Ocuphire Pharma Stock Price, News & Analysis

Ocuphire Pharma, Inc. (Nasdaq: OCUP) historically operated as a clinical-stage ophthalmic biopharmaceutical company focused on therapies for retinal and refractive eye disorders. According to company disclosures, Ocuphire developed small-molecule and gene therapy-based approaches targeting conditions such as non-proliferative diabetic retinopathy, presbyopia, dim light vision disturbances after keratorefractive surgery, and pharmacologically induced mydriasis.

Following an all-stock transaction, Ocuphire Pharma, Inc. combined with Opus Genetics, Inc., a clinical-stage gene therapy company for inherited retinal diseases (IRDs). The combined company operates under the name Opus Genetics, Inc. and trades on Nasdaq under the ticker symbol "IRD." As a result, the OCUP symbol represents the historical listing of Ocuphire Pharma prior to the renaming and ticker change.

Historical focus and pipeline under OCUP

Before the combination, Ocuphire described itself as a clinical-stage ophthalmic biopharmaceutical company developing therapies for patients with retinal and refractive eye disorders. Its lead retinal product candidate, APX3330, is an oral small-molecule inhibitor of Ref-1 (reduction oxidation effector factor-1 protein) in development for non-proliferative diabetic retinopathy (NPDR). Company materials explain that Ref-1 regulates transcription factors such as HIF-1α and NF-κB, and that inhibiting Ref-1 reduces levels of vascular endothelial growth factor (VEGF) and inflammatory cytokines associated with ocular angiogenesis and inflammation.

Ocuphire also advanced Phentolamine Ophthalmic Solution 0.75%, a non-selective alpha-1 and alpha-2 adrenergic antagonist designed to reduce pupil size. This product is approved by the U.S. Food and Drug Administration (FDA) for the treatment of pharmacologically induced mydriasis under the brand name RYZUMVI™, and is being developed further in Phase 3 trials for presbyopia and for decreased visual acuity under low (mesopic) light conditions following keratorefractive surgery. Company communications note that the phentolamine program is partnered under a license agreement, with the partner funding agreed development costs and marketing RYZUMVI for mydriasis.

Beyond these programs, Ocuphire reported work on second-generation analogs APX2009 and APX2014, which are being evaluated for other retinal diseases such as age-related macular degeneration and geographic atrophy. The company has also described a late-stage small-molecule program, APX3330, as an oral tablet intended to be dosed twice per day in diabetic retinopathy.

Transition to Opus Genetics and inherited retinal diseases

With the acquisition of Opus Genetics, Ocuphire’s historical small-molecule portfolio was combined with Opus’s adeno-associated virus (AAV)-based gene therapy pipeline. Company statements describe the combined entity as a clinical-stage ophthalmic biopharmaceutical company developing therapies to treat patients with inherited retinal diseases and other retinal and refractive disorders. The pipeline includes AAV-based gene therapies addressing mutations in genes associated with bestrophinopathy, Leber congenital amaurosis (LCA), retinitis pigmentosa, and other inherited retinal degenerations.

The most advanced gene therapy program highlighted for the combined company is designed to address mutations in the LCA5 gene, which encodes the lebercilin protein, and is being evaluated in an open-label, dose-escalation Phase 1/2 clinical trial. Company reports describe early clinical proof-of-concept data, including visual improvement in adult patients with late-stage disease. Additional programs include candidates for BEST1-associated disease and other IRDs, as well as continued development of Phentolamine Ophthalmic Solution 0.75% in presbyopia and dim light disturbances.

Regulatory and clinical development approach

Ocuphire’s disclosures emphasize the use of Phase 2 and Phase 3 randomized, double-masked, placebo-controlled clinical trials to evaluate both safety and efficacy. For APX3330 in diabetic retinopathy, the company completed the ZETA-1 Phase 2 trial and held an End-of-Phase 2 meeting with the FDA. It then submitted a Special Protocol Assessment (SPA) to align on a Phase 2/3 registration study design using a binocular Diabetic Retinopathy Severity Scale (DRSS) person-level endpoint. Subset analyses from ZETA-1 have been presented at scientific meetings, highlighting potential slowing of disease progression in higher-risk NPDR patients.

For Phentolamine Ophthalmic Solution 0.75%, Ocuphire conducted pivotal Phase 3 trials (MIRA-2 and MIRA-3) in pharmacologically induced mydriasis, which formed the basis of FDA approval for RYZUMVI. The company is also managing the VEGA-3 Phase 3 trial in presbyopia and the LYNX-2 Phase 3 registration trial in dim light disturbances after keratorefractive surgery, both described as randomized, double-masked, placebo-controlled studies with agreed primary endpoints. Under its license agreement, Ocuphire has stated that its partner reimburses budgeted research and development costs for the phentolamine program and shares certain costs after specified thresholds.

Corporate evolution and funding

Ocuphire has reported that its development activities are supported by license and collaboration revenue, primarily from its commercial partner for Phentolamine Ophthalmic Solution 0.75%. Company filings and press releases also reference non-dilutive funding arrangements and collaborations for gene therapy programs under the Opus Genetics name, including agreements with organizations such as the Foundation Fighting Blindness Retinal Degeneration Fund and other funding parties to support specific inherited retinal disease programs.

After the merger and renaming, SEC filings identify the registrant as Opus Genetics, Inc., reflecting the transition from Ocuphire Pharma, Inc. to the combined company. These filings continue to describe the business as focused on gene therapies for inherited retinal diseases alongside late-stage ophthalmic drug candidates originally developed under the Ocuphire name.

How investors can use the OCUP historical record

For investors and researchers, the OCUP ticker now serves as a historical reference for the period when the company operated as Ocuphire Pharma, Inc. The historical record under OCUP includes the evolution of the APX3330 program in diabetic retinopathy, the clinical and regulatory pathway leading to FDA approval of RYZUMVI, and the company’s initial strategy in retinal and refractive eye disorders. Current trading and ongoing development activities are associated with the Opus Genetics, Inc. name and the Nasdaq ticker symbol "IRD," as described in company news regarding the merger and renaming.