Cabaletta Bio (CABA) investor relations material

Strategic vision and pipeline

• Aims to develop and launch the first curative targeted cellular therapies for autoimmune diseases, focusing on rese-cel (resecabtagene autoleucel), a CD19 CAR T cell therapy.

• RESET clinical program includes disease-specific cohorts for myositis, SLE/LN, systemic sclerosis, myasthenia gravis, multiple sclerosis, and pemphigus vulgaris.

• FDA Fast Track and RMAT designations received for multiple indications, supporting accelerated regulatory pathways.

• Registrational cohorts are designed to evolve directly from ongoing Phase 1/2 trials, with myositis registrational trial initiation in 4Q25 and BLA submission planned for 2027.

• Manufacturing partnerships and automation initiatives are in place to support clinical and commercial supply.

Clinical efficacy and safety data

• Transformative clinical responses observed in the majority of rese-cel patients after discontinuing immunomodulators, with durable drug-free remissions in myositis, SLE/LN, and systemic sclerosis.

• In myositis, all DM/ASYS patients with ≥16 weeks follow-up met the primary endpoint; in SSc, all with ≥3 months follow-up achieved rCRISS-25 response off immunomodulators and steroids.

• SLE/LN patients showed high rates of DORIS and renal responses, with significant improvements in SLEDAI-2K and reduction in anti-dsDNA antibodies.

• Safety profile in first 40 patients dosed with preconditioning showed 95% had no or only Grade 1 CRS, and 95% had no ICANS.

• Initial data in pemphigus vulgaris without preconditioning showed compelling clinical activity and similar safety to preconditioned cohorts.

Market opportunity and unmet need

• High unmet need in targeted autoimmune indications: myositis (~80k US patients), SLE/LN (~320k), SSc (~90k), MG (~100k), MS, and PV.

• Existing therapies are limited, often with significant side effects and high rates of refractory disease.

• Rese-cel targets patients with moderate to severe, refractory disease, with market research indicating substantial eligible populations for each indication.