BOSTON--(BUSINESS WIRE)--PureTech Health plc (Nasdaq: PRTC, LSE: PRTC) ('PureTech' or the 'Company'), a clinical-stage biotherapeutics company, noted today that its Founded Entity, Karuna Therapeutics, Inc. (Nasdaq: KRTX) ('Karuna') announced the submission of a New Drug Application (NDA) to the U.S.

临床阶段生物治疗公司BOSTON-（BUSINESS WIRE）-PureTech Health plc（纳斯达克股票代码：PRTC，LSE:PRTC）（'PureTech'或'公司'）今天注意到其创立实体Karuna Therapeutics，Inc.（纳斯达克股票代码：KRTX）（'Karuna'）宣布向美国提交新药申请（NDA）。

Food and Drug Administration (FDA) for KarXT (xanomeline-trospium) for the treatment of schizophrenia..

食品和药物管理局（FDA）用于KarXT（xanomeline trospium）治疗精神分裂症。。

“We are thrilled to note this additional exciting milestone from the KarXT program, following the achievement of three successful, registration enabling studies in schizophrenia. KarXT, which was invented at PureTech, showcases how we invent and advance novel medicines by giving new life to drugs that have validated pharmacology but had previously not reached patients due to an issue that is now addressable with our innovations.

“在三项成功的精神分裂症注册研究取得成果之后，我们很高兴地注意到KarXT计划的另一个令人兴奋的里程碑。由PureTech发明的KarXT展示了我们如何通过赋予新生活来发明和推进新药。已经验证药理学但由于问题不是，以前没有到达患者的药物w可以通过我们的创新解决。

This NDA submission is also an important milestone for patients, as it could represent the first major advance for treating schizophrenia in over 50 years if approved by the FDA,” said Eric Elenko, Chief Innovation Officer at PureTech..

PureTech首席创新官Eric Elenko说，NDA的提交也是患者的一个重要里程碑，因为如果获得FDA的批准，它可能代表50多年来治疗精神分裂症的第一个重大进展。。

The NDA submission is supported by efficacy and long-term safety data from the EMERGENT program, the clinical program evaluating KarXT as a treatment for schizophrenia. The EMERGENT program includes the three completed positive EMERGENT-1, EMERGENT-2, and EMERGENT-3 trials evaluating the efficacy and safety of KarXT compared to placebo, and the ongoing EMERGENT-4 and EMERGENT-5 trials evaluating the long-term safety of KarXT.

NDA的提交得到了来自EMERGENT计划的有效性和长期安全性数据的支持，该计划是评估KarXT治疗精神分裂症的临床计划。紧急计划包括三项完成的积极紧急-1，紧急-2和紧急-3试验，评估KarXT与安慰剂相比的疗效和安全性，以及正在进行的紧急-4和紧急-5试验，评估长期KarXT的安全。

In all three placebo-controlled trials, KarXT met its primary endpoint, demonstrating a statistically significant and clinically meaningful reduction in Positive and Negative Syndrome Scale (PANSS) total score compared to placebo. KarXT also demonstrated reductions in both positive and negative symptoms of schizophrenia as measured by PANSS positive, PANSS negative, and PANSS negative Marder factor subscales, which were secondary endpoints in the trials..

在所有三项安慰剂对照试验中，KarXT均达到其主要终点，与安慰剂相比，阳性和阴性症状量表（PANSS）总分显示统计学显着和临床意义上的降低。通过PANSS阳性，PANSS阴性和PANSS阴性Marder因子分量表测量，KarXT还显示精神分裂症的阳性和阴性症状均减少，这是试验的次要终点。。

As a founder of Karuna and co-inventor of the KarXT program, PureTech retains an equity ownership and has rights to receive milestone payments upon the achievement of certain regulatory approvals and 20% of sublicense income.1

作为Karuna的创始人和KarXT计划的共同发明人，PureTech保留股权，并且在获得某些监管批准和20%的子许可收入后有权获得里程碑式支付.1

If approved, KarXT will be the third therapeutic candidate to be taken from inception at PureTech to FDA regulatory approval.

如果获得批准，KarXT将成为从PureTech开始到FDA监管批准的第三个治疗候选药物。

The full text of the announcement from Karuna is as follows:

卡鲁纳公告的全文如下：

Karuna Therapeutics Submits New Drug Application to U.S. Food and Drug Administration for KarXT for the Treatment of Schizophrenia

Karuna Therapeutics向美国食品和药物管理局提交新药申请，用于治疗精神分裂症

KarXT, a dual M1/M4 muscarinic agonist, represents the first new mechanism of action to treat schizophrenia in several decades, if approved

KarXT是一种双重M1/M4毒蕈碱激动剂，如果获得批准，它代表了几十年来治疗精神分裂症的第一个新的作用机制

Submission is supported by data from three positive registrational trials demonstrating consistent and robust reductions of schizophrenia symptoms

来自三项阳性注册试验的数据支持提交，证明精神分裂症症状持续且稳健地减少

Karuna Therapeutics, Inc. (NASDAQ: KRTX), a biopharmaceutical company driven to discover, develop, and deliver transformative medicines for people living with psychiatric and neurological conditions, today announced the submission of a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for KarXT (xanomeline-trospium) for the treatment of schizophrenia..

Karuna Therapeutics，Inc。（纳斯达克股票代码：KRTX）是一家生物制药公司，致力于为患有精神病和神经疾病的人发现，开发和提供转化药物，今天宣布向美国提交新药申请（NDA）食品和药物管理局（FDA）为KarXT（xanomeline trospium）治疗精神分裂症。。

'Schizophrenia is a serious mental illness that affects how one thinks, feels, and behaves, with symptoms often appearing in patients in early adulthood, during the prime years of their lives,” said Bill Meury, president and chief executive officer of Karuna Therapeutics. “While current therapies have made a difference for many patients, they are not without limitations due to lack of full symptom relief or side effects that may lead to treatment discontinuation.

卡鲁纳治疗公司总裁兼首席执行官比尔·梅里（Bill Meury）说：“精神分裂症是一种严重的精神疾病，会影响人们的思考，感受和行为方式，症状通常出现在成年早期的患者生命的最初几年。“虽然目前的疗法对许多患者有所不同，但由于缺乏可能导致治疗中止的完全症状缓解或副作用，它们并非没有限制。

KarXT, if approved, will represent the first novel pharmacological approach to treating schizophrenia in several decades and provide a new treatment option for patients and their physicians.”.

如果获得批准，KarXT将代表几十年来治疗精神分裂症的第一种新型药理学方法，并为患者及其医生提供新的治疗选择。”。

“The NDA submission represents an important step toward helping patients in need. It is also a defining moment for Karuna Therapeutics. It represents the culmination of years of pre-clinical and clinical development, and a great deal of skill and hard work by our R&D organization. Our priorities over the next year are the regulatory review process, our ongoing development efforts, and building the platform to introduce KarXT to the medical community.

“NDA提交的文件是帮助有需要的患者迈出的重要一步，也是Karuna Therapeutics的决定性时刻，它代表了多年临床前和临床开发的高潮，以及我们研发组织的大量技能和辛勤工作。我们明年的优先事项是监管审查流程，我们正在进行的开发工作和建设向医学界介绍KarXT的平台。

I believe we are uniquely positioned to make a positive impact on how neuropsychiatric conditions are treated,” Meury added..

我相信我们处于独特的地位，可以对神经精神疾病的治疗方式产生积极影响，“Meury补充说。。

The NDA submission is supported by efficacy and long-term safety data from the EMERGENT program, the clinical program evaluating KarXT as a treatment for schizophrenia. The EMERGENT program includes the three completed positive EMERGENT-1, EMERGENT-2, and EMERGENT-3 trials evaluating the efficacy and safety of KarXT compared to placebo, and the ongoing EMERGENT-4 and EMERGENT-5 trials evaluating the long-term safety of KarXT.

NDA的提交得到了来自EMERGENT计划的有效性和长期安全性数据的支持，该计划是评估KarXT治疗精神分裂症的临床计划。紧急计划包括三项完成的积极紧急-1，紧急-2和紧急-3试验，评估KarXT与安慰剂相比的疗效和安全性，以及正在进行的紧急-4和紧急-5试验，评估长期KarXT的安全。

In all three placebo-controlled trials, KarXT met its primary endpoint, demonstrating a statistically significant and clinically meaningful reduction in Positive and Negative Syndrome Scale (PANSS) total score compared to placebo. KarXT also demonstrated reductions in both positive and negative symptoms of schizophrenia as measured by PANSS positive, PANSS negative, and PANSS negative Marder factor subscales, which were secondary endpoints in the trials..

在所有三项安慰剂对照试验中，KarXT均达到其主要终点，与安慰剂相比，阳性和阴性症状量表（PANSS）总分显示统计学显着和临床意义上的降低。通过PANSS阳性，PANSS阴性和PANSS阴性Marder因子分量表测量，KarXT还显示精神分裂症的阳性和阴性症状均减少，这是试验的次要终点。。

KarXT was found to be generally well-tolerated, with the most common adverse events being cholinergic in nature and rated mild to moderate in severity. Discontinuation rates due to treatment emergent adverse events were low and similar between KarXT and placebo across all trials. Notably, KarXT was not associated with common side effects of currently available antipsychotics, including changes in metabolic function, weight gain, somnolence, and extrapyramidal symptoms..

发现KarXT通常具有良好的耐受性，最常见的不良事件本质上是胆碱能，严重程度评定为轻度至中度。在所有试验中，KarXT和安慰剂之间由于治疗紧急不良事件导致的停药率较低且相似。值得注意的是，KarXT与目前可用的抗精神病药物的常见副作用无关，包括代谢功能，体重增加，嗜睡和锥体外系症状的改变。。

About KarXT

关于KarXT

KarXT (xanomeline-trospium) is an investigational muscarinic antipsychotic in development for the treatment of schizophrenia and psychosis related to Alzheimer’s disease. Through its novel mechanism of action, KarXT acts as a dual M1/M4 muscarinic acetylcholine receptor agonist in the central nervous system, which is thought to mediate positive, negative, and cognitive symptoms of schizophrenia.

KarXT（xanomeline trospium）是一种研究性毒蕈碱类抗精神病药物，用于治疗与阿尔茨海默病相关的精神分裂症和精神病。通过其新的作用机制，KarXT在中枢神经系统中充当双重M1/M4毒蕈碱乙酰胆碱受体激动剂，其被认为介导精神分裂症的阳性，阴性和认知症状。

Unlike existing treatments, KarXT does not directly block dopamine receptors, representing a potential new approach to treating schizophrenia..

与现有治疗方法不同，KarXT不直接阻断多巴胺受体，代表了治疗精神分裂症的潜在新方法。。

About Schizophrenia

关于精神分裂症

Schizophrenia is a persistent and often disabling mental illness impacting how a person thinks, feels, and behaves, and affects nearly 24 million people worldwide, including 2.8 million people in the U.S. It is characterized by three symptom domains: positive symptoms (hallucinations and delusions), negative symptoms (difficulty enjoying life and withdrawal from others), and cognitive impairment (deficits in memory, concentration, and decision-making).

精神分裂症是一种持续且经常致残的精神疾病，会影响一个人的思考，感受和行为方式，并影响全球近2400万人，其中包括美国的280万人。它的特征是三个症状领域：阳性症状（幻觉和妄想），阴性症状（享受生活和退学困难），和认知障碍（记忆力，注意力和决策能力不足）。

In part due to limitations with current treatments, people living with schizophrenia often struggle to maintain employment, live independently, and manage relationships. While current treatments can be effective in managing select symptoms, approximately 30% of people to do not respond to therapy, with an additional 50% experiencing only a partial improvement in symptoms or unacceptable side effects..

部分由于目前治疗的局限性，精神分裂症患者往往难以维持就业，独立生活和管理人际关系。虽然目前的治疗方法可以有效地控制某些症状，但大约30%的人对治疗没有反应，另外50%的人仅出现部分症状改善或不可接受的副作用。。

About Karuna

关于卡鲁纳

Karuna Therapeutics is a biopharmaceutical company driven to discover, develop, and deliver transformative medicines for people living with psychiatric and neurological conditions. At Karuna, we understand there is a need for differentiated and more effective treatments that can help patients navigate the challenges presented by serious mental illness.

Karuna Therapeutics是一家生物制药公司，致力于为患有精神病和神经疾病的人发现，开发和提供变革性药物。在卡鲁纳，我们了解需要有区别和更有效的治疗方法来帮助患者应对严重精神疾病带来的挑战。

Utilizing our extensive knowledge of neuroscience, we are harnessing the untapped potential of the brain in pursuit of novel pathways to develop medicines that make meaningful differences in peoples’ lives. For more information, please visit www.karunatx.com..

利用我们广泛的神经科学知识，我们正在利用大脑尚未开发的潜力，寻求新的途径来开发能够在人们生活中产生有意义差异的药物。欲了解更多信息，请访问www.karunatx.com。。

About PureTech Health

关于PureTech健康

PureTech is a clinical-stage biotherapeutics company dedicated to giving life to new classes of medicine to change the lives of patients with devastating diseases. The Company has created a broad and deep pipeline through its experienced research and development team and its extensive network of scientists, clinicians and industry leaders that is being advanced both internally and through its Founded Entities.

PureTech是一家临床阶段的生物治疗公司，致力于为新型药物提供生命，以改变患有破坏性疾病的患者的生活。该公司通过其经验丰富的研发团队及其广泛的科学家，临床医生和行业领导者网络，建立了广泛而深入的渠道，该网络正在内部和通过其创始实体进行发展。

PureTech's R&D engine has resulted in the development of 27 therapeutics and therapeutic candidates, including two (Plenity® and EndeavorRx®) that have received both US FDA clearance and European marketing authorization and a third (KarXT) that has been filed for FDA approval. A number of these programs are being advanced by PureTech or its Founded Entities in various indications and stages of clinical development, including registration enabling studies.

PureTech的研发引擎已经开发出27种治疗药物和治疗候选药物，其中两种（Plenity®和EndeavorRx®）已获得美国FDA批准和欧洲上市许可，第三种（KarXT）已提交FDA批准。PureTech或其创始实体正在临床开发的各种适应症和阶段推进其中一些计划，包括注册研究。

All of the underlying programs and platforms that resulted in this pipeline of therapeutic candidates were initially identified or discovered and then advanced by the PureTech team through key validation points..

所有导致这一治疗候选药物管道的基础程序和平台最初都被识别或发现，然后由PureTech团队通过关键验证点推进。。

For more information, visit www.puretechhealth.com or connect with us on X (formerly Twitter) @puretechh.

欲了解更多信息，请访问www.puretechhealth.com或在X（以前称为Twitter）@puretech上与我们联系。

1As of July 31, 2023, PureTech’s percentage ownership of Karuna was approximately 2.8% on an outstanding voting share basis. As of March 22, 2023, PureTech has sold its right to receive a 3% royalty from Karuna to Royalty Pharma on net sales up to $2 billion annually, after which threshold PureTech will receive 67% of the royalty payments and Royalty Pharma will receive 33%.

1截至2023年7月31日，PureTech在未投票的股份基础上拥有Karuna的百分比约为2.8%。截至2023年3月22日，PureTech已出售其从Karuna获得3%特许权使用费的权利，每年净销售额高达20亿美元，之后PureTech将获得67%的特许权使用费，特许权使用费Pharma将获得33%。

PureTech retains its equity ownership in Karuna. Additionally, under its license agreement with Karuna, PureTech retains the right to receive milestone payments upon the achievement of certain regulatory approvals and 20% of sublicense income..

PureTech保留其在卡鲁纳的股权。此外，根据其与Karuna的许可协议，PureTech保留在实现某些监管批准和20%的子许可收入后获得里程碑付款的权利。。

Cautionary Note Regarding Forward-Looking Statements

关于前瞻性陈述的警示

This press release contains statements that are or may be forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including without limitation those related to submission of a New Drug Application (NDA) to the U.S.

本新闻稿包含1995年“私人证券诉讼改革法”含义范围内或可能是前瞻性声明的声明。本新闻稿中包含的与历史事实事项无关的所有声明均应视为前瞻性声明，包括但不限于与向美国提交新药申请（NDA）有关的声明。

Food and Drug Administration (FDA) for KarXT (xanomeline-trospium) for the treatment of schizophrenia and Karuna’s and PureTech’s future prospects, developments and strategies. The forward-looking statements are based on current expectations and are subject to known and unknown risks, uncertainties and other important factors that could cause actual results, performance and achievements to differ materially from current expectations, including, but not limited to, those risks, uncertainties and other important factors described under the caption 'Risk Factors' in our Annual Report on Form 20-F for the year ended December 31, 2022 filed with the SEC and in our other regulatory filings.

食品和药物管理局（FDA）为KarXT（xanomeline trospium）治疗精神分裂症和Karuna和PureTech的未来前景，发展和战略。前瞻性陈述基于当前预期，并受到已知和未知风险，不确定性和其他重要因素的影响，这些因素可能导致实际结果，绩效和成就与当前预期有很大差异，包括但不限于这些风险，在我们向SEC和我们的其他监管文件提交的截至2022年12月31日的年度20-F年度报告中，“风险因素”标题下描述的不确定性和其他重要因素。

These forward-looking statements are based on assumptions regarding the present and future business strategies of the Company and the environment in which it will operate in the future. Each forward-looking statement speaks only as at the date of this press release. Except as required by law and regulatory requirements, we disclaim any obligation to update or revise these forward-looking statements, whether as a result of new information, future events or otherwise..

这些前瞻性声明基于对公司当前和未来业务策略及其未来运营环境的假设。每个前瞻性声明仅在本新闻稿发布之日才发表。除法律和法规要求外，我们不承担任何更新或修改这些前瞻性声明的义务，无论是由于新信息，未来事件还是其他原因。。