OliX Pharmaceuticals Inc. announced positive results from a Phase 1 study evaluating the safety and tolerability of OLX10212 for the treatment of Age-Related Macular Degeneration (AMD). AMD is the most common cause of blindness in the industrialized world, which affects more than 170 million people worldwide, and currently available treatments are often insufficient to improve existing AMD or mitigate disease progression. The development of small interfering RNAs (siRNAs), like OLX10212 which showed efficacy in non-clinical models for both main forms of AMD, neovascular and dry AMD, allows targeting pathways that are believed to play a critical role in the development of AMD, and therefore present a novel approach for the treatment of this patient population.

This phase 1 study is a multi-center, single-dose, dose-escalating study to evaluate the safety and tolerability of OLX10212 in patients with neovascular AMD. The primary endpoints of this study were safety and tolerability assessments associated with each intravitreal OLX10212 injection. Safety and tolerability were assessed based on a combination of ophthalmologic and systemic evaluations for 2 weeks during the dose-limiting toxicity (DLT) evaluation period followed by an additional 22 weeks of clinical follow-up, for a total duration of 24 weeks.

Preliminary efficacy evaluations included best corrected visual acuity (BCVA) changes from baseline during follow-up. In this study, OLX10212 at dose levels between 100µg/eye/50µL and 950µg/eye/50µL was administered via a single intravitreal injection. Fifteen AMD patients received the treatment.

Neither during the DLT period nor during the follow-up period up to 24 weeks post-injection, there were any observations of adverse effects related to OLX10212. Specifically, there were no signs of inflammation or changes of intraocular homeostasis noted in all patients. In addition, no systemic effects were observed.

Sporadic ophthalmic adverse effects were transient, mild and related to the dose administration procedure as expected for intravitreal injections. Importantly, this study identified dose levels suitable for evaluations of efficacy testing in future clinical trials. Altogether, in this first-in-human study in AMD patients the study objectives were met.

The safety and tolerability evaluations, together with preliminary BCVA improvement of OLX10212 encourage further development of OLX10212 for AMD.