Cabaletta Bio Presents Updated Interim DesCAARTes™ Trial Phase 1 Data at the ASGCT 25th Annual Meeting
PHILADELPHIA, May 18, 2022 (GLOBE NEWSWIRE) -- Cabaletta Bio, Inc. (Nasdaq: CABA), a clinical-stage biotechnology company focused on the discovery and development of targeted cell therapies for patients with autoimmune diseases, today presented updated clinical and translational data through 6 months of follow-up in cohorts A1 through A3, safety data through 3 months and persistence data through 1 month of follow-up in cohorts A1 through A4 from the DesCAARTes™ trial at the American Society of Gene & Cell Therapy (ASGCT) 25th Annual Meeting being held in Washington, D.C. from May 16-19, 2022.
“At ASGCT, we presented updated interim data showing that DSG3-CAART has had a favorable safety profile with no dose limiting toxicities or cytokine release syndrome of any grade through cohort A4, which was a dose of 2.5 billion DSG3-CAART cells. In addition, we have observed a dose dependent increase in DSG3-CAART persistence, which at cohort A4 approached the lower end of the range that is observed in anti-CD19 CART oncology studies in combination with lymphodepletion,” said David Chang, M.D., Chief Medical Officer of Cabaletta. “These data continue to support the planned dose escalation in this trial and our conviction in the potential of this program. As we progress through additional cohorts of the study, we look forward to evaluating the relationship between DSG3-CAART persistence and potential clinical responses in patients with mPV along with the opportunity to explore higher doses and an enhanced manufacturing process to meet our goal of reaching deep, durable and potentially curative responses for these patients.”
Cabaletta’s DesCAARTes™ Phase 1 trial is an open-label, dose escalation, multi-center study of DSG3-CAART in adults with mucosal-dominant pemphigus vulgaris (mPV). The trial is designed to determine the maximum tolerated dose of DSG3-CAART in adult subjects with active, anti-DSG3 Ab positive, biopsy confirmed mPV that is inadequately managed by one or more standard therapies. The primary endpoint is incidence of adverse events (AEs), including dose-limiting toxicities (DLTs), such as cytokine release syndrome (CRS) and neurotoxicity, related to DSG3-CAART within three months of infusion. Secondary endpoints include CAART persistence (qPCR), anti-DSG3 Ab levels (ELISA) and disease activity (PDAI). The trial is currently in cohort A5 (5.0 to 7.5 billion cells) and is being conducted across multiple clinical sites throughout the United States. The Company plans to include two new additional dose cohorts – A5e (enhanced manufacturing process at 5.0 to 7.5 billion cells) and A6m (multi-dose regimen at 10 to 15 billion cells). The prioritization of cohorts following cohort A5 (e.g. A5e or A6m) is subject to evaluation of emerging data and discussions with the FDA, as applicable.
The updated interim DesCAARTes™ trial Phase 1 data included 12 treated subjects, four cohorts with three patients per cohort; nine having completed six months follow up after DSG3-CAART infusion. The posters as presented at ASGCT are available at https://www.cabalettabio.com/technology/posters-publications. The data show:
Data presented on the company’s manufacturing process were as follows:
About Cabaletta Bio
Any forward-looking statements in this press release are based on management’s current expectations and beliefs of future events, and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to: the risk that signs of biologic activity or persistence may not inform long-term results; Cabaletta’s ability to demonstrate sufficient evidence of safety, efficacy and tolerability in its preclinical and clinical trials of DSG3-CAART; the risk that persistence observed with effective CART-19 oncology studies in combination with lymphodepletion is not indicative of, or applicable to, clinical responses in patients with mPV; risks related to clinical trial site activation or enrollment rates that are lower than expected; risks related to unexpected safety or efficacy data observed during clinical studies; risks related to the impact of public health epidemics, such as the ongoing COVID-19 pandemic, affecting countries or regions in which we have operations or do business; Cabaletta’s ability to retain and recognize the intended incentives conferred by Orphan Drug Designation and Fast Track Designation for DSG3-CAART for improving healing of mucosal blisters in patients with mucosal pemphigus vulgaris; Cabaletta’s ability to demonstrate sufficient evidence of safety, efficacy and tolerability in its preclinical and clinical trials of DSG3-CAART; risks related to Cabaletta’s ability to protect and maintain its intellectual property position; uncertainties related to the initiation and conduct of studies and other development requirements for its product candidates; the risk that any one or more of Cabaletta’s product candidates will not be successfully developed and commercialized; and the risk that the initial or interim results of preclinical studies or clinical studies will not be predictive of future results in connection with future studies. For a discussion of these and other risks and uncertainties, and other important factors, any of which could cause Cabaletta’s actual results to differ from those contained in the forward-looking statements, see the section entitled “Risk Factors” in Cabaletta’s most recent annual report on Form 10-K as well as discussions of potential risks, uncertainties, and other important factors in Cabaletta’s other filings with the Securities and Exchange Commission. All information in this press release is as of the date of the release, and Cabaletta undertakes no duty to update this information unless required by law.