Acquisition of Opus Genetics, becoming a public company headquartered in North Carolina

It is based at Research Triangle Park Opus geneticsa clinical-stage gene therapy company treating inherited retinal diseases has been acquired by a small Durham-based ophthalmic biopharmaceutical company, becoming the latest life sciences company to trade on the North Carolina Stock Exchange.

Opus was acquired by Ocuphire Pharma of Farmington Hills, Mich., in an all-stock transaction announced Oct. 22. The combined company will operate under the name Opus Genetics, and its shares began trading on the Nasdaq Stock Exchange under the ticker symbol IRD, short for inherited retinal diseases, which is the company’s focus.

“Opus Genetics has developed an exciting pipeline of transformative therapies for patients with inherited retinal diseases, based on promising early data,” said George Magrath, MD, CEO of Ocuphire, who will continue in this role with the combined company. “This is an opportunity to rapidly advance these treatments, and there will be four major clinical milestones on the horizon in 2025 for the combined company.

“We are excited to bring together a leadership team with deep expertise in developing potentially groundbreaking gene therapies. We look forward to continuing to make progress, creating value together and improving patient outcomes.”

Dr. Ben Yerxa, former president and CEO of Opus, is president of the newly combined company, which will be headquartered in the Research Triangle.

“With our late-stage ophthalmic drug development pipeline and the Ocuphire team’s regulatory approval experience and resources, we believe we are well-positioned to accelerate the development of potentially transformative gene therapies for the treatment of inherited retinal diseases,” Yerxa said. “We see this transaction as a win for IRD patients around the world and look forward to the successful development of our combined project.”

The expanded portfolio includes multiple products from Opus’ adenovirus (AAV)-based gene therapy portfolio for the treatment of IRD, as well as Ocuphire’s Phentolamine Ophthalmic Solution, an eye drop that is being evaluated for presbyopia and low-light vision impairment after surgery keratorefractive. Ocuphire has partnered with Viatris Inc. to commercialize an eye drop solution under the Ryzumvi brand for the reversal of drug-induced eye dilation.

Ocuphire was also developing an oral pill for the treatment of non-proliferative diabetic retinopathy, but Opus will be looking for a strategic partner for this work so that the company can focus its resources on seven gene therapy programs.

Promising early results

The most advanced gene therapy candidate, OPGx-LCA5, is being developed to treat LCA5, an early-onset retinal degeneration that causes vision loss. The ongoing Phase 1/2 clinical trial has demonstrated early clinical proof of concept, and new six-month data showed improved vision in all three adult patients in the study, each of whom has late-stage disease and is legally blind.

Jean Bennett, M.D., scientific co-founder of Opus, said: “This level of effectiveness in patients with late-stage disease is exciting and validates the potential of a one-time OPGx-LCA5 treatment to have a transformative impact on people who have experienced devastating vision loss and for whom “There are alternative treatment options.”

Enrollment of the first pediatric patients in the Phase 1/2 study is expected in the first quarter of 2025, with first data in the third quarter of 2025.

OPGx-LCA5 has received rare pediatric disease and orphan drug designation from the U.S. Food and Drug Administration, so the therapy will be eligible for priority review voucher upon approval of a biologics license application.

The combined company’s expected cash flow has been extended through 2026, the year in which the company anticipates clinical data readouts for pediatric patients in the Phase 1/2 OPGx-LCA5 trial and readouts from three other clinical trials.

As of September 30, the company reported an approximate cash balance of $37 million.

Purchase conditions

Ocuphire issued 5.2 million shares of common stock and 14,100 shares of convertible preferred stock to existing Opus shareholders.

As a result of the issuance, Ocuphire shareholders now own approximately 58% of the fully diluted capitalization of the combined company, and former Opus shareholders now own approximately 42%.

Bennett, Yerxa and Dr. Adrienne Graves, each of whom served on the Opus board prior to the transaction, are now on the expanded board of the combined company.

Bennett is the scientific co-founder of Opus and former scientific founder of Spark Therapeutics. She was one of the first researchers to use viral vectors to deliver transgenes to specific retinal cells and led the first team to demonstrate proof of concept for eye gene therapy.

Yerxa co-founded Opus in 2021 and is the former CEO of the Foundation Fighting Blindness, where he oversaw the creation of the Foundation Fighting Retinal Blindness (RD) Fund. He has over 30 years of experience in biotechnology and ophthalmic drug development, translating promising research discoveries into clinical milestones and treatments. Yerxa also serves on the board of the North Carolina Biotechnology Center.

Graves is the former CEO of Santen Inc. and former president of Iveric Bio. Currently, he serves as the president of the management board of the Rural Development Fund.

Magrath, CEO, previously led Durham-based Lexitas Pharma Services, a contract research organization specializing in ophthalmology.

Earlier this year, he set up a small research lab for Ocuphire at BioLabs North Carolina, a biotech coworking facility in downtown Durham.