Gene Signal announced the publication of interim phase II data that suggests that the antisense oligonucleotide GS-101 is safe and effective at inhibiting and regressing corneal neovascularization. This study was published in the September 2009 issue of Ophthalmology. The group that received the GS-101 dose of 86 µg/day showed regression in 86% of patients compared with progression of neovascularization which occurred in 100% of the placebo group.

Forty patients who were non-responsive to conventional therapy participated in the study. Four groups of 10 patients were treated for three months comparing three doses of GS-101 twice a day to placebo (10 patients per group). The primary endpoint was measured by the reduction in area covered by pathologic corneal blood vessels measured morphometrically on digitized slit-lamp pictures.

GS-101 was found to be well tolerated in this study.

The company is now conducting an international phase III trial with GS-101 for the prevention of pathologic corneal neovascularization and thereby corneal graft rejection. GS-101 has been granted Orphan Drug status for this indication in Europe.

Read ^[1] the release.