ALK-001 for Macular Degeneration

(SAGA Trial)

No longer recruiting at 24 trial locations

Age: 18+

Sex: Any

Trial Phase: Phase 2 & 3

Sponsor: Alkeus Pharmaceuticals, Inc.

Disqualifiers: Medical condition interfering with study, others

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

Duration: 12 months

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new treatment called ALK-001 to determine its effectiveness for Geographic Atrophy (GA), a condition where part of the retina gradually deteriorates, often due to age-related macular degeneration (AMD). The trial aims to assess the safety and effectiveness of ALK-001 compared to a placebo. Participants will receive either ALK-001 or a placebo capsule. This trial may suit individuals with at least one eye affected by GA due to dry AMD. As a Phase 2, Phase 3 trial, the research measures the treatment's effectiveness in an initial, smaller group and represents the final step before FDA approval.

Will I have to stop taking my current medications?

The trial information does not specify if you need to stop taking your current medications. It might depend on whether your medications interfere with the study, so it's best to discuss this with the trial coordinators.

Is there any evidence suggesting that ALK-001 is likely to be safe for humans?

Research has shown that ALK-001, also known as gildeuretinol, was safe in earlier studies. This treatment has been tested for conditions such as geographic atrophy and Stargardt disease, and it did not cause harmful effects related to vitamin A in the body. In simpler terms, it did not affect the body's handling of vitamin A. Overall, safety data from these studies suggest that ALK-001 is generally well-tolerated by patients. While no treatment is completely without risk, current evidence on ALK-001 is encouraging for safety in potential trial participants. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for AMD?

Unlike the standard treatments for geographic atrophy, which focus mainly on slowing disease progression with anti-VEGF injections or vitamins, ALK-001 is unique because it introduces a novel approach targeting vitamin A dimerization. ALK-001 is an oral capsule that aims to reduce toxic byproducts formed from vitamin A, which are believed to contribute to retinal damage in geographic atrophy. Researchers are excited about ALK-001 because it offers a potentially less invasive and more direct method to address the underlying causes of the disease, providing hope for more effective management of this condition.

What evidence suggests that ALK-001 might be an effective treatment for Geographic Atrophy?

Research has shown that ALK-001, also known as oral gildeuretinol acetate, can slow the growth of eye lesions related to geographic atrophy (GA). In earlier studies, ALK-001 significantly reduced lesion growth, with one study noting a 21.6% decrease in growth for Stargardt disease. Additionally, the SAGA study found that ALK-001 significantly slowed the growth rate of GA lesions over 24 months. In this trial, participants will receive either ALK-001 or a placebo. These findings suggest that ALK-001 might slow the progression of GA in people with age-related macular degeneration (AMD). ^{1 4 6 7 8}

Who Is on the Research Team?

Leonide Saad, PhD

Principal Investigator

Alkeus Pharmaceuticals, Inc.

Are You a Good Fit for This Trial?

This trial is for people with a condition called Geographic Atrophy (GA) that's due to dry age-related macular degeneration. Participants need to have at least one eye affected by GA. They shouldn't have any health issues that could mess with the study or stop them from following the trial procedures and staying in the study.

Inclusion Criteria

I have dry AMD causing vision loss in at least one eye.

I have vision loss in at least one eye due to dry AMD.

Exclusion Criteria

I do not have any health issues that would stop me from following the study's requirements.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive ALK-001 or placebo to evaluate the efficacy and safety in treating Geographic Atrophy

12 months

Monthly visits (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment

3 months

What Are the Treatments Tested in This Trial?

Interventions

• ALK-001
• Placebo oral capsule

Trial Overview The trial is testing ALK-001, an oral capsule, against a placebo (a pill without active medication). It's a phase 3 study where participants are randomly chosen to receive either ALK-001 or placebo, and neither they nor the researchers know who gets what—a method known as double-masking.

How Is the Trial Designed?

Group I: ALK-001 Experimental Treatment 1 Intervention

Capsule

Group II: Placebo Placebo Group 1 Intervention

Capsule

ALK-001 is already approved in United States for the following indications:

Approved in United States as Gildeuretinol for:

• Stargardt disease

Who Is Running the Clinical Trial?

Alkeus Pharmaceuticals, Inc.

Lead Sponsor

Trials

4

Recruited

520+

Published Research Related to This Trial

Intravenous liposomal all-trans-retinoic acid (L-ATRA) maintains significantly higher and stable plasma concentrations compared to oral ATRA, with 13- and 22-fold greater AUC values on days 1 and 9, respectively, indicating a more effective delivery method.

Despite higher plasma levels of L-ATRA, the side effects were similar to those of oral ATRA, although L-ATRA was associated with dermal exfoliation in 31% of subjects, while oral ATRA led to abnormal liver function tests in 23% of subjects.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Pharmacokinetics of intravenously administered liposomal all-trans-retinoic acid (ATRA) and orally administered ATRA in healthy volunteers. Ozpolat, B., Lopez-Berestein, G., Adamson, P., et al.[2022]

The review of nine retinoids for cancer prevention found that all-trans-retinoic acid, 13-cis-retinoic acid, and N-ethylretinamide are not suitable for human use due to toxicity, potential to enhance cancer, or ineffectiveness.

Conversely, 9-cis-retinoic acid, etretinate, and acitretin show some potential for cancer prevention, but further research is needed, while all-trans N-(4-hydroxyphenyl)retinamide appears particularly promising.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

An International Evaluation of the Cancer-Preventive Potential of Nine Retinoids. Miller, AB., Nettesheim, P., Stewart, BW.[2019]

Citations

1. alkeuspharma.com alkeuspharma.com/presentation-of-gildeuretinol-data-at-aao-2025/

October 9, 2025 Oral gildeuretinol acetate (ALK-001) is a new molecular entity designed to reduce the dimerization of vitamin A without modulating the visual ...

2. clinicaltrials.gov clinicaltrials.gov/study/NCT02402660

Phase 2 Tolerability and Effects of ALK-001 on Stargardt ... This study evaluates the effects of orally-administered ALK-001 on the progression of Stargardt disease (ABCA4-related).

3. alkeuspharma.com alkeuspharma.com/alkeus-presents-gildeuretinol-data-during-arvo-2025-annual-meeting/

Alkeus Pharmaceuticals Presents Gildeuretinol Data ... The study showed daily oral gildeuretinol significantly slowed the growth of atrophic retinal lesions in Stargardt disease by a 21.6% reduction versus the ...

4. clinicaltrials.gov clinicaltrials.gov/study/NCT03845582

Phase 2/3 Study of ALK-001 in Geographic Atrophy This is a double-masked, multicenter, randomized, placebo-controlled clinical trial, evaluating the efficacy and safety of ALK-001 in participants with ...

5. ophthalmologytimes.com ophthalmologytimes.com/view/alkeus-pharmaceuticals-announces-results-from-the-saga-study-of-oral-gildeuretinol-in-patients-with-ga

Alkeus Pharmaceuticals announces results from the SAGA ... Oral gildeuretinol demonstrated a clinically meaningful reduction in the geographic atrophy lesion growth rate at 24 months, supporting additional clinical ...

6. glance.eyesoneyecare.com glance.eyesoneyecare.com/stories/2025-05-09/favorable-safety-data-supports-alkeus-oral-gildeuretinol-for-dry-amd/

Favorable safety data supports Alkeus' oral gildeuretinol for ... Gildeuretinol has been studied as a potential first-ever treatment for GA secondary to AMD via the phase 3 Study of ALK-001 in Geographic Atrophy (SAGA) trial.

7. alkeuspharma.com alkeuspharma.com/gildeuretinol-data-presentations-during-the-american-society-of-retina-specialists-annual-meeting/

Alkeus Pharmaceuticals Announces Gildeuretinol Data ... The Tolerability and Effects of ALK-001 on Stargardt diseasE (TEASE) studies consist of four independent clinical studies of oral gildeuretinol ...

8. ophthalmologytimes.com ophthalmologytimes.com/view/alkeus-pharmaceuticals-receives-fda-rare-pediatric-disease-and-fast-track-designations-for-gildeuretinol-as-treatment-for-stargardt-disease

Alkeus Pharmaceuticals receives FDA Rare Pediatric ... Gildeuretinol demonstrated a favorable safety profile, with no adverse events related to vitamin A metabolism. Stargardt disease affects 30,000 ...