Wugen, Inc. has begun its pivotal Phase 2 trial of WU-CART-007, an off-the-shelf CAR-T cell therapy for relapsed or refractory T-cell acute lymphoblastic leukemia/lymphoma (T-ALL/LBL) in pediatric and adult patients. This follows promising Phase 1/2 results, which showed a 91% overall response rate and a 73% composite complete remission rate in heavily pre-treated patients. The therapy has received multiple FDA accelerated approval designations, including RMAT, Fast Track, Orphan Drug, and Rare Pediatric Disease, as well as Priority Medicines designation in the EU.
This pivotal trial initiation is particularly important because T-ALL/LBL patients haven’t seen a new treatment approved in 20 years, especially for those with relapsed or refractory disease who face limited options and poor outcomes. The off-the-shelf nature of WU-CART-007 addresses a crucial unmet need by potentially providing faster and more accessible treatment compared to traditional autologous CAR-T therapies, which require individualized manufacturing from the patient’s own cells. The rapid enrollment rate observed so far underscores the high demand for new therapies in this area.
The Phase 2 T-RRex study is a single-arm trial evaluating WU-CART-007’s efficacy and safety in two patient cohorts: one with relapsed/refractory disease and another exploratory cohort with minimal residual disease. WU-CART-007 utilizes CRISPR gene editing to enhance its functionality. By deleting the CD7 target and the TRAC gene in the CAR-T cells, the therapy avoids self-destruction (fratricide) and reduces the risk of graft-versus-host disease, respectively.
Positive results from this pivotal trial could lead to regulatory approval and significantly alter the treatment landscape for T-ALL/LBL. This would offer a readily available and potentially curative therapy for patients who currently have few options. The trial’s success could also further validate Wugen’s allogeneic CAR-T platform and pave the way for the development of similar therapies for other hematological and solid tumor malignancies.
Jon Napitupulu is Director of Media Relations at The Clinical Trial Vanguard. Jon, a computer data scientist, focuses on the latest clinical trial industry news and trends.
