Dr Farid on the Efficacy of MDC-CAR-BCMA001 in R/R Myeloma and AL Amyloidosis

“[Among] these 6 patients with challenging baseline [characteristics], we achieved exceedingly good efficacy with 5 patients responding, 4 achieving a complete response [CR], and all patients who achieved a CR also became minimal residual disease negative.”

Kiavasch Mohammad Nejad Farid, MD, Heidelberg University Hospital, discusses the efficacy findings from a study of the novel second-generation BCMA-directed CAR T-cell construct MDC-CAR-BCMA001 in relapsed/refractory multiple myeloma and systemic light chain (AL) amyloidosis.

During the 51st Annual EBMT Meeting, Farid presented data from a study that showed that at a median follow-up of 13 months, 5 of the patients who received MDC-CAR-BCMA001 (n = 6) achieved a response. Four of these responders achieved a complete response (CR) and all patients who experienced a CR achieved minimal residual disease negativity, Farid said. All responders remained alive at the median follow-up, with 4 remaining in sustained remission without the need for additional therapy, he added. In patients with AL amyloidosis, achieving rapid and deep hematologic response is crucial, he said.

Additional data from the study demonstrated that the median decrease in free light chains was 98.4%. The median time to response was 14 days, and the median time to best response was 41 days. In terms of organ responses, 1 patient each achieved a cardiac very good partial response (VGPR), a renal VGPR, and an improvement in polyneuropathy.

In terms of safety, grade 1 and 2 cytokine release syndrome was reported in 50% and 33% of patients, respectively. One patient experienced grade 3 or higher CRS. The median time to CRS onset was 1 day (range, < 1 to 3), and the median duration of CRS was 4 days (range, 2-6). Sixty-seven percent of patients received tocilizumab (Actemra) and 33% were treated with corticosteroids. Immune effector cell-associated neurotoxicity syndrome and other late neurtoxicities were not reported. Sixty percent of patients experienced grade 1 early immune effector cell-associated hematotoxicity (ICAHT); 20% had grade 2 ICAHT and received granulocyte colony-stimulating factor. Grade 1 ICAHT was reported in 17% of patients and resolved spontaneously.