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In February 2024, Ocuphire submitted a Special Protocol Assessment (SPA) to the U.S. Food and Drug Administration (FDA) to seek agreement on the clinical trial protocol and statistical analysis plan for a Phase 2/3 registration study for APX3330 in DR. This request followed an End-of-Phase 2 meeting held with the FDA late in 2023, during which the company aligned on the registrational primary endpoint of 3-step or more worsening on a binocular Diabetic Retinopathy Severity Scale (DRSS) person-level scale. Dialogue with the FDA is ongoing, and specifics on the study design and the anticipated timing will be announced if and when an agreement is reached with the FDA.

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Earlier this month, a subset analysis from the ZETA-1 trial was presented at the Association for Research in Vision and Ophthalmology (ARVO) Annual Meeting, which took place in Seattle, Washington. This analysis evaluated the efficacy of APX3330 in slowing DR progression on the target population of the planned Phase 2/3 study, and the FDA agreed upon registration endpoint of a 3-step change on a binocular DRSS person-level scale, which is considered clinically meaningful. The presented results in addition to further analyses demonstrated an enhanced treatment effect for APX3330 in this target population.

•	In conjunction with this update, a subpopulation analysis of ZETA-1 data in NPDR will be released.

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In April 2024, Ocuphire’s partner Viatris launched RYZUMVI™ (phentolamine ophthalmic solution) for the treatment of pharmacologically-induced mydriasis in the U.S. in adult and pediatric patients aged 3 and older. Ocuphire has a global license agreement with Viatris to co-develop and commercialize Phentolamine Ophthalmic Solution 0.75% (PS). Under the terms of this agreement, Ocuphire is now recognizing royalties on commercial sales and may be eligible to receive future commercial milestones. For more information on the commercial launch, refer to the announcement on Viatris’ corporate website here.

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In April 2024, the first subject was enrolled in the LYNX-2 Phase 3 registration study evaluating PS for the treatment of decreased visual acuity under low (mesopic) light conditions following keratorefractive surgery. The LYNX-2 trial is being conducted under conditions of a SPA with the FDA. As previously announced, Ocuphire received written agreement from the FDA that the clinical trial protocol and planned statistical analysis of the LYNX-2 Phase 3 trial would adequately address objectives supporting regulatory submission and a potential future marketing application in this indication.

•	In February 2024, Ocuphire appointed Nirav Jhaveri, C.F.A, M.B.A., as Chief Financial Officer and Ashwath (Ash) Jayagopal, Ph.D., M.B.A., as Chief Scientific and Development Officer.

 

About Ocuphire Pharma

 

Ocuphire is also developing APX2009 and APX2014, second-generation analogs of APX3330. These programs are being evaluated for treating other retinal diseases such as age-related macular degeneration and geographic atrophy. For more information, please visit www.ocuphire.com.

 

Forward Looking Statements

 

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Such statements include, but are not limited to, statements concerning the research takeaways from the ZETA-1 trial, the efficacy of APX3330 in slowing the progression of diabetic retinopathy, the safety and tolerability of APX3330, applications of PS in ophthalmology, the registration program for PS, the LYNX-2 Phase 3 registration study, the benefits, uses, and side effects of PS treatment, ongoing discussions with the FDA regarding various of our drug products, continued drug development under our agreement with Viatris, and the sufficiency of cash on hand to meet future funding needs.

 

These forward-looking statements relate to us, our business prospects and our results of operations and are subject to certain risks and uncertainties posed by many factors and events that could cause our actual business, prospects and results of operations to differ materially from those anticipated by such forward-looking statements. Factors that could cause or contribute to such differences include, but are not limited to, those described under the heading “Risk Factors” included in our Annual Report on Form 10-K and in subsequent filing with the U.S. Securities and Exchange Commission (SEC). Readers are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date of this report. In some cases, you can identify forward-looking statements by the following words: “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “intend,” “may,” “ongoing,” “plan,” “potential,” “predict,” “project,” “should,” “will,” “would” or the negative of these terms or other comparable terminology, although not all forward-looking statements contain these words. We undertake no obligation to revise any forward-looking statements in order to reflect events or circumstances that might subsequently arise.

 

•	The success and timing of regulatory submissions and pre-clinical and clinical trials, including enrollment and data readouts;

•	Regulatory requirements or developments;

•	Changes to or unanticipated events in connection with clinical trial designs and regulatory pathways;

•	Delays or difficulties in the enrollment of patients in clinical trials;

•	Substantial competition and rapid technological change;

•	Our development of sales and marketing infrastructure;

•	Future revenue losses and profitability;

•	Our relatively short operating history;

•	Changes in capital resource requirements;

•	Risks related to the inability of Ocuphire to obtain sufficient additional capital to continue to advance its product candidates and its preclinical programs;

•	Domestic and worldwide legislative, regulatory, political and economic developments;

•	Employee misconduct;

•	Changes in market opportunities and acceptance;

•	Reliance on third-parties;

•	Future, potential product liability and securities litigation;

•	System failures, unplanned events, or cyber incidents;

•	The substantial number of shares subject to potential issuance associated with our equity line of credit arrangement;

•	Risks that our partnership with Viatris, or our other licensing arrangements, may not facilitate the commercialization or market acceptance of Ocuphire’s product candidates;

•	Future fluctuations in the market price of our common stock;

•	The success and timing of commercialization of any of Ocuphire’s product candidates; and

•	Obtaining and maintaining Ocuphire’s intellectual property rights.

Ocuphire Pharma, Inc.

Condensed Balance Sheets

(in thousands, except share amounts and par value)

Ocuphire Pharma, Inc.

Condensed Statements of Comprehensive Loss

(in thousands, except share and per share amounts)

(Unaudited)

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