Pigment Epithelium Derived Factor (AdPEDF) for wet macular degeneration

Pigment Epithelium Derived Factor (AdPEDF) - an investigational drug for Wet Macular Degeneration.

Background:

The investigational agent, AdPEDF is a replication-deficient, adenovirus gene transfer vector. The transgene in this vector is the cDNA for human pigment epithelium-derived factor (PEDF). PEDF is one of the most potent known antiangiogenic proteins found in humans. PEDF is the eye's key natural regulator of normal blood vessel growth. It also is a neuro-protective agent. Intravitreal administration of adPEDF is likely to result in a more prolonged duration of effect versus Intravitreal administration of the PEDF protein alone. GenVec, Inc. is developing adPEDF. GenVec has obtained an exclusive license from the National Institutes of Health (NIH) covering worldwide rights to develop and commercialize products using Pigment Epithelium-Derived Factor (PEDF) protein for all ocular indications, including the prevention, diagnosis and treatment of eye diseases.

AdPEDF Clinical Trials

Phase I

Genvec reported the results of the first part of the Phase 1 AdPEDF trial on 10/2004. The first part of Phase 1, multi-center, open-label, dose-escalation study involved 28 patients with advanced disease at 8 dose levels. Seven patients were included at the top dose level. No dose limiting toxicities, drug-related serious adverse events, or cases of infection in the eye (endophthalmitis) were seen. The few adverse events observed during the study were minor and not dose-related. Improvements in retinal appearance and stabilization of visual acuity in patients with very advanced disease were seen.

The second part of the ongoing Phase I clinical trial was announced on 02/2005. A total of 20 patients with less advanced AMD will be enrolled at multiple centers in the U.S. The Phase IB study will compare two doses of to evaluate the drug's safety, tolerability, and effects on vision as well as to establish dose level(s) for possible Phase II clinical trials. Patients will receive a single injection of AdPEDF in the eye most severely affected by the disease.