Newly Issued Patent Expands Medical Uses of APX3330 Oral Therapy in Diabetes Patients and Extends Expiry to 2038
Peer-Reviewed Publication Highlights Vital Role of Inflammation Regulator NFkB as a target for Ref-1 inhibition using APX3330 and APX Pipeline Compounds
Top results from ZETA-1, a multicenter, masked, placebo-controlled Phase 2b trial of APX3330 in diabetic retinopathy with over 100 patients, expected in H2 2022
FARMINGTON HILLS, Mich., June 29, 2022 (GLOBE NEWSWIRE) — Ocuphire Pharma, Inc. (OCUP), a clinical-stage ophthalmic biopharmaceutical company focused on developing and commercializing therapies for the treatment of refractive and retinal eye disorders, has announced today that the United States Patent and Trademark Office (USPTO) has issued a new patent extending the expiration and broadening the coverage of the company’s late-stage oral product candidate, APX3330. Additionally, a new publication supporting the novel mechanism of action inhibiting NFkB and the inflammation pathway for APX3330 and 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 heritage 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 vision-threatening progressive diabetic eye diseases, including diabetic retinopathy (DR) and diabetic macular edema ( MDGs). The timing is perfect as we continue to advance our fully enrolled clinical program of APX3330 in DR/DME, where we have reported masked interim safety results at multiple medical meetings throughout this year, demonstrating a consistent and favorable safety profile for a first-class oral treatment. possibility of treatment. Topline results from the ZETA-1 Phase 2b trial are expected in the second half of 2022.”
US Patent No. 11,351,130 contains claims directed to treatment methods using APX3330, making it eligible for listing in the US FDA Orange Book. It was issued on June 7, 2022 and has a term that expires in 2038. The claims of this patent include methods of treating inflammation and chronic pain in a subject with diabetes. This patent complements the company’s patent area for APX3330 and the company’s pipeline products, including APX2009 and APX2014, which together are the subject of more than 40 patents for ophthalmic and other uses in the US, Europe , Japan and other foreign countries, as well as the subject of pending patent applications in the United States, Europe, Japan, China and other foreign countries.
Ocuphire also announced the publication of a preclinical study describing the central role of NFkB (RelA) in inhibiting Ref-1 in a cell model of pancreatic ductal adenocarcinoma (PDAC) with a Kras genotype and offers evidence of benefits. anti-inflammatories of APX3330 in the PDAC.
The publication titled “RelA is a key target to enhance cellular responses to DNA repair redox signaling protein/Ref-1 and restore disrupted cellular redox homeostasis in mouse PDAC cells” had several key findings such as NFkB being a key target driving the sensitivity of pancreatic cells to redox inhibition of Ref-1, and confirmed that the small molecules APX33330, APX2009 and APX2014 suppress the inflammatory process mediated by the NFkB pathway .
The complete publication online in Frontiers in oncology can be viewed here: https://doi.org/10.3389/fonc.2022.826617
Mark Kelley, PhD, Ocuphire Medical Advisory Board Member, Research Professor of Pediatric Oncology and Professor of Ophthalmology at Indiana University, commented, “The results of this preclinical study continue to support the mechanism of underlying action of Ref-1 inhibitors in reducing inflammation and highlight the broad potential utility of APX3330 in diseases involved in Ref-1 signaling, including diabetic and retinal eye diseases. It is very gratifying to be involved in the advancement of APX3330, especially as we approach a reading of data from the Phase 2b trial in diabetic patients with retinopathy this year. This clinical study represents a milestone that is the result of years of research and translational medicine by many of us.
About the APX3330
APX3330 is an oral small molecule drug candidate and a first-order inhibitor of the transcription factor regulator Ref-1 (reduction-oxidation effector factor-1). With its novel dual mechanism of action, APX3330 blocks downstream pathways regulated by Ref-1, including those involving angiogenesis (VEGF) and inflammation (NFkB), to decrease abnormal activation of angiogenesis and inflammatory pathways that are implicated in several eye diseases, including DR, DME and age-related macular degeneration (AMD).
APX3330 showed a favorable safety and tolerability profile in 11 clinical trials conducted in healthy subjects, with hepatitis and cancer prior to the ZETA-1 trial. APX3330 is currently in a Phase 2 clinical trial in patients with diabetic retinopathy. The most recent interim analysis of masked safety data from the ZETA-1 trial was presented by Dr. David Boyer at the Macula Society Annual Meeting in June 2022 and oral APX3330 continued to demonstrate a favorable safety profile consistent with previous trials. Across all trials, safety results represent over 7,700 subject-days of exposure to the target dose of 600 mg/day of APX3330.
About Ocuphire Pharma
Ocuphire is a publicly traded clinical-stage ophthalmic biopharmaceutical company (OCUP) focused on the development and commercialization of small molecule therapies for the treatment of refractive and retinal ocular 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 eye size. pupil, and is in development for several indications, including reversal of pharmacology-induced mydriasis (MR), presbyopia, and low light or night vision impairment (NVD), and has been studied in 12 clinical trials completed. Ocuphire has reported positive data from the MIRA-2 and MIRA-3 registration trials and the MIRA-4 pediatric safety trial for the treatment of RD. Ocuphire also reported positive early data from the Phase 2 VEGA-1 trial of Nyxol for the treatment of presbyopia, both Nyxol as a single agent and Nyxol with low dose pilocarpine (“LDP”) ) 0.4% as an adjunctive therapy. The company recently announced positive topline results from Nyxol’s LYNX-1 Phase 3 trial for NVD. Ocuphire’s second product candidate, APX3330, is an oral tablet designed to inhibit angiogenesis and inflammatory pathways related to retinal and choroidal vascular diseases, such as diabetic retinopathy (DR) and diabetic macular edema (DME) and was studied in 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/OMD. 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: Registration Phase 3 trial in NVD LYNX -1 (NCT04638660), Phase 3 registrational trials in RM MIRA-2 (NCT04620213) and MIRA-3 (NCT05134974), Phase 3 pediatric safety study MIRA-4 (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 readiness and drug commercialization in key global markets. For more information, visit www.ocuphire.com.
Statements in this press release regarding matters that are not historical facts are “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995. Such statements include, but are not limited to, success and the timing of planned regulatory actions. filings, ZETA-1 Phase 2b trial results and Ocuphire’s indications market, commercial strategy, pre-commercialization activities and commercialization of Ocuphire’s product candidates. These forward-looking statements are based on Ocuphire’s current expectations and involve assumptions that may never materialize or may prove to be incorrect. Actual results and timing of events could differ materially from those anticipated in these forward-looking statements due to various risks and uncertainties, including, without limitation: (i) the success and timing of regulatory submissions and preclinical testing and clinics, including registration and data readouts; (ii) regulatory requirements or changes; (iii) changes to clinical trial designs and regulatory pathways; (iv) changes in capital resource requirements; (v) risks related to Ocuphire’s inability to obtain sufficient additional capital to continue to advance its product candidates and preclinical programs; (vi) legislative, regulatory, political and economic developments, (vii) changes in market opportunities, (viii) effects of COVID-19 on clinical programs and business operations, (ix) success and timing commercialization of any Ocuphire product candidates; and (x) the maintenance of Ocuphire’s intellectual property rights. The foregoing discussion of important factors that could cause actual events to differ from expectations should not be construed as complete and should be read in conjunction with the statements that are included herein and elsewhere, including the risk factors detailed in the documents that have been and can be filed. by Ocuphire from time to time with the SEC. All forward-looking statements contained in this press release speak only as of the date on which they were made. Ocuphire undertakes no obligation to update these statements to reflect events that occur or circumstances that exist after the date on which they were made.
Mina Sooch, President and CEO
Ocuphire Pharma, Inc.
Corey Davis, Ph.D.