- 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
德國美因茨和美國羅克維爾，2025年12月6日 (GLOBE NEWSWIRE) - BioNTech SE（納斯達克代碼：BNTX，
試驗結果顯示，在既往接受抗PD-(L)1及鉑類化療後疾病進展的sqNSCLC人群中，gotistobart較標準化療帶來具有臨床意義的總生存獲益，且安全性可控。相關數據以口頭報告形式在美國芝加哥舉行的國際肺癌研究協會（IASLC）ASCO 2025北美肺癌大會上發布。
“晚期鱗狀非小細胞肺癌患者的中位生存期不足壹年，仍屬侵襲性強、治療棘手的肺癌類型1, 2。近年來，免疫治療及聯合方案雖帶來生存改善，但對抗PD-(L)1抑制劑耐藥的患者預後極差，只剩下化療或姑息治療可供選擇。”首爾延世癌癥中心腫瘤內科教授、首席研究者Byoung Chul Cho博士表示，“隨訪近15個月，gotistobart組的中位總生存期仍未達到，這壹結果令人振奮。我們將繼續推進關鍵臨床試驗階段，進壹步驗證該候選藥物的潛力。”
全球3期PRESERVE-003試驗的非關鍵階段共入組87例轉移性sqNSCLC患者，均接受二線及以上的治療方案，其中45例接受gotistobart單藥治療，42例接受多西他賽化療。截至2025年8月8日，87例sqNSCLC患者被隨機分組：gotistobart（兩次10 mg/kg負荷劑量後維持6 mg/kg，N=45），多西他賽（75 mg/m²，N=42）。
結果顯示，12個月總生存率gotistobart組為63.1%，多西他賽組30.3%；中位隨訪14.5個月時，gotistobart組中位總生存期尚未達到，多西他賽組為10個月。Gotistobart組的死亡風險較化療組降低54%（HR=0.46，95%CI：0.25–0.84；Nominal p value 0.0102）；其安全性與既往結果壹致且可控，≥3級治療相關不良事件發生率42.2%（19/45），低於多西他賽組的48.8%（20/41）。目前，該3期試驗的關鍵階段已在全球160余家臨床試驗中心開展。
BioNTech聯合創始人兼首席醫學官Özlem Türeci教授表示：“Gotistobart專門設計用於選擇性清除腫瘤微環境中的浸潤Treg。今天公布的積極數據驗證了該產品的研發策略，將我們對免疫系統的深度認知轉化為sqNSCLC患者的顯著生存獲益。憑借其獨特的作用機理，我們正探索gotistobart單藥治療及與其他療法的聯用治療方案，目標是為患者帶來突破性的療法，提供顯著、持久的臨床獲益。”
OncoC4聯合創始人兼首席醫學官鄭盼博士指出：“Gotistobart使我們向‘為晚期sqNSCLC患者提供無化療方案’的目標再進壹步，這類患者治療選擇有限，且缺乏可指導治療、可用於臨床實踐的生物標誌物。今日公布的積極臨床數據進壹步證實，gotistobart有望填補當前未被滿足的臨床需求。我們期待繼續攜手共進，深入挖掘這壹全新作用機制的潛力，加速臨床開發，讓更多患者盡早受益。”
關於PRESERVE-003
PRESERVE-003（NCT05671510）是壹項兩階段、開放標簽的Ⅲ期臨床試驗，旨在評估gotistobart單藥與標準化療多西他賽相比，在既往接受PD-(L)1抑制劑聯合鉑類化療後進展的sqNSCLC患者中的療效與安全性。該試驗的非關鍵階段最初納入了所有類型的NSCLC患者，目前正在進行的關鍵階段則僅招募sqNSCLC患者。關鍵階段的臨床試驗，計劃在多個國家和地區的臨床中心入組約500名患者，包括澳大利亞、比利時、加拿大、中國、德國、意大利、荷蘭、西班牙、韓國、土耳其、英國和美國等。主要臨床終點為總生存期（OS），次要終點包括客觀緩解率（ORR）、無進展生存期（PFS）和安全性。
關於gotistobart (BNT316/ONC-392)
Gotistobart（BNT316/ONC-392）是由BioNTech與OncoC4聯合開發的壹種候選藥物，可以選擇性清除腫瘤微環境中的調節性T細胞（Treg）。作為壹種pH敏感型的單克隆抗體，gotistobart的設計旨在實現CTLA-4的循環利用。它與細胞表面CTLA-4受體結合後被內化，隨後因pH變化而解離，使CTLA-4得以返回細胞表面，從而在外周組織和器官保留免疫檢查點功能，同時在腫瘤微環境中增強抗腫瘤的免疫作用3。Gotistobart目前正處於後期臨床開發階段，正同步推進單藥及聯合方案，探索多種癌癥適應癥。Gotistobart於2022年獲得美國FDA授予的快速通道資格，用於治療既往抗PD-(L)1治療後進展的轉移性NSCLC患者，並於2025年獲得中國國家藥監局（NMPA）授予的突破性療法認定。
目前正在進行多項臨床試驗，包括：壹項關鍵3期試驗（PRESERVE-003；NCT05671510），針對免疫耐受的sqNSCLC患者；壹項2期試驗（PRESERVE-004；NCT05446298），針對鉑耐藥卵巢癌患者；壹項2期試驗（PRESERVE-006；NCT05682443），針對轉移性去勢抵抗性前列腺癌患者；以及壹項1/2期開放標簽、劑量遞增試驗（PRESERVE-001；NCT04140526），針對晚期實體瘤患者。此外，BioNTech正在開展的壹項1期試驗（LuCa-MERIT-1；NCT05142189）的信號探索隊列中，評估gotistobart與其mRNA癌癥免疫治療候選藥物BNT116的聯用。
關於非小細胞肺癌（NSCLC）
非小細胞肺癌（NSCLC）泛指除小細胞肺癌外的所有上皮源性肺癌，包括鱗狀細胞癌、大細胞癌和腺癌，占全部肺癌病例的約85%4。其危險因素涵蓋吸煙、石棉暴露及肺纖維化等5。其中鱗狀細胞癌（SCC）約占肺癌總數的25%6。美國2000-2017年的調查數據顯示，晚期鱗狀NSCLC的5年相對生存率僅15%，中位總生存期約11個月，治療選擇極為有限7。當前標準治療為手術、放療聯合化療8；對於壹線免疫聯合化療失敗後進入二線的晚期/轉移性鱗狀NSCLC患者，僅剩化療或姑息治療，其可選方案明顯少於非鱗狀NSCLC5。
關於BioNTech
生物制藥新技術公司（BioNTech）是壹家全球領先的下壹代免疫療法企業，專註於癌癥及其他重癥創新療法的研究與開發。公司依托計算發現平臺及多元藥物類型，加速新型生物藥的研發。其腫瘤產品組合涵蓋mRNA癌癥免疫療法、新壹代免疫調節藥物，以及抗體-藥物偶聯物（ADC）、創新CAR-T細胞療法等靶向藥物，貫穿癌癥治療全周期。憑借在mRNA領域的深厚經驗和自主生產能力，BioNTech與合作夥伴同步推進多款傳染病mRNA疫苗及多樣化腫瘤管線的研究與開發。BioNTech已建立廣泛合作網絡，合作方包括BMS、映恩生物、復星醫藥、Genentech（Roche集團成員）、Genmab、MediLink、OncoC4、Pfizer和Regeneron等公司。
更多信息請訪問 www.BioNTech.com
關於OncoC4
OncoC4總部位於美國馬裏蘭州羅克維爾，是壹家私有、處於後期臨床階段的生物制藥公司，專註於發現和開發用於治療癌癥及免疫疾病的新型生物制劑。公司管線涵蓋多個具有同類首創或同類最佳潛力的項目，靶點新穎且已充分驗證，橫跨腫瘤學與免疫學領域。其中，AI-081為OncoC4完全擁有的PD-1×VEGF雙特異性抗體候選藥物；ONC-841是壹款同類首創的抗SIGLEC10抗體，正在開展針對腫瘤的2期臨床研究，並探索用於神經退行性疾病的可能性。OncoC4與BioNTech 達成戰略合作，共同開發腫瘤微環境選擇性Treg清除候選藥物gotistobart（BNT316/ONC-392，靶向CTLA-4），用於多種實體瘤適應癥，目前雙方正推進針對sqNSCLC的關鍵3期臨床試驗。
更多信息請訪問 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.
聯系方式
OncoC4
投資者關系
Ryan Cui
[email protected]
媒體關系
Helen Schiltz
[email protected]
BioNTech
投資者關系
Douglas Maffei, Ph.D.
[email protected]
媒體關系
Jasmina Alatovic
[email protected]
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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