OliX Pharmaceuticals (226950.KQ) presented clinical development results for its macular degeneration treatment OLX301A at an oligonucleotide therapeutics conference co-hosted by the U.S. Food and Drug Administration and the Drug Information Association. After confirming safety and tolerability in a U.S. Phase 1 trial, the company plans to move into a global Phase 2a study following early signs of efficacy in some patients.
OliX said on Sept. 29 that Dr. Pooja Dua of its U.S. subsidiary OliX US appeared as an invited speaker on Sept. 25 in a session on extrahepatic delivery strategies at the 2026 DIA/FDA Oligonucleotide-Based Therapeutics Conference. The event is a specialized oligonucleotide therapeutics conference jointly run by the FDA and the DIA, bringing together global regulators and experts from the pharmaceutical and biotech industries and academia to discuss research and development and regulatory issues.
Dua introduced preclinical and Phase 1 development results for OLX301A under the title "Inhibition of Ocular Degeneration and Neovascularization Through MyD88-Targeting Cell-Penetrating Asymmetric siRNA."
OLX301A is an RNA interference therapy that targets MyD88, an inflammatory signaling protein involved in the onset and progression of macular degeneration. RNAi is a technology that suppresses the expression of specific genes to reduce production of disease-related proteins. Preclinical studies confirmed that the drug curbed retinal damage and functional decline and reduced abnormal neovascularization.
The Phase 1 trial conducted in the United States showed no drug-related serious adverse events or dose-limiting toxicity. Some patients also showed early signs of efficacy, including improvement in best-corrected visual acuity. Based on those results, OliX is pursuing a global Phase 2a trial to evaluate the efficacy of OLX301A in patients with age-related macular degeneration.
Early research results applying OASIS-D, a next-generation dual-targeting RNAi platform, to eye diseases were also disclosed. OASIS-D is a technology that simultaneously suppresses two disease-related genes in the same tissue with a single therapy. The presentation included early data indicating the potential to suppress two genes at once in ocular tissue.
"It is meaningful that we shared OLX301A's development results and early research findings from our dual-targeting RNAi platform applied to eye diseases at a conference co-hosted by the FDA and the DIA," an OliX official said. "Having submitted an investigational new drug application to enter a global Phase 2a trial, we will push ahead with follow-up studies without setbacks."
