Phase 2 Results: Wugen's CAR-T WU-CART-007 Outperforms Standard in Treating T-ALL/LBL

Wugen, Inc., a U.S.-based biotechnology company specializing in allogeneic, off-the-shelf cell therapies, has reported promising Phase 2 results for its investigational anti-CD7 CAR-T therapy, WU-CART-007 ("W-T7"). The therapy targets relapsed or refractory (R/R) T-cell acute lymphoblastic leukemia/lymphoma (T-ALL/LBL), a particularly aggressive form of cancer with high rates of relapse and mortality among both children and adults.

At the European Hematology Association (EHA) 2024 Hybrid Congress in Madrid, lead investigator Dr. Ibrahim Aldoss, from City of Hope in Duarte, California, presented compelling data from a Phase 2 cohort expansion study. The findings revealed that WU-CART-007 demonstrated a clinically manageable safety profile and significant anti-leukemic activity in patients who had previously been treated with existing cancer therapies.

Dr. Aldoss highlighted the pressing need for effective treatments for R/R T-ALL/LBL, stating, "The results support the potential of this allogeneic CAR-T therapy as a new treatment option for patients who continue to face a refractory illness despite the best available medicines."

Wugen's Chief Medical Officer, Dr. Jan Davidson-Moncada, emphasized the urgency of developing allogeneic treatments that are readily available without delays for patients with challenging blood cancers. He expressed confidence that Wugen's approach could overcome specific challenges associated with CAR-T therapies targeting T-cell diseases and announced plans for further studies of WU-CART-007.

Key Phase 2 Findings
The Phase 2 study administered the recommended dose (RP2D) of WU-CART-007 to 13 heavily pre-treated patients with a high disease burden. Notably, 38% of these patients had relapsed or progressed after undergoing allogeneic stem cell transplants. The study reported outcomes that exceeded the current standard of care for R/R T-ALL/LBL.

Key results included:
- A composite complete remission rate (CRc) of 73% (8 out of 11 patients).
- An overall response rate of 91% (10 out of 11 patients), with 2 partial responses observed in patients with extramedullary disease and R/R T-LBL.
- A median duration of response (mDOR) of 6.2 months, with a median follow-up period of 8.5 months. Nearly half (46%) of the patients remained in continuous remission for periods ranging from 4.3 to 8.6 months.
- Seven patients, including five at RP2D, were successfully transplanted.

Pharmacokinetic analysis showed rapid expansion and persistence of the allogeneic cell therapy, with WU-CART-007 expansion peaking at Day 10 and remaining detectable up to Day 90. No patients developed specific anti-HLA antibodies or anti-drug antibodies.

Additional Data at EHA
Researchers also presented other new findings related to WU-CART-007 and WU-NK-101, an off-the-shelf investigational memory natural killer cell therapy, at EHA. The presentations included data on the preliminary effect of enhanced lymphodepletion on WU-CART-007 in T-ALL/LBL and the anti-tumor phenotype of WU-NK-101 detected in R/R AML patient samples after infusion.

About WU-CART-007
WU-CART-007 is an allogeneic, off-the-shelf CAR-T cell therapy engineered to target CD7+ hematological malignancies. Utilizing CRISPR/Cas9 gene editing technology, Wugen has developed a therapy that overcomes the challenges of CAR-T cell fratricide and minimizes the risk of graft-versus-host disease (GvHD). Currently, WU-CART-007 is being evaluated in a global Phase 1/2 clinical trial for R/R T-ALL/LBL.

WU-CART-007 has received multiple designations from the U.S. Food and Drug Administration and the European Union, including Orphan Drug, Fast Track, Rare Pediatric Disease, and Regenerative Medicine Advanced Therapy (RMAT) designations, as well as PRIME (Priority Medicines) designation for the treatment of R/R T-ALL and T-LBL.

About WU-NK-101
WU-NK-101 is a novel immunotherapy that leverages the efficacy of memory natural killer (NK) cells to target liquid and solid tumors. These hyper-functional, long-lasting immune cells exhibit enhanced anti-tumor activity and a superior phenotype suitable for cancer therapy. Wugen is using its proprietary MonetaTM platform to develop WU-NK-101 as a commercially scalable, off-the-shelf cell therapy, with ongoing development for acute myelogenous leukemia (AML) and planned studies for solid tumors in combination with cetuximab.

Wugen, Inc., a spin-out from Washington University in St. Louis, continues to advance the development of next-generation CAR-T and memory NK cell therapies for cancer.