Last reviewed · How we verify

ALK-001 oral capsule

Alkeus Pharmaceuticals, Inc. · Phase 3 active Small molecule

ALK-001 oral capsule is a Visual cycle modulator Small molecule drug developed by Alkeus Pharmaceuticals, Inc.. It is currently in Phase 3 development for Geographic atrophy secondary to age-related macular degeneration, Stargardt disease, Other inherited retinal dystrophies. Also known as: C20-D3-Retinyl Acetate, C20 Deuterated vitamin A, Gildeuretinol acetate.

ALK-001 is a retinoid that reduces toxic bisretinoid accumulation in the retina by inhibiting the visual cycle.

ALK-001 is a retinoid that reduces toxic bisretinoid accumulation in the retina by inhibiting the visual cycle. Used for Geographic atrophy secondary to age-related macular degeneration, Stargardt disease, Other inherited retinal dystrophies.

At a glance

Generic nameALK-001 oral capsule
Also known asC20-D3-Retinyl Acetate, C20 Deuterated vitamin A, Gildeuretinol acetate
SponsorAlkeus Pharmaceuticals, Inc.
Drug classVisual cycle modulator
TargetVisual cycle (retinoid metabolism pathway)
ModalitySmall molecule
Therapeutic areaOphthalmology
PhasePhase 3

Mechanism of action

ALK-001 works by slowing the regeneration of visual pigment in the retina, thereby reducing the formation and accumulation of lipofuscin and other bisretinoids that contribute to retinal degeneration. By modulating the visual cycle, it decreases the metabolic burden on retinal pigment epithelium cells, potentially slowing progression of inherited retinal dystrophies. This mechanism is particularly relevant in conditions where bisretinoid accumulation drives photoreceptor cell death.

Approved indications

Common side effects

Key clinical trials

Primary sources

Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.

SourceUsed for
ClinicalTrials.govTrial enrolment, design, endpoints, results

Competitive intelligence

For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:

Frequently asked questions about ALK-001 oral capsule

What is ALK-001 oral capsule?

ALK-001 oral capsule is a Visual cycle modulator drug developed by Alkeus Pharmaceuticals, Inc., indicated for Geographic atrophy secondary to age-related macular degeneration, Stargardt disease, Other inherited retinal dystrophies.

How does ALK-001 oral capsule work?

ALK-001 is a retinoid that reduces toxic bisretinoid accumulation in the retina by inhibiting the visual cycle.

What is ALK-001 oral capsule used for?

ALK-001 oral capsule is indicated for Geographic atrophy secondary to age-related macular degeneration, Stargardt disease, Other inherited retinal dystrophies.

Who makes ALK-001 oral capsule?

ALK-001 oral capsule is developed by Alkeus Pharmaceuticals, Inc. (see full Alkeus Pharmaceuticals, Inc. pipeline at /company/alkeus-pharmaceuticals-inc).

Is ALK-001 oral capsule also known as anything else?

ALK-001 oral capsule is also known as C20-D3-Retinyl Acetate, C20 Deuterated vitamin A, Gildeuretinol acetate.

What drug class is ALK-001 oral capsule in?

ALK-001 oral capsule belongs to the Visual cycle modulator class. See all Visual cycle modulator drugs at /class/visual-cycle-modulator.

What development phase is ALK-001 oral capsule in?

ALK-001 oral capsule is in Phase 3.

What are the side effects of ALK-001 oral capsule?

Common side effects of ALK-001 oral capsule include Nyctalopia (impaired night vision), Photopsia (flashing lights), Reduced visual acuity in low light.

What does ALK-001 oral capsule target?

ALK-001 oral capsule targets Visual cycle (retinoid metabolism pathway) and is a Visual cycle modulator.

Related