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OliX Pharmaceuticals have announced positive results for an siRNA experimental treatment for AMD.  

OliX Pharmaceuticals (KOSDAQ: 226950), based in Gyeonggi-do, Seoul, Republic of South Korea, has announced positive results from a phase 1 study evaluating the safety and tolerability of “OLX10212”, for the treatment of age-related macular degeneration (AMD).  The experimental treatment is an siRNA molecule that targets inflammation pathways that are directly involved in nAMD development via intravitreal injection.  The treatment is aimed at efficiently internalize and knock down target gene silencing via local administration and without special formulation or instrumentation.  Previously, OliX signed a license and collaboration agreement with Théa Open Innovation (TOI; a sister company of ophthalmic specialty pharmaceutical company Laboratoires Théa), where TOI secured worldwide rights to the OLX10212 program, except Asia-Pacific, while OliX retains the rights in Asia-Pacific.


Small interfering RNA (siRNA), sometimes known as short interfering RNA or silencing RNA, is a class of double-stranded RNA molecules. siRNA operates within the RNA interference (RNAi) pathway, where it interferes with the expression of specific genes with complementary nucleotide sequences by degrading mRNA after transcription, resulting in no translation.  Chemically synthesized siRNA, a structural mimic of the Dicer-cleavage product of a long dsRNA, could trigger efficient and specific target gene silencing in mammalian cells. According to the company, the novel therapeutics based on siRNA could not be easily utilized because the use of siRNA to inhibit target genes may cause side effects in cells, such as off-target effect. In addition, the company believed it is hard to deliver siRNA to specific cells or organs effectively.  However, OliX’s “cell penetrating asymmetric RNAi technology” proposes a “dramatic reduction in off-target effects and the resolution of problems related to the delivery of siRNA”.


To date, the preliminary results from the phase 1 study, for a multi-center, dose escalation trial, were assessing the safety of recruited 60 participants in AMD, administered with an intravitreal injection with the siRNA. The company reported dose levels between 100μg/eye/50μL and 950μg/eye/50μL, administered via a single intravitreal injection. Fifteen AMD patients received the treatment and at 24 weeks post-injection, there were no observations of adverse effects and no signs of inflammation or changes of intraocular homeostasis among all patients. In addition, no systemic effects were observed and any sporadic ophthalmic adverse effects were reported as being transient, mild and related to the dose administration procedure as expected for intravitreal injections. In addition, Olix stayed that, “the safety and tolerability evaluations, together with preliminary BCVA improvement of OLX10212 encourage further development of OLX10212 for AMD”.


Commenting on the announcement, 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.”