Announcement • Jul 09
Opus Genetics Provides Update On OPGx-BEST1 Program With Detailed Timeline For OPGx-BEST1 Results Opus Genetics provided updates on its ongoing OPGx-BEST1 Phase 1/2 clinical trial (BIRD-1) targeting BEST-1 associated inherited retinal diseases (IRDs). Opus expects to announce three-month topline data from Cohort 1 of the Phase 1/2 trial during the second week of September 2026, assuming all participants complete their assessments as scheduled. The Company plans to present the data at the annual EURETINA Congress taking place in Vienna, Austria from October 1 - 4, 2026. BIRD-1 is an adaptive, open-label, Phase 1/2 study evaluating the safety and efficacy of single-eye subretinal administration of OPGx-BEST1 in adult participants with Best Vitelliform Macular Dystrophy (BVMD) or Autosomal-Recessive Bestrophinopathy (ARB). The trial is designed as a dose escalation trial to evaluate two doses of OPGx-BEST1: 1.5E9 vg/eye (Cohort 1) and 4.5E9 vg/eye (Cohort 2). Enrollment in Cohort 1 was completed in May 2026 with five participants in the study, three with BVMD and two with ARB, who were carefully selected to meet the defined entry criteria. In the BVMD participants, the Company completed the added step of using an in vitro platform to confirm that each participant’s disease mutation is amenable to gene augmentation. After the last participant has completed the Month 3 visit, the Independent Data Monitoring Committee (IDMC) will review all Cohort 1 data and determine next steps. In Cohort 1, the primary endpoint for evaluation is the safety and tolerability of OPGx-BEST1. In addition to safety measures, Opus will be also assessing a number of structural parameters including subretinal fluid as measured by Optical Coherence Tomography (OCT) scans. A reduction in subretinal fluid on OCT would suggest that OPGx-BEST1 has a biological effect demonstrating target engagement. A reduction trending towards 20% may be considered clinically meaningful, and these results, in addition to those on safety, are expected to be used to provide the rationale to advance the trial into Cohort 2 to optimize dose selection, per the trial protocol. Given the high level of patient interest in the trial, potential participants have already been identified to enroll in Cohort 2 if necessary. In the event that OPGx-BEST1 demonstrates a 100% reduction in fluid in the majority of patients in Cohort 1, the trial may be expanded into a potential pivotal trial. Opus expects to present the Month 3 data to the U.S. Food and Drug Administration (FDA) to align on the next steps for clinical development. In addition to OCT structural assessments, functional endpoints will also be evaluated including microperimetry, best corrected visual acuity (BCVA), low luminance visual acuity (LLVA) and contrast sensitivity. Correlation between functional improvements and structural changes would suggest clinically meaningful target engagement by OPGx-BEST1. Due to the open-label nature of the trial, the Company plans to enter an investor relations quiet period beginning July 15, 2026, and continuing until the public announcement of these data. Additional OPGx-BEST1 resources can be found on the Opus Genetics website: Overview, presentations and webcasts can be found including Cohort 1 Baseline Demographics and Key Endpoints for IRDs Presentation and Video Recording by Dr. Mark Pennesi (May 2026) and Preliminary Results from the Sentinel Participant Presentation and Video Recording by Dr. Mark Pennesi (February 2026). Publications and medical conference presentations can be found including the baseline demographics poster presentation from the Association for Research in Vision and Ophthalmology (ARVO) Annual Meeting (May 2026). OPGx-BEST1 leverages Opus Genetics’ proprietary AAV-based gene therapy platform, designed to deliver a functional copy of the BEST1 gene directly to the retinal pigment epithelium (RPE) cells where the defective gene resides. The program builds on extensive preclinical work demonstrating restoration of BEST1 protein expression and improved retinal function in relevant disease models. By restoring BEST1 function, the therapy aims to address the underlying genetic cause of retinal degeneration and support preservation of photoreceptor health and visual function. Estimated global prevalence in BEST1-associated IRDs is approximately 21,800 patients, including roughly 8,400 patients in the U.S. comprised of approximately 8,000 best vitelliform macular dystrophy (BVMD) and approximately 400 autosomal recessive bestrophinopathy (ARB) patients. OPGx-BEST1 is currently being evaluated in an open-label, Phase 1/2 clinical trial. Additional information on the trial can be found by ClinicalTrials.gov Identifier: NCT07185256. There are currently no approved treatments for the disease. Announcement • Jul 06
Opus Genetics Announces FDA Alignment on Phase 3 Registrational Trial Design for Opgx-Lca5 in Lca5-Associated Inherited Retinal Disease Opus Genetics has reached alignment with the U.S. Food and Drug Administration (FDA) in a Type B Rare Disease Evidence Principles (RDEP) meeting on the design of its registrational Phase 3 clinical trial evaluating OPGx-LCA5 for LCA5-associated IRD, an early-onset severe inherited retinal dystrophy. Opus Genetics has received the meeting minutes from the Type B meeting confirming several key elements of the trial. The Phase 3 study is expected to enroll eight participants who are able to complete microperimetry testing with both eyes treated. The study is also expected to include a six-month run-in period, allowing each participant to serve as their own natural history control prior to receiving treatment. Seven of the eight planned participants have already been enrolled and are currently completing the run-in period, and the Company expects to initiate dosing in the Fourth Quarter of 2026. The primary efficacy endpoint is a mean improvement of at least 7 decibels (dB) in retinal sensitivity across the central 16 test loci, a clinically meaningful measure of visual function. The Phase 3 study is designed with greater than 90% statistical power to detect a treatment effect of at least seven decibels. The Phase 1/2 trial supports this outcome measure as those participants able to complete microperimetry demonstrated an average improvement of approximately 10.5 dB. The FDA indicated that Opus Genetics may submit a Biologics License Application (BLA) based on compelling efficacy at the six-month primary endpoint, with 12-month durability data submitted during the BLA review process. OPGx-LCA5 is designed to address a form of Leber congenital amaurosis (LCA) due to biallelic mutations in the LCA5 gene (LCA5), which encodes the lebercilin protein. LCA5-associated inherited retinal disease is an early-onset severe inherited retinal dystrophy. Studies in patients with this mutation have reported evidence for the dissociation of retinal architecture and visual function in this disease, suggesting an opportunity for therapeutic intervention through gene augmentation. OPGx-LCA5 uses an adeno-associated virus 8 (AAV8) vector to precisely deliver a functional LCA5 gene to the outer retina. OPGx-LCA5 is currently being evaluated in a Phase 1/2 clinical trial at the University of Pennsylvania. Data from pediatric participants demonstrated large gains in cone-mediated vision, and the therapy remains well tolerated with no ocular serious adverse events or dose-limiting toxicities. The adult cohort showed durable improvements in cone sensitivity and visual function out to 18 months. OPGx-LCA5 has received Rare Pediatric Disease, Orphan Drug, and Regenerative Medicine Advanced Therapy (RMAT) designations from the FDA and has been accepted into the FDA’s Rare Disease Evidence Principles (RDEP) program. New Risk • Jun 10
New major risk - Shareholder dilution The company's shareholders have been substantially diluted in the past year. Increase in shares outstanding: 36% This is considered a major risk. Shareholder dilution occurs when there is an increase in the number of shares on issue that is not proportionally distributed between all shareholders. Often due to the company raising equity capital or some options being converted into stock. All else being equal, if there are more shares outstanding then each existing share will be entitled to a lower proportion of the company's total earnings, thus reducing earnings per share (EPS). While dilution might not always result in lower EPS (like if the company is using the capital to fund an EPS accretive acquisition) in a lot cases it does, along with lower dividends per share and less voting power at shareholder meetings. Currently, the following risks have been identified for the company: Major Risks Negative equity (-US$21m). Shareholders have been substantially diluted in the past year (36% increase in shares outstanding). Minor Risks Currently unprofitable and not forecast to become profitable over next 3 years (US$45m net loss in 3 years). Significant insider selling over the past 3 months (US$382k sold). 
