Cabaletta Bio (CABA) investor relations material

Strategic program updates and clinical progress

• Initiated enrollment in a pivotal 17-patient myositis trial, aiming for a BLA filing next year.

• Phase 1/2 data in lupus, scleroderma, and myasthenia gravis expected in the first part of the year.

• Demonstrated effective outcomes in pemphigus vulgaris patients using rese-cel without preconditioning.

• Opened a lupus cohort for no-preconditioning studies, with data anticipated this year.

• Monthly spend is $10–13 million, expected to rise with commercial buildout.

Manufacturing innovation and scalability

• Cleared IND amendment for fully automated, human-free manufacturing of rese-cel.

• Automated system enables scalability to 5,000–15,000 patients with minimal capital investment and strong margins.

• Partnership with Cellares allows for rapid scale-up without the need for additional clean rooms.

• Plan to maintain at least two manufacturing sources, including Cellares and existing CDMOs.

• Automated manufacturing expected to deliver industry-low cost of goods and biologic-like margins.

Regulatory and trial design insights

• FDA has endorsed the single-arm pivotal trial design for myositis, requiring 5 of 17 patients to achieve response for statistical significance.

• Trial design optimized for dermatomyositis, with 14 of 17 patients in this subgroup.

• Stringent, objective endpoints include drug-free remission and low-dose steroids, aligning with recent FDA guidance.

• Single-arm trials are increasingly accepted in autoimmune CAR-T studies, with major industry peers following similar approaches.