Cellectis reports promising clinical data for leukemia therapy

New York – Cellectis, a clinical-stage biotechnology company developing cell and gene therapies, presented promising clinical data from its Phase 1 BALLI-01 study of lasme-cel (UCART22). The therapy targets transplant-ineligible patients with relapsed or refractory B-cell acute lymphoblastic leukemia (r/r B-ALL).

The company also unveiled the design for the pivotal Phase 2 study, its planned registration pathway, and commercial opportunity. An R&D Day event featured hematology experts discussing lasme-cel's potential to address the significant unmet need in heavily pretreated patients.

Phase 1 data showed that lasme-cel achieved a response rate of 36% in patients dosed with Cellectis-manufactured product (Process 2). At the recommended Phase 2 dose (RP2D), the complete remission (CR) or CR with incomplete hematologic recovery (CRi) rate was 42%, with 80% of responders achieving minimal residual disease (MRD)-negative status.

For the specific population intended for the pivotal Phase 2 study (Process 2, DL3, age ≤ 50), the CR/CRi rate was 56%, with 80% of responders being MRD-negative. Lasme-cel was generally well-tolerated, with manageable adverse events, including cytokine release syndrome and immune effector cell-associated neurotoxicity syndrome.

Cellectis has initiated the pivotal Phase 2 BALLI-01 study and estimates the potential peak gross sales of lasme-cel to be up to $700 million across the U.S. and Europe. The company stated that lasme-cel could offer a faster and more scalable treatment option for patients where allogeneic CAR-T therapy is indicated.