SUWON, South Korea–(BUSINESS WIRE)–OliX Pharmaceuticals, Inc. (KOSDAQ: 226950), a leading developer of RNAi therapeutics, today 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.
Dong Ki Lee, Ph.D., CEO of OliX, said: “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, M.D., Ph.D., Director Retina Service, Harvard Medical School, and advisor to OliX 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, M.D., 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.”
In 2020, OliX inked a license and collaboration agreement with Théa Open Innovation (TOI) (a sister company of ophthalmic specialty pharmaceutical company Laboratoires Théa), where TOI has secured worldwide rights except Asia-Pacific to the OLX10212 program, while OliX retains the rights in Asia-Pacific.