Precision BioSciences has started treating patients in a Phase I/IIa clinical trial assessing its chimeric antigen receptor (CAR) T cell product candidate PBCAR0191 as an off-the-shelf cell therapy.
The trial involves adults with relapsed or refractory (R/R) non-Hodgkin lymphoma (NHL) or R/R B-cell precursor acute lymphoblastic leukaemia (B-ALL). The first subject treated is an R/R NHL patient.
Being developed in alliance with pharmaceutical firm Servier, PBCAR0191 is a gene-edited allogeneic anti-CD19 CAR T therapy developed with donor-derived T cells altered using Precision’s ARCUS genome editing technology.
The edited cells are engineered to produce CAR T cells that selectively identify CD19 and prevent graft-versus-host disease, which is a major complication with current donor-derived cell-based therapies.
PBCAR0191’s safety, tolerability, and clinical activity will be evaluated during the multi-centre, open-label Phase I/IIa trial, which will enrol up to 80 patients.
Primary outcomes of the trial are safety and maximum tolerated dose, while secondary objectives include anti-tumour activity of PBCAR0191 and objective response rate.
In addition, the CAR T cell therapy’s expansion, trafficking and persistence will be monitored. The company will perform lymphodepletion several days before infusion of the cell therapy.
Outcomes in the trial participants will be tracked for up to one year. The trial is expected to be completed in 2021.
Precision will carry out early-stage research and Phase I activities along with the preparation of clinical supply for any Phase II trials of PBCAR0191.
Meanwhile, Servier holds an exclusive right to opt-in for late-stage development and commercialisation.