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 Jun 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 May 2026·CURRENT OPINION IN OPHTHALMOLOGY

Nutritional supplements: current evidence for retinitis pigmentosa and Stargardt disease

Review

Author: Gregori, Ninel Z. ; Barthelemy, Normila ; Sengillo, Jesse D. ; Lee, Winston ; Lam, Byron L.

Purpose of review:

Inherited retinal diseases (IRDs) are genetically and phenotypically heterogeneous disorders that cause progressive vision loss and lack broadly effective disease-modifying therapies. Increasing evidence implicates metabolic stress, oxidative injury, and photoreceptor–retinal pigment epithelium (RPE) dysfunction in IRD pathophysiology. This review evaluates the evidence and limitations surrounding nutritional and metabolic interventions for retinitis pigmentosa and Stargardt disease.A narrative review of preclinical studies, randomized controlled trials, and contemporary genetic re-analyses was performed. Nutritional interventions reviewed include vitamin A, vitamin E, N-acetylcysteine (NAC), omega-3 fatty acids (docosahexaenoic acid), carotenoids and apocarotenoids, and the deuterated vitamin A analog C20-D3-retinyl acetate (ALK-001).

Recent findings:

Initial studies suggesting a protective effect of vitamin A in retinitis pigmentosa were not confirmed in subsequent trials or genetic re-analyses, which identified baseline differences between groups as a possible contributor to the original findings. In contrast, supplemental vitamin E may accelerate disease progression. NAC demonstrated acceptable tolerability and modest short-term improvements in visual function in early-phase trials, with a phase 3 study ongoing. Omega-3 fatty acids, carotenoids, and apocarotenoids have not shown consistent clinically meaningful benefit in retinitis pigmentosa or Stargardt disease despite strong mechanistic rationale. In Stargardt disease, ALK-001 reduces lipofuscin accumulation in animal models and clinical trials are ongoing to assess efficacy in patients.

Summary:

Most nutritional supplements studied to date have not demonstrated durable or clinically meaningful benefit in IRDs. Vitamin A supplementation in retinitis pigmentosa is no longer supported by current evidence, while NAC remains a promising metabolic therapy under investigation. High-quality, genotype-informed clinical trials with clinically relevant endpoints are needed before nutritional interventions can be incorporated into IRD management guidelines.

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.

News (Medical) associated with Gildeuretinol acetate

30 Apr 2026

·www.biospace.com

Alkeus Pharmaceuticals Announces Presentation of Gildeuretinol Data During the Association for Research in Vision and Ophthalmology (ARVO) 2026 Annual Meeting

CAMBRIDGE, Mass., April 30, 2026 (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) will be presented at the 2026 annual meeting of the Association for Research in Vision and Ophthalmology (ARVO) being held May 3-7 in Denver. Poster Presentation Baseline Characteristics from the Tolerability and Effects of ALK-001 on Stargardt Disease (TEASE) Clinical Program Date: May 7, 2026 Time: 8:00 a.m. to 9:45 a.m. MDT Posterboard Number: 5370 - 0646 Session Number: 512 Session Title: IRD II Presenter: Ryan Yanagihara, M.D., Retina Consultants of Texas About Stargardt Disease Stargardt disease causes severe vision impairment and blindness, primarily in children and young adults, with an estimated 37,000 to 87,000 people affected in the U.S. There is no FDA-approved treatment. In individuals with Stargardt disease, the ABCA4 protein is defective. This defect in the protein results in the accumulation of toxic vitamin A dimers that irreversibly damage the retina, resulting in progressive vision loss. 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. 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 50 patients with Stargardt disease and is complete. The TEASE-2 trial was a randomized, double-masked, placebo-controlled trial in 80 patients with moderate Stargardt disease and is also complete. TEASE-3, the 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 is an open-label extension study. For further information, contact: Media@alkeuspharma.com Website:

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

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 Jun 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

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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)	aigsitcqlt(rtxjqgitbx) = fndjlqnxhc vvzlvvttxb (bdwpfmamxo, 0.081) View more	-	16 Oct 2025
				Placebo oral capsule (Placebo)	aigsitcqlt(rtxjqgitbx) = rrgfehnnhw vvzlvvttxb (bdwpfmamxo, 0.116) View more
NCT04239625 (TEASE-3, GlobeNewswire) Manual	Phase 1/2	Stargardt Disease	5	Gildeuretinol	zdvdcnfxqn(xrghdaouyz) = bzbiiprhki cgcniqftno (mziyoaktzh )	Positive	07 Aug 2025
NCT03845582 (SAGA, ARVO2025)	Phase 3	Age Related Macular Degeneration	198	Gildeuretinol 14 mg/day	qxzxwalkcw(cnlvgaeyaf) = eeviukpsxi ztbjaccymf (etnaottilv ) View more	Positive	04 May 2025
				Placebo	-
NCT03845582 (SAGA, Company_Website) Manual	Phase 3	Geographic Atrophy	198	Gildeuretinol	xmmppbahhw(xcmibwgodl) = fcouxissbt budnjzwpkz (webbaismeg ) View more	Positive	23 Oct 2024
				Placebo	eakhvqgfzf(tuariyrnqf) = fqdvnzordr lsgegkrish (nduaslrxun )
NCT03845582 (SAGA, GlobeNewswire) Manual	Phase 3	Age Related Macular Degeneration \| Geographic Atrophy	-	gildeuretinol acetate	vlrgrmdqkv(ndojfmpklq) = demonstrated a reduction of 0.25 sqmm/year vs. placebo (p=0.07) jkeezghvpz (mudebhepeq ) View more	Positive	17 Sep 2024
				Placebo
NCT04239625 (TEASE-3, Corporate Publications \| GlobeNewswire 1 \| GlobeNewswire 2) Manual	Not Applicable	Stargardt Disease	5	ALK-001	vyyrcritau(lkysdbgqhn) = 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. cegocbtazc (syemyuexda )	Positive	10 Jan 2024
NCT02402660 (TEASE-1, Biospace) Manual	Phase 2	Stargardt Disease	50	ALK-001	taqxbhpwsh(sqwhvjfxle): Difference(%) = -21, P-Value = <0.001 View more	Positive	03 Nov 2023
				Placebo
TEASE (ARVO2017)	Phase 2	Stargardt Disease ABCA4 gene	50	ALK-001	gaknnmynpm(ywygzefbjj) = vytrbwalio olblqrbvbv (zfptnksgde, 8 - 91) View more	-	01 Jun 2017
NCT02402660 (TEASE, ARVO2016)	Phase 2	Stargardt Disease	50	ALK-001	ugimacmgch(thcizmiuri) = zeggjvgovv feikibymer (bauuhdmqat ) View more	Positive	01 Sep 2016

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