NEW YORK, June 11, 2026 — Leads & Copy — Cellectis, a clinical-stage biotechnology company, announced today the presentation of final Phase 1 data from the BALLI-01 clinical trial for lasme-cel, a CD22-directed allogeneic CAR-T therapy, in patients with relapsed/refractory B-cell acute lymphoblastic leukemia (r/r B-ALL). Preliminary data from the NATHALI-01 study evaluating eti-cel, a dual CD20 and CD22 directed CAR-T in relapsed/refractory B-cell non-Hodgkin lymphoma (r/r B-NHL), were also presented at the European Hematology Association (EHA) 2026 Annual Congress.
The BALLI-01 trial results, presented orally by Nitin Jain, M.D., Professor of Medicine at the University of Texas MD Anderson Cancer Center, involved 45 patients in third line and beyond (3L+). Fifteen patients received the recommended Phase 2 dose, and seven were in the target Phase 2 population. These patients were heavily pre-treated, with a median of five prior lines of therapy. A significant majority had previously been treated with blinatumumab (82%), CD19 CAR-T therapy (53%), CD22-directed antibody-drug conjugate (ADC) (56%), and nearly half had undergone hematopoietic stem cell transplantation (HSCT) (47%).
In the target Phase 2 population of the BALLI-01 study, an overall response rate (ORR) of 100% (7/7) was observed, with a complete remission or complete remission with incomplete count recovery (CR/CRi) rate of 57% (4/7). Of these responders, 75% achieved minimal residual disease negative (MRD-ve) status. All patients subsequently proceeded to HSCT. Lasme-cel demonstrated a manageable safety profile, with cytokine release syndrome (CRS) ≥ grade 3 occurring in 4% of patients, immune effector cell-associated neurotoxicity syndrome (ICANS) ≥ grade 3 in 4%, and immune effector cell-associated hemophagocytic lymphohistiocytosis-like syndrome (IEC-HS) ≥ grade 3 in 2%. All reported CRS, ICANS, and IEC-HS events resolved.
Dr. Jain stated, “These final Phase 1 results are particularly meaningful for a patient population that has very limited treatment options. Being able to achieve deep remissions in these patients and allowing them to subsequently receive an HSCT is promising. We look forward to accelerating accrual into the ongoing Pivotal Phase 2 study and bringing this treatment to patients.” The Pivotal Phase 2 BALLI-01 trial is currently recruiting, with the first interim analysis anticipated in Q4 2026. The oral presentation detailed the safety and efficacy of UCART22 in heavily pretreated patients with relapsed or refractory CD22+ B-cell acute lymphoblastic leukemia.
Preliminary data from the NATHALI-01 study, evaluating eti-cel in r/r B-NHL, were presented as a poster by Professor Emmanuel Bachy, M.D., Ph.D., from the Hospices Civils de Lyon, France. Eti-cel is described as a highly differentiated, allogeneic dual CAR-T targeting both CD20 and CD22. As of a February 2026 data cutoff, 14 patients with r/r B-NHL had been treated. This population was heavily pre-treated, with a median of three prior lines of therapy, 93% having received prior CD19-directed CAR-T therapy, and all presenting with stage IV disease at baseline.
In the optimal dose cohort of the NATHALI-01 study, the ORR and complete response (CR) were 88% and 63%, respectively. The analysis indicated a positive correlation between alemtuzumab exposure and clinical outcomes, suggesting that higher alemtuzumab exposure created a favorable lower inflammatory homeostatic milieu prior to eti-cel infusion, which was associated with enhanced eti-cel expansion and higher response rates. Responders also maintained sustained low-level interleukin 2 (IL-2) secretion compared to non-responders. These findings support the investigation of a weight-based alemtuzumab dosing regimen to optimize lymphodepletion and the use of subcutaneous low-dose IL-2 to further enhance eti-cel expansion and treatment response.
Professor Bachy commented, “These encouraging data demonstrate that not only can eti-cel drive responses in a very difficult-to-treat population, but that by optimizing exposure to alemtuzumab we may be able to create a favorable environment for CAR-T expansion and persistence.” The NATHALI-01 study is open for recruitment, with full Phase 1 clinical data expected in Q4 2026. The poster presentation focused on alemtuzumab exposure and sustained IL-2 driving UCART20x22 expansion and clinical response in adults with relapsed or refractory B-cell non-Hodgkin lymphoma.
Cellectis is a clinical-stage biotechnology company focused on developing life-saving cell and gene therapies using its gene-editing platform. The company employs an allogeneic approach for CAR T immunotherapies in oncology, pioneering the concept of off-the-shelf, ready-to-use gene-edited CAR T-cells. Cellectis has headquarters in Paris, France, with operations in New York and Raleigh, NC. The company is listed on the Nasdaq Global Market (ticker: CLLS) and on Euronext Growth (ticker: ALCLS).
Source: Cellectis
