Invectys SAS

▎药明康德内容团队编辑本期看点1. 接受靶向BAFFR的自体CAR-T细胞疗法PMB-CT01的首位患者在治疗后1个月就达到了完全缓解（CR）。2. 创新机制抗肿瘤疫苗AGI-134在不可切除的转移性实体肿瘤患者中的疾病稳定（SD）率达29%。3. 治疗杜氏肌营养不良症（DMD）的反义寡核苷酸（ASO）疗法WVE-N531公布积极早期临床试验结果。4. 胰高血糖素样肽-1（GLP-1）/胰高血糖素双重受体激动剂pemvidutide（ALT-801）治疗非酒精性脂肪性肝炎（NAFLD）患者，治疗24周后，患者肝脏脂肪含量相对减少超过75%。5. KRAS G12C抑制剂Krazati（adagrasib）获突破性疗法认定（BTD），并公布在结直肠癌患者中的1/2期临床试验的积极结果。药明康德内容团队整理PMB-CT01：公布1期临床试验中首例患者的数据PeproMene Bio公司公布了其靶向BAFFR的自体CAR-T细胞疗法PMB-CT01在1期临床试验中的首例患者数据。BAFF-R（B细胞活化因子受体）是肿瘤坏死因子（TNF）受体超家族成员，是几乎只在B细胞上表达的BAFF的主要受体。由于BAFF-R信号传导能够促进正常B细胞增殖，且似乎是B细胞生存所必需的。因此，肿瘤细胞不太可能通过BAFF-R抗原的丢失而逃避免疫应答。这种独特的特性使靶向BAFF-R的CAR-T细胞疗法有望成为治疗B细胞恶性肿瘤的潜在强有力的治疗方法。在体外和动物模型中，PMB-CT01都能杀死人类淋巴瘤和白血病的癌细胞。此次公布的1期临床试验结果显示，首例接受治疗的B细胞非霍奇金淋巴瘤患者在接受治疗1个月后即达到了CR，仅出现了低级的治疗紧急毒性，包括已完全康复的1级细胞因子释放综合征（CRS），未观察到神经毒性/免疫效应细胞相关神经毒性综合征（ICANS）。AGI-134：公布1/2a期临床试验数据BioLineRx公司公布了其研究性抗肿瘤疫苗AGI-134的1/2a期研究结果。AGI-134是一种用于实体肿瘤的合成α-Gal糖脂，通过肿瘤内给药，AGI-134可以用α-Gal标记癌细胞，从而让机体预先存在的大量抗α-Gal抗体，重定向至接受过AGI-134治疗的肿瘤。抗Gal抗体与肿瘤结合后会激活补体级联反应，破坏肿瘤细胞并产生促炎肿瘤微环境。这种微环境会诱导对患者自身肿瘤新抗原的全身性、特异性的抗肿瘤反应。AGI-134已在众多临床前研究中证实了它可以导致原发性肿瘤的消退，并抑制继发性肿瘤（转移）的发展。此外，当与抗PD-1免疫检查点抑制剂联合使用时，AGI-134具有协同作用，有可能扩大这种免疫疗法的效用，并提高在多种癌症类型中的缓解率和持续时间。此次公布的结果显示，AGI-134达到了1/2a期研究的安全性和耐受性的主要终点。放射学评估发现，29%（11/38）的患者的最佳总体缓解为SD，其中7例有既往免疫检查点抑制剂治疗失败的经历。此外，多种生物标志物显示AGI-134能够增加患者的整体免疫活性。WVE-N531：公布1b/2a期临床试验数据Wave Life Sciences公司发布了其反义寡核苷酸（ASO）疗法WVE-N531在三个患有杜氏肌营养不良症（DMD）的男孩身上进行的1b/2a期概念验证研究的积极结果。DMD是一种致命的X连锁遗传性神经肌肉疾病，主要由编码抗肌萎缩蛋白（dystrophin）的基因发生突变所引起。约8%-10%的DMD患儿的基因突变可通过53号外显子跳跃疗法进行治疗。外显子跳跃疗法旨在通过促进抗肌萎缩蛋白的产生来稳定或减缓疾病的进展，从而解决DMD的根本病因。WVE-N531是一种针对DMD的研究性外显子跳跃疗法，用于调控编码抗肌萎缩蛋白的RNA前体的剪接过程。三名非卧床的DMD男性患儿参加了这项开放标签、剂量递增临床试验。研究结果显示，该疗法的安全和耐受性良好，连续三次给药后可导致大量外显子跳跃。新闻稿指出，这是在患有DMD的男孩中进行的临床试验中所报告的外显子跳跃的最早时间点。Pemvidutide：公布1b期临床试验数据Altimmune公司公布了其候选药物pemvidutide治疗NAFLD患者为期24周（其中12周为延长试验）的早期临床试验的顶线结果。Pemvidutide是一款基于多肽的GLP-1/胰高血糖素双重受体激动剂。胰高血糖素能够增加能量消耗，并且促进身体中储存能量的白色脂肪转化为消耗能量的褐色脂肪。它与GLP-1产生协同作用，有望促进比GLP-1受体激动剂单药更有效的体重减轻。胰高血糖素也被认为对肝脏脂肪代谢有直接影响，能够使肝脏脂肪水平迅速降低。此次公布的1b期临床试验结果显示，在所有pemvidutide治疗组中均达到了主要终点及关键次要减肥终点。在第12周和第24周，使用pemvidutide治疗的NAFLD受试者显示出在肝脏脂肪含量、血清ALT水平和体重方面均有降低。在24周时，在1.8 mg和2.4 mg剂量下，患者的肝脏脂肪含量相对减少超过75%。在1.8 mg剂量下，无糖尿病受试者的平均体重减轻7.2%（根据安慰剂组调整后为6.0%），所有受试者的平均体重减轻6.2%（根据安慰剂组调整后4.8%）。此外，血糖控制与糖尿病受试者的空腹血糖和HbA1c改善的趋势依然得到了保持。Krazati（adagrasib）：公布1/2期临床试验数据Mirati Therapeutics公司宣布FDA授予其KRAS G12C抑制剂Krazati（adagrasib）突破性疗法认定（BTD），与EGFR抑制剂cetuximab联用治疗肿瘤带有KRAS G12C突变的晚期结直肠癌患者。Krazati是一款具有高度特异性的强力口服KRAS G12C抑制剂，经过优化设计具有持久的靶点抑制能力。Krazati具有长达24小时的半衰期和广泛的组织分布，而且能够穿过血脑屏障，有助于最大限度地发挥药物效力。此前，美国FDA还授予过它另一项突破性疗法认定，用于治疗携带KRAS G12C突变的经治非小细胞肺癌患者。此次公布的数据为Krazati作为单药或与cetuximab联用治疗携带KRAS G12C突变的晚期结直肠癌患者的1/2期临床试验结果。数据显示，该候选药物对这类患者群体具有显著的临床效力与安全性。在所有28位可评估的患者中，接受Krazati与cetuximab联合疗法的患者具有46%的客观缓解率（ORR），其中位缓解持续时间（DOR）为7.6个月，中位无进展生存期（PFS）为6.9个月。Krazati在此试验中的安全性与以往试验一致，而其与cetuximab的联合疗法并未造成协同不良反应。IVS-3001：IND申请获得FDA许可IVS-3001是Invectys公司与MD安德森癌症中心设立的合资公司CTMC合作开发的HLA-G靶向CAR-T细胞疗法。HLA-G不仅是一种免疫检查点，也是一种肿瘤特异性抗原。IVS-3001能够识别并杀死在细胞表面表达HLA-G的靶细胞。IVS-3001的临床前研究已经产生了足够的数据来支持其有效性和安全性，因而其IND申请获得了FDA许可。该公司正计划在局部晚期不可切除或转移性的HLA-G+选择的恶性实体肿瘤患者中启动1/2a期临床试验。MZE001：公布1期临床试验数据Maze Therapeutics公司公布了其针对庞贝病（Pompe disease）所开发的在研抑制剂MZE001于临床1期试验中的积极结果。庞贝病是由于缺乏酸性α-葡萄糖苷酶（GAA）引起的遗传性溶酶体储存障碍。酶替代疗法是目前唯一获批的疗法，但仍有未满足的临床需求。MZE001是一种口服糖原合成酶1（GYS1）抑制剂，旨在抑制庞贝病的致病原因——糖原累积。此次公布的数据分析显示，MZE001在单剂与多剂量递送时皆呈现良好的耐受性，显示具一天两剂的药代动力学特征，并具有降低生物标志物血细胞糖原（glycogen）水平的能力。STAR-0215：公布1a期临床试验的初步数据Astria Therapeutics公司公布了其用于治疗遗传性血管性水肿（HAE）的血浆激肽释放酶单克隆抗体抑制剂STAR-0215在健康受试者中的1a期临床试验的初步数据。结果显示，STAR-0215具有作为长效血浆激肽释放酶抑制剂的潜力，估计的半衰期长达110天，支持STAR-0215每三个月给药一次或以更低的频率给药。该公司计划在2023年第一季度在HAE患者中启动1b/2期试验。Emraclidine：公布1b期临床试验数据Cerevel Therapeutics公司公布了其新型毒蕈碱M4选择性正变构调节剂emraclidine用于精神分裂症成人患者的早期临床数据。结果显示，与安慰剂相比，emraclidine在阳性和阴性症状量表（PANSS）总分中显示出具有临床意义和统计学意义的改善，总体耐受性良好。动态血压监测试验结果显示，emraclidine长期给药不会引起精神分裂症患者血压升高。同时，研究结果还表明，选择性靶向M4毒蕈碱受体亚型是治疗精神分裂症的潜在新方法。ABI-4334、ABI-H3733：公布1a/1b期临床试验数据Assembly Biosciences公司公布了其下一代HBV核心抑制剂ABI-H3733的1b期临床研究和ABI-4334的1a期临床研究的中期疗效、安全性和药代动力学（PK）结果。结果显示，在为期28天的1b期研究中，8例接受ABI-H3733治疗的HBV患者中有6例在第21天时的HBV DNA低于定量检测的下限，患者的血浆HBV DNA平均减少了3.1个对数值。ABI-4334在1a期试验中的结果表现出支持其每日一次口服给药的PK特征，并且ABI-4334在临床前实验中观察到的高效力在临床试验中也展现出了早期迹象。在这两项研究中均未观察到严重不良事件或实验室异常，包括丙氨酸氨基转移酶（ALT）升高。药明康德为全球生物医药行业提供一体化、端到端的新药研发和生产服务，服务范围涵盖化学药研发和生产、生物学研究、临床前测试和临床试验研发、细胞及基因疗法研发、测试和生产等领域。如您有相关业务需求，欢迎点击下方图片填写具体信息。▲如您有任何业务需求，请长按扫描上方二维码，或点击文末“阅读原文/Read more”，即可访问业务对接平台，填写业务需求信息▲欲了解更多前沿技术在生物医药产业中的应用，请长按扫描上方二维码，即可访问“药明直播间”，观看相关话题的直播讨论与精彩回放参考资料（可上下滑动查看）[1] Astria Therapeutics Announces Positive Preliminary Results from the Phase 1a Clinical Trial of STAR-0215 in Healthy Subjects. Retrieved December 15, 2022, from https://www.businesswire.com/news/home/20221215005251/en[2] Calypso Biotech Announces First Patient with Eosinophilic Esophagitis Dosed in Anti-Interleukin-15 (IL-15) Monoclonal Antibody CALY-002 Phase 1a/b Trial, and Animal Proof of Concept Data in Atopic Dermatitis. Retrieved December 15, 2022, from https://pipelinereview.com/index.php/2022121582298/Antibodies/Calypso-Biotech-Announces-First-Patient-with-Eosinophilic-Esophagitis-Dosed-in-Anti-Interleukin-15-IL-15-Monoclonal-Antibody-CALY-002-Phase-1a/b-Trial-and-Animal-Proof-of-Conc.html[3] Rigel Doses First Patient in Phase 1b Study of R289 for the Treatment of Lower-Risk Myelodysplastic Syndromes. Retrieved December 15, 2022, from https://www.prnewswire.com/news-releases/rigel-doses-first-patient-in-phase-1b-study-of-r289-for-the-treatment-of-lower-risk-myelodysplastic-syndromes-301703773.html[4] Cerevel Therapeutics Announces Publication in The Lancet of Emraclidine Data from Phase 1b Clinical Trial in People Living with Schizophrenia. Retrieved December 15, 2022, from https://investors.cerevel.com/news-releases/news-release-details/cerevel-therapeutics-announces-publication-lancet-emraclidine[5] Maze Therapeutics Announces Completion of Phase 1 First-in-Human Trial Evaluating MZE001 as a Potential Oral Treatment for Pompe Disease. Retrieved December 15, 2022 from https://www.businesswire.com/news/home/20221215005328/en/Maze-Therapeutics-Announces-Completion-of-Phase-1-First-in-Human-Trial-Evaluating-MZE001-as-a-Potential-Oral-Treatment-for-Pompe-Disease[6] Frequency Therapeutics Announces First Patient Dosed in Phase 1b Study of FX-345, the Company’s Second Therapeutic Candidate for Sensorineural Hearing Loss. Retrieved December 15, 2022 from https://www.businesswire.com/news/home/20221215005106/en/[7] SwanBio Therapeutics Initiates First-in-Human Study of AAV Gene Therapy for Adrenomyeloneuropathy. Retrieved December 15, 2022, from https://www.businesswire.com/news/home/20221215005320/en/[8] Nuvation Bio Doses First Patient in Phase 1b Combination Study of NUV-868. Retrieved December 19, 2022, from https://www.businesswire.com/news/home/20221219005673/en/[9] Wave Life Sciences Provides Positive Update on Proof-of-Concept Study for WVE-N531 in Duchenne Muscular Dystrophy. Retrieved December 19, 2022, from https://ir.wavelifesciences.com/news-releases/news-release-details/wave-life-sciences-provides-positive-update-proof-concept-study[10] erevel Therapeutics Announces Positive Results in Emraclidine Ambulatory Blood Pressure Monitoring Trial. Retrieved December 19, 2022, from https://investors.cerevel.com/news-releases/news-release-details/cerevel-therapeutics-announces-positive-results-emraclidine[11] Assembly Biosciences Announces Promising Interim Results from Two Clinical Trials Evaluating Highly Potent Next-Generation Core Inhibitor Candidates ABI-H3733 and ABI-4334. Retrieved December 19, 2022, from https://investor.assemblybio.com/news-releases/news-release-details/assembly-biosciences-announces-promising-interim-results-two[12] BioInvent Initiates Phase 1 Trial with Subcutaneous Formulation of BI-1206. Retrieved December 19, 2022, from https://www.accesswire.com/732321/BioInvent-Initiates-Phase-1-Trial-with-Subcutaneous-Formulation-of-BI-1206[13] Invectys and CTMC announce FDA clearance of IND application for anti-HLA-G CAR-T cell therapy. Retrieved December 19, 2022, from https://www.globenewswire.com/news-release/2022/12/19/2576489/0/en/Invectys-and-CTMC-announce-FDA-clearance-of-IND-application-for-anti-HLA-G-CAR-T-cell-therapy.html[14] BioLineRx Announces Results from Phase 1/2a Study of Investigational Anti-Tumor Vaccine AGI-134 in Metastatic Solid Tumors. Retrieved December 20, 2022, from https://ir.biolinerx.com/news-releases/news-release-details/biolinerx-announces-results-phase-12a-study-investigational-anti[15] PeproMene Bio, Inc. Announced Complete Response of The First Patient Treated in Its B-cell Non-Hodgkin Lymphoma (B-NHL) Phase 1 Clinical Trial of PMB-CT01 (BAFFR-CAR T Cells) at City of Hope. Retrieved December 20, 2022, from https://www.prnewswire.com/news-releases/pepromene-bio-inc-announced-complete-response-of-the-first-patient-treated-in-its-b-cell-non-hodgkin-lymphoma-b-nhl-phase-1-clinical-trial-of-pmb-ct01-baffr-car-t-cells-at-city-of-hope-301707651.html[16] ERASCA ANNOUNCES FIRST PATIENT DOSED IN HERKULES-1 PHASE 1B TRIAL EVALUATING ERAS-007 AND ERAS-601 MAPKLAMP COMBINATION IN RAS/MAPK PATHWAY-ALTERED SOLID TUMORS. Retrieved December 20, 2022, from https://investors.erasca.com/news-releases/news-release-details/erasca-announces-first-patient-dosed-herkules-1-phase-1b-trial[17] Altimmune Announces Positive Topline Results From 24-Week (12-Week Extension) Trial Of Pemvidutide In Subjects With Non-Alcoholic Fatty Liver Disease (NAFLD). Retrieved December 20, 2022, from https://ir.altimmune.com/news-releases/news-release-details/altimmune-announces-positive-topline-results-24-week-12-week[18] BioNTech Initiates Phase 1 Clinical Trial for Malaria Vaccine Program BNT165. Retrieved December 23, 2022, from https://investors.biontech.de/news-releases/news-release-details/biontech-initiates-phase-1-clinical-trial-malaria-vaccine[19] BioNTech Starts Phase 1 Clinical Trial for Prophylactic Herpes Simplex Virus-2 Vaccine Candidate BNT163. Retrieved December 23, 2022, from https://investors.biontech.de/news-releases/news-release-details/biontech-starts-phase-1-clinical-trial-prophylactic-herpes[20] Mirati announces Adagrasib (KRAZATI™) Receives Breakthrough Therapy Designation from FDA for Patients with Advanced, KRAS-Mutated Colorectal Cancer and NEJM Publishes Phase 1b/2 Data from Adagrasib With or Without Cetuximab in Colorectal Cancer. Retrieved December 21, 2022, from https://ir.mirati.com/press-releases/press-release-details/2022/Mirati-announces-Adagrasib-KRAZATI-Receives-Breakthrough-Therapy-Designation-from-FDA-for-Patients-with-Advanced-KRAS-Mutated-Colorectal-Cancer-and-NEJM-Publishes-Phase-1b2-Data-from-Adagrasib-With-or-Without-Cetuximab-in-Colorectal-Cancer/default.aspx免责声明：药明康德内容团队专注介绍全球生物医药健康研究进展。本文仅作信息交流之目的，文中观点不代表药明康德立场，亦不代表药明康德支持或反对文中观点。本文也不是治疗方案推荐。如需获得治疗方案指导，请前往正规医院就诊。版权说明：本文来自药明康德内容团队，欢迎个人转发至朋友圈，谢绝媒体或机构未经授权以任何形式转载至其他平台。转载授权请在「药明康德」微信公众号回复“转载”，获取转载须知。分享，点赞，在看，聚焦全球生物医药健康创新

++++++++++++++++++++++++++++++++++++++++++++++++++++++++++++++++++++++++++++++++本文大概：2000字                                      阅读需要：2分钟佰傲谷靶点药讯专刊，带您纵览全球最新靶点动态国际药闻1CLDN18.2抗体3期临床达主要终点12月18日，安斯泰来宣布，靶向Claudin18.2（CLDN18.2）的在研抗体疗法zolbetuximab与化疗联用，在一线治疗局部晚期不可切除或转移性胃癌和胃食管结合部（GEJ）腺癌患者的3期临床试验中达到主要终点，显著延长患者的无进展生存期（PFS）。这是安斯泰来一个月内所公布关于zolbetuximab在治疗胃癌患者上的第二项临床3期试验积极数据。2礼来进军GPCR礼来与Sosei Group公司达成合作，将联合发现、开发和商业化针对新型G蛋白偶联受体（GPCR）靶点的小分子药物，用于治疗糖尿病和代谢疾病。根据协议条款，礼来将预付3700万美元的现金，以获得多个GPCR药物的权利，而Sosei将根据礼来指定的多个GPCR靶点，发现靶点特异性小分子先导化合物，用于进一步开发和商业化。协议中Sosei将有资格获得高达6.94亿美元的开发和商业里程碑，以及全球分层特许权费用。延伸阅读：礼来花费7亿美元进军GPCR，布局糖尿病等创新疗法3HLA-G CAR-T 治疗实体瘤IND获批12月19日，Invectys公司宣布美国FDA批准了HLA-G靶向CAR-T细胞IVS-3001的IND申请，用于治疗实体瘤。IVS-3001是Invectys与MD安德森癌症中心设立的合资公司CTMC合作开发的，由MD安德森癌症中心研究员Aung Naing教授和共同PI助理教授 Samer Srour领导。4同种异体T细胞免疫疗法上市12月20日，Atara Biotherapeutics和Pierre Fabre联合宣布，欧盟委员会（EC）批准其免疫疗法Ebvallo（Tabelecleucel）上市，作为单药治疗EB病毒（EBV）相关的移植后淋巴增殖性疾病（EBV+PTLD），病患包含曾至少接受过一次前期治疗的成人与2岁以上儿童患者。延伸阅读：全球首个同种异体T细胞免疫疗法Ebvallo获批上市5HER2双抗临床数据积极Zymeworks公布了其靶向HER2的双特异性抗体zanidatamab的2b期研究的积极顶线结果。数据显示：在既往接受过治疗的HER2扩增和表达的BTC患者中，zanidatamab单药治疗确认的客观缓解率（cORR）为41.3%，中位缓解持续时间为12.9个月。Zanidatamab的安全性与先前报道的单药治疗研究中观察到的安全性一致，没有发现新的安全信号。6CD117 ADC 因DLT毒性暂停给药12月20日，Magenta Therapeutics公司宣布，由于剂量限制性毒性问题，公司停止在研药物MGTA-117正在进行的I/II期研究中的最高剂量组给药。MGTA-117是一种靶向CD117的抗体-药物偶联物，是一种通过移除骨髓中导致某些遗传疾病的基因突变细胞而使干细胞移植或基因治疗成为可能的药物（CD117表达于造血干细胞和祖细胞（HSPC）以及约80%的AML和MDS细胞）。MGTA-117由CD117抗体与amanitin（鹅膏蕈碱）偶联而成。延伸阅读：股价下跌50%，CD117-ADC药物因DLT毒性暂停给药国内创新1博生吉CD7 CAR-T获得欧洲孤儿药资格认定近日，博生吉医药科技（苏州）有限公司（以下简称“博生吉”）宣布，其开发的靶向CD7的嵌合抗原受体（CAR）-T细胞注射液（PA3-17注射液）收到欧盟委员会（以下简称“EC”）授予的孤儿药资格认定（Orphan Drug Designation, ODD），用于治疗复发难治的T细胞急性淋巴细胞白血病/淋巴瘤（T-cell Acute Lymphoblastic Leukemia/Lymphoma (T-ALL/LBL)。2复星凯特第二款CAR-T细胞获批临床12月20日，根据国家药品监督管理局药品审评中心官网，复星凯特的CD19 CAR-T细胞FKC889的IND获批，将开展治疗复发或难治性成人前体B细胞急性淋巴细胞白血病（成人R/R ALL）的药物临床试验。这是FKC889继今年3月获批开展治疗复发或难治性套细胞淋巴瘤（R/R MCL）的临床试验以来，再度通过新适应症的临床试验申请。3科望医药CD39/TGF-β获批临床12月21日，科望生物自主研发的注射用ES014获NMPA临床批准，用于实体瘤的治疗。今年5月，ES014已经获得FDA批准开展临床试验。ES014是科望医药开发的一种能够同时靶向CD39和TGF-β的双功能分子，通过将TGF-βⅡ型受体胞外结构域融合到靶向CD39的抗体上设计而成。延伸阅读：科望医药：全球首款CD39/TGF-β双抗获批临床4驯鹿生物BCMA CAR-T细胞治疗IND申请获FDA批准12月22日，驯鹿生物宣布其自主研发的CT103A临床试验IND已完成美国食品及药物管理局（FDA）审评，获准在美国开展针对复发/难治性多发性骨髓瘤（R/R MM）的临床试验。CT103A是一款靶向BCMA的CAR-T细胞疗法，其CAR结构含有全人源的单链可变片段（scFvs）可使其绕过宿主潜在的抗CAR免疫原性，同时保留抗肿瘤活性。近期热门视频更多精彩视频，尽在佰傲谷视频号，欢迎关注~本周好文推荐如需转载请联系佰傲谷并在醒目位置注明出处﹀ 点亮在看，传递信息♥

FDA granted IND clearance for IVS-3001, Invectys’s lead engineered anti-HLA-G CAR-T cell therapy, for the treatment of patients with solid tumors HOUSTON, Texas, Dec. 20, 2022 (GLOBE NEWSWIRE) -- Invectys, Inc. and CTMC, a joint venture between MD Anderson Cancer Center and National Resilience, Inc., today announced Food and Drug Administration (FDA) clearance of an Investigational New Drug (IND) application for a Phase 1/2a clinical study of IVS-3001, Invectys’s lead engineered human leukocyte antigen A (HLA-G) targeting chimeric antigen receptor (CAR) T cell therapy for the treatment of solid tumors. IVS-3001 has been developed in collaboration with CTMC as part of an agreement announced in 2022, and it will now move forward through an MD Anderson-sponsored trial led by principal investigator (PI) Aung Naing, M.D., professor of Investigational Cancer Therapeutics at MD Anderson and co-PI Samer Srour, M.D., assistant professor of Stem Cell Transplantation and Cellular Therapy at MD Anderson. The planned first-in-human, single-arm, open-label, Phase 1/2a study will evaluate the safety, tolerability, pharmacokinetics, and clinical activity of IVS-3001 in patients with histologically or pathologically confirmed diagnosis of a locally advanced unresectable or metastatic HLA-G+ select solid tumor malignancy who failed or was intolerant to standard of care therapies known to confer clinical benefit per treating physician. IVS-3001 may be administered to up to a total of 117 patients under approved protocol. Up to 24 may be treated in Phase 1 Dose Escalation and up to 93 may be treated in Phase 2a. All participants will be asked to enter the long-term follow up (LTFU) study as per FDA standard requirement for all gene and cell therapies. The IVS-3001 therapy for the Phase I trial will be manufactured by CTMC, which was launched to speed the development and manufacturing of innovative cell therapies for patients with cancer. “CTMC was created to accelerate impactful cell therapies reaching patients," said Jason Bock, CEO of CTMC. “We are excited to partner with Invectys to move the HLA-G CAR-T from contract execution to IND safe-to-proceed, in less than a year.” “The FDA clearance of the IND application for IVS-3001 represents an important milestone for Invectys and our colleagues at CTMC, and it is the result of years of commitment to developing a novel class of engineered CAR-T therapy,” said Praveen Tyle, Ph.D., CEO of Invectys. “We believe IVS-3001 therapy has the potential to significantly transform the treatment landscape for cancer patients and the potential to achieve improved clinical outcomes.” About IVS-3001 IVS-3001 is an HLA-G-targeting chimeric antigen receptor (CAR) T cell therapy. HLA-G is not only an immune checkpoint but also a tumor-specific antigen. The CAR construct consists of the HLA-G antigen-targeting domain fused to intracellular signaling domains. The CAR allows IVS-3001 to recognize and kill target cells which express HLA-G on the cell surface. Antigen-specific activation of IVS-3001 results in CAR-T cell proliferation, cytokine secretion, and subsequent cytolytic killing of HLA-G-expressing cells. IVS-3001 preclinical studies have generated adequate data to support efficacy and safety of the CAR-T therapy leading to IND clearance and initiation of First-in-Human clinical trial. About Invectys Invectys, transforming innovative immunotherapies to eradicate cancer, is a clinical stage immuno-oncology company spun-out of the world-renowned Pasteur Institute in Paris. Invectys has two wholly owned subsidiaries, Invectys, S.A. (Paris) which is focused on scientific research and innovation and Invectys USA, Inc. (Houston), whose lead product is the development of a first-in-human HLAG /CAR T initiative. Since 2010, Invectys has raised over $68 million in private funds to develop two innovative platforms of immunotherapy products which that target “universal” tumor antigens. Invectys has also received a grant of $14.2 million from the Cancer Prevention and Research Institute of Texas (Grant ID DP200034) to help fund it’s HLA-G CAR T program. About CTMC CTMC – a joint venture between MD Anderson Cancer Center and Resilience – was created to accelerate the development and manufacturing of impactful cell therapies for patients with cancer. CTMC is uniquely positioned within the Texas Medical Center to provide therapies that begin and end with the patient. By leveraging the strengths of MD Anderson and Resilience we start clinical trials faster and provide a clear path to robust commercialization. Learn more at . Invectys Inc Media Contact: Rosie Williams Rosie.williams@invectys.com 281-384-6699 CTMC Media Contact: Laura Torgerson ltorgerson@ctmc.com 832-295-8533