file:///C:/Users/bozh/Downloads/UCART123_AMELI-01_Phase_1_Oral_Presentation_ASGCT_2023_Final_Upload.pdf
file:///C:/Users/bozh/Downloads/051123_PosterASGCT2023_BAOa.pdf
Evidence of UCART123 anti-tumor activity was observed in four patients out of fifteen at DL2 or above with best overall responses in the FCA arm. Two out of eight patients (25%) at DL2 in the FCA arm achieved meaningful response: * A patient who failed five prior lines of therapy experienced a durable minimal residual disease (MRD) negative complete response (CR) with full count recovery at Day 56 that continues beyond one year.
* A patient with stable disease achieved greater than 90% bone marrow blast reduction (60% to 5%) at Day 28.
The preliminary data show that adding alemtuzumab to the FC LD regimen was associated with sustained lymphodepletion and significantly higher UCART123 cell expansion, which correlated with improved anti-tumor activity.
Patient Enrollment in a 2-Dose Regimen Arm
Overall, these preliminary data support the continued administration of UCART123 after FCA lymphodepletion in patients with r/r AML. Based on observed UCART123 expansion patterns and cytokine profiles, pursuant to an amended protocol, a second dose of UCART123 is given after 10-14 days to allow for additional UCART123 expansion and clinical activity without the use of additional lymphodepletion. The UCART123 cell expansion from the second dose of UCART123, in the setting of reduced disease burden, is expected to be safe and allow for clearance of residual disease.
“These clinically meaningful preliminary data from the AMELI-01 study are very encouraging for patients and for the future of allogeneic CART-cell therapy. AML is a disease with an urgent need for alternative treatment options for patients, and we are excited to be moving the study forward,” said Dr. Mark Frattini, M.D., Ph.D., Chief Medical Officer at Cellectis. “We have now implemented a two-dose regimen arm for our AMELI-01 trial and we look forward to sharing future updates as they become available.”
Bullish
