Cabaletta Bio Inc.
08/07/2025 | Press release | Distributed by Public on 08/07/2025 05:37
FDA meetings to align on the next wave of registrational cohorts for rese-cel anticipated in 3Q25 for lupus, 4Q25 for systemic sclerosis and 1H26 for myasthenia gravis (Form 8-K)
- FDA meetings to align on the next wave of registrational cohorts for rese-cel anticipated in 3Q25 for lupus, 4Q25 for systemic sclerosis and 1H26 for myasthenia gravis -
- Rese-cel clinical data presented at the EULAR 2025 Congress reinforce compelling clinical responses with nearly all patients off immunomodulatory medications and steroids; favorable risk-benefit profile observed across myositis, lupus and systemic sclerosis patients -
- Closed $100 million public offering to support late clinical-stage development and commercial readiness activities for rese-cel; cash runway extended into 2H26 -
PHILADELPHIA, Aug. 7, 2025 -- Cabaletta Bio, Inc. (Nasdaq: CABA), a clinical-stage biotechnology company focused on developing and launching the first curative targeted cell therapies designed specifically for patients with autoimmune diseases, today reported financial results for the second quarter ended June 30, 2025, and provided a business update.
"We achieved significant progress advancing rese-cel in the first half of 2025, including aligning with the FDA on a registrational pathway for rese-cel in myositis to support an anticipated 2027 first BLA submission. We also presented new data that reinforce the therapeutic potential of rese-cel across several autoimmune diseases," said Steven Nichtberger, M.D., Chief Executive Officer of Cabaletta. "As our early clinical data are emerging, enrollment in dermatomyositis, scleroderma and myasthenia gravis has been particularly robust. We look forward to building on this momentum by initiating the myositis registrational program, obtaining regulatory alignment on registrational pathways for two additional indications this year and presenting new clinical data at multiple scientific meetings in the second half of 2025, including complete Phase 1/2 data from RESET-Myositis as well as initial clinical data from RESET-PVTM evaluating rese-cel without pre-conditioning."
Recent Operational Highlights and Upcoming Anticipated Milestones
Rese-cel: Rese-cel (resecabtagene autoleucel, formerly CABA-201) is an investigational, autologous CAR-T cell therapy engineered with a fully human CD19 binder and a 4-1BB co-stimulatory domain, designed specifically for the treatment of autoimmune diseases. Administered as a single, weight-based infusion, rese-cel is intended to transiently and deeply deplete CD19-positive cells, with the goal of resetting the immune system and achieving durable clinical responses without the need for chronic therapy. Cabaletta is evaluating rese-cel in the RESET (REstoring SElf-Tolerance) clinical development program, which includes multiple
ongoing company-sponsored trials across a diverse and growing range of autoimmune diseases in rheumatology, neurology and dermatology.
Clinical Development
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New clinical and translational data presented at the EULAR 2025 Congress: In June 2025, Cabaletta presented new clinical and translational data from 18 evaluable patients in the ongoing RESET-Myositis, RESET-SLE™ and RESET-SSc™ trials in three oral presentations at the EULAR 2025 Congress. The data supported the ability of rese-cel to generate deep B cell depletion and compelling clinical responses in these patients, with nearly all patients off immunomodulators and steroids through the follow-up period.
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On track to initiate enrollment in myositis registrational cohorts: Cabaletta plans to initiate enrollment in two open-label, single-arm, registrational myositis cohorts consisting of approximately 15 patients each in the second half of 2025.
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Upcoming anticipated clinical data presentations in 2025 and 2026: In the second half of 2025, Cabaletta plans to present complete Phase 1/2 clinical data from the RESET-Myositis trial, initial dose data from the RESET-PV trial, which is evaluating rese-cel without preconditioning in patients with pemphigus vulgaris (PV), and initial clinical data from the RESET-MG™ trial. Additionally, Cabaletta expects to present complete Phase 1/2 clinical data from the RESET-SLE and RESET-SSc trials in the first half of 2026.
Regulatory
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First BLA submission planned in 2027 for myositis following FDA alignment on key registrational design elements: In May 2025, Cabaletta announced alignment with the U.S. Food and Drug Administration (FDA) on key design elements for two registrational cohorts in the RESET-Myositis trial. The cohort comprised of patients with dermatomyositis or antisynthetase syndrome represents about 85% of the myositis population, while the IMNM cohort represents about 15%. The two cohorts are independent of each other with regards to regulatory considerations, although as agreed with FDA, safety data from all rese-cel patients will be used to support the Biologics License Application (BLA). The Company plans to submit its first BLA for rese-cel in myositis in 2027 based on either cohort, if successful.
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Additional regulatory discussions planned with FDA to align on additional registrational cohort designs: Cabaletta plans to meet with the FDA to align on key registrational design elements for the RESET-SLE trial in 3Q25, the RESET-SSc trial in 4Q25 and the RESET-MG trial in 1H26.
Manufacturing
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Advancing CMC commercial supply readiness and innovation activities: To support commercial supply readiness, Cabaletta has been advancing BLA-enabling activities both lentiviral vector process and cellular drug product process. The Oxford Biomedica lentiviral process and the commercial drug product process, which has been transferred to Lonza, will be used for initiating registrational enrollment.
Corporate Updates
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In June 2025, Cabaletta closed an underwritten public offering consisting of shares of its common stock, pre-funded warrants and accompanying common stock warrants. The net proceeds from the offering were approximately $94 million, after deducting underwriting discounts, commissions and offering expenses payable by the Company.
Second Quarter 2025 Financial Results
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Research and development expenses were $37.6 million for the three months ended June 30, 2025, compared to $23.4 million for the same period in 2024.
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General and administrative expenses were $8.3 million for the three months ended June 30, 2025, compared to $6.9 million for the same period in 2024.
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As of June 30, 2025, Cabaletta had cash, cash equivalents and short-term investments of $194.7 million, compared to $164.0 million as of December 31, 2024. The Company expects that its cash position as of June 30, 2025, will enable it to fund its operating plan into the second half of 2026.
About Cabaletta Bio
Cabaletta Bio (Nasdaq: CABA) is a clinical-stage biotechnology company focused on developing and launching the first curative targeted cell therapies designed specifically for patients with autoimmune diseases. The CABA™ platform encompasses two complementary strategies which aim to advance the discovery and development of engineered T cell therapies with the potential to become deep and durable, perhaps curative, treatments for a broad range of autoimmune diseases. The lead CARTA (Chimeric Antigen Receptor T cells for Autoimmunity) strategy is prioritizing the development of rese-cel, a 4-1BB-containing fully human CD19-CAR T cell investigational therapy. Rese-cel is currently being evaluated in the RESET™ (REstoring SElf-Tolerance) clinical development program spanning multiple therapeutic areas, including rheumatology, neurology and dermatology. Cabaletta Bio's headquarters and labs are located in Philadelphia, PA. For more information, please visit www.cabalettabio.com and connect with us on LinkedIn.
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