Cabaletta Bio

Cabaletta Bio's rese-cel CAR-T therapy achieved complete B cell depletion and meaningful clinical responses in pemphigus vulgaris patients without requiring preconditioning chemotherapy.

The FDA approved nipocalimab (Imaavy) in May 2025 as the first therapy targeting the underlying cause of generalized myasthenia gravis in patients aged 12 and older with specific antibodies.

Cabaletta Bio announced an underwritten public offering of common stock and warrants on June 11, 2025, with expected closing on June 12, 2025.

• Cellares has successfully completed a Technology Adoption Program with Cabaletta Bio, demonstrating their Cell Shuttle platform can automate and scale manufacturing of rese-cel, a CAR-T therapy for autoimmune diseases including myositis, scleroderma, and lupus nephritis. • The company has also formed a strategic partnership with the University of Wisconsin School of Medicine and Public Health to automate production of a CRISPR-edited GD2 CAR-T therapy for solid tumors, addressing manufacturing bottlenecks in clinical development. • Cellares' Integrated Development and Manufacturing Organization (IDMO) Smart Factories in the US, Europe, and Japan aim to reduce manufacturing costs by up to 50% while increasing production capacity tenfold compared to conventional CDMOs.

Clinical trials demonstrate significant efficacy of rese-cel across multiple autoimmune conditions, with three SLE patients achieving DORIS remission and successful discontinuation of immunosuppressants and steroids.

• Cabaletta Bio has received FDA clearance to proceed with RESET-MS trial, testing their B-cell-targeting CAR T-cell therapy rese-cel in relapsing and progressive multiple sclerosis patients. • The innovative cell therapy, rese-cel (formerly CABA-201), aims to reset the immune system by targeting CD19-positive B-cells, potentially offering a curative approach for autoimmune diseases. • The trial will enroll adults aged 18-60 with either relapsing MS who experienced recent relapses or progressive MS patients showing worsening disability, following specific preconditioning treatment.

Cabaletta Bio will present updated clinical data on rese-cel at upcoming scientific meetings in February 2025, showcasing its potential in autoimmune diseases.

• A phase 1/2 study of CD19 CAR-T therapy in autoimmune diseases, including lupus, myositis, and systemic sclerosis, demonstrates significant clinical responses. • The CASTLE study reports minimal toxicities, with low-grade cytokine release syndrome and no major hematotoxicities or neurotoxicity observed in patients. • Cabaletta Bio's CABA-201 shows potential for immunosuppressant-free clinical responses in refractory autoimmune diseases, with ongoing trials and planned FDA meetings. • Next-generation manufacturing processes for CAR-T therapies, like that used for CC-97540, offer faster production times and require fewer cells for infusion.

• Cabaletta Bio's RESET program, featuring CABA-201, is actively enrolling patients across multiple clinical trials targeting autoimmune diseases like myositis, SLE, and SSc. • The company has expanded its clinical trial program into Europe, securing EMA approval for the RESET-SLE trial, and is actively recruiting at 40 U.S. sites. • Cabaletta Bio anticipates a meeting with the FDA in 2025 to discuss program designs for CABA-201, a cell therapy aimed at resetting the immune system in autoimmune disorders. • With $183.0 million in cash reserves as of September 30, 2024, Cabaletta Bio expects to fund operations into the first half of 2026, supporting ongoing clinical development and strategic expansion.

• Cabaletta Bio reported clinical data from RESET-Myositis™, RESET-SLE™, and initial data from RESET-SSc™ trials at ACR Convergence 2024, showcasing CABA-201's potential in autoimmune disease treatment. • Sixteen patients have been enrolled, with ten dosed across the RESET™ clinical development program, and 40 U.S. clinical sites are actively recruiting patients for CABA-201 trials. • Cabaletta anticipates a meeting with the FDA in 2025 to discuss potential registrational program designs for CABA-201, pending data outcomes. • EMA has authorized clinical trials for CABA-201 in Europe for lupus, expanding the clinical development program internationally.