RXi Pharmaceuticals begins Phase 1/2 clinical trial in ophthalmology

RXi Pharmaceuticals Corporation (NASDAQ: RXII) a biotechnology company focused on discovering and developing innovative therapies primarily in the areas of dermatology and ophthalmology today announced the initiation of a Phase 1/2 clinical trial in ophthalmology. The clinical study RXI-109-1501 will evaluate the safety and clinical activity of RXI-109 to prevent the progression of retinal scarring, a harmful component of numerous retinal diseases.

"We are very pleased to have initiated our first clinical trial in the area of ophthalmology. Currently, there is no effective way to prevent the formation or progression of retinal scars that may occur as a consequence of several devastating ocular diseases. In advanced neovascular or 'wet' age-related macular degeneration, our first area of study, retinal scarring can result in continued vision loss," said Dr. Pamela Pavco, Chief Development Officer of RXi Pharmaceuticals. She further added, "RXI-109 has the potential to fill this unmet medical need by reducing this continuing damage to the retina and, in doing so, helping to preserve vision in these individuals for a longer period of time."

RXI-109-1501 is a multi-center, multi-dose, dose escalation trial conducted in subjects with advanced neovascular or 'wet' age-related macular degeneration (AMD). In this Phase 1/2 trial, each subject will receive a total of four doses of RXI-109 at one month intervals. RXI-109 will be administered by intravitreal injection in one eye only. The dosing period (3 months) will be followed by a four month observation period. The safety and tolerability of RXI-109, as well as the potential for clinical activity, will be evaluated over the course of the study using numerous assessments to monitor ocular health and visual acuity. Several dose levels will be evaluated in a small number of subjects in this first trial in order to establish safety information and to help determine the dosing regimen for continued study.

RXI-109 is a self-delivering RNAi (sd-rxRNA^®) compound developed to target connective tissue growth factor (CTGF), a key regulator of scar formation in the skin and known to be involved in retinal scarring as well. Wet AMD is currently treated with anti-VEGF therapies to block vascular endothelial growth factor (VEGF) from causing blood vessel leakiness and the consequential damage to the retina. However, as the disease progresses, many advanced patients also experience retinal scarring which leads to further vision loss. Our ultimate goal is to reduce the scarring that is secondary to advanced wet AMD and in doing so, preserve vision for a longer period time.

Currently, there are no approved therapeutics in the U.S. for the treatment and prevention of subretinal fibrosis. Such a therapy could benefit patients with the advanced wet AMD as well as those with other ocular indications with a scarring component such as proliferative vitreoretinopathy (PVR) and proliferative diabetic retinopathy (PDR).