CELLECTIS GETS FDA PASS TO CONTINUE ITS CLINICAL TRIAL OF THE NOVEL CAR-T CELL THERAPY

November 6, 2017

The US FDA has lifted the hold on Phase 1 trials of Cellectis’ UCART123 product candidate in acute myeloid leukemia (AML) and blastic plasmacytoid dendritic cell neoplasm (BPDCN).

Cellectis is a clinical-stage biopharmaceutical company focused on developing immunotherapies based on gene-edited allogeneic CAR T-cells (UCART).

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On Sep. 4th 2017, the U.S. Food and Drug Administration (FDA) placed a clinical hold on both UCART123 ongoing Phase 1 studies, respectively in acute myeloid leukemia (AML) and in blastic plasmacytoid dendritic cell neoplasm (BPDCN). The clinical hold was initiated after Cellectis reported one fatality in the BPDCN clinical trial (ABC study). This patient developed Grade 5 Cytokine Release Syndrome (CRS, an immediate complication occurring with the use of anti-T-cell antibody infusions) on Day 8 and passed away on Day 9 despite active CRS management and intensive care unit support.

Cellectis worked closely with the investigators and the FDA to amend the protocols. Main revisions include decrease of the cohort dose level from 6.25x105 to 6.25x104 UCART123 cells/kg and decrease of the cyclophosphamide dose in the lympho-depleting regimen before CAR-T cell infusion.