Givinostat Hydrochloride

罕见病信息网隆重推出《壹周罕见药闻》这一独具特色的品牌栏目。该栏目在每周一更新，以呈现上周罕见病领域的最新消息并同步近期重要活动预告。我们以更高端、更权威的视角，为您剖析最新、最热门的罕见病信息。 罕药快讯 01 BLA审评延期，重磅生物药上市推迟至2027年 10月2日，诺和诺德称FDA告知其血友病A药物denecimig的BLA审评仍在进行中。 该药2025年9月递交申请，原预计2026年三季度获批，延迟系生产基地检查后整改所致；FDA未发现临床疗效或安全性数据缺陷。 Denecimig为皮下注射FVIIIa模拟双特异性抗体，支持每月、每两周或每周一次预防治疗，已于2026年9月获EMA CHMP积极意见。公司预计2027年上半年在美国上市，不影响2026年财务展望。 02 罕见儿科疾病资格认定落地，基因替代疗法再迎新进展 10月1日，MavriX Bio宣布，其在研AAV基因疗法MVX-220用于治疗天使综合征获FDA授予罕见儿科疾病资格认定（RPDD）。该药此前已获孤儿药资格认定和快速通道资格认定，I/II期临床试验已完成首位患者给药。 MVX-220采用新型AAVhu.68衣壳，通过单次枕大池注射将功能性UBE3A基因递送至脑神经元，有望实现长期表达并带来持久临床获益。该药由基因治疗先驱Jim Wilson团队开发，获天使综合征治疗基金会资助后授权MavriX Bio推进临床，"基金会资助+产业转化"模式为罕见病基因治疗提供了可借鉴路径。 天使综合征是一种因UBE3A基因缺陷引发的神经发育障碍性疾病，已纳入我国《第一批罕见病目录》。 03 罕见生长障碍治疗迎新进展，长效IGF-1疗法获批临床 9月30日，CDE官网显示，金赛药业1类新药GenSci155注射液临床试验申请获默示许可，拟定适应症为治疗原发性胰岛素样生长因子-1缺乏症（PIGFD）。 GenSci155是金赛药业基于长效缓释多肽平台Duratide™自主研发的长效化脂肪酸偶联重组人IGF-1类似物，属治疗用生物制品1类，拟皮下注射。该药通过脂肪酸链修饰与白蛋白可逆结合，延长体内半衰期，减少血药浓度波动，有望降低低血糖等副作用。临床前研究显示，其给药频率可达每2-3天一次，较短效重组人IGF-1更便捷，有助于提升患者依从性。 PIGFD又称生长激素不敏感综合征，是一种罕见生长障碍性疾病，表现为非生长激素缺乏性矮小伴低IGF-1血症。 04 关键III期达到主要终点，上市申请正式受理 9月30日，罗氏宣布美国#FDA 已受理其fenebrutinib的新药上市申请（NDA），并授予#优先审评资格，拟用于治疗复发型多发性硬化症（RMS）和原发进展型多发性硬化症（PPMS）。Fenebrutinib是罗氏开发的一款具有中枢神经系统渗透性的可逆、非共价BTK抑制剂，对BTK的选择性显著高于对其他激酶。 该药物可抑制B细胞和小胶质细胞活化，这种双重抑制作用有望减少多发性硬化症的活动性及残疾进展，从而回应患者群体在延缓残疾进展方面的关键未满足需求。 05 基因疗法随访数据未达预期，股价暴跌37% 9月29日，基因治疗公司uniQure公布了亨廷顿病在研疗法ifezuntirgene inilparvovec（AMT-130）的I/II期临床试验最新4年随访数据。 受核心终点结果未达市场预期的冲击，公司股价当日收盘暴跌约37%，早盘开盘最大跌幅一度触及40%，股价下探至24美元以下。 06 这款罕见病新药获FDA批准 9月28日，Egetis Therapeutics公司宣布，美国FDA批准EMCITATE®用于成人及儿童MCT8缺乏症患者外周甲状腺毒症的治疗。这是FDA批准的首款用于治疗MCT8缺乏症的药物。MCT8缺乏症又称Allan-Herndon-Dudley综合征，为X连锁遗传病，患者以男性为主。致病基因为X染色体上的SLC16A2，其编码的MCT8蛋白负责将甲状腺激素转运进入大脑。 07 罕见病在研药斩获日本孤儿药资格认定 9月28日，JCR制药宣布，日本厚生劳动省授予其在研药givinostat#孤儿药资格认定。givinostat由Italfarmaco S.p.A.联合Telethon与意大利Duchenne Parent Project合作研发，为口服HDAC抑制剂，可调节DMD患者肌肉内过度活化的HDAC。该药已在多个国家地区获批，用于6岁及以上DM D患者，暂未在日本上市。 08 暂停半年后重启！罕见病管线迎反转 9月28日，中外制药正式宣布，将接手此前罗氏终止开发的抗潜伏肌抑素抗体emugrobart（研发代号：GYM329），全面重启该药物针对脊髓性肌萎缩症（SMA）的后续研发工作，为长期受疾病困扰的SMA患者带来全新的治疗希望。2026年3月，罗氏曾公开宣布终止 emugrobart 针对#脊髓性肌萎缩症（SMA）和#面肩肱型肌营养不良症（FSHD）的临床开发，相关研发工作一度全面暂停。 09 线粒体靶向新药最新进展 前不久，Mighty Therapeutics公司宣布启动MYTX-255的IND-enabled研究，MYTX-255是一种新型、处于研究阶段的线粒体靶向治疗候选药物。公司计划在2027年底前提交MYTX-255的IND申请。 10 首批患者给药！罕见肾病疗法推进 Eloxx Pharmaceuticals生物制药公司前不久宣布，EXACT研究（一项评估exaluren在无义突变型Alport综合征（NMAS）患者中的疗效的2b期临床试验）的首两名患者已给药。 EXACT研究是一项随机、安慰剂对照、延迟启动的全球性2b期临床试验，旨在评估exaluren对24例由COL4A3、COL4A4或COL4A5无义突变引起的无义突变型Alport综合征（NMAS）患者的安全性和有效性。 11 穿越血脑屏障！小分子新药获欧盟上市许可 西班牙Minoryx医药公司与其在欧洲的合作方Neuraxpharm集团宣布，欧盟委员会（EC）已授予Nezglyal（leriglitazone）特殊情况下的上市许可，用于治疗2～12岁、磁共振成像（MRI）显示脑部病灶无钆增强且神经功能评分（NFS）为0或1的男性脑型肾上腺脑白质营养不良（cALD）患者。 新闻稿指出，Nezglyal是欧盟首个获批用于cALD的药物治疗选择。 罕圈动态 01 760万美元投入罕见病！老药新用已逆转病理 美国国立卫生研究院（NIH）近日向密歇根州立大学人类医学院与Corewell医疗体系授予总金额760万美元的开创性专项资助，支持多胺病（polyaminopathies ）这一罕见遗传病领域的研究。本次项目由两位跨领域学者共同担任首席研究员：一位是密歇根州立大学儿科与人类发育系研究方向副教授André S. Bachmann博士，另一位是Corewell Health 旗下海伦·德沃斯儿童医院医学遗传学与基因组学主任Caleb Bupp博士，双方将依托各自的学术与临床资源，系统推进多胺病发病机制的深度解析。 02 12岁，她身上有14种病 🌍地球Online｜10月3日播报📍坐标：英国👤人物：12岁女孩（由母亲讲述）她出生时便带着多重诊断：先天性膈疝、复杂先天性心脏病（其中一项缺陷极为罕见）、生长发育迟缓、自闭症、因严重吸收不良和喂养困难而长期依赖静脉营养，同时还伴有肾囊肿、肝腺瘤、语言发育迟缓、运动障碍（动作测试仅处于0.5百分位）、腿部挛缩、血小板和白细胞反复波动，以及银屑病。基因检测显示她携带两个意义未明的基因变异：一个与Coffin-Siris综合征（第五指综合征）相关，另一个与歌舞伎综合征相关。 03 不仅仅是生存，更是改变世界的力量 🌍地球Online｜10月2日播报📍 坐标： 美国·威斯康星州议会大厦👤 人物：Aidan近日，在美国威斯康星州麦迪逊市的州议会大厦内，罕见病患者Aidan现身“Beyond the Diagnosis（超越诊断）”艺术展现场。在他身旁展示的，是他童年时期的肖像画。这幅画作已在全美巡展近十年，旨在支持更多罕见病儿童被社会看见。Aidan患有一种名为外胚层发育不良（Ectodermal dysplasia, ED）的罕见病。这是一种先天性遗传病，以毛发、汗腺、牙齿、指甲等外胚层来源的组织发育不全或形态功能缺陷为主要特征。 04 心脏骤停三次后，她“重生”了 🌍地球Online播报｜10月1日 📍坐标：西班牙·洛格罗尼奥 👤人物：Graciela 2025年10月确诊多发性骨髓瘤，同时合并罕见病——淀粉样变性。受疾病影响，她的张口幅度不能超过1.5厘米，舌头活动受限，并伴有吞咽困难。 2025年11月，她接受了心脏移植手术。手术前，她曾三次发生心脏骤停：第一次发生在化疗期间；第二次是在气管切开并连接ECMO时，当时心功能已不足34%；第三次发生在心脏移植当天。 05 这个病想带走她，还没成功 🌍地球Online每日播报｜9月30日📍坐标：英国·南威尔士
👤人物：Jessica👧年龄：21岁Jessica回到了家。从医院出来并不容易——她在医院住了整整一个月，医生一度担心她挺不过来。之后又在床上躺了两周。她挺过了肺炎，挺过了败血症，然后重新站了起来。 06 新西兰发布首个罕见疾病战略实施计划 9月28日消息，新西兰卫生部正式发布《2026-27年罕见疾病战略实施计划》，这是该国2024年7月出台《新西兰罕见疾病战略2024-2034》后推出的首份落地执行文件。 07 25岁因罕见病离世，家人筹款76万支持研究 25岁的英国女孩 Daisy Catterall 去年因罕见病去世。她走之前，“把日子过到了极致”——这是妈妈 Rachel 回忆她时说的话。Daisy 患的是噬血细胞性淋巴组织细胞增生症（HLH， hemophagocytic lymphohistiocytosis），一种罕见的免疫系统疾病：本应保护身体的免疫系统被过度激活，反过来攻击自身的器官和组织。 她走后，她的亲友参加了最强泥人挑战赛（Tough Mudder）募集资金，筹款总额超过85,000英镑（约合人民币 76 万元），全部用于伦敦大学学院医院（UCLH）的 HLH 研究。这笔钱帮助研究团队拿下了一笔科研拨款——研究者说，没有这笔筹款，“这笔拨款就不可能实现”。 08 天后复出的第十天，僵人综合征等来了第一个官方资格认定 9月12日，巴黎近郊丰盛竞技场（原拉德芳斯体育馆）。58岁的#席琳·迪翁 时隔六年重新登台，约3万人到场，演出近两个半小时，后续还有25场。第一首歌开口不久，她就哭了。全球超900万人参与门票抽签，数小时内售罄——观众等的不是《My Heart Will Go On》的大合唱，而是见证一位被疾病困住的歌者，选择重新站上舞台。 活动预告 01【第二期重磅启动】赋能守护·罕见病关爱行动最高4800元救助金开放申请！ 罕见病家庭常面临“诊断难、用药贵、持续治疗负担重”的多重挑战。为切实减轻罕见病患者的用药经济负担，蔻德罕见病中心、阿里巴巴公益和阿里健康公益共同发起“赋能守护·罕见病关爱行动”患者援助第二期项目，计划通过搭建“企业帮一点、公益捐一点、患者付一点”的多方共付机制，继续为经济困难的罕见病患者提供援助。 2026年5月1日至2027年3月31日期间，家庭经济相对困难，并通过罕见病关爱卡模式（详见下 06 援助申请流程）购买过对应罕见病治疗必要且经过罕见病关爱平台认证的药品、低蛋白食品、保健品、器械等的罕见病患者均可申请。 申请次数：同一患者同一疾病，或同一患者不同疾病，本项目周期内最多可提交两次申请，申请金额合计不超过4800元。 02【患者招募】基因治疗 α1 - 抗胰蛋白酶缺乏症（AATD）项目报名啦！ 目前，一项YOLT-202新型基因治疗AATD的安全性和有效性临床研究工作正在进行。YOLT-202 给药修复 SERPINA1 等位基因中的单碱基突变，从而恢复血液中的功能性野生型AAT蛋白，同时减少异常Z-AAT蛋白的存在，以治疗AATD 相关的肺病和/或肝病。这将大大改善受影响患者的生活质量，减少相关并发症的发生，提高患者生存率。该项目已被医院伦理委员会批准用于临床试验。 如果您符合以下标准，则有可能参与此项研究： 1、年龄≥18岁且≤75岁的男性或女性（含边界值）； 2、诊断为AATD且通过基因检测确认为PiZZ突变纯合子； 3、血液中总AAT水平小于11 μM或等效的mg/dL蛋白质； 4、接受增强治疗的患者必须愿意在入组接受给药前至少6周以及在研究期间停止增 强治疗（除非临床上提示需要）。 在参加本研究之前，您将接受额外的检查，经过筛选，最终由研究医生判断您是否符合全部标准。 03 基因治疗苯丙酮尿症(PKU)项目报名来啦！ 苯丙酮尿症（PKU）是一种主要由苯丙氨酸羟化酶（PAH）基因突变引发的代谢性罕见病。因PAH突变，导致无PAH酶蛋白的生成或者酶活性显著下降甚至丧失，导致苯丙氨酸(Phe)在体内蓄积。如果不及时控制，长期可导致严重神经认知功能损伤。 尽管目前已有包括药物与饮食控制在内的治疗方法，但依然存在以下挑战。终身依从性治疗负担沉重，饮食控制难以坚持。血Phe水平难以维持在安全范围，神经损伤风险依旧存在。 目前， 一项GS1168静脉输注治疗苯丙酮尿症的安全性和有效性临床研究正在进行。GS1168是一种基因治疗药物，适用于治疗患有PKU的成年患者。与传统的替代疗法相比，基因治疗药物可以单次给药即有可能达到长期疗效。 它是一种具有肝脏靶向性的重组AAV载体， 在肝特异性启动子的调控下，驱动PAH基因的表达。临床前研究已初步证明本药物的有效性和安全性。 如果您符合以下标准，则有可能参与此项研究： 1.  年龄在18~55岁。 2.  PAH基因突变导致的苯丙酮尿症，现有治疗不佳如饮食控制。 3.  12个月内至少两次血苯丙氨酸浓度≥600μmol/L，其中一次在6个月内。  推 荐 阅 读

- JR-141 is a potentially transformative therapy designed to deliver enzymes to the brain and peripheral tissues to treat neuronopathic and somatic symptoms of Hunter syndrome through intravenous administration - JCR is conducting a global Phase III clinical trial evaluating JR-141 in Hunter syndrome - Italfarmaco will exclusively commercialize JR-141 in the United States, Europe, and Latin America upon regulatory approval MILAN & HYOGO, Japan--(BUSINESS WIRE)-- Italfarmaco S.p.A. (“Italfarmaco”) and JCR Pharmaceuticals Co., Ltd. (TSE 4552; “JCR”) today announced an exclusive license agreement to develop and commercialize JR-141 in the United States, Europe, and Latin America (“licensed territories”) upon regulatory approval. JR-141 is a blood-brain barrier (BBB) penetrating enzyme replacement therapy currently in Phase III ( NCT04573023 ) evaluation as a treatment of Hunter syndrome (also known as mucopolysaccharidosis type II or MPS II). Approved and launched in Japan in 2021 as IZCARGO™ (INN: pabinafusp alfa), JCR and Italfarmaco seek to obtain regulatory approval for JR-141 from the U.S. Food and Drug Administration (FDA), the European Medicines Agency (EMA), the U.K. Medicines and Healthcare products Regulatory Agency (MHRA), and the Brazilian Health Regulatory Agency (ANVISA). Following regulatory approvals by the FDA, EMA, MHRA and ANVISA, Italfarmaco will commercialize and distribute the product in the licensed territories, while JCR will retain responsibility for manufacturing JR-141 upon marketing authorizations. JCR will receive upfront, milestone and royalty payments, in addition to revenues associated with product supply. JR-141 is a next-generation recombinant fusion protein of an antibody against the human transferrin receptor and iduronate-2-sulfatase, the enzyme that is missing or malfunctioning in individuals with Hunter syndrome. JR-141 was developed using J-Brain Cargo ® , JCR’s proprietary BBB-penetration technology. J-Brain Cargo ® is engineered to transport the therapeutic enzyme across the BBB to directly reach the brain and address both the somatic and neuronopathic symptoms of the disease, which can lead to progressive cognitive decline. This agreement expands the relationship between JCR and Italfarmaco, which entered into an exclusive license agreement in December 2025 for the commercialization of givinostat in Japan, a treatment for Duchenne muscular dystrophy, together with a strategic partnership agreement for rare disease therapies. “We are pleased to enter into this strategic agreement with Italfarmaco and collaborate with an ideal global commercialization partner as we work toward our goal of delivering JR-141 to people living with Hunter syndrome around the world,” said Hiroyuki Sonoda, Ph.D., President and Chief Scientific Officer of JCR Pharmaceuticals. “Italfarmaco has expertise in developing and commercializing therapies worldwide, and we look forward to working with Italfarmaco to make JR-141 available to patients outside Japan as soon as possible. This agreement extends our commitment to the partnership with Italfarmaco, developing therapies for rare and genetic diseases for patients around the globe.” “This agreement marks an important milestone in the relationship between Italfarmaco and JCR Pharmaceuticals and reflects a shared commitment to advancing innovative therapies for people living with rare and genetic diseases,” said Francesco Di Marco, Chief Executive Officer of Italfarmaco Group. “By bringing together JCR’s pioneering expertise in blood-brain barrier technologies and Italfarmaco’s global capabilities in rare disease development and commercialization, we are creating a strong foundation to accelerate innovation for underserved patient communities.” “Building on the success of our existing collaboration in Duchenne muscular dystrophy, this agreement further strengthens our partnership and reinforces our shared ambition to be a leading force in the rare and genetic disease field,” said Antonio Nardi, Vice President and Head of Business & Portfolio Development of Italfarmaco. “Alongside our trusted partner, we look forward to advancing our mission of delivering transformative treatments to the patients and families who need them most.” The upfront payment under this agreement has been incorporated into JCR’s consolidated earnings forecast for the fiscal year ending March 31, 2027. About the J-Brain Cargo ® Platform Technology JCR Pharmaceuticals has developed a proprietary blood-brain barrier (BBB)-penetrating technology, J-Brain Cargo ® , to bring biotherapeutics into the central nervous system (CNS). The first drug developed based on this technology is IZCARGO™ (INN: pabinafusp alfa), which is approved in Japan for the treatment of Hunter syndrome, a lysosomal storage disorder (LSD). With J-Brain Cargo ® , JCR seeks to address the unresolved clinical challenges of LSDs by delivering the enzyme to both the body and the brain. About Hunter Syndrome (Mucopolysaccharidosis Type II, or MPS II) Hunter syndrome (mucopolysaccharidosis type II, or MPS II) is an X-linked recessive lysosomal storage disorder caused by a deficiency of iduronate-2-sulfatase, an enzyme that breaks down complex carbohydrates called glycosaminoglycans (GAGs, also known as mucopolysaccharides) in the body. Hunter syndrome, which affects an estimated 2,000-3,000 individuals worldwide (according to JCR research), gives rise to a wide range of somatic and neurological symptoms. The current standard of care for Hunter syndrome is enzyme replacement therapy, which does not address the central nervous system-related symptoms of this lysosomal disease. About JR-141 JR-141 (pabinafusp alfa) is a recombinant fusion protein of an antibody against the human transferrin receptor and iduronate-2-sulfatase, the enzyme that is missing or malfunctioning in subjects with Hunter syndrome. It incorporates J-Brain Cargo ® , JCR’s proprietary blood-brain barrier (BBB)-penetrating technology, to cross the BBB through transferrin receptor-mediated transcytosis, and its uptake into cells is mediated through the mannose-6-phosphate receptor. This novel mechanism of action is expected to make pabinafusp alfa effective against the central nervous system (CNS) symptoms of Hunter syndrome. In pre-clinical trials, JCR has confirmed both high-affinity binding of pabinafusp alfa to transferrin receptors and passage across the BBB into neuronal cells. In addition, JCR has confirmed enzyme uptake in various brain tissues. The company has also confirmed a reduction of substrate accumulation in the CNS and peripheral organs in an animal model of Hunter syndrome. 1,2 In several clinical trials of pabinafusp alfa, JCR obtained evidence of reducing heparan sulfate (HS) concentrations in the cerebrospinal fluid (CSF), a biomarker for assessing effectiveness against CNS symptoms; these results were consistent with those obtained in pre-clinical studies. 3 Clinical studies have also demonstrated the positive effects of pabinafusp alfa on CNS symptoms. 4,5,6 Pabinafusp alfa was approved in Japan by the Ministry of Health, Labour and Welfare and marketed since May 2021 under the brand name “IZCARGO™ I.V. Infusion 10mg.” It was also approved in the United Arab Emirates (UAE) in 2026. Important Safety Information INDICATION: IZCARGO™ is indicated for the treatment of mucopolysaccharidosis type II (MPS II), which is also known as Hunter syndrome. IZCARGO™ is approved in Japan and the United Arab Emirates (UAE). CONTRAINDICATION: IZCARGO™ is contraindicated in patients with a history of anaphylactic shock to its components. WARNINGS AND PRECAUTIONS: Warnings Since serious anaphylaxis and shock may occur with use of IZCARGO™, adequate emergency measures should be made ready for execution before initiation of administration, and the patient should be closely monitored during and after the administration. If a serious infusion associated reaction (IAR) occurs, administration of IZCARGO™ should be discontinued, and appropriate actions should be taken. When IZCARGO™ is administered to patients with severe respiratory failure or acute respiratory disease, an IAR may lead to acute exacerbation of symptoms. A patient’s condition should be closely monitored, and appropriate actions should be taken as needed. Precautions for Use IZCARGO™ is a protein medicinal product and may cause anaphylactic shock, for which close monitoring is required. If any signs of anaphylaxis are noted, discontinue the infusion, and take appropriate actions. Considering the onset of such symptoms, emergency measures should be made ready for execution. IZCARGO™ may cause IARs such as headache, chills, syncope, fatigue, dizziness, pyrexia, rash, erythema, urticaria, or other symptoms. If an IAR occurs, reduce the rate or temporarily discontinue the infusion, and initiate appropriate drug treatment (e.g., corticosteroids, antihistamines, antipyretic analgesics, anti-inflammatory drugs) or emergency procedures (e.g., oxygen administration, securing of airway, adrenaline administration). Premedication with antihistamines, corticosteroids, etc., should be considered for the subsequent infusion of IZCARGO™. ADVERSE REACTIONS: The most commonly reported adverse reactions were pyrexia and urticaria. Prescribing information, indications, contraindications, warnings, and adverse reactions may differ by jurisdiction. Healthcare professionals should consult the locally approved prescribing information. About Italfarmaco S.p.A. Founded in 1938 in Milan, Italy, Italfarmaco is a private global pharmaceutical company that has led the successful development and approval of many pharmaceutical products around the world. The Italfarmaco group has operations in more than 90 countries through directly controlled or affiliated companies. The company is a leader in pharmaceutical research, product development, production and commercialization with proven success in multiple therapeutic areas including oncology, gynecology, neurology, cardiovascular disease and rare diseases. Italfarmaco’s rare disease unit includes programs in Duchenne muscular dystrophy, Becker muscular dystrophy, amyotrophic lateral sclerosis and polycythemia vera. About JCR Pharmaceuticals Co., Ltd. JCR Pharmaceuticals Co., Ltd. (TSE 4552) is a global specialty pharmaceutical company that develops treatments that go beyond rare diseases to solve the world’s most complex healthcare challenges. We continue to build upon our 50-year legacy in Japan while expanding our global footprint into the U.S., Europe, and Latin America. We improve patients’ lives by applying our scientific expertise and unique technologies to research, develop, and deliver next-generation therapies. Our approved products in Japan include therapies for the treatment of growth disorder, MPS II (Hunter syndrome), Fabry disease, acute graft-versus host disease, and renal anemia. Our investigational products in development worldwide are aimed at treating rare diseases including MPS I (Hurler, Hurler-Scheie and Scheie syndrome), MPS II, MPS IIIA and B (Sanfilippo syndrome type A and B), and more. Our core values – Putting people first, Forging our own path, Always advancing, and Committed to excellence – mean that the work we do benefits all our stakeholders, including partners, patients and employees. We strive to expand the possibilities for patients while accelerating medical advancement at a global level. For more information, please visit JCR’s global website: . Cautionary Statement Regarding Forward-Looking Statements This document contains forward-looking statements that are subject to known and unknown risks and uncertainties, many of which are outside our control. Forward-looking statements often contain words such as “believe,” “estimate,” “anticipate,” “intend,” “plan,” “will,” “would,” “target” and similar references to future periods. All forward-looking statements regarding our plans, outlook, strategy and future business, financial performance and financial condition are based on judgments derived from the information available to us at this time. Factors or events that could cause our actual results to be materially different from those expressed in our forward-looking statements include, but are not limited to, a deterioration of economic conditions, a change in the legal or governmental system, a delay in launching a new product, impact on competitors’ pricing and product strategies, a decline in marketing capabilities relating to our products, manufacturing difficulties or delays, an infringement of our intellectual property rights, an adverse court decision in a significant lawsuit and regulatory actions. This document involves information on pharmaceutical products (including those under development). However, it is not intended for advertising or providing medical advice. Furthermore, it is intended to provide information on our company and businesses and not to solicit investment in securities we issue. Except as required by law, we assume no obligation to update these forward-looking statements publicly or to update the factors that could cause actual results to differ materially, even if new information becomes available in the future. References: Sonoda, et al. A blood-brain-barrier-penetrating anti-human transferrin receptor antibody fusion protein for neuronopathic mucopolysaccharidosis II. Mol. Ther. 2018; 26(5): 1366-1374. Morimoto, et al. Clearance of heparin sulfate in the brain prevents neurodegeneration and neurocognitive impairment in MPS II mice. Mol. Ther. 2021; 29(5): 1853-1861. Okuyama, et al. Iduronate-2-sulfatase with Anti-human Transferrin Receptor Antibody for Neuropathic Mucopolysaccharidosis II: A Phase 1/2 Trial. Mol Ther. 2020; 27(2): 456-464. Okuyama, et al. A Phase 2/3 Trial of Pabinafusp Alfa, IDS Fused with Anti-Human Transferrin Receptor Antibody, Targeting Neurodegeneration in MPS-II. Mol Ther. 2021; 29(2): 671-679. Giugliani, et al. Iduronate-2-sulfatase fused with anti-human transferrin receptor antibody, pabinafusp alfa, for treatment of neuronopathic and non-neuronopathic mucopolysaccharidosis II: Report of a phase 2 trial in Brazil. Mol Ther. 2021; 29(7): 2378-2386. Giugliani, et al. Enzyme Replacement Therapy with Pabinafusp Alfa for Neuronopathic Mucopolysaccharidosis II; an Integrated Analysis of Preclinical and Clinical Data. Int. J. Mol. Sci. 2021, Volume 22, Issue 20, 10938. Contacts Media enquiries - Italfarmaco: Gretchen Schweitzer / Adolfo Luna | +49 (0) 151 626 74395 | italfarmaco@trophic.eu Other enquiries - Italfarmaco: Samantha Parker | Patient Advocacy and Communications Lead | RDEnquiries@italfarmacogroup.com Investors & Media - JCR Pharmaceuticals Co., Ltd.: Corporate Communications ir-info@jp.jcrpharm.com