Cellectis (ALCLS) investor relations material

Strategic focus and future plans

• Advancing an allogeneic, off-the-shelf CD22-targeted CAR T therapy (lasme-cel/UCART22) for relapsed/refractory acute lymphoblastic leukemia (ALL), with a pivotal phase 2 trial targeting patients aged 12–50 and a parallel pediatric plan for ages 0–12.

• Manufacturing is fully internalized, with facilities in Europe and the US, enabling rapid scale-up, cost control, and commercial readiness.

• Regulatory alignment achieved with EMA and FDA for a single-arm pivotal trial, using CR/CRi at 3 months as the primary endpoint and transplant eligibility as a key secondary endpoint.

• Global site expansion to 75 centers in North America and Europe, with first patient recruitment expected by year-end and BLA submission targeted for 2028.

• Future label expansions are planned for earlier lines of therapy, including second line and MRD-positive consolidation.

Clinical data and program differentiation

• Phase 1/2 data show high response rates (up to 100% in target phase 2 population) and deep MRD-negative remissions in heavily pretreated ALL patients, including those with prior CD19 and CD22 therapies.

• Median OS of 14.8 months for MRD-negative CR/CRi; most responders proceeded to transplant and showed improved survival outcomes.

• Safety profile is favorable, with low rates of severe cytokine release syndrome, neurotoxicity, and manageable infections.

• Internalized manufacturing process demonstrated superior product consistency and efficacy compared to external manufacturing.

• Allogeneic CAR T enables treatment of patients ineligible for autologous CAR T due to low T cell counts or urgent need, providing a significant clinical advantage.

Commercialization plans and market potential

• Peak annual sales for the initial third-line-plus indication are projected at up to $700 million in the US, EU4, and UK, with potential to reach $1.3 billion with label expansion.

• Pricing assumptions are $840,000 per treatment in the US and $365,000 in the EU by 2035, with a 65% market share expected in the target segment.

• Addressable patient population estimated at 1,900 annually in the US, EU4, and UK for the initial indication, with further upside from global expansion.

• Allogeneic approach and in-house manufacturing expected to drive higher margins and robust growth in the CAR-T market.

• High pricing potential, with illustrative anchor prices of $515,000 (U.S.) and $365,000 (EU4/UK) per treatment.