MB-108

声明：因水平有限，错误不可避免，或有些信息非最及时，欢迎留言指出。本文仅作医疗健康相关药物介绍，非治疗方案推荐（若涉及）;本文不构成任何投资建议。2025年7月7日，Mustang Bio（纳斯达克股票代码：MBIO）宣布美国食品药品监督管理局（FDA）已授予其靶向IL13Rα2的CAR-T细胞疗法MB-101孤儿药资格认证，用于治疗复发性弥漫性和间变性星形细胞瘤和胶质母细胞瘤。该认定为Mustang Bio提供了多项优惠，包括临床试验税收抵免以及在治疗获得监管批准后七年的市场独占权。据悉，其认定范围涵盖美国境内少于20万人的罕见疾病患者。受此消息影响，公司股价昨晚盘中大涨超480%，截至收盘涨超180%，报3.34美元/股。Mustang Bio成立于2015年，是一家临床阶段生物制药公司，专注于开发和商业化新型癌症免疫治疗产品，其开发的MB-101是一种CAR-T细胞疗法，其旨在通过靶向肿瘤细胞表面的IL13Rα2蛋白，训练人体自身的免疫细胞识别并消灭脑癌细胞。公司管线MB-101正与MB-108（一种HSV-1溶瘤病毒，此前已获得恶性胶质瘤治疗孤儿药资格）作为新型联合疗法进行开发。这种组合被命名为MB-109，旨在通过使用MB-108重塑肿瘤微环境来提高疗效。这一治疗策略将MB-101 CAR-T细胞疗法与MB-108溶瘤病毒相结合，有可能使"冷肿瘤变热"，从而提高难治性癌症的治疗效果。希望之城和阿拉巴马大学伯明翰分校的早期数据显示，MB-101和MB-108在复发性胶质母细胞瘤患者中均具有良好的耐受性。希望之城2024年发表在《Nature Medicine》上的I期临床试验数据中，MB-101显示出有希望的结果，50%的患者达到了稳定病情或更好的效果。两名具有"热肿瘤"（肿瘤内CD3+ T细胞水平高）的患者分别实现了持续7.5个月和66+个月的完全缓解—对于这些通常中位生存期为12-15个月的侵袭性脑肿瘤来说，这可谓异常漫长。值得注意的是，这2例完全缓解发生在接受MB-101治疗前肿瘤“最热”的3例患者中。希望之城的MB-101和阿拉巴马大学伯明翰分校的MB-108 1期临床试验仍在持续招募患者。该公司指出，MB-109项目的进一步开发取决于筹集额外资金或建立战略合作伙伴关系。Mustang 总裁兼首席执行官 Manuel Litchman 医学博士表示：“我们很高兴 MB-101能够按时获得孤儿药资格认定，并且该资格认定的适用范围比拟定的适应症更广。MB-101的孤儿药资格认定，加上之前MB-108获得的孤儿药资格认定，有力地证明了我们的科学研究实力。我们希望推进MB-101与MB-108的联合应用，使其成为恶性胶质瘤患者的潜在治疗选择。我们新颖的治疗策略是将MB-101 CAR-T细胞疗法与 MB-108 溶瘤病毒相结合，利用MB-108重塑肿瘤微环境，使冷肿瘤变“热”，从而潜在地提高 MB-101 CAR-T 细胞疗法的疗效。这一进展表明我们致力于探索改善患者预后的新途径。难以治疗的癌症。”总结市场研究数据显示，2025年全球CAR-T疗法的市场规模将达到110亿美元，预计到2034年将攀升至1900亿美元。近年来，多个制药巨头通过合作或收购布局CAR-T赛道：2024年1月，艾伯维和Umoja Biopharma宣布达成潜在总额14亿美元的两项独家选择权和许可协议，双方将利用Umoja专有的VivoVec平台，在肿瘤学领域开发多个原位生成的CAR-T细胞治疗候选药物。2024年2月，安斯泰来宣布与体内CAR-T第一梯队企业Kelonia达成潜在总额8.75亿美元的研究合作和许可协议，将共同开发创新、现货型的体内CAR-T。2024年11月，诺华与Vyriad达成了一项战略合作协议，旨在开发创新的体内CAR-T细胞疗法。2025年6月，艾伯维宣布，将以21亿美元的价格收购体内CAR-T细胞疗法开发商Capstan Therapeutics，以扩大自身免疫性疾病疗法的产品线。后者的主要资产CPTX2309目前正处于一期临床阶段。2025年3月17日，阿斯利康宣布以10亿美元总对价收购比利时生物技术公司EsoBiotec。该公司开发的工程纳米抗体慢病毒平台，能在体内高效地将T淋巴细胞重编程为特定功能细胞。2025年7月，《柳叶刀》杂志上一篇论文中首次报告了EsoBiotec/普瑞金开发的体内CAR T（ESO-T01）在4例多发性骨髓瘤患者中的完整临床数据，四名患者均对治疗产生反应，其中两名患者的癌症完全消退，标志着体内CAR T的重大进展。跨国药企竞速布局体内CAR-T赛道时，国内不乏创新药企迎头赶上。中国创新药企原启生物亦于2025年5月与Umoja Biopharma达成战略合作，在全球多个区域内共同开拓下一代体内CAR-T疗法。云顶新耀依托自研AI+mRNA平台开发“自体生成CAR-T”项目，通过LNP递送mRNA实现体内T细胞编程，适应症覆盖肿瘤及自身免疫疾病‌；石药集团利用 mRNA-LNP 技术生产的BCMA CAR-T疗法已进入临床亦可期待。参考资料：公司公告-END-扫码参与报名

In an ongoing Phase 1 trial published in Nature Medicine, MB-101 was well-tolerated and 50% of patients achieved stable disease or better with two partial responses and two complete responses lasting 7.5 and 66+ months, respectively Preclinical data support a novel combination of MB-101 (IL13Rα2‐targeted CAR-T cell therapy) and MB-108 (HSV-1 oncolytic virus) to optimize clinical results FDA previously granted Orphan Drug Designation to Mustang for MB-108 for the treatment of malignant glioma WORCESTER, Mass., July 07, 2025 (GLOBE NEWSWIRE) -- Mustang Bio, Inc. (“Mustang” or the “Company”) (Nasdaq: MBIO), a clinical-stage biopharmaceutical company focused on translating today’s medical breakthroughs in cell therapies into potential cures for difficult-to-treat cancers, today announced that the U.S. Food and Drug Administration (“FDA”) has granted Orphan Drug Designation to Mustang for MB-101 (IL13Ra2-targeted CAR T-cells) for the treatment of recurrent diffuse and anaplastic astrocytoma (astrocytomas) and glioblastoma (GBM). The FDA grants Orphan Drug Designation to drugs and biologics that are intended for safe and effective treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the U.S. Orphan Drug Designation provides certain incentives, such as tax credits toward the cost of clinical trials upon approval and prescription drug user fee waivers. If a product receives Orphan Drug Status from the FDA, that product is entitled to seven years of market exclusivity for the disease in which it has Orphan Drug designation, which is independent from intellectual property protection. Manuel Litchman, M.D., President and Chief Executive Officer of Mustang, said, “We are thrilled that MB-101 received Orphan Drug Designation on time and with a designation that is broader than the indication proposed. The Orphan Drug Designation for MB-101, coupled with the Orphan Drug Designation granted previously for MB-108, is strong validation for our science, as we hope to advance MB-101, in combination with MB-108, as a potential treatment option for patients living with malignant glioma, including patients with recurrent glioblastoma (“GBM”) and high-grade astrocytomas. Our novel therapeutic strategy, combining our MB-101 CAR-T cell therapy with our MB-108 oncolytic virus, leverages MB-108 to reshape the tumor microenvironment (“TME”) to make cold tumors “hot,” thereby potentially improving the efficacy of MB-101 CAR-T cell therapy. This progress demonstrates our dedication to exploring new possibilities for improving outcomes in patients with challenging-to-treat cancers.” As previously reported, preclinical data presented at the American Association for Cancer Research (“AACR”) Annual Meeting in 2022 supported a combination therapy to potentially optimize results to treat recurrent GBM. The combination leverages MB-108 to reshape the TME and make cold tumors “hot,” thereby potentially improving the efficacy of MB-101 CAR-T cell therapy. Data presented separately on MB-101 and MB-108 showed that administration of these therapies was well tolerated in recurrent GBM patients. As reported in City of Hope’s 2024 Nature Medicine paper, 2 patients treated solely with MB-101 who had high levels of intratumoral CD3+ T cells pre-therapy (i.e., “hot” tumors) achieved complete responses lasting 7.5 and 66+ months, respectively. Importantly, of the 57 City of Hope Phase 1 patients evaluable for survival in that paper, these 2 complete responses were observed in the cohort of 3 patients with the “hottest” tumors prior to treatment with MB-101. Phase 1 clinical trials of MB-101 at City of Hope and of MB-108 at The University of Alabama at Birmingham continue to enroll patients. The Company’s ability to further develop the MB-109 program for recurrent GBM and high-grade astrocytomas is contingent upon raising additional funding and / or consummating a strategic partnership. About MB-109 (MB-101 (IL13Rα2 targeted CAR-T cells) + MB-108 oncolytic virus) MB-109 is Mustang’s designation for the treatment regimen combining MB-101 (IL13Rα2‐targeted CAR-T cell therapy licensed from City of Hope) with MB-108 (HSV-1 oncolytic virus licensed from Nationwide Children’s Hospital). The combination is designed to leverage MB-108 to make cold tumors “hot” and potentially improve the efficacy of MB-101 CAR-T cell therapy. MB-108 oncolytic virus is first injected to infect tumor cells which, in turn, leads to reshaping of the TME through recruitment of endogenous CD8- and CD3-positive effector T-cells. This inflamed TME potentially permits MB-101 CAR-T cells injected into and around the tumor to better infiltrate into and throughout the tumor mass, undergo activation and, ideally, effect tumor cell killing. About Mustang Bio Mustang Bio, Inc. is a clinical-stage biopharmaceutical company focused on translating today’s medical breakthroughs in cell therapies into potential cures for difficult-to-treat cancers. Mustang aims to acquire rights to these technologies by licensing or otherwise acquiring an ownership interest, to fund research and development, and to outlicense or bring the technologies to market. Mustang has partnered with top medical institutions to advance the development of CAR-T therapies. Mustang’s common stock is registered under the Securities Exchange Act of 1934, as amended, and Mustang files periodic reports with the U.S. Securities and Exchange Commission (“SEC”). Mustang was founded by Fortress Biotech, Inc. (Nasdaq: FBIO). For more information, visit www.mustangbio.com. Forward-Looking Statements This press release contains “forward-looking statements” within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934, each as amended. Such statements, which are often indicated by terms such as “anticipate,” “believe,” “could,” “estimate,” “expect,” “goal,” “intend,” “look forward to,” “may,” “plan,” “potential,” “predict,” “project,” “should,” “will,” “would” and similar expressions. The Company’s forward-looking statements, include, but are not limited to, any statements relating to our growth strategy and product development programs, including the timing of and our ability to make regulatory filings such as INDs and other applications and to obtain regulatory approvals for our product candidates, statements concerning the potential of therapies and product candidates and any other statements that are not historical facts. Actual events or results may differ materially from those described in this press release due to a number of risks and uncertainties. Risks and uncertainties include, among other things, our need for substantial additional funds in the immediate future, risks that any actual or potential clinical trials described herein may not initiate or complete in sufficient timeframes to advance the Company’s corporate objectives, or at all, or that promising early results obtained therefrom may not be replicable; whether the purchaser of the Company’s manufacturing facility is able to successfully perform its obligation to produce the Company’s products under the manufacturing services agreement on a timely basis and to acceptable standards; disruption from the sale of the Company’s manufacturing facility making it more difficult to maintain business and operational relationships; negative effects of the announcement or the consummation of the transaction on the market price of the Company’s common stock; significant transaction costs; the development stage of the Company’s primary product candidates, our ability to obtain, perform under, and maintain financing and strategic agreements and relationships; risks relating to the results of research and development activities; risks relating to the timing of starting and completing clinical trials; uncertainties relating to preclinical and clinical testing; our dependence on third-party suppliers; our ability to attract, integrate and retain key personnel; the early stage of products under development; government regulation; patent and intellectual property matters; competition; as well as other risks described in Part I, Item 1A, “Risk Factors,” in our Annual Report on Form 10-K filed on March 28, 2025, subsequent Reports on Form 10-Q, and our other filings we make with the SEC. We expressly disclaim any obligation or undertaking to release publicly any updates or revisions to any forward-looking statements contained herein to reflect any change in our expectations or any changes in events, conditions or circumstances on which any such statement is based, except as required by law, and we claim the protection of the safe harbor for forward-looking statements contained in the Private Securities Litigation Reform Act of 1995. Company Contacts: Jaclyn Jaffe and Nicole McCloskey Mustang Bio, Inc. (781) 652-4500 ir@mustangbio.com Released July 7, 2025 • 8:30 AM EDT