Cabaletta Bio(CABA) - 2025 Q2 - Quarterly Results

Executive Summary Cabaletta Bio reported Q2 2025 financial results and provided a business update, highlighting significant progress in advancing rese-cel, including FDA alignment for a registrational pathway in myositis, compelling clinical data, and a successful $100 million public offering extending cash runway Q2 2025 Business and Financial Highlights Cabaletta Bio reported Q2 2025 financial results and provided a business update, highlighting significant progress in advancing rese-cel, including FDA alignment for a registrational pathway in myositis, compelling clinical data, and a successful $100 million public offering extending cash runway - Registrational cohort enrollment in RESET-Myositis™ trial on track to start in 2H25 with anticipated 2027 BLA submission for rese-cel in myositis[1] - Five disease-specific cohorts fully enrolled in the RESET™ clinical development program from over 70 clinical sites as of July 31, 2025, with expansion phase enrollment continuing[1] - 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[1] - 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[1] - Closed $100 million public offering to support late clinical-stage development and commercial readiness activities for rese-cel; cash runway extended into 2H26[1] Recent Operational Highlights and Upcoming Anticipated Milestones This section details rese-cel's mechanism, clinical progress, regulatory milestones including FDA alignment for myositis, manufacturing advancements, and a recent $100 million public offering Rese-cel Program Overview Rese-cel (resecabtagene autoleucel) is an investigational, autologous CAR-T cell therapy designed to treat autoimmune diseases by transiently and deeply depleting CD19-positive cells, aiming to reset the immune system for durable responses without chronic therapy. It is being evaluated in the RESET clinical development program across rheumatology, neurology, and dermatology - Rese-cel is an investigational, autologous CAR-T cell therapy engineered with a fully human CD19 binder and a 4-1BB co-stimulatory domain[3] - It is designed 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[3] - Rese-cel is being evaluated in the RESET clinical development program, which includes multiple ongoing company-sponsored trials across a diverse and growing range of autoimmune diseases in rheumatology, neurology and dermatology[3][4] Clinical Development Cabaletta presented new clinical and translational data at EULAR 2025, reinforcing rese-cel's ability to achieve deep B cell depletion and compelling clinical responses. The company is on track to initiate myositis registrational cohorts in 2H25 and plans several upcoming data presentations in 2025 and 2026 for various RESET trials EULAR 2025 Congress Data New clinical and translational data from RESET trials presented at EULAR 2025 demonstrated deep B cell depletion and compelling clinical responses - New clinical and translational data from 18 evaluable patients in RESET-Myositis, RESET-SLE™, and RESET-SSc™ trials were presented at the EULAR 2025 Congress in June 2025[8] - The data supported rese-cel's ability to generate deep B cell depletion and compelling clinical responses, with nearly all patients off immunomodulators and steroids through the follow-up period[8] Myositis Registrational Cohorts Cabaletta plans to initiate enrollment in two open-label, single-arm registrational myositis cohorts in the second half of 2025 - Cabaletta plans to initiate enrollment in two open-label, single-arm, registrational myositis cohorts, each consisting of approximately 15 patients, in the second half of 2025[8] Upcoming Clinical Data Presentations Cabaletta plans several upcoming clinical data presentations for RESET-Myositis, RESET-PV, RESET-MG, RESET-SLE, and RESET-SSc trials 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 (evaluating rese-cel without preconditioning), and initial clinical data from the RESET-MG™ trial[8] - Additionally, complete Phase 1/2 clinical data from the RESET-SLE and RESET-SSc trials are expected to be presented in the first half of 2026[8] Regulatory Updates Cabaletta achieved FDA alignment on key design elements for two registrational myositis cohorts, planning a first BLA submission for rese-cel in myositis in 2027. Further FDA meetings are scheduled in 3Q25, 4Q25, and 1H26 to align on registrational designs for lupus, systemic sclerosis, and myasthenia gravis, respectively Myositis BLA Submission Plan Cabaletta achieved FDA alignment for myositis registrational cohorts and plans its first BLA submission for rese-cel in myositis in 2027 - 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[8] - The Company plans to submit its first BLA for rese-cel in myositis in 2027 based on either cohort, if successful[8] Additional Registrational Pathway Discussions Cabaletta plans future FDA meetings to align on registrational designs for lupus, systemic sclerosis, and myasthenia gravis - 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[8] Manufacturing Updates Cabaletta is advancing CMC commercial supply readiness and innovation activities, including BLA-enabling activities for lentiviral vector and cellular drug product processes, with the latter transferred to Lonza for registrational enrollment - Advancing CMC commercial supply readiness and innovation activities to support commercial supply, including BLA-enabling activities for both lentiviral vector process and cellular drug product process[7] - The Oxford Biomedica lentiviral process and the commercial drug product process, transferred to Lonza, will be used for initiating registrational enrollment[7] Corporate Updates In June 2025, Cabaletta completed a public offering, generating approximately $94 million in net proceeds to support its operations - In June 2025, Cabaletta closed an underwritten public offering consisting of shares of its common stock, pre-funded warrants and accompanying common stock warrants[9] - The net proceeds from the offering were approximately $94 million, after deducting underwriting discounts, commissions and offering expenses[9] Second Quarter 2025 Financial Results Cabaletta Bio reported increased operating expenses and net loss for Q2 2025, while a public offering significantly boosted its cash position and extended its runway Financial Summary Cabaletta Bio reported increased R&D and G&A expenses for Q2 2025 compared to Q2 2024, reflecting advanced clinical development. The company's cash position significantly improved to $194.7 million as of June 30, 2025, extending its cash runway into the second half of 2026 Key Financial Metrics (Q2 2025 vs. Q2 2024) | Metric | Q2 2025 | Q2 2024 | Change | | :-------------------------- | :---------- | :---------- | :------- | | Research and development expenses | $37.6 million | $23.4 million | +60.7% | | General and administrative expenses | $8.3 million | $6.9 million | +20.3% | | Cash, cash equivalents and short-term investments (as of period end) | $194.7 million (Jun 30, 2025) | $164.0 million (Dec 31, 2024) | +18.7% | - 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[13] Statements of Operations For the three months ended June 30, 2025, Cabaletta Bio reported a net loss of $45.1 million, an increase from $27.6 million in the same period of 2024, primarily driven by higher operating expenses, particularly in research and development. The net loss for the six months ended June 30, 2025, was $81.1 million, compared to $52.6 million for the prior year period Statements of Operations (Three and Six Months Ended June 30, in thousands) | Metric | Three Months Ended June 30, 2025 (unaudited) | Three Months Ended June 30, 2024 (unaudited) | Six Months Ended June 30, 2025 (unaudited) | Six Months Ended June 30, 2024 (unaudited) | | :------------------------------------ | :------------------------------------------- | :------------------------------------------- | :----------------------------------------- | :----------------------------------------- | | Research and development | $37,638 | $23,427 | $66,656 | $45,381 | | General and administrative | $8,268 | $6,852 | $16,386 | $12,929 | | Total operating expenses | $45,906 | $30,279 | $83,042 | $58,310 | | Loss from operations | $(45,906) | $(30,279) | $(83,042) | $(58,310) | | Interest income | $1,410 | $2,677 | $2,897 | $5,661 | | Interest expense | $(571) | — | $(865) | — | | Other expense | $(61) | — | $(61) | — | | Net loss | $(45,128) | $(27,602) | $(81,071) | $(52,649) | | Net loss per share (basic and diluted) | $(0.73) | $(0.56) | $(1.44) | $(1.07) | Selected Balance Sheet Data As of June 30, 2025, Cabaletta Bio reported a significant increase in cash, cash equivalents, and investments to $194.7 million, up from $164.0 million at December 31, 2024. Total assets and stockholders' equity also increased, while total liabilities saw a moderate rise Selected Balance Sheet Data (as of period end, in thousands) | Metric | June 30, 2025 (unaudited) | December 31, 2024 | | :-------------------------------- | :------------------------ | :---------------- | | Cash, cash equivalents and investments | $194,682 | $163,962 | | Total assets | $224,500 | $185,046 | | Total liabilities | $46,019 | $32,711 | | Total stockholders' equity | $178,481 | $152,335 | About Cabaletta Bio Cabaletta Bio is a clinical-stage biotechnology company developing targeted cell therapies for autoimmune diseases, with rese-cel as its lead CARTA strategy Company Overview Cabaletta Bio is a clinical-stage biotechnology company focused on developing curative targeted cell therapies for autoimmune diseases. Its CABA™ platform includes the lead CARTA strategy, prioritizing rese-cel, a CD19-CAR T cell investigational therapy, currently in the RESET™ clinical development program across multiple therapeutic areas - Cabaletta Bio is a clinical-stage biotechnology company focused on developing and launching the first curative targeted cell therapies designed specifically for patients with autoimmune diseases[11] - The CABA™ platform encompasses two complementary strategies, with the lead CARTA strategy prioritizing the development of rese-cel, a 4-1BB-containing fully human CD19-CAR T cell investigational therapy[11] - Rese-cel is currently being evaluated in the RESET™ clinical development program spanning multiple therapeutic areas, including rheumatology, neurology and dermatology[11] Forward-Looking Statements This section outlines forward-looking statements regarding Cabaletta Bio's future plans and potential risks that could impact actual results Disclaimer and Risk Factors This section contains forward-looking statements regarding Cabaletta Bio's future plans, objectives, clinical trial outcomes, regulatory interactions, financial performance, and ability to fund operations. It also outlines various risks and uncertainties that could cause actual results to differ materially from these statements, including regulatory risks, clinical trial risks, market conditions, and intellectual property risks - Forward-looking statements include express or implied statements regarding Cabaletta's business plans, ability to launch curative cell therapies, successful development and commercialization of drug candidates, timing and results of clinical trials, regulatory interactions, and financial outlook[12][14] - These statements are subject to risks and uncertainties such as regulatory filings, the risk that biologic activity may not inform long-term results, ability to demonstrate safety/efficacy, clinical trial delays, unexpected safety/efficacy data, volatile market conditions, and intellectual property risks[15] - All information is as of the date of the release, and Cabaletta undertakes no duty to update this information unless required by law[15] Contacts This section provides contact information for investor relations inquiries Investor Relations Contact This section provides the contact information for investor relations, specifically Anup Marda, Chief Financial Officer - Contact for investors: Anup Marda, Chief Financial Officer[20] - Email: investors@cabalettabio.com[20]