Ocuphire Granted New US Patent for Late-Stage Oral Drug Candidate 

Ocuphire Pharma, Inc. recently announced the USPTO has issued a new patent extending expiry and broadening coverage of the company’s late-stage oral product candidate, APX3330. In addition, a new publication supporting the novel mechanism of action inhibiting NFkB and the inflammation pathway for APX3330 and the APX pipeline candidates has been published in a peer-reviewed journal.

“We are pleased to announce the issuance of a new US patent that further strengthens our patent estate for APX3330,” said Mina Sooch, MBA, Founder and CEO of Ocuphire Pharma. “If approved, oral APX3330 has the potential to address the high unmet need for early intervention for progressive vision-threatening diabetic eye disease, including diabetic retinopathy (DR) and diabetic macular edema (DME). The timing is ideal as we continue to advance our fully enrolled clinical program investigating APX3330 in DR/DME where we have reported interim masked safety results at multiple medical meetings throughout this year demonstrating a consistent and favorable safety profile for a first-in-class oral treatment option. Top-line results from the Phase 2b ZETA-1 trial are expected in the second half of 2022.”

US Patent No. 11,351,130 has claims directed to methods of treatment using APX3330, which makes it eligible for listing in the US FDA Orange Book. It was issued on June 7, 2022, and has a term that expires in year 2038. Claims in this patent include methods of treating inflammation and chronic pain in a subject suffering from diabetes. This patent complements the company’s patent estate relating to APX3330 and the company’s pipeline products including APX2009 and APX2014, together the subject of over 40 patents directed to ophthalmic and other uses in the US, Europe, Japan, and other foreign countries as well as the subject of pending patent applications in the US, Europe, Japan, China, and other foreign countries.

Ocuphire also announced the publication of a preclinical study outlining the pivotal role of NFkB (RelA) in Ref-1 inhibition in a pancreatic ductal adenocarcinoma (PDAC) cell model with a Kras genotype and offers evidence of anti-inflammatory benefits of APX3330 in PDAC.

The publication, titled, RelA is an Essential Target for Enhancing Cellular Responses to the DNA Repair/Ref-1 Redox Signaling Protein and Restoring Perturbated Cellular Redox Homeostasis in Mouse PDAC Cells, had several key findings such as NFkB being a key target driving pancreatic cell sensitivity to Ref-1 redox inhibition, and confirmed small molecules APX33330, APX2009, and APX2014 suppresses inflammatory process mediated by NFkB pathway.

The full online publication in Frontiers in Oncology can be accessed at https://doi.org/10.3389/fonc.2022.826617.

Mark Kelley, PhD, member of Ocuphire’s Medical Advisory Board Professor of Pediatric Oncology Research, and Professor of Ophthalmology at Indiana University, commented “The results from this preclinical study continue to support the underlying mechanism of action of Ref-1 inhibitors in the reduction of inflammation and highlight the potential broad utility of APX3330 in diseases implicated by Ref-1 signaling, including in diabetic and retinal eye diseases. It is very rewarding to be involved in the advancement of APX3330 especially as we approach a data read-out this year from the Phase 2b trial in diabetic patients with retinopathy. This clinical study represents a significant milestone that is a result of years of research and translational medicine by many of us.”

APX3330 is a small molecule oral drug candidate and a first-in-class inhibitor of the transcription factor regulator Ref-1 (reduction-oxidation effector factor-1). With its novel dual mechanism of action, APX3330 blocks the downstream pathways regulated by Ref-1, including those involving angiogenesis (VEGF) and inflammation (NFkB), to decrease abnormal activation of both angiogenesis and inflammatory pathways that are implicated across several ocular diseases, including DR, DME, and age-related macular degeneration (AMD).

APX3330 has shown a favorable safety and tolerability profile over 11 clinical trials conducted in healthy, hepatitis, and cancer subjects prior to ZETA-1 trial. APX3330 is currently in a Phase 2 clinical trial in diabetic retinopathy patients. The most recent interim analysis of masked safety data from ZETA-1 trial was presented by Dr. David Boyer at the Annual Macula Society Meeting in June 2022 and oral APX3330 continued to demonstrate a favorable safety profile consistent with the prior trials. Across all the trials, the safety findings represent over 7,700 subject-days of exposure at the target dose of 600 mg/day of APX3330.

Ocuphire is a publicly-traded (NASDAQ: OCUP), clinical-stage ophthalmic biopharmaceutical company focused on developing and commercializing small-molecule therapies for the treatment of refractive and retinal eye disorders. The Company’s lead product candidate, Nyxol eye drops (0.75% phentolamine ophthalmic solution), is a once-daily, preservative-free eye drop formulation of phentolamine mesylate, a non-selective alpha-1 and alpha-2 adrenergic antagonist designed to reduce pupil size, and is being developed for several indications, including reversal of pharmacologically-induced mydriasis (RM), presbyopia and dim light or night vision disturbances (NVD), and has been studied in 12 completed clinical trials. Ocuphire has reported positive data from MIRA-2 and MIRA-3 registration trials and MIRA-4 pediatric safety trial for the treatment of RM. Ocuphire also reported positive topline data from the VEGA-1 Phase 2 trial of Nyxol for treatment of presbyopia, both Nyxol as a single agent and Nyxol with low dose pilocarpine (LDP) 0.4% as adjunctive therapy. The company recently reported positive topline results from LYNX-1 Phase 3 trial of Nyxol for NVD. Ocuphire’s second product candidate, APX3330, is an oral tablet designed to inhibit angiogenesis and inflammation pathways relevant to retinal and choroidal vascular diseases, such as diabetic retinopathy (DR) and diabetic macular edema (DME) and has been studied in 11 Phase 1 and 2 trials. The company announced in March the completion of enrollment in the ZETA-1 Phase 2b clinical trial of APX3330 to treat DR/DME. Please visit www.clinicaltrials.gov to learn more about Ocuphire’s ongoing APX3330 Phase 2b trial in DR/DME ZETA-1 (NCT04692688) and completed Nyxol trials: Phase 3 registration trial in NVD LYNX-1 (NCT04638660), Phase 3 registration trials in RM MIRA-2 (NCT04620213) and MIRA-3 (NCT05134974), MIRA-4 Phase 3 pediatric safety study (NCT05223478), and Phase 2 trial in presbyopia VEGA-1 (NCT04675151). As part of its strategy, Ocuphire will continue to explore opportunities to acquire additional ophthalmic assets and seek strategic partners for late-stage development, regulatory preparation, and commercialization of drugs in key global markets. For more information, visit www.ocuphire.com.