WU-NK-101

Data Support Start of First Pivotal Trial for an Off-the-Shelf, Allogeneic CD7-Targeted CAR-T Cell Therapy in Pediatric and Adult PatientsST. LOUIS and SAN DIEGO, Dec. 05, 2024 (GLOBE NEWSWIRE) -- Wugen, Inc., a clinical-stage U.S. biotechnology company developing allogeneic, off-the-shelf cell therapies for the treatment of hematological and solid tumor malignancies, today announced upcoming scientific presentations, including updates from the company’s Phase 1/2 study of WU-CART-007. Researchers will present Phase 1/2 updates and studies for its investigational cell therapies this week at the American Society of Hematology (ASH) Annual Meeting & Exposition, Dec. 7-10 in San Diego. Results from Phase 1/2 showed continued anti-leukemic activity and clinically manageable safety in adults and adolescents. WU-CART-007 is an investigational, potential first-in-class, allogeneic, anti-CD7 CAR-T cell therapy under evaluation for treatment of patients with relapsed or refractory (R/R) T cell acute lymphoblastic leukemia or T cell lymphoblastic lymphoma (T-ALL/LBL). Based on findings to date with WU-CART-007, including this week’s data, Wugen will initiate the first pivotal Phase 2 study for an off-the-shelf CD7-targeted CAR-T cell therapy. “Maturing evidence, including the Phase 1/2 study being presented, suggest WU-CART-007 has the potential to improve current standard of care for patients with these blood cancers. T-ALL/LBL are malignancies associated with high rates of relapse and mortality in patients, many of whom are young,” said Armin Ghobadi, M.D., professor of medicine and clinical director of Center for Gene and Cellular Immunotherapy (CGCI) at the Washington University School of Medicine in St. Louis. “At this year’s ASH meeting, we will present clinical data from adult and adolescent patients that support our soon to open pivotal trial for patients with relapsed or refractory T-ALL/LBL,” said Wugen Chief Medical Officer, Cherry Thomas, M.D. “We are looking forward to working with experienced investigators to provide meaningful clinical benefit to patients with limited treatment options.” The pivotal Phase 2 study is a single-arm trial evaluating the efficacy and safety of WU-CART-007 in patients with R/R T-ALL/LBL and T-ALL/LBL. The study will involve two groups: a R/R cohort and subsequently an exploratory minimal residual disease (MRD)-positive cohort. The trial will enroll pediatric and adult patients at oncology centers in the United States, Europe, Asia and Australia. Wugen Presentations at ASH 3450 WU-CART-007 (WT-7), an Allogeneic CAR T-Cell Targeting CD7 in Relapsed/Refractory (R/R) T-Cell Acute Lymphoblastic Leukemia/Lymphoma (T-ALL/LBL): Phase 2 Results Presenter: Armin Ghobadi, M.D., Washington University School of Medicine, St. Louis Presentation type: PosterTime/location: Sunday, Dec. 8, 6:00-8:00 p.m. PT; Halls G,HSession: 704. Cellular Immunotherapies: Early Phase Clinical Trials and Toxicities: Poster II 3461 WU-CART-007 (WT-7) Is an Effective Treatment for Adolescent Patients with Relapsed or Refractory (R/R) T-Cell Acute Lymphoblastic Leukemia/ Lymphoblastic Lymphoma (T-ALL/LBL) – Subgroup Analysis of WU-CART-007 1001 Presenter: Shannon L. Maude, M.D., Division of Oncology, Center for Childhood Cancer Research and Cancer Immunotherapy Program, Children's Hospital of Philadelphia, Philadelphia Presentation type: PosterTime/location: Sunday, Dec. 8, 6:00-8:00 p.m. PT; Halls G,HSession: 704. Cellular Immunotherapies: Early Phase Clinical Trials and Toxicities: Poster II Wugen researchers will also present the following abstracts for the company’s investigational memory natural killer cell therapy, WU-NK-101: 916 W-NK1 Choreographs Innate and Adaptive Immune Responses to Provide a Robust and Durable Anti-AML Response Presenter: Tom Leedom, Wugen, Inc., St. Louis Presentation type: Oral Time/location: Monday, Dec. 9, 3:30 p.m. PT; Marriott Grand Ballroom 8-9, Marriott Marquis San Diego MarinaSession: 703. Cellular Immunotherapies other than CAR-T Cells: Basic and Translational: Enhancing NK Cell Therapeutics Hematology Disease Topics & Pathways: Research, Translational Research 2:45-4:15 p.m. PT 4257 WUN101-01: First in Human (FIH) Phase 1 Study of WU-NK-101 (W-NK1) in Patients with Relapsed or Refractory (R/R) Acute Myeloid Leukemia (AML) Presenter: Amanda F. Cashen, M.D., Washington University School of Medicine, St. Louis Presentation type: PosterTime/location: Monday, Dec. 9, 6:00-8:00 p.m. PT; Halls G, HSession: 616. Acute Myeloid Leukemias: Investigational Drug and Cellular Therapies: Poster IIIHematology Disease Topics & Pathways: Research, Clinical trials, Translational Research, Clinical Research Investigator-initiated research 2066 Phase 1 Dose-Escalation and Dose-Expansion Study to Evaluate the Safety and Tolerability of Anti-CD7 Allogeneic CAR T-Cells (WU-CART-007) in Patients with CD7+ T-Cell Non-Hodgkin Lymphoma Presenter: Michael H. Kramer, M.D., Ph.D., Washington University School of Medicine, Saint Louis Presentation type: PosterTime/location: Sat., Dec. 7, 5:30-7:30 p.m. PT; Halls G, HSession: 704. Cellular Immunotherapies: Early Phase Clinical Trials and Toxicities: Poster I; About WU-CART-007 WU-CART-007 is an allogeneic, off-the-shelf, fratricide-resistant CD7-targeted CAR-T cell therapy engineered to overcome the technological challenges of harnessing CAR-T cells to treat CD7+ hematological malignancies. Wugen is deploying CRISPR/Cas9 gene editing technology to delete CD7 and the T cell receptor alpha constant (TRAC), preventing CAR-T cell fratricide and mitigating the risk of graft-versus-host-disease (GvHD). WU-CART-007 is manufactured using healthy donor-derived T cells to eliminate the risk of malignant cell contamination historically observed in the autologous CAR-T setting. WU-CART-007 is currently being evaluated in a global Phase 1/2 clinical trial for the treatment of relapsed or refractory (R/R) T cell acute lymphoblastic leukemia (T-ALL)/lymphoblastic lymphoma (LBL). More information on the Phase 1/2 trial is available on clinicaltrials.gov, identifier NCT# 04984356 and on the Phase 2 pivotal trial on clinicaltrials.gov, identifier NCT06514794. WU-CART-007 has received Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug, and Rare Pediatric Disease designations from the U.S. Food and Drug Administration and Priority Medicines (PRIME) Scheme designation in the European Union for the treatment of relapsed/refractory (R/R) T cell acute lymphoblastic leukemia (T-ALL) and T cell lymphoblastic lymphoma (T-LBL). RMAT and PRIME designations provide increased agency support to expedite the development and review of promising therapies for patients in need. About Wugen Wugen, Inc. is a clinical-stage U.S. biotechnology company developing the next generation of off-the-shelf CAR-T and memory natural killer (NK) cell therapies for cancer. For more information, please visit www.wugen.com and follow Wugen’s LinkedIn page. Investor Contact:Mark LewisWugenMlewis@wugen.com314-501-1968 Media Contact:Christine FanelleScient PRchristine@scientpr.com215-595-5211

新 闻 速 览  第66届美国血液学年会（ASH）将于2024年12月7日~10日在美国圣地亚哥召开。ASH作为全球血液学领域规模最大、涵盖最全面的国际学术盛会之一，每年吸引着成千上万名来自世界各地的专家学者，分享最前沿的血液学进展和突破性临床数据。根据ASH最新公布的摘要，Wugen公司有5项研究入选Oral Presentation，将以口头汇报形式报告通用现货型CD7 CAR-T（WU-CART-007)和NK细胞（WU-NK-101）疗法的最新数据。 CD7 CAR-T报告详情如下： 2066 Phase 1 Dose-Escalation and Dose-Expansion Study to Evaluate the Safety and Tolerability of Anti-CD7 Allogeneic CAR T-Cells (WU-CART-007) in Patients with CD7+ T-Cell Non-Hodgkin Lymphoma Program: Oral and Poster Abstracts Session: 704. Cellular Immunotherapies: Early Phase Clinical Trials and Toxicities: Poster I 3450 WU-CART-007 (WT-7), an Allogeneic CAR T-Cell Targeting CD7 in Relapsed/Refractory (R/R) T-Cell Acute Lymphoblastic Leukemia/Lymphoma (T-ALL/LBL): Phase 2 Results Program: Oral and Poster Abstracts Session: 704. Cellular Immunotherapies: Early Phase Clinical Trials and Toxicities: Poster II 3461 WU-CART-007 (WT-7) Is an Effective Treatment for Adolescent Patients with Relapsed or Refractory (R/R) T-Cell Acute Lymphoblastic Leukemia/ Lymphoblastic Lymphoma (T-ALL/LBL) Subgroup Analysis of WU-CART-007 1001 Program: Oral and Poster Abstracts Session: 704. Cellular Immunotherapies: Early Phase Clinical Trials and Toxicities: Poster II WU-CART-007已获FDA授予的孤儿药、快速通道和罕见儿科疾病认定、再生医学先进疗法认定；EMA优先审评药物认定。此次会议上，Wugen将报告通用现货型CD7 CAR-T疗法的最新临床数据,主要亮点包括： CD7 CAR-T治疗（R/R）T-ALL/LBL II期阶段试验结果： 截至2024年7月24日，共13名患者接受了RP2D剂量治疗表现出可控的安全性以及抗白血病活性。 可评估患者中，ORR和CRc分别为91%（10/11）和73%（8/11；6 CR，2 CRi）。中位DOR尚未达到（95% CI: 0.5，NE；范围0.5-9.1个月）；4名患者目前仍持续CR，时间分别为9.1、8.2、6.7和6.7个月。 MRD阴性CR/CRi患者（n=7）的中位DOR为6.6个月（95% CI：1.8，NE），而MRD阳性CR/CRi患者（n=3）为3.7个月（95% CI: 0.5，NE）。MRD阴性患者总生存期（OS）为11.5个月（95% CI：2.7，NE），MRD阳性患者为6.1个月（95% CI：1.4，NE）。 12-18青少年患者亚组分析中，可评估的患者（4/4）CR/CRi率为75%，1例患者达到PR（CD7阴性残留EMD），ORR为100%。CRc患者中，2/3未检测到MRD。达到CR/CRi的3名患者均转为HSCT，其中两名患者有移植史，所有患者在随访9、8.1和6.1个月时仍处于完全缓解状态。 后续关键II期研究NCT06514794计划于2024年末开始招募，包括1岁及以上的儿童患者；在R/R患者队列的安全性得到确认后，可能会启动探索性的MRD阳性队列研究。 CD7 CAR-T治疗CD7+ T-NHL I期剂量递增和剂量扩展研究：： 5名T-NHL患者接受WU-CART-007治疗，无患者出现免疫效应细胞相关神经毒性综合征（ICANS）或GvHD，表现出可控的安全性。 疗效方面：2例完全缓解（CR）；2例部分缓解（PR）；1例病情稳定（SD），显示出早期疗效迹象。本研究（NCT05377827）的招募仍在进行，以进一步确定其在CD7阳性T-NHL和AML患者中的安全性和初步疗效。 NK细胞疗法报告详情如下： 916 W-NK1 Choreographs Innate and Adaptive Immune Responses to Provide a Robust and Durable Anti-AML Response Program: Oral and Poster Abstracts Type: Oral Session: 703. Cellular Immunotherapies other than CAR-T Cells: Basic and Translational: Enhancing NK Cell Therapeutics 4257 WUN101-01: First in Human Human (FIH) Phase 1 Study of WU-NK-101 (W-NK1) in Patients with Relapsed or Refractory (R/R) Acute Myeloid Leukemia (AML) Program: Oral and Poster Abstracts Session: 616. Acute Myeloid Leukemias: Investigational Drug and Cellular Therapies: Poster III WU-NK-101已获得美国食品药品监督管理局（FDA）授予的用于治疗AML的孤儿药认证。此次会议上，Wugen将报告通用现货型NK细胞疗法的最新数据,主要亮点包括： WU-NK-101协调固有和适应性免疫应答，提供强效且持久的抗AML反应： 与供体cNK细胞相比，W-NK1在NKG2A、2B4、DNAM-1以及免疫参与受体的表达上更高；W-NK1与未成熟DC共培养可增加DC的激活标志物表达；分泌的趋化因子在跨膜实验中能有效招募T细胞；W-NK1显著增强T细胞激活和增殖（P<0.01）；促肿瘤M2型巨噬细胞表现出选择性细胞毒性，显示其倾向于改善TME的免疫抑制状态。这些结果同样通过AML异种移植NCG小鼠模型（THP-1和HL-60）体内得到验证。 WU-NK-101在复发或难治性（R/R）急性髓性白血病（AML）患者中的I期研究： 入组9名患者（每个剂量水平3名），表现出可管理的安全性。未报告免ICANS或GvHD；未观察到无疾病情况下的持续性全血细胞减少，也未报告剂量限制性毒性（DLT）。 在DL1水平未观察到缓解，但所有患者外周血母细胞均短暂减少；在DL2及以上水平，除1例外，所有患者骨髓母细胞均减少， ORR为50%（包括2例CRi和1例PR）。 摘要原文链接： 1.    https://ash.confex.com/ash/2024/webprogram/Paper201520.html 2.    https://ash.confex.com/ash/2024/webprogram/Paper201812.html 3.    https://ash.confex.com/ash/2024/webprogram/Paper202005.html 4.    https://ash.confex.com/ash/2024/webprogram/Paper201998.html 5.    https://ash.confex.com/ash/2024/webprogram/Paper200809.html 关于WU-CART-007 WU-CART-007是一种通用现货型CD7靶向CAR-T细胞疗法，旨在克服利用CAR-T细胞治疗CD7+血液系统恶性肿瘤的技术挑战。Wugen利用CRISPR/Cas9基因编辑技术，删除CD7和T细胞受体恒定区（TRAC），防止CAR-T细胞自相残杀，降低移植物抗宿主病（GvHD）的风险。WU-CART-007使用健康供者来源的T细胞制备，消除在自体CAR-T环境中观察到的恶性细胞污染风险。Wugen已与合作伙伴共同开发出稳定且高效扩增的CD7 CAR-T商业化生产工艺，已完成首个I/II 期爬坡及扩展队列研究（NCT04984356）,是目前全球临床进度最快的同类通用现货型细胞产品。 关于WU-NK-101 WU-NK-101是一种新型的免疫疗法，利用记忆自然杀伤细胞（NK）治疗血液和实体肿瘤。记忆NK细胞是一种功能性强、持久力高的免疫细胞，具备增强的抗肿瘤活性和细胞因子诱导的记忆样（CIML）表型。这种罕见的细胞群具有优越的表型、增殖能力和代谢适应性，与其他NK细胞疗法相比更适合用于癌症治疗。Wugen正在应用其专有的MonetaTM平台，将WU-NK-101开发为一种商业规模化、现货型的癌症细胞疗法。 关于上海晨泰森豪医药科技有限公司 晨泰森豪成立于2020年，是一家致力于开发临床急需的通用型细胞治疗药物的创新医疗公司。2021年1月与Wugen达成独家许可和合作协议，共同开发通用现货型记忆NK细胞和CAR-T细胞。用于治疗晚期血液瘤和实体瘤，并积极开发神经退行性疾病和自身免疫疾病的应用。晨泰森豪拥有经验丰富的医学、临床运营、药学开发、注册事务和商业化团队，通过整合肿瘤领域的专业知识，不断寻找创新解决方案，从而改善肿瘤患者的生活。 关于Wugen Inc. Wugen是一家临床阶段的生物技术公司，致力于开发下一代用于治疗癌症的“现货型”免疫记忆自然杀伤 (NK) 细胞疗法和CAR-T细胞疗法。Wugen利用其专有的技术平台和深厚的基因组工程专业技术来开创一种新型的免疫记忆NK细胞和CAR-T细胞疗法，以治疗血液和实体恶性肿瘤。 文章封面来源：Freepik