News & Events zu Opus Genetics Inc (IRD)IRDOpus Genetics Inc

Opus Genetics' OPGx-RDH12 Receives FDA Clearance

Opus Genetics announced that the FDA has cleared its investigational new drug, or IND, application for OPGx-RDH12, an investigational gene therapy for the treatment of RDH12-associated Leber congenital amaurosis, a rare inherited retinal disease that causes progressive vision loss and blindness beginning in early childhood. Under the strategic collaboration announced in July 2025, the Global RDH12 Alliance committed up to $1.6M toward the development of OPGx-RDH12, with eligible development costs reimbursed as activities are completed subject to certain diligence obligations and milestones. The organizations are working together on the clinical and regulatory strategy for the program, integrating the perspectives and experiences of patients and families directly into its development.

Opus Genetics Receives FDA Approval for Presbyopia Drug

Opus Genetics (IRD) announced that the U.S. Food and Drug Administration has approved the company's supplemental New Drug Application for phentolamine ophthalmic solution 0.75% for the treatment of presbyopia in adults. Opus Genetics and Viatris (VTRS) are parties to a global licensing agreement which provides for the development of phentolamine ophthalmic solution 0.75% and grants exclusive rights to Viatris to commercialize the product in the United States. This FDA approval in presbyopia will trigger a milestone payment to Opus Genetics. The FDA approval is supported by positive results from the pivotal Phase 3 VEGA-2 and VEGA-3 clinical trials, both of which met their primary and all key secondary efficacy endpoints, with no treatment-related serious adverse events reported. Phentolamine ophthalmic solution 0.75% is a preservative-free, topical formulation that reduces pupil diameter through a sympatholytic mechanism of action, without engaging the ciliary muscle.

Opus Genetics Presents Clinical Findings for OPGx-BEST1 and OPGx-LCA5 in Brazil

Opus Genetics presented clinical findings from its OPGx-BEST1 and OPGx-LCA5 gene therapy programs at Retina in Rio 2026 in Rio de Janeiro, Brazil. The company presented findings from the low-dose Cohort 1 of its Phase 1/2 BIRD-1 trial of OPGx-BEST1, which showed improved retinal sensitivity in three of four evaluable participants. Additional analyses identified retinal structural and imaging characteristics associated with visual function and treatment response, providing insights that may help guide future clinical development. Additional findings from the ongoing Phase 1/2 trial of OPGx-BEST1 demonstrated encouraging improvements in retinal sensitivity in participants with BEST1-associated retinal diseases, along with new insights into imaging markers that may help predict treatment response. The company also presented findings from the Phase 1/2 clinical trial of OPGx-LCA5, which demonstrated sustained improvements in visual function among adults and children treated with OPGx-LCA5 for Leber congenital amaurosis type 5, or LCA5, a rare inherited retinal disease that causes severe vision loss beginning in childhood. In the OPGx-LCA5 Phase 1/2 study, improvements in visual acuity and light sensitivity were observed in three adult participants through 24 months and three pediatric participants through six months. Participants also reported improvements in everyday activities, including navigating independently, recognizing family members, and identifying objects and seeing colors. Opus presented findings from the ongoing Phase 1/2 BIRD-1 trial of OPGx-BEST1, an investigational gene therapy designed to restore bestrophin-1 function in patients with IRDs caused by mutations in the BEST1 gene, including Best vitelliform macular dystrophy, or BVMD, and autosomal recessive bestrophinopathy, or ARB. The presentation included results from five participants treated in the initial low-dose cohort, with follow-up ranging from three to six months. Among four participants who could complete retinal sensitivity testing, three demonstrated improvements in the treated transitional zone, an area surrounding damaged retinal tissue where light-sensitive cells remain and may be responsive to treatment. Improvements were measured using microperimetry, a test that evaluates the retina's ability to detect light at specific locations. In responding participants, increases in retinal sensitivity were also associated with reductions in intraretinal fluid characteristic of BEST1-associated disease. Additional analyses examined the relationship between retinal structure and function, suggesting that measurements obtained through optical coherence tomography, or OCT, and other imaging techniques may help identify retinal regions most likely to respond to treatment. The presentation also highlighted natural history findings demonstrating that retinal sensitivity declines most noticeably in the transitional zone surrounding damaged tissue, even when measurements across the entire retina appear relatively stable. Together, these findings are informing future clinical development of OPGx-BEST1, including more targeted retinal sensitivity assessments and imaging approaches designed to improve the measurement of treatment response. Rodrigo Jorge of Ribeirao Preto Medical School at the University of Sao Paulo, presented findings from the ongoing Phase 1/2/3 trial of OPGx-LCA5, an investigational gene therapy designed to restore production of lebercilin, a protein essential for normal retinal function. The presentation included data from six participants treated at the low dose of 1E10 vector genomes per eye, including three adults and three adolescents. Adults demonstrated sustained improvements in visual acuity through 24 months, while pediatric participants demonstrated restoration of cone-mediated vision with sensitivity improvements reaching normal ranges through six months. Both groups also demonstrated improved light sensitivity, as measured by full-field stimulus testing, or FST, compared with their untreated eyes. Additional retinal imaging using microperimetry showed increased retinal sensitivity and movement of visual fixation toward the central retina, which is responsible for sharp, detailed vision. Participants also reported meaningful improvements in everyday activities, including recognizing family members, navigating independently without a cane, identifying objects and participating in activities that had previously been difficult because of severe vision loss. Based on the encouraging Phase 1/2 findings, Opus advanced OPGx-LCA5 into Phase 3 development using the low dose without requiring further dose escalation. The Phase 3 clinical trial's natural history and treatment cohorts are fully enrolled, with dosing initiation expected in the fourth quarter of 2026. The clinical trial's primary efficacy endpoint will assess improvements in retinal sensitivity across 16 central retinal test locations. OPGx-LCA5 is an investigational adeno-associated virus serotype 8, or AAV8-based gene therapy designed to deliver a functional copy of the LCA5 gene to retinal photoreceptors. Mutations in LCA5 disrupt production of lebercilin, a protein critical for normal retinal function, resulting in severe visual impairment beginning early in life.