Anti-CD 19 CAR-T Cell Therapy in Patients with ANCA Vasculitis: a Two-stage Interventional, Prospective, Open-label, Phase I/II Trial in Patients with Active, Treatment Refractory, ANCA-IgG-positive Vasculitis

The goal of this phase I/II clinical trial is to investigate anti-CD 19 chimeric antigen receptor T cell (CAR-T cell) therapy in patients with antineutrophil cytoplasmic antibodies (ANCA) immunoglobulin (IgG) positive ANCA associated vasculitis (AAV).
The main questions it aims to answer are:
* To assess the safety of anti-CD19 CAR-T cell therapy in subjects with active, treatment refractory, ANCA-IgG-positive AAV
* To assess the clinical efficacy of anti-CD19 CAR-T cell therapy in subjects with active, treatment refractory ANCA-IgG-positive AAV
* To assess the ANCA seroconversion rate in subjects with active, treatment refractory ANCA-IgG-positive AAV
Participants will receive a single dose of KYV-101 i.v., an autologous fully-human anti-CD19 CAR-T cell immunotherapy. Follow-up time is 52 weeks with regular visits at the site.

开始日期2025-01-01

申办/合作机构

Kyverna Therapeutics, Inc.初创企业

NCT06475495 / Not yet recruiting临床1/2期IIT

Comparison of B-cell Depletion by Rituximab and Anti-CD 19 CAR-T Therapy in Patients With Rheumatoid Arthritis. Two-stage Interventional, Prospective, Randomized, Controlled, Open Label, Parallel-group Phase I/II Trial in Patients With Active, ACPA-positive and Treatment Refractory Rheumatoid Arthritis

The goal of this phase I/II clinical trial is to compare B-cell depletion by rituximab and anti-CD 19 CAR-T therapy in patients with rheumatoid arthritis. The main questions it aims to answer are:
* To assess the safety of anti-CD19 CAR T cell therapy in subjects with active, ACPA positive and treatment refractory RA (Phase-I)
* To assess the safety of anti-CD19 CAR T cell therapy and of rituximab in subjects with active, ACPA positive and treatment refractory RA (Phase-II)
* To assess ACPA seroconversion after anti-CD19 CAR T cell or rituximab therapy in subjects with active, ACPA positive and treatment refractory RA (Phase-II)
Participants in the test-arm will receive a single dose of KYV-101 i.v., an autologous fully-human anti-CD19 CAR T-cell immunotherapy. In the comparator group patients will receive 2x1 g Rituximab i.v.
Follow-up time (both arms) is 52 weeks with regular visits at the site.

开始日期2024-11-01

申办/合作机构

Kyverna Therapeutics, Inc.初创企业

NCT06384976 / Recruiting临床2期

KYSA-7: A Phase 2, Open-Label, Randomized, Multicenter Study of KYV-101, an Autologous Fully Human Anti-CD19 Chimeric Antigen Receptor T-Cell (CD19 CAR T) Therapy, in Subjects With Refractory Primary and Secondary Progressive Multiple Sclerosis

A Study of Anti-CD19 Chimeric Antigen Receptor T-Cell (CD19 CAR T) Therapy, in Subjects with Refractory Primary and Secondary Progressive Multiple Sclerosis

开始日期2024-09-01

申办/合作机构

Kyverna Therapeutics, Inc.初创企业

100 项与 KYV-101 相关的临床结果

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100 项与 KYV-101 相关的转化医学

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100 项与 KYV-101 相关的专利（医药）

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项与 KYV-101 相关的文献（医药）

2024-06-25·PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Successful use of anti-CD19 CAR T cells in severe treatment-refractory stiff-person syndrome

Article

作者: Borie, Dominic ; Gold, Ralf ; Schroers, Roland ; Geis, Christian ; Haghikia, Aiden ; Sgodzai, Melissa ; Mougiakakos, Dimitrios ; Faissner, Simon ; Motte, Jeremias

Treatment with autologous chimeric antigen receptor (CAR) T cells has emerged as a highly effective approach in neuroimmunological disorders such as myasthenia gravis. We report a case of successful anti-CD19 CAR T cell use in treatment-refractory stiff-person syndrome (SPS). To investigate clinical and immunological effects of anti-CD19 CAR T cell use in treatment-refractory SPS, a 69-y-old female with a 9-y history of treatment-refractory SPS with deteriorating episodes of stiffness received an infusion of autologous anti-CD19 CAR T cells (KYV-101) and was monitored clinically and immunologically for more than 6 mo. CAR T cell infusion resulted in reduced leg stiffness, drastic improvement in gait, walking speed increase over 100%, and daily walking distance improvement from less than 50 m to over 6 km within 3 mo. GABAergic medication (benzodiazepines) was reduced by 40%. KYV-101 CAR T cells were well tolerated with only low-grade cytokine release syndrome. This report of successful use of anti-CD19 CAR T cells in treatment-refractory SPS supports continued exploration of this approach in SPS and other B cell–related autoimmune disorders.

2024-06-01·Transplantation and cellular therapy

Fully Human Anti-CD19 CAR T Cells Derived from Systemic Lupus Erythematosus Patients Exhibit Cytotoxicity with Reduced Inflammatory Cytokine Production

Article

作者: Aigner, Michael ; Park, Soo ; Taubmann, Jule ; Lutzny-Geier, Gloria ; Mackensen, Andreas ; Dingfelder, Janin ; Schett, Georg ; Minopoulou, Ioanna ; Cheng, Joseph K ; Kaplan, Charles D ; Van Blarcom, Tom

KYV-101 is an autologous anti-CD19 chimeric antigen receptor (CAR)-T cell therapy under investigation for patients with B-cell driven autoimmune diseases. Hu19-CD828Z is a fully human anti-CD19 CAR designed and demonstrated to have a favorable clinical safety profile. Since anti-CD19 CAR T cells target and kill B cells in both circulation and tissues, the treatment with Hu19-CD828Z CAR T cells offers great potential in depleting autoreactive B cells. Demonstrate that Hu19-CD828Z CAR T cells manufactured from cryopreserved leukaphereses from patients with systemic lupus erythematosus (SLE) exhibit CAR-mediated and CD19-dependent cytokine release, proliferation and cytotoxicity when co-cultured with autologous primary B cells. T cells were enriched from cryopreserved leukaphereses from SLE patients or healthy donors (HD). CAR T cells were generated by transducing these cells with a lentiviral vector encoding Hu19-CD828Z. CAR-mediated and CD19-dependent activity was monitored in vitro in a set of cytotoxicity, cytokine release, and proliferation studies, in response to autologous primary CD19⁺ B cells, a CD19⁺ cell line (NALM-6), or a CD19^- cell line (U937). Hu19-CD828Z CAR T cells produced from SLE patients or HD induced greater proliferation and dose-dependent cytotoxicity against both autologous primary B cells and the CD19⁺ NALM-6 cells than nontransduced control T cells or co-cultures with a CD19^- cell line. Interestingly, there was lower inflammatory cytokine production from SLE patient-derived CAR T cells compared to HD donor-derived CAR T cells with either CD19⁺ cells or primary B cells. Hu19-CD828Z CAR T cells generated from SLE patient lymphocytes demonstrate CAR-mediated and CD19-dependent activity against autologous primary B cells with reduced inflammatory cytokine production supporting KYV-101 as a novel potential therapy for the depletion of pathogenic B cells in SLE patients.

2024-06-01·Med (New York, N.Y.)

CD19-targeted chimeric antigen receptor T cell therapy in two patients with multiple sclerosis

Article

作者: Kuhle, Jens ; Berger, Susanna Carolina ; Rathje, Kristin ; Fischbach, Felix ; Fehse, Boris ; Seibel, Johan ; Badbaran, Anita ; Friese, Manuel A ; Pfeffer, Lena Kristina ; Ayuk, Francis ; Richter, Johanna ; Heesen, Christoph ; Kröger, Nicolaus ; Borie, Dominic ; Reinhardt, Stefanie ; Gagelmann, Nico

BACKGROUND:

Progressive multiple sclerosis (MS) is characterized by compartmentalized smoldering neuroinflammation caused by the proliferation of immune cells residing in the central nervous system (CNS), including B cells. Although inflammatory activity can be prevented by immunomodulatory therapies during early disease, such therapies typically fail to halt disease progression. CD19 chimeric antigen receptor (CAR)-T cell therapies have revolutionized the field of hematologic malignancies. Although generally considered efficacious, serious adverse events associated with CAR-T cell therapies such as immune effector cell-associated neurotoxicity syndrome (ICANS) have been observed. Successful use of CD19 CAR-T cells in rheumatic diseases like systemic lupus erythematosus and neuroimmunological diseases like myasthenia gravis have recently been observed, suggesting possible application in other autoimmune diseases.

METHODS:

Here, we report the first individual treatment with a fully human CD19 CAR-T cell therapy (KYV-101) in two patients with progressive MS.

FINDINGS:

CD19 CAR-T cell administration resulted in acceptable safety profiles for both patients. No ICANS was observed despite detection of CD19 CAR-T cells in the cerebrospinal fluid. In case 1, intrathecal antibody production in the cerebrospinal fluid decreased notably after CAR-T cell infusion and was sustained through day 64.

CONCLUSIONS:

CD19 CAR-T cell administration in progressive MS resulted in an acceptable safety profile. CAR-T cell presence and expansion were observed in the cerebrospinal fluid without clinical signs of neurotoxicity, which, along with intrathecal antibody reduction, indicates expansion-dependent effects of CAR-T cells on CD19⁺ target cells in the CNS. Larger clinical studies assessing CD19 CAR-T cells in MS are warranted.

FUNDING:

Both individual treatments as well the generated data were not based on external funding.

116

项与 KYV-101 相关的新闻（医药）

2024-09-21

·药明康德

让75%患者摆脱胰岛素依赖的细胞疗法；实体瘤CAR-T治疗领域迎来重要进展…… | CGT周报

▎药明康德内容团队编辑近期，全球细胞和基因疗法（CGT）领域迎来系列进展。Vertex Pharmaceuticals公司公布了其在研细胞疗法VX-880的积极临床试验数据，75%的1型糖尿病患者在治疗180天及之后不再使用外源性胰岛素，实现糖尿病“功能性治愈”。用于治疗实体肿瘤的嵌合抗原受体-T（CAR-T）细胞疗法领域迎来重要进展，GCC19CART疗法在经过大量先前治疗的转移性结直肠癌（mCRC）患者中实现了57%的客观缓解率（ORR），患者的中位总生存期（OS）达22.8个月。本文将节选其中部分重要进展做简单介绍，仅供读者参阅。图片来源：123RF ———✦研发进展✦——— ◇ Vertex Pharmaceuticals公司公布了其在研细胞疗法VX-880治疗1型糖尿病的最新临床试验结果。截至2024年5月，共有14例患者接受了VX-880治疗，其中12例患者接受了完整剂量的VX-880单次输注，2例患者接受了一半剂量的VX-880。研究结果显示，单次输注VX-880可显著改善患者的血糖水平且不出现严重低血糖事件，患者有望摆脱对外源胰岛素的依赖，实现糖尿病“功能性治愈”。92%（11/12）患者使用外源性胰岛素的频率大幅减少，75%（9/12）患者在治疗180天及之后不再使用外源性胰岛素。4例随访超过1年的患者达到了研究主要终点，即未发生严重低血糖事件、糖化血红蛋白（HbA1c）水平＜7个百分点，以及不再依赖外源性胰岛素治疗。 VX-880是一种在研干细胞来源、完全分化、产生胰岛素的同种异体胰岛细胞疗法。VX-880有可能通过恢复胰岛细胞功能，包括葡萄糖反应性胰岛素产生，恢复机体调节葡萄糖水平的能力。VX-880通过肝门静脉输注给药，需要长期免疫抑制治疗以保护输入的胰岛细胞免受免疫排斥。图片来源：123RF ◇ Kyverna Therapeutics公司分享了其CAR-T细胞疗法KYV-101正在进行的僵人综合征、重症肌无力和多发性硬化临床试验的病例报告。其中，两名接受KYV-101治疗的僵人综合征患者表现出行动能力和/或行走速度的改善，并且自身抗体滴度降低，其中一名患者在接受治疗一年后仍表现出改善。3名接受KYV-101治疗的重症肌无力患者显示出致病性抗乙酰胆碱受体（anti-AChR）抗体滴度的降低，且病情得到持续的控制，3名患者维持疾病控制超8个月，两名患者维持疾病控制超1年。5名此前接受抗CD20药物治疗失败的多发性硬化患者接受KYV-101治疗后，中枢神经系统中的寡克隆区带数量显著减少，这是多发性硬化疾病进展减缓的潜在替代生物标志物。安全性方面，迄今已有41名自身免疫性疾病患者接受了KYV-101治疗，未观察到严重的细胞因子释放综合征（CRS）或免疫效应细胞相关神经毒性综合征（ICANS）。 KYV-101是一款自体CD19靶向CAR-T细胞疗法。KYV-101中的CAR由美国国立卫生研究院（NIH）设计，旨在提高耐受性。迄今为止，KYV-101在肿瘤和自身免疫性疾病中的临床经验突显了其独特的特性及其为自身免疫性疾病患者提供治疗的潜力。 ◇ BOOST Pharma公司公布了其突破性细胞疗法治疗罕见骨病成骨不全症（OI）的1/2期临床研究的积极顶线结果。该研究中的17名患者确诊为严重的III型和IV型OI，并在卡罗林斯卡大学医院接受了为期12个月的治疗。结果显示，该治疗方案在患儿出生前和出生后给药时均安全且耐受性良好。最后一次给药后的12个月内，患儿的骨折率减少了超过75%。该疗法基于一种使用人类干细胞的全新细胞疗法，具有很高的成骨能力。这种治疗方法旨在确诊后立即进行干预，无论是在出生前还是出生后，这为生命早期（儿童活动增多的阶段）提供了治疗优势。对于OI患儿来说，预防或减少骨折次数是最重要的治疗目标，但目前尚无获批的治疗方法可以实现这一目标。图片来源：123RF ◇ Beacon Therapeutics公司公布了其主打项目基因疗法AGTC-501用以治疗X连锁视网膜色素变性（XLRP）患者的1/2期临床试验的中期结果。结果显示，在纳入研究的29名患者中，AGTC-501总体上安全且耐受性良好，未观察到与该疗法相关的临床显著安全事件。数据还显示，治疗眼与未治疗眼之间的视觉功能差异在36个月时仍然存在。AGTC-501的效益-风险比支持其在由RPGR基因突变引起的XLRP患者中的持续临床开发。 ◇ AAVantgarde Bio公司宣布已在LUCE-1试验中为首位受试者进行了给药。LUCE-1是一项1/2期开放标签临床试验，旨在评估以腺相关病毒8（AAV8）为载体的视网膜内基因疗法AAVB-081用于治疗Usher综合征1B型（USH1B）相关的视网膜色素变性患者的安全性和耐受性。AAVB-081使用该公司的专有的双杂交基因治疗平台，向USH1B相关的视网膜色素变性患者递送MYO7A蛋白。AAVantgarde公司的双杂交平台使用两个AAV8载体，每个载体都携带目标基因的一半信息，两个基因片段在细胞核内重新组合为一个完整的基因，从而能够生成治疗水平的蛋白质。 ◇ 斯丹赛生物（Innovative Cellular Therapeutics）宣布其自主研发的CAR-T细胞疗法GCC19CART在转移性结直肠癌患者中的临床研究成果已发表在JAMA Oncology杂志。结果显示，在2X106 CAR-T细胞/kg的剂量下，GCC19CAR治疗经过大量先前治疗的mCRC患者的ORR为57%，中位总生存期（OS）为22.8个月。新闻稿指出，这项研究在CAR-T细胞疗法用于实体肿瘤（特别是mCRC）的治疗方面具有重要的里程碑意义。斯丹赛还公布了其在美国正在进行的1期临床试验的数据。接受1X106 CAR-T细胞/kg剂量的4例患者中有2例达到部分缓解（PR），ORR为50%。接受2X106 CAR-T细胞/kg剂量的5例患者中有4例达到PR，ORR为80%。 ———✦融资、合作、M&A✦——— 图片来源：123RF ◇ GC Therapeutics公司宣布正式成立，将致力于推进并解锁下一代细胞疗法。该公司的诱导多能干细胞（iPSC）编程平台TFome（转录因子组）由哈佛医学院和Wyss研究所的教授George Church博士的实验室开发，汇集了合成生物学、基因编辑、细胞工程和机器学习的最新进展，以克服与细胞疗法相关的开发和规模化方面的复杂性，并提高患者在多种疾病领域的治疗可及性。TFome平台旨在通过一步式工艺以比传统方法快100倍的速度提供基于iPSC的现货型细胞疗法，以提高效力、效率、质量并降低成本，从而彻底改变细胞疗法。该公司最初的管线将专注于胃肠道、神经和免疫疾病。 GC Therapeutics已完成6500万美元的A轮融资，由Cormorant Asset Management领投，Mubadala Capital、Andreessen Horowitz （a16z） Bio + Health、Medical Excellence Capital、Cercano Management和Pear VC参投，使该公司的总筹资金额达到7500万美元。 ◇ 贝斯生物宣布完成数千万人民币的A2轮融资，由戈壁大湾区（Gobi Partners GBA）管理的AEF大湾区创业基金领投。本轮融资将用于加强贝斯生物的研发团队、优化产品管线、加速临床试验，并为未来的商业化奠定坚实的基础。该公司致力于开发创新的细胞疗法和基因编辑产品，用于治疗癌症和遗传疾病。今年7月，贝斯生物首款产品NK510——一款基于碱基编辑技术的自然杀伤细胞（NK）产品已经向中国国家药品监督管理局（NMPA）申报IND并获得受理。除了NK510之外，贝斯生物的产品管线还包括多个针对血液恶性肿瘤和自身免疫性疾病的碱基编辑NK细胞产品，以及通用CAR免疫疗法和体内基因编辑产品。 ◇ LifeSpan Vision Ventures宣布完成对Remedium Bio的投资。Remedium Bio是一家新型基因疗法开发公司。该公司正在开发一种可调剂量基因治疗平台，可以用单次注射、剂量可调的基因治疗取代广泛的多蛋白注射治疗。这种通过皮下注射进行的治疗更具安全、局部、持久优势，且成本低于许多蛋白质疗法。该轮融资将用于推动该公司主要候选产品RMD-1202进入1期研究。RMD-1202是GLP-1受体激动剂，其减肥、血糖控制和胰岛素反应优于每日GLP-1蛋白注射。 ▲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] T Reichman, P Witkowski, J Markmann, et al. LBA 31Improvement in glycaemic control and elimination of exogenous insulin use in patients with type 1 diabetes infused with fully differentiated islet cells (VX-880). https://cattendee.abstractsonline.com/meeting/20620/session/660 [2] Kyverna Therapeutics Presents Patient Data Reinforcing Potential of KYV-101 for Treatment of Neuroinflammatory Diseases in Symposium at ECTRIMS 2024. Retrieved September 20, 2024, from https://www.prnewswire.com/news-releases/kyverna-therapeutics-presents-patient-data-reinforcing-potential-of-kyv-101-for-treatment-of-neuroinflammatory-diseases-in-symposium-at-ectrims-2024-302251529.html [3] Karolinska Developments portfolio company BOOST Pharma announces positive top-line results from a Phase 1/2 study with over 75% reduction of fracture rates in children with rare bone disease. Retrieved September 20, 2024, from https://www.globenewswire.com/news-release/2024/09/17/2947159/0/en/Karolinska-Developments-portfolio-company-BOOST-Pharma-announces-positive-top-line-results-from-a-Phase-1-2-study-with-over-75-reduction-of-fracture-rates-in-children-with-rare-bon.html [4] Beacon Therapeutics Presents 36-Month Interim Results from Phase I/2 HORIZON Trial of AGTC-501 in Patients with XLRP. Retrieved September 20, 2024, from https://www.prnewswire.com/news-releases/beacon-therapeutics-presents-36-month-interim-results-from-phase-i2-horizon-trial-of-agtc-501-in-patients-with-xlrp-302252375.html [5] AAVantgarde announces first patient dosed in First-In-Human Phase 1/2 LUCE-1 study, evaluating AAVB-081 (Dual-AAV) in retinitis pigmentosa related to Usher Syndrome type 1B. Retrieved September 20, 2024, from https://www.globenewswire.com/news-release/2024/09/16/2946331/0/en/AAVantgarde-announces-first-patient-dosed-in-First-In-Human-Phase-1-2-LUCE-1-study-evaluating-AAVB-081-Dual-AAV-in-retinitis-pigmentosa-related-to-Usher-Syndrome-type-1B.html [6] Innovative Cellular Therapeutics Announces the Publication of Groundbreaking GCC19CART™ Study to Treat mCRC in JAMA Oncology. Retrieved September 20, 2024, from https://www.globenewswire.com/news-release/2024/09/19/2949259/0/en/Innovative-Cellular-Therapeutics-Announces-the-Publication-of-Groundbreaking-GCC19CART-Study-to-Treat-mCRC-in-JAMA-Oncology.html [7] GC Therapeutics Launches to Scale and Unlock the Next Generation of Cell Therapy. Retrieved September 20, 2024, from https://www.businesswire.com/news/home/20240918923504/en/GC-Therapeutics-Launches-to-Scale-and-Unlock-the-Next-Generation-of-Cell-Therapy [8] LifeSpan Vision Ventures Invests in Remedium Bio. Retrieved September 20, 2024, from https://www.prnewswire.com/news-releases/lifespan-vision-ventures-invests-in-remedium-bio-302243756.html 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

细胞疗法临床结果基因疗法免疫疗法

2024-09-18

·PRNewswire

Kyverna Therapeutics Presents Patient Data Reinforcing Potential of KYV-101 for Treatment of Neuroinflammatory Diseases in Symposium at ECTRIMS 2024

Company symposium to highlight case reports from 11 patients with stiff-person syndrome, myasthenia gravis and multiple sclerosis reinforcing KYV-101's initial efficacy and safety profile and broad potential in B cell-driven neuroinflammatory diseases Key biomarkers indicate potential for KYV-101 to durably modify neuroinflammatory diseases with immune system reset Kyverna to present posters on design and methods for company-sponsored clinical trials in neuroinflammatory diseases EMERYVILLE, Calif., Sept. 18, 2024 /PRNewswire/ -- Kyverna Therapeutics, Inc. (Kyverna), a patient-centered, clinical-stage biopharmaceutical company focused on developing cell therapies for patients suffering from autoimmune diseases, today announced the presentation of three posters outlining the design and methods of Kyverna's clinical trials evaluating KYV-101 in neuroinflammatory diseases at the 40th Congress of the European Committee for Treatment and Research in Multiple Sclerosis (ECTRIMS) in Copenhagen, Denmark, taking place September 18-20, 2024. Also today, Kyverna shared case reports from its ongoing clinical trials in stiff-person syndrome, myasthenia gravis and multiple sclerosis in a company-sponsored symposium. These case reports reinforce emerging clinical data from KYV-101, with individual patient vignettes continuing to demonstrate disease modification across trials and durability in some patients extending beyond one year and a well-tolerated safety profile. "The new data contribute to redefine treatment efficacy and support the safety profile of KYV-101," said Ralf Gold, M.D., professor of Medicine, chair of Neurology at Ruhr University Bochum, St. Josef Hospital, in Germany, and one of the presenters at the company's symposium. "By rapidly adding new data across a wide spectrum of severe neuroinflammatory diseases, we can hope to accelerate the development of potentially life-changing therapies." "We are very proud to lead the field in understanding the impact of deep B-cell reset towards a potential paradigm shift in the treatment of neuroinflammatory diseases," said Warner Biddle, Chief Executive Officer at Kyverna. "As we continue to build on KYV-101's leading position in stiff-person syndrome and myasthenia gravis with these results, we are additionally excited by promising case reports in multiple sclerosis, which represents a significant population with high unmet need. Altogether, these results continue to reinforce the broad potential of KYV-101 to durably modify debilitating symptoms of severe neuroinflammatory diseases, and generate insights that will allow us to bring transformative CAR T treatments to even more patients." Across numerous company- and investigator-sponsored studies, KYV-101 is currently being evaluated in a range of B cell-driven autoimmune diseases. Kyverna is currently conducting three ongoing company-sponsored Phase 2 trials in neuroinflammatory diseases: KYSA-6 in myasthenia gravis, KYSA-7 in multiple sclerosis and KYSA-8 in stiff-person syndrome. At a company symposium titled, "KYV-101 Unlocking the Potential of CAR T-cell Therapy in Neuroinflammatory Diseases," to be held at 5:15 pm CET on September 18, 2024, Kyverna and investigators will highlight case report experience from 11 patients from these studies: In stiff-person syndrome, two patients treated with KYV-101 have demonstrated improved mobility and/or walking speed and reduced autoantibody titers, including one patient at greater than one year post-infusion. A third patient demonstrated reduced autoantibody titers but does not yet have mobility scores available. In myasthenia gravis, three patients treated with KYV-101 have shown reduced pathogenic anti-AChR antibody titers and sustained disease control, including two patients at more than one year post-infusion and all three at greater than 8 months post-infusion. In multiple sclerosis, five patients treated with KYV-101 who failed prior anti-CD20 medications have shown a significant and unprecedented average reduction in oligoclonal bands in the central nervous system (CNS), a potential surrogate biomarker for reduced disease progression. Across 41 autoimmune disease patients treated to date with KYV-101, no severe CRS or ICANS has been observed. In addition, the Company will present three posters featuring additional detail on the design and methods used in Kyverna's sponsored clinical trials of KYV-101: "Activity and manufacturing of KYV-101 anti-CD19 chimeric antigen receptor T cells derived from patients with neurologic autoimmune diseases" "Design of KYSA-6, a phase 2, open-label, multicenter study of KYV-101, a novel fully human anti-CD19 chimeric antigen receptor T-cell therapy in refractory generalized myasthenia gravis" "Design of KYSA-7, a phase 2, open-label, randomized, multicenter study of KYV-101, an autologous fully human anti-CD19 chimeric antigen receptor (CAR) T-cell therapy, in treatment refractory primary and secondary progressive multiple sclerosis" The posters presented at ECTRIMS will be available on the publications page of Kyverna's website, and highlights from the case studies presented at Kyverna's symposium will be included in the company's updated corporate presentation, which will be available on the investor relations section of Kyverna's website. About Stiff-Person Syndrome (SPS) SPS is a rare, progressive neurological autoimmune disorder causing debilitating muscle stiffness in the torso, arms, and legs, impacting the ability to walk or move. Patients typically present with muscle spasms and stiffness, resulting in difficulty turning and bending. When stiffness is severe, the patients' posture resembles a statue. Muscle spasms and stiffness can be precipitated by unexpected stimuli, including sounds, like a phone ring or a siren, sudden touches or conditions triggering anxiety and emotional upset which, when severe, are misdiagnosed as a primary anxiety disorder1. About Myasthenia Gravis (MG) Myasthenia gravis is an autoimmune disorder associated with muscle weakness in tissues throughout the body, potentially manifesting in partial paralysis of eye movements, problems in chewing and swallowing, respiratory problems, speech difficulties and weakness in skeletal muscles. MG patients develop antibodies that lead to an immunological attack on critical signaling proteins at the junction between nerve and muscle cells, thereby inhibiting the ability of nerves to communicate properly with muscles. The symptoms of the disease can be transient and in the early stages of the disease can remit spontaneously. However, as the disease progresses, symptom-free periods become less frequent and disease exacerbations can last for months. Disease symptoms reach their maximum levels within two to three years in approximately 80% of patients. Up to 20% of MG patients experience respiratory crisis at least once in their lives2. About Multiple sclerosis (MS) Multiple sclerosis is a chronic neurodegenerative autoimmune disease affecting over 2.8 million individuals worldwide3. It affects more frequently women, people of Northern European descent, and is also associated with certain environmental and genetic factors. Patients with MS can experience a range of symptoms including blurred vision, slurred speech, tremors, numbness, extreme fatigue, problems with memory and concentration, and, in severe cases, the inability to walk or stand. Current disease-modifying treatments for MS aim to reduce the frequency of disease relapses and delay progression of disability, but the disease remains a chronic condition that will progressively worsen for most patients. About KYV-101 KYV-101 is an autologous, fully human CD19 CAR T-cell product candidate for use in B cell-driven autoimmune diseases. The CAR in KYV-101 was designed by the National Institutes of Health (NIH) to improve tolerability and tested in a 20-patient Phase 1 trial in oncology. Results were published by the NIH in Nature Medicine4. KYV-101 is currently being evaluated in sponsored, open-label, Phase 1/2 and Phase 2 trials in the United States and Germany across two broad areas of autoimmune diseases, rheumatologic and neuroinflammatory, as well as in investigator-initiated trials for multiple indications in multiple geographies. The clinical experience to date with KYV-101 in both oncological and autoimmune diseases highlight the differentiated properties of KYV-101 and the potential to treat autoimmune patients. About Kyverna Therapeutics Kyverna Therapeutics, Inc. (Nasdaq: KYTX) is a patient-centered, clinical-stage biopharmaceutical company focused on developing cell therapies for patients suffering from autoimmune diseases. Our lead CAR T-cell therapy candidate, KYV-101 is advancing through clinical development with sponsored clinical trials across two broad areas of autoimmune disease: rheumatology and neurology, including Phase 2 trials for stiff-person syndrome, multiple sclerosis and myasthenia gravis, a Phase 1/2 trial for systemic sclerosis, and two ongoing multi-center, open-label Phase 1/2 trials in the United States and Germany for patients with lupus nephritis. Kyverna's pipeline includes next-generation CAR T-cell therapies in both autologous and allogeneic formats with properties intended to be well suited for use in B cell-driven autoimmune diseases. Forward-Looking Statements Statements in this press release about future expectations, plans and prospects, as well as any other statements regarding matters that are not historical facts, may constitute "forward-looking statements." The words, without limitation, "anticipate," "believe," "continue," "could," "estimate," "expect," "intend," "may," "plan," "potential," "predict," "project," "should," "target," "will," "would" and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these or similar identifying words. Forward-looking statements in this press release include, without limitation, those related to: the potential impact of the clinical outcomes from the ongoing clinical programs; the potential impact of the new data on the treatment efficacy and safety profile of KYV-101; the potential that the results of the ongoing trials could drastically change the treatment landscape for the targeted autoimmune diseases; Kyverna's goals to develop certain paradigm-shifting treatment options; the potential for KYV-101 to provide durable, immunosuppressant-free remission for autoimmune disease patients; Kyverna's beliefs about the differentiated properties of KYV-101; and Kyverna's clinical trials and named-patient activities. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including: uncertainties related to market conditions, and other factors discussed in the "Risk Factors" section of Kyverna's most recent Annual Report on Form 10-K and Quarterly Reports on Form 10-Q that Kyverna has filed or may subsequently file with the U.S. Securities and Exchange Commission. Any forward-looking statements contained in this press release are based on the current expectations of Kyverna's management team and speak only as of the date hereof, and Kyverna specifically disclaims any obligation to update any forward-looking statement, whether as a result of new information, future events or otherwise. For more information, please visit . Contact: Precision AQ on behalf of Kyverna Therapeutics Investors: [email protected] Media: [email protected] 1 Dalakas, M.C., Neurotherapeutics 2022; 19, 832–847. 2 Payus et al., Am J Case Rep. 2021; 22: e928419-1–e928419-4. 3 Walton C, et al., Mult Scler. 2020; 26:1816-1821. 4 Brudno et al., Nature Medicine 2020; 26:270-280. SOURCE Kyverna Therapeutics WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

临床2期细胞疗法临床1期临床结果免疫疗法

2024-09-18

·佰傲谷BioValley

从Kite挖来CEO也没用，股价持续下跌

要论细胞治疗商业化经验，吉利德/Kite可谓是全球顶尖之一，推进了两款CAR-T细胞治疗的上市，旗下Yescarta是全球最热销的CAR-T细胞产品。因此，Kyverna Therapeutics在预期产品将进入后期开发，即将触及商业化道路后，便从吉利德/Kite挖来一位新CEO，以带领其自免CAR-T治疗产品的未来开发和商业化道路。领导层变动 2024年9月16日，Kyverna公司宣布领导层更新，任命Warner Biddle为公司首席执行官（CEO）及董事会成员。Warner Biddle将接替Peter Maag，Peter Maag已经辞去首席执行官和董事会成员的职务，立即生效。 Warner Biddle此前担任Kite Pharma的高级副总裁兼全球商业主管，在2023年被任命为Kite的临时CEO。Warner Biddle具有30年的商业、产品规划和特许经营领导经验，并成功推动血液学CAR-T的上市。此外，Kyverna公司还宣布任命Christi Shaw为董事会成员，接替从董事会辞职的Brian Kotzin。Christi Shaw此前担任Kite的首席执行官。 Kyverna公司的董事会主席Ian Clark在一份声明中欢迎了两位高管的加入，并表示：“Kyverna今天正处于一下章的悬崖上，因为KYV-101正在迅速进入后期开发阶段，并踏上上市之路。” KYV-101的前景 Kyverna的KYV-101，是一款自体CD19 CAR-T细胞治疗，是最早进入临床的自免CAR-T产品之一。 KYV-101的CAR构建体（图片来源：参考资料4） Kyverna公司表示KYV-101在涉及15个自免适应症的50名患者的初始数据中展现了积极的安全性和有效性，尤其在僵人综合征（SPS）和重症肌无力中显示出商业前景。数据截止至2024年7月31日，接受KYV-101治疗的36名自免患者没有报告严重的ICANS或≥3级的CRS。 KYV-101还获得了两个FDA授予的再生医学高级疗法（RMAT），分别用于僵人综合征（SPS）和重症肌无力。 2024年6月，《美国国家科学院院刊》（PNAS）刊登了一份报告，描述了KYV-101用于治疗一名难治性SPS患者后，步行距离的显著改善和GABAergic药物的使用减少40%，同时显示出良好的安全性和耐受性。 Kyverna公司还报告了一位重症肌无力患者的临床获益，该名患者在接受KYV-101治疗后一年无进展。延伸阅读： CAR-T的自免临床进展当乐坛天后遇上CAR-T疗法此外，36名自免患者的KYV-101达到100%的制造成功率。小结 Kyverna公司换将的消息传来，当日股价下跌6%。 Kyverna公司于今年2月登录纳斯达克，完成了3.19亿美元的募资。但是，Kyverna公司的股价却一路下跌，从刚上市时的34美元跌至个位数，下跌幅度超过70%。尽管Kyverna公司给出了KYV-101的前景期许，但是自免CAR-T治疗潜在的复发（今年6月Kyverna公司报道了一位KYV-101低剂量的狼疮患者复发）和致瘤风险，还是让人对自免CAR-T治疗的前景不太乐观。参考资料： 1.https://ir.kyvernatx.com/news-releases/news-release-details/kyverna-therapeutics-announces-leadership-update 2.https://www.biopharmadive.com/news/kyverna-ceo-resign-peter-maag-biddle-kite-shaw/727055/ 3.https://ir.kyvernatx.com/news-releases/news-release-details/kyverna-therapeutics-provides-business-update-and-reports-second 4.https://ir.kyvernatx.com/static-files/348ee07d-e52c-423c-9a99-bf5f4637e682 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 · · · · · · · ·

细胞疗法免疫疗法高管变更ASH会议

100 项与 KYV-101 相关的药物交易

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适应症	最高研发状态	国家/地区	公司	日期
类风湿关节炎	临床2期	-	Kyverna Therapeutics, Inc.初创企业	2024-11-01
原发性进行性多发性硬化	临床2期	美国	Kyverna Therapeutics, Inc.初创企业	2024-09-01
继发进展型多发性硬化	临床2期	美国	Kyverna Therapeutics, Inc.初创企业	2024-09-01
重症肌无力	临床2期	美国	Kyverna Therapeutics, Inc.初创企业	2024-08-28
重症肌无力	临床2期	德国	Kyverna Therapeutics, Inc.初创企业	2024-08-28
弥漫性硬皮病	临床2期	美国	Kyverna Therapeutics, Inc.初创企业	2024-08-06
系统性硬皮病	临床2期	美国	Kyverna Therapeutics, Inc.初创企业	2024-08-06
狼疮性肾炎	临床2期	德国	Kyverna Therapeutics, Inc.初创企业	2022-12-01
ANCA相关性血管炎	临床1期	-	Kyverna Therapeutics, Inc.初创企业	2024-06-01
特发性炎症性肌病	临床1期	-	Kyverna Therapeutics, Inc.初创企业	2024-06-01

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研究	分期	人群特征	评价人数	分组	结果	评价	发布日期


PRNewswire 人工标引	N/A	僵人综合征	1	KYV-101	壓餘齋鏇醖艱鬱繭醖糧(獵廠壓膚鬱築範構構淵) = 艱繭廠艱壓鹹膚製廠願艱齋鹽鑰網廠窪遞襯壓 (鏇簾選齋積餘範蓋夢鏇 )	积极	2024-06-17
PRNewswire 人工标引	N/A	重症肌无力	1	KYV-101	餘觸艱衊簾選製積壓餘(製願範積憲遞壓憲簾齋) = 餘夢衊製廠醖願選醖膚蓋淵繭獵鹹觸淵範襯醖 (廠觸遞繭醖觸醖憲積壓 ) 更多	积极	2023-11-15