Cellectis' R&D Day Highlights Lasme-cel's Potential to Address Significant Unmet Need for Patients with r/r B-ALL

Core Insights - Cellectis has initiated the pivotal Phase 2 BALLI-01 study for lasme-cel (UCART22) targeting relapsed/refractory B-cell acute lymphoblastic leukemia (r/r B-ALL) patients who are ineligible for transplant, with promising clinical data from the Phase 1 study [2][3][13] - The company estimates potential peak gross sales of lasme-cel could reach approximately $700 million across the U.S., EU4, and UK by 2035, with a significant addressable market for heavily pretreated patients [2][19] Clinical Efficacy - In the Phase 1 study, lasme-cel demonstrated an overall response rate (ORR) of 68% with Process 2, increasing to 83% at the recommended Phase 2 dose (RP2D), and achieving 100% in the target Phase 2 population [1][10] - The complete remission (CR) and complete remission with incomplete hematologic recovery (CRi) rate was 56% in the target Phase 2 population, with approximately 80% of these patients achieving minimal residual disease (MRD)-negative status [1][15] - Among patients previously treated with all three targeted therapies, 73% responded to lasme-cel, with 64% achieving MRD-negative status [1][11] Safety Profile - Lasme-cel was generally well-tolerated in the Phase 1 study, with manageable adverse events including cytokine release syndrome (CRS) occurring in 2.5% of patients and immune effector cell-associated neurotoxicity syndrome (ICANS) in 5% [8][14] - The study reported only one case of grade 2 immune effector cell-associated hemophagocytic syndrome (IEC-HS), which resolved [1][8] Commercial Opportunity - Cellectis estimates that lasme-cel could address approximately 1,900 patients annually in the 3L+ B-ALL category by 2035, with a significant portion of the projected incident 1L treated B-ALL population expected to progress to later lines of therapy [17][18] - The company anticipates a pricing strategy that could position lasme-cel at approximately $365,000 in the EU4 and UK, and $515,000 in the U.S. by 2025, with potential for significant revenue growth if approved for commercialization [19][19] Strategic Developments - Cellectis is advancing its pivotal Phase 2 program for lasme-cel and plans to submit a Biologics License Application (BLA) in 2028 following successful meetings with the FDA and EMA [13][19] - The company is also engaged in a strategic partnership with AstraZeneca to develop up to 10 novel cell and gene therapy products, leveraging Cellectis' gene editing expertise [22]