Ocuphire Pharma Acquires Opus Genetics, Expanding Gene Therapy Pipeline and Extending Cash Runway into 2026

New York, October 24, 2024 - PRISM MarketView - Ocuphire Pharma, Inc. (Nasdaq: OCUP), a clinical-stage ophthalmic biopharmaceutical company, has officially acquired Opus Genetics, Inc., a clinical-stage gene therapy company specializing in inherited retinal diseases (IRDs). The all-stock transaction unites the complementary strengths of both companies to create a leading biotech organization focused on gene therapies for retinal and refractive eye disorders. Following the merger, the combined entity will operate under the Opus Genetics name and will trade on Nasdaq under the ticker symbol “IRD,” starting Oct. 24, 2024.

As part of the all-stock acquisition, Ocuphire issued 5.2 million shares of common stock and 14.1 million shares of convertible preferred stock to Opus Genetics’ stockholders. Post-transaction, Ocuphire shareholders will own approximately 58% of the combined company, while Opus Genetics shareholders will hold about 42%.

The deal represents a strategic shift for Ocuphire as it deepens its focus on developing gene therapies, particularly for IRDs, an area with significant unmet medical needs. “A speaker from the FDA recently mentioned that the biggest unmet need in ophthalmology is treatments for inherited retinal disorders,” said Ocuphire CEO George Magrath.

This acquisition significantly expands Ocuphire’s pipeline. Opus Genetics brings seven gene therapy assets, including its most advanced candidate, OPGx-LCA5, currently in a Phase 1/2 trial for Leber congenital amaurosis 5 (LCA5), an early-onset retinal degeneration condition. Recent six-month data from three adult patients showed promising visual improvements, marking an important clinical proof-of-concept for this gene therapy. Testing in pediatric patients is expected to begin in early 2025, with data anticipated in the third quarter of next year.

Dr. Ben Yerxa, former CEO of Opus Genetics and now President of the combined company, explained the importance of securing early data with limited capital: “We took a gamble to use our little bit of available capital to get that early clinical data. That was really key to show that this idea of curated gene therapies could read out in a meaningful way.”

The combined company is poised to achieve multiple clinical milestones in 2025, which include:

The merger highlights Ocuphire’s commitment to the high-growth gene therapy sector, specifically targeting inherited retinal diseases. IRDs, which often result in severe vision loss or blindness, represent a substantial market with limited competition. The global market for gene therapies targeting IRDs is projected to reach $25.7 billion by 2030, according to ResearchAndMarkets. In addition, the potential for Priority Review Vouchers (PRVs) upon regulatory approval could create additional value for the company.

Ocuphire also brings experience in regulatory approvals, having gained FDA clearance for its Phentolamine Ophthalmic Solution for pharmacologically-induced mydriasis in 2023. The company has out-licensed the drug to Viatris, which is funding two additional Phase 3 trials in presbyopia and dim light vision disturbances, with data expected in 2025.

The combined company will continue to be led by George Magrath as CEO, while Opus Genetics co-founder Ben Yerxa will serve as President. The board will include Jean Bennett, M.D., Ph.D., a pioneer in ocular gene therapy and co-developer of Luxturna, the first FDA-approved gene therapy for a form of Leber congenital amaurosis. Adrienne Graves, Ph.D., former CEO of Santen Inc. and former chair of Iveric Bio, will also join the board of directors. This leadership team brings extensive expertise in gene therapy and drug development, positioning the combined company to potentially make breakthroughs in treating inherited retinal diseases.

Following the merger, Ocuphire will shift its focus away from its Ref-1 inhibitor program, including APX3330, which failed to meet endpoints in a Phase 2 trial for diabetic retinopathy. The company plans to seek a strategic partner for the continued development of APX3330 and its follow-on assets, while prioritizing the more capital-efficient gene therapy programs it has acquired from Opus.

With the expanded pipeline and strengthened leadership team, Ocuphire is well-positioned to advance its portfolio of gene therapy assets, particularly in the high-growth IRD space. The combined company’s cash runway has been extended into 2026, providing the resources needed to support operations and advance key clinical programs through critical milestones expected in 2025.

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