Open label, Multicenter, Prospective Study to Characterize the Pharmacokinetics and Pharmacodynamics of Cufence (Trientine Dihydrochloride) and to Investigate the Efficacy and Safety in Wilson’s Disease Patients - TR-004 UNITED Study

开始日期2021-10-08

申办/合作机构-

CTR20211406 / Active, not recruiting临床2期

一项在心力衰竭和射血分数降低患者中评价 INL1（Trientine）效果的随机化、双盲、安慰剂对照研究

目的：主要目的：评价12周时INL1（盐酸曲恩汀）与安慰剂相比在NYHA 心功能分级Ⅱ～Ⅲ级伴左心室射血分数降低（LVEF<40%）的心力衰竭患者中的疗效。次要目的：评价INL1（盐酸曲恩汀）与安慰剂相比在心力衰竭患者中的安全性和耐受性。描述12周治疗期间INL1（盐酸曲恩汀）的药代动力学（PK）特征

开始日期2021-07-01

申办/合作机构

Frontage Laboratories, Inc.

[+2]

NCT04706429 / Active, not recruiting临床2期IIT

A Randomised, Double-blind, Placebo-controlled, Phase 2 Evaluation of the Efficacy and Mechanism of Trientine in Patients With Hypertrophic Cardiomyopathy

This research study has been designed to test whether a drug called trientine dihydrochloride (also called Cufence) reduces heart muscle thickening, improves exercise capacity, improves heart function and reduces abnormal heart rhythms in patients with hypertrophic cardiomyopathy (HCM). The study is also assessing how trientine works in HCM. Participants will be prescribed either trientine or placebo, for a period of 12 months.

开始日期2020-12-01

申办/合作机构

Manchester University NHS Foundation Trust

[+5]

100 项与盐酸曲恩汀相关的临床结果

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100 项与盐酸曲恩汀相关的转化医学

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100 项与盐酸曲恩汀相关的专利（医药）

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460

项与盐酸曲恩汀相关的文献（医药）

2024-12-01·Regenerative therapy

Research progress in stem cell therapy for Wilson disease

Review

作者: Gao, Ce ; Gong, Xin ; Liu, Aihui ; Meng, Xiangying ; Xiong, Xianlang ; Sun, Yi

Wilson disease (WD), also known as hepatolenticular degeneration, is an autosomal recessive disorder characterized by disorganized copper metabolism caused by mutations in the ATP7B gene. Currently, the main treatment options for WD involve medications such as d-penicillamine, trientine hydrochloride, zinc acetate, and liver transplantation. However, there are challenges that encompass issues of poor compliance, adverse effects, and limited availability of liver sources that persist. Stem cell therapy for WD is currently a promising area of research. Due to the advancement in stem cell directed differentiation technology in vitro and the availability of sufficient stem cell donors, it is expected to be a potential treatment option for the permanent correction of abnormal copper metabolism. This article discusses the research progress of stem cell therapy for WD from various sources, as well as the challenges and future prospects of the clinical application of stem cell therapy for WD.

2024-10-01·Drug Research

The Effect of Trientine on AlCl3-Induced Cognitive Dysfunction and Biochemical Changes in the Hippocampus of Rats

Article

作者: Mostafalou, Sara ; Mousavi-Nasab, Kian ; Amani, Mohammad

Abstract:

Cognitive impairments affect millions of people worldwide with an increasing prevalence. Research on their etiology and treatment is developing, nevertheless significant gaps remain. Trientine (TETA), as a copper chelator, has been shown to have beneficial effects in different human chronic diseases such as diabetic cardiomyopathy and neuropathy. Here, we examined the impact of TETA on AlCl3-induced neurocognitive dysfunctions and molecular changes in the hippocampus of rats.Thirty-six male Wistar rats (weighing 200–250 g) were randomly divided into four groups including control, TETA (100 mg/kg/day), AlCl3 (100 mg/kg/day), and AlCl3 (100 mg/kg/day)+TETA (100 mg/kg/day), and received chemicals by gavage for 30 days. At the end of the treatment, the open field maze, elevated plus maze, novel object recognition memory test, and shuttle box test were done. Then after, brain-derived neurotrophic factor (BDNF), glycogen synthase kinase-3 β (GSK-3β), acetylcholinesterase activity, oxidative stress markers, and inflammatory mediators were measured in the hippocampus.AlCl3 increased anxiety-like behaviors and impaired recognition and short-term memory. TETA was able to improve AlCl3-induced anxiety-like behaviors and short-term memory dysfunction. In the AlCl3-treated group, there was a significant increase in GSK-3β, oxidative stress, pro-inflammatory and pro-apoptotic markers, and decreased BDNF in the hippocampus. Co-administration of TETA was able to decrease lipid peroxidation, inflammation, GSK-3β, and acetylcholinesterase activity, and increase BDNF in the hippocampus compared with AlCl3-treated rats.It can be concluded that TETA was able to improve neurobehavioral and neurocognitive functions by alleviating oxidative stress, inflammation, and pro-apoptotic pathways leading to the normalization of BDNF and GSK-3β.

2024-05-01·ARQUIVOS DE NEURO-PSIQUIATRIA

Wilson disease: the diagnostic challenge and treatment outcomes in a series of 262 cases

Article

作者: Porta, Gilda ; Deguti, Marta Mitiko ; Cançado, Eduardo Luiz Rachid ; Araujo, Thiago Ferreira ; Barbosa, Egberto Reis ; Araujo, Fabiana Cordeiro ; Terrabuio, Débora Raquel Benedita

Abstract:

Background Wilson disease (WD) is an autosomal recessive disorder that leads to organ toxicity due to copper overload. Early diagnosis is complicated by the rarity and diversity of manifestations. Objective To describe the diagnostic features and response to treatment in our cohort of WD patients. Methods This was a retrospective analysis of 262 WD patients stratified by clinical presentation, complementary exams, ATP7B genotyping, and response to treatment. Results Symptoms occurred at an average age of 17.4 (7–49) years, and patients were followed up for an average of 9.6 (0–45) years. Patients presented mainly with hepatic (36.3%), neurologic (34.7%), and neuropsychiatric (8.3%) forms. Other presentations were hematologic, renal, or musculoskeletal, and 16.8% of the patients were asymptomatic. Kayser-Fleischer rings occurred in 78.3% of the patients, hypoceruloplasminemia in 98.3%, and elevated cupruria/24h in 73.0%, with an increase after D-penicillamine in 54.0%. Mutations of the ATP7B gene were detected in 84.4% of alleles. Brain magnetic resonance imaging showed abnormalities in the basal ganglia in 77.7% of patients. D-penicillamine was the first choice in 93.6% of the 245 patients, and 21.1% of these patients were switched due to adverse effects. The second-line therapies were zinc and trientine. The therapeutic response did not differ significantly between the drugs (p = 0.2). Nine patients underwent liver transplantation and 82 died. Conclusion Wilson disease is diagnosed at a late stage, and therapeutic options are limited. In people under 40 years of age with compatible manifestations, WD could be considered earlier in the differential diagnosis. There is a need to include ATP7B genotyping and therapeutic alternatives in clinical practice.

项与盐酸曲恩汀相关的新闻（医药）

2024-10-28

·GlobeNewswire-Health Care

Monopar Announces Agreement with Alexion, AstraZeneca Rare Disease For Late-Stage Wilson Disease Drug Candidate

Oct. 24, 2024 -- Monopar Therapeutics Inc. (Nasdaq: MNPR), a clinical-stage biotechnology company focused on developing innovative treatments for patients with unmet medical needs, today announced that it has entered into an agreement with Alexion, AstraZeneca Rare Disease for an exclusive worldwide license to ALXN-1840 (bis-choline tetrathiomolybdate), a drug candidate for Wilson disease that Alexion has progressed through a Phase 3 clinical trial that met its primary endpoint. Monopar will be responsible for all future global development and commercialization activities. Chandler D. Robinson, MD, Co-Founder and Chief Executive Officer of Monopar previously researched tetrathiomolybdate at the laboratory bench, published his results in Science, and helped launch a company around what became known as ALXN-1840. The focus was on Wilson disease. Dr. Robinson came to know the Wilson disease community well through his efforts. At the Wilson Disease Association’s request, he delivered the keynote address at their 2013 Annual Conference celebrating their 30th anniversary, sharing his experience helping advance ALXN-1840 from the laboratory bench to patients. In 2023, Alexion terminated the ALXN-1840 program in Wilson disease based on review of results from Phase II mechanistic trials and discussions with regulatory authorities. Chris Starr, PhD, Co-Founder and Executive Chair of Monopar said, “Upon the 2023 announcement, Chandler, due to his long history with the program and the continued high level of unmet medical need, was contacted by Wilson disease patients, executives and board members of the Wilson Disease Association, as well as physicians regarding the potential for Monopar to obtain rights to ALXN-1840. Due in no small measure to the testimonials Chandler received from clinical trial patients who reported benefit while on the drug for years, we decided that this was an opportunity Monopar needed to pursue, and it fits well with my rare disease drug development and commercialization background as well as Chandler’s background.” Dr. Starr also previously co-founded the orphan drug companies BioMarin and Raptor Pharma (acquired by Horizon Pharma, now a part of Amgen). Dr. Robinson commented, “Alexion has generated a substantial clinical data package on ALXN-1840, including a completed Pivotal Phase 3 clinical trial. The medical data gathered from Alexion’s clinical trials furthers our understanding of Wilson disease and stands to benefit this community.” Under the terms of the license agreement, Monopar will pay Alexion an upfront in the form of a cash payment and equity in Monopar. Future payments are based on tiered royalties on net sales and pre-determined regulatory and sales milestones. “We are excited to have Alexion and AstraZeneca as partners of Monopar as, in addition to Alexion’s work in Wilson disease, AstraZeneca maintains a significant presence in the radiopharma field, in which Monopar is committed to continue growing as Monopar recently announced positive human clinical data with our novel radiopharma program (link),” stated Andrew Cittadine, Chief Operating Officer of Monopar. Wilson disease is a rare and progressive genetic condition in which the body’s pathway for removing excess copper is compromised. 1 It affects one in 30,000 live births in the US.1 Over time this results in the build-up of toxic copper levels in the liver, brain, and other organs, leading to damage that greatly impacts a patient’s life. 1 Patients can develop a wide range of symptoms, including liver disease and/or psychiatric or neurological symptoms, such as personality changes, tremors and difficulty walking, swallowing or talking. 1 In some cases, the damage and loss of function may be irreversible. 1,2,3 ALXN-1840 (bis-choline tetrathiomolybdate) is an investigational once-daily, oral medicine in development for the treatment of Wilson disease. This novel molecule is designed to selectively and tightly bind and remove copper from the body’s tissues and blood. ALXN-1840 has been granted Orphan Drug Designation in the United States and orphan designation in the European Union for Wilson disease. The FoCus trial was a pivotal Phase 3, randomized, rater-blinded, multi-center clinical trial designed to evaluate the efficacy and safety of ALXN-1840 versus standard-of-care (SoC) in patients with Wilson disease aged 12 years and older. The primary endpoint assessed copper mobilization over 48 weeks, defined as daily mean AUEC (Area Under the Effect Curve) for dNCC (directly measured non-ceruloplasmin-bound copper). In the trial, 214 patients were enrolled in one of two cohorts on a 3:1 basis (treatment-experienced:treatment-naïve). Each cohort was then randomized 2:1 (ALXN1840:SoC). The first cohort enrolled 161 patients who received SoC (chelation therapy with penicillamine or trientine, zinc therapy or a combination of both chelation and zinc therapy) for more than 28 days and the second cohort enrolled 53 patients who were treatment-naïve or had received SoC for 28 days or less. The FoCus trial met its primary endpoint demonstrating three-times greater copper mobilization from tissues compared to the SoC arm (Least Square Mean Difference [LSM Diff] 2.18 µmol/L; p Monopar Therapeutics is a clinical-stage biotechnology company with late-stage ALXN-1840 for Wilson disease, and radiopharma programs including Phase 1-stage MNPR-101-Zr for imaging advanced cancers, and Phase 1a-stage MNPR-101-Lu and late preclinical-stage MNPR-101-Ac225 for the treatment of advanced cancers. For more information, visit: www.monopartx.com. References: 1. Patil, M., et al. (2013) J Clin Exp Hepatol, 3, 321-336. 2. Roberts, E.A., Schilsky, M.L. American Association for the Study of Liver D. (2008). Diagnosis and treatment of Wilson disease: An update. Hepatology, 47(6), 2089-2111. 3. European Association for the Study of the Liver. (2012). EASL clinical practice guidelines: Wilson’s disease. J Hepatol, 56(3), 671-685. The content above comes from the network. if any infringement, please contact us to modify.

临床结果引进/卖出孤儿药

2024-08-19

·PRNewswire

Behind The Mystery Unveils Treatment in Wilson Disease with Cuviror® by Orphalan

Wilson Disease, a rare, genetic disorder, hampers the body's ability to eliminate copper, leading to serious health issues. Cuvrior® (trientine tetrahydrochloride) is the only FDA-approved treatment in nearly 30 years. FORT LAUDERDALE, Fla., Aug. 19, 2024 /PRNewswire/ -- This special episode of Behind the Mystery features insightful discussions with a dedicated physician, an advocacy leader, and a CEO whose company has pioneered the first FDA approved treatment for Wilson Disease in nearly 30 years. Mandy, diagnosed with Wilson Disease at a young age, shares her journey living with the condition on the show, addressing the challenges faced by patients in managing their treatment regimens and the need for more convenient options. In addition, Hepatologist Dr. Cheung delves into the complexities of Wilson Disease, including its genetic origins, symptoms, and the critical importance of early diagnosis, management, and adherence to treatment. "I'm proud to be part of the 'Behind the Mystery' team, where we use our platform to raise awareness for the rare disease community. Our series underscored the immense challenges faced by patients, as well as the dedication of physicians, the pharmaceutical industry, and advocacy leaders to provide meaningful solutions to the community," said Senior Writer and Producer of Behind the Mystery, Brittany Cocilova. Our latest episode highlights Orphalan's commitment to developing the only FDA-approved therapy for Wilson Disease in nearly 30 years, showcasing the continued efforts to help patients living with and managing their rare disease. Hosts Montel Williams and Olga Villaverde spotlight the Wilson Disease Association's (WDA) efforts to support patients, improve awareness, and facilitate access to developments. Rhonda Rowland, VP of the WDA, discusses the evolution of treatment options and the organization's role in fostering partnerships between patients, physicians, and the pharmaceutical industry. "Orphalan has a tremendous commitment to educating not only the Wilson Disease community and the general public, but also physicians," says Rhonda Rowland, Vice President of Wilson Disease Association. "We've seen a partnership evolve between advocacy and the pharmaceutical industry which enables an open line of communication addressing the unmet needs in our community." Dr. Naseem Amin, CEO of Orphalan, details how Orphalan is addressing unmet needs in the patient community and emphasizes the importance of empathy in the pharmaceutical industry. CUVRIOR®, a treatment marketed by Orphalan, offers a twice-daily dosing and is a non-refrigerated option for long-term maintenance therapy for Wilson Disease. Behind the Mystery - Wilson Disease will air on Lifetime on August 19th and August 27th at 7:30 AM ET/PT and will remain on the website. By sharing these intimate narratives, the series aims to educate the public, inspire empathy, and advocate for better research, treatments, and support systems. INDICATION CUVRIOR is a copper chelator indicated for the treatment of adult patients with stable Wilson Disease who are de-coppered and tolerant to penicillamine. IMPORTANT SAFETY INFORMATION Do not use CUVRIOR if you are allergic to trientine or to any of the ingredients in CUVRIOR. Your Wilson's Disease symptoms could get worse when you start treatment. This could happen because too much copper is removed from the body in a short period of time. Your doctor may need to reduce your dose or stop CUVRIOR treatment. Copper deficiency may develop following treatment with CUVRIOR. Your doctor will do tests to monitor your urine and blood for copper. Iron deficiency may develop while taking CUVRIOR. If this happens, your doctor may tell you to take iron supplements for a limited time. Allergic reactions, such as a rash, have been reported with trientine, the active ingredient in CUVRIOR. If a rash or other allergic reaction occurs, stop taking CUVRIOR and get emergency medical help. The most common side effects are pain in the abdomen, change in bowel habits, rash, hair loss (alopecia), and mood swings. Take CUVRIOR at least 1 hour apart from any other oral medicine. Tell your doctor about all the medicines you're taking, including over-the-counter medicines, vitamins, and herbal supplements. Avoid taking supplements when taking CUVRIOR. Taking CUVRIOR with mineral supplements (e.g., iron, zinc, calcium, magnesium) can reduce the effectiveness of CUVRIOR. If iron supplementation is necessary, take CUVRIOR at least 2 hours before or 2 hours after taking iron; for other mineral supplements (e.g., zinc, calcium, magnesium), take CUVRIOR at least 1 hour before or 2 hours after. Do not start or stop any medicines while taking CUVRIOR without talking to your doctor. These are not all the possible side effects of CUVRIOR. You are encouraged to report negative side effects of prescription drugs. Contact Orphalan at 1-800-961-8320 or [email protected] or FDA at 1-800-FDA-1088 or medwatch. Please see full Prescribing Information at: About Behind the Mystery: Behind the Mystery™ is the first and only television series dedicated to raising awareness about rare and genetic diseases through powerful patient storytelling. Each episode delves into the personal journeys of individuals and families affected by these conditions, highlighting their struggles, triumphs, and daily realities. By sharing these intimate narratives, the series aims to educate the public, inspire empathy, and advocate for better research, treatments, and support systems. Behind the Mystery not only sheds light on the medical aspects of rare diseases but also celebrates the resilience and strength of the human spirit. Join us weekday mornings on Lifetime®. About Orphalan Orphalan is an international orphan drug development and commercialization company headquartered in Paris. Founded in 2011, the company develops innovative therapies for people living with rare and debilitating diseases. Our trientine tetrahydrochloride product for the treatment of Wilson disease is approved and available in more than 20 countries, branded as CUPRIOR® in EU, the UK, Saudi Arabia, Switzerland, Columbia and China, and as CUVRIOR® in the United States. For more information visit and follow us on LinkedIn. SOURCE Behind The Mystery

孤儿药上市批准

2024-08-09

·艾美达医药咨询

01 行业新闻丨北京上海开启创新药加速审批试点 8月2日，国家药监局发布关于同意在北京上海开展优化创新药临床试验审评审批试点的批复：经审核，同意在北京、上海开展优化创新药临床试验审评审批试点。7月31日，国家药监局发布《优化创新药临床试验审评审批试点工作方案》。方案提到，其工作目标是实现30个工作日内完成创新药临床试验申请审评审批，缩短药物临床试验启动用时。不过，纳入试点工作有严格的条件限制。试点项目范围是1类创新药的临床试验申请，细胞和基因治疗产品、疫苗产品等除外。申请人不受区域限制，但需在境内外至少获批过3个创新药临床试验申请，这就将不少药企排除在外. 此外，试点药物临床试验机构原则上为试点区域内的国家医学中心或者国家临床医学研究中心，承担试点项目相关专业已在药物临床试验机构备案管理信息平台备案，且在该专业领域已作为组长单位牵头完成过至少3项创新药临床试验。主要研究者作为组长单位的主要研究者也需主持完成过至少3项创新药临床试验。经确认的试点项目，药审中心会在受理临床试验申请后30个工作日内完成审评审批。审批通过的，申请人与试点机构应于临床试验申请获批后12周内启动临床试验（第一例受试者签署知情同意书），并在临床试验全过程实施风险管理。试点工作为期1年。试点期间，试点区域内至少完成10个品种的临床试验申请审评审批并启动临床试验。（健识局）丨440个药品通过2024年国家医保药品目录初步形式审查 8月7日，国家医保局对2024年国家医保药品目录初步形式审查结果进行公示，2024年7月1日9时至7月14日17时，共收到企业申报信息626份，涉及药品574个。经审核，440个药品通过初步形式审查。与2023年相比，申报药品数量有所增加。此次公示时间截至8月13日，国家医保局将根据公示期间收到的反馈意见，进一步核实相关信息，确定最终通过形式审查的药品名单，向社会公布，并推进专家评审、谈判竞价等后续工作。值得注意的是，肿瘤治疗领域，康方生物的卡度尼利单抗和依沃西单抗两款全球首创双抗药物入选；CAR-T细胞治疗产品方面，本次合源生物的纳基奥仑赛注射液、科济药业的泽沃基奥仑赛注射液、复星凯特的阿基仑赛注射液、药明巨诺的瑞基奥仑赛注射液通过了初审。本次医保目录初审名单中有不少罕见病药物，如盐酸曲恩汀片（肝豆状核变性）、替度格鲁肽（短肠综合征）、醋酸氟氢可的松片（先天性肾上腺皮质增生症和Addison病）等，显示出国家医保局对罕见病患者群体的关注和支持。同时，维拉苷酶α、氯巴占片、盐酸沙丙蝶呤片等纳入国家鼓励研发儿童药目录的药品也均在本次通过的初审名单中。（医药经济报） 02 企业动态丨恒瑞医药夫那奇珠单抗再获批3项临床 8月5日，中国国家药监局药品审评中心（CDE）官网公示，恒瑞医药夫那奇珠单抗注射液获得临床试验默示许可，拟开发治疗适合接受系统治疗或光疗的6至小于18岁儿童和青少年中重度斑块状银屑病患者。夫那奇珠单抗是恒瑞医药自主研发的一种靶向人IL-17A的重组人源化单克隆抗体，此前已经向NMPA递交多项适应症上市申请，包括治疗成人中重度斑块状银屑病适应症。丨海思科1类创新药获批临床 8月6日，CDE官网公示，海思科1类新药HSK44459片获得临床试验默示许可，拟开发治疗间质性肺疾病。根据海思科此前公告介绍，这是其自主研发的一个全新的治疗间质性肺疾病的药物，为一种高选择性的环核苷酸磷酸二酯酶4B（PDE4B）抑制剂。HSK44459片作为一种高选择性的PDE4B抑制剂，可以通过抑制炎性因子的渗出（主要）和成纤维细胞的增殖活化，达到抗炎和抗纤维化的作用。相较于传统泛靶点PDE4抑制剂，选择性PDE4B抑制剂预期在保留抗炎效果的基础上，能减少胃肠道、中枢神经等不良反应。（海思科）丨英矽智能抗肿瘤新药在美国获批临床 8月7日，英矽智能宣布，该公司自主研发的泛TEAD抑制剂ISM6331于2024年7月获得美国FDA的IND批件，用于治疗间皮瘤。这是该产品继今年6月获得针对间皮瘤适应症的孤儿药资格后，再次取得新进展。根据英矽智能新闻稿介绍，此次IND获批使英矽智能获得临床试验批件的创新分子数量达到9个。ISM6331的研发进程在人工智能的帮助下大大提速。临床前研究显示，该产品在多个细胞系中展现出广谱的抗肿瘤效果，在动物模型中低剂量起效，同时具备良好的安全性和ADMET特征。（英矽智能）丨GSK抗PD-1单抗在新加坡获批上市 8月7日，葛兰素史克（GSK）新加坡公司宣布，新加坡卫生科学管理局（HSA）已批准Jemperli（dostarlimab）的一项新适应症，即与卡铂和紫杉醇联合使用，随后Jemperli作为单一药物用于治疗原发性晚期或复发性子宫内膜癌患者，这些患者存在错配修复缺陷（dMMR）或高度微卫星不稳定性高（MSI-H）。此次获批使得Jemperli成为新加坡dMMR/MSI-H原发性晚期或复发性子宫内膜癌的首个一线免疫肿瘤治疗药物，可用于该类患者的早期治疗。Jemperli早前已获得新加坡HSA的批准，作为dMMR复发或晚期子宫内膜癌成人患者的单药治疗，这些患者在任何情况下在先前含铂方案中或之后进展，并且不适合治疗性手术或放疗。（葛兰素史克）丨精准生物CAR-T突破性疗法拟纳入优先审评 8月7日，CDE官网公示，重庆精准生物的普基仑赛注射液（pCAR-19B细胞自体回输制剂）的新药上市申请拟纳入优先审评，用于治疗3～21岁CD19阳性的复发或难治性B细胞急性淋巴细胞白血病患者，有望在中国加速获批上市。普基仑赛注射液用于儿童、青少年B细胞急性淋巴细胞白血病（B-ALL）适应症于2019年在中国获批开展注册临床研究。（精准生物）丨信达生物一款双抗ADC癌症1类新药申报临床 8月9日，CDE官网公示，信达生物1类新药IBI3001新药临床试验申请获得受理。公开资料显示，IBI3001是一款糖基化定点偶联、针对B7-H3和EGFR的双特异性ADC。该产品的临床前研究结果入选了2024年美国癌症研究协会年会Late-Breaking Research（突破性研究）环节。本次为该产品首次在中国申报IND。（信达生物）丨恒瑞医药5款创新药获批临床 8月9日，恒瑞医药发布公告称，该公司有多款创新药获批临床，其中包括多款抗肿瘤新药以及炎症性疾病、自身免疫性疾病新药。分别为：HRS-4508片，一款化药1类新药，为一种新型、高效、选择性的酪氨酸激酶抑制剂，通过抑制肿瘤细胞增殖发挥抗肿瘤作用；SHR-3821注射液，恒瑞医药自主研发的人源化抗体药物，拟用于治疗晚期恶性实体肿瘤，能够通过在肿瘤组织中特异性激活免疫细胞，发挥特异性抗肿瘤作用；SHR-7787注射液，为1类治疗用生物制品，通过诱导激活T细胞，使其发挥靶向杀伤恶性实体肿瘤细胞的作用；SHR-1819注射液获批开展用于结节性痒疹（PN）患者的2/3期临床试验；夫那奇珠单抗注射液获批开展3期临床试验，适应症为用于治疗适合接受系统治疗或光疗的6至小于18岁儿童和青少年中重度斑块状银屑病患者。（恒瑞医药）丨鼎成肽源生物TCR-T细胞产品获批临床 8月9日，CDE官网公示，鼎成肽源生物申报的1类创新药获批临床，拟用于治疗KRAS G12D突变阳性、基因型为HLA-A 11:01的晚期实体瘤患者。根据鼎成肽源生物早先新闻稿介绍，DCTY1102注射液为一款靶向HLA-A*11:01基因型、KRAS G12D突变的TCR-T细胞治疗产品，适应症为晚期胰腺癌、结直肠癌等恶性实体肿瘤。TCR-T类细胞药物的适用人群与HLA基因型密切相关。人类白细胞抗原（HLA）系统是免疫系统重要组成部分，能将抗原肽递呈给T细胞表面的受体（TCR）。HLA-A*11:01为中国人群中表型频率较高的HLA，占比超过20%。8月6日，东北制药集团股份有限公司（东北制药）宣布拟收购鼎成肽源70%的股权。(鼎成肽源生物) 丨石药集团1类CAR-T疗法获批临床 8月9日，CDE官网公示，石药集团中奇制药申报的1类新药SYS6020注射液获批临床，拟用于难治性活动性系统性红斑狼疮。公开资料显示，SYS6020为一款基于mRNA-LNP的嵌合抗原受体（CAR）-T细胞注射液，也是石药集团布局的首个细胞治疗在研产品。随着此次获批临床，它也将成为石药集团首个迈入自身免疫疾病临床开发的细胞疗法在研产品。SYS6020作为一款基于mRNA-LNP的细胞治疗产品，可通过表达可特异性识别BCMA抗原的CAR，进而靶向识别患者体内BCMA阳性的细胞并对其进行杀灭。与传统CAR-T产品相比，SYS6020具有细胞活率高、CAR阳性率高、无基因组整合引起的致瘤风险，以及细胞因子风暴（CRS）等副作用低的优点。（石药集团） *声明：本文不构成任何投资建议，仅出于传递更多信息之目的。 ------------------------------ 「长按」二维码添加小达「进群」与更多行业伙伴共探市场前沿资讯艾美达医药咨询艾美达（北京）医药信息咨询有限公司，成立于2014年4月，是一家专业的医药行业咨询服务提供商。公司致力于将产业政策研究与真实世界的数据挖掘深度结合，洞悉行业政策对市场的影响，通过专业的研究提供前瞻性的市场分析，为企业产品上市后的市场准入提供整体解决方案。

细胞疗法疫苗免疫疗法加速审批临床研究

100 项与盐酸曲恩汀相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
D00736	盐酸曲恩汀	A16AX	DB06824

研发状态

批准上市

10 条最早获批的记录,

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适应症	国家/地区	公司	日期
肝豆状核变性	美国	Bausch Health Americas, Inc.	1985-11-08

未上市

10 条进展最快的记录,

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适应症	最高研发状态	国家/地区	公司	日期
肥大型心肌病	临床2期	英国	Univar Solutions, Inc.	2020-12-01
心脏衰竭	临床2期	美国	Frontage Laboratories, Inc.	2019-12-30
心脏衰竭	临床2期	美国	成都因诺生物医药科技有限公司	2019-12-30
心脏衰竭	临床2期	美国	CopMed，Inc.	2019-12-30
心肌梗塞	临床2期	中国	成都因诺生物医药科技有限公司	-
心肌缺血	临床2期	中国	成都因诺生物医药科技有限公司	-

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临床结果

适应症

分期

评价

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研究	分期	人群特征	评价人数	分组	结果	评价	发布日期


NCT02426905 (EASL2019)	临床4期	肝豆状核变性	52	Trientine dihydrochloride	構鏇蓋積簾願淵衊憲顧(選齋築膚繭艱獵廠鑰簾) = 8 (15.4%) patients reported serious TEAEs 壓夢窪鹹衊範遞憲鑰鹹 (顧築鏇築願範簾膚艱簾 ) 更多	-	2019-04-12