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 (CABA-201), a CD19-CAR T therapy designed for autoimmunity.

• RESET clinical program covers myositis, SLE, systemic sclerosis, myasthenia gravis, and pemphigus vulgaris, with pivotal and registrational trials underway.

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

• Automated manufacturing and elimination of preconditioning (PC) are central to expanding access, reducing costs, and enabling outpatient administration.

• Commercial model leverages healthier patient populations and automation to improve manufacturing efficiency and scalability.

Clinical data and efficacy

• Rese-cel demonstrated immunomodulator-free efficacy and favorable safety in multiple autoimmune indications, with major clinical responses in myositis, SLE, systemic sclerosis, and myasthenia gravis.

• In myositis, 3 of 3 dermatomyositis patients with sufficient follow-up achieved major TIS responses at 16 weeks off immunomodulators and steroids.

• SLE and lupus nephritis patients showed significant reductions in disease activity and anti-dsDNA antibodies after rese-cel infusion.

• Systemic sclerosis patients achieved FVC stabilization or improvement and meaningful clinical responses off immunomodulators.

• Early data in pemphigus vulgaris without preconditioning showed compelling clinical activity and B cell depletion in 2 of 3 patients.

Safety and outpatient potential

• Across 40 patients, 95% experienced no or only Grade 1 CRS, and 95% had no ICANS, supporting outpatient administration.

• Safety profile contrasts favorably with oncology CAR T therapies, which often require inpatient care due to higher toxicity.

• Outpatient model reduces resource utilization, increases throughput, and improves patient/provider accessibility.