Cabaletta Bio (CABA) Corporate presentation summary

Strategic vision and pipeline

• Aims to develop and launch the first curative targeted cellular therapies for autoimmune diseases, focusing on rese-cel (CD19-CAR T) as a transformative approach for immune reset across multiple indications.

• Pipeline includes programs in myositis, systemic sclerosis (SSc), systemic lupus erythematosus (SLE), lupus nephritis (LN), myasthenia gravis (MG), and pemphigus vulgaris (PV), with plans to expand into larger indications like UC, T1D, and RA.

• RESET clinical trials are advancing in a broad portfolio, targeting high unmet needs and leveraging innovative clinical strategies for accelerated regulatory pathways.

• FDA Fast Track and RMAT designations have been received for multiple indications, supporting expedited development.

• Plans for a BLA submission in 2H27 for myositis, including adult and juvenile dermatomyositis, with potential for a priority review voucher.

Clinical efficacy and safety data

• Rese-cel demonstrated compelling efficacy: 83% of adult DM patients met registrational endpoints, with responses maintained up to 1.5 years; similar outcomes observed in JDM.

• In SSc, most patients with ILD showed FVC improvement or stabilization after discontinuing immunomodulators; 83% achieved rCRISS response at 24 weeks.

• SLE and LN patients experienced meaningful reductions in disease activity (SLEDAI-2K, PGA) after rese-cel, with durable responses off immunomodulators.

• Myasthenia gravis patients (both AChR-positive and negative) showed clinically meaningful improvements in MG-ADL and QMG scores after discontinuing all immunomodulators.

• Favorable safety profile: 94% of patients had no or Grade 1 CRS, and 97% had no ICANS, supporting outpatient administration.

Manufacturing innovation and scalability

• Automated manufacturing via Cellares enables scalable, cost-efficient production, with the potential to treat thousands of patients annually and achieve typical biologic margins.

• Elimination of preconditioning (PC-free) and apheresis (using whole blood collection) further reduces costs, improves patient experience, and expands access.

• Initial automated manufacturing runs met all release specifications, with comparable product quality and in vivo behavior to traditional processes.

• Commercial readiness includes partial automation at launch, with full automation planned post-launch and global expansion facilitated by reduced COGS.