CAR031 study at 9.03-mo median follow up achieves disease control rate (DCR) of 91.3% and objective response rate (ORR) of 56.5% for patients across all dose levels (DLs) and ORR of 75.0% at DL4

CAR031研究在9.03个月的中位随访中，所有剂量水平（DLs）的患者的疾病控制率（DCR）为91.3%，客观缓解率（ORR）为56.5%，DL4的ORR为75.0%

ROCKVILLE, Md., June 4, 2024 /PRNewswire/ -- AbelZeta Pharma, Inc. ('AbelZeta' or the 'Company'), a global clinical-stage biopharmaceutical company focused on the discovery and development of innovative and proprietary cell-based therapeutic products, today announced preliminary safety and efficacy results from its first time in human investigator-initiated trial (IIT) of C-CAR031 in connection with the Company's oral presentation at the 2024 American Society of Clinical Oncology (ASCO) Annual Meeting.

罗克维尔，马里兰州，2024年6月4日/PRNewswire/--AbelZeta Pharma，Inc.（“AbelZeta”或“公司”）是一家全球临床阶段的生物制药公司，专注于发现和开发创新和专有的基于细胞的治疗产品，今天宣布了其首次在人类研究者发起的C-CAR031试验（IIT）中的初步安全性和有效性结果，该试验与该公司在2024年美国临床肿瘤学会（ASCO）年会上的口头介绍有关。

The presentation shared data indicating a manageable safety profile and encouraging anti-tumor activity of C-CAR031 in patients with heavily pretreated advanced hepatocellular carcinoma (HCC) (1-6 lines of prior therapy). C-CAR031 is based on a novel GPC3-targeting CAR-T designed by AstraZeneca (LSE/STO/Nasdaq: AZN) and is manufactured by AbelZeta.

该演讲分享了数据，表明C-CAR031在严重预处理的晚期肝细胞癌（HCC）（1-6行先前治疗）患者中具有可控的安全性和令人鼓舞的抗肿瘤活性。C-CAR031基于阿斯利康（LSE/STO/Nasdaq:AZN）设计的针对CAR-T的新型GPC3，由AbelZeta制造。

C-CAR031 is being co-developed in China by AbelZeta and AstraZeneca..

AbelZeta和AstraZeneca正在中国共同开发C-CAR031。。

'We are encouraged by the first clinical results of C-CAR031 in advanced hepatocellular carcinoma (HCC) patients'

“我们对C-CAR031在晚期肝细胞癌（HCC）患者中的首次临床结果感到鼓舞”

'We are encouraged by the first clinical results of C-CAR031 in advanced hepatocellular carcinoma (HCC) patients,' said Tony (Bizuo) Liu, Chairman and CEO of AbelZeta. 'The early data presented today provide compelling proof-of-concept to potentially redefine therapeutic paradigms in HCC and other GPC3-expressing solid tumors.'.

AbelZeta董事长兼首席执行官Tony（Bizuo）Liu说，我们对C-CAR031在晚期肝细胞癌（HCC）患者中的首次临床结果感到鼓舞今天提供的早期数据提供了令人信服的概念证明，有可能重新定义HCC和其他表达GPC3的实体瘤的治疗范例。”。

Principal Investigator (PI) of the study, Professor Tingbo Liang from the First Affiliated Hospital of Zhejiang University, stated 'C-CAR031 showed a good safety profile and promising efficacy in late-stage hepatocellular carcinoma patients, who typically have a limited number of treatment options available.

该研究的首席研究员（PI）浙江大学第一附属医院的梁廷波教授表示，'C-CAR031在晚期肝细胞癌患者中表现出良好的安全性和有希望的疗效，这些患者通常有有限的治疗选择。

The observed tumor shrinkage in the large/vast majority (91.3%) of the patients suggests that C-CAR031 has the potential to bring clinical value and offer hope to these patients.'.

在大多数/绝大多数（91.3%）患者中观察到的肿瘤缩小表明，C-CAR031有可能为这些患者带来临床价值和希望。”。

As of March 14, 2024, 23 of 24 patients on the study were eligible for efficacy assessment. Tumor reductions were observed in 91.3% patients, in both intrahepatic and extrahepatic lesions, with a median reduction of 42.2% (range, -28.1% 94.4%). The disease control rate was 91.3% and the ORR was 56.5% for patients across all DLs.

截至2024年3月14日，该研究的24名患者中有23名有资格进行疗效评估。在肝内和肝外病变中，91.3%的患者肿瘤减少，中位数减少42.2%（范围-28.1%94.4%）。所有DLs患者的疾病控制率为91.3%，ORR为56.5%。

In DL4, the ORR was 75.0%. With 9.03-month median follow-up, Kaplan-Meier estimation of median overall survival (mOS) is 11.14 months (95% CI, 7.56-NE)..

在DL4中，ORR为75.0%。中位随访9.03个月，Kaplan-Meier估计中位总生存期（mOS）为11.14个月（95%CI，7.56-NE）。。

No dose-limiting toxicity or immune effector cell associated neurotoxicity syndrome (ICANS) was observed. Cytokine release syndrome (CRS) was observed in 22 (91.7%) patients with the majority (87.5%) grade 1/2 CRS  and only 1 (4.2%) grade 3 CRS.

未观察到剂量限制性毒性或免疫效应细胞相关神经毒性综合征（ICANS）。在22例（91.7%）患者中观察到细胞因子释放综合征（CRS），其中大多数（87.5%）为1/2级CRS，只有1例（4.2%）为3级CRS。

About C-CAR031

关于C-CAR031

C-CAR031 is an autologous, armored GPC3-targeting chimeric antigen receptor T-Cell (CAR-T) therapy, being studied for the treatment of HCC. It is based on a novel GPC3-targeting CAR-T designed by AstraZeneca using their dominant negative transforming growth factor-beta receptor II dominant negative (dnTGFβRII) armoring discovery platform and is manufactured by AbelZeta in China.

C-CAR031是一种自体，装甲GPC3靶向嵌合抗原受体T细胞（CAR-T）疗法，正在研究用于治疗HCC。它基于阿斯利康使用其显性负性转化生长因子β受体II显性负性（dnTGFβRII）铠装发现平台设计的新型GPC3靶向CAR-T，由AbelZeta在中国制造。

C-CAR031 is being developed in China under a co-development agreement between AbelZeta and AstraZeneca.1.

根据AbelZeta和AstraZeneca之间的共同开发协议，C-CAR031正在中国开发。

About the Study

关于这项研究

A Phase I clinical study (NCT05155189) aiming to assess the safety and anti-tumor activity of C-CAR031 injection in advanced/unresectable HCC patients is being conducted in China. As of March 14, 2024, a total of 24 patients received C-CAR031 infusion at 4 dose levels (DLs). 83.3% (20/24) had extrahepatic metastasis.

中国正在进行一项I期临床研究（NCT05155189），旨在评估C-CAR031注射液在晚期/不可切除的HCC患者中的安全性和抗肿瘤活性。截至2024年3月14日，共有24名患者接受了4个剂量水平（DLs）的C-CAR031输注。83.3%（20/24）有肝外转移。

The median number of prior lines of systemic therapy was 3.5 (range 1-6)..

先前全身治疗的中位数为3.5（范围1-6）。。

Abstract Title: 'Phase I study of C-CAR031, a GPC3-specific TGFβRIIDN armored autologous CAR-T, in patients with advanced hepatocellular carcinoma (HCC).'

摘要标题：“晚期肝细胞癌（HCC）患者中GPC3特异性TGFβRIIDN装甲自体CAR-T C-CAR031的I期研究。”

Abstract Number: 4019

摘要编号：4019

Session Type and Title: Rapid Oral Abstract – Gastrointestinal Cancer – Gastroesophageal, Pancreatic, and Hepatobiliary

会议类型和标题：快速口头摘要-胃肠道癌-胃食管癌，胰腺癌和肝胆癌

Session Date and Time: 6/3/2024; 9:45 AM-11:15 AM CDT

会议日期和时间：2024年6月3日；9： CDT上午45:00-11:15

About AbelZeta Pharma, Inc.

关于AbelZeta Pharma，Inc。

AbelZeta is a global clinical-stage biopharmaceutical company with centers of excellence in Rockville, Maryland and Shanghai, China. AbelZeta is focusing on developing innovative and proprietary cell-based therapeutic products and is committed to ushering in bespoke treatments that harness the body's own immune system to fight against hematological malignancies and solid tumors, as well as inflammatory and immunological diseases.

AbelZeta是一家全球临床阶段生物制药公司，在马里兰州罗克维尔和中国上海设有卓越中心。AbelZeta专注于开发创新和专有的基于细胞的治疗产品，并致力于开创定制的治疗方法，利用人体自身的免疫系统来对抗血液系统恶性肿瘤和实体瘤，以及炎症和免疫疾病。

AbelZeta advances research and development in its own GMP facilities at its centers of excellence for early-stage clinical studies, with a pipeline comprised of CAR-T and TIL therapies..

AbelZeta在其早期临床研究卓越中心的GMP设施中推进研究和开发，其管道由CAR-T和TIL疗法组成。。

Forward-Looking Statements

前瞻性声明

Statements in this communication relating to plans, strategies, specific activities, and other statements that are not descriptions of historical facts are forward-looking statements. Forward-looking information is inherently subject to risks and uncertainties, and actual results could differ materially from those currently anticipated due to a number of factors, which include any risks detailed from time to time in the Company's reports.

本通讯中有关计划、战略、具体活动以及其他不属于历史事实描述的陈述均为前瞻性陈述。前瞻性信息固有地受到风险和不确定性的影响，由于多种因素，实际结果可能与目前预期的结果存在重大差异，其中包括公司报告中不时详述的任何风险。

Such statements are based on the management's current beliefs and expectations and are subject to significant risks and uncertainties outside of management and the Company's control. Given these uncertainties, you should not place undue reliance on these forward-looking statements, which speak only as of the date hereof.

此类声明基于管理层当前的信念和期望，并受到管理层和公司控制之外的重大风险和不确定性的影响。鉴于这些不确定性，您不应过度依赖这些前瞻性声明，这些声明仅在本协议签署之日起生效。

Except as otherwise required by law, the Company does not undertake any obligation, and expressly disclaims any obligation, to update, alter or otherwise revise any forward-looking statements, whether written or oral, that may be made from time to time, whether as a result of new information, future events or otherwise..

除法律另有规定外，公司不承担任何义务，也明确否认任何义务，更新、更改或以其他方式修改任何前瞻性声明，无论是书面还是口头，无论是由于新信息、未来事件还是其他原因。。

Company Contact:

公司联系人：

Sarah Kelly

莎拉·凯利

Director of Communications

通信总监

AbelZeta Pharma, Inc.

AbelZeta制药股份有限公司。

+1 (240) 552 5870

+1 (240) 552 5870

sarah.kelly@abelzeta.com

sarah.kelly@abelzeta.com

www.abelzeta.com

www.abelzeta.com

References

参考文献

1. AbelZeta Pharma Announces Agreement with AstraZeneca to Co-Develop a novel Glypican 3 (GPC3) Armored CAR-T Therapy in China. December 2023. https://www.abelzeta.com/abelzeta-pharma-announces-agreement-with-astrazeneca-to-co-develop-a-novel-glypican-3-gpc3-armored-car-t-therapy-in-china/

1.AbelZeta Pharma宣布与阿斯利康达成协议，在中国共同开发一种新型磷脂酰肌醇蛋白聚糖3（GPC3）装甲CAR-T疗法。2023年12月。https://www.abelzeta.com/abelzeta-pharma-announces-agreement-with-astrazeneca-to-co-develop-a-novel-glypican-3-gpc3-armored-car-t-therapy-in-china/

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SOURCE AbelZeta Pharma, Inc.

来源AbelZeta Pharma，Inc。

Company Codes: LSE:AZN, NASDAQ-NMS:AZN, Stockholm:AZN

公司代码：LSE:AZN，NASDAQ-NMS:AZN，斯德哥尔摩：AZN