Cabaletta Bio (CABA) Corporate presentation summary

Strategic vision and pipeline

• Aims to develop and launch the first curative targeted cellular therapies for autoimmune diseases, focusing on rese-cel (CD19-CAR T) across multiple indications.

• RESET program includes six trials in myositis, SLE/LN, systemic sclerosis, myasthenia gravis, and pemphigus vulgaris, targeting high unmet needs.

• FDA alignment achieved for registrational trial designs in myositis and SLE, with further alignment for SSc and MG expected in 2026.

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

• Multiple clinical and regulatory catalysts anticipated in 2026, including pivotal data readouts and BLA submission for myositis in 2027.

Clinical data and efficacy

• Rese-cel demonstrated immunomodulator-free efficacy and favorable safety in autoimmune patients, with major clinical responses in myositis, SLE, SSc, MG, and PV.

• In myositis, 3/3 DM patients with sufficient follow-up achieved major TIS responses at 16 weeks; similar efficacy seen in ASYS and SSc cohorts.

• SLE and LN patients showed significant SLEDAI-2K improvements and anti-dsDNA reduction after discontinuing immunomodulators.

• Early data in PV patients without preconditioning showed compelling clinical activity and B cell depletion.

• MG patients demonstrated improvement in MG-ADL and QMG scores off immunomodulators and steroids.

Safety and outpatient potential

• Safety profile in first 40 patients: 95% had no or only Grade 1 CRS, and 95% had no ICANS, supporting outpatient administration.

• Outpatient model reduces resource burden, increases patient access, and supports favorable reimbursement, especially for commercially insured populations.

• Lower incidence and delayed onset of CRS/ICANS compared to oncology CAR T therapies.