RegulationAccelerated Approval (United States), Orphan Drug (United States), Orphan Drug (European Union), Priority Review (China), Breakthrough Therapy (China), Conditional marketing approval (China), Regenerative Medicine Advanced Therapy (United States)

Structure/Sequence

Sequence Code 9718ScFv

Sequence Code 170963ScFv

Sequence Code 13042446CAR construct

Clinical Trials associated with Axicabtagene Ciloleucel

NCT06902012

/ RecruitingPhase 1/2IIT

A Prospective, Single - Arm Clinical Study on the Safety and Efficacy of Early Second Infusion of CD19 CAR - T Based on ctDNA Monitoring in the Treatment of Relapsed/Refractory Large B - Cell Lymphoma

The goal of this clinical trial is to evaluate the efficacy and safety of early secondary infusion of CD19 CAR T-cell therapy in adults with relapsed/refractory diffuse large B-cell lymphoma (DLBCL), guided by ctDNA monitoring. The main questions it aims to answer are:
1. Efficacy: Does early secondary CAR-T infusion improve the 3-month complete remission (CR) rate and long-term survival outcomes (e.g., 1-year PFS, OS)?
2. Safety: What are the adverse events associated with secondary CAR-T infusion, such as cytokine release syndrome (CRS), immune effector cell-associated neurotoxicity (ICANS), and infections?
This is a single-arm, single-center, prospective study. All participants will receive:
* Leukapheresis to collect T cells for CAR-T manufacturing.
* Preconditioning chemotherapy (fludarabine and cyclophosphamide) to prepare the body for CAR-T infusion.
* Two CD19 CAR-T infusions: The first infusion (2×10⁶ cells/kg) followed by a second infusion (same dose) if ctDNA remains positive when PET/CT shows CR or PET/CT shows PR within 60 days post-first infusion.
Participants will undergo:
* Frequent hospital monitoring for ≥14 days post-infusion to manage potential toxicities.
* Regular follow-ups (e.g., blood tests, ctDNA analysis, PET/CT scans) at scheduled intervals up to 12 months.
* Continuous safety assessments, including CRS grading, neurological evaluations, and infection monitoring.

Start Date01 Feb 2027

Sponsor / Collaborator-

NCT06768905

/ Not yet recruitingPhase 1/2IIT

IOMAB-ACT: a Phase Ib/II Multi-institutional Study of 131 I-Apamistamab Followed by CD19-targeted CAR-T Cell Therapy for Patients with Relapsed or Refractory (R/R) Diffuse Large B-cell Lymphoma (DLBCL)

This study is being done to determine the safety, efficacy and tolerability of a single 50 mCi dose of 131I-Apamistamab given prior to CAR-T cell infusion in patients with Relapsed or refractory (R/R) Diffuse large B-cell lymphoma (DLBCL).

Start Date01 Apr 2025

Sponsor / Collaborator

The University of Texas Southwestern Medical Center

[+1]

NCT06912529

/ RecruitingPhase 2

Axicabtagene Ciloleucel CAR T-cells in Patients With Relapsed or Refractory Primary Mediastinal B-cell Lymphoma

This phase II study will evaluate the efficacy, safety and tolerability of second-line treatment with axicabtagene ciloleucel in primary mediastinal B-cell lymphoma patients (PMBCL).

Start Date13 Mar 2025

Sponsor / Collaborator

Universität Leipzig

[+1]

100 Clinical Results associated with Axicabtagene Ciloleucel

100 Translational Medicine associated with Axicabtagene Ciloleucel

100 Patents (Medical) associated with Axicabtagene Ciloleucel

581

Literatures (Medical) associated with Axicabtagene Ciloleucel

01 Apr 2025·BONE MARROW TRANSPLANTATION

Treatment failure patterns in early versus late introduction of CAR T-cell therapy in large B-cell lymphoma

Article

Author: Brown, Samantha ; Kabat, Maciej ; Luttwak, Efrat ; Park, Jae ; Zuckerman, Tsila ; Ip, Andrew ; Rejeski, Kai ; Pavkovic, Emma ; Devlin, Sean M ; Shah, Gunjan L ; Flynn, Jessica R ; Scordo, Michael ; Landego, Ivan ; Shouval, Roni ; Perales, Miguel-Angel ; Lin, Richard J ; Leslie, Lori A ; Salles, Gilles ; Dahi, Parastoo B ; Palomba, M Lia ; Leithner, Doris ; Khatib, Hazim ; Corona, Magdalena ; Luna, Alejandro ; Beyar-Katz, Ofrat ; Cassanello, Giulio ; Schoder, Heiko

CD19-directed chimeric antigen receptor T-cell (CAR-T) therapy has recently been approved as second-line treatment for relapsed/refractory large B-cell lymphoma (LBCL). This study compares patterns of disease relapse and progression across patients receiving CAR-T as second-line (early administration) versus third or subsequent lines (late administration). We analyzed 354 patients treated with Axicabtagene ciloleucel (71%) and Lisocabtagene maraleucel (29%); 80 (23%) received early administration, and 274 (77%) late administration. One-year overall survival was higher in the early group (82% [95% CI 72-93] vs. 71% [95% CI 66-77], p = 0.048). However, the survival benefit was not sustained in multivariable Cox regression modeling and propensity score matching. One-year cumulative incidences of relapse were similar (37% [95% CI 24-50] vs. 43% [95% CI 37-49], p = 0.2), as were 1-year progression-free survival probabilities (62% [95% CI 50-76] vs. 50% [95% CI 44-57], p = 0.14). The early group exhibited a favorable toxicity profile, with lower rate of grade ≥2 cytokine release syndrome (26% vs. 39%, p = 0.031) and reduced cumulative incidence of severe neutropenia (41% [95% CI 30-52] vs. 55% [95% CI 49-60], p = 0.027). Our results indicate favorable outcomes with CAR-T irrespective of treatment line. The equivalence in disease control suggests that CAR-T resistance mechanisms persist in LBCL failing first-line therapy.

01 Apr 2025·Nature Reviews Clinical Oncology

Outcome correlates of approved CD19-targeted CAR T cells for large B cell lymphoma

Review

Author: Bock, Tamara J ; Turtle, Cameron J ; Fiorenza, Salvatore ; Colonne, Chanukya K

CD19-targeted chimeric antigen receptor (CAR) T cells have provided a breakthrough in the treatment of patients with relapsed and/or refractory large B cell lymphoma (LBCL). Currently, three CD19-targeted CAR T cell products are approved by the FDA and various other regulators for the treatment of patients with LBCL: axicabtagene ciloleucel, tisagenlecleucel and lisocabtagene maraleucel. Response rates following infusion of these CD19-targeted CAR T cells have been promising; however, approximately half of treated patients show relapse within 2 years. Furthermore, receiving these agents can be associated with serious toxicities, including cytokine release syndrome and immune effector cell-associated neurotoxicity syndrome. In this Review, we summarize the factors associated with the efficacy, including response and survival outcomes, and toxicity of CD19-targeted CAR T cells in pivotal clinical trials and large real-world datasets describing the outcomes of patients with LBCL who received treatment with these products.

01 Apr 2025·EJHaem

Clinical Impact of CD19 Expression Assessed by Quantitative PCR in Lymphoma Patients Undergoing CAR‐T Therapy

Article

Author: Saus, Ana ; Pérez, Ariadna ; Ferrández‐Izquierdo, Antonio ; Martínez‐Ciarpaglini, Carolina ; Ventura, Laura ; Benzaquén, Ana ; Ortí, Consejo ; Ferrer‐Lores, Blanca ; Solano, Carlos ; Piñana, José Luis ; Teruel, Anabel ; Gómez, Montse ; Heras, Begoña ; Hernández‐Boluda, Juan Carlos ; Remigia, María José ; Amat, Paula ; Goterris, Rosa ; Serrano, Alicia ; Rivada, Marcos ; Pastor‐Galán, Irene ; Arroyo, Ignacio ; Hernani, Rafael ; Terol, María José

ABSTRACT:

Introduction:

Current guidelines do not mandate CD19 tumor expression assessment before chimeric antigen receptor T‐cell (CAR‐T) therapy in large B‐cell lymphoma (LBCL) patients due to limitations of immunohistochemistry (IHC) or flow cytometry. Quantitative polymerase chain reaction (qPCR) offers a more sensitive alternative for detecting CD19 expression, with the primary advantage that mRNA can be easily extracted from paraffin‐embedded tissues.

Methods & Results:

In our study, we included 51 adult patients with LBCL treated with axicabtagene ciloleucel. Among them, 16 were classified as CD19‐negative by IHC; however, qPCR reclassified six (37.5%) as CD19‐positive. We then compared the outcomes between consistently CD19‐negative (IHC−qPCR−) and CD19‐positive (IHC+ and IHC−qPCR+) patients. CD19‐negative cohort showed worse 1‐year progression‐free survival (15 vs. 45%, p = 0.044) and a trend toward shorter duration of response (29 vs. 55%, p = 0.065). Only one (10%) of the CD19‐negative patients remained alive and disease‐free at last follow‐up (6 months), having previously responded to bridge therapy.

Discussion:

If confirmed in a large patient cohort, these findings could form the basis for modifying current patient selection criteria. Consistently negative patients may be suboptimal candidates for anti‐CD19 CAR‐T therapy. Alternative therapeutic options, such as bispecific antibodies or polatuzumab‐based regimens, could be considered for this subset of patients.

416

News (Medical) associated with Axicabtagene Ciloleucel

26 Mar 2025

·www.prnewswire.com

Fosun Pharma Announces 2024 Annual Results

Accelerates Globalization of Innovative Products with Operating Cash Flow Surging 31.13% YoY SHANGHAI, March 25, 2025 /PRNewswire/ -- On March 25, 2025, Shanghai Fosun Pharmaceutical (Group) Co., Ltd. ("Fosun Pharma"or "the Group"; Stock Code: 600196.SH, 02196.HK), a leading global pharmaceutical and healthcare company driven by innovation, announced its annual results for the year ended 31 December 2024 ("the Reporting Period"). In 2024, Fosun Pharma further focused on the business development of innovative drugs and high-value devices, achieving operating revenue of RMB41.07 billion and net profit attributable to shareholders of RMB2.77 billion, representing an increase of 16.08% YoY. Fosun Pharma continuously optimize supply chain management to enhance operational efficiency, recording an operating cash flow of RMB4.48 billion with an increase of 31.13% YoY, surpassing the growth rate of operating profit. In addition, Fosun Pharma continues to advance lean management across quality enhancement, cost control, efficiency improvement, cycle management, and innovation R&D, driving operational efficiency and expanding profitability. During the Reporting Period, the gross margin minus the selling and distribution expense ratio improved by 2.45 percentage points YoY; and, excluding the impact of newly acquired enterprises during the Reporting Period and the same period last year, administrative expenses declined by RMB355 million. Fosun Pharma always takes innovation as a core driving force in its development. Fosun Pharma has established an open and globally integrated pharmaceutical R&D ecosystem, focusing on core therapeutic areas including oncology (solid tumors and hematologic malignancies) and Immune-inflammatory disorders. The company is strategically enhancing its technological leadership in antibody/ADC platforms, cell therapies, and small molecule development, while collaborating with industry funds to pioneer next-generation modalities such as radiopharmaceuticals, RNA therapeutics, gene editing, and AI-powered drug discovery. During the Reporting Period, Fosun Pharma continuously optimized its innovative R&D system while maintaining stable R&D investment, focusing on key pipeline strengths and enhancing efficiency through system integration. In 2024, Fosun Pharma's total R&D expenditure amounted to RMB5.55 billion, with R&D expenses totaling RMB3.64 billion. The R&D expenditure in the pharmaceutical manufacturing segment amounted to RMB4.91 billion, accounting for 16.98% of pharmaceutical business revenue. There are over 80 major pipeline projects on innovative drugs and biosimilars (calculated by indications). In addition, during the Reporting Period, the pharmaceutical manufacturing segment of Fosun Pharma submitted 220 patent applications, including 3 American patent applications, 18 PCT applications, and Fosun Pharma has obtained 66 licensed invention patents authorization. Accelerating Innovation Transformation and the Internationalization of Innovative Products Focusing on unmet clinical needs, Fosun Pharma continuous to push forward its innovation transformation and the development and commercialization of innovative products, expanding into regulatory markets such as the U.S. and the EU. During the Reporting Period, 7 innovative products/biosimilars with a total of 16 indications independently developed and licensed-in by Fosun Pharma were approved for launch, and 8 innovative products/biosimilars had entered the pre-launch approval/critical clinical stage. Fosun Pharma also had 18 innovative products/biosimilar projects (calculated by indications) initiated clinical trials. In the field of lung cancer, Fosun Pharma has developed a comprehensive layout in innovative drugs and precision diagnostics and treatment to benefit patients globally. Fosun Pharma's self-developed innovative anti-PD-1 monoclonal antibody, Han Si Zhuang (Serplulimab Injection), is the world's first anti-PD-1 monoclonal antibody (mAb) approved for the first-line treatment of extensive-stage small cell lung cancer (ES-SCLC), and it remains the first and only anti-PD-1 mAb approved in the EU for the treatment of ES-SCLC. During the Reporting Period, Han Si Zhuang also received approval in Chinese mainland for the treatment of non-squamous non-small cell lung cancer (nsNSCLC), and this also marked its third indication in the field of lung cancer, further expanding its reach. To date, Han Si Zhuang has been approved for marketing in over 30 countries and regions, including China, the European Union, and several Southeast Asian nations, benefiting more than 100,000 patients. In addition, to help more lung cancer patients obtain early diagnosis and treatment in a more minimally invasive way, the Ion Bronchial Navigation Operation Control System (Ion System) of Intuitive Fosun, Fosun Pharma's associated company, received NMPA approval in March 2024 and has been commercially deployed. In high-incidence cancers such as breast and gastric cancers, Fosun Pharma's self-developed monoclonal antibody(mAb) biosimilar Han Qu You (Trastuzumab Injection) has been approved for launch in over 50 countries and regions, including China, the European Union, the U.S., Canada and Australia, and has been included in the health insurance directories in countries such as China, UK, France, and Germany. To date, Han Qu You has benefited over 240,000 patients. The HER2-targeted innovative monoclonal antibody HLX22 for gastric cancer treatment and the novel endocrine therapy HLX78 (lasofoxifene) for breast cancer are currently under international multi-center Phase III clinical trials. Additionally, the biosimilar candidate HLX11 (pertuzumab) has had its new drug applications submitted and accepted in both China and the U.S., injecting new momentum into global expansion. In addition, Fosun Pharma is also advancing the development of rare disease drugs. Its self-developed MEK1/2 selective inhibitor Luvometinib tablets, (Project Code: FCN-159), has had its new drug applications for two indications—treatment of adult dendritic cell and histiocytic tumors, and treatment of plexiform neurofibromas (PN) associated with Neurofibromatosis Type 1 (NF1) in children aged 2 years and older—accepted by the NMPA and placed in the priority review process . In the non-oncology field, during the Reporting Period, Fosun Pharma's self-developed and licensed-in rabies vaccine (Vero cell) for human use (freeze dried), Pu Rui Ni (Pretomanid Tablets), Han Da Yuan (Adalimumab Injection) received approval for four new indications, and Su ke Xin (Avatrombopag Malate Tablets) was approved for its second indication in China; meanwhile, the new drug applications for a biosimilar of denosumab HLX14 under development were accepted by the European Medicines Agency (EMA), Health Canada and the FDA, respectively. Continuously Enhancing Global Operations and Bilateral Licensing Cooperation Fosun Pharma continues to uphold the internationalization strategy in multiple dimensions, such as innovative R&D, licensing, manufacturing and operations as well as commercialization. In 2024, overseas revenue reached RMB11.30 billion, accounting for 27.51% of total revenue. During the reporting period, Fosun Pharma continues to promote the building of production system with international quality standard, laying a solid foundation for the overseas distribution of preparations. As of the end of 2024, all production lines of the domestic subsidiaries under the pharmaceutical manufacturing segment of Fosun Pharma obtained domestic GMP certifications and 10 production lines had passed GMP certification in major regulatory markets such as the U.S. and the EU. Fosun Pharma's pharmaceuticals and medical devices businesses now primarily cover major overseas markets, including the United States, Europe, Africa, India, and Southeast Asia, with an overseas commercialization team of over 1,000 employees. In the U.S., Fosun Pharma's in-house generic drug team has continued to grow, establishing partnerships with major distributors and group purchasing organizations (GPOs) to drive the sales of formulation products. Meanwhile, Fosun Pharma has established its innovative drug team in the U.S. and is preparing for the commercialization of its anti-PD-1 monoclonal antibody (mAb), serplulimab injection. In Europe, Gland Pharma has built localized manufacturing capabilities through its subsidiary, Cenexi. Sisram Medical has expanded its global direct sales offices to 12, with its marketing network now covering over 110 countries and regions. The share of direct sales revenue has further increased to 87%. Meanwhile, Breas Medical's marketing network has expanded across key mature markets, including Europe, the United States, Japan, and Australia. In emerging markets, Fosun Pharma's sales network in Africa now covers over 40 countries and regions. The company continues to advance the Côte d'Ivoire park project to establish localized pharmaceutical manufacturing and supply in Africa. In February 2025, Fosun Pharma launched a new pharmaceutical and medical device sales platform in Nanning, gradually enhancing its registration and commercialization capabilities in Southeast Asia to expand its presence in the region. Fosun Pharma is also actively expanding into the Middle East market. During the reporting period, Henlius, a subsidiary of Fosun Pharma, entered into a strategic partnership with SVAX to establish a joint venture in Saudi Arabia. This collaboration aims to enhance the accessibility of innovative and high-value products in the MENAT region (Middle East, North Africa, and Turkey). Fosun Pharma continues to strengthen its global licensing collaboration, further enhancing its international influence and competitiveness. On the out-licensing front, Henlius, a subsidiary of Fosun Pharma has forged deep partnerships with over 20 leading global biopharmaceutical companies, including Accord, Abbott, Eurofarma, KGbio, Organon, and Dr. Reddy's, to jointly expand global markets. These collaborations comprehensively cover major biologics markets in Europe and the U.S., as well as numerous emerging markets. To date, its approved biologics have benefited more than 750,000 patients worldwide. Fosun Pharma continues to expand its product pipeline in core therapeutic areas through strategic global collaborations. Licensed-in products such as the antiemetic Akynzeo® (netupitant/palonosetron capsules), long-lasting recombinant human granulocyte colony-stimulating factor product (Pei Jin) and the drug for heart failure and hypertension treatment (Yi Xin Tan) have been included in the National Medical Insurance Drugs Catalogue, benefiting a wide range of patients. Fosun Pharma has obtained exclusive development and commercialization rights for Tenapanor Hydrochloride Tablets (Wan Ti Le), a first-in-class phosphate-lowering drug with a novel mechanism. In February 2025, the drug received approval from the National Medical Products Administration (NMPA) in China for the treatment of hyperphosphatemia in adult dialysis patients with chronic kidney disease (CKD). This approval marks a new era of multi-mechanism phosphate control, bringing new hope to dialysis patients with hyperphosphatemia. During the reporting period, Fosun Pharma's licensed product Daxxify® (DaxibotulinumtoxinA-lanm) became the first of its kind approved in China. The product received NMPA approval for the improvement in the appearance of moderate to severe glabellar lines associated with corrugator and/or procerus muscle activity in adult patients. Accelerating Localization of Innovative Products in China to Achieve High-quality Development As a global innovation-driven pharmaceutical and healthcare company, Fosun Pharma actively introduces internationally advanced technologies and products to the Chinese market, benefiting patients and customers. During the reporting period, Fosun-Insightec, a joint venture established with Insightec in China, successfully achieved sales of magneticresonance image guided focused ultrasound brain therapy system ("MRgFUS brain therapy system") for brain treatment. Additionally, the headquarters and industrial base of Intuitive Fosun, an associated company, was officially inaugurated in the Zhangjiang International Medical Park in Shanghai. This facility integrates R&D, manufacturing, and training, further accelerating the localization of the da Vinci Robotic surgical systems in China. To further enhance its leading position in tumor immunotherapy, Fosun Pharma increased its stake in its cell therapy platform, Fosun Kairos, to 100% during the reporting period. The company will continue to collaborate with Kite Pharma to advance the development and commercialization of its licensed products, Axi-Cel (marketed as Yi Kai Da ) and Brexu-Cel (investigational project FKC889), in Chinese mainland, Hong Kong S.A.R and Macao S.A.R. Yi Kai Da became the first in China to introduce an innovative outcome-based payment model. As of the end of 2024, Yi Kai Da has benefited over 800 lymphoma patients, been included in more than 110 provincial and municipal government-backed insurance programs and over 80 commercial insurance plans, and has been registered in more than 180 treatment centers across 28 provinces and municipalities nationwide. While maintaining steady operations, Fosun Pharma remains committed to its corporate social responsibilities. In 2024, the company received multiple domestic and international accolades for its significant progress in ESG, including recognition as one of China's ESG Listed Company Pioneer 100, Deloitte's China Best Managed Companies, Charity Star, and Forbes 2024 China ESG 50 list. Additionally, Fosun Pharma's MSCI ESG rating remained at A. "Fosun Pharma will continue to adhere to innovation-driven and global development strategies, actively embrace AI, concentrate resources on core businesses, and persistently advance lean management and enhance operational efficiency." Wu Yifang, Chairman of Fosun Pharma said, "We aim to grow in tandem with China's biopharmaceutical industry and stride into a new phase of high-quality development. Upholding our mission to 'create better health for families worldwide', we will maintain a patient-centric approach, address unmet clinical needs, deepen innovation transformation and global expansion, and strive to become a global leader in healthcare innovation and integration." About Fosun Pharma Founded in 1994, Shanghai Fosun Pharmaceutical (Group) Co., Ltd. ("Fosun Pharma"; SSE: 600196, HKEX: 02196) is a leading innovation-driven global healthcare company operating in the fields of pharmaceuticals, medical devices & diagnostics, and healthcare services. Through its strategic alliance with Sinopharm Group Co., Ltd., Fosun Pharma further extends its capabilities in pharmaceutical commerce. Over the past 30 years since its establishment, Fosun Pharma has maintained deep roots in China while strategically expanding its global presence. The company has been actively implementing its "4IN" strategy -Innovation, Internationalization, Intelligentization, and Integration, with core business operations now spanning major overseas markets including the United States, Europe, Africa, India, and Southeast Asia. At present, Fosun Pharma has established an open and globally integrated pharmaceutical R&D ecosystem, focusing on core therapeutic areas including oncology (solid tumors and hematologic malignancies) and Immune-inflammatory disorders. The company is strategically enhancing its technological leadership in antibody/ADC platforms, cell therapies, and small molecule development, while collaborating with industry funds to pioneer next-generation modalities such as radiopharmaceuticals, RNA therapeutics, gene editing, and AI-powered drug discovery. This multidimensional approach accelerates the translation of innovative therapies into clinical practice, systematically addressing critical unmet medical needs worldwide. Looking ahead, Fosun Pharma remains anchored in its core values of "Care for life, Continuous innovation, Pursuit of excellence and Sustainable partnership". By advancing its global innovation engine and operational excellence, the company strives to be a global leader to integrate pharmaceutical and healthcare innovations, creating better health for families worldwide. SOURCE Fosun Pharma WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Drug ApprovalPhase 3VaccineLicense out/in

19 Mar 2025

·www.pharmaceutical-technology.com

Allogeneic cell therapies “poised for prime time” says expert

Illustration showing chimeric antigen receptor (CAR) T cells (blue) being used to treat a melanoma (orange). Image source: Getty Images/ NEMES LASZLO/SCIENCE PHOTO LIBRARY As cell and gene therapy developers struggle against an investment slump and competition from established autologous treatments, allogeneic cell therapies may still find a niche. Though still nascent, allogeneic options are “poised for prime time,” said Stefanos Theoharis, CEO of the Barcelona, Spain-based Onechain Immunotherapeutics, at the Advanced Therapies conference, in London on 18 March. One of the primary attractions of allogeneic options is that they have relatively fewer operational challenges than autologous treatments. Theoharis noted that concerns around the limited scalability and lengthy quality controls for autologous manufacture is spurring the sector’s desire for allogeneic alternatives. But allogeneic therapies have their own challenges; namely a lack of demonstrated efficacy, and the difficulty in finding donors. Conversely, despite logistical difficulties, autologous treatments maintain future viability thanks to their demonstrable efficacy and lack of immunogenicity, said Johannes De Munter, PhD, CEO of the Dutch biotech Neuroplast BV. De Munter also suggested the decentralisation of manufacturing and affiliations between CDMOs and hospitals could shorten logistic chains and improve manufacturing and distribution efficacy. Technological innovation was also noted as a potential route to upscale autologous therapy production. Pharmaceutical Technology spoke with Jason Jones, business development lead at the British cell therapy manufacturing company Cellular Origins. The company’s modular robotics system could support small scale, regional manufacturing facilities for cell therapy biotechs to greatly reduce material transport times, said Jones. Given heterogenous needs for patients, both cell therapy approaches can co-exist. There is potential for competition between the two approaches with chimeric antigen receptor (CAR)-T therapeutics in particular. Another topic that was widely discussed at the meeting was in vivo cell therapies. Approved ex-vivo CAR-T therapies like Gilead Sciences’ Yescarta (axicabtagene ciloleucel) or Novartis’s Kymriah (tisagenlecleucel) involve modifying the T cells extracted from a patient outside their body and then re-administering them. In vivo cel therapies are based on emerging science where T cells are engineered into CAR-Ts inside a patient’s body. On 17 March, AstraZeneca announced its planned acquisition of EsoBiotec in a deal worth up to $1bn. The biotech currently has one in vivo cell therapy in clinical development, ESO-T01, which is being developed for multiple myeloma. EsoBiotec is not the only cell therapy developer with research in this space. In January, another biotech, Umoja Biopharma raised $100m to advance its own in vivo CAR-T therapy in the clinic, while Capstan Therapeutics received $175m in funds to develop its in vivo CAR-T therapy CPTX2309 for treating autoimmune disorders. Free Whitepaper Navigating cell therapy process development The cell therapy manufacturing sector is rapidly evolving, driven by the need to balance innovative process development with rigorous GMP standards. This eBook, brought to you by industry leaders Cytiva, explores how biotech firms can address common challenges — including minimizing contamination risks from manual processes, overcoming resource limitations, and creating scalable, robust designs. Industry experts discuss strategies including automation, strategic outsourcing, and flexible equipment selection to streamline processes and reduce regulatory risks. To unlock expert insights on optimizing your cell therapy pathway from clinical development to commercialization, fill in your details now. Thank you. You will receive an email shortly. Please check your inbox to download the Whitepaper. By Cytiva Thematic By downloading this Whitepaper, you acknowledge that GlobalData may share your information with Cytiva Thematic and that your personal data will be used as described in their Privacy Policy

Cell TherapyAcquisitionImmunotherapy

17 Feb 2025

·www.globenewswire.com

Biocartis announces new data on early CAR-T vector load assessment with Idylla™ presented at ASTCT: A Potential Breakthrough in LBCL Treatment Monitoring

14 February 2025 – Biocartis NV (“Biocartis”), an innovative molecular diagnostics company, is pleased to announce that new research on the prototype Idylla™ CD19 CAR-T vector load Assay was presented as a poster at ASTCT in Honolulu, HI, US, on 13 February 2025. The new study1 (Bharadwaj et al, 20252) demonstrated that early CD19 CAR-T vector load quantification in peripheral blood may improve the prediction of clinical outcomes and toxicity management for patients receiving axicabtagene ciloleucel (axi-cel) for Large B-cell Lymphoma (LBCL). The study enrolled 100 patients undergoing axi-cel therapy. Blood samples were analyzed using both droplet digital PCR (ddPCR) centrally, and local testing in the 3 centers with the prototype Idylla™ CAR-T Assay on the Idylla™ Platform. The study positioned the Idylla™ Platform as an automated, local hospital testing system that can provide results in approximately 90 minutes with only 2 minutes of hands-on time, straight from 0.5 ml of blood. The results revealed a 98.6% concordance between the prototype Idylla™ CAR-T Assay and ddPCR, confirming its reliability as a potential early alternative to standard methods. Key Findings: Early CAR-T Vector load increase predicted severe ICANS: Patients experiencing a steep rise in CD19 CAR-T vector load within the first 5 days post-infusion were more likely to develop severe immune effector cell-associated neurotoxicity syndrome (ICANS). Day 3 Idylla™ measurements predicted ICANS severity: Predictive modeling identified day 3 vector load as a significant predictor of ICANS severity using Idylla™. Correlation with Progression-Free Survival (PFS): Patients were stratified into high and low vector load slope groups obtained during the first 5 days after infusion, with significant differences in PFS observed for both Idylla™ and ddPCR measurement methods. Kaplan-Meier curves revealed PFS rates separate by day 50 (100% vs. 70%), and persisted until the end of follow-up beyond day 300 (90% vs. 50%) for high vs. low vector load increases. These findings suggest that, once approved, local hospital testing with Idylla™ could revolutionize toxicity management and treatment monitoring in CAR-T therapy. By providing real-time, accessible vector load measurements, clinicians may better predict toxicity risk, personalize treatment strategies, and improve patient outcomes. W. Michael Korn, M.D., Chief Medical and Scientific Officer of Biocartis, commented: “These findings underscore the power of early, local hospital testing in CAR-T therapy. By providing real-time insights into CAR-T expansion dynamics, Idylla™ may enable clinicians to better predict toxicity risks and optimize treatment strategy - ultimately improving patient outcomes.” With its revolutionary and proprietary Idylla™ Platform, Biocartis aspires to enable personalized medicine for patients around the world through universal access to molecular testing, by making molecular testing actionable, easy, fast and suitable for any lab. The Idylla™ Platform is a fully automated sample-to-result, real-time PCR (Polymerase Chain Reaction) based system designed to offer in-house molecular biomarker testing in only 3 hours, allowing fast and optimal treatment selection. Idylla™'s continuously expanding menu of molecular diagnostic tests and research assays addresses key unmet clinical needs. Today, Biocartis offers tests supporting melanoma, colorectal, lung, breast, thyroid, brain and blood cancer. More information: www.biocartis.com. Follow us on X (Twitter): @Biocartis. The Idylla™ CAR-T Assay is a prototype and is not commercially available. The Biocartis and Idylla™ trademark and logo are used trademarks owned by Biocartis. © February 2025, Biocartis NV. All rights reserved. 1 This study was conducted with direct support from Biocartis. 2 Bharadwaj, S. et al. (2025). Rapid increase in Blood CD19 CAR-T Vector Load during the First 5 Days Post Infusion is Associated with Severe ICANS. ASTCT. The content above comes from the network. if any infringement, please contact us to modify.

Clinical ResultImmunotherapyClinical StudyDiagnostic Reagents

100 Deals associated with Axicabtagene Ciloleucel

External Link

KEGG	Wiki	ATC	Drug Bank
-	-	L01	DB13915

R&D Status

Approved

10 top approved records.

to view more data

Indication	Country/Location	Organization	Date
High grade B-cell lymphoma	Canada	Gilead Sciences Canada, Inc.	13 Feb 2019
Diffuse Large B-Cell Lymphoma	European Union	Kite Pharma EU BV	23 Aug 2018
Diffuse Large B-Cell Lymphoma	Iceland	Kite Pharma EU BV	23 Aug 2018
Diffuse Large B-Cell Lymphoma	Liechtenstein	Kite Pharma EU BV	23 Aug 2018
Diffuse Large B-Cell Lymphoma	Norway	Kite Pharma EU BV	23 Aug 2018
Mediastinal large B-cell lymphoma	European Union	Kite Pharma EU BV	23 Aug 2018
Mediastinal large B-cell lymphoma	Iceland	Kite Pharma EU BV	23 Aug 2018
Mediastinal large B-cell lymphoma	Liechtenstein	Kite Pharma EU BV	23 Aug 2018
Mediastinal large B-cell lymphoma	Norway	Kite Pharma EU BV	23 Aug 2018
Recurrent Follicular Lymphoma	European Union	Kite Pharma EU BV	23 Aug 2018
Recurrent Follicular Lymphoma	Iceland	Kite Pharma EU BV	23 Aug 2018
Recurrent Follicular Lymphoma	Liechtenstein	Kite Pharma EU BV	23 Aug 2018
Recurrent Follicular Lymphoma	Norway	Kite Pharma EU BV	23 Aug 2018
Refractory Follicular Lymphoma	European Union	Kite Pharma EU BV	23 Aug 2018
Refractory Follicular Lymphoma	Iceland	Kite Pharma EU BV	23 Aug 2018
Refractory Follicular Lymphoma	Liechtenstein	Kite Pharma EU BV	23 Aug 2018
Refractory Follicular Lymphoma	Norway	Kite Pharma EU BV	23 Aug 2018
Follicular Lymphoma	United States	Kite Pharma, Inc.	18 Oct 2017
Large B-cell lymphoma	United States	Kite Pharma, Inc.	18 Oct 2017

Developing

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
T-cell/histiocyte rich large B-cell lymphoma	Phase 3	United States	Kite Pharma, Inc.	25 Jan 2018
T-cell/histiocyte rich large B-cell lymphoma	Phase 3	Australia	Kite Pharma, Inc.	25 Jan 2018
T-cell/histiocyte rich large B-cell lymphoma	Phase 3	Austria	Kite Pharma, Inc.	25 Jan 2018
T-cell/histiocyte rich large B-cell lymphoma	Phase 3	Belgium	Kite Pharma, Inc.	25 Jan 2018
T-cell/histiocyte rich large B-cell lymphoma	Phase 3	Canada	Kite Pharma, Inc.	25 Jan 2018
T-cell/histiocyte rich large B-cell lymphoma	Phase 3	France	Kite Pharma, Inc.	25 Jan 2018
T-cell/histiocyte rich large B-cell lymphoma	Phase 3	Germany	Kite Pharma, Inc.	25 Jan 2018
T-cell/histiocyte rich large B-cell lymphoma	Phase 3	Israel	Kite Pharma, Inc.	25 Jan 2018
T-cell/histiocyte rich large B-cell lymphoma	Phase 3	Italy	Kite Pharma, Inc.	25 Jan 2018
T-cell/histiocyte rich large B-cell lymphoma	Phase 3	Netherlands	Kite Pharma, Inc.	25 Jan 2018

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT05108805 (Prospective Clinical Trial of Outpatient Administration of Axicabtagene Ciloleucel, CTgov)	Phase 4	Diffuse Large B-Cell Lymphoma	25	Telemedicine Visit+Axicabtagene Ciloleucel+Fludarabine+cyclophosphamide	xlwwphkbjp = pkwjefhvxz kgblmvplqc (goycocqqme, tiqmsfwswe - kouyrgzjmd) View more	-	27 Mar 2025
ASH2024	Not Applicable	Large B-cell lymphoma	-	Axicabtagene ciloleucel	bmzcccqnwi(fdmtszbvel) = zfpxcsbheb wxglzopjab (emptwodayp ) View more	-	08 Dec 2024
ASH2024 Manual	Not Applicable	Large B-cell lymphoma	-	Chimeric Antigen Receptor T-Cell Therapy (aged ≥80)	puolidluhv(utstjnxpon) = hqbfohjefi qsrcsxhjat (nhpedxkgvn ) View more	Positive	07 Dec 2024
				Chimeric Antigen Receptor T-Cell Therapy (aged 65-79)	puolidluhv(utstjnxpon) = szsqejjcrx qsrcsxhjat (nhpedxkgvn ) View more
ASH2024	Not Applicable	Diffuse Large B-Cell Lymphoma	-	Axi-cel	qtmsodtxok(hddtjrfgqh) = 1 of 335 patients (0.3%) developed secondary T-cell lymphoma 28 days after CAR-T infusion tdjdtwzakf (wmdsvotlgx )	-	07 Dec 2024
NCT04608487 (ASCO2024) Manual	Phase 1	Secondary Central Nervous System Lymphoma CD19	18	Axicabtagene ciloleucel (axi-cel)	edzhqlzjxg(spwhfhcbqq) = hvbretztff wiflqpykhl (qpqaavbekj ) View more	Positive	24 May 2024
NCT05459571 (ZUMA-24, EHA2024) Manual	Phase 2	Large B-cell lymphoma	23	Axicabtagene ciloleucel	qrqdyxywaw(uqghyimetx) = egztsshpqu xbqmynansq (txrhufelot ) View more	Positive	14 May 2024
CART-SIE (EHA2024) Manual	Not Applicable	Diffuse Large B-Cell Lymphoma CD19	485	Axicabtagene Ciloleucel	ifoatwimer(tpaaaeloqv) = fddahxkrnk hhpmxlwoyx (efhzxyortl ) View more	Positive	14 May 2024
				Tisagenlecleucel	ifoatwimer(tpaaaeloqv) = exjeumbjmk hhpmxlwoyx (efhzxyortl ) View more
EHA2024 Manual	Not Applicable	Non-Hodgkin's lymphoma refractory	155	Axicabtagene Ciloleucel (Axi-cel)	wgfogyqkds(mwsjpoksfg) = gmgxfkoryw vmzgdchpmm (tslzznxxaz ) View more	Positive	14 May 2024
				Brexucabtagene Autoleucel (Brexu-cel)	wgfogyqkds(mwsjpoksfg) = pjxlgiesqh vmzgdchpmm (tslzznxxaz ) View more
NCT03391466 \| NCT02348216 (ZUMA-1, EHA2024) Manual	Phase 2/3	Large B-cell lymphoma Second line \| Third line CD19	4,087	Axicabtagene ciloleucel in 2L	agcvwwokza(ktqrhstqvo) = fynzwnoaqm jfppkiydrg (xfnpatcbux ) View more	Positive	14 May 2024
				Axicabtagene ciloleucel in 3L+	agcvwwokza(ktqrhstqvo) = pbjuhvxczi jfppkiydrg (xfnpatcbux ) View more
NCT04531046 (ALYCANTE, EHA2024)	Phase 2	Large B-cell lymphoma [18F]FDG positron emission tomography (PET)	62	axicabtagene ciloleucel (Responders)	uplocxpbzm(iqdlztpjmc) = evwxuuuqgh kvmuspgquu (fdiihkwgsh ) View more	Positive	14 May 2024
				axicabtagene ciloleucel (Non-responders)	uplocxpbzm(iqdlztpjmc) = ccrnfuuxra kvmuspgquu (fdiihkwgsh ) View more

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Axicabtagene Ciloleucel

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