Opus Genetics Announces Presentations at Association for Research in Vision and Ophthalmology (ARVO) 2025 Meeting
New Data from First Three Adult Patients in Phase 1/2 Trial with OPGx-LCA5 Showed that Subjective and Objective Signs of Efficacy Persisted for One Year
Durham, NC., March 05, 2025 (GLOBE NEWSWIRE) -- Opus Genetics, Inc. (Nasdaq: IRD), a clinical-stage ophthalmic biotechnology company developing gene therapies for the treatment of inherited retinal diseases (IRDs) and other ophthalmologic disorders, today announced that three abstracts on its investigational gene therapy candidates have been accepted for presentation at the , to take place May 4-8, 2025 in Salt Lake City, UT. The abstracts feature 12-month data from the first three adult patients in our ongoing Phase 1/2 trial of OPGx-LCA5, as well as pre-clinical results on OPGx-MERTK and OPGx-RDH12.
An abstract summarizing a subset analysis from the previously completed LYNX-1 Phase 3 trial of Phentolamine Ophthalmic Solution 75% has also been accepted for presentation. The full abstracts are available in the ARVO Online Planner, which can be accessed .
“We are pleased to have the opportunity to share data on our gene therapy candidates and to engage with the global ophthalmology community at ARVO 2025,” said George Magrath, M.D., Chief Executive Officer at Opus Genetics. “We look forward to presenting the 12-month data on adult patients being treated in the ongoing Phase 1/2 trial of our most advanced gene therapy candidate OPGx-LCA5. Assuming continued safety and efficacy in the current study, we plan to advance OPGx-LCA5 into a pivotal Phase 3 trial and we are hopeful that, if successful in Phase 3, and approved, OPGx-LCA5 may offer a potentially life-changing therapeutic option for individuals living with LCA5.”
Abstract details
| Title: | Recovery of cone mediated vision in severe ciliopathy after gene augmentation; One year results from a Phase I/II trial of LCA5-LCA (OPGx-LCA5) |
| Author: | Tomas Aleman, M.D., Schele Eye Institute, University of Pennsylvania et al |
| Presentation time: | May 4, 2025 from 4:30 PM to 4:45 PM MT |
| Location: | Room 255E |
OPGx-LCA5 is an investigational gene therapy for the treatment of Leber congenital amaurosis (LCA). The candidate is being evaluated in an ongoing non-randomized single ascending Phase 1/2 dose escalation study. Previously announced results showed OPGx-LCA5 to be well tolerated, with all three adult patients showing visual improvement at six months. New data from the study, to be presented at ARVO, show that subjective and objective signs of efficacy persisted for a year.
| Title: | Evaluation of MERTK gene therapy in RCS rats following a single bilateral subretinal injection |
| Author: | Mayur Choudhary, PhD., Opus Genetics et al |
| Presentation time: | May 8, 2025 from 11:45 AM to 1:30 PM MT |
| Posterboard Number: | 5944 - A0009 |
OPGx-MERTK is a gene therapy being developed by Opus Genetics to treat patients impacted by MERTK-related retinitis pigmentosa (RP). Results from a pre-clinical study which evaluated OPGx-MERTK in a rat model of RP will be presented at ARVO.
| Title: | Evaluation of ocular tolerability of OPGx-RDH12 by subretinal delivery in cynomolgus primates |
| Author: | Ash Jayagopal, PhD., Opus Genetics et al |
| Presentation time: | May 5, 2025 from 8:30 AM to 10:15 AM MT |
| Posterboard Number: | 1621 - B0205 |
OPGx-RDH12 is a gene therapy being developed to treat Leber congenital amaurosis 13 (LCA13), a genetic retinal dystrophy caused by mutations in the RDH12 gene. This pre-clinical study was conducted to evaluate the tolerability of OPGx-RDH12 in primates. Results from this pre-clinical study will be presented at ARVO.
| Title: | LYNX-1 Phase 3 trial of the safety and efficacy of phentolamine ophthalmic solution for the treatment of reduced mesopic low contrast vision: A subset analysis of keratorefractive subjects |
| Author: | Kostas Charizanis, PhD., Opus Genetics et al |
| Presentation time: | May 4, 2025 from 8:00 AM to 9:45 AM MT |
| Posterboard Number: | 173 - A0289 |
LYNX-1 was a randomized, double-masked, placebo-controlled trial of the safety and efficacy of Phentolamine Ophthalmic Solution 0.75% in subjects with dim light disturbances (DLD) of various etiologies. Top-line results were announced in 2022. The results to be presented at ARVO are from a subset analysis of the 25 post-LASIK subjects in LYNX-1 who had reduced mesopic low contrast visual acuity (mLCVA) and photic complaints in order to inform patient population selection for ongoing and future Phase 3 studies.
About Opus Genetics
Opus Genetics is a clinical-stage ophthalmic biotechnology company developing gene therapies to treat patients with inherited retinal diseases (IRDs) and other treatments for ophthalmic disorders. The pipeline includes adeno-associated virus (AAV)-based investigational gene therapies that address mutations in genes that cause different forms of bestrophinopathy, Leber congenital amaurosis (LCA) and retinitis pigmentosa. Our most advanced investigational gene therapy program is designed to address mutations in the LCA5 gene, which encodes the lebercilin protein and is currently being evaluated in a Phase 1/2 open-label, dose-escalation trial, with encouraging early data. BEST1 investigational gene therapy is designed to address mutations in the BEST1 gene, which is associated with retinal degeneration; we expect that a Phase 1/2 study will be initiated in 2025. The pipeline also includes Phentolamine Ophthalmic Solution 0.75%, a non-selective alpha-1 and alpha-2 adrenergic antagonist being investigated to reduce pupil size, and APX3330, a novel small-molecule inhibitor of Ref-1 being investigated to slow the progression of non-proliferative diabetic retinopathy. Phentolamine Ophthalmic Solution 0.75% is currently being evaluated in Phase 3 trials for treatment of presbyopia and reduced dim (mesopic) light low contrast vision following keratorefractive surgery. FDA Fast Track Designation has been granted for Phentolamine Ophthalmic Solution 0.75% as treatment of significant chronic night driving impairment in keratorefractive patients with reduced mesopic vision. We have reached agreement with the FDA on a SPA for a Phase 3 trial to evaluate oral APX3330 for the treatment of DR. For more information, please visit .
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