Drugs / ALK-001
Trials 4
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 11 trial | ||||||
| Phase 1 | NCT02230228 | Apr → Sep 2014 | Stargardt disease, age-related macular degeneration | Alkeus Pharmaceuticals, Inc. | Completed | No outcome recorded |
| Phase 22 trials | ||||||
| Phase 2 | NCT04239625 | Dec 2019 → Dec 2026 expected | ABCA4-related retinopathy, Stargardt disease | Alkeus Pharmaceuticals, Inc. | Active not recruiting | No outcome recorded |
| Phase 2 | NCT02402660 | Aug 2015 → Jun 2026 overdue | ABCA4-related retinopathy, Stargardt disease | Alkeus Pharmaceuticals, Inc. | Enrolling by invitation | No outcome recorded |
| Phase 2/31 trial · 1 missed | ||||||
| Phase 2/3 | NCT03845582 | May 2019 → Jul 2023 | age-related macular degeneration, senile atrophy of choroid | Alkeus Pharmaceuticals, Inc. | Completed | Missed primary |
| Comparator or background therapy1 trial | ||||||
| Phase 3 | NCT07419334 Comparator | Jun 2026 → Oct 2029 expected | Stargardt disease | Alkeus Pharmaceuticals, Inc. | Recruiting | No outcome recorded |
News releases announcing trial results or a regulatory action · 14
| Date | Issuer | Release |
|---|---|---|
| 2026-09-03 | Alkeus Pharmaceuticals, Inc. | Results Alkeus Announces Publication in JAMA Ophthalmology of Efficacy and Safety Results from the TEASE-1 Study of Gildeuretinol in Stargardt Disease alkeuspharma.com ↗
The peer-reviewed publication reports results from the study’s primary efficacy endpoint, two prespecified sensitivity analyses, and safety results over 24 months. |
| 2026-08-24 | Alkeus Pharmaceuticals, Inc. | Regulatory Alkeus Pharmaceuticals Announces Japan Ministry of Health, Labour and Welfare (MHLW) Has Granted Orphan Drug Designation to Gildeuretinol for Stargardt Disease alkeuspharma.com ↗ |
| 2025-08-07 | Alkeus Pharmaceuticals, Inc. | Results Alkeus Pharmaceuticals Announces Presentation of Positive Interim TEASE-3 Study Data in Early-Stage Stargardt Disease Patients Treated with Oral Gildeuretinol alkeuspharma.com ↗
Gildeuretinol acetate demonstrated less loss in ellipsoid zone (EZ) area compared to historical sibling controls. |
| 2025-05-07 | Alkeus Pharmaceuticals, Inc. | Results Alkeus Pharmaceuticals Presents Gildeuretinol Data During the Association for Research in Vision and Ophthalmology (ARVO) 2025 Annual Meeting May 4-8, 2025 alkeuspharma.com ↗
Participants treated with gildeuretinol in the SAGA study in geographic atrophy (GA) secondary to age-related macular degeneration (AMD) showed a slower decline in vision-related quality of life and functional reading scores compared to placebo, demonstrating functional benefit |
| 2025-01-09 | Alkeus Pharmaceuticals, Inc. | Results Alkeus Pharmaceuticals Announces New Positive Interim TEASE-3 Study Results Showing Gildeuretinol Prevented Disease Progression in Early-Stage Stargardt Patients alkeuspharma.com ↗
Two additional TEASE-3 participants who recently completed 24 months of therapy showed no disease progression, consistent with prior results. |
| 2024-12-05 | Alkeus Pharmaceuticals, Inc. | Results Alkeus Pharmaceuticals Announces Positive Gildeuretinol Data Will Be Presented During the 12th International FLORetina ICOOR Congress December 5–8 in Florence, Italy alkeuspharma.com ↗
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. |
| 2024-11-18 | Alkeus Pharmaceuticals, Inc. | Regulatory Alkeus Pharmaceuticals Receives FDA Rare Pediatric Disease and Fast Track Designations for Gildeuretinol as a Treatment for Stargardt Disease alkeuspharma.com ↗
Alkeus Pharmaceuticals, Inc., a biopharmaceutical company dedicated to preserving the sight of individuals impacted by retinal diseases, today announced that gildeuretinol (ALK-001), an investigational oral therapy, has received Rare Pediatric Disease and Fast Track designations from the U.S. Food and Drug Administration (FDA) for the treatment of Stargardt disease. |
| 2024-10-23 | Alkeus Pharmaceuticals, Inc. | Results Topline Results from Alkeus Pharmaceuticals’ Study of Oral Gildeuretinol Demonstrate Significant Trend in Slowing GA Progression and Visual Function Improvement alkeuspharma.com ↗
Oral gildeuretinol demonstrated a statistically significant reduction in the geographic atrophy (GA) lesion growth rate of 15.3% from 6 to 24 months (p=0.047) in a pre-specified analysis. |
| 2024-09-17 | Alkeus Pharmaceuticals, Inc. | Results Alkeus Pharmaceuticals Announces Results from the SAGA Study of Oral Gildeuretinol in Patients with Geographic Atrophy Secondary to Age-Related Macular Degeneration alkeuspharma.com ↗
Gildeuretinol demonstrated a statistically significant reduction in the loss of low luminance visual acuity (LLVA) at 24 months (p=0.03). |
| 2024-07-18 | Alkeus Pharmaceuticals, Inc. | Results Alkeus Pharmaceuticals Announces Presentation of a TEASE-3 Study Update Showing Progression Stalled in Early-Stage Stargardt Disease Patients Treated with Gildeuretinol alkeuspharma.com ↗
Alkeus Pharmaceuticals, Inc. today announced the presentation of interim data from its TEASE-3 study demonstrating that early-stage Stargardt disease patients treated with gildeuretinol acetate showed no disease progression and remained asymptomatic while on therapy ranging between two and six years. |
| 2024-05-08 | Alkeus Pharmaceuticals, Inc. | Results Alkeus Pharmaceuticals Announces Positive Interim Results Demonstrating No Signs of Disease Progression in Early-Stage Stargardt Disease Patients Treated with Gildeuretinol alkeuspharma.com ↗ |
| 2024-01-10 | Alkeus Pharmaceuticals, Inc. | Results Alkeus Pharmaceuticals Presents Positive Interim Data Showing Gildeuretinol Halted Stargardt Disease Progression alkeuspharma.com ↗
Alkeus Pharmaceuticals, Inc. today announced positive interim data showing gildeuretinol halted Stargardt disease progression for up to six years. |
| 2023-11-03 | Alkeus Pharmaceuticals, Inc. | Results Alkeus Pharmaceuticals Presents Positive Gildeuretinol Trial Results at American Academy of Ophthalmology, Demonstrating Significant Slowing of Retinal Atrophic Lesions in Stargardt Disease alkeuspharma.com ↗
Alkeus Pharmaceuticals, Inc. today announced positive data from its TEASE-1 study, a randomized, double-masked, placebo-controlled clinical trial investigating the effects of gildeuretinol (ALK-001) in patients with Stargardt disease. |
| 2021-07-14 | Alkeus Pharmaceuticals, Inc. | Regulatory FDA Grants Alkeus Pharmaceuticals Breakthrough Therapy Designation for ALK-001 (C20-D3-vitamin A) for the Treatment of Stargardt Disease alkeuspharma.com ↗ |
All press releases naming this drug 15 releases
Evidence & citations 3 cited values
Every value below carries the sentence it was read from. 5 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | ALK-001 | ClinicalTrials.gov intervention name — accepted as the source's own label NCT02230228 ↗ |
| Modality | Small molecule | “ALK-001, the investigational drug, is a chemically-modified vitamin A designed as a replacement of vitamin A to prevent the formation of toxic vitamin A dimers in the eye.” NCT02402660 ↗ |
| Route | Oral | “Daily, oral administration of ALK-001 capsules.” NCT02230228 ↗ |