Cabaletta Bio (CABA) Morgan Stanley 22nd Annual Global Healthcare Conference summary

Strategic focus and clinical program design

• Autoimmune CAR-T therapies remain the central focus, with CABA-201 as the lead candidate.

• CABA-201 is a 4-1BB-containing CD19 CAR-T, uniquely designed for autoimmune patients and advanced through multiple INDs for distinct indications.

• Separate INDs for each indication allow for tailored safety monitoring and potential registrational pathways after six patients per cohort.

• Fast Track designations enable real-time FDA engagement and flexible trial design.

• The clinical RESET program uses a single starting dose across all indications, based on preclinical and translational data.

Clinical site expansion and competitive positioning

• 24 company-sponsored, IND-cleared clinical sites are open and enrolling, the most in the U.S. for autoimmune CAR-T.

• Significant head start over competitors, with the next closest having 15 sites; allogeneic players collectively have 10.

• Clinical site engagement is seen as a strategic asset due to increasing competition and limited site capacity.

• Enrollment has accelerated post-initial data presentation, with nine patients enrolled as of August 10 and continued momentum.

Clinical data and safety learnings

• Initial data from myositis and lupus patients showed strong efficacy and pristine safety in early cases.

• A lupus nephritis patient experienced Grade 4 ICANS, which was resolved with standard treatment; this led to protocol refinements, including seizure prophylaxis.

• No protocol changes were required by the DSMB or FDA, and investigator and patient enthusiasm remained high.

• Enrollment and site activation continued to accelerate despite the adverse event.