PHILADELPHIA, Nov. 10, 2020 (GLOBE NEWSWIRE) -- Cabaletta Bio, Inc. (Nasdaq: CABA), a clinical-stage biotechnology company focused on the discovery and development of engineered T cell therapies for patients with B cell-mediated autoimmune diseases, today announced financial results for the third quarter ended September 30, 2020 and provided a business update.

“With patient enrollment now underway for our lead program, DSG3-CAART, for patients with mucosal pemphigus vulgaris, we are on track to report acute safety data from the first cohort of patients in the first half of next year. During the quarter, we opened a second site for the trial and also published comprehensive preclinical proof of concept data in The Journal of Clinical Investigation further validating the mechanism of action of DSG3-CAART,” said Steven Nichtberger, M.D., Chief Executive Officer and co-founder of Cabaletta.

Pipeline Highlights and Anticipated Upcoming Milestones

DSG3-CAART: Desmoglein 3 chimeric autoantibody receptor (CAAR) T cells as potential treatment for patients with mucosal pemphigus vulgaris (mPV).

• The Company’s open-label Phase 1 clinical trial (DesCAARTes™) to evaluate the safety and tolerability of DSG3-CAART in relapsed/refractory mPV patients is actively recruiting patients at the first two sites in the U.S. The Company expects to report acute safety data from the initial cohort in the study in the first half of 2021.
• In August 2020, the Company announced that comprehensive preclinical data for DSG3-CAART were published in The Journal of Clinical Investigation. These data demonstrated that DSG3-CAART achieved autoantibody elimination and resolution of blisters in an active immune mouse model of pemphigus vulgaris and that circulating soluble autoantibodies have the potential to enhance DSG3-CAART efficacy and did not demonstrate off-target toxicity.

MuSK-CAART: Muscle Specific Kinase (MuSK) CAAR T cells as potential treatment for MuSK-associated myasthenia gravis.

• Investigational New Drug (IND)-enabling studies are ongoing with an IND submission to the U.S. Food and Drug Administration (FDA) anticipated in the second half of 2021.

Manufacturing

• Cell processing capacity is contractually secured at the University of Pennsylvania for the Phase 1 study of DSG3-CAART.
• Two to three years of vector supply for DSG3-CAART is secured, and pilot partnerships have been established for the production of additional vector at commercial grade and scale.
• Cabaletta expects to initiate validation of cell processing for MuSK-CAART clinical trials with a commercial grade contract manufacturing partner before the end of 2020.

Upcoming Events

• Cabaletta will participate in a fireside chat at the Piper Sandler Virtual Conference on December 1-3.
• Cabaletta will participate in a fireside chat at the Evercore ISI 3^rd Annual HealthconX Conference on December 1-3.

Third Quarter 2020 Financial Results

The Company expects that its cash and cash equivalents and investments as of September 30, 2020 will enable it to fund its operating plan through at least the third quarter of 2022.

• Research and development (R&D) expenses for the three months ended September 30, 2020 were $5.7 million, compared to $3.2 million for the same period in 2019.
• General and administrative (G&A) expenses for the three months ended September 30, 2020 were $2.8 million, compared to $1.8 million for the same period in 2019.
• As of September 30, 2020, cash and cash equivalents and investments totaled $118.1 million, compared to $136.2 million as of December 31, 2019.

About Cabaletta Bio
Cabaletta Bio is a clinical-stage biotechnology company focused on the discovery and development of engineered T cell therapies, and exploring their potential to provide a deep and durable, perhaps curative, treatment, for patients with B cell-mediated autoimmune diseases. The Cabaletta Approach to selective B cell Ablation (CABA) platform, in combination with Cabaletta’s proprietary technology, utilizes Chimeric AutoAntibody Receptor (CAAR) T cells that are designed to selectively bind and eliminate only specific autoantibody-producing B cells while sparing normal antibody-producing B cells, which are essential for human health. The Company’s lead product candidate, DSG3-CAART, is being evaluated in the DesCAARTes™ phase 1 clinical trial as a potential treatment for patients with mucosal pemphigus vulgaris, a prototypical B cell-mediated autoimmune disease. The FDA granted Fast Track Designation for DSG3-CAART in May 2020. For more information about the clinical trial, please see www.clinicaltrials.gov. The Company’s lead preclinical product candidate, MuSK-CAART, is in IND-enabling studies and is designed as a potential treatment for patients with MuSK-associated myasthenia gravis. For more information, visit www.cabalettabio.com.