PHILADELPHIA, PA — Cabaletta Bio, Inc. (Nasdaq: CABA) has announced key updates on its RESET-Myositis trial and plans for the anticipated 2027 Biologics License Application (BLA) submission for rese-cel (resecabtagene autoleucel), a potential breakthrough therapy for myositis. Following alignment with the U.S. Food and Drug Administration (FDA), the company outlined a registrational study design that could pave the way for an expedited approval process.
The RESET-Myositis trial will now include two subtype-specific single-arm cohorts, each enrolling approximately 15 patients. The first cohort will target dermatomyositis (DM) and antisynthetase syndrome (ASyS), while the second will focus on immune-mediated necrotizing myopathy (IMNM). The FDA also confirmed that pooled safety data from the broader RESET clinical program can supplement myositis-specific data for the BLA submission. The trial’s primary endpoint will measure patient outcomes using a validated Total Improvement Score (TIS) within 26 weeks of rese-cel infusion.
“Myositis is a severe, disabling, and potentially life-threatening autoimmune disease, affecting approximately 80,000 people in the U.S.,” said David J. Chang, M.D., Chief Medical Officer of Cabaletta Bio. “The clinical safety and efficacy data evaluating a single, weight-based dose of rese-cel supported our recent discussions with the FDA. With RMAT designation granted for rese-cel, we are well-positioned to advance on a path that could ultimately deliver a significant new treatment option for patients.”
RMAT designation, granted by the FDA, offers rese-cel significant advantages, including expedited review timelines and enhanced interactions with the FDA aimed at advancing drug development.
The company also shared progress across its broader RESET clinical program, which includes trials for systemic lupus erythematosus (SLE), lupus nephritis (LN), and systemic sclerosis (SSc). Phase 1/2 cohorts are fully enrolled, with 44 patients enrolled and 23 dosed as of May 9, 2025. New clinical data from each study will be presented in three oral sessions at the EULAR 2025 Congress in Barcelona this June.
Cabaletta continues to prioritize manufacturing investments to scale production and improve process efficiencies. Plans include leveraging the capabilities of Oxford Biomedica for lentiviral vector processes and Lonza for commercial production, expected to be operational by Q3 2025.
Financially, the company reported cash and cash equivalents of $131.8 million as of March 31, 2025, down from $164.0 million at the end of 2024. This funding is expected to support Cabaletta’s operations into the first half of 2026.
With its disciplined regulatory and clinical strategy, Cabaletta Bio aims to address unmet needs in autoimmune disease treatment, positioning rese-cel as a potential game-changer for patients suffering from debilitating conditions like myositis.
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