Tipapkinogene sovacivec

- Poster presentation of updated data on BT-001 from the Phase 1 part of the ongoing Phase 1/2a study in solid tumors - Lund, Sweden, and Strasbourg, France, July 28, 2025 – BioInvent International AB (“BioInvent”) (Nasdaq Stockholm: BINV), a biotech company focused on the discovery and development of novel and first-in-class immune-modulatory antibodies for cancer immunotherapy, and Transgene (Euronext Paris: TNG), a biotech company that designs and develops virus-based immunotherapies for the treatment of cancer, and will jointly present a poster on updated data from the Phase 1 part of the Phase 1/2a study of BT-001 at the European Society for Medical Oncology (ESMO) Annual Meeting. The ESMO will take place in Berlin, Germany, from October 17 to 21, 2025. Poster details:Title: “Updated clinical results of BT-001, an oncolytic virus expressing an anti-CTLA-4 mAb, administered in combination with pembrolizumab in patients with advanced solid tumors.”Abstract number: 2828Date: October 18–20, 2025Authors: C. Lebbe, R. Bahleda, E. Ezine, B. Baroudjian, M. Sakkal, E. Rowinski, A. Vinceneux, S. Champiat, K. Bidet Huang, N. Stojkowitz, H. Makhloufi, A. Sadoun, A. Ropenga, M. Semmrich, A. McAllister, M. Chisamore, P. Cassier The abstract will be available on ESMO’s website on October 13, 2025, at 00:05 CEST. BT-001 is an oncolytic virus generated using Transgene’s Invir.IO® platform and its patented large-capacity VVcopTK-RR- oncolytic virus, which has been engineered to encode both a Treg-depleting recombinant human anti-CTLA-4 antibody generated by BioInvent’s proprietary n-CoDeR®/F.I.R.S.T™ platforms, and the human GM-CSF cytokine. BT-001 is being co-developed as part of a 50/50 collaboration on oncolytic viruses between Transgene and BioInvent. In the Phase 1 part of this study, as monotherapy and in combination with MSD’s anti-PD-1 therapy KEYTRUDA® (pembrolizumab), BT-001 has been shown to be well tolerated. BT-001 also showed the first signs of efficacy with clinical response in two out of six refractory patients, thirteen patients evaluated in total, when given in combination with pembrolizumab, with shrinkage of injected and non-injected lesions. Treatment with BT-001 converted “cold” tumors into “hot” ones, and induced T-cell infiltration, as well as PD(L)-1 expression in the tumor microenvironment (See press release from September 14, 2024 here). The ongoing Phase 1/2a study (NCT04725331) is a multicenter, open label, dose-escalation study evaluating BT-001 as a single agent and in combination with MSD’s (Merck & Co., Inc., Rahway, NJ, USA) anti-PD-1 therapy KEYTRUDA® (pembrolizumab). The last patient in the Phase 1 part was enrolled in August 2024. KEYTRUDA® is a registered trademark of Merck Sharp & Dohme LLC, a subsidiary of Merck & Co., Inc., Rahway, NJ, USA.[GC1] ContactsTransgene:Media:Caroline ToschCorporate and Scientific Communications Manager+33 (0)3 68 33 27 38 Investors & Analysts:Lucie LarguierChief Financial Officer (CFO)Nadege BartoliInvestor Relations Analystand Financial Communications Officer+33 (0)3 88 27 91 00/03 MEDiSTRAVAFrazer Hall/Sylvie Berrebi+ 44 (0)203 928 6900 BioInvent:Cecilia HofvanderVP Investor Relations+46 (0)46,286 85 50cecilia.hofvander@bioinvent.com About TransgeneTransgene (Euronext: TNG) is a biotechnology company focused on designing and developing targeted immunotherapies for the treatment of cancer. The Company’s clinical-stage programs consist of a portfolio of viral vector-based immunotherapeutics. TG4050, the first individualized therapeutic vaccine based on the myvac® platform is the Company’s lead asset, with demonstrated proof of principle in patients in the adjuvant treatment of head and neck cancers. The portfolio also includes other viral-vector-based immunotherapies: TG4001 for the treatment of HPV-positive cancers, as well as BT-001 and TG6050, two oncolytic viruses based on the Invir.IO® viral backbone. The Company also conducts innovative discovery and preclinical work, aimed at developing novel viral vector-based modalities.With Transgene’s myvac® platform, therapeutic vaccination enters the field of precision medicine with a novel immunotherapy that is fully tailored to each individual. The myvac® approach allows the generation of a virus-based immunotherapy that encodes patient-specific mutations identified and selected by Artificial Intelligence capabilities provided by its partner NEC.With its proprietary platform Invir.IO®, Transgene is building on its viral vector engineering expertise to design a new generation of multifunctional oncolytic viruses.Additional information about Transgene is available at:www.transgene.comFollow us on social media: X (formerly Twitter): @TransgeneSA — LinkedIn: @Transgene — Bluesky: @Transgene About BioInventBioInvent International AB (Nasdaq Stockholm: BINV) is a clinical-stage biotech company that discovers and develops novel and first-in-class immuno-modulatory antibodies for cancer therapy, with currently five drug candidates in six ongoing clinical programs in Phase 1/2 studies for the treatment of hematological cancer and solid tumors, respectively. The Company’s validated, proprietary F.I.R.S.T.™ technology platform identifies both targets and the antibodies that bind to them, generating many promising new drug candidates to fuel the Company’s own clinical development pipeline and providing licensing and partnering opportunities.The Company generates revenues from research collaborations and license agreements with multiple top-tier pharmaceutical companies, as well as from producing antibodies for third parties in the Company’s fully integrated manufacturing unit. More information is available at http://www.bioinvent.com/ Disclaimer TransgeneThis press release contains forward-looking statements, which are subject to numerous risks and uncertainties, which could cause actual results to differ materially from those anticipated. The occurrence of any of these risks could have a significant negative outcome for the Company’s activities, perspectives, financial situation, results, regulatory authorities’ agreement with development phases, and development. The Company’s ability to commercialize its products depends on but is not limited to the following factors: positive pre-clinical data may not be predictive of human clinical results, the success of clinical studies, the ability to obtain financing and/or partnerships for product manufacturing, development and commercialization, and marketing approval by government regulatory authorities. For a discussion of risks and uncertainties which could cause the Company’s actual results, financial condition, performance or achievements to differ from those contained in the forward-looking statements, please refer to the Risk Factors (“Facteurs de Risque”) section of the Universal Registration Document, available on the AMF website (http://www.amf-france.org) or on Transgene’s website (www.transgene.fr). Forward-looking statements speak only as of the date on which they are made, and Transgene undertakes no obligation to update these forward-looking statements, even if new information becomes available in the future. Disclaimer BioInventThe press release contains statements about the future, consisting of subjective assumptions and forecasts for future scenarios. Predictions for the future only apply as the date they are made and are, by their very nature, in the same way as research and development work in the biotech segment, associated with risk and uncertainty. With this in mind, the actual outcome may deviate significantly from the scenarios described in this press release.

创新药物的研发总是充满失败的可能，本文统计了2024年5款临床试验失败的疫苗，涉及Transgene、GSK、HilleVax、Ultimovacs ASA、Barinthus Biotherapeutics这5家企业。从所处临床阶段看，基本都处于临床2期。下面为具体信息，如有错漏，欢迎留言补充。 1、公司：Transgene 疫苗：TG4001 临床阶段：2期 10月14日，Transgene公布了其治疗性癌症疫苗Tipapkinogen sovacivec（TG4001）最新的2期临床研究结果，与PD-L1抑制剂阿维鲁单抗（Avelumab）联用治疗HPV16阳性宫颈和肛门生殖器肿瘤未达到主要终点——改善无进展生存期。 TG4001是一款基于重组修饰牛痘病毒（MVA）为载体的疫苗，被设计表达HPV-16 E6/E7和佐剂（IL-2）。TG4001通过刺激免疫系统识别HPV-16 E6/E7，以攻击HPV阳性肿瘤细胞。TG4001已在超过350人中进行试验。 不过值得注意的是，虽然该疫苗的2期试验未达到主要终点，但据其预先计划的亚组分析显示，接受TG4001联合治疗方案的宫颈癌患者显示出积极的治疗趋势。但这还需要通过其他分析（包括PD-L1状态）进一步确认并评估。 2、公司：GSK 疫苗：GSK3943104 临床阶段：2期 9月11日，葛兰素史克（GSK）宣布其首款单纯疱疹病毒（HSV）疫苗GSK3943104在2期临床试验中未能达到试验的主要疗效目标，公司将不再推进到3临床，但会继续完成2临床的安全性数据随访和分析，这些数据可以为复发性生殖器疱疹提供有价值的见解，并为未来的研究工作提供参考。 GSK3943104是一款生殖器疱疹的治疗性疫苗，研发目的是减少复发性生殖器疱疹患者的病变。它通过诱导已经感染病毒的患者产生抗病毒免疫力，以限制疱疹的活跃和复制并减少复发。 3、公司：HilleVax 疫苗：HIL-214 临床阶段：2b期 7月8日，HilleVax公司发表声明称，由于2b期NEST-IN1试验未能达到主要或次要疗效终点，公司将停止为婴儿进一步开发HIL-214诺如疫苗。不过，公司会继续探索HIL-214在成人中开发的可能性。 HIL-214是一种诺如病毒二价GI.1/GII.4病毒样颗粒（VLP）疫苗，用于预防由诺如病毒感染引起的中度至重度急性胃肠炎。NEST-IN1是一项2b期随机、双盲、安慰剂对照临床试验，目的是评估HIL-214在美国和拉丁美洲部分地区为约5个月大婴儿首次接种疫苗时的有效性、安全性和免疫原性，涉及约2800名受试者。 在NEST-IN1研究中，有51个主要终点事件，其中疫苗组25个（n=1425），安慰剂组26个（n=1399），疫苗效力为5%（95%置信区间：-64%，45%）。该研究未达到其主要终点，即未能证明对由GI.1或GII.4诺如病毒基因型引起的中度或重度急性胃肠炎（AGE）事件具有疗效。在次要终点方面也未观察到任何临床益处。HIL-214的安全性和免疫原性特征与NEST-IN1中前200名受试者的预设分析以及之前报告的研究中观察到的一致。公司认为，在该试验中多种新兴GII.4毒株的出现可能影响了疫苗在婴儿中的疗效。 4、公司：Ultimovacs ASA 疫苗：UV1 临床阶段：2期 8月6 日，Ultimovacs ASA宣布其UV1治疗头颈部鳞状细胞癌（HNSCC）患者的FOCUS 2期临床试验（NCT05075122）的顶线结果。数据显示，该研究未能达到其主要和次要终点。 UV1是一款肿瘤疫苗，由三种长合成肽组成，表达人类端粒酶（hTERT）逆转录酶亚基的 60 个氨基酸，其作用机制是通过诱导针对 hTERT 的特异性T细胞反应。皮内注射UV1后，皮肤中的抗原呈递细胞（APC）将对这些肽进行处理并激活T细胞。活化的疫苗特异性T细胞进入循环，在HLA复合物背景下搜索显示其同源抗原的细胞，激活免疫系统级联反应，从而增强抗肿瘤反应。 值得注意的是，UV1正在进行5项2期研究，针对5个不同的适应症——黑色素瘤、间皮瘤、卵巢癌、头颈癌、非小细胞肺癌，包括约670名患者。然而，除了间皮瘤外，目前针对黑色素瘤和头颈癌的研究都显示失败了。今年3月，Ultimovacs宣布UV1联用Ipilimumab+nivolumab治疗不可切除或转移性黑色素瘤的2期临床试验INITIUM主要终点和次要终点均未达到。 目前，Ultimovacs公司将重心转移到UV1治疗卵巢癌上，相关的2期数据预计将于2025年上半年公布。 5、公司：Barinthus Biotherapeutics 疫苗：VTP-200 临床阶段：1b/2期 4月18日，Barinthus Biotherapeutics宣布其在研的高危人瘤病毒（hrHPV）相关宫颈癌的疗法VTP-200的1b/2期试验（APOLLO）已达到主要终点，数据表明其对高危人瘤病毒（hrHPV）相关宫颈癌的治疗是安全的。但是，由于未能证明该疗法确实有效，这家生物技术公司正在考虑如何进一步开发候选药物。 VTP-200是一种免疫治疗组合方案，涉及患者使用ChAdOx载体接受初始剂量，使用MVA载体接受第二剂量。两种剂量都编码相同的HPV抗原，目的是产生针对HPV的抗原特异性T细胞免疫反应。 APOLLO试验是一项随机、安慰剂对照的1b/2期多中心试验，在英国和欧盟招募了108名参与者，旨在评估VTP-200在25-55岁患有持续高危（hr）HPV感染和低度宫颈病变的女性中的安全性、耐受性和免疫原性。结果显示，该试验达到主要安全终点，VTP-200总体耐受性良好，无治疗相关的3级或更高级别的不良事件（AE）或严重的AE（SAE）；在接受最高ChAdOx 剂量的组中观察到高危（hr）HPV（60%，第 2 组）和宫颈病变（67%，第2组和第5组） 的清除率呈阳性趋势；来自五个不同活性剂量组的汇总数据显示，与安慰剂组相比，hrHPV 或宫颈病变清除率没有统计学意义改善。 参考资料： [1]汇总|2024年全球值得关注的79个临床试验失败案例.法迈医讯.2024年12月26日. [2]63项失败临床研究汇总|2024 H1.生物药大时代.2024年07月07日. [3]II期失败的实体瘤疫苗.佰傲谷BioValley.2024年10月15日. [4]生殖器疱疹疫苗：GSK宣布失败，但Moderna和BioNTech仍有机会！.艾坦汀官方.2024年12月03日. [5]癌症疫苗 UV1 新数据！头颈鳞癌 II 期临床结果未达终点.Insight数据库.2024年08月06日. [6]接连失败的肿瘤疫苗.生物制药小编.2024年08月09日. [7]安全但无效？！Barinthus正在评估HPV相关癌症疗法的未来.一度医药.2024年04月22日. 识别微信二维码，可添加药时空小编 请注明：姓名+研究方向！