Product Candidates

Ophthotech is developing novel therapeutics to treat diseases of the eye, with a particular focus on diseases of the back of the eye.

Fovista® Anti-PDGF Therapy

Fovista® is designed to target platelet derived growth factor (PDGF) in combination with anti-VEGF drugs to disrupt the formation of abnormal new blood vessels in wet AMD. In clinical trials, the combination of Fovista® 1.5mg and an anti-VEGF therapy demonstrated statistically significant superiority compared to anti-VEGF monotherapy in terms of improvements in visual acuity, providing a 62% comparative benefit from baseline.

Fovista® binds to and inhibits PDGF expression, causing the loss or stripping of pericytes, leaving the endothelial cells unprotected and highly vulnerable to the effects of anti-VEGF therapy. Clinical data have shown that administration of Fovista® 1.5mg in combination with anti-VEGF drugs is likely to inhibit new blood vessel growth in patients with wet AMD more effectively than anti-VEGF monotherapy and may also enhance neovascular regression.

Fovista® Clinical Development   Fovista® fact sheet

Zimura® – Anti-Complement

Zimura® targets complement factor C5, a central component of the complement cascade that is believed to be involved in the development of AMD. In a Phase 2a clinical trial of Zimura®, wet AMD patients who had not previously been treated with anti-VEGF therapy and who received six injections of Zimura® in combination with anti-VEGF treatment showed improvements in vision of a magnitude warranting further evaluation in subsequent studies.

Based on results of this trial, data from a third-party clinical trial and multiple published studies suggesting that the complement pathway plays a significant role in dry AMD, we plan to initiate a Phase 2/3 clinical trial investigating Zimura® for treatment of geographic atrophy, a severe form of dry AMD affecting approximately 8 million patients worldwide.

Zimura® Clinical Development

Aptamer Technology

Both Fovista® and Zimura® are aptamers, single strands of nucleic acid that bind with high specificity and affinity to targets such as PDGF, similar to a monoclonal antibody. Aptamers are synthetically derived, making their production predictable and reproducible. They are also chemically stable and do not generate an immune response that could limit efficacy.