Ciltacabtagene autoleucel

Half of the patients with relapsed or refractory multiple myeloma (RRMM) who received a single dose of Johnson & Johnson and Legend Biotech's CAR-T cell therapy Carvykti (ciltacabtagene autoleucel) remained alive after five years, the partners reported Friday. The data were shared amidst a leadership overhaul for Legend after Ying Huang unexpectedly stepped down as the biotech's chief executive in July. His successor was named earlier this month as Legend tapped former Novartis executive Ingrid Zhang for the role.Legend also recently missed out on a chance to deepen its partnership with J&J. Although the company shared promising early data for its in vivo CAR-T cell therapy LB2501, J&J opted to instead ink an in vivo CAR-T pact with Sail Biomedicines (see – Vital Signs: J&J's Legend(ary) snub weighs on Carvykti).No progression, detectable diseaseThe long-term data for Carvykti, presented at the International Myeloma Society (IMS) meeting, were generated in Cohort A of the Phase II CARTITUDE-2 trial. The study recruited 20 patients with RRMM who had received between one and three prior lines of therapy — and were exposed to a proteasome inhibitor and refractory to Revlimid (lenalidomide) — to receive a single infusion of the BCMA-directed CAR-T. After a median follow-up of 60.7 months, 10 patients were alive and progression-free, without maintenance therapy. Of those patients, five had at least one high-risk disease feature."These results add to the growing body of evidence suggesting that Carvykti may have curative potential for some patients," said Yusri Elsayed, J&J's global therapeutic area head of oncology.Carvykti demonstrated a five-year overall survival rate of 69.2%, with patients achieving a median progression-free survival of 60.5 months. While CARTITUDE-2's primary endpoint was minimal residual disease (MRD) negativity, only three participants were assessed at the five-year mark. All three were MRD negative, with no detectable disease "at the deepest level tested," J&J said. Carvykti was initially approved in 2022 for use in the fourth-line setting based on results from the Phase Ib/II CARTITUDE-1 study, where a one-time infusion in RRMM patients with a median of six prior treatments achieved a 98% response rate. The CAR-T's label was then extended to second-line disease in 2024. "These new Carvykti findings provide additional evidence that when highly effective, established therapies are used earlier in the disease course, more patients may have the opportunity to achieve deep, durable remissions and long-term disease control without maintenance," said Niels van de Donk, a professor of haematology at the University Medical Center in Amsterdam.

New results from cohort A of the CARTITUDE-2 study, among 20 patients, suggest earlier treatment may increase the likelihood of long-term remission 1 Study adds to Johnson & Johnson's overwhelming body of evidence supporting its innovative multiple myeloma therapies in second line, where earlier intervention may increase long-term remission and disease control 2 BEERSE, BELGIUM, Sept. 25, 2026 (GLOBE NEWSWIRE) -- Johnson & Johnson, a worldwide leader in multiple myeloma, announced today new long-term follow-up data from the initial subgroup of cohort A in the Phase 2 CARTITUDE-2 study (N=20) showing that 50% of patients (10/20) in early line relapsed or refractory multiple myeloma (RRMM) who were treated with a single infusion of CARVYKTI ® ▼ (ciltacabtagene autoleucel; cilta-cel) remained alive and progression-free for at least five years without maintenance therapy. 1 These results build on the durable, treatment-free remissions observed in the CARTITUDE-1 study, which evaluated patients in later lines of therapy, and reinforce that earlier treatment with cilta-cel may increase long-term remission and disease control. 1 Together these findings add to the emerging evidence suggesting that cilta-cel may have curative potential in some patients. Findings were presented at the International Myeloma Society (IMS) Annual Meeting (Abstract #PA-288). 1 Expert and company perspectives on earlier treatment-free remissions with cilta-cel "Five years ago, treatment-free remissions of this duration were difficult to imagine for many patients with relapsed or refractory multiple myeloma," said Niels van de Donk, M.D., Ph.D., Professor of Hematology, University Medical Center, Amsterdam, the Netherlands.* "These new cilta-cel findings provide additional evidence that when highly effective, established therapies are used earlier in the disease course, more patients may have the opportunity to achieve deep, durable remissions and long-term disease control without maintenance." “These results add to the growing body of evidence suggesting that cilta-cel may have curative potential for some patients and reinforce the value of bringing highly effective therapies to patients earlier in their treatment journey,” said Yusri Elsayed, M.D., M.H.Sc., Ph.D., Global Therapeutic Area Head, Oncology, Johnson & Johnson. “As we continue to advance a portfolio of complementary and combinable therapies, these findings reinforce our commitment to pursuing curative approaches and redefining what patients can expect from multiple myeloma treatment.” “While outcomes for patients with multiple myeloma have improved significantly in recent years, unmet needs remain for patients with relapsed or refractory disease, when the disease can become harder to control and achieving sustained remission becomes more challenging,” said Ester in ‘t Groen, EMEA Therapeutic Area Head, Haematology, Johnson & Johnson. “A single infusion of cilta-ceI, a CAR-T cell therapy designed to harness a patient’s own immune system to target myeloma cells, has demonstrated the potential to deliver deep and durable responses. Bringing this innovative treatment into earlier lines of care could address an important unmet need at a critical point in the treatment journey and entirely redefine the trajectory, and outcome of disease.” Phase 2 CARTITUDE-2 study build on previous findings CARTITUDE-2 cohort A (N=20) enrolled patients with RRMM who had received one to three prior lines of therapy, were exposed to a proteasome inhibitor, and were refractory to lenalidomide. 1 The primary endpoint was minimal residual disease (MRD) negativity. 1, 3 Patients from initial cohort A were followed to assess long-term outcomes following a single cilta-cel infusion without maintenance therapy. 1 At a median follow-up of 60.7 months: 1 Half of the patients (n=10, 50%) remained alive and progression-free at five years The five-year overall survival rate was 69.2% Median progression-free survival was 60.5 months Patients received a single cilta-cel infusion without maintenance therapy At five years, MRD was assessed by bone marrow in three patients and was not required per protocol. 1 All three patients were MRD-negative at the deepest level tested (10 -6 ), indicating no detectable disease using highly sensitive testing methods. 1 Long-term remissions were observed even among patients with high-risk disease features. Of the 10 patients who remained alive and progression-free at five years, five had at least one high-risk cytogenetic abnormality, including four patients with two or more high-risk features. 1 The safety pro with longer follow-up was consistent with the known safety pro cilta-cel, with no new CAR T-cell-related neurotoxicity reported. 1 Since the previous analysis, one patient developed a new haematologic malignancy (acute myeloid leukaemia) and two deaths occurred due to progressive disease and new cancer. 1 About CARTITUDE-2 CARTITUDE-2 ( NCT04133636 ) is an ongoing, multicohort Phase 2 study evaluating cilta-cel in patients across different multiple myeloma disease settings. Cohort A includes patients with relapsed or refractory multiple myeloma (RRMM) who have received 1–3 prior lines of therapy, including an immunomodulatory drug (IMiD) and a proteasome inhibitor (PI), and are refractory to lenalidomide. 3 The primary endpoint is minimal residual disease (MRD) negativity; secondary endpoints include long-term progression-free survival (PFS), overall survival (OS) and safety. 3 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. 4 BCMA is primarily expressed on the surface of malignant multiple myeloma B-lineage cells, as well as late-stage B-cells and plasma cells. 5 The cilta-cel CAR protein features two BCMA-targeting single domains designed to confer high avidity against human BCMA. 4 Upon binding to BCMA-expressing cells, the CAR promotes T-cell activation, expansion, and elimination of target cells. 6 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 multiple 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. 7 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 . ▼ In line with European Medicines Agency (EMA) regulations for new medicines and those given conditional approval, cilta-cel is subject to additional monitoring. 4 About Multiple Myeloma Multiple myeloma is a complex blood cancer that affects a type of white blood cell called plasma cells, which are found in the bone marrow. 8 , 9 In multiple myeloma, these malignant plasma cells continue to proliferate, accumulating in the body and crowding out normal blood cells, as well as often causing bone destruction and other serious complications. 10 , 11 In the European Union, it is estimated that more than 35,000 people were diagnosed with multiple myeloma in 2024, and more than 21,900 patients died. 12 Patients living with multiple myeloma experience relapses which become more frequent with each line of therapy, while remissions become progressively shorter. 13 , 14 Whilst some patients 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 damage. 15 About Johnson & Johnson’s Multiple Myeloma Portfolio Johnson & Johnson is a global leader in multiple myeloma therapies, with a broad and differentiated portfolio designed to address the complexity and heterogeneity of the disease. Our portfolio spans multiple mechanisms of action, targets and treatment modalities, including CD38-directed therapies, BCMA- and GPRC5D-targeting bispecific antibodies, and cellular therapies. Over the past decade, Johnson & Johnson therapies have helped extend survival for patients with multiple myeloma. With the right medicines, used as early as possible, combined and sequenced for the best results, Johnson & Johnson is expanding treatment options to match the right therapy to the right patient at the right stage of disease and drive deeper and more durable responses. Through ongoing research and a robust clinical program, Johnson & Johnson is committed to transforming multiple myeloma into a more manageable condition that is no longer treated to progression but has the potential to, ultimately, be cured. 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 . Follow us at . Caution 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 , , 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. Key Search Terms CARVYKTI CARTITUDE-1 study Phase 2 CARTITUDE-2 study multiple myeloma long-term remission from multiple myeloma innovative multiple myeloma therapies minimal residual disease (MRD) negativity in multiple myeloma Footnote * Niels van de Donk, M.D., Ph.D., Professor of Hematology, University Medical Center, Amsterdam, Netherlands, has provided consulting, advisory, and speaking services to Johnson & Johnson; he has not been paid for any media work. 1 Cohen AD, et al. Long-Term (≥5-Year) Remission and Survival With Ciltacabtagene Autoleucel (cilta-cel) in Relapsed/Refractory Multiple Myeloma (RRMM) With 1-3 Prior Lines of Therapy: CARTITUDE-2 Cohort A. Poster presented at: 23rd International Myeloma Society Annual Meeting & Exposition; September 23-26, 2026; Glasgow, Scotland. 2 Johnson & Johnson.com. CHMP Recommends Approval of Johnson & Johnson’s TECVAYLI®▼ (teclistamab) in Relapsed/Refractory Multiple Myeloma After at Least One Prior Therapy. Available at: . Last accessed: September 2026. 3 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 Multiple Myeloma (CARTITUDE-2). . Last accessed: September 2026. 4 European Medicines Agency. CARVYKTI (ciltacabtagene autoleucel) Summary of Product Characteristics. Available at: . Last accessed: September 2026. 5 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. 6 Tai YT, et al. Targeting B-cell maturation antigen in multiple myeloma. Immunotherapy 2015;7(11):1187-1199. 7 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:  . Last accessed: September 2026. 8 Abdi J, et al. Drug Resistance in Multiple Myeloma: Latest Findings on Molecular Mechanisms. Oncotarget. 2013;4(12):2186-2207. 9 Myeloma UK. What is myeloma? Available at: . Last accessed: September 2026. 10 Lungu O, et al. Mechanistic insights into bone destruction in multiple myeloma: cellular and molecular perspectives. Journal of Bone Oncology. 2025;51:100668. 11 Mayo Clinic. Multiple Myeloma. Available at: . Last accessed: September 2026. 12 ECIS - European Cancer Information System. Estimates of Cancer Incidence and Mortality in 2024, by Country. Multiple Myeloma. Available at: . Last accessed: September 2026. 13 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. 14 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. 15 American Cancer Society. Multiple Myeloma: Early Detection, Diagnosis and Staging. Available at: . Last accessed: September 2026. CONTACT: Media contact: Jenni Mildon jmildon@its.jnj.com +44 7920 418 552 Investor contact: Jess Margevich investor-relations@its.jnj.com