Cabaletta Bio (CABA) Corporate presentation summary

Strategic vision and pipeline advancement

• 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 program includes trials in myositis, systemic sclerosis (SSc), systemic lupus erythematosus (SLE), myasthenia gravis (MG), and pemphigus vulgaris (PV), with plans for registrational studies and BLA submission in 2H27.

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

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

Clinical efficacy and safety data

• Single rese-cel infusion after discontinuing immunomodulators delivers immune reset with compelling outcomes in myositis, SSc, SLE, MG, and PV.

• In adult dermatomyositis, 83% of Phase 1/2 patients met the registrational endpoint, with responses maintained up to 1.5 years; similar results seen in juvenile DM.

• SSc patients showed FVC improvement or stabilization and high rCRISS response rates after immunomodulator discontinuation.

• Across 63 patients, 94% had no or only Grade 1 CRS, and 97% had no ICANS, supporting outpatient administration.

• PC-free cohorts in PV and lupus demonstrated deep B cell depletion and clinical activity at the lowest dose.

Market opportunity and commercial strategy

• Significant unmet need in targeted autoimmune indications: ~60k DM, ~90k SSc, ~210k SLE, ~110k LN, ~100k MG, and ~80k PV patients in the US.

• Commercial model leverages outpatient administration, automated manufacturing, and reduced COGS to improve scalability and margins.

• Three-phase launch strategy targets academic centers first, then expands to community hospitals and outpatient clinics.

• Automated manufacturing by Cellares enables scale to thousands of patients per year with minimal capital investment.