Melphalan hydrochloride

QUEENSBURY, N.Y.--(BUSINESS WIRE)--Delcath Systems, Inc. (Nasdaq: DCTH), an interventional oncology company focused on the treatment of primary and metastatic liver cancers, today announced the publication of a comparative analysis from the randomized cohort of the Company’s Phase 3 FOCUS study in Annals of Surgical Oncology. The study, titled “An Open-label, Randomized Study of Melphalan/Hepatic Delivery System Versus Best Alternative Care in Patients with Unresectable Metastatic Uveal Melanoma,” indicates that treatment with the Melphalan/Hepatic Delivery System (Melphalan/HDS) shows a trend toward favorable clinical outcomes compared to best alternative care (BAC), which included the physician’s choice of dacarbazine, transarterial chemoembolization (TACE), ipilimumab or pembrolizumab in patients with unresectable metastatic uveal melanoma (mUM) with liver involvement. The FOCUS study was initially designed and conducted as a randomized controlled trial but was amended to a single-arm design. As a result, comparative efficacy analyses were designated as exploratory. A total of 85 patients were enrolled during the randomized portion of the trial, with 72 receiving study treatment (40 Melphalan/HDS; 32 BAC). Key Findings from the Randomized Portion of the FOCUS Study: All efficacy endpoints of the trial demonstrated substantial and consistent improvements in patients treated with Melphalan/HDS over BAC, including: Median progression-free survival in patients treated with Melphalan/HDS was 9.1 months, nearly three times longer than the 3.3 months observed in patients treated with BAC Median overall survival was 18.5 months for Melphalan/HDS compared to 14.5 months with BAC Objective response rate was 27.5% with Melphalan/HDS, nearly three times higher than 9.4% with BAC Disease control rate was 80.0% with Melphalan/HDS, substantially higher than the 46.9% observed with BAC Median hepatic progression-free survival was 11.4 months for Melphalan/HDS – more than three times longer than the 3.3 months for BAC Median progression-free survival in patients treated with Melphalan/HDS was 9.1 months, nearly three times longer than the 3.3 months observed in patients treated with BAC Median overall survival was 18.5 months for Melphalan/HDS compared to 14.5 months with BAC Objective response rate was 27.5% with Melphalan/HDS, nearly three times higher than 9.4% with BAC Disease control rate was 80.0% with Melphalan/HDS, substantially higher than the 46.9% observed with BAC Median hepatic progression-free survival was 11.4 months for Melphalan/HDS – more than three times longer than the 3.3 months for BAC The safety profile of patients treated with Melphalan/HDS was consistent with prior reports and primarily hematologic in nature. No treatment-related deaths were observed. “These results further support the use of our hepatic delivery system as a liver-directed treatment option for patients with metastatic uveal melanoma,” said Dr. Vojislav Vukovic, Chief Medical Officer at Delcath. “Melphalan/HDS delivered meaningful improvements across all clinical endpoints, including progression-free survival, overall survival, disease control, and hepatic outcomes. The findings, while exploratory in nature, reinforce our belief that effective management of liver disease is key to improving outcomes in this patient population, and we are proud to contribute a therapy with both clinical impact and the ability to be administered repeatedly.” The publication is available online at: https://doi.org/10.1245/s10434-025-17231-x About Delcath Systems, Inc., HEPZATO KIT™ and CHEMOSAT® Delcath Systems, Inc. is an interventional oncology company focused on the treatment of primary and metastatic liver cancers. The company’s proprietary products, HEPZATO KIT™ (HEPZATO (melphalan) for Injection/Hepatic Delivery System) and CHEMOSAT® Hepatic Delivery System for Melphalan percutaneous hepatic perfusion (PHP), are designed to administer high-dose chemotherapy to the liver while controlling systemic exposure and associated side effects during a PHP procedure. In the United States, HEPZATO KIT is regulated and approved as a combination drug and device product by the FDA. HEPZATO KIT is approved as a liver-directed treatment for adult patients with metastatic uveal melanoma (mUM) with unresectable hepatic metastases affecting less than 50% of the liver and no extrahepatic disease, or extrahepatic disease limited to the bone, lymph nodes, subcutaneous tissues, or lung that is amenable to resection or radiation. Please see full Prescribing Information, including BOXED WARNING, at www.hepzatokit.com. In Europe, the device-only configuration of the HDS is regulated as a Class III medical device and is marketed under the trade name CHEMOSAT®, where it has been used in major cancer centers to treat a wide range of cancers of the liver. Safe Harbor / Forward-Looking Statements The Private Securities Litigation Reform Act of 1995 provides a safe harbor for forward-looking statements made by the Company or on its behalf. This press release contains forward-looking statements, which are subject to certain risks and uncertainties, that can cause actual results to differ materially from those described. The words “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “intend,” “may,” “plan,” “potential,” “predict,” “project,” “should,” “target,” “will,” “would” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Factors that may cause such differences include, but are not limited to, uncertainties relating to: the Company’s commercialization plans and its ability to successfully commercialize the HEPZATO KIT; contributions to adjusted EBITDA; the Company’s successful management of the HEPZATO KIT supply chain, including securing adequate supply of critical components necessary to manufacture and assemble the HEPZATO KIT and the managing of any trade restrictions; successful FDA inspections of the facilities of the Company and those of its third-party suppliers/manufacturers; the Company’s successful implementation and management of the HEPZATO KIT Risk Evaluation and Mitigation Strategy; the potential benefits of the HEPZATO KIT as a treatment for patients with primary and metastatic disease in the liver; the Company’s ability to obtain reimbursement for the HEPZATO KIT; and the Company’s ability to successfully enter into any necessary purchase and sale agreements with users of the HEPZATO KIT. For additional information about these factors, and others that may impact the Company, please see the Company’s filings with the Securities and Exchange Commission, including those on Forms 10-K, 10-Q, and 8-K. However, new risk factors and uncertainties may emerge from time to time, and it is not possible to predict all risk factors and uncertainties. Accordingly, you should not place undue reliance on these forward-looking statements, which speak only as of the date they are made. We undertake no obligation to publicly update or revise these forward-looking statements to reflect events or circumstances after the date they are made.

2025年3月31日，CASI制药正式发布截至2024年12月31日的年度财务报告，并同步披露核心产品管线亮点及业务进展。 CASI制药董事长兼首席执行官何为无博士表示：“2024年是CASI的重要转型之年，我们通过战略性聚焦CID-103的开发，深化布局器官移植排斥反应及自身免疫疾病领域。CID-103是一种抗CD38抗体，有望成为治疗多种自身免疫性疾病和抗体介导的排斥反应(AMR)的突破性疗法。2025年1月完成的免疫性血小板减少症（ITP）首例患者给药，标志着全球临床开发进程的重要里程碑。目前，我们正与美国食品药品监督管理局（FDA）保持密切沟通，积极推进AMR临床试验暂停事项的解决方案。” 何博士补充道：“展望2025年，我们将继续推进CID-103的临床开发，并预计实现多个关键里程碑。我们将继续贯彻聚焦核心管线的战略，通过审慎的资本配置和高效的执行能力，持续造福患者、创造股东价值。 关键业务进展 • 2024年第四季度收入为1,340万美元，全年累计营收2,850万美元。 • 2024年6月21日，收到何为无博士发出的收购 CASI中国业务的要约。 • 2024年7月15日，与Venrock Healthcare Capital Partners、Foresite Capital、Panacea Venture及何为无博士达成1,500万美元私募融资协议。 核心管线进展 • 2024年5月15日，CID-103治疗ITP的临床试验获得FDA新药临床试验（IND）批准。 • 2024年10月24日，CID-103治疗ITP的临床试验获得中国国家药品监督管理局（NMPA）临床试验申请（CTA）批准。 • 2025年1月3日，CID-103在中国开展的I/II期临床试验完成首例患者给药，患者招募及治疗仍在进行中。 2025年预期里程碑 • 预计2025年第二季度FDA将对AMR临床试验暂停问题作出反馈。 • 计划于2025年第二季度提交CID-103用于再生障碍性贫血的IND申请。 • 预计2025年年中公布CID-103 ITP I期研究的中期数据。 • 计划于2025年第二季度完成Precision Autoimmune Therapeutics（PAT）的股权转让协议，届时CASI将获得CID-103所有适应症的全球独家权益。 产品亮点 CID-103 (抗CD38单克隆抗体) CID-103是一种全人源IgG1抗CD38单克隆抗体，可识别CD38分子上的独特表位。与其他抗CD38单抗相比，CID-103的临床前数据显示出更好的疗效和安全性。CASI拥有CID-103的全球独家权益，并正在推进其在慢性ITP、AMR等适应症的开发。2024年5月，CID-103 治疗成人ITP的I/II期临床试验获得FDA批准。2024年10月，中国CDE批准该研究在中国慢性ITP患者中开展。中国获批的临床试验，是CID-103全球临床试验的重要组成部分。2025年1月，该临床试验完成首例患者给药。此外，我们正全力协同FDA推进AMR临床试验暂停问题的解决。 迈维宁® (注射用盐酸美法仑) 迈维宁®于2018年12月获得中国国家药监局批准进口并上市，2019年8月正式商业化。迈维宁®进入中国市场之前，全国每年仅有800名多发性骨髓瘤患者接受自体移植。而自2019年迈维宁®上市以来，经过CASI团队的不懈努力，2023年已有近10,000名患者因迈维宁®而得到治疗获益。2024年，国内一家生产商推出同质化的注射用美法仑仿制药，导致市场竞争加剧，并对迈维宁®的销售造成一定影响。 富洛特® (普拉曲沙注射液) 2023年7月31日，CASI与Mundipharma International Corporation Limited（MICL），Mundipharma Medical Company (MMCo)及Acrotech Biopharma Inc.(Acrotech)达成转让协议，获得FOLOTYN® （富洛特®）在中国的商业化权益。富洛特®是一种二氢叶酸还原酶抑制剂，已获FDA和NMPA批准用于治疗复发或难治性外周T细胞淋巴瘤（PTCL）。2024年2月15日，富洛特®完成首例中国患者给药。 2024年第四季度及全年财务业绩 • 2024年第四季度收入1,340万美元，较2023年同期的690万美元增长94%，反映出公司下半年商业战略的成功执行。2024年全年收入为2,850万美元，较2023年的3,390万美元下降16%，主要受国产注射用美法仑竞争影响。 • 2024年第四季度研发支出370万美元，较2023年同期的230万美元增长61%，主要用于开发CID-103，以支持公司战略重点转向器官移植排斥和自身免疫疾病。2024年全年研发支出890万美元，较2023年990万美元下降10%，主要因2023年底注销仿制药组合，降低了摊销费用。 • 2024年第四季度管理与行政开支710万美元，较2023年640万美元增长11%，主要因合源生物仲裁相关法律费用增加。2024年全年管理与行政开支2,360万美元，较2023年2,540万美元下降7%，反映出公司在运营效率和成本控制方面的持续优化。 • 2024年净亏损为3,930万美元，2023年为2,630万美元。 • 截至2024年12月31日，公司现金及现金等价物总计1,350万美元，而2023年底为2,910万美元（包含短期投资）。 • 截至2024年12月31日的完整版20-F年报已发布于公司官网 www.casipharmaceuticals.com。需获取纸质版年度报告者，请致函：北京市朝阳区建国路81号华贸写字楼1座1701-1702室，CASI Pharmaceuticals, Inc. 投资者关系部，邮编：100025。