Acucela's Pipeline
Visual Cycle Modulation (VCM) Programs
Acucela is developing orally-delivered VCM therapies that selectively target cells within the retina to protect visual acuity and have the potential to treat blinding eye diseases that affect tens of millions of people.
ACU-4429 for Dry Age-related Macular Degeneration (AMD)
The Company’s lead investigational VCM compound, ACU-4429, is currently in clinical development for dry AMD (Phase 2a has been completed and a Phase 2b/3 trial has been initiated). Dry AMD is a degenerative disease of the eye that affects the part of the retina responsible for fine visual acuity and color vision. There is no approved treatment for dry AMD currently on the market.
Other VCM Programs
Acucela’s VCM technology may provide therapeutic benefit for additional retinal disorders, such as:
Diabetic Retinopathy, the most common diabetic eye disease and a leading cause of blindness in American adults.
Retinopathy of Prematurity (ROP), a potentially blinding eye disease that can occur in premature infants. In ROP, abnormal blood vessels grow and spread throughout the retina. There is no approved treatment for ROP.
Stargardt Disease, a recessive, hereditary retinal disease that results in progressive loss of rod and cone photoreceptors, leading to loss of central vision.
Non-VCM Programs
OPA-6566 for Glaucoma
Glaucoma is a type of progressive or chronic optic neuropathy caused by intraocular pressure or by other factors of unknown origin. Otsuka Pharmaceuticals and Acucela are co-developing this compound in the United States.
Rebamipide for Dry Eye
Dry Eye is usually caused by a problem with the quality of the tear film, the liquid layer that lubricates the cornea and conjunctiva. Rebamipide is in clinical development in the United States.






