PHILADELPHIA, PA — Cabaletta Bio, Inc. (Nasdaq: CABA) has revealed significant progress in its pipeline and operations, underscoring its focus on advancing rese-cel, a cell therapy candidate for autoimmune diseases. The company also outlined key milestones and priorities for 2025, including plans to engage with the U.S. Food and Drug Administration (FDA) on registrational trial designs.
“Our clinical execution in 2024 allowed us to accelerate timelines for registrational discussions and demonstrate the potential of rese-cel to deliver immunosuppressant-free, compelling clinical responses in patients with active, refractory autoimmune disease,” said Steven Nichtberger, M.D., Chief Executive Officer of Cabaletta.
Promising Clinical Progress
Cabaletta announced that 21 patients have already been enrolled across its growing network of 44 clinical trial sites in the U.S. and Europe for the RESET program. Rese-cel has shown a favorable safety profile in the first 10 patients dosed, with 90% experiencing either no cytokine release syndrome (CRS) or only mild grade 1 CRS (fever) and 90% avoiding immune effector cell-associated neurotoxicity syndrome (ICANS). Updated clinical and translational data from these patients will be presented in February 2025.
Recent developments in the pipeline include the first patient enrollment in the RESET-PV trial, which evaluates rese-cel without preconditioning for pemphigus vulgaris, and the initiation of the juvenile myositis cohort in the RESET-Myositis trial. Additionally, rese-cel received FDA clearance to proceed with the RESET-MS trial for multiple sclerosis and was granted Fast Track Designation for treating relapsing and progressive forms of MS.
Strategic Partnerships and Manufacturing
To support its expanding clinical footprint and prepare for potential registrational trials, Cabaletta extended its agreement with Lonza, a leading manufacturing partner. Clinical-grade production of rese-cel is expected in the second half of 2025 under current Good Manufacturing Practices.
2025 Goals
Cabaletta’s top priorities for the year include finalizing discussions with the FDA on registrational trials for rese-cel across disease-specific cohorts and completing patient enrollment and dosing in various RESET trials. New clinical and translational findings on rese-cel will be presented throughout the year.
The company is also exploring innovations to simplify patient access, including testing rese-cel without preconditioning and working toward regulatory alignment on using whole blood collection as an alternative to apheresis.
Financial Outlook
Cabaletta closed 2024 with an unaudited cash balance of $164 million, which it expects will fund operations into the first half of 2026.
“Leveraging a large and growing clinical site footprint in the U.S., recent expansion into Europe, and an increased pace of patient enrollment, we look forward to building on our momentum as we move closer to realizing our vision of launching the first targeted curative cell therapy designed specifically for patients with autoimmune disease,” Nichtberger added.
With a robust pipeline and strategic clarity, Cabaletta Bio is positioned to advance its mission of transforming care for patients with autoimmune diseases.
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