Ocuphire Pharma to Present APX3330 at ARVO 2024

Ocuphire Pharma, Inc., a biopharmaceutical company at the clinical stage, has unveiled significant findings from its ZETA-1 trial for APX3330, a therapeutic candidate aimed at treating diabetic retinopathy (DR). The data was presented at the ARVO Annual Meeting held in Seattle from May 5-9, 2024, by Dr. Daniel Su of Retina-Vitreous Associates Medical Group in Los Angeles.

The ZETA-1 trial was a Phase 2, randomized, double-masked study involving 103 participants, aimed at evaluating the efficacy and safety of APX3330, an oral drug, compared to a placebo. The trial concluded in January 2023. A subset analysis was carried out on 68 participants who had specific baseline DRSS scores in both eyes. This analysis focused on a 17-step binocular diabetic Retinopathy Severity Scale (DRSS) to measure the progression of DR.

The analysis revealed that none of the participants in the APX3330 group experienced a significant worsening of their DRSS scores by 4 steps or more at the 24-week mark, compared to 15.2% in the placebo group—a 100% reduction. Additionally, only 5.7% of the APX3330-treated group had a worsening by 3 steps or more compared to 15.2% in the placebo group, translating to a 62.5% reduction. Moreover, fewer individuals in the APX3330 group developed proliferative diabetic retinopathy (PDR), with only 11% compared to 26% in the placebo group. APX3330 was also found to be safe and well-tolerated, showing similar ocular adverse events in both the treatment and placebo groups.

Dr. George Magrath, the CEO of Ocuphire, expressed satisfaction with these findings, highlighting the drug's potential to slow the progression of DR in patients with moderate to very severe non-proliferative DR, who are at a higher risk for developing proliferative DR. He emphasized that the company is in active discussions with the FDA to finalize the Phase 2/3 protocol under a Special Protocol Assessment, aiming to establish APX3330 as a promising oral treatment option for preventing DR progression.

Ocuphire Pharma is dedicated to developing innovative therapies for retinal and refractive eye conditions. APX3330, their leading candidate, is an oral small-molecule inhibitor targeting the Ref-1 protein, which regulates transcription factors linked to ocular angiogenesis and inflammation. By reducing levels of vascular endothelial growth factor (VEGF) and inflammatory cytokines, APX3330 aims to treat non-proliferative diabetic retinopathy effectively. The drug is administered orally twice a day.

Beyond APX3330, Ocuphire has partnered with Viatris, Inc. to develop and commercialize Phentolamine Ophthalmic Solution 0.75% (PS). This non-selective alpha-1 and alpha-2 adrenergic antagonist was approved by the FDA in September 2023 as RYZUMVI™ for treating pharmacologically-induced mydriasis. PS is also in Phase 3 clinical trials for presbyopia and decreased visual acuity under low light conditions post-keratorefractive surgery.

Furthermore, Ocuphire is advancing second-generation analogs of APX3330, including APX2009 and APX2014, targeting other retinal diseases like age-related macular degeneration and geographic atrophy.

In summary, Ocuphire Pharma’s recent ZETA-1 trial results for APX3330 indicate promising efficacy and safety in slowing the progression of diabetic retinopathy. The company continues to work closely with the FDA and partners like Viatris to bring innovative ocular therapies to market, addressing significant unmet medical needs in retinal and refractive eye disorders.