Opus Genetics, Inc.

Opus Genetics presented positive clinical data from its OPGx-BEST1 and OPGx-LCA5 gene therapy programs at the Retina in Rio 2026 conference. The OPGx-BEST1 Phase 1/2 trial showed improved retinal sensitivity in 75% of evaluable participants in the initial low-dose cohort, with new analyses linking retinal structure to visual function. The OPGx-LCA5 Phase 1/2/3 trial demonstrated sustained improvements in visual acuity and light sensitivity through 24 months in adults and encouraging results in pediatric participants through six months. The company expects to initiate dosing for the Phase 3 OPGx-LCA5 clinical trial in the fourth quarter of 2026.

Opus Genetics reported a significant increase in net loss to $73.6 million for the six months ended June 30, 2026, compared to $15.6 million in the prior year. Revenue from license and collaborations also declined to $2.9 million. The company's stockholders' equity turned negative, reaching $(13.0) million, while total liabilities substantially increased to $106.0 million. Despite these financial challenges, Opus Genetics secured new financing through a Note Purchase Agreement, with an initial $35.0 million funded, contributing to an increase in cash and cash equivalents to $88.8 million.

Opus Genetics reported a net loss of $8.1 million for Q2 2026, with R&D expenses increasing to $11.2 million. The company announced significant pipeline advancements, including expected topline data for OPGx-BEST1 in September 2026 and completed enrollment for the OPGx-LCA5 registrational trial with dosing planned for Q4 2026. Opus Genetics also confirmed a strong cash position of $88.8 million, providing a runway into 2029 to support multiple clinical inflection points.

Opus Genetics, Inc. announced the completion of enrollment in its registrational Phase 3 clinical trial for OPGx-LCA5, an investigational gene therapy for LCA5-associated inherited retinal disease. This milestone follows FDA alignment on the trial design through the Rare Disease Evidence Principles program. The company expects to initiate dosing in Q4 2026 and report topline six-month efficacy data by the end of 2027, with potential for BLA submission based on this data.

Opus Genetics provided an update on its OPGx-BEST1 Phase 1/2 clinical trial (BIRD-1) targeting inherited retinal diseases. The company expects to announce three-month topline data from Cohort 1 in the second week of September 2026 and plans to present this data at the EURETINA Congress in October 2026. Cohort 1 enrollment was completed in May 2026 with five participants, and the trial aims to evaluate the safety and efficacy of OPGx-BEST1.

Opus Genetics announced successful alignment with the FDA on the pivotal Phase 3 study design for OPGx-LCA5, a gene therapy for LCA5-associated inherited retinal disease. The FDA indicated that a Biologics License Application (BLA) may be submitted based on compelling 6-month efficacy data, with 12-month durability data provided during the review process. The company expects to initiate Phase 3 dosing in the fourth quarter of 2026, with 7 of 8 planned participants already enrolled and completing the run-in period. The primary efficacy endpoint is a mean improvement of at least 7 decibels in retinal sensitivity, supported by Phase 1/2 data showing an average improvement of approximately 10.5 dB.

Opus Genetics, Inc. announced its inclusion in the broad-market Russell 3000®, Russell 2000®, and Russell Microcap® Indexes, effective June 29, 2026. This move is part of the June 2026 Russell indexes reconstitution. The company's CEO, George Magrath, highlighted that this inclusion validates their progress in gene therapy development and will enhance their visibility within the investment community. Russell indexes are widely utilized by investment managers, with approximately $12.2 trillion in assets benchmarked against them as of June 2025.

Opus Genetics highlighted five gene therapy programs for inherited retinal diseases (IRDs) at its R&D Science Forum, with four clinical data readouts anticipated in 2027. The company expects three new programs (OPGx-RDH12, OPGx-MERTK, OPGx-RHO) to enter clinical testing within the next 12-18 months. Opus Genetics also reported a cash runway extending into 2029, which is expected to fund multiple clinical milestones and potential product approvals.

Opus Genetics, Inc. presented at its Virtual R&D Science Forum, highlighting its robust pipeline and clinical development strategy for inherited retinal diseases (IRDs). The company announced it is fully-funded to advance five IRD clinical programs, with a cash runway extending into 2029. Key milestones include planned clinical study initiations for RDH12, MERTK, and RHO programs, as well as Phase 3 dosing for LCA5 and initial results for BEST1. The presentation also shared positive efficacy and durability data for OPGx-LCA5, including significant participant-reported improvements.

Opus Genetics, a clinical-stage biopharmaceutical company, announced it will host a virtual Research & Development (R&D) Science Forum on June 16, 2026. The forum will showcase the company's multi-asset gene therapy pipeline for inherited retinal diseases, featuring management and key opinion leaders. It will provide detailed information on three gene therapy programs targeting RDH12, MERTK, and RHO mutations that are entering clinical testing, alongside updates on existing LCA5 and BEST1 clinical programs.