Ocuphire Pharma Announces Presentation on APX3330 at the ARVO 2024 Annual Meeting - Seite 2

“We were pleased to present at ARVO results from the ZETA-1 Phase 2 trial analyzed using both the target population and the FDA-agreed upon registration endpoint for the planned ZETA-2 Phase 2/3 trial,” said George Magrath, M.D., M.B.A., M.S., Chief Executive Officer of Ocuphire. “Results from this subset of participants highlight that APX3330 meaningfully slows DR worsening in patients with moderate to very severe non-proliferative DR (NPDR) in both eyes, representing those who are at higher risk for developing proliferative DR. We are working closely with the FDA regarding our Special Protocol Assessment and aim to finalize the Phase 2/3 protocol soon. We believe that APX3330 has significant potential as a promising oral binocular treatment option for delaying or preventing DR progression in patients who currently lack effective options prior to developing vision-threatening complications.”

About Ocuphire Pharma

Ocuphire is a clinical-stage ophthalmic biopharmaceutical company focused on developing novel therapies for the treatment of retinal and refractive eye disorders.

Ocuphire’s lead retinal product candidate, APX3330, is an oral small-molecule inhibitor of Ref-1 (reduction oxidation effector factor-1 protein) for the treatment of non-proliferative diabetic retinopathy (NPDR). Ref-1 is a regulator of the transcription factors HIF-1α and NF-κB. Inhibiting REF-1 reduces levels of vascular endothelial growth factor (VEGF) and inflammatory cytokines which are known to play key roles in ocular angiogenesis and inflammation. APX3330 is an oral tablet to be administered twice per day for the treatment of diabetic retinopathy (DR). A Phase 2 study in subjects with DR and an End-of-Phase 2 meeting have been completed, and a special protocol assessment (SPA) was submitted to the U.S. Food and Drug Administration (FDA) in February 2024.

In addition, Ocuphire has a partnership with Viatris, Inc. (“Viatris”) to develop and commercialize Phentolamine Ophthalmic Solution 0.75% (“PS”), a non-selective alpha-1 and alpha-2 adrenergic antagonist designed to reduce pupil size. PS was approved by the FDA for the treatment for pharmacologically-induced mydriasis under the brand name RYZUMVI in September 2023. PS is also in Phase 3 clinical development for the treatment of presbyopia and for the treatment of decreased visual acuity under low light (mesopic) conditions after keratorefractive surgery.