-- WU-CART-007 granted FDA Orphan Drug Designation for the treatment of patients with relapsed or refractory (R/R) T-cell acute lymphoblastic leukemia (T-ALL) or lymphoblastic lymphoma (LBL) --
-- WU-CART-007 is the first off-the-shelf allogeneic CAR-T cell therapy targeting T-Cell malignancies to enter the clinic in the United States --
ST. LOUIS, MO and SAN DIEGO, CA, March 15, 2022 – Wugen, Inc., a clinical-stage biotechnology company developing a pipeline of off-the-shelf cell therapies to treat a broad range of hematological and solid tumor malignancies, today announced that the first patient has been dosed in a Phase 1/2 trial of WU-CART-007 for the treatment of relapsed or refractory (R/R) T-cell acute lymphoblastic leukemia (T-ALL)/lymphoblastic lymphoma (LBL). WU-CART-007 is an 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. Additionally, Wugen announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to WU-CART-007 for the treatment of acute lymphoblastic leukemia.
“The dosing of the first patient in our Phase 1/2 trial for R/R T-ALL/LBL is a significant milestone as we continue to advance the first, off-the-shelf allogeneic CAR-T cell therapy for T-Cell malignancies in the United States into the clinic,” said Dan Kemp, Ph.D., President and Chief Executive Officer of Wugen. “We are also pleased to have received FDA Orphan Drug Designation for WU-CART-007, which further augments our efforts to deliver this therapy to patients in an area of great unmet need.”
“Globally, about 11,000 patients are diagnosed with T-ALL/LBL every year. About half of these patients are either resistant to frontline therapy or relapse even after initial remission, and are ultimately left with very limited treatment options,” added Dr. Armin Ghobadi, M.D., Associate Professor of Medicine, Division of Medical Oncology, and principal investigator of the site at the Washington University School of Medicine. “Nelarabine is the only FDA approved therapy for R/R T-ALL, and for more than a decade, no other advances have been made for this patient population. We look forward to advancing WU-CART-007, which has the potential to transform the care paradigm for these patients.”
The Phase 1/2 trial is a first-in-human, multi-site, global, open-label study of WU-CART-007 to evaluate its safety and clinical efficacy in patients with R/R T-ALL/LBL. Phase 1 of the study is designed to characterize the safety and tolerability of a single dose of WU-CART-007 following lymphodepleting conditioning therapy and to define the recommended Phase 2 dose (RP2D), cellular kinetics, and pharmacodynamics. Phase 2 of the study will investigate preliminary anti-tumor activity as measured by objective response rate (ORR) and duration of response (DOR). Additional information is available on clinicaltrials.gov, identifier NCT# 04984356.
The FDA Office of Orphan Products Development grants orphan designation for novel drugs or biologics being developed to treat a rare disease or condition affecting fewer than 200,000 patients in the U.S. ODD qualifies the sponsor of the drug for various development incentives of the Orphan Drug Act, including potentially a seven-year period of U.S. marketing exclusivity, tax credits for clinical research costs, clinical research trial design assistance, the ability to apply for annual grant funding and waiver of Prescription Drug User Fee Act (PDUFA) filing fees.
WU-CART-007 is an 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.
Wugen, Inc., is a clinical-stage biotechnology company developing a pipeline of off-the-shelf memory natural killer (NK) and CAR-T cell therapies to treat a broad range of hematological and solid tumor malignancies. Memory NK cells are hyper-functional, long-lasting immune cells that have evolved to attack cancer and respond to infection. Wugen is harnessing the power of this rare cell population by using its proprietary technologies to create WU-NK-101, currently in development for acute myelogenous leukemia (AML) and solid tumors. Wugen is also advancing WU-CART-007, an allogeneic CAR-T currently in development for T-cell acute lymphoblastic leukemia (T-ALL) and lymphoblastic lymphoma (LBL). For more information, please visit www.wugen.com.