Phase 2 Results: Wugen’s CAR-T WU-CART-007 Outperforms Standard Care in T-ALL/LBL at EHA 2024

Wugen, Inc., a clinical-stage U.S. biotechnology company, has announced positive results from its Phase 2 study of WU-CART-007, an investigational anti-CD7 CAR-T therapy. This therapy targets relapsed/refractory (R/R) T-cell acute lymphoblastic leukemia/lymphoma (T-ALL/LBL). At the European Hematology Association (EHA) 2024 Hybrid Congress in Madrid, lead investigator Dr. Ibrahim Aldoss from City of Hope presented data showing a manageable safety profile and significant anti-leukemic activity with WU-CART-007.

Dr. Aldoss highlighted the aggressive nature of relapsed and refractory T-ALL/LBL, which has high relapse and mortality rates in both children and adults. He emphasized the promising potential of WU-CART-007 as a new treatment for patients who have not responded to existing therapies. Wugen's Chief Medical Officer, Dr. Jan Davidson-Moncada, reiterated the urgent need for effective, readily available allogeneic treatments for hard-to-treat blood cancers and expressed confidence in overcoming challenges associated with CAR-T therapies targeting T-cell diseases. The therapy was developed by Wugen’s Co-founder and Chief Scientific Officer, Dr. Matt Cooper, to specifically target CD7+ malignancies.

The Phase 2 findings were intriguing: 13 heavily pre-treated patients with a median age of 30 years were administered the recommended dose (RP2D) of WU-CART-007. Notably, 38% of these patients had relapsed post-allogeneic stem cell transplant. The treatment outcomes exceeded the current standard of care for R/R T-ALL/LBL, with a composite complete remission rate (CRc) of 73% and an overall response rate of 91%. The median duration of response was 6.2 months, and 46% of patients remained in continuous remission 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, peaking at Day 10 and detectable up to Day 90. Importantly, no patients developed drug product-specific anti-HLA antibodies or anti-drug antibodies.

Phase 1 data from this first-in-human global Phase 1/2 clinical trial of WU-CART-007 were presented last December at the American Society of Hematology meeting, revealing similar encouraging results.

Wugen also showcased additional findings at the EHA involving WU-CART-007 and WU-NK-101. Researchers highlighted the potential impact of Enhanced Lymphodepletion (ELD) on WU-CART-007 in T-ALL/LBL and presented data on WU-NK-101, an off-the-shelf memory NK cell therapy showing promise in treating acute myelogenous leukemia (AML).

WU-CART-007 is designed using CRISPR/Cas9 gene editing to delete CD7 and the T-cell receptor alpha constant (TRAC), thus preventing CAR-T cell fratricide and reducing the risk of graft-versus-host-disease (GvHD). The therapy uses T-cells from healthy donors to avoid malignant cell contamination. It has received Orphan Drug, Fast Track, Rare Pediatric Disease, and Regenerative Medicine Advanced Therapy (RMAT) designations from the U.S. Food and Drug Administration and PRIME designation in the European Union for treating R/R T-ALL and T-LBL.

Meanwhile, WU-NK-101 leverages memory NK cells, known for their enhanced anti-tumor activity and cytokine-induced memory-like (CIML) phenotype, making them suitable for cancer therapy. The therapy is being developed for AML and future studies are planned for solid tumors in combination with cetuximab. Initial studies have shown robust in-vivo activity, resistance to immune suppression, and synergy with checkpoint inhibitors. WU-NK-101 has also received Orphan Drug Designation from the U.S. FDA for AML treatment.

Wugen, Inc., originated from Washington University in St. Louis, is progressing in developing off-the-shelf CAR-T and memory NK cell therapies for cancer treatment.