Ocuphire Pharma to Present APX3330 Diabetic Retinopathy Data at June Retina Meetings

Ocuphire Pharma, Inc., based in Farmington Hills, Michigan, and traded on Nasdaq under the ticker OCUP, announced recent advancements concerning its main therapeutic candidate, APX3330, designed for treating diabetic retinopathy (DR). These updates will be highlighted in presentations at two important forthcoming conferences: the Clinical Trials at the Summit meeting in Park City, Utah, on June 8, and the Retinal Imaging Biomarkers & Endpoints Summit meeting from June 25-27 in Boston.

APX3330 is an orally administered small-molecule inhibitor targeting the Ref-1 protein, aimed specifically at treating non-proliferative diabetic retinopathy (NPDR). Among the 38 million Americans living with diabetes, approximately 10 million develop DR, a leading cause of blindness among working-age adults. Notably, NPDR is present in about 80% of these DR cases.

During the Clinical Trials at the Summit meeting, Veeral Sheth, M.D., M.B.A., a retina specialist from University Retina and a Clinical Assistant Professor at the University of Illinois, will present a session titled “Clinical Update on Oral APX3330 for Diabetic Retinopathy.” This meeting convenes various experts to discuss ongoing clinical trials and the latest data aimed at advancing vitreoretinal care.

At the second annual Retinal Imaging Biomarkers & Endpoints Summit, Ashwath Jayagopal, Ph.D., Ocuphire’s Chief Scientific and Development Officer, will present “Multiplex Analysis of Clinical Imaging & Biomarker Data to Validate Novel Endpoints for Diabetic Retinopathy.” This summit is the sole industry event dedicated to the integration of AI-based predictive measures in imaging data analysis for earlier DR detection and diagnosis, optimizing patient stratification and recruitment for clinical trials.

George Magrath, M.D., M.B.A., M.S., Chief Executive Officer of Ocuphire, remarked on the potential of APX3330 to serve as a pioneering treatment by addressing multiple established disease pathways for NPDR. He emphasized the company's eagerness to share updates on the APX3330 clinical program and the planned ZETA-2 pivotal trial, which seeks to offer an early intervention treatment to delay or prevent DR progression. The discussions will also cover the importance of multimodal imaging in DR, optimizing Phase 2/3 trials, and patient selection.

Ocuphire is set to commence the ZETA-2 Phase 2/3 trial of APX3330 for NPDR in early 2025. The company has already completed a Phase 2 study and an End-of-Phase 2 meeting, and has submitted a special protocol assessment (SPA) to the U.S. Food and Drug Administration (FDA) in February 2024.

Ocuphire Pharma is dedicated to developing innovative therapies for retinal and refractive eye disorders. The company’s lead candidate, APX3330, functions as an oral small-molecule inhibitor of Ref-1, a regulator of transcription factors HIF-1α and NF-κB. By inhibiting Ref-1, APX3330 reduces levels of VEGF and inflammatory cytokines, which are crucial in ocular angiogenesis and inflammation. The drug is designed to be taken orally twice daily for treating DR.

Beyond APX3330, Ocuphire is collaborating on the development and commercialization of Phentolamine Ophthalmic Solution 0.75% (PS), a non-selective alpha-1 and alpha-2 adrenergic antagonist approved by the FDA for treating pharmacologically-induced mydriasis under the brand name RYZUMVI™. PS is also in Phase 3 clinical trials for presbyopia and decreased visual acuity under low light conditions following keratorefractive surgery.

Additionally, Ocuphire is advancing APX2009 and APX2014, second-generation analogs of APX3330, for the treatment of other retinal diseases such as age-related macular degeneration and geographic atrophy.