Age-related macular degeneration (AMD) is a common degenerative disease of the retina, the light-sensitive tissue in the back of the eye.
Wet AMD occurs when abnormal blood vessels (choroidal neovascularization) grow underneath the macula leading to leakage of fluid and blood leading to visual distortion and acute vision loss. If untreated or undertreated, wet AMD leads to scar formation and permanent damage that results in blindness.
Even with approved treatments, significant unmet needs persist related to the following:
- Poor Compliance: This is driven through a combination of patient fatigue with chronic intravitreal injections as well as their caregivers (generally a family member) to consistently take time away from work or other duties in order to drive the patient to the ophthalmologist for their treatment and follow-up appointments.
- Accessibility: Despite the increased burden of retinal disease, the distribution and practice patterns in the United States places significant non-clinical barriers to treatment. Patients living in underserved markets are at higher risk for vision loss because their physical location places them at a geographic disadvantage to large metropolitan areas and more affluent suburbs with a higher concentration of retinal ophthalmologists to provide treatment, further underscoring the value of a product that could be administered by the patient at home.
- Patient administers drug at home with autoinjector up to once per month
(potential for oral administration)
- Patients in rural areas receive convenient treatment (especially parts of China and India)
- Diabetic patients maintain compliance (treated for decades)
- Treatment of earlier stage of AMD
- Lower cost – manufactured at up to 1/10 the cost of other approaches
We have designed and optimized an HDT that:
- Precisely targets reactive macrophages and retinal pigment epithelial cells (RPEs) after systemic administration
- Preclinical models of neovascularization suggest the potential for a superior product profile
- The HD is retained within the CNV lesion of ocular neovascularization for >30 days supporting an optimal dosing and administration profile
- Potential for superior efficacy compared currently approved biologics requiring intravitreal administration at a physician’s office
Laser-induced CNV Mouse Model
D-4517 Alters Pharmacology through Non-Cleavable Dendrimer
|Liver toxicity (decreased size/increased ALT/AST)||No observed effect|
|Proteinuria, decrease in kidney size (males)||No observed effect|
|Hypoglycemia (females)||No observed effect|
|Decrease in heart size (males)||No observed effect|
Same sunitinib exposure in both groups
proprietary Sunitinib analog