RegulationBreakthrough Therapy (United States), Orphan Drug (United States), Rare Pediatric Disease (United States), Orphan Drug (European Union), Fast Track (United States)

Structure/Sequence

Molecular FormulaC22H32O2

InChIKeyQGNJRVVDBSJHIZ-BMSJAHLVSA-N

CAS Registry118139-36-9

Clinical Trials associated with Gildeuretinol acetate

NCT07419334

/ RecruitingPhase 3

A Phase 3 Multicenter, Double-Masked, Randomized, Placebo-Controlled Study to Investigate the Safety, Tolerability, and Efficacy of Gildeuretinol Acetate (ALK 001) on the Progression of Stargardt Disease (STGD)

This study evaluates the efficacy and safety of investigational study drug ALK-001 in participants 8 to 45 years of age, inclusive, with symptoms and signs of autosomal recessive Stargardt disease (STGD)

Start Date01 Apr 2026

Sponsor / Collaborator

Alkeus Pharmaceuticals, Inc.

NCT04239625

/ Active, not recruitingPhase 2

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

Start Date20 Dec 2019

Sponsor / Collaborator

Alkeus Pharmaceuticals, Inc.

NCT03845582

/ CompletedPhase 2/3

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.

Start Date07 May 2019

Sponsor / Collaborator

Alkeus Pharmaceuticals, Inc.

100 Clinical Results associated with Gildeuretinol acetate

100 Translational Medicine associated with Gildeuretinol acetate

100 Patents (Medical) associated with Gildeuretinol acetate

Literatures (Medical) associated with Gildeuretinol acetate

01 Nov 2023·Current medicinal chemistry

Clinical Application and Synthesis Methods of Deuterated Drugs

Article

Author: 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.

01 Sep 2021·The Journal of biological chemistryQ2 · BIOLOGY

Vitamin A cycle byproducts impede dark adaptation

Q2 · BIOLOGY

Article

Author: Dan Zhang ; Kiera Robinson ; Leonide Saad ; Ilyas Washington

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.

01 Jan 2020·Topics in Medicinal Chemistry

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

Author: Petrukhin, Konstantin

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.

News (Medical) associated with Gildeuretinol acetate

24 Feb 2026

·www.biospace.com

Alkeus Pharmaceuticals Announces Presentation of Investigational Oral Gildeuretinol Data During the 49th Annual Meeting of the Macula Society Being Held February 25-28

CAMBRIDGE, Mass., Feb. 24, 2026 (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 Stargardt disease (TEASE) will be presented at the 49th Annual Meeting of the Macula Society being held February 25-28, 2026, in San Diego. Oral Presentation: Safety and Efficacy of Oral Gildeuretinol in Participants with Moderate Stargardt Disease Session: Inherited Dystrophy 1: Clinical Trials and Beyond Date: February 27, 2026 Time: 7:20 a.m. – 7:25 a.m. PST Location: Southpoint Ballroom, Southpoint Event Center, Hotel del Coronado Presenter: SriniVas R. Sadda, M.D., F.A.R.V.O., Doheny Eye Institute, and Professor of Ophthalmology at the University of California – Los Angeles (UCLA) Geffen School of Medicine 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. 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), 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. For more information contact: Media Relations Media@alkeuspharma.com Website:

Orphan DrugFast TrackBreakthrough TherapyClinical Study

04 Dec 2025

·www.globenewswire.com

Alkeus Pharmaceuticals Announces Gildeuretinol Data Will Be Presented During the 13th International FLORetina ICOOR Congress December 4–7 in Florence, Italy

CAMBRIDGE, Mass., Dec. 04, 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 and Geographic Atrophy (GA) will be presented at the 13th International FLORetina ICOOR Congress being held December 4-7 at the Fortezza de Basso in Florence, Italy. Oral Presentations: Gildeuretinol in Stargardt Disease: Results from the TEASE-2 ProgramSession: Retina Futura: Spotlight on Emerging Therapies for IRDsDate: December 6, 2025Time: 11:02 a.m. – 11:06 a.m. CET Location: Santa Croce Room, Fortezza de BassoPresenter: Philip Ferrone, M.D., Vitreoretinal Consultants of New York Safety and Efficacy of Oral Gildeuretinol in Geographic Atrophy: Results of SAGA, a Phase 2/3 Clinical StudySession: Retina Futura: Highlights in GA: Current clinical updates and emerging therapiesDate: December 6, 2025Time: 4:25 p.m. – 4:29 p.m. CET Location: San Giovanni Room, Fortezza de BassoPresenter: Dilsher Dhoot, M.D., FASRS, California Retina ConsultantsAbout 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), 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. About the SAGA study The Study of ALK-001 in GA secondary to age-related macular degeneration (SAGA) (NCT03845582) 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, please contact: Media Relationsmedia@alkeuspharma.comWebsite: www.alkeuspharma.com

Orphan DrugFast TrackClinical ResultBreakthrough Therapy

09 Oct 2025

·www.globenewswire.com

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

Orphan DrugFast TrackClinical Result

100 Deals associated with Gildeuretinol acetate

R&D Status

10 top R&D records.

to view more data

Indication	Highest Phase	Country/Location	Organization	Date
Stargardt Disease	Phase 3	United States	Alkeus Pharmaceuticals, Inc.	01 Apr 2026
dry age-related macular degeneration	Phase 3	United States	Alkeus Pharmaceuticals, Inc.	07 May 2019
Geographic Atrophy	Phase 3	United States	Alkeus Pharmaceuticals, Inc.	07 May 2019
Glycogen Storage Disease Type II	Phase 3	United States	Alkeus Pharmaceuticals, Inc.	07 May 2019
Retinal Dystrophies	Phase 1	-	Alkeus Pharmaceuticals, Inc.	01 Apr 2014

Clinical Result

Indication

Phase

Evaluation

View All Results

Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT03845582 (SAGA, CTgov)	Phase 2/3	dry age-related macular degeneration \| Age Related Macular Degeneration \| Geographic Atrophy ... View more	200	ALK-001 oral capsule (ALK-001)	eplkzizpcb(bthypmzyym) = ffvhygimvi omiwpuqegl (bcdhlsvkrr, 0.081) View more	-	16 Oct 2025
				Placebo oral capsule (Placebo)	eplkzizpcb(bthypmzyym) = jwcavdzcbb omiwpuqegl (bcdhlsvkrr, 0.116) View more
NCT04239625 (TEASE-3, GlobeNewswire) Manual	Phase 1/2	Stargardt Disease	5	Gildeuretinol	envuxerxut(huesepyloq) = aacmrwelzu wnyxbzbejj (dblifsdqry )	Positive	07 Aug 2025
NCT03845582 (SAGA, ARVO2025)	Phase 3	Age Related Macular Degeneration	198	Gildeuretinol 14 mg/day	vtaxhddmsm(xcjxqnlwoy) = bywfdeepnk rwmqwstqen (rgzyjlqave ) View more	Positive	04 May 2025
				Placebo	-
NCT03845582 (SAGA, Company_Website) Manual	Phase 3	Geographic Atrophy	198	Gildeuretinol	fnypkzpqdl(calerodism) = sdaszhssxh inkaxwuccg (bzsykurvcs ) View more	Positive	23 Oct 2024
				Placebo	xdtwswgeqc(rhtrajivez) = dzquscfxgc lhssporqct (ofxiahyyvg )
NCT03845582 (SAGA, GlobeNewswire) Manual	Phase 3	Age Related Macular Degeneration \| Geographic Atrophy	-	gildeuretinol acetate	hqrwtfuiet(kwxgjchrso) = demonstrated a reduction of 0.25 sqmm/year vs. placebo (p=0.07) ozannicubj (rntmgiewbl ) View more	Positive	17 Sep 2024
				Placebo
NCT04239625 (TEASE-3, Corporate Publications \| GlobeNewswire 1 \| GlobeNewswire 2) Manual	Not Applicable	Stargardt Disease	5	ALK-001	psoasyxquz(cdrmdiosvv) = 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. qarqkkxlja (rcmvaqtwrt )	Positive	10 Jan 2024
NCT02402660 (TEASE-1, Biospace) Manual	Phase 2	Stargardt Disease	50	ALK-001	zxwefcnref(kevovqkbir): Difference(%) = -21, P-Value = <0.001 View more	Positive	03 Nov 2023
				Placebo
TEASE (ARVO2017)	Phase 2	Stargardt Disease ABCA4 gene	50	ALK-001	fmipttlppo(escrqbihaq) = ejeaoiuhby zszqhuhlgv (baqcpwwyza, 8 - 91) View more	-	01 Jun 2017
NCT02402660 (TEASE, ARVO2016)	Phase 2	Stargardt Disease	50	ALK-001	sfowmjqrww(uverbkttck) = cisnrnhmee tdcekuhcvu (aqanlrsouu ) View more	Positive	01 Sep 2016

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