Remestemcel-L-rknd

Partnering with Parent Project Muscular Dystrophy to ensure timely access to the trial for eligible patients ~15,000 children are living with DMD in the U.S. NEW YORK, April 07, 2026 (GLOBE NEWSWIRE) -- Mesoblast Limited (Nasdaq:MESO; ASX:MSB), global leader in allogeneic cellular medicines for inflammatory diseases, today announced that the United States Food and Drug Administration (FDA) has granted Investigational New Drug (IND) clearance to directly proceed for a registrational clinical trial evaluating Ryoncil ® (remestemcel-L-rknd) in Duchenne muscular dystrophy (DMD), which affects approximately 15,000 children in the U.S. 1 Ryoncil ® is the first mesenchymal stromal cell (MSC) product approved by FDA, and the only product approved for children under age 12 with steroid-refractory acute graft-versus-host disease (SR-aGvHD) 2 . This new registrational trial builds on Ryoncil’s ® proven safety in children, evidence of efficacy in DMD preclinical models, and FDA-approved manufacturing process. Leveraging Ryoncil's ® anti-inflammatory mechanism of action in SR-aGvHD, Mesoblast aims to reduce the inflammatory cascade characteristic of DMD, preserve muscle function, and slow disease progression. The trial will randomize 76 patients aged 5 to 9 years to either Ryoncil ® (7 infusions of 2 x 10 6 cells/kg over 9 months) or placebo, on top of standard of care. The trial’s primary endpoint will be time-to-stand at nine months, a validated FDA endpoint for approval. To support the successful execution of this study, Mesoblast is collaborating with Parent Project Muscular Dystrophy (PPMD) to foster patient identification and trial awareness through proactive community engagement. “This study represents an important step forward in potentially addressing the inflammatory component of DMD, a major driver of disease progression,” said Aravindhan Veerapandiyan, MD, Director of the Comprehensive Neuromuscular Program at Arkansas Children’s Hospital, and Principal Investigator of the study. “By leveraging the anti-inflammatory effects of Ryoncil, we aim to intervene at a stage where muscle tissue may still be preserved, potentially altering the trajectory of the disease.” “We are very pleased to have received clearance to proceed directly to a registrational study for DMD based on our preclinical data in DMD animal models and our extensive safety data in children with SR-aGvHD. Our experience with Ryoncil suggests that we may have a unique approach to help with this devastating disease in children,” said Silviu Itescu, Chief Executive of Mesoblast About Duchenne Muscular Dystrophy (DMD) Duchenne Muscular Dystrophy (DMD) is a X-linked genetic disorder characterized by progressive muscle degeneration affecting the skeletal, respiratory, and cardiac muscles. It is caused by the absence of functional dystrophin, a key structural protein in muscle cells. DMD affects approximately 15,000 individuals in the United States and primarily impacts boys. Over time, deterioration of the muscle leads to loss of ambulation, respiratory failure and cardiomyopathy ultimately leading to death by the third decade. While gene therapies that replace or increase the missing dystrophin protein are groundbreaking, they are not a complete cure. Chronic inflammation of skeletal and heart muscle remains a major underlying cause of progressive weakness, leading to loss of ambulation, reliance on wheelchair assistance and death in DMD. Corticosteroid use has improved survival but its effects plateau and long-term usage have serious side effects. About Parent Project Muscular Dystrophy Parent Project Muscular Dystrophy (PPMD), a leading patient advocacy organization, to support patient identification, education, and trial awareness. The organization was founded in 1994: . About Mesoblast Mesoblast (the Company) is a world leader in developing allogeneic (off-the-shelf) cellular medicines for the treatment of severe and life-threatening inflammatory conditions. The therapies from the Company’s proprietary mesenchymal lineage cell therapy technology platform respond to severe inflammation by releasing anti-inflammatory factors that counter and modulate multiple effector arms of the immune system, resulting in significant reduction of the damaging inflammatory process. Mesoblast’s Ryoncil ® (remestemcel-L-rknd) for the treatment of steroid-refractory acute graft versus host disease (SR-aGvHD) in pediatric patients 2 months and older is the first FDA-approved mesenchymal stromal cell (MSC) therapy. Please see the full Prescribing Information at . Mesoblast is committed to developing additional cell therapies for distinct indications based on its remestemcel-L and rexlemestrocel-L allogeneic stromal cell technology platforms. Ryoncil ® is being developed for additional inflammatory diseases including SR-aGvHD in adults and biologic-resistant inflammatory bowel disease. Rexlemestrocel-L is being developed for heart failure and chronic low back pain. The Company has established commercial partnerships in Japan, Europe and China. About Mesoblast intellectual property: Mesoblast has a strong and extensive global intellectual property portfolio, with over 1,000 granted patents or patent applications covering mesenchymal stromal cell compositions of matter, methods of manufacturing and indications. These granted patents and patent applications provide commercial protection extending through to at least 2044 in all major markets. About Mesoblast manufacturing: The Company’s proprietary manufacturing processes yield industrial-scale, cryopreserved, off-the-shelf, cellular medicines. These cell therapies, with defined pharmaceutical release criteria, are planned to be readily available to patients worldwide. Mesoblast has locations in Australia, the United States and Singapore and is listed on the Australian Securities Exchange (MSB) and on the Nasdaq (MESO). For more information, please see , LinkedIn: Mesoblast Limited and Twitter: @Mesoblast References / Footnotes Please see the full Prescribing Information at Forward-Looking Statements This press release includes forward-looking statements that relate to future events or our future financial performance and involve known and unknown risks, uncertainties and other factors that may cause our actual results, levels of activity, performance or achievements to differ materially from any future results, levels of activity, performance or achievements expressed or implied by these forward-looking statements. We make such forward-looking statements pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995 and other federal securities laws. Forward-looking statements should not be read as a guarantee of future performance or results, and actual results may differ from the results anticipated in these forward-looking statements, and the differences may be material and adverse. Forward-looking statements include, but are not limited to, statements about: the initiation, timing, progress and results of Mesoblast’s preclinical and clinical studies, and Mesoblast’s research and development programs; Mesoblast’s ability to advance product candidates into, enroll and successfully complete, clinical studies, including multi-national clinical trials; Mesoblast’s ability to advance its manufacturing capabilities; the timing or likelihood of regulatory filings and approvals, manufacturing activities and product marketing activities, if any; the commercialization of Mesoblast’s RYONCIL for pediatric SR-aGVHD and any other product candidates, if approved; regulatory or public perceptions and market acceptance surrounding the use of stem-cell based therapies; the potential for Mesoblast’s product candidates, if any are approved, to be withdrawn from the market due to patient adverse events or deaths; the potential benefits of strategic collaboration agreements and Mesoblast’s ability to enter into and maintain established strategic collaborations; Mesoblast’s ability to establish and maintain intellectual property on its product candidates and Mesoblast’s ability to successfully defend these in cases of alleged infringement; the scope of protection Mesoblast is able to establish and maintain for intellectual property rights covering its product candidates and technology; estimates of Mesoblast’s expenses, future revenues, capital requirements and its needs for additional financing; Mesoblast’s financial performance; developments relating to Mesoblast’s competitors and industry; and the pricing and reimbursement of Mesoblast’s product candidates, if approved. You should read this press release together with our risk factors, in our most recently filed reports with the SEC or on our website. Uncertainties and risks that may cause Mesoblast’s actual results, performance or achievements to be materially different from those which may be expressed or implied by such statements, and accordingly, you should not place undue reliance on these forward-looking statements. We do not undertake any obligations to publicly update or revise any forward-looking statements, whether as a result of new information, future developments or otherwise. Release authorized by the Chief Executive. For more information, please contact: Corporate Communications / Investors Paul Hughes T: +61 3 9639 6036 Media – Global Media – Australia Rubenstein BlueDot Media Caroline Nelson Steve Dabkowski T: +1 703 489 3037 T: +61 419 880 486 E: cnelson@rubenstein.com E: steve@bluedot.net.au

一、一张许可证，一个大信号 国家药品监督管理局数据查询信息显示，上海市药品监督管理局于3月27日核发了一张干细胞《药品生产许可证》（许可证编号：沪20260315）。许可信息明确，其生产范围包含治疗用生物制品：IxCell hUC-MSC-O注射液（仅限注册申报使用）。 这张证的意义，远不止“多了一张许可”这么简单——它意味着，一款针对膝骨关节炎的干细胞药物，正式从临床研发阶段，迈入了产业化准备的全新阶段。 二、从临床到量产，这条路走了多久？ 公开资料完整记录了这款产品的研发历程： 2019年 ：获得临床试验默示许可 2021年 ：在医院端启动II期临床研究 2023年8月 ：II期临床研究完成首次公示，采用随机、双盲、安慰剂对照、多中心设计，适应症为膝骨关节炎 2026年3月 ：成功取得《药品生产许可证》，进入III期临床与产业化准备并行阶段 如果把它放在整个行业坐标里看，这一步的分量会更清晰：2024年，北京药监局核发全国第一张干细胞《药品生产许可证》；2025年1月，国家药监局附条件批准艾米迈托赛注射液上市，被公认为我国干细胞治疗药物“从0到1”的里程碑[reference:0]。 如今，中国干细胞药物早已不再停留在“能不能走药品注册路径”的讨论，而是正式进入“哪些产品能真正走到产业化、商业化前夜”的新阶段。 三、为什么是膝骨关节炎？1.13亿人的痛点 这张许可证之所以备受关注，核心原因在于它瞄准的不是罕见病，而是一个患者基数巨大的常见慢病—— 膝骨关节炎 。 全球骨关节炎患者已近6亿人，而我国骨关节炎患者已突破 1亿人 ，总患病率高达15%[reference:1]。其中，膝骨关节炎占比最高，被称为“头号致残性疾病”[reference:2]。这意味着，有上亿人在忍受着关节疼痛、僵硬、活动受限的折磨，甚至最终走向残疾。 长期以来，膝骨关节炎的治疗始终处于尴尬的困境： 轻中度阶段 ：多依赖止痛、消炎、玻璃酸钠注射、康复训练、减重等方式，仅能缓解症状，无法逆转损伤 晚期阶段 ：多数患者只能选择关节置换手术，不仅创伤大、恢复周期长，还面临术后并发症风险 真正能同时实现炎症控制、症状改善、软骨组织修复的药物，一直是临床的巨大空白。 正因如此，膝骨关节炎成为了国内干细胞药物研发最热门的适应症之一。除了这款IxCell hUC-MSC-O注射液，国内已有多款间充质干细胞（MSC）药物推进到III期临床，赛道正式从“有人试”进入“谁先通”的白热化阶段。 值得一提的是，中山一院正在牵头开展干细胞治疗膝骨关节炎的II期临床研究，遵循国际公认的随机、双盲、平行对照标准，致力构建中国循证医学证据，为全球膝骨关节炎治疗贡献“中国方案”[reference:3]。 四、干细胞治膝关节炎，到底好在哪？ 这款药物采用的人脐带间充质干细胞，是目前干细胞治疗领域最成熟的细胞类型之一，针对膝骨关节炎的作用机制清晰明确[reference:4][reference:5]： 强效抗炎 ：抑制关节内炎症因子释放，减少滑膜积液，从根源缓解关节红肿、疼痛 组织修复 ：促进软骨细胞再生，修复受损的关节软骨与半月板，逆转关节退变 免疫调节 ：平衡关节内免疫微环境，延缓病情进展，降低复发风险 MSCs注射至关节腔内，可明显修复关节软骨，延缓病情进展[reference:6]。早期小规模试验已表明，将干细胞注射至受影响的关节内，不仅能减轻疼痛，还能帮助身体修复软骨[reference:7]。 不同于传统药物“治标不治本”的局限，干细胞治疗从细胞层面修复损伤，为膝骨关节炎患者提供了 “保膝”的全新可能 。 五、许可证≠上市，这一步到底意味着什么？ 很多人会问：拿到生产许可证，是不是就可以直接用了？ 答案是否定的。许可证上标注的 “仅限注册申报使用” 是关键：它不代表药品正式上市销售，也不代表医院可以大规模商业化开展，而是标志着这款产品的生产条件、合规能力，已经达到了注册申报的要求，从“研发中的产品”正式升级为“具备产业化基础的候选药物”。 同时，这款管线的商业化制备体系已完全成型，配套了约 4000平方米的细胞药品产业化基地 ，解决了干细胞药物量产的核心难题——批次一致性、细胞活性、效价测定、冻存复苏、运输交付等全流程标准化，这也是细胞药从实验室走向市场的核心门槛[reference:8]。 六、未来上市，大概要多少钱？ 关于大家最关心的价格问题，目前药品正式获批后的官方定价、支付方式、医院采购价格均未公开，但市场已有两个可参考的坐标： 海南博鳌乐城 ：膝关节炎相关脐带间充质干细胞治疗技术，备案标准为3.6万元/次（单膝） 广州南沙 ：相关企业负责人透露，同类膝骨关节炎干细胞药物未来价格有望控制在3万元左右 值得一提的是，我国首款获批上市的干细胞药物艾米迈托赛注射液，定价为 1.98万元/袋 ，远低于美国同类产品Ryoncil的19万美元/次，体现了中国在干细胞药物成本控制上的优势[reference:9]。 虽然上述价格坐标不能直接等同于未来上市药品的全国统一售价，但明确了一个趋势：这类产品未来未必是大众想象中的“天价”，但短期内也很难成为低价普惠型治疗，仍属于中高端医疗选择。 七、最后 这张《药品生产许可证》，不是终点，而是一个全新的起点。 如果说前些年行业更关注谁拿到了临床批件、谁做出了早期数据，那么现在，真正值得看的，是谁能把II期临床、生产许可、注册申报、质量体系、未来支付路径这整条链条完整打通。 对1亿多膝骨关节炎患者而言，这条消息最珍贵的意义在于： 干细胞药，正在从“遥不可及的前沿技术”，真正走向“可落地的产业化产品” ，而膝骨关节炎，极有可能成为这一轮产业化进程中，最先被验证的大病种之一。 至于最终上市时间、正式价格和临床可及性，我们仍需等待官方披露。真正决定这条管线价值的，从来不是一张证本身，而是它能否顺利走完上市前的最后几步，真正走进医院，造福千万患者。 如果你或家人正在被膝骨关节炎困扰，对这款干细胞药有什么疑问？欢迎在评论区聊聊你的经历。 转发给身边有需要的朋友，一起见证干细胞药从“前沿”走向“落地”。记得关注我们，获取更多前沿、靠谱的医疗科普。 免责声明 ：本文数据来源于公开资料及官方信息，仅供参考，不构成医疗建议。干细胞药物仍处于临床试验及上市前阶段，具体治疗方案请咨询正规医疗机构及专业医生。