WU-CART-007 has been granted fast track, rare pediatric disease, and orphan drug designations.
The FDA has granted fast track and rare pediatric disease designations to Wugen’s WU-CART-007 for the potential treatment of relapsed or refractory (R/R) T-cell acute lymphoblastic leukemia (T-ALL)/lymphoblastic lymphoma (LBL).1
"We are very pleased to have received both fast track and rare pediatric disease designations, which re-affirm the great unmet need for new treatment options for people with R/R T-ALL/LBL," Dan Kemp, PhD, president and chief executive officer, Wugen, said in a statement.1 "Earlier this year, we dosed the first patient in our ongoing Phase 1/2 trial of WU-CART-007 for R/R T-ALL/LBL and are currently in the dose escalation phase of the study. We look forward to working closely with the FDA as we continue to advance WU-CART-007 through clinical development."
WU-CART-007, an allogeneic, fratricide-resistant CD7-targeted, chimeric antigen receptor (CAR) T-cell therapy, is currently being evaluated for safety and efficacy in this patient population in a global, open-label, first-in-human, phase 1/2 study (NCT04984356). The trial dosed its first patient and received orphan drug designation in March 2022.2
“There's huge hurdles that have to be overcome to be able to treat T-cell malignancies. Primarily, it is very difficult to tackle T-cell malignancies with T cells generated from the patient themselves, it is very difficult to be able to generate enough or be able to extract enough healthy T cells since there's always going to be contamination from the malignant clone. Once you generate the CAR T cells to attack T cells, there's a lot of friendly fire or fratricide,” Jan Davidson, MD, PhD, chief medical officer, Wugen, previously told CGTLive.
The first phase of the study is evaluating safety and tolerability, cellular kinetics, and pharmacodynamics of a single dose of the therapy after lymphodepletion and determine a recommended phase 2 dose. This dose will be assessed for preliminary anti-tumor activity in the second phase of the study via objective response rate and duration of response. Secondary outcomes of the study include overall survival and hematopoietic stem cell transplant rate.
“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,” principal investigator Armin Ghobadi, MD, associate professor of medicine, Division of Medical Oncology, Washington University School of Medicine, said in an earlier statement.2 “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.”