Ocuphire Pharma Acquires Opus Genetics

Ocuphire Pharma, Inc., a clinical-stage ophthalmic biopharmaceutical company, announced its all-stock acquisition of Opus Genetics, Inc., a gene therapy company focusing on inherited retinal diseases (IRDs). The merger creates a significant entity in the field of gene therapy for IRDs, with the combined company operating under the name Opus Genetics, Inc. and trading on Nasdaq under the ticker "IRD."

George Magrath, M.D., will continue as the CEO of the combined company, highlighting the promising early data from the OPGx-LCA5 Phase 1/2 trial that showed visual improvement in patients with advanced disease. He expressed optimism about advancing treatments swiftly, with four major clinical milestones anticipated in 2025. Dr. Ben Yerxa, the former President and CEO of Opus Genetics, who will now serve as President of the newly formed company, noted the synergistic benefits of combining Ocuphire's late-stage ophthalmic drug development expertise with Opus Genetics' innovative gene therapies.

The merger expands the pipeline to include multiple gene therapy assets from Opus Genetics' adeno-associated virus (AAV)-based portfolio. These are being developed for IRDs, along with Phentolamine Ophthalmic Solution 0.75%, currently in Phase 3 trials for presbyopia and dim light vision disturbances post-keratorefractive surgery. The company plans to seek a strategic partner for the development of APX3330, an oral small-molecule inhibitor of Ref-1 for non-proliferative diabetic retinopathy, to redirect resources into the gene therapy programs.

The most advanced candidate, OPGx-LCA5, targets LCA5, an early-onset retinal degeneration. The ongoing open-label, dose-escalation Phase 1/2 trial has shown promising early results, with visual improvements in all adult participants. Dr. Jean Bennett, scientific co-founder of Opus Genetics, noted the significant efficacy in these late-stage disease patients, suggesting a transformative potential for the treatment.

Enrollment of pediatric patients in the OPGx-LCA5 Phase 1/2 trial is expected to begin in the first quarter of 2025, with initial data anticipated later that year. This program benefits from receiving Rare Pediatric Disease Designation and Orphan Drug Designation from the FDA, making it eligible for a priority review voucher upon biologics license application (BLA) approval.

Dr. Bennett, Dr. Yerxa, and Dr. Adrienne Graves, who served on the Board of Directors of Opus Genetics, will join the board of the combined company. Dr. Bennett has a notable background in ocular gene therapy, having co-founded Opus Genetics and Spark Therapeutics. Dr. Yerxa co-founded Opus Genetics and has extensive experience in biotechnology and ophthalmic drug development. Dr. Graves brings valuable expertise from her tenure as CEO of Santen Inc. and Iveric Bio.

The combined company projects its cash runway to extend into 2026, covering the period when clinical data readouts are expected for pediatric patients in the OPGx-LCA5 Phase 1/2 trial, the initial patients of the OPGx-BEST1 Phase 1/2 trial, and the LYNX-2 and VEGA-3 Phase 3 trials. The LYNX-2 trial focuses on patients with decreased visual acuity under low light conditions post-keratorefractive surgery, and the VEGA-3 trial addresses presbyopia, with both trials actively enrolling and expecting top-line data in 2025.

In the acquisition, Ocuphire issued 5.2 million shares of its common stock and 14.1 thousand shares of convertible preferred stock to Opus Genetics' stockholders. Upon conversion of the preferred stock, subject to approval at the 2025 annual stockholders meeting, Ocuphire’s pre-acquisition stockholders will own 58% of the combined company on a fully diluted basis, while Opus Genetics’ stockholders will own 42%.

The merger consolidates Ocuphire’s resources and experience in late-stage ophthalmic drug development with Opus Genetics’ pioneering gene therapy programs, aiming to accelerate the advancement of treatments for inherited retinal diseases and improve patient outcomes.