Descartes-08

Initiated Phase 3 AURORA trial of Descartes-08 in myasthenia gravis Preliminary data from Phase 2 trial of Descartes-08 in systemic lupus erythematosus expected in 2H25 Initiation of Phase 2 pediatric basket trial of Descartes-08 in select autoimmune indications expected in 2H25 Approximately $162.1 million cash, cash equivalents and restricted cash as of June 30, 2025, expected to support planned operations into mid-2027, including completion of ongoing Phase 3 AURORA trial FREDERICK, Md., Aug. 07, 2025 (GLOBE NEWSWIRE) -- Cartesian Therapeutics, Inc. (NASDAQ: RNAC) (the “Company” or “Cartesian”), a clinical-stage biotechnology company pioneering cell therapy for autoimmune diseases, today reported financial results for the second quarter ended June 30, 2025, and outlined recent corporate updates. “Following the recent initiation of our pivotal Phase 3 AURORA trial of Descartes-08 in myasthenia gravis (MG), we have entered the second half of the year with significant momentum as we continue to advance our mission to deliver transformative cell therapies to patients with autoimmune diseases,” said Carsten Brunn, Ph.D., President and Chief Executive Officer of Cartesian. “Supported by deep and sustained responses observed through month 12 in the Phase 2b trial along with a clearly defined regulatory pathway, we believe that, if approved, Descartes-08 has the potential to serve as an impactful new MG therapy with the ability to be safely dosed in the outpatient setting and without the need for preconditioning chemotherapy. Additionally, we continue to make progress advancing the balance of our programs and remain on track to share preliminary data from our ongoing Phase 2 trial of Descartes-08 in patients with systemic lupus erythematosus (SLE) and to initiate a pediatric basket trial in select autoimmune indications by the end of this year.” Recent Pipeline Progress and Anticipated Milestones Initiated Phase 3 AURORA Trial of Descartes-08 in Participants with MG. In May 2025, the Company announced that the first participant had been enrolled in the Phase 3 AURORA trial. The randomized, double-blind, placebo-controlled Phase 3 AURORA trial is designed to assess Descartes-08, Cartesian’s autologous anti-B cell maturation antigen (BCMA) chimeric antigen receptor T-cell therapy (CAR-T) versus placebo (1:1 randomization) administered as six once-weekly outpatient infusions without preconditioning chemotherapy in approximately 100 participants with acetylcholine receptor autoantibody positive (AChR Ab+) MG. The primary endpoint will assess the proportion of Descartes-08 participants with an improvement in MG Activities of Daily Living (MG-ADL) score of three points or more at Month 4 compared to placebo.In April 2025, the Company announced updated efficacy and safety data from the Phase 2b trial of Descartes-08 in participants with MG. After a single course of therapy, Descartes-08-treated participants were observed to sustain deep responses through long-term follow-up, with an average 4.8-point reduction in the MG-ADL score at Month 12. The deepest and most compelling sustained responses were observed in Descartes-08-treated participants who did not have prior exposure to biologic therapies, with an average 7.1-point reduction in MG-ADL score and 57% of patients in this subgroup maintaining minimum symptom expression at Month 12. The safety profile of Descartes-08 was consistent with previously reported data and continues to support outpatient administration.An encore presentation of the data, which were originally shared at the 2025 American Academy of Neurology Annual Meeting, was featured during the 15th International Conference on Myasthenia Gravis and Related Disorders on May 15, 2025 in The Hague, Netherlands. Preliminary Data from Ongoing Phase 2 Open-Label Trial of Descartes-08 in Patients with SLE Expected in the Second Half of 2025. The trial is designed to assess the safety, tolerability and clinical activity of outpatient Descartes-08 administration without preconditioning chemotherapy in patients with SLE. SLE is an incurable autoimmune disease marked by systemic inflammation that affects multiple organ systems and impacts approximately 1.5 million people in the United States.Phase 2 Pediatric Basket Trial of Descartes-08 in Select Autoimmune Diseases Expected to Initiate in the Second Half of 2025. This pediatric basket trial will target juvenile SLE, juvenile MG, juvenile dermatomyositis (JDM) and anti-neutrophil cytoplasmic antibody associated vasculitis. The FDA previously granted Rare Pediatric Disease Designation to Descartes-08 for the treatment of JDM, which is a rare pediatric autoimmune disorder.Dosing is On Track in First-in-Human Phase 1 Clinical Trial of Descartes-15. The Phase 1 dose escalation trial of Cartesian’s next-generation, autologous anti-BCMA CAR-T cell therapy is designed to assess the safety and tolerability of outpatient Descartes-15 administration in patients with multiple myeloma. Following the Phase 1 dose escalation trial, the Company expects to subsequently assess Descartes-15 in autoimmune indications. Second Quarter 2025 Financial Results Cash, cash equivalents and restricted cash as of June 30, 2025 was $162.1 million and is expected to support planned operations, including completion of the ongoing Phase 3 AURORA trial for Descartes-08 in MG, into mid-2027.Research and development expenses were $14.9 million for the three months ended June 30, 2025, compared to $12.7 million for the three months ended June 30, 2024. The increase in expenses was primarily a result of increased expenses associated with the ongoing Phase 3 AURORA trial for Descartes-08 for MG.General and administrative expenses were $7.2 million for the three months ended June 30, 2025, compared to $7.0 million for the three months ended June 30, 2024. The increase in expenses was primarily the result of increased facilities expenses.Net income was $15.9 million, or $0.51 net income per share allocable to common stockholders (basic), for the three months ended June 30, 2025, compared to net income of $13.8 million, or $0.58 net income per share allocable to common stockholders (basic), for the three months ended June 30, 2024. About Descartes-08 Descartes-08, Cartesian’s lead cell therapy candidate, is an autologous chimeric antigen receptor T-cell therapy (CAR-T) product targeting B-cell maturation antigen (BCMA) in clinical development for generalized myasthenia gravis (MG) and systemic lupus erythematosus. In contrast to conventional DNA-based CAR T-cell therapies, Cartesian’s CAR-T administration is designed to not require preconditioning chemotherapy, can be administered in the outpatient setting, and does not carry the risk of genomic integration associated with cancerous transformation. Descartes-08 has been granted Orphan Drug Designation and Regenerative Medicine Advanced Therapy Designation by the U.S. Food and Drug Administration for the treatment of MG, and Rare Pediatric Disease Designation for the treatment of juvenile dermatomyositis. About Descartes-15 Descartes-15 is a next-generation, autologous anti-BCMA CAR-T cell therapy. In preclinical studies, Descartes-15 has been observed to achieve an approximately ten-fold increase in CAR expression and selective target-specific killing, relative to Descartes-08. Similar to Descartes-08, Descartes-15 is designed to be administered without preconditioning chemotherapy and does not use integrating vectors. About Cartesian Therapeutics Cartesian Therapeutics is a clinical-stage company pioneering cell therapy for the treatment of autoimmune diseases. The Company’s lead asset, Descartes-08, is a CAR-T in Phase 3 clinical development for patients with generalized myasthenia gravis and Phase 2 development for systemic lupus erythematosus, with a Phase 2 basket trial planned in additional autoimmune indications. The Company’s 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. For more information, please visit www.cartesiantherapeutics.com or follow the Company on LinkedIn or X, formerly known as Twitter. Forward Looking Statements Any statements in this press release about the future expectations, plans and prospects of the Company, including without limitation, statements regarding the Company’s expected cash resources and cash runway, the ability of the Company’s product candidates to be administered in an outpatient setting or without the need for preconditioning lymphodepleting chemotherapy, the potential of Descartes-08, Descartes-15, or any of the Company’s other product candidates to treat myasthenia gravis, juvenile myasthenia gravis, systemic lupus erythematosus, juvenile systemic lupus erythematosus, juvenile dermatomyositis, anti-neutrophil cytoplasmic antibody-associated vasculitis, multiple myeloma, or any other disease, the anticipated timing or the outcome of ongoing and planned clinical trials, studies and data readouts, including the Phase 3 AURORA trial of Descartes-08 in myasthenia gravis, the planned Phase 2 pediatric basket trial of Descartes-08 in juvenile dermatomyositis, juvenile systemic lupus erythematosus, juvenile myasthenia gravis, and anti-neutrophil cytoplasmic antibody-associated vasculitis, and the ongoing Phase 2 trial of Descartes-08 in systemic lupus erythematosus, the anticipated timing or the outcome of the FDA’s review of the Company’s regulatory filings, the Company’s ability to conduct its clinical trials and preclinical studies, the timing or making of any regulatory filings, the anticipated timing or outcome of selection of developmental product candidates, the novelty of treatment paradigms that the Company is able to develop, the potential of any therapies developed by the Company to fulfill unmet medical needs, and enrollment in the Company’s clinical trials and other statements containing the words “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “hypothesize,” “intend,” “may,” “plan,” “potential,” “predict,” “project,” “should,” “target,” “would,” and similar expressions, constitute forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including, but not limited to, the following: the uncertainties inherent in the initiation, completion and cost of clinical trials including proof of concept trials, including uncertain outcomes, the availability and timing of data from ongoing and future clinical trials and the results of such trials, whether preliminary results from a particular clinical trial will be predictive of the final results of that trial and whether results of early clinical trials will be indicative of the results of later clinical trials, the ability to predict results of studies performed on human beings based on results of studies performed on non-human subjects, the unproven approach of the Company’s technology, potential delays in enrollment of patients, undesirable side effects of the Company’s product candidates, political uncertainty, the Company’s reliance on third parties to conduct its clinical trials, the Company’s inability to maintain its existing or future collaborations, licenses or contractual relationships, its inability to protect its proprietary technology and intellectual property, potential delays in regulatory approvals, the availability of funding sufficient for its foreseeable and unforeseeable operating expenses and capital expenditure requirements, the Company’s recurring losses from operations and negative cash flows, substantial fluctuation in the price of the Company’s common stock, risks related to geopolitical conflicts, pandemics, and macroeconomic impacts, and other important factors discussed in the “Risk Factors” section of the Company’s most recent Annual Report on Form 10-K and subsequently filed Quarterly Reports on Form 10-Q, and in other filings that the Company makes with the Securities and Exchange Commission. In addition, any forward-looking statements included in this press release represent the Company’s views only as of the date of its publication and should not be relied upon as representing its views as of any subsequent date. The Company specifically disclaims any intention to update any forward-looking statements included in this press release, except as required by law. Cartesian Therapeutics, Inc. and SubsidiariesConsolidated Balance Sheets(Amounts in thousands, except share data and par value) June 30, 2025 December 31, 2024 (Unaudited) Assets Current assets: Cash and cash equivalents$160,324 $212,610 Accounts receivable 354 872 Prepaid expenses and other current assets 2,717 3,144 Total current assets 163,395 216,626 Non-current assets: Property and equipment, net 12,285 9,912 Right-of-use asset, net 5,164 5,535 In-process research and development assets 150,600 150,600 Goodwill 48,163 48,163 Long-term restricted cash 1,735 1,669 Investments 2,000 2,000 Other assets 5,551 518 Total assets$388,893 $435,023 Liabilities and stockholders’ deficit Current liabilities: Accounts payable$454 $288 Accrued expenses and other current liabilities 8,012 12,076 Lease liability 3,790 2,851 Contingent value right liability — 7,761 Total current liabilities 12,256 22,976 Non-current liabilities: Lease liability, net of current portion 9,559 11,133 Warrant liabilities, net of current portion 1,364 3,836 Contingent value right liability, net of current portion 352,100 387,739 Deferred tax liabilities, net 16,141 16,141 Total liabilities 391,420 441,825 Stockholders’ deficit: Series A Preferred Stock, $0.0001 par value; 134,904.563 shares authorized as of June 30, 2025 and December 31, 2024; 120,790.402 shares issued and outstanding as of June 30, 2025 and December 31, 2024 — — Series B Preferred Stock, $0.0001 par value; 437,927 shares authorized as of June 30, 2025 and December 31, 2024; 437,927 shares issued and outstanding as of June 30, 2025 and December 31, 2024 — — Preferred stock, $0.0001 par value; 9,427,168.437 shares authorized as of June 30, 2025 and December 31, 2024; no shares issued and outstanding as of June 30, 2025 and December 31, 2024 — — Common stock, $0.0001 par value; 350,000,000 shares authorized as of June 30, 2025 and December 31, 2024; 26,000,065 and 25,767,369 shares issued and outstanding as of June 30, 2025 and December 31, 2024, respectively 3 3 Additional paid-in capital 695,942 689,887 Accumulated deficit (693,895) (692,071)Accumulated other comprehensive loss (4,577) (4,621)Total stockholders’ deficit (2,527) (6,802)Total liabilities and stockholders’ deficit$388,893 $435,023 Cartesian Therapeutics, Inc. and Subsidiaries Consolidated Statements of Operations and Comprehensive Income (Loss)(Amounts in thousands, except share and per share data) Three Months EndedJune 30, Six Months EndedJune 30, 2025 2024 2025 2024 (Unaudited)Revenue: Collaboration and license revenue$— $33,271 $400 $39,111 Grant revenue 298 174 998 174 Total revenue 298 33,445 1,398 39,285 Operating expenses: Research and development 14,869 12,661 29,543 22,399 General and administrative 7,240 7,027 15,555 16,477 Total operating expenses 22,109 19,688 45,098 38,876 Operating (loss) income (21,811) 13,757 (43,700) 409 Interest income 1,748 1,195 3,763 2,359 Change in fair value of warrant liabilities 654 (3,908) 2,472 (2,866)Change in fair value of contingent value right liability 35,300 2,500 35,646 (36,800)Change in fair value of forward contract liabilities — — — (6,890)Other (expense) income, net (5) 292 (5) 800 Net income (loss)$15,886 $13,836 $(1,824) $(42,988) Other comprehensive income: Foreign currency translation adjustment 12 14 44 9 Total comprehensive income (loss)$15,898 $13,850 $(1,780) $(42,979) Net income (loss) per share allocable to common stockholders: Basic$0.51 $0.58 $(0.07) $(3.88)Diluted$0.50 $0.54 $(0.07) $(3.88)Weighted-average common shares outstanding: Basic 25,980,262 16,723,479 25,941,670 11,068,749 Diluted 26,447,251 17,791,143 25,941,670 11,068,749 Investor Contact Megan LeDucAssociate Director of Investor Relationsmegan.leduc@cartesiantx.com Media ContactDavid RosenArgot Partnersdavid.rosen@argotpartners.com

近期，先后有两篇报道表明CAR-T可以治愈一种罕见神经自免疾病——慢性炎症性脱髓鞘性多发性神经病（CIDP）。其中一篇来自于华中科技大学同济医院的研究（5月发表于《Cell》），另外一篇来自于德国北莱茵-威斯特法伦州鲁尔大学波鸿分校附属医院的研究（发表于《柳叶刀·神经病学》）。双方分别采用了CD19和BCMA两种靶向不同的CAR-T分别证明了CAR-T治疗该疾病的有效性。这是全球首批关于CAR T细胞疗法用于治疗严重自身免疫性神经病变的临床报告。机制机理CIDP是一种典型的自身免疫性疾病，其根本病因是免疫系统错误地将周围神经系统的髓鞘识别为“外来入侵者”，并发动攻击。例如，B淋巴细胞在CIDP患者中异常活跃，会产生针对髓鞘蛋白（如P0、P2）的自身抗体，或通过分泌促炎细胞因子（如IL-6、TNF-α）直接破坏髓鞘。而髓鞘破坏就会导致肢体无力、感觉异常（如麻木、针刺感），严重时可致呼吸肌麻痹，最终导致死亡。更关键的是，15%的CIDP患者对传统疗法（如激素、免疫球蛋白）无效，且疾病呈慢性进展性。在波鸿中的两例患者都尝试了各种各样的免疫疗法，如静脉免疫球蛋白、皮质类固醇、血浆置换、环磷酰胺、利妥昔单抗、obinutuzumab和硼替佐米等等，但最终因为疾病的难治性都不得不转向CAR-T治疗方案。CAR-T可以通过靶向B细胞表面特异性抗原（如BCMA和CD19），可以直接清除致病性的B淋巴细胞，为神经修复创造条件。治疗状况华中科技大学同济医院的案例中纳入了两名高度难治性CIDP患者，均为男性，年龄分别为52岁和63岁。两名患者均经历多次疾病复发，且接受多种免疫治疗后效果不佳，存在严重功能障碍。两名患者体内的CAR-T细胞均成功扩增，并在输注后10天达到峰值，且能在体内持续存在至少6个月。输注后，两名患者均出现持续约2个月的B细胞谱系完全清除，自身抗体水平可能有所下降。输注后6个月内，两名患者均实现无药缓解，神经功能显著改善，可生活自理。具体表现为肌力提高、感觉障碍减轻、日常生活能力提高等。患者1在由于受到新冠感染再激活B细胞导致注输12个月后复发，患者2则维持24个月持续缓解。波鸿团队的案例中同样纳入了两名难治性CIDP，患者1为72岁男性，患者2为54岁男性。输注后，两名患者均出现显著的临床改善。患者1从间歇性不动改善为进行深蹲和引体向上，炎症性神经病变指标（如总体残疾评分、步行距离和血清神经丝轻链浓度）均有显著改善。患者2的神经功能也有显著改善，具体表现为行走能力提高、感觉功能恢复等。图：治疗10个月后，患者1已经可以自由走动两名患者均出现轻至中度细胞因子释放综合征，但通过tocilizumab和皮质类固醇治疗得到缓解。患者1还出现1级免疫效应细胞相关神经毒性综合征，此后完全缓解。总结此次关于CIDP的CAR-T研究似乎正在揭开重症自身免疫疾病治疗的新篇章。未来可以看到华中科技大学同济医院和波鸿大学医院的更多临床案例，可能将适应症扩展到重症肌无力、僵人综合征等疾病上。参考来源：Jeremias Motte et al, CD19-targeted CAR T-cell therapy for treatment-refractory autoimmune neuropathies, The Lancet Neurology (2025). DOI: 10.1016/S1474-4422(25)00199-1Dong, Ming-Hao et al. “Anti-BCMA CAR-T cell therapy in relapsed/refractory chronic inflammatory demyelinating polyneuropathy.” Med (New York, N.Y.), 100704. 16 May. 2025.