— Positive trends continue as more patients receive treatment with WU-CART-007 in this study exploring the drug’s potential in improving standard of care
— WU-CART-007 continues to demonstrate favorable tolerability profiles in additional patients with R/R T-ALL/LBL
— Initial evidence of anti-leukemic activity and notable clinical improvement reinforced by composite complete remission rate of 60% at dose levels (DL) 2 and 3

ST. LOUIS, MO and SAN DIEGO, CA, USA I December 11, 2023 I Wugen, Inc., a clinical-stage biotechnology company developing allogeneic, off-the-shelf cell therapies for the treatment of hematological and solid tumor malignancies, today shared the latest data from its ongoing Phase 1/2 dose escalation study of WU-CART-007. An investigational allogeneic CAR-T cell therapy, WU-CART-007 is being studied for the treatment of patients with relapsed or refractory T-cell Acute Lymphoblastic Leukemia (T-ALL)/Lymphoblastic Lymphoma (LBL).

Patients with relapsing or refractory (R/R) T-ALL face grim prognoses, with around 30% of patients failing to respond to first-line standard of care treatments, and for those who do respond, approximately 50% relapse.

“For a disease that disproportionately affects younger individuals, the need to find better treatments feels especially urgent,” said Armin Ghobadi, M.D, associate professor of medicine and clinical director of Center for Gene and Cellular Immunotherapy (CGCI) in the Division of Medical Oncology at the Washington University School of Medicine in St. Louis. “It’s encouraging to see positive momentum — with favorable tolerability and efficacy data continuing to be reported as the study has expanded to include more patients with such difficult-to-treat blood cancers.”

“As we at Wugen work diligently to reshape the treatment landscape for patients suffering from diseases with limited treatment options, we believe early results from this study speak to the larger potential of innovative allogeneic cell therapies to enhance the quality of life for patients with more effective and accessible treatments,” said Chief Medical Officer Jan Davidson-Moncada, M.D., Ph.D. “We look forward to reporting topline Phase 2 results in the coming year, which will mark a pivotal milestone in our commitment to redefining the standard of care for these cancers.”

Initial data presented at the European Hematology Association Congress in June 2023 showed that treatment with WU-CART-007 resulted in overall favorable efficacy and tolerability profiles compared to the baseline standard of care. In the latest update, an additional 13 patients were treated with WU-CART-007, which showed clinically acceptable safety profiles and preliminary evidence of anti-leukemic activity, demonstrating a notable clinical improvement. WU-CART-007 remains on track to complete enrollment by the end of this year.

Positive and consistent data from the WU-CART-007 Phase 1/2 trial collectively underscore its potential to address unmet medical needs for difficult-to-treat blood cancers as it advances to the next crucial phase of development. These early results suggest that WU-CART-007 may offer a novel and effective therapeutic avenue, providing hope for improved outcomes in a patient population where viable alternatives are scarce.

In a presentation titled “Phase 1/2 Dose-Escalation/Dose-Expansion Study of Anti-CD7 Allogeneic CAR-T Cells (WU-CART-007) in Relapsed or Refractory (R/R) T-Cell Acute Lymphoblastic Leukemia/ Lymphoblastic Lymphoma (T-ALL/LBL),” Wugen shared these updated data:

  • As of Nov. 28, 2023, 25 patients have been dosed with WU-CART-007. Disease burden at baseline consisted of extramedullary only disease (EMD) in 28% (7/25) of patients, and a median bone marrow (BM) blast count of 63.2% (range 5%-95%) in patients with BM disease.
  • Overall, WU-CART-007 demonstrated a manageable safety profile:
    • No cases of Graft versus Host Disease (GvHD), prolonged T-cell aplasia, or prolonged pancytopenia in the absence of disease were observed. The majority of deaths were due to disease progression. Of three Grade 5 events observed, two were due to fungal infection and were not attributed to WU-CART-007. One event temporally related to WU-CART-007 occurred in the setting of CRS and progressive disease.
    • Most reports of cytokine release syndrome (CRS) were low-grade (84%; 21/25), with the exception of five Grade ≥ 3 report, which were manageable with supportive care.
    • One instance of Grade 1 immune effector cell-associated neurotoxicity syndrome (ICANS), which resolved spontaneously, was reported in a patient at DL3.
  • WU-CART-007 showed dose-dependent anti-tumor activity with no responses seen at DL1. In evaluable patients at DL≥ 2 (18/22), the Composite Complete Remission Rate (CRc) in patients was 67%. At the recommended Phase 2 dose (RP2D), the CRc rate was 73%.
  • No patients developed novel anti-HLA antibodies against the donor, and of the 18 patients who were tested, no anti-drug antibodies against the CAR-construct were detected.
  • Phenotypic analysis revealed in vivo WU-CART-007 cells expressed activation markers (KI67, CD38, HLA-DR) and were largely an effector memory CD45RA+ (EMRA) CM phenotype (CD45RA+, CD197+).

ASH meeting information can be found here: https://www.hematology.org/meetings/annual-meeting.

About WU-CART-007

WU-CART-007 is an allogeneic, off-the-shelf, fratricide-resistant CD7-targeted CAR-T cell therapy engineered to overcome the technological challenges of harnessing CAR-T cells to treat CD7+ hematological malignancies. Wugen is deploying CRISPR/Cas9 gene editing technology to delete CD7 and the T-cell receptor alpha constant (TRAC), preventing CAR-T cell fratricide and mitigating the risk of graft-versus-host-disease (GvHD). WU-CART-007 is manufactured using healthy donor-derived T-cells to eliminate the risk of malignant cell contamination historically observed in the autologous CAR-T setting. WU-CART-007 is currently being evaluated in a global Phase 1/2 clinical trial for the treatment of relapsed or refractory (R/R) T-cell acute lymphoblastic leukemia (T-ALL)/lymphoblastic lymphoma (LBL). Additional information is available on clinicaltrials.gov, identifier NCT# 04984356. WU-CART-007 has received Orphan Drug, Fast Track, and Rare Pediatric Disease Designations from the U.S. Food and Drug Administration for the treatment of R/R T-ALL/LBL.

About Wugen

Wugen, Inc. is a clinical-stage biotechnology company developing the next generation of off-the-shelf memory natural killer (NK) and CAR-T cell therapies for cancer. Wugen is leveraging its proprietary Moneta platform and deep genomic engineering expertise to pioneer a new class of memory NK cell therapies to treat hematological and solid tumor malignancies. For more information, please visit www.wugen.com.