South Korea-based OliX Pharmaceuticals, Inc. announced it has dosed the first patient in its phase 1 clinical trial assessing the investigational RNAi therapeutic OLX10212 for the treatment of age-related macular degeneration (AMD), including geographic atrophy (GA) and neovascular AMD (nAMD).
Give me a quick rundown on OliX Pharmaceuticals.
OliX is a clinical-stage pharmaceutical company focused on developing therapeutics that combat various disorders using its proprietary RNAi technology to downregulate disease-causing gene expression.
Asymmetric siRNA (asiRNA), OliX’s core RNAi platform, is a unique, RNAi-based gene-silencing technology.
Now this RNAi technology.
RNa interference (RNAi) is a biological process in which RNA molecules shut down protein translation via binding to messenger RNAs that code for those specific proteins, effectively protecting against viruses.
OliX’s asiRNA is used to develop a therapeutic RNAi platform called cell-penetrating asiRNA (cp-asiRNA) to target diseases locally, such as dry and wet AMD.
INSERT VIDEO: https://youtu.be/CtkIYuxz_Ew
How about OXL10212?
OLX10212 is a cp-asiRNA that targets inflammation pathways that are directly involved in GA and nAMD development via intravitreal injection.
Talk about the trial.
The phase 1, multi-center, open-label, single- and multi-dose, dose escalation study is assessing the safety and tolerability of OLX10212 administered as an intravitreal injection in 60 participants with AMD.
The trial is divided into two parts: Part A as a single-ascending dose study and Part B as a multiple-dose study.
Tell me more.
Part A consists of 42 participants receiving one injection of OLX10212 at different dosage levels (six patients per level), starting with the lowest dose of 50-μL.
Safety and tolerability of OLX10212 will be evaluated for the first 28 days following injection, with observations for up to 24 weeks post-injection.
Part B includes 18 participants receiving up to three dose-level injections (to be determined following completion of Part A) of OLX10212, with six patients per dose level.
Each injection is being administered four weeks apart, with the safety and tolerability evaluation period covering the first 12 week post-injection (ending 4 weeks after the third injection).
When can we expect results?
In reviewing Clinical Trials, the study is expected to be completed December 2024.
Significance?
This trial is the first in which OLX10212 is being tested on human patients with AMD.
According to investigators, if successful, it has the potential to not only treat wet AMD, but decrease the number of treatments needed—significantly reducing patients’ overall treatment burden.