Cellectis reports Phase 1 data for leukemia therapy lasme-cel

Cellectis SA (NASDAQ: CLLS) reported Phase 1 clinical trial data for lasme-cel, an experimental therapy for patients with relapsed/refractory B-cell acute lymphoblastic leukemia who are ineligible for transplant.

The BALLI-01 Phase 1 study enrolled 40 patients aged 15-70 years with heavily pretreated disease. Results showed an overall response rate of 68% with lasme-cel Process 2 manufacturing (n=22), increasing to 83% at the recommended Phase 2 dose (n=12) and 100% in the target Phase 2 population (n=9).

Among patients who achieved minimal residual disease-negative complete remission, median overall survival was 14.8 months. In the target Phase 2 population, 56% achieved complete remission with approximately 80% reaching minimal residual disease-negative status. All patients in this subset became transplant eligible, with 78% proceeding to transplant.

The therapy was generally well-tolerated, with cytokine release syndrome occurring in 2.5% of patients and immune effector cell-associated neurotoxicity syndrome in 5% of patients. Eight lasme-cel related serious adverse events were reported.

The company has initiated a pivotal Phase 2 trial following regulatory meetings with the FDA and European Medicines Agency. Cellectis expects to submit a biologics license application in 2028.

Cellectis estimates lasme-cel could potentially reach approximately 1,900 addressable third-line B-ALL patients annually by 2035 across the U.S., EU4 countries, and UK. The company projects potential peak gross sales of up to $700 million in these markets.

The company also presented preliminary data on eti-cel for non-Hodgkin lymphoma, showing an overall response rate of 86% and complete response rate of 57% in seven patients.

You May Also Be Interested In