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Clinical data highlight survival benefits and improvement in functional motor outcomes associated with treatment with doxecitine (dC) and doxribtimine (dT) - an investigational pyrimidine nucleoside therapy - in people living with thymidine kinase 2 deficiency (TK2d).1,2,3 Additional patient experience data emphasize the profound physical challenges and severe psychological strain that come with living with TK2d, and the heavy emotional and physical burden experienced by caregivers.4,5 Across all study populations, pyrimidine nucleoside and/or nucleotide therapy was generally well tolerated.1,2,3 TK2d is an ultra-rare, life-threatening, genetic mitochondrial disease with no currently approved treatment options.6,7,8,9,10,11 BRUSSELS, March 19, 2025 /PRNewswire/ -- UCB, a global biopharmaceutical company, today announced positive data from studies involving its investigational pyrimidine nucleoside therapy, doxecitine (dC) and doxribtimine (dT), in people living with thymidine kinase 2 deficiency (TK2d), at this year's MDA Clinical and Scientific Conference, Dallas, Texas, March 16-19, 2025. The data show that in individuals with TK2d who were aged 12 years or less when their symptoms first appeared, treatment with pyrimidine nucleoside and/or nucleotide therapy significantly decreased mortality and increased survival.2,3 In addition, treatment also improved functional outcomes irrespective of age of onset, including retaining or regaining motor milestones, and helped stabilize ventilatory and feeding support use.1,2,3 Across all study populations, pyrimidine nucleoside and/or nucleotide therapy was generally well tolerated with diarrhea being the most common treatment emergent adverse event.1,2,3 Thymidine kinase 2 deficiency is an ultra-rare, life-threatening, genetic mitochondrial disease characterized by progressive and severe muscle weakness (myopathy), which can impact the ability to walk, eat, and breathe independently.6,7,8,9,10 "There are no approved therapies or international clinical guidelines for the management of TK2d, therefore we are very excited to share this data with the medical community at MDA," said Donatello Crocetta, Chief Medical Officer at UCB. "The data highlight the positive impact this investigational treatment could have on the lives of people living with this debilitating and life-threatening condition." Data from participants treated in the doxecitine (dC) and doxribtimine (dT) clinical program were pooled from retrospective and prospective sources and a company-supported expanded access program (EAP). Data from untreated participants were pooled from literature reviews of case series and reports, and a retrospective chart review study. Subgroups were stratified by age of TK2d symptom onset categories and reported for participants with age of TK2d symptom onset ≤12 years and >12 years. Doxecitine and doxribtimine is currently under regulatory review by US and EU regulatory authorities. The safety and efficacy have not been established, and doxecitine and doxribtimine has not been approved by the US Food and Drug Administration (FDA) nor the European Medicines Agency (EMA). Key findings from the studies Improvement in survival: Among people living with TK2d with an age of symptom onset 12 years or less, treatment with nucleoside and/or nucleotide therapy reduced risk of death by 92–94% (hazard ratio=0.06–0.08; p<0.0001) and 87–95% (hazard ratio=0.05–0.13; p<0.0001) in the time from symptom onset and starting treatment, respectively.3 Enhanced functional outcomes: Among people living with TK2d who were aged 12 years or less when their symptoms first appeared, following treatment with pyrimidine nucleoside and/or nucleotide therapy, 75.0% (30/40) regained at least one previously lost motor milestone, with 22.5% (9/40) regaining four or more motor milestones.2 In addition, ventilatory support dependency decreased, with 16.1% (5/31) of patients reducing usage time and 16.1% (5/31) discontinuing ventilatory support altogether after treatment.2 Safety profile: Across all study populations, pyrimidine nucleoside and/or nucleotide therapy was generally well tolerated. Diarrhea (range from 84.6%-90.9%) was the most reported treatment-emergent adverse event.1,2,3 Impact on quality of life on individuals and caregivers: In addition to the data presented on nucleoside and/or nucleotide therapy, patient experience data presented highlight the debilitating physical impacts and severe psychological strain associated with living with TK2d, as well as its impact on caregivers. Many individuals reported the 'extreme' impact the condition has on their quality of life including walking, breathing and eating/swallowing difficulties. Caregivers reported that the constant demands of caregiving and minimal support/respite caused persistent stress and emotional burnout.4,5 Key data presented at MDA About thymidine kinase 2 deficiency (TK2d) Thymidine kinase 2 deficiency is an ultra-rare, life-threatening, genetic mitochondrial disease characterized by progressive and severe muscle weakness (myopathy), which can impact the ability to walk, eat, and breathe independently.6,7,8,9,10 It is estimated that the worldwide prevalence of TK2d is 1.64 [0.5, 3.1] cases per 1,000,000 people.12 The age of TK2d symptom onset is categorized as ≤12 years and >12 years.6,7,8 TK2d profoundly affects multiple health, physical, quality-of-life, and psychosocial domains, as children struggle to achieve developmental milestones or lose them, and adults lose functional independence with challenges in breathing, eating, and walking.11,13 About doxecitine and doxribtimine Doxecitine and doxribtimine is a nucleoside therapy that supports mitochondrial DNA replication resulting in increased or stabilized mitochondrial function in patients with TK2d. Pre-clinical data suggest the primary mechanism of action of doxecitine and doxribtimine is the incorporation of nucleosides deoxycytidine (dC) and deoxythymidine (dT) into skeletal muscle mitochondrial deoxyribonucleic acid (mtDNA), which has the potential to restore mitochondrial DNA copy number and improve skeletal muscle function in patients with TK2d.14,15,16 In the U.S., the application has been granted a priority review, Breakthrough Therapy Designation and Rare Pediatric Disease Designation.17,18 For further information, contact UCB: US Rare Diseases Communications Daphne Teo T: +1 770.880.7655 [email protected] Global Communications Nick Francis T: +44 7769 307745 [email protected] Corporate Communications, Media Relations Laurent Schots T +32.2.559.92.64 [email protected] Investor Relations Antje Witte T +32.2.559.94.14 [email protected] About UCB UCB, Brussels, Belgium () is a global biopharmaceutical company focused on the discovery and development of innovative medicines and solutions to transform the lives of people living with severe diseases of the immune system or of the central nervous system. With more than 9 000 people in approximately 40 countries, the company generated revenue of € 6.1 billion in 2024. UCB is listed on Euronext Brussels (symbol: UCB). Follow us on Twitter: @UCB_news Forward-looking statements This press release may contain forward-looking statements including, without limitation, statements containing the words "believes", "anticipates", "expects", "intends", "plans", "seeks", "estimates", "may", "will", "continue" and similar expressions. These forward-looking statements are based on current plans, estimates and beliefs of management. All statements, other than statements of historical facts, are statements that could be deemed forward-looking statements, including estimates of revenues, operating margins, capital expenditures, cash, other financial information, expected legal, arbitration, political, regulatory or clinical results or practices and other such estimates and results. By their nature, such forward-looking statements are not guarantees of future performance and are subject to known and unknown risks, uncertainties and assumptions which might cause the actual results, financial condition, performance or achievements of UCB, or industry results, to differ materially from those that may be expressed or implied by such forward-looking statements contained in this press release. Important factors that could result in such differences include: the global spread and impact of COVID-19, changes in general economic, business and competitive conditions, the inability to obtain necessary regulatory approvals or to obtain them on acceptable terms or within expected timing, costs associated with research and development, changes in the prospects for products in the pipeline or under development by UCB, effects of future judicial decisions or governmental investigations, safety, quality, data integrity or manufacturing issues; potential or actual data security and data privacy breaches, or disruptions of our information technology systems, product liability claims, challenges to patent protection for products or product candidates, competition from other products including biosimilars, changes in laws or regulations, exchange rate fluctuations, changes or uncertainties in tax laws or the administration of such laws, and hiring and retention of its employees. There is no guarantee that new product candidates will be discovered or identified in the pipeline, will progress to product approval or that new indications for existing products will be developed and approved. Movement from concept to commercial product is uncertain; preclinical results do not guarantee safety and efficacy of product candidates in humans. So far, the complexity of the human body cannot be reproduced in computer models, cell culture systems or animal models. The length of the timing to complete clinical trials and to get regulatory approval for product marketing has varied in the past and UCB expects similar unpredictability going forward. Products or potential products, which are the subject of partnerships, joint ventures or licensing collaborations may be subject to differences disputes between the partners or may prove to be not as safe, effective or commercially successful as UCB may have believed at the start of such partnership. UCB's efforts to acquire other products or companies and to integrate the operations of such acquired companies may not be as successful as UCB may have believed at the moment of acquisition. Also, UCB or others could discover safety, side effects or manufacturing problems with its products and/or devices after they are marketed. The discovery of significant problems with a product similar to one of UCB's products that implicate an entire class of products may have a material adverse effect on sales of the entire class of affected products. Moreover, sales may be impacted by international and domestic trends toward managed care and health care cost containment, including pricing pressure, political and public scrutiny, customer and prescriber patterns or practices, and the reimbursement policies imposed by third-party payers as well as legislation affecting biopharmaceutical pricing and reimbursement activities and outcomes. Finally, a breakdown, cyberattack or information security breach could compromise the confidentiality, integrity and availability of UCB's data and systems. Given these uncertainties, you should not place undue reliance on any of such forward-looking statements. There can be no guarantee that the investigational or approved products described in this press release will be submitted or approved for sale or for any additional indications or labelling in any market, or at any particular time, nor can there be any guarantee that such products will be or will continue to be commercially successful in the future. UCB is providing this information, including forward-looking statements, only as of the date of this press release and it does not reflect any potential impact from the evolving COVID-19 pandemic, unless indicated otherwise. UCB is following the worldwide developments diligently to assess the financial significance of this pandemic to UCB. UCB expressly disclaims any duty to update any information contained in this press release, either to confirm the actual results or to report or reflect any change in its forward-looking statements with regard thereto or any change in events, conditions or circumstances on which any such statement is based, unless such statement is required pursuant to applicable laws and regulations. Additionally, information contained in this document shall not constitute an offer to sell or the solicitation of an offer to buy any securities, nor shall there be any offer, solicitation or sale of securities in any jurisdiction in which such offer, solicitation or sale would be unlawful prior to the registration or qualification under the securities laws of such jurisdiction. References: Scaglia F, et al. Survival and Functional Outcomes in Patients with Thymidine Kinase 2 Deficiency Aged >12 Years at Symptom Onset Who Received Pyrimidine Nucleos(t)ides. . Accessed February 2025. Garone C, et al. Functional Outcomes in Patients with Thymidine Kinase 2 Deficiency Aged ≤12 Years at Symptom Onset Who Received Pyrimidine Nucleos(t)ide Therapy. . Accessed February 2025. Hirano M, et al. Survival Analyses in Patients with Thymidine Kinase 2 Deficiency Aged ≤12 Years at Symptom Onset Who Received Pyrimidine Nucleos(t)ide Therapy. . Accessed February 2025. Yeske P, et al. Patients' Lived Experience of Thymidine Kinase 2 Deficiency (TK2d): Results from the Assessment of TK2d Patient Perspectives (ATP) Study. . Accessed February 2025. Yeske P, et al. Burden and impact of caring for those with thymidine kinase 2 deficiency (TK2d): results from the Assessment of TK2d Patient Perspectives (ATP) study. . Accessed February 2025. Berardo A, et al. Advances in Thymidine Kinase 2 Deficiency: Clinical Aspects, Translational Progress, and Emerging Therapies. J Neuromuscul Dis. 2022;9(2):225-35. Wang J, et al. TK2-Related Mitochondrial DNA Maintenance Defect, Myopathic Form. 2018. In: Adam MP, et al. GeneReviews® [Internet]. Seattle (WA): University of Washington, Seattle; 1993–2025. Garone C, et al. Retrospective natural history of thymidine kinase 2 deficiency. J Med Genet. 2018;55(8):515-21. Domínguez-González C, et al. Late-onset Thymidine Kinase 2 Deficiency: A Review of 18 cases. Orphanet J Rare Dis. 2019;14(1):100. National Institutes of Health. TK2-related mitochondrial DNA depletion syndrome, myopathic form. . Accessed February 2025. U.S. FDA TK2d Patient Listening Session. . Accessed February 2025. Ma Y. Prevalence Estimation of Thymidine Kinase 2 Deficiency: An Ultra-Rare Autosomal Recessive Mitochondrial Disease. Poster presented at: ISPOR Europe; 2023, 12-15 November; Denmark. Amtmann D, et al. The impact of TK2 deficiency syndrome and its treatment by nucleoside therapy on quality of life. Mitochondrion. 2023;68:1-9. Lopez-Gomez C, et al. Deoxycytidine and Deoxythymidine Treatment for Thymidine Kinase 2 Deficiency. Ann Neurol. 2017;81(5):641-52. Lopez-Gomez C, et al. Bioavailability and cytosolic kinases modulate response to deoxynucleoside therapy in TK2 deficiency. EBioMedicine. 2019;46:356-367. National Library of Medicine. A Study of the Efficacy and Safety of MT1621 in Thymidine Kinase 2 (TK2) Deficiency (Treatment naïve). . Accessed February 2025. U.S. FDA. Orphan Drug Designations and Approvals. . Accessed February 2025. UCB Press Release. . Accessed February 2025. ©2025 UCB, Inc., Smyrna, GA 30080. All rights reserved. US-MT-2500041 SOURCE UCB WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

看点 view 本周，2025药品监管新信号释放，中纪委发文直指医药腐败，知名中药企业遭重罚；药品市场迎变局，48个品种集采结果出炉，221个药品价格“跳水”，医药大省集采400个产品拟中选；企业大新闻，创新药“一哥”递表港交所，2家Biotech新品合计大卖超36亿美元，科伦1类新药再下一城......医药行业一周大事件，精彩不容错过！ 米内一周TOP榜 米内数据 齐鲁、科伦大丰收！3个超10亿品种首仿定了 26个补血中成药火了！东阿阿胶、太极上榜 36款清热解毒中成药亮了！独家品种暴涨184% 第十一批国采12个品种备战，石四药、华海蓄力 53个独家中成药申请保护，20亿大品种领跑 药企动态 天士力1类新药进攻2300亿市场 16亿注射剂，科伦获批了 石四药拿下6亿畅销药 58亿大品种，湖南药企获批了 扬子江1类新药来袭，猛攻4000亿市场 重磅会议！释放药品监管新信号 国家药监局官网消息称，1月7日-8日，全国药品监督管理工作会议在北京举行。会议对今年药品监管工作作了七个方面具体部署。其中，在安全保障方面：严格落实药品安全责任，提升药品检查效能，加强重点环节、重点品种、重点区域监管；在产业发展方面：加大对研发创新支持力度，提高研发创新的质量和效率，提升审评审批质效，扩大对外开放合作；在中药监管方面：优化中药审评审批体系，促进中药生产经营便利化，加强中药质量安全监管等。（国家药监局） 临床用药大目录曝光！超1400亿市场波澜再起 为进一步规范新型抗肿瘤药物临床应用，国家卫健委于1月7日发布了《新型抗肿瘤药物临床应用指导原则（2024年版）》，涉及145个常用抗肿瘤药，包括帕博利珠单抗、替雷利珠单抗、信迪利单抗等临床大品种。米内网数据显示，在中国三大终端六大市场（统计范围详见本文末），抗肿瘤药（化+生）2023年销售额超1400亿元，2024上半年以10.42%的增速保持增长。（国家卫健委） 184个药品被鼓励集采？独家品种“霸屏” 近日，一份名为《鼓励地方集采的集采空白大额品种（184个）》的清单在医药圈流传。据悉，184个坊间流出的鼓励集采药品中，独家品种有114个，占比超过60%；仅2家企业获批的品种有22个，占比约11.9%；其余占比不足三成的品种获批企业数均在3家及以上。具体品种方面，贝伐珠单抗、曲妥珠单抗2款畅销抗肿瘤药俨然在列，其2023年在中国公立医疗机构终端销售规模分别超90亿元和60亿元。（趣学术） 中纪委直指医药腐败，揭秘收受回扣大案 1月8日，中纪委发布了《以正风肃纪反腐实际成效赢得党心民心》一文，直指医药等重点领域，强调深化整治各类腐败问题。同日，为期3天的“第二十届中纪委第四次会议”结束并发布公报称，2025年医药、国企仍是反腐的关键领域，制定新时代廉洁文化建设三年行动计划（2025-2027年），讲好中国反腐败故事等。对此，中纪委转载了央视专题片《反腐 为了人民》之《揭开腐败隐身衣》，其中列举了广东省茂名市高州市人民医院原院长王茂生收受回扣的案例。王茂生从医近40年，2013年凭借其专业背景和清廉作风，被委任为高州市人民医院的党委书记兼院长。不料其后利欲熏心，经相关纪委监委查实，王茂生任职期间收受回扣总额高达2亿余元。所有赃款赃物将被如数追缴。（中央纪委国家监委）延伸阅读：据不完全统计，2024年有超350名医药卫生系统干部被查，其中180余名为医院书记或院长。全年通报被查的医药卫生系统干部覆盖范围广，从医疗机构到药企、医学院校，从乡镇卫生院到知名三甲医院，从基层干部到厅官均有涉及，遍布全国30个省市。 1月10日，广州药品集团采购平台发布《关于公示广东联盟阿哌沙班片等药品集中带量采购拟中选结果的通知》显示，由广东、安徽、福建、江西、重庆等13个省份组成的大联盟集采结果出炉。据梳理，此次联盟集采共计48个品种、592个产品拟中选，不乏阿哌沙班口服常释剂型、二甲双胍口服常释剂型、紫杉醇注射剂等临床常用药；从企业层面上看，石药集团、华海药业、倍特药业、扬子江药业等拟中选产品均在9个及以上。（广州药品集团采购平台） 医药大省集采400个产品拟中选 1月9日，江苏省医保局发布《江苏第五轮药品集中带量采购公告（四）》，对拟中选结果进行公示，公示时间为2025年1月9日至11日。据悉，本轮集采共有286家企业的400个产品拟中选，其中222家企业仅1个产品拟中选，占比约为78%。经对比，集采目录中的西药替比夫定口服常释剂、卡替洛尔滴眼剂，以及中成药仙灵骨葆片拟流标。（江苏省医保局） 医保目录重新修正，2个品种保留国谈属性 1月8日，国家医保局发布关于对《国家基本医疗保险、工伤保险和生育保险药品目录（2024年）》部分药品信息进行修正的公告。公告指出，经核查，多种微量元素注射液(Ⅲ)在2024年6月30日前仍为独家药品；阿伐替尼片因原研药品地产化，增加了国内持有人批文，应视为独家药品。根据相关规则，上述2个品种不符合转入常规乙类条件，仍保留在谈判药品部分，其医保支付标准、备注等不变。（国家医保局） 冲刺“A+H”！创新药“一哥”递表港交所 1月6日，港交所披露消息，创新药“一哥”恒瑞医药正式递交上市申请，拟赴港上市，未来有望实现“A+H”两地上市。据其招股书显示，恒瑞目前已有20余款创新药获批上市，管线涵盖逾90款候选新分子实体创新药、30多款处于关键性临床研究及更后期阶段的在研创新药，共开展约400项临床试验，涉及超20项海外临床试验。同日，迈威生物也向港交所递交招股书，拟香港主板挂牌上市。资料显示，迈威生物成立于2017年，专注于肿瘤、与年龄相关疾病的创新药研发，拥有抗体发现技术平台、ADC技术平台、TCE双抗/三抗技术平台，在研管线包括Nectin-4 ADC、B7H3 ADC、CDH17 ADC、IL-11抗体等。（米内网整理） 瞄准超$85亿市场，科伦1类新药再下一城 1月7日，科伦药业发布公告，其控股子公司科伦博泰提交的1类新药博度曲妥珠单抗（A166）上市申请获NMPA受理，拟用于治疗既往至少接受过一种抗HER2治疗的HER2阳性不可切除或转移性乳腺癌。在此之前，该药已于2023年5月申报上市，适应症为既往经二线及以上抗HER2治疗失败的HER2阳性不可切除的局部晚期、复发或转移性乳腺癌。据悉，这是科伦博泰首个提交上市申请的ADC药物。（公司公告）米内数据：截至目前，全球共有16款ADC新药（不含生物类似药）获批上市，包括罗氏的恩美曲妥珠单抗、荣昌制药的维迪西妥单抗、科伦药业的芦康沙妥珠单抗等，其2023年全球销售额合计超过85亿美元。 最高86%！221个药品主动降价 1月6日，云南省药品集采平台发布《关于企业申请降价的公示》。据梳理，此次共有77家企业、221个药品提交了降价申请，基药品种占比接近四成。其中，注射用伏立康唑、非布司他片等降幅较大，均超过70%，而江苏长泰药业的奥氮平氟西汀胶囊价格下降仅0.001%，为本次降幅最小的产品。（米内网整理） 4款新药合计大卖超36亿美元 1月10日，先为达宣布与Verdiva达成一项全球授权协议。根据协议，Verdiva将负责口服伊诺格鲁肽（GLP-1受体激动剂）、口服胰淀素受体激动剂及皮下注射胰淀素受体激动剂在大中华区和韩国以外的全球范围内开发、生产和商业化权利；先为达将获得合计约24.7亿美元的授权总金额，以及产品商业化后销售额提成。1月7日，映恩生物宣布与Avenzo达成一项全球战略合作协议，协议内容显示，映恩生物将授予Avenzo在全球范围内不包括大中华区）开发、生产和商业化DB-1418（EGFR/HER3双抗ADC），同时也将获得相应的首付款及里程碑付款，金额总计达12亿美元。（医药观澜）2025年以来部分创新药出海情况 注：标红为总金额超10亿美元的交易，未披露交易金额用-表示 生产销售劣药！2家中药企业遭重罚 1月8日，四川省药监局发布的两则行政处罚书显示：成都鹤祥天药业有限公司因生产销售劣药土鳖虫（批号：230201），被处以没收违法所得和罚款合计约23.9万元；四川同创康能药业有限公司因生产销售劣药款冬花（批号：230301），被处以没收违法所得约6.59万元，并处罚金10万元。上述两家中药企业被合计罚没超过40万元。（四川省药监局） 一头部药企副总裁官宣离职 1月7日，礼来中国宣布一项重要人事变动——现任礼来中国神经科学事业部副总裁蒋逸飞，决定离开礼来，寻求外部发展机会，最后工作日为下周二（1月14日）。此前，蒋逸飞曾在礼来中国担任南非和撒哈拉以南非洲市场营销总监、糖尿病产品事业部副总裁等多个高管职务，特别是在糖尿病领域，他成功推动了度拉糖肽等重磅产品的上市与持续增长。截至消息官宣之日，蒋逸飞的继任者尚未公布。（医药代表） 注：米内网《中国三大终端六大市场药品竞争格局》，统计范围是：城市公立医院和县级公立医院、城市社区中心和乡镇卫生院、城市实体药店和网上药店，不含民营医院、私人诊所、村卫生室，不含县乡村药店；上述销售额以产品在终端的平均零售价计算。投稿及报料请发邮件到872470254@qq.com稿件要求详询米内微信首页菜单栏商务及内容合作可联系QQ：412539092 【分享、点赞、在看】点一点不失联哦