Latest news with #CAR-T
Yahoo
2 days ago
- Business
- Yahoo
CARsgen Therapeutics Announces NDA Acceptance of Satri-cel by China's NMPA
SHANGHAI, June 25, 2025 /PRNewswire/ -- CARsgen Therapeutics Holdings Limited (Stock Code: a company focused on developing innovative CAR T-cell therapies, announces that the National Medical Products Administration (NMPA) of China has accepted the New Drug Application (NDA) for satricabtagene autoleucel ("satri-cel", CT041) (an autologous CAR T-cell product candidate against protein Claudin18.2) for the treatment of Claudin18.2-positive advanced gastric/gastroesophageal junction adenocarcinoma (G/GEJA) in patients who have failed at least two prior lines of therapy. The NDA submission is mainly based on the results of an open-label, multicenter, randomized controlled confirmatory Phase II clinical trial (CT041-ST-01, NCT04581473) conducted in China. The data have been presented in The Lancet and at the 2025 American Society of Clinical Oncology (ASCO) Annual Meeting. In addition, the company is actively expanding satri-cel application in early-line treatment and perioperative treatment of cancer, including an ongoing Phase Ib registrational trial for pancreatic cancer adjuvant treatment and an ongoing investigator-initiated trial for consolidation treatment following adjuvant therapy in patients with resected G/GEJA. Professor Lin Shen from Beijing Cancer Hospital, the principal investigator of the CT041-ST-01 clinical trial, said, "Gastric cancer is a malignancy with a substantial global disease burden and significant treatment challenges. For patients with advanced gastric cancer, in particular, existing therapeutic options and their efficacy remain severely limited, resulting in extremely poor survival outcomes. Within the current treatment landscape for gastric cancer, a growing number of patients have experienced failure with immunotherapy and anti-angiogenic therapies. Treatment choices and potential benefits become even more constrained in the third-line setting and beyond. Consequently, there exists a significant unmet clinical need for advanced gastric cancer patients after second-line treatment failure. The confirmatory randomized controlled clinical trial of satri-cel has clearly demonstrated that, compared with existing standard therapies, satri-cel offers significant advantages and clinical value in extending both progression-free survival (PFS) and overall survival (OS). The trial results have garnered widespread international attention and recognition, providing a solid evidentiary foundation for satri-cel's New Drug Application (NDA) submission. We look forward to the approval and market launch of satri-cel, which will offer a new treatment option for the broader population of gastric cancer patients." Dr. Zonghai Li, Founder, Chairman of the Board, Chief Executive Officer, and Chief Scientific Officer of CARsgen Therapeutics, said, "We are delighted to announce that the NDA for our self-developed Claudin18.2-targeted CAR T-cell product satri-cel has been accepted for review by China's NMPA. This marks the world's first CAR T-cell therapy product for solid tumors to reach the NDA stage—a major milestone for the CAR-T field. I extend my sincere gratitude to all clinical investigators, trial coordinators, and patients involved in this program. We are hopeful for its timely approval to provide gastric cancer patients with a new treatment option." About Satri-cel Satri-cel is an autologous CAR T-cell product candidate against the protein Claudin18.2 that has the potential to be the first-in-class globally. Satri-cel targets the treatment of Claudin18.2-positive solid tumors with a primary focus on G/GEJA and pancreatic cancer (PC). Initiated trials include investigator-initiated trials (CT041-CG4006, NCT03874897), a confirmatory Phase II clinical trial for advanced G/GEJA in China (CT041-ST-01, NCT04581473), a Phase Ib registrational trial for PC adjuvant therapy in China (CT041-ST-05, NCT05911217), an investigator-initiated trial for satri-cel be used as consolidation treatment following adjuvant therapy in patients with resected G/GEJA (CT041-CG4010, NCT06857786), and a Phase 1b/2 clinical trial for advanced gastric or pancreatic adenocarcinoma in North America (CT041-ST-02, NCT04404595). Satri-cel has been granted Priority Review by the Center for Drug Evaluation (CDE) of China's NMPA for the treatment of Claudin18.2-positive advanced G/GEJA in patients who have failed at least two prior lines of therapy in May 2025. Satri-cel has been granted Breakthrough Therapy Designation by the CDE of China's NMPA for the treatment of Claudin18.2-positive advanced G/GEJA in patients who have failed at least two prior lines of therapy in March 2025. Satri-cel was granted Regenerative Medicine Advanced Therapy designation by U.S. FDA for the treatment of advanced G/GEJA with Claudin18.2-positive tumors in January 2022. Satri-cel received Orphan Drug designation from the U.S. FDA for the treatment of G/GEJA in September 2020. About CARsgen Therapeutics Holdings Limited CARsgen is a biopharmaceutical company focusing on developing innovative CAR T-cell therapies to address the unmet clinical needs including but not limited to hematologic malignancies, solid tumors and autoimmune diseases. CARsgen has established end-to-end capabilities for CAR T-cell research and development covering target discovery, preclinical research, product clinical development, and commercial-scale production. CARsgen has developed novel in-house technologies and a product pipeline with global rights to address challenges faced by existing CAR T-cell therapies. Efforts include improving safety profile, enhancing the efficacy in treating solid tumors, and reducing treatment costs, etc. CARsgen's mission is to be a global biopharmaceutical leader that provides innovative and differentiated cell therapies for patients worldwide and makes cancer and other diseases curable. Forward-looking Statements All statements in this press release that are not historical fact or that do not relate to present facts or current conditions are forward-looking statements. Such forward-looking statements express the Group's current views, projections, beliefs and expectations with respect to future events as of the date of this press release. Such forward-looking statements are based on a number of assumptions and factors beyond the Group's control. As a result, they are subject to significant risks and uncertainties, and actual events or results may differ materially from these forward-looking statements and the forward-looking events discussed in this press release might not occur. Such risks and uncertainties include, but are not limited to, those detailed under the heading "Principal Risks and Uncertainties" in our most recent annual report and interim report and other announcements and reports made available on our corporate website, No representation or warranty is given as to the achievement or reasonableness of, and no reliance should be placed on, any projections, targets, estimates or forecasts contained in this press release. Contact CARsgenFor more information, please visit View original content to download multimedia: SOURCE CARsgen Therapeutics
Medscape
4 days ago
- Health
- Medscape
Lymphoma Highlights From ASCO 2025
Lymphoma highlights from ASCO 2025 are reported by Dr Christopher Flowers from MD Anderson Cancer Center in Houston, Texas. Dr Flowers first reviews a phase 1 study of KITE-363, an anti-CD19/CD20 chimeric antigen receptor (CAR) T-cell therapy, in patients with relapsed or refractory (R/R) B-cell lymphoma. There were no dose-limiting toxicities (a primary endpoint). Grade 3 adverse events occurred in 76% of patients. Patients with primary refractory disease had an overall response rate (ORR) of 80% and a favorable complete response (CR) rate of 67%. Dr Flowers also examines the phase 2/3 clinical trial of zilovertamab vedotin plus standard of care in R/R large B-cell lymphoma. The combination showed meaningful efficacy in ORR rates and CR. Dr Flowers next reviews a phase 2 dose-finding study on AFM13 (CD30/CD16A therapy) plus cord blood allogeneic NK cells. In 24 heavily pretreated patients who received varying doses of both agents, no dose-limiting toxicity was identified and there were no fatal combinations of adverse events. Efficacy and safety extended across several dosages. The ORR was 88% and the CR was 58%. Finally, Dr Flowers reviews early data on multivirus-specific T cells to enhance bispecific antibodies in lymphoma. The T cells, which were enhanced against EBV, CMV, BK virus, and adenovirus, demonstrated ability to kill lymphoma cells in vitro, which may prove a future strategy to enhance tumor activity of bispecific antibodies.
Medscape
17-06-2025
- Health
- Medscape
Integrating CAR T and Bispecific Antibodies in MM Treatment
MILAN — The treatment landscape for relapsed and refractory multiple myeloma (RRMM) has shifted dramatically with the emergence of immunotherapies such as chimeric antigen receptor (CAR) T-cell therapies and bispecific antibodies (bsAbs). These novel approaches have delivered unprecedented outcomes in heavily pretreated patients. Yet determining the optimal treatment strategy remains a clinical challenge. Here at the 2025 European Hematology Association (EHA) Annual Meeting in Milan, Italy, leading experts weighed the strengths and limitations of both approaches, emphasizing that it is not a contest of superiority but a question of sequence and patient selection. Bispecific Antibodies: Off-the-Shelf Convenience With Strong Responses BsAbs work by redirecting T cells toward myeloma cells, binding simultaneously to a tumor antigen and CD3. In his presentation, Philippe Moreau, MD, head of the hematology department at the University Hospital of Nantes, France, reviewed the four agents approved in Europe: teclistamab, elranatamab, and linvoseltamab, which target beta cell maturation antigen (BCMA); and talquetamab, which targets G protein-coupled receptor class C group 5 member D (GPRC5D). These agents deliver overall response rates (ORRs) of 60%-70% in heavily pretreated patients, with median progression-free survival (PFS) of 12-18 months and overall survival (OS) of 24-30 months. Talquetamab in particular induces rapid responses within 1 month but is associated with unique toxicities — such as skin reactions, dysgeusia, and mucosal effects — related to GPRC5D expression in nonhematopoietic tissues like skin. BsAbs offer immediate treatment without the delays associated with CAR T manufacturing. They are also viable for frail patients and more broadly accessible outside of specialized centers. Toxicities, including cytokine release syndrome and infections, are generally manageable with step-up dosing and prophylactic tocilizumab. However, resistance remains a main concern. Roughly one third of patients — particularly those with high-risk cytogenetics, International Staging System stage III disease, or extramedullary disease — exhibit primary resistance. CAR T-Cell Therapy: Durable Outcomes With Earlier Use CAR T-cell therapies have redefined expectations in RRMM, particularly with ciltacabtagene autoleucel, which has achieved a median PFS of 35 months and a median OS approaching 61 months in heavily pretreated populations. Notably, recent data show that one third of patients remain progression-free at 5 years: an unprecedented milestone. 'Phase 3 trials now show improved PFS, OS, and quality of life compared to standard-of-care regimens,' said Paula Rodriguez-Otero, MD, medical coordinator of the Central Unit for Clinical Trials at the University of Navarra, Pamplona, Spain. However, CAR T therapy faces logistical hurdles, including manufacturing delays and the need for specialized infrastructure. High-risk patients with rapid progression or poor bridging therapy responses may still experience suboptimal results, but these challenges are mitigated when CAR T therapy is used earlier in the disease course, before T-cell exhaustion occurs. It's Not One or the Other Both speakers agreed that the future of MM treatment is not about choosing between CAR T and bsAbs but about defining the optimal sequence and integrating both modalities based on patient needs and disease features. BsAbs offer fast, outpatient-ready options for frail or rapidly progressing patients. CAR T therapies, though more complex, offer long remissions and potential treatment-free intervals, especially when used early. Still, Moreau cautioned, many questions remain. What is the best treatment sequence? Should clinicians switch targets — for example from BCMA to GPRC5D — or stick with the same? How should resistance mechanisms, such as antigen loss, be tackled? Can we move toward fixed-duration therapy to reduce costs? Scientific progress must also account for patient priorities. As Solène Clavreul, PhD, noted in an interview with Medscape Medical News , longer survival for myeloma patients means that quality of life is increasingly central. Clavreul is patient advocate and head of medical education and scientific engagement at Myeloma Patients Europe. 'The treatment choices should always be based on research data, but understanding patients' preferences is critical for shared decision-making,' she said. What's Next: BsAb Combinations and Trispecifics 'With 19 new drugs or combinations approved in the past two decades, we've made incredible progress,' said Jesús San Miguel Izquierdo, MD, PhD, director of clinical and translational medicine at the University of Navarra, Pamplona, Spain, in his EHA 2025 Lifetime Achievement Award lecture. Two industry-sponsored studies presented at EHA 2025 point to the next wave of innovation. RedirecTT-1: Dual-Antigen BsAb Combination The phase 2 RedirecTT-1 trial evaluated the combination of talquetamab and teclistamab in patients with extramedullary disease. 'Patients with true extramedullary disease are up to 87% less likely to respond to conventional therapy,' said Shaji Kumar, MD, consultant, professor, and researcher at the Mayo Clinic, Rochester, USA. The combination treatment in the trial yielded an ORR of 78.9% and a complete response rate of 54.4%, with a 12-month PFS rate of 61% and OS rate of 74.5%. Adverse events were not significantly higher than in monotherapy trials, and less frequent dosing (biweekly or monthly) improved tolerability. 'These results showed deep and durable responses in a population with a significant unmet need,' Kumar said. Moreau, who was not involved in the study, added, 'This could be the pivotal trial that leads to approval of the first bsAb combination.' JNJ-5322: First-in-Human Trispecific Antibody JNJ-5322, a trispecific antibody targeting both BCMA and GPRC5D, showed remarkable efficacy in BCMA/GPRC5D-naive patients in a phase 1 trial. 'Despite recent progress, we still need to reduce treatment burden and improve outcomes,' said Rakesh Popat, MD, hematologist at University College Hospital, London, UK. Among patients with triple-class exposed RRMM, the ORR was 100%, with a 70.4% complete response rate and 12-month PFS of 95%. Grade 3/4 infections occurred in 28.6% of patients, but the safety profile — including mild cytokine release syndrome — was manageable. 'JNJ-5322 demonstrated manageable safety and an ORR comparable to CAR T, with convenient, off-the-shelf, weekly dosing, with one step-up dosing to facilitate outpatient dosing,' Popat concluded. Popat, Clavreul, and Kumar reported no relevant financial relationships. Moreaureported honoraria from and advisory board memberships with Janssen, Celgene, Takeda, Amgen, AbbVie, Sanofi, Pfizer, and GSK. Rodriguez Otero reported honoraria from lectures from BMS-Celgene, J&J Innovative Medicines, Sanofi, GSK, Regeneron, and Pfizer; participation in Ad Board meetings for BMS, Janssen, Sanofi, Oncopeptides, Pfizer, Roche, Regeneron, AbbVie, AstraZeneca, H3 Biomedicine, and GSK; consultancy work for BMS-Celgene, AbbVie, Roche, J&J Innovative Medicines, and Pfizer; and research funding and travel support from Pfizer. San Miguel Izquierdo reported participation on advisory boards and consulting services, on behalf of his institution, for AbbVie, Amgen, BMS, Celgene, GSK, Haemalogix, Janssen-Cilag, Karyopharm, MSD, Novartis, Pfizer, Takeda, Regeneron, Roche, Sanofi, Secura Bio, and Gilead-Kite. The two industry-sponsored studies mentioned were funded by Johnson & Johnson.
Yahoo
17-06-2025
- Business
- Yahoo
Hemogenyx Pharmaceuticals PLC Announces Clearance to Proceed with Pediatric Expansion
THIS ANNOUNCEMENT CONTAINS INSIDE INFORMATION AS STIPULATED UNDER THE UK VERSION OF THE MARKET ABUSE REGULATION NO 596/2014 WHICH IS PART OF ENGLISH LAW BY VIRTUE OF THE EUROPEAN (WITHDRAWAL) ACT 2018, AS AMENDED. ON PUBLICATION OF THIS ANNOUNCEMENT VIA A REGULATORY INFORMATION SERVICE, THIS INFORMATION IS CONSIDERED TO BE IN THE PUBLIC DOMAIN. Regulatory Clearance to Proceed with Pediatric Expansion of HG-CT-1 Clinical Trial LONDON, UK / / June 17, 2025 / Hemogenyx Pharmaceuticals plc (LSE:HEMO) is pleased to announce that the 30-day review period by the U.S. Food and Drug Administration (FDA) for the Company's previously submitted amendment to the clinical protocol of its ongoing Phase I trial of HG-CT-1 has concluded without a clinical hold. As a result, the Company is now cleared to proceed with the next steps required to initiate pediatric enrolment in the trial. The protocol amendment expands the eligibility criteria for the Phase I trial of HG-CT-1, Hemogenyx's proprietary CAR-T therapy for relapsed/refractory acute myeloid leukemia (R/R AML), to include children and adolescents with this aggressive and hard-to-treat disease. The Company will now move forward with Institutional Review Board (IRB) submissions and associated site activation procedures to enable the opening of pediatric cohorts. Dr. Vladislav Sandler, CEO & Co-Founder of Hemogenyx Pharmaceuticals, commented:"Regulatory clearance to proceed with the pediatric expansion of our HG-CT-1 clinical trial is a significant milestone. It reflects continued momentum in our development program and underscores our commitment to delivering innovative therapies to patients across age groups. This expansion broadens the potential impact and value of HG-CT-1 as we continue to advance toward key inflection points in the clinic. Importantly, it also reinforces the value of Company's robust intellectual property portfolio, which underpins our pipeline and supports long-term strategic growth." The Company will update the market as further progress is made in pediatric site activation and patient enrolment. Market Abuse Regulation (MAR) Disclosure Certain information contained in this announcement would have been inside information for the purposes of Article 7 of Regulation No 596/2014 (as it forms part of UK domestic law by virtue of the European Union (Withdrawal) Act 2018) until the release of this announcement. The person responsible for arranging for the release of this announcement on behalf of Hemogenyx Pharmaceuticals plc is Dr Vladislav Sandler, Chief Executive Officer & Co-Founder. Enquiries: Hemogenyx Pharmaceuticals plc Dr Vladislav Sandler, Chief Executive Officer & Co-Founder headquarters@ Peter Redmond, Director SP Angel Corporate Finance LLP Tel: +44 (0)20 3470 0470 Matthew Johnson, Vadim Alexandre, Adam Cowl Peterhouse Capital Limited Tel: +44 (0)20 7469 0930 Lucy Williams, Duncan Vasey, Charles Goodfellow About Hemogenyx Pharmaceuticals plc Hemogenyx Pharmaceuticals is a publicly traded company (LSE: HEMO) headquartered in London, with its US operating subsidiaries, Hemogenyx Pharmaceuticals LLC and Immugenyx LLC, located in New York City at its state-of-the-art research facility. The Company is a clinical stage biopharmaceutical group developing new medicines and treatments to treat blood and autoimmune diseases. Hemogenyx Pharmaceuticals is developing several distinct and complementary product candidates, as well as platform technologies that it uses as engines for novel product development. This information is provided by RNS, the news service of the London Stock Exchange. RNS is approved by the Financial Conduct Authority to act as a Primary Information Provider in the United Kingdom. Terms and conditions relating to the use and distribution of this information may apply. For further information, please contact rns@ or visit SOURCE: Hemogenyx Pharmaceuticals PLC View the original press release on ACCESS Newswire
Yahoo
16-06-2025
- Business
- Yahoo
AbelZeta Announces Promising Long-Term Outcomes for C-CAR039, a Novel CD19/CD20 Bi-Specific CAR-T Therapy, in Patients with R/R B NHL at EHA 2025
MILAN, June 16, 2025 /PRNewswire/ -- AbelZeta Pharma, Inc. ("AbelZeta" or the "Company"), a global clinical-stage biopharmaceutical company focused on discovery and development of innovative and proprietary cell-based therapeutic products, presented promising four-year clinical outcomes from 48 relapsed or refractory B-cell non-Hodgkin lymphoma (R/R B-NHL) patients treated with C-CAR039 (also known as Prizloncabtagene Autoleucel) in greater China, at the European Hematology Association (EHA) 2025 Congress. The study evaluated the safety and efficacy of C-CAR039, a novel anti-CD19/CD20 bi-specific CAR-T therapy. The dual-targeting approach to both CD20 and CD19 antigens aims to enhance therapeutic efficacy and reduce the likelihood of antigen escape, a common mechanism of resistance in B-cell malignancies. Key Findings: The overall response rate (ORR) and complete response (CR) rate were 91.5% and 85.1%, respectively for all patients Among the 43 LBCL pts, ORR and CR were 90.7% and 86.0%, respectively With a median follow-up of 45.5 months (range, 3.1–62.8), the median duration of response (DOR), progression-free survival (PFS), and overall survival (OS) were not reached KM estimates of 48-m PFS rate are 52.5% for all patients and 53.4% for LBCL patients, respectively KM estimates of 48-m OS rate are 65.4% for all patients and 66.7% for LBCL patients, respectively Safety Highlights: CRS of any grade: 93.8% | Grade ≥3: 2.1% ICANS of any grade: 6.3% | No severe cases (Grade ≥3) reported No new safety signals were observed with longer follow-up C-CAR039 has continued to show an excellent safety profile and deep and durable responses in R/R B-NHL patients, especially in those with R/R LBCL. These results continue to provide strong support for the curative potential of C-CAR039 as a next-generation therapy for R/R B-NHL. Late-stage clinical development is ongoing. "The long-term clinical outcomes data for C-CAR039 are highly encouraging, demonstrating sustained responses in patients with relapsed or refractory B-cell non-Hodgkin lymphoma," said Tony (Bizuo) Liu, Chairman and Chief Executive Officer of AbelZeta. "These results underscore our commitment and corporate goal to develop transformative therapies that address significant unmet medical needs in cancer." Dr. Yihong Yao, Chief Scientific Officer of AbelZeta, commented, "The strong clinical results of C-CAR039 demonstrate the added value of targeting CD20 in addition to CD19 in aggressive B-NHL. By addressing the limitations of single-antigen therapies - such as CD19 antigen escape - C-CAR039 enables deeper tissue penetration and more complete B-cell depletion. The durable responses observed support the continued advancement of C-CAR039 as a promising new treatment for patients with aggressive B-cell malignancies." For more information on the study and access to the poster presentation, please visit the EHA Library poster here. AbelZeta previously presented 30-month Phase 1 clinical trial results at the 65th American Society of Hematology (ASH) Annual Meeting in December 2023, through oral presentation #1025, as well as at EHA 2024, through poster presentation #P1475. The data with 30 months follow up was published in Blood 2025 Apr 3;145(14):1526-1535. AbelZeta Inc. (formerly known as Cellular Biomedicine Group, Inc.), entered into a worldwide collaboration and licensing agreement with Johnson & Johnson* in 2023 to develop and commercialize next-generation CAR-T cell therapies (excluding Greater China). The Phase 1 study for C-CAR039 was conducted by AbelZeta in China for the treatment of patients with B-cell non-Hodgkin lymphoma. Johnson & Johnson is evaluating the safety and efficacy of this asset (known as JNJ-90014496 outside of Greater China) through a separate study involving a global patient population. Phase 1b data for JNJ-90014496 were presented at EHA 2025 on Friday, June 13, informing the recommended Phase 2 dose and an initial assessment of safety, efficacy and pharmacokinetics in R/R LBCL patients. For more information, please visit Johnson and Johnson website: *The legal entity to the worldwide collaboration and license agreement is Janssen Biotech, Inc., a Johnson & Johnson company. About AbelZeta Pharma, is a global clinical-stage biopharmaceutical company with centers of excellence in Rockville, Maryland and Shanghai, China. AbelZeta is focusing on developing innovative and proprietary cell-based therapeutic products and is committed to ushering in bespoke treatments that harness the body's own immune system to fight against hematological malignancies and solid tumors, as well as inflammatory and immunological diseases. AbelZeta advances research and development in its own GMP facilities at its centers of excellence for early-stage clinical studies, with a pipeline comprised of CAR-T therapies. Forward-Looking StatementsStatements in this communication relating to plans, strategies, specific activities, and other statements that are not descriptions of historical facts are forward-looking statements. Forward-looking information is inherently subject to risks and uncertainties, and actual results could differ materially from those currently anticipated due to a number of factors, which include any risks detailed from time to time in the Company's reports. Such statements are based on the management's current beliefs and expectations and are subject to significant risks and uncertainties outside of management and the Company's control. Given these uncertainties, you should not place undue reliance on these forward-looking statements, which speak only as of the date hereof. Except as otherwise required by law, the Company does not undertake any obligation, and expressly disclaims any obligation, to update, alter or otherwise revise any forward-looking statements, whether written or oral, that may be made from time to time, whether as a result of new information, future events or otherwise. Company Contact:Sarah KellyDirector of CommunicationsAbelZeta Pharma, Inc.+1 (240) 552 View original content to download multimedia: SOURCE AbelZeta Pharma, Inc.
