Sofi-cel has received FDA breakthrough therapy designation for heavily pretreated patients with relapsed/refractory T-ALL and T-LBL.
The FDA has granted breakthrough therapy designation to soficabtagene geleucel (sofi-cel; formerly WU-CART-007), an allogenic CD7-targeted chimeric antigen receptor (CAR) T-cell therapy, for heavily pretreated patients with relapsed/refractory T-cell acute lymphoblastic leukemia (T-ALL) and T-cell lymphoblastic lymphoma (T-LBL).1
Sofi-cel is an investigational, potential first-in-class, allogeneic anti-CD7 CAR T-cell therapy designed to address key technical barriers in the treatment of T-cell malignancies. Using clustered regularly interspaced short palindromic repeats/Cas9 gene editing, CD7 and the T-cell receptor alpha constant genes are deleted to prevent CAR T-cell fratricide and reduce the risk of graft-vs-host disease (GVHD).
This regulatory decision was supported by data from a phase 1 trial (NCT04984356). Findings from the phase 2 portion, presented during the
According to correlative data and long-term follow-up from the study presented at the 2025 ASH Annual Meeting and Exposition, treatment with sofi-cel did not result in any late adverse effects (AEs) of special interest or drug-related serious AEs.4 Furthermore, 3 patients who received a successful allogeneic stem cell transplant in the study were alive approximately 2 years after sofi-cel infusion.
“The FDA’s breakthrough therapy designation underscores the promising clinical data we have generated and the potential for sofi-cel to make a meaningful difference for patients with relapsed/refractory T-ALL/T-LBL,” Cherry Thomas, MD, chief medical officer at Wugen, stated in a news release.1 “This recognition enables close collaboration with the FDA to accelerate development and, ultimately, help bring this innovative therapy to patients as quickly as possible.”
Of note, sofi-cel previously received regenerative medicine advanced therapy, fast track, orphan drug, and rare pediatric disease designations from the FDA, as well as a Priority Medicines designation from the European Medicines Agency for this same patient population.
Sofi-cel is currently being evaluated in patients with relapsed/refractory T-ALL and T-LBL in the pivotal phase 2 T-RRex study (NCT06514794).
This single-arm, multicenter, open-label study is enrolling patients with evidence of T-ALL or T-LBL, as defined by World Health Organization classification, that is either relapsed/refractory or minimal residual disease (MRD) positive.5 Patients are also required to be at least 1 year of age, have adequate organ function, and have an ECOG performance status of 0 or 1 or a Karnofsky performance status of 70 and above at the time of screening. Prior treatment with any anti-CD7 therapy, decompensated hemolytic anemia, or grade 2 to 4 acute or extensive chronic GVHD requiring systemic immunosuppression is not permitted.
Eligible patients will be enrolled into either the relapsed/refractory or MRD cohort, where they will receive a single intravenous infusion of sofi-cel following lymphodepletion.
The study’s primary end points are cCR rate in the relapsed/refractory cohort and MRD-negative CR rate in the MRD cohort.
“Our goal is to bring this investigational off-the-shelf allogeneic CAR-T treatment to patients as soon as possible,” Kumar Srinivasan, PhD, MBA, president and chief executive officer of Wugen, concluded in the news release.1 “Receiving breakthrough therapy designation from the FDA is a significant milestone for our company and a testament to the potential of our therapy to address a critical unmet medical need.”
