Cabaletta Bio (CABA) 2024 Wells Fargo Healthcare Conference summary

Program overview and strategy

• Focused on developing curative cell therapies for autoimmune diseases, leveraging a proprietary CAART platform and in-licensed CABA-201, a fully human CD19 CAR-T product designed exclusively for autoimmune indications.

• Built a broad clinical program with nine cohorts across four studies targeting myositis, lupus, scleroderma, and myasthenia gravis, each under separate INDs for data purity and regulatory flexibility.

• Achieved significant clinical site expansion, now with 26 open sites in the US, outpacing competitors and enabling rapid patient enrollment.

• Emphasized company-sponsored, IND-cleared trials over investigator-initiated studies to prioritize launch readiness and regulatory alignment.

• Designed trials for efficient expansion and rapid transition to registrational studies, especially in rare indications with high unmet need.

Clinical data and safety updates

• Early data from first two patients (myositis and lupus) showed no CRS or ICANS, with translational findings closely matching academic benchmarks.

• A grade four ICANS event occurred in a lupus patient with recent hyperinflammatory history; managed successfully with no sequelae, leading to protocol updates including seizure prophylaxis and stricter infusion timing.

• DSMB and FDA reviewed the adverse event, recommended no dose changes, and enrollment accelerated post-event.

• Hospitalization protocol is four days post-infusion, shorter than typical 10–14 days for similar therapies, though not seen as a key differentiator.

• Safety learnings are being shared with the field, and further data publication is planned.

Differentiation and future directions

• Differentiation centers on breadth of indications, weight-adjusted dosing, and company-sponsored trial rigor, aiming to replicate high-quality academic results.

• Exploring CABA-201 in pemphigus without preconditioning, which could be a major advance if successful, with first sites now enrolling.

• Manufacturing capacity is a current focus due to strong enrollment momentum.

• Upcoming data releases expected by year-end, including updates from lupus, myositis, scleroderma, myasthenia gravis, and pemphigus cohorts.

• Strategic design allows for rapid cohort expansion and efficient regulatory engagement, positioning for multiple approval pathways in 2024–2025.