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 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.

NCT02402660

/ Enrolling by invitationPhase 2

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

Start Date01 Aug 2015

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.

27 Sep 2023·Journal of clinical medicine

Updates on Emerging Interventions for Autosomal Recessive ABCA4-Associated Stargardt Disease.

Review

Author: Gregori, Ninel Z ; Mangwani-Mordani, Simran ; Shah, Serena M ; Wang, Liang

Autosomal recessive Stargardt disease (STGD1) is an inherited retinal degenerative disease associated with a mutated ATP-binding cassette, subfamily A, member 4 (ABCA4) gene. STGD1 is the most common form of juvenile macular degeneration with onset in late childhood to early or middle adulthood and causes progressive, irreversible visual impairment and blindness. No effective treatment is currently available. In the present article, we review the most recent updates in clinical trials targeting the management of STGD1, including gene therapy, small molecule therapy, and stem cell therapy. In gene therapy, dual adeno-associated virus and non-viral vectors have been successful in delivering the human ABCA4 gene in preclinical studies. For pharmaceutical therapies ALK-001, deuterated vitamin A shows promise with preliminary data for phase 2 trial, demonstrating a decreased atrophy growth rate after two years. Stem cell therapy using human pluripotent stem cell-derived retinal pigment epithelium cells demonstrated long-term safety three years after implantation and visual acuity improvements in the first two years after initiation of therapy. Many other treatment options have ongoing investigations and clinical trials. While multiple potential interventions have shown promise in attenuating disease progression, further exploration is necessary to demonstrate treatment safety and efficacy.

01 Sep 2021·Journal of Biological ChemistryQ2 · BIOLOGY

Vitamin A cycle byproducts impede dark adaptation

Q2 · BIOLOGY

Article

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

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

24 Apr 2025

·www.globenewswire.com

Alkeus Pharmaceuticals Announces Presentations of Gildeuretinol Data During the Research in Vision and Ophthalmology (ARVO) 2025 Annual Meeting May 4-8, 2025

CAMBRIDGE, Mass., April 24, 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 studies of investigational oral gildeuretinol for the treatment of Stargardt disease (TEASE) and geographic atrophy secondary to age-related macular degeneration (SAGA) will be presented at the 2025 annual meeting of the Association for Research in Vision and Ophthalmology (ARVO) being held May 4-8 in Salt Lake City. Oral Presentations: Safety and Efficacy of Oral Gildeuretinol for Stargardt Disease: TEASE-1 TrialDate: May 5, 2025Time: 4:30 p.m. to 4:45 p.m. MDTPresentation Number: 1946Session: Session 242: Retinitis pigmentosa and IRDLocation: Calvin L. Rampton Salt Palace Convention Center, Ballroom JPresenter: Benjamin Bakall, M.D., Ph.D., Associated Retina ConsultantsOral Gildeuretinol Treatment in Geographic Atrophy Secondary to AMD in the SAGA Trial: Safety, Efficacy and Patient-reported OutcomesDate: May 6, 2025Time: 4:45 p.m. to 5:00 p.m. MDTPresentation Number: 3623Session: 349: AMD Clinical research ILocation: Calvin L. Rampton Salt Palace Convention Center, Room: 255ABCPresenter: David Boyer, M.D., Retina-Vitreous Associates Medical Group 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), is a new molecular entity currently 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 currently 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, Rare Pediatric Disease, Fast Track and Orphan Drug 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 (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 is an ongoing, fully enrolled, randomized, double-masked, placebo-controlled trial in 80 patients with moderate Stargardt disease, expected to read out topline data in 2025. 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. 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.com Website: www.alkeuspharma.com

Orphan DrugFast TrackClinical ResultBreakthrough Therapy

13 Feb 2025

·www.globenewswire.com

Alkeus Pharmaceuticals Announces Two Presentations of Oral Gildeuretinol Data During the 48th Annual Meeting of the Macula Society Being Held February 12-15, 2025

Feb. 11, 2025 -- Alkeus Pharmaceuticals, Inc., a biopharmaceutical company dedicated to preserving the sight of individuals impacted by retinal diseases, today announced that results from its clinical studies of investigational oral gildeuretinol for the treatment of Stargardt disease (TEASE) and geographic atrophy secondary to age-related macular degeneration (SAGA) will be presented at the 48th Annual Meeting of the Macula Society being held February 12-15 in Charlotte Harbor, Fla. Oral Presentations: Oral Gildeuretinol Slows Progression of Stargardt Disease: Safety and Efficacy Results from The TEASE Program Session: INHERITED RETINAL DYSTROPHY I: TRIALS Date: February 13, 2025 Time: 7:35 a.m. – 7:40 a.m. EST Location: Great Egret Ballroom Presenter: Philip Ferrone, M.D., Vitreoretinal Consultants of New York Oral Gildeuretinol in Geographic Atrophy Secondary to AMD: Safety and Efficacy from SAGA, a 2-Year, Randomized, Double-Masked, Placebo-Controlled Study Session: NON-NV AMD I: TRIALS Date: February 13, 2025 Time: 8:15 a.m. – 8:20 a.m. EST Location: Great Egret Ballroom Presenter: Ivan Jose Suner, M.D., M.B.A., Retina Associates of Florida 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. Gildeuretinol met its prespecified primary efficacy endpoint showing a 21.6% reduction in the growth rate of retinal atrophic lesions area (square root) (p<0.001), and a 29.5% reduction for untransformed areas of retinal atrophic lesions against untreated patients. Gildeuretinol was well-tolerated. The TEASE-2 trial is an ongoing, fully enrolled, randomized, double-masked, placebo-controlled trial in 80 patients with moderate Stargardt disease, expected to read out topline data in 2025. TEASE-3, the first 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. The Study of ALK-001 in GA (SAGA) was a 24-month, double-masked, randomized, placebo-controlled trial to investigate safety, pharmacokinetics, tolerability and efficacy in patients with GA secondary to AMD. The study enrolled 198 patients. The primary efficacy endpoint was the growth rate of GA lesions from baseline to 24 months as assessed by Fundus Autofluorescence (FAF). The first key secondary endpoint was the change in low luminance visual acuity (LLVA) from baseline to 24 months. Oral gildeuretinol acetate (ALK-001) is a new molecular entity designed to reduce the dimerization of vitamin A without modulating the visual cycle. In preclinical studies, gildeuretinol decreased vitamin A dimerization down to the normal rate and prevented retinal degeneration and loss of visual function in animals with Stargardt disease. A randomized, placebo-controlled, double-masked clinical trial of gildeuretinol in late-stage Stargardt patients (TEASE-1) showed clinically and statistically significant slowing of the growth of retinal lesions over two years of treatment. Additional clinical trials of gildeuretinol in Stargardt disease are ongoing. 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. A study (SAGA) of gildeuretinol in 198 patients with geographic atrophy (GA) secondary to age-related macular degeneration (AMD) demonstrated a meaningful trend in the reduction of lesion growth rate and demonstrated a functional benefit in low luminance visual acuity (LLVA). In studies, gildeuretinol demonstrated a favorable safety and tolerability profile. Alkeus Pharmaceuticals, Inc. is a private biopharmaceutical company with headquarters in Cambridge, Mass., backed by institutional investors led by Bain Capital Life Sciences. Founded in 2010, 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), is a new chemical entity currently being evaluated in clinical trials for the treatment of Stargardt disease and for geographic atrophy (GA) secondary to age-related macular degeneration (AMD). The content above comes from the network. if any infringement, please contact us to modify.

Clinical ResultFast TrackOrphan DrugBreakthrough Therapy

23 Jan 2025

·www.prnewswire.com

70+ Key Companies Advancing in Dry Age-Related Macular Degeneration Clinical Trial Therapeutic Space | DelveInsight

Dry macular degeneration is a common eye disorder among people aged above 50 years. It causes blurred or reduced central vision due to thinning of the macula. The macula is the part of the retina responsible for clear vision in a direct line of sight. The pharmaceutical industry is increasingly focused on developing pathway-based therapies for dry AMD, with promising treatments like ALK-001 and LBS-008 in trials, though a combination approach may be needed to fully inhibit disease progression. LAS VEGAS, Jan. 23, 2025 /PRNewswire/ -- DelveInsight's ' Dry Age-Related Macular Degeneration Pipeline Insight 2025 ' report provides comprehensive global coverage of pipeline dry AMD therapies in various stages of clinical development, major pharmaceutical companies are working to advance the pipeline space and future growth potential of the dry AMD pipeline domain. Key Takeaways from the Dry Age-Related Macular Degeneration Pipeline Report DelveInsight's dry AMD pipeline report depicts a robust space with 70+ active players working to develop 80+ pipeline therapies for dry AMD treatment. Key dry AMD companies such as Alkeus Pharmaceuticals, Dobecure, Belite Bio, Cognition Therapeutics, Aviceda Therapeutics, Stealth BioTherapeutics, Allergo Ophthalmics, Annexon, Inc., Johnson & Johnson, InflammX Therapeutics, Lineage Cell Therapeutics, Ionis Pharmaceuticals, Evergreen Therapeutics, Inc., Alexion, Luxa Biotechnology, Astellas Pharma, OliX Pharmaceuticals, Hoffmann-La Roche, Boehringer Ingelheim, Eyevensys and others are evaluating new dry AMD drugs to improve the treatment landscape. Promising dry AMD pipeline therapies such as ALK-001, Etamsylate, Tinlarebant (LBS-008), CT1812, AVD-104, Elamipretide, Risuteganib, ANX 007, JNJ 81201887, Xiflam, OpRegen, IONIS-FB-LRx, EG-301, ALXN2040, RPESC-RPE-4W, MA09-hRPE, OLX301A, RO7303359, BI 771716, EYS 611 and others are under different phases of dry AMD clinical trials. In December 2024, Galimedix Therapeutics announced the initiation of its Phase II eDREAM study to assess the safety and efficacy of GAL-101 eye drops in patients with dry age-related macular degeneration. In December 2024, Ocugen, Inc. announced that the Data and Safety Monitoring Board (DSMB) for the OCU410 ArMaDa clinical trial recently convened and approved continuation of the second phase of the Phase 1/2 study. OCU410 (AAV5-hRORA) is a novel modifier gene therapy candidate being developed for geographic atrophy (GA) secondary to dry age-related macular degeneration (dAMD). In November 2024, Ocugen, Inc. announced positive preliminary efficacy and safety data from the Phase I dose-escalation portion of the Phase 1/2 OCU410 ArMaDa clinical trial for geographic atrophy (GA), secondary to dry age-related macular degeneration (dAMD). Key findings include: no drug-related serious adverse events, reduced lesion growth, preservation of retinal tissue, and most importantly there was a positive effect on the functional visual measure of low luminance visual acuity (LLVA). In October 2024, Eyestem Research, focused on creating scalable cell therapies for incurable diseases, had announced a positive outcome of the Drug Safety Monitoring Board (DSMB) review for the first cohort of its Phase one trial to treat Geographic Atrophy secondary to Dry Age-related Macular Degeneration, marking a milestone for Indian science and medical research. In September 2024, Stealth BioTherapeutics Inc. announced the presentation of new bevemipretide (SBT-272) preclinical data demonstrating topical ocular delivery to the retina with protective effects observed in models of AMD. In June 2024, Stealth BioTherapeutics Inc. announced that it has enrolled and dosed its first patient in the ReNEW trial as part of its Phase III clinical program for elamipretide in patients with dry age-related macular degeneration (dry AMD). The program consists of the two Phase III trials, ReNEW and ReGAIN. Both trials will evaluate the efficacy, safety, and pharmacokinetics of daily subcutaneous injections of elamipretide in participants with dry AMD. In April 2024, Eyestem received approval from India's Central Drugs Standards Control Organisation (CDSCO) to commence human trials for Eyecyte RPE, a potential treatment for geographic atrophy arising from dry age-related macular degeneration (AMD). Request a sample and discover the recent advances in dry AMD treatment drugs @ Dry Age-Related Macular Degeneration Pipeline Report The dry AMD pipeline report provides detailed profiles of pipeline assets, a comparative analysis of clinical and non-clinical stage dry AMD drugs, inactive and dormant assets, a comprehensive assessment of driving and restraining factors, and an assessment of opportunities and risks in the dry AMD clinical trial landscape. Dry Age-Related Macular Degeneration Overview Age-related macular degeneration (AMD) is one of the top four causes of blindness in older adults and the leading cause of irreversible blindness in developed countries. Around 170 million people globally are affected by AMD, making it the third most common cause of vision loss worldwide. Dry AMD typically progresses slowly, but wet AMD can cause severe visual impairment within days or weeks of onset. In the early stages of AMD, there are no noticeable symptoms, though an ophthalmologist can detect drusen before symptoms arise. Some individuals experience slight vision changes during the intermediate stage, but many remain symptom-free. Others may notice dark or grey spots in their central vision or have difficulty transitioning from bright to dim lighting. Clinicians currently use various imaging techniques to monitor changes in the retina, retinal pigment epithelium (RPE), and choroid, including fundus exams, color fundus photography, fundus autofluorescence (FAF), optical coherence tomography (OCT), and infrared reflectance imaging. Over the past two decades, OCT has significantly advanced the early detection, monitoring, and treatment assessment of dry AMD. Modern spectral-domain OCT (SD-OCT) systems provide detailed cross-sectional and volumetric views of the retina, enabling clinicians to visualize, measure, and monitor retinal layers, RPE, hyperreflective foci, geographic atrophy (GA), and drusen. While SD-OCT remains the standard of care, emerging technologies like polarization-sensitive OCT (PS-OCT), which offers tissue-selective imaging, may revolutionize AMD research and clinical practices. Currently, no approved treatments exist for dry AMD. Management involves regular monitoring, timely detection of visual deterioration, appropriate rehabilitation, and early identification of choroidal neovascularization (CNV). Research is underway to explore therapeutic strategies to prevent AMD, slow its progression, or restore vision. Potential treatments include lifestyle and behavioral changes, age-related eye disease study (AREDS) supplements, visual cycle modulators, anti-inflammatory agents, complement inhibitors, gene therapy, cell-based therapies, and approaches to reduce toxic by-products. Find out more about dry AMD treatment drugs @ Drugs for Dry Age-Related Macular Degeneration Treatment A snapshot of the Dry Age-Related Macular Degeneration Pipeline Drugs mentioned in the report: Learn more about the emerging dry AMD pipeline therapies @ Dry Age-Related Macular Degeneration Clinical Trials Dry Age-Related Macular Degeneration Therapeutics Assessment The dry AMD pipeline report proffers an integral view of the dry AMD emerging novel therapies segmented by stage, product type, molecule type, mechanism of action, and route of administration. Scope of the Dry Age-Related Macular Degeneration Pipeline Report Coverage: Global Therapeutic Assessment By Product Type: Mono, Combination, Mono/Combination Therapeutic Assessment By Clinical Stages: Discovery, Pre-clinical, Phase I, Phase II, Phase III Therapeutics Assessment By Route of Administration: Oral, Intravenous, Subcutaneous, Parenteral, Topical Therapeutics Assessment By Molecule Type: Recombinant fusion proteins, Small molecule, Monoclonal antibody, Peptide, Polymer, Gene therapy Therapeutics Assessment By Mechanism of Action: Dimerisation inhibitors, Vitamin A replacements, RBP4 protein inhibitors, Sigma-2 receptor antagonists, Complement factor H stimulants, Macrophage inhibitors, Cell replacements, RNA interference, Key Dry Age-Related Macular Degeneration Companies: Alkeus Pharmaceuticals, Dobecure, Belite Bio, Cognition Therapeutics, Aviceda Therapeutics, Stealth BioTherapeutics, Allergo Ophthalmics, Annexon, Inc., Johnson & Johnson, InflammX Therapeutics, Lineage Cell Therapeutics, Ionis Pharmaceuticals, Evergreen Therapeutics, Inc., Alexion, Luxa Biotechnology, Astellas Pharma, OliX Pharmaceuticals, Hoffmann-La Roche, Boehringer Ingelheim, Eyevensys and others Key Dry Age-Related Macular Degeneration Pipeline Therapies: ALK-001, Etamsylate, Tinlarebant (LBS-008), CT1812, AVD-104, Elamipretide, Risuteganib, ANX 007, JNJ 81201887, Xiflam, OpRegen, IONIS-FB-LRx, EG-301, ALXN2040, RPESC-RPE-4W, MA09-hRPE, OLX301A, RO7303359, BI 771716, EYS 611 and others. Dive deep into rich insights for new drugs for dry AMD treatment, visit @ Dry Age-Related Macular Degeneration Drugs Table of Contents For further information on the dry AMD pipeline therapeutics, reach out @ Dry Age-Related Macular Degeneration Treatment Drugs Related Reports Dry Age-Related Macular Degeneration Market Dry Age-Related Macular Degeneration Market Insights, Epidemiology, and Market Forecast – 2034 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, market share of the individual therapies, and key dry AMD companies including Alkeus Pharmaceuticals, Belite Bio, Aviceda Therapeutics, Johnson & Johnson Innovative Medicine, Allegro Ophthalmics, Lineage Cell Therapeutics (CellCure Neurosciences), Roche, Cognition Therapeutics, Stealth BioTherapeutics, Evergreen Therapeutics, Annexon Biosciences, NGM Biopharmaceuticals, AstraZeneca, Alexion Pharmaceuticals, Ionis Pharmaceuticals, Novartis, Regenerative Patch Technologies, among others. Dry Age-related Macular Degeneration Epidemiology Forecast Dry Age-related Macular Degeneration Epidemiology Forecast – 2034 report delivers an in-depth understanding of the disease, historical and forecasted dry AMD epidemiology in the 7MM, i.e., the United States, EU4 (Germany, France, Italy, and Spain), the United Kingdom, and Japan. Age-related Macular Degeneration Market Age-related Macular Degeneration Market Insights, Epidemiology, and Market Forecast – 2034 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, market share of the individual therapies, and key AMD companies including Regeneron Pharmaceuticals, Novartis, Roche, Opthea Limited, Kodiak Sciences Inc., REGENXBIO, Alkahest Inc, Graybug Vision, Ribomic USA Inc, Outlook Therapeutics, Inc., Unity Biotechnology, Inc, PanOptica, Inc., Clearside Biomedical, Alexion Pharmaceuticals, AstraZeneca, Evergreen Therapeutics, Alkeus Pharmaceuticals, Stealth BioTherapeutics, CellCure Neurosciences, Regenerative Patch Technologies, Allegro Ophthalmics, Annexon Biosciences, NGM Biopharmaceuticals, Ionis Pharmaceuticals, Apellis Pharmaceuticals, Iveric Bio, Gyroscope Therapeutics, Luxa Biotechnology, Gemini Therapeutics , among others. Wet Age-related Macular Degeneration Market Wet Age-related Macular Degeneration Market Insights, Epidemiology, and Market Forecast – 2034 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key Wet Age-related Macular Degeneration companies including EyePoint Pharmaceuticals, Inc., AbbVie, Caregen Co. Ltd., Exegenesis Bio, Shanghai Henlius Biotech, Skyline Therapeutics, 4D Molecular Therapeutics, Ocugenix Corporation, Adverum Biotechnologies, Inc., Ashvattha Therapeutics, Inc., AiViva BioPharma, Inc., Ocular Therapeutix, Inc., Clearside Biomedical, Inc., Hoffmann-La Roche, Kyowa Kirin, Inc., Opthea Limited, AffaMed Therapeutics Limited, EyeBiotech Ltd., Novartis , among others. 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 . Contact Us Shruti Thakur [email protected] +14699457679 Logo: SOURCE DelveInsight Business Research, LLP WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Phase 1Phase 3Phase 2

100 Deals associated with Gildeuretinol acetate

R&D Status

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
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
Stargardt Disease	Phase 2	United States	Alkeus Pharmaceuticals, Inc.	01 Aug 2015
Retinal Dystrophies	Discovery	-	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, Company_Website) Manual	Phase 3	Geographic Atrophy	198	Gildeuretinol	(ifpqkjdxvo) = lmevlpyefj wslpqpyhxa (nqxspkpzsj ) View more	Positive	23 Oct 2024
				Placebo	(zrvysidmpo) = mwmcngnzro vpovxvmeyn (msgujdtuku )
NCT03845582 (SAGA, GlobeNewswire) Manual	Phase 3	Geographic Atrophy	-	gildeuretinol acetate	(zfkzmkemtc) = demonstrated a reduction of 0.25 sqmm/year vs. placebo (p=0.07) kzydvohhcv (exixoaltxs ) View more	Positive	17 Sep 2024
				Placebo
NCT04239625 (TEASE-3, Corporate Publications \| GlobeNewswire 1 \| GlobeNewswire 2) Manual	Not Applicable	Stargardt Disease	5	ALK-001	zkemimfdfs(cpdfdrinyf) = 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. aaefsflthu (lgazkkoruq )	Positive	10 Jan 2024
NCT02402660 (TEASE-1, Biospace) Manual	Phase 2	Stargardt Disease	50	ALK-001	(bngcrwpgna): Difference(%) = -21, P-Value = <0.001 View more	Positive	03 Nov 2023
				Placebo
NCT02402660 (TEASE, ARVO2016)	Phase 2	Stargardt Disease	50	ALK-001	(megxmjpfye) = bfkmbceoof btamfjxabl (koditevilo ) View more	Positive	01 Sep 2016

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