Legend Biotech Unveils Landmark Five-Year Survival Data for CARVYKTI® in Relapsed/Refractory Multiple Myeloma at ASCO 2025
Legend Biotech Corporation,a global leader in cell therapy innovation, presented groundbreaking long-term data at the 2025 American Society of Clinical Oncology (ASCO) Annual Meeting, showcasing the sustained efficacy of its B-cell maturation antigen (BCMA)-targeting CAR T-cell therapy, CARVYKTI® (ciltacabtagene autoleucel; cilta-cel), in patients with relapsed or refractory multiple myeloma (RRMM).
The latest results come from the pivotal Phase 1b/2 CARTITUDE-1 study, which enrolled 97 heavily pretreated patients who received a single infusion of CARVYKTI® without any subsequent maintenance therapy. The most striking finding: 33% (32 of 97) of participants remained progression-free five years post-treatment, an unprecedented outcome in this difficult-to-treat population.
These long-term results, presented during an oral session (Abstract #7505), mark a significant milestone in the evolution of CAR T-cell therapy and provide compelling evidence that a one-time treatment with CARVYKTI® may lead to deep, durable remissions in patients with few remaining therapeutic options.
Five-Year Survival Outcomes Redefine Expectations
At a median follow-up of 61.3 months, the study reported a median overall survival (OS) of 60.7 months (95% confidence interval: 41.9–not estimable), illustrating the long-term benefit of CARVYKTI® in this patient population. Of particular note, the 32 patients who remained progression-free beyond five years did so without receiving any additional therapy for multiple myeloma.
This patient subset included individuals with high-risk disease features, such as:
- High-risk cytogenetics (23.3%)
- Extramedullary disease (12.5%)
- Triple-class refractory disease (90.6%)
- Penta-drug refractory disease (46.9%)
The median age in this long-term responder group was 60 years (range: 43–78), and participants had received a median of 6.5 prior lines of therapy (range: 3–14) before enrolling in the study. Notably, their median time to progression following their last prior therapy was only four months, underscoring the aggressive nature of their disease.
Deep Responses in MRD and Imaging Substudy
An important subset of 12 patients from a single study center underwent serial minimal residual disease (MRD) assessments and annual PET/CT scans. Remarkably, all 12:
- Remained progression-free for at least five years
- Achieved MRD negativity
- Showed no signs of disease recurrence on imaging over the five-year period
This level of durable response, verified through both molecular and imaging assessments, suggests that CARVYKTI® may lead to a state of functional cure in select patients.
Expert Perspective on Durability and Hope
Dr. Sundar Jagannath, a leading myeloma expert and Network Director at the Multiple Myeloma Center of Excellence at The Tisch Cancer Institute, Icahn School of Medicine at Mount Sinai, commented on the findings:
“The durability and consistency we’re seeing with CARVYKTI in the CARTITUDE-1 study is truly remarkable. These data offer real hope for long-term disease control in a population that previously had limited options.”
Such enthusiasm underscores the potential for CARVYKTI® to not only improve survival but also reshape expectations around treatment outcomes in relapsed/refractory multiple myeloma.
Safety Remains Manageable and Consistent
The updated safety profile remained consistent with prior reports. No new cases of movement or neurocognitive treatment-emergent adverse events (MNTs), such as parkinsonism, were observed. Importantly:
- Only two new cases of second primary malignancies (both solid tumors) were reported
- No emergent safety concerns were identified over the extended follow-up period
These findings continue to support a favorable benefit-risk profile for CARVYKTI®, especially when weighed against the otherwise poor prognosis of the enrolled patient population.
Peer-Reviewed Validation
Adding to the significance of the ASCO presentation, these five-year data were simultaneously published in the Journal of Clinical Oncology, reinforcing their impact and scientific rigor. The peer-reviewed publication offers the broader medical community deeper insights into the long-term performance of cilta-cel in a challenging patient setting.
CARVYKTI® in Context: Transforming the Myeloma Landscape
CARVYKTI® is the first and only CAR T-cell therapy for multiple myeloma to demonstrate a statistically significant overall survival benefit over standard of care in previously treated patients. As of the latest update, over 6,500 patients have received CARVYKTI® across various indications and clinical trials globally.
The therapy targets BCMA, a protein commonly expressed on multiple myeloma cells, using dual single-domain antibodies designed to enhance binding avidity and antitumor activity. Manufactured through a personalized process using a patient’s own T cells, CARVYKTI® has become a cornerstone in the rapidly evolving myeloma treatment paradigm.
Dr. Mythili Koneru, Chief Medical Officer at Legend Biotech, summarized the implications of the data:
“This five-year survival data highlights the potential of CARVYKTI to fundamentally change treatment expectations for patients with relapsed or refractory multiple myeloma. For one-third of these heavily pretreated patients to remain progression-free for five years after a single infusion—and without needing further myeloma therapy—represents a potential paradigm shift in how we treat relapsed or refractory multiple myeloma.”
The results from CARTITUDE-1 add momentum to ongoing studies evaluating CARVYKTI® in earlier lines of therapy and broader patient populations. With regulatory approvals in the U.S., Europe, and other global markets, Legend Biotech—along with its development and commercialization partner Janssen Biotech (a Johnson & Johnson company)—continues to expand access and refine CAR T-cell manufacturing to meet rising demand.
The CARTITUDE clinical program now includes studies such as:
- CARTITUDE-2 and CARTITUDE-4, which are exploring cilta-cel in earlier relapse settings
- CARTITUDE-5, which is testing the therapy in newly diagnosed patients ineligible for transplant
These efforts reflect a strategic aim to move CARVYKTI® closer to the frontlines of treatment, possibly even in combination or sequential strategies that sustain long-term remission or achieve cure.
Legend Biotech’s presentation of five-year CARTITUDE-1 results at ASCO 2025 represents a defining moment in the history of multiple myeloma treatment. CARVYKTI® has shown it can offer a substantial proportion of patients with aggressive, refractory disease a real chance at long-term progression-free survival—and potentially, a life free from further treatment. As cell therapy continues to evolve, this data reaffirms CARVYKTI®’s role at the forefront of therapeutic innovation in hematologic cancers.
