- 选择性Treg调节剂gotistobart (BNT316/ONC-392) 的全球3期临床试验PRESERVE-003分为2个阶段，在第1阶段试验中，针对既往接受免疫治疗联合化疗后进展的鳞状非小细胞肺癌（sqNSCLC）患者，相比标准化疗，gotistobart将死亡风险降低了一半以上，且安全性风险可控
- 中位随访近15个月时，gotistobart组的中位总生存期尚未达到，而对照的化疗组为10个月
- 针对高度未被满足的临床需求，gotistobart作为无需化疗的单药治疗方案，有望为导致细胞毒性的传统疗法提供临床替代
- Gotistobart已经获得美国FDA授予的快速通道资格，用于治疗既往接受抗PD-(L)1治疗后疾病进展的转移性NSCLC患者
德国美因茨和美国罗克维尔，2025年12月6日 (GLOBE NEWSWIRE) - BioNTech SE（纳斯达克代码：BNTX，
试验结果显示，在既往接受抗PD-(L)1及铂类化疗后疾病进展的sqNSCLC人群中，gotistobart较标准化疗带来具有临床意义的总生存获益，且安全性可控。相关数据以口头报告形式在美国芝加哥举行的国际肺癌研究协会（IASLC）ASCO 2025北美肺癌大会上发布。
“晚期鳞状非小细胞肺癌患者的中位生存期不足一年，仍属侵袭性强、治疗棘手的肺癌类型1, 2。近年来，免疫治疗及联合方案虽带来生存改善，但对抗PD-(L)1抑制剂耐药的患者预后极差，只剩下化疗或姑息治疗可供选择。”首尔延世癌症中心肿瘤内科教授、首席研究者Byoung Chul Cho博士表示，“随访近15个月，gotistobart组的中位总生存期仍未达到，这一结果令人振奋。我们将继续推进关键临床试验阶段，进一步验证该候选药物的潜力。”
全球3期PRESERVE-003试验的非关键阶段共入组87例转移性sqNSCLC患者，均接受二线及以上的治疗方案，其中45例接受gotistobart单药治疗，42例接受多西他赛化疗。截至2025年8月8日，87例sqNSCLC患者被随机分组：gotistobart（两次10 mg/kg负荷剂量后维持6 mg/kg，N=45），多西他赛（75 mg/m²，N=42）。
结果显示，12个月总生存率gotistobart组为63.1%，多西他赛组30.3%；中位随访14.5个月时，gotistobart组中位总生存期尚未达到，多西他赛组为10个月。Gotistobart组的死亡风险较化疗组降低54%（HR=0.46，95%CI：0.25–0.84；Nominal p value 0.0102）；其安全性与既往结果一致且可控，≥3级治疗相关不良事件发生率42.2%（19/45），低于多西他赛组的48.8%（20/41）。目前，该3期试验的关键阶段已在全球160余家临床试验中心开展。
BioNTech联合创始人兼首席医学官Özlem Türeci教授表示：“Gotistobart专门设计用于选择性清除肿瘤微环境中的浸润Treg。今天公布的积极数据验证了该产品的研发策略，将我们对免疫系统的深度认知转化为sqNSCLC患者的显著生存获益。凭借其独特的作用机理，我们正探索gotistobart单药治疗及与其他疗法的联用治疗方案，目标是为患者带来突破性的疗法，提供显著、持久的临床获益。”
OncoC4联合创始人兼首席医学官郑盼博士指出：“Gotistobart使我们向‘为晚期sqNSCLC患者提供无化疗方案’的目标再进一步，这类患者治疗选择有限，且缺乏可指导治疗、可用于临床实践的生物标志物。今日公布的积极临床数据进一步证实，gotistobart有望填补当前未被满足的临床需求。我们期待继续携手共进，深入挖掘这一全新作用机制的潜力，加速临床开发，让更多患者尽早受益。”
关于PRESERVE-003
PRESERVE-003（NCT05671510）是一项两阶段、开放标签的Ⅲ期临床试验，旨在评估gotistobart单药与标准化疗多西他赛相比，在既往接受PD-(L)1抑制剂联合铂类化疗后进展的sqNSCLC患者中的疗效与安全性。该试验的非关键阶段最初纳入了所有类型的NSCLC患者，目前正在进行的关键阶段则仅招募sqNSCLC患者。关键阶段的临床试验，计划在多个国家和地区的临床中心入组约500名患者，包括澳大利亚、比利时、加拿大、中国、德国、意大利、荷兰、西班牙、韩国、土耳其、英国和美国等。主要临床终点为总生存期（OS），次要终点包括客观缓解率（ORR）、无进展生存期（PFS）和安全性。
关于gotistobart (BNT316/ONC-392)
Gotistobart（BNT316/ONC-392）是由BioNTech与OncoC4联合开发的一种候选药物，可以选择性清除肿瘤微环境中的调节性T细胞（Treg）。作为一种pH敏感型的单克隆抗体，gotistobart的设计旨在实现CTLA-4的循环利用。它与细胞表面CTLA-4受体结合后被内化，随后因pH变化而解离，使CTLA-4得以返回细胞表面，从而在外周组织和器官保留免疫检查点功能，同时在肿瘤微环境中增强抗肿瘤的免疫作用3。Gotistobart目前正处于后期临床开发阶段，正同步推进单药及联合方案，探索多种癌症适应症。Gotistobart于2022年获得美国FDA授予的快速通道资格，用于治疗既往抗PD-(L)1治疗后进展的转移性NSCLC患者，并于2025年获得中国国家药监局（NMPA）授予的突破性疗法认定。
目前正在进行多项临床试验，包括：一项关键3期试验（PRESERVE-003；NCT05671510），针对免疫耐受的sqNSCLC患者；一项2期试验（PRESERVE-004；NCT05446298），针对铂耐药卵巢癌患者；一项2期试验（PRESERVE-006；NCT05682443），针对转移性去势抵抗性前列腺癌患者；以及一项1/2期开放标签、剂量递增试验（PRESERVE-001；NCT04140526），针对晚期实体瘤患者。此外，BioNTech正在开展的一项1期试验（LuCa-MERIT-1；NCT05142189）的信号探索队列中，评估gotistobart与其mRNA癌症免疫治疗候选药物BNT116的联用。
关于非小细胞肺癌（NSCLC）
非小细胞肺癌（NSCLC）泛指除小细胞肺癌外的所有上皮源性肺癌，包括鳞状细胞癌、大细胞癌和腺癌，占全部肺癌病例的约85%4。其危险因素涵盖吸烟、石棉暴露及肺纤维化等5。其中鳞状细胞癌（SCC）约占肺癌总数的25%6。美国2000-2017年的调查数据显示，晚期鳞状NSCLC的5年相对生存率仅15%，中位总生存期约11个月，治疗选择极为有限7。当前标准治疗为手术、放疗联合化疗8；对于一线免疫联合化疗失败后进入二线的晚期/转移性鳞状NSCLC患者，仅剩化疗或姑息治疗，其可选方案明显少于非鳞状NSCLC5。
关于BioNTech
生物制药新技术公司（BioNTech）是一家全球领先的下一代免疫疗法企业，专注于癌症及其他重症创新疗法的研究与开发。公司依托计算发现平台及多元药物类型，加速新型生物药的研发。其肿瘤产品组合涵盖mRNA癌症免疫疗法、新一代免疫调节药物，以及抗体-药物偶联物（ADC）、创新CAR-T细胞疗法等靶向药物，贯穿癌症治疗全周期。凭借在mRNA领域的深厚经验和自主生产能力，BioNTech与合作伙伴同步推进多款传染病mRNA疫苗及多样化肿瘤管线的研究与开发。BioNTech已建立广泛合作网络，合作方包括BMS、映恩生物、复星医药、Genentech（Roche集团成员）、Genmab、MediLink、OncoC4、Pfizer和Regeneron等公司。
更多信息请访问 www.BioNTech.com
关于OncoC4
OncoC4总部位于美国马里兰州罗克维尔，是一家私有、处于后期临床阶段的生物制药公司，专注于发现和开发用于治疗癌症及免疫疾病的新型生物制剂。公司管线涵盖多个具有同类首创或同类最佳潜力的项目，靶点新颖且已充分验证，横跨肿瘤学与免疫学领域。其中，AI-081为OncoC4完全拥有的PD-1×VEGF双特异性抗体候选药物；ONC-841是一款同类首创的抗SIGLEC10抗体，正在开展针对肿瘤的2期临床研究，并探索用于神经退行性疾病的可能性。OncoC4与BioNTech 达成战略合作，共同开发肿瘤微环境选择性Treg清除候选药物gotistobart（BNT316/ONC-392，靶向CTLA-4），用于多种实体瘤适应症，目前双方正推进针对sqNSCLC的关键3期临床试验。
更多信息请访问 www.oncoc4.com
BioNTech前瞻性声明
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including, but not limited to, statements concerning: the collaboration with OncoC4; BioNTech and OncoC4’s ability to successfully co-develop and co-commercialize gotistobart (also known as BNT316 or ONC-392), if approved; the rate and degree of market acceptance of gotistobart, if approved; the initiation, timing, progress, and results of BioNTech’s research and development programs, including data from the non-pivotal dose-confirmation stage of the global randomized Phase 3 trial PRESERVE-003 and statements regarding the expected timing of initiation, enrollment, and completion of trials and related preparatory work and the availability of results, and the timing and outcome of applications for regulatory approvals and marketing authorizations, including expectations regarding the potential indications in which gotistobart may be approved, if at all; the targeted timing and number of additional potentially registrational trials, and the registrational potential of any trial BioNTech may initiate; and discussions with regulatory agencies. In some cases, forward-looking statements can be identified by terminology such as “will,” “may,” “should,” “expects,” “intends,” “plans,” “aims,” “anticipates,” “believes,” “estimates,” “predicts,” “potential,” “continue,” or the negative of these terms or other comparable terminology, although not all forward-looking statements contain these words.
The forward-looking statements in this press release are based on BioNTech’s current expectations and beliefs of future events and are neither promises nor guarantees. You should not place undue reliance on these forward-looking statements because they involve known and unknown risks, uncertainties, and other factors, many of which are beyond BioNTech’s control and which could cause actual results to differ materially and adversely from those expressed or implied by these forward-looking statements. These risks and uncertainties include, but are not limited to: the uncertainties inherent in research and development, including the ability to meet anticipated clinical endpoints, commencement and/or completion dates for clinical trials, regulatory submission dates, regulatory approval dates and/or launch dates, as well as risks associated with clinical data, and including the possibility of unfavorable new preclinical, clinical or safety data and further analyses of existing preclinical, clinical or safety data; the nature of clinical data, which is subject to ongoing peer review, regulatory review and market interpretation; the impact of tariffs and escalations in trade policy; competition related to BioNTech’s product candidates; the timing of and BioNTech’s ability to obtain and maintain regulatory approval for its product candidates; BioNTech’s ability to identify research opportunities and discover and develop investigational medicines; the ability and willingness of BioNTech’s third-party collaborators to continue research and development activities relating to BioNTech’s development candidates and investigational medicines; unforeseen safety issues and potential claims that are alleged to arise from the use of products and product candidates developed or manufactured by BioNTech; BioNTech’s and its collaborators’ ability to commercialize and market its product candidates, if approved; BioNTech’s ability to manage its development and related expenses; regulatory and political developments in the United States and other countries; BioNTech’s ability to effectively scale its production capabilities and manufacture its products and product candidates; and other factors not known to BioNTech at this time.
You should review the risks and uncertainties described under the heading “Risk Factors” in BioNTech’s Report on Form 6-K for the period ended September 30, 2025 and in subsequent filings made by BioNTech with the SEC, which are available on the SEC’s website at www.sec.gov. These forward-looking statements speak only as of the date hereof. Except as required by law, BioNTech disclaims any intention or responsibility for updating or revising any forward-looking statements contained in this press release in the event of new information, future developments or otherwise.
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OncoC4
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Ryan Cui
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Helen Schiltz
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BioNTech
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注：本文为英文新闻稿的中文翻译。若中文版本与英文版本之间存在任何不一致之处，以英文版本为准。
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