PHILADELPHIA, March 21, 2024 (GLOBE NEWSWIRE) — Cabaletta Bio, Inc. (Nasdaq: CABA), a clinical-stage biotechnology company focused on developing and launching the first curative targeted cell therapies designed specifically for patients with autoimmune diseases, today reported financial results for the fourth quarter and full year ended December 31, 2023, and provided a business update.
“Throughout the past year, we set the foundation to enable an efficient development strategy for CABA-201 across a broad range of autoimmune diseases. Clinical sites across the United States are actively recruiting for our myositis and SLE trials, and the first patient has been dosed with no CRS or ICANS of any grade observed as of 21 days following CABA-201 infusion. We look forward to building on this momentum with expansion into additional clinical sites and to delivering data across the RESET program in 2024,” said Steven Nichtberger, M.D., Chief Executive Officer and Co-founder of Cabaletta. “By advancing individual, company-sponsored trials for each autoimmune disease without the need for an initial dose escalation study, we believe we have an accelerated path to initiate discussions with the FDA on registrational cohorts and/or studies following treatment of six patients in any one of the nine cohorts in our current RESET clinical trial program. Our commitment to exploring the broad potential of CABA-201 for patients is further demonstrated by the recently granted Rare Pediatric Disease designation in juvenile dermatomyositis, an indication in which there are no FDA approved therapies. Based on our strong balance sheet and a differentiated CABA-201 development strategy, we believe we are well positioned to develop and launch the first targeted, and perhaps curative, cell therapy specifically designed for patients with autoimmune diseases.”
Recent Operational Highlights and Upcoming Anticipated Milestones
Chimeric Antigen Receptor T cells for Autoimmunity (CARTA) Strategy
CABA-201: Autologous, engineered T cells with a chimeric antigen receptor containing a fully human CD19 binder and a 4-1BB co-stimulatory domain as a potential treatment for a broad range of autoimmune diseases across multiple therapeutic portfolios where B cells contribute to the initiation and/or maintenance of disease.
Rheumatology Portfolio
- CABA-201 in myositis (idiopathic inflammatory myopathies)
- The first patient has been dosed in the Phase 1/2 RESET-Myositis trial with no CRS (cytokine release syndrome) or ICANS (immune effector cell-associated neurotoxicity syndrome) of any grade observed for the first 21 days of a 28-day dose-limiting toxicity observation window following administration.
- Enrollment in the Phase 1/2 RESET-Myositis trial is ongoing across multiple sites in the U.S.
- CABA-201 in systemic lupus erythematosus (SLE)
- Enrollment is underway in the Phase 1/2 RESET-SLE trial, which is being conducted across multiple sites in the U.S.
- In March 2024, Health Canada issued a No Objection Letter (NOL) in response to a Clinical Trial Application for the RESET-SLE trial submitted by Cabaletta. The NOL allows for Cabaletta to begin the process to activate clinical trial sites and pursue patient enrollment for the RESET-SLE trial in Canada.
- CABA-201 in systemic sclerosis (SSc)
- In October 2023, Cabaletta received Investigational New Drug (IND) application clearance from the U.S. Food and Drug Administration (FDA) for the Phase 1/2 RESET-SSc trial.
- Cabaletta anticipates reporting initial clinical data from the Phase 1/2 RESET-SSc trial in the second half of 2024.
- CABA-201 regulatory designation updates
- In January 2024, Cabaletta announced that CABA-201 was granted Fast Track Designations by the FDA for the treatment of patients with dermatomyositis to improve disease activity and for the treatment of patients with SSc to improve associated organ dysfunction.
- In February 2024 and March 2024, Cabaletta announced that CABA-201 was granted Orphan Drug Designation by the FDA for the treatment of idiopathic inflammatory myopathies (IIM, or myositis) and for the treatment of systemic sclerosis, respectively.
- Cabaletta has been recently granted Rare Pediatric Disease Designation for the treatment of juvenile dermatomyositis. This designation may allow the Company to be eligible for a priority review voucher, assuming reauthorization of the program by the U.S. federal government, for a subsequent marketing application at the time of marketing approval for CABA-201.
Neurology Portfolio
- CABA-201 in generalized myasthenia gravis (gMG)
- In November 2023, Cabaletta announced that its IND application for CABA-201 was allowed to proceed by the FDA for the Phase 1/2 RESET-MG trial.
- Cabaletta anticipates reporting initial clinical data from the Phase 1/2 RESET-MG trial in the second half of 2024.
External Scientific Presentations
- In February 2024, Cabaletta presented a poster presentation on new preclinical CABA-201 specificity and activity data for treatment-resistant autoimmune disease at the 2024 Tandem Meetings | Transplantation & Cellular Therapy (TCT) Meetings of ASTCT® (American Society for Transplantation and Cellular Therapy) and CIBMTR® (Center for International Blood and Marrow Transplant Research).
- In March 2024, Cabaletta presented a poster presentation on new preclinical specificity and activity data in treatment resistant myositis at the 5th Global Conference on Myositis. In addition, David J. Chang, M.D., Chief Medical Officer of Cabaletta, moderated a symposium featuring Carl H. June, M.D., and Rohit Aggarwal, M.D., titled “The Next Frontier for CAR T Cells: Autoimmune Disease” on March 14, 2024.
Chimeric AutoAntibody Receptor T (CAART) cells Strategy
- DSG3-CAART: Cabaletta is evaluating desmoglein 3 chimeric autoantibody receptor T (DSG3-CAART) cells as a potential treatment for patients with mucosal pemphigus vulgaris (mPV). Enrollment in the combination cohort of the DesCAARTes™ trial is ongoing, where patients are pre-treated with intravenous immunoglobulin (IVIg), cyclophosphamide and fludarabine prior to DSG3-CAART infusion, with the aim of improving persistence and activation of DSG3-CAART.
- MuSK-CAART: Cabaletta is evaluating muscle-specific kinase (MuSK) chimeric autoantibody receptor T (MuSK-CAART) cells as a potential treatment for patients with MuSK-associated myasthenia gravis (MuSK MG). Enrollment in the Phase 1, open-label MusCAARTes™ study of MuSK-CAART in patients with MuSK autoantibody-positive MG is ongoing in a cohort without preconditioning.
Fourth Quarter and Full Year 2023 Financial Results
- Research and development expenses were $17.4 million and $55.4 million for the three months ended December 31, 2023, and the full year ended December 31, 2023, respectively, compared to $12.4 million and $39.3 million for the three months ended December 31, 2022, and the full year ended December 31, 2022, respectively.
- General and administrative expenses were $5.7 million and $19.2 million for the three months ended December 31, 2023, and the full year ended December 31, 2023, respectively, compared to $3.9 million and $14.8 million for the three months ended December 31, 2022, and the full year ended December 31, 2022, respectively.
- As of December 31, 2023, Cabaletta had cash, cash equivalents and short-term investments of $241.2 million, compared to $106.5 million as of December 31, 2022.
The Company expects that its cash, cash equivalents and short-term investments as of December 31, 2023, will enable it to fund its operating plan into the first half of 2026.
About CABA-201
CABA-201 is designed to deeply and transiently deplete CD19-positive B cells following a one-time infusion, which may enable an “immune system reset” with the potential for durable remission off therapy in patients with autoimmune diseases. To date, Cabaletta has received clearance from the FDA for four Investigational New Drug (IND) applications for CABA-201 in multiple autoimmune conditions including systemic lupus erythematosus (SLE), myositis, systemic sclerosis (SSc) and generalized myasthenia gravis (gMG). Cabaletta is conducting four Phase 1/2 RESET™ clinical trials with a total of nine cohorts that can advance simultaneously, employing a similar parallel cohort design and starting dose of 1 x 106 cells/kg without a dose escalation requirement.
About Cabaletta Bio
Cabaletta Bio (Nasdaq: CABA) is a clinical-stage biotechnology company focused on the discovery and development of engineered T cell therapies that have the potential to provide a deep and durable, perhaps curative, treatment for patients with autoimmune diseases. The CABA™ platform encompasses two strategies: the CARTA (chimeric antigen receptor T cells for autoimmunity) strategy, with CABA-201, a 4-1BB-containing fully human CD19-CAR T, as the lead product candidate being evaluated in the RESET™ (REstoring SElf-Tolerance) clinical trials in systemic lupus erythematosus, myositis, systemic sclerosis and generalized myasthenia gravis, and the CAART (chimeric autoantibody receptor T cells) strategy, with multiple clinical-stage candidates, including DSG3-CAART for mucosal pemphigus vulgaris and MuSK-CAART for MuSK myasthenia gravis. The expanding CABA™ platform is designed to develop potentially curative therapies that offer deep and durable responses for patients with a broad range of autoimmune diseases. Cabaletta Bio’s headquarters and labs are located in Philadelphia, PA.
