RXi Pharmaceuticals announced positive results with RXI-109 in a phase 1/2 clinical trial. RXI-109-1501 is an open-label, multidose, dose escalation study with three dose cohorts, enrolled sequentially, evaluating the safety and tolerability of RXI-109 injections in the eye of subjects with advanced neovascular age-related macular degeneration (AMD), and accompanying subretinal fibrosis. During this study, the clinical effect of these injections was evaluated as a secondary endpoint.
The primary objective was met as shown by the absence of dose-limiting and serious toxicities, and only mild-to-moderate procedure related adverse events. None of the adverse events were drug related. In addition, comprehensive ocular examinations showed no indications of inflammation nor any other tolerability issues related to the treatment. Therefore, these study results show that RXI-109 was safe and well tolerated for all patients included in the three dosage groups.
Even though the primary objective of this study was to evaluate the safety and tolerability of ocular injections with RXI-109, several assessments were included to measure a potential clinical effect. These assessments included measuring the change from baseline in subretinal fibrosis lesion size, as measured by spectral domain (SD) optical coherence tomography (OCT), fundus photography and fluorescein angiography, as well as a potential effect on visual function by measuring the best corrected visual acuity (BCVA), i.e. the best vision a patient could achieve with correction (such as glasses), as measured on the standard eye charts.
“This study is exciting on several levels. First, the impressive safety and tolerability of intravitreal injections of RXI-109 and the promising clinical data, support further development of this compound in retinal scarring. In addition, these positive findings also open significant avenues for the use of our platform technology in ophthalmology, with sd-rxRNA targeting other proteins for other ocular indications. Also, the results contribute to an already extensive set of data that RXI-109, by preventing the overexpression of connective tissue growth factor, can result in a positive impact in various indications where excessive fibrosis is seen,” RXi’s Chief Development Officer, Gerrit Dispersyn, Dr. Med. Sc., said in a company news release.
“Even though this is a small study, it is encouraging that some of the summary statistics on ocular assessments performed in the study eye at the last follow-up visit, suggest a more pronounced change over baseline in the highest dose group (Cohort 3), compared to other groups,” Dr. Dispersyn said.