Phase II Trial Starts for siRNA Therapy for Wet AMD

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Phase II Trial Starts for siRNA Therapy for Wet AMD
October 19, 2005
Genetic Engineering News
Acuity Pharmaceuticals initiated a Phase II trial for Cand5, its lead product candidate for the treatment of wet age-related macular degeneration (wet AMD), following successful completion of Phase I trial.
According to the company, this is the first-ever Phase II program for a small interfering RNA (siRNA) therapy, based on the gene silencing technology of RNA interference (RNAi).
Acuity, founded in 2002, has an exclusive license to several types of RNAi intellectual property from the University of Pennsylvania, including both broad-based and target-focused applications.
?Cand5 is safe and well-tolerated in patients with wet AMD,? said Jonathan L. Prenner M.D., of UMDNJ?Robert Wood Johnson Medical School and an investigator for Acuity?s Phase I study.
Cand5?s RNAi mechanism silences the genes that promote the overgrowth of blood vessels that lead to vision loss in wet AMD by shutting down the production of vascular endothelial growth factor (VEGF), which has been shown to be the central stimulus in the development of wet AMD.
By stopping production of VEGF at the source, Cand5 is expected to have efficacy advantages over other types of therapies for wet AMD, which work by inhibiting VEGF only after it has already been produced in the the eye, explained Dale Pfost, Ph.D., president and CEO of Acuity.
?A single molecule of Cand5 repeatedly stops hundreds of VEGF molecules. This sustained duration of action could result in less frequent delivery, perhaps only four to five times per year,? asserted Dr. Pfost.
Acuity?s Phase I trial, an open label, dose-escalation study that included 15 patients, tested five dose levels administered by intravitreal injection at six-week intervals. Cand5 was shown to be safe and well tolerated following repeat administration of the escalating dose levels, up to 3.0 mg per eye.
This study also included a pharmacokinetic analysis indicating that the study drug was not present in the plasma of any of the patients at any of the doses tested. This absence of systemic exposure to Cand5 is signficant since powerful VEGF inhibitors have the potential to cause serious adverse effects if present systemically, notes Dr. Pfost.