Carvykti (ciltacabtagene autoleucel)

To identify predictive biomarkers, we performed flow cytometry and multiplex assays before and early after CAR-T infusion on 78 patients (ide-cel n = 31; axi-cel n = 24; and cilta-cel n = 23) undergoing CAR-T therapy. Baseline sIL-2R and sVCAM-1 demonstrated robust predictive value for prolonged neutropenia, severe infections, and mortality independently of key clinical variables such as the underlying disease and CAR-T product. Integration of these markers improves existing models and can help to refine risk assessment and guide individualized patient management in CAR-T therapy.

P2, N=210, Active, not recruiting, Janssen Research & Development, LLC | Recruiting --> Active, not recruiting

We analyze 61 RRMM patients receiving idecabtagene vicleucel (Ide-cel; n = 34) or ciltacabtagene autoleucel (Cilta-cel; n = 27) and find that Cilta-cel achieves higher complete response (CR) rates (78% vs. 38%) and longer progression-free survival. Greater reductions in soluble BCMA correlate with enhanced CAR T expansion and systemic inflammation. These findings reveal cellular mechanisms driving differential efficacy and toxicity of BCMA-directed immunotherapy.

Finally, we characterize NINT associated CD4 + CAR T cell populations which are potential therapeutic targets for future exploration. Ciltacabtagene autoleucel associated non-ICANS neurotoxicities are driven by high CD4 + CAR T cell expansion exhibiting memory marker expression and upregulated inflammatory gene signaling pathways.

Locally produced BCMA CAR-T is safe and effective in heavily pretreated R/R MM, inducing deep responses. scRNA-seq/TCR-seq findings highlight interplay of CAR-T heterogeneity, clonal adaptability, and immune regulation. CD8⁺ subset specialization and clonal persistence matter for durable responses; exhaustion and immunosuppression may cause relapse.

P3, N=419, Active, not recruiting, Janssen Research & Development, LLC | Trial completion date: Sep 2036 --> Apr 2029

P2, N=11, Active, not recruiting, Janssen Research & Development, LLC | Recruiting --> Active, not recruiting

High-dose dexamethasone plus intravenous immunoglobulin failed to improve neurologic symptoms and prompted T-cell-depleting cyclophosphamide, which lowered CAR- and non-CAR-T cells. These observations indicate that CIDP is a severe complication of cilta-cel therapy and may arise from bystander expansion of autoreactive CD8⁺ T-cells rather than direct CAR-T cell activity. Timely escalation to T-cell-depleting therapy may improve outcomes.

P3, N=419, Active, not recruiting, Janssen Research & Development, LLC | Trial completion date: Apr 2029 --> Sep 2036

P2, N=43, Active, not recruiting, Janssen Research & Development, LLC | Recruiting --> Active, not recruiting

P3, N=743, Active, not recruiting, Janssen Research & Development, LLC | Trial completion date: Dec 2034 --> Sep 2036

P2, N=10, Recruiting, Janssen Research & Development, LLC | Trial completion date: Aug 2026 --> Nov 2026