PHILADELPHIA, March 31, 2025 – Cabaletta Bio, Inc. (Nasdaq: CABA), a clinical-stage biotechnology company developing curative cell therapies for autoimmune diseases, disclosed a dose-limiting toxicity (DLT) event in its RESET-SSc trial evaluating Rese-cel, a CAR-T cell therapy, and provided a business update. Rese-cel (resecabtagene autoleucel) is an autologous CAR-T cell therapy, utilizing a patient’s own T cells that are genetically engineered to express a chimeric antigen receptor (CAR). This CAR is specifically designed to target CD19, a protein found on the surface of B cells, which play a significant role in the pathogenesis of various autoimmune diseases. By targeting and eliminating these CD19-expressing B cells, Rese-cel aims to disrupt the autoimmune process and potentially provide durable remissions.
Dose-Limiting Toxicity Reported:
- In March 2025, a patient in the RESET-SSc trial experienced a grade 3 immune effector cell-associated neurotoxicity syndrome (ICANS) event nine days following Rese-cel infusion. This occurred after a protocol deviation where a patient with a reported fever three days prior to infusion was not properly evaluated by the medical monitor.
- The ICANS event manifested as a transient period of confusion, with no cerebral edema, seizures, or motor dysfunction. The patient was arousable throughout and the ICANS resolved rapidly following treatment with dexamethasone. The patient was discharged without further symptoms.
- Following a data review, the Independent Data Monitoring Committee (IDMC) recommended that the trial proceed at the current dose.
- Cabaletta has implemented additional safety measures, requiring investigators to confirm in writing the absence of fevers or infections in patients during the two weeks prior to Rese-cel infusion.
Trial and Development Update:
- Despite the DLT event, the IDMC endorsed the continuation of the RESET-SSc trial.
- Cabaletta is continuing to advance Rese-cel across its rheumatology and neurology portfolios, including trials in systemic sclerosis, myositis, systemic lupus erythematosus, lupus nephritis, myasthenia gravis, and multiple sclerosis.
- Cabaletta is preparing to meet with the FDA to finalize registrational trial designs for myositis.
https://www.cabalettabio.com/news-media/press-releases/detail/127/cabaletta-bio-reports-fourth-quarter-and-full-year-2024
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