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] Efficacy - The overall response rate (ORR) for lasme-cel was reported at 68% in Process 2 (n=22), 83% at the recommended Phase 2 dose (RP2D, n=12), and 100% in the target Phase 2 population (n=9) [1][10] - In the target Phase 2 population, the complete remission (CR) and complete remission with incomplete hematologic recovery (CRi) rate was 56%, with approximately 80% of patients achieving minimal residual disease-negative (MRD-negative) status [1][15] Safety - Lasme-cel was generally well-tolerated in the Phase 1 study (n=40), with manageable adverse events including cytokine release syndrome (CRS) and immune effector cell-associated neurotoxicity syndrome (ICANS) [8][14] - Dose-limiting toxicities (DLTs) were uncommon, with only one case reported at Dose Level 3 [14] Durability - Patients who achieved MRD-negative CR/CRi had a median overall survival (OS) of 14.8 months [1] - Among patients who proceeded to hematopoietic stem cell transplantation (HSCT) after lasme-cel therapy, there was a trend towards longer overall survival compared to those who did not undergo transplant [12] Commercial Opportunity - Cellectis estimates potential peak gross sales of lasme-cel could reach approximately $700 million across the U.S., EU4, and UK by 2035, with an addressable market of about 1,900 patients annually [2][20] - The projected pricing for lasme-cel is approximately $365,000 in the EU4 and UK, and $515,000 in the U.S., with a potential compound annual growth rate (CAGR) of ~5% [19][20] Strategic Positioning - Lasme-cel is positioned as a potentially game-changing therapy for r/r B-ALL, offering an alternative to CD19-targeted therapies with off-the-shelf availability and deep MRD-responses [4][18] - The company aims to address the significant unmet need for heavily pretreated patients, with expectations from key opinion leaders indicating a preference for lasme-cel among eligible patients [18]