Earlier use of CARVYKTI®▼ (ciltacabtagene autoleucel) demonstrated lasting treatment-free remissions at 2.5 years in patients with relapsed or refractory multiple myeloma
Follow-up data from CARTITUDE-4 show at least 80 percent of as-treated standard-risk patients remained progression- and treatment-free following a single infusion as early as second line1
Data suggest stronger immune fitness in earlier lines may be associated with longer progression-free survival2
BEERSE, BELGIUM, Dec. 06, 2025 (GLOBE NEWSWIRE) — Johnson & Johnson today announced updated results from the Phase 3 CARTITUDE-4 study supporting durable treatment-free remissions as early as second line treatment with CARVYKTI®▼ (ciltacabtagene autoleucel; cilta-cel).1 In 80 percent of as-treated patients with relapsed or refractory multiple myeloma (RRMM) and standard-risk cytogenetics who were treated with cilta-cel as early as first relapse, the disease did not progress and no further treatment was required at 2.5 years (30 months).1 These results add to the body of clinical and real-world experience established across more than 9,000 patients treated with cilta-cel globally.3
Additional translational analyses demonstrated that patients receiving cilta-cel in earlier lines had improved immune fitness, which suggests a correlation with longer progression-free survival (PFS).2 These data (Abstracts #92, #94) were featured in oral presentations at the 2025 American Society of Hematology (ASH) Annual Meeting.1,2
“These data suggest that a single infusion of cilta-cel for standard-risk patients may provide additional benefit to patients as early as second line of therapy,” said Luciano J. Costa, M.D., Ph.D., Professor of Medicine at the University of Alabama and principal investigator of the CARTITUDE-4 study.* “Treating patients with multiple myeloma after first relapse offers the opportunity to achieve deeper and more durable responses, shifting the treatment paradigm closer to the possibility of long-term remission and, ultimately, cure.”
“Our goal is to treat patients as early as possible, when they have the best chance for lasting remission,” said Jordan Schecter, M.D., Vice President, Research & Development, Multiple Myeloma, Johnson & Johnson Innovative Medicine. “With more than 9,000 patients treated globally, cilta-cel has demonstrated robust efficacy as soon as first relapse and is the only CAR-T to significantly extend overall survival versus standard therapies.”
In the analysis of CARTITUDE-4 data, 176 patients received cilta-cel as early as second line and 59 of those patients had standard-risk cytogenetics.1,4 At a median follow-up of 33.6 months, the 30-month PFS rate among the standard-risk patients in the as-treated population appeared to plateau at 80.5 percent (95 percent confidence interval [CI], 67.2–88.8) following a single infusion of cilta-cel.1,4 Notably, all 26 patients (100 percent) from this group who achieved minimal residual disease (MRD)-negative complete response at 12 months following cilta-cel infusion remained progression-free at 30 months.1
With an additional median follow-up, the safety profile in CARTITUDE-4 was consistent with the known safety profile of cilta-cel, where 97 percent of patients in both the cilta-cel and standard of care treatment arms experienced Grade 3/4 treatment-emergent adverse events (TEAEs), with cytopenia being the most common.1,5 In patients with standard-risk cytogenetics, non-haematologic serious adverse events occurred in 52.5 percent, 28.8 percent experienced Grade 3/4 infections, 74.6 percent experienced cytokine release syndrome (CRS), 1.7 percent experienced immune effector cell (IEC)-associated neurotoxicity syndrome (ICANS) and 6.8 percent cranial nerve palsy (CNP).1 There were no cases of IEC-parkinsonism and 10.2 percent non-relapse mortality after one year.1
Additionally, a translational analysis evaluated the relationship between immune biomarkers and PFS in patients treated with cilta-cel in CARTITUDE-1 and CARTITUDE-4.2 Using cilta-cel after one or two prior lines of therapy demonstrated stronger immune fitness versus patients with three or more prior lines of therapy, characterised by increased baseline CD4+ naïve T-cells (a type of immune cell that has not encountered an antigen) in peripheral blood.2 Bone marrow tumour analyses from patients treated with cilta-cel in CARTITUDE-4 also demonstrated a more immune-activated profile in patients treated after one prior line of therapy versus three.2 These biomarker data identify potential immunologic factors associated with longer PFS and support the improved survival outcomes exhibited with cilta-cel for patients treated as early as second line.2
“Our mission in multiple myeloma is to deliver transformational innovations that fundamentally shift the trajectory of disease and extend and improve patients’ lives,” said Ester in ‘t Groen, EMEA Therapeutic Area Head Haematology, Johnson & Johnson Innovative Medicine. “The data presented at ASH add to the growing body of evidence confirming the potential of cilta-cel to do just that. The important correlation between immune fitness and long-term progression-free survival reinforces our commitment to advancing this therapy earlier in the treatment pathway, where it has the greatest opportunity to maximise deeper, more durable outcomes for patients.”
As cilta-cel use has broadened across academic centres and community practices, Johnson & Johnson continues to collect and analyse clinical and real-world data to further characterise long-term remission outcomes and safety trends. This comprehensive experience across diverse patient populations provides an important foundation for expanding use into earlier treatment settings.
About CARTITUDE-1
CARTITUDE-1 (NCT03548207) is a Phase 1b/2, open-label, multicentre study that evaluated the efficacy and safety of cilta-cel in adults with relapsed and/or refractory multiple myeloma (RRMM), 99 percent of whom were refractory to the last line of treatment; 88 percent of whom were triple-class refractory, meaning their cancer did not or no longer responds to an immunomodulatory agent (IMiD), a proteasome inhibitor (PI) and an anti-CD38 antibody.6,7
The primary objective of the Phase 1b portion of the study, involving 29 patients, was to characterise the safety and confirm the dose of cilta-cel, informed by the first-in-human study with LCAR-B38M CAR-T cells (LEGEND-2).6 Based on the safety profile observed in this portion of the study, outpatient dosing is being evaluated in additional CARTITUDE studies. The Phase 2 portion of the study is evaluating the efficacy of cilta-cel with overall response as the primary endpoint.6 The study involved patients with heavily pretreated RRMM who historically have an expected median progression-free survival (PFS) of <6 months and median overall survival (OS) of ~1 year.6
About CARTITUDE-4
CARTITUDE-4 (NCT04181827) is the first international, randomised, open-label Phase 3 study evaluating the efficacy and safety of cilta-cel versus pomalidomide, bortezomib and dexamethasone (PVd) or daratumumab, pomalidomide and dexamethasone (DPd) in adult patients with relapsed and lenalidomide-refractory multiple myeloma who received one to three prior lines of therapy, including a PI and an IMiD.8 Patients were randomised to receive either a sequence of apheresis, bridging therapy, lymphodepletion and cilta-cel (n=208) or standard of care (SOC), which included PVd or DPd (n=211).9 The primary outcome measure for the study is PFS, defined as the time from the date of randomisation to the date of first documented disease progression, as defined in the International Myeloma Working Group (IMWG) criteria, or death due to any cause.8 Safety, OS, minimal residual disease (MRD) negativity rate and overall response rate are secondary endpoints.8
About Cilta-cel
Cilta-cel is a B-cell maturation antigen (BCMA)-directed, genetically modified autologous T-cell immunotherapy that involves reprogramming a patient’s own T-cells with a transgene encoding chimeric antigen receptor (CAR) that directs the CAR-positive T-cells to eliminate cells that express BCMA.9 BCMA is primarily expressed on the surface of malignant multiple myeloma B-lineage cells, as well as late-stage B-cells and plasma cells.10 The cilta-cel CAR protein features two BCMA-targeting single domains designed to confer high avidity against human BCMA.9 Upon binding to BCMA-expressing cells, the CAR promotes T-cell activation, expansion, and elimination of target cells.11
In April 2024, the European Commission (EC) approved an indication extension for cilta-cel for the treatment of adults with RRMM who have received at least one prior therapy, including an iMiD and a PI, have demonstrated disease progression on the last therapy, and are refractory to lenalidomide. In April 2024, cilta-cel was approved in the U.S. for the second line treatment of adult patients with relapsed or refractory myeloma who have received at least one prior line of therapy including a PI, an iMiD, and who are refractory to lenalidomide.
In December 2017, Janssen Biotech, Inc., a Johnson & Johnson company, entered into an exclusive worldwide licence and collaboration agreement with Legend Biotech USA, Inc., to develop and commercialise cilta-cel.12
For a full list of adverse events and information on dosage and administration, contraindications and other precautions when using cilta-cel please refer to the Summary of Product Characteristics.9 ▼In line with European Medicines Agency (EMA) regulations for new medicines and those given conditional approval, cilta-cel is subject to additional monitoring.9
About Multiple Myeloma
Multiple myeloma is currently an incurable blood cancer that affects a type of white blood cell called plasma cells, which are found in the bone marrow.13,14 In multiple myeloma, these plasma cells continue to proliferate, accumulating in the body and crowding out normal blood cells, often causing bone destruction and other serious complications.13,14 In the European Union, it is estimated that more than 35,000 people were diagnosed with multiple myeloma in 2022, and more than 22,700 patients died.15 Patients living with multiple myeloma experience relapses which become more frequent with each line of therapy while remissions become progressively shorter.16,17,18 Whilst some people diagnosed with multiple myeloma initially have no symptoms, others can have common signs and symptoms of the disease, which can include bone fracture or pain, low red blood cell counts, fatigue, high calcium levels, infections, or kidney problems.19,20
About Johnson & Johnson
At Johnson & Johnson, we believe health is everything. Our strength in healthcare innovation empowers us to build a world where complex diseases are prevented, treated, and cured, where treatments are smarter and less invasive, and solutions are personal. Through our expertise in Innovative Medicine and MedTech, we are uniquely positioned to innovate across the full spectrum of healthcare solutions today to deliver the breakthroughs of tomorrow, and profoundly impact health for humanity.
Learn more at www.innovativemedicine.jnj.com/emea. Follow us at www.linkedin.com/company/jnj-innovative-medicine-emea.
Cautions Concerning Forward-Looking Statements
This press release contains “forward-looking statements” as defined in the Private Securities Litigation Reform Act of 1995 regarding product development and the potential benefits and treatment impact of cilta-cel. The reader is cautioned not to rely on these forward-looking statements. These statements are based on current expectations of future events. If underlying assumptions prove inaccurate or known or unknown risks or uncertainties materialise, actual results could vary materially from the expectations and projections of Johnson & Johnson. Risks and uncertainties include, but are not limited to: challenges and uncertainties inherent in product research and development, including the uncertainty of clinical success and of obtaining regulatory approvals; uncertainty of commercial success; competition, including technological advances, new products and patents attained by competitors; challenges to patents; changes in behaviour and spending patterns of purchasers of health care products and services; changes to applicable laws and regulations, including global health care reforms; and trends toward health care cost containment. A further list and descriptions of these risks, uncertainties and other factors can be found in Johnson & Johnson’s most recent Annual Report on Form 10-K, including in the sections captioned “Cautionary Note Regarding Forward-Looking Statements” and “Item 1A. Risk Factors,” and in Johnson & Johnson’s subsequent Quarterly Reports on Form 10-Q and other filings with the Securities and Exchange Commission. Copies of these filings are available online at http://www.sec.gov/, http://www.jnj.com/ or on request from Johnson & Johnson. Johnson & Johnson does not undertake to update any forward-looking statement as a result of new information or future events or developments.
* Luciano J. Costa, M.D., Ph.D. Professor of Medicine at the University of Alabama and principal investigator of the CARTITUDE-4 study, has provided consulting, advisory, and speaking services to Johnson & Johnson; he has not been paid for any media work.
1 Costa L, et al. Long-Term Progression-Free Survival Benefit With Ciltacabtagene Autoleucel in Standard-Risk Relapsed/Refractory Multiple Myeloma. Oral presentation. American Society of Hematology (ASH) Annual Meeting; 06-09 December, 2025.
2 Parekh S Li K, et al. Earlier use of Ciltacabtagene Autoleucel (Cilta-cel) is Associated With Better Immune Fitness and Stronger Immune Effects as Shown by Correlative Analysis of Peripheral Blood and the Bone Marrow Tumor Microenvironment (TME) From the CARTITUDE-4 Study. Oral presentation. American Society of Hematology (ASH) Annual Meeting; 06-09 December, 2025.
3 Legend Biotech. Legend Biotech announces 9 presentations at the 67th American Society of Hematology Annual Meeting. Available at: https://investors.legendbiotech.com/news-releases/news-release-details/legend-biotech-announces-10-presentations-67th-american-society. Last accessed: December 2025.
4 Costa L, et al. Long-Term Progression-Free Survival Benefit With Ciltacabtagene Autoleucel in Standard-Risk Relapsed/Refractory Multiple Myeloma. Abstract. American Society of Hematology (ASH) Annual Meeting; 06-09 December, 2025.
5 Mateos MV, et al. Overall Survival (OS) With Ciltacabtagene Autoleucel (Cilta-cel) Versus Standard of Care (SoC) in Lenalidomide (Len)-Refractory Multiple Myeloma (MM): Phase 3 CARTITUDE-4 Study Update. International Myeloma Society 2024 Annual Meeting. September 2024.
6 ClinicalTrials.gov. A Study of JNJ-68284528, a Chimeric Antigen Receptor T Cell (CAR-T) Therapy Directed Against B-Cell Maturation Antigen (BCMA) in Participants With Relapsed or Refractory Multiple Myeloma (CARTITUDE-1). Available at: Study Details | A Study of JNJ-68284528, a Chimeric Antigen Receptor T Cell (CAR-T) Therapy Directed Against B-Cell Maturation Antigen (BCMA) in Participants With Relapsed or Refractory Multiple Myeloma | ClinicalTrials.gov. Last accessed: December 2025.
7 Lin Y, et al. CARTITUDE-1 final results: Phase 1b/2 study of ciltacabtagene autoleucel in heavily pretreated patients with relapsed/refractory multiple myeloma. Oral presentation. American Society of Clinical Oncology (ASCO) Annual Meeting 2023.
8 ClinicalTrials.gov. A Study Comparing JNJ-68284528, a CAR-T Therapy Directed Against B-cell Maturation Antigen (BCMA), Versus Pomalidomide, Bortezomib and Dexamethasone (PVd) or Daratumumab, Pomalidomide and Dexamethasone (DPd) in Participants With Relapsed and Lenalidomide-Refractory Multiple Myeloma (CARTITUDE4). Available at: https://clinicaltrials.gov/study/NCT04181827. Last accessed: December 2025.
9 European Medicines Agency. CARVYKTI (ciltacabtagene autoleucel) Summary of Product Characteristics. Available at: https://www.ema.europa.eu/en/documents/product-information/carvykti-epar-product-information_en.pdf. Last accessed: December 2025.
10 Cho SF, et al. Targeting B Cell Maturation Antigen (BCMA) in Multiple Myeloma: Potential Uses of BCMA-Based Immunotherapy. Front Immunol 2018;10(9):1821.
11 Tai YT, et al. Targeting B-cell maturation antigen in multiple myeloma. Immunotherapy 2015;7(11):1187-1199.
12 Johnson & Johnson.com. Janssen Enters Worldwide Collaboration and License Agreement with Chinese Company Legend Biotech to Develop Investigational CAR-T Anti-Cancer Therapy. Available at: https://www.jnj.com/media-center/press-releases/janssen-enters-worldwide-collaboration-and-license-agreement-with-chinese-company-legend-biotech-to-develop-investigational-car-t-anti-cancer-therapy. Last accessed: December 2025.
13 Abdi J, et al. Drug resistance in multiple myeloma: latest findings on molecular mechanisms. Oncotarget 2013;4(12):2186-2207.
14 American Society of Clinical Oncology. Multiple myeloma: introduction. Available at: https://www.cancer.net/cancer-types/multiple-myeloma/introduction. Last accessed: December 2025.
15 ECIS. European Cancer Information System. Estimates of cancer incidence and mortality in 2022, by country. Multiple myeloma. Available at: https://ecis.jrc.ec.europa.eu/explorer.php?$0-0$1-All$2-All$4-1,2$3-51$6-0,85$5-2022,2022$7-7$CEstByCountry$X0_8-3$X0_19-AE27$X0_20-No$CEstBySexByCountry$X1_8-3$X1_19-AE27$X1_-1-1$CEstByIndiByCountry$X2_8-3$X2_19-AE27$X2_20-No$CEstRelative$X3_8-3$X3_9-AE27$X3_19-AE27$CEstByCountryTable$X4_19-AE27. Last accessed: December 2025.
16 Bhatt P, et al. Relapsed/Refractory Multiple Myeloma: A Review of Available Therapies and Clinical Scenarios Encountered in Myeloma Relapse. Curr Oncol. 2023;30(2):2322-2347.
17 Hernández-Rivas JÁ, et al. The changing landscape of relapsed and/or refractory multiple myeloma (MM): fundamentals and controversies. Biomark Res. 2022;10(1):1-23.
18 Gavriatopoulou M, et al. Metabolic Disorders in Multiple Myeloma. Int J Mol Sci. 2021;22(21):11430.
19 American Cancer Society. What is Multiple Myeloma? https://www.cancer.org/cancer/multiple-myeloma/about/what-is-multiple-myeloma.html. Last accessed: December 2025.
20 American Cancer Society. Multiple Myeloma Early Detection, Diagnosis, and Staging. https://www.cancer.org/cancer/types/multiple-myeloma/detection-diagnosis-staging/detection.html. Last accessed: December 2025.
December 2025
CP-554345
CONTACT: Media contact: Jenni Mildon jmildon@its.jnj.com +44 7920 418 552 Investor contact: Lauren Johnson investor-relations@its.jnj.com
