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) as a transformative approach across multiple indications.

• RESET™ clinical program includes pivotal and registrational trials in myositis, SLE, systemic sclerosis, myasthenia gravis, and pemphigus vulgaris, with FDA Fast Track and RMAT designations in key indications.

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

Clinical data and efficacy

• Rese-cel demonstrated immunomodulator-free efficacy and favorable safety in phase 1/2 trials, 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 low/no steroids.

• SLE and lupus nephritis patients showed significant SLEDAI-2K improvements and anti-dsDNA reduction after rese-cel infusion.

• Systemic sclerosis patients achieved FVC stabilization/improvement and skin score reductions, with meaningful responses off immunomodulators.

• Myasthenia gravis patients (both AChR-positive and negative) showed clinically meaningful improvements in MG-ADL and QMG scores after discontinuing immunomodulators.

Safety and outpatient potential

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

• Rese-cel’s safety profile contrasts with oncology CAR T, enabling outpatient use and favorable reimbursement models.

• Outpatient administration reduces hospital resource use, increases patient throughput, and avoids conflicts with cancer patient bed needs.