A Phase 3 Multicenter, Randomized, Double-Masked Study Comparing the Efficacy and Safety of Emixustat Hydrochloride With Placebo for the Treatment of Macular Atrophy Secondary to Stargardt Disease

The purpose of this study is to determine if emixustat hydrochloride reduces the rate of progression of macular atrophy compared to placebo in subjects with Stargardt disease.
Funding Source -- FDA OOPD

开始日期2019-01-07

申办/合作机构

Acucela, Inc.

[+1]

NCT03033108

/ Completed临床2期

A Phase 2a Multicenter, Randomized, Masked Study Evaluating the Pharmacodynamics of Emixustat Hydrochloride in Subjects With Macular Atrophy Secondary to Stargardt Disease

This is a pharmacodynamics study of emixustat hydrochloride in subjects with macular atrophy secondary to Stargardt disease.

开始日期2017-01-01

申办/合作机构

Acucela, Inc.

NCT02753400

/ Completed临床2期

A Multicenter, Randomized, Double-Masked, Placebo-Controlled, Pilot Study to Evaluate Effects of Emixustat Hydrochloride on Aqueous Humor Biomarkers Associated With Proliferative Diabetic Retinopathy

To evaluate the effects of oral emixustat hydrochloride (emixustat) on aqueous humor biomarkers associated with proliferative diabetic retinopathy (PDR) from baseline to week 12.

开始日期2016-05-01

申办/合作机构

Acucela, Inc.

100 项与 Emixustat Hydrochloride 相关的临床结果

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100 项与 Emixustat Hydrochloride 相关的转化医学

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100 项与 Emixustat Hydrochloride 相关的专利（医药）

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项与 Emixustat Hydrochloride 相关的文献（医药）

2025-08-28·JOURNAL OF MEDICINAL CHEMISTRY

Rationally Designed, Short-Acting RPE65 Inhibitors for Visual Cycle-Associated Retinopathies

Article

作者: Saraswat, Vivek ; Chen, Xiuyuan ; Hu, Yulun ; Palczewski, Krzysztof ; Tochtrop, Gregory P. ; Bassetto, Marco ; Smidak, Roman ; Li, Bowen ; Kiser, Philip D. ; Damacio, Francisco

The visual cycle is a metabolic pathway essential for visual function. The bisretinoid byproducts of this pathway can induce retinal toxicity, as occurs in Stargardt disease type 1 (STGD1). Emixustat, which inhibits bisretinoid production, is a visual cycle modulator (VCM) that targets RPE65. However, it causes visual impairment due to its unfavorable duration of action. Here, we report ester-containing analogs of emixustat that are susceptible to hydrolytic clearance and function as short-acting VCMs. We show that the esterase-mediated metabolism of these compounds can be tuned while maintaining high-affinity RPE65 targeting. Compounds 6 (EYE-002) and 7 (EYE-003) containing diethyl acetate and valproate esters, respectively, allowed faster recovery of visual cycle function compared to emixustat. These molecules protected against retinal degeneration in mouse models of photic retinopathy and STGD1. These data demonstrate that shorter attenuation of the visual cycle can therapeutically intervene in retinal diseases with fewer visual side effects compared to emixustat.

2024-06-14·ACS Pharmacology & Translational Science

Unlocking the Therapeutic Potential: Sitagliptin’s Multifaceted Approach in Drug-Resistant Epilepsy through a Novel Mechanism Inhibiting Protein Kinase C-γ and a Long-Term Potentiation Pathway

Article

作者: Raja, Anupam ; Avti, Pramod ; Medhi, Bikash ; Singh, Harvinder ; Bhatia, Alka ; Chauhan, Arushi ; Prakash, Ajay ; Jain, Ashish

Drug-resistant epilepsy is a prominent challenge in chronic neurological disorders. Valproate, commonly used to treat epilepsy, can fail due to various side effects and interactions, necessitating the exploration of alternative treatments. Our study primarily investigated sitagliptin's potential as a therapeutic agent for drug-resistant epilepsy. Employing computational modeling and enzyme assay testing, three lead compounds, emixustat, sitagliptin, and distigmine bromide, were evaluated against the target enzyme protein kinase C-γ. In vivo, experiments on a pentylenetetrazolium-induced lamotrigine-resistant epilepsy model were conducted to test sitagliptin's antiseizure effects, compared with the standard phenobarbital treatment. Emixustat and sitagliptin showcased strong inhibitory properties, while distigmine bromide was less effective in the enzyme assay. Mechanistic insights revealed sitagliptin's ability to modulate the seizure grade and first myoclonic jerk latency via oxidative stress markers, like reduced glutathione and glutathione peroxidase emphasizing its antioxidative role in epilepsy. Additionally, it demonstrated anti-inflammatory effects by significantly reducing proinflammatory markers interleukin-1β and interleukin-6. The modulation of key genes of the long-term potentiation pathway, particularly protein kinase C-γ and metabotropic glutamate receptor 5, was evident through mRNA expression levels. Finally, sitagliptin showed potential neuroprotective properties, limiting pentylenetetrazolium-induced neuronal loss in the hippocampal region. Collectively, our findings suggest sitagliptin's multidimensional therapeutic potential for drug-resistant epilepsy specifically via a long-term potentiation pathway by inhibiting protein kinase C-γ.

2023-12-01·Neural regeneration research

Retinoprotective compounds, current efficacy, and future prospective.

Review

作者: Marino, Rachele ; Sappington, Rebecca ; Feligioni, Marco

Retinal dysfunction is the most common cause of vision loss in several retinal disorders. It has been estimated a great increase in these pathologies that are becoming more globally widespread and numerous over time, also supported by the life expectancy increment. Among different types of retinopathies, we can account some that share causes, symptoms, and treatment including diabetic retinopathy, age-related macular degeneration, glaucoma, and retinitis pigmentosa. Molecular changes, environmental factors, and genetic predisposition might be some of the main causes that drive retinal tissue to chronic inflammation and neurodegeneration in these retinopathies. The treatments available on the market contain compounds that efficiently ameliorate some of the important clinical features of these pathologies like stabilization of the intraocular pressure, reduction of eye inflammation, control of eye oxidative stress which are considered the major molecular mechanisms related to retinal dysfunction. Indeed, the most commonly used drugs are anti-inflammatories, such as corticosteroids, antioxidant, hypotonic molecules and natural neuroprotective compounds. Unfortunately, these drugs, which are fundamental to treating disease symptoms, are not capable to cure the pathologies and so they are not life-changing for patients. This review provided an overview of current treatments on the market, but more interestingly, wants to be a quick window on the new treatments that are now in clinical trials. Additionally, it has been here highlighted that the recent technical enhancement of the investigation methods to identify the various retinopathies causes might be used as a sort of "precise medicine" approach to tailor the identification of molecular pathways involved and potentially study a dedicated treatment for each patient. This approach includes the use of cutting-edge technologies like gene therapy and metabolomics.

项与 Emixustat Hydrochloride 相关的新闻（医药）

2026-05-04

·Business Wire

Kubota Vision Inc. Signs Supply and Licensing Agreement with Laboratoires KÔL for Stargardt Disease Treatment Candidate Under Compassionate Use

CLERMONT-FERRAND, France & TOKYO, Japan--(BUSINESS WIRE)--Kubota Vision Inc. (“Kubota Vision”), a wholly-owned subsidiary of Kubota Pharmaceutical Holdings Co., Ltd. (Minato-ku, Tokyo, Japan) announced signing of a Supply and Licensing Agreement with Laboratoires KÔL (Clermont-Ferrand, France) for the provision of a Stargardt disease (STGD1) treatment candidate under compassionate use authorization. The purpose of this Agreement is for Kubota Vision and KÔL to collaborate on providing “Emixustat” for treatment of Stargardt Disease (STGD1) through compassionate use authorization in France. Kubota Vision will exclusively manufacture and supply Emixustat final products to KÔL and KÔL will have exclusive rights to distribute Emixustat in France under compassionate use access. Ryo Kubota, Chairman, President, and CEO of Kubota Pharmaceutical Holdings, stated, “We are extremely pleased to enter into this Agreement with Laboratoires KÔL for the development, supply and distribution of Emixustat for Stargardt disease (STGD1). By combining Kubota Vision’s clinical development and visual cycle modulation technology with KÔL’s expertise in rare ocular diseases, we aim to deliver meaningful impact for patients and the global ophthalmic community.” Sophie Momège, Founder and CEO of Laboratoires KÔL, commented, “We are honored to collaborate with Kubota Vision to explore the development and commercialization of Emixustat for Stargardt disease, a condition that currently has no cure. By combining our expertise with Kubota Vision’s proven clinical capabilities, we aim to bring one of the first effective treatments to patients worldwide and help reduce vision loss among children and adults.” About Stargardt disease (STGD1) Stargardt disease (STGD1) is a rare hereditary retinal disorder that typically develops in childhood or adolescence and causes gradual loss of vision. It is also known as Stargardt macular dystrophy or juvenile macular degeneration. The condition is primarily caused by mutations in the ABCA4 gene, which lead to progressive damage of the photoreceptor cells and subsequent decline in visual acuity. Symptoms include central vision loss, blurred or distorted vision, reduced visual field, and color vision abnormalities. While most cases appear early in life, symptoms may also emerge in adulthood. The retina enables vision through the visual cycle, a process that converts light into electrical signals sent to the brain. During this cycle, light absorption by visual pigments generates vitamin A–derived byproducts that are toxic if not properly cleared. In a healthy retina, these compounds are removed from photoreceptor cells by a membrane transporter, preventing damage to the retinal pigment epithelium (RPE). In Stargardt disease, mutations in the ABCA4 gene impair this protective mechanism. As a result, toxic metabolites accumulate within RPE cells, leading to cellular dysfunction and triggering apoptosis. The progressive death of RPE cells causes secondary loss of photoreceptors, ultimately resulting in progressive vision loss and, in severe cases, blindness. The accumulation of these toxic metabolites within RPE is considered to be the direct cause of Stargardt Disease. Currently, no approved treatment is available for Stargardt disease. About Emixustat Emixustat is expected to suppress the progression of Stargardt disease (STGD1) by selectively inhibiting RPE65, a key enzyme in the visual cycle, through Kubota group’s proprietary visual cycle modulation (VCM) technology. This selective inhibition reduces the accumulation of metabolic waste products generated during the visual cycle. Visual Cycle Modulation (VCM) is a therapeutic approach designed to reduce the accumulation of toxic byproducts in the retina during the visual cycle. These toxic compounds contribute to oxidative stress and light‑induced retinal damage, particularly within retinal pigment epithelium (RPE) cells, which continuously phagocytose photoreceptor outer segments while accumulating visual cycle byproducts. Emixustat hydrochloride modulates this process by selectively targeting rod cells without affecting cone cells. It inhibits key enzymes involved in the visual cycle, thereby reducing rod cell activity and slowing the formation of toxic metabolites. By decreasing their accumulation in RPE cells, visual cycle modulation helps protect the retina and may delay disease progression. About Laboratoires KÔL Laboratoires KÔL is a French pharmaceutical company focused on rare diseases, with a strong emphasis on ophthalmology. The company possesses deep expertise in corneal grafting, access authorization pathways (AAC/AAP), and preventive eye-care therapies, pioneering innovative treatments for rare corneal disorders. Drawing inspiration from ancient eye-care traditions such as Egyptian “khôl” and integrating cutting-edge medical science, Laboratoires KÔL collaborates with hospitals, research institutions, and regulatory bodies to deliver patient-centric therapeutic solutions worldwide. The company engages in the research, development, manufacturing, and commercialization of drugs for rare diseases. Its proprietary antisense oligonucleotide platform is developed into 2 different eyedrops formulations respectively for corneal disease (Olisens®), and retina. A large clinical program is on-going for this antiangiogenic messenger RNA technology with preliminary promising results in both indications. For more information, visit https://www.laboratoires-kol.com/ About Kubota Vision Inc. Kubota Vision Inc. is a wholly owned subsidiary of Kubota Pharmaceutical Holdings Co., Ltd. (Tokyo 4596), committed to translating innovation into a diverse portfolio of drugs and devices to preserve and restore vision for millions of people worldwide. Kubota Pharmaceutical Group’s research and development pipeline includes Emixustat hydrochloride as a therapeutic candidate for Stargardt disease (STGD1) and proliferative diabetic retinopathy (PDR), as well as a VAP-1 inhibitor targeting Alzheimer’s disease and metabolic dysfunction-associated steatohepatitis (MASH). As medical device and related product portfolio includes Kubota Glass®, a wearable device designed to suppress the progression of myopia, and eyeMO®, a retinal monitoring device developed for home- and tele-ophthalmology use. Kubota Glass® is currently being marketed in both the Japanese and Chinese markets, further expanding the Kubota Pharmaceutical Group’s commitment to advancing ophthalmic innovation and accessible vision care solutions globally. For more information, visit kubotavision.com The “Kubota” logo is registered trademark of Kubota Pharmaceutical Holdings Co., Ltd.

引进/卖出寡核苷酸

2025-11-24

·医药健闻

跨国药企在中国 | 拜耳、强生、武田、飞利浦、卫材、阿斯利康、礼来、赛诺菲、辉瑞、灵北、科利耳、百汇医疗、龙沙、欧彼乐等新动态

跨国药企在中国重点资讯文 | 苏丁企业动态拜耳拜耳·亦庄开放创新中心11月15日在北京正式启用。2023年，拜耳·亦庄开放创新中心宣布将落户位于亦庄的国际医药创新公园（BioPark），这也是拜耳在华设立的首个创新中心。该中心将孵化本土创新药企，助力本土创新药出海，全链条强化中国创新。在启用仪式上，拜耳全球生命科学共创平台拜耳Co.Lab宣布正式落户北京亦庄BioPark，未来将与拜耳·亦庄开放创新中心形成合力，链接全球资源与合作网络，拓深本土创新合作。拜耳Co.Lab在拜耳·亦庄开放创新中心迎来了首批入驻企业——浦合医药与尧景基因。武田武田(Takeda)中国宣布其设立于四川成都的武田中国创新中心(TCIC) 正式投入运营。TCIC是武田数字化创新网络的重要组成部分，也是武田首个将同步推进数字医疗解决方案开发与落地应用的创新中心。自1994年进入中国以来，武田已经完成从新药开发、生产、药品保障供应及商业化运营的完整药品价值链布局。TCIC的成立进一步拓展和加强了武田在华价值链布局。强生强生创新制药中国宣布重要人事变动：公司副总裁、PINS事业部负责人、成熟产品中国负责人何旭正（Alex）将在服务九年多后正式离职，转而寻求外部发展机会。其离职生效日期为2025年12月31日。在强生任职期间，他历任杨森中国香港和澳门地区总经理，2021年底出任杨森中国副总裁、免疫和肺动脉高压业务部负责人，深度参与企业核心业务布局。飞利浦在中华医学会第32次放射学学术大会期间，由上海九州智慧医学影像研究院主办、飞利浦协办的全球代谢影像高峰论坛举行。论坛上，飞利浦（中国）宣布新一代代谢影像解决方案——多核磁共振MR 7700与MetaCT联合亮相。其中飞利浦（苏州）医疗影像基地生产的业内首款3T临床多核磁共振MR7700已通过国家药品监督管理局（NMPA）认证，将在中国市场正式上市。飞利浦新一代多核磁共振MR 7700可在无辐射、无放射性的条件下，提供多维度在体生化代谢图谱。飞利浦MetaCT以高能量成像精度，结合代谢拓展平台，可实现细胞及微环境水平表征成像。卫材2025年产业链供应链国际合作交流会暨企业家太湖论坛在无锡国际会议中心举行。卫材（中国）投资有限公司受邀出席，并由卫材中国副总裁、准入管理本部本部长李云龙代表企业参与江苏省生物医药全产业链开放创新联盟成立仪式。江苏省生物医药全产业链开放创新联盟正式启动，这标志着江苏生物医药产业协同发展迈入新阶段。卫材中国将积极参与联盟的各项协同创新活动，与合作伙伴一同在医药研发、生产、销售、服务等全产业链进行资源整合，为患者提供全方位、一站式的健康解决方案。科利耳11月21日，海南博鳌乐城国际医疗旅游先行区举办首届全球创新药械博览会。科利耳携两大创新产品参展——全球首创的Nucleus Nexa智能人工耳蜗系统，以及业界首款支持3.0T磁共振扫描的最新一代Osia主动式骨传导植入系统。科利耳通过乐城先行区将Nucleus Nexa系统引入中国，真正实现了中国患者“零距离”享有全球领先技术。科利耳最新一代Osia主动式骨传导植入系统，是业界首款支持3.0T磁共振扫描的主动式骨导系统。在本次会议期间，已顺利完成科利耳最新一代Osia主动式骨传导植入系统首批患者植入手术。百汇医疗IHH医疗集团旗下的百汇医疗新天地医疗中心11月20日于上海黄浦区新天地商圈举办开业仪式，标志着百汇医疗在中国市场布局的进一步深化。新启幕的百汇医疗新天地医疗中心坐落于淮海中路核心地段，已于今年10月正式投入运营，中心占地面积近7000平方米，以内科、外科、妇科、儿科等20余个临床专科为基础，集成健康管理中心、内镜中心、手术中心、儿童健康中心、医疗美容中心、运动医学与康复中心六大特色学科，汇聚来自14国的国际化多语种医师团队，支持超过60家国际保险直付。百汇医疗同时宣布与上海健康频道共同签署合作协议，联合成立“健康汇客厅”科普直播站。位于虹桥古北商圈的百汇医疗（虹桥古北）门诊部也将于2026年迎来焕新升级。龙沙继在美国东、西海岸举办CGT创新技术论坛后，龙沙（Lonza）于11月12日在上海张江科学城呈现全球系列第三场盛会。论坛围绕如何将本土创新推向全球市场以及如何实现高效、稳健、可规模化的细胞扩增与基因改造等CGT领域的两大关键议题展开。Lonza细胞工程技术研发经理Valerie Annibaldi博士深入介绍了Lonza在该领域的王牌技术Nucleofector在非病毒、大规模基因编辑工具递送中的应用。Lonza细胞与基因治疗培养基副总监张成康博士重点介绍了Lonza TheraPEAK T-VIVO与NK-VIVO无血清培养基平台。Lonza亚太区培养基领域专家方强毅博士重点介绍了Lonza全新上市的TheraPEAK AmpliCell高性能重组细胞因子以及TheraPEAK 293-GT培养基系统。欧彼乐欧彼乐（Opella）开展2025年“使命日”（Purpose Day）活动，携手上海宋庆龄基金会、上海市儿童医院共同发起“守护肺腑·健康在握 —— 儿童呼吸健康关爱与科学防治行动”。此次活动通过线上公益科普片与线下沉浸式公益活动相结合的方式，致力于提升家庭对儿童常见呼吸道疾病的科学防治认知与自我诊疗意识。此外，欧彼乐捐赠10万元至上海宋庆龄基金会欧彼乐公益项目，通过更多元化的科普教育助力构筑儿童呼吸健康防线，为提升全民健康素养水平贡献力量。窪田制药窪田制药控股株式会社将11月20日至22日在中国厦门国际会展中心举办的“厦门国际光学博览会”参展。参展产品包括：Kubota Glass，一款以关注眼轴长度变化因素为理念的全新近视护理眼镜型可穿戴设备。通过散焦图像刺激视网膜，旨在维持视觉环境的恒定性，每天佩戴2小时，通过对周边视网膜的近视性散焦刺激，促进视网膜前移反应。此外，公司还在推进居家/远程医疗监测设备“eyeMO”的开发；以及针对导致失明的严重眼病的再生和基因治疗药物Emixustat盐酸盐“RETAKU”。德达医疗11月7日，一场聚焦结构性心脏病介入治疗的学术会议在上海德达心血管医院举行。本次会议由中国医药生物技术协会主办，上海德达心血管医院承办，众多心血管领域的权威专家齐聚一堂，共同探讨学科前沿动态，分享前沿研究成果。论坛由上海德达心血管医院副院长副院长黄连军主持，主讲嘉宾阵容堪称豪华，包括孙立忠、龙村、许建屏、万峰、刘亚君、田小园、赵晓琴等心血管领域的权威专家。会议还设置多孔型房缺介入治疗专场交流会议和卵圆孔未闭介入治疗专场学术交流会议。产业动态和铂医药宣布将更新与深化推进其与阿斯利康于2025年3月建立的全球战略合作。双方将基于各自专业技术知识，共同发现和开发包括抗体偶联药物（ADC）和T细胞衔接器（TCE）在内的新一代生物疗法。阿斯利康将在未来四年内，每年持续向和铂医药提名研发项目，并获得这些项目的授权许可选择权。和铂医药将有资格获得选择权费、选择权行使费、开发与商业里程碑付款，以及授权项目基于未来净销售额的分级特许权使用费。相关经济条款与双方于2025年3月达成的财务框架保持一致。礼来中国宣布，中国国家药品监督管理局已批准记能达（多奈单抗）新的推荐剂量滴定方案的说明书更新。记能达是礼来公司每月一次的靶向淀粉样蛋白治疗药物，适用于早期症状性阿尔茨海默病（AD）成人患者，包括轻度认知障碍（MCI）患者以及处于轻度痴呆阶段的AD患者，且需确认存在淀粉样蛋白病理。从2026年1月1日开始，赛诺菲将借助本土合作伙伴上药科园全资子公司宏曜科技力量，在部分广阔市场区域提供潘太欣（五联苗）、凡尔佳（流感疫苗）和凡尔灵（流感疫苗）三款疫苗产品的接种点学术推广服务，进一步提升可及性。赛诺菲将继续负责三款产品全国的进口、分销以及其他所有区域的学术推广工作。辉瑞宣布，全球首款只需每周一次固定剂量皮下注射的非因子创新药物友瑞宁（通用名：马塔西单抗）已获中国国家药品监督管理局（NMPA）正式批准上市，适用于患有不存在凝血因子VIII抑制物的重型A型血友病（先天性凝血因子VIII缺乏，FVIII<1%）或不存在凝血因子IX抑制物的重型B型血友病（先天性凝血因子IX缺乏，FIX<1%）且体重≥35kg的12岁及以上儿童和成人患者的常规预防治疗，以防止出血或降低出血发作的频率。诺纳生物宣布与辉瑞签订一项非独家许可协议，以加速针对一系列潜在疾病适应症的临床前抗体发现研究。辉瑞将获得诺纳生物专有的HCAb技术平台的全球使用权，以生成全人源重链抗体。诺纳生物将获得首付款，并有权获得监管、临床及商业里程碑付款。此外，诺纳生物还有望依托其HCAb平台、先进的B细胞筛选技术及一体化服务体系，与辉瑞在抗体发现、开发及工程化改造等领域开展合作。中国国家药品监督管理局药品审评中心（CDE）已正式受理了灵北公司就偏头痛治疗药物Vyepti(eptinezumab)提交的新药上市申请（NDA），用于成人偏头痛的预防性治疗。若获得批准，这将是灵北公司在中国内地及亚洲重点市场首次推出生物制剂，为符合预防性治疗条件的偏头痛患者提供全新的治疗选择。联系美通社 +86-10-5953 9500 info@prnasia.com

高管变更放射疗法

2023-05-29

·PRNewswire

Diabetic Retinopathy Clinical Trial Pipeline Space Brims With Novel Emerging Therapies with Over 50 Key Companies Working in the Domain | DelveInsight

The prevalence of diabetic retinopathy has been rising over the past few years, which prompts the growing demand for treatment options. The increasing prevalence of diabetic retinopathy and the growing research and development activities to develop novel therapies to treat diabetic retinopathy to drive the market. The companies developing the potential therapies in the last stage of development include Roche, Kodiak Sciences, Novartis, and several others. LAS VEGAS, May 29, 2023 /PRNewswire/ -- DelveInsight's ' Diabetic Retinopathy Pipeline Insight – 2023 ' report provides comprehensive global coverage of pipeline diabetic retinopathy therapies in various stages of clinical development, major pharmaceutical companies working to advance the pipeline space and future growth potential of the diabetic retinopathy pipeline domain. Key Takeaways from the Diabetic Retinopathy Pipeline Report DelveInsight's diabetic retinopathy pipeline report depicts a robust space with 50+ active players working to develop 55+ pipeline therapies for diabetic retinopathy treatment. Key diabetic retinopathy companies such as Kodiak Sciences, Novartis, Regenxbio Inc., OcuTerra Therapeutics, Ocular Therapeutix, Bayer, RemeGen, Roche, Ocuphire Pharma, Adverum Biotechnologies, Boehringer Ingelheim, Palatin Technologies, Valo Health, EyePoint Pharmaceuticals, Kubota Vision, MingSight Pharmaceuticals, Oxurion, Aerie Pharmaceuticals, AsclepiX Therapeutics, Ocugen, Ashvattha Therapeutics, Stealth BioTherapeutics, and others are evaluating new diabetic retinopathy drugs to improve the treatment landscape. Promising diabetic retinopathy pipeline therapies in various stages of development include KSI-301, Brolucizumab, RGX 314, OTT-166, OTX-TKI, Runcaciguat, RC 28 E, RG7774, APX3330, ADVM-022, Faricimab, BI 764524, PL8331, OPL-0401, EYP-1901, emixustat hydrochloride, MS-553, THR-149, AR-13503, AXT107, OCU200, D 4517.2, Elamipretide, and others. In May 2023, Oculic Holding AG announced positive top-line results from Stage 1 of its Phase III DIAMOND trial of OCS-01 eye drops in diabetic macular edema (DME). According to the company, the DIAMOND trial in diabetic macular edema with topical OCS-01 met its stage 1 objective of validating the loading and maintenance dosing regimen designed to optimize OCS-01 efficacy potential with robust statistical significance. In April 2023, Palatin Technologies announced The International Journal of Molecular Sciences published a manuscript, "Stimulating the Melanocortin System in Uveitis and Diabetes Preserves the Structure and Anti-Inflammatory Activity of the Retina" by Tat Fong Ng and Andrew W. Taylor from Department of Ophthalmology, Boston University Chobanian and Avedisian School of Medicine, in Boston, Massachusetts. The manuscript summarizes data demonstrating the effects of PL8331 in two mouse models of retinal disease, experimental autoimmune uveoretinitis (EAU) and diabetic retinopathy (DR). Palatin and the National Institute of Health (NIH) and the Massachusetts Lions Eye Research Foundation provided funding for the study. In February 2023, Ocugen announced that it had submitted an Investigational New Drug application (IND) with the US Food and Drug Administration (FDA) to initiate a Phase 1 clinical trial of OCU200, a fusion protein with a distinct mechanism of action (MOA), for the treatment of diabetic macular edema (DME). This regulatory milestone fulfills the Company's commitment to file the IND for OCU200 within the first quarter of 2023. In January 2023, Ocuphire Pharma announced topline efficacy and safety results from its ZETA-1 Phase II trial evaluating oral APX3330 for the treatment of diabetic retinopathy (DR). Oral APX3330 demonstrated favorable safety and tolerability allowing for a potential attractive non-invasive option for protection of vision in both eyes in DR patients. In May 2022, EyePoint Pharmaceuticals announced the EYP-1901 license to Betta Pharmaceuticals, to develop and commercialize EYP-1901 in China, Hong Kong, Macau, and Taiwan. EyePoint retains all rights for EYP-1901 in the rest of the world and expands its exclusive rights to local delivery of vorolanib for the treatment of all ophthalmic diseases, including diabetic macular edema (DME). Request a sample and discover the recent advances in diabetic retinopathy drug treatment @ Diabetic Retinopathy Pipeline Report The diabetic retinopathy pipeline report provides detailed profiles of pipeline assets, a comparative analysis of clinical and non-clinical stage diabetic retinopathy drugs, inactive and dormant assets, a comprehensive assessment of driving and restraining factors, and an assessment of opportunities and risks in the diabetic retinopathy clinical trial landscape. Diabetic Retinopathy Overview Diabetic retinopathy is a microvascular condition caused by diabetes mellitus's long-term effects. Diabetic retinopathy can cause vision-threatening retinal damage, eventually leading to blindness. It is found in one-third of patients with diabetes and is linked to an increased risk of life-threatening systemic vascular consequences such as stroke, coronary heart disease, and heart failure. Diabetic retinopathy affects an estimated 40% (8% for vision-threatening retinopathy) of people with type 2 diabetes and 86% (42%) of people with type 1 diabetes in the United States. The keys to avoiding diabetic retinopathy blindness are early detection and appropriate intervention. Several large studies have found that the onset of diabetic retinopathy does not usually occur within the first 5 years of type 1 diabetes diagnosis. In contrast, the onset of type 2 diabetes is frequently more subtle, and as a result, retinal damage may be evident at the time of diagnosis. As a result, screening for diabetic retinopathy is advised in type 2 diabetics at the time of diagnosis. Find out more about drugs for diabetic retinopathy @ New Diabetic Retinopathy Drugs A snapshot of the Diabetic Retinopathy Pipeline Drugs mentioned in the report: Learn more about the emerging diabetic retinopathy pipeline therapies @ Diabetic Retinopathy Clinical Trials Diabetic Retinopathy Therapeutics Assessment The diabetic retinopathy pipeline report proffers an integral view of diabetic retinopathy emerging novel therapies segmented by stage, product type, molecule type, mechanism of action, and route of administration. Scope of the Diabetic Retinopathy Pipeline Report Coverage: Global Diabetic Retinopathy Therapeutic Assessment By Product Type: Mono, Combination, Mono/Combination Diabetic Retinopathy Therapeutic Assessment By Clinical Stages: Discovery, Pre-clinical, Phase I, Phase II, Phase III Diabetic Retinopathy Therapeutics Assessment By Route of Administration: Oral, Intravenous, Subcutaneous, Parenteral, Topical Diabetic Retinopathy Therapeutics Assessment By Molecule Type: Recombinant fusion proteins, Small molecule, Monoclonal antibody, Peptide, Polymer, Gene therapy Diabetic Retinopathy Therapeutics Assessment By Mechanism of Action: Vascular endothelial growth factors inhibitors, Angiogenesis inhibitors, Vascular endothelial growth factor A inhibitors, Gene transference, Integrin alphaVbeta3 antagonists, Integrin alphaVbeta6 inhibitors, Integrin alphavbeta8 inhibitors, Platelet-derived growth factor beta receptor antagonists, Protein tyrosine kinase inhibitors, Proto oncogene protein c-kit inhibitors, Vascular endothelial growth factor receptor 3 antagonists, Vascular endothelial growth factor receptor-1 antagonists, Vascular endothelial growth factor receptor-2 antagonists, Guanylate cyclase stimulants, APEX1 protein inhibitors, DNA apurinic apyrimidinic site lyase inhibitors Key Diabetic Retinopathy Companies: Kodiak Sciences, Novartis, Regenxbio Inc., OcuTerra Therapeutics, Ocular Therapeutix, Bayer, RemeGen, Roche, Ocuphire Pharma, Adverum Biotechnologies, Boehringer Ingelheim, Palatin Technologies, Valo Health, EyePoint Pharmaceuticals, Kubota Vision, MingSight Pharmaceuticals, Oxurion, Aerie Pharmaceuticals, AsclepiX Therapeutics, Ocugen, Ashvattha Therapeutics, Stealth BioTherapeutics, and others. Key Diabetic Retinopathy Pipeline Therapies: KSI-301, Brolucizumab, RGX 314, OTT-166, OTX-TKI, Runcaciguat, RC 28 E, RG7774, APX3330, ADVM-022, Faricimab, BI 764524, PL8331, OPL-0401, EYP-1901, emixustat hydrochloride, MS-553, THR-149, AR-13503, AXT107, OCU200, D 4517.2, Elamipretide, and others. Dive deep into rich insights for new drugs for diabetic retinopathy treatment; visit @ Diabetic Retinopathy Medications Table of Contents For further information on the diabetic retinopathy pipeline therapeutics, reach out @ Diabetic Retinopathy Drug Treatment Related Reports Diabetic Retinopathy Market Diabetic Retinopathy Market Insights, Epidemiology, and Market Forecast – 2032 report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key diabetic retinopathy companies, including Genentech, Inc., Regeneron Pharmaceuticals, Roche, Opthea Limited, Regenxbio, Kodiak Sciences Inc, Ocuphire Pharma, Eisai Co Ltd, Apexian Pharmaceuticals, Oculis, among others. Diabetic Retinopathy Epidemiology Forecast Diabetic Retinopathy Epidemiology Forecast – 2032 report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, and diabetic retinopathy epidemiology trends. Proliferative Diabetic Retinopathy Pipeline Proliferative Diabetic Retinopathy Pipeline Insight – 2023 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key proliferative diabetic retinopathy companies, including Kubota Vision, among others. Diabetic Neuropathy Pipeline Diabetic Neuropathy Pipeline Insight – 2023 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key diabetic neuropathy companies, including Goldfinch Bio, Novartis, Serodus, CURACLE, Daiichi Sankyo, NeuroBo Pharmaceuticals, Dong-A Pharmaceutical, among others. Diabetic Neuropathy Market Diabetic Neuropathy Market Insights, Epidemiology, and Market Forecast – 2032 report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key diabetic neuropathy companies, including Goldfinch Bio, Novartis, Serodus, CURACLE, Daiichi Sankyo, NeuroBo Pharmaceuticals, Dong-A Pharmaceutical, among others. Related Healthcare Services Healthcare Consulting Healthcare Competitive Intelligence Services Healthcare Asset Prioritization Services About DelveInsight DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports pharma companies by providing comprehensive end-to-end solutions to improve their performance. Get hassle-free access to all the healthcare and pharma market research reports through our subscription-based platform PharmDelve . Connect with us on LinkedIn |Facebook|Twitter Contact Us Shruti Thakur [email protected] +1(919)321-6187 Logo: SOURCE DelveInsight Business Research, LLP

临床结果临床1期临床3期

100 项与 Emixustat Hydrochloride 相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
D10363	Emixustat Hydrochloride	-	DB12608

研发状态

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适应症	最高研发状态	国家/地区	公司	日期
皮肤松垂	临床3期	美国	Acucela, Inc.	2019-01-07
皮肤松垂	临床3期	巴西	Acucela, Inc.	2019-01-07
皮肤松垂	临床3期	加拿大	Acucela, Inc.	2019-01-07
皮肤松垂	临床3期	丹麦	Acucela, Inc.	2019-01-07
皮肤松垂	临床3期	法国	Acucela, Inc.	2019-01-07
皮肤松垂	临床3期	德国	Acucela, Inc.	2019-01-07
皮肤松垂	临床3期	意大利	Acucela, Inc.	2019-01-07
皮肤松垂	临床3期	荷兰	Acucela, Inc.	2019-01-07
皮肤松垂	临床3期	南非	Acucela, Inc.	2019-01-07
皮肤松垂	临床3期	西班牙	Acucela, Inc.	2019-01-07

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临床结果

适应症

分期

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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


NCT03772665 (SeaSTAR, CTgov)	临床3期	斯塔加特病 \| 皮肤松垂	194	Emixustat (Emixustat)	衊獵糧齋網顧憲蓋鏇獵(淵襯糧鹽範觸憲積鏇壓) = 鹽膚積蓋鬱願鹹獵艱鬱醖簾鹹選鹽壓鏇膚積膚 (顧鹹遞鹹醖範廠糧齋糧, 0.0783) 更多	-	2023-08-03
				Placebo (Placebo)	衊獵糧齋網顧憲蓋鏇獵(淵襯糧鹽範觸憲積鏇壓) = 顧構醖憲鑰艱壓網淵膚醖簾鹹選鹽壓鏇膚積膚 (顧鹹遞鹹醖範廠糧齋糧, 0.1002) 更多
NCT02753400 (CTgov)	临床2期	伴有糖尿病的增殖性视网膜病变	24	Placebo+emixustat hydrochloride (Emixustat Hydrochloride)	鑰顧憲壓鹽艱襯範鬱鑰(願選鏇壓鏇觸衊範衊蓋) = 製壓淵蓋繭繭夢淵鏇顧廠壓淵鏇獵鏇鏇壓遞鹹 (蓋簾蓋夢膚醖廠衊顧繭, 137.1) 更多	-	2021-04-27
				Placebo (Placebo)	鑰顧憲壓鹽艱襯範鬱鑰(願選鏇壓鏇觸衊範衊蓋) = 觸窪膚鬱獵繭製獵製網廠壓淵鏇獵鏇鏇壓遞鹹 (蓋簾蓋夢膚醖廠衊顧繭, 12.6) 更多
NCT03033108 (CTgov)	临床2期	斯塔加特病 \| 皮肤松垂	23	Emixustat (Emixustat Dose 1)	窪選糧膚蓋鏇構壓獵襯(廠糧繭憲選簾憲窪構壓) = 糧製遞廠壓獵鹽艱淵蓋鬱鹹齋範顧齋顧壓糧選 (醖淵鑰糧鹽獵鑰鹽壓蓋, 窪糧鑰糧願簾壓構壓淵 ~ 廠醖窪網選齋膚網鬱糧) 更多	-	2021-04-27
				Emixustat (Emixustat Dose 2)	窪選糧膚蓋鏇構壓獵襯(廠糧繭憲選簾憲窪構壓) = 壓壓觸製顧餘糧鏇糧鬱鬱鹹齋範顧齋顧壓糧選 (醖淵鑰糧鹽獵鑰鹽壓蓋, 遞齋齋鑰衊夢觸廠衊膚 ~ 窪襯蓋鹹壓膚艱蓋窪積) 更多
NCT02753400 (Pubmed \| Graefes Arch Clin Exp Ophthalmol) 人工标引	临床2期	伴有糖尿病的增殖性视网膜病变	18	emixustat hydrochloride	鹽繭構製製衊鏇構鏇願(遞繭構獵夢鬱餘淵築積) = 構簾觸鏇餘膚膚範糧選廠齋蓋積鹹願遞壓顧艱 (蓋夢製構膚遞餘憲簾餘 ) 更多	积极	2021-02-01
				Placebo	鹽繭構製製衊鏇構鏇願(遞繭構獵夢鬱餘淵築積) = 鏇獵遞鹽糧願窪壓積範廠齋蓋積鹹願遞壓顧艱 (蓋夢製構膚遞餘憲簾餘 ) 更多
NCT01802866 (Pubmed \| Ophthalmology) 人工标引	临床2/3期	年龄相关性黄斑变性 \| 地图样萎缩	508	emixustat hydrochloride	蓋淵壓獵膚衊夢顧夢鹽(衊窪顧觸網淵廠淵築願) = 蓋選壓鏇壓積繭衊淵窪齋醖願衊觸遞夢餘鑰製 (蓋醖夢壓構壓鹹範膚遞 )	不佳	2018-10-01
				Placebo	蓋淵壓獵膚衊夢顧夢鹽(衊窪顧觸網淵廠淵築願) = 鹹衊簾襯糧窪醖淵淵壓齋醖願衊觸遞夢餘鑰製 (蓋醖夢壓構壓鹹範膚遞 )
NCT00942240 (Phase I Dose-Ranging Study of ACU-4429, ARVO2012)	临床1期	-	40	ACU-4429 (5-40 mg)	觸壓積廠鑰鏇築膚獵觸(獵鏇願鹽廠構廠餘膚襯) = The incidence of ocular adverse events (AEs) increased in a dose-dependent manner; all participants exposed to ≥20 mg ACU-4429 reported mild, drug-related ocular AEs and numerous D-28 color vision testing abnormal results; all ocular AEs and abnormal results resolved within 7-10 days of study completion. 襯襯積鹽簾簾繭廠憲製 (醖蓋願網鑰構醖積繭積 )	积极	2012-03-01
				Placebo