NEW YORK – Ocuphire Pharma this week announced an all-stock acquisition of Opus Genetics, a company developing gene therapies for inherited retinal diseases.
As part of the acquisition, Farmington Hills, Michigan-based Ocuphire issued 5.2 million shares of its common stock and 14,100 shares of its convertible preferred stock to existing stockholders of Opus Genetics. The shares of convertible preferred stock can be converted into shares of common stock after stockholder approval at the company's annual meeting in April 2025. Pre-acquisition stockholders of Ocuphire and Opus Genetics will own approximately 58 percent and 42 percent, respectively, of the combined company's fully diluted capitalization.
The combined company will be called Opus Genetics and trade on the Nasdaq under the ticker symbol "IRD" starting Thursday.
Both Ocuphire and Opus Genetics focus on developing treatments for eye disorders. The merger bolsters the combined company's pipeline with multiple clinical stage adeno-associated virus (AAV)-based gene therapies for inherited retinal diseases, including OPGx-LCA5, a gene therapy candidate undergoing a dose-escalation Phase I/II trial in patients with Leber congenital amaurosis 5. The inherited eye disease is caused by mutations in the LCA5 gene, and the gene therapy is designed to use an AAV8 vector to deliver a functional LCA5 gene to the outer retina.
According to Opus Genetics, the Phase I/II trial has shown early clinical proof of concept for OPGx-LCA5, by showing that it improves vision in all three adult patients with late-stage disease in the trial. Opus Genetics expects to enroll the first pediatric patients in this study in the first quarter of 2025. OPGx-LCA5 also has rare pediatric disease designation and orphan drug status from the US Food and Drug Administration, which makes the therapy eligible for a priority review voucher if it gains regulatory approval and reaches the market.
Citing "capitalization requirements," Ocuphire and Opus Genetics are expecting to seek a strategic partner to advance APX3330, a Ref-1 inhibitor under development for treating nonproliferative diabetic retinopathy and use the resources to advance gene therapy programs. The merger is expected to extend the combined company's cash runway into 2026, during which multiple data readouts are slated, including the first pediatric data readout in the third quarter of 2025 from the Phase I/II OPGx-LCA5 trial.
In connection with the acquisition, Ocuphire CEO George Magrath will continue to serve as CEO of the combined company. Ben Yerxa, former president and CEO of Opus Genetics, will be president of the newly combined company. Former Opus Genetics board members Jean Bennett, Yerxa, and Adrienne Graves will join the combined firm's board of directors.
Leerink Partners is Ocuphire's exclusive financial adviser in this transaction, and Sidley Austin is its legal counsel. Smith Anderson is Opus Genetics' legal counsel.