Ocuphire Pharma to Present APX3330 for Diabetic Retinopathy at July Scientific Meetings

15 July 2024
Ocuphire Pharma, Inc., a clinical-stage biopharmaceutical company specializing in ophthalmic conditions, announced its involvement in two significant upcoming events: the Association for Research in Vision and Ophthalmology Special Interest Group (ARVO SIG) panel and the 42nd Annual Scientific Meeting of the American Society of Retina Specialists (ASRS). These events will spotlight the company's lead oral candidate, APX3330, which is being developed for diabetic retinopathy (DR).

APX3330 is designed as an oral small-molecule inhibitor that targets Ref-1 (reduction oxidation effector factor-1), a protein implicated in retinal diseases. Diabetic retinopathy, the leading cause of blindness among working-age adults, affects roughly 10 million out of 38 million Americans with diabetes. Ocuphire's CEO, George Magrath, M.D., will discuss oral medications for retinal diseases in an ARVO SIG panel on July 11. Topics will include the potential of oral therapies in early disease stages, the need for innovative treatments with unique mechanisms, and the hurdles and opportunities in developing these medications. This virtual panel discussion will subsequently be available on-demand on ARVO's website.

The efficacy of APX3330 in treating non-proliferative diabetic retinopathy (NPDR) will be a focal point of the ASRS 42nd Annual Scientific Meeting. Dr. Kareem Sioufi will present a subset analysis from Ocuphire’s ZETA-1 Phase 2 clinical trial virtually. The analysis, co-authored by several notable experts including Dr. Magrath, assesses APX3330's ability to slow DR progression using a binocular DRSS person-level scale in high-risk NPDR patients. This paper will be accessible to ASRS attendees through a mobile app and at viewing stations starting July 17, and later in the ASRS member on-demand content library.

Dr. Magrath expressed his eagerness to partake in the ARVO SIG panel, emphasizing the significance of non-invasive treatment options that could enhance patient compliance and alleviate the treatment burden associated with injectable therapies. He also highlighted the potential of oral APX3330 in revolutionizing diabetic retinopathy treatment by addressing angiogenesis, oxidative stress, and inflammation simultaneously.

APX3330 is a novel small-molecule inhibitor that uniquely targets three related processes involved in retinal diseases: inflammation, angiogenesis, and oxidative stress. By inhibiting Ref-1, APX3330 aims to promote retinal health. It is initially being developed as an oral tablet intended to slow DR progression, potentially delaying the need for more invasive treatments like anti-VEGF injections. Data from the ZETA-1 Phase 2 trial indicated that APX3330 could significantly slow DR progression.

Diabetic retinopathy impacts about 10 million Americans and is projected to affect over 14 million by 2050. DR is categorized into non-proliferative (NPDR) and proliferative (PDR) stages, with NPDR being the early, often asymptomatic stage, and PDR being the advanced stage associated with significant vision loss. Approximately 80% of DR patients have NPDR, which can progress to PDR if untreated. Despite the risk of visual impairment, over 90% of NPDR patients currently do not receive treatment beyond regular observation due to the burden of frequent injections required by existing therapies.

Ocuphire Pharma is committed to developing and commercializing innovative therapies for retinal and refractive eye disorders. Their lead candidate, APX3330, is focused on diabetic retinopathy, while another late-stage program, Phentolamine Ophthalmic Solution 0.75%, is designed for presbyopia and dim light vision disturbances and is marketed as RYZUMVI™ for the reversal of pharmacologically induced mydriasis.

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