Cellectis SA (NASDAQ:CLLS) saw its stock rise approximately 5% in premarket trading on Wednesday after the company disclosed that the U.S. Food and Drug Administration has granted Regenerative Medicine Advanced Therapy, or RMAT, designation to its blood cancer therapy candidate.
The designation applies specifically to lasmecabtagene timgedleucel, an allogeneic CAR-T cell therapy designed to target the CD22 antigen. The regulatory recognition covers the intended treatment of patients with relapsed or refractory B-cell acute lymphoblastic leukemia, according to the company announcement.
Cellectis said the FDA's RMAT decision is supported by data from the Phase 1 BALLI-01 trial, which demonstrated an efficacy and safety profile that underpins the regulatory submission. The company intends to present the final Phase 1 results from BALLI-01 at the 2026 Congress of the European Hematology Association on June 13. The scheduled presentation will be delivered by Nitin Jain, Professor of Medicine in the Department of Leukemia at MD Anderson Cancer Center.
In parallel with the RMAT designation and the forthcoming data presentation, Cellectis noted that the pivotal Phase 2 portion of the BALLI-01 trial is actively enrolling patients. Information on participant eligibility criteria and the clinical centers taking part in the study is posted on clinicaltrials.gov under identifier NCT04150497.
Founded as a clinical-stage biotechnology company, Cellectis uses gene editing platforms to develop cell and gene therapies. The company said the RMAT designation relates to its CD22-targeting allogeneic CAR-T program and follows the Phase 1 evidence the firm presented.
Context and next steps
The immediate market reaction reflected investor attention to the regulatory milestone and the upcoming presentation of the final Phase 1 data. The pivotal Phase 2 enrollment status means additional clinical data may follow as the trial progresses, and details on eligibility and participating centers are available publicly through the ClinicalTrials.gov record cited by the company.
This article reports the facts provided in the company announcement and related trial documentation. It does not add information beyond the statements and trial identifiers Cellectis disclosed.