Anti-platelet-derived growth factor found safe

April 15, 2010

An anti-platelet-derived growth factor aptamer is safe and well-tolerated when used in combination with anti-vascular endothelial growth factor therapy in patients with neovascular age-related macular degeneration.

The multicenter, phase I, dose-escalating study enrolled 22 patients who received anti-VEGF therapy with ranibizumab 0.5 mg (Lucentis, Genentech) in combination with E10030. The two therapies were given as separate sequential injections, and the evaluated doses of E10030 were 0.03, 0.3, 1.5, and 3 mg. Patients received three sets of injections at monthly intervals. Primary endpoint evaluation was at week 12, 4 weeks after the last treatment.

No dose-limiting toxicity was observed, and there were no significant adverse events related to the study drug. Data from secondary endpoints showed positive anatomic and functional effects, including a novel and dramatic reduction in choroidal neovascularization (CNV) size.

"This research has the limitations of a phase I study, including a nonrandomized design, small sample size, and limited follow-up," he said. "It is too early to tell if anti-PDGF treatment combined with anti-VEGF therapy is a reasonable target. However, safety data so far are positive."

This study also provides confirmation of the fascinating anatomic findings observed in preclinical animal models, he said. Based on these promising data, a phase II trial comparing the combination regimen against ranibizumab monotherapy is about to begin.