The autologous B-cell maturation antigen (BCMA)-directed CAR T-cell therapy anito-cel was tested at 2 dose levels in a phase 1 trial among participants with RR MM who had received at least 3 prior lines of therapy. The lower dose level was administered to 32 participants, and 6 received the higher dose. Dr Binod Dhakal (Medical College of Wisconsin, WI, USA) shared findings concerning safety and efficacy1.
The overall response rate was 100 %, with a stringent complete remission (sCR)/CR rate of 76 %. Moreover, this result was consistent among participants with extramedullary disease (n=13) and those with high-risk cytogenetics (n=11). Dr Dhakal added that the median progression-free survival was not reached after a median follow-up of 26.5 months. The corresponding 24-month progression-free survival rate was 56 %. Furthermore, 25 out of 28 evaluable participants were minimal residual disease-negative (10-5). “Remarkably, these findings are consistent in participants with extramedullary disease, a feature associated with notoriously poor prognosis,” added Dr Dhakal.
The toxicity profile was somewhat more favourable in the lower dose group, whereas the efficacy of the lower dose appeared to be similar to the higher dose. “We did not see any delayed neurotoxicities, Guillain-Barre syndrome, cranial nerve palsies, or Parkinsonian-like syndrome,” highlighted Dr Dhakal. Also, there were no grade 3 cytokine release syndrome cases in the low dose group and only 1 case of grade 3 immune effector cell-associated neurotoxicity syndrome.
Altogether, anito-cel delivered encouraging results in a heavily pre-treated population of patients with MM in a phase 1 trial, supporting further assessment of this novel CAR T-cell therapy in MM.
Medical writing support was provided by Robert van den Heuvel.