACP-101 clinical development program for those living with this debilitating syndrome and their families.”.

“随着我们继续为患有这种使人衰弱的综合症的人及其家人推进ACP-101临床开发计划，我们期待与Prader-Willi社区和临床研究机构合作。”。

More information about the COMPASS PWS study is available at www.CompassPWS.com.

有关COMPASS PWS研究的更多信息，请访问www.CompassPWS.com。

About Prader-Willi Syndrome

关于普拉德-威利综合症

Prader-Willi syndrome (PWS) is a rare neurobehavioral genetic disorder that affects both males and females.6 PWS is estimated to affect approximately 8,000 to 10,000 patients in the United States.3,4 PWS affects the functioning of the hypothalamus and other aspects of the brain with symptoms varying by individual.5,6 The most common symptom is hyperphagia, which is an unrelenting lack of satiety, to which a deficiency in oxytocin is believed to be contributory.5,7 Individuals living with PWS have fewer oxytocin-producing neurons in the brain.7 Other defining features of the syndrome may include altered metabolism, developmental delays, behavioral challenges and moderate cognitive deficits.3 Patients may also experience high pain tolerance, sleep disturbances, gastrointestinal issues, respiratory and temperature regulation abnormalities.5,7-9 There is no FDA-approved treatment for the hyperphagia associated with PWS.5.

Prader-Willi综合征（PWS）是一种罕见的神经行为遗传疾病，影响男性和女性.6 PWS估计影响美国约8000至10000名患者.3,4 PWS影响下丘脑和其他方面的功能。大脑的症状因人而异.5,6最常见的症状是食欲过盛，这是一种持续缺乏饱腹感，认为催产素缺乏是造成这种情况的原因.5,7患有PWS的个体大脑中产生催产素的神经元较少.7该综合征的其他定义特征可能包括代谢改变，发育迟缓，行为挑战和中度认知缺陷。.3患者也可能出现高疼痛耐受性，睡眠障碍，胃肠道问题，呼吸和温度调节异常.5,7-9没有FDA批准的与PWS相关的食欲过盛的治疗方法。

About Carbetocin Nasal Spray (ACP-101)

关于卡贝霉素鼻腔喷雾剂（ACP-101）

Carbetocin nasal spray is an investigational drug being developed for the treatment of hyperphagia in Prader-Willi syndrome (PWS). Carbetocin has improved drug qualities relative to oxytocin, including an extended half-life and greater specificity for the oxytocin receptor compared to vasopressin receptors which could provide meaningful efficacy with an attractive safety profile in patients with PWS.10 For the treatment of PWS specifically, a central nervous system disorder, an intranasal formulation of carbetocin was developed, which provides direct delivery of the drug to the brain, greatly reducing systemic exposure and the potential for side effects.

Carbetocin鼻喷雾剂是一种正在开发用于治疗Prader-Willi综合征（PWS）中的食欲过盛的研究药物。与催产素相比，Carbetocin具有改善的药物质量，包括与加压素受体相比延长的半衰期和对催产素受体的更高特异性，这可以为PWS患者提供有意义的疗效和有吸引力的安全性.10特别是对于PWS的治疗，中枢神经系统疾病，开发了卡贝霉素的鼻内制剂，其提供药物直接递送至大脑，大大减少全身暴露和副作用的可能性。

Acadia acquired Levo Therapeutics and worldwide rights to carbetocin nasal spray in June 2022. Carbetocin nasal spray has been granted Orphan Drug, Fast Track, and Rare Pediatric Disease designations by the FDA..

Acadia于2022年6月收购了Levo Therapeutics和carbetocin鼻喷雾剂的全球权利。Carbetocin鼻腔喷雾剂已被FDA授予孤儿药，快速通道和罕见的儿科疾病名称。。

About Acadia Pharmaceuticals

关于阿卡迪亚制药

Acadia is advancing breakthroughs in neuroscience to elevate life. For 30 years we have been working at the forefront of healthcare to bring vital solutions to people who need them most. We developed and commercialized the first and only approved therapies for hallucinations and delusions associated with Parkinson’s disease psychosis and for the treatment of Rett syndrome.

阿卡迪亚正在推进神经科学的突破，以提升生命。30年来，我们一直致力于医疗保健的前沿，为最需要他们的人提供重要的解决方案。我们开发并商业化了第一个也是唯一批准的用于与帕金森病精神病相关的幻觉和妄想以及用于治疗Rett综合征的疗法。

Our clinical-stage development efforts are focused on treating the negative symptoms of schizophrenia, Prader-Willi syndrome, Alzheimer’s disease psychosis and neuropsychiatric symptoms in central nervous system disorders. For more information, visit us at www.acadia.com and follow us on LinkedIn and Twitter..

我们的临床阶段开发工作集中于治疗中枢神经系统疾病中精神分裂症，Prader-Willi综合征，阿尔茨海默病精神病和神经精神症状的阴性症状。欲了解更多信息，请访问我们的www.acadia.com，并在LinkedIn和Twitter上关注我们。。

Forward-Looking Statements

前瞻性声明

Statements in this press release that are not strictly historical in nature are forward-looking statements. These statements include but are not limited to statements regarding the timing of future events. These statements are only predictions based on current information and expectations and involve a number of risks and uncertainties.

本新闻稿中并非严格具有历史意义的陈述是前瞻性陈述。这些陈述包括但不限于关于未来事件发生时间的陈述。这些陈述只是基于当前信息和期望的预测，涉及许多风险和不确定性。

Actual events or results may differ materially from those projected in any of such statements due to various factors, including the risks and uncertainties inherent in drug development, approval and commercialization. For a discussion of these and other factors, please refer to Acadia’s annual report on Form 10-K for the year ended December 31, 2022, as well as Acadia’s subsequent filings with the Securities and Exchange Commission.

由于各种因素，包括药物开发，批准和商业化固有的风险和不确定性，实际事件或结果可能与任何此类声明中预测的事件或结果有很大差异。有关这些因素和其他因素的讨论，请参阅截至2022年12月31日的Acadia 10-K年度报告，以及Acadia随后向证券交易委员会提交的报告。

You are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof. This caution is made under the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. All forward-looking statements are qualified in their entirety by this cautionary statement and Acadia undertakes no obligation to revise or update this press release to reflect events or circumstances after the date hereof, except as required by law..

警告您不要过分依赖这些前瞻性声明，这些声明仅在本声明发布之日起生效。这种谨慎是根据1995年“私人证券诉讼改革法”的安全港规定作出的。除法律规定外，所有前瞻性声明均受本警示性声明的整体约束，Acadia不承担修改或更新本新闻稿的义务，以反映本声明签署之日以后的事件或情况。。

References

工具书类

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