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Short interfering RNA drug shows early promise in retinal disease studies
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A new type of drug technology has shown promising early results for the treatment of both age-related macular degeneration and diabetic macular edema, according to Alexander Brucker, MD. He spoke about phase 1 and phase 2 trials of Cand5, Acuity Pharmaceuticals’ short interfering RNA therapy that targets vascular endothelial growth factor, during the World Ophthalmology Congress in São Paulo, Brazil. Phase 1 safety studies are complete, and phase 2 trials are ongoing, he said.
An important finding of the phase 1 safety trial of the drug for AMD, Dr. Brucker said, was that no evidence of short interfering RNA circulating in the circulating blood was found. He added that mean visual acuity was stable at 6 weeks, and there was “very high stability over a period of 12 weeks.”
“We have a very powerful anti-VEGF molecule,” Dr. Brucker said. “It appears to have no systemic exposure and no significant ocular side effects.”
In a phase 2 trial in 129 patients with choroidal neovascularization, subjects are being injected every 6 weeks with one of three doses of Cand5. A primary outcome of the phase 2 trial will be best corrected visual acuity; Dr. Brucker said. The last patient in that trial will receive the final injection during the first quarter of this year.
“We’re going to have results and data on the efficacy of this drug probably by the summertime of this year,” he said.
In addition to the AMD trial, Cand5 is under investigation as a potential treatment for diabetic macular edema as well, Dr. Brucker said.
“If we can inhibit messenger RNA inside the cell from releasing the VEGF into the vitreous, we can then perhaps decrease this vascular permeability molecule and vascular endothelial growth factor and decrease neovascularization and leakage,” he told attendees.
In the phase 2 diabetic macular edema trial, patients are being treated every 4 weeks, Dr. Brucker said. Based on the rapid rate of enrollment in the trial, he expects the recruitment for the study will be completed “by the end of April or May, rather than in the third quarter,” he said.
Dr. Brucker said Acuity plans to begin phase 3 trials for each indication some time in the first half of 2007.