Ocuphire Acquires Gene Therapy Company Opus Genetics
Ocuphire Pharma announced an all-stock acquisition of Opus Genetics, a clinical-stage gene therapy company specializing in inherited retinal diseases (IRDs). As part of the deal, the combined company will be renamed Opus Genetics, effective October 23, 2024, and will begin trading under the Nasdaq ticker symbol “IRD” on October 24, 2024.
“Opus Genetics has developed a compelling pipeline of transformative therapies for patients with inherited retinal diseases, and this merger provides an opportunity to advance these treatments quickly,” George Magrath, MD, CEO of the combined company, said in a company news release. “With four major clinical milestones anticipated in 2025, we are encouraged by the promising early data, including new 6-month proof-of-concept results showing visual improvements in three out of three patients with advanced LCA5 disease. Together, we aim to deliver groundbreaking gene therapies and improve patient outcomes.”
Ocuphire issued 5.2 million shares of common stock and 14.1 thousand shares of convertible preferred stock to Opus Genetics stockholders. Following the acquisition, Ocuphire stockholders will own approximately 58% of the combined company, with Opus Genetics stockholders holding 42%. Stockholder approval for the conversion of preferred shares into common stock is expected at Ocuphire’s annual meeting in April 2025.
Ben Yerxa, PhD, former President and CEO of Opus Genetics and now President of the newly combined company, added, “Ocuphire’s experience in late-stage ophthalmic drug development, coupled with our gene therapy expertise, positions us well to accelerate our pipeline for IRDs. This merger is a win for patients around the world, and we are committed to progressing our combined programs efficiently.”
The new Opus Genetics brings a pipeline of assets, including an adeno-associated virus (AAV)-based gene therapy portfolio for IRDs. The company will also continue developing phentolamine ophthalmic solution 0.75% for presbyopia and dim light vision disturbances following keratorefractive surgery. However, the company will seek a strategic partner to advance APX3330, its late-stage diabetic retinopathy program, while focusing its resources on gene therapy programs.
The most advanced candidate, OPGx-LCA5, targets LCA5, an early-onset retinal degeneration. Recent 6-month data from an open-label, dose-escalation phase 1/2 trial showed visual improvement in three adult patients with late-stage disease. Enrollment of pediatric patients in the trial is expected in early 2025, with initial data anticipated by Q3 2025. The FDA granted OPGx-LCA5 Rare Pediatric Disease and Orphan Drug Designations, making it eligible for a priority review voucher upon approval.
Dr. Bennett, Dr. Yerxa, and Adrienne Graves, PhD, will join the Board of Directors of the combined company.
With a cash runway extending into 2026, the company anticipates key clinical data readouts from multiple trials, including pediatric results from the OPGx-LCA5 trial and topline data from the phase 3 LYNX-2 and VEGA-3 trials. Both the LYNX-2 trial (for patients with decreased visual acuity in low light) and the VEGA-3 trial (for presbyopia) are actively enrolling, with results expected in early to mid-2025.