A Phase 2 Multicenter, Double-Masked, Randomized, Placebo-Controlled Study to Investigate the Long Term Safety, Tolerability, Pharmacokinetics and Effects of ALK-001 on the Progression of Stargardt Disease

The purpose of this open-label, multicenter study is to determine the long-term safety, pharmacokinetics and effects of ALK-001 (C20-D3-retinyl acetate) on the progression of Stargardt disease. This study is an extension of NCT02402660 and enrolls participants who are at least 8 years old. Enrollment is by invitation only.
Funding Source - FDA OOPD

开始日期2019-12-20

申办/合作机构

Alkeus Pharmaceuticals, Inc.

NCT03845582

/ Completed临床2/3期

A Phase 2/3 Multicenter, Randomized, Double-masked, Parallel-group, Placebo-controlled Study to Investigate the Safety, Pharmacokinetics, Tolerability, and Efficacy of ALK-001 in Geographic Atrophy Secondary to Age-related Macular Degeneration

This is a double-masked, multicenter, randomized, placebo-controlled clinical trial, evaluating the efficacy and safety of ALK-001 in participants with Geographic Atrophy (GA) secondary to age-related macular degeneration (AMD).
Up to 200 participants will receive ALK-001 while up to 100 participants will receive a placebo.

开始日期2019-05-07

申办/合作机构

Alkeus Pharmaceuticals, Inc.

NCT02402660

/ Enrolling by invitation临床2期

The purpose of this study is to determine the long term safety and tolerability of ALK-001 (C20-D3-retinyl acetate), and to explore the effects of ALK-001 on the progression of Stargardt disease in patients between the ages of 8 and 70 years old.
Funding Source - FDA OOPD

开始日期2015-08-01

申办/合作机构

Alkeus Pharmaceuticals, Inc.

100 项与 Gildeuretinol acetate 相关的临床结果

登录后查看更多信息

100 项与 Gildeuretinol acetate 相关的转化医学

登录后查看更多信息

100 项与 Gildeuretinol acetate 相关的专利（医药）

登录后查看更多信息

项与 Gildeuretinol acetate 相关的文献（医药）

2023-11-01·Current medicinal chemistry

Clinical Application and Synthesis Methods of Deuterated Drugs

Article

作者: Rao, Guo-Wu ; Zhang, Wen ; Xu, Xiao-Liang

Abstract::

Many drugs have adverse absorption, distribution, metabolism, and excretory (ADME) properties that prevent their widespread use or limit their use in some indications. In addition to preparation techniques and prodrug strategies, deuterium modification is a viable method for improving ADME properties. Deuterated drugs have attracted increasing attention from the pharmaceutical industry in recent years. To date, two deuterated drugs have been approved by the FDA. In 2017, austedo was approved by the FDA as a new drug for Huntington's disease in the United States, the first deuterium drug to be marketed worldwide. Recently (June 9, 2021), donafinil has been listed in China; this result has caused major pharmaceutical companies and the pharmaceutical industry to pay attention to deuterium technology again. In addition, BMS-986165, RT001, ALK-001, HC-1119, AVP-786 and other drugs are in phase III clinical studies, and some solid deuterium compounds have entered phase I and II clinical trials. The deuterium strategy has been widely used in pharmaceutical research and has become a hot spot in pharmaceutical research in recent years. In this paper, the research and development of deuterated drugs are reviewed, and the influence of deuterium modification on drugs, the advantages of deuterium strategies and the synthesis strategies of deuterated drugs are mainly introduced. Hoping to provide references for clinical application, the discovery of new deuterium chemical entities and research and development of new deuterated drugs.

2021-09-01·The Journal of biological chemistry2区 · 生物学

Vitamin A cycle byproducts impede dark adaptation

2区 · 生物学

Article

作者: Washington, Ilyas ; Zhang, Dan ; Robinson, Kiera ; Saad, Leonide

Impaired dark adaptation (DA), a defect in the ability to adjust to dimly lit settings, is a universal hallmark of aging. However, the mechanisms responsible for impaired DA are poorly understood. Vitamin A byproducts, such as vitamin A dimers, are small molecules that form in the retina during the vitamin A cycle. We show that later in life, in the human eye, these byproducts reach levels commensurate with those of vitamin A. In mice, selectively inhibiting the formation of these byproducts, with the investigational drug C20D₃-vitamin A, results in faster DA. In contrast, acutely increasing these ocular byproducts through exogenous delivery leads to slower DA, with otherwise preserved retinal function and morphology. Our findings reveal that vitamin A cycle byproducts alone are sufficient to cause delays in DA and suggest that they may contribute to universal age-related DA impairment. Our data further indicate that the age-related decline in DA may be tractable to pharmacological intervention by C20D₃-vitamin A.

2020-01-01·Topics in Medicinal Chemistry

Recent Developments in Agents for the Treatment of Age-Related Macular Degeneration and Stargardt Disease

作者: Konstantin Petrukhin

A review.Age-related macular degeneration (AMD) is the leading cause of irreversible blindness in older individuals living in developed countries.There are two major clin. presentations of AMD: atrophic or "dry" and neovascular or "wet." Geog. atrophy (GA) is the most severe manifestation of dry AMD which represents the form of the disease characterized by the highest prevalence.A smaller fraction of AMD patients (10-20%) develop choroidal neovascularization (CNV) which represents the key feature of neovascular AMD.Historically, laser photocoagulation, surgery, and photodynamic therapy were the first treatment options for patients with CNV.In recent years, the emergence of anti-VEGF therapeutics has transformed the treatment of patients with neovascular AMD.Current anti-VEGF biologics that represent a standard of care (ranibizumab, bevacizumab, and aflibercept) are very effective in improving or maintaining visual acuity in CNV patients over long periods of time.The work on the new generation of anti-VEGF agents with higher potency and long-lasting efficacy is ongoing with the goal of reducing the frequency of intravitreal injections required for achieving good visual acuity outcomes.Brolucizumab, abicipar pegol, and faricimab exemplify the efforts toward the development of novel therapeutic agents with lower frequency of intravitreal injections.While neovascular AMD can be effectively managed with current anti-VEGF therapeutics, there are no FDA-approved treatments for the atrophic form of AMD.Similarly, there is no therapy for inherited Stargardt disease, an orphan genetic form of macular dystrophy that shares phenotypic similarities with atrophic AMD.Due to the multigenic and multifactorial nature of AMD, it is believed that a combination of several factors may contribute to pathogenesis of atrophic AMD.This includes a complement system dysregulation in the retina and exposure of retinal cells to toxins produced in the visual retinoid cycle reactions (lipofuscin bisretinoids and retinaldehydes).Several therapeutic agents are currently being evaluated in clin. trials for geog. atrophy related to atrophic AMD or Stargardt disease.This includes pegcetacoplan (C3 inhibitor), avacincaptad pegol (C5 inhibitor), emixustat (RPE65 inhibitor), ALK-001 (deuterated form of vitamin A: C20-D3-retinyl acetate), STG-001 (RBP4 antagonist), and tinlarebant (RBP4 antagonist).These ongoing clin. trials may yield a therapy that will address a significant unmet medical need that exists for the currently untreatable forms of macular degeneration.

项与 Gildeuretinol acetate 相关的新闻（医药）

2025-10-09

·GlobeNewswire-Health Care

Alkeus Pharmaceuticals Announces Presentation of Gildeuretinol Data at the American Academy of Ophthalmology (AAO) 2025 Annual Meeting, October 18-21 in Orlando

Results from the study of oral gildeuretinol in moderate Stargardt disease to be presented as late-breaker during Retina Subspecialty DayCAMBRIDGE, Mass., Oct. 09, 2025 (GLOBE NEWSWIRE) -- Alkeus Pharmaceuticals, Inc., a biopharmaceutical company dedicated to preserving the sight of individuals impacted by retinal diseases, today announced that results from its clinical study of investigational oral gildeuretinol for the treatment of moderate Stargardt disease (TEASE-2) will be presented at the 129th annual meeting of the American Academy of Ophthalmology (AAO) being held in Orlando, October 18-21, 2025. Oral presentation Safety and Efficacy of Oral Gildeuretinol in Participants with Intermediate-Stage Stargardt Disease Date/Time: Retina Subspecialty Day 2025, October 18, 8:23 a.m. – 8:30 a.m. EDTSession: Section XI: Late Breaking Developments, Part IILocation: Orange County Convention Center, OrlandoPresenter: Philip J. Ferrone, M.D., Vitreoretinal Consultants of New York About Alkeus Pharmaceuticals Alkeus Pharmaceuticals, Inc. is a private biopharmaceutical company dedicated to preserving the sight of individuals impacted by retinal diseases. Based in Cambridge, Mass., Alkeus is backed by institutional investors led by Bain Capital Life Sciences. Alkeus is developing therapies for serious diseases of the eye with high unmet need, with the purpose to preserve the sight of individuals impacted by retinal diseases. Alkeus’ breakthrough-designated lead candidate, gildeuretinol acetate (ALK-001), currently is being evaluated in clinical trials for the treatment of Stargardt disease. About Gildeuretinol Acetate (ALK-001) Oral gildeuretinol acetate (ALK-001) is a new molecular entity designed to reduce the dimerization of vitamin A without modulating the visual cycle. Gildeuretinol is being evaluated in clinical trials for the treatment of Stargardt disease and has been studied for geographic atrophy secondary to age-related macular degeneration. Gildeuretinol has received Breakthrough Therapy, Rare Pediatric Disease, Fast Track and Orphan Drug designations for Stargardt disease from the U.S. Food and Drug Administration (FDA). The European Medicines Agency (EMA) has designated gildeuretinol as an orphan medicinal product for the treatment of non-syndromic inherited retinal dystrophies due to defects in the ABCA4 gene, which includes Stargardt disease. About the TEASE Program The Tolerability and Effects of ALK-001 on Stargardt diseasE (TEASE) studies consist of four independent clinical studies of oral gildeuretinol (ALK-001) in Stargardt disease, denoted as TEASE-1, TEASE-2, TEASE-3 and TEASE-4. The TEASE-1 study was a randomized, double-masked, placebo-controlled trial in patients with advanced Stargardt disease. The TEASE-2 trial was a randomized, double-masked, placebo-controlled trial in patients with moderate Stargardt disease. TEASE-1 and TEASE-2 have been completed. TEASE-3 (NCT02402660), an ongoing clinical trial in early-stage Stargardt disease, is an open-label study of gildeuretinol in genetically confirmed patients with early signs of disease visible on retinal imaging, but who have not begun experiencing symptoms of vision loss. TEASE-4 (NCT04239625) is an open-label extension study. For further information, please contact: Media Relationsmedia@alkeuspharma.comWebsite: www.alkeuspharma.com

孤儿药快速通道临床结果

2025-08-08

·GlobeNewswire-Health Care

Alkeus Pharmaceuticals Announces Presentation of Positive Interim TEASE-3 Study Data in Early-Stage Stargardt Disease Patients Treated with Oral Gildeuretinol

Gildeuretinol acetate demonstrated less loss in ellipsoid zone (EZ) area compared to historical sibling controls. Early-stage Stargardt disease patients treated with gildeuretinol for two to seven years exhibited relatively stable disease over the course therapy. Gildeuretinol was well tolerated and had a safety profile consistent with prior gildeuretinol studies, with the majority of adverse events being mild or moderate. Aug. 07, 2025 -- Alkeus Pharmaceuticals, Inc. today announced the presentation of an interim data update from its TEASE-3 study demonstrating that five early-stage Stargardt disease patients treated with investigational oral gildeuretinol acetate experienced less loss in ellipsoid zone (EZ) area compared to historical sibling controls and overall disease remained relatively stable while on therapy ranging between two to seven years. The study update was presented during the 43rd American Society of Retina Specialists (ASRS) Annual Scientific Meeting, held July 30 – Aug. 2 in Long Beach, Calif. “We are encouraged by this new interim data from the TEASE-3 study of gildeuretinol in early-stage Stargardt patients with confirmed disease-causing ABCA4 genetic mutations,” said Seemi Khan, M.D., M.P.H., M.B.A., Chief Medical Officer of Alkeus Pharmaceuticals. “These additional data further support the potential of investigational gildeuretinol to slow disease progression and stabilize vision in individuals living with Stargardt disease. We are excited to continue advancing our efforts towards delivering a treatment for Stargardt disease.” TEASE-3, the first clinical trial in early-stage Stargardt disease, is an open-label study of gildeuretinol in genetically confirmed patients who exhibit early retinal changes on imaging but have not yet developed meaningful symptoms of vision loss. TEASE-3 study participants each have a sibling who was previously diagnosed with Stargardt disease. The disease progression over two years is assessed by retinal imaging and functional outcome measures. After the initial two-year treatment period, patients continue to receive gildeuretinol while enrolled in an open label long-term extension study. In the TEASE-3 study, five patients have completed two years of treatment with once-daily oral gildeuretinol. Gildeuretinol was well tolerated and demonstrated a safety profile consistent with prior gildeuretinol studies, with the majority of adverse events being mild or moderate in severity. “Time is of the essence for patients losing vision from early-onset Stargardt disease,” said Kenneth Fan, M.D., M.B.A., Retina Consultants of Texas. “With no approved treatments available, these data inform our understanding of retinal health in early-stage disease and underscore the importance of ongoing clinical evaluation.” Stargardt disease is a serious cause of blindness in children and adults, with an estimated 30,000 people affected in the U.S. and more than 150,000 worldwide. There is no approved treatment. In individuals with Stargardt disease, the ABCA4 protein is defective. This defect in the protein results in the accelerated dimerization of vitamin A, forming toxic by-products that irreversibly damage the retina, resulting in progressive vision loss. Alkeus Pharmaceuticals, Inc. is a private biopharmaceutical company dedicated to preserving the sight of individuals impacted by retinal diseases. Based in Cambridge, Mass., Alkeus is backed by institutional investors led by Bain Capital Life Sciences. Alkeus is developing therapies for serious diseases of the eye with high unmet need. Alkeus’ breakthrough-designated lead candidate, oral gildeuretinol acetate (ALK-001), is being evaluated in clinical trials for the treatment of Stargardt disease and for geographic atrophy secondary to age-related macular degeneration. Oral gildeuretinol acetate (ALK-001) is a new molecular entity designed to reduce the dimerization of vitamin A without modulating the visual cycle. Gildeuretinol is being evaluated in clinical trials for the treatment of Stargardt disease and for geographic atrophy secondary to age-related macular degeneration. Gildeuretinol has received Breakthrough Therapy, Fast Track, Orphan Drug and Rare Pediatric Disease designations for Stargardt disease from the U.S. Food and Drug Administration. The Tolerability and Effects of ALK-001 on Stargardt diseasE (TEASE) studies consist of four independent clinical studies of oral gildeuretinol acetate (ALK-001) in Stargardt disease, denoted as TEASE-1, TEASE-2, TEASE-3 and TEASE-4. The TEASE-1 study was a randomized, double-masked, placebo-controlled trial in 50 patients with advanced Stargardt disease and is complete. The TEASE-2 trial is a randomized, double-masked, placebo-controlled trial in 80 patients with moderate Stargardt disease, expected to read out topline data in 2025. TEASE-3, a clinical trial in early-stage Stargardt disease, is an ongoing open-label study of gildeuretinol in genetically confirmed patients with early signs of disease visible on retinal imaging, but who have not begun experiencing meaningful symptoms of vision loss. TEASE-4 is an ongoing open-label extension study. The content above comes from the network. if any infringement, please contact us to modify.

临床结果孤儿药快速通道突破性疗法临床研究

2025-07-23

·GlobeNewswire-Health Care

Alkeus Pharmaceuticals Announces Gildeuretinol Data Presentations During the American Society of Retina Specialists (ASRS) 43rd Annual Meeting July 29-August 2

CAMBRIDGE, Mass., July 23, 2025 (GLOBE NEWSWIRE) -- Alkeus Pharmaceuticals, Inc., a biopharmaceutical company dedicated to preserving the sight of individuals impacted by retinal diseases, today announced that data from its clinical studies of investigational oral gildeuretinol for the treatment of Stargardt disease (TEASE) and for geographic atrophy secondary to age-related macular degeneration (SAGA) will be presented during the American Society of Retina Specialists (ASRS) 43rd Annual Scientific Meeting being held July 29 to Aug. 2, 2025, in Long Beach, Calif. Oral Presentation: Oral Gildeuretinol Slows Disease Progression in Early Stargardt Disease: Updates From the TEASE-3 StudyDate: Aug. 1, 2025Session: Hereditary Retinal Disease & Genetics Symposium 1Time: 4:53 p.m. – 4:56 p.m.Location: Hall B, Long Beach Convention CenterPresenter: Kenneth Fan, M.D., M.B.A., Retina Consultants of Texas Paper on Demand (Recorded Video Presentation) Safety Profile of Oral Gildeuretinol Acetate: Safety Results Across TEASE-1, TEASE-3, and SAGA Clinical Studies Date: Available on demand for meeting registrantsPresenter: Mathew MacCumber, M.D., Ph.D., FASRS, Illinois Retina Associates About Alkeus Pharmaceuticals Alkeus Pharmaceuticals, Inc. is a private biopharmaceutical company dedicated to preserving the sight of individuals impacted by retinal diseases. Based in Cambridge, Mass., Alkeus is backed by institutional investors led by Bain Capital Life Sciences. Alkeus is developing therapies for serious diseases of the eye with high unmet need. Alkeus’ breakthrough-designated lead candidate, gildeuretinol acetate (ALK-001), is a new molecular entity being evaluated in clinical trials for the treatment of Stargardt disease and for geographic atrophy secondary to age-related macular degeneration. About Gildeuretinol Acetate (ALK-001) Oral gildeuretinol acetate (ALK-001) is a new molecular entity designed to reduce the dimerization of vitamin A without modulating the visual cycle. Gildeuretinol is being evaluated in clinical trials for the treatment of Stargardt disease and for geographic atrophy secondary to age-related macular degeneration. Gildeuretinol has received Breakthrough Therapy, Fast Track, Orphan Drug and Rare Pediatric Disease designations for Stargardt disease from the U.S. Food and Drug Administration. About the TEASE Program The Tolerability and Effects of ALK-001 on Stargardt diseasE (TEASE) studies consist of four independent clinical studies of oral gildeuretinol acetate (ALK-001) in Stargardt disease, denoted as TEASE-1, TEASE-2, TEASE-3 and TEASE-4. The TEASE-1 study was a randomized, double-masked, placebo-controlled trial in 50 patients with advanced Stargardt disease and is complete. The TEASE-2 trial is a randomized, double-masked, placebo-controlled trial in 80 patients with moderate Stargardt disease, expected to read out topline data in 2025. TEASE-3, a clinical trial in early-stage Stargardt disease, is an ongoing open-label study of gildeuretinol in genetically confirmed patients with early signs of disease visible on retinal imaging, but who have not begun experiencing symptoms of vision loss. TEASE-4 is an ongoing open-label extension study. About the SAGA study The Study of ALK-001 in GA secondary to age-related macular degeneration (SAGA) was a 24-month, double-masked, randomized, placebo-controlled trial to investigate safety, pharmacokinetics, tolerability and efficacy in 198 patients with geographic atrophy secondary to age-related macular degeneration and is complete. For further information, contact: Media@alkeuspharma.comWebsite: www.alkeuspharma.com

快速通道孤儿药临床结果

100 项与 Gildeuretinol acetate 相关的药物交易

登录后查看更多信息

研发状态

10 条进展最快的记录,

后查看更多信息

适应症	最高研发状态	国家/地区	公司	日期
干性年龄相关性黄斑病变	临床3期	美国	Alkeus Pharmaceuticals, Inc.	2019-05-07
地图样萎缩	临床3期	美国	Alkeus Pharmaceuticals, Inc.	2019-05-07
II型糖原贮积病	临床3期	美国	Alkeus Pharmaceuticals, Inc.	2019-05-07
斯塔加特病	临床2期	美国	Alkeus Pharmaceuticals, Inc.	2015-08-01
视网膜变性疾病	临床1期	-	Alkeus Pharmaceuticals, Inc.	2014-04-01

登录后查看更多信息

临床结果

适应症

分期

评价

查看全部结果

研究	分期	人群特征	评价人数	分组	结果	评价	发布日期


NCT03845582 (SAGA, CTgov)	临床2/3期	干性年龄相关性黄斑病变 \| 年龄相关性黄斑变性 \| 地图样萎缩 ... 更多	200	ALK-001 oral capsule (ALK-001)	顧願衊膚構構淵齋淵蓋(夢鏇遞膚醖獵網齋襯簾) = 鹽衊顧齋選憲襯獵獵鏇淵膚積鹹繭夢膚餘鑰壓 (鹹選積衊蓋膚積構構顧, 0.081) 更多	-	2025-10-16
				Placebo oral capsule (Placebo)	顧願衊膚構構淵齋淵蓋(夢鏇遞膚醖獵網齋襯簾) = 築壓憲獵廠願夢鹹餘壓淵膚積鹹繭夢膚餘鑰壓 (鹹選積衊蓋膚積構構顧, 0.116) 更多
NCT04239625 (TEASE-3, GlobeNewswire) 人工标引	临床1/2期	斯塔加特病	5	Gildeuretinol	壓夢糧鏇膚構觸鏇製網(鏇選築築選鹽蓋顧繭願) = 顧廠構憲鬱遞淵膚襯蓋齋壓網鹽鹹糧鑰餘遞鑰 (築膚蓋觸網鹽遞選觸蓋 )	积极	2025-08-07
NCT03845582 (SAGA, Company_Website) 人工标引	临床3期	地图样萎缩	198	Gildeuretinol	衊鏇夢獵糧鑰襯鏇獵鏇(淵壓鏇觸糧願膚窪鑰遞) = 願膚淵醖鹹艱鬱鏇窪窪夢築壓網憲蓋糧襯衊範 (網獵淵蓋遞夢醖鬱鹹膚 ) 更多	积极	2024-10-23
				Placebo	窪獵鹹鑰壓艱範艱夢觸(餘製網襯窪衊簾範顧膚) = 膚顧簾網遞遞繭願壓鬱淵選鹽築壓壓餘顧積顧 (積遞選範獵夢鏇鏇鹽築 )
NCT03845582 (SAGA, GlobeNewswire) 人工标引	临床3期	年龄相关性黄斑变性 \| 地图样萎缩	-	gildeuretinol acetate	醖憲壓網鹹獵顧鬱夢鏇(築製鏇鏇鹹糧遞範夢襯) = demonstrated a reduction of 0.25 sqmm/year vs. placebo (p=0.07) 鏇鏇鏇網鹽衊鬱範簾廠 (鬱繭廠願顧襯顧範築鹽 ) 更多	积极	2024-09-17
				Placebo
NCT04239625 (TEASE-3, Corporate Publications \| GlobeNewswire 1 \| GlobeNewswire 2) 人工标引	N/A	斯塔加特病	5	ALK-001	鑰蓋壓壓鹽鏇壓製網艱(顧願糧鹽願鹹築艱蓋獵) = A total of five patients have completed the 24-month study. Following the study, these patients have remained on treatment and continue to show no disease progression, with the longest duration of therapy to date being more than seven years. 齋製繭積糧簾淵餘獵夢 (觸觸選鏇鑰鹽淵網簾獵 )	积极	2024-01-10
NCT02402660 (TEASE-1, Biospace) 人工标引	临床2期	斯塔加特病	50	ALK-001	鹹製齋構鹽糧襯獵網衊(願構鏇築憲鏇鬱顧廠築): Difference(%) = -21, P-Value = <0.001 更多	积极	2023-11-03
				Placebo
TEASE (ARVO2017)	临床2期	斯塔加特病 ABCA4 gene	50	ALK-001	膚積醖膚鬱製糧淵衊夢(鑰憲鏇積憲膚壓鹹築蓋) = 積廠夢衊網範構窪憲鹹蓋衊築窪憲夢醖簾艱積 (鑰遞膚鏇築鬱鏇糧製膚, 8 ~ 91) 更多	-	2017-06-01
NCT02402660 (TEASE, ARVO2016)	临床2期	斯塔加特病	50	ALK-001	糧廠夢觸顧鏇獵獵範願(鑰壓選鹽鏇鹽醖衊鹽夢) = 艱鹽憲獵繭壓糧積顧範衊憲網範鹽鹽鬱築壓構 (範選鏇簾鹹齋膚壓獵膚 ) 更多	积极	2016-09-01