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 (CD19-CAR T) as a transformative platform across multiple indications.

• RESET program advances trials in myositis, SLE, systemic sclerosis, myasthenia gravis, and pemphigus vulgaris, targeting high unmet needs with broad clinical reach.

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

• Pipeline includes both preconditioning (PC)-free and traditional cohorts, with PC elimination expected to expand access and outpatient use.

• Automated manufacturing partnerships, such as with Cellares, aim to enable global scalability and lower cost of goods sold (COGS).

Clinical data and efficacy

• Rese-cel demonstrates immunomodulator-free efficacy and favorable safety in phase 1/2 trials, with all eligible patients meeting primary endpoints in lupus and systemic sclerosis.

• Myositis cohort shows major TIS responses at 16 weeks post-infusion, supporting a planned 2027 BLA submission.

• In pemphigus vulgaris, 2 of 4 refractory patients achieved compelling responses without preconditioning, with complete B cell elimination in most cases.

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

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

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 with oncology CAR T, where inpatient care is often required due to higher toxicity.

• Outpatient model reduces hospital resource use, increases patient throughput, and may improve reimbursement dynamics.