A Phase 1, Sequential Drug Interaction Study to Evaluate the Effect of CYP3A4 and P-glycoprotein Inhibition by Itraconazole or Induction by Rifampicin on the Pharmacokinetics of Ecopipam and Its Metabolites in Healthy Subjects

This is a single center, 2-Part, Phase 1, open-label, fixed-sequence, drug-drug interaction study designed to compare the PK of ecopipam when administered alone and in combination with itraconazole (Part 1) or rifampicin (Part 2) in healthy subjects.

开始日期2024-01-18

申办/合作机构

Emalex Biosciences, Inc.初创企业

[+2]

NCT06021522 / Recruiting临床3期

A Multicenter, Open-Label, Study to Evaluate the Long-term Safety of Ecopipam Tablets in Children, Adolescents and Adults With Tourette's Disorder

The primary objective of this study is to evaluate the long-term safety and tolerability of ecopipam tablets in children (greater than or equal to [>=] 6 and less than [<] 12 years of age), adolescents (>=12 and <18 years of age), and adults (>=18 years of age) with Tourette's Syndrome (TS).

开始日期2023-08-16

申办/合作机构

Emalex Biosciences, Inc.初创企业

NCT05841160 / Completed临床1期

A Randomized, Double-Blind, Placebo- and Positive Controlled, Crossover, Thorough QT/QTc Study to Evaluate the Effects of a Therapeutic and Supratherapeutic Dose of Ecopipam (EBS-101) on Cardiac Repolarization in Healthy Subjects

THOROUGH QT/QTc STUDY TO EVALUATE THE EFFECTS OF ECOPIPAM (EBS-101) ON CARDIAC REPOLARIZATION

开始日期2023-03-15

申办/合作机构

Emalex Biosciences, Inc.初创企业

[+2]

100 项与依考匹泮相关的临床结果

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100 项与依考匹泮相关的转化医学

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100 项与依考匹泮相关的专利（医药）

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214

项与依考匹泮相关的文献（医药）

2023-09-26·The Journal of neuroscience : the official journal of the Society for Neuroscience

The role of retinal dopamine D1 receptors in ocular growth and myopia development in mice.

Article

作者: Ziheng Shu ; Kaijie Chen ; Qiongsi Wang ; Honglin Wu ; Yangfeifei Zhu ; Ruikang Tian ; Wenjun Yan ; Qin Huang ; Chunlan Zhang ; Weiwei Xiong ; Jia Qu ; Xiangtian Zhou ; Furong Huang

Dopamine is a key neurotransmitter in the signaling cascade controlling ocular refractive development, but the exact role and site of action of dopamine D1 receptors (D1Rs) involved in myopia remains unclear. Here, we determine whether retinal D1Rs exclusively mediate the effects of endogenous dopamine and systemically delivered D1R agonist or antagonist in the mouse form deprivation myopia (FDM) model. Male C57BL/6 mice subjected to unilateral FDM or unobstructed vision were divided into four groups: one non-injected and three groups that received intraperitoneal injections of either a vehicle, D1R agonist SKF38393 (18 and 59 nmol/g) or D1R antagonist SCH39166 (0.1 and 1 nmol/g). The effects of these drugs on FDM were further assessed in Drd1-knockout (Drd1-KO), retina-specific conditional Drd1-knockout (Drd1-CKO) mice and corresponding wild-type littermates. In the visually unobstructed group, neither SKF38393 nor SCH39166 affected normal refractive development, whereas myopia development was attenuated by SKF38393 and enhanced by SCH39166 injections. In Drd1-KO or Drd1-CKO mice, however, these drugs had no effect on FDM development, suggesting that activation of retinal D1Rs is pertinent to myopia suppression by the D1R agonist. Interestingly, the development of myopia was unchanged by either Drd1-KO or Drd1-CKO, and neither SKF38393 or SCH39166 injections, nor Drd1-KO, affected the retinal or vitreal dopamine and the dopamine metabolite DOPAC levels. Effects on axial length were less marked than effects on refraction. Therefore, activation of D1Rs, specifically retinal D1Rs, inhibits myopia development in mice. These results also suggest that multiple dopamine-D1R mechanisms play roles in emmetropization and myopia development.Significance StatementWhile dopamine is recognized as a "stop" signal that inhibits myopia development (myopization), the location of the dopamine D1 receptors (D1Rs) that mediate this action remains to be addressed. Answers to this key question are critical for understanding how dopaminergic systems regulate ocular growth and refraction. We report here the results of our study showing that D1Rs are essential for controlling ocular growth and myopia development in mice, and identifying the retina as the site of action for dopaminergic control via D1Rs. These findings highlight the importance of intrinsic retinal dopaminergic mechanisms for the regulation of ocular growth and suggest specific avenues for exploring the retinal mechanisms involved in the dopaminergic control of emmetropization and myopization.

2023-04-01·Pharmacology research & perspectives

Ultra-high-field pharmacological functional MRI of dopamine D1 receptor-related interventions in anesthetized rats.

Article

作者: Yuka Kimura ; Shunsuke Nakazawa ; Kantaro Nishigori ; Yuki Mori ; Junji Ichihara ; Yoshichika Yoshioka

The dopamine D1 receptor (D1R) is associated with schizophrenia, Parkinson's disease, and attention deficit hyperactivity disorder. Although the receptor is considered a therapeutic target for these diseases, its neurophysiological function has not been fully elucidated. Pharmacological functional MRI (phfMRI) has been used to evaluate regional brain hemodynamic changes induced by neurovascular coupling resulting from pharmacological interventions, thus phfMRI studies can be used to help understand the neurophysiological function of specific receptors. Herein, the blood oxygenation level-dependent (BOLD) signal changes associated with D1R action in anesthetized rats was investigated by using a preclinical ultra-high-field 11.7-T MRI scanner. PhfMRI was performed before and after administration of the D1-like receptor agonist (SKF82958), antagonist (SCH39166), or physiological saline subcutaneously. Compared to saline, the D1-agonist induced a BOLD signal increase in the striatum, thalamus, prefrontal cortex, and cerebellum. At the same time, the D1-antagonist reduced the BOLD signal in the striatum, thalamus, and cerebellum by evaluating temporal profiles. PhfMRI detected D1R-related BOLD signal changes in the brain regions associated with high expression of D1R. We also measured the early expression of c-fos at the mRNA level to evaluate the effects of SKF82958 and isoflurane anesthesia on neuronal activity. Regardless of the presence of isoflurane anesthesia, c-fos expression level was increased in the region where positive BOLD responses were observed with administration of SKF82958. These findings demonstrated that phfMRI could be used to identify the effects of direct D1 blockade on physiological brain functions and also for neurophysiological assessment of dopamine receptor functions in living animals.

2023-01-11·Pediatrics

Ecopipam for Tourette Syndrome: A Randomized Trial.

Article

作者: Donald L Gilbert ; Jordan S Dubow ; Timothy M Cunniff ; Stephen P Wanaski ; Sarah D Atkinson ; Atul R Mahableshwarkar

BACKGROUND AND OBJECTIVES:

All US Food and Drug Administration-approved medications for Tourette syndrome are antipsychotics, and their use is limited by the risk of weight gain, metabolic changes, and drug-induced movement disorders. Several small trials suggest that ecopipam, a first-in-class, selective dopamine 1 receptor antagonist, reduces tics with a low risk for these adverse events. This trial sought to further evaluate the efficacy, safety, and tolerability of ecopipam in children and adolescents with moderate to severe Tourette syndrome.

METHODS:

This was a multicenter, randomized, double-blind, placebo-controlled, phase 2b trial. Subjects aged ≥6 to <18 years with a baseline Yale Global Tic Severity Score Total Tic Score of ≥20 were randomly assigned 1:1 to ecopipam (n = 76) or placebo (n = 77). The primary endpoint was mean change over 12 weeks in the Yale Global Tic Severity Score Total Tic Score. The Clinical Global Impression of Tourette Syndrome Severity was the secondary endpoint. Safety and tolerability were evaluated at each study visit.

RESULTS:

Total tic scores were significantly reduced from baseline to 12 weeks in the ecopipam group compared with placebo (least squares mean differences -3.44, 95% confidence interval -6.09 to -0.79, P = .01). Improvement in Clinical Global Impression of Tourette Syndrome Severity was also greater in the ecopipam group (P = .03). More weight gain was seen in subjects assigned to placebo. No metabolic or electrocardiogram changes were identified. Headache (15.8%), insomnia (14.5%), fatigue (7.9%), and somnolence (7.9%) were the most common adverse events.

CONCLUSIONS:

Among children and adolescents with TS, ecopipam reduces tics to a greater extent than placebo, without observable evidence of common antipsychotic-associated side effects.

项与依考匹泮相关的新闻（医药）

2023-10-05

·BioSpace

Emalex Biosciences Releases Results of 12-Month Open-Label Extension Study for Tourette Syndrome Candidate

Long-term safety data collected in pediatric patients treated with investigational drug ecopipam; durable treatment effect reported with no evidence of tachyphylaxis CHICAGO--(BUSINESS WIRE)-- An open-label extension study of 121 pediatric patients with Tourette syndrome evaluated the safety pro the investigational drug ecopipam for up to 12-months of treatment. No new safety concerns were identified based on an evaluation of adverse events, physical exams, vital signs, laboratory results and safety scales, according to Emalex Biosciences. A durable effect was also observed over the course of the study based on evaluation of the patients’ motor and vocal tics, which were assessed every three months. The findings were first presented at the International Congress of Parkinson’s Disease and Movement Disorder Society in Copenhagen and will be highlighted at the Child Neurology Society Annual Meeting this week in Vancouver. Patients in the open-label study previously completed a Phase 2b clinical trial involving more than 150 children and adolescents that found ecopipam significantly reduced the total number of motor and vocal tics compared to placebo, according to a study published in the journal Pediatrics. In this open-label study, ecopipam was generally well tolerated, with 33.1% (40/121) of patients reporting treatment-related adverse reactions (ARs). The most common ARs were anxiety (6.6%), insomnia (5.8%), and somnolence (5.8%). One patient experienced a serious adverse reaction (obsessive thoughts) and 11.6% (14/121) of patients discontinued the study due to adverse events. In addition, patients did not have observable evidence of excessive weight gain or metabolic side effects commonly reported with antipsychotic agents, which are often used to treat patients with Tourette syndrome. Ecopipam is a first-in-class dopamine-1 receptor antagonist. The antipsychotic agents currently approved to treat Tourette syndrome target the dopamine-2 receptor. Ecopipam is the lead candidate for Emalex Biosciences, a clinical-stage biopharmaceutical company founded by Paragon Biosciences. Emalex is currently conducting a Phase 3 clinical trial of ecopipam for the treatment of Tourette syndrome at more than 90 sites in North America and Europe. Tourette syndrome is a chronic, childhood-onset neurodevelopmental disorder characterized by motor and vocal tics. The condition is associated with both increased mortality and significant morbidity. In the majority of individuals, Tourette syndrome substantially impacts day-to-day physical and social function. “The fact that most patients who completed the Phase 2b study asked to participate in the open-label extension points to the unmet medical needs for people with Tourette syndrome. The 12-month data supports the continued development of ecopipam as a first-in-class novel therapy for children and adolescents with Tourette syndrome,” said Emalex Biosciences Chief Medical Officer Frederick Munschauer, MD. About Ecopipam Emalex’s first-in-class development candidate ecopipam, a novel investigational compound that is being studied as a potential treatment for certain central nervous system disorders, blocks the actions of the neurotransmitter dopamine at the D1 receptor. Dopamine is a neurotransmitter in the central nervous system, and its receptors have been classified into two “families” based on their genetic structure: “D1” (including subtypes D1 and D5) and “D2” (including subtypes D2, D3, and D4). D1 receptor super-sensitivity may be a mechanism for the repetitive and compulsive behaviors associated with Tourette syndrome. Currently approved therapies for the treatment of Tourette syndrome act at D2 receptors. Ecopipam has been shown to be generally well tolerated in clinical trials conducted to date and has received Orphan Drug and Fast Track designation from the FDA for the treatment of patients with Tourette syndrome. Adverse events affecting primarily the central nervous system have been reported in clinical trials conducted to date, including headache, fatigue, somnolence, insomnia, restlessness, anxiety, depression and rarely, suicidal ideation. About Emalex Biosciences Emalex Biosciences was created by Paragon Biosciences to develop new treatments for central nervous system disorders. Emalex is in late-stage development of a new class of drug targeting the D1 receptor for patients with Tourette syndrome and other conditions that have limited treatment options. Visit to learn more. About Paragon Biosciences, LLC Paragon Biosciences is a global life science leader that creates, builds and funds innovative biology-based companies in three key areas: cell and gene therapy, biology engineering and advanced biotechnology. Our portfolio companies use biology to accelerate scientific breakthroughs that solve some of society’s most challenging problems. Learn more at .

临床2期孤儿药快速通道临床结果临床3期

2023-07-25

·生物谷

《自然·神经科学》：颠覆认知！美国西北大学团队发现，抗精神分裂药物的作用机制与预想的不一样

近日，美国西北大学的研究团队获得了精神病神经基础理解的里程碑式发现——与抗精神病药物相结合调控纹状体输出并不是D2R，而是D1R。精神分裂症是一种严重的精神障碍，患者可能出现感知、思维和情感方面的严重紊乱，对日常生活产生十分恶劣的影响。多巴胺假说是精神分裂症发病机制的重要假说之一。多巴胺假说认为，精神分裂症可能与大脑中多巴胺的过度活跃有关。过度活跃的神经递质导致特定神经回路的失衡，从而引发幻觉、妄想、思维紊乱、情感疏离等症状。抑制多巴胺活性被认为是抗精神病药物的主要作用机制之一。大脑纹状体区域的棘状投射神经元（SPNs）分别表达多巴胺受体D1（D1R）和多巴胺受体D2（D2R），受体与Gαs偶联可以增加或降低SPN活动。通过调节SPN，可以达到平衡纹状体输出的目的。以往科学家认为，抗精神病药物是通过与D2R结合来抑制多巴胺活性的。事实上，D2R在纹状体之外的脑细胞中也有表达，根据受体表达水平预测的药物治疗效果往往与实际存在误差甚至完全相反。近日，美国西北大学的研究团队获得了精神病神经基础理解的里程碑式发现——与抗精神病药物相结合调控纹状体输出并不是D2R，而是D1R。研究发现，有些药物虽然能够使D2-SPN动态正常化，但是却没有产生临床效果。药物的抗精神病功效与D1-SPN的动力学逆转有关。选择性抑制D1-SPN可以减弱安非他命驱动的过度运动、幻觉等症状。研究发表在《自然·神经科学》上。研究人员通过神经群体成像方法记录小鼠大脑D1-SPN和D2-SPN的活动。使用安非他命诱导多巴胺释放，神经元活动与之前观察的一致，D1-SPN活动水平增加，D2-SPN活动水平降低。接下来，研究人员选择了4种抗精神病药物，评估其对神经元活动的影响。其中，氟哌啶醇有显著的运动障碍副作用，氯氮平和奥氮平运动障碍副作用较轻，MP-10为临床失败的精神分裂症候选药物。根据小鼠用药后的运动能力，设置高、低两种药物剂量。结果显示，3种临床有效药物均可以减轻安非他命引起的D1-SPN神经元动态紊乱、过度运动和感觉运动门控异常（即感觉和运动之间的调节失常）。相比之下，MP-10虽然减轻了安非他命引起的D2-SPN活动水平降低，但是加剧了D1-SPN的过度活动，且并未纠正小鼠的感觉运动门控异常。抗精神病药物改善小鼠的过度运动这样的结果提示我们，是不是对D1-SPN活动的纠正，才是药物起效的关键呢？研究人员用其他新型抗精神病药物候选化合物进行了测试。虽然这些化合物的作用机制并不是与多巴胺受体结合，但所有产生临床疗效的化合物，都可以减轻安非他命引起的D1-SPN过度活动。而通过化学方法抑制D1-SPN活性，也可以减轻安非他命导致的小鼠幻听。药物对D1-SPN的影响能更好地解释抗精神病药物的疗效分别使用D1R部分激动剂SKF38393、D1R拮抗剂SCH23390/SCH39166处理小鼠，结果显示，3种化合物可以减轻安非他命引起的过度运动和感觉运动门控异常。其中，SKF38393在基线条件下促进D1-SPN活动，在多巴胺过度释放的条件下抑制D1-SPN活动，表现出多巴胺状态依赖性的D1-SPN选择性抑制。总的来说，研究证明，抗精神病药物的疗效与其恢复D1-SPN的正常活动有关，为开发副作用更小的药物开辟了新的选择。研究人员表示，“这是一个里程碑式发现，彻底改变了我们对精神病神经基础的理解”。

核酸药物

2023-04-11

·药明康德

2022年全球生物技术公司融资TOP11

▎药明康德内容团队编辑今日，行业媒体Fierce Biotech发布了一份2022年生物技术公司融资榜单，揭示了在过去一年获投资者青睐的公司。在这份榜单中，每一家公司皆获得至少2亿美元的大额融资，显示其所专注开发的新兴技术、管线被市场所看好，标志着生物产业界未来发展的潜在方向。公司：Altos Labs融资金额：30亿美元融资轮：A获得融资月份：1月投资公司：Jeff Bezos、Arch Venture Partners等根据行业媒体Fierce Biotech报道，Altos Labs在去年1月完成生物技术领域有史以来规模最大的一笔投资。除了达10位数的融资金额外，Altos Labs公司的明星阵容更是吸引产业界关注的一大原因。前GSK首席科学官兼研发总裁Hal Barron博士担任公司的首席执行官。创立Juno Therapeutics与Grail公司的Rick Klausner博士担任首席科学官，另一名创始人Hans Bishop先生则出任总裁。原基因泰克高级副总裁Ann Lee-Karlon博士出任首席运营官。CRISPR科技先驱，也是诺奖得主的Jennifer Doudna博士则是董事会成员。▲Rick Klausner博士在2020年药明康德全球论坛上指出：“我们对细胞疗法的理解才刚刚起步。未来几十年，随着科学和技术的创新发展，细胞疗法的研发、生产制造将还有极大的想象空间。”Altos Labs的目标是通过让细胞返老还童，以带来变革性的创新疗法。Altos Labs通过对细胞进行多样的编程，使这些细胞具有更为优化的抵抗力，这种抵抗力指的是抵抗应激，迎来修复或是细胞再生。公司：Areteia Therapeutics融资金额：3.5亿美元融资轮：A获得融资月份：7月投资公司：Bain Capital Life Sciences、Access Biotechnology、GV、Arch Venture Partners、Saturn Partners、Sanofi、Maverick Capital与Population Health Partners2022年7月，Areteia Therapeutics获得3.5亿美元A轮融资，以推进治疗哮喘的小分子药物研发。该公司管线中的先导候选药物dexpramipexole是一种口服小分子药物，通过抑制骨髓中的嗜酸性粒细胞的成熟和释放，治疗嗜酸性粒细胞性哮喘。针对重度嗜酸性粒细胞性哮喘患者的2期临床结果显示，该药物耐受性良好，用药后患者血液中的嗜酸性粒细胞数量显著降低。Areteia利用这笔资金启动了一对3期试验，将共招募2000多名严重嗜酸性粒细胞性哮喘患者，并计划启动第三项试验。两项正在进行中的临床试验中较大的一项是检视dexpramipexole对重度哮喘急性加重发生率影响长达52周的评估，Areteia计划启动第二项具有相同终点的3期试验。而另一项进行中的临床试验则是对dexpramipexole在肺功能改善上的24周评估。公司：Acelyrin融资金额：3亿美元融资轮：C获得融资月份：9月投资公司：Access Biotechnology、AyurMaya、Westlake Village BioPartners、Cowen Healthcare Investments、Decheng Capital、Marshall Wace、OrbiMed、Samsara BioCapital、Surveyor Capital、Tybourne Capital Management与venBio PartnersAcelyrin的主要项目izokibep是一种抗体模拟白细胞介素-17A（IL-17A）抑制剂。去年9月，Acelyrin所获得的3亿美元Ｃ轮资金，主要用于推动izokibep进入银屑病关节炎和中轴型脊柱关节炎的3期试验。今年1月，公司报道了izokibep用于治疗中度至重度化脓性汗腺炎2b/3期研究的积极顶线数据。此外，Acelyrin在今年1月时并购ValenzaBio，这项交易为Acelyrin带来两项候选药物，包含lonigutamab——一种正在进行甲状腺眼病1期研究的抗IGF-1R单克隆抗体，以及VB-517——一种正在为过敏性皮肤病慢性荨麻疹排队的临床前抗c-KIT单抗。公司：Tessera Therapeutics融资金额：3亿美元融资轮：C获得融资月份：4月投资公司：Abu Dhabi Investment Authority、Alaska Permanent Fund Corp.、Altitude Life Science Ventures、Artis Ventures、Cormorant Asset Management、Flagship Pioneering、Hanwha Impact Partners、Longevity Vision Fund、March Capital、Salt Fund与SoftBank Vision Fund 2Tessera在去年4月获得的3亿美元C轮融资被用于支持该公司进一步构建其基因书写（GENE WRITING）技术平台，并且推动多个治疗项目进入临床开发。Tessera的基因书写技术旨在通过书写生命密码来治愈疾病。这一平台可以将任何碱基对改变为任何其他碱基对，进行小片段插入或删减，只需递送RNA就可以将整个基因写入基因组。这具有治愈几乎任何遗传疾病的潜力。▲原AbbVie总裁，Flagship Pioneering的CEO合伙人，Tessera Therapeutics首席执行官Michael Severino博士在2023年药明康德全球论坛上指出，未来的产业要取得创新突破，必须超越用小鼠模型来研究人类疾病。Tessera尚未公布正式的管线或早期临床候选项目，但公司表示正在开展体内和体外基因改变的研究，范围从短片段到外显子长度序列。一些最初关注的领域包括肝脏、肺部和免疫细胞。公司：Kriya Therapeutics融资金额：2.7亿美元融资轮：C获得融资月份：5月投资公司：Patient Square Capital、Bluebird Ventures、CAM Capital、Dexcel Pharma、Foresite Capital、JDRF T1D Fund、Lightswitch Capital、Narya Capital、QVT、Transhuman Capital等Kriya在去年C轮融资中获得高达2.7亿美元的巨额资金，使得其在三轮募资中共筹集了4.5亿美元。Kriya专有的计算引擎SIRVE能够整合该公司工程化和高通量筛选平台产生的大量数据，支持机器学习赋能的分析和智能产品设计。该公司的研发管线专注于4个疾病领域，分别为眼科、肿瘤学、罕见病和慢性病。该公司曾登上行业媒体BioSpace评选的2021年度新一代生物新锐公司榜单。该公司的研发管线中，KT-A112是一款通过肌肉注射给药的基因疗法，它可以产生治疗1型和2型糖尿病患者所需胰岛素和葡萄糖激酶；KT-A522是一款通过唾液腺注射给药的疗法，它能够产生一种胰高血糖素样肽1（GLP-1）受体激动剂，用于治疗2型糖尿病和严重肥胖症患者。KT-A832是一种通过胰腺内注射给药的疗法，它能够产生经修饰的胰岛素生长因子1（IGF-1），治疗1型糖尿病患者。Kriya在去年11月份以未公开的金额收购了Redpin。此次收购将神经病学治疗纳入Kriya的研发管线，主要针对癫痫和三叉神经痛。公司：Emalex Biosciences融资金额：2.5亿美元融资轮：D获得融资月份：11月投资公司：Bain Capital Life Sciences、Paragon Biosciences、Valor Equity Partners、Fidelity Management & Research Co.等Emalex Biosciences所获得的2.5亿美元D轮融资，将协助其开发治疗抽动秽语综合症（TS）这项神经病学疾病。资金将主要用于启动其在研药物ecopipam的一项3期试验。这种创新型多巴胺-1受体拮抗剂已获得FDA对TS患者的孤儿药和快速通道指定。Emalex于今年3月给对首位患者进行给药，试验预计将在北美和欧洲的90个研究中心招募超过200名患者。虽然目前批准的TS治疗方法作用于多巴胺的D2受体，Emalex的药物则靶向神经递质的D1受体。该公司认为，D1受体可能是造成该病状相关重复和强迫行为的机制。公司：Kallyope融资金额：2.36亿美元融资轮：D获得融资月份：2月投资公司：Mubadala Investment Co.与The Column Group领投Kallyope去年2月完成的2.36亿美元D轮融资主要用于推进公司的“肠-脑轴”开创性药物发现平台、临床试验和跨越广泛治疗领域的多个管线项目。通过揭示肠道和大脑之间的连接，Kallyope平台已被用来生成跨越几个相关治疗领域的新型治疗方案，包括代谢、免疫学和炎症、以及中枢神经系统疾病。其策略的一个关键要素，是用肠道限制性分子靶向肠道和肠-脑生理学相关靶标，这是一种有望提高药物发现和开发速度和成功几率的全新方法。通过这笔新的资金支持，Kallyope将继续推进其治疗2型糖尿病、肥胖和胃肠道屏障疾病的两个先导项目的临床试验，以及加快管线中20多个项目和分子的进展，并进一步发展其平台。公司：Orna Therapeutics融资金额：2.21亿美元融资轮：B获得融资月份：8月投资公司：MPM Capital、BioImpact Capital、Merck & Co等Orna Therapeutics在去年8月份同时宣布完成2.21亿美元的B轮融资和与默沙东（MSD）的大额交易。Orna自默沙东获得1.5亿美元前期付款和高达35亿美元的里程碑付款。环形RNA是一种在哺乳动物细胞中天然存在的RNA。它们通过RNA前体的不同剪接方式而生成。科学家们发现，在环形RNA中加入病毒RNA中负责启动蛋白合成的IRES序列，可以让环形RNA与核糖体结合，启动蛋白合成。与线形mRNA相比，环形RNA的结构让它们能够避免被先天免疫系统和核酸外切酶识别，不但显著降低了免疫原性，而且具有更高的稳定性。▲Orna Therapeutics首席执行官Tom Barnes博士在2023年药明康德全球论坛上表示，RNA是一个非常令人兴奋的领域。”但疗法的递送仍然是需要解决的重大挑战。“Orna Therapeutics公司内部管线的主攻方向是利用环形RNA的稳定性，通过在T细胞中引入表达抗体嵌合受体（CAR）的环形RNA，在人体内直接生成CAR-T细胞疗法，从而避免从患者体内分离细胞，在体外进行工程化改造的繁琐过程。此外，该公司还首次使用非病毒递送方式，将编码全长抗肌萎缩蛋白（dystrophin）的环形RNA递送到了人类细胞内部，并且表达抗肌萎缩蛋白。自从筹集到B轮资金后，Orna在中国为其抗CD19候选药物和基于其isCAR技术的其他项目找到了合作伙伴。上海显博生物科技与Orna达成协议，支付了预付款，并承诺实现里程碑款项，以便在大中华区获得这些项目的使用权。公司：Arsenal Biosciences（ArsenalBio）融资金额：2.20亿美元融资轮：B获得融资月份：9月投资公司：Softbank Vision Fund 2、Bristol Myers Squibb、Byers Capital、Emerson Collective Investments、Green Sands、Hitachi Ventures、Sixth Street、the Parker Institute for Cancer Immunotherapy、Westlake Village BioPartners、the University of California、San Francisco Foundation Investment Co.、Euclidean Capital、Waycross Ventures与Kleiner PerkinsArsenal Biosciences是一家专注于开发细胞疗法的公司，其去年所获得2.2亿美元融资帮助公司扩大团队并推动早期项目进入临床，公司的先导项目为AB-1015。去年11月，ArsenalBio启动了AB-1015在铂类耐药性上皮性卵巢癌患者的1期临床试验。ArsenalBio首席执行官Ken Drazan博士在去年9月接受行业媒体Fierce Biotech采访时表示，公司针对肾癌和前列腺癌的第二和第三个项目将分别在2024年初和2025年初准备进入临床阶段。▲Arsenal Biosciences首席执行官Ken Drazan博士在2023年药明康德全球论坛上指出，“细胞能够接受基因编辑的次数并不是无限的”，需要考虑每个细胞能够接受的基因编辑次数和AAV载体的剂量。ArsenalBio所开发疗法的独特之处在于不使用病毒载体的情况下进行基因编辑。ArsenalBio的策略包含三个类别：开发非病毒、基因编辑的自体T细胞，用于治疗实体瘤，建立一份指令数据库以找出如何最好地编辑这些细胞，以及与其他公司进行合作。公司：维泰瑞隆（Sironax）融资金额：2亿美元融资轮：B获得融资月份：8月投资公司：Gaorong Capital、Yunfeng Capital、Temasek, Invus、F-Prime Capital、Eight Roads、Arch Venture Partners、K2 Venture Partners、MSA Capital、CBC Group、Long River Investments、LSV Capital、Superstring Capital、Future Innovation Fund等去年8月，维泰瑞隆宣布完成2亿美元的B轮融资。所获得的资金主要用于受体相互作用蛋白激酶1（RIPK1）抑制剂的临床开发，并进一步扩大公司研发规模、拓展研发管线，针对衰老相关退行性疾病开发潜在“first-in-class”或“best-in-class”创新疗法。该公司由百济神州联合创始人王晓东博士和北京生命科学研究所资深研究员张志远博士联合创建。研究方向包括细胞程序性死亡、神经保护性通路和神经炎症。其主打疗法SIR0365是一款RIPK1抑制剂。RIPK1是一种丝氨酸/苏氨酸蛋白激酶，可调节多种生物信号转导通路，例如肿瘤坏死因子α介导的NFκB信号转导、细胞凋亡、细胞程序性坏死和炎症反应。公司：Upstream Bio融资金额：2亿美元融资轮：A获得融资月份：6月投资公司：OrbiMed、Maruho Co.、Access Biotechnology、Decheng Capital、HBM Healthcare Investments、TCG X、Omega Funds、Samsara BioCapital与Altshuler Shaham Provident FundsUpstream Bio公司去年6月宣布完成2亿美元A轮融资，获得资金主要用于推动其主打在研疗法UPB-101的临床开发。UPB-101是一款靶向抗胸腺基质淋巴细胞生成素（TSLP）受体的单克隆抗体，TSLP是一种上皮细胞因子，位于多个炎症级联反应的顶端。它可能影响多种过敏性和炎症疾病，包括哮喘。Upstream在获得融资两个月后，将UPB-101推进至治疗哮喘的1b试验，以确定试验中期阶段的合适剂量。Upstream的首席执行官Samantha Truex女士在6月接受Fierce Biotech采访时指出，该公司也计划使用所获得的资金建立治疗免疫介导疾病的管线。大家都在看▲欲了解更多前沿技术在生物医药产业中的应用，请长按扫描上方二维码，即可访问“药明直播间”，观看相关话题的直播讨论与精彩回放参考资料：[1] Biotech's top money raisers of 2022. Retrieved April 11, 2023 from https://www.fiercebiotech.com/biotech/biotechs-top-10-money-raisers-2022免责声明：药明康德内容团队专注介绍全球生物医药健康研究进展。本文仅作信息交流之目的，文中观点不代表药明康德立场，亦不代表药明康德支持或反对文中观点。本文也不是治疗方案推荐。如需获得治疗方案指导，请前往正规医院就诊。版权说明：本文来自药明康德内容团队，欢迎个人转发至朋友圈，谢绝媒体或机构未经授权以任何形式转载至其他平台。转载授权请在「药明康德」微信公众号回复“转载”，获取转载须知。分享，点赞，在看，聚焦全球生物医药健康创新

高管变更临床2期细胞疗法

100 项与依考匹泮相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
D03937	依考匹泮	-	DB12273

研发状态

适应症	最高研发状态	国家/地区	公司
Lesch-Nyhan综合征	临床3期	西班牙更多	Emalex Biosciences, Inc.初创企业更多
Tourette 综合征	临床3期	美国更多	Emalex Biosciences, Inc.初创企业更多
口吃	临床2期	美国更多	Emalex Biosciences, Inc.初创企业更多
不宁腿综合征	临床2期	美国更多	Emalex Biosciences, Inc.初创企业更多
自残行为	终止	西班牙更多	Psyadon Pharmaceuticals, Inc. 更多

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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


MDS2021	临床2期	Tourette 综合征	26	Ecopipam	(製觸鏇遞鑰廠壓構憲憲) = 觸觸餘蓋選鹹構廠夢簾範膚衊壓衊網願選顧糧 (網鏇憲憲觸鏇廠製觸繭 )	积极	2021-09-17
NCT01244633 (CTgov)	临床1/2期	Tourette 综合征	18	Ecopipam	(鏇壓壓網觸餘衊網鹽選) = 簾壓窪鹹選夢顧襯醖築憲觸構憲壓鹽獵廠淵鏇 (齋繭願膚艱製齋選蓋窪, 網鹹蓋蓋鏇鹹觸觸積膚 ~ 範鏇蓋夢簾蓋獵獵壓壓) 更多	-	2015-09-29
Corporate Publications 人工标引	临床2期	Tourette 综合征	-	Ecopipam	(鑰衊夢膚鑰觸壓夢淵範) = 願壓獵衊艱膚遞鏇選醖築顧鑰夢糧鬱窪醖築窪 (窪醖餘築構醖鑰蓋鹽餘 ) 更多	积极	2023-10-05
NCT04007991 (CTgov)	临床2期	Tourette 综合征	153	Ecopipam (Ecopipam HCI 2 mg/kg/Day)	製構憲選範鬱獵糧構選(遞淵淵範糧鑰鏇夢壓遞) = 廠淵願醖構齋製製窪淵餘鹽鬱糧鬱糧膚範壓積 (遞襯齋淵鑰願蓋顧艱鹹, 簾夢簾壓鑰觸餘鑰簾鑰 ~ 鹹蓋膚齋繭選築網願鏇) 更多	-	2023-10-04
NCT04007991 (CTgov)	临床2期	Tourette 综合征	153	Placebo (Placebo)	製構憲選範鬱獵糧構選(遞淵淵範糧鑰鏇夢壓遞) = 襯艱繭淵夢膚膚淵餘觸餘鹽鬱糧鬱糧膚範壓積 (遞襯齋淵鑰願蓋顧艱鹹, 餘鬱繭選繭願夢簾窪夢 ~ 觸鬱鬱選窪築壓憲憲淵) 更多	-	2023-10-04
NCT01065558 (Pubmed \| Mol Genet Metab) 人工标引	临床1期	Lesch-Nyhan综合征	5	ecopipam	(蓋壓窪齋齋繭觸憲觸獵) = sedation being the most common dose-limiting event 網獵遞選襯簾齋簾壓鹽 (構觸鬱顧獵顧願糧願網 )	积极	2016-04-01
MDS2019	N/A	不宁腿综合征	10	Ecopipam	(築艱鑰壓積壓鑰艱鬱網) = Tolerability was very good 願醖壓繭艱蓋夢壓廠構 (廠醖遞鏇鏇憲襯積觸繭 ) 更多	积极	2019-09-22
MDS2019	N/A	不宁腿综合征	10	Placebo		积极	2019-09-22
Phase 2b Study of Ecopipam (MDS2022)	临床2期	Tourette 综合征	153	Ecopipam	(獵齋觸齋鬱遞積鑰選醖): LS mean [SE] difference = -3.4 (95% CI, -6.1 ~ -0.8) 更多	积极	2022-09-15
Phase 2b Study of Ecopipam (MDS2022)	临床2期	Tourette 综合征	153	Placebo		积极	2022-09-15
NCT02909088 (Pubmed \| Clin Neuropharmacol)	临床2/3期	Tourette 综合征	18	Ecopipam 50 mg	(範蓋鑰醖構選淵膚鹽築) = 22% 鏇製構衊構窪糧淵構夢 (觸夢遞顧築築願淵範選 ) 更多	-	2014-01-01
NCT01751802 (CTgov)	临床3期	自残行为 \| Lesch-Nyhan综合征	9	Ecopipam (Ecopipam)	獵餘繭鏇襯憲選獵積鑰(蓋鏇獵選繭夢糧鹹選艱) = 願觸淵壓糧鬱衊觸齋觸憲膚鏇網艱餘鹹積選繭 (衊窪網淵範淵憲範構襯, 衊範窪鏇淵廠淵襯遞廠 ~ 願廠膚蓋築築鹽選鹹鬱) 更多	-	2015-10-08
NCT01751802 (CTgov)	临床3期	自残行为 \| Lesch-Nyhan综合征	9	Placebo (Placebo)	獵餘繭鏇襯憲選獵積鑰(蓋鏇獵選繭夢糧鹹選艱) = 鹽憲壓選築鹹鏇襯觸願憲膚鏇網艱餘鹹積選繭 (衊窪網淵範淵憲範構襯, 艱蓋願鬱淵製壓醖鏇憲 ~ 願糧餘鏇鹽願鬱糧壓願) 更多	-	2015-10-08
P02.058 (AAN2013)	N/A	Tourette 综合征	15	Ecopipam 50 mg	(膚觸壓鏇範鹽蓋鏇遞衊) = Three patients each reported fatigue 衊膚廠餘鹹簾簾鏇繭齋 (觸願獵壓鬱築夢製範網 ) 更多	积极	2013-02-12