SCD-1 inhibitor (Pfizer Inc)

The eye drop maker Ocuphire Pharma and startup Opus Genetics are merging in an all-stock deal, the companies told Endpoints News . Ocuphire $OCUP will acquire privately-held Opus and adopt its name, and will trade under the new stock ticker $IRD effective Thursday, reflecting its focus on inherited retinal diseases. The combined company will have a late-stage eye drop and a pipeline of seven AAV-based gene therapies. Ocuphire CEO George Magrath will remain in his post and Opus CEO Ben Yerxa will become president. Magrath said that at a recent conference, a speaker from the FDA “mentioned that the biggest unmet need in ophthalmology right now [is] treatments for inherited retinal disorders, so that was music to my ears.” Ocuphire shareholders will own about 58% of the combined company and Opus’ shareholders will get about 42%, according to the announcement. The combined company will have about 20 employees. Michigan-based Ocuphire went public through a reverse merger with Rexahn Pharmaceuticals in the fall of 2020. North Carolina-based Opus came out of stealth three years ago, backed with $19 million in seed funding. The company’s science is based on work from Jean Bennett, who co-founded Spark Therapeutics and co-developed its treatment Luxturna. Bennett will become a board member of the combined company. After its founding, Opus bought two preclinical gene therapies from Iveric Bio for $500,000 upfront in December 2022. Opus has raised a total of about $26 million, Yerxa told Endpoints Tuesday. Opus’ lead gene therapy is OPGx-LCA5, which is in Phase 1/2 for an early-onset retinal degeneration condition known as Leber congenital amaurosis 5. On Tuesday, the companies said three of three adults in the trial had visual improvement at six months. “We took a gamble to use our little bit of available capital to get that early clinical data,” Yerxa said. “That was really key to get to that proof-of-concept to show that this idea of curated gene therapies, hand-selected, that they would read out in a meaningful way.” The next step entails testing the therapy in three pediatric patients, and data could come in the third quarter of next year. Magrath said that if those data show similar improvement, “the door is open to discuss with the FDA what the accelerated approval would look like.” Ocuphire has experience with regulatory approval from its eye drop, which was cleared by the FDA last year for pharmacologically-induced mydriasis. The drug has been out-licensed to Viatris, which is also fully funding two Phase 3 tests of the phentolamine ophthalmic solution in dim light vision disturbances and presbyopia, a common condition that causes blurry vision. Those studies are slated to have data readouts in the first quarter and first half of 2025, respectively. Ocuphire is running the clinical development and is responsible for regulatory filings before handing off to Viatris to lead commercialization, Magrath said. The presbyopia market has had its challenges. AbbVie pulled back on promoting a product for the disease and cut a next-generation drug, while the company Orasis hasn’t yet started selling its approved drug. On Monday, Lenz Therapeutics said the FDA set an approval deadline of Aug. 8 for its eye drop LNZ100. After the merger, Ocuphire will stop working on the Ref-1 inhibitor APX3330, an oral small molecule that had failed a Phase 2 trial in diabetic retinopathy last year. It plans to look for a partner for the drug, as well as for its follow-on Ref-1 inhibitors APX2009 and APX2014 for geographic atrophy and other retina conditions, Magrath said.