Eye drug maker Ocuphire Pharma is acquiring gene therapy developer Opus Genetics in an all-stock transaction that will see the commercial-stage company adopt the biotech’s identity.
The resulting entity, which will operate as Opus Genetics, will pitch itself as a “biotech company committed to being a leader in the development of gene therapies for the treatment of inherited retinal diseases,” Ocuphire said in an Oct. 22 release.
The acquisition will see Nasdaq-listed Ocuphire, which markets the Viatris-partnered pupil dilation drug Ryzumvi, take over Opus’ pipeline of adeno-associated virus (AAV)-based retinal gene therapies. They will be headed up by OPGx-LCA5at, which is currently undergoing a phase 1/2 trial for a type of early-onset retinal degeneration.
The study’s three adult participants to date have all shown visual improvement after six months, Ocuphire pointed out in the release. The first pediatric patients are due to be enrolled in the first quarter of 2025, with an initial readout penciled in for the third quarter of that year.
Opus’ scientific co-founder Jean Bennett, M.D., Ph.D., said the level of efficacy shown by OPGx-LCA5 among the first three patients, all of whom have late-stage disease, is “exciting and supportive of the potential for a one-time treatment.”
This could have “a transformative impact on individuals who have experienced devastating vision loss and for whom no alternative treatment options exist,” added Bennett, who was a former scientific founder of Spark Therapeutics and will join the board of the new Opus.
As part of the deal, Ocuphire is offloading a clinical-stage candidate in the form of APX3330, an oral small-molecule inhibitor of Ref-1 for the treatment of non-proliferative diabetic retinopathy. The company had still been hoping for a path to FDA approval despite a phase 2 fail last year but said in yesterday’s release that, “due to the capital requirements and developmental timelines,” it will now search for a partner for the drug so it can “redirect its existing resources towards the acquired gene therapy programs.”
Ocuphire's Ryzumvi, also known as phentolamine ophthalmic solution, was approved by the FDA a year ago to treat pharmacologically induced mydriasis. The biopharma has two phase 3 trials with the drug ongoing in dim light disturbances and loss of focus, with readouts expected in the first quarter and first half of 2025, respectively.
The merged company will list on the Nasdaq under the ticker “IRD” from Oct. 24 and have a cash runway stretching into 2026. Ocuphire’s current shareholders will own 58% of the new entity, while Opus’ shareholders will own the remaining 42%.
“Opus Genetics has created a compelling pipeline of transformative therapies for patients with inherited retinal diseases, with promising early data,” said Ocuphire’s CEO George Magrath, M.D., who will continue to helm the merged company. “This is an opportunity to advance these treatments quickly, with four major clinical milestones on the horizon in 2025 for the combined company.”
Opus CEO Ben Yerxa, Ph.D., who will be president of the merged company, said Ocuphire’s “late-stage ophthalmic drug development and regulatory approval experience and resources” would ensure the resulting company will be “well-positioned to accelerate our pipeline of potentially transformative gene therapies for inherited retinal diseases.”