FDA grants the Investigational New Drug designation for PBCAR20A - a CAR T-cell therapy

On 16 September 2019, the US Food and Drug Administration (FDA) accepted the Investigational New Drug (IND) application for the off-the-shelf chimeric antigen receptor (CAR) T-cell therapy, PBCAR20A.  

PBCAR20A is a CD20-targeting genome-edited allogeneic CAR T-cell therapy candidate that can now be investigated for the treatment of non-Hodgkin lymphoma (NHL), chronic lymphocytic leukemia (CLL), and small lymphocytic lymphoma (SLL). The phase 1/2a clinical trial is planned to start by the end of the year and aims to recruit patients with CLL, SLL, and NHL, including mantle cell lymphoma (MCL), for which the company has received Orphan Drug Designation. The initial report of this nonrandomized, open-label study is expected in 2020.

The approval is based on the preclinical data showing PBCAR20A efficacy in vivo against CD20+ tumor cells. The allogeneic CAR T-cells are created by inserting the CAR gene at the T-cell receptor (TCR) locus of T cells derived from healthy donors. The proprietary manufacturing platform allows fast manufacture of a consistent product, maintaining a high proportion of naïve and central memory CAR T-cells. The technology aims to prevent graft-versus-host disease.

This is the second off-the-shelf CAR T-cell IND product; PBCAR0191, an anti-CD19 CAR T-cell therapy, is currently being investigated in patients with relapsed/refractory (R/R) NHL and R/R B-cell precursor acute lymphoblastic leukemia (B-ALL).