NEW YORK – Precigen on Tuesday said the US Food and Drug Administration has cleared an investigational new drug application for a Phase I/Ib trial of its investigational CAR T-cell therapy PRGN-3007 for patients with advanced ROR1-positive cancers.
In the study, Precigen and collaborators at the Moffitt Cancer Center will evaluate the autologous treatment's safety and efficacy in two patient populations. One arm will include patients with ROR1-positive blood cancers, including relapsed or refractory chronic lymphocytic leukemia, mantle cell lymphoma, acute lymphoblastic leukemia, and diffuse large B-cell lymphoma. A second arm will include patients with locally advanced, unresectable, or metastatic triple-negative breast cancer.
In the first part of the trial, patients in both arms will receive PRGN-3007 in an initial dose escalation phase, and in the second part, patients will receive the maximum tolerated dose of the treatment.
According to the Germantown, Maryland-based firm, PRGN is a "next-generation UltraCAR-T" that incorporates PD-1 blockade. This eliminates the need to give patients a checkpoint inhibitor alongside the CAR T-cell therapy, which could add significant toxicity and cost.
Precigen manufactures the autologous therapy using its non-viral gene delivery system and overnight, decentralized manufacturing process. The firm uses a single multicistronic transposon plasmid to engineer patients' harvested cells such that they express a CAR targeting ROR1, membrane-bound interleukin-15, a kill switch, and a novel mechanism to block PD-1 gene expression.