On September 25, 2026 Legend Biotech Corporation (NASDAQ: LEGN) (Legend Biotech), a global leader in cell therapy, reported five-year follow-up data from CARTITUDE-2 Cohort A (n=20) evaluating CARVYKTI (ciltacabtagene autoleucel; cilta-cel) in patients with relapsed/refractory multiple myeloma (RRMM) after one to three prior lines of therapy after a median follow-up of 60.7 months. These data were presented today at the 2026 International Myeloma Society (IMS) Annual Meeting in Glasgow, Scotland (Abstract #PA-288).
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The data showed that five years after a single CARVYKTI infusion, 10 of 20 patients (50%) remained alive and progression-free without maintenance therapy. The findings build on long-term outcomes previously observed in CARTITUDE-1 and suggest that earlier use of CARVYKTI may increase the likelihood of achieving durable treatment-free remission, further strengthening evidence supporting its potential to transform expectations in multiple myeloma.
"These five-year data, showing that 50% of patients remained alive and progression-free after a single CARVYKTI infusion, reinforce the growing body of evidence supporting the unique curative potential of CARVYKTI," said Alan Bash, President, CARVYKTI at Legend Biotech.
Multiple myeloma is a blood cancer that can place a significant physical and emotional burden on patients throughout the course of their disease. Patients may experience symptoms including bone pain, fatigue, weakness, infections, and kidney problems, which can affect their ability to work, remain active, and participate in everyday life. Multiple myeloma often requires ongoing treatment and may recur after periods of remission, leading patients to undergo multiple cycles of therapy over many years.i,ii
"To be more than five years out from a single treatment and still able to focus on living my life rather than my next therapy is something I never imagined when I was diagnosed," said Joe Rader, CARTITUDE-2 Cohort A participant. "When you live with multiple myeloma, you learn to measure time differently – from one treatment to the next, one scan to the next."
"For people living with multiple myeloma, the possibility of spending years without disease progression or the need for ongoing treatment can be incredibly meaningful," said Niels van de Donk, M.D., Ph.D., Professor of Hematology at Amsterdam UMC. "I believe these findings, that show half of the patients in the cohort remain progression- and treatment-free at five years, are unique in multiple myeloma and give us reason for optimism and deepen our understanding of what may be possible when CARVYKTI is used earlier in the treatment journey. Ultimately, our hope is to help more patients achieve lasting disease control and spend less time cycling through treatments."‡
Five-Year CARTITUDE-2 Cohort A Data Show Sustained Remission in Earlier-Line RRMM
With extended follow-up, patients continued to experience durable clinical benefit, with a median PFS of 60.5 months. Median overall survival was not reached at the time of analysis, and 69.2% of patients were alive at five years. With a median follow-up of 60.7 months, 10 of 20 patients (50%) remained alive and progression-free without further anti-myeloma treatment ≥5 years after CARVYKTI infusion.
The safety profile observed with longer follow-up was consistent with the known safety profile of CARVYKTI, with no new CAR T-cell-related neurotoxicity reported. Since the previous analysis of CARTITUDE-2 Cohort A with a median follow-up of approximately 30 months,iii one patient developed a new hematologic malignancy (acute myeloid leukemia) and two deaths occurred due to progressive disease and new cancer.
ABOUT CARVYKTI (CILTACABTAGENE AUTOLEUCEL; CILTA-CEL)
Ciltacabtagene autoleucel is a BCMA-directed, genetically modified autologous T-cell immunotherapy, which involves reprogramming a patient’s own T-cells with a transgene encoding a chimeric antigen receptor (CAR) that identifies and eliminates cells that express BCMA. The cilta-cel CAR protein features two BCMA-targeting single-domain antibodies designed to confer high avidity against human BCMA. Upon binding to BCMA-expressing cells, the CAR promotes T-cell activation, expansion, and elimination of target cells.iv
In December 2017, Legend Biotech entered into an exclusive worldwide license and collaboration agreement with Janssen Biotech, Inc., a Johnson & Johnson company, to develop and commercialize cilta-cel. In February 2022, cilta-cel was approved by the U.S. Food and Drug Administration (FDA) under the brand name CARVYKTI for the treatment of adults with relapsed or refractory multiple myeloma. In April 2024, cilta-cel was approved for the second-line treatment of patients with relapsed/refractory myeloma who have received at least one prior line of therapy, including a proteasome inhibitor, an immunomodulatory agent, and are refractory to lenalidomide.
In May 2022, the European Commission (EC) granted conditional marketing authorization of CARVYKTI for the treatment of adults with relapsed and refractory multiple myeloma. In September 2022, Japan’s Ministry of Health, Labour and Welfare (MHLW) approved CARVYKTI. Cilta-cel was granted Breakthrough Therapy Designation in the U.S. in December 2019 and in China in August 2020. In addition, cilta-cel received a PRIority MEdicines (PRIME) designation from the European Commission in April 2019. Cilta-cel also received Orphan Drug Designation from the U.S. FDA in February 2019, from the European Commission in February 2020, and from the Pharmaceuticals and Medicinal Devices Agency (PMDA) in Japan in June 2020. In March 2022, the European Medicines Agency’s Committee for Orphan Medicinal Products recommended by consensus that the orphan designation for cilta-cel be maintained on the basis of clinical data demonstrating improved and sustained complete response rates following treatment.
ABOUT MULTIPLE MYELOMA
Multiple myeloma is a blood cancer that starts when plasma cells, a type of white blood cell found in the bone marrow, become cancerous and grow.v In 2026, it is estimated that more than 36,000 people will be diagnosed with multiple myeloma, and more than 10,000 people will die from the disease in the U.S.vi While some patients with multiple myeloma initially have no symptoms, most patients are diagnosed due to symptoms that can include bone problems, low blood counts, calcium elevation, kidney problems, or infections.vii
ABOUT CARTITUDE-1
CARTITUDE-1 (NCT03548207) is a Phase 1b/2, open-label, multicenter study evaluating the safety and efficacy of cilta-cel in adults with relapsed and/or refractory with multiple myeloma who have received at least 3 prior lines of therapy or are double refractory to a PI and IMiD, received a PI, an IMiD, and anti-CD38 antibody and documented disease progression within 12 months of starting the most recent therapy. The primary objective of the Phase 1b portion of the study was to characterize the safety and confirm the recommended Phase 2 dose of cilta-cel, informed by the first-in-human study with LCAR-B38M CAR-T cells (LEGEND-2). The Phase 2 portion further evaluated the efficacy of cilta-cel with overall response rate as the primary endpoint.viii
ABOUT CARTITUDE-2
CARTITUDE-2 (NCT04133636) is an ongoing Phase 2 multicohort study evaluating the safety and efficacy of cilta-cel in patients with multiple myeloma across various clinical settings. CARTITUDE-2 Cohort A evaluated cilta-cel in patients who had received one to three prior lines of therapy, including a proteasome inhibitor and an immunomodulatory drug, and lenalidomide refractory. The primary objective of Cohort A was to evaluate the efficacy of cilta-cel, with minimal residual disease (MRD) negativity serving as the primary endpoint.
(Press release, Legend Biotech, SEP 25, 2026, View Source [SID1234671101])