Cabaletta Bio Q1 2024 Financial Results and Business Update

Cabaletta Bio, Inc., a clinical-stage biotech firm specializing in targeted cell therapies for autoimmune diseases, released its financial report for Q1 2024 and provided updates on its ongoing projects. The company has reported significant progress in its clinical trials and innovations aimed at improving patient care and therapy delivery.

Steven Nichtberger, M.D., CEO and Co-founder of Cabaletta, highlighted the absence of cytokine release syndrome (CRS) or immune effector cell-associated neurotoxicity syndrome (ICANS) in the first patients treated in the RESET-Myositis and RESET-SLE trials. He anticipates presenting initial data from these trials at the EULAR 2024 Congress in June. Additionally, the company is exploring the use of CABA-201 without preconditioning by incorporating the RESET-PV sub-study within the ongoing DesCAARTes trial for pemphigus vulgaris. Another innovation includes the potential elimination of apheresis by using a blood draw for the CABA-201 manufacturing process, which was presented at the ASGCT meeting.

CABA-201 is an engineered T cell therapy with a chimeric antigen receptor targeting CD19, designed to treat various autoimmune diseases. The company is conducting multiple Phase 1/2 trials across several autoimmune conditions including myositis, systemic lupus erythematosus (SLE), systemic sclerosis (SSc), generalized myasthenia gravis (gMG), and pemphigus vulgaris (PV).

In the realm of myositis, the Phase 1/2 RESET-Myositis trial began dosing patients in March 2024. No severe side effects were observed, and patient enrollment continues. Clinical data from the first patient in this trial will be presented at the EULAR 2024 Congress. The U.S. FDA has granted CABA-201 several regulatory designations for myositis, including Fast Track and Orphan Drug Designations.

Regarding SLE, the RESET-SLE trial also dosed its first patient with no adverse CRS or ICANS observed. Enrollment is ongoing, and initial data will be presented in June 2024. Health Canada has approved the initiation of clinical trial sites for this trial in Canada.

For systemic sclerosis, the FDA has granted Fast Track and Orphan Drug Designations for CABA-201, and initial data from the Phase 1/2 RESET-SSc trial is expected in the latter half of 2024.

In dermatology, the RESET-PV sub-study within the DesCAARTes trial will evaluate CABA-201 without preconditioning in patients with mucosal and mucocutaneous pemphigus vulgaris.

In neurology, the RESET-MG trial for generalized myasthenia gravis expects to report initial data in the second half of 2024.

Cabaletta has shared preclinical data demonstrating the feasibility of using blood draws instead of apheresis to manufacture autologous CD19-CAR T cells, which could simplify the process significantly. This data was presented at the ASGCT 27th Annual Meeting, showing successful CAR T cell production from blood draws from both healthy donors and lupus patients.

The company will also present initial clinical data from the first patients treated in the RESET-Myositis and RESET-SLE trials at the EULAR Congress in June 2024 in Vienna, Austria.

In addition to CABA-201, Cabaletta is exploring other cell therapies like DSG3-CAART for mucosal pemphigus vulgaris and MuSK-CAART for MuSK-associated myasthenia gravis.

Financially, Cabaletta reported research and development expenses of $22 million for Q1 2024, up from $12.4 million in the same period in 2023. General and administrative expenses were $6.1 million, up from $4.5 million the previous year. As of March 31, 2024, the company had $223.8 million in cash and short-term investments, expected to fund operations into the first half of 2026.

CABA-201 aims to deplete CD19-positive B cells with a single infusion, potentially leading to long-term remission without ongoing therapy in autoimmune disease patients. The company is conducting four Phase 1/2 RESET trials with nine cohorts moving concurrently, using a parallel cohort design with a fixed starting dose. A separate sub-study within the DesCAARTes trial is also evaluating CABA-201 without preconditioning for PV patients.

Cabaletta Bio, headquartered in Philadelphia, PA, is committed to developing engineered T cell therapies targeting autoimmune diseases. The company's platform focuses on CARTA and CAART strategies, with the lead product CABA-201 being tested in multiple autoimmune conditions.