Cabaletta Bio (CABA) investor relations material

Key data presentations and clinical milestones

• Upcoming data releases in October will cover phase 1/2 myositis patients, scleroderma, lupus, and myasthenia gravis, with pivotal program agreements expected by year-end.

• Myositis is the lead indication, with a registrational approach agreed upon with the FDA, targeting a BLA filing in 2027.

• The pivotal myositis trial will enroll 14–15 patients per cohort, aiming to access a large US patient population.

• No preconditioning data for ResiCel will be presented, potentially offering a significant market advantage if successful.

• Enrollment rates are strong, with over 60% of the 100-patient safety target already met.

Competitive landscape and strategic positioning

• Myositis was chosen over lupus due to less competition and a clearer path to first-to-market status.

• Most competitors in lupus use preconditioning; success in a no preconditioning regimen could provide a multi-year lead.

• The company’s translational research and unique clinical insights are seen as key differentiators.

• Rapid manufacturing is less critical to physicians than efficacy, safety, and the need for lymphodepletion.

Market opportunity and patient impact

• Myositis represents a large, underserved market with high unmet need and limited approved therapies.

• Current therapies are expensive and not curative; new data suggest a transformative impact for patients.

• The autologous business model is improving, with lower costs and better supply, supporting commercial viability.

• Patient and physician enthusiasm is high, with strong demand and positive clinical outcomes driving enrollment.