Cabaletta Bio (CABA) H.C. Wainwright 26th Annual Global Investment Conference 2024 summary

Company overview and clinical program

• Focused on developing targeted curative cellular therapies for autoimmune diseases since 2018.

• In-licensed CABA-201 in 2022, rapidly advancing to multiple IND filings for various indications.

• Clinical programs address myositis, lupus, myasthenia gravis, scleroderma, and pemphigus.

• Largest US network of company-sponsored, IND-cleared clinical sites actively enrolling.

• Currently operating 28 open clinical sites, with rapid recent expansion.

Clinical data and safety learnings

• Initial data from first two patients showed correct dose selection and clean safety profile at EULAR.

• A third patient with severe lupus nephritis experienced a grade four ICANS event, which resolved with routine care.

• Key learning: patients with recent disease exacerbation are at higher risk for adverse events.

• Protocol updated to require at least two weeks from last active disease event before infusion and added seizure prophylaxis.

• Seizure prophylaxis now standard, aligning with leading academic practices.

Enrollment, site strategy, and regulatory approach

• Despite safety event, clinical site openings and patient enrollment have accelerated.

• FDA and DSMB supported continuation without major protocol changes.

• Separate INDs for each indication allow precise allocation of safety data and flexible trial design.

• Extensive site network positions program for rapid phase III transition and registration discussions.

• Enrollment pace now exceeds expectations, supporting robust data generation.