Cabaletta Bio (CABA) 12th Annual Cantor Fitzgerald Global Healthcare Conference summary

Key program updates and clinical progress

• Rese-cel, an autologous CD19 CAR-T, uses a nine-day manufacturing process with a 4-1BB-containing, fully human binder and weight-adjusted dosing, showing durable efficacy in autoimmune diseases.

• Over 90 patients have been treated, with 94% experiencing no or only mild, transient CRS and 97% showing no ICANS; no IEC-HS events have been observed.

• Plans include initiating a pivotal scleroderma program by year-end, reporting pivotal myositis data by mid-next year, and filing for pediatric myositis by end of 2027.

• Outpatient administration and the potential for preconditioning-free regimens are key differentiators, aiming for broader access and improved reimbursement.

• A vision for apheresis-free, single-dose outpatient therapy is being pursued, with ongoing innovations to expand market opportunity.

Manufacturing strategy and differentiation

• The nine-day manufacturing process mirrors approved CAR-Ts and is distinct from competitors using shorter, riskier processes.

• Shorter manufacturing increases pro-inflammatory cytokines, raising safety risks in autoimmune patients; the standard process minimizes these risks.

• Redundant manufacturing partnerships with Soliris and ElevateBio ensure scalability, supply security, and competitive cost of goods.

• Soliris and ElevateBio have demonstrated process comparability, with FDA recognizing interchangeability for clinical supply.

• Current market conditions favor autologous CAR-T manufacturing, with multiple CDMOs competing to support rese-cel.

Safety, market positioning, and future outlook

• Safety profile is a major differentiator, especially after recent competitor safety events; rese-cel is the only actively enrolling trial among major players.

• Outpatient treatment is enabled by safety data, addressing capacity and reimbursement challenges in large autoimmune populations.

• Preconditioning-free regimens are a top patient and physician priority, with ongoing studies to validate this approach.

• Upcoming milestones include pivotal data releases, BLA filings, and potential for a priority review voucher in pediatric indications.

• The team’s UPenn roots and focus on data integrity have guided strategic decisions and product differentiation.