Cartesian Therapeutics(RNAC) - 2025 Q1 - Quarterly Results

Executive Summary & Business Update This section provides an overview of Cartesian Therapeutics' recent pipeline advancements, anticipated milestones, and detailed descriptions of its lead product candidates, Descartes-08 and Descartes-15 - Initiation of Phase 3 AURORA trial of Descartes-08 in myasthenia gravis expected in 2Q25[1][3] - Preliminary data from Phase 2 trial of Descartes-08 in systemic lupus erythematosus expected in 2H25[1][9] - Initiation of Phase 2 pediatric basket trial of Descartes-08 in select autoimmune indications expected in 2H25[1][9] Recent Pipeline Progress and Anticipated Milestones Cartesian Therapeutics reported significant pipeline advancements, including positive long-term Phase 2b results for Descartes-08 in myasthenia gravis (MG) and the planned initiation of the Phase 3 AURORA trial in Q2 2025 Descartes-08 in Myasthenia Gravis (MG) Updated long-term results from the Phase 2b trial of Descartes-08 in MG showed deep and sustained benefits through 12 months after a single course of therapy, particularly in participants without prior biologic exposure - Positive updated long-term results from Phase 2b trial of Descartes-08 in MG, with sustained benefits observed through 12 months after a single course of therapy[3] - Average 4.8-point reduction in MG Activities of Daily Living Scale (MG-ADL) at Month 12 for Descartes-08-treated participants[3] - Initiation of Phase 3 AURORA Trial of Descartes-08 in MG on track for 2Q25, designed to assess Descartes-08 versus placebo in approximately 100 participants[3] Descartes-08 in Systemic Lupus Erythematosus (SLE) Preliminary data from the ongoing Phase 2 open-label trial of Descartes-08 in patients with SLE is expected in the second half of 2025, assessing safety, tolerability, and clinical activity of outpatient Descartes-08 administration without preconditioning chemotherapy - Preliminary data from ongoing Phase 2 open-label trial of Descartes-08 in patients with SLE expected in the second half of 2025[9] - The trial assesses safety, tolerability, and clinical activity of outpatient Descartes-08 administration without preconditioning chemotherapy[9] Descartes-08 Pediatric Basket Trial A Phase 2 pediatric basket trial of Descartes-08 in select autoimmune diseases, including juvenile SLE, juvenile MG, juvenile dermatomyositis (JDM), and anti-neutrophil cytoplasmic antibody associated vasculitis, is expected to initiate in the second half of 2025 - Phase 2 Pediatric Basket Trial of Descartes-08 in select autoimmune diseases expected to initiate in the second half of 2025[9] - This trial will target juvenile SLE, juvenile MG, juvenile dermatomyositis (JDM) and anti-neutrophil cytoplasmic antibody associated vasculitis[9] - The FDA previously granted Rare Pediatric Disease Designation to Descartes-08 for the treatment of JDM[9] Descartes-15 Development Dosing continues in the first-in-human Phase 1 clinical trial of Descartes-15 for multiple myeloma, with plans to subsequently assess this next-generation CAR-T cell therapy in autoimmune indications - Dosing continues in First-in-Human Phase 1 Clinical Trial of Descartes-15 for multiple myeloma[9] - The Company expects to subsequently assess Descartes-15 in autoimmune indications after the Phase 1 dose escalation trial[9] Product Candidate Overviews Cartesian Therapeutics is developing two key autologous CAR-T cell therapy candidates, Descartes-08 and Descartes-15, both designed for outpatient administration without preconditioning chemotherapy and without the risk of genomic integration About Descartes-08 Descartes-08 is Cartesian's lead autologous anti-BCMA CAR-T cell therapy candidate, in clinical development for generalized myasthenia gravis (MG) and systemic lupus erythematosus (SLE), and has received multiple FDA designations - Descartes-08 is an autologous chimeric antigen receptor T-cell therapy (CAR-T) product targeting B-cell maturation antigen (BCMA)[6] - Designed to not require preconditioning chemotherapy, can be administered in the outpatient setting, and does not carry the risk of genomic integration[6] - Granted Orphan Drug Designation, Regenerative Medicine Advanced Therapy Designation for MG, and Rare Pediatric Disease Designation for juvenile dermatomyositis[6] About Descartes-15 Descartes-15 is a next-generation, autologous anti-BCMA CAR-T cell therapy that has shown an approximately ten-fold increase in CAR expression and selective target-specific killing in preclinical studies compared to Descartes-08, sharing its design benefits - Descartes-15 is a next-generation, autologous anti-BCMA CAR-T cell therapy[7] - In preclinical studies, Descartes-15 achieved an approximately ten-fold increase in CAR expression and selective target-specific killing, relative to Descartes-08[7] - Designed to be administered without preconditioning chemotherapy and does not use integrating vectors[7] First Quarter 2025 Financial Results This section details Cartesian Therapeutics' financial performance for the first quarter of 2025, including key financial highlights, balance sheet figures, and statements of operations Financial Highlights Cartesian Therapeutics reported a cash position of $182.1 million as of March 31, 2025, expected to fund operations into mid-2027, with increased R&D expenses and a substantially improved net loss per share - Cash, cash equivalents and restricted cash as of March 31, 2025 was $182.1 million, expected to support planned operations into mid-2027[1][9] Key Financial Highlights (Q1 2025 vs Q1 2024) | Metric | Q1 2025 (in millions) | Q1 2024 (in millions) | Change (YoY) (in millions) | | :-------------------------------- | :-------------------- | :-------------------- | :----------- | | Research and development expenses | $14.7 | $9.7 | +$5.0 | | General and administrative expenses | $8.3 | $9.5 | -$1.2 | | Net loss | $(17.7) | $(56.8) | +$39.1 | | Net loss per share (basic) | $(0.68) | $(10.50) | +$9.82 | Consolidated Balance Sheets As of March 31, 2025, Cartesian Therapeutics reported total assets of $409.1 million, a decrease from $435.0 million at December 31, 2024, primarily due to reduced cash and cash equivalents, alongside decreased total liabilities and an increased stockholders' deficit Consolidated Balance Sheet Highlights (Amounts in thousands) | Metric | March 31, 2025 (Unaudited) | December 31, 2024 | | :------------------------------------ | :------------------------- | :---------------- | | Cash and cash equivalents | $180,434 | $212,610 | | Total current assets | $185,120 | $216,626 | | Total assets | $409,130 | $435,023 | | Total current liabilities | $14,998 | $22,976 | | Total liabilities | $430,919 | $441,825 | | Total stockholders' deficit | $(21,789) | $(6,802) | Consolidated Statements of Operations and Comprehensive Loss For the three months ended March 31, 2025, total revenue decreased significantly to $1.1 million from $5.8 million in the prior year, primarily due to lower collaboration and license revenue, while operating expenses increased, leading to a reduced net loss of $(17.7) million compared to $(56.8) million in Q1 2024 due to a positive change in contingent value right liability Consolidated Statements of Operations Highlights (Amounts in thousands) | Metric | Three Months Ended March 31, 2025 (Unaudited) | Three Months Ended March 31, 2024 | | :------------------------------------------ | :-------------------------------------------- | :-------------------------------- | | Collaboration and license revenue | $400 | $5,840 | | Grant revenue | $700 | $— | | Total revenue | $1,100 | $5,840 | | Research and development | $14,674 | $9,738 | | General and administrative | $8,315 | $9,450 | | Total operating expenses | $22,989 | $19,188 | | Operating loss | $(21,889) | $(13,348) | | Change in fair value of contingent value right liability | $346 | $(39,300) | | Net loss | $(17,710) | $(56,824) | | Basic and diluted net loss per share | $(0.68) | $(10.50) | About Cartesian Therapeutics Cartesian Therapeutics is a clinical-stage biotechnology company focused on pioneering cell therapy for autoimmune diseases, with its lead asset, Descartes-08, entering Phase 3 for generalized myasthenia gravis and Phase 2 for systemic lupus erythematosus, and Descartes-15 in Phase 1 development for multiple myeloma - Cartesian Therapeutics is a clinical-stage company pioneering cell therapy for the treatment of autoimmune diseases[1][10] - The Company's lead asset, Descartes-08, is a CAR-T entering Phase 3 clinical development for generalized myasthenia gravis and Phase 2 development for systemic lupus erythematosus[10] - The clinical-stage pipeline also includes Descartes-15, a next-generation, autologous anti-BCMA CAR-T currently being evaluated in a Phase 1 trial in patients with multiple myeloma[10] Forward Looking Statements This section contains standard forward-looking statements regarding the company's future expectations, plans, and prospects, including clinical trial outcomes, regulatory approvals, and financial resources, emphasizing that actual results may differ materially due to various important factors and risks - Statements about future expectations, plans and prospects, including cash resources, product candidate administration, potential to treat diseases, and timing/outcome of clinical trials, constitute forward-looking statements[11] - Actual results may differ materially due to various important factors, including uncertainties in clinical trials, data availability, regulatory approvals, funding, and other risks discussed in SEC filings[11] - The Company specifically disclaims any intention to update any forward-looking statements, except as required by law[11] Contact Information This section provides contact details for investor and media inquiries - Investor Contact: Megan LeDuc, Associate Director of Investor Relations (megan.leduc@cartesiantx.com)[16] - Media Contact: David Rosen, Argot Partners (david.rosen@argotpartners.com)[16]