附录99.1

protara therapeutics宣布2024年第三季度财务业绩并提供业务更新。

NEW YORk，2024年11月12日 - protara therapeutics（纳斯达克代码：TARA）公布，这家临床阶段公司为治疗癌症和罕见疾病开发转变性疗法，今天提供了业务更新和截至2024年9月30日的第三季度财务业绩。

“我们持续取得重大进展，推进所有进行中的项目，努力实现将改变生命的疗法带给受癌症和罕见疾病影响的患者的目标，” protara therapeutics首席执行官Jesse Shefferman说。“值得一提的是，我们仍在计划中，计划在2024年第4季度从我们对非肌肉浸润性膀胱癌（NMIBC）患者的TARA-002的ADVANCED-2研究中报告中期数据。考虑到其易于使用、良好的安全性概况以及令人鼓舞的早期临床数据，我们相信TARA-002可能成为NMIBC治疗格局中有影响力的补充物。”

Shefferman先生补充说，“除了我们的NMIBC计划外，我们仍然按计划将于2025年第一季度开始最关键的THRIVE-3研究，该研究探讨静脉注射(IV)氯化胆碱，我们最近从美国食品和药物管理局(FDA)获得了快速通道指定。最后，我们预计将于2025年上半年从进行中的TARA-002对淋巴管畸形(LMs)进行的第2期STARBORN-1试验中获得中期数据，这是一种罕见的疾病，主要影响儿童，目前尚无FDA批准的治疗方案。

最新进展和亮点

NMIBC中的TARA-002

用于接受输液支持(PS)的静脉注射氯化胆碱

TARA-002 in LMs

2024年第三季度财务结果

关于TARA-002

TARA-002是一种研究性细胞治疗，用于治疗NMIBC和LMS，并已获得美国食品和药物管理局授予的罕见小儿疾病认定。TARA-002是从日本中外制药市场上市的广泛免疫增强剂Picibanil®所使用的一种基因不同的A群链球菌主细胞库开发而来。protara therapeutics已成功展示了TARA-002与Ok-432之间的制造可比性。

当TARA-002被施用时，假设囊肿或肿瘤内的固有和适应性免疫细胞被激活，并释放细胞激素如肿瘤坏死因子（TNF）-alpha、干扰素（IFN）-γ IL-6、IL-10、IL-12等进行炎症反应。TARA-002也直接杀死肿瘤细胞，并通过诱导免疫原性细胞死亡触发宿主免疫反应，从而进一步增强抗肿瘤的免疫反应。

关于非肌层侵袭性膀胱癌（NMIBC）

膀胱癌在美国是第六大常见的癌症，非肌侵性膀胱癌约占膀胱癌诊断的约80％。每年在美国约有6.5万名患者被诊断为非肌侵性膀胱癌。非肌侵性膀胱癌是指发现在膀胱内表面内层组织中的癌症，并且未扩散到膀胱肌层。

关于淋巴管畸形（LMs）

LMs是淋巴管的罕见先天畸形，导致这些结构无法连接或排出到静脉系统。大多数LMs存在于头部和颈部区域，并在早期儿童中进行诊断，期间淋巴生长活跃，超过50％在出生时检测到，90％在三岁之前诊断。该病最常见的弊端和严重表现包括压迫上呼吸消化道，包括需要插管和可能需要气管造口依赖；局部出血；对重要结构的侵袭，包括神经，血管，淋巴管；反复感染，以及外观和其他功能残疾。

关于IV磷脂醯胆碱（Choline Chloride）

IV磷脂醯胆碱是一种正在开发中的探索性药物，用于接受输液支持的病人的静脉磷脂质替代疗法。胆碱是磷脂质的已知重要基质，对于健康的肝脏功能至关重要，同时在调节基因表达，细胞膜信号，大脑发育和神经传导，肌肉功能和骨骼健康中起著重要作用。接受输液支持的病人无法从肠道营养来源合成胆碱，目前没有含有胆碱的输液支持制剂。依赖输液支持的患者约80％缺乏胆碱，并且有一定程度的肝损伤，可能导致肝功能衰竭。每年在美国约有9万人需要在家接受输液支持，其中约有3万人长期需要输液支持。IV磷脂醯胆碱有潜力成为首个获得美国食品和药物管理局（FDA）批准的用于输液支持患者的IV胆碱配方，并获得FDA授予孤儿药品称号，用于预防输液支持患者的胆碱缺乏。美国专利商标局已向该公司授予了请求胆碱组成的美国专利和一种用胆碱组成治疗胆碱缺乏的方法的美国专利，每个专利期均至2041年到期。

About Protara Therapeutics, Inc.

Protara is a clinical-stage biotechnology company committed to advancing transformative therapies for people with cancer and rare diseases. Protara’s portfolio includes its lead candidate, TARA-002, an investigational cell-based therapy in development for the treatment of non-muscle invasive bladder cancer (NMIBC) and lymphatic malformations (LMs). The Company is evaluating TARA-002 in an ongoing Phase 2 trial in NMIBC patients with carcinoma in situ (CIS) who are unresponsive or naïve to treatment with Bacillus Calmette-Guérin (BCG), as well as a Phase 2 trial in pediatric patients with LMs. Additionally, Protara is developing IV Choline Chloride, an investigational phospholipid substrate replacement for patients on parenteral nutrition who are otherwise unable to meet their choline needs via oral or enteral routes. For more information, visit www.protaratx.com.

参考文献

1. Klinische Einheit, or KE, is a German term indicating a specified weight of dried cells in a vial.

前瞻性陈述

Statements contained in this press release regarding matters that are not historical facts are “forward looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995. Protara may, in some cases, use terms such as “predicts,” “believes,” “potential,” “proposed,” “continue,” “designed,” “estimates,” “anticipates,” “expects,” “plans,” “intends,” “may,” “could,” “might,” “will,” “should” or other words or expressions referencing future events, conditions or circumstances that convey uncertainty of future events or outcomes to identify these forward-looking statements. Such forward-looking statements include but are not limited to, statements regarding Protara’s intentions, beliefs, projections, outlook, analyses or current expectations concerning, among other things: Protara’s business strategy, including its development plans for its product candidates and plans regarding the timing or outcome of existing or future clinical trials; statements related to expectations regarding interactions with the FDA; Protara’s financial position; statements regarding the anticipated safety or efficacy of Protara’s product candidates; and Protara’s outlook for the remainder of the year. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Factors that contribute to the uncertain nature of the forward-looking statements include: risks that Protara’s financial guidance may not be as expected, as well as risks and uncertainties associated with: Protara’s development programs, including the initiation and completion of non-clinical studies and clinical trials and the timing of required filings with the FDA and other regulatory agencies; general market conditions; changes in the competitive landscape; changes in Protara’s strategic and commercial plans; Protara’s ability to obtain sufficient financing to fund its strategic plans and commercialization efforts; having to use cash in ways or on timing other than expected; the impact of market volatility on cash reserves; failure to attract and retain management and key personnel; the impact of general U.S. and foreign, economic, industry, market, regulatory, political or public health conditions; and the risks and uncertainties associated with Protara’s business and financial condition in general, including the risks and uncertainties described more fully under the caption “Risk Factors” and elsewhere in Protara’s filings and reports with the United States Securities and Exchange Commission. All forward-looking statements contained in this press release speak only as of the date on which they were made and are based on management’s assumptions and estimates as of such date. Protara undertakes no obligation to update any forward-looking statements, whether as a result of the receipt of new information, the occurrence of future events or otherwise, except as required by law.

protara therapeutics, inc.及其子（或附属）公司
未经审核的简化合并资产负债表
(以千为单位，每股数据除外)

protara therapeutics, inc.及其附属公司
未经审计之综合损益及综合亏损表
(以千为单位，每股数据除外)

公司联络人:

Justine O’Malley
protara therapeutics
Justine.OMalley@protaratx.com
646-817-2836