Cabaletta Bio (CABA) investor relations material

Company progress and clinical pipeline

• Enrollment initiated in a pivotal 17-patient myositis trial using a single-arm design agreed with FDA; fully automated manufacturing process implemented with Cellares.

• Acute data in pemphigus showed elimination or reduction of severe disease at starting dose without preconditioning; higher doses being explored due to favorable safety profile.

• Durability data in pemphigus and lupus expected later this year; lupus cohort now includes patients treated without preconditioning at the same initial dose.

• Complete phase I/II data in scleroderma, lupus nephritis, and myasthenia gravis to be presented in the first half of the year; alignment with FDA on a scleroderma pivotal trial targeted for this year.

• Acute and durability data from Cellares-manufactured product and high-dose, no-preconditioning cohorts in lupus and pemphigus expected in the near term.

Manufacturing and business model innovation

• Fully automated Cellares Cell Shuttle enables simultaneous manufacturing of 16 patient samples, reducing cost, capital investment, and need for skilled technicians.

• Manufacturing process allows for five doses per run, enabling redosing with minimal incremental cost and supporting outpatient treatment.

• Minimal capital investment required for scaling; two Cell Shuttles can support 1,000 patients per year, with cost structure competitive with any industry player.

• Automated manufacturing and outpatient focus create a fundamentally different, scalable, and profitable business model for autologous CAR T.

Safety, efficacy, and differentiation

• Across 40 patients in four indications, 95% experienced only grade 1 or no CRS, with no ICANS in over a year after stricter patient selection protocols.

• Safety profile compares favorably to existing autoimmune therapies; main side effect is transient fever, manageable without prophylactic steroids or tocilizumab.

• Differentiators include 4-1BB costimulatory domain, weight-based dosing, and a shortened manufacturing process yielding more activated CAR T-cells.

• No preconditioning regimen could significantly expand market opportunity, especially in lupus, by avoiding risks like ovarian failure and increasing patient willingness to enroll.

• Durable, drug-free remission of 18 months in 60–85% of patients would be a game changer and primary modality for cell therapy in lupus.