Adds ABS-1230, a clinical-stage, potential first-in-class precision therapy for KCNT1+ epilepsy

新增ABS-1230，一种处于临床阶段、有望成为首个同类精准疗法的KCNT1+癫痫治疗药物

Leverages Jazz development expertise and commercial strength in rare and severe epilepsies

利用Jazz在罕见和严重癫痫领域的开发专长和商业实力

Transaction includes $820 million upfront payment

交易包括8.2亿美元的预付款

DUBLIN and SAN DIEGO

都柏林和圣迭戈

,

，

Aug. 10, 2026

2026年8月10日

/PRNewswire/ -- Jazz Pharmaceuticals plc (Nasdaq:

/美通社/ -- Jazz Pharmaceuticals plc（纳斯达克：

JAZZ

爵士乐

) and Actio Biosciences, Inc. (Actio Biosciences) today announced the companies have entered into a definitive agreement for Jazz to acquire privately-held Actio Biosciences for $820 million upfront and up to $500 million in contingent consideration.

) 与 Actio Biosciences, Inc.（Actio Biosciences）今日宣布，双方已签署最终协议，Jazz 将以 8.2 亿美元的首付款及最高 5 亿美元的或有对价收购私营公司 Actio Biosciences。

Actio Biosciences' lead clinical asset is ABS-1230, a novel, first-in-class small molecule precision therapy KCNT1 ion channel inhibitor. KCNT1+ epilepsy is a rare genetic developmental and epileptic encephalopathy (DEE) affecting approximately 2,500 patients in the United States. Patients suffering from KCNT1+ epilepsy typically endure a profound seizure burden, with most experiencing dozens to hundreds of episodes daily that remain highly resistant to antiseizure medications.

Actio Biosciences 的核心临床资产是 ABS-1230，这是一种新颖的、首创新类的小分子精准疗法，为 KCNT1 离子通道抑制剂。KCNT1+ 癫痫是一种罕见的遗传性发育性和癫痫性脑病（DEE），在美国影响约 2,500 名患者。KCNT1+ 癫痫患者通常承受严重的癫痫发作负担，大多数患者每天经历数十次至数百次发作，且对抗癫痫药物高度耐药。

Approximately 80% of individuals with KCNT1+ epilepsy experience disease onset during infancy; many of these children never achieve fundamental developmental milestones, such as walking or speaking, and tragically, some do not survive into adulthood. For those with later-onset disease, the condition typically manifests as disruptive nocturnal seizures, often accompanied by significant cognitive and psychiatric comorbidities..

约80%的KCNT1相关癫痫患者在婴儿期发病；许多患儿从未达到行走或说话等基本发育里程碑，令人痛心的是，部分患者无法存活至成年。对于晚发型患者，该病通常表现为破坏性的夜间癫痫发作，并常伴有严重的认知和精神共患病。

There are currently no U.S. Food and Drug Administration (FDA) approved therapies for KCNT1+ epilepsy. ABS-1230 recently demonstrated meaningful seizure reductions in an early clinical proof-of-concept trial in children with KCNT1 epilepsy. The ongoing Phase 1b/2a KYRON trial is designed to serve as the registrational study to support a new drug application submission in the U.S.

目前，美国食品药品监督管理局（FDA）尚未批准任何用于治疗KCNT1+癫痫的疗法。ABS-1230在最近一项针对KCNT1癫痫患儿的早期临床概念验证试验中，显示出具有临床意义的癫痫发作减少。正在进行的KYRON 1b/2a期临床试验旨在作为注册研究，以支持在美国提交新药申请。

In addition to having FDA Fast Track, Rare Pediatric Disease and Orphan Drug Product designations, ABS-1230 was accepted into the FDA's new Rare Disease Evidence Principles (RDEP) program which aims to facilitate rapid development of ultra-rare disease therapies. .

除了获得美国食品药品监督管理局（FDA）的快速通道、罕见儿科疾病和孤儿药产品认定外，ABS-1230 还被纳入 FDA 新设立的罕见病证据原则（RDEP）计划，该计划旨在促进超罕见疾病疗法的快速开发。

'The acquisition of ABS-1230 represents a highly strategic expansion of our rare epilepsy portfolio, building upon the global success of

收购ABS-1230代表了我们罕见癫痫产品组合的高度战略性扩展，建立在...全球成功的基础上

Epidiolex

埃皮迪奥克斯

and deepening our leadership in rare and severe epilepsies,' said Renee Gala, president and chief executive officer, Jazz Pharmaceuticals. 'The emerging clinical profile of ABS-1230 is highly encouraging, and we look forward to closing the proposed transaction and collaborating with our new Actio Biosciences colleagues to address an urgent patient need.

“并深化我们在罕见且严重癫痫领域的领导地位，”Jazz Pharmaceuticals 总裁兼首席执行官 Renee Gala 表示。“ABS-1230 初步展现出的临床特征令人备受鼓舞，我们期待完成拟议的交易，并与新加入的 Actio Biosciences 同事携手合作，以满足患者迫切的医疗需求。”

Together, we are committed to working alongside the epilepsy patient community and regulators to bring this important medicine to children and families, who currently have no treatment options for a devastating disease.'.

我们承诺与癫痫患者社区和监管机构携手合作，将这一重要药物带给目前对这种毁灭性疾病没有任何治疗选择的儿童和家庭。

'We chose to partner with Jazz because they combine a purpose-led culture with a world-class development engine and the commercial scale to ensure ABS-1230 is brought to patients as quickly and efficiently as possible,' said David Goldstein, Ph.D., chief executive officer, Actio Biosciences. 'We started Actio with one goal in mind: to bring meaningful therapies to those who need them most.

“我们选择与Jazz合作，是因为他们将使命驱动的文化、世界级的研发引擎以及确保ABS-1230能够尽快且高效地惠及患者的商业化规模相结合。”Actio Biosciences首席执行官David Goldstein博士表示。“我们创立Actio时秉持一个目标：为最需要的患者提供有意义的疗法。

Guided by our expertise in genetics, we set out to leverage a deep understanding of disease biology to develop highly effective targeted therapies for rare diseases with high unmet need. ABS-1230 is that vision personified, and we are thrilled to partner with Jazz on the next leg of the journey to bring this important medicine to patients.'.

凭借我们在遗传学领域的专业优势，我们致力于通过对疾病生物学的深刻理解，开发针对具有高未满足需求的罕见病的高效靶向疗法。ABS-1230 正是这一愿景的体现，我们非常高兴能与 Jazz 制药携手合作，共同推进下一阶段的研发进程，将这一重要药物带给患者。

Key Highlights

关键亮点

Strengthens Jazz's leadership in rare epilepsy. Builds on durable Epidiolex franchise and growing epilepsy pipeline.

巩固Jazz在罕见癫痫领域的领导地位。依托持久的Epidiolex品牌组合和不断增长的癫痫产品线。

Meaningful early clinical data support developing ABS-1230 for KCNT1+ epilepsy, an ultra-rare disease with no approved therapies.

有意义的早期临床数据支持开发ABS-1230用于治疗KCNT1+癫痫，这是一种没有获批疗法的超罕见疾病。

ABS-1230 has been accepted into FDA's RDEP program, which accelerates ultra-rare disease development.

ABS-1230 已获准加入美国食品药品监督管理局（FDA）的罕见疾病评估计划（RDEP），该计划旨在加速超罕见疾病的药物研发。

Opportunity for development of ABS-1230 for additional genetic epilepsy indications.

开发ABS-1230用于更多遗传性癫痫适应症的机会。

Transaction Terms

交易条款

Under the terms of the merger agreement, Actio Biosciences shareholders will receive an $820 million upfront payment and up to $500 million in potential approval and sales milestones. As part of the transaction, and concurrently with closing, Actio Biosciences will spin out a new privately-held entity with certain management, employees and assets (not including ABS-1230 which remains with Actio Biosciences and is being acquired by Jazz).

根据合并协议的条款，Actio Biosciences 的股东将获得 8.2 亿美元的预付款，以及高达 5 亿美元的潜在监管批准和销售里程碑付款。作为交易的一部分，并在交割同时，Actio Biosciences 将分拆出一家新的私营实体，包含部分管理人员、员工和资产（不包括 ABS-1230，该资产仍保留在 Actio Biosciences，并由 Jazz 收购）。

The new, independent private company will be funded by existing investors, with Jazz receiving a minority stake and certain related rights. The focus of the new company will be on genetic rare neurological diseases, which will include a clinical-stage small molecule TRPV4 inhibitor, ABS-0871, for Charcot-Marie-Tooth type 2C and other early-stage programs.

这家新的独立私营公司将由现有投资者提供资金，Jazz将获得少数股权及某些相关权利。新公司的重心将放在遗传性罕见神经系统疾病上，其中包括用于2C型夏科-马里-托思病的临床阶段小分子TRPV4抑制剂ABS-0871，以及其他早期研发项目。

With the focus on rare disease, this investment in the new entity represents a strong strategic fit for Jazz's long-term strategy..

鉴于对罕见病的关注，对新实体的这项投资与Jazz的长期战略高度契合。

The transaction has been unanimously approved by the boards of both companies and is expected to close by the fourth quarter of 2026, subject to customary closing conditions. Jazz will fund the transaction through a combination of cash on hand and drawing on existing financing facilities.

该交易已获得两家公司董事会的一致批准，预计将在2026年第四季度完成，但需满足惯常的交割条件。Jazz将通过手头现金和提取现有融资设施的组合方式为该交易提供资金。

Closing Conditions

交割条件

The transaction is subject to customary closing conditions

本次交易须满足惯例交割条件

.

。

Advisors

顾问

Moelis & Company LLC is serving as financial advisor to Jazz Pharmaceuticals, and Hogan Lovells Cadwalader US LLP is serving as legal advisor.

莫利斯公司（Moelis & Company LLC）担任爵士制药公司（Jazz Pharmaceuticals）的财务顾问，霍金路伟卡德瓦拉德美国律师事务所（Hogan Lovells Cadwalader US LLP）担任法律顾问。

J.P. Morgan Securities LLC and Centerview Partners LLC are serving as financial advisors to Actio Biosciences, and Cooley LLP is serving as legal advisor.

摩根大通证券有限责任公司和 Centerview Partners LLC 担任 Actio Biosciences 的财务顾问，Cooley LLP 担任法律顾问。

About ABS-1230

关于ABS-1230

ABS-1230 is designed to be a potent and selective orally available small molecule KCNT1 inhibitor for the treatment of KCNT1-related epilepsy. In preclinical studies, ABS-1230 inhibited KCNT1 across all evaluable pathogenic mutations, indicating potential to treat all patients with KCNT1-related epilepsy.

ABS-1230 旨在成为一种强效、选择性高且可口服的小分子 KCNT1 抑制剂，用于治疗 KCNT1 相关癫痫。在临床前研究中，ABS-1230 对所有可评估的致病性突变均表现出 KCNT1 抑制作用，表明其具有治疗所有 KCNT1 相关癫痫患者的潜力。

In an early clinical proof-of-concept trial, children with KCNT1-related epilepsy who received ABS-1230 experienced meaningful seizure reductions. ABS-1230 has the potential to provide patients with the convenience of an oral therapy. ABS-1230 has FDA Fast Track, Rare Pediatric Disease and Orphan Drug Product designations and has been accepted into the Rare Disease Evidence Principles process..

在一项早期的临床概念验证试验中，接受ABS-1230治疗的KCNT1相关癫痫患儿实现了具有临床意义的发作减少。ABS-1230有望为患者提供口服治疗的便利性。ABS-1230已获得美国食品药品监督管理局（FDA）的快速通道认定、罕见儿科疾病认定和孤儿药产品认定，并已被纳入罕见病证据原则流程。

About KCNT1-related Epilepsy

关于 KCNT1 相关癫痫

KCNT1-related epilepsy is a rare and often fatal pediatric epileptic encephalopathy. Patients with KCNT1-related epilepsy experience frequent treatment-resistant seizures that typically begin in early infancy and are accompanied by profound developmental delays and neurological impairments. Those with later onset of disease face disruptive night-time seizures, often with cognitive and psychiatric comorbidities.

KCNT1相关癫痫是一种罕见且常致死的儿童癫痫性脑病。KCNT1相关癫痫患者会出现频繁的难治性癫痫发作，通常始于婴儿早期，并伴有严重的发育迟缓和神经功能损害。晚发型患者则面临破坏性的夜间癫痫发作，常合并认知和精神障碍。

General antiepileptic drugs have limited benefit in patients with this genetic epilepsy and are often associated with debilitating side effects. There are currently no approved therapies for KCNT1+ epilepsy..

常规抗癫痫药物在此类遗传性癫痫患者中的疗效有限，且常伴随令人衰弱的副作用。目前尚无针对KCNT1相关癫痫的获批疗法。

About

关于

Jazz Pharmaceuticals

爵士制药公司

Jazz Pharmaceuticals plc (Nasdaq:

Jazz Pharmaceuticals plc（纳斯达克：

JAZZ

爵士乐

) is a global biopharma company whose purpose is to innovate to transform the lives of patients and their families. We are dedicated to developing life-changing medicines for people with rare disease — often with limited or no therapeutic options. We have a diverse portfolio of medicines, including leading therapies addressing epilepsies, cancers and sleep disorders.

）是一家全球性生物制药公司，其使命是通过创新改变患者及其家人的生活。我们致力于为罕见病患者开发能够改变生命的药物——这些患者往往面临治疗选择有限甚至无药可治的困境。我们拥有多元化的产品组合，包括针对癫痫、癌症和睡眠障碍等疾病的领先疗法。

Our patient-focused and science-driven approach powers pioneering research and development advancements across our robust pipeline of innovative therapeutics. Jazz is headquartered in Dublin, Ireland with research and development laboratories, manufacturing facilities and employees in multiple countries committed to serving patients worldwide.

我们以患者为中心、以科学为驱动的方法，推动了我们在创新疗法强大产品管线中前沿的研发进展。Jazz 总部位于爱尔兰都柏林，在多个国家设有研发实验室、生产设施并拥有员工，致力于服务全球患者。

Please visit .

请访问。

www.jazzpharmaceuticals.com

www.jazzpharmaceuticals.com

for more information.

以获取更多信息。

About Actio Biosciences

关于Actio Biosciences

Actio Biosciences is a clinical-stage biotechnology company advancing the translation of genetic insights into novel small molecule precision medicines. The company applies deep expertise in Mendelian genetics, drug discovery and data science to identify targets whose biology underlies rare diseases with high unmet need and hold promise for the treatment of more prevalent indications.

Actio Biosciences 是一家处于临床阶段的生物技术公司，致力于将遗传学见解转化为新型小分子精准药物。该公司凭借在孟德尔遗传学、药物发现和数据科学领域的深厚专业知识，识别那些生物学机制 underlying 高未满足需求的罕见疾病、并有望用于治疗更广泛适应症的靶点。

By precisely targeting the root causes of rare diseases, Actio generates biological insights to enable expansion of development into more prevalent indications..

通过精准针对罕见疾病的根本病因，Actio 生成生物学洞察，从而推动开发工作拓展至更常见的适应症。

The company is advancing two clinical-stage programs: ABS-1230 for the treatment of KCNT1-related epilepsy, and ABS-0871 for the treatment of Charcot-Marie-Tooth disease type 2C including other TRPV4-related neuromuscular disorders (collectively, CMT2C). Actio will also pursue development of both programs in more prevalent indications such as additional genetic epilepsies for ABS-1230 and overactive bladder for ABS-0871.

该公司正在推进两个处于临床阶段的项目：ABS-1230，用于治疗KCNT1相关癫痫；以及ABS-0871，用于治疗夏科-马里-图斯病2C型（Charcot-Marie-Tooth disease type 2C），包括其他TRPV4相关的神经肌肉疾病（统称为CMT2C）。Actio还将推动这两个项目在更常见的适应症中的开发，例如针对ABS-1230的其他遗传性癫痫，以及针对ABS-0871的膀胱过度活动症。

In addition, the company has launched a third program for a rare genetic epilepsy with indication expansion potential in a common central nervous system disorder. For more information, please visit .

此外，公司还启动了一项针对罕见遗传性癫痫的第三项计划，该计划在常见中枢神经系统疾病中具有适应症扩展潜力。欲了解更多信息，请访问。

ActioBiosciences.com

ActioBiosciences.com

and follow the company on

并在以下平台关注该公司

LinkedIn

领英

and

和

X

X

.

。

Cautionary Note Concerning Forward-Looking Statements

关于前瞻性陈述的警示性说明

This Current Report on Form 8-K contains forward-looking statements that involve risks and uncertainties relating to future events and the future performance of Jazz and its subsidiaries, including statements regarding Jazz's proposed acquisition of the Company, the anticipated timing of the closing of the proposed acquisition, the prospective benefits of the proposed acquisition, and the potential of ABS-1230 for the treatment of KCNT1-Related Epilepsy.

本8-K表格当前报告包含前瞻性陈述，这些陈述涉及与未来事件及Jazz及其子公司未来表现相关的风险和不确定性，包括关于Jazz拟议收购本公司、拟议收购预计完成时间、拟议收购的预期利益以及ABS-1230在治疗KCNT1相关癫痫方面的潜力的陈述。

These statements, which represent Jazz's current expectations or beliefs concerning various future events, may contain words such as 'may,' 'will,' 'would,' 'could,' 'expect,' 'anticipate,' 'intend,' 'plan,' 'believe,' 'estimate,' 'project,' 'seek,' 'should,' 'strategy,' 'future,' 'opportunity,' 'potential' or other similar words and expressions indicating future results.

这些陈述代表了 Jazz 对各类未来事件的当前预期或信念，可能包含“可能”、“将”、“将会”、“能够”、“预期”、“预计”、“打算”、“计划”、“相信”、“估计”、“预测”、“寻求”、“应当”、“战略”、“未来”、“机会”、“潜力”等词语，或其他指示未来结果的类似词语和表述。

Actual results could differ materially from those anticipated in these forward-looking statements. Risks that may cause these forward-looking statements to be inaccurate include, without limitation: uncertainties as to the timing of the Merger; the possibility that various closing conditions for the transaction may not be satisfied or waived, including that a governmental entity may prohibit, delay, or refuse to grant approval for the consummation of the transaction; uncertainties related to the achievement of the Development Milestone and the Sales Milestones; the possibility that the transaction does not close; risks related to the parties' ability to realize the anticipated benefits of the proposed acquisition; the risk of disruption to Jazz's or the Company's business; the effects of the transaction on relationships with employees, customers, suppliers, or other business partners; the costly and time-consuming pharmaceutical product development and.

实际结果可能与这些前瞻性陈述中预期的结果存在重大差异。可能导致这些前瞻性陈述不准确的风险包括但不限于：合并时间的不确定性；交易的各项交割条件可能无法满足或被豁免的可能性，包括政府实体可能禁止、延迟或拒绝批准交易的完成；与实现开发里程碑和销售里程碑相关的不确定性；交易未能完成的可能性；与各方实现拟议收购预期收益的能力相关的风险；对Jazz或公司业务造成中断的风险；交易对与员工、客户、供应商或其他业务合作伙伴关系的影响；以及耗时且成本高昂的医药产品开发。

Jazz Contacts:

爵士乐联系人：

Media:

媒体：

[email protected]

[email protected]

; Ireland +353 1 637 2141; U.S. +1 215 867 4948

爱尔兰 +353 1 637 2141；美国 +1 215 867 4948

Investor:

投资者：

[email protected]

[email protected]

; Ireland +353 1 634 7800; U.S. +1 650 496 2717

爱尔兰 +353 1 634 7800；美国 +1 650 496 2717

Actio Biosciences Contacts:

Actio Biosciences 联系方式：

Media:

媒体：

Katie Engleman, 1AB

凯蒂·恩格尔曼，1AB

[email protected]

[email protected]

Investor:

投资者：

Renee Leck, THRUST

蕾妮·莱克，THRUST

[email protected]

[email protected]

SOURCE Jazz Pharmaceuticals plc

来源：Jazz Pharmaceuticals plc

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