Eye drop company merges with retinal gene therapy startup from Spark co-founder

Ocuphire Pharma, a company specializing in eye drops, and Opus Genetics, a startup focusing on gene therapies, have announced a merger that will see Ocuphire acquiring Opus in an all-stock transaction. This move will result in the newly formed entity taking on the name Opus Genetics and trading under the stock ticker $IRD from Thursday, with a focus on inherited retinal diseases. George Magrath, the current CEO of Ocuphire, will remain in his role, while Ben Yerxa, CEO of Opus, will become the president of the combined company.

Magrath mentioned that the FDA has identified a significant unmet need for treatments targeting inherited retinal disorders, aligning perfectly with the new company's mission. The shareholders of Ocuphire will possess approximately 58% of the merged entity, with Opus shareholders holding the remaining 42%. The combined workforce will consist of around 20 employees.

Ocuphire, based in Michigan, entered the public market via a reverse merger with Rexahn Pharmaceuticals in late 2020. Opus, headquartered in North Carolina, emerged from stealth mode three years ago with $19 million in seed funding. The company's scientific foundation is built on the work of Jean Bennett, a co-founder of Spark Therapeutics and co-developer of Luxturna, a gene therapy for retinal diseases. Bennett will join the board of the merged company.

In December 2022, Opus acquired two preclinical gene therapies from Iveric Bio for an upfront payment of $500,000. To date, Opus has raised approximately $26 million. The lead gene therapy of Opus, OPGx-LCA5, is currently in Phase 1/2 clinical trials for Leber congenital amaurosis 5, a type of early-onset retinal degeneration. Preliminary results have shown visual improvements in three adult participants at the six-month mark.

Yerxa highlighted the strategic risk they took to secure initial clinical data with limited capital, which proved crucial in validating their gene therapy approach. The next phase involves testing the therapy on three pediatric patients, with results expected in the third quarter of next year. If these results demonstrate similar improvements, discussions with the FDA regarding accelerated approval could commence.

Ocuphire brings experience in regulatory processes, having received FDA approval last year for its eye drop used to treat pharmacologically-induced mydriasis. This drug has been licensed to Viatris, which is also funding two Phase 3 trials of the phentolamine ophthalmic solution for dim light vision disturbances and presbyopia. The results of these studies are anticipated in the first quarter and first half of 2025, respectively. Ocuphire is handling the clinical development and regulatory submissions, after which Viatris will manage commercialization.

The presbyopia market has faced various challenges. For instance, AbbVie has scaled back the promotion of its product for the condition and discontinued a next-generation drug. Meanwhile, Orasis has not yet launched its approved drug. Recently, Lenz Therapeutics announced that the FDA has set an approval deadline for its eye drop LNZ100 on August 8.

Post-merger, Ocuphire will cease development of the Ref-1 inhibitor APX3330, an oral small molecule that did not succeed in a Phase 2 trial for diabetic retinopathy last year. The company intends to seek a partner for this drug, as well as for its follow-on Ref-1 inhibitors, APX2009 and APX2014, aimed at treating geographic atrophy and other retinal conditions.