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]

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]