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）

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.

2023-06-22·Journal of medicinal chemistry1区 · 医学

Tuning the Metabolic Stability of Visual Cycle Modulators through Modification of an RPE65 Recognition Motif

1区 · 医学

Article

作者: Kiser, Philip D. ; Bassetto, Marco ; Tochtrop, Gregory P. ; Badiee, Mohsen ; Li, Bowen ; Zhang, Jianye ; Zaluski, Jordan

In the eye, the isomerization of all-trans-retinal to 11-cis-retinal is accomplished by a metabolic pathway termed the visual cycle that is critical for vision. RPE65 is the essential trans-cis isomerase of this pathway. Emixustat, a retinoid-mimetic RPE65 inhibitor, was developed as a therapeutic visual cycle modulator and used for the treatment of retinopathies. However, pharmacokinetic liabilities limit its further development including: (1) metabolic deamination of the γ-amino-α-aryl alcohol, which mediates targeted RPE65 inhibition, and (2) unwanted long-lasting RPE65 inhibition. We sought to address these issues by more broadly defining the structure-activity relationships of the RPE65 recognition motif via the synthesis of a family of novel derivatives, which were tested in vitro and in vivo for RPE65 inhibition. We identified a potent secondary amine derivative with resistance to deamination and preserved RPE65 inhibitory activity. Our data provide insights into activity-preserving modifications of the emixustat molecule that can be employed to tune its pharmacological properties.

2022-03-01·BRITISH JOURNAL OF OPHTHALMOLOGY

Randomised study evaluating the pharmacodynamics of emixustat hydrochloride in subjects with macular atrophy secondary to Stargardt disease.

Article

作者: Gregory, Jeff K ; Birch, David G ; Koester, John M ; Kubota, Ryo

BACKGROUND/AIMS:

Stargardt disease is a rare, inherited, degenerative disease of the retina that is the most common type of hereditary macular dystrophy. Currently, no approved treatments for the disease exist. The purpose of this study was to characterise the pharmacodynamics of emixustat, an orally available small molecule that targets the retinal pigment epithelium-specific 65 kDa protein (RPE65), in subjects with macular atrophy secondary to Stargardt disease.

METHODS:

In this multicentre study conducted at six study sites in the USA, 23 subjects with macular atrophy secondary to Stargardt disease were randomised to one of three doses of daily emixustat (2.5 mg, 5 mg or 10 mg) and treated for 1 month. The primary outcome was the suppression of the rod b-wave recovery rate on electroretinography after photobleaching, which is an indirect measure of RPE65 inhibition.

RESULTS:

Subjects who received 10 mg emixustat showed near-complete suppression of the rod b-wave amplitude recovery rate postphotobleaching (mean=91.86%, median=96.69%), whereas those who received 5 mg showed moderate suppression (mean=52.2%, median=68.0%). No effect was observed for subjects who received 2.5 mg emixustat (mean=-3.31%, median=-12.23%). The adverse event profile was consistent with prior studies in other patient populations and consisted primarily of ocular adverse events likely related to RPE65 inhibition.

CONCLUSION:

This study demonstrated dose-dependent suppression of rod b-wave amplitude recovery postphotobleaching, confirming emixustat's biological activity in patients with Stargardt disease. These findings informed dose selection for a 24-month phase 3 trial (SeaSTAR Study) that is now comparing emixustat to placebo in the treatment of Stargardt disease-associated macular atrophy.

项与 Emixustat Hydrochloride 相关的新闻（医药）

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

2022-07-27

·BioSpace

Kubota Vision Announces Database Lock for Phase 3 Clinical Trial of Emixustat in Patients with Stargardt Disease and Expected Release of Top-Line Results in August 2022

SEATTLE--(BUSINESS WIRE)-- Kubota Vision Inc. (“Kubota Vision”), a clinical-stage ophthalmology company and wholly-owned subsidiary of Kubota Pharmaceutical Holdings Co., Ltd. (Tokyo 4596), announced today the database lock for the phase 3 clinical trial of the investigational visual cycle modulator emixustat hydrochloride (emixustat) in patients with Stargardt Disease. Kubota Vision previously announced that the trial was completed in June 2022, as the last patient underwent their last visit. This was followed by completion of data entry, confirmation of the accuracy and completeness of the database, and final sign-off by all investigators. Now that the database is locked, the datasets are being transferred to an independent statistical group, who will unmask patient treatment and analyze the data. Top-line results will be reported by Kubota Vision in August 2022. Ryo Kubota, MD, PhD, Chairman, President, and CEO of Kubota Vision Inc., stated, “Database lock marks an important step towards being able to report the most important results from our phase 3 trial in Stargardt disease. We would like to thank all those who have been involved in this clinical trial over the years and are excited to soon be able to share the outcome of this trial of emixustat for the treatment of this blinding disease.” About Stargardt Disease Stargardt disease is a rare, genetically inherited disease that directly affects the retina of the eye, often resulting in the slow progression of vision loss in children. It may also be referred to as Stargardt macular dystrophy or juvenile macular degeneration and affects approximately 1 in 8,000 - 10,000 individuals worldwide.*1 The most common form of the disease is caused by a genetic mutation of the ABCA4 gene leading to the accumulation of toxic vitamin A byproducts (primarily A2E) in the retina, which results in the gradual deterioration of photoreceptors and vision. Symptoms of Stargardt disease typically appear during childhood or adolescence, but in some cases difficulty with eyesight and vision loss may not be identified until later in life. Stargardt disease affects less than 150,000 patients in total in the U.S., Europe and Japan where it is recognized as an orphan disease. Currently, there are no known therapies that slow the advance of the disease, and it is recognized as a serious unmet medical need. Stargardt disease market is expected to grow to US$1,600M in 2027.*2 *1 Facts About Stargardt Disease, National Eye Institute. , accessed on 14 September 2018. *2 WISEGUY RESEARCH CONSULTANTS PVT LTD Global Juvenile Degeneration (Stargardt Disease) Market Research Report- Forecast to 2027. About Emixustat Hydrochloride Emixustat modulates the visual cycle by inhibiting a critical enzyme of this pathway, retinal pigment epithelium protein 65 (RPE65). The visual cycle is the process by which vitamin A is recycled in the eye; vitamin A is crucial to the visual process. Slowing the visual cycle reduces the availability of vitamin A derivatives (11-cis- and all-trans-retinal) to form precursors of toxic A2E and related compounds. In addition, reducing the availability of 11-cis-retinal decreases retinal metabolic demands under dark conditions. Emixustat when delivered orally was found to be generally well tolerated in human clinical studies with delayed dark adaptation being the most common adverse event. Kubota Vision is exploring emixustat’s potential to stop or slow the progression of vision loss in patients diagnosed with Stargardt disease in an ongoing clinical study. The FDA and EMA granted orphan drug designation to emixustat for the treatment of Stargardt disease. (See January 5, 2017 press release titled “Acucela Receives Orphan Drug Designation from the FDA for the Treatment of Stargardt Disease” and June 9, 2019 press release titled “Acucela Receives Orphan Designation from the EMA for Emixustat for the Treatment of Stargardt Disease”) 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 development pipeline includes drug candidates for the treatment of diabetic retinopathy and Stargardt disease. The Company is also developing a handheld OCT device for the monitoring of neovascular retinal diseases, to be used directly by patients, and a wearable device for myopia control. ; Cautionary Statements: “Kubota Vision,” the Kubota Vision logo and “Kubota” are registered trademarks or trademarks of Kubota Vision Inc. or Kubota Pharmaceutical Holdings in various jurisdictions.

孤儿药

2022-06-24

·BioSpace

Kubota Vision Announced End of Phase 3 Clinical Trial of Emixustat in Patients with Stargardt Disease

SEATTLE--(BUSINESS WIRE)-- Kubota Vision Inc. (“Kubota Vision”), a clinical-stage ophthalmology company and wholly-owned subsidiary of Kubota Pharmaceutical Holdings Co., Ltd. (Tokyo 4596), announced today the completion of the phase 3 clinical trial investigating emixustat hydrochloride (“emixustat”) in patients with macular atrophy secondary to Stargardt disease. The study was a multi-center, randomized, double-masked, and placebo-controlled phase 3 clinical study in which subjects were randomly assigned to emixustat 10 mg or placebo (2:1 ratio) once daily for 24 months. The target total number of subjects was 162; however, due to high interest in the study, a total of 194 subjects were enrolled in this study across 29 sites in 11 countries worldwide. The last patient has completed the study. The primary objective of this study is to determine if emixustat reduces the rate of macular atrophy progression, in comparison to placebo, in subjects with Stargardt disease. Secondary objectives include assessing changes in visual function parameters such as BCVA (best-corrected visual acuity) letter score and reading speed. The database of this study will be locked in the third quarter 2022. Ryo Kubota, MD, PhD, Chairman, President and CEO of Kubota Vision Inc., stated, “Despite Stargardt being a rare disease, we were able to enroll more patients than originally planned and feel grateful that the patients trusted our drug with great expectation and continued taking the drug for two years during the pandemic. We would like to sincerely thank all of the participants and their families for their cooperation and are very pleased that the long-term, large-scale clinical trial has been successfully completed. We are also looking forward to receiving the results of the trial in the coming months.” The FDA (U.S. Food and Drug Administration) and EMA (European Medicines Agency) granted orphan drug designation to emixustat for the treatment of Stargardt disease. (See January 5, 2017 press release titled “Acucela Receives Orphan Drug Designation from the FDA for the Treatment of Stargardt Disease” and June 9, 2019 press release titled “Acucela Receives Orphan Designation from the EMA for Emixustat for the Treatment of Stargardt Disease”) About Stargardt Disease Stargardt disease is a rare, genetically inherited disease that directly affects the retina of the eye, often resulting in the slow progression of vision loss in children. It may also be referred to as Stargardt macular dystrophy or juvenile macular degeneration and affects approximately 1 in 8,000 - 10,000 individuals worldwide.*1 The most common form of the disease is caused by a genetic mutation of the ABCA4 gene leading to the accumulation of toxic vitamin A byproducts (primarily A2E) in the retina, which results in the gradual deterioration of photoreceptors and vision. Symptoms of Stargardt disease typically appear during childhood or adolescence, but in some cases difficulty with eyesight and vision loss may not be identified until later in life. Stargardt disease affects less than 150,000 patients in total in the U.S., Europe and Japan where it is recognized as an orphan disease. Currently, there are no known therapies that slow the advance of the disease, and it is recognized as a serious unmet medical need. *1 Facts About Stargardt Disease, National Eye Institute. , accessed on 14 September 2018. About Emixustat Hydrochloride Emixustat modulates the visual cycle by inhibiting a critical enzyme of this pathway, retinal pigment epithelium protein 65 (RPE65). The visual cycle is the process by which vitamin A is recycled in the eye; vitamin A is crucial to the visual process. Slowing the visual cycle reduces the availability of vitamin A derivatives (11-cis- and all-trans-retinal) to form precursors of toxic A2E and related compounds. In addition, reducing the availability of 11-cis-retinal decreases retinal metabolic demands under dark conditions. Emixustat when delivered orally was found to be generally well tolerated in human clinical studies with delayed dark adaptation being the most common adverse event. Kubota Vision is exploring emixustat’s potential to stop or slow the progression of vision loss in patients diagnosed with Stargardt disease in an ongoing clinical study. 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 development pipeline includes drug candidates for the treatment of diabetic retinopathy and Stargardt disease. The company is also developing a handheld OCT device for the monitoring of neovascular retinal diseases, to be used directly by patients, and wearable device for myopia control. ; Cautionary Statements: “Kubota Vision”, the Kubota Vision logo and “Kubota” are registered trademarks or trademarks of Kubota Vision Inc. or Kubota Pharmaceutical Holdings in various jurisdictions.

孤儿药

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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适应症

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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)	製鬱鏇顧獵衊襯網鹽鹽(顧艱鏇顧築餘壓鬱鹹網) = 選蓋鬱網鹹鑰餘餘觸鬱窪艱醖鹹繭鏇範膚顧範 (獵鹽選製餘積壓觸鹽廠, 齋艱遞簾鬱膚蓋網鹽築 ~ 繭齋糧鹹憲餘鹽廠衊糧) 更多	-	2023-08-03
				Placebo (Placebo)	製鬱鏇顧獵衊襯網鹽鹽(顧艱鏇顧築餘壓鬱鹹網) = 鏇築襯網鹽顧選鏇製繭窪艱醖鹹繭鏇範膚顧範 (獵鹽選製餘積壓觸鹽廠, 網鏇獵窪築齋繭襯廠獵 ~ 鑰繭艱鹹齋鹽觸鏇衊獵) 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)	蓋選廠憲選艱醖鹹範製(窪願網顧顧範襯築網憲) = 觸鬱觸艱醖衊築選壓顧製餘遞憲鏇構襯壓遞艱 (壓鏇獵艱壓齋壓構膚憲, 構願鬱選鑰齋獵製衊鏇 ~ 製繭壓範網蓋積鏇壓顧) 更多	-	2021-04-27
				Placebo (Placebo)	蓋選廠憲選艱醖鹹範製(窪願網顧顧範襯築網憲) = 壓築選襯窪觸構顧繭蓋製餘遞憲鏇構襯壓遞艱 (壓鏇獵艱壓齋壓構膚憲, 糧簾膚鬱艱鹽蓋齋簾憲 ~ 鑰衊壓蓋網鹽網鹽醖糧) 更多
NCT03033108 (CTgov)	临床2期	斯塔加特病 \| 皮肤松垂	23	Emixustat (Emixustat Dose 1)	夢顧網製選鑰艱獵繭範(夢衊窪壓觸夢網衊遞壓) = 網獵襯夢淵繭糧鹹鬱網鬱鹹餘範襯夢蓋觸製積 (網願鑰觸鬱襯顧壓鹹蓋, 襯網齋繭積廠築顧鏇鏇 ~ 選夢膚鏇顧觸窪觸廠遞) 更多	-	2021-04-27
				Emixustat (Emixustat Dose 2)	夢顧網製選鑰艱獵繭範(夢衊窪壓觸夢網衊遞壓) = 蓋獵餘淵夢糧鹽壓憲觸鬱鹹餘範襯夢蓋觸製積 (網願鑰觸鬱襯顧壓鹹蓋, 淵選構艱遞鹹製窪簾鹹 ~ 衊獵積蓋遞鏇窪夢糧醖) 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	壓餘鑰鹹遞夢願繭夢構(獵夢夢獵築鬱壓繭簾襯) = 蓋顧窪鬱繭艱製顧淵網窪遞艱廠築構遞鹹憲範 (憲醖夢蓋製鏇築願積膚 ) 更多	积极	2021-02-01
				Placebo	壓餘鑰鹹遞夢願繭夢構(獵夢夢獵築鬱壓繭簾襯) = 繭膚膚蓋襯鬱鬱醖憲遞窪遞艱廠築構遞鹹憲範 (憲醖夢蓋製鏇築願積膚 ) 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	淵齋網獵願齋夢蓋鏇選(齋鹽遞鹹獵鏇鑰壓鬱鑰) = 範積鏇範鏇糧艱窪衊範選壓願積蓋鏇顧製廠鑰 (網襯選窪襯積繭淵獵鹽 )	不佳	2018-10-01
				Placebo	淵齋網獵願齋夢蓋鏇選(齋鹽遞鹹獵鏇鑰壓鬱鑰) = 壓積願選觸壓夢選鹹餘選壓願積蓋鏇顧製廠鑰 (網襯選窪襯積繭淵獵鹽 )
NCT00942240 (Phase I Dose-Ranging Study of ACU-4429, ARVO2012)	临床1期	-	40	ACU-4429 (5-40 mg)	艱襯鹹衊觸鬱範壓膚膚(餘範鏇齋遞繭餘製鏇廠) = 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. 蓋蓋簾積網鹹醖蓋膚鑰 (鹹膚簾襯製鹽鏇積築蓋 )	积极	2012-03-01
				Placebo