Research results: OliX Pharmaceuticals announces positive results of phase 1 safety and tolerability results of OLX10212 for AMD

The safety and tolerability data from the Phase 1 study provided "substantial optimism" for the advancement of the treatment. Image credit: ©Liubomir – stock.adobe.com

OliX Pharmaceuticals, Inc. announced positive results from a phase 1 study of the safety and tolerability of OLX10212 for the treatment of age-related macular degeneration (AMD). This phase 1 study was a multi-centre, single-dose, dose-escalating study to evaluate the safety and tolerability of OLX10212 in patients with neovascular AMD. Safety and tolerability of the study were assessed 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.¹

The 15 study participants received treatments of OLX10212 at dose levels between 100μg/eye/50μL and 950μg/eye/50μL administered via a single intravitreal injection. There were any observations of adverse effects related to OLX10212 during the DLT period or during the follow-up period up to 24 weeks post-injection. No systemic effects were observed. Sporadic ophthalmic adverse effects were transient, mild and related to the dose administration procedure as expected for intravitreal injections.¹

Preliminary efficacy evaluations included best corrected visual acuity (BCVA) changes from baseline during follow-up. This study identified dose levels suitable for evaluations of efficacy testing in future clinical trials.¹

Dong Ki Lee, PhD, CEO of OliX, shared his excitement for this study in the press release, saying: “OliX and its Ophthalmology Division are strongly committed to use our proprietary siRNA platform technology to advance the development of novel, safe and effective treatments for our patients. We are thrilled about the outcome of this first-in-human trial in patients with wet AMD who were treated with OLX10212. The excellent safety and tolerability data, paired with encouraging preliminary BCVA improvement reassure the next steps in this program, and we are confident that OLX10212 and its novel mechanism of action may have potential to provide benefits in the treatment of AMD patients.”

Demetrios G. Vavvas, MD, PhD, Director of Retina Service at Harvard Medical School and advisor to OliX, also noted the importance of this trial in the press release. He said, “The safety data from this first in human eyes of asymmetric siRNA is a very important step in having this technology to our arsenal of therapeutics. I am looking forward to the next phase of these trials.”

Veeral Sheth, MD, University Retina and Macula Associates P.C., who participated in the trial, said, “Our commitment to addressing the challenge of blindness caused by macular degeneration has led to substantial advancements, though there remains a notable divergence between the efficacy observed in controlled clinical trials and the outcomes in everyday clinical practice. This reality motivates our search for novel therapeutic options that promise safety, effectiveness and an enhanced quality of life for our patients. The novel mechanism of OLX10212, coupled with the encouraging safety, tolerability, data emerging from the Phase 1 study, provides substantial optimism for the advancement of this treatment in the realm of wet macular degeneration therapy.”¹

Reference:

1. OliX Pharmaceuticals Announces Positive Safety Data and Preliminary Efficacy Effects in a Phase 1 Trial of OLX10212 for Age-Related Macular Degeneration. OliX Pharmaceuticals Inc. November 29, 2023. Accessed December 11, 2023. https://www.businesswire.com/news/home/20231129820286/en/OliX-Pharmaceuticals-Announces-Positive-Safety-Data-and-Preliminary-Efficacy-Effects-in-a-Phase-1-Trial-of-OLX10212-for-Age-Related-Macular-Degeneration