- NDA is supported by data evaluating seladelpar efficacy and safety in over 500 patients -

-NDA得到了评估500多名患者seladelpar疗效和安全性的数据的支持-

- Seladelpar is the first New Drug Application from CymaBay in its ongoing mission to help people living with PBC -

-Seladelpar是CymaBay正在进行的帮助PBC患者的任务中的第一个新药申请-

NEWARK, Calif., Dec. 15, 2023 /PRNewswire/ -- CymaBay Therapeutics, Inc. (NASDAQ: CBAY), a biopharmaceutical company focused on innovative therapies for patients with liver and other chronic diseases, today announced it has submitted a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for investigational treatment, seladelpar, for the management of primary biliary cholangitis, including pruritus in adults without cirrhosis or with compensated cirrhosis (Child Pugh A) who are inadequate responders or intolerant to ursodeoxycholic acid (UDCA).

加利福尼亚州纽瓦克，2023年12月15日，一家专注于肝脏和其他慢性病患者创新疗法的生物制药公司CymaBay Therapeutics，Inc.（纳斯达克：CBAY），今天宣布已向美国食品和药物管理局（FDA）提交新药申请（NDA），以进行研究性治疗，seladelpar，用于治疗原发性胆汁性胆管炎，包括对熊去氧胆酸（UDCA）反应不足或不耐受的无肝硬化或代偿性肝硬化（Child-Pugh A）的成年人的瘙痒症。

CymaBay has requested a Priority Review of the NDA that, if accepted, would mean the FDA would plan to complete its review within six months of accepting the application..

CymaBay要求优先审查NDA，如果被接受，意味着FDA将计划在接受申请后的六个月内完成审查。。

Seladelpar has been granted Breakthrough Therapy Designation by the FDA and is the only potent, selective, orally active delpar, or PPARδ agonist, with Phase 3 trial results demonstrating a statistically significant improvement in markers of cholestasis related to risk of progression and PBC-related pruritus.

Seladelpar已被FDA授予突破性治疗称号，是唯一有效的，选择性的，口服活性的delpar或PPARδ激动剂，3期试验结果表明，与进展风险和PBC相关瘙痒症相关的胆汁淤积标志物有统计学显着改善。

The comprehensive NDA application is supported by robust data evaluating the efficacy and tolerability profile of seladelpar in more than 500 participants with PBC in the placebo-controlled Phase 3 RESPONSE and ENHANCE studies, the long-term open-label ASSURE study, as well as in prior Phase 2 studies..

全面的NDA应用得到了可靠数据的支持，这些数据评估了seladelpar在安慰剂对照的3期反应和强化研究，长期开放标签ASSURE研究以及之前的2期研究中对500多名PBC参与者的疗效和耐受性概况。。

Breakthrough Therapy Designation is granted by the FDA to investigational agents intended to treat a serious condition and preliminary clinical evidence indicates that it may demonstrate substantial improvement over available therapy on a clinically significant endpoint. FDA updated the Breakthrough Therapy Designation for seladelpar in October 2023 in recognition of data that indicates seladelpar may provide meaningful improvement over existing therapy based on a reduction in alkaline phosphatase (ALP) and improvement in pruritus in patients without cirrhosis or with compensated cirrhosis..

FDA将突破性治疗指定给旨在治疗严重疾病的研究药物，初步临床证据表明，它可能会在临床上显着改善可用治疗的终点。FDA于2023年10月更新了seladelpar的突破性治疗名称，因为有数据表明，基于无肝硬化或代偿性肝硬化患者碱性磷酸酶（ALP）的降低和瘙痒的改善，seladelpar可能比现有治疗提供有意义的改善。。

'People living with PBC need new treatment options to reduce the risk of disease progression and the daily impact of their disease from debilitating symptoms like pruritus. Today's announcement of our submission of the NDA for seladelpar is an important milestone in our ongoing work to bring forward new innovative therapies with the potential to help people living with PBC,' said Klara Dickinson, Chief Regulatory and Compliance Officer, CymaBay Therapeutics.

“患有PBC的人需要新的治疗选择，以降低疾病进展的风险以及瘙痒等衰弱症状对疾病的日常影响。CymaBay Therapeutics首席监管和合规官克拉拉·迪金森（KlaraDickinson）说，我们今天宣布向塞拉德尔帕尔提交保密协议，这是我们正在进行的工作中的一个重要里程碑，我们正在努力提出新的创新疗法，有可能帮助患有PBC的人。

'We would like to extend our thanks to the participants, their families, and all the investigators who have taken part in studies of seladelpar to date, which together have enabled the clinical development and submission of seladelpar for FDA approval. We now look forward to working with the FDA to secure validation of the application and throughout its review.'.

“我们要感谢参与者，他们的家人，以及迄今为止参与seladelpar研究的所有研究人员，他们共同促成了seladelpar的临床开发和提交FDA批准。我们现在期待着与FDA合作，确保应用程序的验证和整个审查过程。”。

CymaBay intends to file marketing authorization applications to the European Medicines Agency (EMA) and U.K. Medicines and Healthcare products Regulatory Agency (MHRA) in the first half of 2024. Seladelpar has received Priority Medicines (PRIME) status from the EMA, as part of the program to optimize development plans and speed up evaluation of priority medicines so they can reach patients earlier..

CymaBay计划在2024年上半年向欧洲药品管理局（EMA）和英国药品和保健品管理局（MHRA）提交上市授权申请。Seladelpar已获得欧洲药品管理局（EMA）的优先药物（PRIME）地位，作为优化发展计划和加快优先药物评估的计划的一部分，以便能够更早地到达患者。。

About PBCPBC is a rare, chronic inflammatory liver disease primarily affecting women (1 in 1,000 women over the age of 40 or about 130,000 total people in the U.S.). PBC is characterized by impaired bile flow (known as cholestasis) and the accumulation of toxic bile acids in the liver, leading to inflammation and destruction of the bile ducts within the liver and causing increased levels of ALP, alanine aminotransferase (ALT), and gamma-glutamyl transferase (GGT), enzymes found primarily in the liver, as well as total bilirubin.

关于PBCPBC是一种罕见的慢性炎症性肝病，主要影响女性（40岁以上女性中有1/1000，美国总人口约13万）。PBC的特征是胆汁流量受损（称为胆汁淤积）和肝脏中有毒胆汁酸的积累，导致肝脏内胆管的炎症和破坏，并导致ALP水平升高，丙氨酸氨基转移酶（ALT）和γ-谷氨酰转移酶（GGT）是主要存在于肝脏以及总胆红素中的酶。

The most common early symptoms of PBC are pruritus (itching) and fatigue, which can be debilitating for some patients. Progression of PBC is associated with an increased risk of liver-related mortality..

PBC最常见的早期症状是瘙痒和疲劳，这可能会使一些患者虚弱。PBC的进展与肝脏相关死亡风险增加有关。。

About SeladelparSeladelpar, an investigational treatment for people with PBC, is a first-in-class oral, selective peroxisome proliferator-activated receptor delta (PPARδ) agonist, or delpar, shown to regulate critical metabolic and liver disease pathways in indications with high unmet medical need. Preclinical and clinical data support its ability to regulate genes involved in bile acid synthesis, inflammation, fibrosis and lipid metabolism, storage, and transport..

关于SeladelparSeladelpar，一种针对PBC患者的研究性治疗方法，是一种一流的口服选择性过氧化物酶体增殖物激活受体δ（PPARδ）激动剂或delpar，可调节严重未满足医疗需求的关键代谢和肝病途径。临床前和临床数据支持其调节胆汁酸合成，炎症，纤维化和脂质代谢，储存和运输相关基因的能力。。

About CymaBayCymaBay Therapeutics, Inc. is a clinical-stage biopharmaceutical company focused on improving the lives of people with liver and other chronic diseases that have high unmet medical need through a pipeline of innovative therapies. Our deep understanding of the underlying mechanisms of liver inflammation and fibrosis, and the unique targets that play a role in their progression, have helped us receive breakthrough therapy designation (FDA), Priority Medicines status (EMA), and orphan drug status (U.S.

关于CymaBayCymaBay Therapeutics，Inc.是一家临床阶段的生物制药公司，专注于通过创新疗法的渠道改善肝脏和其他慢性疾病患者的生活，这些患者的医疗需求尚未得到满足。我们对肝脏炎症和纤维化的潜在机制以及在其进展中发挥作用的独特靶点的深入了解，帮助我们获得了突破性治疗指定（FDA），优先药物状态（EMA）和孤儿药物状态（美国）。

and Europe) for seladelpar, a first-in-class investigational treatment for people with PBC. Our evidence-based decision-making and commitment to the highest quality standards reflect our relentless dedication to the people, families, and communities we serve. To learn more, visit www.cymabay.com and follow us on X (formerly Twitter) and LinkedIn..

和欧洲）用于seladelpar，这是针对PBC患者的一流研究治疗。我们基于证据的决策和对最高质量标准的承诺反映了我们对我们服务的人民、家庭和社区的不懈奉献。要了解更多信息，请访问www.cymabay.com，并在X（以前的Twitter）和LinkedIn上关注我们。。

Cautionary StatementsAny statements made in this press release regarding potential FDA acceptance of the seladelpar NDA, its subsequent review and the timing thereof, the potential for seladelpar to treat PBC and potentially improve ALP levels, clinical symptoms or outcomes of the disease, the future EMA and MHRA filing plans of CymaBay and the timing thereof are forward-looking statements that are subject to risks and uncertainties.

警示声明本新闻稿中关于FDA可能接受seladelpar NDA，随后的审查及其时间安排，seladelpar治疗PBC并可能改善ALP水平，临床症状或疾病结局的所有声明，CymaBay未来的EMA和MHRA备案计划及其时间安排是前瞻性陈述，具有风险和不确定性。

Actual results and the timing of events regarding the further development of seladelpar could differ materially from those anticipated in such forward-looking statements as a result of risks and uncertainties, which include, without limitation, risks related to: the success, cost and timing of any of CymaBay's product development activities, including clinical trials; and effects observed in trials to date that may not be repeated in the future.

由于风险和不确定性，seladelpar进一步开发的实际结果和事件发生的时间可能与此类前瞻性声明中预期的结果有很大差异，其中包括但不限于与以下相关的风险：CymaBay任何产品开发活动（包括临床试验）的成功，成本和时间；以及迄今为止在试验中观察到的未来可能不会重复的效果。

Additional risks relating to CymaBay are contained in CymaBay's filings with the Securities and Exchange Commission, including without limitation its most recent Annual Report on Form 10-K, its Quarterly Reports on Form 10-Q and other documents subsequently filed with or furnished to the Securities and Exchange Commission.

与CymaBay相关的其他风险包含在CymaBay向美国证券交易委员会提交的文件中，包括但不限于其最新的10-K表年度报告、10-Q表季度报告以及随后向美国证券交易委员会提交或提供的其他文件。

CymaBay disclaims any obligation to update these forward-looking statements except as required by law..

CymaBay不承担更新这些前瞻性声明的任何义务，除非法律要求。。

For additional information about CymaBay visit www.cymabay.com.

有关CymaBay的更多信息，请访问www.CymaBay.com。

Public Relations Contact:

公共关系联系人：

Theresa DolgeEvoke Kyne(609) 915-2156  [email protected]

Theresa DolgeEvoke Kyne（609）915-2156[受电子邮件保护]

Investor Relations Contact:

投资者关系联系人：

PJ KelleherLifeSci Advisors, LLC(617) 430-7579[email protected]

PJ KelleherLifeSci Advisors，LLC（617）430-7579[受电子邮件保护]

SOURCE CymaBay Therapeutics

来源：CymaBay Therapeutics