Current phase 1/2 research for neovascular age-related macular degeneration

Area of Science:

• Ophthalmology
• Genetics
• Oncology

Background:

• Neovascular age-related macular degeneration (nAMD) is a leading cause of vision loss.
• Current treatments focus on inhibiting vascular endothelial growth factor (VEGF).
• There is a continuous need for novel therapeutic strategies to improve outcomes in nAMD.

Purpose of the Study:

• To provide an update on Phase 1 and 2 clinical trials for nAMD.
• To review recently completed and ongoing trials by the end of 2014.
• To summarize emerging therapeutic modalities for nAMD.

Main Methods:

• Review of early-phase (Phase 1/2) clinical trials.
• Categorization of therapies by administration route (gene therapy, topical, systemic, radiation, intravitreal).
• Inclusion of trials completed or underway by end of 2014.

Main Results:

• Gene therapy trials (AAV2-sFLT01, AVA-101, RetinoStat) aim to express angiogenesis inhibitors.
• Development of topical eye drops (regorafenib, squalamine lactate, PAN-90806) for angiogenesis inhibition.
• Early-stage systemic (iSONEP, X-82) and local radiation therapies (proton beam, stereotactic radiotherapy) are under investigation.
• Multiple intravitreal injections (hl-con1, abicipar pegol, PF582, DE-120, ESBA 1008, REGN2176-3) are in early trials.

Conclusions:

• A wide array of treatment options for nAMD are in Phase 1/2 clinical trials.
• Emerging therapies include gene therapy, eye drops, systemic agents, radiation, and intravitreal injections.
• Further Phase 3 trials are necessary to validate the efficacy and safety of these novel nAMD treatments.