FDA Grants Fast Track and Rare Pediatric Disease Designations for WU-CART-007 in T-ALL/LBL

The FDA has granted fast track designation and rare pediatric disease designation for WU-CART-007, a chimeric antigen receptor (CAR) T-cell therapy for the treatment of patients with relapsed or refractory T-cell acute lymphoblastic leukemia (T-ALL) or lymphoblastic lymphoma (LBL), according to Wugen, Inc.¹

WU-CART-007 is an allogeneic, off-the-shelf, fratricide-resistant CD7-targeted CAR T-cell therapy. The product works to overcome the challenges of harnessing CAR T cells which aim to treat CD7+ hematological malignancies.

Currently, WU-CART-007 is being evaluated for its safety and efficacy in patients with relapsed/refractory T-ALL/LBL in a phase 1/2 study (NCT04984356).

“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,” said Dan Kemp, PhD, president and chief executive officer of Wugen, in the press release. “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.”

In the global, open-label, first-in-human, phase 1/2 study, experts aim to evaluate the safety, recommended dose, and preliminary anti-tumor activity of WU-CART-007 in this patient population.²

Those enrolled include patients aged 12 years and older with evidence of relapsed or refractory T-ALL or LBL. Patients must have bone marrow with 5% or greater lymphoblasts by morphologic assessment or evidence of extramedullary disease at screening, adequate renal, hepatic, respiratory, and cardiovascular function, a life expectancy of over 12 weeks, and an ECOG/Karnofsky performance status 0 or 1 at screening (adults age >16) or Lansky performance status of 60 and above (adolescents ≤ 16).

An estimated 44 patients will be enrolled, and the study will consist of 2 phases. Within phase 1, the dose-escalation portion, up to 24 patients will be treated with 1 dose of WU-CART-007, using 4 or less dose levels until the maximum tolerated dose (MTD) or maximum administered dose (MAD) is determined. In this dose escalation part, cohorts of 3 to 6 patients will be enrolled using a standard 3 + 3 design. When the recommended phase 2 dose is determined, the phase 2 portion cohort expansion portion of the trial will begin to enroll expansion cohorts.

Primary end points of the trial include safety based on the incidence of adverse events, MTD, overall response rate, duration of response, and progression-free survival. Secondary end points of the study consist of overall survival and hematopoietic stem cell transplant rate.

The trial is actively recruiting in California, Missouri, Pennsylvania, and Australia with an expected study completion date of August 2026.

References:

Wugen receives U.S. FDA fast track and rare pediatric disease designations for WU-CART-007 for the treatment of R/R T-ALL/LBL. News release. Wugen Inc. July 19, 2022. Accessed July 20, 2022. https://bwnews.pr/3PGPxa1

A phase 1/2 study of the safety and efficacy of anti-CD7 allogeneic CAR-T cells (WU-CART-007) in patients with relapsed or refractory T-ALL/LBL. ClinicalTrials.gov. Updated June 23, 2022. Accessed July 20, 2022. https://clinicaltrials.gov/ct2/show/NCT04984356