Ocuphire completes acquisition of Opus Genetics

(Image credit: AdobeStock/Yingyaipumi)

Ocuphire Pharma, Inc. announced the all-stock acquisition of Opus Genetics, Inc., a clinical-stage gene therapy company for inherited retinal diseases (IRDs).

According to a news release, the deal would position the combined companies as a key player in the development of gene therapies for the treatment of IRDs. In connection with the merger, the combined company will be renamed Opus Genetics Inc., effective October 23, 2024, and will trade on Nasdaq under the ticker symbol IRD effective October 24, 2024.

George Magrath, MD, who will continue to serve as CEO of the combined company, said Opus Genetics has created a compelling pipeline of transformative therapies for patients with inherited retinal diseases, with promising early data.

“This is an opportunity to advance these treatments quickly, with four major clinical milestones on the horizon in 2025 for the combined company,” Magrath said in the news release. “We are encouraged by the new LCA5 six-month proof-of-concept data showing visual improvement in three out of three patients with advanced disease and are excited to bring together a leadership team with deep expertise in the development of potentially groundbreaking 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 Genetics and president of the newly combined company, said the merger cold impact the combined companies’ pipeline.

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

According to a recent news release, the merged company has expanded its pipeline, incorporating several promising assets from its adeno-associated virus (AAV)-based gene therapy portfolio. These assets are being developed for inherited retinal diseases (IRDs), alongside Phentolamine Ophthalmic Solution 0.75%, which is currently under evaluation for the treatment of presbyopia and dim (mesopic) light vision disturbances (DLD) following keratorefractive surgery. Given the capital demands and developmental timelines for APX3330, an oral small-molecule inhibitor of Ref-1 targeting non-proliferative diabetic retinopathy (NPDR), the company plans to seek a strategic partner to advance the clinical development of this late-stage diabetic retinopathy program. Meanwhile, resources will be reallocated toward the recently acquired gene therapy initiatives.¹

The most advanced gene therapy candidate, OPGx-LCA5, is aimed at treating LCA5, an early-onset retinal degeneration. An open-label, dose-escalation Phase 1/2 clinical trial is currently underway, providing early clinical proof-of-concept. New six-month data show visual improvements in all three adult patients with late-stage disease participating in the trial.¹

Jean Bennett, MD, PhD, scientific co-founder of Opus Genetics, lauded the potential impact OPGx-LCA5 could have on patients.

“This level of efficacy in patients with late-stage disease is exciting and supportive of the potential for a one-time treatment with OPGx-LCA5, which could have a transformative impact on individuals who have experienced devastating vision loss and for whom no alternative treatment options exist,” Bennett said in the news release.

Enrollment of the first pediatric patients in the Phase 1/2 trial is expected in the first quarter of 2025, with the first data anticipated in the third quarter of 2025. As the program has received Rare Pediatric Disease Designation and Orphan Drug Designation from the U.S. Food and Drug Administration (FDA), OPGx-LCA5 will be eligible to receive a priority review voucher upon biologics license application (BLA) approval.

Bennett, Yerxa, and Adrienne Graves, Ph.D., all of whom previously served on the Board of Directors of Opus Genetics, will join the Board of the newly combined company.

Bennett, the scientific co-founder of Opus Genetics and former scientific founder of Spark Therapeutics, was among the pioneers in using viral vectors to deliver transgenes to specific retinal cells, leading the first team to demonstrate ocular gene therapy proof-of-principle. Yerxa, co-founder of Opus Genetics and former CEO of the Foundation Fighting Blindness, played a key role in establishing the Foundation's Retinal Degeneration (RD) Fund. With over 30 years of experience in biotechnology and ophthalmic drug development, Yerxa has successfully translated research breakthroughs into clinical advancements. Graves, former CEO of Santen Inc. and chair of Iveric Bio, currently chairs the RD Fund’s board.

The combined company has extended its cash runway into 2026, a period during which it expects to achieve several clinical milestones. These include data readouts from pediatric patients in the OPGx-LCA5 Phase 1/2 trial, the first patients in the OPGx-BEST1 Phase 1/2 trial, and from the ongoing LYNX-2 and VEGA-3 Phase 3 trials. Both the LYNX-2 trial, targeting patients with decreased visual acuity in low-light conditions after keratorefractive surgery, and the VEGA-3 trial for presbyopia, are actively enrolling, with top-line results anticipated in early and mid-2025, respectively.¹

According to the news release, as part of the acquisition, Ocuphire issued 5.2 million shares of common stock and 14,100 shares of convertible preferred stock to Opus Genetics’ existing stockholders. The convertible preferred shares will convert to common stock pending approval at Ocuphire’s 2025 annual stockholders' meeting. Post-issuance, Ocuphire’s pre-acquisition stockholders will own approximately 58% of the combined company’s fully diluted capitalization, with Opus Genetics’ pre-acquisition stockholders holding around 42%.

Reference

1. Ocuphire Pharma Announces Acquisition of Opus Genetics. Ocuphire Pharma, Inc. Published October 22, 2024. Accessed October 22, 2024. https://www.ocuphire.com/news-media/press-releases/detail/444/ocuphire-pharma-announces-acquisition-of-opus-genetics