Emerging AMD Treatments Currently in Clinical Trials

AdPEDF — GenVec has just completed a Phase I human study of a gene therapy for the treatment of wet AMD. The name of the treatment is adenovirus-based Pigment Epithelium Derived Factor (AdPEDF). The company believes that AdPEDF will preserve vision in people with a variety of retinal degenerative diseases including dry AMD. The AdPEDF treatment involves the delivery of a gene that leads to the production of the protein PEDF, which helps keep photoreceptors healthy, thereby preserving vision. A Phase II study of AdPEDF is underway for treating patients with early to moderate wet AMD. FFB funded earlier, preclinical studies of PEDF.

Encapsulated Cell Technology (ECT) — Developed by Neurotech, the ECT is a tiny capsule (6 millimeters) implanted into the eye. The capsule contains retinal cells that produce a vision-preserving protein called Ciliary Neurotrophic Factor (CNTF). The protein helps keep photoreceptors alive and healthy, thereby preserving vision. The ECT is currently in a Phase II human clinical trial for people with dry AMD. FFB funded earlier, preclinical studies of this therapy.

EVIZON™ (squalamine lactate) — Developed by Genaera, EVIZON is currently in a Phase III human study for the treatment of wet AMD. The drug works by blocking numerous factors that lead to the growth of unhealthy, vision-robbing blood vessels under the retina. Derived from the liver of the dogfish shark, EVIZON is a small molecule drug administered intravenously (no eye injection).

OT-551 (antioxidant eye drops) — Othera’s OT-551 is intended to supplement the eye’s natural defense system against disease and injury. Permeating tissues at both the front of the eye (lens) and back of the eye (retina), researchers hope that OT-551 will provide antioxidant protection against both cataract and dry AMD. OT-551 is currently in a clinical study for the treatment of geographic atrophy (advanced dry AMD).

Retaane® (anecortave acetate) — Developed by Alcon, Retaane is a modified steroid that has shown promise in reducing the risk of vision loss due to the growth of unhealthy blood vessels in wet AMD. Alcon is currently studying the drug in a clinical trial to see if it prevents dry AMD from transforming into wet AMD.

siRNA (bevasiranib) — Developed by Acuity Pharmaceuticals, siRNA is an innovative technology that silences the genes that lead to the growth of unhealthy, vision-robbing blood vessels under the retina. The treatment has showed safety and efficacy in a Phase II study. A Phase III clinical trial is planned.

VEGF Trap — Regeneron’s wet AMD treatment, VEGF Trap, blocks the development of unhealthy blood vessels that lead to vision loss. Regeneron has successfully completed a Phase I clinical trial with the VEGF Trap in patients with wet AMD utilizing systemic delivery, but is presently pursuing development of the VEGF Trap in wet AMD using an injection into the eye.