CI Scientists Remarks: 

About PBCAR0191:

• Made from donor-derived T cells modified using Precision Bio’s ARCUS genome editing technology

• • Recognizes CD19 and avoids graft-versus-host disease (GvHD) which is significant complication associated with donor-derived, cell-based therapies

• PBCAR0191 is being evaluated in a Phase 1/2a multicenter, non-randomized, open-label, parallel assignment, dose-escalation, and dose-expansion study to evaluate the safety and tolerability in adult patients with R/R B-cell acute lymphoblastic leukemia (B-ALL) and R/R non-Hodgkin lymphoma (NHL)
• Initial data from this study from NHL and B-ALL cohorts was promising. Based on the data observed at dose level (DL)1 and DL2, company had filed for a protocol amendment for this trial with the FDA in Dec’19. Following feedback from the FDA in Jan’20, the protocol amendment is now being implemented
• The company is actively assessing key clinical questions including the impact of higher total doses of cells, repeat dose of cells, and/or the impact of modified lymphodepletion on the ability to achieve durable clinical benefit
• PBCAR0191 has received orphan drug designation for the treatment of ALL, mantle cell lymphoma

About the collaboration:

• This product is being developed in collaboration with Servier, which has exclusive rights to opt in for late-stage development and commercialization
• PBCAR0191 is one of three CAR-T products in Precision’s pipeline. The others are: PBCAR20A CD20 CAR-T for hematological malignancies, PBCAR269A B-cell maturation antigen (BCMA) CAR-T for R/R multiple myeloma