Promising Results Emerge in Clinical Trials of CABA-201 for Autoimmune Diseases
CABA-201, an investigational chimeric antigen receptor T-cell (CAR-T) therapy made by Cabaletta Bio, is showing promise in treating a variety of autoimmune diseases. Data presented recently at the 2024 American College of Rheumatology (ACR) Convergence conference highlights positive results in patients participating in phase 1/2 clinical trials for lupus, myositis, and systemic sclerosis.
## Positive Steps Forward in Treating Lupus
In patients who received CABA-201 as part of the RESET-SLE trial targeting systemic lupus erythematosus and lupus nephritis, a patient in the severe skin cohort exhibited a significant improvement.
Forty-two days after treatment, this patient demonstrated a substantial decline in skin disease severity, dropping from a baseline score of 42 on the modified Rodnan Skin Score to 36. This represents continued improvement, as prior to starting CABA-201, the patient ceased receiving any disease-specific therapies while still actively undergoing treatment. This suggests the patient might be experiencing a durable response without needing ongoing medication specific to their lupus.
## Safety Profile
Early reporting indicates a favorable safety profile for CABA-201. five out of eight patients across the three initial trials did not experience any occurrences of cytokine release syndrome (CRS).
The remaining three evaluable patients experienced mild CRS (grade 1-2) and recovered after receiving standard treatment.
The first patient with lupus nephritis treated in the RESET-SLE trial experienced a case of immune effector cell-associated neurotoxicity syndrome (ICANS). Initial reports of this case份 were released in August 2024.
Cabaletta highlighted that the ICANS case appeared connected to a possible underlying infection.
The patient had history of acute inflammatory events before receiving the CABA-201 therapy, and an unusual and inflammatory cytokine profile was observed both before and after starting treatment.
## Expanding Clinical Trials and Future Prospects
Moving forward, research on CABA-201 is expanding. The FDA granted clearance for a phase 1/2 trial (RESET-MG; NCT06359041) for generalized myasthenia gravis. Additionally, CABA-201 will be used as a monotherapy in a substudy (RESET-PV, NCT04422912) within another ongoing trial for pemphigus vulgaris.
This substudy builds upon the success of the phase 1 DesCAARTes study. Sixteen patients have been enrolled across the RESET clinical trials and ten have received their assigned treatment.
More promising news came when the European Medicines Agency cleared the trial application assessing CABA-201 in lupus. This should soon clear the way for expansion of clinical development for the 💪treatment
in Europe.
Clinically, this is encouraging:
It was stated “‘Focused clinical execution has resulted in 40 US clinical sites actively recruiting patients for the RESET clinical trial program for CABA-201…” Steven Nichtberger, MD, CEO of Cabaletta Bio, said in a November 2024 statement.
“We are encouraged by the accelerating pace of enrollment, and, data permitting, anticipate meeting with the FDA next year regarding registrational program designs for CABA-201
This is a sizable jump, as the company previously reported 10 sites actively recruiting participants in May. After completed phases 1 and 2,
