Opus Genetics becomes a limited liability company based in NC

Research Triangle Park-based Opus Geneticsa clinical-stage gene therapy company focused on treating inherited retinal diseases has been acquired by a small ophthalmology biopharmaceutical company with operations in Durham, becoming the newest publicly traded life sciences company in North Carolina.

Opus was acquired by Ocuphire Pharma of Farmington Hills, Michigan, in an all-stock transaction announced on October 22. The combined company will operate as Opus Genetics and its shares will trade on the Nasdaq stock market under the ticker symbol IRD, shorthand for hereditary retinal diseases, the company’s focus.

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

“We are excited to bring together a leadership team with deep expertise in the development of potentially game-changing gene therapies. We look forward to continuing our progress, creating value and improving patient outcomes together.”

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

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

The expanded pipeline includes multiple assets from Opus’ adeno-associated virus (AAV)-based gene therapy portfolio for IRDs, as well as Ocuphire’s phentolamine ophthalmic solution, an eye drop being evaluated in presbyopia and low-light vision disturbances following keratorefractive surgery. Ocuphire collaborated with Viatris Inc. to market the eye drop solution, branded as Ryzumvi, for reversing drug-induced eye dilations.

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

Promising first results

The most advanced gene therapy candidate, OPGx-LCA5, is being developed to treat LCA5, a premature degeneration of the retina that causes vision loss. An ongoing Phase 1/2 clinical trial has demonstrated early clinical proof-of-concept, with new six-month data demonstrating visual improvement in all three adult patients enrolled in the trial, each with late-stage disease and being legally blind.

Jean Bennett, M.D., Ph.D., scientific co-founder of Opus, said: “This level of efficacy in patients with late-stage disease is exciting and supports the potential for a single treatment with OPGx-LCA5, which could have a transformative impacting individuals who have experienced devastating vision loss and for whom no alternative treatment options exist.”

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

OPGx-LCA5 has received rare pediatric disease designation and orphan drug designation from the U.S. Food and Drug Administration, so the therapy is eligible for treatment a voucher for priority assessment after approval of the marketing authorization application for biological medicinal products.

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

The company reported a cash balance of approximately $37 million as of September 30.

Conditions for acquisition

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

Following the issuances, 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 Adrienne Graves, Ph.D., each of whom served on Opus’ board of directors prior to the transaction, now sits on the expanded board of directors 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 cells in the retina and led the first team to demonstrate the proof-of-principle of ocular gene therapy.

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

Graves is the former CEO of Santen Inc. and former chairman of Iveric Bio. She is currently chairman of the board of the RD Fund.

Magrath, the 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 co-working facility in downtown Durham.