Replay and MD Anderson announce FDA clearance of IND application for first-in-class PRAME-targeted T-Cell Receptor Natural Killer (TCR-NK) cell therapy for hematological malignancies

Replay和MD Anderson宣布FDA批准IND申请，用于针对血液系统恶性肿瘤的一流PRAME靶向T细胞受体自然杀伤（TCR-NK）细胞疗法

Phase 1/2 study in patients with relapsed/refractory AML and MDS, anticipated to commence Q3 2024

复发/难治性AML和MDS患者的1/2期研究，预计将于2024年第3季度开始

PRAME is Syena’s second TCR-NK target and follows the NY-ESO-1-targeted TCR-NK program, which has an ongoing clinical study in multiple myeloma with a clinical study in advanced synovial sarcoma, and myxoid/round cell liposarcoma anticipated to commence imminently.

PRAME是Syena的第二个TCR-NK靶标，遵循NY-ESO-1靶向TCR-NK计划，该计划正在进行多发性骨髓瘤的临床研究，并对晚期滑膜肉瘤和粘液样/圆形细胞脂肪肉瘤进行临床研究，预计即将开始。

San Diego, California, London, UK, and Houston, Texas, April 30, 2024 – Replay, a genome writing company reprogramming biology by writing and delivering big DNA, and The University of Texas MD Anderson Cancer Center, today announced that the Food & Drug Administration (FDA) has issued a ‘safe to proceed’ for the Investigational New Drug (IND) application for PRAME TCR/IL-15 NK (SY-307), an engineered T-Cell Receptor Natural Killer (TCR-NK) cell therapy for relapsed/refractory myeloid malignancies.

2024年4月30日，加利福尼亚州圣地亚哥、英国伦敦和德克萨斯州休斯顿——Replay，一家通过编写和传递大DNA对生物学进行重编程的基因组编写公司，以及德克萨斯大学MD安德森癌症中心，今天宣布，美国食品和药物管理局（FDA）已经发布了一份针对恶作剧TCR/IL-15 NK（SY-307）的研究性新药（IND）申请的“安全程序”，这是一种针对复发/难治性骨髓恶性肿瘤的工程化T细胞受体自然杀伤（TCR-NK）细胞疗法。

MD Anderson is the IND sponsor..

MD Anderson是IND赞助商。。

PRAME TCR/IL-15 NK (SY-307) is being developed by Syena, an oncology-focused product company launched by Replay and MD Anderson based on the scientific discoveries of Katy Rezvani, M.D., Ph.D., Professor of Stem Cell Transplantation & Cellular Therapy at MD Anderson. Syena has an exclusive license to Miltenyi Biotec’s PRAME (PReferentially expressed Antigen in MElanoma) T-cell receptor (TCR).

PRAME TCR/IL-15 NK（SY-307）由Syena开发，Syena是一家专注于肿瘤学的产品公司，由Replay和MD Anderson根据MD Anderson干细胞移植和细胞治疗教授Katy Rezvani的科学发现推出。Syena拥有Miltenyi Biotec的PRAME（黑色素瘤中优先表达的抗原）T细胞受体（TCR）的独家许可证。

The engineered PRAME-targeted TCR-NK cell therapy is developed from cord blood-derived natural killer cells that express a high affinity TCR targeting the PRAME tumor-associated neoantigen. PRAME is highly immunogenic and expressed on numerous different cancer types, including hematologic malignancies such as acute myeloid leukemia (AML) and myelodysplastic syndromes (MDS), and solid tumors such as melanoma, sarcoma, and ovarian, endometrial, lung, and breast cancer.

工程化的PRAME靶向TCR-NK细胞疗法是由脐带血衍生的自然杀伤细胞开发的，该细胞表达靶向PRAME肿瘤相关新抗原的高亲和力TCR。PRAME具有高度免疫原性，并在许多不同的癌症类型上表达，包括血液系统恶性肿瘤，如急性髓细胞白血病（AML）和骨髓增生异常综合征（MDS），以及实体瘤，如黑色素瘤，肉瘤，卵巢癌，子宫内膜癌，肺癌和乳腺癌。

The ability of PRAME to elicit humoral and cellular immune responses along with its restricted tissue expression, establishes it as a compelling target for cell therapy-mediated cancer immunotherapy..

PRAME引发体液和细胞免疫反应的能力及其有限的组织表达，使其成为细胞疗法介导的癌症免疫疗法的引人注目的靶标。。

The phase 1/2 open-label study will assess the safety, tolerability, and preliminary efficacy of PRAME TCR/IL-15 NK (SY-307) in patients with relapsed/refractory AML and MDS. It will be administered following lymphodepletion with standard doses of fludarabine/cyclophosphamide (Flu/Cy) and decitabine.

1/2期开放标签研究将评估PRAME TCR/IL-15 NK（SY-307）在复发/难治性AML和MDS患者中的安全性，耐受性和初步疗效。它将在标准剂量的氟达拉滨/环磷酰胺（Flu/Cy）和地西他滨进行淋巴清除后服用。

Up to 44 patients will be enrolled into the study, which is anticipated to commence in Q3 2024..

预计2024年第三季度将有多达44名患者参加这项研究。。

Adrian Woolfson, Executive Chairman, President, and Co-Founder of Replay, said: “PRAME is expressed at high levels in multiple different tumor types, making it a compelling target for engineered TCR-NK cancer immunotherapy. The recent IND clearance of our PRAME TCR/IL-15 NK (SY-307) program for AML and MDS represents a significant expansion of Syena’s growing pipeline of ‘off-the-shelf’ engineered TCR-NK therapies and complements our existing NY-ESO-1 targeted programs in myeloma and sarcoma.

Replay的执行主席、总裁兼联合创始人阿德里安·伍尔夫森（AdrianWoolfson）表示：“PRAME在多种不同的肿瘤类型中高水平表达，使其成为工程化TCR-NK癌症免疫治疗的引人注目的目标。最近IND批准了我们针对AML和MDS的PRAME TCR/IL-15 NK（SY-307）计划，这代表了Syena不断增长的“现成”工程化TCR-NK疗法管道的重大扩展，并补充了我们现有的针对骨髓瘤和肉瘤的NY-ESO-1计划。

This diversification of Syena’s TCR-NK portfolio takes us a step closer to democratizing cell therapy for cancer patients with high unmet medical needs and limited treatment options.”.

Syena TCR-NK组合的这种多样化使我们更接近于为医疗需求未得到满足且治疗选择有限的癌症患者提供民主化的细胞治疗。”。

Lachlan MacKinnon, CEO and Co-Founder of Replay, said: “The recent FDA IND clearance of our PRAME TCR/IL-15 NK (SY-307) cell therapy is demonstrative of the growing momentum at Syena, as the second program enters the clinic. This is illustrative of the strength of our business model to rapidly progress the development of a commercial-scale supply of cell therapy products with significant potential for patients.”.

Replay首席执行官兼联合创始人拉克兰·麦金农（LachlanMacKinnon）表示：“随着第二个项目进入临床，我们的PRAME TCR/IL-15 NK（SY-307）细胞疗法最近获得FDA IND批准，这表明Syena的发展势头越来越强劲。这说明了我们商业模式的优势，可以快速推进商业规模的细胞疗法产品的开发，为患者提供巨大的潜力。”。

Dr Arun Balakumaran, Chief Medical Officer of Replay, said: “PRAME is a well-known cancer-testis antigen with re-expression in multiple cancer types, including AML and solid tumors, and restricted expression on normal tissues. This dichotomous expression pattern and its ability to elicit spontaneous humoral and cellular immune responses render it a promising target for cancer immunotherapy.”.

Replay首席医疗官Arun Balakumaran博士说：“PRAME是一种众所周知的癌症睾丸抗原，在多种癌症类型（包括AML和实体瘤）中重新表达，在正常组织中表达受限。这种二分表达模式及其引发自发体液和细胞免疫反应的能力使其成为癌症免疫治疗的有希望的靶标。”。

Dr Katy Rezvani Professor of Stem Cell Transplantation & Cellular Therapy at MD Anderson said: “This is an exciting milestone in the development of 'off-the-shelf' engineered TCR-NK cell therapies to address significant unmet medical needs. Our hope is the PRAME studies, initially in hematological malignancies and then in solid tumors, will further advance our understanding of the potential for engineered TCR-modified NK cells to benefit patients with relapsed/refractory myeloid malignancies.”.

MD Anderson干细胞移植与细胞治疗教授Katy Rezvani博士说：“这是开发‘现成’工程化TCR-NK细胞疗法以解决重大未满足的医疗需求的一个令人兴奋的里程碑。我们希望PRAME研究，最初是在血液系统恶性肿瘤中，然后是在实体瘤中，将进一步提高我们对工程化TCR修饰的NK细胞对复发/难治性骨髓恶性肿瘤患者有益的潜力的理解。”。

Ends

结束

Disclosure

披露

MD Anderson has an institutional conflict of interest with Replay and Syena, and MD Anderson has implemented an Institutional Conflict of Interest Management and Monitoring Plan to manage these relationships.

MD Anderson与Replay和Syena存在机构利益冲突，MD Anderson实施了机构利益冲突管理和监控计划来管理这些关系。

About Replay

关于Replay

Replay is a genome writing company, which aims to define the future of genomic medicine through reprogramming biology by writing, designing, and delivering big DNA. The Company has assembled a toolkit of platform technologies, including a high payload capacity HSV platform, to address the challenges that currently limit clinical progress and prevent genomic medicine from realizing its full potential..

Replay是一家基因组写作公司，旨在通过编写、设计和交付大DNA来重新编程生物学，从而定义基因组医学的未来。该公司已经组装了一套平台技术工具包，包括一个高负载容量的HSV平台，以应对目前限制临床进展并阻止基因组医学充分发挥其潜力的挑战。。

The Company’s hub-and-spoke business model separates technology development within Replay from therapeutic development in a portfolio of product companies that leverage its technology platforms. Replay’s first-in-class engineered TCR-NK cell therapy product company, Syena, uses technology developed by Katy Rezvani, M.D., Ph.D., at The University of Texas MD Anderson Cancer Center in Houston, Texas.

该公司的中心辐射业务模式将Replay中的技术开发与利用其技术平台的产品公司组合中的治疗开发分开。Replay的第一家一流工程化TCR-NK细胞治疗产品公司Syena使用德克萨斯州休斯顿德克萨斯大学安德森癌症中心医学博士KatyRezvani开发的技术。

Replay’s high payload capacity HSV vector capable of delivering up to 30 times the payload of AAV, is utilized by Replay’s gene therapy product companies, bringing big DNA treatments to diseases affecting the skin, and eye. Replay is led by a distinguished team of academics, entrepreneurs, and seasoned industry experts..

Replay的高有效载荷容量HSV载体能够提供高达AAV有效载荷30倍的有效载荷，被Replay的基因治疗产品公司利用，为影响皮肤和眼睛的疾病带来了大规模的DNA治疗。Replay由杰出的学者、企业家和经验丰富的行业专家组成的团队领导。。

The Company raised $55 million in seed financing in July 2022 and is supported by an international syndicate of investors including: KKR, OMX Ventures, ARTIS Ventures, and Lansdowne Partners.

该公司于2022年7月筹集了5500万美元的种子融资，并得到了包括KKR、OMX Ventures、ARTIS Ventures和Lansdowne Partners在内的国际投资者财团的支持。

Replay is headquartered in San Diego, California. For further information please visit www.replay.bio and follow us on LinkedIn and X.

Replay总部位于加利福尼亚州圣地亚哥。欲了解更多信息，请访问www.replay.bio，并在LinkedIn和X上关注我们。

About MD Anderson

关于MD Anderson

The University of Texas MD Anderson Cancer Center in Houston ranks as one of the world's most respected centers focused on cancer patient care, research, education and prevention. The institution’s sole mission is to end cancer for patients and their families around the world. MD Anderson is one of only 53 comprehensive cancer centers designated by the National Cancer Institute (NCI).

位于休斯顿的德克萨斯大学MD安德森癌症中心是世界上最受尊敬的癌症患者护理、研究、教育和预防中心之一。该机构的唯一使命是为世界各地的患者及其家人终止癌症。MD Anderson是美国国家癌症研究所（NCI）指定的仅有的53个综合癌症中心之一。

MD Anderson is No. 1 for cancer in U.S. News & World Report’s “Best Hospitals” rankings and has been named one of the nation’s top two hospitals for cancer since the rankings began in 1990. MD Anderson receives a cancer center support grant from the NCI of the National Institutes of Health (P30 CA016672)..

MD Anderson在《美国新闻与世界报道》的“最佳医院”排名中排名第一，自1990年开始排名以来，已被评为美国两大癌症医院之一。MD Anderson获得了美国国立卫生研究院NCI（P30 CA016672）的癌症中心支持拨款。。

Contacts:

联系人：

Replay

重播

Dr Adrian Woolfson/Lachlan MacKinnon

Adrian Woolfson博士/Lachlan MacKinnon

info@replay.bio

info@replay.bio

ICR Consilium – media relations

ICR Consilium–媒体关系

Amber Fennell/ David Daley

Amber Fennell/David Daley

replay@consilium-comms.com

replay@consilium-comms.com

MD Anderson

MD安德森

Clayton Boldt, Ph.D.

Clayton Boldt博士。

CRBoldt@MDAnderson.org

CRBoldt@MDAnderson.org