Key Takeaways
- Carvykti Delivers Long-Term Remission: One-third of patients in the CARTITUDE-1 trial achieved at least five years of treatment-free survival after a single CAR T-cell therapy infusion.
- Sustained Efficacy in High-Risk Patients: The trial included heavily pretreated and triple-class refractory populations, with median overall survival reaching over five years.
- Data Supports Curative Potential: Johnson & Johnson aims to move from treatment to cure, with future results from CARTITUDE-4 expected to reinforce Carvykti’s benefit across subgroups.
Results from the Phase Ib/II CARTITUDE-1 trial show that a single infusion of Johnson & Johnson’s (J&J) Carvykti (ciltacabtagene autoleucel; cilta-cel) delivered treatment-free remissions for at least five years in a third of patients with relapsed or refractory multiple myeloma (RRMM). These findings were presented at the 2025 American Society of Clinical Oncology Annual Meeting and published in The Journal of Clinical Oncology.1
Can a Single CAR T-cell Therapy Infusion Offer Lasting Remission in Multiple Myeloma?
“This new evidence shows how a single infusion of Carvykti can help patients survive without disease progression much longer than previously thought possible in this setting, and without any maintenance or subsequent treatment,” said Peter M. Voorhees*, MD, clinical professor, hematology and oncology, Atrium Health, Levine Cancer Institute at Wake Forest University School of Medicine, in a press release. “In a heavily pre-treated population, a third of patients remained treatment- and progression-free for at least five years.”
Design and Objectives of the CARTITUDE-1 Trial
- The open-label, multicenter CARTITUDE-1 trial evaluated the efficacy and safety of Carvykti in 97 patients.
- Ninety-nine percent of patients in the trial were refractory to their last line of treatment, and 88% were triple-class refractory.
- For the Phase Ib portion, the primary endpoint was to characterize safety and confirm the dose of Carvykti, informed by the first-in-human study with LCAR-B38M CAR T cells.
- For the Phase II portion, the primary endpoint was overall response (OR). This phase included heavily pretreated patients, who historically experienced median progression-free survival (PFS) of over six months and median overall survival (OS) of around one year.
Durable Results Show Deep and Lasting Responses
- Results showed that of the 97 enrolled patients, 32 achieved five-year PFS without needing maintenance or subsequent anti-myeloma therapy.
- Median OS was 60.7 months at a median follow-up of 61.3 months.
Consistent Safety Profile and Adverse Event Monitoring
- The safety profile of Carvykti was consistent with previous studies after longer follow up, with no new safety signals identified.
- Nineteen deaths occurred post-infusion, with three due to disease progression and 16 due to adverse events (AEs).
- The most common AEs resulted from infections.
- Common nonlaboratory AEs included pyrexia; cytokine release syndrome; hypogammaglobulinemia; hypotension; musculoskeletal pain; fatigue; infections-pathogen unspecified; cough; chills; diarrhea; nausea; encephalopathy; decreased appetite; upper respiratory tract infection; headache; tachycardia; dizziness; dyspnea; edema; viral infections; coagulopathy; constipation; and vomiting.1
Multiple Myeloma Remains a Significant Clinical Challenge
According to the American Cancer Society, the lifetime risk of developing multiple myeloma (MM) in the United States is less than 1%, including a one in 108 chance for men and a one in 133 chance for women. By the end of 2025, an estimated 36,110 new cases will be diagnosed, with approximately 12,030 deaths. MM is most common in individuals over 65 years of age, with an average diagnosis age of 69 years. Diagnosis in people under 35 years of age is rare, at less than 1%.2
“Across our multiple myeloma portfolio and pipeline, we are shifting from treating to progression to treating to cure,” said Jordan Schecter, MD, VP, research & development, multiple myeloma, Johnson & Johnson Innovative Medicine, in the press release. “Our focus is to extend patient survival, and based on our expertise of the disease biology, develop treatment regimens with curative potential.”
Next Steps and Future Data from CARTITUDE-4
J&J stated that additional data from the CARTITUDE-4 trial confirmed Carvykti’s benefit over standard of care across key patient subgroups. Results are also expected to be presented at the European Hematology Association 2025 Congress.1
References
1. Single infusion of CARVYKTI® (ciltacabtagene autoleucel) delivered lasting treatment-free remissions for at least five years in patients with relapsed or refractory multiple myeloma. J&J. June 3, 2025. Accessed June 5, 2025. https://www.jnj.com/media-center/press-releases/single-infusion-of-carvykti-ciltacabtagene-autoleucel-delivered-lasting-treatment-free-remissions-for-at-least-five-years-in-patients-with-relapsed-or-refractory-multiple-myeloma
2. Key Statistics About Multiple Myeloma. American Cancer Society. Accessed June 5, 2025. https://www.cancer.org/cancer/types/multiple-myeloma/about/key-statistics.html
