Th20 - An Academic BCMA-directed CAR-T Therapy (ARI0002h) with Fractionated and Booster Dose in Patients with Relapsed/Refractory Multiple Myeloma: First Data About Efficacy and Safety

Abstract Text: ARI0002h is an autologous and academic CAR T-cell product with a 4-1BB co-stimulatory domain and a humanized single chain variable fragment targeting BCMA.
Here, we report safety and efficacy results of the CARTBCMA-HCB-01 multicenter clinical trial for patients with relapsed/refractory multiple myeloma (NCT04309981) receiving ARI0002h in 5 Spanish center, while productions were done in 2 of them, by using CliniMACS Prodigy.
Patients (pts) eligible had measurable disease and refractory to previous conventional treatments. Targeted dose was 3x106/kg CAR+ cells, being administered in fractionated manner (10%/30%/60%). A second dose (3x106 CAR+ cells/kg) was planned at least 4 months after the first dose in pts with any grade of response without serious complications after first administration.
30 pts received ARI0002h cells. Median CAR-T cell production time was 11 days (range 9-14) with a 100% manufacture success. The ORR of 30 evaluable pts was 100%, with a stringent complete remission (sCR) plus very good partial response (VGPR) rate of 90%. Median time to first response was one month. Of 26 MRD-evaluable pts at day +100, 92% were MRD-negative in bone marrow.
53% of patients were alive and without progression at 16 months. Median overall survival (OS) was not reached and the 16-month OS rate was 80%.
ARI0002h cells demonstrated peak expansion on day 14. 24 out of 28 eligible pts (86%) received the second dose. 7 pts (29%) improved their response after reinfusion.
A second cohort of additional 30 pts has been started, trying to contrast with the first 30 patients.