An Open-label, Single-arm, Multicohort, Phase 2 Study to Assess the Efficacy and Safety of Tabelecleucel in Subjects With Epstein-Barr Virus-associated Diseases (EBVision)

The purpose of this study is to assess the efficacy and safety of tabelecleucel in participants with EBV-associated diseases.

开始日期2021-07-14

申办/合作机构

Pierre Fabre Medicament Information

NCT03988582

/ Withdrawn临床2期IIT

A Phase II Trial of EBV-Specific Cytotoxic T Cells for the Treatment of EBV Lymphomas or Other EBV-associated Malignancies

The purpose of this study is to test whether treatment with EBV-specific cytotoxic T cells (EBV-CTLs) is effective, and to test any good and bad effects of treatment with EBV-CTLs. EBV-CTLs are a special immune cells that may attack abnormal cells. EBV-CTLs are made by taking cells from a healthy person, growing them in a laboratory for several weeks to educate them to recognize and destroy EBV infected cells, and then storing them in a freezer until they are required for treatment.

开始日期2019-06-11

申办/合作机构

Memorial Sloan Kettering Cancer Center

NCT03769467

/ Terminated临床1/2期

An Open-Label Phase 1B/2 Study to Evaluate the Safety and Efficacy of Tabelecleucel in Combination With Pembrolizumab in Subjects With Platinum-pretreated, Recurrent/Metastatic Epstein-Barr Virus-Associated Nasopharyngeal Carcinoma

This is a multicenter, open-label, single-arm Phase 1B/2 study to assess the safety and efficacy of tabelecleucel in combination with pembrolizumab for the treatment of subjects with platinum-pretreated, recurrent/metastatic Epstein-Barr Virus-associated Nasopharyngeal Carcinoma (EBV+ NPC).

开始日期2019-02-19

申办/合作机构

Atara Biotherapeutics, Inc.

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100 项与 Tabelecleucel 相关的临床结果

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100 项与 Tabelecleucel 相关的转化医学

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100 项与 Tabelecleucel 相关的专利（医药）

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项与 Tabelecleucel 相关的文献（医药）

2025-07-01·ADVANCES IN THERAPY

Tabelecleucel in Post-transplant Epstein–Barr Virus-Associated Lymphoproliferative Disease: Patient and Oncologist Perspectives

Article

作者: Perez-Martinez, Antonio ; Ramos Boluda, Esther ; Hernandez Oliveros, Francisco ; Gonzalez Martinez, Berta ; Lastra Rodrigo, Anarbella ; Alcolea Sánchez, Alida

This article is coauthored by the parents of a young patient and the medical team who cared for her.Together, they recount their shared experience with tabelecleucel, a novel therapy for Epstein-Barr virus-associated post-transplant lymphoproliferative disease (EBV + PTLD). The patient's parents offer a deeply personal perspective, reflecting on several years of struggle marked by unwavering hope and trust in the medical professionals who have supported their daughter throughout her journey. The pediatric hemato-oncologist who advocated for the use of tabelecleucel described the case as unique worldwide.Their joint testimony tells the remarkable story of how tabelecleucel, despite an initially unclear biological rationale, achieved a reversal of a severe case of EBV + PTLD that had progressed to a point considered beyond recovery and associated with a dire prognosis.

2025-04-01·Journal of Managed Care & Specialty Pharmacy

The effectiveness and value of tabelecleucel for the treatment of Epstein-Barr virus–positive posttransplant lymphoproliferative disease

Article

作者: Richardson, Marina ; Lin, Grace A. ; McKenna, Avery ; Lee, Woojung ; Raymond, Finn ; Agboola, Foluso

2024-12-31·JOURNAL OF MEDICAL ECONOMICS

Comparative analysis of tabelecleucel and current treatment in patients with Epstein-Barr virus-positive post-transplant lymphoproliferative disease following hematopoietic cell transplant or solid organ transplant

Article

作者: Macabeo, Bérengère ; Xing, Baodong ; Zimmermann, Heiner ; Guzman-Becerra, Norma ; Brookhart, M. Alan ; Barlev, Arie ; Thivolet, Marion ; Mehta, Aditi

AIMS:

With recent European Union marketing authorization, tabelecleucel is the first off-the-shelf, allogeneic Epstein-Barr virus (EBV)-specific T-cell immunotherapy approved for the treatment of relapsed/refractory EBV-positive post-transplant lymphoproliferative disease (EBV⁺ PTLD). In the absence of a control arm, real-world evidence can provide a comparative benchmark for single-arm studies in ultra-rare populations. This study assessed the treatment effect of tabelecleucel in the single-arm phase 3 ALLELE study (NCT03394365) versus a treatment group from a multinational, multicenter retrospective chart review study (RS002) of patients with EBV⁺ PTLD.

METHODS:

In ALLELE, patients had disease relapsed/refractory to rituximab ± chemotherapy and received tabelecleucel 2x10⁶ cells/kg on days 1, 8, and 15 in 35-day cycles. Patients in RS002 had disease relapsed/refractory to rituximab ± chemotherapy and received next line of systemic therapy between January 2000 and December 2018. Propensity score-based standardized mortality/morbidity ratio weighting was used to achieve balance between treatment and comparator arms. Kaplan-Meier estimators and Cox regression models were used to compare overall survival (OS) in the re-weighted sample.

RESULTS:

30 patients (n = 14 hematopoietic cell transplant [HCT], n = 16 solid organ transplant [SOT]) from ALLELE (data cutoff: November 2021) and 84 patients (n = 36 HCT, n = 48 SOT) from RS002 (data lock: January 2021) were included. Median time from diagnosis to first tabelecleucel dose (ALLELE) or start date of next line of systemic therapy (RS002) was 3.6 months. Tabelecleucel was associated with a substantial OS benefit compared with current treatment, with an unadjusted HR of 0.47 (95% confidence interval [CI] 0.25-0.88) and adjusted HR of 0.37 (95% CI 0.20-0.71) when using the start date of the next line of therapy as the index date. Sensitivity analyses yielded consistent results.

CONCLUSIONS:

In this study of real-world data, tabelecleucel was associated with an OS benefit among patients with R/R EBV⁺ PTLD for whom there is high unmet need.

151

项与 Tabelecleucel 相关的新闻（医药）

2025-10-16

·丁香园 Insight 数据库

近十年：中国抗肿瘤新药获 FDA 批准成功率仅为 5.2%，出海挑战究竟为何？

最近十年，中国创新药研发取得了长足进步，尤其是抗肿瘤领域。根据丁香园 Insight 数据库，从 2015 年 1 月 1 日到 2025 年 1 月 1 日，中国 NMPA 共批准了 165 个新分子实体抗肿瘤药，其中 69 个由国内企业开发，占比 41.8%。随着国内新药研发能力的崛起，「In China for China」早已不再是中国药企的目标，越来越多的药企将目光瞄向海外，希望实现「In China for Global」的更大愿景。不过，出海并非易事。截至 2025 年 5 月 25 日，仅有 10 个新分子实体药物在海外获批，数量屈指可数。近日，来自清华大学、丁香园 Insight 数据库等的研究人员联合在《柳叶刀-肿瘤学》发表了一篇文章，通过对近 10 年国产抗肿瘤新药的国际多中心临床试验（MRCT）开展状况、海外获批情况等进行深度分析，剖析了中国抗癌药物走向海外市场面临的关键挑战。（扫描文末二维码可获得论文全文）截图来源：《柳叶刀-肿瘤学》官网注：论文数据来源丁香园 Insight 数据库；临床试验统计范围为在 ClinicalTrials.gov、ChinaDrugTrials.org.cn 上登记的试验；MRCT 定义为试验开展地区≥2 个国家。 82 家中国药企开展 MRCT 百济神州最多根据文章，在 2015 年 1 月 1 日--2024 年 10 月 1 日期间，共计有 82 家中国药企开展了 259 项 MRCT，涉及 183 个肿瘤领域的新分子实体。时间方面，近一半（47.9%）试验是在 2021 年--2024 年期间开展。企业维度，百济神州开展的 MRCT 数量最多，有 70 项（占比 27.0%）。从试验分期来看，Ⅰ 期试验有 102 项，Ⅱ 期试验有 109 项，Ⅲ 期试验有 48 项。成分类别方面，有 78 个小分子药物、32 个抗体偶联药物（ADC）、30 个单抗、22 个双抗、8 个融合蛋白、4 个 PROTAC、2 个细胞疗法、2 个三特异性抗体。图：中国药企开展的 MRCT 中药物的成分类别分布图源：参考资料[1] 从靶点分布来看，最常见的靶点是 PD-1 和 PD-L1（13 项试验），其次是 HER2（8 项试验）和 EGFR（7 项试验）。多靶点药物有 36 个（19.6%），其中有 15 个同时靶向 PD-1 和 PD-L1。图：中国药企开展的 MRCT 中药物的靶点分布图源：参考资料[1] 试验开展地点分布方面，这些 MRCT 共涉及全球 51 个国家，包含中国地区的试验最多（207 项），其次是美国（187 项）和澳大利亚（127 项）。从承办方来看，大多数 MRCT 由一家中国公司开展，而与其它中国本土公司或者国外企业合作开展的试验比例较小。国产抗癌新药美国成功率仅为 5.2% 低于中国从试验设计来看，大多数 I 期临床试验包含剂量探索和扩展队列研究。II 期试验主要侧重于初步疗效评估和剂量优化。在 48 项 III 期 MRCT 中，有 46 项（95.8%）为随机试验，2 项（4.2%）为单臂试验。适应症上，最常见的癌症类型是肺癌、肝癌和淋巴瘤。试验状态方面，45 项 MRCT 已完成，127 项正在招募患者，13 项终止，1 项暂停，5 项撤回。根据临床试验的进度和最终的药物获批状态，文章对这些临床试验的成功率进行了分析。包含中国人群的 MRCT 在中国不同阶段的成功率分别为：从 Ⅰ 期进入 Ⅱ 期临床为 61.5%（8/13）；从 Ⅱ 期进入 Ⅲ 期为 19.0%（4/21）；从 Ⅲ 期进入 NDA/BLA 为 69.2%（9/13）；从 NDA/BLA 到获批为 100%（9/9）。总体来看，从 Ⅰ 期到获得中国 NMPA 批准的成功率为 8.1%，其中化学新药的获批成功率低于生物新药（5.6% vs 8.3%）。而在含美国人群的 MRCT 中：从 I 期临床试验到 FDA 批准的成功率为 5.2%，低于中国（8.1%）；不过，与中国相比，化学新药获 FDA 批准的成功率高于生物新药。图：中国公司开展的抗癌药 MRCT 成功率图源：参考资料[1] 中国抗癌药出海面临两大关键挑战截至 2025 年 5 月 25 日，共计有 11 个中国本土抗癌新药曾在美国或者欧盟申报上市，其中有 8 个获得批准（表 1），涉及 20 个适应症（不同药物的相同适应症重复计数）；3 款药物收到了 FDA 的完整回复函（CRL），未能获批。进一步分析 FDA 和 EMA 的审评报告发现，国产抗癌新药能否获批主要面临两大挑战：一是支持药物获批的关键试验中患者多样性问题；二是生产问题。表1：向 FDA 和 EMA 递交上市申请的中国抗癌新药图源：参考资料[1] 挑战一：关键临床中患者群体的多样性问题百济神州的泽布替尼（BTK 抑制剂）是首个获得 FDA 和 EMA 批准上市的中国本土小分子药物。在中国，该药在 2021 年基于一项在国内开展的单臂临床研究 (NCT03332173) 获得附条件批准上市，用于治疗华氏巨球蛋白血症。后来基于一项头对头伊布替尼的关键 Ⅲ 期 MRCT (NCT03053440) 研究，该药相继获得了 FDA、EMA 和 NMPA 的常规批准。不过，FDA 在审评报告指出，美国临床试验人群的多样性存在局限性，特别是某些种族和族裔亚群的代表性不足。为了弥补这一证据缺口，FDA 要求上市后收集更多安全性和有效性数据，特别是针对患有华氏巨球蛋白血症的少数族裔和种族群体的数据。百济神州的替雷利珠单抗（PD-1 抑制剂）是第二个获得 FDA 批准的中国自主研发的免疫治疗药物。替雷利珠单抗的 BLA 申请在中国和美国几乎同时提交，但其在中国（2022 年 4 月 8 日）的常规批准速度比美国早了近两年（2024 年 3 月 14 日）。 FDA 在审评报告中指出，在关键性试验中，亚裔患者占入组人群的 79%，不足以代表美国少数族裔群体。不过，根据美国监测、流行病学和最终结果数据库 (SEER) 的分析，食管鳞状细胞癌的治疗结果未显示出显著的种族差异。此外，其它靶向疗法的安全性数据也支持 FDA 的结论——该试验结果可以外推至美国人群。和黄医药的呋喹替尼（VEGF 抑制剂）是第二个获得 FDA 批准用于治疗癌症的中国本土小分子药物。2018 年，NMPA 批准呋喹替尼用于治疗晚期结直肠癌，批准依据的是 FRESCO 试验 (NCT02314819) 的数据，该试验仅招募了中国患者。 FDA 和 EMA 认为，基于单一国家试验的 FRESCO 试验无法充分代表其患者群体和医疗实践。因此，和黄医药又启动了一项额外的全球 FRESCO-2 试验 (NCT04322539)，以支持呋喹替尼的海外获批。这项试验招募了来自 15 个国家的 691 名患者，其中欧洲患者 495 名、美国患者 124 名、日本患者 56 名、澳大利亚患者 16 名。国际多中心试验结果显示，与安慰剂相比，呋喹替尼在总生存期方面具有显著且具有临床意义的改善，呋喹替尼的中位总生存期为 7.4 个月，而安慰剂为 4.8 个月，结果与 FRESCO 试验一致。不过，由于额外的国际临床开发要求，呋喹替尼获得 FDA 和 EMA 的批准时间比中国 NMPA 延迟了 5 年以上。信达生物的信迪利单抗（PD-1 抑制剂）于 2021 年获得 NMPA 批准，用于 NSCLC 一线治疗，该批准仅基于来自中国人群的临床试验数据 (NCT03607539)。信达和礼来曾试图用来自中国人群的临床试验数据寻求 FDA 的批准。然而，FDA 认为该试验数据未能充分代表美国人群的多样性，且不符合美国临床实践标准或监管要求。FDA 要求进行一项额外的 MRCT 试验，该试验设计为信迪利单抗联合化疗对比标准治疗用于治疗转移性 NSCLC 的一线治疗，采用非劣效性设计，主要终点为总生存期。同样，和黄医药的索凡替尼在中国基于其关键性试验（NCT02589821 和 NCT02588170）获批用于治疗神经内分泌肿瘤，因为这些试验只包含中国患者的数据，它也未能获得 FDA 批准。挑战二：生产问题生产问题是本土抗癌新药能否在海外获批所面临的第二大挑战。君实的特瑞普利单抗（PD-1 抑制剂）曾在 2022 年 4 月收到了 FDA 的 CRL。FDA 在上市前检查中发现了生产质量缺陷，包括潜在的病毒污染风险和工艺验证不足。经过数月的全面整改，特瑞普利单抗重新提交了 BLA，最终在 2023 年 10 月获得 FDA 批准上市用于治疗鼻咽癌。值得注意的是，支持特瑞普利单抗获批的关键试验虽然采用了 MRCT 设计，但其研究对象并未包括美国人群。不过，由于鼻咽癌在美国是一种罕见病，存在大量未满足的临床需求，所以特瑞普利单抗最终通过联邦法规第 21 条 314.106(b)，即允许依赖国外临床数据而获批上市。康方生物/正大天晴的派安普利单抗是第三个获得 FDA 批准的国产 PD-1 抑制剂。该药最初于 2024 年 4 月基于单臂试验数据 (NCT03866967) 获得 NMPA 批准，用于治疗鼻咽癌成人患者的三线治疗。为了支持全球上市申请，康方/正大天晴于 2019 年启动了一项包含美国患者群体的 MRCT 试验 (NCT04974398)。该 MRCT 试验结果显示，与对照组相比，派安普利单抗显著延长了患者的无进展生存期。不过，在 2023 年 11 月的上市前检查中，FDA 发现存在多项生产质量缺陷，主要包括微生物污染风险、偏差调查记录不完整，以及实际生产工艺与此前提交的工艺不一致的情况，所以拒绝批准派安普利单抗上市。经过数月的整改，FDA 于 2025 年 1 月对该生产设施进行了重新评估，并确认质量问题已得到解决。2025 年 4 月，派安普利单抗正式获得 FDA 批准上市，用于鼻咽癌治疗。恒瑞的卡瑞利珠单抗（PD-1 抑制剂）+阿帕替尼（VEGFR 抑制剂）联合疗法于 2023 年获得 NMPA 批准，用于晚期肝细胞癌一线治疗，该批准基于一项 MRCT （NCT03764293）。在海外，卡瑞利珠单抗曾在 2023 年 5 月、2024 年 9 月先后两次向 FDA 递交 BLA 申请，但均未获得批准。在首次拒批的 CRL 中，FDA 指出生产基地存在缺陷，加上旅行限制未能对一些生产基地进行检查。第二次 CRL 中，FDA 再次提到了生产问题未得到完全解决。当然，因生产问题被 FDA 拒绝批准并非只是中国新药面临的挑战，不少海外公司的产品也曾因生产问题未能批准。单是 2025 年以来就有多个案例，例如在 FDA 发给 Atara 公司的现货型 T 细胞疗法 Ebvallo、Ultragenyx 公司的 AAV9 基因疗法 UX111、再生元阿柏西普 (8 mg) 的新适应症申请等多个药物的 CRL 中，均提到了生产问题。小结近年来，虽然中国药企开展的抗癌药 MRCT 数量显著增长，但与跨国药企相比，数量仍然不足。NMPA 发布的《中国新药注册临床试验进展年度报告（2023 年）》显示，在中国 3988 项新药研究试验中，MRCT 仅占 290 项（6.7%）。MRCT 数量偏低的主要原因是资金和资源限制、全球研发能力受限以及应对不同监管政策的挑战。目前，中国公司通过 MRCT 在美国获批的药物成功率低于中国。商业战略层面，许多中国公司在药物研发中采取了跟随策略，这种方式虽然风险更低、研发周期更快，但在一定程度上直接导致了靶点内卷和适应症重叠。大多数正在进行 MRCT 的药物并非首创药物，主要集中在一些常见的靶点（例如 PD-1 和 PD-L1），这可能导致药物在海外市场上缺乏竞争力。目前海外获批的国产抗癌新药中，只有泽布替尼展现出了 BIC 实力，并成为首个由中国药企研发的销售额超过 10 亿美元的重磅炸弹药物（24 年销售额超 20 亿美元）。长远来看，中国药企应专注于研发具有差异化优势的抗癌药物，临床试验设计上充分考虑入组患者群体的多样性，并解决好生产问题，方能提升产品的海外获批速度，从而在国际市场占据一席之地。限于篇幅有限，文章仅分享了论文中的部分精华内容。扫描下方二维码添加 Insight 情报助手小音，回复出海挑战，即可获得论文全文。参考资料： [1]Challenges for Chinese innovative cancer drugs in going global: insights from multiregional clinical trials. https://www-thelancet-com.libproxy1.nus.edu.sg/journals/lanonc/article/PIIS1470-2045(25)00350-X/abstract [2] 丁香园 Insight 数据库封面来源：站酷海洛 Plus 免责声明：本文仅作信息分享，不代表 Insight 立场和观点，也不作治疗方案推荐和介绍。如有需求，请咨询和联系正规医疗机构。编辑：馨药 PR 稿对接：微信 insightxb 投稿：微信 insightxb；邮箱 insight@dxy.cn

抗体药物偶联物蛋白降解靶向嵌合体上市批准

2025-08-11

·investors.atarabio.com

Atara Biotherapeutics Announces Second Quarter Financial Results and Operational Progress

Tab-cel Prescription Drug User Fee Act (PDUFA) Target Action Date of January 10, 2026 Atara has transferred substantially all operational activities and associated costs related to tab-cel to Pierre Fabre Laboratories Atara has resumed its evaluation of strategic options Approval of BLA would unlock a $40 Million milestone payment from Pierre Fabre Laboratories, materially extending cash runway THOUSAND OAKS, Calif.--(BUSINESS WIRE)-- Atara Biotherapeutics, Inc. (Nasdaq: ATRA), a leader in T-cell immunotherapy, leveraging its novel allogeneic Epstein-Barr virus (EBV) T-cell platform to develop transformative therapies for patients with cancer and autoimmune diseases, today reported financial results for the second quarter 2025 and business updates. Tabelecleucel (tab-cel® or Ebvallo™) for Post-Transplant Lymphoproliferative Disease (PTLD) The U.S. Food and Drug Administration (FDA) has accepted the filing of Atara’s Biologics License Application (BLA) for tabelecleucel (tab-cel®) indicated as monotherapy for treatment of adult and pediatric patients two years of age and older with Epstein-Barr virus positive post-transplant lymphoproliferative disease (EBV+ PTLD) who have received at least one prior therapy. There are no FDA approved therapies in this treatment setting. The BLA has been granted Priority Review with a Class 2 Resubmission Prescription Drug User Fee Act (PDUFA) target action date of January 10, 2026. In July, the Company completed transferring substantially all operational activities and associated costs related to tab-cel to Pierre Fabre Laboratories. The sponsorship of the BLA continues to be maintained by the Company. Corporate Updates Strategic Options Evaluation: In April 2025, the Company temporarily paused its review of strategic alternatives pending resubmission of the tab-cel BLA. The Company has resumed its evaluation of strategic options following the resubmission of the tab-cel BLA. These options may include, but are not limited to, an acquisition, merger, reverse merger, other business combinations, licensing, sale of assets, or other strategic transactions. It is possible that Atara may not pursue a strategic alternative or transaction or that any strategic alternative or transaction, if pursued, will not be completed on attractive terms, or that a strategic alternative or transaction may not ultimately be consummated. Financial Update: Second Quarter 2025 Financial Results: Cash, cash equivalents and short-term investments as of June 30, 2025, totaled $22.3 million, as compared to $13.8 million as of March 31, 2025 Net cash used in operating activities was $7.4 million for the second quarter 2025, as compared to $10.6 million in the same period in 2024 Total revenues were $17.6 million for the second quarter 2025, as compared to $28.6 million for the same period in 2024. Total revenues decreased by $11.0 million year-over-year, primarily due to the accelerated recognition of deferred revenue in the first quarter 2025 following the transition of manufacturing responsibilities to Pierre Fabre Laboratories. As a result, less deferred revenue remained available for recognition in the comparative period. Total costs and operating expenses include non-cash stock-based compensation, depreciation and amortization expenses of $3.0 million for the second quarter 2025, as compared to $7.7 million for the same period in 2024 Research and development expenses were $7.3 million for the second quarter 2025, as compared to $33.3 million for the same period in 2024 Research and development expenses include $0.7 million of non-cash stock-based compensation expenses for the second quarter 2025, as compared to $3.3 million for the same period in 2024 General and administrative expenses were $6.5 million for the second quarter 2025, as compared to $8.9 million for the same period in 2024 General and administrative expenses include $2.1 million of non-cash stock-based compensation expenses for the second quarter 2025, as compared to $3.0 million for the same period in 2024 Atara reported net income of $2.4 million, or $0.20 basic earnings per share and $0.19 diluted earnings per share for the second quarter 2025. The net income position is due to the acceleration of revenue recognized following the transition of tab-cel development and safety responsibilities to Pierre Fabre Laboratories in July 2025 2025 Outlook and Cash Runway: Under its commercialization agreement with Pierre Fabre Medicament, Atara is eligible to receive a $40 million milestone payment upon FDA approval of the tab-cel BLA. In addition, Atara will be eligible to receive double-digit tiered royalties as a percentage of net sales and milestones related to commercial sales of EBVALLO. We anticipate the full-year 2025 operating expenses will decrease by at least 60% compared to 2024, driven by the transition of substantially all tab-cel activities and associated costs to Pierre Fabre Laboratories as well as the implementation of operational efficiencies in the first half of the year. Atara projects that cash, cash equivalents and short-term investments as of June 30, 2025, combined with the proceeds of the milestone payment upon tab-cel BLA approval under its commercialization agreement with Pierre Fabre Medicament, will provide significant cash runway and flexibility for the company to execute on its strategic priorities. About Atara Biotherapeutics, Inc. Atara is harnessing the natural power of the immune system to develop off-the-shelf cell therapies for difficult-to-treat cancers and autoimmune conditions that can be rapidly delivered to patients from inventory. With cutting-edge science and differentiated approach, Atara is the first company in the world to receive regulatory approval of an allogeneic T-cell immunotherapy. Our advanced and versatile T-cell platform does not require T-cell receptor or HLA gene editing and forms the basis of a diverse portfolio of investigational therapies that target EBV, the root cause of certain diseases. Atara is headquartered in Southern California. For more information, visit atarabio.com and follow @Atarabio on X and LinkedIn. Forward-Looking Statements This press release contains or may imply “forward-looking statements” within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934. For example, forward-looking statements include statements regarding: (1) the development, timing and progress of tab-cel, including the timing for FDA review of the resubmission of the BLA, the potential characteristics and benefits of tab-cel, and the results of, and prospects for, the global partnership with Pierre Fabre Medicament involving tab-cel, and the potential financial benefits to Atara as a result of the global partnership with Pierre Fabre Medicament, including the receipt, timing and amount of any payments to be received by Atara thereunder; (2) Atara’s cash runway, receipt of potential milestone payments, and estimated reduction in operating expenses; and (3) Atara’s evaluation of strategic alternatives and ability to consummate one or more strategic transactions. Because such statements deal with future events and are based on Atara’s current expectations, they are subject to various risks and uncertainties and actual results, performance or achievements of Atara could differ materially from those described in or implied by the statements in this press release. These forward-looking statements are subject to risks and uncertainties, including, without limitation, risks and uncertainties associated with the costly and time-consuming pharmaceutical product development process and the uncertainty of clinical success; risks related to FDA’s review of the resubmitted BLA for tab-cel; our ability to access capital, and the sufficiency of Atara’s cash resources and access to additional capital on favorable terms or at all; the timing of the strategic review process; whether Atara will pursue any strategic alternatives; in the event Atara pursues a strategic alternative, that the strategic alternative may not be attractive or ultimately consummated; whether any strategic alternative will result in additional value for Atara and its stockholders; whether the process will have an adverse impact on Atara and other risks and uncertainties affecting Atara, including those discussed in Atara’s filings with the Securities and Exchange Commission, including in the “Risk Factors” and “Management’s Discussion and Analysis of Financial Condition and Results of Operations” sections of the Company’s most recently filed periodic reports on Form 10-K and Form 10-Q and subsequent filings and in the documents incorporated by reference therein. Except as otherwise required by law, Atara disclaims any intention or obligation to update or revise any forward-looking statements, which speak only as of the date hereof, whether as a result of new information, future events or circumstances or otherwise. Financials ATARA BIOTHERAPEUTICS, INC. Condensed Consolidated Balance Sheets (Unaudited) (In thousands) June 30, December 31, 2025 2024 Assets Current assets: Cash and cash equivalents $ 16,903 $ 25,030 Short-term investments 5,420 17,466 Restricted cash — 146 Accounts receivable — 1,482 Inventories — 10,655 Other current assets 2,449 10,115 Total current assets 24,772 64,894 Property and equipment, net 176 1,294 Operating lease assets 11,027 39,807 Other assets 927 3,103 Total assets $ 36,902 $ 109,098 Liabilities and stockholders’ equity (deficit) Current liabilities: Accounts payable $ 1,762 $ 4,367 Accrued compensation 2,552 6,589 Accrued research and development expenses 971 7,984 Deferred revenue 1,607 95,092 Other current liabilities 7,671 20,542 Total current liabilities 14,563 134,574 Deferred revenue - long-term — — Operating lease liabilities - long-term 15,426 29,914 Liability related to the sale of future revenues - long-term 40,222 38,624 Other long-term liabilities 1,732 3,269 Total liabilities $ 71,943 $ 206,381 Stockholders’ (deficit) equity: Common stock 1 1 Additional paid-in capital 1,979,114 1,957,261 Accumulated other comprehensive loss — 8 Accumulated deficit (2,014,156 ) (2,054,553 ) Total stockholders’ (deficit) equity (35,041 ) (97,283 ) Total liabilities and stockholders’ (deficit) equity $ 36,902 $ 109,098 ATARA BIOTHERAPEUTICS, INC. Condensed Consolidated Statements of Operations and Comprehensive Loss (Unaudited) (In thousands, except per share amounts) Three Months Ended June 30, Six Months Ended June 30, 2025 2024 2025 2024 Commercialization revenue $ 17,575 $ 28,640 $ 115,724 $ 55,997 Costs and operating expenses: Cost of commercialization revenue 554 4,627 20,993 6,612 Research and development expenses 7,310 33,332 34,743 78,838 General and administrative expenses 6,514 8,912 17,989 20,025 Total costs and operating expenses 14,378 46,871 73,725 105,475 Income (loss) from operations 3,197 (18,231 ) 41,999 (49,478 ) Interest and other income (expense), net (807 ) (818 ) (1,599 ) (1,299 ) Loss before provision for income taxes 2,390 (19,049 ) 40,400 (50,777 ) Provision for income taxes 3 — 3 24 Net income (loss) $ 2,387 $ (19,049 ) $ 40,397 $ (50,801 ) Other comprehensive gain (loss): Unrealized gain (loss) on available-for-sale securities — 41 (8 ) 190 Comprehensive income (loss) $ 2,387 $ (19,008 ) $ 40,389 $ (50,611 ) Basic net income (loss) per common share $ 0.20 $ (3.10 ) $ 3.52 $ (8.64 ) Diluted net income (loss) per common share $ 0.19 $ (3.10 ) $ 3.49 $ (8.64 ) Basic weighted-average shares outstanding 12,197 6,143 11,484 5,883 Diluted weighted-average shares outstanding 12,310 6,143 11,576 5,883 View source version on businesswire.com: https://www.businesswire.com/news/home/20250811258417/en/ Investor and Media Relations Amber Daugherty Sr. Director, Strategy and Operations adaugherty@atarabio.com Source: Atara Biotherapeutics, Inc. Released August 11, 2025

免疫疗法财报引进/卖出

2025-07-28

·细胞基因治疗前沿

首款现货型T细胞疗法上市申请获FDA受理

声明：因水平有限，错误不可避免，或有些信息非最及时，欢迎留言指出。本文仅作医疗健康相关药物介绍，非治疗方案推荐（若涉及）;本文不构成任何投资建议。2025年7月25日，Atara Biotherapeutics的Pierre Fabre Pharmaceuticals共同宣布，美国FDA已受理为tabelecleucel（tab-cel）提交的生物制品许可申请（BLA），作为单药，用于治疗爱泼斯坦-巴尔病毒（EBV）阳性的移植后淋巴增殖性疾病（PTLD）的成人及2岁及以上儿童患者，这些患者至少接受过一种既往治疗。FDA并授予该申请优先审评资格，PDUFA日期为2026年1月10日。值得一提的是，目前该适应症尚无FDA批准的治疗方案。Tab-cel是一种同种异体、“现货型”EBV特异性T细胞免疫疗法，旨在靶向并清除EBV感染细胞。此次申报资料包括覆盖430余例患者的关键性与支持性临床数据，其中关键3期ALLELE研究显示接受治疗患者的客观缓解率达48.8%（p<0.0001），安全性良好且与既往研究结果一致。此前，tab-cel已获得FDA授予的突破性疗法认定及孤儿药资格。Tabelecleucel 于2022年12月获得欧盟委员会 (EC) 授予的 EBVALLO™ 品牌上市许可。此外，该药物还于2023年5月获得英国药品和保健产品管理局 (MDPA) 和 2024 年 5 月获得瑞士医药管理局(Swissmedic) 的上市许可。自2025年3月31日起，PFP承担了为欧洲市场生产商业产品以及全球临床试验供应tabeleculeucel的全球责任。2025年7月15日，Atara Biotherapeutics将tabeleculeucel新药临床试验申请转让给Pierre Fabre Laboratories的子公司Pierre Fabre Medicament。根据Atara与Pierre Fabre Laboratories于2023年11月宣布的全球独家许可协议的条款，Atara将负责监管程序，直至BLA转让给Pierre Fabre Pharmaceuticals。参考资料：公司公告

优先审批免疫疗法孤儿药临床3期突破性疗法

100 项与 Tabelecleucel 相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
D11146	-	-	-

研发状态

批准上市

10 条最早获批的记录,

后查看更多信息

适应症	国家/地区	公司	日期
EBV 相关的移植后淋巴增殖性疾病	欧盟	Pierre Fabre Medicament Information	2022-12-16
EBV 相关的移植后淋巴增殖性疾病	冰岛	Pierre Fabre Medicament Information	2022-12-16
EBV 相关的移植后淋巴增殖性疾病	列支敦士登	Pierre Fabre Medicament Information	2022-12-16
EBV 相关的移植后淋巴增殖性疾病	挪威	Pierre Fabre Medicament Information	2022-12-16

未上市

10 条进展最快的记录,

后查看更多信息

适应症	最高研发状态	国家/地区	公司	日期
爱泼斯坦-巴尔病毒感染	申请上市	欧盟	Atara Biotherapeutics, Inc.	2021-11-30
造血干细胞移植	临床2期	美国	Pierre Fabre Medicament Information	2021-07-14
造血干细胞移植	临床2期	奥地利	Pierre Fabre Medicament Information	2021-07-14
造血干细胞移植	临床2期	比利时	Pierre Fabre Medicament Information	2021-07-14
造血干细胞移植	临床2期	法国	Pierre Fabre Medicament Information	2021-07-14
造血干细胞移植	临床2期	意大利	Pierre Fabre Medicament Information	2021-07-14
造血干细胞移植	临床2期	西班牙	Pierre Fabre Medicament Information	2021-07-14
造血干细胞移植	临床2期	英国	Pierre Fabre Medicament Information	2021-07-14
平滑肌肉瘤	临床2期	美国	Pierre Fabre Medicament Information	2021-07-14
平滑肌肉瘤	临床2期	奥地利	Pierre Fabre Medicament Information	2021-07-14

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临床结果

适应症

分期

评价

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研究	分期	人群特征	评价人数	分组	结果	评价	发布日期


NCT03394365 (ALLELE, Company_Website) 人工标引	临床2期	EBV 相关的移植后淋巴增殖性疾病	-	Tabelecleucel	願艱網艱遞淵衊積糧鑰(鏇築膚鏇顧積鑰願餘醖) = 製獵夢簾醖築憲廠廠觸壓壓鹹壓築蓋構膚製構 (鬱鑰製憲製積築積餘襯 )	积极	2025-07-14
ASH2024	N/A	EBV 相关的移植后淋巴增殖性疾病	-	Tabelecleucel	繭齋範餘積艱鹽襯憲鹽(鹹鹹壓獵網鏇鹽糧廠淵) = reported in 65.4% and 61.2% of HCT and SOT pts, respectively 蓋觸範積構獵鏇繭選簾 (憲齋蓋衊鹹廠鏇壓鑰鬱 ) 更多	-	2024-12-07
NCT03769467 (CTgov)	临床1/2期	爱泼斯坦-巴尔病毒感染 \| 鼻咽癌 \| 爱泼斯坦-巴尔病毒相关鼻咽癌	12	Pembrolizumab+Tabelecleucel (Cohort 1B: Checkpoint Inhibitor Naïve)	鑰範選遞衊衊鏇窪膚壓 = 鏇蓋網醖淵獵蓋築艱網顧鏇鏇淵願艱製淵構衊 (鹹遞蓋顧選製襯遞鬱鬱, 鬱鏇衊築醖範鑰蓋鏇艱 ~ 憲襯衊蓋淵淵夢製願鏇) 更多	-	2024-11-14
NCT03769467 (CTgov)	临床1/2期	爱泼斯坦-巴尔病毒感染 \| 鼻咽癌 \| 爱泼斯坦-巴尔病毒相关鼻咽癌	12	Pembrolizumab+Tabelecleucel (Cohort 1B: Checkpoint Inhibitor PD-1/PD-L1 Failure)	鑰範選遞衊衊鏇窪膚壓 = 鹽繭築壓築願鹽憲築鏇顧鏇鏇淵願艱製淵構衊 (鹹遞蓋顧選製襯遞鬱鬱, 積顧壓鹽網遞簾醖艱襯 ~ 鹽糧夢襯窪獵積製鬱構) 更多	-	2024-11-14
SOHO2024	N/A	EBV 相关的移植后淋巴增殖性疾病	-	Tabelecleucel	壓廠餘膚獵築憲齋獵簾(艱糧齋繭構廠淵艱衊簾) = 鹹壓鹹製鬱淵簾選鑰衊憲鑰積蓋廠醖範遞鏇膚 (衊憲範夢鑰觸襯憲膚願, 52.4 ~ 93.6) 更多	-	2024-09-04
NCT02822495 (NCT02822495, Pubmed \| Blood Adv)	N/A	移植后淋巴增生性疾病	26	Tabelecleucel	顧觸觸網獵餘餘範廠製(範繭網襯壓餘網選餘網) = 淵窪築範窪獵憲鹽襯壓製衊繭醖鬱遞願鹹選衊 (鏇網窪範簾鑰製簾醖鏇, 46.5 ~ 84.7) 更多	积极	2024-06-25
NCT01498484 \| NCT00002663 \| NCT04554914 \| NCT02822495 (Tandem Meetings ASTCT and CIBMTR2024) 人工标引	临床1/2期	EBV 相关的移植后淋巴增殖性疾病	-	Tabelecleucel	鏇獵襯構鑰憲窪衊鏇築(夢築襯製範蓋鬱淵繭膚) = There were no treatment-related fatal or life-threatening treatment-emergent adverse events (TEAEs) reported or serious treatment-related TEAEs of neurotoxicity, organ rejection, graft-versus-host disease, or tumor flare reaction of any grade 網鑰範願積顧顧鏇淵獵 (蓋醖窪餘鑰鬱蓋蓋艱簾 )	积极	2024-02-01
NCT01498484 \| NCT00002663 \| NCT04554914 \| NCT02822495 (EBVision, Biospace) 人工标引	临床1/2期	EBV 相关的移植后淋巴增殖性疾病	18	tab-cel 2x106 cells/kg	憲襯鬱蓋鑰艱構齋範顧(繭襯鏇襯遞鑰範醖糧鹹) = 憲鏇窪鑰鹹憲襯憲鏇衊蓋廠膚蓋構構鏇鑰糧鹹 (鏇製簾積糧鏇衊觸壓範, 52.4 ~ 93.6) 更多	积极	2023-11-29
	临床1/2期	EBV 相关的移植后淋巴增殖性疾病	18	tab-cel 2x106 cells/kg (responders)	糧衊窪觸製窪壓遞觸襯(齋齋鏇觸願遞觸衊窪觸) = 鹽蓋艱鏇蓋鏇醖製淵憲鏇醖願醖鹹鹽積窪糧襯 (構襯願鏇獵遞繭鹽鹹齋 )	积极	2023-11-29
EMA (ICML2023)	N/A	EBV 相关的移植后淋巴增殖性疾病 EBV +	74	Tabelecleucel	範製製繭窪積築遞觸積(蓋鑰繭窪獵鬱鹽積積製) = 淵壓鏇獵遞積製範構廠顧蓋蓋製鹽醖憲壓願鹽 (鬱糧襯簾齋膚網選壓網, 47.3 ~ 88.3) 更多	积极	2023-06-09
NCT03394365 (ALLELE, EHA2023)	临床3期	移植后淋巴增生性疾病 \| EBV 相关的移植后淋巴增殖性疾病 \| 造血干细胞移植 ... 更多	43	Tab-cel	憲簾憲選窪膚網膚糧遞(網糧遞廠糧憲窪膚築衊) = 廠繭糧鏇鑰鹽積憲築簾艱壓鏇鹹鑰構壓窪鬱夢 (衊醖願獵壓鬱餘鏇繭築, 35.5 ~ 66.7) 更多	-	2023-06-08
ASCO 12023 \| ASCO 22023 人工标引	N/A	EBV 相关的移植后淋巴增殖性疾病 EBV Positive	24	tabelecleucel	壓範鏇簾糧鏇選獵網遞(選鹹艱鏇顧選範壓夢繭) = 憲襯遞蓋窪衊襯夢餘選齋蓋繭淵衊繭築餘餘網 (淵艱鏇觸鬱衊廠顧簾憲, 44.7 ~ 84.4) 更多	积极	2023-05-31
ASCO 12023 \| ASCO 22023 人工标引	N/A	EBV 相关的移植后淋巴增殖性疾病 EBV Positive	24	tabelecleucel (EBV+ PTLD post-HCT)	壓範鏇簾糧鏇選獵網遞(選鹹艱鏇顧選範壓夢繭) = 積餘顧壓夢壓顧製廠積齋蓋繭淵衊繭築餘餘網 (淵艱鏇觸鬱衊廠顧簾憲, 47.3 ~ 99.7) 更多	积极	2023-05-31