Study of Novel Off-the-Shelf CAR-T in T-ALL/LBL Launching in 2025

A phase 2 study evaluating WU-CART-007, a first-in-class, investigational, anti-CD7 chimeric antigen receptor (CAR) T-cell therapy, for the treatment of adult and pediatric patients with relapsed/refractory (R/R) T-cell acute lymphoblastic leukemia or T-cell lymphoblastic lymphoma (T-ALL/LBL) is planned to begin in the first quarter of 2025.¹

This pivotal phase 2 study will assess WU-CART-007’s safety and efficacy in patients with R/R T-ALL/LBL and T-ALL/LBL. The single-arm study will include 2 cohorts: one of patients with R/R disease and an exploratory cohort of patients with minimal residual disease (MRD)-positive status.

The trial, which is enrolling patients at centers across the US, Europe, Asia, and Australia, marks the first pivotal study of an off-the-shelf, allogeneic CD7-targeted CAR T-cell therapy for this patient population.

“T-ALL/LBL is an aggressive cancer with a significant need for new therapies given that many patients will progress on standard of care,” said Kumar Srinivasan, PhD, Wugen president and chief executive officer, in a press release. “Wugen is well positioned for this critical stage of research and development for WU-CART-007.”

Prior Data on WU-CART-007

WU-CART-007, an allogeneic, off-the-shelf, fratricide-resistant CD7-targeted CAR T-cell therapy, works to overcome the technological challenges of harnessing CAR T cells that aim to treat CD7-positive hematological malignancies. The agent is manufactured through the use of healthy donor-derived T cells and aims to remove the risk of malignant cell contamination that has historically been seen in the autologous CAR T setting.¹

In July 2022, the FDA granted WU-CART-007 fast track designation.² Additionally, the agent has been granted regenerative medicine advanced therapy, orphan drug, and rare pediatric disease designations from the FDA and PRIME designation in the European Union for the treatment of R/R T-ALL and T-LBL.

WU-CART-007 has already been examined in a phase 1/2 trial (NCT04984356) for the treatment of R/R T-ALL/LBL.³ The global, open-label, first-in-human study assessed the safety, recommended dose, and preliminary antitumor activity of WU-CART-007 in this patient population, which includes those aged 12 years and older with adequate renal, hepatic, respiratory, and cardiovascular function, a life expectancy of over 12 weeks, and an ECOG/Karnofsky performance status 0 or 1 at screening (adults age >16) or Lansky performance status of 60 and above (adolescents ≤16).³

The primary end points included safety based on the incidence of adverse events, MTD, overall response rate, duration of response, and progression-free survival. The secondary end points of the study were overall survival and hematopoietic stem cell transplant rate.

Results from the phase 1/2 cohort expansion study showed clinically manageable safety and strong anti-leukemic activity.¹ The overall response rate was 91% in heavily pretreated patients with R/R T-ALL/LBL. The updated data from the study are expected to be presented at the 2024 American Society of Hematology Annual Meeting.

REFERENCES:

1. Wugen to initiate a pivotal trial for its investigational, off-the-shelf, allogeneic, CD7-targeted CAR-T cell therapy, WU-CART-007, in patients with relapsed or refractory T cell acute lymphoblastic leukemia or T cell lymphoblastic lymphoma. News release. Wugen, Inc. November 12, 2024. Accessed November 12, 2024. https://tinyurl.com/2bbnwpwf

2. Wugen receives U.S. FDA fast track and rare pediatric disease designations for WU-CART-007 for the treatment of R/R T-ALL/LBL. News release. Wugen Inc. July 19, 2022. Accessed November 12, 2024. https://bwnews.pr/3PGPxa1

3. A phase 1/2 study of the safety and efficacy of anti-CD7 allogeneic CAR-T cells (WU-CART-007) in patients with relapsed or refractory T-ALL/LBL. ClinicalTrials.gov. Updated August 12, 2024. Accessed November 12, 2024. https://clinicaltrials.gov/ct2/show/NCT04984356