Cabaletta Bio, Inc. (Nasdaq: CABA) has announced its third-quarter 2024 financial results and provided a business update, highlighting significant progress in its clinical development programs and corporate strategy. The company is focused on developing targeted cell therapies for autoimmune diseases.
Clinical Trial Advancements
Cabaletta Bio's RESET clinical trial program for CABA-201 has expanded to 40 active clinical sites across the United States. As of November 12, 2024, 16 patients have been enrolled, and 10 have been dosed. The company plans to present clinical data from the RESET-Myositis and RESET-SLE trials, along with initial data from the RESET-SSc trial, at the ACR Convergence 2024 conference.
Steven Nichtberger, M.D., Chief Executive Officer of Cabaletta, stated, "Focused clinical execution has resulted in 40 U.S. clinical sites actively recruiting patients for the RESET clinical trial program for CABA-201... We are looking forward to sharing clinical safety and efficacy data from the first 8 myositis, lupus and scleroderma patients..."
European Expansion
Cabaletta Bio has received Clinical Trial Application (CTA) authorization from the European Medicines Agency (EMA) for CABA-201 in systemic lupus erythematosus (SLE). This authorization allows the company to activate clinical trial sites and pursue patient enrollment in Europe, marking a significant expansion of its clinical development program.
To support this expansion, Cabaletta has appointed Gerwin Winter as Senior Vice President and Head of International. Winter's experience at BeiGene, Portola, Celgene, and BMS is expected to be valuable in establishing and growing Cabaletta's presence in Europe.
Financial Position
As of September 30, 2024, Cabaletta Bio reported cash, cash equivalents, and short-term investments totaling $183.0 million. The company anticipates that these funds will be sufficient to support operations into the first half of 2026.
While the cash position remains strong, research and development expenses increased to $26.3 million for the three months ended September 30, 2024, compared to $13.8 million for the same period in 2023. General and administrative expenses also rose to $6.8 million from $4.9 million in the prior year.
CABA-201: A Potential Curative Therapy
CABA-201 is an autologous, engineered T cell therapy designed with a chimeric antigen receptor (CAR) that targets CD19-positive cells. The therapy aims to deplete B cells, which contribute to the initiation and maintenance of various autoimmune diseases. Cabaletta believes that a single dose of CABA-201 has the potential to reset the immune system, leading to compelling clinical responses without the need for chronic immunosuppressant therapy.
Cabaletta is currently evaluating CABA-201 in the RESET clinical development program, which includes multiple disease-specific clinical trials across rheumatology, neurology, and dermatology.
Upcoming Milestones
Cabaletta Bio anticipates meeting with the FDA in 2025 to discuss potential registrational program designs for CABA-201, contingent on data availability. The company is also planning to present initial clinical data from the RESET-MG trial in generalized myasthenia gravis in the first half of 2025.
Cabaletta's manufacturing capabilities have been enhanced by demonstrating that large-scale runs using 200mL whole blood collections yield similar amounts of CD19-CAR T cells as runs using leukapheresis material, with similar cytotoxicity.
