Cabaletta Bio (CABA) Guggenheim Securities Emerging Outlook: Biotech Summit 2026 summary

Strategic program updates

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

• Automated manufacturing for rese-cel enables scalable production with minimal capital investment.

• 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 without preconditioning.

• Outpatient administration is being adopted due to a strong safety profile and low rates of severe adverse events.

Clinical trial design and regulatory alignment

• Myositis pivotal trial uses a single-arm, 17-patient design, with statistical significance achieved if 5 patients respond.

• Focused on dermatomyositis patients to maximize probability of success based on prior data.

• FDA has endorsed the single-arm trial approach for CAR-T in autoimmune diseases, aligning with industry trends.

• Stringent, objective endpoints require patients to be off immunomodulatory medications and on low-dose steroids.

• Regulatory acceptance is reinforced by similar approaches from other major industry players.

Manufacturing and scalability

• Fully automated manufacturing with Cellares allows for rapid scaling to thousands of patients without major capital outlay.

• Partnership with Cellares positions the company years ahead in automation compared to competitors.

• Plan to maintain at least two sources of product supply, including both automated and manual CDMOs.

• Automated manufacturing reduces cost of goods by eliminating unused capacity and minimizing failures.

• Expectation of industry-low cost of goods due to efficient processes and healthier patient population.